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转甲状腺素蛋白淀粉样心肌病风险评估在心力衰竭患者队列中的应用。

Transthyretin Amyloid Cardiomyopathy Risk Evaluation in a Cohort of Patients With Heart Failure.

机构信息

Pharmacy Department, Kaiser Permanente Colorado, Aurora, CO, USA.

Cardiology Department, Colorado Permanente Medical Group, Aurora, CO, USA.

出版信息

Perm J. 2023 Jun 15;27(2):51-60. doi: 10.7812/TPP/22.135. Epub 2023 Mar 27.

DOI:10.7812/TPP/22.135
PMID:36970848
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC10266840/
Abstract

Introduction Transthyretin amyloid cardiomyopathy (ATTR-CM) is a progressive, infiltrative form of heart failure (HF). Nevertheless, ATTR-CM is a largely underrecognized and misdiagnosed condition. This study's objective was to develop an efficient model to assess the chance of ATTR-CM in patients with HF. Methods This was an observational study of patients with HF who had a confirmed diagnosis of ATTR-CM and those with HF but without known ATTR-CM between January 1, 2019, and July 1, 2021. Patient characteristics were extracted from administrative and claims electronic databases and compared between the groups. A propensity score for having ATTR-CM was modeled. Samples of 50 control patients with the highest and lowest propensity scores were adjudicated to assess whether further workup to evaluate for ATTR-CM was warranted for each patient. The sensitivity and specificity of the model were calculated. Results Thirty-one patients with confirmed ATTR-CM and 7620 patients without known ATTR-CM were included in the study. Patients with ATTR-CM were more likely to be Black and to have atrial flutter/fibrillation, cardiomegaly, HF with preserved ejection fraction, pericardial effusion, carpal tunnel syndrome, joint disorders, and lumbar spinal stenosis and to use a diuretic (all p < 0.05). A propensity model with 16 inputs was developed (c-statistic = 0.875). The model's sensitivity and specificity were 71.9% and 95.2%, respectively. Conclusion The propensity model developed in this study provided an efficient means for identifying patients with HF who are more likely to have ATTR-CM and may warrant further workup.

摘要

简介

转甲状腺素蛋白淀粉样心肌病(ATTR-CM)是一种进行性、浸润性心力衰竭(HF)形式。然而,ATTR-CM 是一种很大程度上未被认识和误诊的疾病。本研究的目的是开发一种有效的模型来评估 HF 患者发生 ATTR-CM 的可能性。

方法

这是一项观察性研究,纳入了 2019 年 1 月 1 日至 2021 年 7 月 1 日期间确诊为 ATTR-CM 的 HF 患者和 HF 但无已知 ATTR-CM 的患者。从行政和索赔电子数据库中提取患者特征,并在两组之间进行比较。对是否患有 ATTR-CM 进行倾向评分建模。对具有最高和最低倾向评分的 50 名对照患者进行样本裁决,以评估是否需要对每位患者进行进一步检查以评估是否存在 ATTR-CM。计算模型的敏感性和特异性。

结果

研究纳入了 31 例确诊为 ATTR-CM 的患者和 7620 例无已知 ATTR-CM 的患者。ATTR-CM 患者更可能为黑人,更可能患有心房颤动/扑动、心脏扩大、射血分数保留的心力衰竭、心包积液、腕管综合征、关节疾病和腰椎椎管狭窄,并且更可能使用利尿剂(均 P<0.05)。开发了一个具有 16 个输入的倾向模型(c 统计量=0.875)。该模型的敏感性和特异性分别为 71.9%和 95.2%。

结论

本研究中开发的倾向模型为识别更有可能患有 ATTR-CM 的 HF 患者提供了一种有效的方法,可能需要进一步检查。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/23fb/10266840/d4a7e82a3dad/tpp_22.135-g001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/23fb/10266840/d4a7e82a3dad/tpp_22.135-g001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/23fb/10266840/d4a7e82a3dad/tpp_22.135-g001.jpg

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本文引用的文献

1
A Simple Score to Identify Increased Risk of Transthyretin Amyloid Cardiomyopathy in Heart Failure With Preserved Ejection Fraction.一种简单评分可识别射血分数保留的心力衰竭患者转甲状腺素蛋白淀粉样心肌病的风险增加。
JAMA Cardiol. 2022 Oct 1;7(10):1036-1044. doi: 10.1001/jamacardio.2022.1781.
2
Healthcare resource use of patients with transthyretin amyloid cardiomyopathy.转甲状腺素蛋白淀粉样变性心肌病患者的医疗资源利用情况。
ESC Heart Fail. 2022 Jun;9(3):1636-1642. doi: 10.1002/ehf2.13913. Epub 2022 Apr 1.
3
ATTR Amyloidosis: Current and Emerging Management Strategies: State-of-the-Art Review.
转甲状腺素蛋白淀粉样变性:当前及新出现的管理策略:最新综述
JACC CardioOncol. 2021 Oct 19;3(4):488-505. doi: 10.1016/j.jaccao.2021.06.006. eCollection 2021 Oct.
4
A machine learning model for identifying patients at risk for wild-type transthyretin amyloid cardiomyopathy.一种用于识别野生型转甲状腺素蛋白淀粉样心肌病风险患者的机器学习模型。
Nat Commun. 2021 May 11;12(1):2725. doi: 10.1038/s41467-021-22876-9.
5
Impact of Delayed Diagnosis and Misdiagnosis for Patients with Transthyretin Amyloid Cardiomyopathy (ATTR-CM): A Targeted Literature Review.转甲状腺素蛋白淀粉样变心肌病(ATTR-CM)患者延迟诊断和误诊的影响:一项针对性文献综述
Cardiol Ther. 2021 Jun;10(1):141-159. doi: 10.1007/s40119-021-00219-5. Epub 2021 Apr 20.
6
Diuretic Therapy for Patients With Heart Failure: JACC State-of-the-Art Review.心力衰竭患者的利尿剂治疗:JACC 最新综述。
J Am Coll Cardiol. 2020 Mar 17;75(10):1178-1195. doi: 10.1016/j.jacc.2019.12.059.
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ESC Heart Fail. 2019 Dec;6(6):1128-1139. doi: 10.1002/ehf2.12518. Epub 2019 Sep 25.
8
Transthyretin Amyloid Cardiomyopathy: JACC State-of-the-Art Review.转甲状腺素蛋白淀粉样心肌病:美国心脏病学会最新临床综述
J Am Coll Cardiol. 2019 Jun 11;73(22):2872-2891. doi: 10.1016/j.jacc.2019.04.003.
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