Longitudinal timed function tests in Duchenne muscular dystrophy: ImagingDMD cohort natural history

Harneet Arora, Rebecca J. Willcocks, Donovan J. Lott, Ann T. Harrington, Claudia R. Senesac, Kirsten L. Zilke, Michael J. Daniels, Dandan Xu, Gihan I. Tennekoon, Erika Finanger, Barry S. Russman, Richard S. Finkel, William T. Triplett, Barry J. Byrne, Glenn A. Walter, H. Lee Sweeney, Krista Vandenborne

Research output: Contribution to journalArticle

7 Scopus citations

Abstract

INTRODUCTION: Tests of ambulatory function are common clinical trial endpoints in Duchenne muscular dystrophy (DMD). Using these tests, the ImagingDMD study has generated a large data set that can describe the contemporary natural history of DMD in 5-12.9-year-olds. METHODS: Ninety-two corticosteroid-treated boys with DMD and 45 controls participated in this longitudinal study. Participants performed the 6-minute walk test (6MWT) and timed function tests (TFT: 10-m walk/run, climbing 4 stairs, supine to stand). RESULTS: Boys with DMD had impaired functional performance even at 5-6.9 years old. Boys older than 7 had significant declines in function over 1 year for 10-m walk/run and 6MWT. Eighty percent of participants could perform all functional tests at 9 years old. TFTs appear to be slightly more responsive and predictive of disease progression than the 6MWT in 7-12.9 year olds. DISCUSSION: This study provides insight into the contemporary natural history of key functional endpoints in DMD. Muscle Nerve 58: 631-638, 2018.

Original languageEnglish (US)
Pages (from-to)631-638
Number of pages8
JournalMuscle & nerve
Volume58
Issue number5
DOIs
StatePublished - Nov 1 2018

Keywords

  • 6-minute walk test
  • ambulatory function
  • Duchenne muscular dystrophy
  • functional endpoints
  • loss of ambulation
  • outcome measures

ASJC Scopus subject areas

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

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    Arora, H., Willcocks, R. J., Lott, D. J., Harrington, A. T., Senesac, C. R., Zilke, K. L., Daniels, M. J., Xu, D., Tennekoon, G. I., Finanger, E., Russman, B. S., Finkel, R. S., Triplett, W. T., Byrne, B. J., Walter, G. A., Sweeney, H. L., & Vandenborne, K. (2018). Longitudinal timed function tests in Duchenne muscular dystrophy: ImagingDMD cohort natural history. Muscle & nerve, 58(5), 631-638. https://doi.org/10.1002/mus.26161