P.2.11 CINRG Duchenne Natural History Study: Relationship of longitudinal measures of ambulatory timed function tests and loss of clinical milestones

• DOI: 10.1016/j.nmd.2013.06.416
• Publication Date: 2013-08-28T20:41:55Z
• Authors: C.M. McDonald; T. Duong; E. Henricson; T. Abresch; F. Hu; A. Cnaan
• Keywords: 03 medical and health sciences; 0302 clinical medicine

The Cooperative International Neuromuscular Research Group (CINRG) DMD Natural History Study enrolled 340 individuals with DMD aged 2–28 years in a longitudinal observational study. Clinical assessments were obtained every 3 months for the first year and annually, thereafter. The aim of this sub analysis was to evaluate the predictive capabilities of timed tests in assessing loss of clinically meaningful milestones. We created a 6-level composite of individual “milestone” tasks combining the results from the ability to perform the timed function tests and the Brooke and Vignos functional scales. Three GC groups (naive, current user, past user) were compared on timed function velocities at ages Timed function velocities increased with age in children 10 s. predicted loss of ability to stand (log rank p p 8 s. predicted loss of stair climbing (log rank p p 12 s. predicts loss of ambulation over 12 months (log rank p 10% over a year predicted the likelihood of loss of ambulation over the following 4 years (log rank p Longitudinal studies of timed function tests show predictable maturational changes and improved timed function velocities in those currently treated with GC. Timed function tests are clinically meaningful in terms of predictive ability for loss of function.