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Left main revascularization with optical coherence tomography in a young male with newly diagnosed homozygous familial hypercholesterolemia.

ABSTRACT: Familial hypercholesterolemia is the most common primary lipid disorder responsible for coronary artery disease. A 22-year-old male presented with history of accelerating angina and extensive xanthomatous lesions all over the body. Coronary angiography showed significant left main and left anterior descending artery disease. Optical coherence tomography (OCT) imaging revealed areas of lipid rich plaque with thin capped fibrous atheroma, and inflammatory cell infiltration. Area of maximum stenosis showed fibrous plaque with dense lipid pool. OCT in this patient clarified plaque characteristics and achieved optimal procedural results. There were only few case reports which described OCT findings in patients with familial hypercholesterolemia. <Learning objective: Undiagnosed familial hypercholesterolemia may present with life-threatening complications. The index case presented with critical left main disease. Intravascular imaging is necessary in patients with primary lipid disorders undergoing percutaneous coronary intervention, which characterizes plaque, the index case had densely lipid rich plaque with areas of inflammation. Characterizing plaque characteristics in patients with lipid disorders might have an impact on the management of these patients, which needs to be clarified in appropriately sized studies.>.

SUBMITTER: Revaiah PC

PROVIDER: S-EPMC8721252 | biostudies-literature |

REPOSITORIES: biostudies-literature

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Project description: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.RegistrationURL: https://www.clinicaltrials.gov; Unique identifier: NCT04233918.

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Left main revascularization with optical coherence tomography in a young male with newly diagnosed homozygous familial hypercholesterolemia.

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