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Published on: August 2, 2016
Liquid formulation of pentoxifylline is a poorly tolerated treatment for duchenne dystrophy
Angela Zimmerman1, Paula R Clemens, Carolina Tesi-Rocha
1Children's National Medical Center, Washington, DC, USA.
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.
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