Long-term outcomes of steroid therapy for Duchenne muscular dystrophy in Japan.
BACKGROUND
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 0.75mg/kg/day prednisolone every day (daily group, n=51); those treated with 1mg/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 9.6years of age (to 94.1%) in the daily group, until 8.8years of age (to 96.9%) in the intermittent group, and until 7.2years 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.


CONCLUSIONS
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.
Department of Child Neurology, National Center Hospital, National Center of Neurology and Psychiatry, Tokyo, Japan.
Masahide
M
Goto
Department of Child Neurology, National Center Hospital, National Center of Neurology and Psychiatry, Tokyo, Japan. Electronic address: komakih@ncnp.go.jp.
Hirofumi
H
Komaki
Department of Child Neurology, National Center Hospital, National Center of Neurology and Psychiatry, Tokyo, Japan.
Eri
E
Takeshita
Faculty of Medicine, University of Tsukuba, Tsukuba, Japan.
Yoshiki
Y
Abe
Department of Child Neurology, National Center Hospital, National Center of Neurology and Psychiatry, Tokyo, Japan.
Akihiko
A
Ishiyama
Department of Child Neurology, National Center Hospital, National Center of Neurology and Psychiatry, Tokyo, Japan.
Kenji
K
Sugai
Department of Child Neurology, National Center Hospital, National Center of Neurology and Psychiatry, Tokyo, Japan.
Masayuki
M
Sasaki
Department of Mental Retardation and Birth Defect Research, National Institute of Neuroscience, National Center of Neurology and Psychiatry, Tokyo, Japan.
Yu-Ichi
Y
Goto
Department of Neuromuscular Research, National Institute of Neuroscience, National Center of Neurology and Psychiatry, Tokyo, Japan.
Ikuya
I
Nonaka
D000293:Adolescent; D000305:Adrenal Cortex Hormones / Q000009:adverse effects / Q000627:therapeutic use*; D001827:Body Height / Q000187:drug effects; D002648:Child; D002675:Child, Preschool; D004185:Disability Evaluation; D005500:Follow-Up Studies; D006801:Humans; D007564:Japan / Q000453:epidemiology; D053208:Kaplan-Meier Estimate; D009043:Motor Activity / Q000187:drug effects; D020388:Muscular Dystrophy, Duchenne / Q000188:drug therapy* / Q000453:epidemiology / Q000235:genetics; D009465:Neuromuscular Agents / Q000009:adverse effects / Q000627:therapeutic use*; D011239:Prednisolone / Q000009:adverse effects / Q000627:therapeutic use*; D012044:Regression Analysis; D012119:Respiration / Q000187:drug effects; D012189:Retrospective Studies; D013997:Time Factors; D016896:Treatment Outcome; D014797:Vital Capacity / Q000187:drug effects
D000305:Adrenal Cortex Hormones; D009465:Neuromuscular Agents; D011239:Prednisolone
Ambulation; Corticosteroid treatment; Height; Mixed model; Respiratory function; Side effect