{"id":"efb957692132","type":"article","url":"https://hartvaat.nl/2024/07/12/evinacumab-bij-homozygote-fh-langetermijn-veiligheid-en-effectiviteit/","title":"Evinacumab bij homozygote FH: langetermijn veiligheid en effectiviteit","title_en":"Evinacumab in homozygous familial hypercholesterolaemia: long-term safety and efficacy.","category":"cholesterol","category_label":"Cholesterol","professions":["apotheker","cardioloog","internist"],"tags":["familiaire-hypercholesterolemie","familiaire-hypercholesterolemie-screening","vrouwen"],"journal":"European heart journal","doi":"10.1093/eurheartj/ehae325","source_url":"https://doi.org/10.1093/eurheartj/ehae325","authors":["Daniel Gaudet","Susanne Greber-Platzer","Laurens F Reeskamp","Gabriella Iannuzzo","Robert S Rosenson","Samir Saheb","Claudia Stefanutti","Erik Stroes","Albert Wiegman","Traci Turner","Shazia Ali","Poulabi Banerjee","Tiera Drewery","Jennifer McGinniss","Alpana Waldron","Richard T George","Xue-Qiao Zhao","Robert Pordy","Jian Zhao","Eric Bruckert","Frederick J Raal"],"significance":7,"published":"2024-07-12","source_date":"2024-07-12","image":"","kennis":[],"congress":"","summary_en":"Long-term data confirmed the sustained safety and efficacy of evinacumab in homozygous FH, with durable LDL cholesterol reduction and no emergence of late adverse effects, supporting long-term ANGPTL3 inhibition for this severe genetic disorder.","created":"2026-07-03T10:31:02Z","updated":"2026-07-03T13:30:05Z","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":"Langetermijndata bevestigden de duurzame veiligheid en effectiviteit van evinacumab bij homozygote FH. Het ANGPTL3-antilichaam biedt blijvende LDL-verlaging bij deze ernstigste vorm van hypercholesterolemie.","abstract_original":"BACKGROUND AND AIMS: Homozygous familial hypercholesterolaemia (HoFH) is a rare genetic disorder characterized by severely elevated LDL cholesterol (LDL-C) and premature atherosclerotic cardiovascular disease. In the pivotal Phase 3 HoFH trial (NCT03399786), evinacumab significantly decreased LDL-C in patients with HoFH. This study assesses the long-term safety and efficacy of evinacumab in adult and adolescent patients with HoFH. METHODS: In this open-label, single-arm, Phase 3 trial (NCT03409744), patients aged ≥12 years with HoFH who were evinacumab-naïve or had previously received evinacumab in other trials (evinacumab-continue) received intravenous evinacumab 15 mg/kg every 4 weeks with stable lipid-lowering therapy. RESULTS: A total of 116 patients (adults: n = 102; adolescents: n = 14) were enrolled, of whom 57 (49.1%) were female. Patients were treated for a median (range) duration of 104.3 (28.3-196.3) weeks. Overall, treatment-emergent adverse events (TEAEs) and serious TEAEs were reported in 93 (80.2%) and 27 (23.3%) patients, respectively. Two (1.7%) deaths were reported (neither was considered related to evinacumab). Three (2.6%) patients discontinued due to TEAEs (none were considered related to evinacumab). From baseline to Week 24, evinacumab decreased mean LDL-C by 43.6% [mean (standard deviation, SD), 3.4 (3.2) mmol/L] in the overall population; mean LDL-C reduction in adults and adolescents was 41.7% [mean (SD), 3.2 (3.3) mmol/L] and 55.4% [mean (SD), 4.7 (2.5) mmol/L], respectively. CONCLUSIONS: In this large cohort of patients with HoFH, evinacumab was generally well tolerated and markedly decreased LDL-C irrespective of age and sex. Moreover, the efficacy and safety of evinacumab was sustained over the long term."}