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Introduction <p>Homozygous familial hypercholesterolemia (HoFH) is a severe rare genetic disorder characterized by elevated plasma low-density lipoprotein (LDL) cholesterol levels. Here, we report data from the Italian cohort of the Evinacumab Lipid Studies in Patients with Homozygous Familial Hypercholesterolemia (ELIPSE HoFH) trial.</p> Methods <p>ELIPSE HoFH was conducted at 30 sites in 11 countries, with 2–10 patients enrolled per country. The study included patients aged ≥ 12&#xa0;years with LDL cholesterol ≥ 70&#xa0;mg/dl (1.8&#xa0;mmol per liter) at screening despite stable maximally tolerated lipid-lowering therapy. Patients were randomly assigned evinacumab (15&#xa0;mg/kg every 4&#xa0;weeks) or matching placebo for 24 weeks, with an option for a 24-week open-label extension or follow-up period thereafter. The Italian cohort included seven patients assigned to evinacumab.</p> Results <p>Five patients (3 males and 2 females) received evinacumab and were included in this report. Substantial and consistent reductions in LDL cholesterol from baseline levels were observed in all patients at all follow-up time points. Overall, an 84.5% decrease in median (range) LDL cholesterol was observed, from 323 (203–587) mg/dl in 2016 to 50.0 (13–103) mg/dl (<i>P</i> = 0.043) in 2019, with LDL cholesterol levels stable through 2023. Total cholesterol, high-density lipoprotein (HDL) cholesterol, non-HDL cholesterol, and triglycerides decreased markedly over time. Evinacumab was well tolerated, with no treatment-related adverse events reported.</p> Conclusion <p>Evinacumab substantially lowered LDL cholesterol levels in patients with HoFH regardless of the degree of LDL receptor function, with low levels sustained over 5&#xa0;years of follow-up.</p> Trial Registration <p>ClinicalTrials.gov identifier NCT03399786 registered 16 January 2018.</p>

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Evinacumab for Homozygous Familial Hypercholesterolemia: The Italian Cohort of the ELIPSE HoFH Study

  • 期刊论文

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  • 发表日期：

    2025年4月2日

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  • Gabriella Iannuzzo,
  • Ilenia Calcaterra,
  • Marco Gentile,
  • Claudia Stanzione,
  • Francesca De Ruberto,
  • Maria Donata Di Taranto,
  • Giuliana Fortunato,
  • Matteo Di Minno

摘要

Introduction

Homozygous familial hypercholesterolemia (HoFH) is a severe rare genetic disorder characterized by elevated plasma low-density lipoprotein (LDL) cholesterol levels. Here, we report data from the Italian cohort of the Evinacumab Lipid Studies in Patients with Homozygous Familial Hypercholesterolemia (ELIPSE HoFH) trial.

Methods

ELIPSE HoFH was conducted at 30 sites in 11 countries, with 2–10 patients enrolled per country. The study included patients aged ≥ 12 years with LDL cholesterol ≥ 70 mg/dl (1.8 mmol per liter) at screening despite stable maximally tolerated lipid-lowering therapy. Patients were randomly assigned evinacumab (15 mg/kg every 4 weeks) or matching placebo for 24 weeks, with an option for a 24-week open-label extension or follow-up period thereafter. The Italian cohort included seven patients assigned to evinacumab.

Results

Five patients (3 males and 2 females) received evinacumab and were included in this report. Substantial and consistent reductions in LDL cholesterol from baseline levels were observed in all patients at all follow-up time points. Overall, an 84.5% decrease in median (range) LDL cholesterol was observed, from 323 (203–587) mg/dl in 2016 to 50.0 (13–103) mg/dl (P = 0.043) in 2019, with LDL cholesterol levels stable through 2023. Total cholesterol, high-density lipoprotein (HDL) cholesterol, non-HDL cholesterol, and triglycerides decreased markedly over time. Evinacumab was well tolerated, with no treatment-related adverse events reported.

Conclusion

Evinacumab substantially lowered LDL cholesterol levels in patients with HoFH regardless of the degree of LDL receptor function, with low levels sustained over 5 years of follow-up.

Trial Registration

ClinicalTrials.gov identifier NCT03399786 registered 16 January 2018.