The cooperative international neuromuscular research group Duchenne natural history study: Glucocorticoid treatment preserves clinically meaningful functional milestones and reduces rate of disease progression as measured by manual muscle testing and other commonly used clinical trial outcome measures

Erik K Henricson; R. Ted Abresch; Avital Cnaan; Fengming Hu; Tina Duong; Adrienne Arrieta; Jay Han; Diana M Escolar; Julaine M Florence; Paula R Clemens; Eric P Hoffman; Craig M McDonald; Cooperative International Neuromuscular Research Group (CINRG) Investigators

doi:10.1002/mus.23808

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The cooperative international neuromuscular research group Duchenne natural history study: Glucocorticoid treatment preserves clinically meaningful functional milestones and reduces rate of disease progression as measured by manual muscle testing and other commonly used clinical trial outcome measures

Journal article

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The cooperative international neuromuscular research group Duchenne natural history study: Glucocorticoid treatment preserves clinically meaningful functional milestones and reduces rate of disease progression as measured by manual muscle testing and other commonly used clinical trial outcome measures

Erik K Henricson, R. Ted Abresch, Avital Cnaan, Fengming Hu, Tina Duong, Adrienne Arrieta, Jay Han, Diana M Escolar, Julaine M Florence, Paula R Clemens, …

Muscle & nerve, Vol.48(1), pp.55-67

07/2013

DOI: 10.1002/mus.23808

PMCID: PMC4103170

PMID: 23649481

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http://doi.org/10.1002/mus.23808View

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Abstract

ABSTRACT Introduction: Glucocorticoid (GC) therapy in Duchenne muscular dystrophy (DMD) has altered disease progression, necessitating contemporary natural history studies. Methods: The Cooperative Neuromuscular Research Group (CINRG) DMD Natural History Study (DMD‐NHS) enrolled 340 DMD males, ages 2–28 years. A comprehensive battery of measures was obtained. Results: A novel composite functional “milestone” scale scale showed clinically meaningful mobility and upper limb abilities were significantly preserved in GC‐treated adolescents/young adults. Manual muscle test (MMT)‐based calculations of global strength showed that those patients <10 years of age treated with steroids declined by 0.4±0.39 MMT unit/year, compared with −0.4±0.39 MMT unit/year in historical steroid‐naive subjects. Pulmonary function tests (PFTs) were relatively preserved in steroid‐treated adolescents. The linearity and magnitude of decline in measures were affected by maturational changes and functional status. Conclusions: In DMD, long‐term use of GCs showed reduced strength loss and preserved functional capabilities and PFTs compared with previous natural history studies performed prior to the widespread use of GC therapy. Muscle Nerve, 2013

Phenotype

quality of life/psychology

child/preschool

locomotion

muscular dystrophies/Duchenne/physiopathology

muscle strength/physiology

health status

muscular dystrophies/therapy

respiratory function tests

adolescent

follow‐up studies

adult

humans

male

muscular dystrophies/classification

Details

Title: Subtitle: The cooperative international neuromuscular research group Duchenne natural history study: Glucocorticoid treatment preserves clinically meaningful functional milestones and reduces rate of disease progression as measured by manual muscle testing and other commonly used clinical trial outcome measures
Creators: Erik K Henricson - University of California
R. Ted Abresch - University of California
Avital Cnaan - Departments of Pediatrics, Epidemiology, and Biostatistics, George Washington University
Fengming Hu - Center for Genetic Medicine Research, Children's National Medical Center
Tina Duong - Center for Genetic Medicine Research, Children's National Medical Center
Adrienne Arrieta - Center for Genetic Medicine Research, Children's National Medical Center
Jay Han - University of California
Diana M Escolar - Department of Neurology, Kennedy Krieger Institute
Julaine M Florence - Department of Neurology, Washington University
Paula R Clemens - Department of Neurology, University of Pittsburgh and Department of Veterans Affairs Medical Center
Eric P Hoffman - Department of Integrative Systems Biology, George Washington University
Craig M McDonald - University of California
Cooperative International Neuromuscular Research Group (CINRG) Investigators
Contributors: Katherine D Mathews (Contributor) - University of Iowa, Stead Family Department of Pediatrics
Resource Type: Journal article
Publication Details: Muscle & nerve, Vol.48(1), pp.55-67
DOI: 10.1002/mus.23808
PMID: 23649481
PMCID: PMC4103170
NLM abbreviation: Muscle Nerve
ISSN: 0148-639X
eISSN: 1097-4598
Number of pages: 13
Language: English
Date published: 07/2013
Academic Unit: Neurology; Stead Family Department of Pediatrics; Iowa Neuroscience Institute; Neurology (Pediatrics)
Record Identifier: 9984070713702771

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