Evidence map›Paper›PMID 31102204›Full record

ArticleAdvances in therapy2019

Real-World Outcomes with Lomitapide Use in Paediatric Patients with Homozygous Familial Hypercholesterolaemia.

Tawfeg Ben-Omran, Luis Masana, Genovefa Kolovou, Gema Ariceta, F Javier Nóvoa, Allan M Lund, Martin P Bogsrud, María Araujo, Osamah Hussein, Daiana Ibarretxe and 2 more

Erratum issuedOpen access · hybridAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Advances in therapy, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. Cited by 24 papers, 3 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
24citing papers in PubMed, 3 pooled it
7.1field-weighted citation impact, top 2% 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.

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

24 citing papers in PubMed, 3 syntheses or guidelines pooled it, 53 citations in OpenAlex.

  1. Pooled it
  2. Pooled it
  3. Familial hypercholesterolemia - treatment update in children, systematic review.Pediatric endocrinology, diabetes, and metabolism · 2022
    Pooled it
  4. Review
  5. Review
  6. Review
  7. Review
  8. Review
  9. Review
  10. Review
  11. Review
  12. Review
  13. Article
  14. Review
  15. Article
  16. Case Studies in Pediatric Lipid Disorders and Their Management.The Journal of clinical endocrinology and metabolism · 2021
    Article
  17. Review
  18. Review
  19. Novel therapeutic targets and agents for pediatric dyslipidemia.Therapeutic advances in endocrinology and metabolism · 2021
    Review
  20. Review
4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

12 authors at 10 institutions in 8 countries.

Tawfeg Ben-OmranDivision of Clinical and Metabolic Genetics, Department of Pediatrics, Hamad Medical Corporation and Sidra Medicine, Doha, Qatar.
Luis MasanaVascular Medicine and Metabolism Unit, Universitat Rovira i Virgili, IISPV, CIBERDEM, Reus, Spain.
Genovefa KolovouOnassis Cardiac Surgery Center, Athens, Greece.
Gema AricetaPediatric Kidney Diseases, University Hospital Vall d' Hebron, Barcelona, Spain.
F Javier NóvoaEndocrinology Department, University Hospital Insular de Gran Canaria, University Institute of Biomedical and Health Research of the University of Las Palmas de Gran Canaria, Las Palmas, Spain.
Allan M LundCenter for Inherited Metabolic Diseases, Departments of Paediatrics and Clinical Genetics, Copenhagen University Hospital, Rigshospitalet, Copenhagen, Denmark.
Martin P BogsrudNational Advisory Unit for Familial Hypercholesterolemia, Oslo University Hospital, Oslo, Norway.
María AraujoNutrition Service, Hospital Nacional de Pediatría 'Dr Juan P. Garrahan', Buenos Aires, Argentina.
Osamah HusseinInternal Medicine Department 'A, Ziv Medical Centre, Azreili Faculty of Medicine, Bar Ilan University, Safed, Israel.
Daiana IbarretxeVascular Medicine and Metabolism Unit, Universitat Rovira i Virgili, IISPV, CIBERDEM, Reus, Spain.
Rosa M Sanchez-HernándezEndocrinology Department, University Hospital Insular de Gran Canaria, University Institute of Biomedical and Health Research of the University of Las Palmas de Gran Canaria, Las Palmas, Spain.
Raul D SantosLipid Clinic, Heart Institute (InCor), University of Sao Paulo and Hospital Israelita Albert Einstein, São Paulo, Brazil. raul.santos@incor.usp.br.ORCID http://orcid.org/0000-0002-9860-6582
Institut de Recerca Biomèdica Catalunya Sud · ESUniversidad de Las Palmas de Gran Canaria · ESBar-Ilan University · ILCopenhagen University Hospital · DKGarrahan Hospital · ARHamad Medical Corporation · QAOnassis Cardiac Surgery Center · GROslo University Hospital · NOUniversidade de São Paulo · BRVall d'Hebron Hospital Universitari · ES

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

introductionHomozygous familial hypercholesterolaemia (HoFH) is a rare, autosomal disease affecting the clearance of low-density lipoprotein cholesterol (LDL-C) from circulation, and leading to early-onset atherosclerotic cardiovascular disease (ASCVD). Treatment consists mainly of statins, lipoprotein apheresis (LA) and, more recently, the microsomal triglyceride transfer protein inhibitor lomitapide. Lomitapide is not licensed for use in children, but has been made available through an expanded access programme or on a named patient basis.

methodsThis case series includes 11 HoFH patients in 10 different centres in eight countries, less than 18 years of age (mean 11.6 ± 1.1 years, 64% male), with signs of ASCVD, and who have received treatment with lomitapide (mean dose 24.5 ± 4.3 mg/day; mean exposure 20.0 ± 2.9 months). Background lipid-lowering therapy was given according to local protocols. Lomitapide was commenced with a stepwise dose escalation from 2.5 mg or 5 mg/day; dietary advice and vitamin supplements were provided as per the product label for adults. Laboratory analysis was conducted as part of regular clinical care.

resultsIn the 11 cases, mean baseline LDL-C was 419 ± 74.6 mg/dL and was markedly reduced by lomitapide to a nadir of 176.7 ± 46.3 mg/dL (58.4 ± 6.8% decrease). Six patients achieved recommended target levels for children below 135 mg/dL, five of whom had LA frequency reduced. In one case, LDL-C levels were close to target when lomitapide was started but remained stable despite 75% reduction in LA frequency (from twice weekly to biweekly). Adverse events were mainly gastrointestinal in nature, occurred early in the treatment course and were well managed. Three patients with excursions in liver function tests were managed chiefly without intervention; two patients had decreases in lomitapide dose.

conclusionsLomitapide demonstrated promising effectiveness in paediatric HoFH patients. Adverse events were manageable, and the clinical profile of the drug is apparently similar to that in adult patients.

fundingAmryt Pharma.

Indexed as

HomozygoteAdolescentAdultAnticholesteremic AgentsChildCholesterol, LDLFemaleHumansHydroxymethylglutaryl-CoA Reductase InhibitorsHyperlipoproteinemia Type IIHypolipidemic AgentsMaleAnticholesteremic AgentsCholesterol, LDLHydroxymethylglutaryl-CoA Reductase InhibitorsHypolipidemic AgentsAdverse eventsAtherosclerosisCardiologyHomozygous familial hypercholesterolaemiaLipidologyLomitapideLow-density lipoprotein cholesterolPaediatricPatient casesReal-world data

Identifiers

PMID31102204
PMCPMC6824397
OpenAlexW2945967174

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC
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 OpenQuestion graph.