Real-world safety and efficacy of lomitapide in homozygous familial hypercholesterolemia: interim report of special-use survey in Japan.

IF 1.6 Q3 CARDIAC & CARDIOVASCULAR SYSTEMS

Future cardiology Pub Date : 2024-02-01 Epub Date: 2024-02-29 DOI:10.2217/fca-2023-0136

Mariko Harada-Shiba, Shigenori Haruna, Noriaki Kogawa

引用次数: 0

Abstract

Aim: To evaluate the safety and efficacy of lomitapide in real-world clinical practice in Japan. Patients & methods: Interim analysis of 39 patients with homozygous familial hypercholesterolemia from an all-case surveillance study. Results: Median lomitapide dose (across 42 months) was 9.8 mg/day. 74 drug-related adverse events (AEs) were reported in 24 (61.5%) patients, including 14 (35.9%) with liver-related AEs, 19 (48.7%) with gastrointestinal disorders and 1 (2.6%) bleeding disorder. Lomitapide dose was reduced for 39.2% of drug-related AEs, withdrawn temporarily for 12.2%, and discontinued for 1 event (1.4%). Mean ± SD blood LDL-C level decreased from 225.9 ± 172.0 mg/dl (5.8 ± 4.5 mmol/l) predose to 159.4 ± 93.0 mg/dl (4.1 ± 2.4 mmol/l) at 12 months (p = 0.0245). Conclusion: This interim analysis suggests lomitapide is safe and effective in real-world clinical practice in Japan.

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洛米他匹对同型家族性高胆固醇血症的实际安全性和疗效：日本特殊用途调查中期报告。

目的：评估洛美他匹在日本实际临床实践中的安全性和有效性。患者和方法对一项全病例监测研究中的 39 例同源家族性高胆固醇血症患者进行中期分析。研究结果洛米他匹中位剂量（42个月）为9.8毫克/天。24例（61.5%）患者报告了74例药物相关不良事件（AEs），其中14例（35.9%）为肝脏相关不良事件，19例（48.7%）为胃肠道疾病，1例（2.6%）为出血性疾病。在39.2%的药物相关不良反应中，洛米他匹被减量，12.2%暂时停药，1例（1.4%）停药。平均±标清血液低密度脂蛋白胆固醇水平从用药前的 225.9 ± 172.0 mg/dl (5.8 ± 4.5 mmol/l)降至 12 个月时的 159.4 ± 93.0 mg/dl (4.1 ± 2.4 mmol/l)（p = 0.0245）。结论这项中期分析表明，在日本的实际临床实践中，洛美他匹是安全有效的。

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

Future cardiology CARDIAC & CARDIOVASCULAR SYSTEMS-

CiteScore

2.80

自引率

5.90%

发文量

期刊介绍： Research advances have contributed to improved outcomes across all specialties, but the rate of advancement in cardiology has been exceptional. Concurrently, the population of patients with cardiac conditions continues to grow and greater public awareness has increased patients" expectations of new drugs and devices. Future Cardiology (ISSN 1479-6678) reflects this new era of cardiology and highlights the new molecular approach to advancing cardiovascular therapy. Coverage will also reflect the major technological advances in bioengineering in cardiology in terms of advanced and robust devices, miniaturization, imaging, system modeling and information management issues.