Goto Masahide, Komaki Hirofumi, Takeshita Eri, Abe Yoshiki, Ishiyama Akihiko, Sugai Kenji, Sasaki Masayuki, Goto Yu-Ichi, Nonaka Ikuya
Department of Child Neurology, National Center Hospital, National Center of Neurology and Psychiatry, Tokyo, Japan.
Department of Child Neurology, National Center Hospital, National Center of Neurology and Psychiatry, Tokyo, Japan.
Brain Dev. 2016 Oct;38(9):785-91. doi: 10.1016/j.braindev.2016.04.001. Epub 2016 Apr 21.
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.
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).
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.
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.
皮质类固醇对改善杜氏肌营养不良症（DMD）患者的运动功能有效，但在平衡疗效和副作用的治疗方案上尚未达成共识。
回顾性分析三组DMD患者的数据：每天接受0.75mg/kg/天泼尼松龙治疗的患者（每日治疗组，n = 51）；隔日接受1mg/kg/天泼尼松龙治疗的患者（间歇治疗组，n = 36），以及未接受类固醇治疗的患者（未治疗组，n = 42）。
尽管各组间丧失行走能力的年龄无显著差异，但每日治疗组相对于未治疗组丧失行走能力的风险比为0.24（95%置信区间[CI]：0.11 - 0.54），间歇治疗组为0.34（95%CI：0.19 - 0.62）。每日治疗组预计用力肺活量百分比在9.6岁时增加至94.1%，间歇治疗组在8.8岁时增加至96.9%，未治疗组在7.2岁时增加至87.6%。体重增加是治疗组中最常观察到的副作用。每日治疗组的身高显著低于未治疗组。还观察到其他副作用，但无患者停止治疗。两个治疗组在益处和副作用方面无明显差异。
这是对日本DMD患者不同类固醇治疗方案长期疗效的首次评估。除身高外，不同类固醇治疗方案在益处和副作用方面无显著差异。
