Physicians' Academy for Cardiovascular Education

Long-term PCSK9i treatment in pediatric HeFH patients safe and reduces LDL-c

Paediatric patients with heterozygous familial hypercholesterolaemia treated with evolocumab for 80 weeks (HAUSER-OLE): a single-arm, multicentre, open-label extension of HAUSER-RCT

Literature - Santos RD, Ruzza A, Hovingh GK, et al. - Lancet Diabetes Endocrinol. 2022 Oct;10(10):732-740. doi: 10.1016/S2213-8587(22)00221-2.

Introduction and methods


The HAUSER-RCT study showed that in pediatric patients with heterozygous familial hypercholesterolemia (HeFH), treatment with the PCSK9 inhibitor evolocumab for 24 weeks is safe and reduces LDL-c and other lipid parameter levels, compared with placebo [1]. However, it is unclear whether long-term treatment with evolocumab is safe and effective in pediatric patients with HeFH.

Aim of the study

The aim of this study was to evaluate the safety and efficacy of long-term treatment with evolocumab in pediatric patients with HeFH.


This 80-week open-label extension study (HAUSER-OLE) was a follow-up to the HAUSER-RCT study. In this previous multicenter, double-blind, placebo-controlled phase 3 study, 157 pediatric patients with HeFH were randomized (2:1) to monthly, subcutaneous administration of evolocumab 420 mg or placebo for 24 weeks. Inclusion criteria were: age 10-17 years, HeFH by genetic testing or according to clinical diagnostic criteria and LDL-c levels ≥3.4 mmol/L, triglyceride levels ≤4.5 mmol/L and optimized statin therapy at screening.

Patients were eligible to participate in the HAUSER-OLE study if they had been treated with evolocumab or placebo for 24 weeks and no serious treatment-emergent adverse events (TEAEs) had occurred during treatment. In this single-arm study, 150 patients received monthly open-label evolocumab 420 mg subcutaneously for 80 weeks, in addition to statin therapy with or without ezetimibe.


The primary endpoint was the incidence of TEAEs. The secondary endpoint was efficacy, assessed by percentage and absolute changes in the levels of LDL-c, other lipid parameters (including non-HDL-c and apolipoprotein B) and PCSK9 between the start of the HAUSER-RCT study and the end of the HAUSER-OLE study (a total of 104 weeks).

Main results




This open-label extension study (HAUSER-OLE) showed that monthly subcutaneous administration of evolocumab 420 mg for up to 104 weeks, in addition to statin therapy with or without ezetimibe, in pediatric patients with HeFH was safe, well tolerated and resulted in a reduction of LDL-c and other lipid parameter levels.


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Find this article online at Lancet Diabetes Endocrinol.

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