[Glucocorticoid therapy in Duchenne muscular dystrophy]

Abstract

In 1974 Drachman and his colleagues first raised the possibility of beneficial effect of glucocorticoid in an uncontrolled trial of Duchenne muscular dystrophy (DMD) patients. In contrast, Siegel et al. could not find any significant effect of glucocorticoid in a controlled study. There then followed notable absence in formal trials until Brooke et al. published interesting results in a trial of prednisone in 1987. This was subsequently followed by a series of short term, randomized, double-blind, controlled trials. These studies demonstrated that glucocorticoid could slow the rate of decline of muscle strength. We also investigated the effect of glucocorticoid in 10 DMD patients taking a dosage of prednisolone 1 mg/kg on alternate day. Seven patients of those maintained activities of daily living after 48 weeks of treatment, but the benefits did not last over 192 weeks in any patient. Glucocorticoid therapy may be a valuable option in DMD patients because that no effective medical treatment is currently available. It does significantly slow the progress of disease for at least 3 years. Benefits must be weighed against expected side effects from glucocorticoid. Further studies are needed to establish optimal treatment schedules using prednisolone and develop therapeutic agents with fewer side-effects. The mechanism of beneficial effect of glucocorticoid is unknown.