Simulation model to estimate pretreatment (baseline) low-density lipoprotein cholesterol levels in people living with homozygous familial hypercholesterolemia.

IF 4.6 3区医学 Q2 PHARMACOLOGY & PHARMACY

Journal of clinical lipidology Pub Date : 2025-08-19 DOI:10.1016/j.jacl.2025.08.011

Jing Gu, Robert J Sanchez, Alexandra Koumas, Sandeep Tripathi, Harshit Dixit, Adam Amer, Laurence Sperling, Christie M Ballantyne

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引用次数: 0

Abstract

Introduction: Pretreatment levels of low-density lipoprotein cholesterol (LDL-C) are a primary consideration for the clinical diagnosis of homozygous familial hypercholesterolemia (HoFH). However, determination of pretreatment LDL-C can be challenging in this population because many individuals begin lipid-lowering therapy (LLT) early in life before diagnosis of HoFH.

Methods: A previously developed simulation model was adapted to estimate pretreatment LDL-C in people living with HoFH, based on their treated LDL-C value and current LLT (statin, ezetimibe, alirocumab, evolocumab, and/or lipoprotein apheresis).

Results: The tool includes an interface for users to provide relevant details for estimating non-clinical pretreatment LDL-C, specifically for patients suspected of having HoFH.

Conclusion: This HoFH-specific calculator is provided as a supplemental tool to facilitate education about and awareness of the potential of having HoFH, which may lead to reduced time to appropriate treatments for people living with HoFH and, ultimately, optimize their outcomes.

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纯合子家族性高胆固醇血症患者预处理（基线）低密度脂蛋白胆固醇水平的模拟模型

前言：低密度脂蛋白胆固醇（LDL-C）预处理水平是纯合子家族性高胆固醇血症（HoFH）临床诊断的主要考虑因素。然而，在这一人群中，确定预处理LDL-C可能具有挑战性，因为许多人在诊断出HoFH之前就开始了降脂治疗（LLT）。方法：采用先前开发的模拟模型，根据治疗后的LDL-C值和当前LLT（他汀类药物、依折麦布、alirocumab、evolocumab和/或脂蛋白单采）估计HoFH患者的预处理LDL-C。结果：该工具包括一个界面，供用户提供估算非临床预处理LDL-C的相关细节，特别是疑似患有HoFH的患者。结论：该HoFH特异性计算器可作为一种辅助工具，促进对HoFH潜在患者的教育和认识，从而减少HoFH患者接受适当治疗的时间，并最终优化其预后。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Journal of clinical lipidology 医学-药学

CiteScore

7.00

自引率

6.80%

发文量

209

审稿时长

49 days

期刊介绍： Because the scope of clinical lipidology is broad, the topics addressed by the Journal are equally diverse. Typical articles explore lipidology as it is practiced in the treatment setting, recent developments in pharmacological research, reports of treatment and trials, case studies, the impact of lifestyle modification, and similar academic material of interest to the practitioner. Sections of Journal of clinical lipidology will address pioneering studies and the clinicians who conduct them, case studies, ethical standards and conduct, professional guidance such as ATP and NCEP, editorial commentary, letters from readers, National Lipid Association (NLA) news and upcoming event information, as well as abstracts from the NLA annual scientific sessions and the scientific forums held by its chapters, when appropriate.