Table 2

DCA treatment of 53 infants and children with CLA

No of evaluable subjects
Sex (M/F)34/1751
Mean (range) age at start of treatment5.4 years (2 days-18 years)52
Mean (range) dose (mg/kg/day)85 (10-1500)39
Mean (range) treatment duration11 months (1 day-5 years)45
Response (yes/no)2-150
 Biochemical40/11 (78%)51
 Clinical15/24 (38%)39
  • 2-150 A biochemical response is defined as at least a 20% decrease in blood and/or cerebrospinal fluid lactate from pretreatment levels after DCA administration. A clinical response is defined as an improvement in exercise endurance or in neuromuscular, neurobehavioural or cardiac function, stimulation of linear growth, or reduction in the severity and/or frequency of acid-base decompensation.