Evidence map›Paper›PMID 38545781›Full record

ArticleArteriosclerosis, thrombosis, and vascular biology2024

Evolocumab Treatment in Pediatric Patients With Homozygous Familial Hypercholesterolemia: Pooled Data From Three Open-Label Studies.

Frederick J Raal, Robert A Hegele, Andrea Ruzza, J Antonio G López, Ajay K Bhatia, Johnny Wu, Huei Wang, Daniel Gaudet, Albert Wiegman, Jian Wang and 1 more

3 registry-linked trialsOpen access · hybridAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Arteriosclerosis, thrombosis, and vascular biology, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 3 registered trials, which are not on this map. Cited by 10 papers, 2 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
10citing papers in PubMed, 2 pooled it
10.3field-weighted citation impact, top 1% of its field
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.

NCT01624142 phase2 / phase3completednot on this map

A Multicenter, Open-label Study to Assess the Long-term Safety, Tolerability, and Efficacy of Evolocumab (AMG145) on LDL-C in Subjects With Severe Familial Hypercholesterolemia

TypeinterventionalSponsorAmgenRan2012 to 2018Enrolled300ConditionsSevere Familial HypercholesterolemiaArmsEvolocumab
NCT02624869 phase3completednot on this map

Open-label, Single-Arm, Multicenter Study to Evaluate the Safety, Tolerability and Efficacy of Evolocumab for LDL-C Reduction, as Add-on to Diet and Lipid-lowering Therapy, in Pediatric Subjects From 10 to 17 Years of Age With Heterozygous Familial Hypercholesterolemia (HeFH) or Homozygous Familial Hypercholesterolemia (HoFH)

TypeinterventionalSponsorAmgenRan2016 to 2021Enrolled163ConditionsFamilial HypercholesterolemiaArmsEvolocumab
NCT03403374 phase4completednot on this map

A Multicenter, Open-label, Single-arm, Study to Evaluate Safety and Tolerability of Repatha in Patients With Homozygous Familial Hypercholesterolemia (HoFH) in India

TypeinterventionalSponsorAmgenRan2018 to 2019Enrolled30ConditionsHomozygous Familial Hypercholesterolemia HoFHArmsevolocumab
3 · Its place in the literature

Who cites it

10 citing papers in PubMed, 2 syntheses or guidelines pooled it, 18 citations in OpenAlex.

  1. Pooled it
  2. Pooled it
  3. Article
  4. Review
  5. Advanced therapy in familial hypercholesterolemia.Canadian family physician Medecin de famille canadien · 2026
    Review
  6. Review
  7. Review
  8. Review
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  10. 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

11 authors at 7 institutions in 5 countries.

Frederick J RaalFaculty of Health Sciences, University of the Witwatersrand, Johannesburg, South Africa (F.J.R.).
Robert A HegeleDepartment of Medicine and Biochemistry, Robarts Research Institute, University of Western Ontario, London, Canada (R.A.H., J.W.).ORCID 0000-0003-2861-5325
Andrea RuzzaGlobal Development (A.R., J.A.G.L., A.K.B.), Amgen Inc., Thousand Oaks, CA.ORCID 0000-0001-6827-0718
J Antonio G LópezGlobal Development (A.R., J.A.G.L., A.K.B.), Amgen Inc., Thousand Oaks, CA.ORCID 0000-0001-8604-5549
Ajay K BhatiaGlobal Development (A.R., J.A.G.L., A.K.B.), Amgen Inc., Thousand Oaks, CA.
Johnny WuDepartment of Medicine and Biochemistry, Robarts Research Institute, University of Western Ontario, London, Canada (R.A.H., J.W.).
Huei WangGlobal Biostatistics (J.W., H.W.), Amgen Inc., Thousand Oaks, CA.
Daniel GaudetClinical Lipidology and Rare Lipid Disorders Unit, Community Genomic Medicine Centre and ECOGENE-21, Department of Medicine, Université de Montreal, Chicoutimi, Quebec, Canada (D.G.).ORCID 0000-0002-5185-3666
Albert WiegmanDepartment of Paediatrics, Amsterdam UMC, Location AMC, the Netherlands (A.W.).ORCID 0000-0001-6223-5671
Jian WangGlobal Biostatistics (J.W., H.W.), Amgen Inc., Thousand Oaks, CA.ORCID 0000-0002-6759-0522
Raul D SantosLipid Clinic Heart Institute (InCor), University of Sao Paulo Medical School Hospital and Hospital Israelita Albert Einstein, Sao Paulo, Brazil (R.D.S.).
Amgen (United States) · USWestern University · CACenter for Global Development · USGGD Amsterdam · NLHospital Israelita Albert Einstein · BRUniversité du Québec à Chicoutimi · CAUniversity of the Witwatersrand · ZA

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundPediatric patients with homozygous familial hypercholesterolemia (HoFH) have an increased risk of atherosclerotic cardiovascular disease and difficulty meeting low-density lipoprotein cholesterol (LDL-C) goals. In this post hoc analysis, we evaluated pooled safety and efficacy data from 3 studies in pediatric patients with HoFH treated with the PCSK9 (proprotein convertase subtilisin/kexin type 9) monoclonal antibody inhibitor evolocumab.

methodsPatients with HoFH aged 10 to 17 years received treatment with open-label evolocumab 420 mg subcutaneously monthly or biweekly in the TAUSSIG, RAMAN, or HAUSER-OLE clinical studies. All patients received background statins with or without ezetimibe. Study duration ranged from 12 to 260 weeks. The primary end point was treatment-emergent adverse events per 100 patient-years. Efficacy end points were changes from baseline to week 12 in lipids and PCSK9.

resultsOf the 39 patients in the pooled analysis, 69.2% were males, median age was 13.0 years, and 79.5% (31/39) had genotyped HoFH with

conclusionsIn this pooled data analysis from 3 studies in pediatric patients with HoFH, evolocumab was well tolerated, with no new safety signals reported. These safety findings are consistent with findings from previous studies of evolocumab. Patients showed marked variability in LDL-C reduction. Results from this pooled analysis support guidelines suggesting a trial of PCSK9 inhibitor therapy regardless of estimated residual LDLR function. REGISTRATION: URL: https://www.clinicaltrials.gov; Unique identifier: NCT01624142, NCT03403374, and NCT02624869.

Indexed as

Antibodies, Monoclonal, HumanizedAnticholesteremic AgentsCholesterol, LDLHomozygoteHyperlipoproteinemia Type IIPCSK9 InhibitorsAdolescentAge FactorsAntibodies, MonoclonalBiomarkersChildClinical Studies as TopicDrug Therapy, CombinationEzetimibeFemaleGenetic Predisposition to DiseaseAntibodies, MonoclonalAntibodies, Monoclonal, HumanizedAnticholesteremic AgentsBiomarkersCholesterol, LDLevolocumabEzetimibeHydroxymethylglutaryl-CoA Reductase InhibitorsPCSK9 InhibitorsPCSK9 protein, humanProprotein Convertase 9Serine Proteinase Inhibitorsatherosclerosiscardiovascular diseasehyperlipidemialipoproteins, LDL

Identifiers

PMID38545781
PMCPMC11042515
OpenAlexW4393265812

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC-ND
Read underepoch 390

Registered trials

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.