title: Change in natural history of Duchenne muscular dystrophy with long-term corticosteroid treatment: implications for management.
abstract: In 2005, the American Academy of Neurology and the Child Neurology Society published a practice parameter, based primarily on studies that involved 6 to 18 months of treatment, indicating that prednisone has a beneficial effect on muscle strength and function in patients with Duchenne muscular dystrophy and recommended that corticosteroids be offered (prednisone 0.75 mg/kg/d and deflazacort 0.9 mg/kg/d) as treatment. Recent reports emphasize that longer term treatment with corticosteroids (greater than 3 years) produces important sustained benefits in neuromuscular function without causing major side effects. This review highlights these reports and indicates that long-term corticosteroid therapy (1) prolongs ambulation by 2 to 5 years, (2) reduces the need for spinal stabilization surgery, (3) improves cardiopulmonary function, (4) delays the need for noninvasive nasal ventilation, and (5) increases survival and the quality of life of patients with Duchenne muscular dystrophy. Educational, vocational, and other social counseling is now a vital part of management for Duchenne muscular dystrophy.
authors.affiliation: Department of Neurology, University of Rochester, Rochester, NY 14642, USA. tracy_forrester@urmc.rochester.edu
authors.forename: Richard T
authors.initials: RT
authors.lastname: Moxley
authors.forename: Shree
authors.initials: S
authors.lastname: Pandya
authors.forename: Emma
authors.initials: E
authors.lastname: Ciafaloni
authors.forename: Deborah J
authors.initials: DJ
authors.lastname: Fox
authors.forename: Kim
authors.initials: K
authors.lastname: Campbell
mesh_terms: D002648:Child; D018450:Disease Progression; D004334:Drug Administration Schedule; D006801:Humans; D008297:Male; D020388:Muscular Dystrophy, Duchenne / Q000175:diagnosis* / Q000188:drug therapy* / Q000503:physiopathology; D011241:Prednisone / Q000008:administration & dosage* / Q000627:therapeutic use; D011282:Pregnenediones / Q000008:administration & dosage* / Q000627:therapeutic use; D013997:Time Factors; D016896:Treatment Outcome
chemical_list: D011282:Pregnenediones; C021988:deflazacort; D011241:Prednisone