Lysosomal Disorders Unit, Royal Free Hospital.
Centre for Cardiovascular Genetics, Institute Cardiovascular Science, University College London, London, UK.
Curr Opin Pediatr. 2020 Oct;32(5):633-640. doi: 10.1097/MOP.0000000000000943.
All guidelines for the management of heterozygous familial hypercholesterolaemia in children and young people recommend statins to lower LDL-cholesterol (LDL-C) concentrations, to reduce the individual's adult risk of developing cardiovascular disease (CVD). Here, we review recent findings regarding the efficacy and safety of the use of stains in childhood.
As expected from their safety profile in adults, there is no evidence from short-term trials or long-term follow-up that statin use in children is associated with any adverse effects on growth, pubertal development or muscle or liver toxicity. Long-term follow-up indicates benefits with respect to lower CVD rates. Factors that influence adherence are discussed, as is the role of the underlying genetic causes for hypercholesterolaemia and of variation at other genes in determining the LDL-C-lowering effect.
Based on the good safety profile, and the expert opinion guidelines, clinicians should consider prescribing statins for children with hypercholesterolaemia from the age of at least 10 years (and earlier if CVD risk is particularly high in the family). Uptitrating statin dosage and the use of additional lipid-lowering therapies should be considered so that LDL-C concentrations are lowered to recommended targets.
所有关于杂合子家族性高胆固醇血症儿童和青少年管理的指南都建议使用他汀类药物降低 LDL 胆固醇(LDL-C)浓度,以降低个体成年后患心血管疾病(CVD)的风险。在这里,我们回顾了他汀类药物在儿童中的疗效和安全性的最新发现。
由于他汀类药物在成人中的安全性,短期试验或长期随访均未发现他汀类药物在儿童中使用与生长、青春期发育或肌肉或肝脏毒性的任何不良影响相关。长期随访表明其对降低 CVD 发生率有获益。讨论了影响依从性的因素,以及高胆固醇血症的潜在遗传原因和其他基因变异在确定 LDL-C 降低效果方面的作用。
基于良好的安全性和专家意见指南,临床医生应考虑从至少 10 岁(如果家族中 CVD 风险特别高,则更早)开始为高胆固醇血症的儿童开他汀类药物。应考虑增加他汀类药物剂量和使用其他降脂治疗,以使 LDL-C 浓度降低至推荐目标。