Multicenter trial with ubidecarenone: treatment of 44 patients with mitochondrial myopathies.
Fourty four patients with mitochondrial myopathies were treated with Ubidecarenone (CoQ10) for six months in an open multicentric trial. No side effects due to the drug administration were observed. Sixteen patients showing at least 25% decrease of post exercise lactate levels were selected as responders. Responsiveness was apparently not related to CoQ10 level in serum and platelets or to the presence or absence of mtDNA deletions. The responders were further treated for 3 months with CoQ10 or placebo in the second blind part of the trial; no significant differences between the 2 groups were observed. It is not clear why CoQ10 had therapeutic effects in some patients and not in others with the same clinical presentation and biochemical defect, and we failed to identify candidate responders before treatment. At the dosage of CoQ10 used in the study (2 mg/kg/day) the therapy requires long administration time before a response is demonstrable.
Scarlato
G
G
Institute of Clinical Neurology, University of Milan, Italy.
Bresolin
N
N
Moroni
I
I
Doriguzzi
C
C
Castelli
E
E
Comi
G
G
Angelini
C
C
Carenzi
A
A
D000293:Adolescent; D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D003067:Coenzymes; D004272:DNA, Mitochondrial / Q000235:genetics; D005260:Female; D006801:Humans; D007773:Lactates / Q000097:blood; D019344:Lactic Acid; D008297:Male; D008875:Middle Aged; D008931:Mitochondria, Muscle / Q000187:drug effects* / Q000201:enzymology; D009135:Muscular Diseases / Q000188:drug therapy* / Q000201:enzymology / Q000235:genetics; D014451:Ubiquinone / Q000031:analogs & derivatives* / Q000627:therapeutic use
D003067:Coenzymes; D004272:DNA, Mitochondrial; D007773:Lactates; D014451:Ubiquinone; D019344:Lactic Acid; C024989:coenzyme Q10