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

摘要

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