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Contemp Clin Trials. 2017 Jul;58:34-39. doi: 10.1016/j.cct.2017.04.008. Epub 2017 Apr 24.

Developing standardized corticosteroid treatment for Duchenne muscular dystrophy.

Author information

1
John Walton Muscular Dystrophy Research Centre, Newcastle University, United Kingdom. Electronic address: michela.guglieri@ncl.ac.uk.
2
John Walton Muscular Dystrophy Research Centre, Newcastle University, United Kingdom.
3
University of Rochester Medical Center, United States.
4
Newcastle University, United Kingdom.
5
University Medical Center, Freiburg, Germany.
6
The Robert Jones and Agnes Hunt Orthopaedic Hospital, NHS Foundation Trust, Oswestry, United Kingdom.
7
National Institutes of Health, United States.
8
UCLA, United States.
9
Leeds Teaching Hospitals, United Kingdom.
10
University of Utah, United States.
11
Greater Glasgow and Clyde NHS Yorkhill Hospital, United Kingdom.
12
Alder Hey Children's Hospital, United Kingdom.
13
Nationwide Children's Hospital, United States.
14
Ann and Robert H. Lurie Children's Hospital, United States.
15
Neurological Institute "Carlo Besta", Germany.
16
Birmingham Heartlands Hospital, United Kingdom.
17
University of Mexico, Mexico.
18
University of Calgary, Canada.
19
GOSH, UCL, United Kingdom.
20
UC Davis Medical Center, United States.
21
University of Essen, Germany.
22
Children's Hospital, Technical University Dresden, Germany.
23
University of Kansas Medical Center, United States.
24
Children's Hospital London Health Sciences Centre, Canada.
25
Boston Children's Hospital, United States.
26
Nemours Children's Hospital, United States.
27
University of Messina AOU Policlinico Gaetano Martino, Italy.
28
Royal Manchester Children's Hospital, United Kingdom.
29
University of Torino, Italy.
30
University of Padova, Italy.
31
Penn State College of Medicine, United States.
32
Children's University Hospital, Göttingen, Germany.
33
Vanderbilt Children's Hospital, United States.
34
University of North Carolina School of Medicine, United States.
35
Children's Hospital of Eastern Ontario, Canada.
36
Children's National Medical Center, Washington, United States.

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.

KEYWORDS:

Deflazacort; Duchenne muscular dystrophy; Prednisolone; Randomized; Standards of care

PMID:
28450193
PMCID:
PMC6279424
DOI:
10.1016/j.cct.2017.04.008
[Indexed for MEDLINE]
Free PMC Article

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