{"title":"培马布特治疗高脂血症的长期安全性和有效性：在日本上市后24个月的观察监测","authors":"Mao Watanabe, Michiko Nakanishi, Wataru Shingaki, Ryoji Gunji, Yuichi Makinose, Takashi Kanno, Shun Ishibashi","doi":"10.1007/s12325-025-03294-5","DOIUrl":null,"url":null,"abstract":"<div><h3>Introduction</h3><p>Pemafibrate was well tolerated and effective in clinical trials that led to its approval to treat hyperlipidemia. However, these clinical trials typically have limitations and may not sufficiently assess the performance of pemafibrate in the real-world setting. Therefore, the aim of this study was to evaluate the long-term safety and efficacy of pemafibrate in patients with hyperlipidemia in Japan.</p><h3>Methods</h3><p>This was a prospective, multicenter, open-label, observational post-marketing surveillance (PMS) study. Patients with hyperlipidemia were observed for 24 months from the start of pemafibrate treatment. Safety evaluations included the incidence of adverse drug reactions (ADRs), subgroup analysis of ADRs, and changes in laboratory test values. Efficacy was determined by the changes in lipid test values from baseline.</p><h3>Results</h3><p>A total of 3672 cases were registered from 612 facilities in Japan. In the safety analysis, 68.1% of patients were male, mean age was 60.8 years, and 37.8% were switched to pemafibrate from other dyslipidemia agents. In total, 147 patients (4.07%) experienced ADRs; serious ADRs were reported in 8 patients (0.22%). The most common ADRs included abnormalities in laboratory tests (<i>n</i> = 60; 1.66%), hepatobiliary disorders (<i>n</i> = 24, 0.66%), musculoskeletal and connective tissue disorders (<i>n</i> = 14, 0.39%), and metabolic and nutritional disorders (<i>n</i> = 14, 0.39%). No incidents of rhabdomyolysis occurred, and 37 (1.02%) patients experienced rhabdomyolysis-related ADRs. Renal-related ADRs occurred in 13 patients (0.36%). When subdivided by patient background, the incidence of ADRs was significantly higher in patients aged ≥ 75 years than < 75 years (<i>P</i> = 0.028). There were no significant differences in other subgroups. The mean percent change in mixed fasting and non-fasting triglyceride levels was − 35.2% (<i>P</i> < 0.001). High- and low-density lipoprotein cholesterol levels both increased significantly.</p><h3>Conclusion</h3><p>This PMS study demonstrated the safety and efficacy of pemafibrate after long-term administration in patients with hyperlipidemia.</p></div>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":"42 10","pages":"4950 - 4963"},"PeriodicalIF":4.0000,"publicationDate":"2025-07-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://link.springer.com/content/pdf/10.1007/s12325-025-03294-5.pdf","citationCount":"0","resultStr":"{\"title\":\"Long-Term Safety and Efficacy of Pemafibrate to Treat Hyperlipidemia: Post-marketing Surveillance Over a 24-Month Observation Period in Japan\",\"authors\":\"Mao Watanabe, Michiko Nakanishi, Wataru Shingaki, Ryoji Gunji, Yuichi Makinose, Takashi Kanno, Shun Ishibashi\",\"doi\":\"10.1007/s12325-025-03294-5\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<div><h3>Introduction</h3><p>Pemafibrate was well tolerated and effective in clinical trials that led to its approval to treat hyperlipidemia. However, these clinical trials typically have limitations and may not sufficiently assess the performance of pemafibrate in the real-world setting. Therefore, the aim of this study was to evaluate the long-term safety and efficacy of pemafibrate in patients with hyperlipidemia in Japan.</p><h3>Methods</h3><p>This was a prospective, multicenter, open-label, observational post-marketing surveillance (PMS) study. Patients with hyperlipidemia were observed for 24 months from the start of pemafibrate treatment. Safety evaluations included the incidence of adverse drug reactions (ADRs), subgroup analysis of ADRs, and changes in laboratory test values. Efficacy was determined by the changes in lipid test values from baseline.</p><h3>Results</h3><p>A total of 3672 cases were registered from 612 facilities in Japan. In the safety analysis, 68.1% of patients were male, mean age was 60.8 years, and 37.8% were switched to pemafibrate from other dyslipidemia agents. In total, 147 patients (4.07%) experienced ADRs; serious ADRs were reported in 8 patients (0.22%). The most common ADRs included abnormalities in laboratory tests (<i>n</i> = 60; 1.66%), hepatobiliary disorders (<i>n</i> = 24, 0.66%), musculoskeletal and connective tissue disorders (<i>n</i> = 14, 0.39%), and metabolic and nutritional disorders (<i>n</i> = 14, 0.39%). No incidents of rhabdomyolysis occurred, and 37 (1.02%) patients experienced rhabdomyolysis-related ADRs. Renal-related ADRs occurred in 13 patients (0.36%). When subdivided by patient background, the incidence of ADRs was significantly higher in patients aged ≥ 75 years than < 75 years (<i>P</i> = 0.028). There were no significant differences in other subgroups. The mean percent change in mixed fasting and non-fasting triglyceride levels was − 35.2% (<i>P</i> < 0.001). High- and low-density lipoprotein cholesterol levels both increased significantly.</p><h3>Conclusion</h3><p>This PMS study demonstrated the safety and efficacy of pemafibrate after long-term administration in patients with hyperlipidemia.</p></div>\",\"PeriodicalId\":7482,\"journal\":{\"name\":\"Advances in Therapy\",\"volume\":\"42 10\",\"pages\":\"4950 - 4963\"},\"PeriodicalIF\":4.0000,\"publicationDate\":\"2025-07-31\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"https://link.springer.com/content/pdf/10.1007/s12325-025-03294-5.pdf\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Advances in Therapy\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://link.springer.com/article/10.1007/s12325-025-03294-5\",\"RegionNum\":3,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q2\",\"JCRName\":\"MEDICINE, RESEARCH & EXPERIMENTAL\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Advances in Therapy","FirstCategoryId":"3","ListUrlMain":"https://link.springer.com/article/10.1007/s12325-025-03294-5","RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q2","JCRName":"MEDICINE, RESEARCH & EXPERIMENTAL","Score":null,"Total":0}
Long-Term Safety and Efficacy of Pemafibrate to Treat Hyperlipidemia: Post-marketing Surveillance Over a 24-Month Observation Period in Japan
Introduction
Pemafibrate was well tolerated and effective in clinical trials that led to its approval to treat hyperlipidemia. However, these clinical trials typically have limitations and may not sufficiently assess the performance of pemafibrate in the real-world setting. Therefore, the aim of this study was to evaluate the long-term safety and efficacy of pemafibrate in patients with hyperlipidemia in Japan.
Methods
This was a prospective, multicenter, open-label, observational post-marketing surveillance (PMS) study. Patients with hyperlipidemia were observed for 24 months from the start of pemafibrate treatment. Safety evaluations included the incidence of adverse drug reactions (ADRs), subgroup analysis of ADRs, and changes in laboratory test values. Efficacy was determined by the changes in lipid test values from baseline.
Results
A total of 3672 cases were registered from 612 facilities in Japan. In the safety analysis, 68.1% of patients were male, mean age was 60.8 years, and 37.8% were switched to pemafibrate from other dyslipidemia agents. In total, 147 patients (4.07%) experienced ADRs; serious ADRs were reported in 8 patients (0.22%). The most common ADRs included abnormalities in laboratory tests (n = 60; 1.66%), hepatobiliary disorders (n = 24, 0.66%), musculoskeletal and connective tissue disorders (n = 14, 0.39%), and metabolic and nutritional disorders (n = 14, 0.39%). No incidents of rhabdomyolysis occurred, and 37 (1.02%) patients experienced rhabdomyolysis-related ADRs. Renal-related ADRs occurred in 13 patients (0.36%). When subdivided by patient background, the incidence of ADRs was significantly higher in patients aged ≥ 75 years than < 75 years (P = 0.028). There were no significant differences in other subgroups. The mean percent change in mixed fasting and non-fasting triglyceride levels was − 35.2% (P < 0.001). High- and low-density lipoprotein cholesterol levels both increased significantly.
Conclusion
This PMS study demonstrated the safety and efficacy of pemafibrate after long-term administration in patients with hyperlipidemia.
期刊介绍：
Advances in Therapy is an international, peer reviewed, rapid-publication (peer review in 2 weeks, published 3–4 weeks from acceptance) journal dedicated to the publication of high-quality clinical (all phases), observational, real-world, and health outcomes research around the discovery, development, and use of therapeutics and interventions (including devices) across all therapeutic areas. Studies relating to diagnostics and diagnosis, pharmacoeconomics, public health, epidemiology, quality of life, and patient care, management, and education are also encouraged.
The journal is of interest to a broad audience of healthcare professionals and publishes original research, reviews, communications and letters. The journal is read by a global audience and receives submissions from all over the world. Advances in Therapy will consider all scientifically sound research be it positive, confirmatory or negative data. Submissions are welcomed whether they relate to an international and/or a country-specific audience, something that is crucially important when researchers are trying to target more specific patient populations. This inclusive approach allows the journal to assist in the dissemination of all scientifically and ethically sound research.
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