Impact of evolocumab treatment on low-density lipoprotein cholesterol levels in heterozygous familial hypercholesterolemic patients withdrawing from regular apheresis

• Published in: Atherosclerosis Vol. 265; pp. 225 - 230
• Main Authors: Kawashiri, Masa-aki, Nohara, Atsushi, Higashikata, Toshinori, Tada, Hayato, Nakanishi, Chiaki, Okada, Hirofumi, Konno, Tetsuo, Sakata, Kenji, Hayashi, Kenshi, Inazu, Akihiro, Mabuchi, Hiroshi, Yamagishi, Masakazu
• Format: Journal Article
• Language: English
• Published: Ireland Elsevier B.V 01.10.2017
• Subjects: Aged; Aged, 80 and over; Antibodies, Monoclonal - therapeutic use; Anticholesteremic Agents - therapeutic use; Apolipoprotein B; Blood Component Removal; Cholesterol, LDL - blood; Cholesterol, LDL - drug effects; Familial hypercholesterolemia; Female; Heterozygote; Humans; Hyperlipoproteinemia Type II - blood; Hyperlipoproteinemia Type II - drug therapy; Hyperlipoproteinemia Type II - genetics; Hyperlipoproteinemia Type II - therapy; Low-density lipoprotein apheresis; Low-density lipoprotein cholesterol; Male; Middle Aged; Proprotein convertase subtilisin/kexin type 9 inhibitor; Prospective Studies; Treatment Outcome; Apolipoprotein B; Low-density lipoprotein apheresis; Familial hypercholesterolemia; Proprotein convertase subtilisin/kexin type 9 inhibitor; Low-density lipoprotein cholesterol
• ISSN: 0021-9150; 1879-1484
• DOI: 10.1016/j.atherosclerosis.2017.09.011

Low-density lipoprotein (LDL) apheresis has been used to treat refractory hyperlipidemia such as familial hypercholesterolemia (FH). Evolocumab, a proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitor used in clinical settings, can reduce LDL cholesterol (LDL-C) levels by >70%. Therefore, this study aimed to assess the impact of evolocumab on withdrawal from regular LDL apheresis in patients with heterozygous FH (HeFH). Eleven patients with HeFH undergoing biweekly LDL apheresis were enrolled and were subsequently switched to a biweekly subcutaneous injection of 140 mg of evolocumab. The primary endpoints were percent changes in mean LDL-C and apolipoprotein B (apoB) serum levels, which were averages of two different time point measurements, due to the switch in the treatment method. The mean LDL-C and apoB serum levels significantly reduced from 2.55 ± 0.62 mmol/L to 0.96 ± 0.40 mmol/L (−62.5%, p < 0.0001) and from 82.8 ± 12.3 mg/dL to 45.4 ± 10.9 mg/dL (−45.2%, p < 0.0001), respectively. Serum lipoprotein (a) levels also significantly reduced from 148 (116–351) mg/L to 91 (53–289) mg/L (−38.5%, p < 0.01). The reduction in LDL-C and apoB levels was not associated with the basal serum levels of PCSK9 or cholesterol production/absorption markers. Although evolocumab significantly reduced serum vitamin E levels, they were still within the normal range, and no subjective or objective side effects were observed. Compared to biweekly LDL apheresis, biweekly evolocumab injection therapy is less expensive, less invasive, less time-consuming, and more effective in reducing atherogenic lipoprotein levels without severe adverse side effects. •Eleven patients with heterozygous FH under biweekly LDL apheresis were enrolled.•All the patients were switched from LDL apheresis to evolocumab injection therapy.•Evolocumab significantly reduces LDL-C by 62.5% compared with LDL apheresis.•No side effects were documented other than mild reduction in vitamin E serum levels.