title: 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.
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.
authors: Department of Physical Medicine and Rehabilitation, School of Medicine, University of California, Davis, 4860 Y Street, Suite 3850, Sacramento, California 95817, USA.
authors: Erik K
authors: EK
authors: Henricson
authors: R Ted
authors: RT
authors: Abresch
authors: Avital
authors: A
authors: Cnaan
authors: Fengming
authors: F
authors: Hu
authors: Tina
authors: T
authors: Duong
authors: Adrienne
authors: A
authors: Arrieta
authors: Jay
authors: J
authors: Han
authors: Diana M
authors: DM
authors: Escolar
authors: Julaine M
authors: JM
authors: Florence
authors: Paula R
authors: PR
authors: Clemens
authors: Eric P
authors: EP
authors: Hoffman
authors: Craig M
authors: CM
authors: McDonald
mesh_terms: D000293:Adolescent; D000328:Adult; D035843:Biomedical Research / Q000379:methods / Q000592:standards; D002648:Child; D002675:Child, Preschool; D002986:Clinical Trials as Topic / Q000379:methods / Q000592:standards; D015331:Cohort Studies; D003430:Cross-Sectional Studies; D003943:Diagnostic Techniques, Neurological / Q000592:standards; D018450:Disease Progression; D005938:Glucocorticoids / Q000627:therapeutic use*; D006801:Humans; D007391:International Cooperation; D008137:Longitudinal Studies; D008297:Male; D053580:Muscle Strength / Q000502:physiology; D020388:Muscular Dystrophy, Duchenne / Q000175:diagnosis / Q000188:drug therapy* / Q000453:epidemiology; D017063:Outcome Assessment, Health Care / Q000379:methods* / Q000592:standards; D011446:Prospective Studies; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: D005938:Glucocorticoids
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