LIQUID FORMULATION OF PENTOXIFYLLINE IS A POORLY TOLERATED TREATMENT FOR DUCHENNE DYSTROPHY

作者：Zimmerman Angela; Clemens Paula R; Tesi Rocha Carolina; Connolly Anne; Iannaccone Susan T; Kuntz Nancy; Arrieta Adrienne; Hache Lauren; Henricson Erik; Hu Fengming; Mayhew Jill; Escolar Diana M^*

来源：Muscle & Nerve, 2011, 44(2): 170-173.

DOI：10.1002/mus.22127

• clinical trial
• Duchenne
• leukopenia
• muscular dystrophy
• pentoxifylline

Introduction: In this study we performed an open-label, pilot study of an orally administered liquid formulation of immediate-release pentoxifylline (PTX) on patients with Duchenne muscular dystrophy (DMD). Treatment efficacy, safety, and tolerability were assessed. Methods: The tolerability and safety of PTX and measures of muscle strength and function were evaluated during 12 months of treatment. Results: Seventeen boys with DMD, between 4 and 8 years of age, were enrolled at one of five Cooperative International Neuromuscular Research Group (CINRG) centers. Only 9 were able to complete the 12-month PTX treatment phase; the primary reason for discontinuation was adverse events. Intolerable gastrointestinal side effects were experienced by 65% of participants. Two participants had severe leukopenia that resolved with medication withdrawal. Conclusions: Open-label treatment with a liquid formulation of immediate-release PTX resulted in a high incidence of adverse events in boys with DMD. Poor tolerability of this PTX formulation precluded adequate assessment of efficacy. Muscle Nerve 44: 170-173, 2011

• 出版日期2011-8