Deflazacort use in Duchenne muscular dystrophy: an 8-year follow-up.
Data reported here were collected over an 8-year period for 79 Duchenne muscular dystrophy patients, 37 of whom were treated with deflazacort. Mean length of treatment was 66 months. Treated boys stopped walking at 11.5 +/- 1.9 years, compared with 9.6 +/- 1.4 years for untreated boys. Cardiac function was better preserved with the use of deflazacort, as shown by a normal shortening fraction in treated (30.8 +/- 4.5%) vs untreated boys (26.6 +/- 5.7%, P < 0.05), a higher ejection fraction (52.9 +/- 6.3% treated vs 46 +/- 10% untreated), and lower frequency of dilated cardiomyopathy (32% treated vs 58% untreated). Scoliosis was much less severe in treated (14 +/- 2.5 degrees ) than in untreated boys (46 +/- 24 degrees ). No spinal surgery was necessary in treated boys. Limb fractures were similarly frequent in treated (24%) and untreated (26%) boys, but vertebral fractures occurred only in the treated group (7/37) (compared with zero for the untreated group). In both groups, body weight excess tripled between the ages of 8 and 12 years. All untreated patients grew normally (>4 cm/year), as opposed to only 15% of treated boys. Deflazacort improves cardiac function, prolongs walking, and seems to eliminate the need for spinal surgery, although vertebral fractures and stunted growth occur. The overall impact on quality of life appears positive.
Houde
Sylvie
S
Department of Pediatrics, Division of Rehabilitation Medicine, Marie-Enfant Rehabilitation Center, 5200 Bélanger East, Montreal, Quebec, Canada. sylvie.houde.irm@ssss.gouv.ca
Filiatrault
Michèle
M
Fournier
Anne
A
Dubé
Julie
J
D'Arcy
Sylvie
S
Bérubé
Denis
D
Brousseau
Yves
Y
Lapierre
Guy
G
Vanasse
Michel
M
D000293:Adolescent; D000328:Adult; D000893:Anti-Inflammatory Agents / Q000627:therapeutic use*; D001827:Body Height / Q000187:drug effects; D001835:Body Weight / Q000187:drug effects; D015519:Bone Density / Q000187:drug effects; D002386:Cataract / Q000209:etiology / Q000517:prevention & control; D050723:Fractures, Bone / Q000209:etiology / Q000517:prevention & control; D006801:Humans; D008137:Longitudinal Studies; D008297:Male; D020388:Muscular Dystrophy, Duchenne / Q000150:complications / Q000188:drug therapy*; D011282:Pregnenediones / Q000627:therapeutic use*; D012600:Scoliosis / Q000209:etiology / Q000517:prevention & control; D016896:Treatment Outcome
D000893:Anti-Inflammatory Agents; D011282:Pregnenediones; C021988:deflazacort