Preserving function in Duchenne dystrophy with long-term pulse prednisone therapy.
We report a case of a patient with Duchenne muscular dystrophy who was treated with intermittent pulse prednisone for severe asthma from age 3 to 17 yr and had remarkable preservation of skeletal muscle function. He had a maternal uncle with Duchenne muscular dystrophy who had the identical familial deletion mutation and died at age 19 of respiratory failure. Compared with his untreated uncle, our patient remains partially ambulatory at age 20. This case provides interesting, albeit anecdotal, evidence of considerable clinical benefit from pulse prednisone used on a much longer term basis than has been previously studied and promotes the need for further investigation on this type of therapy in Duchenne muscular dystrophy.
Carter
G T
GT
Department of Rehabilitation Medicine, School of Medicine, University of Washington, Seattle, USA.
McDonald
C M
CM
D000203:Activities of Daily Living; D000293:Adolescent; D000893:Anti-Inflammatory Agents / Q000627:therapeutic use*; D001249:Asthma / Q000150:complications* / Q000188:drug therapy*; D018450:Disease Progression; D004334:Drug Administration Schedule; D006801:Humans; D008297:Male; D018908:Muscle Weakness / Q000209:etiology; D018482:Muscle, Skeletal / Q000187:drug effects* / Q000503:physiopathology*; D020388:Muscular Dystrophy, Duchenne / Q000150:complications* / Q000235:genetics / Q000503:physiopathology*; D011241:Prednisone / Q000627:therapeutic use*; D013997:Time Factors
D000893:Anti-Inflammatory Agents; D011241:Prednisone