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[Preprint]. 2023 Nov 15:2023.11.14.23298547.
doi: 10.1101/2023.11.14.23298547.

Clinical practice recommendations on lipoprotein apheresis for children with homozygous familial hypercholesterolemia: 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   14 , Claudia Stefanutti  15 , Christina Taylan  16 , Albert Wiegman  1 , Claus Peter Schmitt  17
Affiliations

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

M Doortje Reijman et al. medRxiv. .

Update in

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 well established since three decades, lowering serum LDL-C levels by more than 70% per session. 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 targets and follow-up of lipoprotein apheresis in children with homozygous familial hypercholesterolaemia and on the role of lipoprotein(a) and liver transplantation.

Keywords: LDL; cholesterol; consensus statement; homozygous familial hypercholesterolemia; lipoprotein apheresis; paediatrics.

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