Vissing 2001.
| Methods | Randomised, placebo‐controlled, double‐blind cross‐over | |
| Participants | 8 participants (3 CPEO, 1 MERRF, 5 mitochondrial myopathy) | |
| Interventions | Dichloroacetate 25 mg/kg twice daily or placebo for 15 days. Washout of 6 weeks in all participants | |
| Outcomes | VO2, workload and blood pressure during cycle ergometry, with measurements of venous lactate and pyruvate. 31P‐MRS of forearm muscles before and after forearm exercise was measured. All participants filled a 20‐item questionnaire commenting on activity levels and symptoms. Outcome measurements were performed before and at the end of each treatment phase | |
| Notes | Normalisation of resting and exercise lactate and pyruvate levels. All other outcomes were non‐significant or did not favour treatment. One participant withdrew due to sedation on treatment | |
| 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 | Low risk | All participants completed 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 | This was a double‐blind study. Placebo was "the same volume, color and flavor as the DCA mixture." |
| Blinding of outcome assessment (detection bias) All outcomes | Low risk | Participants were randomly assigned to start in either the treatment or placebo arm |
ADL: activities of daily living
ATP: adenosine triphosphate
CPEO: chronic progressive external ophthalmoplegia
DCA: dichloroacetate
DMG: dimethylglycine
GATE: Global Assessment of Treatment Efficacy score, based on a health‐related inventory, neurological and psychological evaluation, and activities of daily living
1H‐MRS: proton magnetic resonance spectroscopy
KSS: Kearns‐Sayre syndrome
MELAS: mitochondrial encephalomyopathy with lactic acidosis and stroke‐like episodes
MERRF: myoclonic epilepsy with ragged‐red fibres
MRC: UK Medical Research Council
mtDNA: mitochondrial DNA
NIDFT: nonischaemic isometric dorsiflexion torque 31P‐MRS: phosphorus magnetic resonance spectroscopy
PCr: phosphocreatine
SD: standard deviation
SF‐36 Short Form 36 Health Survey
SLSJ‐COX: Saguenay‐Lac‐Saint‐Jean cytochrome c oxidase deficiency
VO2: oxygen consumption