Treatment for mitochondrial disorders.

Participants: males and females of any age with confirmed mitochondrial disease based on muscle histochemistry, respiratory chain complex analysis of tissues or cell lines, or DNA studies. Study design: randomized controlled trials (including cross-over).

Coenzyme Q10 600 mg orally twice daily for 60 days; Creatine monohydrate 3 g daily with 2 g dextrose, 300 mg alpha-lipoic acid, and 120 mg coenzyme Q10, taken daily for two months; Dichloroacetate (DCA) regimens included: 25 mg/kg twice daily for 1 week; 25 mg/kg twice daily for 15 days; and 12.5 mg/kg every 12 hours for six months; Dimethylglycine (DMG) 50 mg/kg/day in three divided doses for <33 kg or 5 g/day in three divided doses for ≥33 kg for three days; Whey-based cysteine supplement 10 g/day for 30 days.

No clear evidence that any intervention improves clinical outcomes in mitochondrial disorders. Some regimens produced biochemical or physiological improvements but these did not translate into meaningful gains in muscle strength, endurance, or quality of life. Adverse events were uncommon except peripheral nerve toxicity with long-term DCA in adults. Therefore, no treatment can be recommended; further research should use homogeneous populations and clinically relevant, limited primary outcomes.

Small, heterogeneous studies with diverse diseases and regimens; risk of bias unclear in several domains; cross-over designs with potential carryover; varied and often non-clinical endpoints; no meta-analysis; short durations; limited statistical power.