Duca Franz, Poledniczek Michael, Kronberger Christina, Binder Christina, Rettl René, Camuz-Ligios Luciana, Agis Hermine, Koschutnik Matthias, Donà Carolina, Badr-Eslam Roza, Beitzke Dietrich, Loewe Christian, Nitsche Christian, Hengstenberg Christian, Kastner Johannes, Bergler-Klein Jutta, Kammerlander Andreas Anselm
Division of Cardiology, Department of Internal Medicine II, Medical University of Vienna, Vienna, Austria.
Division of Hematology, Department of Internal Medicine I, Medical University of Vienna, Vienna, Austria.
Eur Radiol. 2025 Jul 16. doi: 10.1007/s00330-025-11792-x.
Cardiac transthyretin amyloidosis (ATTR CA) has been increasingly recognized as an important heart failure (HF) entity, and cardiac magnetic resonance (CMR) imaging is a mainstay in the clinical evaluation of this disease. However, studies evaluating the prognostic values of longitudinal data in ATTR CA patients with disease-modifying therapies are lacking. We aimed to assess the prognostic significance of serial quantification of extracellular volume (ECV) in ATTR CA patients treated with tafamidis.
The present study included ATTR CA patients who received ≥ 3 months of tafamidis treatment and underwent baseline and CMR, including ECV quantification. The primary endpoint was a composite of all-cause mortality, cardiac transplantation, or hospitalization due to HF.
Between June 2016 and June 2020, 54 patients were included in the present analysis and were representative of a typical ATTR CA cohort (median age: 76.7 years, male participants: 79.6%). The median time on tafamidis before follow-up CMR was 6.0 months (interquartile range (IQR): 6.0-8.3). Participants depicted typical structural changes for ATTR CA patients with markedly elevated ECV (51.4% (IQR: 41.3-57.6), normal range: 20-32%) and myocardial ventricular hypertrophy (intraventricular septum: 19 mm (16.0-22.0), normal range: 5-12 mm). Change of ECV was the only parameter among clinical, laboratory, and CMR parameters that was independently associated with the composite endpoint (HR: 1.077, 95% CI: 1.013-1.145, p = 0.017).
Change of ECV was the only predictor of adverse outcome among clinical, laboratory, and imaging parameters in our cohort of tafamidis-treated ATTR CA patients.
Question What is the prognostic significance of serial ECV quantification in transthyretin amyloidosis patients on tafamidis treatment? Findings Change of ECV is a strong predictor of a combined endpoint consisting of all-cause mortality, cardiac transplantation, or hospitalization due to HF. Clinical relevance Patients with increasing ECV might potentially benefit from a change in amyloid-specific treatment to a different agent.
心脏转甲状腺素蛋白淀粉样变（ATTR CA）已越来越被认为是一种重要的心力衰竭（HF）类型，心脏磁共振（CMR）成像在该疾病的临床评估中是主要手段。然而，缺乏评估疾病修饰治疗的ATTR CA患者纵向数据预后价值的研究。我们旨在评估在接受塔法米迪斯治疗的ATTR CA患者中，细胞外容积（ECV）连续定量的预后意义。
本研究纳入了接受塔法米迪斯治疗≥3个月并接受基线和CMR检查（包括ECV定量）的ATTR CA患者。主要终点是全因死亡率、心脏移植或因HF住院的复合终点。
在2016年6月至2020年6月期间，54例患者纳入本分析，代表了典型的ATTR CA队列（中位年龄：76.7岁，男性参与者：79.6%）。随访CMR前接受塔法米迪斯治疗的中位时间为6.0个月（四分位间距（IQR）：6.0 - 8.3）。参与者表现出ATTR CA患者典型的结构变化，ECV显著升高（51.4%（IQR：41.3 - 57.6），正常范围：20 - 32%）以及心肌心室肥厚（室间隔：19 mm（16.0 - 22.0），正常范围：5 - 12 mm）。ECV变化是临床、实验室和CMR参数中唯一与复合终点独立相关的参数（风险比：1.077，95%置信区间：1.013 - 1.145，p = 0.017）。
在我们接受塔法米迪斯治疗的ATTR CA患者队列中，ECV变化是临床、实验室和成像参数中不良结局的唯一预测因素。
问题 在接受塔法米迪斯治疗的转甲状腺素蛋白淀粉样变患者中，ECV连续定量的预后意义是什么？发现 ECV变化是全因死亡率、心脏移植或因HF住院组成的复合终点的有力预测因素。临床意义 ECV升高的患者可能会从将淀粉样特异性治疗更换为不同药物中获益。
