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Long-term safety of dichloroacetate in congenital lactic acidosis.
- Source :
-
Molecular genetics and metabolism [Mol Genet Metab] 2013 Jun; Vol. 109 (2), pp. 139-43. Date of Electronic Publication: 2013 Apr 06. - Publication Year :
- 2013
-
Abstract
- We followed 8 patients (4 males) with biochemically and/or molecular genetically proven deficiencies of the E1α subunit of the pyruvate dehydrogenase complex (PDC; 3 patients) or respiratory chain complexes I (1 patient), IV (3 patients) or I+IV (1 patient) who received oral dichloroacetate (DCA; 12.5 mg/kg/12 h) for 9.7 to 16.5 years. All subjects originally participated in randomized controlled trials of DCA and were continued on an open-label chronic safety study. Patients (1 adult) ranged in age from 3.5 to 40.2 years at the start of DCA administration and are currently aged 16.9 to 49.9 years (mean ± SD: 23.5 ± 10.9 years). Subjects were either normal or below normal body weight for age and gender. The 3 PDC deficient patients did not consume high fat (ketogenic) diets. DCA maintained normal blood lactate concentrations, even in PDC deficient children on essentially unrestricted diets. Hematological, electrolyte, renal and hepatic status remained stable. Nerve conduction either did not change or decreased modestly and led to reduction or temporary discontinuation of DCA in 3 patients, although symptomatic worsening of peripheral neuropathy did not occur. We conclude that chronic DCA administration is generally well-tolerated in patients with congenital causes of lactic acidosis and is effective in maintaining normal blood lactate levels, even in PDC-deficient children not consuming strict ketogenic diets.<br /> (Copyright © 2013 Elsevier Inc. All rights reserved.)
- Subjects :
- Acidosis, Lactic blood
Acidosis, Lactic congenital
Adolescent
Adult
Child
Child, Preschool
Dichloroacetic Acid administration & dosage
Female
Humans
Lactic Acid blood
Male
Middle Aged
Randomized Controlled Trials as Topic
Treatment Outcome
Young Adult
Acidosis, Lactic drug therapy
Dichloroacetic Acid adverse effects
Subjects
Details
- Language :
- English
- ISSN :
- 1096-7206
- Volume :
- 109
- Issue :
- 2
- Database :
- MEDLINE
- Journal :
- Molecular genetics and metabolism
- Publication Type :
- Academic Journal
- Accession number :
- 23611579
- Full Text :
- https://doi.org/10.1016/j.ymgme.2013.03.019