Evidence map›Paper›PMID 38938723›Full record

ArticleJACC. Advances2023

The Long-Term Efficacy and Safety of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia.

Frederick J Raal, Robert S Rosenson, Laurens F Reeskamp, John J P Kastelein, Paolo Rubba, P Barton Duell, Masahiro Koseki, Erik Stroes, Shazia Ali, Poulabi Banerjee and 6 more

Abstract read
In one paragraph

Article in JACC. Advances, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 13 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
13citing papers in PubMed, 1 pooled it
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

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.

2 · The registry

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.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

13 citing papers in PubMed, 1 synthesis or guideline pooled it.

  1. Pooled it
  2. Article
  3. Advanced therapy in familial hypercholesterolemia.Canadian family physician Medecin de famille canadien · 2026
    Review
  4. Review
  5. Article
  6. Review
  7. Review
  8. Review
  9. Antibody-Based Therapeutics for Hypercholesterolemia.Biologics : targets & therapy · 2025
    Review
  10. Review
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  13. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

16 authors.

Frederick J RaalFaculty of Health Sciences, University of the Witwatersrand, Johannesburg, South Africa.
Robert S RosensonCardiometabolics Unit, Zena and Michael A Wiener Cardiovascular Institute, Marie-Josee and Henry R. Kravis Center for Cardiovascular Health, Icahn School of Medicine at Mount Sinai, New York, New York, USA.
Laurens F ReeskampDepartment of Vascular Medicine, University of Amsterdam, Amsterdam, The Netherlands.
John J P KasteleinDepartment of Vascular Medicine, University of Amsterdam, Amsterdam, The Netherlands.
Paolo RubbaDepartment of Internal Medicine and Surgery, Federico II University, Naples, Italy.
P Barton DuellKnight Cardiovascular Institute and Division of Endocrinology, Diabetes and Clinical Nutrition, Oregon Health & Science University, Portland, Oregon, USA.
Masahiro KosekiDivision of Cardiovascular Medicine, Department of Medicine, Osaka University Graduate School of Medicine, Osaka, Japan.
Erik StroesAmsterdam Cardiovascular Sciences, Amsterdam, The Netherlands.
Shazia AliRegeneron Pharmaceuticals, Inc, Tarrytown, New York, USA.
Poulabi BanerjeeRegeneron Pharmaceuticals, Inc, Tarrytown, New York, USA.
Kuo-Chen ChanRegeneron Pharmaceuticals, Inc, Tarrytown, New York, USA.
Nagwa KhillaRegeneron Pharmaceuticals, Inc, Tarrytown, New York, USA.
Jennifer McGinnissRegeneron Pharmaceuticals, Inc, Tarrytown, New York, USA.
Robert PordyRegeneron Pharmaceuticals, Inc, Tarrytown, New York, USA.
Yi ZhangRegeneron Pharmaceuticals, Inc, Tarrytown, New York, USA.
Daniel GaudetClinical Lipidology and Rare Lipid Disorders Unit, Department of Medicine, Université de Montréal Community Gene Medicine Center, Lipid Clinic Chicoutimi Hospital and ECOGENE-21 Clinical and Translational Research Center, Chicoutimi, Quebec, Canada.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Background: Homozygous familial hypercholesterolemia (HoFH) is characterized by early-onset atherosclerotic cardiovascular disease due to the high low-density lipoprotein cholesterol (LDL-C) burden. Patients with null-null low-density lipoprotein receptor ( Objectives: The purpose of this study was to evaluate longer-term efficacy and safety of evinacumab in patients with HoFH from the ELIPSE HoFH study. Methods: Patients with HoFH on stable lipid-lowering therapies (LLTs) ± lipoprotein apheresis and screening LDL-C ≥70 mg/dL who completed the DBTP entered the 24-week open-label treatment period (OLTP) and received intravenous evinacumab 15 mg/kg every 4 weeks. OLTP results were summarized descriptively. Results: A total of 64 patients completed the DBTP and received open-label evinacumab. Despite multiple LLTs, the mean baseline LDL-C at DBTP entry was 250.5 ± 162.3 mg/dL. From baseline to week 48 (end of OLTP), evinacumab reduced mean LDL-C by 46.3% (mean reduction, 134.3 ± 117.3 mg/dL), with similar mean LDL-C reductions for patients with null-null (47.2%) and non-null variants (45.9%). Adverse events occurred in 47 (73.4%) patients; 4 (6.3%) patients experienced adverse events considered evinacumab-related (drug hypersensitivity, infusion-related reaction and asthenia, generalized pruritis, and muscle spasms). Conclusions: In patients with HoFH, evinacumab demonstrated substantial and sustained LDL-C reduction regardless of LDLR function, and was generally well tolerated.

Indexed as

angiopoietin-like protein 3clinical trialevinacumabhomozygous familial hypercholesterolemialipidslipoprotein

Identifiers

PMID38938723
PMCPMC11198175

What Socratic holds

Textmetadata
LicenceCC BY-NC-ND
Read underepoch 390

Registered trials

None linked

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 Socratic graph.