一名4.5岁纯合子家族性高胆固醇血症儿童的低密度脂蛋白单采术

LDL apheresis in a homozygous familial hypercholesterolemic child aged 4.5.

作者信息

Stefanutti C, Notarbartolo A, Colloridi V, Nigri A, Vivenzio A, Bertolini S, Bosco G, Berni A, Di Giacomo S, Mazzarella B

机构信息

Istituto di Terapia Medica Sistematica, University of Rome La Sapienza, Policlinico Umberto I, Italy.

出版信息

Artif Organs. 1997 Oct;21(10):1126-7. doi: 10.1111/j.1525-1594.1997.tb00453.x.

DOI:10.1111/j.1525-1594.1997.tb00453.x

PMID:9335372

Abstract

Preliminary experience with the efficacy and safety of dextran sulfate cellulose low-density lipoprotein (LDL) apheresis for the treatment of a 4.5-year-old girl with homozygous familial hypercholesterolemia and coronary artery disease is reported. The decrease of the most atherogenic apolipoprotein B-containing lipoproteins, low-density lipoprotein (LDL) and lipoprotein(a) (Lp [a]), were in the ranges of 63.1-68.7%, and 52.5-58.6%, respectively. The child tolerated LDL apheresis without any clinically significant complications. Therefore, she was submitted to a long-term program of treatment at intervals of 15 days. The experience suggests the possibility of an early beginning of extracorporeal treatment with LDL apheresis in children severely affected by homozygous or double heterozygous familial hypercholesterolemia.

摘要

报告了硫酸葡聚糖纤维素低密度脂蛋白（LDL）血液成分分离术治疗一名4.5岁纯合子家族性高胆固醇血症和冠状动脉疾病女孩的疗效和安全性的初步经验。最具致动脉粥样硬化性的含载脂蛋白B的脂蛋白，即低密度脂蛋白（LDL）和脂蛋白(a) [Lp(a)] 的降低幅度分别在63.1% - 68.7%和52.5% - 58.6%范围内。该患儿耐受LDL血液成分分离术，未出现任何具有临床意义的并发症。因此，她接受了为期15天的长期治疗方案。该经验表明，对于受纯合子或双重杂合子家族性高胆固醇血症严重影响的儿童，早期开始进行LDL血液成分分离术体外治疗是有可能的。