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乳糜微粒血症的治疗。

Treatment of chylomicronemia.

机构信息

Unidad de Lípidos, Servicio de Medicina Interna, Hospital Virgen de la Victoria, Málaga, España.

Unidad de Lípidos, Servicio de Medicina Interna, Hospital Virgen de la Victoria, Málaga, España; Departamento de Medicina y Dermatología, Universidad de Málaga, Málaga, España; Instituto de Investigación Biomédica de Málaga, Málaga, España.

出版信息

Clin Investig Arterioscler. 2021 May;33 Suppl 2:75-79. doi: 10.1016/j.arteri.2021.01.004.

Abstract

Fasting chylomicronaemia appears in type V (multifactorial chylomicronaemia syndrome, MCS), and in type I (familial chylomicronaemia syndrome, FCS). MCS needs to be treated as in any general hypertriglyceridaemia: low-calorie diet, avoid sugar and alcohol, reduce body weight, control of diabetes and, in some cases, common lipid lowering-drugs, such as fibrates or omega-3 fatty acids. For type I HLP, FCS, patients should adhere to a strict very low fat diet, usually less than 15-20 g per day. In spite of this, many patients with FCS suffer from recurrent abdominal pain and/or acute pancreatitis. Volanesorsen, an antisense oligonucleotide against apolipoprotein C-III, is the only drug approved to control the disease. As shown in the APPROACH study, the administration of volanesorsen at a weekly dose of 285 mg induced at three month a reduction of triglycerides of 77% (primary end-point) and a reduction of 1712 mg/dL from the baseline. Among patient receiving volanesorsen, 77% reached a fasting triglyceride value below 750 mg/dL. The most frequent side effects were a skin reaction at injection site and low platelet levels, which should be monitored.

摘要

空腹乳糜微粒血症见于 V 型(多因素乳糜微粒血症综合征,MCS)和 I 型(家族性乳糜微粒血症综合征,FCS)。MCS 需要像治疗任何一般高甘油三酯血症一样进行治疗:低热量饮食,避免糖和酒精,减轻体重,控制糖尿病,在某些情况下,使用常见的降脂药物,如贝特类或ω-3 脂肪酸。对于 I 型高脂蛋白血症,FCS 患者应坚持严格的低脂饮食,通常每天少于 15-20 克。尽管如此,许多 FCS 患者仍会反复发作腹痛和/或急性胰腺炎。Volanesorsen 是一种针对载脂蛋白 C-III 的反义寡核苷酸,是唯一获准控制该疾病的药物。如 APPROACH 研究所示,每周 285 毫克的 volanesorsen 给药在三个月时可使甘油三酯降低 77%(主要终点),并使基线时的甘油三酯降低 1712 毫克/分升。在接受 volanesorsen 治疗的患者中,77%的患者空腹甘油三酯值低于 750 毫克/分升。最常见的副作用是注射部位皮肤反应和血小板计数低,应进行监测。

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