Coenzyme Q10 and vitamin E deficiency in Friedreich's ataxia: predictor of efficacy of vitamin E and coenzyme Q10 therapy.
OBJECTIVE
A pilot study of high dose coenzyme Q(10) (CoQ(10))/vitamin E therapy in Friedreich's ataxia (FRDA) patients resulted in significant clinical improvements in most patients. This study investigated the potential for this treatment to modify clinical progression in FRDA in a randomized double blind trial.


METHODS
Fifty FRDA patients were randomly divided into high or low dose CoQ(10)/ vitamin E groups. The change in International Co-operative Ataxia Ratings Scale (ICARS) was assessed over 2 years as the primary end-point. A post hoc analysis was made using cross-sectional data.


RESULTS
At baseline serum CoQ(10) and vitamin E levels were significantly decreased in the FRDA patients (P < 0.001). During the trial CoQ(10) and vitamin E levels significantly increased in both groups (P < 0.01). The primary and secondary end-points were not significantly different between the therapy groups. When compared to cross-sectional data 49% of all patients demonstrated improved ICARS scores. This responder group had significantly lower baseline serum CoQ(10) levels.


CONCLUSIONS
A high proportion of FRDA patients have a decreased serum CoQ(10) level which was the best predictor of a positive clinical response to CoQ(10)/vitamin E therapy. Low and high dose CoQ(10)/vitamin E therapies were equally effective in improving ICARS scores.
Cooper
J M
JM
University Department of Clinical Neurosciences, Institute of Neurology, University College London, London, UK. j.cooper@medsch.ucl.ac.uk
Korlipara
L V P
LV
Hart
P E
PE
Bradley
J L
JL
Schapira
A H V
AH
D000293:Adolescent; D000328:Adult; D000975:Antioxidants / Q000008:administration & dosage; D004305:Dose-Response Relationship, Drug; D045222:Electron Transport Chain Complex Proteins / Q000235:genetics / Q000378:metabolism; D023381:Endpoint Determination; D004734:Energy Metabolism / Q000187:drug effects / Q000502:physiology; D005260:Female; D005621:Friedreich Ataxia / Q000188:drug therapy* / Q000378:metabolism / Q000503:physiopathology; D006801:Humans; D008297:Male; D008928:Mitochondria / Q000235:genetics / Q000378:metabolism; D054792:Muscle, Striated / Q000378:metabolism / Q000503:physiopathology; D018384:Oxidative Stress / Q000187:drug effects / Q000502:physiology; D011237:Predictive Value of Tests; D016896:Treatment Outcome; D014451:Ubiquinone / Q000008:administration & dosage / Q000031:analogs & derivatives* / Q000097:blood / Q000172:deficiency; D015854:Up-Regulation / Q000187:drug effects / Q000502:physiology; D014810:Vitamin E / Q000008:administration & dosage*; D014811:Vitamin E Deficiency / Q000097:blood / Q000188:drug therapy* / Q000503:physiopathology; D055815:Young Adult
D000975:Antioxidants; D045222:Electron Transport Chain Complex Proteins; D014451:Ubiquinone; D014810:Vitamin E; C024989:coenzyme Q10