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Developing standardized corticosteroid treatment for Duchenne muscular dystrophy.

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.

SUBMITTER: Guglieri M

PROVIDER: S-EPMC6279424 | biostudies-literature | 2017 Jul

REPOSITORIES: biostudies-literature

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Developing standardized corticosteroid treatment for Duchenne muscular dystrophy.

Guglieri Michela M Bushby Kate K McDermott Michael P MP Hart Kimberly A KA Tawil Rabi R Martens William B WB Herr Barbara E BE McColl Elaine E Wilkinson Jennifer J Kirschner Janbernd J King Wendy M WM Eagle Michele M Brown Mary W MW Willis Tracey T Hirtz Deborah D Shieh Perry B PB Straub Volker V Childs Anne-Marie AM Ciafaloni Emma E Butterfield Russell J RJ Horrocks Iain I Spinty Stefan S Flanigan Kevin M KM Kuntz Nancy L NL Baranello Giovanni G Roper Helen H Morrison Leslie L Mah Jean K JK Manzur Adnan Y AY McDonald Craig M CM Schara Ulrike U von der Hagen Maja M Barohn Richard J RJ Campbell Craig C Darras Basil T BT Finkel Richard S RS Vita Giuseppe G Hughes Imelda I Mongini Tiziana T Pegoraro Elena E Wicklund Matthew M Wilichowski Ekkehard E Bryan Burnette W W Howard James F JF McMillan Hugh J HJ Thangarajh Mathula M Griggs Robert C RC

Contemporary clinical trials 20170424

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 cou ...[more]

PMID: 28450193

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Project description:BackgroundDuchenne muscular dystrophy (DMD) is a severe rare progressive inherited neuromuscular disorder, leading to loss of ambulation (LOA) and premature mortality. The standard of care for patients with DMD has been treatment with corticosteroids for the past decade; however a synthesis of contemporary data describing the clinical course of DMD is lacking. The objective was to summarize age at key clinical milestones (loss of ambulation, scoliosis, ventilation, cardiomyopathy, and mortality) in the corticosteroid-treatment-era.MethodsA systematic review was conducted using MEDLINE and EMBASE. The percentage experiencing key clinical milestones, and the mean or median age at those milestones, was synthesized from studies from North American populations, published between 2007 and 2018.ResultsFrom 5637 abstracts, 29 studies were included. Estimates of the percentage experiencing key clinical milestones, and age at those milestones, showed heterogeneity. Up to 30% of patients lost ambulation by age 10 years, and up to 90% by 15 years of age. The mean age at scoliosis onset was approximately 14 years. Ventilatory support began from 15 to 18 years, and up to half of patients required ventilation by 20 years of age. Registry-based estimates suggest that 70% had evidence of cardiomyopathy by 15 years and almost all by 20 years of age. Finally, mortality rates up to 16% by age 20 years were reported; among those surviving to adulthood mortality was up to 60% by age 30 years.ConclusionsContemporary natural history studies from North America report that LOA on average occurs in the early teens, need for ventilation and cardiomyopathy in the late teens, and death in the third or fourth decade of life. Variability in rates may be due to differences in study design, treatment with corticosteroids or other disease-modifying agents, variations in clinical practices, and dystrophin mutations. Despite challenges in synthesizing estimates, these findings help characterize disease progression among contemporary North American DMD patients.

Dataset Information

Developing standardized corticosteroid treatment for Duchenne muscular dystrophy.

Publications

Developing standardized corticosteroid treatment for Duchenne muscular dystrophy.

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