Articles producció científica> Medicina i Cirurgia

A Case Series Assessing the Effects of Lomitapide on Carotid Intima-Media Thickness in Adult Patients with Homozygous Familial Hypercholesterolaemia in a Real-World Setting

  • Identification data

    Identifier: imarina:9246567
  • Authors:

    Blom DJ
    Gaudet D
    Hegele RA
    Patel DS
    Cegla J
    Kolovou G
    Marin LM
  • Others:

    Author, as appears in the article.: Blom DJ; Gaudet D; Hegele RA; Patel DS; Cegla J; Kolovou G; Marin LM
    Department: Medicina i Cirurgia
    URV's Author/s: Masana Marín, Luis
    Keywords: Triglyceride transfer protein Low-density lipoprotein cholesterol Lomitapide Homozygous familial hypercholesterolemia Carotid intima-media thickness Cardiovascular surrogate marker risk-factors management low-density lipoprotein cholesterol lomitapide guidance efficacy disease clinician carotid intima-media thickness cardiovascular association arterial-wall thickness
    Abstract: Introduction: Homozygous familial hypercholesterolaemia (HoFH) is characterised by extremely elevated levels of low-density lipoprotein cholesterol (LDL-C) and results from multiple mutations in genes affecting the LDL receptor pathway. Patients are at risk of premature atherosclerotic cardiovascular disease (ASCVD) and premature death. Lomitapide is a microsomal triglyceride transfer protein inhibitor developed to treat HoFH, but cardiovascular outcome data are lacking. Methods: We evaluated detailed data from five HoFH patients and one patient with heterozygous FH (HeFH) and a very severe phenotype. We also analysed confirmatory data from a further 8 HoFH cases. In total, we analysed data from patients in seven global centres in six countries who were all treated with lomitapide with long-term follow-up. Carotid intima-media thickness (CIMT) imaging was recorded on an ad hoc basis to monitor ASCVD in HoFH. Results: Lomitapide resulted in marked decreases in LDL-C of 56.8–93.9% [77.7–93.9% in the 6 initial cases (mean nadir 64.8 ± 30.1 mg/dL); 56.8–86.0% in the 8 confirmatory cases (mean nadir 131.4 ± 38.2 mg/dL)]. CIMT regressed in 50% of cases (mean follow-up 5.0 ± 3.1 years in initial six cases, and 4.4 ± 1.4 years in confirmatory cases). In the remaining patients, CIMT showed little further change. In patients where assessments of plaque area were available, regression or stabilisation in CIMT was accompanied by clinically significant regression of plaque area. Conclusions: Lomitapide reduces LDL-C levels in patients with HoFH and severe LDL-C phenotypes, and results in stabilisation and/or regression of CIMT, which is an established marker of ASCVD risk. Additional data are needed to determine if this confers a survival benefit in these very high-risk patients.
    Thematic Areas: Pharmacology (medical) Pharmacology & pharmacy Medicine, research & experimental Medicine (miscellaneous) Medicina iii Medicina ii Medicina i General medicine
    licence for use: https://creativecommons.org/licenses/by/3.0/es/
    Author's mail: luis.masana@urv.cat
    Author identifier: 0000-0002-0789-4954
    Record's date: 2024-07-20
    Papper version: info:eu-repo/semantics/publishedVersion
    Link to the original source: https://link.springer.com/article/10.1007/s12325-021-02031-y
    Licence document URL: http://repositori.urv.cat/ca/proteccio-de-dades/
    Papper original source: Advances In Therapy. 39 (4): 1857-1870
    APA: Blom DJ; Gaudet D; Hegele RA; Patel DS; Cegla J; Kolovou G; Marin LM (2022). A Case Series Assessing the Effects of Lomitapide on Carotid Intima-Media Thickness in Adult Patients with Homozygous Familial Hypercholesterolaemia in a Real-World Setting. Advances In Therapy, 39(4), 1857-1870. DOI: 10.1007/s12325-021-02031-y
    Article's DOI: 10.1007/s12325-021-02031-y
    Entity: Universitat Rovira i Virgili
    Journal publication year: 2022
    Publication Type: Journal Publications
  • Keywords:

    Medicine (Miscellaneous),Medicine, Research & Experimental,Pharmacology & Pharmacy,Pharmacology (Medical)
    Triglyceride transfer protein
    Low-density lipoprotein cholesterol
    Lomitapide
    Homozygous familial hypercholesterolemia
    Carotid intima-media thickness
    Cardiovascular
    surrogate marker
    risk-factors
    management
    low-density lipoprotein cholesterol
    lomitapide
    guidance
    efficacy
    disease
    clinician
    carotid intima-media thickness
    cardiovascular
    association
    arterial-wall thickness
    Pharmacology (medical)
    Pharmacology & pharmacy
    Medicine, research & experimental
    Medicine (miscellaneous)
    Medicina iii
    Medicina ii
    Medicina i
    General medicine
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