Controlled clinical trial of dichloroacetate for treatment of congenital lactic acidosis in children
PW Stacpoole, DS Kerr, C Barnes, ST Bunch… - …, 2006 - publications.aap.org
OBJECTIVE. Open-label studies indicate that oral dichloroacetate (DCA) may be effective in
treating patients with congenital lactic acidosis. We tested this hypothesis by conducting the
first double-blind, randomized, control trial of DCA in this disease. METHODS. Forty-three
patients who ranged in age from 0.9 to 19 years were enrolled. All patients had persistent or
intermittent hyperlactatemia, and most had severe psychomotor delay. Eleven patients had
pyruvate dehydrogenase deficiency, 25 patients had 1 or more defects in enzymes of the …
treating patients with congenital lactic acidosis. We tested this hypothesis by conducting the
first double-blind, randomized, control trial of DCA in this disease. METHODS. Forty-three
patients who ranged in age from 0.9 to 19 years were enrolled. All patients had persistent or
intermittent hyperlactatemia, and most had severe psychomotor delay. Eleven patients had
pyruvate dehydrogenase deficiency, 25 patients had 1 or more defects in enzymes of the …
