Clinical practice recommendations on lipoprotein apheresis for children with homozygous familial hypercholesterolaemia: An expert consensus statement from ERKNet and ESPN
Year: 2024
Type: 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.
Source: Atherosclerosis
Authors: M. Doortje Reijman, D. Meeike Kusters, Jaap W. Groothoff, Klaus Arbeiter, Eldad J. Dann, Lotte M. de Boer, Sarah D. de Ferranti, Antonio Gallo, Susanne Greber-Platzer, Jacob Hartz, Lisa C. Hudgins, Daiana Ibarretxe, Meral Kayikcioglu, Reinhard Klingel, Genovefa D. Kolovou, Jun Oh, R. Nils Planken, Claudia Stefanutti, Christina Taylan, Albert Wiegman, Claus Peter Schmitt
Institutions: Amsterdam University Medical Centers, Amsterdam Gastroenterology Endocrinology Metabolism, Amsterdam Cardiovascular Sciences, University of Amsterdam, Medical University of Vienna, Rambam Health Care Campus, Boston Children's Hospital, Harvard University, Inserm, Sorbonne Université, Pitié-Salpêtrière Hospital, Fondation pour l’innovation en Cadiométabolisme et Nutrition, Weill Cornell Medicine, The Rogosin Institute, Instituto de Salud Carlos III, Centro de Investigación Biomédica en Red Diabetes y Enfermedades Metabólicas Asociadas, Institut de Recerca Biomèdica Catalunya Sud, Centro de Investigación Biomédica en Red, Universitat Rovira i Virgili, Ege University, Apherese Forschungsinstitut, Metropolitan Hospital, University Medical Center Hamburg-Eppendorf, Amsterdam UMC Location University of Amsterdam, Policlinico Umberto I, Sapienza University of Rome, University of Cologne, University Hospital Cologne, Heidelberg University
Language: English
Subfield: Cardiology and Cardiovascular Medicine
Field: Medicine
Domain: Health Sciences
Open Access status: hybrid
Estimated APC paid: $3,790
Funders: Amgen, Sanofi, University of Pennsylvania, Helsingin Yliopisto, Regeneron Pharmaceuticals, Ultragenyx Pharmaceutical, McGill University Health Centre, Mylan, Amryt Pharma, Imperial College London, University College London, University of Toronto, Novo Nordisk, Hacettepe Üniversitesi, Medizinischen Hochschule Hannover, Medizinische Universität Wien, Hospital for Sick Children, Servier, Swedish Orphan Biovitrum, Sorbonne Université, Silence Therapeutics, Esperion Therapeutics, National Institutes of Health, Perelman School of Medicine, University of Pennsylvania, National Heart, Lung, and Blood Institute
OpenAlex ID: W4393227439