Dichloroacetate treatment for mitochondrial cytopathy: long-term effects in MELAS.
The long-term effects of the sodium salt of dichloroacetic acid (DCA) were evaluated in four patients with mitochondrial encephalomyelopathy with lactic acidosis and stroke-like episodes (MELAS) carrying A3243G mutation. Oral administration of DCA in MELAS patients was followed for an average of 5 years 4 months. Serum levels of lactate and pyruvate were maintained at around 10 and 0.6 mg/dl, respectively. Serum levels of DCA were 40-136 microg/ml. Symptoms responding to treatment included persistent headache, abdominal pain, muscle weakness, and stroke-like episodes. In contrast, no improvements in mental status, deafness, short stature, or neuroelectrophysiological findings were observed. Adverse effects included mild liver dysfunction in all patients, hypocalcemia in three and peripheral neuropathy in one. None of these adverse events was severe enough to require discontinuation of treatment. To determine suitable indications for DCA therapy, analysis of many more patients who have undergone DCA administration is required.
Mori
Masato
M
Department of Pediatrics, Jichi Medical School, 3311-1 Yakushiji, Minamikawachi, Tochigi 329-0498, Japan. morim@jichi.ac.jp
Yamagata
Takanori
T
Goto
Tamako
T
Saito
Shigeko
S
Momoi
Mariko Y
MY
D015746:Abdominal Pain / Q000188:drug therapy; D000284:Administration, Oral; D000293:Adolescent; D056486:Chemical and Drug Induced Liver Injury; D002648:Child; D003999:Dichloroacetic Acid / Q000008:administration & dosage* / Q000009:adverse effects* / Q000097:blood; D005260:Female; D006261:Headache / Q000188:drug therapy; D006801:Humans; D006996:Hypocalcemia / Q000139:chemically induced; D019344:Lactic Acid / Q000097:blood; D017241:MELAS Syndrome / Q000097:blood / Q000188:drug therapy* / Q000503:physiopathology*; D008928:Mitochondria / Q000187:drug effects; D018908:Muscle Weakness / Q000188:drug therapy; D010523:Peripheral Nervous System Diseases / Q000139:chemically induced; D019289:Pyruvic Acid / Q000097:blood; D020521:Stroke / Q000188:drug therapy / Q000517:prevention & control; D013997:Time Factors; D016896:Treatment Outcome
D019344:Lactic Acid; D019289:Pyruvic Acid; D003999:Dichloroacetic Acid