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Abstract：
BACKGROUND: Results from ATTR-ACT (Safety and Efficacy of Tafamidis in Patients With Transthyretin Cardiomyopathy) indicate that tafamidis prolongs survival and reduces cardiovascular hospitalizations in cardiac transthyretin amyloidosis (ATTR-CA). However, real-world data supporting these findings are scarce. Thus, we sought to characterize the clinical outcome of patients with ATTR-CA treated with tafamidis in a real-world setting and assess the prognostic role of the New York Heart Association (NYHA) classification.
RESULTS: We conducted a retrospective observational study, enrolling a consecutive sample of patients with ATTR-CA (wild-type or variant) treated with tafamidis. Clinical outcome was tracked through follow-up visits or phone calls. Primary outcomes were death and major adverse cardiac events (MACE), a composite end point of death and hospitalizations for acute cardiac decompensation, myocardial infarction, severe arrythmias, or stroke. Kaplan-Meier analysis estimated overall and MACE-free survival including NYHA subgroups (NYHA I/II versus NYHA III). One hundred sixty-seven patients with ATTR-CA (94.6% wild-type) were enrolled and followed for a median of 539 [323-869] days. Median overall survival was not reached. Estimated 1-year, 2-year, and 5-year overall survival among the whole cohort was 93.5%, 85.9%, and 70.2%, respectively. Overall survival was higher in the NYHA I/II subgroup (P=0.002). Median MACE-free survival time was 1082 (95% CI, 962-1202) days. MACE-free survival was higher in the NYHA I/II subgroup (P<0.001). With respective hazard ratios of 5.85 (95% CI, 1.48-23.18; P=0.012) and 3.95 (95% CI, 1.99-7.84; P<0.001), NYHA III was an independent predictor of death and MACE.
CONCLUSIONS: Treatment of ATTR-CA with tafamidis led to substantial improvements of clinical outcome. NYHA classification at treatment initiation is a reliable tool to provide patients with individualized prognostic information.
RESULTS: We conducted a retrospective observational study, enrolling a consecutive sample of patients with ATTR-CA (wild-type or variant) treated with tafamidis. Clinical outcome was tracked through follow-up visits or phone calls. Primary outcomes were death and major adverse cardiac events (MACE), a composite end point of death and hospitalizations for acute cardiac decompensation, myocardial infarction, severe arrythmias, or stroke. Kaplan-Meier analysis estimated overall and MACE-free survival including NYHA subgroups (NYHA I/II versus NYHA III). One hundred sixty-seven patients with ATTR-CA (94.6% wild-type) were enrolled and followed for a median of 539 [323-869] days. Median overall survival was not reached. Estimated 1-year, 2-year, and 5-year overall survival among the whole cohort was 93.5%, 85.9%, and 70.2%, respectively. Overall survival was higher in the NYHA I/II subgroup (P=0.002). Median MACE-free survival time was 1082 (95% CI, 962-1202) days. MACE-free survival was higher in the NYHA I/II subgroup (P<0.001). With respective hazard ratios of 5.85 (95% CI, 1.48-23.18; P=0.012) and 3.95 (95% CI, 1.99-7.84; P<0.001), NYHA III was an independent predictor of death and MACE.
CONCLUSIONS: Treatment of ATTR-CA with tafamidis led to substantial improvements of clinical outcome. NYHA classification at treatment initiation is a reliable tool to provide patients with individualized prognostic information.
摘要：
背景：来自ATTR-ACT（Tafamidis在转甲状腺素蛋白心肌病患者中的安全性和有效性）的结果表明，tafamidis可延长心脏转甲状腺素蛋白淀粉样变性（ATTR-CA）的生存期并减少心血管住院。然而,支持这些发现的真实世界数据很少。因此，我们试图在真实世界环境中表征使用tafamidis治疗的ATTR-CA患者的临床结局,并评估纽约心脏协会(NYHA)分类的预后作用.
结果：我们进行了一项回顾性观察研究，对接受tafamidis治疗的ATTR-CA（野生型或变体）患者进行连续采样。通过随访或电话跟踪临床结果。主要结果是死亡和主要不良心脏事件（MACE），急性心脏代偿失调的死亡和住院的复合终点,心肌梗塞,严重的心律失常,或中风。Kaplan-Meier分析估计总体生存率和无MACE生存率，包括NYHA亚组（NYHAI/II与NYHAIII）。纳入167名ATTR-CA患者（94.6%野生型），随访中位数为539[323-869]天。未达到中位总生存期。估计1年，2年,整个队列的5年总生存率为93.5%，85.9%,70.2%,分别。NYHAI/II亚组的总生存率较高(P=0.002)。中位无MACE生存时间为1082(95%CI,962-1202)天。NYHAI/II亚组无MACE生存率较高(P<0.001)。风险比分别为5.85（95％CI，1.48-23.18；P=0.012）和3.95（95％CI，1.99-7.84；P<0.001），NYHAIII是死亡和MACE的独立预测因子。
结论：用tafamidis治疗ATTR-CA可显著改善临床结果。治疗开始时的NYHA分类是为患者提供个性化预后信息的可靠工具。
结果：我们进行了一项回顾性观察研究，对接受tafamidis治疗的ATTR-CA（野生型或变体）患者进行连续采样。通过随访或电话跟踪临床结果。主要结果是死亡和主要不良心脏事件（MACE），急性心脏代偿失调的死亡和住院的复合终点,心肌梗塞,严重的心律失常,或中风。Kaplan-Meier分析估计总体生存率和无MACE生存率，包括NYHA亚组（NYHAI/II与NYHAIII）。纳入167名ATTR-CA患者（94.6%野生型），随访中位数为539[323-869]天。未达到中位总生存期。估计1年，2年,整个队列的5年总生存率为93.5%，85.9%,70.2%,分别。NYHAI/II亚组的总生存率较高(P=0.002)。中位无MACE生存时间为1082(95%CI,962-1202)天。NYHAI/II亚组无MACE生存率较高(P<0.001)。风险比分别为5.85（95％CI，1.48-23.18；P=0.012）和3.95（95％CI，1.99-7.84；P<0.001），NYHAIII是死亡和MACE的独立预测因子。
结论：用tafamidis治疗ATTR-CA可显著改善临床结果。治疗开始时的NYHA分类是为患者提供个性化预后信息的可靠工具。
