Real-world characteristics and treatment of cardiac transthyretin amyloidosis: A multicentre, observational study.

IF 3.2 2区医学 Q2 CARDIAC & CARDIOVASCULAR SYSTEMS

ESC Heart Failure Pub Date : 2024-11-06 DOI:10.1002/ehf2.15126

Richard J Nies, Svenja Ney, Ingrid Kindermann, Yvonne Bewarder, Angela Zimmer, Fabian Knebel, Katrin Hahn, Sebastian Spethmann, Peter Luedike, Lars Michel, Tienush Rassaf, Maria Papathanasiou, Stefan Störk, Vladimir Cejka, Amin Polzin, Fabian Voss, Malte Kelm, Bernhard Unsöld, Christine Meindl, Michael Paulus, Ali Yilmaz, Bishwas Chamling, Caroline Morbach, Roman Pfister

{"title":"Real-world characteristics and treatment of cardiac transthyretin amyloidosis: A multicentre, observational study.","authors":"Richard J Nies, Svenja Ney, Ingrid Kindermann, Yvonne Bewarder, Angela Zimmer, Fabian Knebel, Katrin Hahn, Sebastian Spethmann, Peter Luedike, Lars Michel, Tienush Rassaf, Maria Papathanasiou, Stefan Störk, Vladimir Cejka, Amin Polzin, Fabian Voss, Malte Kelm, Bernhard Unsöld, Christine Meindl, Michael Paulus, Ali Yilmaz, Bishwas Chamling, Caroline Morbach, Roman Pfister","doi":"10.1002/ehf2.15126","DOIUrl":null,"url":null,"abstract":"Aims: Data on the clinical profiles of patients with transthyretin amyloidosis cardiomyopathy (ATTR-CM) in the post-approval era of tafamidis 61 mg are lacking. Study aims were characterization of contemporary ATTR-CM patients, analysis of potential eligibility for the 'Transthyretin Amyloidosis Cardiomyopathy Clinical Trial' (ATTR-ACT) and identification of factors associated with the decision on tafamidis 61 mg treatment.Methods and results: This retrospective study analysed ATTR-CM patients seen at eight University Hospitals in the first year after approval of tafamidis 61 mg for ATTR-CM in Germany (April 2020 to March 2021). The cohort comprised 366 patients (median age 79 [74; 82] years, 84% male), with 47% and 45% of the cohort being in National Amyloidosis Centre ATTR stage ≥ II and NYHA class ≥ III, respectively. Sixty-four per cent of patients met key eligibility criteria of the pivotal ATTR-ACT. In recently diagnosed patients (58% with diagnosis ≤6 months), the rate of variant ATTR was significantly lower than in patients diagnosed more than 6 months ago (9.3% vs. 19.7%). Of the 293 patients without prior ATTR specific treatment, tafamidis 61 mg was newly initiated in 77%. Patients with tafamidis 61 mg treatment were significantly younger, were more often eligible for ATTR-ACT, had lower NYHA class and higher serum albumin levels. These variables explained 16% of the variance of treatment decision. Unadjusted survival was higher in patients with than those without treatment (1-year survival 98.6% vs. 87.3%, P < 0.001).Conclusions: Wild-type ATTR was the primary aetiology amongst contemporary ATTR-CM patients and almost two-thirds of patients were in an advanced disease stage. Clinical profiles of 64% of patients in routine care matched those of the ATTR-ACT. Further effort is needed to detect patients at an earlier disease stage and to validate criteria justifying treatment initiation.","PeriodicalId":11864,"journal":{"name":"ESC Heart Failure","volume":" ","pages":""},"PeriodicalIF":3.2000,"publicationDate":"2024-11-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"ESC Heart Failure","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1002/ehf2.15126","RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q2","JCRName":"CARDIAC & CARDIOVASCULAR SYSTEMS","Score":null,"Total":0}

引用次数: 0

Abstract

Aims: Data on the clinical profiles of patients with transthyretin amyloidosis cardiomyopathy (ATTR-CM) in the post-approval era of tafamidis 61 mg are lacking. Study aims were characterization of contemporary ATTR-CM patients, analysis of potential eligibility for the 'Transthyretin Amyloidosis Cardiomyopathy Clinical Trial' (ATTR-ACT) and identification of factors associated with the decision on tafamidis 61 mg treatment.

Methods and results: This retrospective study analysed ATTR-CM patients seen at eight University Hospitals in the first year after approval of tafamidis 61 mg for ATTR-CM in Germany (April 2020 to March 2021). The cohort comprised 366 patients (median age 79 [74; 82] years, 84% male), with 47% and 45% of the cohort being in National Amyloidosis Centre ATTR stage ≥ II and NYHA class ≥ III, respectively. Sixty-four per cent of patients met key eligibility criteria of the pivotal ATTR-ACT. In recently diagnosed patients (58% with diagnosis ≤6 months), the rate of variant ATTR was significantly lower than in patients diagnosed more than 6 months ago (9.3% vs. 19.7%). Of the 293 patients without prior ATTR specific treatment, tafamidis 61 mg was newly initiated in 77%. Patients with tafamidis 61 mg treatment were significantly younger, were more often eligible for ATTR-ACT, had lower NYHA class and higher serum albumin levels. These variables explained 16% of the variance of treatment decision. Unadjusted survival was higher in patients with than those without treatment (1-year survival 98.6% vs. 87.3%, P < 0.001).

Conclusions: Wild-type ATTR was the primary aetiology amongst contemporary ATTR-CM patients and almost two-thirds of patients were in an advanced disease stage. Clinical profiles of 64% of patients in routine care matched those of the ATTR-ACT. Further effort is needed to detect patients at an earlier disease stage and to validate criteria justifying treatment initiation.

查看原文本刊更多论文

心脏转甲状腺素淀粉样变性的真实世界特征与治疗：一项多中心观察研究。

研究目的：在他法米迪 61 毫克获批后的时代，有关经淀粉样蛋白淀粉样变性心肌病（ATTR-CM）患者临床概况的数据还很缺乏。研究目的是描述当代 ATTR-CM 患者的特征，分析 "转甲状腺素淀粉样变性心肌病临床试验"（ATTR-ACT）的潜在资格，并确定与他法米地 61 毫克治疗决定相关的因素：这项回顾性研究分析了德国批准塔法米地61毫克治疗ATTR-CM后第一年（2020年4月至2021年3月）在八家大学医院就诊的ATTR-CM患者。队列中有366名患者（中位年龄79 [74; 82]岁，84%为男性），其中47%和45%的患者分别处于国家淀粉样变性中心ATTR≥II期和NYHA≥III级。64%的患者符合关键ATTR-ACT的主要资格标准。在最近确诊的患者中（58%确诊时间≤6个月），变异ATTR的发生率明显低于6个月前确诊的患者（9.3%对19.7%）。在293名之前未接受过ATTR特异性治疗的患者中，77%的患者新近开始使用他法米迪61毫克。接受他法米迪 61 毫克治疗的患者明显更年轻，更常符合 ATTR-ACT 的条件，NYHA 分级更低，血清白蛋白水平更高。这些变量解释了 16% 的治疗决策变异。接受治疗的患者未经调整的存活率高于未接受治疗的患者（1 年存活率为 98.6% 对 87.3%，P 结论）：野生型 ATTR 是当代 ATTR-CM 患者的主要病因，近三分之二的患者处于疾病晚期。64%的常规治疗患者的临床特征与ATTR-ACT相符。我们需要进一步努力，检测出处于疾病早期阶段的患者，并验证启动治疗的标准。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

求助全文

约1分钟内获得全文求助全文

来源期刊

ESC Heart Failure Medicine-Cardiology and Cardiovascular Medicine

CiteScore

7.00

自引率

7.90%

发文量

461

审稿时长

12 weeks

期刊介绍： ESC Heart Failure is the open access journal of the Heart Failure Association of the European Society of Cardiology dedicated to the advancement of knowledge in the field of heart failure. The journal aims to improve the understanding, prevention, investigation and treatment of heart failure. Molecular and cellular biology, pathology, physiology, electrophysiology, pharmacology, as well as the clinical, social and population sciences all form part of the discipline that is heart failure. Accordingly, submission of manuscripts on basic, translational, clinical and population sciences is invited. Original contributions on nursing, care of the elderly, primary care, health economics and other specialist fields related to heart failure are also welcome, as are case reports that highlight interesting aspects of heart failure care and treatment.