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.
PDF(Pubmed)