Corticosteroid treatment and functional improvement in Duchenne muscular dystrophy: long-term effect.
OBJECTIVE
To determine and compare the long-term effects of prednisone and deflazacort on the functional status of children with Duchenne muscular dystrophy.


METHODS
A total of 49 boys with Duchenne muscular dystrophy, between the age of 12 and 15 yrs, who were observed over a 7-yr period were reviewed retrospectively. Eighteen had been treated with prednisone, 12 with deflazacort, and 19 had no drug treatment. All boys treated with steroids received medication for >2 yrs before losing their ambulation. Lower and upper limb motor functions, pulmonary function, prevalence of surgery for scoliosis, and side effects were compared.


RESULTS
Boys in the steroid groups were significantly more functional and performed better on all tests than boys not treated (P < 0.05). There was no significant difference between the deflazacort- and prednisone-treated groups (P > 0.05). The number of boys having scoliosis surgery in treated groups was significantly less than nontreated boys (P < 0.05). The control group's pulmonary capacity was decreasing and significantly less than both prednisone- and deflazacort-treated boys. Both deflazacort and prednisone had beneficial effect on pulmonary function and scoliosis. Cataracts, hypertension, behavioral changes, excessive weight gain, and vertebral fracture were noted as serious side effects.


CONCLUSIONS
Prednisone and deflazacort have a significant beneficial effect on slowing the disease progress. Their usage in Duchenne muscular dystrophy may prolong ambulation and upper limb function with similar potency. Both steroids also improve pulmonary function, in addition to delaying the need for spinal interventions, with similar therapeutic profiles.
Balaban
Birol
B
Department of Physical Medicine and Rehabilitation, Gulhane Military Medical Academy, Etlik-Ankara, Turkey.
Matthews
Dennis J
DJ
Clayton
Gerald H
GH
Carry
Terri
T
D000293:Adolescent; D000893:Anti-Inflammatory Agents / Q000627:therapeutic use*; D001827:Body Height / Q000187:drug effects; D001835:Body Weight / Q000187:drug effects; D018450:Disease Progression; D004305:Dose-Response Relationship, Drug; D005500:Follow-Up Studies; D005938:Glucocorticoids / Q000627:therapeutic use*; D006801:Humans; D008297:Male; D009043:Motor Activity / Q000187:drug effects*; D009048:Motor Skills / Q000187:drug effects; D018908:Muscle Weakness / Q000209:etiology / Q000517:prevention & control; D020388:Muscular Dystrophy, Duchenne / Q000188:drug therapy* / Q000503:physiopathology; D011241:Prednisone / Q000627:therapeutic use*; D011282:Pregnenediones / Q000627:therapeutic use*; D012189:Retrospective Studies; D018709:Statistics, Nonparametric
D000893:Anti-Inflammatory Agents; D005938:Glucocorticoids; D011282:Pregnenediones; C021988:deflazacort; D011241:Prednisone