ArticleCirculation2024
Evinacumab for Pediatric Patients With Homozygous Familial Hypercholesterolemia.
Article in Circulation, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. It is linked to trial NCT04233918 (A Three-Part, Single-Arm, Open-Label Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Evinacumab in Pediatric Patients With Homozygous Familial Hypercholesterolemia), which is not on this map. Cited by 41 papers, 1 of them a synthesis that pooled it.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
A Three-Part, Single-Arm, Open-Label Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Evinacumab in Pediatric Patients With Homozygous Familial Hypercholesterolemia
Who cites it
41 citing papers in PubMed, 1 synthesis or guideline pooled it, 77 citations in OpenAlex.
- Pooled it
- Evinacumab for Homozygous Familial Hypercholesterolemia: The Italian Cohort of the ELIPSE HoFH Study.Advances in therapy · 2025Trial
- Clinical considerations on the use of evinacumab for the treatment of homozygous familial hypercholesterolemia.American journal of preventive cardiology · 2026Review
- Evinacumab Across Different Lipid Phenotypes: A Systematic Review and Meta-Analysis.Advances in therapy · 2026Article
- The Angiopoietin-like Protein (ANGPTL) Axis in Dyslipidemia: Mechanisms, Cardiovascular Risk, and Emerging Therapies.Current atherosclerosis reports · 2026Review
- Familial hypercholesterolaemia in children and adolescents: a European Atherosclerosis Society consensus statement.European heart journal · 2026Article
- [Austrian lipid consensus : Interdisciplinary evidence-based recommendations on prevention and treatment of lipid-associated diseases from 15 medical specialist societies].Wiener klinische Wochenschrift · 2026Article
- Biologics for cardiovascular diseases: from bench to bedside.Signal transduction and targeted therapy · 2026Review
- Distinct From the Adult Paradigm: Re-Evaluate the Pathophysiological and Therapeutic Framework in Children and Adolescent Hyperlipidaemia.Current obesity reports · 2026Review
- Genetic Testing for Children with Familial Hypercholesterolemia.Journal of atherosclerosis and thrombosis · 2026Review
- Atherogenic Dyslipidemia in Children and Adolescents: Current Evidence, Clinical Challenges, and Future Perspectives.Journal of cardiovascular development and disease · 2026Review
- Advanced therapy in familial hypercholesterolemia.Canadian family physician Medecin de famille canadien · 2026Review
- The High Price of Interrupted Follow-Up: Catastrophic Progression of Homozygous Familial Hypercholesterolemia-A Case Report and Literature Review.Clinical case reports · 2026Article
- Angiopoietin-like Protein 3 (ANGPTL3) Targeting in the Management of Dyslipidemias.International journal of molecular sciences · 2026Review
- Investigation of Monoclonal Antibody Pharmacokinetics in Pediatric Population and Characterization Using a Platform PBPK Model.The AAPS journal · 2026Article
- Advances in Non-statin Lipid Therapies: A Narrative Review of Evolving Strategies for Cardiovascular Risk Reduction.American journal of cardiovascular drugs : drugs, devices, and other interventions · 2026Review
- Homozygous familial hypercholesterolemia, experience with Evinacumab treatment in two Mexican pediatric patients: case report.Frontiers in genetics · 2026Article
- Brazilian Guideline on Dyslipidemias and Prevention of Atherosclerosis - 2025.Arquivos brasileiros de cardiologia · 2025Article
- Pediatric Hypertriglyceridemia: Lipoprotein Metabolism, Etiology, and Management.Current pediatrics reports · 2025Article
- Diagnostic and Therapeutic Challenges of Homozygous and Severe Heterozygous Familial Hypercholesterolemia from Clinical Aspect-A Single-Center Study.Journal of clinical medicine · 2025Article
Corrections and comments
- Commented on by
- Erratum issued
Authors and funding
18 authors at 12 institutions in 4 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundHomozygous familial hypercholesterolemia (HoFH) is a rare genetic disorder characterized by severely elevated low-density lipoprotein cholesterol (LDL-C) levels due to profoundly defective LDL receptor (LDLR) function. Given that severely elevated LDL-C starts in utero, atherosclerosis often presents during childhood or adolescence, creating a largely unmet need for aggressive LDLR-independent lipid-lowering therapies in young patients with HoFH. Here we present the first evaluation of the efficacy and safety of evinacumab, a novel LDLR-independent lipid-lowering therapy, in pediatric patients with HoFH from parts A and B of a 3-part study.
methodsThe phase 3, part B, open-label study treated 14 patients 5 to 11 years of age with genetically proven HoFH (true homozygotes and compound heterozygotes) with LDL-C >130 mg/dL, despite optimized lipid-lowering therapy (including LDLR-independent apheresis and lomitapide), with intravenous evinacumab 15 mg/kg every 4 weeks.
resultsEvinacumab treatment rapidly and durably (through week 24) decreased LDL-C with profound reduction in the first week, with a mean (SE) LDL-C reduction of -48.3% (10.4%) from baseline to week 24. ApoB (mean [SE], -41.3% [9.0%]), non-high-density lipoprotein cholesterol (-48.9% [9.8%]), and total cholesterol (-49.1% [8.1%]) were similarly decreased. Treatment-emergent adverse events were reported in 10 (71.4%) patients; however, only 2 (14.3%) reported events that were considered to be treatment-related (nausea and abdominal pain). One serious treatment-emergent adverse event of tonsillitis occurred (n=1), but this was not considered treatment-related.
conclusionsEvinacumab constitutes a new treatment for pediatric patients with HoFH and inadequately controlled LDL-C despite optimized lipid-lowering therapy, lowering LDL-C levels by nearly half in these extremely high-risk and difficult-to-treat individuals. REGISTRATION: URL: https://www.clinicaltrials.gov; Unique identifier: NCT04233918.
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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.