The Cooperative International Neuromuscular Research Group Duchenne Natural History Study-A Longitudinal Investigation In The Era Of Glucocorticoid Therapy: Design Of Protocol And The Methods Used

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

Research output: Contribution to journalReview ArticleResearchpeer-review

90 Citations (Scopus)

Abstract

Contemporary natural history data in Duchenne muscular dystrophy (DMD) is needed to assess care recommendations and aid in planning future trials. Methods: The Cooperative International Neuromuscular Research Group (CINRG) DMD Natural History Study (DMD-NHS) enrolled 340 individuals, aged 2-28 years, with DMD in a longitudinal, observational study at 20 centers. Assessments obtained every 3 months for 1 year, at 18 months, and annually thereafter included: clinical history; anthropometrics; goniometry; manual muscle testing; quantitative muscle strength; timed function tests; pulmonary function; and patient-reported outcomes/health-related quality-of-life instruments. Results: Glucocorticoid (GC) use at baseline was 62% present, 14% past, and 24% GC-naive. In those ≥6 years of age, 16% lost ambulation over the first 12 months (mean age 10.8 years). Conclusions: Detailed information on the study methodology of the CINRG DMD-NHS lays the groundwork for future analyses of prospective longitudinal natural history data. These data will assist investigators in designing clinical trials of novel therapeutics.

Original languageEnglish
Pages (from-to)32-54
Number of pages23
JournalMuscle and Nerve
Volume48
Issue number1
DOIs
Publication statusPublished - Jul 2013
Externally publishedYes

Keywords

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

Cite this

McDonald, C. M., Henricson, E. K., Abresch, R. T., Han, J. J., Escolar, D. M., Florence, J. M., Duong, T., Arrieta, A., Clemens, P. R., Hoffman, E. P., Cnaan, A., & the Cooperative International Neuromuscular Research Group (CINRG) Investigators (2013). The Cooperative International Neuromuscular Research Group Duchenne Natural History Study-A Longitudinal Investigation In The Era Of Glucocorticoid Therapy: Design Of Protocol And The Methods Used. Muscle and Nerve, 48(1), 32-54. https://doi.org/10.1002/mus.23807