Developing standardized corticosteroid treatment for Duchenne muscular dystrophy

作者：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

来源：Contemporary Clinical Trials, 2017, 58: 34-39.

DOI：10.1016/j.cct.2017.04.008

• Duchenne muscular dystrophy
• Standards of care
• Prednisolone
• Deflazacort
• Randomized

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.

• 出版日期2017-7
• 单位NIH; UC Davis; UCLA