Abstract：
BACKGROUND: The 2019 European guidelines (ESC/EAS) for the treatment of dyslipidaemias recommend more aggressive targets for low-density lipoprotein cholesterol (LDL-C) in patients with familial hypercholesterolemia (FH). Current lipid-lowering treatment is often inadequate to achieve these targets.
METHODS: Data from the HELLAS-FH registry were analysed to assess achievement of LDL-C targets in adults with FH based on the 2019 ESC/EAS guidelines. In patients who had not achieved LDL-C target, the maximally reduced LDL-C value was calculated after theoretical switch to rosuvastatin/ezetimibe 40/10 mg/day. The percentage of patients who remained candidates for proprotein convertase subtilisin/kexin type 9 inhibitors (PCSK9i) was then calculated.
RESULTS: Patients (n = 1694, mean age 50.8 ± 14.7 years) had LDL-C levels 242 ± 71 mg/dL (6.3 ± 1.8 mmol/L) at diagnosis. Most treated patients were receiving statins (97.5%) and about half were on additional ezetimibe (47.5%). Based on the 2019 ESC/EAS guidelines the percentage of patients achieving LDL-C goals was only 2.7%. Following theoretical up titration to rosuvastatin/ezetimibe 40/10 mg, LDL-C target achievement rate would increase to 5.9%. In this scenario, most patients (55.9%) would be eligible for PCSK9i treatment. Following theoretical administration of a PCSK9i, LDL-C target achievement rate would rise to 57.6%. However, 42.4% of patients would still be eligible for further LDL-C lowering treatment.
CONCLUSIONS: Most FH patients do not reach new LDL-C targets even if on maximum intensity statin/ezetimibe treatment. In this case, more than half of FH patients are candidates for PCSK9i therapy and a considerable proportion may still require additional LDL-C lowering.
METHODS: Data from the HELLAS-FH registry were analysed to assess achievement of LDL-C targets in adults with FH based on the 2019 ESC/EAS guidelines. In patients who had not achieved LDL-C target, the maximally reduced LDL-C value was calculated after theoretical switch to rosuvastatin/ezetimibe 40/10 mg/day. The percentage of patients who remained candidates for proprotein convertase subtilisin/kexin type 9 inhibitors (PCSK9i) was then calculated.
RESULTS: Patients (n = 1694, mean age 50.8 ± 14.7 years) had LDL-C levels 242 ± 71 mg/dL (6.3 ± 1.8 mmol/L) at diagnosis. Most treated patients were receiving statins (97.5%) and about half were on additional ezetimibe (47.5%). Based on the 2019 ESC/EAS guidelines the percentage of patients achieving LDL-C goals was only 2.7%. Following theoretical up titration to rosuvastatin/ezetimibe 40/10 mg, LDL-C target achievement rate would increase to 5.9%. In this scenario, most patients (55.9%) would be eligible for PCSK9i treatment. Following theoretical administration of a PCSK9i, LDL-C target achievement rate would rise to 57.6%. However, 42.4% of patients would still be eligible for further LDL-C lowering treatment.
CONCLUSIONS: Most FH patients do not reach new LDL-C targets even if on maximum intensity statin/ezetimibe treatment. In this case, more than half of FH patients are candidates for PCSK9i therapy and a considerable proportion may still require additional LDL-C lowering.
摘要：
背景：关于治疗血脂异常的2019年欧洲指南（ESC/EAS）推荐了家族性高胆固醇血症（FH）患者的低密度脂蛋白胆固醇（LDL-C）更积极的目标。目前的降脂治疗通常不足以实现这些目标。
方法：根据2019年ESC/EAS指南，分析了HELLAS-FH注册数据，以评估FH成人LDL-C目标的实现情况。在未达到LDL-C目标的患者中，在理论转换为瑞舒伐他汀/依泽替米贝40/10mg/d后,计算LDL-C的最大降低值.然后计算仍然是前蛋白转化酶枯草杆菌蛋白酶/kexin9型抑制剂(PCSK9i)候选者的患者的百分比。
结果：患者（n=1694，平均年龄50.8±14.7岁）在诊断时LDL-C水平为242±71mg/dL（6.3±1.8mmol/L）。大多数接受治疗的患者正在接受他汀类药物（97.5％），约一半的患者正在接受其他依泽替米贝（47.5％）。根据2019年ESC/EAS指南，达到LDL-C目标的患者比例仅为2.7％。按照瑞舒伐他汀/依泽替米贝40/10mg的理论滴定，LDL-C目标达标率将增至5.9%。在这种情况下，大多数患者(55.9%)符合PCSK9i治疗的条件.在理论上管理PCSK9i之后，LDL-C目标完成率将上升至57.6%。然而,42.4％的患者仍有资格接受进一步的LDL-C降低治疗。
结论：大多数FH患者即使接受最大强度他汀类药物/依泽替米贝治疗，也无法达到新的LDL-C指标。在这种情况下,超过一半的FH患者是PCSK9i治疗的候选人,相当一部分患者可能仍需要额外降低LDL-C.
方法：根据2019年ESC/EAS指南，分析了HELLAS-FH注册数据，以评估FH成人LDL-C目标的实现情况。在未达到LDL-C目标的患者中，在理论转换为瑞舒伐他汀/依泽替米贝40/10mg/d后,计算LDL-C的最大降低值.然后计算仍然是前蛋白转化酶枯草杆菌蛋白酶/kexin9型抑制剂(PCSK9i)候选者的患者的百分比。
结果：患者（n=1694，平均年龄50.8±14.7岁）在诊断时LDL-C水平为242±71mg/dL（6.3±1.8mmol/L）。大多数接受治疗的患者正在接受他汀类药物（97.5％），约一半的患者正在接受其他依泽替米贝（47.5％）。根据2019年ESC/EAS指南，达到LDL-C目标的患者比例仅为2.7％。按照瑞舒伐他汀/依泽替米贝40/10mg的理论滴定，LDL-C目标达标率将增至5.9%。在这种情况下，大多数患者(55.9%)符合PCSK9i治疗的条件.在理论上管理PCSK9i之后，LDL-C目标完成率将上升至57.6%。然而,42.4％的患者仍有资格接受进一步的LDL-C降低治疗。
结论：大多数FH患者即使接受最大强度他汀类药物/依泽替米贝治疗，也无法达到新的LDL-C指标。在这种情况下,超过一半的FH患者是PCSK9i治疗的候选人,相当一部分患者可能仍需要额外降低LDL-C.
