Developing standardized corticosteroid treatment for Duchenne muscular dystrophy

Publikation: Beitrag in FachzeitschriftForschungsartikelBeigetragenBegutachtung

Beitragende

  • Michela Guglieri - , Newcastle University (Autor:in)
  • Kate Bushby - , Newcastle University (Autor:in)
  • Michael P. McDermott - , University of Rochester (Autor:in)
  • Kimberly A. Hart - , University of Rochester (Autor:in)
  • Rabi Tawil - , University of Rochester (Autor:in)
  • William B. Martens - , University of Rochester (Autor:in)
  • Barbara E. Herr - , University of Rochester (Autor:in)
  • Elaine McColl - , Newcastle University (Autor:in)
  • Jennifer Wilkinson - , Newcastle University (Autor:in)
  • Janbernd Kirschner - , Albert-Ludwigs-Universität Freiburg (Autor:in)
  • Wendy M. King - , University of Rochester (Autor:in)
  • Michele Eagle - , Newcastle University (Autor:in)
  • Mary W. Brown - , University of Rochester (Autor:in)
  • Tracey Willis - , The Robert Jones and Agnes Hunt Orthopaedic and District Hospital NHS Trust (Autor:in)
  • Deborah Hirtz - , National Institutes of Health (NIH) (Autor:in)
  • Perry B. Shieh - , University of California at Los Angeles (Autor:in)
  • Volker Straub - , Newcastle University (Autor:in)
  • Anne Marie Childs - , Leeds Teaching Hospitals NHS Trust (Autor:in)
  • Emma Ciafaloni - , University of Rochester (Autor:in)
  • Russell J. Butterfield - , University of Utah (Autor:in)
  • Iain Horrocks - , The Shrewsbury and Telford Hospital NHS Trust (Autor:in)
  • Stefan Spinty - , Alder Hey Children's NHS Foundation Trust (Autor:in)
  • Kevin M. Flanigan - , Nationwide Children’s Hospital (Autor:in)
  • Nancy L. Kuntz - , Ann and Robert H. Lurie Children's Hospital of Chicago (Autor:in)
  • Giovanni Baranello - , Neurological Institute “Carlo Besta” (Autor:in)
  • Helen Roper - , University Hospitals Birmingham NHS Foundation Trust (Autor:in)
  • Leslie Morrison - , Universidad Nacional Autónoma de México (Autor:in)
  • Jean K. Mah - , University of Calgary (Autor:in)
  • Adnan Y. Manzur - , University College London (Autor:in)
  • Craig M. McDonald - , University of California at Davis (Autor:in)
  • Ulrike Schara - , Universität Duisburg-Essen (Autor:in)
  • Maja von der Hagen - , Klinik und Poliklinik für Kinder- und Jugendmedizin, Abteilung für Neuropädiatrie (Autor:in)
  • Richard J. Barohn - , University of Kansas (Autor:in)
  • Craig Campbell - , Western University (Autor:in)
  • Basil T. Darras - , Harvard University (Autor:in)
  • Richard S. Finkel - , Nemours Children's Hospital (Autor:in)
  • Giuseppe Vita - , Università degli Studi di Messina (Autor:in)
  • Imelda Hughes - , Manchester University NHS Foundation Trust (Autor:in)
  • Tiziana Mongini - , University of Turin (Autor:in)
  • Elena Pegoraro - , Università degli studi di Padova (Autor:in)
  • Matthew Wicklund - , Pennsylvania State University (Autor:in)
  • Ekkehard Wilichowski - , Georg-August-Universität Göttingen (Autor:in)
  • W. Bryan Burnette - , Vanderbilt University (Autor:in)
  • James F. Howard - , University of North Carolina at Chapel Hill (Autor:in)
  • Hugh J. McMillan - , University of Ottawa (Autor:in)
  • Mathula Thangarajh - , Children's National Medical Center (Autor:in)
  • Robert C. Griggs - , University of Rochester (Autor:in)

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 (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.

Details

OriginalspracheEnglisch
Seiten (von - bis)34-39
Seitenumfang6
FachzeitschriftContemporary Clinical Trials
Jahrgang58
PublikationsstatusVeröffentlicht - 1 Juli 2017
Peer-Review-StatusJa

Externe IDs

Scopus 85018333312
PubMed 28450193

Schlagworte

Schlagwörter

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