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Muscle Nerve. 2011 Aug;44(2):170-3. doi: 10.1002/mus.22127. Epub 2011 Jun 14.

Liquid formulation of pentoxifylline is a poorly tolerated treatment for duchenne dystrophy.

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1
Children's National Medical Center, Washington, DC, USA.

Abstract

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.

Comment in

PMID:
21674534
PMCID:
PMC3136640
DOI:
10.1002/mus.22127
[Indexed for MEDLINE]
Free PMC Article

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