背景转甲状腺素蛋白心脏淀粉样变性(ATTR-CM),在6%到15%的射血分数保留的心力衰竭队列中发现,长期以来一直被认为是一种预后不良的罕见疾病。新的治疗方法使其成为心力衰竭的少数可直接治疗的原因之一。这项研究试图确定是否患有ATTR-CM的患者,特别是那些用tafamidis治疗的,与未选择的射血分数保留的心力衰竭队列具有相当的生存率。方法和结果我们比较了ATTR-CM患者的单中心队列(n=114)和TOPCAT(醛固酮拮抗剂治疗保留心功能心力衰竭)试验(n=1761,不包括俄罗斯和格鲁吉亚)中射血分数保留的心力衰竭患者的临床特征和结果。主要结果是全因死亡的复合结果,心力衰竭住院,心肌梗塞,和中风。还对用他法米米治疗的ATTR-CM患者进行了亚组分析。与参加TOPCAT试验的患者相比,ATTR-CM患者的主要复合结局发生率更高(风险比[HR],1.44[95%CI,1.09-1.91];P=0.01),全因死亡率相似(HR,1.43[95%CI,0.99-2.06];P=0.06),但心力衰竭住院率较高(HR,1.62[95%CI,1.15-2.28];P<0.01)。与参加TOPCAT的患者相比,接受tafamidis治疗的ATTR-CM患者的主要复合结局发生率相似(HR,1.30[95%CI,0.86-1.96];P=0.21)和全因死亡(HR,1.10[95%CI,0.57-2.14];P=0.78),但心力衰竭住院率较高(HR,1.96[95%CI,1.27-3.02];P<0.01)。结论与射血分数保留的心力衰竭患者相比,使用tafamidis治疗的ATTR-CM患者的全因死亡率相似。心力衰竭住院率较高。
Background Transthyretin cardiac amyloidosis (ATTR-CM), found in 6% to 15% of cohorts with heart failure with preserved ejection fraction, has long been considered a rare disease with poor prognosis. New treatments have made it one of the few directly treatable causes of heart failure. This
study sought to determine whether patients with ATTR-CM, particularly those treated with tafamidis, have comparable survival to an unselected cohort with heart failure with preserved ejection fraction. Methods and Results We compared the clinical characteristics and outcomes between a single-center cohort of patients with ATTR-CM (n=114) and patients with heart failure with preserved ejection fraction enrolled in the TOPCAT (Treatment of Preserved Cardiac Function Heart Failure With an Aldosterone Antagonist)
trial (n=1761, excluding Russia and Georgia). The primary outcome was a composite of all-cause death, heart failure hospitalization, myocardial infarction, and stroke. Subgroup analysis of patients with ATTR-CM treated with tafamidis was also performed. Patients with ATTR-CM had higher rates of the primary composite outcome compared with patients enrolled in the TOPCAT
trial (hazard ratio [HR], 1.44 [95% CI, 1.09-1.91]; P=0.01), with similar rates of all-cause death (HR, 1.43 [95% CI, 0.99-2.06]; P=0.06) but higher rates of heart failure hospitalizations (HR, 1.62 [95% CI, 1.15-2.28]; P<0.01). Compared with patients enrolled in TOPCAT, patients with ATTR-CM treated with tafamidis had similar rates of the primary composite outcome (HR, 1.30 [95% CI, 0.86-1.96]; P=0.21) and all-cause death (HR, 1.10 [95% CI, 0.57-2.14]; P=0.78) but higher rates of heart failure hospitalizations (HR, 1.96 [95% CI, 1.27-3.02]; P<0.01). Conclusions Patients with ATTR-CM treated with tafamidis have similar rates of all-cause death compared with patients with heart failure with preserved ejection fraction, with higher rates of heart failure hospitalizations.