@article{89f1313be77f400e8053f5ab698012cb,
title = "Pentoxifylline as a rescue treatment for DMD: A randomized double-blind clinical trial",
abstract = "Objective: To determine whether pentoxifylline (PTX) slows the decline of muscle strength and function in ambulatory boys with Duchenne muscular dystrophy (DMD). Methods: This was a multicenter, randomized, double-blinded, controlled trial comparing 12 months of daily treatment with PTX or placebo in corticosteroid-treated boys with DMD using a slow-release PTX formulation {\~(}20 mg/kg/day). The primary outcome was the change in mean total quantitative muscle testing (QMT) score. Secondary outcomes included changes in QMT subscales, manual muscle strength, pulmonary function, and timed function tests. Outcomes were compared using Student t tests and a linear mixed-effects model. Adverse events (AEs) were compared using the Fisher exact test. Results: A total of 64 boys with DMD with a mean age of 9.9 ± 2.9 years were randomly assigned to PTX or placebo in 11 participating Cooperative International Neuromuscular Research Group centers. There was no significant difference between PTX and the placebo group in total QMT scores (p > 0.14) or in most of the secondary outcomes after a 12-month treatment. The use of PTX was associated with mild to moderate gastrointestinal or hematologic AEs. Conclusion: The addition of PTX to corticosteroid-treated boys with DMD at a moderate to late ambulatory stage of disease did not improve or halt the deterioration of muscle strength and function over a 12-month study period. Classification of evidence: This study provides Class I evidence that treatment with PTX does not prevent deterioration in muscle function or strength in corticosteroid-treated boys with DMD.",
author = "Escolar, {D. M.} and A. Zimmerman and T. Bertorini and Clemens, {P. R.} and Connolly, {A. M.} and L. Mesa and K. Gorni and A. Kornberg and H. Kolski and N. Kuntz and Y. Nevo and C. Tesi-Rocha and K. Nagaraju and S. Rayavarapu and Hache, {L. P.} and Mayhew, {J. E.} and J. Florence and F. Hu and A. Arrieta and E. Henricson and Leshner, {R. T.} and Mah, {J. K.}",
note = "Funding Information: 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). A. Zimmerman serves as a consultant for Halo Therapeutics. Dr. Bertorini serves on the speakers' bureaus of Teva, Biogen Idec, Allergan, and Merck Serono and serves on scientific advisory boards for and receives speaker honoraria from Pfeiffer, Allergan, and Athena. Dr. Clemens receives research support from Genzyme Corporation, Amicus, NIH, Veterans Administration, and Department of Defense. Dr. Connolly receives research support from PTC Pharmaceutical, the Muscular Dystrophy Association and NIH and serves on scientific advisory boards for Acceleron and Halo Therapeutics. Dr. Mesa and Dr. Gorni report no disclosures. Dr. Kornberg has received funding for travel from Genzyme and Biogen Idec and serves as a Section Editor for BMC Neurology . Dr. Kolski receives research support from Talecris Biotherapeutics. Dr. Kuntz receives research support from Cooperative International Neuromuscular Research Group/DOD and NIH. Dr. Nevo is listed as an author on a patent re: The use of Glatiramer Acetate in muscular dystrophy and receives research support from AFM and Israeli Ministry of Health and Little Steps (Israeli Parents organization). Dr. Tesi-Rocha reports no disclosures. Dr. Nagaraju serves as an Associate Editor for the Journal of Neurological Sciences and is founder of Reveragen Biophrama. Dr. Rayavarapu reports no disclosures. L.P. Hache has received funding for travel and speaker honoraria from Genzyme and receives staff grant funding from NIH and Department of Defense. J.E. Mayhew has received funding for travel and speaker honoraria from Genzyme and serves as a consultant for Enobia Pharma, Inc. and Genzyme. J. Florence has served on scientific advisory boards and/or as a consultant for Prosensa, GlaxoSmithKline, Acceleron, PTC Therapeutics, and DART Therapeutics. F. Hu receives research support from US Department of Defense, NIH, US Department of Education, and the Muscular Dystrophy Association. A. Arrieta receives research support from the US Department of Defense and the Muscular Dystrophy Association. E. Henricson served as a consultant for Genzyme and PTC Therapeutics. Dr. Leshner serves on a scientific board and speakers' bureau for Genzyme and receives research support from Genzyme, Wyeth, and US Department of Defense. Dr. Mah receives research support from PTC Therapeutics, Acceleron Pharma, US Department of Defense, and US FSH Society and Muscular Dystrophy Canada. Funding Information: The study was funded by General Clinical Research Center 5M01 RR020359 (Washington, DC) and M01 RR00084 (Pittsburgh, PA), Department of Defense (DoD) W81XWH-09-1-0592, and NIH K23 RR16281-01 and the Federation to Eradicate Duchenne . This publication was made possible by Grant UL1 RR024992 from the National Center for Research Resources (NCRR), a component of the NIH, and NIH Roadmap for Medical Research. The authors take full responsibility for the contents of this paper, which do not represent the views of the Department of Veterans Affairs, the US Government, the NCRR, the DoD, or the NIH. ",
year = "2012",
month = mar,
day = "20",
doi = "10.1212/WNL.0b013e31824c46be",
language = "English (US)",
volume = "78",
pages = "904--913",
journal = "Neurology",
issn = "0028-3878",
publisher = "Lippincott Williams and Wilkins",
number = "12",
}