Dichloroacetate causes toxic neuropathy in MELAS: A randomized, controlled clinical trial
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To the Editor:
We read with interest the report by Kaufmann et al.1 The investigators studied the efficacy of dichloroacetate (DCA) in the treatment of 30 patients with mitochondrial myopathy, encephalopathy, lactic acidosis and stroke-like episodes (MELAS). At a DCA dose of 25 mg/kg/day, they detected no therapeutic benefit and documented peripheral nerve toxicity resulting in premature study termination. They conclude that peripheral nerve toxicity overshadows any potential benefit from DCA in patients harboring the A3243G mutation.
The high rate of DCA-induced peripheral neuropathy in the Kaufmann et al. study was unexpected because previous studies in a sample of 97 patients with congenital lactic acidosis (including 10 patients with MELAS) did not reveal a high incidence …
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