Developing standardized corticosteroid treatment for Duchenne muscular dystrophy
Developing standardized corticosteroid treatment for Duchenne muscular dystrophy
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DOI：
10.1016/j.cct.2017.04.008
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发表时间：
2017-07-01
影响因子：
2.2
通讯作者：
Griggs, Robert C.
中科院分区：
文献类型：
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作者：
Guglieri, Michela;Bushby, Kate;Griggs, Robert C.
尽管皮质类固醇是唯一记录的改善杜氏肌营养不良症（DMD）男孩力量和功能的治疗方法，但皮质类固醇处方不一致，在一些国家，不开皮质类固醇处方。我们正在进行一项临床试验，（1）比较3种最常用的皮质类固醇方案;（2）简化DMD并发症的治疗;（3）简化皮质类固醇副作用的预防。来自5个国家的38个研究中心的研究人员计划招募300名4-7岁的男孩，他们被随机分配到三种治疗方案中的一种：每日泼尼松;每日地夫可特;或间歇泼尼松（10天治疗/10天停药）。男孩被随访至少3年，以评估不同治疗方案的相对有效性和不良事件特征。主要结局是一个三维变量，包括对数转换的从地板上起身的时间、用力肺活量和受试者/父母对治疗的满意度，每个变量均在所有基线后访视期间取平均值。该研究方案包括基于证据和共识的DMD并发症和皮质类固醇副作用的治疗。本研究旨在建立DMD的标准皮质类固醇方案。由于DMD的所有新干预措施都是作为皮质类固醇的补充疗法开发的，因此确定最佳方案对所有新治疗都很重要。
Despite corticosteroids being the only treatment documented to improve strength and function in boys with Duchenne muscular dystrophy (DMD) corticosteroid prescription is inconsistent and in some countries, corticosteroids are not prescribed. We are conducting a clinical trial that (1) compares the 3 most frequently prescribed corticosteroid regimes; (2) standardizes treatment of DMD complications; and (3) standardizes prevention of corticosteroid side effects. Investigators at 38 sites in 5 countries plan to recruit 300 boys aged 4-7 who are randomly assigned to one of three regimens: daily prednisone; daily deflazacort; or intermittent prednisone (10 days on/10 days off).Boys are followed for a minimum of 3 years to assess the relative effectiveness and adverse event profiles of the different regimens. The primary outcome is a 3-dimensional variable consisting of log-transformed time to rise from the floor, forced vital capacity, and subject/parent satisfaction with treatment, each averaged over all post-baseline visits. The study protocol includes evidence- and consensus-based treatment of DMD complications and of corticosteroid side effects.This study seeks to establish a standard corticosteroid regimen for DMD. Since all new interventions for DMD are being developed as add-an therapies to corticosteroids, defining the optimum regimen is of importance for all new treatments.