Outcome of familial hypercholesterolemia at Vietnam National Children’s Hospital
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Abstract
Familial hypercholesterolemia is a genetic disease that leads to elevated LDL-C, resulting in the development of atherosclerosis and premature coronary heart disease. The study aimed to evaluate the outcomes of patients with familial hypercholesterolemia treated at Vietnam National Children's Hospital from March 2015 to September 2024. A total of 24 children with familial hypercholesterolemia were identified by genetic testing, 17 of whom required lipid-lowering medication. The median age at treatment was 10 years old (2.7 - 15.7). Twelve patients with heterozygous familial hypercholesterolemia responded well to moderate or high-dose statin therapy alone, reducing LDL-C by 41.3% and total cholesterol by 35%, with 6/12 patients reaching the treatment goal. Five patients with homozygous/compound heterozygous familial hypercholesterolemia responded poorly to moderate or high-dose statin therapy combined with the cholesterol absorption inhibitor ezetimibe, with only 1/5 patients reaching the treatment goal. GOT, GPT, and CK concentrations in the 17 patients did not increase when treated with lipid-lowering medication.
Article Details
Keywords
Familial hypercholesterolemia, outcome
References
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