Longitudinal timed function tests in Duchenne muscular dystrophy: ImagingDMD cohort natural history.
Muscle Nerve, 2018/11;58(5):631-638.
Arora H[1], Willcocks RJ[1], Lott DJ[1], Harrington AT[2], Senesac CR[1], Zilke KL[3], Daniels MJ[4], Xu D[5], Tennekoon GI[2], Finanger EL[3], Russman BS[3], Finkel RS[6], Triplett WT[1], Byrne BJ[7], Walter GA[8], Sweeney HL[9], Vandenborne K[1]
Affiliations
PMID: 29742798DOI: 10.1002/mus.26161
Impact factor: 3.852
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.
Keywords: 6-minute walk test; Duchenne muscular dystrophy; ambulatory function; functional endpoints; loss of ambulation; outcome measures
MeSH terms
Adolescent; Age Factors; Child; Child, Preschool; Cohort Studies; Disease Progression; Female; Humans; Image Processing, Computer-Assisted; Magnetic Resonance Imaging; Male; Muscular Dystrophy, Duchenne; Outcome Assessment, Health Care; Time Factors; Walk Test; Walking
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