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Scientific Articles

Developing standardized corticosteroid treatment for Duchenne muscular dystrophy

key information

source: Contemporary Clinical Trials

year: 2017

authors: Guglieri M, Bushby K, McDermott MP, Hart KA, Tawil R, Martens WB, Herr BE, McColl E, Wilkinson J, Kirschner J, King WM, Eagle M, Brown MW, Willis T, Hirtz D, Shieh PB, Straub V, Childs AM, Ciafaloni E, Butterfield RJ, Horrocks I, Spinty S, Flanigan KM, Kuntz NL, Baranello G, Roper H, Morrison L, Mah JK, Manzur AY, McDonald CM, Schara U, von der Hagen M, Barohn RJ, Campbell C, Darras BT, Finkel RS, Vita G, Hughes I, Mongini T, Pegoraro E, Wicklund M, Wilichowski E, Bryan Burnette W, Howard JF, McMillan HJ, Thangarajh M, Griggs RC


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.

organization: Newcastle University, UK; University of Rochester Medical Center, USA; University Medical Center, Germany; The Robert Jones and Agnes Hunt Orthopaedic Hospital, NHS Foundation Trust, UK; National Institutes of Health, USA; UCLA, USA; Leeds Teaching Hospitals, UK; University of Utah, USA; Greater Glasgow and Clyde NHS Yorkhill Hospital, UK; Alder Hey Children's Hospital, UK; Nationwide Children's Hospital, USA; Ann and Robert H. Lurie Children's Hospital, USA; Neurological Institute "Carlo Besta", Germany; Birmingham Heartlands Hospital, UK; University of Mexico, Mexico; University of Calgary, Canada; GOSH, UCL, UK; UC Davis Medical Center, USA; University of Essen, Germany; Children's Hospital, Technical University Dresden, Germany; University of Kansas Medical Center, USA; Children's Hospital London Health Sciences Centre, Canada; Boston Children's Hospital, USA; Nemours Children's Hospital, USA; University of Messina AOU Policlinico Gaetano Martino, Italy; Royal Manchester Children's Hospital, UK; University of Torino, Italy; University of Padova, Italy; Penn State College of Medicine, USA; Children's University Hospital, Germany; Vanderbilt Children's Hospital, USA; University of North Carolina School of Medicine, USA; Children's Hospital of Eastern Ontario, Canada; Children's National Medical Center, USA

DOI: 10.1016/j.cct.2017.04.008

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