Pentoxifylline as a rescue treatment for DMD
Neurology®,  Clinical Article

Escolar DM et al. – The addition of pentoxifylline (PTX) to corticosteroid–treated boys with Duchenne muscular dystrophy (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.

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.

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