Toggle Main Menu Toggle Search

Open Access padlockePrints

Developing standardized corticosteroid treatment for Duchenne muscular dystrophy

Lookup NU author(s): Dr Michela Guglieri, Professor Katherine Bushby, Professor Elaine McColl, Dr Jennifer Wilkinson, Dr Michelle Eagle, Professor Volker Straub

Downloads

Full text for this publication is not currently held within this repository. Alternative links are provided below where available.


Abstract

© 2017 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-on therapies to corticosteroids, defining the optimum regimen is of importance for all new treatments.


Publication metadata

Author(s): 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 A-M, 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

Publication type: Article

Publication status: Published

Journal: Contemporary Clinical Trials

Year: 2017

Volume: 58

Pages: 34-39

Print publication date: 01/07/2017

Online publication date: 24/04/2017

Acceptance date: 24/04/2017

ISSN (print): 1551-7144

ISSN (electronic): 1559-2030

Publisher: Elsevier

URL: https://doi.org/10.1016/j.cct.2017.04.008

DOI: 10.1016/j.cct.2017.04.008


Altmetrics

Altmetrics provided by Altmetric


Actions

Find at Newcastle University icon    Link to this publication


Share