EFFECTS OF DICHLOROACETATE IN 3 PATIENTS WITH MELAS

We present the clinical and laboratory effects of dichloroacetate (DCA ) in three children with mitochondrial myopathy, encephalopathy, lacti c acidosis, and strokelike episodes (MELAS) who had not responded to o ther medications. Administration of DCA lowered the elevated levels of lactate and pyruvate in the serum and CSF. DCA ameliorated abdominal pain, headache, and strokelike episodes, and improved cognitive functi on and fatigability in the three patients during the study period. Som e transient liver dysfunction, hypocalcemia, and peripheral neuropathy were observed. The use of DCA in MELAS merits further study.