PMID: 9440797Jan 24, 1998Paper

Clinical and radiologic improvements in mitochondrial encephalomyelopathy following sodium dichloroacetate therapy

Brain & Development
S KimuraS Takeshita

Abstract

We administered sodium dichloroacetate (DCA), which reduces the circulating lactate and pyruvate concentrations by stimulating the activity of the pyruvate dehydrogenase complex (PDHC), to three children with mitochondrial encephalomyelopathy. Significant clinical, biochemical and radiologic improvements were obtained following DCA therapy (approximately 30 mg/kg per day, divided into three doses). All three patients had non-pyruvate dehydrogenase complex (PDHC) deficiencies: two exhibited Leigh syndrome (complex I deficiency and unknown etiology), and one abnormal myelination (multienzyme deficiency), demonstrated on magnetic resonance imaging (MRI). The lactic and pyruvic acid concentrations in serum and cerebrospinal fluid (CSF) were decreased significantly by the oral DCA treatment. The lactic acid peak on MR spectroscopy also markedly decreased in parallel with the CSF level. In addition, the brain lesions observed on MRI were improved in all patients. No exacerbation was observed in any of the patients, who have been followed-up more than 21 months following the DCA therapy. These results suggest that DCA therapy should be considered in all patients with a mitochondria-related enzyme deficiency.

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Citations

Feb 27, 2004·Current Neurology and Neuroscience Reports·Andrea Gropman
Apr 3, 2001·Current Treatment Options in Neurology·Roser Pons, Darryl C. De Vivo
Aug 6, 2002·Pediatric Neurology·Tatsuya FujiiTakehiko Okuno
Mar 18, 2006·Neuroimaging Clinics of North America·Kim M Cecil
Sep 8, 2010·Developmental Disabilities Research Reviews·Seth D FriedmanRussell P Saneto
Mar 12, 2010·Journal of Applied Toxicology : JAT·Ezdihar A HassounJessica Spildener
Oct 7, 2004·Molecular Genetics and Metabolism·Bruce A BarshopRichard H Haas
Jul 2, 2008·Mitochondrion·Russell P SanetoDennis W W Shaw
May 22, 2008·Journal of Applied Toxicology : JAT·Ezdihar A Hassoun, Jatin Mehta
Apr 22, 2009·Pediatrics International : Official Journal of the Japan Pediatric Society·Yoshiki KatsumiTohru Sugimoto
Jul 8, 2000·Brain Pathology·G Manfredi, M F Beal

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