Real-World Characteristics of Patients with Wild-Type Transthyretin Amyloid Cardiomyopathy: An Analysis of Electronic Healthcare Records in the United States,American Journal of Cardiovascular Drugs

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Real-World Characteristics of Patients with Wild-Type Transthyretin Amyloid Cardiomyopathy: An Analysis of Electronic Healthcare Records in the United States
American Journal of Cardiovascular Drugs ( IF 2.8 ) Pub Date : 2023-02-13 , DOI: 10.1007/s40256-022-00563-4
Rahul Bhambri ₁ , A Carmine Colavecchia ₂ , Marianna Bruno ₁ , Yong Chen ₃ , Jose Alvir ₁ , Anuja Roy ₁ , Jason Kemner ₃ , Aaron Crowley ₄ , Darrin Benjumea ₄ , Lauren Gilstrap ₅

Affiliation

Background

Tafamidis was approved for the treatment of hereditary and wild-type transthyretin amyloid cardiomyopathy (ATTRwt-CM) in May 2019, based on findings from the Tafamidis in Transthyretin Cardiomyopathy Clinical Trial (ATTR-ACT).

Methods

This retrospective cohort study evaluated the factors associated with tafamidis prescription after diagnosis of ATTRwt-CM in the real world. Between May 2019 and December 2020, 430 patients with 6 months’ database activity were indexed from the de-identified US Optum electronic healthcare records at first diagnosis of ATTRwt-CM or prescription of tafamidis, then followed until last activity or death. Of these, 209 patients were prescribed tafamidis during follow-up, 167 (80%) within 1 month, 98% by 6 months, and 100% by 9 months. Median time from index to tafamidis prescription, calculated using the Kaplan–Meier method, was 5.8 months (95% confidence interval [CI] 2.4–not evaluable).

Results

Factors associated with tafamidis prescription in a multivariable Cox proportional hazards regression (hazard ratio [95% CI]) included age ≥ 65 years (2.1 [1.07–4.05]), male sex (1.6 [1.07–2.28]), having heart failure/cardiomyopathy (2.4 [1.54–3.82]), and having had technetium-99m pyrophosphate myocardial scintigraphy (1.7 [1.28–2.28]).

Conclusions

The clinical characteristics of patients with ATTRwt-CM who were prescribed tafamidis in the real world were broadly comparable with those who took part in ATTR-ACT. Further studies are needed to evaluate hereditary and ATTRwt-CM patient populations in the real world and assess the long-term outcomes associated with disease management pathways.

Clinical Trials Registration

ClinicalTrials.gov identifier: NCT01994889.

中文翻译：

野生型转甲状腺素蛋白淀粉样变性心肌病患者的真实世界特征：美国电子医疗记录分析

背景

根据 Tafamidis 在转甲状腺素蛋白心肌病临床试验 (ATTR-ACT) 中的发现，Tafamidis 于 2019 年 5 月获准用于治疗遗传性和野生型转甲状腺素蛋白淀粉样变性心肌病 (ATTRwt-CM)。

方法

这项回顾性队列研究评估了在现实世界中诊断出 ATTRwt-CM 后与 tafamidis 处方相关的因素。在 2019 年 5 月至 2020 年 12 月期间，430 名具有 6 个月数据库活动的患者在首次诊断为 ATTRwt-CM 或开出 tafamidis 处方时从去识别化的美国 Optum 电子医疗记录中编制索引，然后进行跟踪直至最后一次活动或死亡。其中，209 名患者在随访期间服用了 tafamidis，1 个月内有 167 名（80%），6 个月时为 98%，9 个月时为 100%。使用 Kaplan–Meier 方法计算，从索引到 tafamidis 处方的中位时间为 5.8 个月（95% 置信区间 [CI] 2.4 – 不可评估）。

结果

在多变量 Cox 比例风险回归（风险比 [95% CI]）中与 tafamidis 处方相关的因素包括年龄≥65 岁（2.1 [1.07–4.05]）、男性（1.6 [1.07–2.28]）、心力衰竭/心肌病 (2.4 [1.54–3.82])，并进行过锝 99m 焦磷酸心肌闪烁显像 (1.7 [1.28–2.28])。