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

Background and aims: 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). Methods: 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. Results: 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. Conclusions: 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. (C) 2017 Elsevier B.V. All rights reserved.