DCA treatment of 53 infants and children with CLA
No of evaluable subjects | ||
---|---|---|
Sex (M/F) | 34/17 | 51 |
Mean (range) age at start of treatment | 5.4 years (2 days-18 years) | 52 |
Mean (range) dose (mg/kg/day) | 85 (10-1500) | 39 |
Mean (range) treatment duration | 11 months (1 day-5 years) | 45 |
Response (yes/no)2-150 | ||
Biochemical | 40/11 (78%) | 51 |
Clinical | 15/24 (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.