Purpose:

In this study Pentoxifylline was added as a rescue treatment to patients who were taking steroids (the same amount for at least 1 year), but were either stable (no improvement of muscle strength over three months) or had continued to slowly deteriorate. Patients were not excluded if they are taking any combination of herbal supplements or vitamins as long as the dosing had been stable for 2 months.

Further Study Details:

Duchenne muscular dystrophy (DMD) is the most common lethal inherited disorder worldwide. Despite the exponential increase in our understanding of the disorder since the discovery and characterization of the causative gene and its product dystrophin in 1987, current therapeutic management remains largely supportive. Awaiting a final genetic cure to be available in the future, further investments in developing better drug therapies for DMD remain important. This study was designed as a randomized, placebo controlled study. Patients were enrolled in the study after showing similar muscle testing results during the screening visit. Sixty-four patients were randomized into the pentoxifylline arm or a placebo arm. Subjects were evaluated by quantitative muscle testing (QMT), manual muscle testing (MMT), timed function testing, functional evaluation and pulmonary function tests, and measurement of contractures. At each visit, subjects also underwent medical history review and safety evaluation.

Results

The results of this study show that Pentoxifylline was reasonably well tolerated over the study year. However, the data did not confirm any additional benefit in terms of increasing muscle strength or function, or slowing disease progression in the study population of ambulant boys 7 years and older with DMD who have been on a stable steroid dose. In addition, we were unable to find any benefit in prevention of contractures. The group of boys who received Pentoxifylline showed significantly more mild side effects which included increased bruising, mild nose bleeds and nausea. Consequently, we do not recommend Pentoxifylline as an adjunct to steroid treatment.