每周两次糖皮质激素治疗杜氏肌营养不良症婴儿和男童。
Twice-weekly glucocorticosteroids in infants and young boys with Duchenne muscular dystrophy.
机构信息
Department of Neurology, Washington University School of Medicine in Saint Louis, St Louis, Missouri, 63110, USA.
Department of Pediatrics, Washington University School of Medicine in Saint Louis, St Louis, Missouri, USA.
出版信息
Muscle Nerve. 2019 Jun;59(6):650-657. doi: 10.1002/mus.26441. Epub 2019 Feb 23.
INTRODUCTION
Glucocorticosteroids (GC) are effective in slowing weakness in boys with Duchenne muscular dystrophy (DMD).
METHODS
This is a multisite, 1-year, open-label trial of twice-weekly prednisolone (5 mg/kg/dose) in infants/young boys (0.4-2.4 years) with DMD. We compared changes in Bayley III Scales of Infant Development (Bayley-III) with untreated boys followed for 1 year (historical control cohort [HCC]). Twenty-three of 25 participants completed the study.
RESULTS
Treated boys gained an average of 0.5 points on the Bayley-III gross motor scaled score (GMSS) compared with the HCC who, on average, declined 1.3 points (P = 0.03). All boys maintained linear growth, and none developed Cushingoid features. Excessive weight gain occurred in 13 of 23 (56%) boys.
DISCUSSION
This study provides evidence that twice-weekly GC is well tolerated in infants and young boys with DMD and improves GMSS. Excessive weight gain is a potential risk. Longer follow-up is required to determine whether early GC initiation is feasible in most infants/boys with DMD. Muscle Nerve 59:650-657, 2019.
简介
糖皮质激素（GC）可有效减缓杜氏肌营养不良症（DMD）男孩的肌无力症状。
方法
这是一项为期 1 年、多中心、开放性标签的试验，研究对象为患有 DMD 的婴儿和幼儿（0.4-2.4 岁），给予其每周两次的泼尼松龙（5mg/kg/剂量）治疗。我们比较了接受治疗的男孩与未经治疗、随访 1 年的男孩（历史对照组 [HCC]）的贝利婴幼儿发展量表第三版（Bayley-III）的变化。25 名参与者中有 23 名完成了研究。
结果
与 HCC 相比，接受治疗的男孩在贝利婴幼儿发展量表第三版粗大运动量表评分（GMSS）上平均增加了 0.5 分，而 HCC 平均下降了 1.3 分（P=0.03）。所有男孩均保持线性生长，且均未出现库欣样特征。23 名男孩中有 13 名（56%）出现了体重过度增加。
讨论
本研究提供了证据表明，每周两次 GC 治疗可耐受良好，且可改善 DMD 婴儿和幼儿的 GMSS。体重过度增加是一个潜在的风险。需要更长时间的随访来确定在大多数 DMD 婴儿/男孩中早期 GC 治疗是否可行。肌肉神经 59:650-657, 2019.
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