Article

OBJECTIVE: To determine the exercise capacity of children and adolescents with
Friedreich's Ataxia (FA) and to evaluate the effects of 6 months of idebenone
treatment on exercise capacity. DESIGN: Exploratory endpoint in a randomized
double-blind, placebo-controlled, phase II clinical trial designed to investigate
the effects of idebenone on a biomarker of oxidative stress. SETTING: Exercise
physiology laboratory in a single clinical research center. PARTICIPANTS:
Ambulatory subjects (N=48; age range, 9-17 y) with genetically confirmed FA.
INTERVENTION: Idebenone administered orally 3 times a day for a total daily dose
of approximately 5, 15, and 45 mg/kg or matching placebo for 6 months. MAIN
OUTCOME MEASURES: Peak oxygen consumption per unit time (peak VO(2)) and peak
work rate (WR) were measured during incremental exercise testing at baseline and
after treatment. Echocardiography and neurologic assessments were also completed
before and after treatment. RESULTS: Baseline mean peak VO(2) +/- SD was
746+/-246 mL/min (16.2+/-5.8 mL/kg/min), and WR was 40+/-23 W for all subjects.
Peak VO(2) and WR were correlated with short guanine-adenine-adenine allele
length and neurologic function. Relative left ventricular wall thickness was
increased but left ventricular ejection fraction was normal in most subjects;
there was no relationship between any exercise and echocardiographic measures.
There were no significant changes in mean peak VO(2) or WR after idebenone
treatment at any dose level relative to placebo. CONCLUSIONS: Exercise capacity
in children and adolescents with FA was significantly impaired. The basis for the
impairment appears to be multifactorial and correlated to the degree of
neurologic impairment. Although idebenone has previously been shown potentially
to improve features of FA, idebenone treatment did not increase exercise capacity
relative to placebo.
CI - Copyright 2010 American Congress of Rehabilitation Medicine. Published by
Elsevier Inc. All rights reserved.

Langue : ANGLAIS