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

ABSTRACT:

Objective

To determine whether pentoxifylline (PTX) slows the decline of muscle strength and function in ambulatory boys with Duchenne muscular dystrophy (DMD).

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 wit

SUBMITTER: Escolar DM 

PROVIDER: S-EPMC3306159 | biostudies-literature | 2012 Mar

REPOSITORIES: biostudies-literature