Prednisone 10 days on/10 days off in patients with Duchenne muscular dystrophy.
Corticosteroids are effective in improving motor function in Duchenne muscular dystrophy (DMD) patients within 6 months-2 years of treatment initiation, but there is as yet no consensus on which treatment scheme is the best. We retrospectively analyzed data of 35 DMD patients who were treated with prednisone 0.75 mg/kg per day intermittently 10 days on/10 days off. Prednisone was started during the ambulant phase at age 3.5-9.7 years (median 6.5 years). The median period of treatment was 27 months (range 3-123 months). The median age at which ambulation was lost was 10.8 years (mean 10.9 years; 95% confidence interval 10.0-11.8 years). Nine patients (26%) had excessive weight gain. Eight boys (21%) had a bone fracture, which was when four of these eight children lost the ability to walk. Treatment was stopped in two obese patients, two hyperactive boys and one patient following a fracture. Our data suggest that prednisone 10 on/10 off has relatively few side effects and extends the ambulant phase by 1 year compared to historical controls.
Straathof
Chiara S M
CS
Department of Neurology, Leiden University Medical Centre, Leiden, The Netherlands. c.s.m.straathof@lumc.nl
Overweg-Plandsoen
W C G Truus
WC
van den Burg
Gert Jan
GJ
van der Kooi
Anneke J
AJ
Verschuuren
Jan J G M
JJ
de Groot
Imelda J M
IJ
D000293:Adolescent; D000367:Age Factors; D017668:Age of Onset; D017109:Akathisia, Drug-Induced; D000893:Anti-Inflammatory Agents / Q000008:administration & dosage; D002648:Child; D002675:Child, Preschool; D018450:Disease Progression; D004334:Drug Administration Schedule; D050723:Fractures, Bone / Q000139:chemically induced; D005938:Glucocorticoids / Q000008:administration & dosage; D006801:Humans; D008297:Male; D051346:Mobility Limitation; D018908:Muscle Weakness / Q000188:drug therapy* / Q000209:etiology / Q000517:prevention & control; D018482:Muscle, Skeletal / Q000187:drug effects* / Q000503:physiopathology; D020388:Muscular Dystrophy, Duchenne / Q000188:drug therapy* / Q000503:physiopathology; D009765:Obesity / Q000139:chemically induced / Q000503:physiopathology; D010024:Osteoporosis / Q000139:chemically induced; D011241:Prednisone / Q000008:administration & dosage*; D012189:Retrospective Studies; D016896:Treatment Outcome
D000893:Anti-Inflammatory Agents; D005938:Glucocorticoids; D011241:Prednisone