A PILOT TRIAL OF PREDNISONE IN FACIOSCAPULOHUMERAL MUSCULAR-DYSTROPHY

Facioscapulohumeral muscular dystrophy (FSHD) is an autosomal dominant , 4q35-linked, slowly progressive muscular dystrophy with no known eff ective treatment. Since prednisone improves strength in Duchenne dystr ophy, we performed a pilot, open-label trial of prednisone in eight su bjects fulfilling strict diagnostic criteria for FSHD. Prednisone (1.5 mg/kg/day; maximum 80 mg/day) was administered for 12 weeks. Manual m uscle testing, maximum voluntary isometric contraction testing, and mu scle mass estimations by dual energy x-ray absorptiometry and urinary creatinine excretion were performed at baseline and at 12 weeks. There were no significant changes in strength or muscle mass. We conclude t hat prednisone given for 12 weeks does not produce major improvement i n strength or increase muscle mass in FSHD. The study did not have suf ficient power or length of follow-up to address the possibility that p rednisone might arrest or slow disease progression.