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.
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.
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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