Journal article

Randomized, blinded trial of weekend vs daily prednisone in Duchenne muscular dystrophy

DM Escolar, LP Hache, PR Clemens, A Cnaan, CM McDonald, V Viswanathan, AJ Kornberg, TE Bertorini, Y Nevo, T Lotze, A Pestronk, MM Ryan, E Monasterio, JW Day, A Zimmerman, A Arrieta, E Henricson, J Mayhew, J Florence, F Hu Show all

Neurology | LIPPINCOTT WILLIAMS & WILKINS | Published : 2011

Abstract

OBJECTIVE: To perform a double-blind, randomized study comparing efficacy and safety of daily and weekend prednisone in boys with Duchenne muscular dystrophy (DMD). METHODS: A total of 64 boys with DMD who were between 4 and 10 years of age were randomized at 1 of 12 centers of the Cooperative International Neuromuscular Research Group. Efficacy and safety of 2 prednisone schedules (daily 0.75 mg/kg/day and weekend 10 mg/kg/wk) were evaluated over 12 months. RESULTS: Equivalence was met for weekend and daily dosing of prednisone for the primary outcomes of quantitative muscle testing (QMT) arm score and QMT leg score. Secondary strength scores for QMT elbow flexors also showed equivalence be..

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Grants

Awarded by General Clinical Research Center (GCRC)


Awarded by NIH


Awarded by NATIONAL CENTER FOR ADVANCING TRANSLATIONAL SCIENCES


Awarded by NATIONAL CENTER FOR RESEARCH RESOURCES


Funding Acknowledgements

Supported by the Muscular Dystrophy Association (MDA), General Clinical Research Center (GCRC) 5M01 RR020359, and the NIH (K23 RR16281-01).Dr. Escolar serves on a scientific advisory board for the NIH/NINDS; serves on the speakers' bureau for and has received funding for travel and speaker honoraria from Athena Diagnostics, Inc.; serves as a consultant for Acceleron Pharma, HALO therapeutics, AVI Biopharma, Gerson Lheman Group (GLC), and Medacorp; and has received research support from the NIH, the Muscular Dystrophy Association, and the Foundation to Eradicate Duchenne (FED). L. P. Hache serves on the CINRG Executive Committee, CINRG Publication and Outcomes Subcommittees, and Treat-NMD Global Database Oversight Committee; and has received research/salary support from Genzyme Corporation, the US Department of Defense, and the NIH. Dr. Clemens receives/has received research support from Genzyme Corporation, Amicus Therapeutics, Inc., the US Department of Defense, the US Department of Veterans Affairs, and the NIH. Dr. Cnaan serves on scientific advisory boards for the NIH (NIGMS, NIDDK, NCRR) and the FDA; and receives research support from the NIH (NINDS, NCRR), the US Department of Defense, the US Department of Education, and the Gilbert Family Neurofibromatosis Institute. Dr. McDonald serves on scientific advisory boards for PTC Therapeutics, Inc., GlaxoSmithKline, BioMarin Pharmaceutical Inc., and Gilead Sciences, Inc.; and receives research support from PTC Therapeutics, Inc., Insmed Inc., the NIH/NIDRR, the US Department of Education, Shriner's Hospital for Children, the Muscular Dystrophy Association, and Clinical Research Network in Duchenne Muscular Dystrophy. Dr. Viswanathan reports no disclosures. Dr. Kornberg has received funding for travel from Genzyme Corporation and Biogen Idec; and has received research support from Multiple Sclerosis Research Australia. Dr. Bertorini serves on speakers' bureaus for and has received funding for travel and speaker honoraria from Teva Pharmaceutical Industries Ltd., EMD Serono, Inc., Pfeiffer Pharmaceuticals Inc., Allergan, Inc., Pfizer Inc, and Athena Diagnostics, Inc.; serves on the editorial board of the Journal of Clinical Neuromuscular Disorders; and receives publishing royalties from editing three books with Elsevier (2002, 2008, 2010). Dr. Nevo served on the CINRG Executive Committee and serves on CINRG Sub-Therapeutic Subcommittee; has served as a consultant for Teva Pharmaceutical Industries Ltd.; holds a provisional patent on the use of glatiramer acetate in muscular dystrophy; and receives research support from AFM, the Israeli Ministry of Health and the Israel Science Foundation. Dr. Lotze serves on a scientific advisory board for Opexa Therapeutics. Dr. Pestronk serves on the scientific advisory board of the Myositis Association; has served on the speakers' bureau for and received speaker honoraria from Athena Diagnostics, Inc.; holds stock in Johnson & Johnson; is director of the Washington University Neuromuscular Clinical Laboratory which performs antibody testing and muscle and nerve pathology analysis, procedures for which the Washington University Neurology Department bills; may accrue revenue on patents re: TS-HDS antibody, GALOP antibody, GM1 ganglioside antibody, and Sulfatide antibody; has received license fee payments from Athena Diagnostics, Inc. for patents re: antibody testing; and receives/has received research support from Genzyme Corporation, Insmed Inc., Knopp Neurosciences Inc., Prosensa, Isis Pharmaceuticals, Inc., sanofi-aventis, Cytokinetics, Incorporated, the NIH, CINRG Children's Hospital Washington DC, the Myositis Association, and the Muscular Dystrophy Association. Dr.Ryan serves on atherapeutic advisory committee for Treat-NMD; serves as an Associate Editor for the Journal of Pediatric Neurology and on the editorial board of the Journal of Clinical Neuroscience; and receives research support from PTC Therapeutics, Inc. Dr. Monasterio reports no disclosures. Dr. Day serves on a scientific advisory board for PTC Therapeutics, Inc.; receives research support from Genzyme Corporation, PTC Therapeutics, Inc., the NIH (NIAMS, NINDS), and the Muscular Dystrophy Association; receives royalties for patents on genetic testing for myotonic dystrophy type 2 and spinocerebellar ataxia type 5 that are licensed to Athena Diagnostics; and serves on the MDA Medical Advisory Committee. A. Zimmerman receives salary support from the US Department of Defense and the US Department of Education. A. Arrieta receives salary support from the US Department of Defense, the US Department of Education, the NIH, and the Muscular Dystrophy Association. E. Henricson serves as a consultant for PTC Therapeutics, Inc. and receives salary support from the US Department of Education. J. Mayhew has received funding for travel and speaker honoraria from Genzyme Corporation and serves/has served as a consultant for Genzyme Corporation and Enobia Pharma Inc. Dr. Florence serves on a scientific advisory board for Prosensa; serves on the editorial board of Neuromuscular Disorders; and has serves/has served as a consultant for Prosensa, GlaxoSmithKline, Genzyme Corporation, PTC Therapeutics, Inc., and Acceleron Pharma. F. Hu receives salary support from the US Department of Defense, the US Department of Education, and the NIH/NCRR. Dr. Connolly serves as a Contributing Editor for the Journal of Child Neurology and receives research support from PTC Therapeutics, Inc., the NIH, and the Muscular Dystrophy Association.