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Harish Petnikota, Vrisha Madhuri, Sangeet Gangadharan, Indira Agarwal, Belavendra Antonisamy
BACKGROUND: Muscular dystrophies are inherited myogenic disorders characterized by progressive muscle wasting and weakness of variable distribution and severity. They are a heterogeneous group characterized by variable degree of skeletal and cardiac muscle involvement. The most common and the most severe form of muscular dystrophy is DMD. Currently, there is no curative treatment for muscular dystrophies. Several drugs have been studied to retard the progression of the muscle weakness...
September 2016: Indian Journal of Orthopaedics
Robert C Griggs, J Phillip Miller, Cheryl R Greenberg, Darcy L Fehlings, Alan Pestronk, Jerry R Mendell, Richard T Moxley, Wendy King, John T Kissel, Valerie Cwik, Michel Vanasse, Julaine M Florence, Shree Pandya, Jordan S Dubow, James M Meyer
OBJECTIVE: To assess safety and efficacy of deflazacort (DFZ) and prednisone (PRED) vs placebo in Duchenne muscular dystrophy (DMD). METHODS: This phase III, double-blind, randomized, placebo-controlled, multicenter study evaluated muscle strength among 196 boys aged 5-15 years with DMD during a 52-week period. In phase 1, participants were randomly assigned to receive treatment with DFZ 0.9 mg/kg/d, DFZ 1.2 mg/kg/d, PRED 0.75 mg/kg/d, or placebo for 12 weeks. In phase 2, placebo participants were randomly assigned to 1 of the 3 active treatment groups...
August 26, 2016: Neurology
Chet R Villa, Ahmad Kaddourah, Jacob Mathew, Thomas D Ryan, Brenda L Wong, Stuart L Goldstein, John L Jefferies
Patients with Duchenne muscular dystrophy (DMD) develop dilated cardiomyopathy and are at risk for kidney injury. Creatinine based estimated glomerular filtration rate (eGFR) is limited by low muscle mass with low serum creatinine levels in DMD. We assessed the relationship between cardiac function, modified Schwartz eGFR and cystatin C eGFR in patients with DMD. Ninety-three patients with DMD were screened for renal dysfunction in an outpatient neuromuscular clinic. Patients with new nephrotoxic medications, recent hospitalization or decompensated heart failure were excluded from the analysis...
October 2016: Neuromuscular Disorders: NMD
Susan Sienko, Cathleen Buckon, Eileen Fowler, Anita Bagley, Loretta Staudt, Mitell Sison-Williamson, Kathy Zebracki, Craig M McDonald, Michael Sussman
The aim of this study was to determine whether prednisone and deflazacort play a different role in child behavior and perceived health related psychosocial quality of life in ambulant boys with Duchenne Muscular Dystrophy. As part of a prospective natural-history study, parents of sixty-seven ambulant boys with DMD (27 taking prednisone, 15 taking deflazacort, 25 were steroid naïve) completed the Child Behavior Checklist (CBCL) for assessment of behavioral, emotional and social problems and both parents and boys with DMD completed the PedsQL™4...
2016: PLoS Currents
Cathleen Buckon, Susan Sienko, Anita Bagley, Mitell Sison-Williamson, Eileen Fowler, Loretta Staudt, Kent Heberer, Craig M McDonald, Michael Sussman
BACKGROUND: In the absence of a curative treatment for Duchenne Muscular Dystrophy (DMD), corticosteroid therapy (prednisone, deflazacort) has been adopted as the standard of care, as it slows the progression of muscle weakness and enables longer retention of functional mobility. The ongoing development of novel pharmacological agents that target the genetic defect underlying DMD offer hope for a significant alteration in disease progression; however, substantiation of therapeutic efficacy has proved challenging...
2016: PLoS Currents
Amardeep Singh, Emily K Schaeffer, Christopher W Reilly
BACKGROUND: Corticosteroids are widely used in the management of patients with Duchenne muscular dystrophy (DMD). They improve quality of life in these patients by prolonging ambulation and preserving cardiorespiratory status. However, corticosteroid treatment is associated with a decrease in bone mineral density (BMD) and an increased risk of vertebral fractures (VF). The purpose of this study was to investigate the prevalence of VF in patients with DMD undergoing long-term treatment with the corticosteroid deflazacort...
June 18, 2016: Journal of Pediatric Orthopedics
Leonardo Bianchi, Katharina Hansel, Elettra Antonelli, Veronica Bellini, Luigi Rigano, Luca Stingeni
No abstract text is available yet for this article.
July 2016: Contact Dermatitis
Emma Matthews, Ruth Brassington, Thierry Kuntzer, Fatima Jichi, Adnan Y Manzur
BACKGROUND: Duchenne muscular dystrophy (DMD) is the most common muscular dystrophy of childhood. Untreated, this incurable disease, which has an X-linked recessive inheritance, is characterised by muscle wasting and loss of walking ability, leading to complete wheelchair dependence by 13 years of age. Prolongation of walking is a major aim of treatment. Evidence from randomised controlled trials (RCTs) indicates that corticosteroids significantly improve muscle strength and function in boys with DMD in the short term (six months), and strength at two years (two-year data on function are very limited)...
2016: Cochrane Database of Systematic Reviews
Molly M Lamb, Nancy A West, Lijing Ouyang, Michele Yang, David Weitzenkamp, Katherine James, Emma Ciafaloni, Shree Pandya, Carolyn DiGuiseppi
OBJECTIVES: To evaluate growth patterns of ambulatory males with Duchenne muscular dystrophy (DMD) treated with corticosteroids compared with ambulatory, steroid-naïve males with DMD and age-matched unaffected general-population males and to test associations between growth and steroid treatment patterns among treated males. STUDY DESIGN: Using data from the Muscular Dystrophy Surveillance, Tracking, and Research Network, we identified a total of 1768 height, 2246 weight, and 1755 body mass index (BMI) measurements between age 2 and 12 years for 324 ambulatory males who were treated with corticosteroids for at least 6 months...
June 2016: Journal of Pediatrics
Eun Chae Lee, Geun A Kim, Ja Wook Koo
Toxic epidermal necrolysis (TEN) is a drug-related fatal disease. Extensive necrosis of the epidermis can lead to serious complications. This report describes two cases of TEN, associated with deflazacort (DFZ), in two boys, aged 4 years and 14 years, with nephrotic syndrome (NS). The 14-year-old male teenager received DFZ following NS relapse. After 17 days, pruritic papules appeared on the lower extremities. Another case involved a 4-year-old boy receiving DFZ and enalapril. After a 41-day DFZ treatment period, erythematous papules appeared on the palms and soles...
December 2014: Kidney Research and Clinical Practice
David Gloss, Richard T Moxley, Stephen Ashwal, Maryam Oskoui
OBJECTIVE: To update the 2005 American Academy of Neurology (AAN) guideline on corticosteroid treatment of Duchenne muscular dystrophy (DMD). METHODS: We systematically reviewed the literature from January 2004 to July 2014 using the AAN classification scheme for therapeutic articles and predicated recommendations on the strength of the evidence. RESULTS: Thirty-four studies met inclusion criteria. RECOMMENDATIONS: In children with DMD, prednisone should be offered for improving strength (Level B) and pulmonary function (Level B)...
February 2, 2016: Neurology
María Rico, Javier Villafani, Alberto Tuñón, Valentín Mateos, Pedro Oliva-Nacarino
BACKGROUND: Chronic lymphocytic inflammation with pontine perivascular enhancement responsive to steroids (CLIPPERS) is a recently described inflammatory disease of the central nervous system, distinguished by brainstem- and spinal cord-centered lesions with a characteristic contrast enhancement on MRI, a lymphocytic perivascular infiltrate on pathological exam, and a dramatic response to and dependence on steroids therapy. Since its initial description in 2010, different glucocorticoid-sparing agents, mostly immunosuppressant drugs, have been used to minimize the dosage, but these therapies also carry the risk of important secondary effects...
2016: American Journal of Case Reports
Nicolas Brouilly, Claire Lecroisey, Edwige Martin, Laura Pierson, Marie-Christine Mariol, Hiroshi Qadota, Michel Labouesse, Nathalie Streichenberger, Nicole Mounier, Kathrin Gieseler
Duchenne muscular dystrophy (DMD) is a genetic disease characterized by progressive muscle degeneration due to mutations in the dystrophin gene. In spite of great advances in the design of curative treatments, most patients currently receive palliative therapies with steroid molecules such as prednisone or deflazacort thought to act through their immunosuppressive properties. These molecules only slightly slow down the progression of the disease and lead to severe side effects. Fundamental research is still needed to reveal the mechanisms involved in the disease that could be exploited as therapeutic targets...
November 15, 2015: Human Molecular Genetics
Vera Bernardino, Pedro Mendes-Bastos, Ana Rodrigues, Nuno Riso
We report a case of a 65-year-old man with systemic lupus erythematosus (SLE) and antiphospholipid syndrome, presenting palpable purpuric lesions, necrotic blisters and swelling ankles, after a previous tracheobronchitis episode. Laboratory data were remarkable for mild proteinuria and imaging studies were normal. A skin biopsy showed IgA deposits on superficial dermal capillaries and IgA vasculitis (IgAV) (former Henoch-Schönlein purpura) was assumed. The patient was treated with colchicine, deflazacort and azathioprine, but as a regression in the purpuric lesions was noted, a decline in renal function was detected...
2015: BMJ Case Reports
Luca Bello, Heather Gordish-Dressman, Lauren P Morgenroth, Erik K Henricson, Tina Duong, Eric P Hoffman, Avital Cnaan, Craig M McDonald
OBJECTIVE: We aimed to perform an observational study of age at loss of independent ambulation (LoA) and side-effect profiles associated with different glucocorticoid corticosteroid (GC) regimens in Duchenne muscular dystrophy (DMD). METHODS: We studied 340 participants in the Cooperative International Neuromuscular Research Group Duchenne Natural History Study (CINRG-DNHS). LoA was defined as continuous wheelchair use. Effects of prednisone or prednisolone (PRED)/deflazacort (DFZ), administration frequency, and dose were analyzed by time-varying Cox regression...
September 22, 2015: Neurology
Ravish Singhal, Sadbhavna Pandit, Neeraj Dhawan
BACKGROUND: Corticosteroids are the main therapy of nephrotic syndrome and goal of corticosteroid therapy is to obtain maximum clinical benefit with minimum adverse effects. Children are more vulnerable to side effects of corticosteroids related to growth and adrenal suppression, so a search for an alternative steroid with fewer side-effects is underway. Deflazacort is an oxazoline derivative and preliminary data suggest reduced osteoporosis, lesser growth retardation and weight gain with deflazacort...
April 2015: Iranian Journal of Pediatrics
Juliano Alves Pereira, Maria Julia Marques, Humberto Santo Neto
The standard therapy used in the treatment of Duchenne muscle dystrophy (DMD) is corticoids, such as deflazacort and prednisone. However, they have limited therapeutic value, and their combination with drugs already in use to treat other human diseases could potentially increase corticoid outcomes in DMD. In the present study, we evaluated whether a combined therapy of the corticoid deflazacort with doxycycline could result in greater improvement in mdx dystrophy than deflazacort alone. Deflazacort alone or deflazacort/doxycycline were administered for 36 days (starting on postnatal day 0) in drinking water...
July 2015: Clinical and Experimental Pharmacology & Physiology
Bijit Lodh, Kaku Akoijam Singh, Rajendra Singh Sinam
OBJECTIVE: The objective of the following study is to assess the effect of steroidal anti-inflammatory agent on the outcome of ureterorenoscopic lithotripsy (URSL) for ureterovesical junction (UVJ) calculus. SETTINGS AND DESIGN: This was a prospective randomized controlled study conducted at the Department of Urology, Regional Institute of Medical Sciences, Imphal. SUBJECTS AND METHODS: One hundred and twenty-six patients requiring ureteroscopic lithotripsy for UVJ calculus were randomly assigned into two groups...
April 2015: Urology Annals
Jerry Lewis, Lauren Bacall, Humphrey Bogart, Gene Kelly
BACKGROUND: . Dysferlinopathies are autosomal recessive disorders caused by mutations in the dysferlin (DYSF) gene encoding the dysferlin protein. DYSF mutations lead to a wide range of muscular phenotypes, with the most prominent being Miyoshi myopathy (MM) and limb girdle muscular dystrophy type 2B (LGMD2B). METHODS: . We assessed the one-year-natural course of dysferlinopathy, and the safety and efficacy of deflazacort treatment in a double-blind, placebo-controlled cross-over trial...
January 27, 2015: Gene
Richard T Wang, Cheri A Silverstein Fadlon, J Wes Ulm, Ivana Jankovic, Ascia Eskin, Ake Lu, Vanessa Rangel Miller, Rita M Cantor, Ning Li, Robert Elashoff, Anne S Martin, Holly L Peay, Nancy Halnon, Stanley F Nelson
To assess the utility of online patient self-report outcomes in a rare disease, we attempted to observe the effects of corticosteroids in delaying age at fulltime wheelchair use in Duchenne muscular dystrophy (DMD) using data from 1,057 males from DuchenneConnect, an online registry. Data collected were compared to prior natural history data in regard to age at diagnosis, mutation spectrum, and age at loss of ambulation. Because registrants reported differences in steroid and other medication usage, as well as age and ambulation status, we could explore these data for correlations with age at loss of ambulation...
2014: PLoS Currents
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