{"title":"他法米迪对肝移植后遗传性转甲状腺素心脏淀粉样变性患者的初步疗效和安全性分析","authors":"Takahiro Okumura, Kenji Furusawa, Ryota Ito, Hiroaki Hiraiwa, Toyoaki Murohara","doi":"10.1016/j.arcmed.2024.103083","DOIUrl":null,"url":null,"abstract":"<div><p>Hereditary transthyretin cardiac amyloidosis (ATTRv-CA) after liver transplantation remains challenging to treat due to residual amyloid deposits in extrahepatic organs, including the heart. Tafamidis, a transthyretin tetramer stabilizer, has shown promise in the treatment of ATTRv-CA; however, its efficacy and safety after liver transplantation are uncertain. In this preliminary retrospective review, we assessed the efficacy and safety of tafamidis (80 mg) in three ATTRv-CA cases after liver transplantation. Following one year of treatment, all patients experienced improvement in dyspnea, New York Heart Association functional class, brain natriuretic peptide levels, and cardiac troponin T levels. No significant changes in echocardiographic parameters were observed. Notably, no cardiovascular or drug-related adverse events occurred during treatment. Our findings suggest that tafamidis may benefit post-liver transplant patients with ATTRv-CA and warrant further investigation through randomized controlled trials with larger cohorts. This study highlights a potential therapeutic avenue for the management of cardiovascular involvement in this challenging patient population.</p></div>","PeriodicalId":8318,"journal":{"name":"Archives of Medical Research","volume":"56 1","pages":"Article 103083"},"PeriodicalIF":4.7000,"publicationDate":"2024-09-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Preliminary efficacy and safety analysis of tafamidis in post-liver transplant patients with hereditary transthyretin cardiac amyloidosis\",\"authors\":\"Takahiro Okumura, Kenji Furusawa, Ryota Ito, Hiroaki Hiraiwa, Toyoaki Murohara\",\"doi\":\"10.1016/j.arcmed.2024.103083\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<div><p>Hereditary transthyretin cardiac amyloidosis (ATTRv-CA) after liver transplantation remains challenging to treat due to residual amyloid deposits in extrahepatic organs, including the heart. Tafamidis, a transthyretin tetramer stabilizer, has shown promise in the treatment of ATTRv-CA; however, its efficacy and safety after liver transplantation are uncertain. In this preliminary retrospective review, we assessed the efficacy and safety of tafamidis (80 mg) in three ATTRv-CA cases after liver transplantation. Following one year of treatment, all patients experienced improvement in dyspnea, New York Heart Association functional class, brain natriuretic peptide levels, and cardiac troponin T levels. No significant changes in echocardiographic parameters were observed. Notably, no cardiovascular or drug-related adverse events occurred during treatment. Our findings suggest that tafamidis may benefit post-liver transplant patients with ATTRv-CA and warrant further investigation through randomized controlled trials with larger cohorts. This study highlights a potential therapeutic avenue for the management of cardiovascular involvement in this challenging patient population.</p></div>\",\"PeriodicalId\":8318,\"journal\":{\"name\":\"Archives of Medical Research\",\"volume\":\"56 1\",\"pages\":\"Article 103083\"},\"PeriodicalIF\":4.7000,\"publicationDate\":\"2024-09-17\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Archives of Medical Research\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://www.sciencedirect.com/science/article/pii/S0188440924001358\",\"RegionNum\":3,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q1\",\"JCRName\":\"MEDICINE, RESEARCH & EXPERIMENTAL\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Archives of Medical Research","FirstCategoryId":"3","ListUrlMain":"https://www.sciencedirect.com/science/article/pii/S0188440924001358","RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"MEDICINE, RESEARCH & EXPERIMENTAL","Score":null,"Total":0}
引用次数: 0
摘要
肝移植后的遗传性转甲状腺素心脏淀粉样变性(ATTRv-CA)由于在包括心脏在内的肝外器官中残留淀粉样蛋白沉积物,其治疗仍具有挑战性。Tafamidis是一种转甲状腺素四聚体稳定剂,已显示出治疗ATTRv-CA的前景;然而,其在肝移植后的疗效和安全性尚不确定。在这项初步回顾性研究中,我们评估了他法米迪(80 毫克)在三例 ATTRv-CA 肝移植后的疗效和安全性。治疗一年后,所有患者的呼吸困难、纽约心脏协会功能分级、脑钠肽水平和心肌肌钙蛋白 T 水平均有所改善。超声心动图参数未见明显变化。值得注意的是,治疗期间未发生心血管或药物相关不良事件。我们的研究结果表明,他法米迪可能对肝移植后的 ATTRv-CA 患者有益,值得通过更大规模的随机对照试验进行进一步研究。这项研究为治疗这一具有挑战性的患者群体的心血管疾病提供了一条潜在的治疗途径。
Preliminary efficacy and safety analysis of tafamidis in post-liver transplant patients with hereditary transthyretin cardiac amyloidosis
Hereditary transthyretin cardiac amyloidosis (ATTRv-CA) after liver transplantation remains challenging to treat due to residual amyloid deposits in extrahepatic organs, including the heart. Tafamidis, a transthyretin tetramer stabilizer, has shown promise in the treatment of ATTRv-CA; however, its efficacy and safety after liver transplantation are uncertain. In this preliminary retrospective review, we assessed the efficacy and safety of tafamidis (80 mg) in three ATTRv-CA cases after liver transplantation. Following one year of treatment, all patients experienced improvement in dyspnea, New York Heart Association functional class, brain natriuretic peptide levels, and cardiac troponin T levels. No significant changes in echocardiographic parameters were observed. Notably, no cardiovascular or drug-related adverse events occurred during treatment. Our findings suggest that tafamidis may benefit post-liver transplant patients with ATTRv-CA and warrant further investigation through randomized controlled trials with larger cohorts. This study highlights a potential therapeutic avenue for the management of cardiovascular involvement in this challenging patient population.
期刊介绍:
Archives of Medical Research serves as a platform for publishing original peer-reviewed medical research, aiming to bridge gaps created by medical specialization. The journal covers three main categories - biomedical, clinical, and epidemiological contributions, along with review articles and preliminary communications. With an international scope, it presents the study of diseases from diverse perspectives, offering the medical community original investigations ranging from molecular biology to clinical epidemiology in a single publication.