未经证实:转甲状腺素蛋白淀粉样心肌病(ATTR-CM)越来越被认为是心力衰竭(HF)的可治疗原因。诊断和治疗的进展增加了早期诊断的患者数量,但缺乏无HF症状患者的预后数据.此外,目前尚不清楚无症状患者是否从早期启动转甲状腺素蛋白(TTR)稳定剂中获益.
未经证实:本研究的目的是描述无HF症状患者ATTR-CM的自然史和预后。
UNASSIGNED:回顾性收集6个国际淀粉样变性中心无HF症状的ATTR-CM患者的临床特征和结局。
未经批准:共有118名患者(78.8%为男性,中位年龄66岁[IQR:53.8-75岁],68[57.6%]有变异型甲状腺素运载蛋白淀粉样变性,平均左心室射血分数60.5%±9.9%,左心室平均壁厚15.4±3.1mm,包括在基线或随访期间用TTR稳定剂治疗的53[45%]。在3.7年的中位随访期间(IQR:1-6年),38名患者出现HF症状(23名纽约心脏协会功能II级和14名功能III或IV级),32死了,和2需要心脏移植。此外,20名患者接受了起搏器,13开发的AF,1人中风了。总生存率为96.5%(95%CI:91%-99%),90.4%(95%CI:82%-95%),在1、3和5年时为82%(95%CI:71%-89%),分别。TTR稳定剂治疗与生存率改善相关(HR:0.31;95%CI:0.12-0.82;P=0.019),在调整性别后仍然显着。年龄,ATTR-CM型,和估计的肾小球滤过率(HR:0.18;95%CI:0.06-0.55;P=0.002)。
未经评估:经过3.7年的中位随访期,3例无症状ATTR-CM患者中有1例出现HF症状,几乎同样多的人死亡或需要心脏移植。用TTR稳定剂治疗与改善预后相关。
UNASSIGNED: Transthyretin amyloid cardiomyopathy (ATTR-CM) is increasingly recognized as a treatable cause of heart failure (HF). Advances in diagnosis and therapy have increased the number of patients diagnosed at early stages, but prognostic data on patients without HF symptoms are lacking. Moreover, it is unknown whether asymptomatic patients benefit from early initiation of transthyretin (TTR) stabilizers.
UNASSIGNED: The aim of this study was to describe the natural history and prognosis of ATTR-CM in patients without HF symptoms.
UNASSIGNED: Clinical characteristics and outcomes of patients with ATTR-CM without HF symptoms were retrospectively collected at 6 international amyloidosis centers.
UNASSIGNED: A total of 118 patients (78.8% men, median age 66 years [IQR: 53.8-75 years], 68 [57.6%] with variant transthyretin amyloidosis, mean left ventricular ejection fraction 60.5% ± 9.9%, mean left ventricular wall thickness 15.4 ± 3.1 mm, and 53 [45%] treated with TTR stabilizers at baseline or during follow-up) were included. During a median follow-up period of 3.7 years (IQR: 1-6 years), 38 patients developed HF symptoms (23 New York Heart Association functional class II and 14 functional class III or IV), 32 died, and 2 required cardiac transplantation. Additionally, 20 patients received pacemakers, 13 developed AF, and 1 had a stroke. Overall survival was 96.5% (95% CI: 91%-99%), 90.4% (95% CI: 82%-95%), and 82% (95% CI: 71%-89%) at 1, 3, and 5 years, respectively. Treatment with TTR stabilizers was associated with improved survival (HR: 0.31; 95% CI: 0.12-0.82; P = 0.019) and remained significant after adjusting for sex, age, ATTR-CM type, and estimated glomerular filtration rate (HR: 0.18; 95% CI: 0.06-0.55; P = 0.002).
UNASSIGNED: After a median follow-up period of 3.7 years, 1 in 3 patients with asymptomatic ATTR-CM developed HF symptoms, and nearly as many died or required cardiac transplantation. Treatment with TTR stabilizers was associated with improved prognosis.