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.
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。