{"id":"19d0baa6873c","type":"article","url":"https://hartvaat.nl/2026/09/19/nieuwe-lipidendalers-bieden-meer-opties-voor-kinderen-met-familiaire-hypercholes/","title":"Nieuwe lipidendalers bieden meer opties voor kinderen met familiaire hypercholesterolemie","title_en":"Current, novel and emerging lipid-lowering therapies in pediatric familial hypercholesterolemia.","category":"cholesterol","category_label":"Cholesterol","professions":["apotheker","cardioloog","internist"],"tags":[],"journal":"Current opinion in lipidology","doi":"10.1097/MOL.0000000000001060","source_url":"https://doi.org/10.1097/MOL.0000000000001060","authors":["Eelke Houter","Sibbeliene E van den Bosch","Barbara A Hutten","Willemijn E Corpeleijn"],"significance":5,"published":"2026-09-26","source_date":"2026-09-19","image":"https://hartvaat.nl/global/img/6e2cc0469790.webp","kennis":["https://hartvaat.nl/kennis/lipiden/familiaire-hypercholesterolemie/","https://hartvaat.nl/kennis/lipiden/lp-a-verhoogd/"],"congress":"","summary_en":"This narrative review outlines current and emerging lipid-lowering therapies for pediatric familial hypercholesterolemia (FH). While conventional treatments and injectable PCSK9 inhibitors remain foundational, novel oral PCSK9 inhibitors, fixed-dose combinations, and emerging agents targeting ANGPTL3 and MTP are in development and offer potent LDL-C reductions, particularly for homozygous FH. With updated European consensus guidelines setting stricter LDL-C targets, clinicians should anticipate a shift toward earlier, more intensive, and personalized treatment strategies to improve long-term cardiovascular outcomes in this population.","created":"2026-09-20T01:05:47Z","updated":"2026-09-20T01:05:51Z","licence":"Citeer vrij, met bronvermelding en een link naar hartvaat.nl (de url van het record). Samenvattingen zijn redactioneel werk van HartVaat; de oorspronkelijke publicaties blijven van hun uitgevers (doi). Geen medisch advies.","body_markdown":"Deze review schetst het huidige en toekomstige landschap van lipidendalers bij kinderen met familiaire hypercholesterolemie (FH). Naast standaardtherapie worden PCSK9-remmers al ingezet, maar injectie en kosten beperken het gebruik; orale PCSK9-remmers, vaste combinaties en nieuwe remmers van ANGPTL3 en MTP staan in de startblokken en beloven sterkere LDL-C-dalingen, ook bij homozygote FH. Door de aangescherpte Europese richtlijnen worden meer kinderen in aanmerking voor behandeling, wat een verschuiving naar vroege, intensieve en gepersonaliseerde therapieën rechtvaardigt.","abstract_original":"PURPOSE OF REVIEW: Over the past decade, pharmacological treatment options for children with familial hypercholesterolemia (FH) have expanded considerably. The recent European Atherosclerosis Society consensus recommends more stringent LDL cholesterol (LDL-C) targets than previously advocated. Consequently, more children with FH are expected to become eligible for novel therapeutic options. This review summarizes established and emerging lipid-lowering therapies (LLT) evaluated in children, focusing on recent and ongoing clinical trials. RECENT FINDINGS: Conventional LLT remains the cornerstone of FH treatment, and proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitors are approved for pediatric use and achieve significant LDL-C reductions; however, injectable administration and high costs limit their widespread use. Novel oral PCSK9 inhibitors are currently under investigation in clinical trials, and fixed-dose combinations may offer a more practical and cost-effective approach. Additionally, angiopoietin-like protein 3 inhibitors and microsomal triglyceride transfer protein inhibitors lower LDL-C in dependently of LDL receptor activity, making them particularly suitable for homozygous FH patients. SUMMARY: The management of FH is entering a new therapeutic era. As therapeutic options expand and agents with greater LDL-C reductions become available, personalized treatment strategies will become increasingly important to achieve LDL-C targets and may ultimately shift from stepwise intensification approaches toward early intensive treatment."}