Guglieri Michela, Bushby Kate, McDermott Michael P, Hart Kimberly A, Tawil Rabi, Martens William B, Herr Barbara E, McColl Elaine, Wilkinson Jennifer, Kirschner Janbernd, King Wendy M, Eagle Michele, Brown Mary W, Willis Tracey, Hirtz Deborah, Shieh Perry B, Straub Volker, Childs Anne-Marie, Ciafaloni Emma, Butterfield Russell J, Horrocks Iain, Spinty Stefan, Flanigan Kevin M, Kuntz Nancy L, Baranello Giovanni, Roper Helen, Morrison Leslie, Mah Jean K, Manzur Adnan Y, McDonald Craig M, Schara Ulrike, von der Hagen Maja, Barohn Richard J, Campbell Craig, Darras Basil T, Finkel Richard S, Vita Giuseppe, Hughes Imelda, Mongini Tiziana, Pegoraro Elena, Wicklund Matthew, Wilichowski Ekkehard, Bryan Burnette W, Howard James F, McMillan Hugh J, Thangarajh Mathula, Griggs Robert C
John Walton Muscular Dystrophy Research Centre, Newcastle University, United Kingdom.
John Walton Muscular Dystrophy Research Centre, Newcastle University, United Kingdom.
Contemp Clin Trials. 2017 Jul;58:34-39. doi: 10.1016/j.cct.2017.04.008. Epub 2017 Apr 24.
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 (10days on/10days off). Boys are followed for a minimum of 3years 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-on therapies to corticosteroids, defining the optimum regimen is of importance for all new treatments.
尽管皮质类固醇是唯一被证明可改善杜氏肌营养不良症（DMD）男孩力量和功能的治疗方法，但皮质类固醇的处方并不一致，在一些国家，并不开具皮质类固醇药物。我们正在进行一项临床试验，该试验（1）比较三种最常用的皮质类固醇治疗方案；（2）规范DMD并发症的治疗；（3）规范皮质类固醇副作用的预防。来自5个国家38个地点的研究人员计划招募300名4至7岁的男孩，他们被随机分配到三种治疗方案之一：每日服用泼尼松；每日服用地夫可特；或间歇性服用泼尼松（服用10天/停用10天）。对男孩进行至少3年的随访，以评估不同治疗方案的相对有效性和不良事件情况。主要结局是一个三维变量，包括从地面起身的对数转换时间、用力肺活量以及受试者/家长对治疗的满意度，每个指标均在基线后的所有随访中进行平均。研究方案包括基于证据和共识的DMD并发症及皮质类固醇副作用的治疗。本研究旨在为DMD确立一种标准的皮质类固醇治疗方案。由于所有针对DMD的新干预措施都是作为皮质类固醇的附加疗法开发的，因此确定最佳治疗方案对所有新疗法都很重要。
