转甲状腺素淀粉样心肌病的当代长期他法米底治疗结果的多中心研究

IF 12 1区医学 Q1 CARDIAC & CARDIOVASCULAR SYSTEMS

Jacc: Cardiooncology Pub Date : 2025-04-01 DOI:10.1016/j.jaccao.2024.12.005

Ahmad Masri MD, MS , Priyanka Bhattacharya MD , Brent Medoff MD , Ain U. Ejaz MD , Miriam R. Elman MS, MPH , Pranav Chandrashekar MD , Lauren Ives MPH , Alfonsina Mirabal Santos MD , Sergio L. Teruya MD , Yuanzi Zhao MD, PhD , Shuaiqi Huang PhD , Xiaofeng Wang PhD , Brett W. Sperry MD , Mathew S. Maurer MD , Prem Soman MD, PhD , Mazen Hanna MD

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引用次数: 0

摘要

背景：在attri - act试验中，他法非底能提高生存率并降低心血管住院率。由于认识和早期诊断的提高，转甲状腺素淀粉样心肌病（atr - cm）的流行病学正在迅速发展。目的：作者试图评估接受他非他胺治疗的atr - cm患者的当代长期预后。方法纳入2018年至2021年期间在美国5个淀粉样变中心接受至少1剂他法米地的atr - cm患者。主要结局为全因死亡率。结果624例患者平均年龄76.9±8.4岁，女性12.5%，黑人17.5%，atr - cm变异17.5%。起始时，52%的患者NYHA功能为II级，34%的患者NYHA功能为III级，40%的患者处于国家淀粉样变性中心(NAC)≥II期，38%的患者处于哥伦比亚≥II期，NT-proBNP水平中位数为1,914 (Q1-Q3: 957-3914) pg/mL。中位随访43.2个月（Q1-Q3: 25.2-52.8个月），241例患者（38.6%）死亡。65个月时免于死亡的概率为54.1% （95% CI: 47.4%-60.4%）。同样，将队列限制为在atr - cm诊断后6个月内接受他法米地的患者（n = 397, 63.6%），结果相似，65个月时生存率为49.6% （95% CI: 37.6%-60.5%）。结论：在一组接受他非他汀治疗的atr - cm患者中，39%的患者在43个月内死亡。需要进一步的工作来提高我们对atr - cm的理解，其自然史，以及如何进一步提高生存率和预防心力衰竭的进展。

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A Multicenter Study of Contemporary Long-Term Tafamidis Outcomes in Transthyretin Amyloid Cardiomyopathy

Background

Tafamidis improved survival and decreased cardiovascular hospitalizations in the ATTR-ACT trial. Due to improved recognition and earlier diagnosis, the epidemiology of transthyretin amyloid cardiomyopathy (ATTR-CM) is rapidly evolving.

Objectives

The authors sought to evaluate the contemporary long-term outcomes of patients with ATTR-CM treated with tafamidis.

Methods

Patients with ATTR-CM who received at least 1 dose of tafamidis between 2018 and 2021 at 5 amyloidosis centers in the United States were enrolled. Primary outcome was all-cause mortality.

Results

Among 624 patients, mean age was 76.9 ± 8.4 years, 12.5% were female, 17.5% were Black, and 17.5% had variant ATTR-CM. At the time of tafamidis start, 52% had NYHA functional class II, 34% had NYHA functional class III, 40% were in National Amyloidosis Center (NAC) Stage ≥II, 38% were in Columbia Stage ≥II, and the median NT-proBNP level was 1,914 (Q1-Q3: 957-3914) pg/mL. Over a median follow-up of 43.2 months (Q1-Q3: 25.2-52.8 months), 241 patients (38.6%) died. The probability of freedom from death at 65 months was 54.1% (95% CI: 47.4%-60.4%). Similarly, restricting the cohort to patients who received tafamidis within 6 months of their ATTR-CM diagnosis (n = 397, 63.6%) showed similar results, with a survival probability of 49.6% (95% CI: 37.6%-60.5%) at 65 months.

Conclusions

In a contemporary cohort of tafamidis-treated patients with ATTR-CM, 39% of patients died over a median of 43 months. Further work is needed to improve our understanding of ATTR-CM, its natural history, and how to further improve survival and prevent progression of heart failure.

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来源期刊

Jacc: Cardiooncology Multiple-

CiteScore

12.50

自引率

6.30%

发文量

106

期刊介绍： JACC: CardioOncology is a specialized journal that belongs to the esteemed Journal of the American College of Cardiology (JACC) family. Its purpose is to enhance cardiovascular care for cancer patients by publishing high-quality, innovative scientific research and sharing evidence-based knowledge. The journal aims to revolutionize the field of cardio-oncology and actively involve and educate professionals in both cardiovascular and oncology fields. It covers a wide range of topics including pre-clinical, translational, and clinical research, as well as best practices in cardio-oncology. Key areas of focus include understanding disease mechanisms, utilizing in vitro and in vivo models, exploring novel and traditional therapeutics (across Phase I-IV trials), studying epidemiology, employing precision medicine, and investigating primary and secondary prevention. Amyloidosis, cardiovascular risk factors, heart failure, and vascular disease are some examples of the disease states that are of particular interest to the journal. However, it welcomes research on other relevant conditions as well.