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

Research output: Contribution to journalArticle

112 Citations (Scopus)

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.

Original languageEnglish (US)
Pages (from-to)55-67
Number of pages13
JournalMuscle and Nerve
Volume48
Issue number1
DOIs
StatePublished - Jul 2013

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Duchenne Muscular Dystrophy
Natural History
Glucocorticoids
Disease Progression
Outcome Assessment (Health Care)
Clinical Trials
Muscles
Respiratory Function Tests
Steroids
Research
Therapeutics
Upper Extremity
Young Adult

Keywords

  • Adolescent
  • Adult
  • Child/preschool
  • Follow-up studies
  • Health status
  • Humans
  • Locomotion
  • Male
  • Muscle strength/physiology
  • Muscular dystrophies/classification
  • Muscular dystrophies/Duchenne/physiopathology
  • Muscular dystrophies/therapy
  • Phenotype
  • Quality of life/psychology
  • Respiratory function tests

ASJC Scopus subject areas

  • Clinical Neurology
  • Cellular and Molecular Neuroscience
  • Physiology (medical)
  • Physiology

Cite this

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. / Henricson, Erik K; Abresch, R. Ted; Cnaan, Avital; Hu, Fengming; Duong, Tina; Arrieta, Adrienne; Han, Jay; Escolar, Diana M.; Florence, Julaine M.; Clemens, Paula R.; Hoffman, Eric P.; McDonald, Craig M.

In: Muscle and Nerve, Vol. 48, No. 1, 07.2013, p. 55-67.

Research output: Contribution to journalArticle

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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.",
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AU - Duong, Tina

AU - Arrieta, Adrienne

AU - Han, Jay

AU - Escolar, Diana M.

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AU - Hoffman, Eric P.

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