Dichloroacetate treatment for mitochondrial cytopathy: Long-term effects in MELAS

Brain and Development

Volumn 26, Issue 7, 2004, Pages 453-458

  Mori, Masato ^a   Yamagata, Takanori ^a   Goto, Tamako ^a   Saito, Shigeko ^a   Momoi, Mariko Y ^a  

^a JICHI MEDICAL UNIVERSITY   (Japan)

Author keywords

Dichloroacetate; Long term effects; Mitochondrial encephalomyelopathy with lactic acidosis and stroke like episodes; Treatment

Indexed keywords

DICHLOROACETIC ACID; LACTIC ACID; PYRUVIC ACID; THIAMINE;

ABDOMINAL PAIN; ADOLESCENT; ARTICLE; CASE REPORT; CHILD; CONTROLLED STUDY; DISORDERS OF MITOCHONDRIAL FUNCTIONS; DRUG EFFECT; ELECTROPHYSIOLOGY; FEMALE; GENE MUTATION; HEADACHE; HEARING IMPAIRMENT; HUMAN; HYPOCALCEMIA; LACTATE BLOOD LEVEL; LACTIC ACIDOSIS; LIVER DYSFUNCTION; LONG TERM CARE; MELAS SYNDROME; MENTAL HEALTH; MUSCLE WEAKNESS; PERIPHERAL NEUROPATHY; SHORT STATURE; SIDE EFFECT; SYMPTOM; TREATMENT OUTCOME;

ABDOMINAL PAIN; ADMINISTRATION, ORAL; ADOLESCENT; CEREBROVASCULAR ACCIDENT; CHILD; DICHLOROACETATE; FEMALE; HEADACHE; HUMANS; HYPOCALCEMIA; LACTIC ACID; LIVER DISEASES; MELAS SYNDROME; MITOCHONDRIA; MUSCLE WEAKNESS; PERIPHERAL NERVOUS SYSTEM DISEASES; PYRUVIC ACID; TIME FACTORS; TREATMENT OUTCOME;

EID: 4444228301     PISSN: 03877604     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.braindev.2003.12.009     Document Type: Article

References (32)

1. Pavlakis S.G., Rowland L.P., DeVivo D.L., Rowland L.P. Mitochondrial myopathy, encephalopathy, lactic acidosis, and stroke-like episodes: a distinctive clinical syndrome associated with ragged-red fibers. Ann Neurol. 16:1984;481-488
2. Goto Y., Nonaka I., Horai S. A mutation in the tRNALeu (UUR) gene associated with the MELAS subgroup of mitochondrial encephalomyopathies. Nature. 348:1990;651-653
3. Tanaka J., Nagai T., Arai H., Inui K., Yamanouchi H., Goto Y., et al. Treatment of mitochondrial encephalomyopathy with a combination of cytochrome C and vitamins B1 and B2. Brain Dev. 19:1997;262-267
4. Kuroda Y., Ito M., Naito E., Yokota I., Matsuda J., Saijo T., et al. Concomitant administration of sodium dichloroacetate and vitamin B1 for lactic acidemia in children with MELAS syndrome. J Pediatr. 131:1997;450-452
5. Yamamoto M., Sato T., Anno M., Ujike H., Takemoto M. Mitochondrial myopathy, encephalopathy, lactic acidosis, and stroke-like episodes with recurrent abdominal symptoms and coenzyme Q10 administration. J Neurol Neurosurg Psychiatry. 50:1987;1475-1481
6. Ihara Y., Namba R., Kuroda S., Sato T., Shirabe T. Mitochondrial encephalopathy (MELAS): pathological study and successful therapy with coenzyme Q10 and idebenone. J Neurol Sci. 90:1989;263-271
7. Chen R.S., Huang C.C., Chu N.C. Coenzyme Q10 treatment in mitochondrial encephalomyopathies. Eur Neurol. 37:1997;212-218
8. Abe K., Matsuo Y., Kadekawa J., Inoue S., Yanagihara T. Effect of coenzyme Q10 in patients with mitochondrial myopathy, encephalopathy, lactic acidosis, and stroke-like episodes (MELAS): evaluation by noninvasive tissue oximetry. J Neurol Sci. 162:1999;65-68
9. Kuroda Y., Ito M., Toshima K., Takeda E., Naito E., Hwang T.J., et al. Treatment of chronic congenital lactic acidosis by oral administration of dichloroacetate. J Inherit Metab Dis. 9:1986;244-252
10. Toth P.P., El-Shanti H., Eivins S., Rheas W.J., Klein J.M. Transient improvement of congenital lactic acidosis in a male infant with pyruvate decarboxylase deficiency treated with dichloroacetate. J Pediatr. 123:1993;427-430
11. Byrd D.J., Krohn H.P., Winkler L., Steinborn C., Hadam M., Brodehl J., et al. Neonatal pyruvate dehydrogenase deficiency with lipoate responsive lactic acidaemia and hyperammonaemia. Eur J Pediatr. 148:1989;543-547
12. Kimura S., Osaka H., Saitou K., Otsuki N., Kobayashi T., Nezu A. Improvement of lesions shown on MRI and CT scan by administration of dichloroacetate in patients with Leigh syndrome. J Neurol Sci. 134:1995;103-107
13. Cragen W.J. Leigh disease with deficiency of lipoamide dehydrogenase: treatment failure with dichloroacetate. Pediatr Neurol. 14:1996;69-71
14. Takanashi J., Sugita K., Tanabe Y., Maemoto T., Niimi H. Dichloroacetate treatment in Leigh syndrome caused by mitochondrial DNA mutation. J Neurol Sci. 145:1997;83-86
15. Kimura S., Otsuki N., Nezu A., Tanaka M., Takeshita S. Clinical and radiologic improvements in mitochondrial encephalomyopathy following sodium dichloroacetate therapy. Brain Dev. 19:1997;535-540
16. Stacpoole P.W., Lorenz A.C., Thomas R.G., Harman E.M. Dichloroacetate in the treatment of lactic acidosis. Ann Intern Med. 108:1988;58-63
17. Stacpoole P.W., Wright E.C., Baumgartner T.G., Bersin R.M., Buchalter S., Curry S.H., et al. A controlled clinical trial of dichloroacetate for treatment of lactic acidosis in adults. N Engl J Med. 327:1992;1564-1569
18. Stacpoole P.W., Greene Y.J. Dichloroacetate. Diabetes Care. 15:1992;785-791
19. Bersin R.M., Stacpoole P.W. Dichloroacetate as metabolic therapy for myocardial ischemia and failure. Am Heart J. 134:1997;841-855
20. Lewis J.F., DaCosta M., Wargowich T., Stacpoole P. Effects of dichloroacetate in patients with congestive heart failure. Clin Cardiol. 21:1998;888-892
21. Stacpoole P.W., Henderson G.N., Yan Z., James M.O. Clinical pharmacology and toxicology of dichloroacetate. Environ Health Perspect. 106:(Suppl 4):1998;989-994
22. Saitoh S., Momoi M.Y., Yamagata T., Mori Y., Imai M. Effects of dichloroacetate in three patients with MELAS. Neurology. 50:1998;531-534
23. Przyrembel H. Therapy of mitochondrial disorders. J Inherit Metab Dis. 10:1987;129-146
24. Nakagawa E., Osari S., Yamanouchi H., Matsuda H., Goto Y., Nonaka I. Long-term therapy with cytochrome c, flavin mononucleotide and thiamine diphosphate for a patient with Kearns-Sayre syndrome. Brain Dev. 18:1996;68-70
25. DeStefano N., Matthews P.M., Ford B., Genge A., Karpati G., Arnold D.L. Short-term dichloroacetate treatment improves indices of cerebral metabolism in patients with mitochondrial disorders. Neurology. 46:1995;1586-1590
26. Saijo T., Naito E., Ito M., Takeda E., Hashimoto T., Kuroda Y. Therapeutic effect of sodium dichloroacetate on visual and auditory hallucinations in a patient with MELAS. Neuropediatrics. 22:1991;166-167
27. Chandy M.J., Ravindra J. Effect of dichloracetate on infarct size in a primate model of focal cerebral ischaemia. Neurol India. 48:(3):2000;227-230
28. Taniguchi M., Wilson C., Hunter C.A., Pehowich D.J., Clanachan A.S., Lopaschuk G.D. Dichloroacetate improves cardiac efficiency after ischemia independent of changes in mitochondrial proton leak. Am J Physiol Heart Circ Physiol. 280:(4):2001;H1762-H1769
29. DeAngelo A.B., Daniel F.B., Most B.M., Olson G.R. The carcinogenicity of dichloroacetic acid in the male Fischer 344 rat. Toxicology. 114:1996;207-221
30. Chen C.W. Biologically based dose-response model for liver tumors induced by trichloroethylene. Environ Health Perspect. 108:(Suppl 2):2000;335-342
31. Walgren J.E., Kurtz D.T., McMillan J.M. The effect of the trichloroethylene metabolites trichloroacetate and dichloroacetate on peroxisome proliferation and DNA synthesis in cultured human hepatocytes. Cell Biol Toxicol. 16:(4):2000;257-273
32. Kato-Weinstein J., Lingohr M.K., Orner G.A., Thrall B.D., Bull R.J. Effects of dichloroacetate on glycogen metabolism in B6C3F1 mice. Toxicology. 130:1998;141-154