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Developing standardized corticosteroid treatment for Duchenne muscular dystrophy.

Guglieri, Michela and Bushby, Kate and McDermott, Michael P and Hart, Kimberly A and Tawil, Rabi and Martens, William B and Herr, Barbara E and McColl, Elaine and Wilkinson, Jennifer and Kirschner, Janbernd and King, Wendy M and Eagle, Michele and Brown, Mary W and Willis, Tracey and Hirtz, Deborah and Shieh, Perry B and Straub, Volker and Childs, Anne-Marie and Ciafaloni, Emma and Butterfield, Russell J and Horrocks, Iain and Spinty, Stefan and Flanigan, Kevin M and Kuntz, Nancy L and Baranello, Giovanni and Roper, Helen P and Morrison, Leslie and Mah, Jean K and Manzur, Adnan Y and McDonald, Craig M and Schara, Ulrike and von der Hagen, Maja and Barohn, Richard J and Campbell, Craig and Darras, Basil T and Finkel, Richard S and Vita, Giuseppe and Hughes, Imelda and Mongini, Tiziana and Pegoraro, Elena and Wicklund, Matthew and Wilichowski, Ekkehard and Bryan Burnette, W and Howard, James F and McMillan, Hugh J and Thangarajh, Mathula and Griggs, Robert C (2017) Developing standardized corticosteroid treatment for Duchenne muscular dystrophy. Contemporary clinical trials, 58. pp. 34-39. ISSN 1559-2030. This article is available to all HEFT staff and students via ASK Discovery tool http://tinyurl.com/z795c8c by using their HEFT Athens login IDs

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Official URL: http://www.contemporaryclinicaltrials.com/article/...

Abstract

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.

Item Type: Article
Additional Information: This article is available to all HEFT staff and students via ASK Discovery tool http://tinyurl.com/z795c8c by using their HEFT Athens login IDs
Subjects: WE Musculoskeletal. Orthopaedics
WS Paediatrics. Child health
Divisions: Womens and Childrens > Paediatrics
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Depositing User: Mrs Yolande Brookes
Date Deposited: 24 May 2017 14:17
Last Modified: 24 May 2017 14:17
URI: http://www.repository.heartofengland.nhs.uk/id/eprint/1372

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