National Amyloidosis Centre, Division of Medicine, University College London, Royal Free Campus, London, UK.
Heart Failure Unit, Cardiology University Department, IRCCS Policlinico San Donato, Milan, Italy.
Eur J Heart Fail. 2022 Sep;24(9):1700-1712. doi: 10.1002/ejhf.2606. Epub 2022 Jul 27.
Transthyretin amyloid cardiomyopathy (ATTR-CM) is an increasingly diagnosed disease. Echocardiography is widely utilized, but studies to confirm the value of echocardiography for tracking changes over time are not available. We sought to describe (i) changes in multiple echocardiographic parameters; (ii) differences in rate of progression of three predominant genotypes; and (iii) the ability of changes in echocardiographic parameters to predict prognosis.
We prospectively studied 877 ATTR-CM patients attending our centre between 2000 and 2020. Serial echocardiography findings at baseline, 12 months and 24 months were compared with survival. Overall, 565 patients had wild-type ATTR-CM and 312 hereditary ATTR-CM (201 with V122I; 90 with T60A). There was progressive worsening of structural and functional parameters over time, patients with V122I ATTR-CM showing more rapid worsening of left and right ventricular structural and functional parameters compared to both wild-type and T60A ATTR-CM. Among a wide range of echocardiographic analyses, including deformation-based parameters, only worsening in the degree of mitral (MR) and tricuspid regurgitation (TR) at 12- and 24-month assessments was associated with worse prognosis (change at 12 months: MR, hazard ratio 1.43 [95% confidence interval 1.14-1.80], p = 0.002; TR, hazard ratio 1.38 [95% confidence interval 1.10-1.75], p = 0.006). Worsening in MR remained independently associated with poor prognosis after adjusting for known predictors.
In ATTR-CM, echocardiographic parameters progressively worsen over time. Patients with V122I ATTR-CM demonstrate the most rapid deterioration. Worsening of MR and TR were the only parameters associated with mortality, MR remaining independent after adjusting for known predictors.
转甲状腺素蛋白淀粉样心肌病（ATTR-CM）是一种日益被诊断出的疾病。超声心动图被广泛应用，但尚无研究证实其用于随时间追踪变化的价值。我们旨在描述：（i）多个超声心动图参数的变化；（ii）三种主要基因型进展速度的差异；以及（iii）超声心动图参数变化预测预后的能力。
我们前瞻性地研究了 2000 年至 2020 年期间在我们中心就诊的 877 名ATTR-CM 患者。将基线、12 个月和 24 个月的连续超声心动图结果与生存情况进行比较。总体而言，565 名患者为野生型ATTR-CM，312 名患者为遗传性ATTR-CM（201 名为 V122I；90 名为 T60A）。随着时间的推移，结构和功能参数逐渐恶化，与野生型和 T60AATTR-CM 相比，V122IATTR-CM 患者的左、右心室结构和功能参数恶化速度更快。在广泛的超声心动图分析中，包括基于变形的参数，只有在 12 个月和 24 个月评估时二尖瓣（MR）和三尖瓣反流（TR）的恶化程度与预后更差相关（12 个月时的变化：MR，风险比 1.43 [95%置信区间 1.14-1.80]，p=0.002；TR，风险比 1.38 [95%置信区间 1.10-1.75]，p=0.006）。在调整已知预测因素后，MR 的恶化仍然与不良预后独立相关。
在ATTR-CM 中，超声心动图参数随时间逐渐恶化。V122IATTR-CM 患者表现出最快的恶化。MR 和 TR 的恶化是唯一与死亡率相关的参数，在调整已知预测因素后，MR 仍然独立。
