NEJM:他汀类药物对儿童家族性高胆固醇血症的长期影响

2019-10-17 MedSci MedSci原创

研究认为,家族性高胆固醇血症患者儿童时期开始使用他汀类药物,延缓了颈动脉内膜中层厚度的进展,降低了成年后患心血管疾病的风险

家族性高胆固醇血症的特点是严重升高的低密度脂蛋白(LDL)胆固醇和过早的心血管疾病。他汀类药物治疗儿童患者的短期疗效已得到证实,但较长期的心血管疾病风险变化评估尚不充分。

近日研究人员公布了20年他汀类药物治疗儿童家族性高胆固醇血症的疗效。214名患者参与研究,先前接受过2年的普伐他汀疗效研究。患者完成问卷调查,提供血样,并进行颈动脉内膜-中膜厚度测量,以无病兄弟及患病父母为对照。

研究期间,患者平均LDL胆固醇含量由237.3mg每分升下降至160.7mg每分升,较基线下降32%。37名患者(20%)实现LDL胆固醇含量小于100mg每分升的治疗目标。在整个随访期间,家族性高胆固醇血症患者颈动脉内中膜厚度的平均进展为每年0.0056毫米,对照兄弟姐妹的平均进展为每年0.0057毫米。家族性高胆固醇血症患者39岁时心血管事件和心血管疾病死亡的累积发生率低于其父母(1% vs. 26% ;0% vs. 7%)。

研究认为,家族性高胆固醇血症患者儿童时期开始使用他汀类药物,延缓了颈动脉内膜中层厚度的进展,降低了成年后患心血管疾病的风险。

原始出处:

Ilse K. Luirink et al. 20-Year Follow-up of Statins in Children with Familial Hypercholesterolemia. N Engl J Med, October 17, 2019.

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    2019-10-19 wetgdt
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    2019-10-17 旺医

    顶刊就是顶刊,谢谢梅斯带来这么高水平的研究报道,我们科里同事经常看梅斯,分享梅斯上的信息

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香港的两组包括治疗血脂异常及家族性高胆固醇血症的专家和儿科专科医生在2016年举行两次会议,讨论香港患有家族性高胆固醇血症的成年人和儿童的多方面管理。在回顾家族性高胆固醇血症的诊断、筛查和管理的临床数据及指引后,他们就家族性高胆固醇血症的管理在下列方面达成共识,包括临床特征、诊断标准、成人筛查、儿童筛查、管理血液低密度脂蛋白胆固醇的目标,动脉粥样硬化的检测、生活方式和行为改变,以及药物治疗。

JACC:每10位年轻心梗患者就有1人为家族性高胆固醇血症,心梗前四成人没用他汀

一提家族性高胆固醇血症,好多人都会觉得是一种遗传性疾病,普通人离得较远。但实际上在早发心梗人群中并不少见。

Regeneron的ANGPTL3单抗evinacumab治疗家族性高胆固醇血症的III期临床成功

Regeneron Pharmaceuticals宣布其ANGPTL3抗体evinacumab达到了III期ELIPSE HoFH研究的主要终点,与单纯降脂治疗相比,evinacumab将纯合子家族性高胆固醇血症(HoFH)患者的LDL胆固醇从基线降低49%。该公司表示,数据将于2020年向FDA提交。