title: A pilot trial of prednisone in facioscapulohumeral muscular dystrophy. FSH-DY Group.
abstract: Facioscapulohumeral muscular dystrophy (FSHD) is an autosomal dominant, 4q35-linked, slowly progressive muscular dystrophy with no known effective treatment. Since prednisone improves strength in Duchenne dystrophy, we performed a pilot, open-label trial of prednisone in eight subjects fulfilling strict diagnostic criteria for FSHD. Prednisone (1.5 mg/kg/day; maximum 80 mg/day) was administered for 12 weeks. Manual muscle testing, maximum voluntary isometric contraction testing, and muscle 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 that prednisone given for 12 weeks does not produce major improvement in strength or increase muscle mass in FSHD. The study did not have sufficient power or length of follow-up to address the possibility that prednisone might arrest or slow disease progression.
authors.affiliation: Department of Neurology, University of Rochester School of Medicine and Dentistry, NY 14642, USA.
authors.forename: R
authors.initials: R
authors.lastname: Tawil
authors.forename: M P
authors.initials: MP
authors.lastname: McDermott
authors.forename: S
authors.initials: S
authors.lastname: Pandya
authors.forename: W
authors.initials: W
authors.lastname: King
authors.forename: J
authors.initials: J
authors.lastname: Kissel
authors.forename: J R
authors.initials: JR
authors.lastname: Mendell
authors.forename: R C
authors.initials: RC
authors.lastname: Griggs
mesh_terms: D000293:Adolescent; D000328:Adult; D001132:Arm; D005145:Face; D005260:Female; D006801:Humans; D008297:Male; D008875:Middle Aged; D009132:Muscles / Q000503:physiopathology; D009136:Muscular Dystrophies / Q000188:drug therapy* / Q000503:physiopathology; D010865:Pilot Projects; D011241:Prednisone / Q000009:adverse effects / Q000627:therapeutic use*; D012782:Shoulder; D016896:Treatment Outcome
chemical_list: D011241:Prednisone