Department of Cardiology, Aarhus University Hospital, Aarhus N, Denmark.
Ann Med. 2024 Dec;56(1):2398735. doi: 10.1080/07853890.2024.2398735. Epub 2024 Sep 9.
Increased diagnostic awareness and specific disease treatments have changed the landscape of transthyretin cardiac amyloidosis (ATTR). Patients with wild-type ATTR (ATTRwt) are increasingly being diagnosed, potentially changing the clinical profile and prognosis compared with existing retrospective data. We aimed to study the clinical characteristics, distribution of red flags and prognosis of contemporary ATTRwt patients.
From January 1 2017, to December 31 2022, 213 consecutive patients were diagnosed with ATTRwt and prospectively followed up. Data on clinical characteristics, biomarkers, echocardiography findings, hospitalization due to worsening heart failure (WHF) and all-cause mortality were collected.
A 37% increase in newly diagnosed patients from 2017-2019 ( = 90) vs. 2020-2022 ( = 123) was observed. The majority of patients presented with NAC disease stage I in the latter period (49% in 2017-2019 vs. 58% in 2020-2022, = .16). Red flags were primarily cardiac-related, including elevated NT-proBNP, impaired left ventricular longitudinal systolic strain with an apical sparing pattern, heart failure with increased left ventricular wall thickness and elevated troponins. NAC disease stage I as well as low NT-proBNP levels (<1000 ng/L) were significantly associated with better survival (both < .001). When compared with NAC disease stage II + III combined, patients with NAC disease stage I had a significantly lower risk of WHF hospitalization or death (log rank test: = .0001). Independent predictors of the combined endpoint WHF hospitalization or death were NT-proBNP (HR 1.03 [95% CI 1.00-1.07], < .049) and prior implantation of permanent pacemaker (HR 2.01 [1.30-3.11], = .002).
Increased diagnostic awareness resulted in a 37% increase in newly diagnosed patients in 2020-2022 vs. 2017-2019. As expected all-cause mortality but also the morbidity in terms of risk of hospitalization with WHF were significantly lower in patients with NAC disease stage I, as well as in those with low NT-proBNP levels <1000 ng/L. These findings underline the importance of continuous attention to diagnostic awareness, as early diagnosis is critical for initiating both general and specific ATTR treatment, thus improving prognosis.
诊断意识的提高和特定疾病的治疗改变了转甲状腺素蛋白心脏淀粉样变（ATTR）的格局。越来越多的野生型 ATTR（ATTRwt）患者被诊断出来，与现有的回顾性数据相比，这可能改变了临床特征和预后。我们旨在研究当代 ATTRwt 患者的临床特征、红色标志分布和预后。
从 2017 年 1 月 1 日至 2022 年 12 月 31 日，连续诊断了 213 例 ATTRwt 患者，并对其进行了前瞻性随访。收集了临床特征、生物标志物、超声心动图结果、因心力衰竭恶化（WHF）住院和全因死亡率的数据。
2017-2019 年（ = 90）新诊断患者增加了 37%，而 2020-2022 年（ = 123）增加了 37%。在后一时期，大多数患者的 NAC 疾病分期为 I 期（2017-2019 年为 49%，2020-2022 年为 58%， = .16）。红色标志主要与心脏有关，包括升高的 NT-proBNP、心尖保留模式的左心室纵向收缩应变受损、左心室壁增厚和升高的肌钙蛋白的心力衰竭。NAC 疾病 I 期和低 NT-proBNP 水平（<1000ng/L）与更好的生存显著相关（均 < .001）。与 NAC 疾病 II+III 期相比，NAC 疾病 I 期患者因 WHF 住院或死亡的风险显著降低（对数秩检验： = .0001）。WHF 住院或死亡的综合终点的独立预测因素是 NT-proBNP（HR 1.03[95%CI 1.00-1.07]， < .049）和先前植入永久性起搏器（HR 2.01[1.30-3.11]， = .002）。
诊断意识的提高导致 2020-2022 年新诊断患者比 2017-2019 年增加了 37%。不出所料，所有原因的死亡率以及因 WHF 住院的发病率在 NAC 疾病 I 期和 NT-proBNP 水平<1000ng/L 的患者中显著降低。这些发现强调了持续关注诊断意识的重要性，因为早期诊断对于启动一般和特定的 ATTR 治疗至关重要，从而改善预后。
