Article

Duchenne muscular dystrophy (DMD) is an X-linked genetic neuromuscular disorder
characterized by progressive proximal to distal muscle weakness. The success of
randomized clinical trials for novel therapeutics depends on outcome measurements
that are sensitive to change.
As the development of motor skills may lead to
functional improvements in young boys with DMD, their inclusion may potentially
confound clinical trials.
Three-dimensional gait analysis is an under-utilized
approach that can quantify joint moments and powers, which reflect functional
muscle strength. In this study, gait kinetics, kinematics, spatial-temporal
parameters, and timed functional tests were quantified over a one-year period for
21 boys between 4 and 8 years old who were enrolled in a multisite natural
history study. At baseline, hip moments and powers were inadequate. Between the
two visits, 12 boys began a corticosteroid regimen (mean duration 10.8+/-2.4
months) while 9 boys remained steroid-naive. Significant between-group
differences favoring steroid use were found for primary kinetic outcomes (peak
hip extensor moments (p=.007), duration of hip extensor moments (p=.007), peak
hip power generation (p=.028)), and spatial-temporal parameters (walking speed
(p=.016) and cadence (p=.021)). Significant between-group differences were not
found for kinematics or timed functional tests with the exception of the 10m walk
test (p=.03), which improves in typically developing children within this age
range. These results indicate that hip joint kinetics can be used to identify
weakness in young boys with DMD and are sensitive to corticosteroid intervention.
Inclusion of gait analysis may enhance detection of a treatment effect in clinical trials particularly for young boys with more preserved muscle function.
CI - Copyright (c) 2016 Elsevier B.V. All rights reserved.

Langue : ANGLAIS