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Longitudinal timed function tests in Duchenne muscular dystrophy: ImagingDMD cohort natural history
Author(s) -
Arora Harneet,
Willcocks Rebecca J.,
Lott Donovan J.,
Harrington Ann T.,
Senesac Claudia R.,
Zilke Kirsten L.,
Daniels Michael J.,
Xu Dandan,
Tennekoon Gihan I.,
Finanger Erika L.,
Russman Barry S.,
Finkel Richard S.,
Triplett William T.,
Byrne Barry J.,
Walter Glenn A.,
Sweeney H. Lee,
Vandenborne Krista
Publication year - 2018
Publication title -
muscle and nerve
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.025
H-Index - 145
eISSN - 1097-4598
pISSN - 0148-639X
DOI - 10.1002/mus.26161
Subject(s) - duchenne muscular dystrophy , natural history , supine position , physical medicine and rehabilitation , medicine , physical therapy , ambulatory , natural history study , cohort , neuromuscular disease , muscular dystrophy , disease
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