Abstract
Introduction
Corticosteroids are effective for improving motor function in patients with Duchenne
muscular dystrophy (DMD), but there is no consensus on a regimen that balances efficacy
and side effects.
Methods
Data from three groups of DMD patients were retrospectively analyzed: those treated
with 0.75 mg/kg/day prednisolone every day (daily group, n = 51); those treated with 1 mg/kg/day prednisolone on alternate days (intermittent group, n = 36), and those not treated with steroids (nontreatment group, n = 42).
Results
Although the age of ambulation loss did not differ significantly among the groups,
the hazard ratios for ambulation loss relative to the nontreatment group were 0.24
(95% confidence interval [CI]: 0.11–0.54) in the daily group and 0.34 (95% CI: 0.19–0.62)
in the intermittent group. The percentage of predicted forced vital capacity increased
until 9.6 years of age (to 94.1%) in the daily group, until 8.8 years of age (to 96.9%) in the intermittent group, and until 7.2 years of age (to 87.6%) in the nontreatment group. Weight gain was the most frequently
observed side effect in the treated groups. Height was significantly lower in the
daily than in the nontreatment group. Other side effects were observed, but no patient
discontinued therapy. There were no marked differences in benefits and side effects
between the two treated groups.
Discussion
This is the first assessment of long-term outcomes of different steroid therapy regimens
in Japanese DMD patients. Benefits and side effects, except height, did not differ
significantly between steroid regimens.
Keywords
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Article Info
Publication History
Published online: April 21, 2016
Accepted:
April 4,
2016
Received in revised form:
March 30,
2016
Received:
February 19,
2016
Identification
Copyright
© 2016 The Japanese Society of Child Neurology. Published by Elsevier B.V. All rights reserved.
