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Review
. 2024 May:392:117525.
doi: 10.1016/j.atherosclerosis.2024.117525. Epub 2024 Mar 27.

Clinical practice recommendations on lipoprotein apheresis for children with homozygous familial hypercholesterolaemia: An expert consensus statement from ERKNet and ESPN

M Doortje Reijman  1 , D Meeike Kusters  1 , Jaap W Groothoff  1 , Klaus Arbeiter  2 , Eldad J Dann  3 , Lotte M de Boer  1 , Sarah D de Ferranti  4 , Antonio Gallo  5 , Susanne Greber-Platzer  6 , Jacob Hartz  4 , Lisa C Hudgins  7 , Daiana Ibarretxe  8 , Meral Kayikcioglu  9 , Reinhard Klingel  10 , Genovefa D Kolovou  11 , Jun Oh  12 , R Nils Planken  13 , Claudia Stefanutti  14 , Christina Taylan  15 , Albert Wiegman  16 , Claus Peter Schmitt  17
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Free article
Review

Clinical practice recommendations on lipoprotein apheresis for children with homozygous familial hypercholesterolaemia: An expert consensus statement from ERKNet and ESPN

M Doortje Reijman et al. Atherosclerosis. 2024 May.
Free article

Abstract

Homozygous familial hypercholesterolaemia is a life-threatening genetic condition, which causes extremely elevated LDL-C levels and atherosclerotic cardiovascular disease very early in life. It is vital to start effective lipid-lowering treatment from diagnosis onwards. Even with dietary and current multimodal pharmaceutical lipid-lowering therapies, LDL-C treatment goals cannot be achieved in many children. Lipoprotein apheresis is an extracorporeal lipid-lowering treatment, which is used for decades, lowering serum LDL-C levels by more than 70% directly after the treatment. Data on the use of lipoprotein apheresis in children with homozygous familial hypercholesterolaemia mainly consists of case-reports and case-series, precluding strong evidence-based guidelines. We present a consensus statement on lipoprotein apheresis in children based on the current available evidence and opinions from experts in lipoprotein apheresis from over the world. It comprises practical statements regarding the indication, methods, treatment goals and follow-up of lipoprotein apheresis in children with homozygous familial hypercholesterolaemia and on the role of lipoprotein(a) and liver transplantation.

Keywords: Cholesterol; Consensus statement; Homozygous familial hypercholesterolaemia; LDL; Lipoprotein apheresis; Paediatrics.

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Conflict of interest statement

Declaration of competing interest SDdF received grants from the National Institutes of Health/National Heart, Lung, and Blood Institute of the National Institutes of Health, the Family Heart Foundation and holds a patent for UpToDate, with royalties paid. AG received grants and personal fees from Amgen, Sanofi and Regeneron, Mylan Viatris, MSD, Akcea Therapeutics, Amryt, Servier, Novartis and Ultragenyx. SGP received study funding from Amgen. JH received a research grant from the National Heart, Lung, And Blood Institute of the National Institutes of Health under Award Number K23HL145109. DI received advisory honoraria from Amryt, and honoraria for lectures from Sanofi, Sobi and Novartis. CPS received advisory honoraria from Baxter, Iperboreal and Stadapharma, lecturing honoraria from Fresenius and research funding from Baxter. MK received honoraria from Abbott, Abdi Ibrahim, Amryt, LIB Therapeutics, NovoNordisk, and TR-pharma; research funding from Amryt Pharma, and participated in clinical trials with Amgen, Ionis, LIB Therapeutics, Novo Nordisk, Sanofi. RK, as head of the Apheresis Research GmbH, Cologne, Germany received financial grants for scientific research projects, honoraria for consulting and presentation of lectures including travel expenses from the companies Diamed, Cologne, Germany, and Asahi Kasei Medical, Tokyo, Japan. GK has given talks, attended conferences, received consultancy fees and participated in trials sponsored by Amgen, Novartis, Sanofi, Servier, Amryt. AW received research grants from Amgen, Regeneron, Novartis, Silence Therapeutics, Esperion, and Ultragenyx and advisory honoraria from Amryt, and Novartis. KA, EJD, LMdB, JWG, LCH, GDK, JO, RNP, MDR, CS and CT have nothing to declare.

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