The results of a randomized controlled trial of daily Prednisone conducted in 99 boys aged five to 15 years with Duchenne dystrophy are reported from the Department of Neurology, University of Rochester, NY and five collaborating institutions. Prednisone at 0.3 mg/kg or 0.75 mg/kg and placebo were administered for six months. Patients were evaluated at ten days, one month, two months, three months, and six months of treatment. At the three month visit those receiving 0.75 mg/kg were significantly strongly than those given 0.3 mg/kg Prednisone and both Prednisone groups were stronger than those treated with placebo. At six months side-effects occurred in the group treated with 0.75 mg/kg and these included weight gain, Cushingoid appearance, and excessive hair growth. This study confirmed previous reports from these Centers showing that Prednisone produces a significant increase in muscle strength, pulmonary function, and functional ability in patients with Duchenne dystrophy. The present study also defines the minimum Prednisone dose required to produce the maximal increase in strength at 0.3 to 0.75 mg/kg. Improvement is rapid occurring within ten days and is maximal by two months of treatment. The authors recommend that a Prednisone dosage of 0.75 mg/kg/day be considered for patients with Duchenne dystrophy who experience functional decline. [1]

COMMENT: Clinical trials of myoblast transfer are investigational and limited in application. Studies of the benefits and mechanism of Prednisone in muscle protein synthesis are important particularly in young children showing a rapid functional decline. Alternative schedules of Prednisone administration such as pulse dosage are under investigation and may have fewer side effects. The beneficial effects of prednisone, 0.65 mg/kg/day, extending over a two year observation period has been demonstrated in 89 boys with DMD. [2]