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1

Therapy for the Brugada Syndrome  

PubMed Central

The Brugada syndrome is a congenital syndrome of sudden cardiac death first described as a new clinical entity in 1992. Electrocardiographically characterized by a distinct coved-type ST segment elevation in the right precordial leads, the syndrome is associated with a high risk for sudden cardiac death in young and otherwise healthy adults, and less frequently in infants and children. The ECG manifestations of the Brugada syndrome are often dynamic or concealed and maybe revealed or modulated by sodium channel blockers. The syndrome may also be unmasked or precipitated by a febrile state, vagotonic agents, ?-adrenergic agonists, ?-adrenergic blockers, tricyclic ortetracyclic antidepressants, a combination of glucose and insulin, and hypokalemia, as well as by alcohol and cocaine toxicity. An implantable cardioverter-defibrillator (ICD) is the most widely accepted approach to therapy. Pharmacological therapy aimed at rebalancing the currents active during phase 1 of the right ventricular action potential is used to abort electrical storms, as an adjunct to device therapy, and as an alternative to device therapy when use of an ICD is not possible. Isoproterenol and cilostazol boost calcium channel current, and drugs like quinidine inhibit the transient outward current, acting to diminish the action potential notch and thus suppress the substrate and trigger for ventricular tachycardia/fibrillation (VT/VF). PMID:16610350

Fish, J. M.

2006-01-01

2

Current therapy of myelodysplastic syndromes?  

PubMed Central

After being a neglected and poorly-understood disorder for many years, there has been a recent explosion of data regarding the complex pathogenesis of myelodysplastic syndromes (MDS). On the therapeutic front, the approval of azacitidine, decitabine, and lenalidomide in the last decade was a major breakthrough. Nonetheless, the responses to these agents are limited and most patients progress within 2 years. Allogeneic stem cell transplantation remains the only potentially curative therapy, but it is associated with significant toxicity and limited efficacy. Lack or loss of response after standard therapies is associated with dismal outcomes. Many unanswered questions remain regarding the optimal use of current therapies including patient selection, response prediction, therapy sequencing and combinations, and management of resistance. It is hoped that the improved understanding of the underpinnings of the complex mechanisms of pathogenesis will be translated into novel therapeutic approaches and better prognostic/predictive tools that would facilitate accurate risk-adaptive therapy. PMID:23954262

Zeidan, Amer M.; Linhares, Yuliya; Gore, Steven D.

2014-01-01

3

Directed Therapy for Exfoliation Syndrome  

PubMed Central

Exfoliation syndrome (XFS) is an age-related disorder of the extracellular matrix that leads the production of abnormal fibrillar material that leads to elevated intraocular pressure and a relatively severe glaucoma. Exfoliation material is deposited in numerous ocular tissues and extraocular organs. XFS is associated with ocular ischemia, cerebrovascular disease, neurodegenerative disease and cardiovascular disease. Current modalities of treatment include intraocular pressure lowering with topical antihypertensives, laser trabeculoplasty and filtration surgery. The disease paradigm for XFS should be expanded to include directed therapy designed specifically to target the underlying disease process. Potential targets include preventing the formation or promoting the depolymerization of exfoliation material. Novel therapies targeting trabecular meshwork may prove particularly useful in the care of exfoliative glaucoma. The systemic and ocular associations of XFS underscore the need for a comprehensive search for neuroprotective agents in its treatment. PMID:19888433

Angelilli, Allison; Ritch, Robert

2009-01-01

4

Complementary and Alternative Therapies for Down Syndrome  

ERIC Educational Resources Information Center

In their role as committed advocates, parents of children with Down syndrome have always sought alternative therapies, mainly to enhance cognitive function but also to improve their appearance. Nutritional supplements have been the most frequent type of complementary and alternative therapy used. Cell therapy, plastic surgery, hormonal therapy,…

Roizen, Nancy J.

2005-01-01

5

Music therapy for children with Rett syndrome  

Microsoft Academic Search

The Rett syndrome is good reactivity for sound and music. We enforced active music therapy (MT) individual session to patients with Rett syndrome. The patients were 4, 5 and 6-year-old. The active music therapy and individual session of 30 min\\/week were performed. It was recorded in video and description, and using the original evaluation list, six items of fact. On

Akihiro Yasuhara; Yuriko Sugiyama

2001-01-01

6

Growth hormone therapy in Turner syndrome.  

PubMed

Short stature is the single most common physical abnormality in Turner syndrome (TS) with adult stature averaging 20 cm shorter than that of the general population. Randomized, placebo-controlled studies to final adult height have proven that GH therapy is effective in increasing stature in TS. Recently, randomized, controlled studies have demonstrated that adjunctive therapies with low-dose estrogen or low-dose oxandrolone enhance stature further. These therapies may provide benefits beyond height augmentation. PMID:22946284

Davenport, Marsha L

2012-05-01

7

Therapy for the Brugada Syndrome  

Microsoft Academic Search

The Brugada syndrome is a congenital syndrome of sudden cardiac death first described as a new clinical entity in 1992. Electrocardiographically\\u000a characterized by a distinct coved-type ST segment elevation in the right precordial leads, the syndrome is associated with\\u000a a high risk for sudden cardiac death in young and otherwise healthy adults, and less frequently in infants and children. The

C. Antzelevitch; J. M. Fish

8

Response of Paraneoplastic Syndromes to Antineoplastic Therapy  

PubMed Central

The development of a clinically apparent paraneoplastic syndrome in a patient with malignant disease is cause for considerable concern as the symptoms can be as serious and difficult to deal with as those produced by the cancer itself. While the information in the medical literature concerning the response of paraneoplastic processes to specific antineoplastic therapy is limited, case reports and small series would suggest that a number of syndromes are alleviated with successful treatment of the underlying neoplasm. As treatment programs for advanced malignant disease improve, a larger percentage of patients suffering from the effects of paraneoplastic processes will benefit from therapy directed at their cancer. PMID:3521097

Markman, Maurie

1986-01-01

9

Cognitive behaviour therapy in chronic fatigue syndrome  

Microsoft Academic Search

Fifty patients fulfilling operational criteria for the chronic fatigue syndrome (CFS), and who had been ill for a mean of five years, were offered cognitive behaviour therapy in an open trial. Those fulfilling operational criteria for depressive illness were also offered tricyclic antidepressants. The rationale was that a distinction be drawn between factors that precipitate the illness and those that

S Butler; T Chalder; M Ron; S Wessely

1991-01-01

10

Therapies of mucopolysaccharidosis IVA (Morquio A syndrome)  

PubMed Central

Introduction Morquio A syndrome (mucopolysaccharidosis type IVA, MPS IVA) is one of the lysosomal storage diseases and is caused by the deficiency of N-acetylgalactosamine-6-sulfate sulfatase (GALNS). Deficiency of this enzyme leads to accumulation of glycosaminoglycans (GAGs), keratan sulfate (KS) and chondroitin-6-sulfate (C6S). The majority of KS is produced by chondrocytes, and therefore, the undegraded substrates accumulate mainly in cells and extracelluar matrix (ECM) of cartilage. This has a direct impact on cartilage and bone development, leading to systemic skeletal dysplasia. In patients with Morquio A, cartilage cells are vacuolated, and this results in abnormal chondrogenesis and/or endochondral ossification. Areas covered This article describes the advanced therapies of Morquio A, focused on enzyme replacement therapy (ERT) and gene therapy to deliver the drug to avascular bone lesions. ERT and gene therapies for other types of MPS are also discussed, which provide therapeutic efficacy to bone lesions. Expert opinion ERT, gene therapy and hematopietic stem therapy are clinically and/or experimentally conducted. However, there is no effective curative therapy for bone lesion to date. One of the limitations for Morquio A therapy is that targeting avascular cartilage tissues remains an unmet challenge. ERT or gene therapy with bone-targeting system will improve the bone pathology and skeletal manifestations more efficiently. PMID:25419501

Tomatsu, Shunji; Alméciga-Díaz, Carlos J; Barbosa, Hector; Montańo, Adriana M; Barrera, Luis A; Shimada, Tsutomu; Yasuda, Eriko; Mackenzie, William G; Mason, Robert W; Suzuki, Yasuyuki; Orii, Kenji E; Orii, Tadao

2014-01-01

11

[Intensive therapy for patients with Guillian-Barré syndrome.  

PubMed

Guillain-Barré syndrome is the leading cause of acute flaccid paralysis in the industrialized world. Approximately 25% of the patients suffering from Guillain-Barré syndrome develop respiratory failure requiring mechanical ventilation and intensive therapy. We seek answers to when it is optimal to start respiratory supportive therapy and review various complications associated with Guillain-Barré syndrome. PMID:25316360

Buus, Lone; Třnnesen, Else K

2014-10-13

12

[Differentiated antiplatelet therapy for acute coronary syndromes].  

PubMed

Dual antiplatelet therapy is the cornerstone of maintenance medication following invasive treatment of patients with acute coronary syndromes (ST elevation myocardial infarction, non-ST elevation myocardial infarction, unstable angina). Over the last decade, P2Y12 inhibition in addition to low-dose acetylsalicylic acid has been intensively debated. The debate was enriched by the results of the large phase III clinical trials for prasugrel (TRITON) and ticagrelor (PLATO) compared to clopidogrel in patients with acute coronary syndromes. This article summarizes the critical details und subanalyses of both study programmes and highlights on clinical decision making when using the three P2Y12 blockers in acute coronary syndromes. A special focus is on higher risk patients such as those with ST elevation myocardial infarction and those with coexisting diabetes, but also on minimizing relevant bleedings, which are common during more intense platelet inhibition. PMID:24430955

Schäfer, A; Arntz, H R; Boudriot, E; Garlichs, C; Hoffmann, S; Ince, H; Klingenheben, T; Weil, J; Zugck, C; Helms, T M; Silber, S

2014-01-01

13

Weight Loss and Medication in Polycystic Ovary Syndrome Therapy.  

PubMed

Polycystic ovary syndrome, which is a complex syndrome, affects approximately 6% of reproductive-age women. Many abnormalities are associated with polycystic ovary syndrome, but confusion still exists about their causation. Diagnosis of polycystic ovary syndrome is by exclusion. Management of the metabolic aspects of polycystic ovary syndrome focuses on minimizing insulin resistance and hyperinsulinemia with diet therapy or insulin-lowering drugs. PMID:11984434

Tolstoi, Linda G.; Josimovich, John B.

2002-01-01

14

Emerging Therapies for Acute Coronary Syndromes  

PubMed Central

In the majority of cases acute coronary syndromes (ACS) are caused by activation and aggregation of platelets and subsequent thrombus formation leading to a decrease in coronary artery blood flow. Recent focus on the treatment of ACS has centered on reducing the response of platelets to vascular injury as well as inhibiting fibrin deposition. Novel therapies include more effective P2Y12 receptor blockers thereby reducing inter-individual variability, targeting the platelet thrombin receptor (protease activated receptor 1) as well as directly inhibiting factor Xa or thrombin activity. In this review we discuss the clinical data evaluating the effectiveness of these various new ACS treatment options. PMID:22028691

Lilly, Scott M.; Wilensky, Robert L.

2011-01-01

15

Iron Chelation Therapy in Myelodysplastic Syndromes  

PubMed Central

Myelodysplastic syndromes (MDS) are a heterogeneous disorder of the hematopoietic stem cells, frequently characterized by anemia and transfusion dependency. In low-risk patients, transfusion dependency can be long lasting, leading to iron overload. Iron chelation therapy may be a therapeutic option for these patients, especially since the approval of oral iron chelators, which are easier to use and better accepted by the patients. The usefulness of iron chelation in MDS patients is still under debate, mainly because of the lack of solid prospective clinical trials that should take place in the future. This review aims to summarize what is currently known about the incidence and clinical consequences of iron overload in MDS patients and the state-of the-art of iron chelation therapy in this setting. We also give an overview of clinical guidelines for chelation in MDS published to date and some perspectives for the future. PMID:20672005

Messa, Emanuela; Cilloni, Daniela; Saglio, Giuseppe

2010-01-01

16

Review of pharmacological therapies in fibromyalgia syndrome  

PubMed Central

This review addresses the current status of drug therapy for the management of fibromyalgia syndrome (FMS) and is based on interdisciplinary FMS management guidelines, meta-analyses of drug trial data, and observational studies. In the absence of a single gold-standard medication, patients are treated with a variety of drugs from different categories, often with limited evidence. Drug therapy is not mandatory for the management of FMS. Pregabalin, duloxetine, milnacipran, and amitriptyline are the current first-line prescribed agents but have had a mostly modest effect. With only a minority of patients expected to experience substantial benefit, most will discontinue therapy because of either a lack of efficacy or tolerability problems. Many drug treatments have undergone limited study and have had negative results. It is unlikely that these failed pilot trials will undergo future study. However, medications, though imperfect, will continue to be a component of treatment strategy for these patients. Both the potential for medication therapy to relieve symptoms and the potential to cause harm should be carefully considered in their administration. PMID:24433463

2014-01-01

17

Jellyfish envenoming syndromes: unknown toxic mechanisms and unproven therapies.  

PubMed

Interest in envenoming syndromes caused by Australian jellyfish has been intense since the deaths in early 2002 of two tourists in Queensland, attributed to the Irukandji syndrome. We review current knowledge of these envenoming syndromes, mechanisms of venom action and therapy, focusing on the deadly box jellyfish, Chironex fleckeri, and the array of jellyfish thought to cause the Irukandji syndrome. Current understanding of jellyfish venom activity is very limited, and many treatments are unproven and based on anecdote. PMID:12492389

Bailey, Paul M; Little, Mark; Jelinek, George A; Wilce, Jacqueline A

2003-01-01

18

Triple antiplatelet therapy in acute coronary syndromes.  

PubMed

Heightened platelet activity plays a critical role in thrombus formation, which is central to acute coronary syndromes (ACS), including non-ST-segment elevation (NSTE)-ACS (comprising unstable angina pectoris and non-ST-segment elevation myocardial infarction [NSTEMI]) and ST-segment elevation myocardial infarction (STEMI), and has been implicated in poor clinical outcome. Platelets not only impact coronary thrombus but are major contributors to microcirculatory dysfunction and vascular inflammation. Efforts to inhibit platelet function, including antiplatelet therapy, are paramount to the management of ACS; thus, a growing recognition of the various pathways driving platelet activity has given rise to the need for multiple agents that impart complimentary mechanisms of action. While only inhibiting platelet activation will still allow for aggregation, i.e. the binding of glycoprotein (GP) IIb/IIIa receptors to fibrinogen, solely blocking aggregation may leave platelet-activating pathways free to sustain the production and release of various pro-inflammatory and pro-thrombotic compounds. The benefit of 'triple antiplatelet therapy', referring to the combination of aspirin, a thienopyridine or non-thienopyridine adenosine diphosphate (ADP)/P2Y12 receptor blocker and a GPIIb/IIIa inhibitor (GPI), has been demonstrated in patients with NSTE-ACS who ultimately undergo percutaneous coronary intervention (PCI) and are determined to be at an elevated risk for ischaemic events, and in patients undergoing primary PCI. It is therefore recommended by the European Society of Cardiology (ESC) and American College of Cardiology/American Heart Association. Furthermore, the rationale for adding a GPI, particularly in patients with STEMI, is backed by studies that have shown negligible effects of a 600?mg clopidogrel loading dose, despite being administered 4 hours prior to PCI. Moreover, it has been observed that the physiological state of STEMI may deem dual antiplatelet therapy ineffective, because during an acute event the absorption of clopidogrel may be impaired. Nonetheless, there is still considerable variability with respect to the use of triple antiplatelet therapy such as that documented in the Euro Heart Survey. The perception that the mortality benefit afforded by adding a GPI to dual oral antiplatelet therapy does not outweigh the risk is a likely factor. This may be fuelled by results of trials such as BRAVE-3, which, inconsistent with those for On-TIME 2, failed to prove the value of adding a GPI to dual oral antiplatelet therapy in patients with STEMI. Subsequent analyses have indeed demonstrated the positive benefit-risk ratio associated with adding a GPI and determined that the timing of GPI administration could have an impact on clinical outcome related to its impact on infarct size in patients with STEMI. Additionally, it has been presumed that a synergistic effect exists between P2Y12 inhibitors and GPIs. Triple antiplatelet therapy has a significant role to play in the management of patients with ACS managed with PCI. An understanding of patient risk status and timing of symptoms and bleeding risk is crucial to patient selection and ensuring that this therapy is optimized. Though no interaction has been noted in trials of newer, more potent antiplatelet agents, future studies are key to determining the role of this strategy in the era of these more potent agents. PMID:21902293

Valgimigli, Marco; Minarelli, Monica

2011-09-10

19

Churg-Strauss syndrome associated with montelukast therapy  

Microsoft Academic Search

Churg-Strauss syndrome is a rare form of eosinophilic vasculitis associated with asthma. There have been several recent case reports of the condition in association with leukotriene antagonists and it has been speculated that the Churg-Strauss syndrome was unmasked when oral corticosteroids were withdrawn. We report a case of Churg-Strauss syndrome associated with montelukast therapy in an asthmatic patient in whom

J M Tuggey; H S R Hosker

2000-01-01

20

Systemic therapy of Cushing’s syndrome  

PubMed Central

Cushing’s disease (CD) in a stricter sense derives from pathologic adrenocorticotropic hormone (ACTH) secretion usually triggered by micro- or macroadenoma of the pituitary gland. It is, thus, a form of secondary hypercortisolism. In contrast, Cushing’s syndrome (CS) describes the complexity of clinical consequences triggered by excessive cortisol blood levels over extended periods of time irrespective of their origin. CS is a rare disease according to the European orphan regulation affecting not more than 5/10,000 persons in Europe. CD most commonly affects adults aged 20–50 years with a marked female preponderance (1:5 ratio of male vs. female). Patient presentation and clinical symptoms substantially vary depending on duration and plasma levels of cortisol. In 80% of cases CS is ACTH-dependent and in 20% of cases it is ACTH-independent, respectively. Endogenous CS usually is a result of a pituitary tumor. Clinical manifestation of CS, apart from corticotropin-releasing hormone (CRH-), ACTH-, and cortisol-producing (malign and benign) tumors may also be by exogenous glucocorticoid intake. Diagnosis of hypercortisolism (irrespective of its origin) comprises the following: Complete blood count including serum electrolytes, blood sugar etc., urinary free cortisol (UFC) from 24 h-urine sampling and circadian profile of plasma cortisol, plasma ACTH, dehydroepiandrosterone, testosterone itself, and urine steroid profile, Low-Dose-Dexamethasone-Test, High-Dose-Dexamethasone-Test, after endocrine diagnostic tests: magnetic resonance imaging (MRI), ultra-sound, computer tomography (CT) and other localization diagnostics. First-line therapy is trans-sphenoidal surgery (TSS) of the pituitary adenoma (in case of ACTH-producing tumors). In patients not amenable for surgery radiotherapy remains an option. Pharmacological therapy applies when these two options are not amenable or refused. In cases when pharmacological therapy becomes necessary, Pasireotide should be used in first-line in CD. CS patients are at an overall 4-fold higher mortality rate than age- and gender-matched subjects in the general population. The following article describes the most prominent substances used for clinical management of CS and gives a systematic overview of safety profiles, pharmacokinetic (PK)-parameters, and regulatory framework. PMID:25091295

2014-01-01

21

[Successful electroconvulsive therapy of Cotard syndrome with bitemporal hypoperfusion].  

PubMed

A case study is presented to illustrate a rare condition described by Cotard as "délire de négation". The central symptom is a nihilistic delusion with denial of one's own existence of oneself and that of the external world. In the present case, the syndrome became manifest as an escalation of a recurrent depressive disorder late in life. After initial resistance to therapy, the syndrome was successfully treated with electroconvulsive therapy. For the first time, we report the regional cerebral blood flow measured by 99mTc-HMPAO-SPECT before and after therapy. Before treatment, significant bitemporal hypoperfusion relative to the cerebellum was found, which was no longer demonstrable on remission. PMID:9005351

Lohmann, T; Nishimura, K; Sabri, O; Klosterkötter, J

1996-05-01

22

Sick sinus syndrome as a complication of mediastinal radiation therapy  

SciTech Connect

A 33-year-old man who had received mediastinal radiation therapy for Hodgkin's disease 12 years earlier developed a symptomatic sick sinus syndrome requiring the implantation of a permanent pacemaker. The sick sinus syndrome and a finding of an occult constrictive pericarditis were considered to be due to the previous mediastinal irradiation. A ventricular pacemaker was chosen because mediastinal radiotherapy also increases the risk of developing atrioventricular conduction defects.

Pohjola-Sintonen, S.; Toetterman, K.J.K.; Kupari, M. (Helsinki Univ. Central Hospital (Finland))

1990-06-01

23

[Iatrogenic influence of testosterone therapy among persons with Klinefelter Syndrome].  

PubMed

The purpose of this work is a presentation of cases of persons with Klinefelter Syndrome where, most probably--as a result of testosterone supplementation therapy, some aggressive behaviours occurred requiring implementation of pharmacotherapy and psychiatric hospitalisation. The authors contemplate adequacy of standard hormone treatment where the main purpose is to improve the quality of life of the karyotype 47, XXY patients described. The article has been enhanced by a theoretical introduction into the basic subject which is the Klinefelter Syndrome. PMID:21614835

Radko, Magdalena; ?ucka, Izabela; Zió?kowski, Jacek

2011-01-01

24

Bortezomib therapy for nephrotic syndrome due to idiopathic membranous nephropathy.  

PubMed

Idiopathic membranous nephropathy is a common cause of nephrotic syndrome in adults. The nephrotic syndrome due to idiopathic membranous nephropathy is often resistant to glucocorticosteroids and requires an alkylating agent such as chlorambucil or cyclophosphamide to induce remission. Recent studies illustrate that antibodies against the autoantigen M-type phospholipase A2 receptor contribute to a vast majority but not all cases of idiopathic membranous nephropathy. Herein, we report a patient with nephrotic syndrome due to membranous nephropathy that was resistant to 6 months of therapy with ramipril and high-dose glucocorticosteroids but responded to a single cycle of bortezomib infusion. PMID:24430762

Hartono, Choli; Chung, Miriam; Kuo, Sheng F; Seshan, Surya V; Muthukumar, Thangamani

2014-02-01

25

Bortezomib therapy for nephrotic syndrome due to idiopathic membranous nephropathy  

PubMed Central

Idiopathic membranous nephropathy is a common cause of nephrotic syndrome in adults. The nephrotic syndrome due to idiopathic membranous nephropathy is often resistant to glucocorticosteroids and requires an alkylating agent such as chlorambucil or cyclophosphamide to induce remission. Recent studies illustrate that antibodies against the autoantigen M-type phospholipase A2 receptor contribute to a vast majority but not all cases of idiopathic membranous nephropathy. Herein, we report a patient with nephrotic syndrome due to membranous nephropathy that was resistant to 6 months of therapy with ramipril and high-dose glucocorticosteroids but responded to a single cycle of bortezomib infusion. PMID:24430762

Chung, Miriam; Kuo, Sheng F.; Seshan, Surya V.; Muthukumar, Thangamani

2014-01-01

26

Rituximab therapy for steroid-dependent minimal change nephrotic syndrome  

Microsoft Academic Search

We present a patient with steroid-sensitive but high-dose steroid-dependent nephrotic syndrome who was treated with rituximab. For 9 months following therapy she had undetectable CD19 cells in the peripheral circulation. She remained in remission during this period even though therapy was reduced to low-dose, alternate day prednisolone only. After 9 months, CD19 cells were once again detectable. Shortly after CD19

Rodney D. Gilbert; Eleanor Hulse; Susan Rigden

2006-01-01

27

Cognitive behavior therapy for chronic fatigue syndrome: efficacy and implications  

Microsoft Academic Search

Cognitive behavior therapy (CBT) is a form of nonpharmacologic treatment. It is based on a model of chronic fatigue syndrome (CFS) that hypothesizes that certain cognitions and behavior may perpetuate symptoms and disability–that is, act as obstacles to recovery. Treatment emphasizes self-help and aims to help the patient to recover by changing these unhelpful cognitions and behavior. There is now

Michael Sharpe

1998-01-01

28

Dance Therapy with Physical Therapy for Children with Down Syndrome.  

ERIC Educational Resources Information Center

This study sought to investigate effects of a dance program on bilateral toe-standing balance and single-point static balance skills of a group of children with Down Syndrome. Thirteen experimental and 10 control group students between the ages of 3 and 13 years were assessed on toe-standing balance and single-point standing balance on the right…

Dupont, Blanche Burt; Schulmann, Diana

29

Behçet syndrome: from pathogenesis to novel therapies.  

PubMed

Behçet syndrome is a chronic disease hallmarked by inflammation of the blood vessels that is related to an autoimmune reaction caused by inherited susceptibility due to specific genes and environmental factors, probably components of infectious microorganisms, which turn on or get going the disease in genetically susceptible subjects. The more common clinical expression of the disease is represented by a triple-symptom complex of recurrent oral aphthous ulcers, genital ulcers, and uveitis, sometimes associated with inflammatory arthritis, phlebitis, iritis, as well as inflammation of the digestive tract, brain, and spinal cord. The treatment strategies used to manage the manifestations of Behçet syndrome have gradually progressed, and a number of new therapeutic resources have been implemented in recent years, allowing better control of pathogenic mechanisms, reducing symptoms and suffering, and ameliorating patient's outcome. PMID:25447032

Mazzoccoli, Gianluigi; Matarangolo, Angela; Rubino, Rosa; Inglese, Michele; De Cata, Angelo

2014-12-01

30

Asperger's syndrome: diagnosis, comorbidity and therapy.  

PubMed

Asperger's syndrome (AS), a behavioral disorder that is related to autism, is associated with abnormal social functioning and repetitive behaviors but not with a decrease in intelligence or linguistic functionality. This article reviews the clinical diagnosis of AS and discusses the comorbid disorders that may be present with AS, as well as the efficacy, safety, and tolerability of pharmacotherapies given to AS patients, as reported in preclinical and clinical studies. AS may be present with several comorbid disorders including: attention deficit hyperactivity disorder, anxiety, schizophrenia, bipolar disorder, depression, and Tourette's syndrome. The difficulty in distinguishing AS from autism results in treating the comorbid disorder symptoms, rather than treating the symptoms of AS. Accordingly, there is a great need to further understand the psychobiology of AS and its association with other disorders, which should expand the pharmacological and non-pharmacological therapeutic options and improve the quality of life for AS patients. PMID:25655905

Tarazi, Fi; Sahli, Zt; Pleskow, J; Mousa, Sa

2015-03-01

31

Pharmacologic therapies for complex regional pain syndrome  

Microsoft Academic Search

Complex regional pain syndrome (CRPS) remains a challenging condition to diagnose and treat. There are few large-scale, randomized\\u000a trials of pharmacologic agents, and most published studies are small, uncontrolled, or presented only in abstract form at\\u000a meetings. The most commonly used agents, such as anticonvulsants, antidepressants, and opiates, have been found to be useful\\u000a for other neuropathic pain conditions in

Sean Mackey; Steven Feinberg

2007-01-01

32

Complementary Therapy in Polycystic Ovary Syndrome  

PubMed Central

Polycystic Ovary Syndrome (PCOS) is an endocrine disease. PCOS afflicts 5 to 10 % of women of reproductive age. The symptoms are: amenorrhea, oligomenorrhea, hirsutism, obesity, infertility, chronic hyperandrogenic anovulation and acne. Other risk factors aggravate this condition: insulin resistance, obesity, hypertension, dyslipidemia, inflammation and subclinical cardiovascular disease. Anxiety, depression and reduced quality of life are also common. This review highlights the mechanisms and the beneficial effects of acupuncture, exercise and resveratrol on animal models and on humans affected by PCOS. PMID:24809037

Aquino, C. I.; Nori, S. L.

2014-01-01

33

HIV therapy, metabolic syndrome, and cardiovascular risk  

Microsoft Academic Search

People with HIV infection have metabolic abnormalities that resemble metabolic syndrome (hypertriglyceridemia, low high-density\\u000a lipoprotein cholesterol, and insulin resistance), which is known to predict increased risk of cardiovascular disease (CVD).\\u000a However, there is not one underlying cause for these abnormalities and they are not linked to each other. Rather, individual\\u000a abnormalities can be affected by the host response to HIV

Vivian Pao; Grace A. Lee; Carl Grunfeld

2008-01-01

34

Therapies for steroid-resistant nephrotic syndrome  

Microsoft Academic Search

Between 10 and 20% of children with primary nephrotic syndrome are steroid-resistant (SRNS). From earlier studies in children\\u000a with SRNS, we know that cyclosporin (with or without alternate-day prednisone) and cyclophosphamide (with pulse intravenous\\u000a corticosteroids) result in comparable complete or partial remission rates of about 60%. An evaluation of the relative effectiveness\\u000a of cyclophosphamide and cyclosporin has not been possible

Elisabeth M. Hodson; Jonathan C. Craig

2008-01-01

35

Electroconvulsive therapy for lycanthropy and Cotard syndrome: a case report.  

PubMed

We present a case of psychotic depression presenting with lycanthropy (being converted to a pig) and Cotard syndrome simultaneously and treated with electroconvulsive therapy. A 37-year-old female patient developed psychotic depression after a stressor (a possibility of having a malignancy). As her depression worsened, she developed delusional belief of self being metamorphosed to a pig and her children also being metamorphosed into pig. In addition, she had the delusional belief that her own body and body of her children was rotting away. She was treated with electroconvulsive therapy along with venlafaxine and olanzapine, with which she improved completely. PMID:20562636

Grover, Sandeep; Shah, Ruchita; Ghosh, Abhishek

2010-12-01

36

Shoulder Impingement Syndromes: Implications on Physical Therapy Examination and Intervention  

PubMed Central

A painful shoulder presents challenges in examination, diagnosis and intervention for the physical therapist because of the complexity of the structures involved. A common cause of shoulder pain is shoulder impingement syndrome. This was first described as a condition in which the soft tissues of the subacromial space were chronically entrapped and compressed between the humeral head and the subacromial arch. This definition does not account for the myriad potential causes of shoulder impingement conditions, as forms of impingement other than subacromial soft tissue compression may explain different symptomatic shoulder injuries. This paper describes shoulder impingement syndromes that have been hypothesized, identified and analyzed in the literature. Physical Therapy examination and intervention for these syndromes are also discussed.

2005-01-01

37

Therapies in Aicardi–Goutičres syndrome  

PubMed Central

Aicardi–Goutičres syndrome (AGS) is a genetically determined disorder, affecting most particularly the brain and the skin, characterized by the inappropriate induction of a type I interferon-mediated immune response. In most, but not all, cases the condition is severe, with a high associated morbidity and mortality. A number of important recent advances have helped to elucidate the biology of the AGS-related proteins, thus providing considerable insight into disease pathology. In this study, we outline the clinical phenotype of AGS, paying particular attention to factors relevant to therapeutic intervention. We then discuss the pathogenesis of AGS from a molecular and cell biology perspective. Finally, we suggest possible treatment strategies in light of these emerging insights. PMID:23607857

Crow, Y J; Vanderver, A; Orcesi, S; Kuijpers, T W; Rice, G I

2014-01-01

38

Should we consider MMF therapy after rituximab for nephrotic syndrome?  

Microsoft Academic Search

The management of steroid-dependent nephrotic syndrome, especially in patients who have failed to respond to cytotoxic drugs,\\u000a such as cyclophosphamide, remains challenging. Rituximab represents a new (off-label) therapeutic option. In a significant\\u000a portion of patients, it has a short serum half-life following the recovery of CD20-positive cells. The addition of mycophenolate\\u000a mofetil (MMF) as a maintenance therapy is also an

Guido Filler; Shih-Han Susan Huang; Ajay P. Sharma

39

Diagnostic test for prenatal identification of Down's syndrome and mental retardation and gene therapy therefor  

DOEpatents

A a diagnostic test useful for prenatal identification of Down syndrome and mental retardation. A method for gene therapy for correction and treatment of Down syndrome. DYRK gene involved in the ability to learn. A method for diagnosing Down's syndrome and mental retardation and an assay therefor. A pharmaceutical composition for treatment of Down's syndrome mental retardation.

Smith, Desmond J. (Oakland, CA); Rubin, Edward M. (Berkeley, CA)

2000-01-01

40

[Syndrome of Inadequate ADH Secretion: Pitfalls in diagnosis and therapy].  

PubMed

Euvolemic hyponatremia is most frequently caused by the syndrome of inappropriate antidiu-retic hormone secretion (SIADH). Causes of SIADH-induced hyponatremia are myriad and include tumors, pulmonary diseases or central nervous system disorders. SIADH can also be induced by a broad spectrum of drugs such as antidepressants and antiepileptics. The unfavorable prognostic relevance of hyponatremia is often underestimated. SIADH therapy focuses on the treatment of the underlying disease. Thus, a thorough differential diagnostic assessment of the genesis of SIADH is crucial. Therapy options for euvolemic hyponatremia include fluid restriction, administration of hyperosmolar saline solution in case of severe symptoms, or therapy with tolvaptan. Tolvaptan is a selective, oral vasopressin-V2-receptor-antagonist that inhibits ADH-induced retention of electrolyte-free water in the connecting duct of the kidney. This inhibition results in an increased serum sodium level. Close monitoring of serum sodium levels and volume status is imperative, especially during the initial phase of therapy. Fluid restriction is unnecessary during tolvaptan therapy; a previously prescribed fluid re-striction should be stopped when therapy begins. Treatment with tolvaptan can often result in a rapid and controlled improvement of the symptoms. Different cases presented in this article illustrate the diversity of SIADH in clinical practice relating to its diagnosis and its therapy as well as difficulties in identifying the underlying cause in clinical practice. PMID:25734677

Schäffler, Andreas; Lindner, Uwe

2015-03-01

41

Epigenetics, fragile X syndrome and transcriptional therapy.  

PubMed

Epigenetics refers to the study of heritable changes in gene expression that occur without a change in DNA sequence. Epigenetic mechanisms therefore include all transcriptional controls that determine how genes are expressed during development and differentiation, but also in individual cells responding to environmental stimuli. The purpose of this review is to examine the basic principles of epigenetic mechanisms and their contribution to human disorders with a particular focus on fragile X syndrome (FXS), the most common monogenic form of developmental cognitive impairment. FXS represents a prototype of the so-called repeat expansion disorders due to "dynamic" mutations, namely the expansion (known as "full mutation") of a CGG repeat in the 5'UTR of the FMR1 gene. This genetic anomaly is accompanied by epigenetic modifications (mainly DNA methylation and histone deacetylation), resulting in the inactivation of the FMR1 gene. The presence of an intact FMR1 coding sequence allowed pharmacological reactivation of gene transcription, particularly through the use of the DNA demethylating agent 5'-aza-2'-deoxycytydine and/or inhibitors of histone deacetylases. These treatments suggested that DNA methylation is dominant over histone acetylation in silencing the FMR1 gene. The importance of DNA methylation in repressing FMR1 transcription is confirmed by the existence of rare unaffected males carrying unmethylated full mutations. Finally, we address the potential use of epigenetic approaches to targeted treatment of other genetic conditions. PMID:24123753

Tabolacci, Elisabetta; Chiurazzi, Pietro

2013-11-01

42

Corticosteroid Injections Versus Manual Physical Therapy for Treatment of the Shoulder Impingement Syndrome  

MedlinePLUS

Corticosteroid Injections Versus Manual Physical Therapy for Treatment of the Shoulder Impingement Syndrome The full report is titled “OneYear Outcome of Subacromial Corticosteroid Injection Compared With ...

43

Fibromyalgia Syndrome and Spa Therapy: Myth or Reality?  

PubMed Central

Fibromyalgia syndrome (FS) is a common musculoskeletal disorder characterized by otherwise unexplained chronic widespread pain, a lowered pain threshold, high tender point counts, sleep disturbances, fatigue, headache, irritable bowel syndrome, morning stiffness, paraesthesias in the extremities, often psychological distress and depressed mood. Consequently, FS has a negative impact on working capacity, family life, social functioning and quality of life. Because of unknown etiology and not clearly understood pathogenesis, there is no standard therapy regime for FS. A variety of medical treatments, including antidepressants, opioids, analgesic or non-steroidal anti-inflammatory drugs, sedatives, muscle relaxants and antiepileptics, have been used to treat FS. Currently, no pharmacological treatment for FS is consistently successful. According to recent guidelines, the optimal treatment of FS requires a multidisciplinary approach with a combination of non-pharmacological and pharmacological treatment modalities. Spa therapy is a popular treatment for FS in many European countries, as well as in Japan and Israel. However, despite their long history and popularity spa treatments are still the subject of debate and their role in modern medicine is still not clear. The objective of this review is to summarize the currently available information on clinical effects and mechanism of action of spa therapy in FS. We also provide some suggestions for further development in this area. PMID:22408369

Guidelli, Giacomo M.; Tenti, Sara; De Nobili, Emanuele; Fioravanti, Antonella

2012-01-01

44

[Successful long-term therapy of Stewart-Bluefarb syndrome].  

PubMed

The Stewart-Bluefarb syndrome is defined as an unilateral angiodermatitis due to multiple arterio-venous fistules accompanied by acroangiodermatitis resembling Kaposi sarcoma (pseudo-kaposi sarcoma). The acroangiodermatitis is most common on the lower limb. It leads to ulcerated nodules with a high risk of bleeding and infection, as well as edema, pain and seldom limb hypertrophy. Curative therapy requires elimination of the arteriovenous shunts. Surgical destruction of the multiple small fistulae is a limitating factor. A better alternative is embolisation, but this approach carries the risk of ischemia and necrosis. A 32 year old female patient with Stewart-Bluefarb syndrome is presented; she has been successfully treated with embolisation on eight occasions. PMID:10875071

Utermann, S; Kahle, B; Petzoldt, D

2000-05-01

45

Treating irritable bowel syndrome: overview, perspective and future therapies  

PubMed Central

This article summarizes the ongoing challenges in irritable bowel syndrome and the exciting opportunities for development of novel therapies for this common, enigmatic condition. The challenges include insufficient understanding of mechanisms, lack of specificity of symptoms, differentiation from other conditions, and lack of availability of noninvasive tests to identify dysfunctions. However, significant opportunities are reflected by the advances in clinical trial design and, particularly, clinically relevant end points for such trials, and the increasing understanding of basic neuroenteric science. The latter has delivered two new medications to the practice (alosetron and tegaserod), and other candidate therapies (other serotonergic, tachykininergic, opioid, cannabinoid modulators) are being carefully appraised as potential drugs for the future. PMID:15037521

Camilleri, Michael

2004-01-01

46

Oral antiplatelet therapy in acute coronary syndromes: recent developments.  

PubMed

The purpose of this article is to summarize the current knowledge about treatment with oral platelet inhibitors in patients with acute coronary syndrome (ACS). Antiplatelet therapy has been shown to improve the prognosis of patients with ACS with ST segment elevation myocardial infarction (STEMI) and non-ST segment elevation ACS (NSTE-ACS). Aspirin should be given with a loading dose of 250-500 mg, followed by 75-100 mg/day. Dual antiplatelet therapy is recommended for all patients with ACS for 12 months regardless of the initial revascularization strategy. Clopidogrel should be administered at first medical contact in STEMI with a loading dose of 600 mg. In patients with ACS and percutaneous coronary intervention (PCI) 2 × 75 mg clopidogrel should be given daily over 7 days, while in all other patients 75 mg per day appears to be sufficient. The two newer adenosine diphosphate-receptor antagonists prasugrel and ticagrelor lead to a more rapid and effective inhibition of platelet aggregation compared with clopidogrel, which was associated with an improved clinical outcome in two large randomized studies. Prasugrel is indicated in patients with ACS undergoing PCI and was most effective in diabetics and in patients with STEMI. In the recent TaRgeted platelet Inhibition to cLarify the Optimal strateGy to medicallY manage Acute Coronary Syndromes trial in medically treated patients with NSTE-ACS, prasugrel did not significantly reduce ischemic events compared with clopidogrel. Ticagrelor has been studied in the whole spectrum of ACS patients and reduced cardiovascular and total mortality in comparison with clopidogrel. The greatest benefit has been observed in patients with planned conservative treatment and in patients with impaired renal function. Expanding antiplatelet therapy from dual to triple therapy including a platelet thrombin receptor antagonist in the thrombin receptor antagonist for clinical event reduction in acute coronary syndrome trial was not associated with a significant reduction in the primary combined endpoint but an increase in bleeding complications. However, in the Thrombin Receptor Antagonist in Secondary Prevention of atherothrombotic ischemic events study in patients with prior myocardial infarction, vorapaxar on top of standard antiplatelet therapy was effective. PMID:25135288

Zeymer, Uwe

2013-06-01

47

Children with Down Syndrome Improved in Motor Functioning and Muscle Tone Following Massage Therapy  

ERIC Educational Resources Information Center

Twenty-one moderate to high functioning young children (mean age, two years) with Down syndrome receiving early intervention (physical therapy, occupational therapy and speech therapy) were randomly assigned to additionally receive two 0.5-hour massage therapy or reading sessions (control group) per week for two months. On the first and last day…

Hernandez-Reif, Maria; Field, Tiffany; Largie, Shay; Mora, Dana; Bornstein, Joan; Waldman, Ronnie

2006-01-01

48

044. Sleep apnoea syndrome after growth hormone therapy in a patient with Prader-Willi syndrome  

PubMed Central

Background Prader-Willi syndrome (PWS) is a rare genetic disorder, which incidence is 1/10,000-1/30,000. It is characterized by hypotonia, short stature, delayed motor and mental development, hypogonadism, hyperphagia, obesity and specific physical appearance. It has been observed increased frequency of obstructive apneas-hypopnoeas and sleep architecture disorder in patients with PWS. Prescription of growth hormone (GH) is indicated in childhood, in patients with genetic confirmed syndrome and growth disorders. However, several case reports raise the question of interaction between the growth hormone therapy and apnea syndrome. Case presentation A case of a 7-year-old girl with PWS is examined, who started treatment with GH in 2012 and her respiratory function has been followed up for two years with continuous sleep studies. The first sleep study type III showed AHI 9.5/h, average SpO2 97.8%, minimum SpO2 91% and oxygen desaturation index (ODI) 4.8/h. A year later, a new polysomnography (PSG) study demonstrated AHI 3.1/h, average SpO2 96%, minimum SpO2 91% and ODI 4/h. On 5th/2014, while she was at the 4th semester of therapy with GH, the PSG study recorded deterioration with AHI 42.9/h, ODI 32.4/h, average SpO2 95.8% and minimum 83%. The hormone therapy was blamed for this worsening and consequently the therapy was terminated. The patient underwent a sleep study type III, three months after the therapy disruption and there has been reported an important improvement: AHI 11.2/h, average SpO2 95%, minimum SpO2 92%. Conclusions The therapy with growth hormone in patients with PWS may cause sleep breathing disorder and result in apneas. Patients should be strictly observed with continuous sleep studies in order to continue the therapy or not. The deterioration of respiratory function during sleep is an indication for disruption of hormone therapy, something that can gradually restore the observed disorder.

Nikolaou, Konstantina; Papagianni, Maria; Siopi, Dimitra; Markopoulou, Katerina; Kareklas, Nikos; Galanou, Artemis; Agrafiotis, Michalis; Tsara, Venetia

2015-01-01

49

Cervical Syndrome – the Effectiveness of Physical Therapy Interventions  

PubMed Central

ABSTRACT Introduction: The cervical syndrome refers to a set of disorders caused by the changes in the cervical spine and the soft-tissue surrounding it, with pain as the predominant symptom. Sore neck has been a common problem among a large section of today`s population. The factors contributing to this issue include the modern lifestyle, prolonged sitting and incorrect, fixed or constrained working postures. The root of these difficulties is found in the mechanical disorders of the cervical spine structures, poor body posture and jerky body movements. In the Scandinavian countries neck pain is considered to be a public health problem. Methods: The study evaluated 25 patients with an established diagnosis of cervical syndrome. The research was conducted at the PI Institute of Occupational and Sports Medicine of Zenica–Doboj Canton. Each patient received twenty physical therapy treatment sessions. Results and conclusions: The study included 25 patients suffering from the cervical syndrome. The statistical analysis of gender distribution indicated that 36% of the patients were male, while 64% were female. The mean age of study participants was 46.76±4,23. The patients ranged in age from 39 to 54 years, with no statistically significant difference in the mean age of male and female patients, p=0.691. Analysing the types of occupational activities performed by the patients, the study found a positive relation between neck pain and prolonged sitting at work. The patients who performed office work made up 76% of the total number. Each method of physical therapy applied in the treatment of neck pain patients proved useful. However, the combination of electrotherapy, kinesiotherapy and manual massage proved to be most effective. Conclusion: The cervical syndrome is a common medical condition primarily affecting adult population, with prevalence being higher among women and office workers. The condition places a considerable socioeconomic burden on the afflicted. Cervical pain ranges greatly in severity – from moderate to unbearable, thus leading to high levels of work absence as well as to a decrease in the quality of life. Proper physical therapy program can help the patients with neck pain return to their normal everyday activities, improve their quality of life, as well as reduce the absence from work. PMID:25568511

Kasumovic, Mersija; Gorcevic, Emir; Gorcevic, Semir; Osmanovic, Jasna

2013-01-01

50

Adjuvant physical therapy versus occupational therapy in patients with reflex sympathetic dystrophy\\/complex regional pain syndrome type I  

Microsoft Academic Search

Oerlemans HM, Oostendorp RAB, de Boo T, van der Laan L, Severens JL, Goris RJA. Adjuvant physical therapy versus occupational therapy in patients with reflex sympathetic dystrophy\\/complex regional pain syndrome type I. Arch Phys Med Rehabil 2000;81:49-56. Objective: To investigate the effectiveness and cost of physical therapy (PT) or occupational therapy (OT) in patients with reflex sympathetic dystrophy (RSD). Design:

H. Margreet Oerlemans; Rob A. B. Oostendorp; Theo de Boo; Lyckle van der Laan; Johan L. Severens; R. Jan A. Goris

2000-01-01

51

[Common and not so common nerve entrapment syndromes: diagnostics, clinical aspects and therapy].  

PubMed

Altogether, nerve entrapment syndromes have a very high incidence. Neurological deficits attributable to a focal peripheral nerve lesion lead to the clinical diagnosis. Frequently, pain is the dominant symptom but is often not confined to the nerve supply area. Electroneurography, electromyography, and more recently also neurosonography are the most important diagnostic tools. In most patients surgical therapy is necessary, which should be carried out in a timely manner. The entrapment syndromes discussed are suprascapular nerve entrapment, carpal tunnel syndrome, cubital tunnel syndrome, meralgia paraesthetica, thoracic outlet syndrome and anterior interosseous nerve syndrome. PMID:25526716

Schulte-Mattler, W J; Grimm, T

2015-02-01

52

Mucopolysaccharidosis III (Sanfilippo Syndrome)- disease presentation and experimental therapies.  

PubMed

Sanfilippo Syndrome or Mucopolysaccharidosis Ill (MPS Ill) is a group of lysosomal storage diseases resulting from a deficiency of one of four lysosomal enzymes: Type A - heparan N-sulfatase (SGSH), Type B - a-N-acetylglucosaminidase (NAGLU), Type C - acetyl CoA a-glucosaminide acetyltransferase (HGSNAT) and Type D - N-acetylglucosamine-6-sulfatase (GNS). Each of these enzymes is necessary for degradation of heparan sulfate. Deficiency of any of these enzymes manifests as a neurodegenerative disorder with accompanying somatic manifestations. Typically this presents early in life with developmental delays followed by developmental regression and usually results in death sometime during the second decade of life, though several less severe cases have been described living into late adulthood (30's to 60's). Often there is a delay of several years from time of symptom onset to diagnosis. Currently treatment is limited to supportive care. We will briefly discuss the typical natural history and presumed pathophysiology of the disease. We will also discuss current experimental therapies being pursued for treatment of this devastating disease. These include enzyme replacement, gene therapy, stem cell therapy, and substrate reduction approaches. PMID:25345095

Gilkes, Janine A; Heldermon, Coy D

2014-09-01

53

Iron chelation therapy in myelodysplastic syndromes: where do we stand?  

PubMed Central

Anemia leading to transfusion dependency (TD) and iron overload (IO) is commonly observed in patients with myelodysplastic syndromes (MDS). In MDS, TD and IO have been retrospectively associated with inferior survival and worse clinical outcomes, including cardiac, hepatic and endocrine dysfunction, and, in some analyses, with leukemic progression and infectious complications. Although suggested by retrospective analyses, clear prospective documentation of the beneficial effects of iron chelation therapy (ICT) on organ function and survival in MDS patients with TD and IO is currently lacking. Consequently, the role of ICT in MDS patients with TD and IO remains a very controversial aspect in the management of MDS. In this review, the authors summarize the current knowledge regarding IO in MDS and the role of ICT. PMID:23991926

Mitchell, Mhairi; Gore, Steven D; Zeidan, Amer M

2014-01-01

54

More is better: combination therapies for myelodysplastic syndromes.  

PubMed

The myelodysplastic syndromes (MDS) are a heterogenous collection of clonal hematopoietic malignancies that exist as a subgroup of the myeloid neoplasms as classified by the World Health Organization (WHO). They are characterized by ineffective hematopoiesis, subsequent cytopenias, transformation to acute myeloid leukemia (AML), and poor overall survival. There are currently three FDA-approved medications for MDS; lenalidomide, azacitidine, and decitabine. The role of these agents is to diminish the clinical impact of MDS and delay its progression to AML. However, despite known results with these monotherapies, recent clinical trials with a variety of combinations for MDS have demonstrated promising results. These trials include combinations of hypomethylating agents, histone deacetylase inhibitors, growth factors, and chemotherapy among others. In this paper we review the current literature on combination therapies in MDS, analyze on-going and concluded trials, and suggest future possibilities for combination strategies in MDS. PMID:25659727

Ornstein, Moshe C; Mukherjee, Sudipto; Sekeres, Mikkael A

2015-03-01

55

Electroconvulsive therapy in a patient with moyamoya syndrome.  

PubMed

We report on a 30-year-old woman diagnosed with moyamoya syndrome resulting from sickle cell disease who developed catatonia and was successfully treated with electroconvulsive therapy (ECT). Neuroimaging revealed severe tandem narrowing of the left internal carotid artery with diminished cerebral blood flow, moderate narrowing of the right supraclinoid aspect of the right internal carotid artery, and associated numerous lenticulostriate collaterals bilaterally, consistent with moyamoya. The patient presented with mutism; posturing; immobility; stupor; withdrawal; refusal to eat, drink, or speak; and staring, supporting a diagnosis of catatonia. It initially responded to a lorazepam challenge; however, a complicated hospital course and deterioration of the patient's condition, including septic shock, delirium, and continued catatonic symptoms, led to the pursuit of ECT to treat her symptoms. We discuss the risks involved with the administration of ECT in a patient with fragile cerebral vasculature and the successful treatment of catatonia in this patient without resultant stroke or cerebral hemorrhage. PMID:24901428

Ghignone, Erica; Rosenthal, Lisa; Lloyd, Robert Brett; Mouli, Samdeep; Dinwiddie, Stephen

2015-03-01

56

Adherence to secondary prevention therapies in acute coronary syndrome.  

PubMed

Despite overwhelming evidence of the effectiveness of secondary prevention therapies, surveys indicate poor adherence to medical treatments and lifestyle recommendations after an acute coronary syndrome. The term adherence is preferred over compliance, as the former suggests a therapeutic alliance, whereas the latter reflects passive patient obedience. Poor adherence results from a complex interplay of multiple factors at patient, practitioner and system levels. Poor adherence among patients with stable coronary artery disease is associated with increased risk of cardiovascular admissions (10%-40%), coronary interventions (10%-30%) and cardiovascular mortality (50%-80%). Improving adherence is a complex process. A range of interventions that target modifiable factors influencing adherence have been explored, but there are no guidelines to guide the choice, and multidisciplinary efforts may be needed. Future research in the area should focus on comparative efficacy of interventions to enhance adherence. PMID:25390496

Thakkar, Jay B; Chow, Clara K

2014-11-17

57

Metabolic syndrome – Removing roadblocks to therapy: Antigenic immunotherapies?  

PubMed Central

Up to 25 per cent of the world?s adult population may have the metabolic syndrome, a condition closely associated with central obesity. The metabolic syndrome is a major risk factor for cardiovascular disease and type 2 diabetes and therefore represents an important worldwide health problem. In addition to metabolic abnormalities such as raised fasting plasma glucose, high cholesterol and high blood pressure, there is consensus that obese subjects develop a state of low-grade chronic immune activation. This sustained pro-inflammatory response in fat tissue is thought to worsen insulin resistance and dyslipidemia. Likewise, the immune system contributes to the detrimental cascade of events leading to plaque formation in atherosclerosis. It has long been assumed that the innate arm of the immune system was the only key player, but emerging evidence suggests that there is in fact a sizeable adaptive immune component to obesity and cardiovascular disease. From a therapeutic perspective, it could be envisioned that immune modulation drugs such as cytokine inhibitors, co-stimulation blockers or anti-T cell agents could offer benefit. It is questionable, however, whether chronic treatment with for instance biologicals will have a favorable risk/benefit profile in a silent condition such as the metabolic syndrome. An attractive alternative could be the development of antigen-specific T cell therapies, not unlike those currently in various phases of development for type 1 diabetes. In this article, we will give an overview of antigen-specific treatment modalities in type 1 diabetes, followed by a review of the evidence for T cell involvement in obesity and atherosclerosis. PMID:24749057

Coppieters, Ken T.; von Herrath, Matthias G.

2014-01-01

58

Morquio A syndrome: diagnosis and current and future therapies.  

PubMed

Morquio A syndrome is an autosomal recessive disorder, one of 50 lysosomal storage diseases (LSDs), and is caused by the deficiency of N-acetylgalactosamine-6-sulfate sulfatase (GALNS). Deficiency of this enzyme causes specific glycosaminoglycan (GAG) accumulation: keratan sulfate (KS) and chondroitin-6-sulfate (C6S). The majority of KS is produced in the cartilage, therefore, the undegraded substrates accumulate mainly in cartilage and in its extracelluar matrix (ECM), causing direct leads to direct impact on cartilage and bone development and leading to the resultant systemic skeletal spondyloepiphyseal dysplasia. Chondrogenesis ,the earliest phase of skeletal formation that leads to cartilage and bone formation is controlled by cellular interactions with the ECM, growth and differentiation factors and other molecules that affect signaling pathways and transcription factors in a temporal-spatial manner. In Morquio A patients, in early childhood or even at birth, the cartilage is disrupted presumably as a result of abnormal chondrogenesis and/ or endochondral ossification. The unique clinical features are characterized by a marked short stature, odontoid hypoplasia, protrusion of the chest, kyphoscoliosis, platyspondyly, coxa valga, abnormal gait, and laxity of joints. In spite of many descriptions of the unique clinical manifestations, diagnosis delay still occurs. The pathogenesis of systemic skeletal dysplasia in Morquio A syndrome remains an enigmatic challenge. In this review article, screening, diagnosis, pathogenesis and current and future therapies of Morquio A are discussed. PMID:25345096

Tomatsu, Shunji; Yasuda, Eriko; Patel, Pravin; Ruhnke, Kristen; Shimada, Tsutomu; Mackenzie, William G; Mason, Robert; Thacker, Mihir M; Theroux, Mary; Montańo, Adriana M; Alméciga-Díaz, Carlos J; Barrera, Luis A; Chinen, Yasutsugu; Sly, William S; Rowan, Daniel; Suzuki, Yasuyuki; Orii, Tado

2014-09-01

59

Morquio A Syndrome: Diagnosis and Current and Future Therapies  

PubMed Central

Morquio A syndrome is an autosomal recessive disorder, one of 50 lysosomal storage diseases (LSDs), and is caused by the deficiency of N-acetylgalactosamine-6-sulfate sulfatase (GALNS). Deficiency of this enzyme causes specific glycosaminoglycan (GAG) accumulation: keratan sulfate (KS) and chondroitin-6-sulfate (C6S). The majority of KS is produced in the cartilage, therefore, the undegraded substrates accumulate mainly in cartilage and in its extracelluar matrix (ECM), causing direct leads to direct impact on cartilage and bone development and leading to the resultant systemic skeletal spondyloepiphyseal dysplasia. Chondrogenesis, the earliest phase of skeletal formation that leads to cartilage and bone formation is controlled by cellular interactions with the ECM, growth and differentiation factors and other molecules that affect signaling pathways and transcription factors in a temporal-spatial manner. In Morquio A patients, in early childhood or even at birth, the cartilage is disrupted presumably as a result of abnormal chondrogenesis and/or endochondral ossification. The unique clinical features are characterized by a marked short stature, odontoid hypoplasia, protrusion of the chest, kyphoscoliosis, platyspondyly, coxa valga, abnormal gait, and laxity of joints. In spite of many descriptions of the unique clinical manifestations, diagnosis delay still occurs. The pathogenesis of systemic skeletal dysplasia in Morquio A syndrome remains an enigmatic challenge. In this review article, screening, diagnosis, pathogenesis and current and future therapies of Morquio A are discussed. PMID:25345096

Tomatsu, Shunji; Yasuda, Eriko; Patel, Pravin; Ruhnke, Kristen; Shimada, Tsutomu; Mackenzie, William G.; Mason, Robert; Thacker, Mihir M.; Theroux, Mary; Montańo, Adriana M.; Alméciga-Díaz, Carlos J.; Barrera, Luis A.; Chinen, Yasutsugu; Sly, William S.; Rowan, Daniel; Suzuki, Yasuyuki; Orii, Tadao

2014-01-01

60

[Burning mouth syndrome--etiology, differentialdiagnostical aspects and therapy].  

PubMed

Burning Mouth Syndrome (BMS) or glossodynia is a chronic pain syndrome that mainly affects middle-aged/elderly women. This condition is probably of multifactorial origin, often idiopathic, and its etiopathogenesis remains largely enigmatic. Visible pathologic lesions of the oral mucosa or processes are usually not evident. Etiologic factors that have been reported include several disorders (e. g. hematologic disorders, denture factors, the climacteric, infections, endocrinological, neurological or psychiatric disorders). On the other hand psychological factors, such as anxiety, depression and phobias have been reported to play a significant role at the beginning of this nosological entity. BMS related to nutritional deficiency is uncommon. A systemic approach to assessment is essential and the opinion of other specialities, particularly internal medicine, dentistry or neurology, may be of help. Treatment is of the underlying cause but in many cases no specific aetiological factor is identified. Non-indicated substitution therapy of suspected but unproved deficiency states must be avoided. Treatment approaches were divided into topical and systemic strategies. However, there is little research evidence that provides clear guidance for those treating patients with BMS. PMID:15195716

Reiss, M; Reiss, G

2004-05-01

61

Metabolic Syndromes Associated with HIV: Mitigating the Side Effects of Drug Therapy.  

ERIC Educational Resources Information Center

HIV infection and highly active antiretroviral therapy (HAART) are associated with such metabolic disorders as AIDS wasting syndrome, metabolic dysregulation, and abnormalities of serum lipids. Adjunctive therapies (e.g., diet and antilipid therapy); risk factor modification (e.g., smoking cessation and blood pressure control); aerobic exercise;…

Stringer, William W.; Sattler, Fred R.

2001-01-01

62

Differentiation Therapy With Decitabine in Treating Patients With Myelodysplastic Syndrome  

ClinicalTrials.gov

Chronic Myelomonocytic Leukemia; de Novo Myelodysplastic Syndromes; Myelodysplastic Syndromes; Refractory Anemia; Refractory Anemia With Excess Blasts; Refractory Anemia With Ringed Sideroblasts; Refractory Cytopenia With Multilineage Dysplasia; Thrombocytopenia

2013-02-25

63

A meta-analysis of behavior therapy for Tourette Syndrome.  

PubMed

Individual randomized controlled trials (RCTs) of habit reversal training and a Comprehensive Behavioral Intervention for Tics (collectively referred to as behavior therapy, BT) have demonstrated efficacy in reducing tic severity for individuals with Tourette Syndrome and Chronic Tic Disorders (collectively referred to as TS), with no examination of treatment moderators. The present meta-analysis synthesized the treatment effect sizes (ES) of BT relative to comparison conditions, and examined moderators of treatment. A comprehensive literature search identified eight RCTs that met inclusion criteria, and produced a total sample of 438 participants. A random effects meta-analysis found a medium to large ES for BT relative to comparison conditions. Participant mean age, average number of therapy sessions, and the percentage of participants with co-occurring attention deficit hyperactivity disorder (ADHD) were found to moderate treatment effects. Participants receiving BT were more likely to exhibit a treatment response compared to control interventions, and identified a number needed to treat (NNT) of three. Sensitivity analyses failed to identify publication bias. Overall, BT trials yield medium to large effects for TS that are comparable to treatment effects identified by meta-analyses of antipsychotic medication RCTs. Larger treatment effects may be observed among BT trials with older participants, more therapeutic contact, and less co-occurring ADHD. PMID:24398255

McGuire, Joseph F; Piacentini, John; Brennan, Erin A; Lewin, Adam B; Murphy, Tanya K; Small, Brent J; Storch, Eric A

2014-03-01

64

Children with Down syndrome improved in motor functioning and muscle tone following massage therapy  

Microsoft Academic Search

Twenty?one moderate to high functioning young children (mean age, two years) with Down syndrome receiving early intervention (physical therapy, occupational therapy and speech therapy) were randomly assigned to additionally receive two 0.5?hour massage therapy or reading sessions (control group) per week for two months. On the first and last day of the study, the children’s functioning levels were assessed using

Tiffany Field; Shay Largie; Dana Mora; Joan Bornstein; Ronnie Waldman

2006-01-01

65

Positive Airway Pressure Therapy for Obstructive Sleep Apnea\\/Hypopnea Syndrome  

Microsoft Academic Search

Continuous positive airway therapy (CPAP) remains the mainstay of therapy for obstructive sleep apnea syndrome (OSAS). CPAP\\u000a therapy has been demonstrated to resolve sleep-disordered breathing events and improve several clinical outcomes. The first\\u000a part of this review will concentrate on conventional fixed-pressure CPAP therapy with the second portion of this chapter focussing\\u000a on newer technological advancements in the delivery of

Neil S. Freedman

66

Photodynamic therapy of subfoveal choroidal neovascularization with verteporfin in the ocular histoplasmosis syndrome  

Microsoft Academic Search

ObjectiveTo evaluate the safety and effect on visual acuity of photodynamic therapy with verteporfin (Visudyne, Novartis AG) in patients with subfoveal choroidal neovascularization (CNV) secondary to the ocular histoplasmosis syndrome (OHS).

David A Saperstein; Philip J Rosenfeld; Neil M Bressler; Robert H Rosa; Michel Sickenberg; Paul Sternberg; Thomas M Aaberg; Troy A Reaves

2002-01-01

67

Down's syndrome-associated Single Minded 2 gene as a pancreatic cancer drug therapy target  

Microsoft Academic Search

We report here a pancreatic cancer drug therapy utility of a gene involved in Down's syndrome. Single Minded 2 gene (SIM2) from Down's Syndrome Critical Region was expressed in pancreatic cancer-derived cell lines as well as in tumor tissues, but not in the normal pancreas. A related member of the SIM family, SIM1, did not show similar specificity. Inhibition by

Maurice Phil DeYoung; Matthew Tress; Ramaswamy Narayanan

2003-01-01

68

Studies on the interrelation between Zollinger-Ellison syndrome, Helicobacter pylori, and proton pump inhibitor therapy  

Microsoft Academic Search

BACKGROUND & AIMS: The interrelation between Helicobacter pylori infection and proton pump inhibitor therapy in patients with Zollinger- Ellison syndrome is unknown. The aim of this study was to evaluate the influence of these factors on parameters of Zollinger-Ellison syndrome. METHODS: Prevalence of H. pylori was determined by biopsy and antibody testing in 84 patients. The influence of H. pylori

HC Weber; DJ Venzon; RT Jensen; DC Metz

1997-01-01

69

Animal Models of Alport Syndrome: Advancing the Prospects for Effective Human Gene Therapy  

Microsoft Academic Search

Several animal models for Alport syndrome have been described. These are available for studies on the pathogenetic mechanisms of the disease, as well as for the development of new technologies for gene therapy in this progressive hereditary kidney disease. This review summarizes current knowledge on the molecular basis of Alport syndrome, and on the animal models which all remarkably well

Pirkko Heikkilä; Karl Tryggvason; Paul Thorner

2000-01-01

70

Cognitive behavior therapy for night eating syndrome: a pilot study.  

PubMed

Because no studies of psychotherapy treatments for night eating syndrome (NES) have been published, we conducted a pilot study of a 10-session cognitive behavior therapy (CBT) for NES. Twenty-five patients (19 female, 6 male) were screened and comprehensively assessed before being enrolled. At each visit, patients completed the Night Eating Symptom Scale (NESS), were weighed, and number of awakenings and the number of nocturnal ingestions and daily caloric intake were calculated from weekly food and sleep records. Mixed model regression analyses [of the data] showed significant decreases in caloric intake after dinner (35.0% to 24.9%); number of nocturnal ingestions (8.7 to 2.6 per week); weight (82.5 to 79.4 kg); and NESS score (28.7 to 16.3; all p values <0.0001). Number of awakenings per week, depressed mood, and quality of life also improved significantly (p values <.02). This first clinical trial of CBT for NES shows significant improvements in the core aspects of NES and weight reduction, suggesting the need for a controlled treatment trial. PMID:20405767

Allison, Kelly C; Lundgren, Jennifer D; Moore, Reneé H; O'Reardon, John P; Stunkard, Albert J

2010-01-01

71

Intravenous Ulinastatin Therapy for Stevens-Johnson Syndrome and Toxic Epidermal Necrolysis in Pediatric Patients  

Microsoft Academic Search

Background: More effective therapy is needed for the treatment of Stevens-Johnson syndrome (SJS) and toxic epidermal necrolysis (TEN). The clinical efficacy of intravenous ulinastatin therapy was investigated in 3 Japanese pediatric patients with SJS or TEN. Methods: Ulinastatin was given to 1 pediatric SJS patient and 2 pediatric TEN patients within 7 days (patient 1; SJS), 6 days (patient 2;

Yasuji Inamo; Takahiro Okubo; Mika Wada; Sachiko Fuchigami; Kouji Hashimoto; Tatsuo Fuchigami; Shigeru Takahashi; Shigemasa Sawada; Kensuke Harada

2002-01-01

72

SMART syndrome (stroke-like migraine attacks after radiation therapy) in adult and pediatric patients.  

PubMed

SMART syndrome (stroke-like migraine attacks after radiation therapy) is a rare condition that involves complex migraines with focal neurologic findings in patients following cranial irradiation for central nervous system malignancies. Little is known about the mechanisms behind the disorder, making successful treatment challenging. We report 2 new cases of SMART syndrome in pediatric patients as well as review all documented cases of the syndrome. Each of our 2 pediatric patients suffered multiple episodes. Attacks were characterized by severe headache, visual disturbance, aphasia, and weakness. Recovery occurred over several days to weeks. The data from all documented reports of SMART syndrome indicate a greater prevalence for male gender. An age-dependent pattern of onset was also observed, with a greater variability of syndrome onset in patients who received cranial irradiation at a younger age. SMART appears to be a reversible, recurrent long-term complication of radiation therapy with possible age- and gender-related influences. PMID:23364656

Armstrong, Amy E; Gillan, Eileen; DiMario, Francis Joseph

2014-03-01

73

Immunonutritional effects during synbiotics therapy in pediatric patients with short bowel syndrome  

Microsoft Academic Search

The aim of this study was to evaluate the effects of synbiotic therapy in patients with short bowel syndrome (SBS). Four pediatric\\u000a patients with SBS, who were receiving synbiotics therapy including Bifidobacterium breve, Lactobacillus casei and galactooligosaccharides, were enrolled in this study. We evaluated changes in immunonutritional parameters before and\\u000a after receiving synbiotics therapy. Four normal, healthy, age-matched children were

Keiichi Uchida; Takuya Takahashi; Mikihiro Inoue; Masami Morotomi; Kohei Otake; Makoto Nakazawa; Yoshihide Tsukamoto; Chikao Miki; Masato Kusunoki

2007-01-01

74

Minimally Invasive Therapies for Chronic Pelvic Pain Syndrome  

Microsoft Academic Search

Chronic pelvic pain syndrome (CPPS) is a common problem among men and women worldwide. It is a symptoms-complex term for interstitial\\u000a cystitis\\/painful bladder syndrome in women and chronic prostatitis\\/chronic pelvic pain syndrome in men. Patients often present\\u000a with a combination of lower urinary tract symptoms with pelvic pain and sexual dysfunction. No gold standard exists for diagnosis\\u000a or treatment of

Salim A. Wehbe; Jennifer Y. Fariello; Kristene Whitmore

2010-01-01

75

Myelodysplastic syndrome evolving from aplastic anemia treated with immunosuppressive therapy: efficacy of hematopoietic stem cell transplantation  

PubMed Central

A proportion of patients with aplastic anemia who are treated with immunosuppressive therapy develop clonal hematologic disorders, including post-aplastic anemia myelodysplastic syndrome. Many will proceed to allogeneic hematopoietic stem cell transplantation. We identified 123 patients with post-aplastic anemia myelodysplastic syndrome who from 1991 through 2011 underwent allogeneic hematopoietic stem cell transplantation, and in a matched-pair analysis compared outcome to that in 393 patients with de novo myelodysplastic syndrome. There was no difference in overall survival. There were no significant differences with regard to 5-year probabilities of relapse, non-relapse mortality, relapse-free survival and overall survival; these were 14%, 40%, 46% and 49% for post-aplastic anemia myelodysplastic syndrome, and 20%, 33%, 47% and 49% for de novo myelodysplastic syndrome, respectively. In multivariate analysis, relapse (hazard ratio 0.71; P=0.18), non-relapse mortality (hazard ratio 1.28; P=0.18), relapse-free survival (hazard ratio 0.97; P=0.80) and overall survival (hazard ratio 1.02; P=0.88) of post-aplastic anemia myelodysplastic syndrome were similar to those of patients with de novo myelodysplastic syndrome. Cytogenetic risk was independently associated with overall survival in both groups. Thus, transplant success in patients with post-aplastic anemia myelodysplastic syndrome was similar to that in patients with de novo myelodysplastic syndrome, and cytogenetics was the only significant prognostic factor for post-aplastic anemia myelodysplastic syndrome patients. PMID:25107891

Kim, Sung-Yong; Le Rademacher, Jennifer; Antin, Joseph H.; Anderlini, Paolo; Ayas, Mouhab; Battiwalla, Minoo; Carreras, Jeanette; Kurtzberg, Joanne; Nakamura, Ryotaro; Eapen, Mary; Deeg, H. Joachim

2014-01-01

76

Chronic Fatigue Syndrome and Women: Can Therapy Help?  

ERIC Educational Resources Information Center

Presents current research on chronic fatigue syndrome, which currently afflicts mostly females between ages of 25 and 55. Notes that, because depression is common symptom of chronic fatigue syndrome, mental health practitioners are often involved with victims and must formulate appropriate treatment strategy that considers physiological,…

Burke, Susan G.

1992-01-01

77

Correction of hypokalemia with antialdosterone therapy in Gitelman's syndrome.  

PubMed

Six adult patients (4 females and 2 males, age range 26-57 years) with Gitelman's syndrome (GS) were treated with spironolactone 200-300 mg/day (n = 5) and/or amiloride 10-30 mg/day (n = 3) for 1-18 months. The patients had hypokalemia, hyperreninemia, chloride-resistant metabolic alkalosis, renal hypomagnesemia (n = 5), and hypocalciuria (n = 5). Free water clearance studies during maximal water diuresis and furosemide administration were suggestive of a solute reabsorptive defect beyond the loop of Henle. Antialdosterone therapy induced a significant increase of PK (from 2.6 +/- 0.4 to 3.4 +/- 0.4 mM; p < 0.0001) and a decrease of CK (from 21.4 +/- 13.2 to 10.6 +/- 4.8 ml/min, p < 0.02) and FEK (from 21.0 +/- 13.6 to 13.4 +/- 5.7%; p < 0.03); PMg increased from 1.38 +/- 0.38 to 1.64 +/- 0.21 mg/dl (p < 0.03) with a parallel fall of CMg (from 5.5 +/- 2.3 to 2.9 +/- 1.5 ml/min; p < 0.02) and FEMg (from 5.7 +/- 2.6 to 2.9 +/- 0.6%; p < 0.05); arterial blood pH and HCO3- did not change (P = plasma, C = clearance, FE = fractional excretion). The creatinine clearance fell (from 90.5 +/- 16.8 to 65.8 +/- 20.9 ml/min; p < 0.05), and Prenin rose (from 16.6 +/- 8.9 to 35.3 +/- 25.3 ng/ml/h; p < 0.02, as did Paldo (from 26.1 +/- 12.3 to 109 +/- 82.6 ng/dl; p < 0.01), indicating extracellular fluid volume contraction; however no significant clinical symptoms of hypovolemia ensued.(ABSTRACT TRUNCATED AT 250 WORDS) PMID:8080005

Colussi, G; Rombolŕ, G; De Ferrari, M E; Macaluso, M; Minetti, L

1994-01-01

78

The metabolic syndrome and mind-body therapies: a systematic review.  

PubMed

The metabolic syndrome, affecting a substantial and increasing percentage of the worldwide population, is comprised of a cluster of symptoms associated with increased risk of type 2 diabetes, cardiovascular disease, and other chronic conditions. Mind-body modalities based on Eastern philosophy, such as yoga, tai chi, qigong, and meditation, have become increasingly popular worldwide. These complementary therapies have many reported benefits for improving symptoms and physiological measures associated with the metabolic syndrome. However, clinical trial data concerning the effectiveness of these practices on the syndrome as a whole have not been evaluated using a systematic and synthesizing approach. A systematic review was conducted to critically evaluate the data from clinical trials examining the efficacy of mind-body therapies as supportive care modalities for management of the metabolic syndrome. Three clinical trials addressing the use of mind-body therapies for management of the metabolic syndrome were identified. Findings from the studies reviewed support the potential clinical effectiveness of mind-body practices in improving indices of the metabolic syndrome. PMID:21773016

Anderson, Joel G; Taylor, Ann Gill

2011-01-01

79

Speech & Language Therapy for Children and Adolescents with Down Syndrome  

MedlinePLUS

... and interact with people. Syntax and morphology (including grammar, verb tenses, word roots, suffixes and prefixes) are ... Children with Down syndrome frequently have difficulty with grammar, tenses and word endings and use shorter sentences ...

80

Cotard's syndrome with schizophreniform disorder can be successfully treated with electroconvulsive therapy: case report  

PubMed Central

We report a case of Cotard's syndrome associated with psychotic symptoms. A 27-year-old man was admitted to hospital with the diagnosis of schizophreniform disorder. His presenting symptoms, which had started 1 month before hospital admission, were somatic delusions of gastrointestinal and cardiovascular malfunction and the absence of a stomach, which resulted in a decrease in weight from 75 kg to 63 kg in 1 month. Cranial computed tomographic images showed dilatation of the lateral and third ventricles, whereas magnetic resonance imaging revealed central atrophy and lateral ventricle dilatation. Single- photon emission computed tomography demonstrated left temporal, left frontal and left parietal hypoperfusion. The patient did not respond to antipsychotic therapies, but he was successfully treated with electroconvulsive therapy. This report emphasizes that Cotard's syndrome may be accompanied by lesions of the left hemisphere and that electroconvulsive therapy could be the first-line therapy in such patients with psychotic disorder. PMID:15069468

Caliyurt, Okan; Vardar, Erdal; Tuglu, Cengiz

2004-01-01

81

Scheie syndrome: Enzyme replacement therapy does not prevent progression of cervical myelopathy due to spinal cord compression  

Microsoft Academic Search

Summary  Hurler–Scheie syndrome is caused by alpha-l-iduronidase deficiency. Enzyme replacement therapy (ERT) can improve physical\\u000a capacity and reduces organomegaly. However, the effect on bradytrophic connective tissue is limited. As intravenously administered\\u000a enzyme cannot cross the blood–brain barrier, the therapy of choice for the more severe Hurler syndrome is haematopoietic stem\\u000a cell transplantation (HCT). In the more attenuated Scheie syndrome, neurological impairment

S. Illsinger; T. Lücke; H. Hartmann; E. Mengel; W. Müller-Forell; F. Donnerstag; A. M. Das

82

Investigation of the effect of GaAs laser therapy on cervical myofascial pain syndrome  

Microsoft Academic Search

Low-energy laser therapy has been applied in several rheumatoid and soft tissue disorders with varying rates of success. The objective of our study was to investigate the effect of laser therapy on cervical myofascial pain syndrome with a placebo-controlled double-blind prospective study model. It was performed with a total of 53 patients (35 females and 18 males) with cervical myofascial

Lale Altan; Umit Bingöl; Mehtap Aykaç; Merih Yurtkuran

2005-01-01

83

Hyperbaric oxygen therapy prevents coagulation disorders in an experimental model of multiple organ failure syndrome  

Microsoft Academic Search

Objective  To evaluate the effects of hyperbaric oxygen (HBO) therapy on the coagulation cascade using an experimental model of multiple organ failure syndrome (MOFS).Design  MOFS was induced by zymosan (500?mg\\/kg i.?p.) in rats. HBO therapy (2?ATA) was administered in a cylindrical steel chamber 4 and 11?h after zymosan administration. In a separate set of experiments animals were monitored for 72?h, and systemic toxicity was scored.Intervention  Eighteen

Francesco Imperatore; Salvatore Cuzzocrea; Domenico De Lucia; Marcella Sessa; Barbara Rinaldi; Annalisa Capuano; Giovanni Liguori; Amelia Filippelli; Francesco Rossi

2006-01-01

84

Impact of Enzyme Replacement Therapy and Hematopoietic Stem Cell Therapy on Growth in Patients with Hunter Syndrome  

PubMed Central

Patients with Hunter syndrome (mucopolysaccharidosis II) present with skeletal dysplasia including short stature as well as CNS and visceral organ involvement. A previous study on Hunter syndrome indicated an impact on brain and heart involvement after hematopoietic stem cell therapy (HSCT) at an early stage but little impact after enzyme replacement therapy (ERT) (Tanaka et al 2012). Meanwhile, impact on growth in patients with Hunter syndrome treated with ERT and HSCT has not been compared until now. We recently developed baseline growth charts for untreated patients with Hunter syndrome to evaluate the natural history of growth of these patients compared to unaffected controls (Patel et al, 2014). To assess impact of ERT and HSCT on growth, clinical data were obtained from 44 Japanese male patients with MPS II; 26 patients had been treated with ERT, 12 patients had been treated with HSCT, and 6 had been treated with both ERT and HSCT. Height and weight were compared to untreated patients and unaffected controls from the previous study. We demonstrated 1) that MPS II patients, who had been treated with either ERT or HSCT, had increased height and weight when compared to untreated patients, and 2) that HSCT and ERT were equally effective in restoring growth of MPS II patients. In conclusion, HSCT should be considered as one of the primary therapeutic options for early stage treatment of MPS II, as HSCT has also been reported to have a positive effect on brain and heart valve development (Tanaka et al 2012). PMID:25061571

Patel, Pravin; Suzuki, Yasuyuki; Tanaka, Akemi; Yabe, Hiromasa; Kato, Shunichi; Shimada, Tsutomu; Mason, Robert W.; Orii, Kenji E.; Fukao, Toshiyuki; Orii, Tadao; Tomatsu, Shunji

2014-01-01

85

Prospects for Gene Therapy in the Fragile X Syndrome  

ERIC Educational Resources Information Center

Gene therapy is unarguably the definitive way to treat, and possibly cure, genetic diseases. A straightforward concept in theory, in practice it has proven difficult to realize, even when directed to easily accessed somatic cell systems. Gene therapy for diseases in which the central nervous system (CNS) is the target organ presents even greater…

Rattazzi, Mario C.; LaFauci, Giuseppe; Brown, W. Ted

2004-01-01

86

Effects of waon therapy on chronic fatigue syndrome: a pilot study.  

PubMed

Objective Chronic fatigue syndrome (CFS) is a disabling condition of unknown etiology, and no definitive therapy has been identified to date. We developed Waon therapy, a form of thermal therapy using a far-infrared dry sauna, and in this study herein examined its feasibility and safety in patients with CFS. Methods Ten consecutive inpatients with CFS stayed in a 60°C sauna for 15 minutes and then rested on a bed under a blanket for an additional 30 minutes outside the sauna room. The treatments were performed once a day, five days a week for four weeks. Perceived fatigue, the primary outcome measure, was evaluated using a numerical rating scale before, during (two weeks after the commencement of therapy) and after therapy. The pain level, evaluated using a numerical rating scale, mood, assessed using the Profile of Mood States questionnaire, and performance status, assessed using a scale developed for CFS patients were also examined before and after therapy. Results Perceived fatigue significantly decreased after therapy, although no significant reductions were observed during therapy. In addition, a negative mood, including anxiety, depression and fatigue, and the performance status significantly improved after therapy. However, the levels of pain and vigor did not change significantly. No patients reported any adverse effects during the therapy. Conclusion These findings suggest that Waon therapy may be a useful and safe treatment for CFS. PMID:25748743

Soejima, Yuji; Munemoto, Takao; Masuda, Akinori; Uwatoko, Yuuki; Miyata, Masaaki; Tei, Chuwa

2015-01-01

87

Remission of refractory minimal change nephrotic syndrome after basiliximab therapy  

Microsoft Academic Search

Minimal change nephrotic syndrome has been proposed to be a disorder of T cell dysfunction. It is hypothesized that a circulating\\u000a factor(s) from activated T cells might alter glomerular permeability to protein. Some studies have provided evidence that\\u000a up-regulation of interleukin-2 may be involved, not only in the pathophysiology of minimal change nephrotic syndrome, but\\u000a also in steroid resistance. Basiliximab, an anti-interleukin-2

Sung-Shin Park; Won-Ho Hahn; Sung-Do Kim; Byoung-Soo Cho

2009-01-01

88

Oral carnitine therapy in children with cystinosis and renal Fanconi syndrome  

SciTech Connect

11 children with either cystinosis or Lowe's syndrome had a reduced content of plasma and muscle carnitine due to renal Fanconi syndrome. After treatment with oral L-carnitine, 100 mg/kg per d divided every 6 h, plasma carnitine concentrations became normal in all subjects within 2 d. Initial plasma free fatty acid concentrations, inversely related to free carnitine concentrations, were reduced after 7-20 mo of carnitine therapy. Muscle lipid accumulation, which varied directly with duration of carnitine deficiency (r = 0.73), improved significantly in three of seven rebiopsied patients after carnitine therapy. One Lowe's syndrome patient achieved a normal muscle carnitine level after therapy. Muscle carnitine levels remained low in all cystinosis patients, even though cystinotic muscle cells in culture took up L-(/sup 3/H)carnitine normally. The half-life of plasma carnitine for cystinotic children given a single oral dose approximated 6.3 h; 14% of ingested L-carnitine was excreted within 24 h. Studies in a uremic patient with cystinosis showed that her plasma carnitine was in equilibrium with some larger compartment and may have been maintained by release of carnitine from the muscle during dialysis. Because oral L-carnitine corrects plasma carnitine deficiency, lowers plasma free fatty acid concentrations, and reverses muscle lipid accumulation in some patients, its use as therapy in renal Fanconi syndrome should be considered. However, its efficacy in restoring muscle carnitine to normal, and the optimal dosage regimen, have yet to be determined.

Gahl, W.A.; Bernardini, I.; Dalakas, M.; Rizzo, W.B.; Harper, G.S.; Hoeg, J.M.; Hurko, O.; Bernar, J.

1988-02-01

89

Role of Treadmill Training Versus Suspension Therapy On Balance In Children With Down Syndrome  

Microsoft Academic Search

Background and PurposeMaintaining balance is a subordinate but necessary requirement for most human actions. Most Down syndrome (DS) children, who constitute a large portion in our country, continue to evidence deficits in balance, co-ordination, and gait throughout childhood and adulthood. So, it is essential to seek an ideal physical therapy program to help in solving such widespread problem. The present

Gehan H. Elmeniway; Hebatallah M. Kamal; Samah A. Elshemy

90

Music Therapy for Children with Down Syndrome: Perceptions of Caregivers in a Special School Setting  

ERIC Educational Resources Information Center

Down syndrome (DS) is a genetic disorder resulting from chromosome 21 having three copies (trisomy 21). Cognitive functioning and anatomical features cause speech and language development delay (Kumin, 2003). Children with DS generally enjoy communication (Schoenbrodt, 2004), and respond well to interaction and social scripts. Music therapy has…

Pienaar, Dorothea

2012-01-01

91

Primary therapy for small cell lung cancer reversing the Eaton-Lambert syndrome  

SciTech Connect

A case report is presented of a patient with small cell carcinoma of the lung associated with the classic Eaton-Lambert syndrome. He received intermittent anticholinesterase therapy, with minimal improvement. Combined radiotherapy and chemotherapy for the primary neoplasm produced considerable improvement, with normal EMG findings after complete remission was achieved. 7 references, 1 table.

Kalter, S.; Dhingra, H.M.; Farha, P.

1985-02-01

92

Successful Treatment of Olfactory Reference Syndrome with Cognitive Behavioral Therapy: A Case Study  

ERIC Educational Resources Information Center

Olfactory reference syndrome (ORS) is characterized by a preoccupation with the belief that one's body emits a foul odor. Cognitive behavioral therapy (CBT) was used to treat a woman in her 50s who presented in our outpatient anxiety disorders specialty clinic with ORS, accompanied by embarrassment, shame, distress, avoidance behavior, and social…

Martin-Pichora, Andrea L.; Antony, Martin M.

2011-01-01

93

Efficacy of low level laser therapy in myofascial pain syndrome: An algometric and thermographic evaluation  

Microsoft Academic Search

Background and objectives: The efficacy of low level laser therapy (LLLT) in myofascial pain syndrome (MPS) seems controversial. Our aim was to clarify the effect of LLLT in MPS by using algometry and thermography. Study Design\\/Materials and Methods: Sixty-two patients with MPS having an active trigger point in the neck or upper back region were randomly divided into two equal

Murat Birtane; Fatma Nesrin Turan

2003-01-01

94

Effect of low level laser therapy in rheumatoid arthritis patients with carpal tunnel syndrome  

Microsoft Academic Search

Objective: the aim of the present study was to evaluate the efficacy of low level laser therapy (LLLT) in patients with rheumatoid arthritis (RA) with carpal tunnel syndrome (CTS). Material and methods: a total of 19 patients with the diagnosis of CTS in 19 hands were included and randomly assigned to two treatment groups; LLLT (Group 1) (10 hands) with

Cengiz Onera; Meric Colak

95

Preliminary experience with dance movement therapy in patients with chronic fatigue syndrome  

Microsoft Academic Search

To evaluate the influence of dance movement therapy (DMT) on the perception of well-being and functional capacity in women with chronic fatigue syndrome (CFS). Previous studies have analysed the effects of DMT in fibromyalgia but not specifically in CFS. Seven women diagnosed with CFS attended a 4-month DMT program. All of them performed a maximal physical test before and after

A. Blázquez; E. Guillamó; C. Javierre

2010-01-01

96

Haematopoietic cell transplantation (HCT) in combination with enzyme replacement therapy (ERT) in patients with Hurler syndrome  

Microsoft Academic Search

Hurler syndrome (MPS 1H) is the severe form of mucopolysaccharidosis type 1 (MPS 1). Haematopoietic cell transplantation (HCT) is the treatment of choice, but carries a high incidence of graft failure and morbidity. The use of enzyme replacement therapy (ERT) might improve the clinical signs and symptoms before HCT, resulting in less transplantation-related complications. Moreover, clearance of glycosaminoglycans (GAG's) from

J Cox-Brinkman; J. J. Boelens; J. E. Wraith; A. O'Meara; P. Veys; F. A. Wijburg; N. Wulffraat; R. F. Wynn

2006-01-01

97

Cognitive Behavior Therapy for Relatively Active and for Passive Chronic Fatigue Syndrome Patients  

ERIC Educational Resources Information Center

In chronic fatigue syndrome (CFS), facilitating, initiating, and perpetuating factors are distinguished. Although somatic factors might have initiated symptoms in CFS, they do not explain the persistence of fatigue. Cognitive behavior therapy (CBT) for CFS focuses on factors that perpetuate and prolong symptoms. Recently it has been shown that,…

Bazelmans, Ellen; Prins, Judith; Bleijenberg, Gijs

2006-01-01

98

Implementing Cognitive Behavioral Therapy for Chronic Fatigue Syndrome in a Mental Health Center: A Benchmarking Evaluation  

ERIC Educational Resources Information Center

Objective: This study evaluated the success of implementing cognitive behavioral therapy (CBT) for chronic fatigue syndrome (CFS) in a representative clinical practice setting and compared the patient outcomes with those of previously published randomized controlled trials (RCTs) of CBT for CFS. Method: The implementation interventions were the…

Scheeres, Korine; Wensing, Michel; Knoop, Hans; Bleijenberg, Gijs

2008-01-01

99

Is a Full Recovery Possible after Cognitive Behavioural Therapy for Chronic Fatigue Syndrome?  

Microsoft Academic Search

Background: Cognitive behavioural therapy (CBT) for chronic fatigue syndrome (CFS) leads to a decrease in symptoms and disabilities. There is controversy about the nature of the change following treatment; some suggest that patients improve by learning to adapt to a chronic condition, others think that recovery is possible. The objective of this study was to find out whether recovery from

Hans Knoop; Gijs Bleijenberg; Marieke F. M. Gielissen; Peter D. White

2007-01-01

100

Increase in prefrontal cortical volume following cognitive behavioural therapy in patients with chronic fatigue syndrome  

Microsoft Academic Search

Chronic fatigue syndrome (CFS) is a disabling disorder, characterized by persistent or relapsing fatigue. Recent studies have detected a decrease in cortical grey matter volume in patients with CFS, but it is unclear whether this cerebral atrophy constitutes a cause or a consequence of the disease. Cognitive behavioural therapy (CBT) is an effective behavioural intervention for CFS, which combines a

Floris P. de Lange; Anda Koers; Joke S. Kalkman; Gijs Bleijenberg; Peter Hagoort; Ivan Toni

2008-01-01

101

Increase in Prefrontal Cortical Volume following Cognitive Behavioural Therapy in Patients with Chronic Fatigue Syndrome  

ERIC Educational Resources Information Center

Chronic fatigue syndrome (CFS) is a disabling disorder, characterized by persistent or relapsing fatigue. Recent studies have detected a decrease in cortical grey matter volume in patients with CFS, but it is unclear whether this cerebral atrophy constitutes a cause or a consequence of the disease. Cognitive behavioural therapy (CBT) is an…

de Lange, Floris P.; Koers, Anda; Kalkman, Joke S.; Bleijenberg, Gijs; Hagoort, Peter; van der Meer, Jos W. M.; Toni, Ivan

2008-01-01

102

Intravenous immunoglobulin replacement therapy to prevent pulmonary infection in a patient with Good's syndrome.  

PubMed

Good's syndrome is an acquired immunodeficiency state associated with thymoma and characterized by recurrent pulmonary infections. We describe a 67-year-old woman who presented with respiratory symptoms caused by concomitant disseminated cytomegalovirus infection and Pneumocystis jiroveci pneumonia 38 months after thymectomy for a thymoma. Immunologic analysis revealed hypogammaglobulinemia with absent B-cell population as demonstrated by flow cytometry, consistent with Good's syndrome. Following treatment with sulfamethoxazole/trimethoprim and ganciclovir, the patient improved with resolution of her respiratory symptoms. However, the patient subsequently experienced additional infections, necessitating additional subsequent hospital admissions. During the last admission, intravenous immunoglobulin (IVIG) replacement therapy was initiated and continued after discharge. Infection has been prevented for one year after beginning IVIG replacement therapy. This case reveals that in patients with combined humoral and cell-mediated immune deficiency, concomitant infection with different pathogens is not unusual, and immediate specific therapy is important. Periodic IVIG infusion, to maintain adequate Ig levels, is recommended. PMID:23200552

Wang, Ching-Hsun; Chan, Edward D; Perng, Cherng-Lih; Chian, Chih-Feng; Chen, Chien-Wen; Perng, Wann-Cherng; Su, Wen-Lin

2015-04-01

103

Atypical cytogenetics in therapy-related myelodysplastic syndrome secondary to indolent B-cell lymphoma.  

PubMed

A case of therapy-related myelodysplastic syndrome (t-MDS) with unusual cytogenetics is presented. While therapy related myeloid neoplasms account for 10-20% of all myeloid neoplasms, 90% of therapy related myelodysplastic syndromes (MDS) present with a del(7q) or a del(5q) and fewer than 1% present with a del(20q). In this case, the common cytogenetic pattern of either del(7q) or del(5q) is absent while presenting with an abnormal del(20q). Also demonstrated is the potently poor prognostic indicator of cytomegalovirus (CMV) seropositivity, even when seropositivity is matched between donor and recipient of hematopoietic stem-cell transplant. The patient also continues to demonstrate the inherent dangers of a stem-cell transplant, presenting with graft-versus-host disease (GvHD) while being a haploidentical 10 out of 10 HLA match to the allogeneic stem cell donor. PMID:21905579

Sinsabaugh, Christopher

2011-01-01

104

Menopause, the metabolic syndrome, and mind-body therapies.  

PubMed

Cardiovascular disease risk rises sharply with menopause, likely due to the coincident increase in insulin resistance and related atherogenic changes that together comprise the metabolic or insulin resistance syndrome, a cluster of metabolic and hemodynamic abnormalities strongly implicated in the pathogenesis and progression of cardiovascular disease. A growing body of research suggests that traditional mind-body practices such as yoga, tai chi, and qigong may offer safe and cost-effective strategies for reducing insulin resistance syndrome-related risk factors for cardiovascular disease in older populations, including postmenopausal women. Current evidence suggests that these practices may reduce insulin resistance and related physiological risk factors for cardiovascular disease; improve mood, well-being, and sleep; decrease sympathetic activation; and enhance cardiovagal function. However, additional rigorous studies are needed to confirm existing findings and to examine long-term effects on cardiovascular health. PMID:18779682

Innes, Kim E; Selfe, Terry Kit; Taylor, Ann Gill

2008-01-01

105

Menopause, the metabolic syndrome, and mind-body therapies  

PubMed Central

Cardiovascular disease risk rises sharply with menopause, likely due to the coincident increase in insulin resistance and related atherogenic changes that together comprise the metabolic or insulin resistance syndrome, a cluster of metabolic and hemodynamic abnormalities strongly implicated in the pathogenesis and progression of cardiovascular disease. A growing body of research suggests that traditional mind-body practices such as yoga, tai chi, and qigong may offer safe and cost-effective strategies for reducing insulin resistance syndrome-related risk factors for cardiovascular disease in older populations, including postmenopausal women. Current evidence suggests that these practices may reduce insulin resistance and related physiological risk factors for cardiovascular disease; improve mood, well-being, and sleep; decrease sympathetic activation; and enhance cardiovagal function. However, additional rigorous studies are needed to confirm existing findings and to examine long-term effects on cardiovascular health. PMID:18779682

Innes, Kim E.; Selfe, Terry Kit; Taylor, Ann Gill

2009-01-01

106

Thalidomide therapy for myelodysplastic syndromes: current status and future perspectives  

Microsoft Academic Search

Thalidomide exerts in vitro heterogeneous biological effects on hematopoiesis which have supported its possible use in treating myelodysplastic syndromes (MDS). Some recent clinical trials have confirmed that thalidomide may improve anemia and, less frequently, other cytopenias, in a proportion of younger patients with low-risk MDS (11–56%, on intention-to-treat analysis). Of interest, erythroid responses may be achieved also in transfusion-dependent subjects

Pellegrino Musto

2004-01-01

107

Fatty Acids in the Causation and Therapy of Metabolic Syndrome  

Microsoft Academic Search

The role of fatty acids in the prevention and pathogenesis of metabolic syndrome leading to cardiovascular diseases, type\\u000a 2 diabetes and insulin resistance are reviewed. We did Medline, PubMed search till March, 2007. Excess of linoleic acid, trans\\u000a fatty acids (TFA), saturated and total fat as well as refined starches and sugar are proinflammatory. Low dietary monounsaturated\\u000a fatty acids (MUFA)

Ram B. Singh; Fabien De Meester; Viola Mechirova; Daniel Pella; Kuniaki Otsuka

108

[Drug therapy in complex regional pain syndrome type I].  

PubMed

Pharmacologic treatment options for complex regional pain syndrome (CRPS) type I are discussed and drug effectiveness is graded according to the level of evidence available. Various drug strategies in the treatment of CRPS type I, i.e. NSAIDs, corticosteroids, free radical scavengers, antidepressants, anticonvulsants, local anesthetics, opioid analgesics, clonidine, capsaicin, NMDA receptor antagonists, calcitonin, bisphosphonates, GABA(B)-agonists, alpha-blockers, IVRA (bretylium/ketanserin), IVRA (clonidine), IVRSB, local anesthetics sympathetic blockade, GLOA and iloprost are discussed. PMID:15150686

Von Eisenhart-Rothe, R; Rittmeister, M

2004-07-01

109

Menstrual Cyclicity After Metformin Therapy in Polycystic Ovary Syndrome  

Microsoft Academic Search

Objective: To assess the effect of insulin-lowering treatment on menstrual cyclicity in polycystic ovary syndrome (PCOS).Methods: Forty oligoamenorrheic women with PCOS were recruited in a prospective clinical study to receive metformin for a minimum period of 6 months. Twenty-two women completed the study. Serum LH, FSH, free testosterone, and glucose and insulin response to oral glucose load were measured both

Elsy Velázquez M; Arnaldo Acosta; Soaira G Mendoza

1997-01-01

110

Pelvic Floor Therapies in Chronic Pelvic Pain Syndrome  

Microsoft Academic Search

Chronic pelvic pain syndrome is a poorly understood clinical entity associated with urinary symptoms, pelvic floor dysfunction,\\u000a and multisystem disorders. Treatment of pelvic floor dysfunction is difficult and often frustrating for the patient as well\\u000a as for the involved physician. The purpose of this review is to update clinicians on the latest research for the treatment\\u000a of pelvic floor dysfunction

Ragi Doggweiler; Adam F. Stewart

2011-01-01

111

Olanzapine therapy in hallucinatory visions related to Bonnet syndrome  

Microsoft Academic Search

Charles Bonnet syndrome (CBS) is characterised\\u000aby the triad of complex visual hallucinations, ocular pathology\\u000acausing visual deterioration and preserved cognitive status.\\u000aWe report a case of a 62–year–old man with a brief history\\u000aof visual hallucinations. The patient complained of amaurosis\\u000awith optic nerve atrophy in his left eye and a severe\\u000aimpairment of visual acuity in the right

M. Coletti Moja; E. Milano; S. Gasverde; M. Gianelli; M. T. Giordana

2005-01-01

112

Gaucher disease with nephrotic syndrome: Response to enzyme replacement therapy  

Microsoft Academic Search

Nephrotic syndrome in patients with Gaucher disease is rare; most of the few reported cases have had a well-defined glomerulopathy often with Gaucher cells in the glomeruli. We report the case of a 54-year-old woman with Gaucher disease, who had splenectomy at age 25, preeclampsia with renal biopsy disclosing only endotheliosis at age 32, and improvement of proteinuria and reappearance

Domenico Santoro; Barry E. Rosenbloom; Arthur H. Cohen

2002-01-01

113

A practical guide to the therapy of narcolepsy and hypersomnia syndromes.  

PubMed

Narcolepsy and other syndromes associated with excessive daytime sleepiness can be challenging to treat. New classifications now distinguish narcolepsy/hypocretin deficiency (also called type 1 narcolepsy), a lifelong disorder with well-established diagnostic procedures and etiology, from other syndromes with hypersomnolence of unknown causes. Klein-Levin Syndrome, a periodic hypersomnia associated with cognitive and behavioral abnormalities, is also considered a separate entity with separate therapeutic protocols. Non hypocretin-related hypersomnia syndromes are diagnoses of exclusion. These diagnoses are only made after eliminating sleep deprivation, sleep apnea, disturbed nocturnal sleep, and psychiatric comorbidities as the primary cause of daytime sleepiness. The treatment of narcolepsy/hypocretin deficiency is well-codified, and involves pharmacotherapies using sodium oxybate, stimulants, and/or antidepressants, plus behavioral modifications. These therapies are almost always needed, and the risk-to-benefit ratio is clear, notably in children. Detailed knowledge of the pharmacological profile of each compound is needed to optimize use. Treatment for other syndromes with hypersomnolence is more challenging and less codified. Preferably, therapy should be conservative (such as modafinil, atomoxetine, behavioral modifications), but it may have to be more aggressive (high-dose stimulants, sodium oxybate, etc.) on a case-by-case, empirical trial basis. As cause and evolution are unknown in these conditions, it is important to challenge diagnosis and therapy over time, keeping in mind the possibility of tolerance and the development of stimulant addiction. Kleine-Levin Syndrome is usually best left untreated, although lithium can be considered in severe cases with frequent episodes. Guidelines are provided based on the literature and personal experience of the author. PMID:23065655

Mignot, Emmanuel J M

2012-10-01

114

May-Thurner syndrome: case report and review of the literature involving modern endovascular therapy.  

PubMed

May-Thurner syndrome is a rare clinical entity involving venous obstruction of the left lower extremity. Obstruction occurs secondary to compression of the left common iliac vein between the right common iliac artery and the underlying vertebral body. Current management largely involves endovascular therapy. A review was conducted of six studies containing at least five patients with May-Thurner syndrome treated by endovascular therapy. We compiled data on 113 patients, analyzing patient demographics, treatment details, and outcome. An 18-year-old female presented 1 week after the onset of left lower extremity pain and swelling. Duplex ultrasonography revealed extensive left-sided deep venous thrombosis (DVT). Thrombolysis followed by iliac vein stent placement restored patency to the venous system, with subsequent resolution of symptoms. Review of 113 patients revealed that the majority were females (72%) presenting with DVT (77%), most of which was acute in onset (73%). Therapy consisted of catheter-directed thrombolysis and subsequent stent placement in the majority of patients, resulting in a mean technical success of 95% and a mean 1-year patency of 96%. Endovascular therapy is the current mainstay of treatment for May-Thurner syndrome. Review of the current literature supports treatment via catheter-directed thrombolysis followed by stent placement with good early results. PMID:19909680

Moudgill, Neil; Hager, Eric; Gonsalves, Carin; Larson, Robert; Lombardi, Joseph; DiMuzio, Paul

2009-01-01

115

FRAGILE X SYNDROME: PSYCHIATRIC MANIFESTATIONS, ASSESSMENT AND EMERGING THERAPIES  

PubMed Central

Fragile X Syndrome (FXS), the most common inherited cause of intellectual disabilities, is an X-linked dominant disorder caused by the amplification of a CGG repeat in the 5? untranslated region of the fragile X mental retardation gene 1 (FMR1). Prevalence estimates of the disorder are approximately 1/3600. Psychiatric manifestations of the disorder include anxiety, attention deficit hyperactivity disorder, autism, mood instability and aggression. In this article we review the above psychiatric manifestations and challenges to accurate assessment. We also discuss how the neurobiological underpinnings of these symptoms are beginning to be understood and can help guide treatment. PMID:25632275

Wadell, Paula M.; Hagerman, Randi J.; Hessl, David R.

2015-01-01

116

Complement therapy in atypical haemolytic uraemic syndrome (aHUS)  

PubMed Central

Central to the pathogenesis of atypical haemolytic uraemic syndrome (aHUS) is over-activation of the alternative pathway of complement. Inherited defects in complement genes and autoantibodies against complement regulatory proteins have been described. The use of plasma exchange to replace non-functioning complement regulators and hyper-functional complement components in addition to the removal of CFH-autoantibodies made this the ‘gold-standard’ for management of aHUS. In the last 4 years the introduction of the complement inhibitor Eculizumab has revolutionised the management of aHUS. In this review we shall discuss the available literature on treatment strategies to date. PMID:23810412

Wong, Edwin K.S.; Goodship, Tim H.J.; Kavanagh, David

2013-01-01

117

Australian venomous jellyfish, envenomation syndromes, toxins and therapy.  

PubMed

The seas and oceans around Australia harbour numerous venomous jellyfish. Chironex fleckeri, the box jellyfish, is the most lethal causing rapid cardiorespiratory depression and although its venom has been characterised, its toxins remain to be identified. A moderately effective antivenom exists which is also partially effective against another chirodropid, Chiropsalmus sp. Numerous carybdeids, some unidentified, cause less severe illness, including Carybdea rastoni whose toxins CrTX-A and CrTX-B are large proteins. Carukia barnesi, another small carybdeid is one cause of the 'Irukandji' syndrome which includes delayed pain from severe muscle cramping, vomiting, anxiety, restlessness, sweating and prostration, and occasionally severe hypertension and acute cardiac failure. The syndrome is in part caused by release of catecholamines but the cause of heart failure is undefined. The venom contains a sodium channel modulator. Two species of Physalia are present and although one is potentially lethal, has not caused death in Australian waters. Other significant genera of jellyfish include Tamoya, Pelagia, Cyanea, Aurelia and Chyrosaora. PMID:16928389

Tibballs, James

2006-12-01

118

Pain and reduced mobility in complex regional pain syndrome I: outcome of a prospective randomised controlled clinical trial of adjuvant physical therapy versus occupational therapy  

Microsoft Academic Search

There are no adequate comparative studies on physical therapy (PT) versus occupational therapy (OT) in patients with complex regional pain syndrome I (CRPS I). Therefore, we conducted a prospective randomised clinical trial to assess their effectiveness. The outcomes regarding reducing pain and normalising active range of motion (AROM) are discussed. Included in the study were 135 patients who had been

H. Margreet Oerlemans; Rob A. B. Oostendorp; Theo de Boo; R. Jan. A. Goris

1999-01-01

119

Genotype- and phenotype-directed antiplatelet therapy selection in patients with acute coronary syndromes.  

PubMed

Although dual antiplatelet therapy (DAPT) has been a standard treatment in patients with acute coronary syndrome (ACS) for over a decade, only recently have therapeutic options beyond aspirin and clopidogrel become available. Additional treatment options are particularly useful because of the documented history of variability in antiplatelet response. This article reviews the current treatment options for DAPT in ACS, and reviews both genotype- and phenotype-guided methods for determining optimal antiplatelet therapy for patients with ACS. Additionally, recommendations from current guidelines as well as expert commentary are provided for the use of available testing methods to determine optimal DAPT for ACS patients. PMID:25340283

Ismail, Sahar; Lee, Yee Ming; Patel, Meet; Duarte, Julio D; Ardati, Amer K

2014-11-01

120

Effectiveness of Electroconvulsive Therapy for Depression and Cotard's Syndrome in a Patient with Frontotemporal Lobe Dementia  

PubMed Central

In the field of psychogeriatrics, the differential diagnosis of depression and dementia, as well as the treatment of depression and comorbid dementia, is an important issue. In this paper, the authors present the case of a 72-year-old woman with Cotard's syndrome and frontotemporal dementia (FTD) who was admitted to a psychiatric hospital with delusions of negation accompanied by depressive symptoms. Pharmacotherapy over a 2-year hospitalization was unsuccessful, and she was subsequently transferred to our university hospital. A total of 18 sessions of electroconvulsive therapy released her from psychomotor inhibition, appetite loss, and Cotard's delusions. The indication for electroconvulsive therapy in patients with dementia is discussed. PMID:23119214

Kobayashi, Toshiyuki; Inoue, Koju; Shioda, Katsutoshi; Kato, Satoshi

2012-01-01

121

Metformin therapy decreases hyperandrogenism and hyperinsulinemia in women with polycystic ovary syndrome  

Microsoft Academic Search

Objective: To evaluate the effects of 12 weeks of metformin therapy on hormonal and clinical indices in polycystic ovary syndrome (PCOS).Design: Prospective study.Setting: University hospital.Patient(s): Thirty-nine women with PCOS and fasting hyperinsulinemia.Intervention(s): Twelve weeks of therapy with oral metformin (500 mg three times per day).Main Outcome Measure(s): Levels of insulin, T, DHEAS, insulin-like growth factor-I (IGF-I), gonadotropins, and sex hormone-binding

Beata Kolodziejczyk; Antoni J Duleba; Robert Z Spaczynski; Leszek Pawelczyk

2000-01-01

122

Loading dose of clopidogrel in combination with other antithrombotic therapy for capsular warning syndrome.  

PubMed

A 66-year-old man had repeated stereotypical transient ischemic attacks (TIAs) of dysarthria and left hemiparesis. His symptoms were considered capsular warning syndrome (CWS), for which the responsible lesion was found on magnetic resonance imaging to be in the right putamen expanding to the corona radiata. Although administration of antithrombotic therapy including aspirin was ineffective, no further TIAs occurred after a loading dose of clopidogrel was added. A loading dose of clopidogrel combined with other antithrombotic therapy might be an effective treatment for CWS. PMID:24139407

Kawano, Hiroyuki; Nakajima, Makoto; Inatomi, Yuichiro; Yonehara, Toshiro; Ando, Yukio

2014-01-01

123

Endoscopic therapy for esophageal hematoma with blue rubber bleb nevus syndrome  

PubMed Central

A 57-year-old woman previously diagnosed with blue rubber bleb nevus syndrome (BRBNS) reported hematemesis. BRBNS is a rare vascular anomaly syndrome consisting of multifocal hemangiomas of the skin and gastrointestinal (GI) tract but her GI tract had never been examined. An upper gastrointestinal endoscopy revealed a large bleeding esophageal hematoma positioned between the thoracic esophagus and the gastric cardia. An endoscopic injection of polidocanol was used to stop the hematoma from bleeding. The hematoma was incised using the injection needle to reduce the pressure within it. Finally, argon plasma coagulation (APC) was applied to the edge of the incision. The esophageal hematoma disappeared seven days later. Two months after the endoscopic therapy, the esophageal ulcer healed and the hemangioma did not relapse. This rare case of a large esophageal hematoma originating from a hemangioma with BRBNS was treated using a combination of endoscopic therapy with polidocanol injection, incision, and APC. PMID:25512774

Takasumi, Mika; Hikichi, Takuto; Takagi, Tadayuki; Sato, Masaki; Suzuki, Rei; Watanabe, Ko; Nakamura, Jun; Sugimoto, Mitsuru; Waragai, Yuichi; Kikuchi, Hitomi; Konno, Naoki; Watanabe, Hiroshi; Obara, Katsutoshi; Ohira, Hiromasa

2014-01-01

124

The spectrum of monogenic autoinflammatory syndromes: understanding disease mechanisms and use of targeted therapies.  

PubMed

Monogenic autoinflammatory diseases encompass a distinct and growing clinical entity of multisystem inflammatory diseases with known genetic defects in the innate immune system. The diseases present clinically with episodes of seemingly unprovoked inflammation (fever, rashes, and elevation of acute phase reactants). Understanding the genetics has led to discovery of new molecules involved in recognizing exogenous and endogenous danger signals, and the inflammatory response to these stimuli. These advances have furthered understanding of innate inflammatory pathways and spurred collaborative research in rheumatology and infectious diseases. The pivotal roles of interleukin (IL)-1beta in cryopyrin-associated periodic syndromes, tumor necrosis factor (TNF) in TNF receptor-associated periodic syndrome, and links to inflammatory cytokine dysregulation in other monogenic autoinflammatory diseases have resulted in effective therapies targeting proinflammatory cytokines IL-1beta and TNF and uncovered other new potential targets for anti-inflammatory therapies. PMID:18606080

Glaser, Rachel L; Goldbach-Mansky, Raphaela

2008-07-01

125

McKenzie diagnosis and therapy in the evaluation and management of a lumbar disc derangement syndrome: A case study  

Microsoft Academic Search

ObjectiveTo discuss the case of a patient whose lumbar disc derangement syndrome resolved after treatment that included McKenzie diagnosis and therapy, spinal mobilization, and spinal manipulation. Also, to give an overview of the McKenzie method in general, and more specifically for evaluation and management of derangement syndrome.

Steven M Santolin

2003-01-01

126

Development of antiviral therapy for severe acute respiratory syndrome.  

PubMed

A new disease, the severe acute respiratory distress syndrome (SARS), caused by the SARS coronavirus (SARS-CoV), emerged at the beginning of 2003 and rapidly spread throughout the world. Although the disease had disappeared in June 2003 its re-emergence cannot be excluded. The development of vaccines against SARS-CoV may take years. Therefore, the availability of effective antiviral drugs against SARS-CoV may be crucial for the control of future SARS outbreaks. In this review, experimental and clinical data about potential anti-SARS drugs is summarised and discussed. Animal model studies will be needed to help to determine which interventions warrant controlled clinical testing. PMID:15878786

Cinatl, Jindrich; Michaelis, Martin; Hoever, Gerold; Preiser, Wolfgang; Doerr, Hans Wilhelm

2005-06-01

127

Kinesio taping compared to physical therapy modalities for the treatment of shoulder impingement syndrome  

Microsoft Academic Search

The purpose of this study was to determine and compare the efficacy of kinesio tape and physical therapy modalities in patients\\u000a with shoulder impingement syndrome. Patients (n?=?55) were treated with kinesio tape (n?=?30) three times by intervals of 3 days or a daily program of local modalities (n?=?25) for 2 weeks. Response to treatment was evaluated with the Disability of Arm, Shoulder,

Erkan Kaya; Murat Zinnuroglu; Ilknur Tugcu

2011-01-01

128

Cognitive-behavioral therapy for premenstrual syndrome and premenstrual dysphoric disorder: a systematic review  

Microsoft Academic Search

We systematically reviewed empirical studies that investigated the use of cognitive-behavioral therapy (CBT) for premenstrual\\u000a syndrome (PMS) or premenstrual dysphoric disorder (PMDD). Our multi-database search identified seven published empirical reports.\\u000a Three were identified as randomized controlled trials (RCTs). The methods utilized to investigate therapeutic efficacy of\\u000a CBT in these studies varied widely from case reports to RCTs with pharmacotherapy comparison

M. Kathleen B. Lustyk; Winslow G. Gerrish; Shelley Shaver; Shaunie L. Keys

2009-01-01

129

Effectiveness and Limitations of b-Blocker Therapy in Congenital Long-QT Syndrome  

Microsoft Academic Search

Background— b-blockers are routinely prescribed in congenital long-QT syndrome (LQTS), but the effectiveness and limitations of b-blockers in this disorder have not been evaluated. Methods and Results—The study population comprised 869 LQTS patients treated with b-blockers. Effectiveness of b-blockers was analyzed during matched periods before and after starting b-blocker therapy, and by survivorship methods to determine factors associated with cardiac

Arthur J. Moss; Wojciech Zareba; Peter J. Schwartz; Richard S. Crampton; Jesaia Benhorin; G. Michael Vincent; Emanuela H. Locati; Silvia G. Priori; Carlo Napolitano; Aharon Medina; Li Zhang; Jennifer L. Robinson; Katherine W. Timothy; Jeffrey A. Towbin; Mark L. Andrews

2000-01-01

130

New therapies in steroid-sensitive and steroid-resistant idiopathic nephrotic syndrome  

Microsoft Academic Search

Although many children with idiopathic nephrotic syndrome (INS) respond initially to steroid therapy, repeated courses for\\u000a patients with relapses often cause significant steroid toxicity. Patients with frequent relapses who develop steroid dependency\\u000a thus require alternative treatment. The first such options have been considered to be cyclophosphamide or levamisole, although\\u000a the latter is no longer available in many countries. There is

Michael van Husen; Markus J. Kemper

2011-01-01

131

Maintenance therapy with mycophenolate mofetil after rituximab in pediatric patients with steroid-dependent nephrotic syndrome  

Microsoft Academic Search

Rituximab (RTX) has a significant steroid-sparing effect in children with steroid-dependent nephrotic syndrome (SDNS). However,\\u000a patients are likely to relapse with the recovery of CD20+ cells. We conducted a small prospective cohort study with a historical\\u000a control to evaluate the effect of RTX infusion followed by mycophenolate mofetil (MMF) as a maintenance therapy. Nine patients\\u000a with SDNS who stopped their

Shuichi Ito; Koichi Kamei; Masao Ogura; Mai Sato; Takuya Fujimaru; Tomoaki Ishikawa; Tomohiro Udagawa; Kazumoto Iijima

132

Antiplatelet therapy in acute coronary syndromes: focus on ticagrelor  

PubMed Central

The use of antiplatelet agents, specifically the thienopyridines, has become a standard of care in the approach to the patient presenting with an acute coronary syndrome. These drugs irreversibly inhibit the platelet by permanently binding to the surface P2Y12 receptor and blocking the downstream fibrinogen cross-linking between platelets, which leads to aggregation and thrombus. However, currently available therapeutic choices are limited by potential interaction with other medications, slow hepatic conversion to active metabolite, genetic resistance, and narrow therapeutic safety margin. In order to overcome these disadvantages, there has been an interest in developing alternatives to thienopyridines. Recent investigations have included ticagrelor, a reversible inhibitor of the P2Y12 platelet receptor, which appears to have overcome several drawbacks of the current thienopyridines. Its unique pharmacokinetic and pharmacodynamic profiles result in an inhibition of platelet aggregation that is rapid, high, consistent, and less susceptible to interpatient variability than currently available P2Y12 inhibitors. In addition, ticagrelor offers a potential mortality advantage not apparent with current agents. Although questions regarding the nature, magnitude, and clinical significance of several observed adverse effects (dyspnea and ventricular pauses) remain unanswered, it appears that ticagrelor may represent a significant advancement over currently available oral antiplatelet agents. PMID:22282698

Birkeland, Kade; Parra, David; Rosenstein, Robert

2010-01-01

133

Inositol: history of an effective therapy for Polycystic Ovary Syndrome.  

PubMed

Inositol is a physiological compound belonging to the sugar family. The two inositol stereoisomers, myo-inositol and D-chiroinositol are the two main stereisomers present in our body. Myo-inositol is the precursor of inositol triphosphate, a second messenger regulating many hormones such as TSH, FSH and insulin. D-chiroinositol is synthetized by an insulin dependent epimerase that converts myo-inositol into D-chiro-inositol. Polycistic Ovary Syndrome (PCOS) is a metabolic and hormonal disorder and a common cause of infertility. Insulin resistance and the consequent hyperinsulinaemia contribute to hyperandrogenism development, typical marker of PCOS. In these patients myo and/or D-chiro-inositol administration improves insulin sensivity while only myo-inositol is a quality marker for oocytes evaluation. Myo-inositol produces second messengers for FSH and glucose uptake, while D-chiroinositol provides second messengers promoting glucose uptake and glycogen synthesis. The physiological ratio of these two isomers is 40:1 (MI/DCI) and seems to be an optimal approach for the treatment of PCOS disorders. PMID:25010620

Bizzarri, M; Carlomagno, G

2014-07-01

134

Meconium aspiration syndrome: possible pathophysiological mechanisms and future potential therapies.  

PubMed

Does meconium cause meconium aspiration syndrome (MAS) or is meconium discharge only a marker of fetal hypoxia? This dispute has lasted for centuries, but since the 1960s, detrimental effects of meconium itself on the lungs have been demonstrated in animal experiments. In clinical MAS, persistent pulmonary hypertension of the newborn is the leading cause of death in MAS. Regarding the complex chemical composition of meconium, it is difficult to identify a single agent responsible for the pathophysiology. However, considering that meconium is stored in the intestines, partly unexposed to the immune system, aspirated meconium could be recognized as ‘danger', representing damaged self. The common denominator in the pathophysiology could therefore be activation of innate immunity. Thus, a bulk of evidence implies that meconium is a potent activator of inflammatory mediators, including cytokines, complement, prostaglandins and reactive oxygen species. We hypothesize that the two main recognition systems of innate immunity, the Toll-like receptors and the complement system, recognize meconium as ‘danger', which leads not only to lung dysfunction but also to a systemic inflammatory response. This might have therapeutic implications in the future. PMID:25721501

Lindenskov, Paal Helge Haakonsen; Castellheim, Albert; Saugstad, Ola Didrik; Mollnes, Tom Eirik

2015-01-01

135

The Effect of Polarized Polychromatic Noncoherent Light (Bioptron) Therapy on Patients with Carpal Tunnel Syndrome  

PubMed Central

Introduction: To study the effects of Polarized Polychromatic Noncoherent Light (Bioptron) therapy on patients with carpal tunnel syndrome (CTS). Methods: This study was designed as a randomized clinical trial. Forty four patients with mild or moderate CTS (confirmed by clinical and electrodiagnostic studies) were assigned randomly into two groups (intervention and control goups). At the beginning of the study, both groups received wrist splinting for 8 weeks. Bioptron light was applied for the intervention group (eight sessions, for 3/weeks). Bioptron was applied perpendicularly to the wrist from a 10 centimeter sdistance. Pain severity and electrodiagnostic measurements were compared from before to 8 weeks after initiating each treatment. Results: Eight weeks after starting the treatments, the mean of pain severity based on Visual Analogue Scale (VAS) scores decreased significantly in both groups. Median Sensory Nerve Action Potential (SNAP) latency decreased significantly in both groups. However, other electrophysiological findings (median Compound Motor Action Potential (CMAP) latency and amplitude, also SNAP amplitude) did not change after the therapy in both groups. There was no meaningful difference between two groups regarding the changes in the pain severity. Conclusion: Bioptron with the above mentioned parameters led to therapeutic effects equal to splinting alone in patients with carpal tunnel syndrome. However, applying Bioptron with different therapeutic protocols and light parameters other than used in this study, perhaps longer duration of therapy and long term assessment may reveal different results favoring Bioptron therapy. PMID:25606338

Raeissadat, Seyed Ahmad; Rayegani, Seyed Mansoor; Rezaei, Sajad; Bahrami, Mohammad Hasan; Eliaspour, Dariush; Karimzadeh, Afshin

2014-01-01

136

Impact of massage therapy in the treatment of linked pathologies: Scoliosis, costovertebral dysfunction, and thoracic outlet syndrome  

Microsoft Academic Search

Summary Objectives: To investigate the efficacy of massage therapy in the concurrent treatment of three related, but discrete, disorders: scoliosis, costovertebral dysfunction, and thoracic outlet syndrome. Methods: A 34-year-old female subject reported steadily increasing pain in the right shoulder over the previous 8 months. Chiropractic diagnosis and assessment by the author's clinical supervisor had identified these three conditions. Massage therapy

Michael Hamm

2006-01-01

137

Combination therapy of ursodeoxycholic acid and budesonide for PBC–AIH overlap syndrome: a meta-analysis  

PubMed Central

In this study, a meta-analysis of randomized controlled trials comparing ursodeoxycholic acid (UDCA) monotherapy with combination therapies utilizing UDCA and budesonide was performed. We found that combination therapy with UDCA and budesonide was more effective than UDCA monotherapy for primary biliary cirrhosis–autoimmune hepatitis overlap syndrome. Moreover, compared to prednisone, budesonide has fewer side effects. PMID:25632224

Zhang, Huawei; Yang, Jing; Zhu, Rong; Zheng, Yuanyuan; Zhou, Yuqing; Dai, Weiqi; Wang, Fan; Chen, Kan; Li, Jingjing; Wang, Chengfen; Li, Sainan; Liu, Tong; Abudumijiti, Huerxidan; Zhou, Zheng; Wang, Jianrong; Lu, Wenxia; Wang, Junshan; Xia, Yujing; Zhou, Yingqun; Lu, Jie; Guo, Chuanyong

2015-01-01

138

Combination therapy of ursodeoxycholic acid and budesonide for PBC-AIH overlap syndrome: a meta-analysis.  

PubMed

In this study, a meta-analysis of randomized controlled trials comparing ursodeoxycholic acid (UDCA) monotherapy with combination therapies utilizing UDCA and budesonide was performed. We found that combination therapy with UDCA and budesonide was more effective than UDCA monotherapy for primary biliary cirrhosis-autoimmune hepatitis overlap syndrome. Moreover, compared to prednisone, budesonide has fewer side effects. PMID:25632224

Zhang, Huawei; Yang, Jing; Zhu, Rong; Zheng, Yuanyuan; Zhou, Yuqing; Dai, Weiqi; Wang, Fan; Chen, Kan; Li, Jingjing; Wang, Chengfen; Li, Sainan; Liu, Tong; Abudumijiti, Huerxidan; Zhou, Zheng; Wang, Jianrong; Lu, Wenxia; Wang, Junshan; Xia, Yujing; Zhou, Yingqun; Lu, Jie; Guo, Chuanyong

2015-01-01

139

International data on supportive therapies at 1 year in acute coronary syndrome patients undergoing PCI: results from the APTOR study  

Microsoft Academic Search

Background: Supportive therapies of exercise and diet-modifying secondary prevention programmes are associated with reduced morbidity and mortality in acute coronary syndrome (ACS) patients. We sought to evaluate the frequency and correlates of referral to these supportive therapies, and their impact on concordance with prescribed secondary prevention medications at 1 year among ACS patients undergoing percutaneous coronary intervention (PCI) in three

Ameet Bakhai; Jean Ferrieres; Andres Ińiguez; Claude Schmitt; Magali Sartral; Mark Belger; Uwe Zeymer

2011-01-01

140

Tacrolimus rescue therapy for severe respiratory failure in the anti-synthetase syndrome  

PubMed Central

Interstitial lung disease (ILD) is the major determinant of morbidity and mortality in the anti-synthetase syndrome (ASS). The therapeutic efficacy of corticosteroids for the ILD component is limited; hence, additional immunosuppressive and immunomodulatory therapies have been tried with a modicum of success in recent years. Tacrolimus, a calcineurin inhibitor, is one potential therapy. We describe four consecutive patients with ASS whom we treated with tacrolimus at a quaternary referral hospital in 2009–2013. All four patients had significant ILD, three had severe and progressive ILD, and two had been referred for consideration of lung transplantation. Tacrolimus use was associated with improvement in ILD in all four patients with a mean follow-up of 3 years. Our case series adds further evidence to support the use of tacrolimus as salvage therapy for severe respiratory failure due to ILD in ASS, which may be associated with a dramatic and enduring response. PMID:25473571

Rigby, Amy L; Plit, Marshall; Glanville, Allan R

2014-01-01

141

Clinical review: Acute respiratory distress syndrome - clinical ventilator management and adjunct therapy  

PubMed Central

Acute respiratory distress syndrome (ARDS) is a potentially devastating form of acute inflammatory lung injury with a high short-term mortality rate and significant long-term consequences among survivors. Supportive care, principally with mechanical ventilation, remains the cornerstone of therapy - although the goals of this support have changed in recent years - from maintaining normal physiological parameters to avoiding ventilator-induced lung injury while providing adequate gas exchange. In this article we discuss the current evidence base for ventilatory support and adjunctive therapies in patients with ARDS. Key components of such a strategy include avoiding lung overdistension by limiting tidal volumes and airway pressures, and the use of positive end-expiratory pressure with or without lung recruitment manoeuvres in patients with severe ARDS. Adjunctive therapies discussed include pharmacologic techniques (for example, vasodilators, diuretics, neuromuscular blockade) and nonpharmacologic techniques (for example, prone position, alternative modes of ventilation). PMID:23672857

2013-01-01

142

Metabolic syndrome in chronic hepatitis C infection: does it still matter in the era of directly acting antiviral therapy?  

PubMed Central

Metabolic syndrome is prevalent in patients with hepatitis C virus (HCV) infection. Given the pandemic spread of HCV infection and metabolic syndrome, the burden of their interaction is a major public health issue. The presence of metabolic syndrome accelerates the progression of liver disease in patients with HCV infection. New drug development in HCV has seen an unprecedented rise in the last year, which resulted in better efficacy, better tolerance, and a shorter treatment duration. This review describes the underlying mechanisms and clinical effects of metabolic syndrome in HCV infection, as well as their importance in the era of new directly acting antiviral therapy. PMID:25506251

Lim, TR

2014-01-01

143

Pharmacologic and Complementary and Alternative Medicine Therapies for Irritable Bowel Syndrome  

PubMed Central

Irritable bowel syndrome (IBS) is a chronic functional gastrointestinal disorder characterized by episodic abdominal pain or discomfort in association with altered bowel habits (diarrhea and/or constipation). Other gastrointestinal symptoms, such as bloating and flatulence, are also common. A variety of factors are believed to play a role in the development of IBS symptoms, including altered bowel motility, visceral hypersensitivity, psychosocial stressors, altered brain-gut interactions, immune activation/low grade inflammation, alterations in the gut microbiome, and genetic factors. In the absence of biomarkers that can distinguish between IBS subgroups on the basis of pathophysiology, treatment of this condition is predicated upon a patient's most bothersome symptoms. In clinical trials, effective therapies have only offered a therapeutic gain over placebos of 7-15%. Evidence based therapies for the global symptoms of constipation predominant IBS (IBS-C) include lubiprostone and tegaserod; evidence based therapies for the global symptoms of diarrhea predominant IBS (IBS-D) include the probiotic Bifidobacter infantis, the nonabsorbable antibiotic rifaximin, and alosetron. Additionally, there is persuasive evidence to suggest that selected antispasmodics and antidepressants are of benefit for the treatment of abdominal pain in IBS patients. Finally, several emerging therapies with novel mechanisms of action are in development. Complementary and alternative medicine therapies including probiotics, herbal therapies and acupuncture are gaining popularity among IBS sufferers, although concerns regarding manufacturing standards and the paucity of high quality efficacy and safety data remain. PMID:21927652

Maneerattaporn, Monthira; Saad, Richard

2011-01-01

144

Resolution of HIV-associated nephrotic syndrome with highly active antiretroviral therapy delivered by gastrostomy tube.  

PubMed

There is no consensus regarding the specific management of HIV-associated nephrotic syndrome. We report a child whose first manifestation of human immunodeficiency virus type 1 (HIV-1) infection was nephropathy and wasting syndrome associated with profound immunodeficiency. The patient had a dramatic clinical and immunologic response to triple antiretroviral therapy delivered through a gastrostomy tube, with complete resolution of nephrotic syndrome. A 51/2-year-old African-American girl presented with a 2-week history of cough, chest pain, vomiting, loose stools, abdominal distention, anorexia, and fever. In addition, she had recurrent oral thrush. Her weight and height were below the 5th percentile. She was chronically ill, appearing with oropharyngeal thrush and pitting edema in lower extremities. She had scattered rhonchi and decreased breath sounds on both lung bases. Her abdomen was distended and diffusely tender. A chest radiograph showed consolidation of the right upper and left lower lobes with bilateral pleural effusion. Admission laboratories were consistent with nephrotic syndrome. Streptococcus pneumoniae grew from the blood culture and the child responded well to treatment with intravenous ceftriaxone. She was found to be HIV-infected, her CD4(+) cell count was 3 cells/mcL and her plasma HIV-1 RNA was >750 000 copies/mL. A percutaneous gastrostomy tube was placed for supplemental nutrition. She was treated with stavudine, lamivudine, and nelfinavir via gastrostomy tube with good clinical response. Twenty-one months after instituting antiretroviral therapy, her weight and height had increased to the 50th and 10th percentile respectively, and she had complete resolution of her nephrotic syndrome. Her CD4(+) cell count increased to 1116 cells/mcL and her viral load has remained undetectable. HIV-1 associated nephrotic syndrome has been described in children with profound immunodeficiency. The course of untreated HIV-associated nephrotic syndrome is rapid progression to renal failure in up to 40% of the children. Regardless of the presence of renal insufficiency, if untreated, it is uniformly fatal. A modest improvement of HIV-1 associated nephrotic syndrome has been observed in patients treated with zidovudine. Steroid and cyclosporine treatment have resulted in improved renal function but long-term use of immunosuppressive therapy has raised concerns about safety. We have described, to our knowledge, the first child with HIV-associated nephrotic syndrome who had a remarkable clinical, immunologic, and virologic response to triple-drug combination therapy given by gastrostomy tube, with complete resolution of proteinuria and normalization of the serum albumin. She also had a striking improvement in weight, height, and quality-of-life. Whether the presence of a gastrostomy tube contributed to the excellent response because of improved compliance is unknown, but warrants systematic evaluation. PMID:10585995

Viani, R M; Dankner, W M; Muelenaer, P A; Spector, S A

1999-12-01

145

Managing Sjögren’s Syndrome and non-Sjögren Syndrome dry eye with anti-inflammatory therapy  

PubMed Central

Dry eye from Sjögren’s syndrome is a multifactorial disease that results in dysfunction of the lacrimal functional unit. Studies have shown changes in tear composition, including inflammatory cytokines, chemokines, and metalloproteinase. T-lymphocytes have been shown to increase in the conjunctiva and lacrimal glands in patient and animal models. This inflammation is in part responsible for the pathogenesis of the disease, which results in symptoms of eye irritation, ocular surface epithelial disease, and loss of corneal barrier function. There are a number of anti-inflammatory approaches for treating this disease. The current study reviews details of immune response and anti–inflammatory therapies used to control this disease. PMID:25120351

Coursey, Terry G; de Paiva, Cintia S

2014-01-01

146

The Effect of Extracorporeal Shock Wave Therapy on Myofascial Pain Syndrome  

PubMed Central

Objective To investigate the effect of extracorporeal shock wave therapy (ESWT) on myofascial pain syndrome (MPS). Method Thirty patients with MPS in trapezius muscle were randomly divided into two groups, ESWT group (n=15), and trigger point injections (TPI)+transcutaneous electrical nerve stimulation (TENS) group (n=15). For a total of 3 weeks, ESWT was undertaken with 1,500 pulse each time at one week interval totaling 4,500 pulse, TPI for once a week totaling three times and TENS for five times a week totaling three weeks. Results The changes in pain threshold (lb/cm2) showed the values of 6.86±1.35 before first therapy, 11.43±0.27 after first therapy, and 12.57±0.72 after third therapy, while TPI+TENS group showed the values of 6.20±1.92 before first therapy, 8.80±0.48 after first therapy, and 9.60±2.19 after third therapy, and the changes between the groups were significantly different (p=0.045). The changes in visual analog scale were estimated to be 6.86±0.90 before first therapy, 2.86±0.90 after first therapy, and 1.86±0.69 after third therapy in case of ESWT group, whereas the figures were estimated to be 7.20±1.30 before first therapy, 4.60±0.55 after first therapy, and 2.80±0.84 after third therapy in case of TPI+TENS group, and the changes between the groups were significantly different (p=0.010). The changes in McGill pain questionnaire (p=0.816) and pain rating scale (p=0.644) between the groups were not significantly different. The changes in neck ROM were also not significantly different between the groups (p>0.05). Conclusion The ESWT in patients with MPS in trapezius muscle are as effective as TPI and TENS for the purpose of pain relief and improving cervical range of motion. PMID:23185731

Jeon, Jong Hyun; Jung, Yun Jae; Lee, Ju Youn; Choi, Ji Soo; Mun, Jeong Hyeon; Park, Won Yong; Seo, Cheong Hoon

2012-01-01

147

Conus magus vs. Irukandji syndrome: a computational approach of a possible new therapy.  

PubMed

The Irukandji syndrome is caused by the sting of some small jellyfish species. The syndrome has severe life-threatening consequences. The exacerbating pain and cardiovascular symptoms (tachycardia and hypertension) are hard to control in many cases. We suggest a way to experiment a new possible therapy with an FDA approved analgesic, ziconotide, a synthetic derivative from a marine cone snail (Conus magus) venom component, which is administrated intravenously. The proposed experimental plasma concentration of ziconotide for rats is in the range of 0-6?gml(-1). Based on a molecular biological scenario of the venom action mechanism at cellular level, we suggest that the proposed method should be functional in re-establishing the normal cardiovascular parameters of the experimental animals and concomitantly it should abolish the severe pain caused by envenomation. We expect that positive experimental results in agreement with our theory will lead to the possibility of a new therapy for the Irukandji syndrome and possibly for other envenomations with similar ethyology. PMID:21777663

András, Csaba D; Albert, Csilla; Salamon, Szidónia; Gálicza, Judit; András, Réka; András, Emil

2011-10-10

148

Cardiorenal Syndrome: Ultrafiltration Therapy for Heart Failure—Trials and Tribulations  

PubMed Central

Summary Heart failure remains the leading cause of hospitalization in older patients and is considered a growing public health problem with a significant financial burden on the health care system. The suboptimal efficacy and safety profile of diuretic-based therapeutic regimens coupled with unsatisfactory results of the studies on novel pharmacologic agents have positioned ultrafiltration on the forefront as an appealing therapeutic option for patients with acute decompensated heart failure (ADHF). In recent years, substantial interest in the use of ultrafiltration has been generated due to the advent of dedicated portable devices and promising results of trials focusing both on mechanistic and clinical aspects of this therapeutic modality. This article briefly reviews the proposed benefits of ultrafiltration therapy in the setting of ADHF and summarizes the major findings of the currently available studies in this field. The results of more recent trials on cardiorenal syndrome that present a counterpoint to previous observations and highlight certain limitations of ultrafiltration therapy are then discussed, followed by identification of major challenges and unanswered questions that could potentially hinder its more widespread use. Future studies are warranted to shed light on less well characterized aspects of ultrafiltration therapy and to further define its role in ADHF and cardiorenal syndrome. PMID:23723339

2013-01-01

149

Cognitive-behavioral therapy for the management of irritable bowel syndrome  

PubMed Central

Irritable bowel syndrome (IBS) is a common disorder, reported to be found in 5%-20% of the general population. Its management accounts for up to 25% of a gastroenterologist’s workload in the outpatient department, and the main symptoms are abdominal pain, bloating, and altered bowel habits. Despite a great amount of available pharmacological treatments aimed at a wide variety of gastrointestinal and brain targets, many patients have not shown adequate symptom relief. In recent years, there has been increasing evidence to suggest that psychological treatments, in particular cognitive-behavioral therapy (CBT), are effective for the management of IBS. This review discusses CBT for the management of IBS. CBT has proved to be effective in alleviating the physical and psychological symptoms of IBS and has thus been recommended as a treatment option for the syndrome. PMID:24379577

Tang, Qing-Lin; Lin, Guo-Yao; Zhang, Ming-Qing

2013-01-01

150

Eculizumab Therapy Leads to Rapid Resolution of Thrombocytopenia in Atypical Hemolytic Uremic Syndrome  

PubMed Central

Eculizumab is highly effective in controlling complement activation in patients with the atypical hemolytic uremic syndrome (aHUS). However, the course of responses to the treatment is not well understood. We reviewed the responses to eculizumab therapy for aHUS. The results show that, in patients with aHUS, eculizumab therapy, when not accompanied with concurrent plasma exchange therapy, led to steady increase in the platelet count and improvement in extra-renal complications within 3 days. By day 7, the platelet count was normal in 15 of 17 cases. The resolution of hemolytic anemia and improvement in renal function were less predictable and were not apparent for weeks to months in two patients. The swift response in the platelet counts was only observed in one of five cases who received concurrent plasma exchange therapy and was not observed in a case of TMA due to gemcitabine/carboplatin. In summary, eculizumab leads to rapid increase in the platelet counts and resolution of extrarenal symptoms in patients with aHUS. Concurrent plasma exchange greatly impedes the response of aHUS to eculizumab therapy. Eculizumab is ineffective for gemcitabine/carboplatin associated TMA. PMID:25400666

Kuo, Elizabeth

2014-01-01

151

Effects of Temperature on Chronic Trapezius Myofascial Pain Syndrome during Dry Needling Therapy  

PubMed Central

The purpose of this study was to investigate the effects of temperature on chronic trapezius myofascial pain syndrome during dry needling therapy. Sixty patients were randomized into two groups of dry needling (DN) alone (group A) and DN combined with heat therapy group (group B). Each patient was treated once and the therapeutic effect was assessed by the visual analogue scale (VAS), pressure pain threshold (PPT), and the 36-item short form health survey (SF-36) at seven days, one month, and three months after treatment. Evaluation based on VAS and PPT showed that the pain of patients in groups A and B was significantly (P < 0.05) relieved at seven days, one month, and three months after treatment Compared to before treatment. There was significantly (P < 0.05) less pain in group B than group A at one and three months after treatment. The SF-36 evaluation demonstrated that the physical condition of patients in both groups showed significant (P < 0.05) improvement at one month and three months after treatment than before treatment. Our study suggests that both DN and DN heating therapy were effective in the treatment of trapezius MPS, and that DN heating therapy had better long-term effects than DN therapy. PMID:25383083

2014-01-01

152

Extracorporeal Shock Wave Therapy in Myofascial Pain Syndrome of Upper Trapezius  

PubMed Central

Objective To evaluate the effect of extracorporeal shock wave therapy (ESWT) in myofascial pain syndrome of upper trapezius with visual analogue scale (VAS) and pressure threshold by digital algometer. Method Twenty-two patients diagnosed with myofascial pain syndrome in upper trapezius were selected. They were assigned to treatment and standard care (control) groups balanced by age and sex, with eleven subjects in each group. The treated group had done four sessions of ESWT (0.056 mJ/mm2, 1,000 impulses, semiweekly) while the control group was treated by the same protocol but with different energy levels applied, 0.001 mJ/mm2. The VAS and pressure threshold were measured twice: before and after last therapy. We evaluated VAS of patients and measured the pressure threshold by using algometer. Results There were two withdrawals and the remaining 20 patients were three men and 17 women. Age was distributed with 11 patients in their twenties and 9 over 30 years old. There was no significant difference of age, sex, pre-VAS and pre-pressure threshold between 2 groups (p>0.05) found. The VAS significantly decreased from 4.91±1.76 to 2.27±1.27 in the treated group (p<0.01). The control group did not show any significant changes of VAS score. The pressure threshold significantly increased from 40.4±9.94 N to 61.2±12.16 N in the treated group (p<0.05), but there was no significant change in the control group. Conclusion ESWT in myofascial pain syndrome of upper trapezius is effective to relieve pain after four times therapies in two weeks. But further study will be required with more patients, a broader age range and more males. PMID:23185732

Ji, Hye Min; Kim, Ho Jeong

2012-01-01

153

Analysis of Factors Associated With Radiation-Induced Bronchiolitis Obliterans Organizing Pneumonia Syndrome After Breast-Conserving Therapy  

SciTech Connect

Purpose: To evaluate factors associated with radiation-induced bronchiolitis obliterans organizing pneumonia (BOOP) syndrome after breast-conserving therapy. Methods and Materials: A total of 702 women with breast cancer who received radiotherapy after breast-conserving surgery at seven institutions between July 1995 and December 2006 were analyzed. In all patients, the whole breast was irradiated with two tangential photon beams. The criteria used for the diagnosis of radiation-induced BOOP syndrome were as follows: (1) radiotherapy to the breast within 12 months, (2) general and/or respiratory symptoms lasting for {>=}2 weeks, (3) radiographs showing lung infiltration outside the radiation port, and (4) no evidence of a specific cause. Results: Radiation-induced BOOP syndrome was seen in 16 patients (2.3%). Eleven patients (68.8%) were administered steroids. The duration of steroid administration ranged from 1 week to 3.7 years (median, 1.1 years). Multivariate analysis revealed that age ({>=}50 years; odds ratio [OR] 8.88; 95% confidence interval [CI] 1.16-67.76; p = 0.04) and concurrent endocrine therapy (OR 3.05; 95% CI 1.09-8.54; p = 0.03) were significantly associated with BOOP syndrome. Of the 161 patients whose age was {>=}50 years and who received concurrent endocrine therapy, 10 (6.2%) developed BOOP syndrome. Conclusions: Age ({>=}50 years) and concurrent endocrine therapy can promote the development of radiation-induced BOOP syndrome after breast-conserving therapy. Physicians should carefully follow patients who received breast-conserving therapy, especially those who are older than 50 years and received concurrent endocrine therapy during radiotherapy.

Katayama, Norihisa [Department of Radiology, Okayama University Hospital, Okayama (Japan)], E-mail: n-katayama@bea.hi-ho.ne.jp; Sato, Shuhei [Department of Radiology, Okayama University Hospital, Okayama (Japan); Katsui, Kuniaki [Department of Radiology, Fukuyama City Hospital, Fukuyama (Japan); Takemoto, Mitsuhiro [Department of Radiology, Okayama University Hospital, Okayama (Japan); Tsuda, Toshihide [Department of Environmental Epidemiology, Graduate School of Environmental Science, Okayama University Graduate School, Okayama (Japan); Yoshida, Atsushi [Department of Radiology, Kure Kyosai Hospital, Kure (Japan); Morito, Tsuneharu [Department of Radiology, Okayama Saiseikai General Hospital, Okayama (Japan); Nakagawa, Tomio [Department of Radiology, NHO Fukuyama Medical Center, Fukuyama (Japan); Mizuta, Akifumi [Department of Radiology, Chugoku Central Hospital, Fukuyama (Japan); Waki, Takahiro; Niiya, Harutaka [Department of Radiology, NHO Okayama Medical Center, Okayama (Japan); Kanazawa, Susumu [Department of Radiology, Okayama University Hospital, Okayama (Japan)

2009-03-15

154

Cauda equina syndrome associated with multiple lumbar arachnoid cysts in ankylosing spondylitis: improvement following surgical therapy.  

PubMed Central

A case of cauda equina syndrome with multiple lumbar arachnoid cysts complicating ankylosing spondylitis (AS) is described. The value of computerised tomography (CT) and magnetic resonance imaging (MRI) as a non-invasive means of establishing the diagnosis is emphasised. In contrast to previously reported cases the patient showed neurological improvement following surgical therapy. Surgery may be indicated in some patients, particularly when there is nerve root compression by the arachnoid cysts and when the patient is seen early before irreversible damage to the cauda equina has occurred. Images PMID:2292702

Shaw, P J; Allcutt, D A; Bates, D; Crawford, P J

1990-01-01

155

[Application of hyperbaric oxygen therapy for post-cardiac arrest syndrome].  

PubMed

Therapeutic mechanisms of hyperbaric oxygen therapy (HBOT) are based on elevation of both the partial pressure of oxygen and hydrostatic pressure. It has been reported that HBOT supply much amount of oxygen and might prevent from brain tissue after global ischemia in patients with post-cardiac arrest syndrome(PCAS). On the other hand, high levels of oxygen produce reactive oxygen species(ROS). ROS occurs secondary brain damage after ischemia. The clinical evidence of HBOT has not been established. In this paper, we review some reports about the advantages and disadvantages of HBOT after global ischemia with PCAS. PMID:21591418

Miyamoto, Kazuyuki; Dohi, Kenji

2011-04-01

156

Treating metabolic syndrome's metaflammation with low level light therapy: preliminary results  

NASA Astrophysics Data System (ADS)

Metabolic syndrome comprises a constellation of morbidities such as insulin resistance, hyperinsulinemia, atherogenic dyslipidemia, dysglycemia and obesity (especially abdominal). Metabolic alterations are observed in major insulin target organs, increasing the risk of cardiovascular diseases, type-2 diabetes and therefore mortality. Tissue alterations are characterized by immune cells infiltrates (especially activated macrophages). Released inflammatory mediators such as TNF-? induce chronic inflammation in subjects with metabolic syndrome, since inflammatory pathways are activated in the neighboring cells. The intra-abdominal adipose tissue appears to be of particular importance in the onset of the inflammatory state, and strategies contributing to modulate the inflammatory process within this adipose tissue can mitigate the metabolic syndrome consequences. Considering the low level light therapy (LLLT) recognized benefits in inflammatory conditions, we hypothesized this therapeutic approach could promote positive effects in modulating the inflammatory state of metabolic syndrome. That being the scope of this study, male C57BL/6 mice were submitted to a high-fat/high-fructose diet among 8 weeks to induce metabolic syndrome. Animals were then irradiated on the abdominal region during 21 days using an 850 nm LED (6 sessions, 300 seconds per session, 60 mW output power, ~6 J/cm2 fluence, ~19 mW/cm2 fluence rate). Before and during treatment, blood was sampled either from the retroorbital plexus or from tail puncture for glucose, total cholesterol and triglycerides analysis. So far our results indicate no alterations on these metabolic parameters after LLLT. For further investigations, blood was collected for plasma inflammatory cytokine quantification and fresh ex vivo samples of liver and intra-abdominal adipose tissue were harvested for immunohistochemistry purposes.

Yoshimura, Tania M.; Kato, Ilka T.; Deana, Alessandro M.; Ribeiro, Martha S.

2014-02-01

157

NTOS symptoms and mobility: a case study on neurogenic thoracic outlet syndrome involving massage therapy.  

PubMed

Neurogenic thoracic outlet syndrome (NTOS) is a neuromuscular condition affecting brachial plexus functionality. NTOS is characterized by paresthesia, pain, muscle fatigue, and restricted mobility in the upper extremity. This study quantified massage therapy's possible contribution to treatment of NTOS. A 24-year-old female with NTOS received eight treatments over 35 days. Treatment included myofascial release, trigger point therapy, cross fiber friction, muscle stripping, and gentle passive stretching. Abduction and lateral rotation at the glenohumeral (GH joint) assessments measured range of motion (ROM). A resisted muscle test evaluated upper extremity strength. The client rated symptoms daily via a visual analog scale (VAS). Findings showed improvement in ROM at the GH joint. VAS ratings revealed a reduction in muscle weakness, pain, numbness, and 'paresthesia'. Results suggest massage may be useful as part of a broad approach to managing NTOS symptoms and improving mobility. PMID:24411148

Streit, Robin S

2014-01-01

158

Plasmapheresis as adjuvant therapy in Stevens-Johnson syndrome and hepatic encephalopathy.  

PubMed

Stevens-Johnson syndrome (SJS) is a severe idiosyncratic reaction, most commonly triggered by medications, which is characterized by fever and mucocutaneous lesions, leading to necrosis and sloughing of the epidermis. Aside from skin and mucosal manifestations, SJS may also compromise heart, liver, kidney, lung, and gastrointestinal tract. Although cholestatic liver disease has been reported to occur in SJS, hepatic encephalopathy (HE) as a delayed complication has never been reported. We report a 4-year-old female child with anticonvulsant-induced SJS complicated by HE who was completely cured with a combination of systemic corticosteroid, intravenous immunoglobulin (IVIG), and plasmapheresis therapy. We suggested that plasmapheresis may be used as an adjuvant therapy for SJS with HE. PMID:23777679

Hung, Po-Cheng; Wang, Huei-Shyong; Hsia, Shao-Hsuan; Wong, Alex M-C

2014-04-01

159

Journees Automates Cellulaires 2008 (Uz`es), pp. 54-64 SOFIC ONE HEAD MACHINES  

E-print Network

Head Machine" an automaton that lives in a discrete space. It can walk, read and write symbols, and itsJourn´ees Automates Cellulaires 2008 (Uz`es), pp. 54-64 SOFIC ONE HEAD MACHINES A. GAJARDO-dimensional Turing machines with only one head is adopted. A subshift is associated to each Turing machine, and its

Paris-Sud XI, Université de

160

TUDE DE LA CYTOTOXICIT MDIATION CELLULAIRE DANS LE SARCOME DE ROUS CHEZ LE HAMSTER  

E-print Network

Ă?TUDE DE LA CYTOTOXICITĂ? Ă? MĂ?DIATION CELLULAIRE DANS LE SARCOME DE ROUS CHEZ LE HAMSTER Ginette, France Summary CELL-MEDIATED CYTOTOXICITY AGAINST ROUS SARCOMA IN THE HAMSTER. - The cytotoxic response of lymphoid cells from hamsters immunized against RS 2/3 cells, transformed by Rous sarcoma virus

Paris-Sud XI, Université de

161

Journes Automates Cellulaires 2008 (Uzs), pp. 216-227 TOPOLOGICAL PROPERTIES OF SAND AUTOMATA  

E-print Network

Journées Automates Cellulaires 2008 (Uzès), pp. 216-227 TOPOLOGICAL PROPERTIES OF SAND AUTOMATA.masson@lif.univ-mrs.fr Abstract. In this paper, we exhibit a strong relation between the sand automata con- figuration space and the cellular automata configuration space. This relation induces a compact topology for sand automata

Paris-Sud XI, Université de

162

Multimodal therapy for category III chronic prostatitis/chronic pelvic pain syndrome in UPOINTS phenotyped patients  

PubMed Central

The complex network of etiological factors, signals and tissue responses involved in chronic prostatitis/chronic pelvic pain syndrome (CP/CPPS) cannot be successfully targeted by a single therapeutic agent. Multimodal approaches to the therapy of CP/CPPS have been and are currently being tested, as in the frame of complex diagnostic-therapeutic phenotypic approaches such as the urinary, psychosocial, organ-specific, infection, neurological and muscle tenderness (UPOINTS) system. In this study, the effect of combination therapy on 914 patients diagnosed, phenotyped and treated in a single specialized prostatitis clinic was analyzed. Patients received ?-blockers, Serenoa repens (S. repens) extracts combined or not with supplements (lycopene and selenium) and, in the presence of documented or highly suspected infection, antibacterial agents. Combination treatment induced marked and significant improvements of National Institutes of Health Chronic Prostatitis Symptom Index (NIH-CPSI) prostatitis symptom scores, International Index of Erectile Function (IIEF) sexual dysfunction scores, urinary peak flow rates and bladder voiding efficiency. These improvements, assessed after a 6-month course of therapy, were sustained throughout a follow-up period of 18 months. A clinically appreciable reduction of ?6 points of the total NIH-CPSI score was achieved in 77.5% of patients subjected to combination therapy for a period of 6 months. When the patients were divided in two cohorts, depending on the diagnosis of CP/CPPS [inflammatory (IIIa) vs. non-inflammatory (IIIb) subtypes], significant improvements of all signs and symptoms of the syndrome were observed in both cohorts at the end of therapy. Intergroup comparison showed that patients affected by the IIIa sub-category of CP/CPPS showed more severe signs and symptoms (NIH-CPSI total, pain and quality of life impact scores, and Qmax) at baseline when compared with IIIb patients. However, the improvement of symptoms after therapy was significantly more pronounced in IIIa patients when compared with IIIb patients. In contrast to current opinion, the evidence emerging from the present investigation suggests that the inflammatory and non-inflammatory sub-categories of CP/CPPS may represent two distinct pathological conditions or, alternatively, two different stages of the same condition. In conclusion, a simple protocol based on ?-blockers, S. repens extracts and supplements and antibacterial agents, targeting the urinary, organ specific and infection domains of UPOINTS, may induce a clinically appreciable improvement of the signs and symptoms of CP/CPPS in a considerable percentage of patients. In patients not responding sufficiently to such therapy, second-line agents (antidepressants, anxiolytics, muscle relaxants, 5-phosphodiesterase inhibitors and others) may be administered in order to achieve a satisfactory therapeutic response. PMID:25667610

MAGRI, VITTORIO; MARRAS, EMANUELA; RESTELLI, ANTONELLA; WAGENLEHNER, FLORIAN M.E.; PERLETTI, GIANPAOLO

2015-01-01

163

Opsoclonus-myoclonus in an HIV-infected child on antiretroviral therapy—possible immune reconstitution inflammatory syndrome  

Microsoft Academic Search

The exact immunopathogenesis and neuroanatomical localization of opsoclonus-myoclonus ataxia syndrome remains unclear. We describe a 1 year 9 month old girl who, shortly after commencement of highly active antiretroviral therapy developed opsoclonus-myoclonus syndrome and subsequently died of disseminated cytomegalovirus infection. We postulate on the etiological factors that may have played a role in the disease pathogenesis of the patient's opsoclonus-myoclonus

Ronald van Toorn; Helena Rabie; James M. Warwick

2005-01-01

164

Case Report: The Effects of Massage Therapy on a Woman with Thoracic Outlet Syndrome  

PubMed Central

Introduction Thoracic outlet syndrome (TOS) refers to a group of conditions resulting from compression of the neurovascular structures of the thoracic outlet. The parameters for physical therapy include myofascial release (MFR), neuromuscular therapy (NMT), muscle strengthening, and stretching. This case study examined the effects of neuromuscular therapy, massage, and other manual therapies on a 56-year-old female presenting with bilateral numbness over the forearms and hands on waking. Numbness occurred most days, progressing to “dead rubbery” forearms and hands once or twice a month. Methods The treatment plan was implemented over eight weeks and consisted of six, 50-minute bodywork sessions. Several nonbodywork strategies were also employed to address potential contributing factors to the TOS symptomology experienced by the client. Objective measurements included posture analysis (PA), range of movement (ROM), and Roos and Adson’s tests. The Measure Your Own Medical Outcome Profile (MYMOP2), a client-generated measure of clinical outcome, was used to measure clinical change. Results MYMOP2 overall profile score results demonstrated an improvement of 2.25 from pretreatment to post-treatment measurement. Clinically meaningful change was measured by the individual and was indicative of substantial symptom improvement where a score change of over one was considered as meaningful. Conclusions A course of massage was effective for numbness symptoms in an individual with TOS, and results lasted over a year without additional treatments. Further research is needed to fully understand the effects of massage for TOS symptoms. PMID:25452819

Wakefield, Mary Lillias

2014-01-01

165

Mesenchymal stem cell therapy for acute radiation syndrome: innovative medical approaches in military medicine.  

PubMed

After a radiological or nuclear event, acute radiation syndrome (ARS) will present complex medical challenges that could involve the treatment of hundreds to thousands of patients. Current medical doctrine is based on limited clinical data and remains inadequate. Efforts to develop medical innovations that address ARS complications are unlikely to be generated by industry because of market uncertainties specific to this type of injury. A prospective strategy could be the integration of cellular therapy to meet the medical demands of ARS. The most clinically advanced cellular therapy to date is the administration of mesenchymal stem cells (MSCs). Results of currently published investigations describing MSC safety and efficacy in a variety of injury and disease models demonstrate the unique qualities of this reparative cell population in adapting to the specific requirements of the damaged tissue in which the cells integrate. This report puts forward a rationale for the further evaluation of MSC therapy to address the current unmet medical needs of ARS. We propose that the exploration of this novel therapy for the treatment of the multivariate complications of ARS could be of invaluable benefit to military medicine. PMID:25722881

Eaton, Erik B; Varney, Timothy R

2015-01-01

166

Enzyme replacement therapy in a feline model of Maroteaux-Lamy syndrome.  

PubMed Central

We report studies that suggest enzyme replacement therapy will result in a significant reduction in disease progression and tissue pathology in patients with Maroteaux-Lamy syndrome (Mucopolysaccharidosis type VI, MPS VI). A feline model for MPS VI was used to evaluate tissue distribution and clinical efficacy of three forms of recombinant human N-acetylgalactosamine-4-sulfatase (rh4S, EC 3.1.6.1). Intravenously administered rh4S was rapidly cleared from circulation. The majority of rh4S was distributed to liver, but was also detected in most other tissues. Tissue half-life was approximately 2-4 d. Three MPS VI cats given regular intravenous infusions of rh4S for up to 20 mo showed variable reduction of storage vacuoles in Kupffer cells and connective tissues, however cartilage chondrocytes remained vacuolated. Vertebral bone mineral volume was improved in two MPS VI cats in which therapy was initiated before skeletal maturity, and increased bone volume appeared to correlate with earlier age of onset of therapy. One cat showed greater mobility in response to therapy. PMID:8621770

Crawley, A C; Brooks, D A; Muller, V J; Petersen, B A; Isaac, E L; Bielicki, J; King, B M; Boulter, C D; Moore, A J; Fazzalari, N L; Anson, D S; Byers, S; Hopwood, J J

1996-01-01

167

A case of multiple evanescent white dot syndrome treated by steroid pulse therapy.  

PubMed

Multiple evanescent white dot syndrome (MEWDS) is characterized by acute visual loss which is usually spontaneously restored after a few months. While occasional treatments with local or oral steroids have been reported, these are hardly effective. A 25-year-old man, a Sumo wrestler, was diagnosed with MEWDS, and as he wanted a quick recovery to compete in an upcoming tournament treatment with steroid pulse therapy was initiated 8 days after the symptoms appeared. Before treatment, visual acuity in his left eye was 20/400. Multiple white dots were seen at the level of the deep retina or retinal pigment epithelium, and the Mariotte's blind spot extended to the center of the visual field. Immediately after the end of steroid pulse therapy, left visual acuity increased to 20/25, and left visual field recovered remarkably. No white dots were seen funduscopically. Steroid pulse therapy might provide early improvement of visual functions, and we believe it could be a treatment option for initiating an early recovery from MEWDS. However, steroid pulse therapy may also result in lethal damage including disorders of the circulatory organs. Thus, it should only be applied in limited situations in which patients had rapid decline in visual function and needs for social return at an early stage like that of our patient. PMID:17330396

Takahashi, Yasuhiro; Ataka, Shinsuke; Wada, Sonomi; Kohno, Takeya; Nomura, Yuya; Shiraki, Kunihiko

2006-12-01

168

Statin Therapy in Metabolic Syndrome and Hypertension Post-JUPITER: What is the Value of CRP?  

PubMed Central

Much evidence supports a pivotal role for inflammation in atherosclerosis. C-reactive protein (CRP), the prototypic marker of inflammation in humans, is a cardiovascular risk marker and may also promote atherogenesis. CRP levels are increased in metabolic syndrome and hypertension and confer increased risk of cardiovascular events in patients in these subgroups. Statins have been shown to lower low-density lipoproteins and CRP independently, and reduce cardiovascular events in subjects with and without metabolic syndrome and hypertension. In this review, we focus on the results from the primary prevention statin trial, Justification for the Use of statins in Primary prevention: an Intervention Trial Evaluating Rosuvastatin (JUPITER), which showed reductions in LDL, CRP, and cardiovascular events. Post-JUPITER, the new guidelines will now need to consider recommending high-sensitivity CRP testing to intermediate-risk metabolic syndrome patients and those with hypertension and intermediate risk so that we can better identify candidates at greater risk and reduce cardiovascular burden in these subjects with statin therapy. PMID:21046291

Devaraj, Sridevi; Siegel, David

2010-01-01

169

Immuno-therapy of Acute Radiation Syndromes : Extracorporeal Immuno-Lympho-Plasmo-Sorption.  

NASA Astrophysics Data System (ADS)

Methods Results Summary and conclusions Introduction: Existing Medical Management of the Acute Radiation Syndromes (ARS) does not include methods of specific immunotherapy and active detoxication. Though the Acute Radiation Syndromes were defined as an acute toxic poisonous with development of pathological processes: Systemic Inflammatory Response Syndrome (SIRS), Toxic Multiple Organ Injury (TMOI), Toxic Multiple Organ Dysfunction Syndrome(TMODS), Toxic Multiple Organ Failure (TMOF). Radiation Toxins of SRD Group play an important role as the trigger mechanisms in development of the ARS clinical symptoms. Methods: Immuno-Lympho-Plasmo-Sorption is a type of Immuno-therapy which includes prin-ciples of immunochromato-graphy, plasmopheresis, and hemodialysis. Specific Antiradiation Antitoxic Antibodies are the active pharmacological agents of immunotherapy . Antiradia-tion Antitoxic Antibodies bind selectively to Radiation Neurotoxins, Cytotoxins, Hematotox-ins and neutralize their toxic activity. We have developed the highly sensitive method and system for extracorporeal-immune-lypmh-plasmo-sorption with antigen-specific IgG which is clinically important for treatment of the toxic and immunologic phases of the ARS. The method of extracorporeal-immune-lypmh-plasmo-sorption includes Antiradiation Antitoxic Antibodies (AAA) immobilized on microporous polymeric membranes with a pore size that is capable to provide diffusion of blood-lymph plasma. Plasma of blood or lymph of irradiated mammals contains Radiation Toxins (RT) that have toxic and antigenic properties. Radiation Toxins are Antigen-specific to Antitoxic blocking antibodies (Immunoglobulin G). Plasma diffuses through membranes with immobilized AAA and AA-antibodies bind to the polysaccharide chain of tox-ins molecules and complexes of AAA-RT that are captured on membrane surfaces. RT were removed from plasma. Re-transfusion of plasma of blood and lymph had been provided. We show a statistical significant reduction in postradiation lethality.

Popov, Dmitri; Maliev, Slava

170

Metformin therapy improves ovulatory rates, cervical scores, and pregnancy rates in clomiphene citrate-resistant women with polycystic ovary syndrome  

Microsoft Academic Search

Objective: To evaluate the effect of metformin therapy on hyperandrogenism, insulin resistance, cervical scores, ovulation, and pregnancy rates in clomiphene citrate-resistant women with polycystic ovary syndrome (PCOS).Design: Prospective, randomized, double-blind, placebo-controlled study.Setting: Infertility clinic of a tertiary referral center.Patient(s): Fifty-six women with clomiphene citrate-resistant PCOS.Intervention(s): Two cycles of oral metformin therapy (850 mg, twice daily) in group I and placebo

Müberra Kocak; Eray Caliskan; Coskun Simsir; Ali Haberal

2002-01-01

171

Comparison the efficacy of phonophoresis and ultrasound therapy in myofascial pain syndrome.  

PubMed

The aim of this study is to compare the effect of phonophoresis, ultrasound and placebo ultrasound therapies in the treatment of myofascial pain syndrome (MPS). This is a randomized, double-blind placebo controlled study. Sixty patients (48 women, 12 men, mean age 37.9 ± 12.2 years) with MPS were included in this study. Patients were allocated into three groups. Group 1(n = 20) was received diclofenac phonophoresis, group 2(n = 20) was received ultrasound and group 3(n = 20) was received placebo ultrasound therapies over trigger points, 10 min a day for 15 session during 3 weeks (1 MHz-1,5 watt/cm˛). Additionally, all patients were given neck exercise program including isotonic, isometric and stretching. Patients were assessed by means of pain, range of motion (ROM) of neck, number of trigger points (NTP), algometric measurement and disability. Pain severity was measured by visual analog scale (VAS) and Likert scale. The neck pain disability index (NPDI) was used for assessing disability. Measurements were taken before and after treatment. After treatment, there were statistically significant improvements in pain severity, NTP, pressure pain threshold (PPT), ROM and NPDI scores both in phonophoresis and in ultrasound therapy groups (P < 0.05). Statistically significant increase in cervical lateral flexion and rotation was observed in the placebo US group. While there was no statistically significant improvement in the cervical flexion-extension joint movement, pain levels, number of trigger points and NPDI score, pressure pain threshold (P > 0.05), also there were no significant differences in all parameters between group 1 and 2 (P = 0.05). Both diclofenac phonophoresis and ultrasound therapy were effective in the treatment of patients with MPS. Phonophoresis was not found to be superior over ultrasound therapy. PMID:20354859

Ay, Saime; Do?an, Sebnem Kolda?; Evcik, Deniz; Ba?er, Ozgün Cakmak

2011-09-01

172

Therapy strategies for Usher syndrome Type 1C in the retina.  

PubMed

The Usher syndrome (USH) is the most common form of inherited deaf-blindness with a prevalence of ~?1/6,000. Three clinical subtypes (USH1-USH3) are defined according to the severity of the hearing impairment, the presence or absence of vestibular dysfunction and the age of onset of retinitis pigmentosa (RP). USH1 is the most severe subtype with congenital severe to profound hearing loss and onset of RP before puberty. Currently only the amelioration of the hearing deficiency is implemented, but no treatment of the senso-neuronal degeneration in the eye exists.In our studies we are focusing on the evaluation of gene-based therapies to cure the retinal degeneration of USH1C patients: (i) gene augmentation using recombinant adeno-associated virus, (ii) genome editing by homologous recombination mediated by zinc-finger nucleases and, (iii) read-through therapy using novel designer aminoglycosides and PTC124. Latter compounds target in-frame nonsense mutations which account for ~?20?% of all USH cases.All analyzed gene-based therapy strategies lead to the restoration of USH protein expression. These adjustments may be sufficient to reduce the progression of retinal degeneration, which would greatly improve the life quality of USH patients. PMID:24664766

Nagel-Wolfrum, Kerstin; Baasov, Timor; Wolfrum, Uwe

2014-01-01

173

Lentivirus-based Gene Therapy of Hematopoietic Stem Cells in Wiskott-Aldrich Syndrome  

PubMed Central

Wiskott-Aldrich Syndrome (WAS) is an inherited immunodeficiency caused by mutations in the gene encoding WASP, a protein regulating the cytoskeleton. Hematopoietic stem/progenitor cell (HSPC) transplants can be curative but, when matched donors are unavailable, infusion of autologous HSPCs modified ex vivo by gene therapy is an alternative approach. We used a lentiviral vector encoding functional WASP to genetically correct HSPCs from three WAS patients and re-infused the cells after reduced-intensity conditioning regimen. All three patients showed stable engraftment of WASP-expressing cells and improvements in platelet counts, immune functions, and clinical score. Vector integration analyses revealed highly polyclonal and multi-lineage haematopoiesis resulting from the gene corrected HSPCs. Lentiviral gene therapy did not induce selection of integrations near oncogenes and no aberrant clonal expansion was observed after 20–32 months. Although extended clinical observation is required to establish long-term safety, lentiviral gene therapy represents a promising treatment for WAS. PMID:23845947

Aiuti, Alessandro; Biasco, Luca; Scaramuzza, Samantha; Ferrua, Francesca; Cicalese, Maria Pia; Baricordi, Cristina; Dionisio, Francesca; Calabria, Andrea; Giannelli, Stefania; Castiello, Maria Carmina; Bosticardo, Marita; Evangelio, Costanza; Assanelli, Andrea; Casiraghi, Miriam; Di Nunzio, Sara; Callegaro, Luciano; Benati, Claudia; Rizzardi, Paolo; Pellin, Danilo; Di Serio, Clelia; Schmidt, Manfred; Von Kalle, Christof; Gardner, Jason; Mehta, Nalini; Neduva, Victor; Dow, David J.; Galy, Anne; Miniero, Roberto; Finocchi, Andrea; Metin, Ayse; Banerjee, Pinaki; Orange, Jordan; Galimberti, Stefania; Valsecchi, Maria Grazia; Biffi, Alessandra; Montini, Eugenio; Villa, Anna; Ciceri, Fabio; Roncarolo, Maria Grazia; Naldini, Luigi

2015-01-01

174

Directed Therapy: An Approach to the Improved Treatment of Exfoliation syndrome  

PubMed Central

Exfoliation syndrome (XFS) is an age-related, generalized disorder of the extracellular matrix characterized by the production and progressive accumulation of a fibrillar extracellular material in many ocular tissues and is the most common identifiable cause of open-angle glaucoma worldwide. Exfoliation syndrome plays an etiologic role in open-angle glaucoma, angle-closure glaucoma, cataract, and retinal vein occlusion. It is accompanied by an increase in serious complications at the time of cataract extraction, such as zonular dialysis, capsular rupture, and vitreous loss. It is associated systemically with an increasing number of vascular disorders, hearing loss, and Alzheimer's disease. Exfoliation syndrome appears to be a disease of elastic tissue microfibrils. Directed therapy simply means devising specific treatments for specific diseases. There was little incentive to attempt to distinguish between various open-angle glaucomas if the treatments were essentially the same. However, this view also prevented the application of directed therapy in those instances in which such was available and applicable. Pilocarpine has multiple beneficial actions in eyes with XFS. Not only does it lower IOP, but by increasing aqueous outflow, it should enable the trabecular meshwork to clear more rapidly, and by limiting pupillary movement, should slow the progression of the disease. Theoretically, miotics should be the first line of treatment. Pilocarpine 2% q.h.s. can provide sufficient limitation of pupillary mobility without causing these side effects. In 2007, two common single nucleotide polymorphisms in the coding region of the lysyl oxidase-like 1 (LOXL1) gene located on chromosome 15 were specifically associated with XFS and XFG. LOXL1 is a member of the lysyl oxidase family of enzymes, which are essential for the formation, stabilization, maintenance, and remodelling of elastic fibers and prevent age-related loss of elasticity of tissues. LOXL1 protein is a major component of exfoliation deposits and appears to play a role in its accumulation and in concomitant elastotic processes in intra- and extraocular tissues of XFS patients. This discovery should open the way to new approaches and directions of therapy for this protean disorder. PMID:20142958

Angelilli, Allison; Ritch, Robert

2009-01-01

175

Shockwave Therapy in the Management of Complex Regional Pain Syndrome in Medial Femoral Condyle of the Knee  

Microsoft Academic Search

The aim of this prospective study was to assess the efficacy of shockwave (SW) therapy in the management of complex regional pain syndrome (CRPS). In this study, 30 patients (pts) who were affected by CRPS of the medial femoral condyle and unresponsive to previous standard physiotherapeutic and pharmacological treatment underwent 3 SW sessions at 72-h intervals, each consisting of 4000

Angela Notarnicola; Lorenzo Moretti; Silvio Tafuri; Antonio Panella; Marco Filipponi; Alessio Casalino; Michele Panella; Biagio Moretti

2010-01-01

176

Rituximab and Chronic Plasmapheresis Therapy of Nephrotic Syndrome in Renal Transplantation Patients With Recurrent Focal Segmental Glomerulosclerosis  

Microsoft Academic Search

Focal segmental golmerulosclerosis (FSGS) recurs in 30% of patients with FSGS who received a first renal transplant and in more than 80% of patients receiving a second transplant after a recurrence. Plasmapheresis (PP) can reduce proteinuria and even induce complete remission of proteinuria. We studied the effects of rituximab therapy associated with chronic PP treatment of nephrotic syndrome among 3

M. Rodríguez-Ferrero; J. Ampuero; F. Anaya

2009-01-01

177

Novel Development of Remitting Seronegative Symmetrical Synovitis with Pitting Edema (RS3PE) Syndrome due to Insulin Therapy  

PubMed Central

Patient: Male, 67 Final Diagnosis: Remitting seronegative symmetrical synovitis with pitting edema (RS3PE) syndrome Symptoms: Bilateral wrist swelling Medication: — Clinical Procedure: — Specialty: Rheumatology Objective: Unusual or unexpected effect of treatment Background: Remitting seronegative symmetrical synovitis with pitting edema (RS3PE) syndrome is a rare clinical entity characterized by the sudden onset of inflammatory arthritis and marked pitting edema on upper and lower extremities. RS3PE is considered a rheumatic process distinct from rheumatoid arthritis, which may occasionally represent a paraneoplastic syndrome. Case Report: Herein, we describe a rare case of RS3PE associated with insulin therapy in a patient with no evidence of underlying malignancy. Conclusions: To the best of our knowledge, this is the first case report of RS3PE associated with insulin therapy. Physicians should look at the introduction of drugs as possible triggers for the development of RS3PE. PMID:24696753

Mainali, Naba Raj; Schmidt, Torrey R.; Alweis, Richard; George, David L.

2014-01-01

178

Cross-Sectional Study of Patients With Onset of Acute Coronary Syndrome During Statin Therapy  

PubMed Central

Background Although statin therapy significantly reduces cardiovascular morbidity and mortality, atherosclerotic plaque progresses in some patients taking statins. This study investigated the factors associated with onset of acute coronary syndrome (ACS) early after the initiation of statin therapy. Methods Consecutive patients taking statins who presented with ACS (n = 64) were divided into < 1-year and > 1-year groups based on the duration of statin therapy. Patient characteristics, coronary risk factors, lesion locations, and percutaneous intervention procedures were compared between groups. Results The < 1-year group was significantly younger (57.6 ± 11.9 years vs. 76.6 ± 9.1 years, P < 0.01), had significantly higher body mass index (27.22 ± 4.20 kg/m2 vs. 24.60 ± 4.65 kg/m2, P < 0.05), higher proportion of males (94% vs. 70%, P < 0.05), higher proportion of current smokers (61% vs. 17%, P < 0.01), and lower proportions taking aspirin and calcium antagonists (both 17% vs. 57%, P < 0.05) than the > 1-year group. In the < 1-year group, there were significant correlations between the low-density lipoprotein cholesterol (LDL-C) and triglyceride (TG) levels (r = 0.649, P = 0.004) and between the TG and hemoglobin (Hb)A1c levels (r = 0.552, P = 0.018), but these correlations were not observed a year before admission. TG level was the only parameter associated with LDL-C and HbA1c levels. Conclusions A linear correlation between the LDL-C and TG levels, obesity, older age, male sex, and smoking may be associated with increased risk of onset of ACS early after the initiation of statin therapy. Prospective cohort studies are needed to further explore these interactions. PMID:25780481

Akuzawa, Nobuhiro; Hatori, Takashi; Imai, Kunihiko; Kitahara, Yonosuke; Kurabayashi, Masahiko

2015-01-01

179

Increased Atherothrombotic Burden in Patients with Diabetes Mellitus and Acute Coronary Syndrome: A Review of Antiplatelet Therapy  

PubMed Central

Patients with diabetes mellitus presenting with acute coronary syndrome have a higher risk of cardiovascular complications and recurrent ischemic events when compared to nondiabetic counterparts. Different mechanisms including endothelial dysfunction, platelet hyperactivity, and abnormalities in coagulation and fibrinolysis have been implicated for this increased atherothrombotic risk. Platelets play an important role in atherogenesis and its thrombotic complications in diabetic patients with acute coronary syndrome. Hence, potent platelet inhibition is of paramount importance in order to optimise outcomes of diabetic patients with acute coronary syndrome. The aim of this paper is to provide an overview of the increased thrombotic burden in diabetes and acute coronary syndrome, the underlying pathophysiology focussing on endothelial and platelet abnormalities, currently available antiplatelet therapies, their benefits and limitations in diabetic patients, and to describe potential future therapeutic strategies to overcome these limitations. PMID:22347666

Balasubramaniam, Karthik; Viswanathan, Girish N.; Marshall, Sally M.; Zaman, Azfar G.

2012-01-01

180

Synthesis of ganglioside epitopes for oligosaccharide specific immunoadsorption therapy of Guillian-Barré syndrome.  

PubMed

Guillain-Barré syndrome is a postinfectious, autoimmune neuropathy resulting in neuromuscular paralysis. Auto-antibodies, often induced by bacterial infection, bind to human gangliosides possessing monosialoside and diasialoside epitopes and impair the function of nerve junctions, where these ganglioside structures are highly enriched. Truncated gangliosides representive of GD3, GQ1b and GM2 epitopes have been synthesized as methyl glycosides and as a glycosides of an eleven carbon tether. The synthetic oligosaccharide ligands are structural mimics of these highly complex ganglioside epitopes and via their ability to neutralize or remove auto-antibodies have the potential for therapy, either as soluble blocking ligands administered systemically, or as immuno-affinity ligands for use as extracorporeal immunoadsorbents. PMID:15064799

Andersen, Sřren M; Ling, Chang-Chun; Zhang, Ping; Townson, Kate; Willison, Hugh J; Bundle, David R

2004-04-21

181

Complete atrioventricular block associated with dapsone therapy: a rare complication of dapsone-induced hypersensitivity syndrome.  

PubMed

Dapsone is still widely used for a range of infectious and inflammatory diseases. A potential severe side-effect, known as dapsone-induced hypersensitivity syndrome (DHS), may occur. DHS is characterized by fever, skin rashes, lymphadenopathy and multiorgan dysfunction manifesting as hepatitis, cholangitis, pneumonitis, colitis, thyroiditis and myocarditis. However, DHS-associated complete atrioventricular block has not previously been reported. We describe here a 45-year-old Chinese woman who developed DHS after 5 weeks of dapsone therapy for pustular palmoplantar psoriasis. In addition to typical DHS symptoms, she experienced several episodes of syncope as a result of complete atrioventricular block which was successfully reversed with a permanent pacemaker implantation. PMID:19583683

Zhu, K J; He, F T; Jin, N; Lou, J X; Cheng, H

2009-08-01

182

Neurologic improvement without angiographic improvement after antithyroid therapy in a patient with Moyamoya syndrome.  

PubMed

Moyamoya disease with special complications, including Graves' disease, is called as moyamoya syndrome. A 22-year-old Japanese woman had left middle cerebral artery (MCA) territory infarction complicated with Graves' disease. She had right-sided hemiparesis that deteriorated on day 8 with the infarct growth and thyrotoxicosis. On angiogram, the left MCA was occluded at the origin without moyamoya vessels. Positron emission tomography (PET) revealed misery-perfusion phenomenon in the left MCA territory. After initiation of the antithyroid therapy, her hemiparesis became milder. Seventeen months later, her thyroid function was normalized and net-like collateral moyamoya vessels proliferated in the left MCA territory. Misery-perfusion phenomenon persisted on PET. This report is unique in the point of neurologic recovery of the moyamoya patient right after initiation of antithyroid medication without radiological improvement. PMID:24119625

Ishigami, Akiko; Toyoda, Kazunori; Suzuki, Rieko; Miyashita, Fumio; Iihara, Koji; Minematsu, Kazuo

2014-01-01

183

Habit Reversal Therapy for Body-Focused Repetitive Behaviors in Williams Syndrome: A Case Study  

PubMed Central

Williams syndrome (WS) is genetic neurodevelopmental disorder with a well-characterized cognitive and behavioral phenotype. Research has consistently demonstrated high rates of psychopathology in this population; however, little research has examined the use of empirically-supported psychosocial interventions in those with WS. The current case study reports on the use of Habit Reversal Therapy (HRT) to treat multiple body-focused repetitive behaviors in a child with WS. Although HRT is a well-established cognitive-behavioral intervention for body-focused repetitive behaviors, it has been infrequently used in populations with developmental disabilities. An etiologically-informed approach was used to adapt HRT to fit the known behavioral and cognitive phenotype of WS. Results suggest that HRT may be beneficial for this population. Modified treatment elements are described and future research areas highlighted. PMID:24357918

Klein-Tasman, Bonita P.

2013-01-01

184

Clobazam as an adjunctive therapy in treating seizures associated with Lennox–Gastaut syndrome  

PubMed Central

Lennox–Gastaut syndrome (LGS) is a devastating childhood epilepsy syndrome characterized by the occurrence of multiple types of seizures and cognitive decline. Most children suffer from frequent seizures that are refractory to current medical management. Recent clinical trials have suggested that addition of clobazam may improve the clinical outcome for some LGS patients. Although clobazam has been available for over five decades, it has only recently been approved by the US Food and Drug Administration for this indication. As a 1,5-benzodiazepine, clobazam is structurally related to the widely used 1,4-benzodiazepines, which include diazepam. Clobazam has been shown to modulate GABAergic neurotransmission by positive allosteric modulation of GABAA receptors, and to increase expression of transporters for both GABA and glutamate. The active metabolite n-desmethylclobazam (norclobazam) also modulates GABAA receptors, and the relative importance of these two compounds in the clinical effectiveness of clobazam remains an open question. Clinical trials involving clobazam as an addon therapy in a variety of pediatric epilepsy populations have found a significant improvement in seizure control. In patients with LGS, clobazam may have greatest efficacy for drop seizures. Longstanding clinical experience suggests that clobazam is a safe and well tolerated antiepileptic drug with infrequent and mild adverse effects. These results suggest that adjunctive treatment with clobazam may be a reasonable option for LGS patients, particularly those who are treatment-resistant. PMID:22128252

Leahy, Jennifer T; Chu-Shore, Catherine J; Fisher, Janet L

2011-01-01

185

Genome sequencing in myelodysplastic syndromes: can molecular mutations predict benefit from hypomethylating agent therapy?  

PubMed

Evaluation of: Bejar R, Lord A, Stevenson K, et al. TET2 mutations predict response to hypomethylating agents in myelodysplastic syndrome patients. Blood 2014 Oct 23;124(17):2705-12. Patients with myelodysplastic syndromes (MDS) have clinically variable courses even within the same prognostic subgroups. Although hypomethylating agents (HMAs) have been shown to improve outcomes in patients with high-risk MDS, many patients do not derive benefit. There is an urgent clinical need to identify patients with low probability of benefiting from HMAs but no reliable clinical predictors or biomarkers have been discovered to date. Although some recurrent molecular mutations in MDS carry independent prognostic value, their ability to predict benefit from HMAs is not clear. Here, we discuss an important article in which sequencing from samples of 213 patients identified recurrent mutations associated with response to HMAs. Although an important step in the right direction, the clinical implications of these findings are far from optimal and identification of biomarkers that can reliably predict benefit from HMAs and other therapies in patients with MDS remains a top clinical and a research priority. PMID:25697572

Lee, Eun-Ju; Zeidan, Amer M

2015-04-01

186

Molecular analysis, pathogenic mechanisms, and readthrough therapy on a large cohort of Kabuki syndrome patients.  

PubMed

Kabuki syndrome (KS) is a multiple congenital anomalies syndrome characterized by characteristic facial features and varying degrees of mental retardation, caused by mutations in KMT2D/MLL2 and KDM6A/UTX genes. In this study, we performed a mutational screening on 303 Kabuki patients by direct sequencing, MLPA, and quantitative PCR identifying 133 KMT2D, 62 never described before, and four KDM6A mutations, three of them are novel. We found that a number of KMT2D truncating mutations result in mRNA degradation through the nonsense-mediated mRNA decay, contributing to protein haploinsufficiency. Furthermore, we demonstrated that the reduction of KMT2D protein level in patients' lymphoblastoid and skin fibroblast cell lines carrying KMT2D-truncating mutations affects the expression levels of known KMT2D target genes. Finally, we hypothesized that the KS patients may benefit from a readthrough therapy to restore physiological levels of KMT2D and KDM6A proteins. To assess this, we performed a proof-of-principle study on 14 KMT2D and two KDM6A nonsense mutations using specific compounds that mediate translational readthrough and thereby stimulate the re-expression of full-length functional proteins. Our experimental data showed that both KMT2D and KDM6A nonsense mutations displayed high levels of readthrough in response to gentamicin treatment, paving the way to further studies aimed at eventually treating some Kabuki patients with readthrough inducers. PMID:24633898

Micale, Lucia; Augello, Bartolomeo; Maffeo, Claudia; Selicorni, Angelo; Zucchetti, Federica; Fusco, Carmela; De Nittis, Pasquelena; Pellico, Maria Teresa; Mandriani, Barbara; Fischetto, Rita; Boccone, Loredana; Silengo, Margherita; Biamino, Elisa; Perria, Chiara; Sotgiu, Stefano; Serra, Gigliola; Lapi, Elisabetta; Neri, Marcella; Ferlini, Alessandra; Cavaliere, Maria Luigia; Chiurazzi, Pietro; Monica, Matteo Della; Scarano, Gioacchino; Faravelli, Francesca; Ferrari, Paola; Mazzanti, Laura; Pilotta, Alba; Patricelli, Maria Grazia; Bedeschi, Maria Francesca; Benedicenti, Francesco; Prontera, Paolo; Toschi, Benedetta; Salviati, Leonardo; Melis, Daniela; Di Battista, Eliana; Vancini, Alessandra; Garavelli, Livia; Zelante, Leopoldo; Merla, Giuseppe

2014-07-01

187

Molecular Analysis, Pathogenic Mechanisms, and Readthrough Therapy on a Large Cohort of Kabuki Syndrome Patients  

PubMed Central

Kabuki syndrome (KS) is a multiple congenital anomalies syndrome characterized by characteristic facial features and varying degrees of mental retardation, caused by mutations in KMT2D/MLL2 and KDM6A/UTX genes. In this study, we performed a mutational screening on 303 Kabuki patients by direct sequencing, MLPA, and quantitative PCR identifying 133 KMT2D, 62 never described before, and four KDM6A mutations, three of them are novel. We found that a number of KMT2D truncating mutations result in mRNA degradation through the nonsense-mediated mRNA decay, contributing to protein haploinsufficiency. Furthermore, we demonstrated that the reduction of KMT2D protein level in patients’ lymphoblastoid and skin fibroblast cell lines carrying KMT2D-truncating mutations affects the expression levels of known KMT2D target genes. Finally, we hypothesized that the KS patients may benefit from a readthrough therapy to restore physiological levels of KMT2D and KDM6A proteins. To assess this, we performed a proof-of-principle study on 14 KMT2D and two KDM6A nonsense mutations using specific compounds that mediate translational readthrough and thereby stimulate the re-expression of full-length functional proteins. Our experimental data showed that both KMT2D and KDM6A nonsense mutations displayed high levels of readthrough in response to gentamicin treatment, paving the way to further studies aimed at eventually treating some Kabuki patients with readthrough inducers. PMID:24633898

Micale, Lucia; Augello, Bartolomeo; Maffeo, Claudia; Selicorni, Angelo; Zucchetti, Federica; Fusco, Carmela; De Nittis, Pasquelena; Pellico, Maria Teresa; Mandriani, Barbara; Fischetto, Rita; Boccone, Loredana; Silengo, Margherita; Biamino, Elisa; Perria, Chiara; Sotgiu, Stefano; Serra, Gigliola; Lapi, Elisabetta; Neri, Marcella; Ferlini, Alessandra; Cavaliere, Maria Luigia; Chiurazzi, Pietro; Monica, Matteo Della; Scarano, Gioacchino; Faravelli, Francesca; Ferrari, Paola; Mazzanti, Laura; Pilotta, Alba; Patricelli, Maria Grazia; Bedeschi, Maria Francesca; Benedicenti, Francesco; Prontera, Paolo; Toschi, Benedetta; Salviati, Leonardo; Melis, Daniela; Di Battista, Eliana; Vancini, Alessandra; Garavelli, Livia; Zelante, Leopoldo; Merla, Giuseppe

2014-01-01

188

Emerging antiplatelet therapy for coronary artery disease and acute coronary syndrome.  

PubMed

Antiplatelet therapy is used widely with proven benefit for the prevention of further ischemic cardiac complications in patients with known coronary artery disease (CAD) and a history of acute coronary syndrome (ACS). The limitations of conventional antiplatelet therapy with aspirin, clopidogrel, or prasugrel, as well as the fact that rates of recurrent ischemic events still remain high with use of these agents, underscore the need to investigate alternate agents that may further reduce event rates while limiting bleeding risk. The selection of antiplatelet therapy is further influenced by the following: ticagrelor was approved in July 2011 by the United States Food and Drug Administration (FDA), and clopidogrel is slated to become available as a generic productin 2012. We provide an overview of emerging agents for the treatment of CAD and ACS, including the reversible P2Y(12) antagonists ticagrelor, cangrelor, and elinogrel, and a new class of oral protease-activated receptor-1 (PAR-1) inhibitors, vorapaxar and atopaxar.The recently approved P2Y(12) antagonists prasugrel and ticagrelor demonstrate enhanced ability to prevent adverse cardiac outcomes. However, this comes at a cost of a potential increased risk of bleeding. New adverse effects have also emerged, including dyspnea for all of the reversible P2Y(12) antagonists (ticagrelor, cangrelor, and elinogrel) and ventricular pauses for ticagrelor. In addition, the newer P2Y(12) antagonists have a faster onset and offset. Two of these agents, cangrelor and elinogrel, are available as intravenous formulations, which may provide additional benefits in patients who undergo coronary artery bypass graft (CABG) surgery. Trials with the PAR-1 inhibitors have also shown trends toward reductions in cardiac events, but not without the possibility of increased bleeding. More than ever, as the arsenal of antiplatelet therapy expands, health care providers need to understand the pharmacologic and pharmacodynamic differences between conventional and emerging antiplatelet therapies for patients with ACS and CAD. Health care providers must also carefully assess patient-specific factors such as risk of thrombosis, concomitant disease states, age, drug adherence, and aspirin dose, and plan for those patients who will be undergoing CABG when selecting antiplatelet therapy in order to optimally balance bleeding and thrombosis risk. PMID:22392457

Packard, Kathleen A; Campbell, Jennifer A; Knezevich, Jon T; Davis, Estella M

2012-03-01

189

Cell and Gene Therapy for Genetic Diseases: Inherited Disorders Affecting the Lung and Those Mimicking Sudden Infant Death Syndrome  

PubMed Central

Abstract Some of the first human gene therapy trials targeted diseases of the lung and provided important information that will continue to help shape future trials. Here we describe both cell and gene therapies for lung diseases such as cystic fibrosis and alpha-1 antitrypsin disorder as well as fatty acid oxidation disorders that mimic sudden infant death syndrome (SIDS). Human clinical gene therapy trials for cystic fibrosis and alpha-1 antitrypsin have been performed using a variety of vectors including adenovirus, adeno-associated virus, and nonviral vectors. No human clinical gene therapy trials have been performed for disorders of fatty acid oxidation; however, important proof-of-principle studies have been completed for multiple fatty acid oxidation disorders. Important achievements have been made and have yet to come for cell and gene therapies for disorders of the lung and those mimicking SIDS. PMID:22642257

Keeler, Allison M.

2012-01-01

190

Un mod`ele non-lineaire de proliferation cellulaire : extinction des cellules et invariance  

E-print Network

Un mod`ele non-lin´eaire de prolif´eration cellulaire : extinction des cellules et invariance ´etudions un syst`eme non-lin´eaire structur´e en ^age et maturit´e d´ecrivant la production des cellules-locale dans la variable maturit´e. Nous prouvons que l'unicit´e des solutions ne d´epend que des cellules de

Paris-Sud XI, Université de

191

Correction of Murine Bernard–Soulier Syndrome by Lentivirus-mediated Gene Therapy  

PubMed Central

Bernard–Soulier syndrome (BSS) is an inherited bleeding disorder caused by a defect in the platelet glycoprotein (GP) Ib-IX-V complex. The main treatment for BSS is platelet transfusion but it is often limited to severe bleeding episodes or surgical interventions due to the risk of alloimmunization. We have previously reported successful expression of human GPIb? (hGPIb?) in human megakaryocytes using a lentiviral vector (LV) encoding human GP1BA under control of the platelet-specific integrin ?IIb promoter (2bIb?). In this study, we examined the efficacy of this strategy for the gene therapy of BSS using GPIb?null as a murine model of BSS. GPIb?null hematopoietic stem cells (HSC) transduced with 2bIb? LV were transplanted into lethally irradiated GPIb?null littermates. Therapeutic levels of hGPIb? expression were achieved that corrected the tail bleeding time and improved the macrothrombocytopenia. Sequential bone marrow (BM) transplants showed sustained expression of hGPIb? with similar phenotypic correction. Antibody response to hGPIb? was documented in 1 of 17 total recipient mice but was tolerated without any further treatment. These results demonstrate that lentivirus-mediated gene transfer can provide sustained phenotypic correction of murine BSS, indicating that this approach may be a promising strategy for gene therapy of BSS patients. PMID:22044935

Kanaji, Sachiko; Kuether, Erin L; Fahs, Scot A; Schroeder, Jocelyn A; Ware, Jerry; Montgomery, Robert R; Shi, Qizhen

2012-01-01

192

[Long-term management of post acute coronary syndrome with oral antiplatelet therapy].  

PubMed

Cardiovascular disease is the primary cause of early death and morbidity in the industrialized world and is becoming a growing problem in many developing countries. Coagulation inhibitors play a major role in the management of the acute phase of ACS whether in association with reperfusion strategies or not. Currently, and in accordance with the results of major randomised studies, for medium and long-term management, the association of Clopidogrel and aspirin is the treatment of choice. However, despite the recognised benefits of this therapeutic strategy and above all the recommendations of learned societies, which have placed this bi-therapy in class I, according to national and international registries it is still underused. Moreover, all of these registries have confirmed, in the real world, the negative impact of not prescribing this antiplatelet therapy on morbidity and mortality after both ST and non-ST elevation acute coronary syndrome. which shows the difficulty of applying to everyday clinical practice the results of major randomised cohorts. PMID:17719355

Lorgis, L; Zeller, M; Beer, J-C; Buffet, P; L'Huillier, I; Nguyen, C; Schaad, F; Obbee, P; Lagrost, A-C; Cottin, Y

2007-06-01

193

Antithrombotic Therapy in Patients with Acute Coronary Syndrome in the Intermountain Heart Collaborative Study  

PubMed Central

Objective. To determine factors associated with single antiplatelet (SAP) or dual antiplatelet (DAP) therapy and anticoagulants (AC) use in hospital and after discharge among patients with acute coronary syndrome (ACS). Methods. We evaluated 5,294 ACS patients in the Intermountain Heart Collaborative Study from 2004 to 2009. Multivariable logistic regressions were used to determine predictors of AC or AP use. Results. In hospital, 99% received an AC, 79% DAP, and 19% SAP; 78% had DAP + AC. Coronary stents were the strongest predictors of DAP use in hospital compared to SAP (P < 0.001). After discharge, 77% received DAP, 20% SAP, and 9% AC; 5% had DAP + AC. DAP compared to SAP was less likely for patients on AC (odds ratio [OR] = 0.30, P < 0.0001) after discharge. Placement of a stent increased the likelihood of DAP (bare metal: OR = 54.8, P < 0.0001; drug eluting: OR = 59.4, P < 0.0001). 923 had atrial fibrillation and 337 had a history of venous thromboembolism; these patients had increased use of AC (29% and 40%, resp.). Conclusion. While in-hospital use of AC was nearly universal, postdischarge AC use was rare. Concern for providing the best antithrombotic therapy, while maintaining an acceptable bleeding risk, may explain the selection decisions. PMID:25632367

Klaskala, Winslow; Woller, Scott C.; Horne, Benjamin D.; Bunch, T. Jared; Le, Viet T.; Mills, Roger M.; Muhlestein, Joseph B.

2015-01-01

194

Russell Silver syndrome: a perspective on growth and the influence of growth hormone therapy.  

PubMed

A 6 years male child was referred to our Endocrinology clinic with complaints of failure to thrive and he displayed the characteristic features of Russell Silver Syndrome which included short stature, relative macrocephaly, triangular facies and bilateral clinodactyly. He had a birth weight of 2.14 kg and an expected target height of 170 cm. He was subjected to a hormonal analysis which revealed a normal thyroid profile, but low serum markers of growth namely IGF-1=68 ng/ml (52-297 ng/ml) and basal growth hormone (GH) (1.5 ?g/l). No defects were detected on MRI of the sella. Therefore a growth hormone stimulation test with Clonidine was performed which confirmed complete GH deficiency (at 0 min=0.16 ?g/l, 60 min=0.27 ?g/l, 120 min=4.73 ?g/l). He was commenced on rhGH therapy at 8 years of age (height=102 cm, SDS=-4.53), due to financial restraints. Following initiation of GH therapy (1.5 IU/day) for 19 months, a height gain of 15 cm was obtained (Height=117 cm, SDS=-3.05). Bone age at 9 yr. was between 7-8 years. PMID:23087879

Mascarenhas, J V; Ayyar, Vageesh S

2012-09-01

195

A severe course of Cronkhite-Canada syndrome and the review of clinical features and therapy in 49 Chinese patients.  

PubMed

We describe a severe case of Cronkhite-Canada syndrome and review the clinical features and therapy in 49 Chinese patients. A 67-year-old man who underwent severe chronic diarrhea had typical clinical manifestations of hyperpigmentation, hair loss, and dystrophic changes in the fingernails. Although sufficient nutrition support and other therapies reported in the literature were provided, the patient died of systemic failure one year later. Cronkhite-Canada syndrome is characterized by generalized gastrointestinal polyps associated with hyperpigmentation, hair loss, and onycholysis. Anemia, positive stool occult blood, serum electrolyte disturbances, and low serum proteins are the main clinical features of patients with Cronkhite-Canada syndrome. Most patients were diagnosed by esophagogastroduodenoscopy and/or colonoscopy, and polyps were found in the entire alimentary tract, except the notable exception of the esophagus. The polyp-like samples of mucosa, hyperplasia, and adenoma were characterized by acute/chronic inflammation. Four cases were complicated with cancer. The treatment of Cronkhite-Canada syndrome includes symptomatic and support therapy, administration of corticosteroids, antibiotics and acid inhibitors, therapeutic endoscopy, and surgery. While the mortality rate was reported as 47.3% (9/19), some patients may live a long life with controlled symptoms. PMID:24226722

She, Qiang; Jiang, Jian-Xia; Si, Xin-Min; Tian, Xue-Ying; Shi, Rui-Hua; Zhang, Guo-Xin

2013-01-01

196

Conventional insulin vs insulin infusion therapy in acute coronary syndrome diabetic patients  

PubMed Central

AIM: To evaluate the impact on glucose variability (GLUCV) of an nurse-implemented insulin infusion protocol when compared with a conventional insulin treatment during the day-to-day clinical activity. METHODS: We enrolled 44 type 2 diabetic patients (n = 32 males; n = 12 females) with acute coronary syndrome (ACS) and randomy assigned to standard a subcutaneous insulin treatment (n = 23) or a nurse-implemented continuous intravenous insulin infusion protocol (n = 21). We utilized some parameters of GLUCV representing well-known surrogate markers of prognosis, i.e., glucose standard deviation (SD), the mean daily ? glucose (mean of daily difference between maximum and minimum glucose), and the coefficient of variation (CV) of glucose, expressed as percent glucose (SD)/glucose (mean). RESULTS: At the admission, first fasting blood glucose, pharmacological treatments (insulin and/or anti-diabetic drugs) prior to entering the study and basal glycated hemoglobin (HbA1c) were observed in the two groups treated with subcutaneous or intravenous insulin infusion, respectively. When compared with patients submitted to standard therapy, insulin-infused patients showed both increased first 24-h (median 6.9 mmol/L vs 5.7 mmol/L P < 0.045) and overall hospitalization ? glucose (median 10.9 mmol/L vs 9.3 mmol/L, P < 0.028), with a tendency to a significant increase in first 24-h glycaemic CV (23.1% vs 19.6%, P < 0.053). Severe hypoglycaemia was rare (14.3%), and it was observed only in 3 patients receiving insulin infusion therapy. HbA1c values measured during hospitalization and 3 mo after discharge did not differ in the two groups of treatment. CONCLUSION: Our pilot data suggest that no real benefit in terms of GLUCV is observed when routinely managing blood glucose by insulin infusion therapy in type 2 diabetic ACS hospitalized patients in respect to conventional insulin treatment PMID:25126402

Arvia, Caterina; Siciliano, Valeria; Chatzianagnostou, Kyriazoula; Laws, Gillian; Quinones Galvan, Alfredo; Mammini, Chiara; Berti, Sergio; Molinaro, Sabrina; Iervasi, Giorgio

2014-01-01

197

Clinical pilot study: efficacy of triple antibiotic therapy in Blastocystis positive irritable bowel syndrome patients  

PubMed Central

Background Blastocystis species are common human enteric parasites. Carriage has been linked to Irritable Bowel Syndrome (IBS). Treatment of Blastocystis spp. with antimicrobials is problematic and insensitive diagnostic methods and re-infection complicate assessment of eradication. We investigated whether triple antibiotic therapy comprising diloxanide furoate, trimethoprim/sulfamethoxazole and secnidazole (TAB) given to diarrhoea-predominant IBS (D-IBS) patients positive for Blastocystis would achieve eradication. Methods In a longitudinal, prospective case study 10 D-IBS Blastocystis-positive patients took 14 days of diloxanide furoate 500 mg thrice daily, trimethoprim/sulfamethoxazole 160/80 mg twice daily and secnidazole 400 mg thrice daily. Faecal specimens were collected at baseline, day 15 and 4 weeks after completion of TAB. Specimens were analysed using faecal smear, culture and polymerase chain reaction (PCR) of the 16 SSU rRNA. Patients kept a concurrent clinical diary. Results Six (60%) patients cleared Blastocystis spp. after TAB, including three who had failed previous therapy. Subtypes detected were ST3 (60%), ST4 (40%), ST1 (20%) and ST7, 8 (10%); four patients had mixed ST infections. Serum immunoglobulin A (IgA) levels were low in 40% of patients. Higher rates of Blastocystis clearance were observed in patients symptomatic for less than a year (Mann–Whitney, p?=?0.032, 95% confidence) with no associations found with age, previous antibiotic therapy, faecal parasite load, ST, IgA level or clinical improvement. Conclusions Clearance of Blastocystis spp. was achieved with TAB in 60% of D-IBS patients, an improvement over conventional monotherapy. Higher clearance rates are needed to facilitate investigation of the relevance of this parasite in clinically heterogenous IBS. PMID:25349629

2014-01-01

198

[A case of Isaacs' syndrome preceding the recurrence of malignant thymoma--generating site of ectopic activity and therapy].  

PubMed

We described a-44-year old male patient with Isaacs' syndrome occurring 8 months before the recurrence of malignant thymoma. Electrophysiological examination suggested that spontaneous muscle activities were generated at the distal terminals of the motor nerves. Double filtration plasmapheresis alleviated the symptoms of neuromyotonia for a short term. Administration of valproic acid had more prolonged effects lasting for 2 months until his death from relapsed thymoma. Our results support that autoimmune mechanisms, especially humoral factors, are playing an important role in the pathogenesis of Isaacs' syndrome and that anti-convulsants therapy is important. PMID:9490901

Matsuda, S; Takahashi, N; Kuwabara, S

1997-10-01

199

Novel Anti-platelet Agents in Acute Coronary Syndrome: Mechanisms of Action and Opportunities to Tailor Therapy.  

PubMed

Dual anti-platelet therapy, most commonly aspirin and clopidogrel, has been the standard of care for over a decade in patients who have experienced acute coronary syndrome, particularly when treated with coronary stenting. However, residual risk in patients receiving dual anti-platelet therapy post-acute coronary syndrome raises an unmet need for alternative therapy to clopidogrel. Consequently, novel anti-platelets agents including the P2Y12 receptor antagonists, such as prasugrel and ticagrelor, have emerged. Furthermore, using new methods to assess genetic polymorphisms and functional phenotypic assessments of platelet reactivity may become important in the development of personalized medicine and in developing tailored approaches to individual treatment. While robust large-scale evidence for genotypic- and phenotypic-guided therapy in improving outcomes is currently lacking, tremendous interest from various stakeholders including researchers, funding agencies, and industry continues to spur research endeavors in this arena. Further investigation is required in this emerging field to potentially offer improved platelet inhibition that may optimize cardioprotection and minimize bleeding risk in patients with acute coronary syndrome. PMID:25732742

Quatromoni, Neha; Tuteja, Sony; Kolanksy, Daniel M; Matthai, William H; Giri, Jay

2015-05-01

200

Forearm Compartment Syndrome following Thrombolytic Therapy for Massive Pulmonary Embolism: A Case Report and Review of Literature.  

PubMed

Use of thrombolytic therapy in pulmonary embolism is restricted in cases of massive embolism. It achieves faster lysis of the thrombus than the conventional heparin therapy thus reducing the morbidity and mortality associated with PE. The compartment syndrome is a well-documented, potentially lethal complication of thrombolytic therapy and known to occur in the limbs involved for vascular lines or venepunctures. The compartment syndrome in a conscious and well-oriented patient is mainly diagnosed on clinical ground with its classical signs and symptoms like disproportionate pain, tense swollen limb and pain on passive stretch. However these findings may not be appropriately assessed in an unconscious patient and therefore the clinicians should have high index of suspicion in a patient with an acutely swollen tense limb. In such scenarios a prompt orthopaedic opinion should be considered. In this report, we present a case of acute compartment syndrome of the right forearm in a 78 years old male patient following repeated attempts to secure an arterial line for initiating the thrombolytic therapy for the management of massive pulmonary embolism. The patient underwent urgent surgical decompression of the forearm compartments and thus managed to save his limb. PMID:23198222

Badge, Ravi; Hemmady, Mukesh

2011-01-01

201

Combination Systemic and Intravitreal Antiviral Therapy in The Management of Acute Retinal Necrosis Syndrome (An American Ophthalmological Society Thesis)  

PubMed Central

Purpose: To compare the outcomes of combination systemic and intravitreal antiviral therapy vs systemic antiviral therapy alone for treating acute retinal necrosis syndrome (ARN). We hypothesize that combination therapy might result in superior visual acuity (VA) and retinal detachment (RD) outcomes vs traditional systemic antiviral therapy alone. Methods: A retrospective, interventional, comparative single-center study of patients with ARN. We reviewed demographic data, herpesvirus diagnoses, polymerase chain reaction (PCR) results, VA, RD, and the use of systemic and intravitreal antiviral therapy. Outcome measures included VA improvement by 2 or more lines, severe visual loss, VA ?20/200, and RD. Results: We studied 29 eyes of 24 patients, treated from 1987 through 2009. Mean age was 42.6 years and mean follow-up was 44.0 months. Twelve patients (14 eyes) were treated with combined systemic and intravitreal antiviral therapy and 12 patients (15 eyes) with systemic therapy alone. Kaplan-Meier survival analysis revealed that patients receiving combination intravitreal and systemic antiviral therapy were more likely to have VA improved by 2 lines or greater (P=.006). Patients receiving combination therapy also showed a decreased incidence of progression to severe visual loss (0.13/patient-years [PY]) compared to patients receiving systemic therapy alone (0.54/PY, P=.02) and had decreased incidence of RD (0.29/PY vs 0.74/PY, P=.03). Conclusions: Combination oral and intravitreal antiviral therapy may improve visual and functional outcomes in patients with ARN. Clinicians should consider prompt administration of combination systemic and intravitreal antiviral therapy as first-line treatment for patients with clinical features of ARN. PMID:24385671

Flaxel, Christina J.; Yeh, Steven; Lauer, Andreas K.

2013-01-01

202

Recurrent Stent Thrombosis in a Patient with Antiphospholipid Syndrome and Dual Anti-Platelet Therapy Non-Responsiveness  

PubMed Central

Antiphospholipid syndrome (APS), the most common acquired hypercoagulable condition, is diagnosed by persistent presence of antiphospholipid antibodies and episodes of vascular thrombosis. It may be an important predisposing factor for stent thrombosis, resulting in poor outcomes. Also, anti-platelet therapy non-responsiveness is associated with stent thrombosis. We report a case of a 39-year-old man who after undergoing successful percutaneous coronary intervention for significant coronary artery disease suffered repeated stent thrombosis events leading to ST-segment elevation myocardial infarction. Eventually, he underwent coronary artery bypass surgery because of uncontrolled thrombosis and was diagnosed as having APS and dual antiplatelet therapy non-responsiveness. PMID:25653707

Lee, You-Hong; Tahk, Seung-Jea; Hong, You-Sun; Park, Jin-Sun; Seo, Kyoung-Woo; Choi, Yong-Woo; Noh, Choong-Kyun

2015-01-01

203

Metformin therapy improves the menstrual pattern with minimal endocrine and metabolic effects in women with polycystic ovary syndrome  

Microsoft Academic Search

Objective: To determine the clinical, hormonal, and biochemical effects of 4–6 months of metformin therapy in obese patients with polycystic ovary syndrome (PCOS).Design: Prospective study.Setting: The Gynecological Endocrine Unit of University Central Hospital, Oulu, Finland.Patient(s): Twenty obese patients with PCOS.Intervention(s): Patients were treated with 0.5 g of metformin three times daily for 4–6 months.Main Outcome Measure(s): Clinical symptoms, menstrual pattern,

Laure C. Morin-Papunen; Riitta M. Koivunen; Aimo Ruokonen; Hannu K. Martikainen

1998-01-01

204

Therapy-related patterns of cytogenetic abnormalities in acute myeloid leukemia and myelodysplastic syndrome post polycythemia vera  

Microsoft Academic Search

A minor fraction of patients with polycythemia vera (PV) develop a terminal acute myeloid leukemia (AML) or myelodysplastic\\u000a syndrome (MDS). Analysis of the cytogenetic abnormalities during AML or MDS may help in understanding if this development\\u000a is part of the natural course of the disease or induced by myelosuppressive therapy. Thirty-six cases with AML or MDS post\\u000a PV, collected in

Birgitta Swolin; Stig Rödjer; Jan Westin

2008-01-01

205

Autologous stem cell transplantation in refractory Asherman's syndrome: A novel cell based therapy  

PubMed Central

BACKGROUND: There is substantial evidence that adult stem cell populations exist in human endometrium, and hence it is suggested that either endogenous endometrial stem/progenitor cells can be activated or bone marrow derived stem cells can be transplanted in the uterine cavity for endometrial regeneration in Asherman's syndrome (AS). AIMS AND OBJECTIVES: The objective was to evaluate the role of sub-endometrial autologous stem cell implantation in women with refractory AS in attaining menstruation and fertility. SETTING: Tertiary care referral center. DESIGN: Prospective case series. MATERIALS AND METHODS: Six cases of refractory AS with failed standard treatment option of hysteroscopic adhesiolysis in the past were included. Mononuclear stem cells (MNCs) were implanted in sub-endometrial zone followed by exogenous oral estrogen therapy. Endometrial thickness (ET) was assessed at 3, 6, and 9 months. RESULTS: Descriptive statistics and statistical analysis of study variables was carried out using STATA version 9.0. The mean MNC count was 103.3 × 106 (±20.45) with mean CD34+ count being 203,642 (±269,274). Mean of ET (mm) at 3 months (4.05 ± 1.40), 6 months (5.46 ± 1.36) and 9 months (5.48 ± 1.14) were significantly (P < 0.05) increased from pretreatment level (1.38 ± 0.39). Five out of six patients resumed menstruation. CONCLUSION: The autologous stem cell implantation leads to endometrial regeneration reflected by restoration of menstruation in five out of six cases. Autologous stem cell implantation is a promising novel cell based therapy for refractory AS. PMID:25191021

Singh, Neeta; Mohanty, Sujata; Seth, Tulika; Shankar, Meenakshi; Bhaskaran, Sruthi; Dharmendra, Sona

2014-01-01

206

Molecular pathogenesis and targets for therapy in myelodysplastic syndrome (MDS) and MDS-related leukemias.  

PubMed

The myelodysplastic syndromes (MDS) are a family of disorders that are characterized by ineffective hematopoiesis and evolution to acute myelogenous leukemias (AMLs) that are strikingly refractory to current therapeutic approaches. A substantial proportion of these complex diseases arise in the setting of exposures to environmental or occupational toxins, including cytotoxic therapy for a prior malignancy or other disorder (secondary MDS/AML). On the genomic level, MDS is typified by losses and translocations involving certain key gene segments, with disruption of the normal structure and function of genes that control the balance of proliferation and differentiation in hematopoietic precursors. In addition, MDS cells display impaired responses to diverse cytokines in terms of activating signaling intermediaries that trigger both proliferation and differentiation, and the disruption of the normal flow of biochemical information along the pathways translates into ineffective multilineage hematopoiesis and bone marrow failure. MDS/AMLs provide a fertile testing ground for the development of novel agents and the concomitant molecular dissection of the mechanisms by which these agents induce growth inhibition, terminal differentiation, and eventual cell death. PMID:9466478

Karp, J E

1998-01-01

207

Atypical hemolytic uremic syndrome: from the rediscovery of complement to targeted therapy.  

PubMed

Atypical hemolytic uremic syndrome (aHUS) is a devastating form of renal thrombotic microangiopathy. In the last five years, we have finally witnessed a dramatic improvement in the management of aHUS patients, and three breakthroughs in our understanding of aHUS have led to such an improvement. The first breakthrough was the emergence of a new clinical picture of aHUS (frequency of adult cases, and overall poor renal prognosis despite plasma therapy). The second breakthrough was the identification of complement alternative pathway dysregulation as a major risk factor for aHUS. The third breakthrough was the availability in clinical practice of the first complement inhibitor, the anti-C5 monoclonal antibody, eculizumab. Available data from case series and prospective studies indicate that eculizumab use has dramatically improved the renal prognosis of aHUS. These breakthroughs have prompted the French working group on aHUS to propose a new algorithm for the management of aHUS in children and in adults. This algorithm will evolve as we gain new insights in the pathogenesis and evolution of aHUS in the eculizumab era. PMID:23756030

Fakhouri, Fadi; Frémeaux-Bacchi, Véronique; Loirat, Chantal

2013-09-01

208

Effect of various drugs used in conservative therapy of hepatorenal syndrome: A retrospective drug utilization study  

PubMed Central

Objectives: To analyze the impact of various drugs used in conservative therapy on renal failure and mortality in hepatorenal syndrome (HRS) at a tertiary care teaching hospital. Materials and Methods: Retrospective review of hospital admission records was conducted for case records with HRS as diagnosis. Demographic and clinical data and drug utilization pattern were collected in a pre-designed patient information sheet. Impact of various drugs especially hepatoprotector antioxidant, silymarin, on survival benefits in terms of number of patients alive, change in mean arterial pressure (MAP) and change in serum creatinine at the end of treatment period were estimated by univariate and followed by multivariate analysis. Results: Of the total 89 case records, 31 met the eligibility criteria and were included in the analysis. On multivariate analysis, a significant correlation between use of intravenous fluids (IVFs) and survival benefits was observed (P < 0.05); wherein patients treated with IVFs had an increase in log odds of survival by 2.42 (95% CI = 1.06 to 121.13) as compared to patient not treated with IVF. However, MAP was not affected by any of the treatment modalities. While change in serum creatinine level was not significantly (P = 0.06, regression correlation = ?0.3) correlated with duration of treatment with IVFs. Conclusion: Use of IVFs may be associated with better short-term survival benefits and favor HRS reversal. Use of silymarin as hepatoprotector antioxidant has no beneficial effects on HRS reversal or survival benefits. PMID:25298585

Hiremath, Sharanabasayyaswamy B.; Kamdod, Mohammed Ameeruddin

2014-01-01

209

Comparison of efficacy of kinesiological taping and subacromial injection therapy in subacromial impingement syndrome.  

PubMed

The aim of the study was to compare the efficacy of kinesiological taping and subacromial injection therapy in patients with subacromial impingement syndrome (SIS). Seventy patients diagnosed with SIS were randomly assigned to group 1 (n?=?35, injection group) or group 2 (n?=?35, kinesiological taping group). Betamethasone plus prilocaine was injected to subacromial space in the patients in group 1. In group 2, tape was applied three times for a period of five consecutive days with a 2-day recovery interval. A 3-month exercise program was prescribed for both groups including stretching and strengthening exercises. All patients were assessed at baseline and at 1 and 3 months post-intervention. Assessments were made by visual analog scale (VAS) for pain, range of motion (ROM) measurements, specific tests, and Shoulder Pain and Disability Index (SPADI). Significant differences were detected in VAS and SPADI scores as well as ROM measurements in both groups when compared to baseline (p?>?0.05). No significant differences were detected between the groups except for active flexion degree in favor of group 1 (p?=?0.004). Both kinesiological taping and steroid injection in conjunction with an exercise program were found to be effective in the treatment of SIS. Kinesio taping may be an alternative treatment option in the rehabilitation of SIS especially when a non-invasive technique is needed. PMID:25403253

Suba??, Volkan; Cak?r, Tuncay; Ar?ca, Zuhal; Sar?er, Rahime Nur; Bilgilisoy Filiz, Meral; Kolda? Do?an, Sebnem; Toraman, Naciye Füsun

2014-11-18

210

Gene therapy for Wiskott-Aldrich syndrome--long-term efficacy and genotoxicity.  

PubMed

Wiskott-Aldrich syndrome (WAS) is characterized by microthrombocytopenia, immunodeficiency, autoimmunity, and susceptibility to malignancies. In our hematopoietic stem cell gene therapy (GT) trial using a ?-retroviral vector, 9 of 10 patients showed sustained engraftment and correction of WAS protein (WASP) expression in lymphoid and myeloid cells and platelets. GT resulted in partial or complete resolution of immunodeficiency, autoimmunity, and bleeding diathesis. Analysis of retroviral insertion sites revealed >140,000 unambiguous integration sites and a polyclonal pattern of hematopoiesis in all patients early after GT. Seven patients developed acute leukemia [one acute myeloid leukemia (AML), four T cell acute lymphoblastic leukemia (T-ALL), and two primary T-ALL with secondary AML associated with a dominant clone with vector integration at the LMO2 (six T-ALL), MDS1 (two AML), or MN1 (one AML) locus]. Cytogenetic analysis revealed additional genetic alterations such as chromosomal translocations. This study shows that hematopoietic stem cell GT for WAS is feasible and effective, but the use of ?-retroviral vectors is associated with a substantial risk of leukemogenesis. PMID:24622513

Braun, Christian Jörg; Boztug, Kaan; Paruzynski, Anna; Witzel, Maximilian; Schwarzer, Adrian; Rothe, Michael; Modlich, Ute; Beier, Rita; Göhring, Gudrun; Steinemann, Doris; Fronza, Raffaele; Ball, Claudia Regina; Haemmerle, Reinhard; Naundorf, Sonja; Kühlcke, Klaus; Rose, Martina; Fraser, Chris; Mathias, Liesl; Ferrari, Rudolf; Abboud, Miguel R; Al-Herz, Waleed; Kondratenko, Irina; Maródi, László; Glimm, Hanno; Schlegelberger, Brigitte; Schambach, Axel; Albert, Michael Heinrich; Schmidt, Manfred; von Kalle, Christof; Klein, Christoph

2014-03-12

211

Long-term outcome of immunosuppressive therapy for Japanese patients with lower-risk myelodysplastic syndromes.  

PubMed

To investigate the long-term usefulness of immunosuppressive therapy (IST) for Japanese patients with lower-risk myelodysplastic syndromes, we retrospectively analyzed 29 MDS patients who were treated with cyclosporine A alone or with anti-thymocyte globulin at a single institute in Japan. A total of 58.6 % of patients showed hematological response to IST. Overall survival of all patients was 74.5 % at 5 years and 48.3 % at 10 years. The major adverse event was the elevation of creatinine level (grade 1 and 2). Eleven patients were still on IST at the time of analysis with, at least, some clinical benefits. Pneumonia was the most frequent cause of death (eight of 12 deaths), followed by bleeding (three of 12); most of the patients who died were non-responders. The presence of paroxysmal nocturnal hemoglobinuria-type cells was significantly associated with both response to IST and long-term survival by univariate analysis. The 10-year overall survival of responders (72.2 %) was significantly superior to that of non-responders (15.6 %, P < 0.0001). These results suggest that IST using cyclosporine A provides long-term benefit for Japanese patients with lower-risk MDS. PMID:24254637

Hata, Tomoko; Tsushima, Hideki; Baba, Maki; Imaizumi, Yoshitaka; Taguchi, Jun; Imanishi, Daisuke; Nagai, Kazuhiro; Tomonaga, Masao; Miyazaki, Yasushi

2013-12-01

212

Conservative treatment of carpal tunnel syndrome: comparison between laser therapy and Fascial Manipulation(®).  

PubMed

The etiopathogenesis of Carpal Tunnel Syndrome (CTS) is multifactorial and most cases are classified as idiopathic (Thurston 2013). A randomized controlled trial was performed to compare the effectiveness of Fascial Manipulation(®) (FM) and Low-Level Laser Therapy (LLLT) for CTS. This prospective trial included 42 patients (70 hands with symptoms) with clinical and electroneuromyographic diagnosis of CTS. The patients were randomly assigned to receive multiple sessions of FM or multiple session of LLLT. The Visual Analogic Scale (VAS) and Boston Carpal Tunnel Questionnaire (BCTQ) were performed at baseline, end of treatment and after three months. The group that received FM showed a significant reduction in subjective pain perception and an increased function assessed by BCTQ at the end of the treatment and follow-up. The group that received LLLT showed an improvement in the BCTQ at the end of the treatment but the improvement level was not sustained at the three month follow-up. FM is a valid alternative treatment for CTS. PMID:25603750

Pratelli, Elisa; Pintucci, Marco; Cultrera, Pina; Baldini, Enrico; Stecco, Antonio; Petrocelli, Antonio; Pasquetti, Pietro

2015-01-01

213

Cellules souches et thérapie cellulaire. Contribution au débat éthique  

Microsoft Academic Search

Divergent and sometimes conflicting positions with respect to human stem cells and cell therapy do not merely reflect disagreement among scientists and conflicts of interest. They attest the ethical tension resulting from recent progress in understanding the earliest stages of development of the human being that can be observed in vitro. Can the extremely potent notion of the human person

Denys Pellerin

2002-01-01

214

Risk factors for therapy-related myelodysplastic syndrome and acute myeloid leukemia treated with allogeneic stem cell transplantation  

PubMed Central

Background After successful treatment of malignant diseases, therapy-related myelodysplastic syndrome and acute myeloid leukemia have emerged as significant problems. Design and Methods The aim of this study was to investigate outcome and risk factors in patients with therapy-related myelodysplastic syndrome or acute myeloid leukemia who underwent allogeneic stem cell transplantation. Between 1981 and 2006, 461 patients with therapy-related myelodysplastic syndrome or acute myeloid, a median age of 40 years and a history of solid tumor (n=163), malignant lymphoma (n=133), or other hematologic diseases (n=57) underwent stem cell transplantation and their data were reported to the European Group for Blood and Marrow Transplantation. Results The cumulative incidence of non-relapse mortality and relapse at 3 years was 37% and 31%, respectively. In a multivariate analysis significant factors for relapse were not being in complete remission at the time of transplantation (p=0.002), abnormal cytogenetics (p=0.005), higher patients’ age (p=0.03) and therapy-related myelodysplastic syndrome (p=0.04), while higher non-relapse mortality was influenced by higher patients’ age. Furthermore, there was a marked reduction in non-relapse mortality per calendar year during the study period (p<0.001). The 3-year relapse-free and overall survival rates were 33% and 35%, respectively. In a multivariate analysis significant higher overall survival rates were seen per calendar year (p<0.001), for younger age (<40 years) and normal cytogenetics (p=0.05). Using age (<40 years), abnormal cytogenetics and not being in complete remission at the time of transplantation as risk factors, three different risk groups with overall survival rates of 62%, 33% and 24% could be easily distinguished. Conclusions Allogeneic stem cell transplantation can cure patients with therapy-related myelodysplastic syndrome and acute myeloid leukemia and has markedly improved over time. Non-complete remission, abnormal cytogenetics and higher patients’ age are the most significant factors predicting survival. PMID:19278968

Kröger, Nicolaus; Brand, Ronald; van Biezen, Anja; Zander, Axel; Dierlamm, Judith; Niederwieser, Dietger; Devergie, Agnčs; Ruutu, Tapani; Cornish, Jackie; Ljungman, Per; Gratwohl, Alois; Cordonnier, Catherine; Beelen, Dietrich; Deconinck, Eric; Symeonidis, Argiris; de Witte, Theo

2009-01-01

215

LEGO[R] Therapy and the Social Use of Language Programme: An Evaluation of Two Social Skills Interventions for Children with High Functioning Autism and Asperger Syndrome  

ERIC Educational Resources Information Center

LEGO[R] therapy and the Social Use of Language Programme (SULP) were evaluated as social skills interventions for 6-11 year olds with high functioning autism and Asperger Syndrome. Children were matched on CA, IQ, and autistic symptoms before being randomly assigned to LEGO or SULP. Therapy occurred for 1 h/week over 18 weeks. A no-intervention…

Owens, Gina; Granader, Yael; Humphrey, Ayla; Baron-Cohen, Simon

2008-01-01

216

Multimodal physical therapy management of a 48-year-old female with post-stroke complex regional pain syndrome.  

PubMed

This case report describes a 48-year-old female who presented with complaints of right shoulder pain, hyperesthesias and swelling of the hand along with added symptoms of pain centralization following a cerebrovascular accident. On clinical evaluation, the patient satisfied the Budapest diagnostic criteria for Complex Regional Pain Syndrome (CRPS) type-1. Physical therapy management (1st three sessions) was initially focused on pain neurophysiology education with an aim to reduce kinesiophobia and reconceptualise her pain perception. The patient had an immediate significant improvement in her pain and functional status. Following this, pain modulation in the form of transcutaneous electrical nerve stimulation, kinesio tape application, "pain exposure" physical therapy and exercise therapy was carried out for a period of 7 weeks. The patient had complete resolution of her symptoms which was maintained at a six-month follow-up. PMID:23879307

Anandkumar, Sudarshan; Manivasagam, Murugavel

2014-01-01

217

Upper extremity acute compartment syndrome during tissue plasminogen activator therapy for pulmonary embolism in a morbidly obese patient  

PubMed Central

Introduction Deep vein thrombosis (DVT) and pulmonary embolism (PE) are more frequently observed in morbidly obese patients. Tissue plasminogen activator (tPA) is a thrombolytic agent which dissolves the thrombus more rapidly than conventional heparin therapy and reduces the mortality and morbidity rates associated with PE. Compartment syndrome is a well-known and documented complication of thrombolytic treatment. In awake, oriented and cooperative patients, the diagnosis of compartment syndrome is made based on clinical findings including swelling, tautness, irrational and continuous pain, altered sensation, and severe pain due to passive stretching. These clinical findings may not be able to be adequately assessed in unconscious patients. Presentation of case In this case report, we present compartment syndrome observed, for which fasciotomy was performed on the upper right extremity of a 46-year old morbidly obese, conscious female patient who was receiving tPA due to a massive pulmonary embolism. Discussion Compartment syndrome had occurred due to the damage caused by the repeated unsuccessful catheterisation attempts to the brachial artery and the accompanying tPA treatment. Thus, the bleeding that occurred in the volar compartment of the forearm and the anterior compartment of the arm led to acute compartment syndrome (ACS). After relaxation was brought about in the volar compartment of the forearm and the anterior compartment of the arm, the circulation in the limb was restored. Conclusion As soon as the diagnosis of compartment syndrome is made, an emergency fasciotomy should be performed. Close follow-up is required to avoid wound healing problems after the fasciotomy. PMID:25618841

Tuna, Serkan; Duymus, Tahir Mutlu; Mutlu, Serhat; Ketenci, Ismail Emre; Ulusoy, Ayhan

2015-01-01

218

Cyclosporine therapy monitored with abbreviated area under curve in nephrotic syndrome.  

PubMed

Cyclosporin A (CsA) is an effective therapy for children with long-lasting nephrotic syndrome (NS). Long-term treatment can result in chronic CsA nephropathy (CsAN) and there is controversy concerning its incidence and severity. Trough levels are commonly used to monitor the drug concentration. We report a retrospective clinical and histological analysis of 18 children (12 males, 6 females) with steroid-dependent nephrotic syndrome (15 patients) and partially steroid-sensitive nephrotic syndrome (3 patients) treated with CsA for a long-term period (mean 4.9 years, range 2.2-6.9). Before CsA treatment all patients had normal creatinine clearance. CsA was started at a dose of 5 mg/kg per day administered orally in two divided doses and adjusted to maintain the mean CsA blood concentration between 250 and 350 ng/ml obtained from abbreviated area under the curve (AUC). A renal biopsy was performed after a mean period of 3.9 years (range 2.2-6.2) from the start of CsA treatment. Tubular, interstitial, and arteriolar lesions were evaluated in order to assess CsAN. The mean CsA dose and the mean CsA blood concentration were 4.4 mg/kg per day (range 3.6-5.8) and 276.6 ng/ml (range 162-346), respectively. No child had a worsening creatinine clearance during CsA treatment and follow-up after CsA discontinuation. If compared with the year before the start of CsA treatment, NS relapses and prednisone (PDN) dose significantly decreased during CsA treatment, 4/year versus 0.8/year (P <0.0001) and 0.9 mg/kg per day versus 0.2 mg/kg per day (P <0.0001), respectively. Histological analysis showed 15 patients with minimal change disease and 3 with focal segmental glomerulosclerosis. Clear-cut lesions diagnostic of CsAN were never found and only mild lesions were observed in 5 children (suggestive of CsAN in 2 patients and consistent with CsAN in 3 patients). Long-term CsA treatment is confirmed to be effective in preventing NS relapses and reducing PDN dose. Renal function is not a reliable indicator of CsAN. With the mean CsA blood concentrations used in our patients CsAN presented a low incidence (28%) and was generally mild. Renal biopsy should be performed 2-3 years from the start of long-term CsA treatment, especially if the mean CsA blood concentrations are not regularly monitored. PMID:15602664

Rinaldi, Stefano; Sesto, Antonella; Barsotti, Paola; Faraggiana, Tullio; Sera, Francesco; Rizzoni, Gianfranco

2005-01-01

219

Targeted therapy of short-bowel syndrome with teduglutide: the new kid on the block  

PubMed Central

Extensive intestinal resection impairs the absorptive capacity and results in short-bowel syndrome-associated intestinal failure (SBS-IF), when fluid, electrolyte, acid-base, micro-, and macronutrient homeostasis cannot be maintained on a conventional oral diet. Several factors, including the length and site of the resected intestine, anatomical conformation of the remnant bowel, and the degree of postresection intestinal adaptation determine the disease severity. While mild SBS patients achieve nutritional autonomy with dietary modification (eg, hyperphagia, small frequent meals, and oral rehydration fluids), those with moderate-to-severe disease may develop SBS-IF and become dependent on parenteral support (PS) in the form of intravenous fluids and/or nutrition for sustenance of life. SBS-IF is a chronic debilitating disease associated with a poor quality of life, and carries significant morbidity and health care costs. Medical management of SBS-IF is primarily focused on individually tailored symptomatic treatment strategies, such as antisecretory and antidiarrheal agents to mitigate fluid losses, and PS. However, PS administration is associated with potentially life-threatening complications, such as central venous thromboses, bloodstream infections, and liver disease. In pursuit of a targeted therapy to augment intestinal adaptation, research over the past 2 decades has identified glucagon-like peptide, an intestinotrophic gut peptide that has been shown to enhance intestinal absorptive capacity by causing an increase in the villus length, crypt depth, and mesenteric blood flow and by decreasing gastrointestinal motility and secretions. Teduglutide, a recombinant analog of glucagon-like peptide-2, is the first targeted therapeutic agent to gain approval for use in adult SBS-IF. Teduglutide was shown to result in significant (20%–100%) reduction in PS-volume requirement and have a satisfactory safety profile in three randomized control trials. Further research is warranted to see if reduction in PS dependency translates to improved quality of life and reduced PS-associated complications. PMID:25525380

Vipperla, Kishore; O’Keefe, Stephen J

2014-01-01

220

Diagnosis and therapy of acute respiratory distress syndrome in adults: an international survey.  

PubMed

In an attempt to identify the range of opinions influencing the diagnosis and therapy of patients with the adult respiratory distress syndrome (ARDS), a postal survey was mailed to 3,164 physician members of the American Thoracic Society Critical Care Assembly. The questionnaire asked opinions regarding the factors important in the diagnosis of ARDS and its treatment. Thirty-one percent of physicians surveyed responded within 4 weeks, the vast majority of which were board certified or eligible in Internal Medicine, Pulmonary Disease, and/or Critical Care Medicine. A known predisposing cause, measure of oxygenation efficiency, and a chest radiograph depicting pulmonary edema were reported to be the most important criteria for a clinical and research diagnosis of ARDS. Lung compliance and bronchoalveolar lavage neutrophil or protein content were reportedly less important. The initial treatment of patients with ARDS was reported to be most commonly accomplished using volume-cycled ventilation in the assist/control mode. Nearly half the responders reported using lower tidal volumes (5 to 9 mL/kg) than the traditionally recommended 10 to 15 mL/kg. Most respondents indicated they have intentionally allowed CO2 retention. On average, oxygen toxicity was thought to begin at an FIO2 between 0.5 and 0.6. It was reported that modest levels of positive end-expiratory pressure (PEEP) were used in incremental fashion as FiO2 requirements increased. Perceived indications for insertion of pulmonary artery catheters and compensation of the effects of PEEP on the pulmonary artery occlusion pressure varied widely among the responders. We conclude that reported practice patterns regarding the care of ARDS patients vary widely even within a relatively homogenous group of critical care practitioners. PMID:8904279

Carmichael, L C; Dorinsky, P M; Higgins, S B; Bernard, G R; Dupont, W D; Swindell, B; Wheeler, A P

1996-03-01

221

Subretinal Gene Therapy of Mice With Bardet-Biedl Syndrome Type 1  

PubMed Central

Purpose. To study safety and efficacy of subretinal adeno-associated virus (AAV) vector AAV-Bbs1 injection for treatment of a mouse model of Bardet-Biedl syndrome type 1 (BBS1). Methods. Constructs containing a wild-type (WT) Bbs1 gene with and without a FLAG tag in AAV2/5 vectors were generated. Viral genomes were delivered by subretinal injection to right eyes and sham injections to left eyes at postnatal day 30 (P30) to P60. Transgene expression and BBSome reconstitution were evaluated by immunohistochemistry and Western blotting following sucrose gradient ultracentrifugation. Retinal function was analyzed by electroretinogram (ERG) and structure by optical coherence tomography (OCT). Histology and immunohistochemistry were performed on selected eyes. Results. Expression of FLAG-tagged Bbs1 was demonstrated in photoreceptor cells using antibody directed against the FLAG tag. Coinjection of AAV-GFP demonstrated transduction of 24% to 32% of the retina. Western blotting demonstrated BBS1 protein expression and reconstitution of the BBSome. ERG dark-adapted bright flash b-wave amplitudes were higher in AAV-Bbs1–injected eyes than in sham-injected fellow eyes in more than 50% of 19 animals. Anti-rhodopsin staining demonstrated improved localization of rhodopsin in AAV-Bbs1–treated eyes. WT retinas injected with AAV-Bbs1 with or without a FLAG tag showed outer retinal degeneration on ERG, OCT, and histology. Conclusions. In a knock-in model of BBS1, subretinal delivery of AAV-Bbs1 rescues BBSome formation and rhodopsin localization, and shows a trend toward improved ERG. BBS is challenging to treat with gene therapy due to the stoichiometry of the BBSome protein complex and overexpression toxicity. PMID:23900607

Seo, Seongjin; Mullins, Robert F.; Dumitrescu, Alina V.; Bhattarai, Sajag; Gratie, Daniel; Wang, Kai; Stone, Edwin M.; Sheffield, Val; Drack, Arlene V.

2013-01-01

222

Progesterone - new therapy in mild carpal tunnel syndrome? Study design of a randomized clinical trial for local therapy  

Microsoft Academic Search

BACKGROUND: Local corticosteroid injection for carpal tunnel syndrome (CTS) provides greater clinical improvement in symptoms one month after injection compared to placebo but significant symptom relief beyond one month has not been demonstrated and the relapse of symptoms is possible. Neuroprotection and myelin repair actions of the progesterone was demonstrated in vivo and in vitro study. We report the design

Paolo Milani; Mauro Mondelli; Federica Ginanneschi; Riccardo Mazzocchio; Alessandro Rossi

2010-01-01

223

Treatment results of high dose cabergoline as an adjuvant therapy in six patients with established severe ovarian hyper stimulation syndrome  

PubMed Central

Background: The beneficial role of cabergoline as a prophylactic agent to prevent ovarian hyper stimulation syndrome (OHSS) among high-risk patients has been demonstrated in previous studies. But data for its role as a treatment for established severe OHSS is still limited. We represent the treatment results of high dose oral cabergoline in management of six patients after the syndrome is established. Case: High-dose oral cabergoline (1 mg daily for eight days) was prescribed as an adjuvant to symptomatic treatment for six hospitalized patients with established severe OHSS following infertility treatment cycles. In two cases OHSS resolved rapidly despite the occurrence of ongoing pregnancy. Conclusion: Considering the treatment outcomes of our patients, high dose cabergoline did not eliminate the need for traditional treatments, but it was a relatively effective and safe therapy in management of established severe OHSS, and prevented the increase in its severity following the occurrence of pregnancy. PMID:25469130

Saharkhiz, Nasrin; Akbari Sene, Azadeh; Salehpour, Saghar; Tamimi, Maryam; Vasheghani Farahani, Masoumeh; Sheibani, Kourosh

2014-01-01

224

Antithrombotic therapy for long-term secondary prevention of acute coronary syndrome in high-risk patients.  

PubMed

Patients with acute coronary syndrome (ACS) represent a major clinical burden, because they tend to experience recurrent ischemic events. Acute management of patients with ACS includes combination antithrombotic therapy composed of a parenteral anticoagulant and dual-antiplatelet therapy. Dual-antiplatelet therapy is also recommended for long-term secondary prevention of ACS. Despite advances in the antithrombotic therapies available, clinical trials suggest that patients with ACS still faceă10% risk of another event within 12-15 months of the index event. Certain patient populations, such as elderly patients and those with renal impairment or heart failure, are at higher risk of recurrent ACS events, because these patients have more vascular ischemic and bleeding risk factors than most other patients. Evidence from the GRACE and CRUSADE registries suggests underuse of the guideline-recommended evidence-based therapies for the management of ACS in such patients. This review summarizes the current standard of care for patients with ACS, focusing on long-term secondary antithrombotic strategies. Registry data are used to identify high-risk patient populations; the recent antiplatelet and anticoagulant Phase III trial data are summarized to highlight any patient populations who receive greater or lesser benefit from specific long-term antithrombotic strategies. Guideline recommendations are discussed and suggestions are provided to help improve implementation of long-term secondary prevention strategies and patient prognosis after an ACS event. PMID:25733842

Husted, Steen

2015-01-01

225

Antithrombotic therapy for long-term secondary prevention of acute coronary syndrome in high-risk patients  

PubMed Central

Patients with acute coronary syndrome (ACS) represent a major clinical burden, because they tend to experience recurrent ischemic events. Acute management of patients with ACS includes combination antithrombotic therapy composed of a parenteral anticoagulant and dual-antiplatelet therapy. Dual-antiplatelet therapy is also recommended for long-term secondary prevention of ACS. Despite advances in the antithrombotic therapies available, clinical trials suggest that patients with ACS still faceă10% risk of another event within 12–15 months of the index event. Certain patient populations, such as elderly patients and those with renal impairment or heart failure, are at higher risk of recurrent ACS events, because these patients have more vascular ischemic and bleeding risk factors than most other patients. Evidence from the GRACE and CRUSADE registries suggests underuse of the guideline-recommended evidence-based therapies for the management of ACS in such patients. This review summarizes the current standard of care for patients with ACS, focusing on long-term secondary antithrombotic strategies. Registry data are used to identify high-risk patient populations; the recent antiplatelet and anticoagulant Phase III trial data are summarized to highlight any patient populations who receive greater or lesser benefit from specific long-term antithrombotic strategies. Guideline recommendations are discussed and suggestions are provided to help improve implementation of long-term secondary prevention strategies and patient prognosis after an ACS event. PMID:25733842

Husted, Steen

2015-01-01

226

Psychological Therapies in Patients with Irritable Bowel Syndrome: A Systematic Review and Meta-Analysis of Randomized Controlled Trials  

PubMed Central

Background. Irritable bowel syndrome (IBS) is a poorly understood disease with few effective treatments. Psychosocial factors are believed to contribute to the pathogenesis of IBS. Objective. To evaluate the evidence for psychological therapies in IBS treatment. Methods. We searched six medical databases through February 6, 2014, for randomized controlled trials (RCTs) of psychological therapies for the treatment of IBS. Two independent reviewers identified the RCTs, extracted the data, and assessed trial quality. We used the random-effect model to pool standardized mean difference (SMD) and 95% confidence interval (CI) across trials. Results. 15 RCTs that mostly evaluated cognitive behavioral therapy were included. Psychological therapies were associated with improvement in IBS symptoms severity scales (SMD ?0.618; 95% CI: ?0.853 to ?0.383), IBS-Quality of Life (SMD 0.604; 95% CI: 0.440 to 0.768), and abdominal pain (SMD ?0.282; 95% CI: ?0.562 to ?0.001). No statistically significant effect was observed on diarrhea or constipation. Limitations. The trials were at increased risk of bias and the overall sample size was small leading to imprecision. Conclusion. Psychological therapies may improve the quality of life and symptom severity in IBS. The effect size noted is moderate to large and is clinically meaningful. PMID:25802514

Altayar, Osama; Prokop, Larry J.; Sood, Amit; Murad, Mohammad Hassan

2015-01-01

227

Optimizing therapy for iron overload in the myelodysplastic syndromes: recent developments.  

PubMed

The myelodysplastic syndromes (MDS) are characterized by cytopenias and risk of progression to acute myeloid leukaemia (AML). Most MDS patients eventually require transfusion of red blood cells for anaemia, placing them at risk of transfusional iron overload. In ?-thalassaemia major, transfusional iron overload leads to organ dysfunction and death; however, with iron chelation therapy, organ function is improved, and survival improved to near normal and correlated with the degree of compliance with chelation. In lower-risk MDS, several nonrandomized studies suggest an adverse effect of iron overload on survival and that lowering iron with chelation may minimize this impact. Emerging data indicate that chelation may improve organ function, particularly hepatic function, and a minority of patients may have improvement in cell counts and decreased transfusion requirements. While guidelines for MDS generally recommend chelation in selected lower-risk patients, data from nonrandomized trials suggest iron overload may impact adversely on the outcome of higher-risk MDS and stem cell transplantation (SCT). This effect may be due to increased transplant-related mortality, infection and AML progression, and preliminary data suggest that lowering iron may be beneficial in this patient group. Other areas of active and future investigation include optimizing the monitoring of iron overload using imaging such as T2* MRI and measures of labile iron and oxidative stress; correlating new methods of measuring iron to clinical outcomes; clarifying the contribution of different cellular and extracellular iron pools to iron toxicity; optimizing chelation by using agents that access the appropriate iron pools to minimize the relevant clinical consequences in individual patients; and incorporating measures of quality of life and co-morbidities into clinical trials of chelation in MDS. It should be noted that chelation is costly and potentially toxic, and in MDS should be initiated after weighing potential risks and benefits for each patient until more definitive data are available. In this review, data on the impact of iron overload in MDS and SCT are discussed; for example, several noncontrolled studies show inferior survival in patients with iron overload in these clinical settings, including an increase in transplant-related mortality and infection risk. Possible mechanisms of iron toxicity include oxidative stress, which can damage cellular components, and the documented impact of lowering iron on organ function with measures such as iron chelation therapy includes an improvement in elevated liver transaminases. Lowering iron also appears to improve survival in both lower-risk MDS and SCT in nonrandomized studies. Selected aspects of iron metabolism, transport, storage and distribution that may be amenable to future intervention and improved removal of iron from important cellular sites are discussed, as are attempts to quantify quality of life and the importance of co-morbidities in measures to treat MDS, including chelation therapy. PMID:21275444

Leitch, Heather A

2011-01-22

228

Plates-formes de microscopie et fluorescence par resonance de plasmons de surface appliquees a l'imagerie cellulaire  

NASA Astrophysics Data System (ADS)

L'elaboration de nouveaux medicaments repose sur les etudes pharmacologiques, dont le role est d'identifier de nouveaux composes actifs ou de nouvelles cibles pharmacologiques agissant entre autres au niveau cellulaire. Recemment, la detection basee sur la resonance des plasmons de surface (SPR) a ete appliquee a l'etude de reponses cellulaires. Cette methode de detection, permettant d'observer des variations d'indice de refraction associes a de faibles changements de masse a la surface d'un metal, a l'avantage de permettre l'etude d'une population de cellules vivantes en temps reel, sans necessiter l'introduction d'agents de marquage. Pour effectuer la detection au niveau de cellules individuelles, on peut employer la microscopie SPR, qui consiste a localiser spatialement la detection par un systeme d'imagerie. Cependant, la detection basee sur la SPR est une mesure sans marquage et les signaux mesures sont attribues a une reponse moyennee des differentes sources cellulaires. Afin de mieux comprendre et identifier les composantes cellulaires generant le signal mesure en SPR, il est pertinent de combiner la microscopie SPR avec une modalite complementaire, soit l'imagerie de fluorescence. C'est dans cette problematique que s'insere ce projet de these, consistant a concevoir deux plates-formes distinctes de microscopie SPR et de fluorescence optimisees pour l'etude cellulaire, de sorte a evaluer les possibilites d'integration de ces deux modalites en un seul systeme. Des substrats adaptes pour chaque plate-forme ont ete concus et realises. Ces substrats employaient une couche d'argent passivee par l'ajout d'une mince couche d'or. La stabilite et la biocompatibilite des substrats ont ete validees pour l'etude cellulaire. Deux configurations permettant d'ameliorer la sensibilite en sondant les cellules plus profondement ont ete evaluees, soit l'emploi de plasmons de surface a longue portee et de guides d'onde a gaine metallique. La sensibilite accrue de ces configurations a aussi ete demontree pour un usage en biodetection cellulaire. Une plate-forme permettant de mesurer la spectroscopie SPR simultanement avec l'acquisition d'images de fluorescence a ete realisee. Cette plate-forme a ensuite ete validee par l'etude de reponses cellulaires suite a une stimulation pharmacologique. Puis, un systeme base sur la microscopie SPR a ete concu et caracterise. Son emploi pour l'etude de reponses au niveau de cellules individuelles a ete demontre. Finalement, les forces et faiblesses des substrats et des plates-formes realisees au cours de la these ont ete evaluees. Des possibilites d'amelioration sont mises de l'avant et l'integration des modalites de microscopie SPR et de fluorescence suite aux travaux de la these est discutee. Les realisations au cours de cette etude ont donc permis d'identifier les composantes cellulaires impliquees dans la generation du signal mesure en biodetection SPR. Mots-cles : Resonance des plasmons de surface, microscopie SPR, plasmons de surface a longue portee LRSPR, guide d'onde a gaine metallique MCWG, fluorescence exaltee par plasmons de surface SPEF, biodetection cellulaire, imagerie SPR.

Chabot, Vincent

229

Oral carnitine therapy in children with cystinosis and renal Fanconi syndrome  

Microsoft Academic Search

11 children with either cystinosis or Lowe's syndrome had a reduced content of plasma and muscle carnitine due to renal Fanconi syndrome. After treatment with oral L-carnitine, 100 mg\\/kg per d divided every 6 h, plasma carnitine concentrations became normal in all subjects within 2 d. Initial plasma free fatty acid concentrations, inversely related to free carnitine concentrations, were reduced

W. A. Gahl; I. Bernardini; M. Dalakas; W. B. Rizzo; G. S. Harper; J. M. Hoeg; O. Hurko; J. Bernar

1988-01-01

230

Presentation of familial Mediterranean fever in a heterozygous MEFV mutation triggered by immunosuppressive therapy for myelodysplastic syndrome.  

PubMed

Familial Mediterranean fever (FMF) is a recessively inherited disease characterized by recurrent episodes of systemic inflammation. The cause of this disease is the mutations affecting both the alleles of MEFV gene. We describe here a case in a heterozygous MEFV mutation complicated with myelodysplastic syndrome (MDS). Clinical symptoms and the effectiveness of colchicines in this patient are typical for FMF. The first attack of FMF in this patient was observed during immunosuppressive therapy for MDS. This case suggests the possibility that certain immunosuppressants may trigger FMF attack in asymptomatic cases carrying MEFV heterozygous mutation. PMID:19466506

Sasaki, Ko; Tahara, Toshiyuki; Mitani, Kinuko

2009-07-01

231

Exercise therapy and cognitive behavioural therapy to improve fatigue, daily activity performance and quality of life in Postpoliomyelitis Syndrome: the protocol of the FACTS-2-PPS trial  

PubMed Central

Background Postpoliomyelitis Syndrome (PPS) is a complex of late onset neuromuscular symptoms with new or increased muscle weakness and muscle fatigability as key symptoms. Main clinical complaints are severe fatigue, deterioration in functional abilities and health related quality of life. Rehabilitation management is the mainstay of treatment. Two different therapeutic interventions may be prescribed (1) exercise therapy or (2) cognitive behavioural therapy (CBT). However, the evidence on the effectiveness of both interventions is limited. The primary aim of the FACTS-2-PPS trial is to study the efficacy of exercise therapy and CBT for reducing fatigue and improving activities and quality of life in patients with PPS. Additionally, the working mechanisms, patients' and therapists' expectations of and experiences with both interventions and cost-effectiveness will be evaluated. Methods/Design A multi-centre, single-blinded, randomized controlled trial will be conducted. A sample of 81 severely fatigued patients with PPS will be recruited from 3 different university hospitals and their affiliate rehabilitation centres. Patients will be randomized to one of three groups i.e. (1) exercise therapy + usual care, (2) CBT + usual care, (3) usual care. At baseline, immediately post-intervention and at 3- and 6-months follow-up, fatigue, activities, quality of life and secondary outcomes will be assessed. Costs will be based on a cost questionnaire, and statistical analyses on GEE (generalized estimated equations). Analysis will also consider mechanisms of change during therapy. A responsive evaluation will be conducted to monitor the implementation process and to investigate the perspectives of patients and therapists on both interventions. Discussion A major strength of the FACTS-2-PPS study is the use of a mixed methods design in which a responsive and economic evaluation runs parallel to the trial. The results of this study will generate new evidence for the rehabilitation treatment of persons with PPS. Trial registration Dutch Trial Register NTR1371. PMID:20082714

2010-01-01

232

The acute phase reactant, fibrinogen, as a guide to plasma exchange therapy for acute Guillain-Barré syndrome.  

PubMed

The Guillian Barré syndrome is an acute inflammatory disorder for which plasma exchange is effective treatment. Up to 10% relapse after plasma exchange suggesting that treatment sometimes finishes before disease activity has resolved. We studied whether plasma fibrinogen, an inflammatory marker, might be used to determine when to discontinue plasma exchange in patients with acute Guillain-Barré syndrome. We conducted a post-hoc analysis of apheresis database and hospital records of patients treated with plasma exchange for acute Guillain-Barré syndrome during 1999-2004. Data were analyzed from 28 patients who underwent a total of 29 courses of plasma exchange for acute Guillain-Barré syndrome. The mean (+/-SD) plasma fibrinogen concentration was 422.5 (+/-96.4) mg/dl at the time of presentation and, in 17 of the 29, it was above 400 mg/dl (reference range 200-400). Twenty of the 21 patients whose fibrinogen fell by more than 30% from baseline by the time of the final plasma exchange treatment had neurological improvement. There was improvement in only 3 of the 8 instances where fibrinogen decreased by less than 30% by the end of plasma exchange therapy. A > or =30% decrease in fibrinogen by the conclusion of plasma exchange was significantly associated with sustained neurological improvement (P = 0.0025). The plasma fibrinogen level appears to reflect disease activity in acute Guillain-Barré syndrome. A <30% fall in fibrinogen level despite plasma exchange may indicate the need to continue plasma exchange to maximize the benefit of treatment or minimize the risk of relapse. Therapeutic plasma exchange need not be extended when plasma fibrinogen remains > or =30% below its level at presentation by the time of the final planned plasma exchange procedure. PMID:16342192

Sanjay, Rashmi; Flanagan, Janice; Sodano, Donata; Gorson, Kenneth C; Ropper, Allan H; Weinstein, Robert

2006-07-01

233

Recurrent giant molluscum contagiosum immune reconstitution inflammatory syndrome (IRIS) after initiation of antiretroviral therapy in an HIV-infected man.  

PubMed

We describe an HIV-infected South African man who experienced two distinct episodes of disseminated giant molluscum contagiosum immune reconstitution inflammatory syndrome (IRIS) over a six-year period. The first episode of molluscum contagiosum IRIS occurred with rapid virologic suppression following antiretroviral therapy initiation. The second episode occurred during a rapid increase in CD4 cells following stable viral suppression with second-line antiretroviral therapy. His molluscum contagiosum lesions then completely resolved during a reduction in CD4 count, despite maintaining virologic suppression. Nearly one year after the resolution of his giant molluscum contagiosum IRIS lesions, he maintains an undetectable viral load, but his level of immune deficiency has not improved. In the absence of well-controlled therapeutic trials, molluscum contagiosum IRIS presents important management challenges. PMID:23970647

Drain, Paul K; Mosam, Anisa; Gounder, Lilishia; Gosnell, Bernadett; Manzini, Thandekile; Moosa, Mahomed-Yunus S

2014-03-01

234

Salvage therapy with high dose Intravenous Immunoglobulins in acquired Von Willebrand Syndrome and unresponsive severe intestinal bleeding  

PubMed Central

A 91-year-old woman affected with acquired Von Willebrand (VW) syndrome and intestinal angiodysplasias presented with severe gastrointestinal bleeding (hemoglobin 5 g/dl). Despite replacement therapy with VW factor/factor VIII concentrate qid, bleeding did not stop (eleven packed red blood cell units were transfused over three days). High circulating levels of anti-VW factor immunoglobulin M were documented immunoenzimatically. Heart ultrasound showed abnormalities of the mitral and aortic valves with severe flow alterations. When intravenous immunoglobulins were added to therapy, prompt clinical and laboratory responses occurred: complete cessation of bleeding, raise in hemoglobin, VW factor antigen, VW ristocetin cofactor and factor VIII levels as well as progressive reduction of the anti-VWF autoantibody levels. PMID:24926417

2014-01-01

235

Oral anticoagulant use in addition to antiplatelet therapy for secondary prevention in acute coronary syndrome: current perspectives.  

PubMed

Patients with acute coronary syndrome (ACS) are typically managed with long-term dual antiplatelet therapy of acetylsalicylic acid plus a P2Y12 platelet receptor antagonist; however, although effective, the risk of another vascular event within 12 months remains at approximately 10%. Considerable efforts have been made to find improved therapeutic approaches to secondary prevention in ACS. The ATLAS ACS 2-TIMI 51 trial demonstrated that rivaroxaban (2.5 mg twice daily) significantly reduced recurrent vascular events, increased the risk of major bleeding but not the risk of fatal bleeding, and resulted in reduced rates of death from cardiovascular causes. These results formed the basis for approval in Europe of rivaroxaban (2.5 mg twice daily) in conjunction with standard antiplatelet therapy for the secondary prevention of ACS. PMID:25017622

Turpie, Alexander G G

2014-08-01

236

Cytogenetic evidence of metastatic myxoid liposarcoma and therapy-related myelodysplastic syndrome in a bone marrow biopsy.  

PubMed

Myxoid liposarcoma exhibits a peculiar clinical behavior, with a tendency to spread to serosal membranes, distant soft tissues, and bones, even in the absence of lung metastases. Therapy-related hematological neoplasms are well-known side effects of cytotoxic chemotherapy. We describe an exceptional case of metastatic myxoid liposarcoma of the spine associated with therapy-related refractory anemia with excess of blasts in a 37-year-old woman who underwent multi-agent chemotherapy for a myxoid liposarcoma of the left thigh. Microscopic examination of the bone marrow biopsy revealed dysplastic features, with abnormal localization of immature precursors and micromegakaryocytes, and islands of undifferentiated oval small/medium-size cells, suggestive of acute myeloid leukemia arising in the setting of a myelodysplastic syndrome. Immunohistochemistry was not discriminant. Cytogenetic analyses of bone marrow aspirate disclosed the presence of 2 different rearrangements, subsequently confirmed by fluorescent in situ hybridization and was crucial in making the correct diagnosis. PMID:19439345

Rossi, Sabrina; Canal, Fabio; Licci, Stefano; Zanatta, Lucia; Laurino, Licia; Gottardi, Michele; Gherlinzoni, Filippo; Dei Tos, Angelo Paolo

2009-07-01

237

Pain exposure physical therapy may be a safe and effective treatment for longstanding complex regional pain syndrome type 1: a case series  

Microsoft Academic Search

Objective: To determine if treatment of longstanding complex regional pain syndrome type 1, focusing on functional improvement only while neglecting pain, results in clinical improvement of this syndrome.Design: Prospective description of a case series of 106 patients.Setting: Outpatient clinic for rehabilitation.Interventions: Physical therapy of the affected limb directed at a functional improvement only while neglecting the pain, was performed following

Jan-Willem Ek; Jan C van Gijn; Han Samwel; Jan van Egmond; Frank PAJ Klomp; Robert TM van Dongen

2009-01-01

238

Comparison of conservative treatment with and without manual physical therapy for patients with shoulder impingement syndrome: a prospective, randomized clinical trial  

Microsoft Academic Search

The aim of this prospective, randomized clinical study was to compare the effectiveness of two physical therapy treatment\\u000a approaches for impingement syndrome, either by joint and soft tissue mobilization techniques or by a self-training program.\\u000a Thirty patients (Group 1, n = 15; Group 2, n = 15) with the diagnosis of an outlet impingement syndrome of the shoulder were treated either by strengthening the

Gamze Senbursa; Gul Baltac?; Ahmet Atay

2007-01-01

239

Evaluation of noncytotoxic DNMT1-depleting therapy in patients with myelodysplastic syndromes.  

PubMed

BACKGROUND. Mutational inactivation in cancer of key apoptotic pathway components, such as TP53/p53, undermines cytotoxic therapies that aim to increase apoptosis. Accordingly, TP53 mutations are reproducibly associated with poor treatment outcomes. Moreover, cytotoxic treatments destroy normal stem cells with intact p53 systems, a problem especially for myeloid neoplasms, as these cells reverse the low blood counts that cause morbidity and death. Preclinical studies suggest that noncytotoxic concentrations of the DNA methyltransferase 1 (DNMT1) inhibitor decitabine produce p53-independent cell-cycle exits by reversing aberrant epigenetic repression of proliferation-terminating (MYC-antagonizing) differentiation genes in cancer cells. METHODS. In this clinical trial, patients with myelodysplastic syndrome (n = 25) received reduced decitabine dosages (0.1-0.2 mg/kg/day compared with the FDA-approved 20-45 mg/m2/day dosage, a 75%-90% reduction) to avoid cytotoxicity. These well-tolerated doses were frequently administered 1-3 days per week, instead of pulse cycled for 3 to 5 days over a 4- to 6-week period, to increase the probability that cancer S-phase entries would coincide with drug exposure, which is required for S-phase-dependent DNMT1 depletion. RESULTS. The median subject age was 73 years (range, 46-85 years), 9 subjects had relapsed disease or were refractory to 5-azacytidine and/or lenalidomide, and 3 had received intensive chemoradiation to treat other cancers. Adverse events were related to neutropenia present at baseline: neutropenic fever (13 of 25 subjects) and septic death (1 of 25 subjects). Blood count improvements meeting the International Working Group criteria for response occurred in 11 of 25 (44%) subjects and were highly durable. Treatment-induced freedom from transfusion lasted a median of 1,025 days (range, 186-1,152 days; 3 ongoing), and 20% of subjects were treated for more than 3 years. Mutations and/or deletions of key apoptosis genes were frequent (present in 55% of responders and in 36% of nonresponders). Noncytotoxic DNMT1 depletion was confirmed by serial BM ?-H2AX (DNA repair/damage marker) and DNMT1 analyses. MYC master oncoprotein levels were markedly decreased. CONCLUSION. Decitabine regimens can be redesigned to minimize cytotoxicity and increase exposure time for DNMT1 depletion, to safely and effectively circumvent mutational apoptotic defects. TRIAL REGISTRATION. Clinicaltrials.gov NCT01165996. FUNDING. NIH (R01CA138858, CA043703); Department of Defense (PR081404); Clinical and Translational Science Award (CTSA) (UL1RR024989); and the Leukemia and Lymphoma Society (Translational Research Program). PMID:25621498

Saunthararajah, Yogen; Sekeres, Mikkael; Advani, Anjali; Mahfouz, Reda; Durkin, Lisa; Radivoyevitch, Tomas; Englehaupt, Ricki; Juersivich, Joy; Cooper, Kathleen; Husseinzadeh, Holleh; Przychodzen, Bartlomiej; Rump, Matthew; Hobson, Sean; Earl, Marc; Sobecks, Ronald; Dean, Robert; Reu, Frederic; Tiu, Ramon; Hamilton, Betty; Copelan, Edward; Lichtin, Alan; Hsi, Eric; Kalaycio, Matt; Maciejewski, Jaroslaw

2015-03-01

240

The effectiveness and cost evaluation of pain exposure physical therapy and conventional therapy in patients with complex regional pain syndrome type 1. Rationale and design of a randomized controlled trial  

PubMed Central

Background Pain Exposure Physical Therapy is a new treatment option for patients with Complex Regional Pain Syndrome type 1. It has been evaluated in retrospective as well as in prospective studies and proven to be safe and possibly effective. This indicates that Pain Exposure Physical Therapy is now ready for clinical evaluation. The results of an earlier performed pilot study with an n?=?1 design, in which 20 patients with Complex Regional Pain Syndrome type 1 were treated with Pain Exposure Physical Therapy, were used for the design and power calculation of the present study. After completion and evaluation of this phase III study, a multi-centre implementation study will be conducted. The aim of this study is to determine whether Pain Exposure Physical Therapy can improve functional outcomes in patients with Complex Regional Pain Syndrome type 1. Methods/design This study is designed as a single-blinded, randomized clinical trial. 62 patients will be randomized with a follow-up of 9?months to demonstrate the expected treatment effect. Complex Regional Pain Syndrome type 1 is diagnosed in accordance with the Bruehl/International Association for the Study of Pain criteria. Conventional therapy in accordance with the Dutch guideline will be compared with Pain Exposure Physical Therapy. Primary outcome measure is the Impairment level SumScore, restricted version. Discussion This is the first randomized controlled study with single blinding that has ever been planned in patients with Complex Regional Pain Syndrome type 1 and does not focus on a single aspect of the pain syndrome but compares treatment strategies based on completely different pathophysiological and cognitive theories. Trial registration Clinical trials NCT00817128; National Trial Register NTR2090 PMID:22515496

2012-01-01

241

Modeling severely discordant hematocrits and normal amniotic fluids after incomplete laser therapy in twin-to-twin transfusion syndrome.  

PubMed

Our objective was to explain the clinical presentations of sustained arteriovenous anastomotic transfusion of blood after incomplete laser therapy in twin-to-twin transfusion syndrome (TTTS). We extended our mathematical model of TTTS by adding the dynamics of hematocrit, and simulated incomplete laser therapy, first, by leaving one patent opposite arteriovenous anastomosis from the recipient to the donor and, second, by leaving one patent arteriovenous anastomosis from the donor to the recipient. In both simulations we reproduced the clinical observation of severe hematocrit discordance preceding delayed amniotic fluid imbalance. In conclusion, incomplete laser therapy may cause a severe circulatory imbalance between the twins which presents predominantly as discordant hematocrits rather than discordant amniotic fluid volumes as in primary TTTS. These results imply that the anemia-polycythemia sequence is a sensitive mechanism to identify transfusion reversal after complicated laser therapy, confirming the suggested role of middle cerebral artery peak systolic velocity Doppler measurements as a useful method of follow-up. PMID:17098282

van den Wijngaard, Jeroen P H M; Lewi, Liesbeth; Lopriore, Enrico; Robyr, Romaine; Middeldorp, Johanna M; Vandenbussche, Frank P H A; Devlieger, Roland; Deprest, Jan; Ville, Yves; van Gemert, Martin J C

2007-07-01

242

Combination therapy for segmental craniocervical dystonia (Meige syndrome) with aripiprazole, trihexyphenidyl, and botulinum toxin: three cases reports.  

PubMed

Segmental craniocervical dystonia is characterized by blephalospasm and oromandibular dystonia and is also called Meige syndrome. The current treatment strategy including botulinum toxin (BTX) injections has not yet attained an acceptable level. We describe a long-term favorable response of a novel combination therapy with aripiprazole (ARP), trihexyphenidyl (THP), and BTX in three patients with segmental craniocervical dystonia. The symptoms of three patients responded promptly to the combination therapy with ARP 3-6 mg daily, THP 2-8 mg daily, and BTX. Although the patients were required to receive a BTX 50-100 IU injection every 3-6 months, their symptoms were kept in a satisfactory condition for up to 2 years without any adverse effects. ARP possesses the potential for dramatically improving dystonia. THP has the possibility to enhance the efficacy of ARP and prolong the effective period of BTX. It may be an important requisite to give all three agents together for a successful treatment. The combination therapy with ARP, THP, and BTX was well-tolerated and useful in controlling the symptoms of segmental craniocervical dystonia, however, the reason why this combination therapy succeeded is unknown. A further long-term follow-up is required to monitor the delayed neurological adverse effects. PMID:25151364

Saito, Tsukasa; Katayama, Takayuki; Sawada, Jun; Kano, Kohei; Asanome, Asuka; Takahashi, Kae; Sato, Nobuyuki; Hasebe, Naoyuki

2015-02-01

243

Alport syndrome from bench to bedside: the potential of current treatment beyond RAAS blockade and the horizon of future therapies.  

PubMed

The hereditary type IV collagen disease Alport syndrome (AS) always leads to end-stage renal failure. Yesterday, for the past 90 years, this course was described as 'inevitable'. Today, RAAS blockade has changed the 'inevitable' course to a treatable disease. Tomorrow, researchers hope to erase the 'always' from 'always leads to renal failure' in the textbooks. This review elucidates therapeutic targets that evolve from research: (i) kidney embryogenesis and pathogenesis; (ii) phenotype-genotype correlation and the role of collagen receptors and podocytes; (iii) the malfunctioning Alport-GBM; (iv) tubulointerstitial fibrosis; (v) the role of proteinuria in pathogenesis and prognosis; and (vi) secondary events such as infections, hyperparathyroidism and hypercholesterolaemia. Therefore, moderate lifestyle, therapy of bacterial infections, Paricalcitol in adult patients with hyperparathyroidism and HMG-CoA-reductase inhibitors in adult patients with dyslipoproteinemia might contribute to a slower progression of AS and less cardiovascular events. In the future, upcoming treatments including stem cells, chaperon therapy, collagen receptor blockade and anti-microRNA therapy will expand our perspective in protecting the kidneys of Alport patients from further damage. This perspective on current and future therapies is naturally limited by our personal focus in research, but aims to motivate young scientists and clinicians to find a multimodal cure for AS. PMID:25165179

Gross, Oliver; Perin, Laura; Deltas, Constantinos

2014-09-01

244

Acute Human Immunodeficiency Virus (HIV) Syndrome After Nonadherence to Antiretroviral Therapy in a Patient With Chronic HIV Infection: A Case Report  

PubMed Central

We report a rare case of acute human immunodeficiency virus (HIV) syndrome in a patient with chronic HIV infection with acute illness indistinguishable from acute retroviral syndrome. The patient presented with an acute febrile mononucleosis-like illness after increasing nonadherence to antiretroviral therapy. A marked increase in HIV RNA level of 1 220 000 copies/mL from less than 20 copies/mL occurred within 3 weeks. The diagnosis of acute HIV syndrome was made after alternative causes of illness were ruled out. PMID:25734180

Choi, Seong K.; Graber, Christopher J.

2014-01-01

245

Successful Medical Therapy for Hypophosphatemic Rickets due to Mitochondrial Complex I Deficiency Induced de Toni-Debré-Fanconi Syndrome  

PubMed Central

Primary de Toni-Debré-Fanconi syndrome is a non-FGF23-mediated hypophosphatemic disorder due to a primary defect in renal proximal tubule cell function resulting in hyperphosphaturia, renal tubular acidosis, glycosuria, and generalized aminoaciduria. The orthopaedic sequela and response to treatment of this rare disorder are limited in the literature. Herein we report a long term followup of a 10-year-old female presenting at 1 year of age with rickets initially misdiagnosed as vitamin D deficiency rickets. She was referred to the metabolic bone and genetics clinics at 5 years of age with severe genu valgum deformities of 24 degrees and worsening rickets. She had polyuria, polydipsia, enuresis, and bone pain. Diagnosis of hypophosphatemic rickets due to de Toni-Debré-Fanconi syndrome was subsequently made. Respiratory chain enzyme analysis identified a complex I mitochondrial deficiency as the underlying cause. She was treated with phosphate (50–70?mg/kg/day), calcitriol (30?ng/kg/day), and sodium citrate with resolution of bone pain and normal growth. By 10 years of age, her genu valgus deformities were 4 degrees with healing of rickets. Her excellent orthopaedic outcome despite late proper medical therapy is likely due to the intrinsic renal tubular defect that is more responsive to combined alkali, phosphate, and calcitriol therapy. PMID:24386581

Bowden, Sasigarn A.; Patel, Hiren P.; Beebe, Allan; McBride, Kim L.

2013-01-01

246

Effects of Levothyroxine Replacement Therapy on Parameters of Metabolic Syndrome and Atherosclerosis in Hypothyroid Patients: A Prospective Pilot Study  

PubMed Central

The aim of this study was to investigate the effect of levothyroxine (LT4) replacement therapy during three months on some parameters of metabolic syndrome and atherosclerosis in patients with increased thyroid-stimulating hormone (TSH) level. This study included a group of 30 female patients with TSH level >4?mIU/L and 15 matched healthy controls. Intima media complex thickness (IMCT) and peak systolic flow velocity (PSFV) of superficial femoral artery were determined by Color Doppler scan. In hypothyroid subjects, BMI, SBP, DBP, and TSH were significantly increased versus controls and decreased after LT4 administration. FT4 was significantly lower in hypothyroid subjects compared with controls and significantly higher by treatment. TC, Tg, HDL-C, and LDL-C were similar to controls at baseline but TC and LDL-C were significantly decreased by LH4 treatment. IMCT was significantly increased versus controls at baseline and significantly reduced by treatment. PSFV was similar to controls at baseline and significantly decreased on treatment. In this study, we have demonstrated the effects of LT4 replacement therapy during three months of treatment on correction of risk factors of metabolic syndrome and atherosclerosis. PMID:25821465

Gluvic, Zoran; Sudar, Emina; Tica, Jelena; Jovanovic, Aleksandra; Zafirovic, Sonja; Tomasevic, Ratko; Isenovic, Esma R.

2015-01-01

247

Effects of levothyroxine replacement therapy on parameters of metabolic syndrome and atherosclerosis in hypothyroid patients: a prospective pilot study.  

PubMed

The aim of this study was to investigate the effect of levothyroxine (LT4) replacement therapy during three months on some parameters of metabolic syndrome and atherosclerosis in patients with increased thyroid-stimulating hormone (TSH) level. This study included a group of 30 female patients with TSH level >4?mIU/L and 15 matched healthy controls. Intima media complex thickness (IMCT) and peak systolic flow velocity (PSFV) of superficial femoral artery were determined by Color Doppler scan. In hypothyroid subjects, BMI, SBP, DBP, and TSH were significantly increased versus controls and decreased after LT4 administration. FT4 was significantly lower in hypothyroid subjects compared with controls and significantly higher by treatment. TC, Tg, HDL-C, and LDL-C were similar to controls at baseline but TC and LDL-C were significantly decreased by LH4 treatment. IMCT was significantly increased versus controls at baseline and significantly reduced by treatment. PSFV was similar to controls at baseline and significantly decreased on treatment. In this study, we have demonstrated the effects of LT4 replacement therapy during three months of treatment on correction of risk factors of metabolic syndrome and atherosclerosis. PMID:25821465

Gluvic, Zoran; Sudar, Emina; Tica, Jelena; Jovanovic, Aleksandra; Zafirovic, Sonja; Tomasevic, Ratko; Isenovic, Esma R

2015-01-01

248

A Case of Childhood Vitrectomy Performed for Dense Vitreous Hemorrhage Secondary to Leukemia Therapy and Tumor Lysis Syndrome  

PubMed Central

Purpose To report a case of vitrectomy performed in a child with dense massive vitreous hemorrhage due to secondary acute myelogenous leukemia (AML) and tumor lysis syndrome. Case A 4-year-old boy with clear-cell renal cell carcinoma was successfully treated with chemotherapy in 2011. However, in May 2012, he developed secondary AML. Although he was treated with combined chemotherapy and radiation, tumor lysis syndrome occurred with renal and heart failure complications. After an ultrasound examination by pediatricians found bilateral subretinal protrusions, he was referred to our clinic. Fundus examinations confirmed that the protrusions were bilateral subretinal or choroidal hemorrhages. A few weeks later, dense vitreous hemorrhages occurred bilaterally, and he completely lost vision in both eyes. Electroretinograms were extinguished in both eyes. After improvement of his general condition, we performed a 25-gauge vitrectomy combined with lens extraction in his left eye in December 2012. After removal of the vitreous hemorrhage, we found the subretinal hemorrhage had already been absorbed, leaving a mottled fundus color. However, the optic disc was not pale. Nine months after the surgery, his best-corrected visual acuity finally improved to 0.1. Conclusion We successfully treated a case of severe vitreous hemorrhage secondary to leukemia therapy and tumor lysis syndrome using 25-gauge vitrectomy. This procedure may be safe and effective to perform, even in children with complications.

Kudo, Takashi; Suzuki, Yukihiko; Metoki, Tomomi; Nakazawa, Mitsuru

2015-01-01

249

Improved Identification and Antihypertension Pharmacotherapy in Cardiorenal Metabolic Syndrome: Focus on Racial/Ethnic Minorities, Olmesartan Medoxomil, and Combination Therapy.  

PubMed

Cardiorenal metabolic syndrome (CRS) is a global health care concern in view of aging in certain populations, increased obesity, changing lifestyles, and its close association with type 2 diabetes mellitus and cardiovascular morbidity and mortality. Determining the appropriate criteria for CRS has been somewhat controversial, and efforts to fully describe and define the syndrome are still ongoing. Nonetheless, improving knowledge of the syndrome among health care professionals will help to identify patients who may require pharmacological and therapeutic lifestyle intervention, particularly with regards to addressing high-normal blood pressure and hypertension. This article reviews current clinical guidelines with a focus on the identification, especially in racial/ethnic minorities, treatment, and associated cardiovascular morbidity and mortality of high blood pressure and hypertension in patients with CRS. Efficacy and outcomes studies that provide insight into the selection of an initial antihypertensive regimen in this population will be discussed. Finally, a brief review of the benefits of olmesartan medoxomil and combination therapy and patient factors in the management of hypertension with CRS will be addressed. PMID:23380878

Ferdinand, Keith C

2012-12-01

250

Impact of enzyme replacement therapy on linear growth in Korean patients with mucopolysaccharidosis type II (Hunter syndrome).  

PubMed

Hunter syndrome (or mucopolysaccharidosis type II [MPS II]) arises because of a deficiency in the lysosomal enzyme iduronate-2-sulfatase. Short stature is a prominent and consistent feature in MPS II. Enzyme replacement therapy (ERT) with idursulfase (Elaprase®) or idursulfase beta (Hunterase®) have been developed for these patients. The effect of ERT on the growth of Korean patients with Hunter syndrome was evaluated at a single center. This study comprised 32 patients, who had received ERT for at least 2 yr; they were divided into three groups according to their ages at the start of ERT: group 1 (<6 yr, n=14), group 2 (6-10 yr, n=11), and group 3 (10-20 yr, n=7). The patients showed marked growth retardation as they got older. ERT may have less effect on the growth of patients with the severe form of Hunter syndrome. The height z-scores in groups 2 and 3 revealed a significant change (the estimated slopes before and after the treatment were -0.047 and -0.007, respectively: difference in the slope, 0.04; P<0.001). Growth in response to ERT could be an important treatment outcome or an endpoint for future studies. PMID:24550654

Cho, Sung Yoon; Huh, Rimm; Chang, Mi Sun; Lee, Jieun; Kwun, Younghee; Maeng, Se Hyun; Kim, Su Jin; Sohn, Young Bae; Park, Sung Won; Kwon, Eun-Kyung; Han, Sun Ju; Jung, Jooyoun; Jin, Dong-Kyu

2014-02-01

251

Prediction of response to therapy with ezatiostat in lower risk myelodysplastic syndrome  

E-print Network

Background: Approximately 70% of all patients with myelodysplastic syndrome (MDS) present with lower-risk disease. Some of these patients will initially respond to treatment with growth factors to improve anemia but will ...

Galili, Naomi

252

Change of the course of steroid-dependent nephrotic syndrome after rituximab therapy  

Microsoft Academic Search

A 16-year-old patient with steroid-dependent nephrotic syndrome with more than 35 relapses developed severe relapsing idiopathic thrombocytopenic purpura (ITP). At the age of 2 years, nephrotic syndrome was diagnosed and successfully treated with a standard prednisone regimen. Frequent relapses occurred. Treatment with oral cyclophosphamide followed by cyclosporine was successful, but several attempts to withdraw steroids failed and the patient suffered from

Kerstin Benz; Jörg Dötsch; Wolfgang Rascher; Daniel Stachel

2004-01-01

253

Therapy Insight: cancer anorexia–cachexia syndrome—when all you can eat is yourself  

Microsoft Academic Search

Tumor growth is associated with profound metabolic and neurochemical alterations, which can lead to the onset of anorexia–cachexia syndrome. Anorexia is defined as the loss of the desire to eat, while cachexia results from progressive wasting of skeletal muscle mass—and to a lesser extent adipose tissue—occurring even before weight loss becomes apparent. Cancer anorexia–cachexia syndrome is highly prevalent among cancer

Michael M Meguid; Akio Inui; Maurizio Muscaritoli; Filippo Rossi-Fanelli; Alessandro Laviano

2005-01-01

254

The effect of gallium arsenide aluminum laser therapy in the management of cervical myofascial pain syndrome: a double blind, placebo-controlled study  

Microsoft Academic Search

The efficacy of low-level laser therapy (LLLT) in myofascial pain syndrome (MPS) seems controversial. A prospective, double-blind,\\u000a randomized controlled trial was conducted in patients with chronic MPS in the neck to evaluate the effects of low-level 830-nm\\u000a gallium arsenide aluminum (Ga–As–Al) laser therapy. The study group consisted of 64 MPS patients. The patients were randomly\\u000a assigned into two groups. In

U. Dundar; D. Evcik; F. Samli; H. Pusak; V. Kavuncu

2007-01-01

255

Long-term effects of recombinant human growth hormone therapy in children with Prader–Willi syndrome  

PubMed Central

Purpose of review Recombinant human growth hormone (hGH) therapy in children with Prader–Willi syndrome (PWS) improves linear growth, body composition, physical strength and agility, and other metabolic parameters. These benefits must be weighed against potential adverse effects, including rare occurrences of sudden death. This review summarizes recent evidence important to a benefit–risk analysis of hGH use in children with PWS. Recent findings Studies consistently show that hGH improves stature, body composition, fat percentage and distribution, and other metabolic markers in children with PWS. Preliminary reports of improved cognitive development during hGH have also emerged. Scoliosis progression is influenced by growth rate, but frequency of occurrence and severity are not increased by hGH exposure. PWS genotype does not appear to affect response to hGH. Concerns about hGH-associated sudden death persist, but recent studies show either absence of change in sleep-disordered breathing or improved sleep cardiovascular function during hGH therapy. Summary Recent studies confirm and expand reported benefits of hGH therapy in children with PWS, including a possible salutary role in cognitive development. These findings support previous assertions that hGH can reduce morbidity and improve function in children with PWS, and suggest that potential risks of such treatment are favorably balanced by its benefits. PMID:23782572

Wolfgram, Peter M.; Carrel, Aaron L.; Allen, David B.

2015-01-01

256

Treatment of Fibromyalgia Syndrome: Recommendations of Recent Evidence-Based Interdisciplinary Guidelines with Special Emphasis on Complementary and Alternative Therapies  

PubMed Central

Objective. Current evidence indicates that there is no single ideal treatment for fibromyalgia syndrome (FMS). First choice treatment options remain debatable, especially concerning the importance of complementary and alternative medicine (CAM) treatments. Methods. Three evidence-based interdisciplinary guidelines on FMS in Canada, Germany, and Israel were compared for their first choice and CAM-recommendations. Results. All three guidelines emphasized a patient-tailored approach according to the key symptoms. Aerobic exercise, cognitive behavioral therapy, and multicomponent therapy were first choice treatments. The guidelines differed in the grade of recommendation for drug treatment. Anticonvulsants (gabapentin, pregabalin) and serotonin noradrenaline reuptake inhibitors (duloxetine, milnacipran) were strongly recommended by the Canadian and the Israeli guidelines. These drugs received only a weak recommendation by the German guideline. In consideration of CAM-treatments, acupuncture, hypnosis/guided imagery, and Tai Chi were recommended by the German and Israeli guidelines. The Canadian guidelines did not recommend any CAM therapy. Discussion. Recent evidence-based interdisciplinary guidelines concur on the importance of treatment tailored to the individual patient and further emphasize the need of self-management strategies (exercise, and psychological techniques). PMID:24348701

Fitzcharles, Mary-Ann; Buskila, Dan; Shir, Yoram; Sommer, Claudia

2013-01-01

257

Adverse events and deterioration reported by participants in the PACE trial of therapies for chronic fatigue syndrome  

PubMed Central

Objective Adverse events (AEs) are health related events, reported by participants in clinical trials. We describe AEs in the PACE trial of treatments for chronic fatigue syndrome (CFS) and baseline characteristics associated with them. Methods AEs were recorded on three occasions over one year in 641 participants. We compared the numbers and nature of AEs between treatment arms of specialist medical care (SMC) alone, or SMC supplemented by adaptive pacing therapy (APT), cognitive behaviour therapy (CBT) or graded exercise therapy (GET). We examined associations with baseline measures by binary logistic regression analyses, and compared the proportions of participants who deteriorated by clinically important amounts. Results Serious adverse events and reactions were infrequent. Non-serious adverse events were common; the median (quartiles) number was 4 (2, 8) per participant, with no significant differences between treatments (P = .47). A greater number of NSAEs were associated with recruitment centre, and baseline physical symptom count, body mass index, and depressive disorder. Physical function deteriorated in 39 (25%) participants after APT, 15 (9%) after CBT, 18 (11%) after GET, and 28 (18%) after SMC (P < .001), with no significant differences in worsening fatigue. Conclusions The numbers of adverse events did not differ significantly between trial treatments, but physical deterioration occurred most often after APT. The reporting of non-serious adverse events may reflect the nature of the illness rather than the effect of treatments. Differences between centres suggest that both standardisation of ascertainment methods and training are important when collecting adverse event data. PMID:24913337

Dougall, Dominic; Johnson, Anthony; Goldsmith, Kimberley; Sharpe, Michael; Angus, Brian; Chalder, Trudie; White, Peter

2014-01-01

258

Orbital Lipomatosis: A Complication of Steroid Therapy in the Sweet Syndrome.  

PubMed

The description of a Sweet syndrome steroid dependant-induced orbital lipomatosis is reported. A 76-year-old-man with history of Sweet syndrome presented with severe bilateral proptosis (Hertel value, 25 mm) with decreased visual acuity and evoked potentials lengthened. A bilateral transpalpebral orbital decompression was performed by resection of intraorbital fat without bone removal. The surgery was uneventful. The volume of resected orbital fat was 15 ml for both sides. Proptosis reduction was 6 mm. Postoperative Hertel values were 19 mm, and evoked potentials were improved. The proptosis was managed successfully. Orbital lipectomy led to minimal sequelae and may be repeated if necessary in this case. PMID:25120096

Da Costa, Gwendoline; Pare, Arnaud; Sury, Florent; Goga, Dominque; Laure, Boris

2014-09-12

259

Global patterns of use of antithrombotic and antiplatelet therapies in patients with acute coronary syndromes: insights from the global registry of acute coronary events (grace)  

Microsoft Academic Search

Background Many agents are available to treat acute coronary syndromes (ACS), yet limited information is avail- able about their use from a multinational perspective. The objective of this report was to describe patterns of use of anti- thrombotic and antiplatelet therapies in patients with the spectrum of ACS through the use of data from the Global Regis- try of Acute

Andrzej Budaj; David Brieger; Gabriel Steg

2004-01-01

260

Ambulatory Arterial Stiffness Index in Turner Syndrome: The Impact of Sex Hormone Replacement Therapy  

Microsoft Academic Search

Background: Morbidity and mortality from congenital and acquired cardiovascular (CV) disease is increased in Turner syndrome (TS), where traditional indices of CV risk are widely present but the single most common feature remains estrogen deficiency. Aim: To investigate CV risk in TS as expressed by the widely available ambulatory arterial stiffness index (AASI) and the impact of female sex hormone

Kristian H. Mortensen; Klavs W. Hansen; Mogens Erlandsen; Jens S. Christiansen; Claus H. Gravholt

2009-01-01

261

Modified Atkins diet therapy for a case with glucose transporter type 1 deficiency syndrome  

Microsoft Academic Search

Glucose transporter type 1 deficiency syndrome (GLUT-1 DS), giving rise to impaired glucose transport across the blood–brain barrier, is characterized by infantile seizures, complex motor disorders, global developmental delay, acquired microcephaly, and hypoglycorrhachia. GLUT-1 DS can be treated effectively with a ketogenic diet because it can provide an alternative fuel for brain metabolism; however, the excessive restriction of food intake

Susumu Ito; Hirokazu Oguni; Yasushi Ito; Keiko Ishigaki; Junko Ohinata; Makiko Osawa

2008-01-01

262

Towards a therapy for Angelman syndrome by targeting a long non-coding RNA.  

PubMed

Angelman syndrome is a single-gene disorder characterized by intellectual disability, developmental delay, behavioural uniqueness, speech impairment, seizures and ataxia. It is caused by maternal deficiency of the imprinted gene UBE3A, encoding an E3 ubiquitin ligase. All patients carry at least one copy of paternal UBE3A, which is intact but silenced by a nuclear-localized long non-coding RNA, UBE3A antisense transcript (UBE3A-ATS). Murine Ube3a-ATS reduction by either transcription termination or topoisomerase I inhibition has been shown to increase paternal Ube3a expression. Despite a clear understanding of the disease-causing event in Angelman syndrome and the potential to harness the intact paternal allele to correct the disease, no gene-specific treatment exists for patients. Here we developed a potential therapeutic intervention for Angelman syndrome by reducing Ube3a-ATS with antisense oligonucleotides (ASOs). ASO treatment achieved specific reduction of Ube3a-ATS and sustained unsilencing of paternal Ube3a in neurons in vitro and in vivo. Partial restoration of UBE3A protein in an Angelman syndrome mouse model ameliorated some cognitive deficits associated with the disease. Although additional studies of phenotypic correction are needed, we have developed a sequence-specific and clinically feasible method to activate expression of the paternal Ube3a allele. PMID:25470045

Meng, Linyan; Ward, Amanda J; Chun, Seung; Bennett, C Frank; Beaudet, Arthur L; Rigo, Frank

2015-02-19

263

Psychodynamic interpersonal therapy and improvement in interpersonal difficulties in people with severe irritable bowel syndrome  

Microsoft Academic Search

The aim of the present study was to assess the relationship between change in interpersonal difficulties with change in chronic pain, health status and psychological state in 257 irritable bowel syndrome (IBS) patients in a randomized control trial comparing psychotherapy, antidepressant and usual care. We assessed at three time points interpersonal problems (IIP-32), abdominal pain and bowel symptoms, psychological distress

Thomas Hyphantis; Else Guthrie; Barbara Tomenson; Francis Creed

2009-01-01

264

Hyperreninemia Characterizing Women with Polycystic Ovary Syndrome Improves after Metformin Therapy  

Microsoft Academic Search

Background: Polycystic ovary syndrome (PCOS) is characterized by chronic anovulation, hyperandrogenemia and insulin resistance. Hyperreninemia is observed in insulin resistance and hyperandrogenemic states. Aims: To investigate the levels of total plasma renin and their possible relationship with insulin resistance and hyperandrogenemia and to explore the effect of metformin on these parameters in PCOS women. Methods: 48 PCOS women who were

Evanthia Diamanti-Kandarakis; Frangiskos N. Economou; Sarantis Livadas; Evangelia Tantalaki; Christina Piperi; Athanasios G. Papavassiliou; Dimitrios Panidis

2009-01-01

265

The use of Simvastatin Plus Metformin Therapy in Patients With Polycystic Ovarian Syndrome  

Microsoft Academic Search

Background: Polycystic ovarian syndrome (PCOS) is a common endocrine disorder that affects the fertility of reproductive-aged women due to high levels of testosterone. Metformin is currently used to treat patients with PCOS in order to improve the biochemical markers of the disease. New studies show that statin medications like simvastatin may prove efficacious with reduction of testosterone levels, and therefore,

Erin Carrick

2012-01-01

266

Increased uptake of guideline-recommended oral antiplatelet therapy: insights from the Canadian acute coronary syndrome reflective.  

PubMed

Current guideline-based recommendations for oral dual-antiplatelet therapy in an acute coronary syndrome (ACS) include the use of newer adenosine diphosphate receptor inhibitor (ADPri) regimens and agents. The Canadian ACS Reflective Program is a multicenter observational quality-enhancement project that compared the use of ADPri therapy in 2 phases (November 2011-March 2013 and April 2013-November 2013) and also compared ADPri use with previous national data from the Canadian Global Registry of Acute Coronary Events (2000-2008). Of 3099 patients with ACS, 30.6% had ST-segment elevation myocardial infarction (STEMI), 52.3% had non-STEMI, and 17% had unstable angina. There was high use of dual-antiplatelet therapy for ? 24 hours, with important increases noted when compared with previous national experience (P for trend, < 0.0001). Clopidogrel was the most commonly used ADPri (82.2%), with lower use of the newer agents ticagrelor (9.0%) and prasugrel (3.1%). Ticagrelor and prasugrel use was most frequent in patients with STEMI undergoing percutaneous coronary intervention PCI (34.3%). There was relatively lower use of ADPri therapy at discharge; it was given mainly to patients who did not undergo PCI (68.2%) and to those with non-ST-elevation ACS (82%). When comparing the 2 consecutive phases of data collection in the ACS Reflective, there was an approximate 3- and 2-fold increase in the early and discharge use of the newer ADPri agents, respectively. In conclusion, there has been a temporal increase in ADPri use compared with previous national experience and an increased uptake of newer ADPri agents. Additional work is needed to identify and address barriers limiting optimal implementation of these newer guideline-recommended agents into routine Canadian practice. PMID:25475475

Gandhi, Sumeet; Zile, Brigita; Tan, Mary K; Saranu, Jhansi; Bucci, Claudia; Yan, Andrew T; Robertson, Patrick; Quantz, Mackenzie A; Letovsky, Eric; Tanguay, Jean-Francois; Dery, Jean-Pierre; Fitchett, David; Madan, Mina; Cantor, Warren J; Heffernan, Michael; Natarajan, Madhu K; Wong, Graham C; Welsh, Robert C; Goodman, Shaun G

2014-12-01

267

Physical Therapy and Occupational Therapy in Progeria  

MedlinePLUS

... rights reserved. Page 2 of 5 Physical and Occupational Therapy in Progeria Hutchinson-Gilford Progeria Syndrome (“Progeria”, or “ ... Children with Progeria need Physical Therapy (PT) and Occupational Therapy (OT) as often as possible (optimally 2- 3 ...

268

Cyclophosphamide and methotrexate in Susac's Syndrome: a successful sequential therapy in a case with involvement of the cerebellum.  

PubMed

Susac's Syndrome (SS) is a rare disease with unknown aetiology due to a microangiopathy affecting the precapillary arterioles of the brain, retina, cochlea and semicircular canals. Neurological manifestations, visual dysfunction and hearing loss represent the classical clinical triad of SS. Diagnosis is confirmed by laboratory investigations, neuroimaging findings, fluoroangiography and inner-ear studies. An early treatment with steroids and immunosuppressors limits the sequelae of disease. We report a case of SS in which the clinical triad occurred in a very short period of time. Brain MRI showed the involvement of cerebellum, this representing a rare neuroradiological finding in SS. A full remission of disease was obtained by using corticosteroids and cyclophosphamide in the acute-subacute phase and methotrexate as maintenance therapy. This latter has never been used before in SS. PMID:24794491

Catarsi, Eleonora; Pelliccia, Veronica; Pizzanelli, Chiara; Pesaresi, Ilaria; Cosottini, Mirco; Migliorini, Paola; Tavoni, Antonio

2014-05-01

269

The clinical practice of Cognitive Behavioural Therapy for children and young people with a diagnosis of Asperger's Syndrome.  

PubMed

Children and young people diagnosed with Asperger's Syndrome (AS) have significant social-communication difficulties and impaired empathy and theory of mind skills. These difficulties place them at risk of developing mental health problems, particularly anxiety, depression and obsessive compulsive disorder. Although Cognitive Behavioural Therapy (CBT) is recognised as an effective intervention for these problems in both child and adult populations, little research has specifically looked at the use of CBT with children and young people with an AS diagnosis. However, limited evidence suggests that CBT, if suitably adapted, is a feasible and potentially helpful treatment option. This paper focuses on the clinical practice of CBT and explores how the underpinning therapeutic relationship can be modified to meet the cognitive needs of this particular group of young clients. PMID:20516059

Donoghue, Kate; Stallard, Paul; Kucia, Joanna

2011-01-01

270

Diffuse choroidal hemangioma associated with exudative retinal detachment in a Sturge-Weber syndrome case: Photodynamic therapy and intravitreous bevacizumab.  

PubMed

We report the case of a young female patient with a diffuse choroidal hemangioma (DCH) and glaucoma as part of Sturge-Weber syndrome (SWS) and symptomatic retinal detachment that was treated successfully with photodynamic therapy (PDT) and intravitreal bevacizumab (IVB). The patient was treated with a single session of PDT, a 689-nm laser was used to deliver 50J/cm(2) with a maximum spot size of 6400?m, for 166s. IVB was administered 3 days later. The exudative retinal detachment (ERD), macular edema and visual acuity improved one week after treatment. The patient was followed for 18 months with no recurrence of ERD, and her visual acuity was preserved. PDT followed by IVB may be an effective treatment option for visual deterioration due to ERD in patients with DCHs, as are found in SWS. PMID:25560419

Anaya-Pava, Edwin J; Saenz-Bocanegra, Carlos H; Flores-Trejo, Alejandro; Castro-Santana, Norma A

2015-03-01

271

Refractory postsurgical pyoderma gangrenosum in a patient with Beckwith Wiedemann syndrome: response to multimodal therapy.  

PubMed

Pyoderma gangrenosum (PG) is a rare neutrophilic dermatosis that may be difficult to diagnose and treat. We presented a 41-year-old woman who required skin grafting following third-degree burns to her left breast. She suffered recurrent graft dehiscence and infections over many years, prompting elective bilateral reduction mammoplasty. She subsequently developed suture margin ulcerations unresponsive to topical therapies and antibiotics. Skin biopsies were non-specific, and a clinical diagnosis of PG was established. Although initially responsive to corticosteroids, wounds promptly recurred following steroid taper. She was treated unsuccessfully with various immunomodulatory agents and underwent elective bilateral mastectomy. Following a mastectomy, she developed progressive deep chest wall ulcerations. She failed numerous immunomodulatory treatments, surgical wound closure and negative pressure wound therapy. Ultimately, treatment with adalimumab, mycophenolate mofetil and prednisone, in addition to hyperbaric oxygen therapy facilitated progressive healing. Our case highlights the role of collaborative multimodal therapy for the treatment of refractory PG. PMID:24154999

Fakhar, Faiza; Memon, Sehrish; Deitz, Diane; Abramowitz, Richard; Alpert, Deborah R

2013-01-01

272

Safety and efficacy of enzyme replacement therapy with idursulfase beta in children aged younger than 6years with Hunter syndrome.  

PubMed

Idursulfase beta (Hunterase®) has been used for enzyme replacement therapy (ERT) of patients with mucopolysaccharidosis II (MPS II, Hunter syndrome) aged 6years or older since 2012 in Korea. The objective of this study was to evaluate the safety and efficacy of ERT with idursulfase beta in Hunter syndrome children younger than 6years. This study was a 52-week, single center, single arm, open-label clinical trial (NCT01645189). Idursulfase beta (0.5mg/kg/week) was administered intravenously for 52weeks. The primary endpoint was safety assessed by adverse events (AEs). Secondary endpoints included vital signs, physical examination, ECG, laboratory tests, anti-idursulfase antibodies, and efficacy represented by changes in urinary glycosaminoglycan (GAG) at week 53 from baseline. In addition, growth indices and developmental milestones (Denver II test) were evaluated as exploratory variables. All six patients experienced at least one AE. A total of 109 AEs were reported. One patient experienced a serious AE (hospitalization due to gastroenteritis) that was considered not to be treatment related. One patient (16.7%) experienced infusion-related adverse drug reactions (ADRs), developing urticaria six times and a cough five times. There were no serious ADRs and no clinically significant changes in vital signs, physical exam, laboratory parameters, or ECG. Of the six patients, four (66.7%) showed anti-idursulfase antibodies and neutralizing antibodies on at least one occasion during the study. At week 53, urinary GAG was significantly reduced by -35.1±30.6mgGAG/g creatine from baseline (P=0.038). This study indicates that the safety and efficacy of idursulfase beta are similar to those reported in Hunter syndrome patients aged 6years or older. PMID:25219292

Sohn, Young Bae; Cho, Sung Yoon; Lee, Jieun; Kwun, Yonghee; Huh, Rimm; Jin, Dong-Kyu

2015-02-01

273

Activities of daily living in patients with Hunter syndrome: Impact of enzyme replacement therapy and hematopoietic stem cell transplantation.  

PubMed

The aim of this study was to assess the activities of daily living (ADL) in patients with Hunter syndrome (mucopolysaccharidosis II; MPS II) using a newly designed ADL questionnaire. We applied the questionnaire to evaluate clinical phenotypes and therapeutic efficacies of enzyme replacement therapy (ERT) and hematopoietic stem cell transplantation (HSCT). We also explored early signs and symptoms to make early diagnosis feasible. We devised a new ADL questionnaire with three domains: "movement," "movement with cognition," and "cognition." Each domain has four subcategories rated on a 5-point scale based on level of assistance. We also scored signs and symptoms unique to MPS by 12 subcategories (five points per category), providing 60 points in total. The questionnaire was first administered to 138 healthy Japanese controls (0.33-50years), and successively, to 74 Japanese patients with Hunter syndrome (4-49years). The patient cohort consisted of 51 severe and 23 attenuated phenotypes; 20 patients treated with HSCT, 23 patients treated early with ERT (?8years), 25 patients treated late with ERT (>8years), and 4 untreated patients. Among 18 severe phenotypic patients treated by HSCT, 10 were designated as early HSCT (?5years), while 8 were designated as late HSCT (>5years). Scores from patients with severe phenotypes were lower than controls and attenuated phenotypes in all categories. Among patients with severe phenotypes, there was a trend that HSCT provides a higher ADL score than early ERT, and there was a significant difference in ADL scores between late ERT and HSCT groups. Early ERT and early HSCT provided a higher score than late ERT and late HSCT, respectively. In conclusion, we have evaluated the feasibility of a new questionnaire in control population and patients with Hunter syndrome, leading to a novel evaluation method for clinical phenotypes and therapeutic efficacy. Early treatment with HSCT provides a better consequence in ADL of patients. PMID:25468646

Tanjuakio, Julian; Suzuki, Yasuyuki; Patel, Pravin; Yasuda, Eriko; Kubaski, Francyne; Tanaka, Akemi; Yabe, Hiromasa; Mason, Robert W; Montańo, Adriana M; Orii, Kenji E; Orii, Koji O; Fukao, Toshiyuki; Orii, Tadao; Tomatsu, Shunji

2015-02-01

274

Improvement of hypercortisolism by ?-blocker therapy in subclinical Cushing's syndrome associated with ACTH-independent macronodular adrenocortical hyperplasia.  

PubMed

A 61-year-old man with hypertension and diabetes was referred for the evaluation of multiple bilateral adrenal tumors. While Cushingoid features were not apparent, an elevated cortisol level in response to a low-dose dexamethasone suppression test (187.7 nmol/l), an elevated urinary cortisol level (170.9 nmol/day), and a weak response to a cosyntropin-releasing hormone (CRH) provocation test were observed. Furthermore, the serum cortisol level increased in response to a posture test or isoproterenol infusion. Accordingly, the patient was diagnosed as having ACTH-independent macronodular adrenal hyperplasia (AIMAH) with subclinical Cushing's syndrome associated with the aberrant expression of ?-adrenergic receptors. After 2 months of propranolol therapy, the serum cortisol responses to a posture test and isoproterenol infusion, the cortisol level in response to a low-dose dexamethasone suppression test (102.1 nmol/l), and the urinary cortisol level (165.9 nmol/day) all normalized. While the suppression of cortisol secretion was sustained for 24 months, glucose metabolism and adrenal size were unaffected. To our knowledge, this is the first report of AIMAH accompanied by subclinical Cushing's syndrome associated with the aberrant expression of ?-adrenergic receptors. Furthermore, propranolol inhibited cortisol hypersecretion in the present case. Additional cases or controlled studies are needed to determine the potential effect of propranolol on metabolic disorders and adrenal size in patients with AIMAH. PMID:19813002

Oki, Kenji; Yamane, Kiminori; Nakanishi, Shuhei; Nakashima, Reiko; Jitsuiki, Kuniaki; Kohno, Nobuoki

2009-12-01

275

Updates on the myo-inositol plus D-chiro-inositol combined therapy in polycystic ovary syndrome.  

PubMed

Polycystic ovary syndrome (PCOS) is one of the most common endocrine disorders affecting women of reproductive age. It is characterized by chronic anovulation, hyperandrogenism, and insulin resistance. It is the main cause of infertility due to the menstrual dysfunction and metabolic disorders. Women with PCOS also have an increased cardiovascular risk because of dyslipidemia and insulin resistance. So far, we have a lot of information about the etiology of PCOS, and many steps forward have been made about the diagnosis of this syndrome, but there is still no certainty about the therapy. Myo-inositol (MI) and D-chiro-inositol, two inositol stereoisomers, have been proven to be effective in PCOS treatment. However, only MI has been shown to have beneficial effects on reproductive function, whereas the administration of MI/D-chiro-inositol, in the physiological plasma ratio (i.e., 40:1) ensures better clinical results, such as the reduction of insulin resistance, androgens' blood levels, cardiovascular risk and regularization of menstrual cycle with spontaneous ovulation. PMID:24898153

Unfer, Vittorio; Porcaro, Giuseppina

2014-09-01

276

[Congenital myasthenic syndromes: difficulties in the diagnosis, course and prognosis, and therapy--The French National Congenital Myasthenic Syndrome Network experience].  

PubMed

Congenital myasthenic syndromes (CMS) are a heterogeneous group of disorders caused by genetic defects affecting neuromuscular transmission and leading to muscle weakness accentuated by exertion. Three different aspects have been investigated by members of the national French CMS Network: the difficulties in making a proper diagnosis; the course and long-term prognosis; and the response to therapy, especially for CMS that do not respond to cholinesterase inhibitors. CMS diagnosis is late in most cases because of confusion with other entities such as: congenital myopathies, due to the frequent presentation in patients of myopathies such as permanent muscle weakness, atrophy and scoliosis, and the abnormalities of internal structure, diameter and distribution of fibers (type I predominance, type II atrophy) seen on biopsy; seronegative autoimmune myasthenia gravis, when CMS is of late onset; and metabolic myopathy, with the presence of lipidosis in muscle. The long-term prognosis of CMS was studied in a series of 79 patients recruited with the following gene mutations: CHRNA; CHRNE; DOK7; COLQ; RAPSN; AGRN; and MUSK. Disease-course patterns (progressive worsening, exacerbation, stability, improvement) could be variable throughout life in a given patient. DOK7 patients had the most severe disease course with progressive worsening: of the eight wheelchair-bound and ventilated patients, six had mutations of this gene. Pregnancy was a frequent cause of exacerbation. Anticholinesterase agents are the first-line therapy for CMS patients, except for cases of slow-channel CMS, COLQ and DOK7. In our experience, 3,4-DAP was a useful complement for several patients harboring CMS with AChR loss or RAPSN gene mutations. Ephedrine was given to 18 patients (eight DOK7, five COLQ, four AGRN and one RAPSN). Tolerability was good. Therapeutic responses were encouraging even in the most severely affected patients, particularly with DOK7 and COLQ. Salbutamol was a good alternative in one patient who was allergic to ephedrine. PMID:23452772

Eymard, B; Stojkovic, T; Sternberg, D; Richard, P; Nicole, S; Fournier, E; Béhin, A; Laforęt, P; Servais, L; Romero, N; Fardeau, M; Hantaď, D

2013-02-01

277

Temporal patterns of lipid testing and statin therapy in acute coronary syndrome patients (from the Canadian GRACE Experience).  

PubMed

Current guidelines recommend the measurement of fasting lipid profile and use of statins in all patients with acute coronary syndrome (ACS). However, the temporal trends of lipid testing and statin therapy in "real-world" patients with ACS are unclear. From January 1999 through December 2008, the prospective, multicenter, Global Registry of Acute Coronary Events (GRACE/GRACE(2)/CANRACE) enrolled 13,947 patients with ACS in Canada. We stratified the study population based on year of presentation into 3 groups (1999 to 2004, 2005 to 2006, and 2007 to 2008) and compared the use of lipid testing and use of statin therapy in hospital. Overall, 70.8% of patients underwent lipid testing and 79.4% received in-hospital statin therapy; these patients were younger and had lower GRACE risk scores (p <0.001 for the 2 comparisons) compared to those who did not. Over time there was a significant increase in rates of in-hospital statin therapy (70% in 1999 to 2004 to 84.5% in 2007 to 2008, p for trend < 0.001) but only a minor increase in rates of lipid testing (69.4% in 1999 to 2004 to 72.4% in 2007 to 2008, p for trend = 0.003). After adjusting for confounders, this increasing temporal trend remained statistically significant for statin therapy (p <0.001) but not for lipid testing. Lipid testing was independently associated with in-hospital statin use (adjusted odds ratio 1.62, 95% confidence interval 1.27 to 2.08, p <0.001). In patients who did have lipid testing, those with low-density lipoprotein cholesterol level >130 mg/dl (3.4 mmol/L) were more likely to be treated with in-hospital statins. In conclusion, there has been a significant temporal increase in the use of in-hospital statin therapy but only a minor increase in lipid testing. Lipid testing was strongly associated with in-hospital statin use. A substantial proportion of patients with ACS, especially those at higher risk, still do not receive these guideline-recommended interventions in contemporary practice. PMID:22381155

Elbarouni, Basem; Banihashemi, S Behnam; Yan, Raymond T; Welsh, Robert C; Kornder, Jan M; Wong, Graham C; Anderson, Frederick A; Spencer, Frederick A; Grondin, François R; Goodman, Shaun G; Yan, Andrew T

2012-05-15

278

Hurler syndrome  

MedlinePLUS

Enzyme replacement therapy for Hurler syndrome adds a working form of the missing enzyme to the body. The medication, called laronidase (Aldurazyme), is given through a vein (intravenously). Talk to your ...

279

Yang/Qi Invigoration: An Herbal Therapy for Chronic Fatigue Syndrome with Yang Deficiency?  

PubMed Central

According to traditional Chinese medicine (TCM) theory, Yang and Qi are driving forces of biological activities in the human body. Based on the crucial role of the mitochondrion in energy metabolism, we propose an extended view of Yang and Qi in the context of mitochondrion-driven cellular and body function. It is of interest that the clinical manifestations of Yang/Qi deficiencies in TCM resemble those of chronic fatigue syndrome in Western medicine, which is pathologically associated with mitochondrial dysfunction. By virtue of their ability to enhance mitochondrial function and its regulation, Yang- and Qi-invigorating tonic herbs, such as Cistanches Herba and Schisandrae Fructus, may therefore prove to be beneficial in the treatment of chronic fatigue syndrome with Yang deficiency.

Wong, Hoi Shan; Chen, Jihang; Ko, Kam Ming

2015-01-01

280

[Therapy of Frey syndrome with botulinum toxin A. Experiences with a new method of treatment].  

PubMed

The effectiveness of botulinum toxin for the treatment of Frey's syndrome is demonstrated. Since December 1993, 14 patients with severe symptomatic gustatory sweating have been treated at the ENT Department, University of Göttingen. Botulinum toxin A (approximately 0.5 U/cm2) was injected intracutaneously into the affected skin area as determined by Minor's starch iodine test. Gustatory sweating in the treated skin area ceased completely within 2 days and did not reappear during the period of following (13 months maximum follow-up). There were no side effects. Findings show that local botulinum toxin injections are a highly effective, safe and minimally invasive treatment for Frey's syndrome. Moreover, this could be a new therapeutic tool for other forms of hyperhidrosis. PMID:8530311

Drobik, C; Laskawi, R; Schwab, S

1995-11-01

281

Therapy of the neonatal abstinence syndrome with tincture of opium or morphine drops  

Microsoft Academic Search

Background:Treating opioid-addicted women with methadone in pregnancy increased the number of newborns suffering from neonatal abstinence syndrome (NAS). High-pitch crying, insomnia, tremor, myoclonic jerks, vomiting, diarrhoea and poor weight gain were reported symptoms, which were evaluated using the Finnegan (F)-score. Earlier phenobarbital or paregoric had been used to suppress symptoms. We surveyed the administration of pure ?-agonist morphine (MO) in

Stefan Langenfeld; Larissa Birkenfeld; Peter Herkenrath; Carsten Müller; Martin Hellmich; Martin Theisohn

2005-01-01

282

Syndrom der persistierenden Müller-StrukturenEndokrinologische Aspekte und Empfehlungen zur Therapie  

Microsoft Academic Search

Zusammenfassung  \\u000a Das Syndrom der persistierenden Müller-Strukturen (SPMS) ist definiert durch den Nachweis von Müller-Strukturen (Tuben, Uterus,\\u000a proximale Vagina) bei Patienten mit vollständig männlich differenziertem äußeren Genitale und normalem, männlichen Chromosomensatz.\\u000a Bei typischer Klinik – gleichzeitiges Vorliegen von Leistenhernie und kontralateralem Kryptorchismus – sollte daher eine gründliche\\u000a sonographische Untersuchung des Abdomens erfolgen. Wir berichten über 3 Patienten, bei denen im Rahmen

S. A. Wudy; B. Töpke; J. Homoki; W. Sorgo; B. Höhmann; W. M. Teller

1997-01-01

283

A Biopsychosocial Therapy Model for Chronic Prostatitis\\/Chronic Pelvic Pain Syndrome  

Microsoft Academic Search

\\u000a Chronic prostatitis\\/chronic pelvic pain syndrome (CP\\/CPPS) has long been considered a frustrating and prevalent condition\\u000a by patients and treating physicians alike. Pain is noted as the cardinal symptom in CP\\/CPPS and is strongly associated with\\u000a a host of intra and interpersonal difficulties reported by patients in domains such as pain, disability, relations, and overall\\u000a quality of life. With no tenable

Dean A. Tripp

284

Deep brain stimulation therapy for treatment-refractory Tourette’s syndrome  

Microsoft Academic Search

Tourette’s syndrome is a chronic neurobehavioral disorder that can demonstrate refractoriness to conservative treatments,\\u000a or to invasive nonsurgical treatments such as botulinum toxin infiltration, or to psychobehavioral treatments. In these cases,\\u000a the surgical option is often proposed, either with lesional interventions, or more recently with deep brain stimulation (DBS).\\u000a This latter modality is currently preferred because of its reversibility and

Marco Sassi; Mauro Porta; Domenico Servello

2011-01-01

285

Bone mass in androgen-insensitivity syndrome: Response to hormonal replacement therapy  

Microsoft Academic Search

The response of bone mass to long-term treatment with estrogen and progesterone in patients with complete androgen-insensitivity syndrome (AIS) is unknown. We report a 17-year-old female patient (karyotype 46 X, Y) with AIS studied during a 4-year period. Bone mineral density (BMD) measured by dual X-ray absorptiometry in lumbar spine and proximal femur was sharply reduced at the initial visit,

M. Mufioz-Torres; E. Jódar; M. Quesada; F. Escobar-Jiménez

1995-01-01

286

Metformin therapy throughout pregnancy reduces the development of gestational diabetes in women with polycystic ovary syndrome  

Microsoft Academic Search

Objective: To assess whether metformin safely reduced development of gestational diabetes in women with the polycystic ovary syndrome (PCOS).Design: Prospective and retrospective study.Setting: Outpatient clinical research center.Patient(s): The prospective study included 33 nondiabetic women with PCOS who conceived while taking metformin and had live births; of these, 28 were taking metformin through delivery. The retrospective study included 39 nondiabetic women

C. J Glueck; Ping Wang; Suichi Kobayashi; Harvey Phillips; Luann Sieve-Smith

2002-01-01

287

An Unusual Combination of Phototoxicity and Stevens-Johnson Syndrome due to Antimalarial Therapy  

Microsoft Academic Search

A 12-year-old boy developed a phototoxic rash with subsequent progression to Stevens-Johnson syndrome due to prophylactic ingestion of antimalarials (chloroquine and sulfadoxine-pyrimethamine; Fansidar®). The patient recovered from his skin symptoms after 4 weeks during which he received systemic corticosteroids and antibiotics. This unusual combination of two different patterns of adverse cutaneous drug reactions was most probably caused by the sulfonamide

B. Ordel; A. Sivayathorn; H. Hönigsmann

1989-01-01

288

AMP kinase and malonyl-CoA: targets for therapy of the metabolic syndrome  

Microsoft Academic Search

Patients with the metabolic syndrome are characterized by insulin resistance, obesity and a predisposition to hypertension, dyslipidaemia, pancreatic ?-cell dysfunction, type 2 diabetes and premature atherosclerosis. Here we review the hypothesis that a common feature linking these multiple abnormalities is dysregulation of the AMP-activated protein kinase (AMPK)\\/malonyl-CoA fuel-sensing and signalling network. It is proposed that such dysregulation leads to alterations

Neil Ruderman; Marc Prentki

2004-01-01

289

Frequency of impulse control behaviours associated with dopaminergic therapy in restless legs syndrome  

E-print Network

-spinal dopaminer- gic system exists [3] and that it interacts with the iron metabolism [4]. Low doses of dopamine agonists (DA) are effective for improving sleep disturbances, daytime symptoms and quality of life of RLS sufferers [5]. The DAs ropinirole... patients. Table 1 Impulse control and compulsive behaviours diagnoses and medication data in patients with restless legs syndrome Patient ICB diagnosis DA type/dose L-dopa dose 1 Compulsive shopping, binge eating Ropinirole/2mg 100 mg 2 Computer gambling...

Voon, Valerie; Schoerling, Andrea; Wenzel, Sascha; Ekanayake, Vindhya; Reiff, Julia; Trenkwalder, Claudia; Sixel-Doring, Friederike

2011-09-28

290

Successful treatment of Bing-Neel syndrome using combination therapy with fludarabine and rituximab.  

PubMed

Bing-Neel syndrome is known as Waldenström's macroglobulinemia with central nervous system infiltration by neoplastic lymphoplasmacytoid and plasma cells. A 74-year-old man was admitted because of progressive cognitive impairment. Serum immunoelectrophoresis showed a monoclonal IgM-kappa component. Bone marrow aspiration revealed 59% small lymphocytes showing plasmacytoid differentiation. Bone marrow flow cytometry disclosed a population of kappa light-chain positive lymphoid cells expressing CD19, CD20, CD38, and CD138. Magnetic resonance imaging of the brain demonstrated gadolinium-enhancement in the right temporo-parieto-occipital meninges with sulcal enhancement. Cerebrospinal fluid cytology showed a population of lymphoplasmacytoid cells, positive for CD19, CD20, CD25, and kappa light-chain. Based on these findings, Bing-Neel syndrome was diagnosed. Although combination chemotherapy consisting of intrathecal methotrexate and oral cyclophosphamide was started, his symptoms continued to worsen. Then, we initiated treatment with a regimen consisting of fludarabine/rituximab (FR). After 6 courses of this FR regimen, a complete remission was achieved. Our case suggests the FR regimen to potentially be an effective treatment option for Bing-Neel syndrome of the scattered type. PMID:25744044

Nagaharu, Keiki; Miyazami, Kana; Imai, Hiroshi; Tamura, Asako; Umino, Akira; Fujieda, Atsushi; Sugimoto, Yuka; Yamaguchi, Motoko; Masuya, Masahiro; Katayama, Naoyuki

2014-12-01

291

Grade 4 febrile neutropenia and Fournier’s Syndrome associated with triple therapy for hepatitis C virus: A case report  

PubMed Central

The use of triple therapy for hepatitis C not only increases the rate of sustained virological responses compared with the use of only interferon and ribavirin (RBV) but also leads to an increased number of side effects. The subject of this study was a 53-year-old male who was cirrhotic with hepatitis C virus genotype 1 A and was a previous null non-responder. We initially attempted retreatment with boceprevir (BOC), Peg-interferon and RBV, and a decrease in viral load was observed in the 8th week. In week 12, he presented with disorientation, flapping, fever, tachypnea, arterial hypotension and tachycardia. He also exhibited leucopenia with neutropenia. Cefepime and filgrastim were initiated, and treatment for hepatitis C was suspended. A myelogram revealed hypoplasia, cytotoxicity and maturational retardation. After 48 h, he developed bilateral inguinal erythema that evolved throughout the perineal area to the root of the thighs, with exulcerations and an outflow of seropurulent secretions. Because we hypothesized that he was suffering from Fournier’s Syndrome, treatment was replaced with the antibiotics imipenem, linezolid and clindamycin. After this new treatment paradigm was initiated, his lesions regressed without requiring surgical debridement. Triple therapy requires knowledge regarding the management of adverse effects and drug interactions; it also requires an understanding of the importance of respecting the guidelines for the withdrawal of treatment. In this case report, we observed an adverse event that had not been previously reported in the literature with the use of BOC. PMID:25018856

Oliveira, Kelly Cristhian Lima; Cardoso, Emili de Oliveira Bortolon; de Souza, Suzana Carla Pereira; Machado, Flávia Souza; Zangirolami, Carlos Eduardo Alves; Moreira, Alecsandro; Silva, Giovanni Faria; de Oliveira, Cássio Vieira

2014-01-01

292

Ex-vivo Gene Therapy Restores LEKTI Activity and Corrects the Architecture of Netherton Syndrome-derived Skin Grafts  

PubMed Central

Netherton syndrome (NS) is a debilitating congenital skin disorder caused by mutations in the SPINK5 gene encoding the lymphoepithelial Kazal-type-related inhibitor (LEKTI). It is characterized by defective keratinization, recurrent infections, and hypernatraemic dehydration with a mortality rate of about 10% in the first year of life. Currently, there are no curative treatments for NS. We have developed a HIV-1 based, self-inactivating lentiviral vector to express SPINK5 in keratinocytes as part of an ex-vivo gene therapy strategy for NS. High transduction efficiency was achieved in NS keratinocytes and reconstitution of LEKTI expression was confirmed in previously deficient cells. These genetically corrected keratinocytes were further tested in an in vitro organotypic culture (OTC) system and in vivo mouse/human skin engraftment model. Results showed correction of epidermal architecture in both OTCs and regenerated skin grafts. Importantly, the results from corrected skin grafts indicated that even where detectable LEKTI expression was restored to a limited numbers of cells, a wider bystander benefit occurred around these small populations. As LEKTI is a secreted protein, the genetically modified graft may provide not only an immediate local protective barrier, but also act as a source of secreted LEKTI providing a generalized benefit following ex-vivo gene therapy. PMID:20877344

Di, Wei-Li; Larcher, Fernado; Semenova, Ekaterina; Talbot, Gill E; Harper, John I; Del Rio, Marcela; Thrasher, Adrian J; Qasim, Waseem

2011-01-01

293

Lack of durable disease control with chemotherapy for mycosis fungoides and Sézary syndrome: a comparative study of systemic therapy.  

PubMed

Numerous systemic treatment options exist for patients with mycosis fungoides (MF) and Sézary syndrome (SS), but no large comparative studies are published. To study the efficacy of treatments, a retrospective analysis of our cutaneous lymphoma database was undertaken, with 198 MF/SS patients undergoing systemic therapies. The primary end point was time to next treatment (TTNT). Patients with advanced-stage disease made up 53%. The median follow-up time from diagnosis for all alive patients was 4.9 years (range 0.3-39.6), with a median survival of 11.4 years. Patients received a median of 3 lines of therapy (range 1-13), resulting in 709 treatment episodes. Twenty-eight treatment modalities were analyzed. The median TTNT for single- or multiagent chemotherapy was only 3.9 months (95% confidence interval [CI] 3.2-5.1), with few durable remissions. ?-interferon gave a median TTNT of 8.7 months (95% CI 6.0-18.0), and histone deacetylase inhibitors (HDACi) gave a median TTNT of 4.5 months (95% CI 4.0-6.1). When compared directly with chemotherapy, interferon and HDACi both had greater TTNT (P < .00001 and P = .01, respectively). This study confirms that all chemotherapy regimens assessed have very modest efficacy; we recommend their use be restricted until other options are exhausted. PMID:25336628

Hughes, Charlotte F M; Khot, Amit; McCormack, Christopher; Lade, Stephen; Westerman, David A; Twigger, Robert; Buelens, Odette; Newland, Kate; Tam, Constantine; Dickinson, Michael; Ryan, Gail; Ritchie, David; Wood, Colin; Prince, H Miles

2015-01-01

294

Genetic Syndromes and Genes Involved in the Development of the Female Reproductive Tract: A Possible Role for Gene Therapy  

PubMed Central

Müllerian and vaginal anomalies are congenital malformations of the female reproductive tract resulting from alterations in the normal developmental pathway of the uterus, cervix, fallopian tubes, and vagina. The most common of the Müllerian anomalies affect the uterus and may adversely impact reproductive outcomes highlighting the importance of gaining understanding of the genetic mechanisms that govern normal and abnormal development of the female reproductive tract. Modern molecular genetics with study of knock out animal models as well as several genetic syndromes featuring abnormalities of the female reproductive tract have identified candidate genes significant to this developmental pathway. Further emphasizing the importance of understanding female reproductive tract development, recent evidence has demonstrated expression of embryologically significant genes in the endometrium of adult mice and humans. This recent work suggests that these genes not only play a role in the proper structural development of the female reproductive tract but also may persist in adults to regulate proper function of the endometrium of the uterus. As endometrial function is critical for successful implantation and pregnancy maintenance, these recent data suggest a target for gene therapy. Future research will be needed to determine if gene therapy may improve reproductive outcomes for patients with demonstrated deficient endometrial expression related to abnormal gene expression. PMID:25506511

Connell, MT; Owen, CM; Segars, JH

2014-01-01

295

Turner syndrome  

MedlinePLUS

Growth hormone may help a child with Turner syndrome grow taller. Estrogen replacement therapy is often started when the girl is 12 or 13 years old. This helps trigger the growth of breasts, ... Women with Turner syndrome who wish to become pregnant ...

296

A randomised trial of adaptive pacing therapy, cognitive behaviour therapy, graded exercise, and specialist medical care for chronic fatigue syndrome (PACE): statistical analysis plan  

PubMed Central

Background The publication of protocols by medical journals is increasingly becoming an accepted means for promoting good quality research and maximising transparency. Recently, Finfer and Bellomo have suggested the publication of statistical analysis plans (SAPs).The aim of this paper is to make public and to report in detail the planned analyses that were approved by the Trial Steering Committee in May 2010 for the principal papers of the PACE (Pacing, graded Activity, and Cognitive behaviour therapy: a randomised Evaluation) trial, a treatment trial for chronic fatigue syndrome. It illustrates planned analyses of a complex intervention trial that allows for the impact of clustering by care providers, where multiple care-providers are present for each patient in some but not all arms of the trial. Results The trial design, objectives and data collection are reported. Considerations relating to blinding, samples, adherence to the protocol, stratification, centre and other clustering effects, missing data, multiplicity and compliance are described. Descriptive, interim and final analyses of the primary and secondary outcomes are then outlined. Conclusions This SAP maximises transparency, providing a record of all planned analyses, and it may be a resource for those who are developing SAPs, acting as an illustrative example for teaching and methodological research. It is not the sum of the statistical analysis sections of the principal papers, being completed well before individual papers were drafted. Trial registration ISRCTN54285094 assigned 22 May 2003; First participant was randomised on 18 March 2005. PMID:24225069

2013-01-01

297

Therapy Insight: congestive heart failure, chronic kidney disease and anemia, the cardio–renal–anemia syndrome  

Microsoft Academic Search

Congestive heart failure (CHF) and chronic kidney disease (CKD) often progress to end stage even with optimum medical therapy. One factor that is common to both conditions is anemia, which is present in about a third of CHF patients. CHF can cause or worsen both anemia and CKD, and CKD can cause or worsen both anemia and CHF. Thus, a

Donald S Silverberg; Dov Wexler; Adrian Iaina

2005-01-01

298

Cognitive-Behavioral Therapy for Somatization and Symptom Syndromes: A Critical Review of Controlled Clinical Trials  

Microsoft Academic Search

Objective: Few treatments for somatization have been proven effective. In the past decade, however, clinical trials of cognitive-behavioral therapy (CBT) have been promising. Our aim was to critically review and synthesize the evidence from these trials. Methods: A search of the Medline database from 1966 through July 1999 was conducted to identify controlled trials designed to evaluate the efficacy of

Kurt Kroenke; Ralph Swindle

2000-01-01

299

[Metabolic therapy with Actovegin in case of ischemic syndrome of limbs].  

PubMed

Actovegin was used in complex conservative and surgical treatment of lower limb chronic ischemia and diabetic foot syndrome in 200 patients. In 120 patients Actovegin was used in combination with reconstructive operations, angioplasty and stenting for preparation for surgery. In 80 patients Actovegin was used as independent method of treatment. In severe cases the medicine was injected intravenously 1000-2000 mg ?10-15, and in mild degrees (IIA-IIB) - 400-800 mg intramuscularly with conversion on the tablet form in the future. Clinical picture, dopplerographic and microcirculatory data evidence about absolute safety, efficiency and comparative cheapness of the parenteral and enteral forms of Actovegine. PMID:24736538

Dibirov, M D

2014-01-01

300

Therapy-related myelodysplastic syndrome presenting as fulminant heart failure secondary to myeloid sarcoma  

PubMed Central

Rapidly progressive heart failure is commonly caused by an extensive myocardial infarction, a mechanical complication of infarction, myocarditis, or acute valvular insufficiency. We present an unusual case that was caused by a diffuse infiltration of the myocardium with leukemic cells (myeloid sarcoma). The patient presented with episodic shortness of breath, he was anemic and thrombocytopenic, and his bone marrow biopsy revealed myelodysplastic syndrome from treatment for oligodendroglioma. His clinical course was characterized by a chronic leak of cardiac enzymes, a new right bundle branch block, and a large pericardial effusion causing tamponade and death from fulminant heart failure and ventricular arrhythmias within 2 weeks. At autopsy, the heart was massively infiltrated with myeloblasts and other immature myeloid cells. There was no evidence of acute leukemia in the bone marrow or peripheral blood. Cardiac infiltration in a patient with myelodysplastic syndrome is extremely rare, especially in the absence of bone marrow involvement by blasts. The recognition of this entity is becoming increasingly important as the incidence of cardiac myeloid sarcoma may be on the rise as the number of patients receiving chemotherapy increases. PMID:21544187

Matkowskyj, Kristina A.; Wiseman, William R.; Robin, Jason C.; Norvell, John P.; Puthumana, Jyothy; Nelson, Beverly; Peterson, LoAnn; McGarry, Thomas J.

2010-01-01

301

[Current therapy of bile duct cysts. II. Intrahepatic cysts (Caroli syndrome)].  

PubMed

17 patients with a Caroli's syndrome are reviewed with emphasis on clinical features and late results (follow-up: 2-10 years). The group consists of 9 women and 8 men ranging in age from 17 to 80 years (mean of 42.6). 14 patients had a diffuse form, 5 of whom had periportal fibrosis with secondary biliary cirrhosis, which in 3 cases resulted in a portal hypertension with hepatic failure. In only 3 cases the dilatation was limited to the left lobe of the liver. 9 of the patients had previously undergone surgery in the form of cholecystectomy and choledocholithotomy elsewhere. The disease was complicated by lithiasis (14 intrahepatic, 9 extrahepatic). Curative treatment was only possible in the 3 patients with unilobar Caroli's syndrome (partial liver resection). In 5 patients we performed a cholecystectomy and choledocholithotomy combined in 2 cases with a transduodenal sphincterotomy. There was no operative death. Endoscopic treatment consisted in removal of stones and decompression of the biliary tree by sphincterotomy in 9 cases and endoscopic retrograde biliary drainage (endoprosthesis) in 5 cases. Excepting the 3 curative operated patients who are asymptomatic respectively 2, 4 and 5 years after surgery, the remaining cases had repeated bouts of acute pains accompanied by recurrent episodes of cholangitis. A late mortality of 23.5% (4 patients) is proof of the poor prognosis of this disease. PMID:8681697

Benhidjeb, T; Müller, J M; Gellert, K; Zanow, J; Rudolph, B

1996-03-01

302

Calcul par simulation des paramčtres dosimétriques pour le noyau cellulaire aprčs irradiation ? in vitro  

NASA Astrophysics Data System (ADS)

Determination of absorbed dose in biological targets after high LET ? particules irradiation needs heavy calculations. A softwave has been developed in order to allow everyone to calculate hit probability and absorbed dose. It is particularly adapted to the study of cell cultures irradiated with electrodeposited source or ?-beam accelerator. It is based first, on a random generator of ?-track homogeneously distributed in 4?, second, on the evaluation of energy loss in the different media along the track and then on a statistical analysis of the results. This method is accurate and low time consuming. The target is either modelised by an ellipsoid or represented by its 3D shape recorded using confocal microscopy. Des calculs dosimétriques complexes sont nécessaires pour l'évaluation des doses délivrées dans des cibles biologiques aprčs irradiation par des particules ? de haut TEL. Un logiciel a été développé pour rendre facilement accessible le calcul de la probabilité pour atteindre la cible et de la dose absorbée. il est particuličrement adapté ŕ l'étude des cultures cellulaires irradiées par des sources électrodéposées de radionucléides ou des accélérateurs de particules. Il repose sur un générateur de traces aléatoires, sur une approximation de la perte d'énergie dans les différents milieux traversés et sur une exploitation statistique des résultats obtenus. Cette méthode s'avčre précise et rapide. La cible est modélisée par un ellipsoďde ou représentée par son image 3D obtenue en microscopie confocale.

Le Foll, L.; Bailly, I.; Fritsch, P.

1998-04-01

303

Enzyme replacement therapy in a murine model of Morquio A syndrome  

Microsoft Academic Search

Mucopolysaccharidosis IVA (MPS IVA) is an autosomal recessive disorder caused by a deficiency of N-acetylgalactosamine-6-sulfate sulfatase (GALNS), leading to accumulation of keratan sulfate (KS) and chrondroitin-6-sulfate. The pharmacokinetics and biodistributions were determined for two recombinant human GALNSs produced in CHO cell lines: native GALNS and sulfatase-modifier-factor 1 (SUMF1) modified GALNS. Preclinical studies of enzyme replacement therapy (ERT) by using two

Shunji Tomatsu; Adriana M. Montano; Amiko Ohashi; Monica A. Gutierrez; Hirotaka Oikawa; Toshihiro Oguma; Vu Chi Dung; Tatsuo Nishioka; Tadao Orii; William S. Sly

2008-01-01

304

Transformation of Sézary syndrome into CD30+ anaplastic large T-cell lymphoma after alemtuzumab therapy with evidence of clonal unity.  

PubMed

Alemtuzumab is a humanized mouse antibody targeting the CD52 cell surface, which has been effective in patients with advanced stage mycosis fungoides (MF) including erythrodermic MF and Sézary syndrome. There are a few descriptions of large cell transformation after its administration. A young patient with an acute onset of Sézary syndrome treated initially unsuccessfully with fludarabine and cyclophosphamide and later on successfully with alemtuzumab has been described. Three weeks after the beginning of therapy, however, she developed transformed T-cell lymphoma indistinguishable from CD30 anaplastic large-cell lymphoma. After bone marrow transplantation, the transformed CD30 cutaneous T-cell lymphoma recurred as a transformed CD30 plaque MF. All 3 types of lesions showed the same T-cell receptor clonal gene rearrangement, which supports the notion that Sézary syndrome, CD30 anaplastic large-cell lymphoma, and MF are interrelated. PMID:25548993

Nevet, Mariela Judith; Zuckerman, Tsila; Sahar, Dvora; Bergman, Reuven

2015-01-01

305

Omega-3 Essential Fatty Acids Therapy for Dry Eye Syndrome: A Meta-Analysis of Randomized Controlled Studies  

PubMed Central

Background Dry eye is a common, complex condition that can reduce ocular comfort and visual performance. The impact on quality of life has been rated as similar to the effect of moderate angina and, in more severe cases, dialysis and severe angina. This study aimed to use meta-analysis to compare omega-3 fatty acid and placebo fatty acid in the management of dry eye syndrome. Material/Methods Comparative studies published until 1 June 2014 were searched through a comprehensive search of the Medline, Embase, Web of Science, and the Cochrane Library electronic databases. A systematic review and cumulative analysis of comparative studies reporting the effect of omega-3 fatty acid on dry eye syndrome was conducted. All analyses were performed using the Review Manager (RevMan) v.5 software (Nordic Cochrane Centre, Copenhagen, Denmark). Results The trials involved a total of 790 participants in 7 independent studies. All the studies are published between 2007 and 2013. Meta-analysis of the 5 studies that reported data in mean SD values revealed that the tear break-up time (TBUT) was significantly greater by 1.58 s (WMD=1.58, 95% CI=0.60 to 2.55; P=0.007). Combination of all the Schirmer’s test data showed that omega-3 fatty acid supplementation could significantly improve the Schirmer’s test (WMD=0.74, 95% CI=0.29 to 1.19; P=0.001). However, the combination of all the OSDI test data showed that omega-3 fatty acid supplementation did not significantly improve the OSDI test results (WMD=?4.54, 95% CI=?9.85 to 0.78; P=0.09). Conclusions Based on the data included in our meta-analysis, omega-3 fatty acid was associated with better TBUT and Schirmer’s. No significant differences were detected in OSDI test results. Consequently, our findings suggest that omega-3 fatty acid offers is an effective therapy for dry eye syndrome. PMID:25193932

Liu, Aihua; Ji, Jian

2014-01-01

306

Gitelman-like syndrome after cisplatin therapy: a case report and literature review  

PubMed Central

Background Cisplatin is a well-known nephrotoxic antineoplastic drug. Chronic hypokalemic metabolic alkalosis with hypomagnesemia and hypocalciuria is one of the rare complications associated with its use. Case presentation A 42- year-old woman presented with a 20 year-history of hypokalemic metabolic alkalosis with hypomagnesemia and hypocalciuria after cisplatin-based chemotherapy for ovarian cancer. This patient has had chronic muscle aches and fatigue and has had episodic seizure-like activity and periodic paralysis. Only thirteen other patients with similar electrolyte abnormalities have been described in the literature. This case has the longest follow-up. Conclusion Cisplatin can cause permanent nephrotoxicity, including Gitelman-like syndrome. This drug should be considered among the possible causes of chronic unexplained electrolyte disorders. PMID:16723030

Panichpisal, Kessarin; Angulo-Pernett, Freddy; Selhi, Sharmila; Nugent, Kenneth M

2006-01-01

307

The role of aspirin resistance on outcome in patients with acute coronary syndrome and the effect of clopidogrel therapy in the prevention of major cardiovascular events  

Microsoft Academic Search

Background: Aspirin resistance may increase up to more then threefold the risk of major cardiovascular events (MACE) in patients with\\u000a stable coronary artery disease.\\u000a \\u000a \\u000a Aim:The aim of our study was to determine; the prevalence of aspirin resistance in patients with acute coronary syndromes, the\\u000a role of aspirin resistance on outcome in the follow-up and the effect of clopidogrel therapy in

Burak Pamukcu; Huseyin Oflaz; Aytac Oncul; Berrin Umman; Fehmi Mercanoglu; Mustafa Ozcan; Mehmet Meric; Yilmaz Nisanci

2006-01-01

308

Central sensitization does not identify patients with carpal tunnel syndrome who are likely to achieve short-term success with physical therapy  

Microsoft Academic Search

The aim of the current study was to identify whether hyperexcitability of the central nervous system is a prognostic factor\\u000a for individuals with carpal tunnel syndrome (CTS) likely to experience rapid and clinical self-reported improvement following\\u000a a physical therapy program including soft tissue mobilization and nerve slider neurodynamic interventions. Women presenting\\u000a with clinical and electrophysiological findings of CTS were involved

César Fernández-de-las-Peńas; Joshua A. Cleland; Ricardo Ortega-Santiago; Ana Isabel de-la-Llave-Rincon; Almudena Martínez-Perez; Juan A. Pareja

2010-01-01

309

Impact of metformin therapy on ovarian stimulation and outcome in ‘coasted’ patients with polycystic ovary syndrome undergoing in-vitro fertilization  

Microsoft Academic Search

This study was designed to determine if metformin therapy improves ovarian stimulation and IVF outcomes in coasted patients with clomiphene-resistant polycystic ovarian syndrome (PCOS). A retrospective data analysis was performed on women with clomiphene citrate-resistant PCOS treated with or without metformin, who underwent 72 cycles of IVF-embryo transfer with intracytoplasmic sperm injection (ICSI). In 59 cycles, patients were coasted to

LA Stadtmauer; SK Toma; RM Riehl; LM Talbert

2002-01-01

310

Metformin therapy is associated with a decrease in plasma plasminogen activator inhibitor-1, lipoprotein(a), and immunoreactive insulin levels in patients with the polycystic ovary syndrome  

Microsoft Academic Search

Sixteen nondiabetic women with polycystic ovary syndrome (PCOS) aged 18 to 33 years were studied before and after 8 weeks on metformin (1.5 g\\/d) therapy to assess whether reducing hyperinsulinemia would reduce the levels of the major inhibitor of fibrinolysis, antigenic plasminogen activator inhibitor type 1 (PAI-1). Compared with six normal control women, PCOS women had a higher body mass

Elsy M. Velazquez; Soaira G. Mendoza; Ping Wang; Charles J. Glueck

1997-01-01

311

The Effect of an Occupational Therapy Role-Playing Intervention on the Social Skills of Adolescents With Asperger's Syndrome: A Pilot Study  

Microsoft Academic Search

The purpose of the authors in this pilot study was to assess the effects of an occupational therapy role-playing intervention for adolescents with Asperger's syndrome. An ABA multiple baseline single-subject design across participants was used with three adolescents to determine if a six-week intervention could increase targeted social behaviors. Data were collected over baseline, intervention, and probe phases. Three methods

Sharon A. Gutman; Emily I. Raphael-Greenfield; Sabrina Salvant

2012-01-01

312

Type 2 diabetes mellitus and the cardiometabolic syndrome: impact of incretin-based therapies  

PubMed Central

The rates of type 2 diabetes mellitus, obesity, and cardiovascular disease (CVD) continue to increase at epidemic proportions. It has become clear that these disease states are not independent but are frequently interrelated. By addressing conditions such as obesity, insulin resistance, stress hyperglycemia, impaired glucose tolerance, and diabetes mellitus, with its micro- and macrovascular complications, a specific treatment strategy can be developed. These conditions can be addressed by early identification of patients at high risk for type 2 diabetes, prompt and aggressive treatment of their hyperglycemia, recognition of the pleiotropic and synergistic benefits of certain antidiabetes agents on CVD, and thus, avoiding potential complications including hypoglycemia and weight gain. Incretin-based therapies, which include glucagon-like peptide-1 (GLP-1) receptor agonists and dipeptidyl peptidase-IV (DPP-IV) inhibitors, have the potential to alter the course of type 2 diabetes and associated CVD complications. Advantages of these therapies include glucose-dependent enhancement of insulin secretion, infrequent instances of hypoglycemia, weight loss with GLP-1 receptor agonists, weight maintenance with DPP-IV inhibitors, decreased blood pressure, improvements in dyslipidemia, and potential beneficial effects on CV function. PMID:21437091

Schwartz, Stanley; Kohl, Benjamin A

2010-01-01

313

Chronic pelvic pain syndrome: reduction of medication use after pelvic floor physical therapy with an internal myofascial trigger point wand.  

PubMed

This study documents the voluntary reduction in medication use in patients with refractory chronic pelvic pain syndrome utilizing a protocol of pelvic floor myofascial trigger point release with an FDA approved internal trigger point wand and paradoxical relaxation therapy. Self-referred patients were enrolled in a 6-day training clinic from October, 2008 to May, 2011 and followed the protocol for 6 months. Medication usage and symptom scores on a 1-10 scale (10 = most severe) were collected at baseline, and 1 and 6 months. All changes in medication use were at the patient's discretion. Changes in medication use were assessed by McNemar's test in both complete case and modified intention to treat (mITT) analyses. 374 out of 396 patients met inclusion criteria; 79.7 % were male, median age of 43 years and median symptom duration of 5 years. In the complete case analysis, the percent of patients using medications at baseline was 63.6 %. After 6 months of treatment the percentage was 40.1 %, a 36.9 % reduction (p < 0.001). In the mITT analysis, there was a 22.7 % overall reduction from baseline (p < 0.001). Medication cessation at 6 months was significantly associated with a reduction in total symptoms (p = 0.03). PMID:25708131

Anderson, Rodney U; Harvey, Richard H; Wise, David; Nevin Smith, J; Nathanson, Brian H; Sawyer, Tim

2015-03-01

314

A non-complement-fixing antibody to ?2 glycoprotein I as a novel therapy for antiphospholipid syndrome.  

PubMed

A single-chain fragment variable (scFv) recognizing ?2-glycoprotein 1 (?2GPI) from humans and other species was isolated from a human phage display library and engineered to contain an IgG1 hinge-CH2-CH3 domain. The scFv-Fc directed against ?2GPI domain I-induced thrombosis and fetal loss, thus mimicking the effect of antibodies from patients with antiphospholipid syndrome (APS). Complement is involved in the biological effect of anti-?2GPI scFv-Fc, as demonstrated by its ability to promote in vitro and in vivo complement deposition and the failure to induce vascular thrombosis in C6-deficient rats and fetal loss in C5-depleted mice. A critical role for complement was also supported by the inability of the CH2-deleted scFv-Fc to cause vessel occlusion and pregnancy failure. This antibody prevented the pathological effects of anti-?2GPI antibodies from APS patients and displaced ?2GPI-bound patient antibodies. The CH2-deleted antibody represents an innovative approach potentially useful to treat APS patients refractory to standard therapy. PMID:24642748

Agostinis, Chiara; Durigutto, Paolo; Sblattero, Daniele; Borghi, Maria O; Grossi, Claudia; Guida, Filomena; Bulla, Roberta; Macor, Paolo; Pregnolato, Francesca; Meroni, Pier Luigi; Tedesco, Francesco

2014-05-29

315

In vitro evaluation of mismatched double-stranded RNA (ampligen) for combination therapy in the treatment of acquired immunodeficiency syndrome.  

PubMed

Multiple drug effect analyses with mismatched double-stranded RNA (mismatched dsRNA or Ampligen) as a core drug were performed to identify other agents and mechanisms through which mismatched dsRNA may potentiate effective therapeutic intervention in human immunodeficiency virus (HIV) infection. Antiviral activities were defined by a microtiter infection assay utilizing MT-2 cells as targets and HTLV-III-B produced in H9 cells as a virus source. The scope of agents tested included rIFN-alpha A, rIFN-beta Ser 17, and rIFN-gamma as cytokines; azidothymidine and phosphonoformate (Foscarnet) as inhibitors of reverse transcription; ribavirin as a putative inhibitor of proper HIV mRNA capping; amphotericin B as a lipophile; and castanospermine as a glycoprotein processing (glucosidase I) inhibitor. Separately, each drug demonstrated dose-dependent anti-HIV activity and, when used in combination with mismatched dsRNA, demonstrated synergism. Although mismatched dsRNA was synergistic with all three IFNs for anti-HIV activity in microtiter infection assays, it did not potentiate the transient inhibition of virus production observed for IFN in cultures of H9/HTLV-III-B cells. The results of these studies suggest that the pleiotropic activities of dsRNAs differ from those of IFN and may provide synergism in combination therapy with a wide range of antiviral drugs for the treatment of the acquired immunodeficiency syndrome (AIDS). PMID:2469450

Montefiori, D C; Robinson, W E; Mitchell, W M

1989-04-01

316

Pulsed vs. CW low level light therapy on osteoarticular signs and symptoms in limited scleroderma (CREST syndrome)  

NASA Astrophysics Data System (ADS)

Limited cutaneous systemic sclerosis (lcSSc) was formerly known as CREST syndrome in reference to the associated clinical features: Calcinosis, Raynaud's phenomenon, Esophageal dysfunction, Sclerodactyly, and Telangiectasias. The transforming growth factor beta (TGF-?) has been identified has a major player in the pathogenic process, while low level light therapy (LLLT) has been shown to modulate this cytokine superfamily. This case study was conducted to assess the efficacy of 940nm using microsecond domain pulsing and continuous wave mode (CW) on osteoarticular signs and symptoms associated with lcSSc. The patient was treated two to three times a week for 13 weeks, using a sequential pulsing mode on one elbow, and a CW mode on the other. Efficacy assessments included inflammation, symptoms, pain, and health scales, patient satisfaction, clinical global impression, and adverse effects monitoring. Significant functional and morphologic improvements were observed after LLLT, with best results seen with the pulsing mode. No significant adverse effects were noted. Two mechanisms of action may be at play. The 940nm wavelength provides inside-out heating possibly vasodilating capillaries which in turn increases catabolic processes leading to a reduction of in situ calcinosis. LLLT may also improve symptoms by triggering a cascade of cellular reactions, including the modulation of inflammatory mediators.

Barolet, Daniel

2012-03-01

317

The IDEAL DVT study, individualised duration elastic compression therapy against long-term duration of therapy for the prevention of post-thrombotic syndrome: protocol of a randomised controlled trial  

PubMed Central

Introduction Post-thrombotic syndrome (PTS) is a serious complication of deep vein thrombosis (DVT) of the leg that affects 20–50% of patients. Once a patient experiences PTS there is no treatment that effectively reduces the debilitating complaints. Two randomised controlled trials showed that elastic compression stocking (ECS) therapy after DVT for 24?months can reduce the incidence of PTS by 50%. However, it is unclear whether all patients benefit to the same extent from ECS therapy or what the optimal duration of therapy for individual patients should be. ECS therapy is costly, inconvenient, demanding and sometimes even debilitating. Tailoring therapy to individual needs could save substantial costs. The objective of the IDEAL DVT study, therefore, is to evaluate whether tailoring the duration of ECS therapy on signs and symptoms of the individual patient is a safe and effective method to prevent PTS, compared with standard ECS therapy. Methods and analysis A multicentre, single-blinded, allocation concealed, randomised, non-inferiority trial. A total of 864 consecutive patients with acute objectively documented proximal DVT of the leg are randomised to either standard duration of 24?months or tailored duration of ECS therapy following an initial therapeutic period of 6?months. Signs and symptoms of PTS are recorded at regular clinic visits. Furthermore, quality of life, costs, patient preferences and compliance are measured. The primary outcome is the proportion of patients with PTS at 24?months. Ethics and dissemination Based on current knowledge the standard application of ECS therapy is questioned. The IDEAL DVT study will address the central questions that remain unanswered: Which individual patients benefit from ECS therapy and what is the optimal individual treatment duration? Primary ethics approval was received from the Maastricht University Medical Centre. Results Results of the study will be disseminated via peer-reviewed publications and presentations at scientific conferences. Trial registration number NCT01429714 and NTR 2597. PMID:25190617

ten Cate-Hoek, Arina J; Bouman, Annemieke C; Joore, Manuela A; Prins, Martin; ten Cate, Hugo

2014-01-01

318

Evolution of iron overload in patients with low-risk myelodysplastic syndrome: iron chelation therapy and organ complications.  

PubMed

This study aimed to evaluate the evolution of iron overload, assessed by serum ferritin (SF), in transfusion-dependent lower risk patients with myelodysplastic syndrome (MDS), as well as to describe the occurrence of organ complications, and to analyze its relationship with iron chelation therapy. This observational retrospective study was conducted from March 2010 to March 2011 in 47 Spanish hospitals. A total of 263 patients with lower risk MDS (International Prognostic Scoring System [IPSS] low/intermediate-1 risk or Spanish Prognostic Index [SPI] 0-1 risk), transfusion-dependent, and who had received ?10 packed red blood cells (PRBC) were included. At MDS diagnosis, patients received a mean of 2.8?±?3.9 PRBC/month, and 8.7 % of patients showed SF ?1000 ?g/L. Over the course of the disease, patients received a mean of 83.4?±?83.3 PRBC, and 36.1 % of patients presented SF ?2500 ?g/L. Cardiac, hepatic, endocrine, or arthropathy complications appeared/worsened in 20.2, 11.4, 9.9, and 3.8 % of patients, respectively. According to investigator, iron overload was a main cause of hepatic (70.0 %) and endocrine (26.9 %) complications. A total of 96 (36.5 %) patients received iron chelation therapy for ?6 months, being deferasirox the most frequent first chelation treatment (71.9 %). Chelation-treated patients showed longer overall survival (p?therapy and provide additional evidence on the deleterious effect of iron overload in lower risk MDS patients. PMID:25516455

Remacha, Ángel F; Arrizabalaga, Beatriz; Villegas, Ana; Durán, María Soledad; Hermosín, Lourdes; de Paz, Raquel; Garcia, Marta; Campelo, Maria Diez; Sanz, Guillermo

2015-05-01

319

Constipation-predominant irritable bowel syndrome: A review of current and emerging drug therapies  

PubMed Central

Irritable bowel syndrome (IBS) is a highly prevalent medical condition that adversely affects patient quality of life and constitutes a significant economic burden on healthcare resources. A large proportion of patients suffer from the constipation subtype of IBS (IBS-C), most commonly afflicting older individuals and those with a lower socioeconomic status. Conventional pharmacologic and nonpharmacologic treatment options have limited efficacies and/or significant adverse events, which lead to increased long-term health care expenditures. Failure to effectively treat IBS-C patients over the past decades has largely been due to a poor understanding of disease pathophysiology, lack of a global view of the patient, and an inappropriate selection of patients and treatment endpoints in clinical trials. In recent years, however, more effective and safer drugs have been developed for the treatment of IBS-C. The advancement in the area of pharmacologic treatment is based on new knowledge of the pathophysiologic basis of IBS-C and the development of drugs with increased selectivity within pharmacologic classes with recognized efficacies. This narrative review covers the spectrum of available drugs and their mechanisms of action, as well as the efficacy and safety profiles of each as determined in relevant clinical trials that have investigated treatment options for IBS-C and chronic constipation. A brief summary of laxative-based treatment options is presented, followed by up-to-date assessments for three classes of drugs: prokinetics, prosecretory agents, and bile acid modulators. PMID:25083062

Jadallah, Khaled A; Kullab, Susan M; Sanders, David S

2014-01-01

320

Hedgehog agonist therapy corrects structural and cognitive deficits in a Down syndrome mouse model  

PubMed Central

Down syndrome (DS) is among the most frequent genetic causes of intellectual disability, and ameliorating this deficit is a major goal in support of people with trisomy 21. The Ts65Dn mouse recapitulates some major brain structural and behavioral phenotypes of DS, including reduced size and cellularity of the cerebellum and learning deficits associated with the hippocampus. We show that a single treatment of newborn mice with the sonic hedgehog pathway agonist, SAG1.1 (SAG), results in normal cerebellar morphology in adults. Further, SAG treatment at birth rescued phenotypes associated with hippocampal deficits that occur in untreated adult Ts65Dn mice. This treatment resulted in behavioral improvements and normalized performance in the Morris Water Maze task for learning and memory. SAG treatment also produced physiological effects and partially rescued both NMDA receptor dependent synaptic plasticity and NMDA/AMPA receptor ratio, physiological measures associated with memory. These outcomes confirm an important role for the hedgehog pathway in cerebellar development and raise the possibility for its direct influence in hippocampal function. The positive results from this approach suggest a possible direction for therapeutic intervention to improve cognitive function for this population. PMID:24005160

Das, Ishita; Park, Joo-Min; Shin, Jung H.; Jeon, Soo Kyeong; Lorenzi, Hernan; Linden, David J.; Worley, Paul F.; Reeves, Roger H.

2014-01-01

321

Intradermal Therapy (Mesotherapy) for the Treatment of Acute Pain in Carpal Tunnel Syndrome: A Preliminary Study  

PubMed Central

Background The carpal tunnel syndrome (CTS) is the most common cause of severe hand pain. In this study we treated acute pain in CTS patients by means of local intradermal injections of anti-inflammatory drugs (mesotherapy). Methods In twenty-five patients (forty-five hands), CTS diagnosis was confirmed by clinical and neurophysiological examination prior to mesotherapy. A mixture containing lidocaine 10 mg, ketoprophen lysine-acetylsalycilate 80 mg, xantinol nicotinate 100 mg, cyanocobalamine 1,000 mcg plus injectable water was used. Sites of injection were three parallel lines above the transverse carpal ligament and two v-shaped lines, one at the base of the thenar eminence, and the other at the base of the hypothenar eminence. Results The day after the treatment, all but four patients reported a significant reduction in pain and paresthesias. After 12 months, 17 patients had a complete pain relief, eight patients reported recurrence of pain and sensory symptoms and four out of them underwent surgical treatment. Conclusions With the obvious limits of a small-size open-label study, our results suggest that mesotherapy can temporary relieve pain and paresthesias in most CTS patients and in some cases its effect seems to be long-lasting. Further controlled studies are needed to confirm our preliminary findings and to compare mesotherapy to conventional approaches for the treatment of CTS. PMID:24478901

Conforti, Giorgio; Capone, Loredana

2014-01-01

322

Posterior Reversible Encephalopathy Syndrome (PRES) as a Complication of Immunosuppressive Therapy in Renal Transplantation in Children.  

PubMed

Although kidney transplantation is by far the best method of renal replacement therapy, organ receiver is still not spared of eventual toxic consequences of drugs that are in charge of keeping the transplanted kidney functional. Both calcineurin inhibitors, of which tacrolimus more often, occasionally lead to neurotoxic side effects, mostly mild and reversible and dose-dependent in nature, but they can also be very severe or even fatal. It is very important to be aware of possible neurotoxic effects, to confirm them radiologically, and to prevent or reduce drug effects on nervous system. Sometimes the reduction of dose or substitution with another drug with similar mechanism effect is sufficient to terminate the neurotoxic effects of the drug and still not jeopardize the function of transplanted organ. PMID:25568539

Hodzic, Emir; Brcic, Majda; Atic, Mirza; Halilcevic, Alma; Jasarevic, Amila; Aleckovic-Halilovic, Mirna; Trojak, Davor; Atic, Nedima; Zulic, Snezana; Mehmedovic, Zlatan; Iveljic, Ivana

2014-06-01

323

Posterior Reversible Encephalopathy Syndrome (PRES) as a Complication of Immunosuppressive Therapy in Renal Transplantation in Children  

PubMed Central

Although kidney transplantation is by far the best method of renal replacement therapy, organ receiver is still not spared of eventual toxic consequences of drugs that are in charge of keeping the transplanted kidney functional. Both calcineurin inhibitors, of which tacrolimus more often, occasionally lead to neurotoxic side effects, mostly mild and reversible and dose-dependent in nature, but they can also be very severe or even fatal. It is very important to be aware of possible neurotoxic effects, to confirm them radiologically, and to prevent or reduce drug effects on nervous system. Sometimes the reduction of dose or substitution with another drug with similar mechanism effect is sufficient to terminate the neurotoxic effects of the drug and still not jeopardize the function of transplanted organ. PMID:25568539

Hodzic, Emir; Brcic, Majda; Atic, Mirza; Halilcevic, Alma; Jasarevic, Amila; Aleckovic-Halilovic, Mirna; Trojak, Davor; Atic, Nedima; Zulic, Snezana; Mehmedovic, Zlatan; Iveljic, Ivana

2014-01-01

324

Autonomic modulation and antiarrhythmic therapy in a model of long QT syndrome type 3  

PubMed Central

Aims Clinical observations in patients with long QT syndrome carrying sodium channel mutations (LQT3) suggest that bradycardia caused by parasympathetic stimulation may provoke torsades de pointes (TdP). ?-Adrenoceptor blockers appear less effective in LQT3 than in other forms of the disease. Methods and results We studied effects of autonomic modulation on arrhythmias in vivo and in vitro and quantified sympathetic innervation by autoradiography in heterozygous mice with a knock-in deletion (?KPQ) in the Scn5a gene coding for the cardiac sodium channel and increased late sodium current (LQT3 mice). Cholinergic stimulation by carbachol provoked bigemini and TdP in freely roaming LQT3 mice. No arrhythmias were provoked by physical stress, mental stress, isoproterenol, or atropine. In isolated, beating hearts, carbachol did not prolong action potentials per se, but caused bradycardia and rate-dependent action potential prolongation. The muscarinic inhibitor AFDX116 prevented effects of carbachol on heart rate and arrhythmias. ?-Adrenoceptor stimulation suppressed arrhythmias, shortened rate-corrected action potential duration, increased rate, and minimized difference in late sodium current between genotypes. ?-Adrenoceptor density was reduced in LQT3 hearts. Acute ?-adrenoceptor blockade by esmolol, propranolol or chronic propranolol in vivo did not suppress arrhythmias. Chronic flecainide pre-treatment prevented arrhythmias (all P < 0.05). Conclusion Cholinergic stimulation provokes arrhythmias in this model of LQT3 by triggering bradycardia. ?-Adrenoceptor density is reduced, and ?-adrenoceptor blockade does not prevent arrhythmias. Sodium channel blockade and ?-adrenoceptor stimulation suppress arrhythmias by shortening repolarization and minimizing difference in late sodium current. PMID:20110334

Fabritz, Larissa; Damke, Dierk; Emmerich, Markus; Kaufmann, Susann G.; Theis, Kathrin; Blana, Andreas; Fortmüller, Lisa; Laakmann, Sandra; Hermann, Sven; Aleynichenko, Elena; Steinfurt, Johannes; Volkery, Daniela; Riemann, Burkhard; Kirchhefer, Uwe; Franz, Michael R.; Breithardt, Günter; Carmeliet, Edward; Schäfers, Michael; Maier, Sebastian K.G.; Carmeliet, Peter; Kirchhof, Paulus

2010-01-01

325

Restrictive transfusion practice during extracorporeal membrane oxygenation therapy for severe acute respiratory distress syndrome.  

PubMed

Recommendations concerning the management of hemoglobin levels and hematocrit in patients on extracorporeal membrane oxygenation (ECMO) still advise maintenance of a normal hematocrit. In contrast, current transfusion guidelines for critically ill patients support restrictive transfusion practice. We report on a series of patients receiving venovenous ECMO (vvECMO) for acute respiratory distress syndrome (ARDS) treated according to the restrictive transfusion regimen recommended for critically ill patients. We retrospectively analyzed 18 patients receiving vvECMO due to severe ARDS. Hemoglobin concentrations were kept between 7 and 9?g/dL with a transfusion trigger at 7?g/dL or when physiological transfusion triggers were apparent. We assessed baseline data, hospital mortality, time on ECMO, hemoglobin levels, hematocrit, quantities of packed red blood cells received, and lactate concentrations and compared survivors and nonsurvivors. The overall mortality of all patients on vvECMO was 38.9%. Mean hemoglobin concentration over all patients and ECMO days was 8.30?±?0.51?g/dL, and hematocrit was 0.25?±?0.01, with no difference between survivors and nonsurvivors. Mean numbers of given PRBCs showed a trend towards higher quantities in the group of nonsurvivors, but the difference was not significant (1.97?±?1.47 vs. 0.96?±?0.76 units; P?=?0.07). Mean lactate clearance from the first to the third day was 45.4?±?28.3%, with no significant difference between survivors and nonsurvivors (P?=?0.19). In our cohort of patients treated with ECMO due to severe ARDS, the application of a restrictive transfusion protocol did not result in an increased mortality. Safety and feasibility of the application of a restrictive transfusion protocol in patients on ECMO must further be evaluated in randomized controlled trials. PMID:25349127

Voelker, Maria T; Busch, Thilo; Bercker, Sven; Fichtner, Falk; Kaisers, Udo X; Laudi, Sven

2015-04-01

326

Irritable bowel syndrome: A disease still searching for pathogenesis, diagnosis and therapy  

PubMed Central

Irritable bowel syndrome (IBS) is the most frequently diagnosed functional gastrointestinal disorder in primary and secondary care. It is characterised by abdominal discomfort, pain and changes in bowel habits that can have a serious impact on the patient’s quality of life. The pathophysiology of IBS is not yet completely clear. Genetic, immune, environmental, inflammatory, neurological and psychological factors, in addition to visceral hypersensitivity, can all play an important role, one that most likely involves the complex interactions between the gut and the brain (gut-brain axis). The diagnosis of IBS can only be made on the basis of the symptoms of the Rome III criteria. Because the probability of organic disease in patients fulfilling the IBS criteria is very low, a careful medical history is critical and should pay particular attention to the possible comorbidities. Nevertheless, the severity of the patient’s symptoms or concerns sometimes compels the physician to perform useless and/or expensive diagnostic tests, transforming IBS into a diagnosis of exclusion. The presence of alarming symptoms (fever, weight loss, rectal bleeding, significant changes in blood chemistry), the presence of palpable abdominal masses, any recent onset of symptoms in patient aged over 50 years, the presence of symptoms at night, and a familial history of celiac disease, colorectal cancer and/or inflammatory bowel diseases all warrant investigation. Treatment strategies are based on the nature and severity of the symptoms, the degree of functional impairment of the bowel habits, and the presence of psychosocial disorders. This review examines and discusses the pathophysiological aspects and the diagnostic and therapeutic approaches available for patients with symptoms possibly related to IBS, pointing out controversial issues and the strengths and weaknesses of the current knowledge. PMID:25083055

Bellini, Massimo; Gambaccini, Dario; Stasi, Cristina; Urbano, Maria Teresa; Marchi, Santino; Usai-Satta, Paolo

2014-01-01

327

Skeletal findings in the first 12 months following initiation of glucocorticoid therapy for pediatric nephrotic syndrome  

PubMed Central

Introduction Vertebral fracture (VF) incidence following glucocorticoid (GC) initiation has not been previously reported in pediatric nephrotic syndrome. Methods VF were assessed on radiographs (Genant method); lumbar spine bone mineral density (LS BMD) was evaluated by dual-energy x-ray absorptiometry. Results Sixty-five children were followed to 12 months post-GC initiation (median age: 5.4 years, range 2.3 to 17.9). Three of 54 children with radiographs (6%, 95% CI 2 to 15%) had incident VF at 1 year. The mean LS BMD Z-score was below the healthy average at baseline (mean ± SD ?0.5 ± 1.1 p=0.001) and at 3 months (?0.6 ± 1.1 p<0.001), but not at 6 months (?0.3 ± 1.3, p=0.066) or 12 months (?0.3 ± 1.2, p=0.066). Mixed effect modeling showed a significant increase in LS BMD Z-scores between 3 and 12 months (0.22 SD, 95% CI 0.08 to 0.36, p=0.003). A sub-group (N=16; 25%) had LS BMD Z-scores that were ? ?1.0 at 12 months. In these children, each additional 1000 mg/m2 of GC received in the first 3 months was associated with a decrease in LS BMD Z-score by 0.39 at 12 months (95% CI, ?0.71 to ?0.07; p=0.017). Conclusions The incidence of VF at 1 year was low and LS BMD Z-scores improved by 12 months in the majority. Twenty-five percent of children had LS BMD Z-scores ? ?1.0 at 12 months. In these children, LS BMD Z-scores were inversely associated with early GC exposure, despite similar GC exposure compared to the rest of the cohort. PMID:23948876

Phan, V; Blydt-Hansen, T; Feber, J; Alos, N; Arora, S; Atkinson, S; Bell, L; Clarson, C; Couch, R; Cummings, EA; Filler, G; Grant, RM; Grimmer, J; Hebert, D; Lentle, B; Ma, J; Matzinger, M; Midgley, J; Pinsk, M; Rodd, C; Shenouda, N; Stein, R; Stephure, D; Taback, S; Williams, K; Rauch, F; Siminoski, K; Ward, LM

2014-01-01

328

Cerebral hemodynamics in patients with obstructive sleep apnea syndrome monitored with near-infrared spectroscopy (NIRS) during positive airways pressure (CPAP) therapy: a pilot study  

NASA Astrophysics Data System (ADS)

In obstructive sleep apnea syndrome (OSA) the periodic reduction or cessation of breathing due to narrowing or occlusion of the upper airway during sleep leads to daytime symptoms and increased cardiovascular risk, including stroke. The higher risk of stroke is related to the impairment in cerebral vascular autoregulation. Continuous positive airways pressure (CPAP) therapy at night is the most effective treatment for OSA. However, there is no suitable bedside monitoring method evaluating the treatment efficacy of CPAP therapy, especially to monitor the recovery of cerebral hemodynamics. NIRS is ideally suited for non-invasive monitoring the cerebral hemodynamics during sleep. In this study, we will for first time assess dynamic changes of cerebral hemodynamics during nocturnal CPAP therapy in 3 patients with OSA using NIRS. We found periodic oscillations in HbO2, HHb, tissue oxygenation index (TOI) and blood volume associated with periodic apnea events without CPAP in all OSA patients. These oscillations were gradually attenuated and finally eliminated with the stepwise increments of CPAP pressures. The oscillations were totally eliminated in blood volume earlier than in other hemodynamic parameters. These results suggested that 1) the cerebral hemodynamic oscillations induced by OSA events can effectively be attenuated by CPAP therapy, and 2) blood flow and blood volume recovered first during CPAP therapy, followed by the recovery of oxygen consumption. Our study suggested that NIRS is a useful tool to evaluate the efficacy of CPAP therapy in patients with OSA bedside and in real time.

Zhang, Zhongxing; Schneider, Maja; Laures, Marco; Fritschi, Ursula; Lehner, Isabella; Qi, Ming; Khatami, Ramin

2014-03-01

329

Advances in the development of novel antioxidant therapies as an approach for fetal alcohol syndrome prevention.  

PubMed

Ethanol is the most common human teratogen, and its consumption during pregnancy can produce a wide range of abnormalities in infants known as fetal alcohol spectrum disorder (FASD). The major characteristics of FASD can be divided into: (i) growth retardation, (ii) craniofacial abnormalities, and (iii) central nervous system (CNS) dysfunction. FASD is the most common cause of nongenetic mental retardation in Western countries. Although the underlying molecular mechanisms of ethanol neurotoxicity are not completely determined, the induction of oxidative stress is believed to be one central process linked to the development of the disease. Currently, there is no known effective strategy for prevention (other than alcohol avoidance) or treatment. In the present review we will provide the state of art in the evidence for the use of antioxidants as a potential therapeutic strategy for the treatment using whole-embryo and culture cells models of FASD. We conclude that the imbalance of the intracellular redox state contributes to the pathogenesis observed in FASD models, and we suggest that antioxidant therapy can be considered a new efficient strategy to mitigate the effects of prenatal ethanol exposure. Birth Defects Research (Part A) 103:163-177, 2015. © 2014 Wiley Periodicals, Inc. PMID:25131946

Joya, Xavier; Garcia-Algar, Oscar; Salat-Batlle, Judith; Pujades, Cristina; Vall, Oriol

2015-03-01

330

160 High dose octreotide; a novel therapy for the treatment of drug refractory postural orthostatic tachycardia syndrome in patients with joint hypermobility syndrome  

Microsoft Academic Search

IntroductionPostural orthostatic tachycardia syndrome (POTS) is defined as symptomatic orthostatic intolerance with an increase in heart rate of 30 beats per minute within 10 min of head up tilt (HUT). This dysautonomia causes wide-ranging symptoms including palpitations, presyncope, chronic fatigue, headache and cognitive difficulties. When POTS occurs in patients with pre-existing Joint Hypermobility Syndrome (JHS), symptoms begin approximately a decade

A E French; C Shepherd; A Horne; C Parker; J Tagney; J Pitts-Crick; T Johnson; G Thomas

2011-01-01

331

Impact of enzyme replacement therapy and hematopoietic stem cell transplantation in patients with Morquio A syndrome  

PubMed Central

Patients with mucopolysaccharidosis IVA (MPS IVA) can present with systemic skeletal dysplasia, leading to a need for multiple orthopedic surgical procedures, and often become wheelchair bound in their teenage years. Studies on patients with MPS IVA treated by enzyme replacement therapy (ERT) showed a sharp reduction on urinary keratan sulfate, but only modest improvement based on a 6-minute walk test and no significant improvement on a 3-minute climb-up test and lung function test compared with the placebo group, at least in the short-term. Surgical remnants from ERT-treated patients did not show reduction of storage materials in chondrocytes. The impact of ERT on bone lesions in patients with MPS IVA remains limited. ERT seems to be enhanced in a mouse model of MPS IVA by a novel form of the enzyme tagged with a bone-targeting moiety. The tagged enzyme remained in the circulation much longer than untagged native enzyme and was delivered to and retained in bone. Three-month-old MPS IVA mice treated with 23 weekly infusions of tagged enzyme showed marked clearance of the storage materials in bone, bone marrow, and heart valves. When treatment was initiated at birth, reduction of storage materials in tissues was even greater. These findings indicate that specific targeting of the enzyme to bone at an early stage may improve efficacy of ERT for MPS IVA. Recombinant N-acetylgalactosamine-6-sulfate sulfatase (GALNS) in Escherichia coli BL21 (DE3) (erGALNS) and in the methylotrophic yeast Pichia pastoris (prGALNS) has been produced as an alternative to the conventional production in Chinese hamster ovary cells. Recombinant GALNS produced in microorganisms may help to reduce the high cost of ERT and the introduction of modifications to enhance targeting. Although only a limited number of patients with MPS IVA have been treated with hematopoietic stem cell transplantation (HSCT), beneficial effects have been reported. A wheelchair-bound patient with a severe form of MPS IVA was treated with HSCT at 15 years of age and followed up for 10 years. Radiographs showed that the figures of major and minor trochanter appeared. Loud snoring and apnea disappeared. In all, 1 year after bone marrow transplantation, bone mineral density at L2–L4 was increased from 0.372 g/cm2 to 0.548 g/cm2 and was maintained at a level of 0.48±0.054 for the following 9 years. Pulmonary vital capacity increased approximately 20% from a baseline of 1.08 L to around 1.31 L over the first 2 years and was maintained thereafter. Activity of daily living was improved similar to the normal control group. After bilateral osteotomies, a patient can walk over 400 m using hip–knee–ankle–foot orthoses. This long-term observation of a patient shows that this treatment can produce clinical improvements although bone deformity remained unchanged. In conclusion, ERT is a therapeutic option for MPS IVA patients, and there are some indications that HSCT may be an alternative to treat this disease. However, as neither seems to be a curative therapy, at least for the skeletal dysplasia in MPS IVA patients, new approaches are investigated to enhance efficacy and reduce costs to benefit MPS IVA patients.

Tomatsu, Shunji; Sawamoto, Kazuki; Alméciga-Díaz, Carlos J; Shimada, Tsutomu; Bober, Michael B; Chinen, Yasutsugu; Yabe, Hiromasa; Montańo, Adriana M; Giugliani, Roberto; Kubaski, Francyne; Yasuda, Eriko; Rodríguez-López, Alexander; Espejo-Mojica, Angela J; Sánchez, Oscar F; Mason, Robert W; Barrera, Luis A; Mackenzie, William G; Orii, Tadao

2015-01-01

332

Paraneoplastic Syndromes  

PubMed Central

Neoplasms can produce a variety of remote effects on the host; these are referred to as paraneoplastic syndromes. The syndromes may affect any of the systems of the body, may precede or follow the diagnosis of the underlying neoplasm, and may or may not parallel the course of the neoplasm in severity. The diagnosis of and therapy for these syndromes can be challenging to a physician, but successful therapy may bring about worthwhile relief for the patient. In addition, the syndromes and the substances that cause them are sometimes useful in diagnosing and in following the course of certain neoplasms. Perhaps of greater importance, study of these remote effects of neoplasia may shed light on the nature of the neoplastic process itself. PMID:6990627

Stolinsky, David C.

1980-01-01

333

Chinese Herbal Decoction Based on Syndrome Differentiation as Maintenance Therapy in Patients with Extensive-Stage Small-Cell Lung Cancer: An Exploratory and Small Prospective Cohort Study  

PubMed Central

Objective. To investigate the treatment effect and treatment length of Chinese herbal decoction (CHD) as maintenance therapy on patients with extensive-stage small-cell lung cancer (ES-SCLC) and to reflect the real syndrome differentiation (Bian Zheng) practices of traditional Chinese medicine (TCM). Patients and Methods. Different CHDs were prescribed for each patient based on syndrome differentiation. The length of CHD treatment was divided into two phases for analyzing progression-free survival (PFS) and postprogression survival (PPS). Results. Three hundred and fifty-seven CHDs were prescribed based on syndrome differentiation during the study period. Median PFS was significantly longer in patients who received CHD >3 months than patients who received CHD ?3 months in the first phase (8.7 months versus 4.5 months; hazard ratio (HR), 0.52; 95% confidence interval (CI), 0.41–0.99; P = 0.0009). Median PPS was significantly longer in patients who received CHD >7 months than patients who received CHD ?7 months in the second phase (11.7 months versus 5.1 months; HR, 2.32; 95% CI, 1.90–2.74; P = 0.002). Conclusion. CHD could improve PFS and PPS, which are closely related to treatment time and deepness of response of first-line therapy. In addition, CHD could improve body function and keep patients in a relatively stable state. PMID:25815038

Liu, Rui; He, Shu lin; Zhao, Yuan chen; Zheng, Hong gang; Li, Cong huang; Bao, Yan ju; Qin, Ying gang; Hou, Wei; Hua, Bao Jin

2015-01-01

334

Education, progressive muscle relaxation therapy, and exercise for the treatment of night eating syndrome. A pilot study.  

PubMed

Night eating syndrome (NES) is a circadian rhythm disorder in which food intake is shifted toward the end of the day, interfering with sleep. According to the biobehavioral model of NES, the disorder is the result of a genetic predisposition that, coupled with stress, leads to enhanced reuptake of serotonin, thereby dysregulating circadian rhythms and decreasing satiety. Using the biobehavioral model as a guide, we developed a brief behavioral intervention using education, relaxation strategies, and exercise to address the core symptoms of NES. In this pilot randomized controlled clinical trial, 44 participants with NES were randomly assigned to an educational group (E; n?=?14), E plus progressive muscle relaxation therapy (PMR; n?=?15); or PMR plus exercise (PMR Plus, n?=?15). Participants received a baseline intervention with 1- and 3-week follow-up sessions. Effectiveness analyses showed that participants in all three groups evidenced significant reductions on measures of NES symptoms (p?

Vander Wal, Jillon S; Maraldo, Toni M; Vercellone, Allison C; Gagne, Danielle A

2015-06-01

335

Efficacy and safety of oral tolvaptan therapy in patients with the syndrome of inappropriate antidiuretic hormone secretion  

PubMed Central

Objective Tolvaptan, an oral antagonist of the vasopressin V2 receptor, has been found to improve hyponatremia in patients with mixed etiologies. This study analyzed a subgroup of patients with the syndrome of inappropriate antidiuretic hormone secretion (SIADH) to evaluate the efficacy and safety of tolvaptan in this group. Design and patients Hyponatremic patients in the SALT-1 and SALT-2 studies with a diagnosis of SIADH were identified based on clinical diagnosis by individual study investigators. Subjects were randomized to receive oral placebo (n=52) or tolvaptan 15 mg daily, with further titration to 30 and 60 mg daily, if necessary, based on the response of serum [Na+] (n=58). Results In patients with SIADH, improvement in serum [Na+] was significantly greater (P<0.0001) with tolvaptan than placebo over the first 4 days of therapy as well as the entire 30-day study, with minimal side effects of increased thirst, dry mouth, and urination. Only 5.9% of tolvaptan-treated patients had overly rapid correction of hyponatremia as defined by current guidelines. After discontinuation of tolvaptan, serum [Na+] declined to values similar to placebo. A significant positive treatment effect favoring tolvaptan on the physical component, and a near-significant trend on the mental component, was found using the SF-12 Health Survey. Tolvaptan was associated with a significantly reduced incidence of fluid restriction. Conclusions Results for the SIADH subgroup were analogous to those of the combined SALT population regarding efficacy and safety but demonstrated a greater improvement in the physical component of the SF-12 Health Survey than in the full mixed etiology SALT patient group. PMID:21317283

Verbalis, Joseph G; Adler, Suzanne; Schrier, Robert W; Berl, Tomas; Zhao, Qiong; Czerwiec, Frank S

2013-01-01

336

Hyperbaric Oxygen Therapy Can Improve Post Concussion Syndrome Years after Mild Traumatic Brain Injury - Randomized Prospective Trial  

PubMed Central

Background Traumatic brain injury (TBI) is the leading cause of death and disability in the US. Approximately 70-90% of the TBI cases are classified as mild, and up to 25% of them will not recover and suffer chronic neurocognitive impairments. The main pathology in these cases involves diffuse brain injuries, which are hard to detect by anatomical imaging yet noticeable in metabolic imaging. The current study tested the effectiveness of Hyperbaric Oxygen Therapy (HBOT) in improving brain function and quality of life in mTBI patients suffering chronic neurocognitive impairments. Methods and Findings The trial population included 56 mTBI patients 1–5 years after injury with prolonged post-concussion syndrome (PCS). The HBOT effect was evaluated by means of prospective, randomized, crossover controlled trial: the patients were randomly assigned to treated or crossover groups. Patients in the treated group were evaluated at baseline and following 40 HBOT sessions; patients in the crossover group were evaluated three times: at baseline, following a 2-month control period of no treatment, and following subsequent 2-months of 40 HBOT sessions. The HBOT protocol included 40 treatment sessions (5 days/week), 60 minutes each, with 100% oxygen at 1.5 ATA. “Mindstreams” was used for cognitive evaluations, quality of life (QOL) was evaluated by the EQ-5D, and changes in brain activity were assessed by SPECT imaging. Significant improvements were demonstrated in cognitive function and QOL in both groups following HBOT but no significant improvement was observed following the control period. SPECT imaging revealed elevated brain activity in good agreement with the cognitive improvements. Conclusions HBOT can induce neuroplasticity leading to repair of chronically impaired brain functions and improved quality of life in mTBI patients with prolonged PCS at late chronic stage. Trial Registration ClinicalTrials.gov NCT00715052 PMID:24260334

Fishlev, Gregori; Bechor, Yair; Volkov, Olga; Bergan, Jacob; Friedman, Mony; Hoofien, Dan; Shlamkovitch, Nathan; Ben-Jacob, Eshel; Efrati, Shai

2013-01-01

337

Growth Hormone Research Society Workshop Summary: Consensus Guidelines for Recombinant Human Growth Hormone Therapy in Prader-Willi Syndrome  

PubMed Central

Context: Recombinant human GH (rhGH) therapy in Prader-Willi syndrome (PWS) has been used by the medical community and advocated by parental support groups since its approval in the United States in 2000 and in Europe in 2001. Its use in PWS represents a unique therapeutic challenge that includes treating individuals with cognitive disability, varied therapeutic goals that are not focused exclusively on increased height, and concerns about potential life-threatening adverse events. Objective: The aim of the study was to formulate recommendations for the use of rhGH in children and adult patients with PWS. Evidence: We performed a systematic review of the clinical evidence in the pediatric population, including randomized controlled trials, comparative observational studies, and long-term studies (>3.5 y). Adult studies included randomized controlled trials of rhGH treatment for ? 6 months and uncontrolled trials. Safety data were obtained from case reports, clinical trials, and pharmaceutical registries. Methodology: Forty-three international experts and stakeholders followed clinical practice guideline development recommendations outlined by the AGREE Collaboration (www.agreetrust.org). Evidence was synthesized and graded using a comprehensive multicriteria methodology (EVIDEM) (http://bit.ly.PWGHIN). Conclusions: Following a multidisciplinary evaluation, preferably by experts, rhGH treatment should be considered for patients with genetically confirmed PWS in conjunction with dietary, environmental, and lifestyle interventions. Cognitive impairment should not be a barrier to treatment, and informed consent/assent should include benefit/risk information. Exclusion criteria should include severe obesity, uncontrolled diabetes mellitus, untreated severe obstructive sleep apnea, active cancer, or psychosis. Clinical outcome priorities should vary depending upon age and the presence of physical, mental, and social disability, and treatment should be continued for as long as demonstrated benefits outweigh the risks. PMID:23543664

Tony, Michčle; Höybye, Charlotte; Allen, David B.; Tauber, Maďthé; Christiansen, Jens Sandahl; Ambler, Geoffrey R.; Battista, Renaldo; Beauloye, Véronique; Berall, Glenn; Biller, Beverly M. K.; Butler, Merlin G.; Cassidy, Suzanne B.; Chihara, Kazuo; Cohen, Pinchas; Craig, Maria; Farholt, Stense; Goetghebeur, Mireille; Goldstone, Anthony P.; Greggi, Tiziana; Grugni, Graziano; Hokken-Koelega, Anita C.; Johannsson, Gudmundur; Johnson, Keegan; Kemper, Alex; Kopchick, John J.; Malozowski, Saul; Miller, Jennifer; Mogul, Harriette R.; Muscatelli, Françoise; Nergĺrdh, Ricard; Nicholls, Robert D.; Radovick, Sally; Rosenthal, M. Sara; Sipilä, Ilkka; Tarride, Jean-Eric; Vogels, Annick; Waters, Michael J.

2013-01-01

338

Acute coronary syndrome (Takotsubo cardiomyopathy) following electroconvulsive therapy in the absence of significant coronary artery disease: case report and review of the literature.  

PubMed

We report a case of myocardial infarction occurring in a 45-year-old woman in the absence of coronary artery disease during a course of electroconvulsive therapy (ECT) for major depression. After the third ECT session, the patient reported substernal chest pain, and although the electrocardiogram was normal, cardiac enzymes were found to be elevated. Cardiac workup to determine etiology during hospital stay showed no evidence of coronary artery disease on catheterization. Cardiac echocardiograph and computed tomography findings were consistent with a diagnosis of Takotsubo cardiomyopathy. This cardiac syndrome is a stress-induced syndrome occurring in the absence of coronary artery stenosis and associated with surging of catecholamines. It is diagnosed on the basis of the shape of the apical ballooning observed on cardiac imaging, which resembles a "takotsubo" or octopus fishing pot in Japan, where this syndrome was first recognized. Coincidentally, after termination of her ECT course, this patient was also diagnosed with Loeys-Dietz syndrome, an inherited connective tissue disorder that can predispose sufferers to aortic dissection. This case illustrates the importance of careful workup of chest pain in the setting of ECT, even if electrocardiogram findings seem unremarkable initially. PMID:18955900

O'Reardon, John P; Lott, Jasonc P; Akhtar, Umair W; Cristancho, Pilar; Weiss, David; Jones, Niya

2008-12-01

339

Olmsted Syndrome  

PubMed Central

Olmsted syndrome is a rare congenital, sharply circumscribed transgredient palmoplantar keratoderma. It was first described by Olmsted in 1927. The diagnosis of this rare disease depends on clinical features like symmetrical involvement of keratoderma of the palms and soles and the symmetrical hyperkeratotic plaques around the body orifices. It starts in the neonatal period or in childhood. The disease has a slow but progressive and extremely disabling course. Treatment of Olmsted syndrome is often based on topical therapy with retinoic acid, corticosteroid, emollients, and keratolytics. The present paper describes a case of Olmsted syndrome and its treatment. PMID:23320205

Elise Tonoli, Renata; De Villa, Damię; Hübner Frainer, Renata; Pizzarro Meneghello, Luana; Ricachnevsky, Nelson; de Quadros, Maurício

2012-01-01

340

Effects of immunosuppressive therapy in a patient with aplastic anemia-paroxysmal nocturnal hemoglobinuria (AA-PNH) syndrome during ongoing eculizumab treatment.  

PubMed

A 65-year-old woman experienced a hemolytic attack triggered by sepsis. She presented with markedly increased CD55(-) CD59(-) erythrocytes and the signs of bone marrow failure, which led to a diagnosis of aplastic anemia-paroxysmal nocturnal hemoglobinuria (AA-PNH) syndrome. There was a possibility of increasing hemolysis, as large PNH clones remained after immunosuppressive therapy (IST). Accordingly, eculizumab was first used to control the hemolytic attack followed by IST with antithymocyte globulin and cyclosporine A. The patient was successfully weaned from blood transfusions and has been followed up without any recurrence of hemolytic attacks. PMID:24429452

Asano, Jin; Ueda, Ryosuke; Tanaka, Yasuhiro; Shinzato, Isaku; Takafuta, Toshiro

2014-01-01

341

Therapeutic options for Middle East respiratory syndrome coronavirus (MERS-CoV)--possible lessons from a systematic review of SARS-CoV therapy.  

PubMed

The Middle East Respiratory Syndrome coronavirus (MERS-CoV) has been detected in a number of countries in the Middle East and Europe with an apparently high mortality rate. It is phylogenetically related to the SARS coronavirus and has also been associated with severe respiratory illness as well as nosocomial transmission in healthcare settings. Current international recommendations do not support any specific therapies; however, there are a number of agents, which were used during the SARS epidemic of 2003. It is possible that these might be active against the related MERS coronavirus. We have reviewed the literature on the safety and efficacy of therapies used in patients with SARS with a view to their potential use in patients with MERS-CoV infections. PMID:23993766

Momattin, Hisham; Mohammed, Khurram; Zumla, Alimuddin; Memish, Ziad A; Al-Tawfiq, Jaffar A

2013-10-01

342

Cost-utility analysis of genotype-guided antiplatelet therapy in patients with moderate-to-high risk acute coronary syndrome and planned percutaneous coronary intervention  

PubMed Central

Background Prasugrel is recommended over clopidogrel in poor/intermediate CYP2C19 metabolizers with acute coronary syndrome (ACS) and planned percutaneous coronary intervention (PCI), reducing the risk of ischemic events. CYP2C19 genetic testing can guide antiplatelet therapy in ACS patients. Objective The purpose of this study was to evaluate the cost-utility of genotype-guided treatment, compared with prasugrel or generic clopidogrel treatment without genotyping, from the US healthcare provider’s perspective. Methods A decision model was developed to project lifetime economic and humanistic burden associated with clinical outcomes (myocardial infarction [MI], stroke and major bleeding) for the three strategies in patients with ACS. Probabilities, costs and age-adjusted quality of life were identified through systematic literature review. Incremental cost-utility ratios (ICURs) were calculated for the treatment strategies, with quality-adjusted life years (QALYs) as the primary effectiveness outcome. Relative risk of developing myocardial infarction and stroke between patients with and without variant CYP2C19 when receiving clopidogrel were estimated to be 1.34 and 3.66, respectively. One-way and probabilistic sensitivity analyses were performed. Results Clopidogrel cost USD19,147 and provided 10.03 QALYs versus prasugrel (USD21,425, 10.04 QALYs) and genotype-guided therapy (USD19,231, 10.05 QALYs). The ICUR of genotype-guided therapy compared with clopidogrel was USD4,200. Genotype-guided therapy provided more QALYs at lower costs compared with prasugrel. Results were sensitive to the cost of clopidogrel and relative risk of myocardial infarction and stroke between CYP2C19 variant vs. non-variant. Net monetary benefit curves showed that genotype-guided therapy had at least 70% likelihood of being the most cost-effective alternative at a willingness-to-pay of USD100,000/QALY. In comparison with clopidogrel, prasugrel therapy was more cost-effective with <21% certainty at willingness-to-pay of >USD170,000/QALY. Conclusions Our modeling analyses suggest that genotype-guided therapy is a cost-effective strategy in patients with acute coronary syndrome undergoing planned percutaneous coronary intervention. PMID:25243032

Patel, Vardhaman; Lin, Fang-Ju; Ojo, Olaitan; Rao, Sapna; Yu, Shengsheng; Zhan, Lin; Touchette, Daniel R.

2014-01-01

343

Protocol for the PACE trial: A randomised controlled trial of adaptive pacing, cognitive behaviour therapy, and graded exercise as supplements to standardised specialist medical care versus standardised specialist medical care alone for patients with the chronic fatigue syndrome\\/myalgic encephalomyelitis or encephalopathy  

Microsoft Academic Search

BACKGROUND: Chronic fatigue syndrome (CFS, also called myalgic encephalomyelitis\\/encephalopathy or ME) is a debilitating condition with no known cause or cure. Improvement may occur with medical care and additional therapies of pacing, cognitive behavioural therapy and graded exercise therapy. The latter two therapies have been found to be efficacious in small trials, but patient organisations' surveys have reported adverse effects.

Peter D White; Michael C Sharpe; Trudie Chalder; Julia C DeCesare; Rebecca Walwyn

2007-01-01

344

Statin therapy in patients with acute coronary syndrome: low-density lipoprotein cholesterol goal attainment and effect of statin potency  

PubMed Central

Background Elevated low-density lipoprotein cholesterol (LDL-C) is associated with an increased risk of coronary artery disease. Current guidelines recommend an LDL-C target of <70 mg/dL (<1.8 mmol/L) for acute coronary syndrome (ACS) patients, and the first-line treatment to lower lipids is statin therapy. Despite current guidelines and the efficacious lipid-lowering agents available, about half of patients at very high risk, including ACS patients, fail to achieve their LDL-C goal. This study assessed LDL-C goal attainment according to use of high and low potency statins in routine practice in Thailand. Methods A retrospective cohort study was performed by retrieving data from medical records and the electronic hospital database for a tertiary care hospital in Thailand between 2009 and 2011. Included were ACS patients treated with statins at baseline and with follow-up of LDL-C levels. Patients were divided into high or low potency statin users, and the proportion reaching the LDL-C goal of <70 mg/dL was determined. A Cox proportional hazard model was applied to determine the relationship between statin potency and LDL-C goal attainment. Propensity score adjustment was used to control for confounding by indication. Results Of 396 ACS patients (60% males, mean age 64.3±11.6 years), 229 (58%) were treated with high potency statins and 167 (42%) with low potency statins. A quarter reached their target LDL-C goal (25% for patients on high potency statins and 23% on low potency statins). High potency statins were not associated with increased LDL-C goal attainment (adjusted hazards ratio 1.22, 95% confidence interval 0.79–1.88; P=0.363). Conclusion There was no significant effect of high potency statins on LDL-C goal attainment. Moreover, this study showed low LDL-C goal attainment for patients on either low or high potency statins. The reasons for the low LDL-C goal attainment rate warrants further investigation. PMID:25670902

Chinwong, Dujrudee; Patumanond, Jayanton; Chinwong, Surarong; Siriwattana, Khanchai; Gunaparn, Siriluck; Hall, John Joseph; Phrommintikul, Arintaya

2015-01-01

345

Humoral and cellular immune parameters before and during immunosuppressive therapy of a patient with stiff-man syndrome and insulin dependent diabetes mellitus  

PubMed Central

OBJECTIVES—Humoral and cellular immune reactivity are reported for two neuroendocrine autoantigens—glutamic acid decarboxylase (GAD) and the protein tyrosine phosphatase IA-2—in a patient with the autoimmune type of stiff-man syndrome and insulin dependent diabetes (IDDM).?METHODS—Antibodies and T cell proliferation against GAD and IA-2 and cytokine release of antigen stimulated T cells (IFN-?) were determined before and several times during immunosuppressive therapy with prednisolone.?RESULTS—Raised GAD antibodies against full length GAD65 or chimeric constructs were detected before therapy and they remained at a high concentration despite a marked clinical improvement during cortisone treatment. Antibodies to IA-2 were undetectable, but weak T cell responses to both GAD and IA-2 were seen before therapy and once on reduction of high cortisone dosages when the patient showed signs of clinical deterioration. Cytokine profiles showed increased IFN-? production after stimulation with GAD or IA-2 suggesting increased activation of TH1 cells.?CONCLUSION—Immunosuppressive therapy —even with extremely high doses of 500 mg a day—does not lead to the reduction of antibody concentrations in the periphery nor to a switch in epitope recognition of such antibodies despite clinical improvement. The amount of T cell reactivity to various antigens, however, may be a useful marker to monitor the effectiveness of immunotherapy.?? PMID:9703171

Hummel, M; Durinovic-Bello, I; Bonifacio, E; Lampasona, V; Endl, J; Fessele, S; Bergh, F; Trenkwalder, C; Standl, E; Ziegler, A

1998-01-01

346

Using a digital game for training desirable behavior in cognitive-behavioral therapy of burnout syndrome: a controlled study.  

PubMed

Burnout is a globally increasing illness, and as a result, many forms of burnout therapy have arisen. The use of digital games can be psychotherapeutically effective because they can transform exercises that are by themselves unattractive into intrinsically motivated action. This pilot study aims to test whether a specially designed game contributes to patients learning desired behavior and achieving other specific therapeutic goals in an online cognitive-behavioral therapy (CBT)-based burnout treatment context. In total, 101 participants took part in the experiment, under four conditions: (a) Game+Therapy, (b) Therapy Only, (c) Game Only, and (d) No Game+No Therapy. Pre- and postmeasures were taken online. Results showed that the two therapy conditions (Game+Therapy and Therapy Only) showed a greater decrease in complaints and disengagement, and a stronger increase in coping skills than the nontherapy conditions (Game Only and No Game+No Therapy). As expected, the Game+Therapy condition outperformed the Therapy Only condition on combined improvement measures of burnout symptoms. However, analyses of individual measures showed no effects. It can be cautiously concluded that the therapeutic digital game may be a useful tool when embedded in a therapeutic burnout treatment program and is probably more efficient than CBT, as it is used in current practice. PMID:25684611

Zielhorst, Thomas; van den Brule, Daphne; Visch, Valentijn; Melles, Marijke; van Tienhoven, Sam; Sinkbaek, Helle; Schrieken, Bart; Tan, Eduard S-H; Lange, Alfred

2015-02-01

347

Quantifying the Evidence for the Risk of Metabolic Syndrome and Its Components following Androgen Deprivation Therapy for Prostate Cancer: A Meta-Analysis  

PubMed Central

Background No meta-analysis is yet available for the risk of metabolic syndrome (MetS) following androgen deprivation therapy (ADT) for men with prostate cancer. To summarize the evidence for the link between ADT and MetS or its components quantitatively with a meta-analysis including all studies published to date. Methods PubMed and Embase were searched using predefined inclusion criteria to perform meta-analyses on the association between metabolic syndrome, hyperglycemia, diabetes, hypertension, dyslipidemia or obesity and androgen deprivation therapy in patients with prostate cancer. Random effects methods were used to estimate pooled relative risks (RRs) and 95% confidence intervals (CI). Results A total of nine studies was included. There was a positive association between ADT and risk of MetS (RR: 1.75 (95% CI: 1.27–2.41)). Diabetes was the only MetS component present in more than 3 studies, and also showed an increased risk following ADT (RR: 1.36 (95% CI: 1.17–1.58)). Conclusion This is the first quantitative summary addressing the potential risk of MetS following ADT in men with PCa. The positive RRs indicate that there is a need to further elucidate how type and duration of ADT affect these increased risks of MetS and diabetes as the number of men with PCa treated with ADT is increasing. PMID:25794005

Bosco, Cecilia; Crawley, Danielle; Adolfsson, Jan; Rudman, Sarah; Van Hemelrijck, Mieke

2015-01-01

348

Effect of Combining Therapy with Traditional Chinese Medicine-Based Psychotherapy and Herbal Medicines in Women with Menopausal Syndrome: A Randomized Controlled Clinical Trial  

PubMed Central

This multicenter, randomized, controlled clinical study was designed to address the effectiveness of combined traditional-Chinese-medicine- (TCM-) based psychotherapy and Chinese herbal medicine (CHM) in the treatment of menopausal syndrome. Altogether 424 eligible women diagnosed as menopausal syndrome and categorized as Kidney-Yin/Kidney-Yang deficiency pattern in TCM were randomly assigned into 4 groups and accepted TCM-based psychotherapy (PSY), CHM, PSY + CHM, or placebo therapies, respectively, for 12 weeks, and another 12 weeks were taken as the followup. Kupperman Index (KI) and the Menopause-Specific Quality of Life (MENQOL) with its four subscales (vasomotor, physical, psychosocial, and sexual) were employed for efficacy assessment. Results showed that 400 participants completed 12-week treatment, of which 380 finished the record of KI and MENQOF at week 24. The average adjusted number of KI score decreased between baseline and 12 weeks in all groups. Statistically significant differences were detected in the average adjusted change between the PSY + CHM group and placebo at overall time points (P < 0.05). No severe adverse events occurred in each group and no significant differences were indicated between any of the three groups and placebo in adverse event proportion. We concluded that TCM psychotherapy combined with CHM has a favorable outcome in treating menopausal syndrome. PMID:23304198

Yang, Hongyan; Yang, Jing; Wen, Zehuai; Zha, Qinglin; Nie, Guangning; Huang, Xuchun; Zhang, Chunlin; Lu, Aiping; Jiang, Miao; Wang, Xiaoyun

2012-01-01

349

Vaccine Therapy Plus Immune Adjuvant in Treating Patients With Chronic Myeloid Leukemia, Acute Myeloid Leukemia, or Myelodysplastic Syndrome  

ClinicalTrials.gov

Accelerated Phase Chronic Myelogenous Leukemia; Adult Acute Myeloid Leukemia in Remission; Chronic Phase Chronic Myelogenous Leukemia; Previously Treated Myelodysplastic Syndromes; Refractory Anemia With Excess Blasts; Refractory Anemia With Excess Blasts in Transformation; Relapsing Chronic Myelogenous Leukemia

2013-01-04

350

Treatment Options for Myelodysplastic Syndromes  

MedlinePLUS

... myelodysplastic syndromes includes supportive care, drug therapy, and stem cell transplantation. Patients with a myelodysplastic syndrome who have ... in patients with acute myeloid leukemia. Chemotherapy with stem cell transplant Stem cell transplant is a method of ...

351

Iron chelation therapy in the myelodysplastic syndromes and aplastic anemia: a review of experience in South Korea  

Microsoft Academic Search

Emerging clinical data indicate that transfusion-dependent patients with bone marrow-failure syndromes (BMFS) are at risk\\u000a of the consequences of iron overload, including progressive damage to hepatic, endocrine, and cardiac organs. Despite the\\u000a availability of deferoxamine (DFO) in Korea since 1998, data from patients with myelodysplastic syndromes, aplastic anemia,\\u000a and other BMFS show significant iron overload and damage to the heart

Jong Wook Lee

2008-01-01

352

Fluvastatin in the therapy of acute coronary syndrome: Rationale and design of a multicenter, randomized, double-blind, placebo-controlled trial (The FACS Trial)[ISRCTN81331696].  

PubMed

BACKGROUND: Activation of inflammatory pathways plays an important contributory role in coronary plaque instability and subsequent rupture, which can lead to the development of acute coronary syndrome (ACS). Elevated levels of serum inflammatory markers such as C-reactive protein (CRP) represent independent risk factors for further cardiovascular events. Recent evidence indicates that in addition to lowering cholesterol levels, statins also decrease levels of inflammatory markers. Previous controlled clinical trials reporting the positive effects of statins in participants with ACS were designed for very early secondary prevention. To our knowledge, no controlled trials have evaluated the potential benefits of statin therapy, beginning immediately at the time of hospital admission. A previous pilot study performed by our group focused on early initiation of cerivastatin therapy. We demonstrated a highly significant reduction in levels of inflammatory markers (CRP and interleukin-6). Based on these preliminary findings, we are conducting a clinical trial to evaluate the efficacy of another statin, fluvastatin, as an early intervention in patients with ACS. METHODS: The FACS-trial (Fluvastatin in the therapy of Acute Coronary Syndrome) is a multicenter, randomized, double-blind, placebo-controlled study evaluating the effects of fluvastatin therapy initiated at the time of hospital admission. The study will enroll 1,000 participants admitted to hospital for ACS (both with and without ST elevation). The primary endpoint for the study is the influence of fluvastatin therapy on levels of inflammatory markers (CRP and interleukin-6) and on pregnancy associated plasma protein A (PAPP-A). A combined secondary endpoint is 30-day and one-year occurrence of death, nonfatal myocardial infarction, recurrent symptomatic ischemia, urgent revascularization, and cardiac arrest. CONCLUSION: The primary objective of the FACS trial is to demonstrate that statin therapy, when started immediately after hospital admission for ACS, results in reduction of inflammation and improvement of prognosis. This study may contribute to new knowledge regarding therapeutic strategies for patients suffering from ACS and may offer additional clinical indications for the use of statins. PMID:15790413

Ostadal, Petr; Alan, David; Hajek, Petr; Vejvoda, Jiri; Mates, Martin; Blasko, Peter; Veselka, Josef; Kvapil, Milan; Kettner, Jiri; Wiendl, Martin; Aschermann, Ondrej; Slaby, Josef; Nemecek, Eduard; Holm, Frantisek; Rac, Marek; Macek, Milan; Cepova, Jana

2005-03-24

353

A Survey of Radiation-Induced Bronchiolitis Obliterans Organizing Pneumonia Syndrome After Breast-Conserving Therapy in Japan  

SciTech Connect

Purpose: We observed a rare and unique occurrence of radiation-induced pulmonary injury outside the tangential field for early breast cancer treatment. The findings appeared to be idiopathic and were called radiation-induced bronchiolitis obliterans organizing pneumonia (BOOP) syndrome. We surveyed major hospitals in Japan to review their findings of radiation-induced BOOP, in particular the clinical and pictorial characteristics of the entity. Methods and Materials: We reviewed surveys completed and returned by 20 institutions. The survey responses were based on a total of 37 cases of BOOP syndrome. We also reviewed X-ray and computed tomography scans provided by these institutions. We discussed the information derived from the questionnaire and analyzed patients' characteristics, methods used in the treatment of BOOP syndrome, and prognosis. Results: The incidence of the radiation-induced BOOP syndrome was about 1.8% (37 of 2,056). We did not find a relationship between the characteristics of patients and the occurrence of radiation-induced BOOP syndrome. The pulmonary findings were classified into four patterns on chest computed tomography scans. Progression of the pulmonary lesions observed on chest X-ray was classified into three patterns. Pneumonitis appeared within 6 months after radiotherapy was completed and disappeared within 6-12 months after its onset. At 5-year follow-up, 2 patients had died, 1 of breast cancer and the other of interstitial pneumonitis, which seemed to be idiopathic and unrelated to the radiation-induced BOOP syndrome. Conclusions: Although the incidence of BOOP syndrome and its associated prognosis are not significant, the patients' clinical condition must be carefully followed.

Ogo, Etsuyo [Department of Radiology, Kurume University School of Medicine, Kurume (Japan)], E-mail: etsuogo@med.kurume-u.ac.jp; Komaki, Ritsuko [Department of Radiation Oncology, The University of Texas, M.D. Anderson Cancer Center, Houston, TX (United States); Fujimoto, Kiminori; Uchida, Masafumi; Abe, Toshi [Department of Radiology, Kurume University School of Medicine, Kurume (Japan); Nakamura, Katsumasa [Department of Radiology, Faculty of Medicine, Fukuoka University, Fukuoka (Japan); Mitsumori, Michihide [Department of Radiology, Faculty of Medicine, Kyoto University, Kyoto (Japan); Sekiguchi, Kenji [Department of Radiation Oncology, St. Luke's International Hospital, Tokyo (Japan); Kaneyasu, Yuko [Department of Radiology, Faculty of Medicine, Hiroshima University, Hiroshima (Japan); Hayabuchi, Naofumi [Department of Radiology, Kurume University School of Medicine, Kurume (Japan)

2008-05-01

354

Long-term effect of extracorporeal shock wave therapy on the treatment of chronic pelvic pain syndrome due to non bacterial prostatitis  

PubMed Central

Background: There is limited evidence about the chronic pelvic pain syndrome (CPPS) treatment by shockwave therapy, and the aim of this study was to evaluate the effect of extracorporeal shockwave therapy on CPPS due to non bacterial prostatitis in a long-term period. Materials and Methods: In a follow-up survey, 40 patients with CPPS (that were randomly distributed into the treatment or sham groups were evaluated at 16, 20, and 24 weeks. In the treatment group, patients were treated by extracorporeal shock wave therapy (ESWT) once a week for 4 weeks by a protocol of 3000 impulses, 0.25 mJ/m2 and 3 Hz of frequency. 0.05 mJ/m2 were added in each week. In the sham group, the same protocol was applied, but with the probe being turned off. The follow-up assessments were done by visual analog scale for pain and National Institutes of Health-developed Chronic Prostatitis Symptom Index (NIH-CPSI). Data were compared using independent t-test or analysis of variences. Results: Three patients did not complete the study protocol, 37 patients were evlauated (19 patients in treatment and 18 patients in the sham group). At week 24, the mean of pain score, urinary score, quality-of-life and NIH-CPSI score between two groups were not statistically different. Conclusion: Although, ESWT therapy as a safe and effective therapy in CPPS in short-term follow-up has been established, its long-term efficacy was not supported by this study. PMID:25097599

Moayednia, Amir; Haghdani, Saeid; Khosrawi, Saeid; Yousefi, Elham; Vahdatpour, Babak

2014-01-01

355

Therapy-related patterns of cytogenetic abnormalities in acute myeloid leukemia and myelodysplastic syndrome post polycythemia vera: single center experience and review of literature.  

PubMed

A minor fraction of patients with polycythemia vera (PV) develop a terminal acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS). Analysis of the cytogenetic abnormalities during AML or MDS may help in understanding if this development is part of the natural course of the disease or induced by myelosuppressive therapy. Thirty-six cases with AML or MDS post PV, collected in a single Swedish institution during a 33-year period, are described with special regard to time to development of AML or MDS, therapy given during active PV, and cytogenetic findings during AML or MDS. A further 118 cases of AML or MDS post PV, in whom type of therapy during active PV and cytogenetic findings during AML or MDS were reported, were collected from the literature. AML or MDS developed in our own series after 1-30 years with a fairly constant rate (two cases per year). The most frequent cytogenetic abnormalities were +1q, -5, 5q-, -7, 7q-, +8, +9, 11q-, 13q-, and 20q-. When patients in the total material (n = 154) were divided with regard to treatment during active PV, marked differences were observed. The highest frequency of abnormalities was found in patients given multiple lines of therapy (n = 61), dominating features being -5/5q- in 28 patients (46%), -7/7q- in 19 patients (31%), numerous translocations in 24 patients (39%), and unidentified markers in 22 patients (36%). Half of the patients treated with hydroxyurea alone showed a -5 or 5q- abnormality. In patients treated with phlebotomy alone, +8 and +9 were the most frequent findings. The type of therapy given during active PV influences the type of chromosome abnormalities present during terminal AML or MDS and can also be instrumental in the development of leukemia. PMID:18351338

Swolin, Birgitta; Rödjer, Stig; Westin, Jan

2008-06-01

356

Efficacy and safety of individually tailored antiplatelet therapy in patients with acute coronary syndrome after coronary stenting: a single center, randomized, feasibility study  

PubMed Central

Background Low responsiveness to clopidogrel (LRC) is associated with increased risk of ischemic events. This study was aimed to explore the feasibility of tailored antiplatelet therapy according to the responsiveness to clopidogrel. Methods A total of 305 clopidogrel naďve patients with acute coronary syndromes (ACS) undergoing coronary stenting were randomly assigned to receive standard (n = 151) or tailored (n = 154) antiplatelet therapy. The ADP-induced platelet aggregation tests by light transmission aggregometry were performed to identify LRC patients assigned to the tailored group. The standard antiplatelet regimen was dual antiplatelet therapy with aspirin and clopidogrel. The tailored antiplatelet therapy was standard regimen for non-LRC patients and an additional 6-month cilostazol treatment for LRC patients. The primary efficacy outcome was the composite of cardiovascular death, myocardial infarction or stroke at one year. Results LCR was present in 26.6% (41/154) of patients in the tailored group. The percentage platelet aggregation for LCR patients was significantly decreased at three days after adjunctive cilostazol treatment (77.5% ± 12.1% vs. 64.5% ± 12.1%, P < 0.001). At one year follow-up, a non-significant 37% relative risk reduction of primary events were observed in the tailored group as compared to the standard group (5.8% vs. 9.3%, P = 0.257). There were no differences in the rates of stent thrombosis and hemorrhagic events between the two groups. Conclusions Tailored antiplatelet therapy for ACS patients after coronary stenting according to responsiveness to clopidogrel is feasible. However, its efficacy and safety need further confirmation by clinical trials with larger sample sizes. PMID:25678901

Zhu, Hong-Chang; Li, Yi; Guan, Shao-Yi; Li, Jing; Wang, Xiao-Zeng; Jing, Quan-Min; Wang, Zu-Lu; Han, Ya-Ling

2015-01-01

357

Vertebral axial decompression therapy for pain associated with herniated or degenerated discs or facet syndrome: An outcome study  

Microsoft Academic Search

The outcomes of vertebral axial decompression (DECOMPRESSION) therapy for patients with low back pain from various causes are reported. Data was collected from twenty-two medical centers for patients who received DECOMPRESSION therapy for low back pain, which was sometimes accompanied by referred leg pain. Only patients who received at least ten sessions and had a diagnosis of herniated disc, degenerative

Earl E. Gose; William K. Naguszewski; Robert K. Naguszewski

358

Effect of high-intensity laser therapy in the management of myofascial pain syndrome of the trapezius: a double-blind, placebo-controlled study.  

PubMed

Myofascial pain syndrome (MPS) of the trapezius muscle is one of the main causes of neck pain. In this randomized, double-blind study, we evaluated the effects of high-intensity laser therapy (HILT) in female patients with chronic MPS of the trapezius muscle. The patients were assigned to two groups. The HILT group was treated with HILT and exercise, and the sham therapy group was treated with placebo HILT and exercise. The patients were assessed for pain, cervical active range of motion, disability, and quality of life. Evaluations were performed before treatment (week 0) and after treatment (weeks 4 and 12). Both groups showed significant improvement in all parameters at weeks 4 and 12. However, in a comparison of the percentage changes in the parameters at weeks 4 and 12 relative to pretreatment values, the HILT group showed greater improvement in pain scores, the neck disability index, and several subparts of the short-form 36 health survey (SF-36) (physical functioning, role limitations due to physical functioning, bodily pain, general health perceptions, social functioning, and role limitations due to emotional problems) than did the sham therapy group. We conclude that HILT is an effective therapeutic method in the treatment of patients with chronic MPS of the trapezius muscle. PMID:25274197

Dundar, Umit; Turkmen, Utku; Toktas, Hasan; Solak, Ozlem; Ulasli, Alper Murat

2015-01-01

359

Therapy-related myeloid neoplasms with isolated del(20q): comparison with cases of de novo myelodysplastic syndrome with del(20q).  

PubMed

The isolated deletion of chromosome 20q [del(20q)] has been observed in both de novo and therapy-related cases of myelodysplastic syndrome (MDS). The clinicopathologic features of de novo MDS with isolated del(20q) are well characterized. However, relatively little is known about therapy-related myeloid neoplasms (t-MNs) with isolated del(20q). In this study, we identified five cases of t-MN and 26 cases of de novo MDS with isolated del(20q) over a 10-year period. All cases had a long latency interval from the treatment of the primary malignancy to the onset of t-MN, and all were associated with frequent bone marrow dysplasia. The del(20q) was the sole abnormality detected at the time of diagnosis of t-MN in three cases, six years prior to diagnosis in one case, and at the time of relapse of acute myeloid leukemia (AML) in one case. Three patients with therapy-related MDS (t-MDS) had a relatively indolent clinical course, whereas two patients presented with AML or developed AML shortly after t-MDS. The patients with de novo MDS with isolated del(20q) presented frequently with anemia and thrombocytopenia which were associated with bone marrow dysplasia. The median overall survival was 64 months. In all cases, del(20q) was present at the time of diagnosis. PMID:23357231

Kanagal-Shamanna, Rashmi; Yin, C Cameron; Miranda, Roberto N; Bueso-Ramos, Carlos E; Wang, Xiaohong Iris; Muddasani, Ramya; Medeiros, L Jeffrey; Lu, Gary

2013-01-01

360

[Metabolic syndrome].  

PubMed

The metabolic syndrome consists of a cluster of metabolic diseases which often coexist: abdominal obesity, glucoseintolerance, diabetes mellitus type II, dyslipidemia, hypertension and impaired fibrinolysis. The common pathophysiologic link of these diseases in insulin resistance. All clinical disorders of the metabolic syndrome are risk factors for the vascular system. Since several diseases are present at the same time the risk for atherosclerotic complications such as coronary artery disease and apoplexy is potentiated. As a consequence the costs for direct and indirect health care are high. Besides a genetic predisposition the metabolic syndrome is mainly caused by the typical life style in industrialized countries with high energy and fat intake, physical inactivity, alcohol consumption, smoking, and stress. Therefore, prophylaxis and therapy imply the removal of these factors. In order to be successful experienced physicians and motivated patients are prerequisites. Even more affective than therapy is prophylaxis which is, however, not established in Germany. The metabolic syndrome is up to now not identified as a major health problem neither by the medical profession nor by health insurances and politicians. An effective therapy and prophylaxis would induce far-reaching changes in our health system and diminish health costs. PMID:9082643

Wirth, A; Rothenfelde, B

1996-12-01

361

The growing teratoma syndrome: results of therapy and long-term follow-up of 33 patients  

Microsoft Academic Search

Growing teratoma syndrome (GTS) is defined as an increase in tumour size during or after chemotherapy for germ cell tumour (GCT), and only mature teratoma at histological analysis of the resected tumour specimen. Between 1985 and 1997, 30 male patients fulfilling GTS criteria were included in the present study. 3 female patients were also included but analysed separately. A mature

F André; K Fizazi; S Culine; J.-P Droz; P Taupin; C Lhommé; M.-J Terrier-Lacombe; C Théodore

2000-01-01

362

Pentosan polysulfate therapy for chronic nonbacterial prostatitis (chronic pelvic pain syndrome category IIIA): a prospective multicenter clinical trial  

Microsoft Academic Search

Objectives. Chronic nonbacterial prostatitis\\/chronic pelvic pain syndrome (CPPS) has clinical and perhaps etiologic characteristics similar to interstitial cystitis. Pentosan polysulfate sodium (PPS), an oral medication indicated for the treatment of interstitial cystitis, has shown moderate benefit in reducing chronic pelvic pain and voiding symptoms in patients with interstitial cystitis. We undertook a prospective open-label, multicenter Phase II pilot study to

J. Curtis Nickel; Brenda Johnston; Joe Downey; Jack Barkin; Peter Pommerville; Mireille Gregoire; Ernest Ramsey

2000-01-01

363

The effects of scapular stabilization based exercise therapy on pain, posture, flexibility and shoulder mobility in patients with shoulder impingement syndrome: a controlled randomized clinical trial  

PubMed Central

Background: Dysfunction in the kinetic chain caused by poor scapula stabilization can contribute to shoulder injuries and Shoulder Impingement Syndrome (SIS). The purpose of this study was to compare the effectiveness of two treatment approaches scapular stabilization based exercise therapy and physical therapy in patients with SIS. Methods: The study is a randomized clinical trial in which 68 patients with SIS were randomly assigned in two groups of exercise therapy (ET) and physical therapy (PT) and received 18 sessions of treatment. Pain, shoulders' range of abduction and external rotation, shoulder protraction, scapular rotation and symmetry as well as postural assessment and Pectoralis minor length were evaluated pre and post intervention. The paired-sample t test and the independent sample t test were applied respectively to determine the differences in each group and between two groups. Results: Our findings indicated significant differences in abduction and external rotation range, improvement of forward shoulder translation and increase in the flexibility of the involved shoulder between the two groups (respectively ; p=0.024, p=0.001, p<0/0001, p<0/0001). No significant difference was detected in pain reduction between the groups (p=0.576). Protraction of the shoulder (p<0.0001), forward head posture (p<0/0001) and mid thoracic curvature (p<0.0001) revealed a significant improvement in the ET group. Apparent changes occurred in scapular rotation and symmetry in both groups but no significant differences were observed between the two groups (respectively; p=0.183, p=0.578). Conclusion: The scapular stabilization based exercise intervention was successful in increasing shoulder range, decreasing forward head and shoulder postures and Pectoralis minor flexibility. PMID:25664288

Moezy, Azar; Sepehrifar, Saeed; Solaymani Dodaran, Masoud

2014-01-01

364

Insult-dependent effect of bone marrow cell therapy on inflammatory response in a murine model of extrapulmonary acute respiratory distress syndrome  

PubMed Central

Introduction Administration of bone marrow-derived cells produces beneficial effects in experimental extrapulmonary acute respiratory distress syndrome (ARDS). However, there are controversies regarding the effects of timing of cell administration and initial insult severity on inflammatory response. We evaluated the effects of bone marrow-derived mononuclear cells (BMDMC) in two models of extrapulmonary ARDS once lung morphofunctional changes had already been installed. Methods BALB/c mice received lipopolysaccharide (LPS) intraperitoneally (5 mg/kg in 0.5 ml saline) or underwent cecal ligation and puncture (CLP). Control mice received saline intraperitoneally (0.5 ml) or underwent sham surgery. At 24 hours, groups were further randomized to receive saline or BMDMC (2 × 106) intravenously. Lung mechanics, histology, and humoral and cellular parameters of lung inflammation and remodeling were analyzed 1, 3 and 7 days after ARDS induction. Results BMDMC therapy led to improved survival in the CLP group, reduced lung elastance, alveolar collapse, tissue and bronchoalveolar lavage fluid cellularity, collagen fiber content, and interleukin-1? and increased chemokine (keratinocyte-derived chemokine and monocyte chemotactic protein-1) expression in lung tissue regardless of the experimental ARDS model. Intercellular adhesion molecule-1 and vascular cell adhesion molecule-1 expression in lung tissue increased after cell therapy depending on the insult (LPS or CLP). Conclusions BMDMC therapy at day 1 successfully reduced lung inflammation and remodeling, thus contributing to improvement of lung mechanics in both extrapulmonary ARDS models. Nevertheless, the different inflammatory responses induced by LPS and CLP resulted in distinct effects of BMDMC therapy. These data may be useful in the clinical setting, as they suggest that the type of initial insult plays a key role in the outcome of treatment. PMID:24406030

2013-01-01

365

Recurrent Giant Molluscum Contagiosum Immune Reconstitution Inflammatory Syndrome (IRIS) after Initiation of Antiretroviral Therapy in an HIV-infected South African Male  

PubMed Central

Summary We describe an HIV-infected South African male who experienced two distinct episodes of disseminated giant molluscum contagiosum (MC) immune reconstitution inflammatory syndrome (IRIS) over a 6-year period. The first episode of MC-IRIS occurred with rapid virologic suppression following antiretroviral therapy (ART) initiation. The second episode occurred during a rapid increase in CD4 cells following stable viral suppression with second-line ART. His MC lesions then completely resolved during a reduction in CD4 count, despite maintaining virologic suppression. Nearly one year after the resolution of his giant MC-IRIS lesions, he maintains an undetectable viral load, but his level of immune deficiency has not improved. In the absence of well-controlled therapeutic trials, MC-IRIS presents important management challenges. PMID:23970647

Drain, Paul K.; Mosam, Anisa; Gounder, Lilishia; Gosnell, Bernadett; Manzini, Thandekile; Moosa, Mahomed-Yunus S.

2014-01-01

366

Beta-adrenergic modulation of arrhythmogenesis and identification of targeted sites of antiarrhythmic therapy in Timothy (LQT8) syndrome: a theoretical study.  

PubMed

Timothy syndrome (TS) is a malignant form of congenital long QT syndrome with a mode of arrhythmia onset often triggered by enhanced sympathetic tone. We sought to explore mechanisms by which beta-adrenergic stimulation (BAS) modulates arrhythmogenesis and to identify potential targeted sites of antiarrhythmic therapy in TS. Using a dynamic Luo-Rudy ventricular myocyte model incorporated with detailed intracellular Ca(2+) cycling, along with its one-dimensional multicellular strand, we simulated various clinical scenarios of TS, with stepwise increase in the percentage of G406R Ca(v)1.2 channels from 0 to 11.5 and 23%, and to 38.5 and 77%, respectively, for heterozygous and homozygous states of TS1 and TS2. Progressive prolongation of action potential duration (APD) and QT interval, accompanied by amplification of transmural dispersion of repolarization, steepening of APD restitution, induction of delayed afterdepolariztions (DADs), and both DAD and phase 3 early afterdepolariztion-mediated triggered activities, correlated well with the extent of G406R Ca(v)1.2 channel mutation. BAS amplified transmural dispersion of repolarization, steepened APD restitution, and facilitated inducibility of DAD-mediated triggered activity. Systematic analysis of intracellular Ca(2+) cycling revealed that sarcoplasmic reticulum Ca(2+) ATPase (uptake current) played an essential role in BAS-induced facilitation of DAD-mediated triggered activity and, in addition to L-type calcium current, it could be an effective site of antiarrhythmic therapy under the influence of BAS. Thus G406R Ca(v)1.2 channel mutation confers not only a trigger, but also a substrate for lethal ventricular arrhythmias, which can be exaggerated by BAS. It is suggested that, besides beta-adrenergic blockers and L-type calcium current channel blockers, an agent aimed at reduction of sarcoplasmic reticulum Ca(2+) ATPase uptake current may provide additional antiarrhythmic effect in patients with TS. PMID:19855067

Sung, Ruey J; Wu, Yung-Han; Lai, Nathan Hsing-Jung; Teng, Chun-Hao; Luo, Ching-Hsing; Tien, Hui-Chun; Lo, Chu-Pin; Wu, Sheng-Nan

2010-01-01

367

Characteristics of children and young adults with Marfan syndrome and aortic root dilation in a randomized trial comparing atenolol and losartan therapy  

PubMed Central

Background The Pediatric Heart Network designed a clinical trial to compare aortic root growth and other short-term cardiovascular outcomes in children and young adults with Marfan syndrome randomized to receive atenolol or losartan. We report here the characteristics of the screened population and enrolled subjects. Methods and results Between 2007 and 2011, 21 clinical sites randomized 608 subjects, aged 6 months to 25 years who met the original Ghent criteria and had a body surface area–adjusted aortic root diameter z-score >3.0. The mean age at study entry was 11.2 years, 60% were male, and 25% were older teenagers and young adults. The median aortic root diameter z-score was 4.0. Aortic root diameter z-score did not vary with age. Mitral valve prolapse and mitral regurgitation were more common in females. Among those with a positive family history, 56% had a family member with aortic surgery, and 32% had a family member with a history of aortic dissection. Conclusions Baseline demographic, clinical, and anthropometric characteristics of the randomized cohort are representative of patients in this population with moderate to severe aortic root dilation. The high percentage of young subjects with relatives who have had aortic dissection or surgery illustrates the need for more definitive therapy; we expect that the results of the study and the wealth of systematic data collected will make an important contribution to the management of individuals with Marfan syndrome. PMID:23622922

Lacro, Ronald V.; Guey, Lin T.; Dietz, Harry C.; Pearson, Gail D.; Yetman, Anji T.; Gelb, Bruce D.; Loeys, Bart L.; Benson, D. Woodrow; Bradley, Timothy J.; De Backer, Julie; Forbus, Geoffrey A.; Klein, Gloria L.; Lai, Wyman W.; Levine, Jami C.; Lewin, Mark B.; Markham, Larry W.; Paridon, Stephen M.; Pierpont, Mary Ella; Radojewski, Elizabeth; Selamet Tierney, Elif Seda; Sharkey, Angela M.; Wechsler, Stephanie Burns; Mahony, Lynn

2013-01-01

368

Single strand conformation polymorphism analysis of androgen receptor gene mutations in patients with androgen insensitivity syndromes: Application for diagnosis, genetic counseling, and therapy  

SciTech Connect

Recent studies indicate that mutations in the androgen receptor gene are associated with androgen insensitivity syndromes, a heterogeneous group of related disorders involving defective sexual differentiation in karyotypic males. In this report, the authors address the possibility of rapid mutational analysis of the androgen receptor gene for initial diagnosis, genetic counseling, and molecular subclassification of affected patients and their families. DNA from peripheral blood leukocytes of six patients from five families with various degrees of androgen insensitivity was studied. Exons 2 to 8 of the androgen receptor gene were analyzed using a combination of single strand conformation polymorphism analysis and direct DNA sequencing. Female family members were also studied to identify heterozygote carriers. Point mutations in the AR gene were identified in all six patients, and all mutations caused amino acid substitutions. One patient with incomplete androgen insensitivity was a mosaic for the mutation. Four of the five mothers, as well as a young sister of one patient, were carriers of the mutation present in the affected child. The data show that new mutations may occur in the androgen receptor gene leading to sporadic androgen insensitivity syndrome. Molecular genetic characterization of the variant allele can serve as a primary tool for diagnosis and subsequent therapy, and can provide a basis for distinguishing heterozygous carriers in familial androgen resistance. The identification of carriers is of substantial clinical importance for genetic counseling. 29 refs., 2 figs., 1 tab.

Hiort, O. (Medizinische Universitaet zu Luebeck (Germany) Tufts-New England Medical Center, Boston, MA (United States)); Huang, Q. (Massachusetts Eye and Ear Infirmary, Boston, MA (United States)); Sinnecker, G.H.G.; Kruse, K. (Medizinische Universitaet zu Luebeck (Germany)); Sadeghi-Nejad, A.; Wolfe, H.J. (Tufts-New England Medical Center, Boston, MA (United States)); Yandell, D.W. (Massachusetts Eye and Ear Infirmary, Boston, MA (United States))(Harvard Medical School, Boston, MA (United States) Harvard School of Public Health, Boston, MA (United States))

1993-07-01

369

Pulsed versus continuous wave low-level light therapy on osteoarticular signs and symptoms in limited scleroderma (CREST syndrome): a case report  

NASA Astrophysics Data System (ADS)

Limited cutaneous systemic sclerosis (lcSSc) was formerly known as CREST syndrome in reference to the associated clinical features: calcinosis, Raynaud's phenomenon, esophageal dysfunction, sclerodactyly, and telangiectasias. The transforming growth factor beta has been identified as a major player in the pathogenic process, where low-level light therapy (LLLT) has been shown to modulate this cytokine superfamily. This case study was conducted to assess the efficacy of 940 nm using millisecond pulsing and continuous wave (CW) modes on osteoarticular signs and symptoms associated with lcSSc. The patient was treated two to three times a week for 13 weeks using a sequential pulsing mode on one elbow and a CW mode on the other. Efficacy assessments included inflammation, symptoms, pain, health scales, patient satisfaction, clinical global impression, and adverse effects monitoring. Considerable functional and morphologic improvements were observed after LLLT, with the best results seen with the pulsing mode. No adverse effects were noted. Pulsed LLLT represents a treatment alternative for osteoarticular signs and symptoms in limited scleroderma (CREST syndrome).

Barolet, Daniel

2014-11-01

370

A review on cognitive behavorial therapy (CBT) and graded exercise therapy (GET) in myalgic encephalomyelitis (ME) \\/ chronic fatigue syndrome (CFS): CBT\\/GET is not only ineffective and not evidence-based, but also potentially harmful for many patients with ME\\/CFS  

Microsoft Academic Search

Benign Myalgic Encephalomyelitis (ME) \\/ Chronic Fatigue Syndrome (CFS) is a debilitating disease which, despite numerous biological abnormalities has remained highly controversial. Notwithstanding the medical pathogenesis of ME\\/CFS, the (bio)psychosocial model is adopted by many governmental organizations and medical profes sio nals to legitimize the combination of Cognitive Behavioral Therapy (CBT) and Graded Exercise Therapy (GET) for ME\\/CFS. Justified by

Frank N. M. Twisk; Michael Maes

371

Neuroacanthocytosis Syndromes  

PubMed Central

Neuroacanthocytosis (NA) syndromes are a group of genetically defined diseases characterized by the association of red blood cell acanthocytosis and progressive degeneration of the basal ganglia. NA syndromes are exceptionally rare with an estimated prevalence of less than 1 to 5 per 1'000'000 inhabitants for each disorder. The core NA syndromes include autosomal recessive chorea-acanthocytosis and X-linked McLeod syndrome which have a Huntington´s disease-like phenotype consisting of a choreatic movement disorder, psychiatric manifestations and cognitive decline, and additional multi-system features including myopathy and axonal neuropathy. In addition, cardiomyopathy may occur in McLeod syndrome. Acanthocytes are also found in a proportion of patients with autosomal dominant Huntington's disease-like 2, autosomal recessive pantothenate kinase-associated neurodegeneration and several inherited disorders of lipoprotein metabolism, namely abetalipoproteinemia (Bassen-Kornzweig syndrome) and hypobetalipoproteinemia leading to vitamin E malabsorption. The latter disorders are characterized by a peripheral neuropathy and sensory ataxia due to dorsal column degeneration, but movement disorders and cognitive impairment are not present. NA syndromes are caused by disease-specific genetic mutations. The mechanism by which these mutations cause neurodegeneration is not known. The association of the acanthocytic membrane abnormality with selective degeneration of the basal ganglia, however, suggests a common pathogenetic pathway. Laboratory tests include blood smears to detect acanthocytosis and determination of serum creatine kinase. Cerebral magnetic resonance imaging may demonstrate striatal atrophy. Kell and Kx blood group antigens are reduced or absent in McLeod syndrome. Western blot for chorein demonstrates absence of this protein in red blood cells of chorea-acanthocytosis patients. Specific genetic testing is possible in all NA syndromes. Differential diagnoses include Huntington disease and other causes of progressive hyperkinetic movement disorders. There are no curative therapies for NA syndromes. Regular cardiologic studies and avoidance of transfusion complications are mandatory in McLeod syndrome. The hyperkinetic movement disorder may be treated as in Huntington disease. Other symptoms including psychiatric manifestations should be managed in a symptom-oriented manner. NA syndromes have a relentlessly progressive course usually over two to three decades. PMID:22027213

2011-01-01

372

Evaluation of antisecretory drug therapy of zollinger-ellison syndrome (ZES) using 24-hour pH monitoring  

Microsoft Academic Search

Clinical and endoscopic findings were compared with 24-hr pH profiles in 8 patients with Zollinger-Ellison syndrome (ZES), who were being treated, during consecutive periods, with doses of cimetidine or ranitidine. There was a positive correlation between the effect on gastric pH and the outcome of treatment, with ranitidine proving to be more effective. The reduction of gastric acidity was correlated

T. Vallot; M. Mignon; R. Mazure; S. Bonfils

1983-01-01

373

Mucopolysaccharidosis type II (Hunter syndrome): a clinical review and recommendations for treatment in the era of enzyme replacement therapy  

Microsoft Academic Search

Mucopolysaccharidosis type II (MPS II; Hunter syndrome) is a rare X-linked recessive disease caused by deficiency of the lysosomal\\u000a enzyme iduronate-2-sulphatase, leading to progressive accumulation of glycosaminoglycans in nearly all cell types, tissues\\u000a and organs. Clinical manifestations include severe airway obstruction, skeletal deformities, cardiomyopathy and, in most patients,\\u000a neurological decline. Death usually occurs in the second decade of life, although

J. Edmond Wraith; Maurizio Scarpa; Michael Beck; Olaf A. Bodamer; Linda De Meirleir; Nathalie Guffon; Allan Meldgaard Lund; Gunilla Malm; Ans T. Van der Ploeg; Jiri Zeman

2008-01-01

374

Metformin therapy in a hyperandrogenic anovulatory mutant murine model with polycystic ovarian syndrome characteristics improves oocyte maturity during superovulation  

Microsoft Academic Search

Background  Metformin, an oral biguanide traditionally used for the treatment of type 2 diabetes, is widely used for the management of\\u000a polycystic ovary syndrome (PCOS)-related anovulation. Because of the significant prevalence of insulin resistance and glucose\\u000a intolerance in PCOS patients, and their putative role in ovulatory dysfunction, the use of metformin was touted as a means\\u000a to improve ovulatory function and

Mary E Sabatini; Lankai Guo; Maureen P Lynch; Joseph O Doyle; HoJoon Lee; Bo R Rueda; Aaron K Styer

2011-01-01

375

Long-Term Effectiveness and Compliance of Positional Therapy with the Sleep Position Trainer in the Treatment of Positional Obstructive Sleep Apnea Syndrome  

PubMed Central

Study Objectives: To investigate effectiveness, long-term compliance, and effects on subjective sleep of the Sleep Position Trainer (SPT) in patients with position-dependent obstructive sleep apnea syndrome (POSAS). Design: Prospective, multicenter cohort study. Patients or Participants: Adult patients with mild and moderate POSAS were included. Interventions: Patients asked to use the SPT for 6 mo. At baseline and after 1, 3, and 6 mo, questionnaires would be completed: Epworth Sleepiness Scale (ESS), Pittsburgh Sleep Quality Index (PSQI), Functional Outcomes of Sleep Questionnaire (FOSQ), and questions related to SPT use. Measurements and Results: One hundred forty-five patients were included. SPT use and SPT data could not be retrieved in 39 patients. In the remaining 106 patients, median percentage of supine sleep decreased rapidly during SPT's training phase (day 3 to 9) to near-total avoidance of supine sleep. This decrease was maintained during the following months of treatment (21% at baseline versus 3% at 6 mo). SPT compliance, defined as more than 4 h of nightly use, was 64.4%. Regular use, defined as more than 4 h of usage over 5 nights/w, was 71.2%. Subjective compliance and regular use were 59.8% and 74.4%, respectively. Median ESS (11 to 8), PSQI (8 to 6), and FOSQ (87 to 103) values significantly improved compared with baseline. Conclusions: Positional therapy using the Sleep Position Trainer (SPT) effectively diminished the percentage of supine sleep and subjective sleepiness and improved sleep related quality of life in patients with mild to moderate position-dependent obstructive sleep apnea syndrome. SPT treatment appeared to have sustained effects over 6 months. SPT compliance and regular use rate were relatively good. Subjective and objective compliance data corresponded well. The lack of a placebo-controlled group limited the efficacy of conclusions. Citation: van Maamen JP, de Vries N. Long-term effectiveness and compliance of positional therapy with the Sleep Position Trainer in the treatment of positional obstructive sleep apnea syndrome. SLEEP 2014;37(7):1209-1215. PMID:25061249

van Maanen, J. Peter; de Vries, Nico

2014-01-01

376

The TRACTISS Protocol: a randomised double blind placebo controlled clinical TRial of Anti-B-Cell Therapy In patients with primary Sjögren’s Syndrome  

PubMed Central

Background Primary Sjögren’s Syndrome (PSS) mainly affects women (9:1 female:male ratio) and is one of the commonest autoimmune diseases with a prevalence of 0.1 – 0.6% of adult women. For patients with PSS there is currently no effective therapy that can alter the progression of the disease. The aim of the TRACTISS study is to establish whether in patients with PSS, treatment with rituximab improves clinical outcomes. Methods/design TRACTISS is a UK multi-centre, double-blind, randomised, controlled, parallel group trial of 110 patients with PSS. Patients will be randomised on a 1:1 basis to receive two courses of either rituximab or placebo infusion in addition to standard therapy, and will be followed up for up to 48 weeks. The primary objective is to assess the extent to which rituximab improves symptoms of fatigue and oral dryness. Secondary outcomes include ocular dryness, salivary flow rates, lacrimal flow, patient quality of life, measures of disease damage and disease activity, serological and peripheral blood biomarkers, and glandular histology and composition. Discussion The TRACTISS trial will provide direct evidence as to whether rituximab in patients with PSS leads to an improvement in patient symptoms and a reduction in disease damage and activity. Trial registration UKCRN Portfolio ID: 9809 ISRCTN65360827. PMID:24438039

2014-01-01

377

Effect of blood hematocrit and erythrocyte deformability on adenosine 5'-diphosphate platelet reactivity in patients with acute coronary syndromes on dual antiplatelet therapy.  

PubMed

Previous studies have explored the association between hemorheologic alterations and aspirin resistance, pointing out the possible interaction between hematologic components and platelet responsiveness to antiplatelet drugs. The aim of this study was to evaluate the association between hemorheologic variables and residual platelet reactivity in patients with acute coronary syndromes (ACSs) who underwent percutaneous coronary intervention on dual antiplatelet therapy. The study population included 528 patients with ACSs. Hemorheologic studies were performed by assessing whole blood viscosity at 0.512 and 94.5/second, plasma viscosity, and erythrocyte deformability index. Post-treatment platelet reactivity was investigated by measuring platelet aggregation by adenosine 5'-diphosphate (ADP) 10 mumol and a value >70% was defined as high ADP platelet reactivity. Significantly (p <0.01) lower values of hematocrit and erythrocyte deformability and higher values of whole blood viscosity at 94.5/second were found in patients with high ADP platelet reactivity. At multivariate analysis, lower values of hematocrit and erythrocyte deformability index and higher values of whole blood viscosity at 94.5/second and leukocytes (highest vs lowest tertile) also resulted in an independent association with high platelet reactivity, except for leukocytes, after simultaneous adjustment for hematocrit, leukocyte count, and erythrocyte deformability index. In conclusion, these results demonstrate the influence of hematocrit and of erythrocyte deformability on ADP platelet reactivity. These variables could be considered to optimize treatment with antiplatelet therapy in these patients. PMID:19733708

Cecchi, Emanuele; Marcucci, Rossella; Paniccia, Rita; Bandinelli, Brunella; Valente, Serafina; Giglioli, Cristina; Lazzeri, Chiara; Gensini, Gian Franco; Abbate, Rosanna; Mannini, Lucia

2009-09-15

378

Successful management of insulin allergy and autoimmune polyendocrine syndrome type 4 with desensitization therapy and glucocorticoid treatment: a case report and review of the literature.  

PubMed

Introduction. Insulin allergy is a rare complication of insulin therapy, especially in type 1 diabetes mellitus (T1DM). Key manifestations are hypersensitivity-related symptoms and poor metabolic control. T1DM, as well as insulin allergy, may develop in the context of autoimmune polyendocrine syndrome (APS), further complicating management. Case Report. A 17-year-old male patient, diagnosed with T1DM, was treated with various insulin therapy schemes over several months, which resulted in recurrent anaphylactoid reactions and poor glycemic control, after which he was referred to our Endocrinology and Immunology Department. A prick test was carried out for all commercially available insulin presentations and another insulin scheme was designed but proved unsuccessful. A desensitization protocol was started with Glargine alongside administration of Prednisone, which successfully induced tolerance. Observation of skin lesions typical of vitiligo prompted laboratory workup for other autoimmune disorders, which returned positive for autoimmune gastritis/pernicious anemia. These findings are compatible with APS type 4. Discussion. To our knowledge, this is the first documented case of insulin allergy in type 4 APS, as well as this particular combination in APS. Etiopathogenic components shared by insulin allergy and APS beg for further research in immunogenetics to further comprehend pathophysiologic aspects of these diseases. PMID:25548690

Rojas, Joselyn; Villalobos, Marjorie; Martínez, María Sofía; Chávez-Castillo, Mervin; Torres, Wheeler; Mejías, José Carlos; Miquilena, Edgar; Bermúdez, Valmore

2014-01-01

379

Successful Management of Insulin Allergy and Autoimmune Polyendocrine Syndrome Type 4 with Desensitization Therapy and Glucocorticoid Treatment: A Case Report and Review of the Literature  

PubMed Central

Introduction. Insulin allergy is a rare complication of insulin therapy, especially in type 1 diabetes mellitus (T1DM). Key manifestations are hypersensitivity-related symptoms and poor metabolic control. T1DM, as well as insulin allergy, may develop in the context of autoimmune polyendocrine syndrome (APS), further complicating management. Case Report. A 17-year-old male patient, diagnosed with T1DM, was treated with various insulin therapy schemes over several months, which resulted in recurrent anaphylactoid reactions and poor glycemic control, after which he was referred to our Endocrinology and Immunology Department. A prick test was carried out for all commercially available insulin presentations and another insulin scheme was designed but proved unsuccessful. A desensitization protocol was started with Glargine alongside administration of Prednisone, which successfully induced tolerance. Observation of skin lesions typical of vitiligo prompted laboratory workup for other autoimmune disorders, which returned positive for autoimmune gastritis/pernicious anemia. These findings are compatible with APS type 4. Discussion. To our knowledge, this is the first documented case of insulin allergy in type 4 APS, as well as this particular combination in APS. Etiopathogenic components shared by insulin allergy and APS beg for further research in immunogenetics to further comprehend pathophysiologic aspects of these diseases. PMID:25548690

Villalobos, Marjorie; Martínez, María Sofía; Chávez-Castillo, Mervin; Mejías, José Carlos; Miquilena, Edgar; Bermúdez, Valmore

2014-01-01

380

The Othello Syndrome  

PubMed Central

A case of the Othello syndrome is presented. In its classical form the syndrome is rare, but as with other allied paranoid states, its medicosocial implications are great. Rational management should include pharmacotherapy, conjoint family therapy after symptom remission, and long-term individual psychotherapy. PMID:6827614

Famuyiwa, Oluwole O.; Ekpo, Micheal

1983-01-01

381

The Othello syndrome.  

PubMed

A case of the Othello syndrome is presented. In its classical form the syndrome is rare, but as with other allied paranoid states, its medicosocial implications are great. Rational management should include pharmacotherapy, conjoint family therapy after symptom remission, and long-term individual psychotherapy. PMID:6827614

Famuyiwa, O O; Ekpo, M

1983-02-01

382

Lymphedema in Noonan's syndrome.  

PubMed

Chronic edema can be debilitating in Noonan's syndrome. Transient childhood lymphedema may be followed by elephantiasis as early as adolescence. Recurrent streptococcal cellulitis exacerbates this problem. Hygenic measures and prophylactic antiobiotic therapy may slow progression of lymphedema of the legs in Noonan's syndrome. PMID:6396246

White, S W

1984-12-01

383

Dual antiplatelet therapy with or without oral anticoagulation in the postdischarge management of acute coronary syndrome patients with an indication for long term anticoagulation: a systematic review.  

PubMed

Currently, there is a lack of consensus among guidelines for the postdischarge treatment of patients presenting with acute coronary syndrome (ACS) who have a long-term indication for anticoagulation. We conducted a systematic review comparing the safety and effectiveness of dual antiplatelet therapy (DAPT) and triple therapy (TT; defined as DAPT plus an oral anticoagulant) in patients with ACS and a long-term indication for anticoagulation. We searched for clinical studies in MEDLINE, EMBASE, and the Cochrane Database of Systematic Reviews published between January 1995 and September 2013. Each investigator screened and abstracted data, assessed applicability and quality, and graded the strength of evidence. Meta-analysis of direct comparison was performed when outcomes and follow-up periods were comparable. Fourteen observational studies were identified that contained comparative effectiveness data on DAPT versus TT. No difference in the odds of mortality (OR 1.04, 95% CI 0.59-1.83) or stroke (OR 1.01, 95% CI 0.38-2.67) at 1-5 years was found between TT and DAPT. Major bleeding at 1-5 years (OR 1.46, 95% CI 1.07-2.00) and nonfatal MI at 1-5 years (OR 1.85, 95% CI 1.13-3.02) occurred more frequently in patients receiving TT. The results of this systematic review demonstrate that treatment with TT was associated with increased rates of nonfatal MI and major bleeding when compared with treatment with DAPT in the postdischarge management of ACS patients with an indication for oral anticoagulation. Until results of ongoing randomized trials assessing antithrombotic therapies define optimal management strategies, the current analysis suggests using caution when prescribing TT to these patients. PMID:24891133

Washam, Jeffrey B; Dolor, Rowena J; Jones, W Schuyler; Halim, Sharif A; Hasselblad, Victor; Mayer, Stephanie B; Heidenfelder, Brooke L; Melloni, Chiara

2014-10-01

384

Mutational profiling of therapy-related myelodysplastic syndromes and acute myeloid leukemia by next generation sequencing, a comparison with de novo diseases.  

PubMed

In this study we used a next generation sequencing-based approach to profile gene mutations in therapy-related myelodysplastic syndromes (t-MDS) and acute myeloid leukemia (t-AML); and compared these findings with de novo MDS/AML. Consecutive bone marrow samples of 498 patients, including 70 therapy-related (28 MDS and 42 AML) and 428 de novo (147 MDS and 281 AML) were analyzed using a modified-TruSeq Amplicon Cancer Panel (Illumina) covering mutation hotspots of 53 genes. Overall, mutation(s) were detected in 58.6% of t-MDS/AML and 56.8% of de novo MDS/AML. Of therapy-related cases, mutations were detected in 71.4% of t-AML versus 39.3% t-MDS (p=0.0127). TP53 was the most common mutated gene in t-MDS (35.7%) as well as t-AML (33.3%), significantly higher than de novo MDS (17.7%) (p=0.0410) and de novo AML (12.8%) (p=0.0020). t-AML showed more frequent PTPN11 but less NPM1 and FLT3 mutations than de novo AML. In summary, t-MDS/AML shows a mutation profile different from their de novo counterparts. TP53 mutations are highly and similarly prevalent in t-MDS and t-AML but mutations in genes other than TP53 were more frequent in t-AML than t-MDS. The molecular genetic profiling further expands our understanding in this group of clinically aggressive yet heterogeneous myeloid neoplasms. PMID:25573287

Ok, Chi Young; Patel, Keyur P; Garcia-Manero, Guillermo; Routbort, Mark J; Fu, Bin; Tang, Guilin; Goswami, Maitrayee; Singh, Rajesh; Kanagal-Shamanna, Rashmi; Pierce, Sherry A; Young, Ken H; Kantarjian, Hagop M; Medeiros, L Jeffrey; Luthra, Rajyalakshmi; Wang, Sa A

2015-03-01

385

Efficacy of EMLA cream phonophoresis comparison with ultrasound therapy on myofascial pain syndrome of the trapezius: a single-blind, randomized clinical study.  

PubMed

The aim of this study is to investigate whether eutectic mixture of local anesthetics (EMLA) cream phonophoresis superior to conventional US over the trigger points (TPs) in terms of improvements of pain, range of motion and disability in myofascial pain syndrome (MPS). Fifty patients (42 female, 8 male) diagnosed with MPS were included in the study. Patients were randomly assigned into two treatment groups including phonophoresis (PH) group (n = 25) and ultrasound (US) group (n = 25). PH group received EMLA cream phonophoresis (2.5 % lidocaine, 2.5 % prilocaine); US group received conventional ultrasound therapy over the all active TPs on trapezius muscle for 10 min a day for 15 sessions. Outcome measures were performed before the treatment course and at the end of a 15-session course of treatment. Student T, Mann-Whitney U, chi-square and Wilcoxon tests were used for statistical analysis. At the end of the therapy, there was statistically significant decrease in both PH group and US group in terms of number of trigger point (NTP) (p = 0.001, p = 0.029), pain intensity on movement (p = 0.001 vs. 0.002) and right/left cervical lateral ROMs (p = 0.001/p = 0.001, p = 0.009/p = 0.020) relative to baseline. The NTP decrease in PH group was significantly higher than that in US group (1.84 ± 1.46 vs. 0.72 ± 1.45; p = 0.01). Pain intensity at rest (p = 0.001) and NPDI scores (p = 0.001) were statistically improvement in only PH group. EMLA cream phonophoresis is more effective than conventional ultrasound therapy in terms of pain and associated neck disability, and it seems the complementary treatment option for MPS. PMID:24149990

Ustun, Nilgun; Arslan, Fatma; Mansuroglu, Ayhan; Inanoglu, Deniz; Yag?z, Abdullah Erman; Guler, Hayal; Turhanoglu, Ayse Dicle

2014-04-01

386

Laser therapy of painful shoulder and shoulder-hand syndrome in treatment of patients after the stroke.  

PubMed

The common complication after stroke is pain and dysfunction of shoulder of paralyzed arm, as well as the swelling of the hand. The aim of this study was to determine the effects of LASER therapy and to correlate with electrotherapy (TENS, stabile galvanization) in subjects after stroke. We analyzed 70 subjects after stroke with pain in shoulder and oedema of paralyzed hand. The examinees were divided in two groups of 35, and they were treated in the Clinic for Physical Medicine and Rehabilitation in Tuzla during 2006 and 2007. Experimental group (EG) had a treatment with LASER, while the control group (CG) was treated with electrotherapy. Both groups had kinesis therapy and ice massage. All patients were examined on the admission and discharge by using the VAS, DASH, Barthel index and FIM. The pain intensity in shoulder was significantly reduced in EG (p<0,0001), swelling is lowered in EG (p=0,01). Barthel index in both groups was significant higher (p<0,01). DASH was significantly improved after LASER therapy in EG (p<0,01). EG had higher level of independency (p<0,01). LASER therapy used on EG shows significantly better results in reducing pain, swelling, disability and improvement of independency. PMID:19284397

Karabegovi?, Azra; Kapidzi?-Durakovi?, Suada; Ljuca, Farid

2009-02-01

387

Implantable Cardioverter?Defibrillator Therapy in Patients With Ventricular Fibrillation out of Hospital Cardiac Arrest Secondary to Acute Coronary Syndrome  

PubMed Central

Background Survivors of ventricular fibrillation out of hospital cardiac arrest (VF?OHCA) due to a potentially reversible cause such as acute myocardial infarction (MI) or ischemia are considered to be at low risk of recurrent arrhythmia. Implantable cardioverter defibrillators (ICD) are not routinely recommended in such patients. However, the outcome of these patients in the era of rapid coronary revascularization and ICD therapy is not known. Methods and Results We examined the outcome of 114 consecutive survivors of VF OHCA due to acute MI or ischemia in Olmsted County, MN from 1990 to 2011. An ICD was implanted in 45/114 patients. ICD recipients had lower EF [median (IQR) 38 (26 to 54) versus 48 (35 to 58) %, P=0.04]. During a median (IQR) follow?up of 9.9 (4.4 to 14.6) years, ICD implantation was associated with reduced cardiac mortality (HR 0.24 [0.07 to 0.88], P=0.031) and a trend towards reduced all?cause mortality (HR 0.56 [0.30 to 1.02], P=0.059) after adjusting for the first principal component. One or more appropriate ICD therapies were delivered in 19/45, with half of the patients receiving therapy within 1 year. Patients with EF ?35% at discharge continued to be at long?term risk for ICD therapy compared with those with EF >35% who were at increased risk predominantly in the first 8 months. EF and revascularization were not significantly associated with ICD therapy in the multivariable analysis. Conclusions Patients with VF?OHCA in the setting of acute MI or myocardial ischemia remain at high risk of recurrent ventricular arrhythmias, particularly if EF ?35%. This suggests that ICD implantation may be reasonable if EF ?35%. PMID:25713292

Madhavan, Malini; Friedman, Paul A.; Lennon, Ryan J.; Prasad, Abhiram; White, Roger D.; Sriram, Chenni S.; Gulati, Rajiv; Gersh, Bernard J.

2015-01-01

388

Gut-directed hypnotherapy in children with irritable bowel syndrome or functional abdominal pain (syndrome): a randomized controlled trial on self exercises at home using CD versus individual therapy by qualified therapists  

PubMed Central

Background Irritable bowel syndrome (IBS) and functional abdominal pain (syndrome) (FAP(S)) are common pediatric disorders, characterized by chronic or recurrent abdominal pain. Treatment is challenging, especially in children with persisting symptoms. Gut-directed hypnotherapy (HT) performed by a therapist has been shown to be effective in these children, but is still unavailable to many children due to costs, a lack of qualified child-hypnotherapists and because it requires a significant investment of time by child and parent(s). Home-based hypnotherapy by means of exercises on CD has been shown effective as well, and has potential benefits, such as lower costs and less time investment. The aim of this randomized controlled trial (RCT) is to compare cost-effectiveness of individual HT performed by a qualified therapist with HT by means of CD recorded self-exercises at home in children with IBS or FAP(S). Methods/Design 260 children, aged 8-18 years with IBS or FAP(S) according to Rome III criteria are included in this currently conducted RCT with a follow-up period of one year. Children are randomized to either 6 sessions of individual HT given by a qualified therapist over a 3-month period or HT through self-exercises at home with CD for 3 months. The primary outcome is the proportion of patients in which treatment is successful at the end of treatment and after one year follow-up. Treatment success is defined as at least 50% reduction in both abdominal pain frequency and intensity scores. Secondary outcomes include adequate relief, cost-effectiveness and effects of both therapies on depression and anxiety scores, somatization scores, QoL, pain beliefs and coping strategies. Discussion If the effectiveness of home-based HT with CD is comparable to, or only slightly lower, than HT by a therapist, this treatment may become an attractive form of therapy in children with IBS or FAP(S), because of its low costs and direct availability. Trial registration Dutch Trial Register number NTR2725 (date of registration: 1 February 2011) PMID:24894077

2014-01-01

389

Lifestyle intervention and anti-obesity therapies in the polycystic ovary syndrome: impact on metabolism and fertility.  

PubMed

Obesity is frequently present in patients with polycystic ovary syndrome (PCOS) and plays an important role in the pathogenesis of the metabolic, endocrine, and reproductive abnormalities associated with this syndrome. We aimed to summarize the effects of lifestyle changes and anti-obesity pharmacotherapy in patients with PCOS. We reviewed the literature regarding the effects of lifestyle changes and anti-obesity agents on the metabolic and endocrine abnormalities of PCOS. Lifestyle changes, including diet, exercise, and behavioral modification, appear to improve the metabolic and reproductive abnormalities of overweight and obese patients with PCOS. Therefore, lifestyle changes appear to represent the first-line management for all overweight and obese patients with PCOS. However, the optimal composition of diet and the optimal type of exercise in these patients are unknown. Anti-obesity agents that have been studied in PCOS include orlistat, sibutramine, and rimonabant. However, the latter two agents have been withdrawn from the market because of side effects. Long-term studies with orlistat in overweight and obese diabetic patients showed greater weight loss and metabolic and cardiovascular benefits than those achieved with lifestyle changes alone. However, there are limited data on the efficacy of orlistat in women with PCOS. In conclusion, lifestyle changes (diet, exercise and behavioral modification), particularly when combined with anti-obesity agents, exert beneficial effects on the endocrine abnormalities of obese patients with PCOS and improve metabolic parameters. PMID:23625194

Panidis, Dimitrios; Tziomalos, Konstantinos; Papadakis, Efstathios; Vosnakis, Christos; Chatzis, Panagiotis; Katsikis, Ilias

2013-12-01

390

Syndromic Disorders with Short Stature  

PubMed Central

Short stature is one of the major components of many dysmorphic syndromes. Growth failure may be due to a wide variety of mechanisms, either related to the growth hormone (GH)/insulin-like growth factor axis or to underlying unknown pathologies. In this review, the relatively more frequently seen syndromes with short stature (Noonan syndrome, Prader-Willi syndrome, Silver-Russell syndrome and Aarskog-Scott syndrome) were discussed. These disorders are associated with a number of endocrinopathies, as well as with developmental, systemic and behavioral issues. At present, GH therapy is used in most syndromic disorders, although long-term studies evaluating this treatment are insufficient and some controversies exist with regard to GH dose, optimal age to begin therapy and adverse effects. Before starting GH treatment, patients with syndromic disorders should be evaluated extensively. PMID:24637303

??klar, Zeynep; Berbero?lu, Merih

2014-01-01

391

Isolated venlafaxine-induced serotonin syndrome  

Microsoft Academic Search

Serotonin syndrome is a potentially fatal complication of serotonergic drug therapy. Usually, serotonin syndrome occurs with the concomitant use of two serotonergic drugs; this case report describes a patient with a classic presentation of serotonin syndrome induced solely by a venlafaxine overdose. Emergency physicians need to be aware that the serotonin syndrome may occur not only with serotonergic drug combinations

Paul Kolecki

1997-01-01

392

A trial of Scrambler therapy in the treatment of cancer pain syndromes and chronic chemotherapy-induced peripheral neuropathy.  

PubMed

Neuropathic pain is common among cancer patients and often difficult to treat. This study used Scrambler therapy, a patient-specific electrocutaneous nerve stimulation device, to treat cancer patients with pain. Patients received Scrambler therapy for 10 sessions (one daily) over a two-week period. The primary outcome was changed in pain numerical rating scale (NRS) at one month; secondary outcomes were changes in the Brief Pain Inventory and European Organization for Treatment and Cancer QLC-CIPN-20(EORTC CIPN-20), over time. Thirty-nine patients, mean age 56.5 yr, 16 men and 23 women, were treated over an 18-month period for an average of 9.3 days each. The "now" pain scores reduced from 6.6 before treatment to 4.5 at 14 days, 4.6, 4.8, and 4.6 at 1, 2, and 3 months, respectively (p < 0.001). Clinically important and statistically significant improvements were seen in average, least, and worst pain; BPI interference with life scores, and motor and sensory scales on the EORTC CIPN-20. No adverse effects were observed. In this single arm trial, Scrambler therapy appeared to relieve cancer-associated chronic neuropathic pain both acutely and chronically, and provided sustained improvements in many indicators of quality of life. PMID:24143893

Coyne, Patrick J; Wan, Wen; Dodson, Patricia; Swainey, Craig; Smith, Thomas J

2013-12-01

393

An Expanded Trial of Scrambler Therapy in the Treatment of Cancer Pain Syndromes And Chronic Chemotherapy-Induced Peripheral Neuropathy  

PubMed Central

Background Neuropathic pain is common among cancer patients and often difficult to treat. This study used Scrambler Therapy, a patient specific electrocutaneous nerve stimulation device, to treat cancer patient with pain. Methods Patients received Scrambler Therapy for ten sessions (one daily) over a two- week period. The primary outcome was change in pain numeric rating scale (NRS) at 1 month; secondary outcomes were changes in the Brief Pain Inventory and European Organization for Treatment and Cancer QLC-CIPN-20 over time. Results 39 patients, mean age 56.5, 16 men and 23 women, were treated over an 18 month period for an average of 9.3 days each. The “now” pain scores reduced from 6.6 before treatment to 4.5 at 14 days, 4.6, 4.8 and 4.6 at 1, 2 and 3 months. (p<0.001) Clinically important and statistically significant improvements were seen in average, least, and worst pain; BPI interference with life scores, and motor and sensory scales on the EORTC CIPN-20. No adverse effects were observed. Conclusions In this single arm trial, Scrambler therapy appeared to relieve cancer associated chronic neuropathic pain both acutely and chronically, and provided sustained improvements in many indicators of quality of life. PMID:24143893

Coyne, Patrick J; Wan, Wen; Dodson, Patricia A; Swainey, Craig; Smith, Thomas J

2015-01-01

394

Long-term testosterone therapy in hypogonadal men ameliorates elements of the metabolic syndrome: an observational, long-term registry study  

PubMed Central

Aim The goal of this study was to determine if long-term testosterone (T) therapy in men with hypogonadism, henceforth referred to as testosterone deficiency (TD), ameliorates or improves metabolic syndrome (MetS) components. Methods We performed a cumulative registry study of 255 men, aged between 33 and 69 years (mean 58.02 ± 6.30) with subnormal plasma total T levels (mean: 9.93 ± 1.38; range: 5.89–12.13 nmol/l) as well as at least mild symptoms of TD assessed by the Aging Males' symptoms scale. All men received treatment with parenteral T undecanoate 1000 mg (Nebido®, Bayer Pharma, Berlin, Germany), administered at baseline and 6 weeks and thereafter every 12 weeks for up to 60 months. Lipids, glucose, liver enzymes and haemoglobin A1c analyses were carried out in a commercial laboratory. Anthropometric measurements were also made throughout the study period. Results Testosterone therapy restored physiological T levels and resulted in reductions in total cholesterol (TC) [7.29 ± 1.03 to 4.87 ± 0.29 mmol/l (281.58 ± 39.8 to 188.12 ± 11.31 mg/dl)], low-density lipoprotein cholesterol [4.24 ± 1.07 to 2.84 ± 0.92 mmol/l (163.79 ± 41.44 to 109.84 ± 35.41 mg/dl)], triglycerides [3.14 ± 0.58 to 2.16 ± 0.13 mmol/l (276.16 ± 51.32 to 189.78 ± 11.33 mg/dl)] and increased high-density lipoprotein levels [1.45 ± 0.46 to 1.52 ± 0.45 mmol/l (56.17 ± 17.79 to 58.85 ± 17.51 mg/dl)] (p < 0.0001 for all). There were marked reductions in systolic and diastolic blood pressure, blood glucose, haemoglobin A1c, C-reactive protein (6.29 ± 7.96 to 1.03 ± 1.87 U/l), alanine aminotransferase and aspartate aminotransferase (p < 0.0001 for all). Conclusions Long-term T therapy, at physiological levels, ameliorates MetS components. These findings strongly suggest that T therapy in hypogonadal men may prove useful in reducing the risk of cardiometabolic diseases. PMID:24127736

Traish, A M; Haider, A; Doros, G; Saad, F

2014-01-01

395

Central sensitization does not identify patients with carpal tunnel syndrome who are likely to achieve short-term success with physical therapy.  

PubMed

The aim of the current study was to identify whether hyperexcitability of the central nervous system is a prognostic factor for individuals with carpal tunnel syndrome (CTS) likely to experience rapid and clinical self-reported improvement following a physical therapy program including soft tissue mobilization and nerve slider neurodynamic interventions. Women presenting with clinical and electrophysiological findings of CTS were involved in a prospective single-arm trial. Participants underwent a standardized examination and then a physical therapy session. The physical therapy sessions included both soft tissue mobilization directed at the anatomical sites of potential median nerve entrapment and a passive nerve slider neurodynamic technique targeted to the median nerve. Pressure pain thresholds (PPT) over the median, radial and ulnar nerves, C5-C6 zygapophyseal joint, carpal tunnel and tibialis anterior muscle were assessed bilaterally. Additionally, thermal detection and pain thresholds were measured over the carpal tunnel and thenar eminence bilaterally to evaluate central nervous system excitability. Subjects were classified as responders (having achieved a successful outcome) or non-responders based on self-perceived recovery. Variables were entered into a stepwise logistic regression model to determine the most accurate variables for determining prognosis. Data from 72 women were included in the analysis, of which 35 experienced a successful outcome (48.6%). Three variables including PPT over the C5-C6 joint affected side <137 kPa, HPT carpal tunnel affected side <39.6ş and general health >66 points were identified. If 2 out of 3 variables were present (LR + 14.8), the likelihood of success increased from 48.6 to 93.3%. We identified 3 factors that may be associated with a rapid clinical response to both soft tissue mobilization and nerve slider neurodynamic techniques targeted to the median nerve in women presenting with CTS. Our results support that widespread central sensitization may not be present in women with CTS who are likely to achieve a successful outcome with physical therapy. Future studies are now necessary to validate these findings. PMID:20953591

Fernández-de-Las-Peńas, César; Cleland, Joshua A; Ortega-Santiago, Ricardo; de-la-Llave-Rincon, Ana Isabel; Martínez-Perez, Almudena; Pareja, Juan A

2010-11-01

396

Persistence and selection of an expanded B-cell clone in the setting of rituximab therapy for Sjögren’s syndrome  

PubMed Central

Introduction Subjects with primary Sjögren’s syndrome (SjS) have an increased risk of developing B-cell lymphoma and may harbor monoclonal B-cell expansions in the peripheral blood. Expanded B-cell clones could be pathogenic, and their persistence could exacerbate disease or predispose toward the development of lymphoma. Therapy with anti-CD20 (rituximab) has the potential to eliminate expanded B-cell clones and thereby potentially ameliorate disease. This study was undertaken to identify and track expanded B-cell clones in the blood of subjects with primary SjS who were treated with rituximab. Methods To determine whether circulating B-cell clones in subjects with primary SjS emerge or remain after B cell-depleting therapy with rituximab, we studied the antibody heavy-chain repertoire. We performed single-memory B-cell and plasmablast sorting and antibody heavy-chain sequencing in six rituximab-treated SjS subjects over the course of a 1-year follow-up period. Results Expanded B-cell clones were identified in four out of the six rituximab-treated SjS subjects, based upon the independent amplification of sequences with identical or highly similar VH, DH, and JH gene segments. We identified one SjS subject with a large expanded B-cell clone that was present prior to therapy and persisted after therapy. Somatic mutations in the clone were numerous but did not increase in frequency over the course of the 1-year follow-up, suggesting that the clone had been present for a long period of time. Intriguingly, a majority of the somatic mutations in the clone were silent, suggesting that the clone was under chronic negative selection. Conclusions For some subjects with primary SjS, these data show that (a) expanded B-cell clones are readily identified in the peripheral blood, (b) some clones are not eliminated by rituximab, and (c) persistent clones may be under chronic negative selection or may not be antigen-driven. The analysis of sequence variation among members of an expanded clone may provide a novel means of measuring the chronicity and selection of expanded B-cell populations in humans. PMID:24517398

2014-01-01

397

Improvement of cardiac function by short-term enzyme replacement therapy in a murine model of cardiomyopathy associated with Hunter syndrome evaluated by serial echocardiography with speckle tracking 2-D strain analysis.  

PubMed

Cardiac systolic function is significantly decreased in a proportion of patients with Hunter syndrome. This study was performed to evaluate the change in myocardial function associated with enzyme replacement therapy (ERT) in a mouse model of cardiomyopathy associated with Hunter syndrome. Thirty 9-week-old iduronate-2-sulfatase (IDS) knockout mice received either intravenous injection of human recombinant IDS (ERT group, N=15) or saline (control group, N=15) for 5 weeks. Echocardiography was performed at baseline and after treatment. Echocardiographic parameters of left ventricular (LV) systolic function and 2-dimensional radial and circumferential strain were assessed. At follow-up, there was a significant increase in LV fractional shortening and radial and circumferential strain in the ERT group only. Notable myocardial fibrosis was observed in the control group only. In the murine model of Hunter syndrome, ERT exerts beneficial effects on cardiac function, which can be evaluated by serial echocardiographic evaluation including 2-dimensional strain analysis. PMID:24836711

Lee, Sang-Chol; Lee, Jieun; Jin, Dong-Kyu; Kim, Jung-Sun; Jeon, Eun-Seok; Kwun, Young Hee; Chang, Mi Sun; Ko, Ah-ra; Yook, Yeon Joo; Sohn, Young Bae

2014-07-01

398

Phase I study protocol for ex vivo lentiviral gene therapy for the inherited skin disease, Netherton syndrome.  

PubMed

Netherton syndrome (NS) is a serious inherited skin disorder caused by mutations in the serine protease inhibitor Kazal type 5 gene (SPINK5), which encodes for a serine protease inhibitor lymphoepithelial Kazal type-related inhibitor (LEKTI). Patients with NS have defective keratinization, hair shaft defects, recurrent infections, atopy, and a predisposition to skin malignancies. Historically, 1 in 10 infants has died before their first birthday. Currently, there are no proven treatments to cure this condition. A SIN-lentiviral vector encoding the codon-optimized SPINK5 gene under the control of a 572 bp element derived from the human involucrin promoter can confer compartment-specific LEKTI expression in NS keratinocytes with restoration of normal skin architecture. Here we detail a study protocol for a phase I trial for feasibility and safety evaluations of autologous epidermal sheets generated from ex vivo gene-corrected keratinocyte stem cells, which will be grafted onto patients with mutation-proven NS. PMID:24329107

Di, Wei-Li; Mellerio, Jemima E; Bernadis, Catina; Harper, John; Abdul-Wahab, Alya; Ghani, Sumera; Chan, Lucas; Martinez-Queipo, Magdalena; Hara, Havinder; McNicol, Anne-Marie; Farzaneh, Farzin; McGrath, John; Thrasher, Adrian; Qasim, Waseem

2013-12-01

399

Fatigue In Teenagers on the interNET - The FITNET Trial. A randomized clinical trial of web-based cognitive behavioural therapy for adolescents with chronic fatigue syndrome: study protocol. [ISRCTN59878666  

Microsoft Academic Search

Background  Chronic Fatigue Syndrome (CFS) is increasingly recognized as a cause of disability and inactivity in adolescents in the Netherlands.\\u000a CFS is characterized by unexplained fatigue lasting more than 6 months. Cognitive Behavioural Therapy (CBT) has proven to\\u000a be effective. However, CBT availability for adolescents with CFS is limited and requires special therapeutic skills not always\\u000a readily available. An alternative to

Sanne L Nijhof; Gijs Bleijenberg; Cuno SPM Uiterwaal; Jan LL Kimpen; Elise M van de Putte

2011-01-01

400

Increased leptin supply to hypothalamus by gene therapy confers life-long benefits on energy homeostasis, disease cluster of metabolic syndrome- diabetes type 1 and 2, dyslipidemia and cardiovascular ailments- and bone remodeling  

Microsoft Academic Search

Leptin insufficiency in the hypothalamus is causally linked to increased fat accrual, diseases of the metabolic syndrome,\\u000a skeletal abnormalities and shortened life-span. We show that leptin sufficiency attained with hypothalamic leptin gene therapy\\u000a (i) suppressed food intake, the agerelated and energy enriched diet-induced fat accrual, and dyslipidemia, (ii) attenuated\\u000a episodic insulin secretion and enhanced insulin sensitivity, (iii) enhanced glucose tolerance

Satya P. Kalra

401

The white dot syndromes.  

PubMed

The white dot syndromes are a heterogeneous group of rare inflammatory disorders affecting the retina, the retinal pigment epithelium, and the choroid. Not all of these diseases actually cause white dots, but they all have unique lesions in the fundus. We describe acute posterior multifocal placoid pigment epitheliopathy, serpiginous choroiditis, birdshot chorioretinopathy, multifocal choroiditis with panuveitis, diffuse subretinal fibrosis syndrome, punctate inner choroidopathy, multiple evanescent white dot syndrome, and diffuse unilateral subacute neuroretinitis as the white dot syndromes in this review. Some of these conditions share an association with systemic infectious diseases. In addition, treatment of these diseases is similar. Some can be treated with immunosuppressive therapy. Other treatment options include laser photocoagulation, topical or systemic steroid therapy, photodynamic therapy, and, most recently, anti-vascular endothelial growth factor agents. The new development in treatment may alter the visual prognosis of the patients, leading to a better outcome in visual acuity. PMID:17999832

Matsumoto, Yoko; Haen, Sebastian P; Spaide, Richard F

2007-01-01

402

Paraneoplastic neurological syndromes  

PubMed Central

Paraneoplastic neurological syndromes are immune-mediated erroneous attacks on the central or peripheral nervous systems, or both, directed originally against the tumour itself. They have been known for more than 40 years, but recently the discovery of new subgroups of paraneoplastic encephalitis syndromes with a remarkably good response to immune therapy has ignited new clinical and scientific interest. Knowledge of these subgroups and their associated autoantibodies is important in therapeutic decision-making. However, the abundance of new autoantibodies and syndromes can be confusing. This review paper summarizes current knowledge and new developments in the field of paraneoplastic neurological syndromes, their classification, pathophysiology and treatment. PMID:23937626

Leypoldt, F; Wandinger, K-P

2014-01-01

403

Hypertension due to antiangiogenic cancer therapy with vascular endothelial growth factor inhibitors: understanding and managing a new syndrome.  

PubMed

Novel antiangiogenic cancer therapies, particularly agents that block vascular endothelial growth factor (VEGF) signalling, have improved outcomes in patients with cancers and are now used as first-line therapies for some tumours. However, with VEGF inhibitors (VEGFIs) are new complications, particularly hypertension. VEGFI-induced hypertension is a dose-dependent phenomenon due to on-target effects rather than off-target effects. Increased blood pressure occurs in almost 100% of patients who take VEGFIs, with a subset who develop severe hypertension. Molecular mechanisms underlying VEGFI-induced hypertension are unclear, but endothelial dysfunction and increased vascular resistance, due to impaired nitric oxide signalling, reduced prostacyclin production, endothelin-1 (ET-1) upregulation, oxidative stress, and rarefaction have been implicated. Treatment of hypertension should be aimed at reducing the risk of short-term morbidity associated with hypertension while maintaining effective dosing of antiangiogenic therapy for optimal cancer treatment. Although specific guidelines are not yet available for the management of VEGFI-induced hypertension, angiotensin-converting enzyme inhibitors and dihydropyridine calcium channel blockers are commonly used. Severe hypertension might require reduction of VEGFI dosing, or in some cases, interruption of treatment. As more potent VEGFIs are developed and as more cancer patients are treated with VEGFIs, the burden of hypertension toxicity will increase. This will be further compounded as the use of antiangiogenic drugs broadens to include older patients and those with pre-existing cardiovascular disease. Here we focus on VEGF as a target for antiangiogenesis and how this affects increased blood pressure. Putative mechanisms underlying VEGFI-induced hypertension are highlighted and therapeutic strategies to manage such hypertension are discussed. PMID:24786444

Small, Heather Yvonne; Montezano, Augusto C; Rios, Francisco J; Savoia, Carmine; Touyz, Rhian M

2014-05-01

404

Phase I-II Clinical Trial of Oxaliplatin, Fludarabine, Cytarabine, and Rituximab Therapy in Aggressive, Relapsed/Refractory Chronic Lymphocytic Leukemia or Richter’s Syndrome  

PubMed Central

Introduction To improve outcomes of patients with Richter's syndrome (RS) and relapsed/refractory chronic lymphocytic leukemia (CLL), we modified the oxaliplatin and cytarabine doses of the oxaliplatin, fludarabine, cytarabine, and rituximab (OFAR1) regimen for this phase I-II study (OFAR2). Patients and Methods OFAR2 consisted of oxaliplatin at 30 mg/m2 on days (D) 1–4, fludarabine at 30 mg/m2, cytarabine at 0.5 g/m2, rituximab at 375 mg/m2 (D3), and pelfigrastim at 6 mg (D6). Fludarabine and cytarabine were given on D2–3 (cohort 1), D2–4 (cohort 2), or D2–5 (cohort 3) every 4 weeks. Phase II followed the "3+3" design of phase I. Results The 102 patients (CLL, 67; RS, 35) treated had heavily pretreated, high-risk disease. Twelve patients were treated in phase I; cohort 2 was the phase II recommended dose. The most common toxicities were hematologic. Response rates (phase II) were 38.7% for RS (CR, 6.5%) and 50.8% for relapsed/refractory CLL (CR, 4.6%). The median survival durations were 6.6 (RS) and 20.6 (CLL) months. Among 9 patients who underwent allogeneic stem cell transplantation (SCT) as post-remission therapy, none has died (median follow-up, 15.9 months). Conclusion OFAR2 had significant antileukemic activity in RS and relapsed/refractory CLL. Patients undergoing SCT as post-remission therapy had favorable outcomes PMID:23810245

Tsimberidou, Apostolia M.; Wierda, William G.; Wen, Sijin; Plunkett, William; O’Brien, Susan; Kipps, Thomas J.; Jones, Jeffrey A.; Badoux, Xavier; Kantarjian, Hagop; Keating, Michael J.

2014-01-01

405

Randomized Multicenter Clinical Trial of Myofascial Physical Therapy in Women with Interstitial Cystitis/Painful Bladder Syndrome (IC/PBS) and Pelvic Floor Tenderness  

PubMed Central

Objectives To determine the efficacy and safety of pelvic floor Myofascial Physical Therapy (MPT) in women with newly-symptomatic IC/PBS, as compared to Global Therapeutic Massage (GTM). Materials and Methods A randomized controlled trial of 10 scheduled treatments of MPT vs. GTM was performed at 11 clinical centers located in North America. We recruited women with IC/PBS with demonstrable pelvic floor tenderness on physical examination and a limitation of no more than 3 years symptom duration. The primary outcome was the proportion of responders defined as ‘moderately improved’ or ‘markedly improved’ in overall symptoms compared to baseline on a 7-point scale Global Response Assessment (GRA). Secondary outcomes included ratings for pain, urgency, frequency; the O'Leary-Sant IC Symptom and Problem Index (ICSI/ICPI) and reports of adverse events. We compared response rates between treatment arms using the exact conditional version of the Mantel-Haenszel test to control for clustering by clinical center. For secondary efficacy outcomes, cross-sectional descriptive statistics and changes from baseline were calculated. Results Eighty-one women randomized to the two treatment groups had similar symptoms at baseline. The GRA response rate was 26% in the GTM group and 59% in the MPT group (p=0.0012). Pain, urgency, and frequency ratings and in ICSI/ICPI decreased in both groups during follow-up and were not significantly different between the groups. Pain was the most common adverse event, occurring at similar rates in both groups. There were no serious adverse events reported. Conclusions A significantly higher proportion of women with IC/PBS reponded to treatment with MPT than with GTM. MPT may be a beneficial therapy in women with this syndrome. PMID:22503015

FitzGerald, MP; Payne, CK; Lukacz, ES; Yang, CC; Peters, KM; Chai, TC; Nickel, JC; Hanno, PM; Kreder, KJ; Burks, DA; Mayer, R; Kotarinos, R; Fortman, C; Allen, TM; Fraser, L; Mason-Cover, M; Furey, C; Odabachian, L; Sanfield, A; Chu, J; Huestis, K; Tata, GE; Dugan, N; Sheth, H; Bewyer, K; Anaeme, A; Newton, K; Featherstone, W; Halle-Podell, R; Cen, L; Landis, JR; Propert, KJ; Foster, HE; Kusek, JW; Nyberg, *LM

2012-01-01

406

The Efficacy of Shugan Jianpi Zhixie Therapy for Diarrhea-Predominant Irritable Bowel Syndrome: A Meta-Analysis of Randomized, Double-Blind, Placebo-Controlled Trials  

PubMed Central

Background Shugan Jianpi Zhixie therapy (SJZT) has been widely used to treat diarrhea-predominant irritable bowel syndrome (IBS-D), but the results are still controversial. A meta-analysis of randomized, double-blind, placebo-controlled trials was performed to assess the efficacy and tolerability of SJZT for IBS-D. Methods The MEDLINE, EMBASE, Cochrane Library, the China National Knowledge Infrastructure database, the Chinese Biomedical Literature database and the Wanfang database were searched up to June 2014 with no language restrictions. Summary estimates, including 95% confidence intervals (CI), were calculated for global symptom improvement, abdominal pain improvement, and Symptom Severity Scale (BSS) score. Results Seven trials (N=954) were included. The overall risk of bias assessment was low. SJZT showed significant improvement for global symptom compared to placebo (RR 1.61; 95% CI 1.24, 2.10; P =0.0004; therapeutic gain = 33.0%; number needed to treat (NNT) = 3.0). SJZT was significantly more likely to reduce overall BSS score (SMD –0.67; 95% CI –0.94, –0.40; P < 0.00001) and improve abdominal pain (RR 4.34; 95% CI 2.64, 7.14; P < 0.00001) than placebo. The adverse events of SJZT were no different from those of placebo. Conclusions This meta-analysis suggests that SJZT is an effective and safe therapy option for patients with IBS-D. However, due to the high clinical heterogeneity and small sample size of the included trials, further standardized preparation, large-scale and rigorously designed trials are needed. PMID:25853241

Sun, Xiaomin; Tang, Yang; Cheng, Jingru; Wang, Tian; Li, Fei; Kuang, Yuxiang; Luo, Ren; Zhao, Xiaoshan

2015-01-01

407

Leptin replacement therapy for the treatment of non-HAART associated lipodystrophy syndromes: a meta-analysis into the effects of leptin on metabolic and hepatic endpoints.  

PubMed

The clinical manifestations of lipodystrophy syndromes (LS) are hypoleptinemia, hyperglycemia, insulin resistance, dyslipidemia and hepatic steatosis. Leptin replacement therapy (LRT) is effective at improving these pathologies. Currently, there are no data compiling the evidence from the literature, and demonstrating the effect of LRT in LS patients. A systematic review of the MEDLINE and Cochrane Library databases was conducted to identify studies assessing the effect of LRT on metabolic and hepatic endpoints in patients with LS not associated with highly active antiretroviral therapy (HAART) use. Standardized mean differences (SMD) and 95% confidence intervals of pooled results were calculated for overall changes in glucose homeostasis, lipid profile, and hepatic physiology, using an inverse-variance random-effects model. After screening, 12 studies were included for review. Meta-analysis of results from 226 patients showed that LRT decreased fasting glucose [0.75 SMD units (range 0.36-1.13), p=0.0001], HbA1c [0.49 (0.17-0.81), p=0.003], triglycerides [1.00 (0.69-1.31), p<0.00001], total cholesterol [0.62 (0.21-1.02), p=0.003], liver volume [1.06 (0.51-1.61), p=0.0002] and AST [0.41 (0.10-0.73) p=0.01]. In patients with non-HAART LS, LRT improves the outcome of several metabolic and hepatic parameters. Studies were limited by small populations and therefore large prospective trials are needed to validate these findings. PMID:25465598

Rodríguez, Alexander J; Neeman, Teresa; Giles, Aaron G; Mastronardi, Claudio A; Paz Filho, Gilberto

2014-11-01

408

LA COOPERATION D'ONCOGENES DANS LA TRANSFORMATION CELLULAIRE Samarut J. 1.(J!J(Jrll{)in! (Ir liinloyicMol(!clJlain! el C(!Julairc, [cole Normale 5lJpl!ricLJrc  

E-print Network

LA COOPERATION D'ONCOGENES DANS LA TRANSFORMATION CELLULAIRE Samarut J. 1.(J!J(JrĂŤll{)in! (Ir'HEPATOCYTES DE RAT PAR TRANSFECTION IN VITRO OU IN VIVO A L'AIDE DE PLASMIDES ONCOGENES (Ha-ras ou BPV) VEHICULES l'AR DES LIPOSOMES : EXPRESSION D'UN ONCOGENE ET DE PHENOTYPES METABOLIQUES DU FOIE. Fischbach M

Paris-Sud XI, Université de

409

When is iron overload deleterious, and when and how should iron chelation therapy be administered in myelodysplastic syndromes?  

PubMed

Iron overload in MDS starts even before patients become red-blood cell transfusion dependent, because disease-associated ineffective erythropoiesis suppresses hepcidin production in the liver and thus causes unrestrained iron absorption in the duodenum. However, the main cause of iron overload is regular transfusion therapy, which in MDS is associated with a risk of unclear magnitude for iron-related complications. Iron deposition in tissues can now be detected with non-invasive techniques such as T2* MRI. Iron toxicity in MDS may not only depend on the degree of tissue iron accumulation but also on the extent of chronic exposure to non-transferrin-bound iron (NTBI), including labile plasma iron (LPI) and intracellular labile iron pools, which increase the level of oxidative stress. Iron chelation therapy (ICT) can rapidly lower NTBI and LPI and more slowly mobilizes tissue iron stores. Further studies, including the ongoing TELESTO controlled trial, will more clearly define the role of ICT in MDS, including any effect on specific morbidities or mortality in the MDS setting. PMID:24507819

Steensma, David P; Gattermann, Norbert

2013-12-01

410

Characteristics of novel myeloid precursor cell line, PC-MDS, established from a bone marrow of the patient with therapy-related myelodysplastic syndrome.  

PubMed

We report on characteristics of the first human cell line, PC-MDS, derived from a bone marrow of a patient with therapy-related myelodysplastic syndrome (t-MDS) who had no overt post-MDS leukemia. Classic cytology analyses, immunophenotyping, cytogenetic and molecular genetic procedures were used for characterization of the cell line. PC-MDS cells are positive for the expression of CD13, CD15, CD30, CD33, and CD45 antigen. Positive cytochemical staining and immunophenotype analyses indicated that PC-MDS cells have some characteristics of the early myeloid precursor cell. The karyotype analysis of PC-MDS cell line revealed various numerical and structural changes including those typically associated with t-MDS: del(5)(q13)[7], der(5)t(5;11)(p11;q11)[13], -7[6], del(7)(q31)[2], +20[3], -20[4]. Evaluation of methylation status in a promoter region of p15, p16 and MGMT genes showed biallelic hypermethylation pattern of 5' promoter region only in MGMT gene. PC-MDS is the first t-MDS derived cell line, and based on its immunological, cytogenetic and molecular characterization could be a new tool in evaluation of complex biology of MDS and a model for methylation studies. PMID:17350682

Bogdanovi?, Gordana; Jurisi?, Vladimir; Kraguljac, Nada; Mrdjanovi?, Jasminka; Jakimov, Dimitar; Krtolica, Koviljka; Krajnovi?, Milena; Magi?, Zvonko; Stojiljkovi?, Bratislav; Andrijevi?, Ljiljana; Srdi?, Tatjana; Balti?, Mirjana; Popovi?, Stevan

2007-08-01

411

Long-term therapy of a patient with summerskill-walshe-tygstrup syndrome by applying prometheus® liver dialysis: a case report.  

PubMed

Summerskill-Walshe-Tygstrup syndrome is a rare benign chronic liver disease characterized by recurring cholestasis with jaundice and severe pruritus. Due to insufficient conservative treatment, liver dialysis by Prometheus(®) was applied to a 45-year-old female patient with resistant pruritus. Initially, other possible liver diseases were excluded and the patient was treated symptomatically since the diagnosis of Summerskill-Walshe-Tygstrup was stated in 1998. As conservative and endoscopic methods progressively failed to relieve the patient's suffering, Prometheus(®) liver dialysis was performed regularly since 2006 at 3-month intervals and successfully led to a decrease in the patient's symptoms. Cholestatic liver enzymes and also serum bile acids could be lowered significantly from an average of 22.5 ± 2.7 to 7.3 ± 1.7 µmol/l. Consequently, Prometheus(®) liver dialysis may be a beneficial option for patients with benign recurrent intrahepatic cholestasis suffering from therapy-resistant symptoms and may be used as well as other extracorporeal liver support devices which have already been reported to improve cholestatic pruritus. PMID:22949896

Walensi, Mikolaj; Canbay, Ali; Witzke, Oliver; Gerken, Guido; Kahraman, Alisan

2012-05-01

412

Long-Term Therapy of a Patient with Summerskill-Walshe-Tygstrup Syndrome by Applying Prometheus® Liver Dialysis: A Case Report  

PubMed Central

Summerskill-Walshe-Tygstrup syndrome is a rare benign chronic liver disease characterized by recurring cholestasis with jaundice and severe pruritus. Due to insufficient conservative treatment, liver dialysis by Prometheus® was applied to a 45-year-old female patient with resistant pruritus. Initially, other possible liver diseases were excluded and the patient was treated symptomatically since the diagnosis of Summerskill-Walshe-Tygstrup was stated in 1998. As conservative and endoscopic methods progressively failed to relieve the patient's suffering, Prometheus® liver dialysis was performed regularly since 2006 at 3-month intervals and successfully led to a decrease in the patient's symptoms. Cholestatic liver enzymes and also serum bile acids could be lowered significantly from an average of 22.5 ± 2.7 to 7.3 ± 1.7 µmol/l. Consequently, Prometheus® liver dialysis may be a beneficial option for patients with benign recurrent intrahepatic cholestasis suffering from therapy-resistant symptoms and may be used as well as other extracorporeal liver support devices which have already been reported to improve cholestatic pruritus. PMID:22949896

Walensi, Mikolaj; Canbay, Ali; Witzke, Oliver; Gerken, Guido; Kahraman, Alisan

2012-01-01

413

Analysis of the cost-effectiveness of surfactant treatment (Curosurf®) in respiratory distress syndrome therapy in preterm infants: early treatment compared to late treatment  

PubMed Central

Background The best criteria for surfactant treatment in the perinatal period are unknown and this makes it of interest to consider the possible economic implications of lessening the use of more restrictive criteria. Objective The objective of this study is the evaluation of the costs of respiratory care for preterm infants with Respiratory Distress Syndrome (RDS) treated with "early rescue" surfactant compared to a "late rescue" strategy. Methods The study was carried out applying the costs of materials used, of staff and pharmacological therapy calculated in the Neonatal Intensive Care Unit (NICU) of an Italian hospital to the Verder et al. study (Pediatrics 1999) clinical data. Results The cost for patients treated with early strategy was slightly lower than for patients treated with late strategy (Euro 4,901.70 vs. Euro 4,960.07). The cost of treatment with surfactant was greater in the early group (Euro 458.49 vs. Euro 311.74), but this was compensated by the greater cost of treatment with Mechanical Ventilation (MV) in the late group (respectively Euro 108.85 vs. Euro 259.25). Conclusions The cost-effectiveness analysis performed in this study shows how early treatment with surfactant in preterm infants with RDS, as well as being clinically more effective, is associated with a slightly lower cost. PMID:24886906

2014-01-01

414

Impact of Dual Antiplatelet Therapy with Proton Pump Inhibitors on the Outcome of Patients with Acute Coronary Syndrome Undergoing Drug-Eluting Stent Implantation  

PubMed Central

This study aimed to assess if proton pump inhibitors (PPIs) may reduce the effectiveness of clopidogrel, than H2 antagonist (anti-H2) in order to determine rehospitalization for acute coronary syndrome (re-ACS), target vessel revascularization (TVR) and cardiac death. This case-control study included 176 patients with ACS undergoing angioplasty (PCI) with drug-eluting stent implantation. The population was divided into two groups: PPI group (n = 121) consisting of patients receiving at discharge dual antiplatelet therapy (DAT) plus PPI and anti-H2 group (n = 55), consisting of patients receiving at discharge DAT?+?H2 receptor antagonist (H2RA). In a followup of 36 months the prevalence of ACS event (P = 0.014), TVR (P = 0.031) was higher in the PPI group than in the anti-H2 group; instead there was no statistically significant difference between groups for death. The variables independently associated with ACS were the diabetes, omeprazole, and esomeprazole; instead the variables independently associated with TVR were only omeprazole. Our data shows that the use of omeprazole and esomeprazole, with clopidogrel, is associated with increased risk of adverse outcomes after PCI with drug-eluting stent implantation. PMID:22792485

Macaione, Francesca; Montaina, Carla; Evola, Salvatore; Novo, Giuseppina; Novo, Salvatore

2012-01-01

415

Sotos syndrome  

PubMed Central

Sotos syndrome is an overgrowth condition characterized by cardinal features including excessive growth during childhood, macrocephaly, distinctive facial gestalt and various degrees of learning difficulty, and associated with variable minor features. The exact prevalence remains unknown but hundreds of cases have been reported. The diagnosis is usually suspected after birth because of excessive height and occipitofrontal circumference (OFC), advanced bone age, neonatal complications including hypotonia and feeding difficulties, and facial gestalt. Other inconstant clinical abnormalities include scoliosis, cardiac and genitourinary anomalies, seizures and brisk deep tendon reflexes. Variable delays in cognitive and motor development are also observed. The syndrome may also be associated with an increased risk of tumors. Mutations and deletions of the NSD1 gene (located at chromosome 5q35 and coding for a histone methyltransferase implicated in transcriptional regulation) are responsible for more than 75% of cases. FISH analysis, MLPA or multiplex quantitative PCR allow the dete