| Methods |
Randomised, placebo‐controlled, double‐blind cross‐over |
| Participants |
11 participants (4 myopathy, 1 CPEO, 2 KSS, 2 Leigh syndrome, one MELAS) |
| Interventions |
Dichloroacetate 25 mg/kg twice daily or placebo for one week. 3 month washout |
| Outcomes |
Neurological exam, dynamometry, gait evaluation, venous blood lactate, alanine and pyruvate at rest and on incremental exercise; 31P‐MRS of muscle and 1H‐MRS of the brain |
| Notes |
Statistically significantly decreased blood lactate, pyruvate and alanine at rest and after exercise (P < 0.05), and statistically significant improvements on brain MRS (brain lactate/creatine ratio fell by 42%, P < 0.05; brain choline/creatine ratio increased by 18%, P < 0.01; brain acetylaspartate/creatine ratio increased by 18%, P < 0.05) (SD not provided for these values) |
| Risk of bias |
| Bias |
Authors' judgement |
Support for judgement |
| Random sequence generation (selection bias) |
Unclear risk |
Method of randomisation not specified |
| Allocation concealment (selection bias) |
Unclear risk |
Method of allocation not specified |
| Incomplete outcome data (attrition bias)
All outcomes |
Unclear risk |
Not all participants underwent all outcome assessments |
| Selective reporting (reporting bias) |
Low risk |
All outcome measures in methods section were reported in results section |
| Other bias |
Low risk |
None identified |
| Blinding of participants and personnel (performance bias)
All outcomes |
Low risk |
Groups were "randomized and unknown to the patient, physicians, or other evaluators." |
| Blinding of outcome assessment (detection bias)
All outcomes |
Low risk |
"Each patient underwent two treatment courses, one with DCA and one with placebo. The order of these was randomized..." |
