Controlled Clinical Trial of Dichloroacetate for Treatment of Congenital Lactic Acidosis in Children

  title={Controlled Clinical Trial of Dichloroacetate for Treatment of Congenital Lactic Acidosis in Children},
  author={Peter W. Stacpoole and D. Steven Kerr and Carie L Barnes and S Terri Bunch and Paul R. Carney and Eileen Fennell and Natalia M Felitsyn and Robin L. Gilmore and Melvin Greer and George N. Henderson and Alan David Hutson and Richard E. Neiberger and R O'Brien and Leigh Ann Perkins and Ronald G. Quisling and Albert L. Shroads and Jonathan J Shuster and Janet H Silverstein and Douglas W. Theriaque and Edward Valenstein},
  pages={1519 - 1531}
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… 

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