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Pentoxifylline as a rescue treatment for DMD: a randomized double-blind clinical trial

D M Escolar1, A Zimmerman, T Bertorini

  • 1Children’s National Medical Center, Washington, DC, USA. diana.escolar@gmail.com

Neurology
|March 10, 2012
PubMed

Insights

Pentoxifylline (PTX) did not slow muscle strength decline in boys with Duchenne muscular dystrophy (DMD). This 12-month trial found no significant benefit of PTX compared to placebo in maintaining muscle function.

Area of Science:

  • Neurology
  • Pediatrics
  • Pharmacology

Background:

  • Duchenne muscular dystrophy (DMD) is a progressive genetic disorder causing muscle weakness.
  • Current treatments for DMD, including corticosteroids, aim to slow disease progression.
  • The potential of pentoxifylline (PTX) to modify DMD progression requires investigation.

Purpose of the Study:

  • To evaluate if pentoxifylline (PTX) can slow the decline of muscle strength and function in ambulatory boys with Duchenne muscular dystrophy (DMD).

Main Methods:

  • A 12-month, multicenter, randomized, double-blind, placebo-controlled trial.
  • Participants were corticosteroid-treated boys with DMD receiving daily slow-release PTX or placebo.
  • Primary outcome: change in quantitative muscle testing (QMT) total score; secondary outcomes included muscle strength, pulmonary function, and timed function tests.

Main Results:

  • Sixty-four boys with DMD were randomized; no significant difference in total QMT scores between PTX and placebo groups after 12 months (p = 0.14).
  • Most secondary outcomes also showed no significant differences between treatment groups.
  • Pentoxifylline use was associated with mild to moderate gastrointestinal and hematologic adverse events.

Conclusions:

  • Adding pentoxifylline to corticosteroid therapy did not improve or halt the deterioration of muscle strength and function in ambulatory boys with DMD over 12 months.
  • This study provides Class I evidence that PTX is not effective in preventing functional decline in this patient population.
Abstract

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