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塔非酰胺对老年患者生存的影响:来自欧洲医疗保健淀粉样变性登记处的见解。

Impact of Tafamidis on survival in elderly patients: Insights from the Healthcare European Amyloidosis Registry.

作者信息

Jobbé-Duval Antoine, Kharoubi Mounira, Donal Erwan, Bauer Fabrice, Broussier Amaury, Bisson Arnaud, Bouchot Océane, Charron Phillipe, Costa Jérôme, Courand Pierre-Yves, Dagrenat Charlotte, Delelis François, Eicher Jean-Christophe, Fraix Antoine, Gellen Barnabas, Gueffet Jean-Pierre, Habib Gilbert, Inamo Jocelyn, Jeanneteau Julien, Legallois Damien, Margerit Léa, Mouhat Basile, Piriou Nicolas, Puscas Tania, Réant Patricia, Roubille François, Trésorier Romain, Von Hunolestein Jean-Jacques, Taieb Charles, Zaroui Amira, Lairez Olivier, Damy Thibaud

机构信息

Department of Cardiology, Médipôle Hôpital Mutualiste, 69100 Villeurbanne, France; Department of Cardiology, Hôpital Louis Pradel, Hospices Civil de Lyon, 69500 Bron, France.

Department of Cardiology, Referral Center for Cardiac Amyloidosis, Filiere Cardiogen, GRC Amyloid Research Institute all at APHP CHU Henri Mondor, 94010 Créteil, France; HEART'S Foundation, 94300 Vincennes, France; Clinical Epidemiology and Ageing (CEpiA) at Henri Mondor University Hospital and Institut National de la Santé et de la Recherche Médicale (INSERM) U955, Institut Henri Mondor University Hospital, F-94010 Créteil, France.

出版信息

Int J Cardiol. 2025 Oct 15;437:133522. doi: 10.1016/j.ijcard.2025.133522. Epub 2025 Jun 16.

Abstract

BACKGROUND

Cardiac transthyretin amyloidosis (ATTR-CM) is a life-threatening cardiomyopathy. Tafamidis has been demonstrated to be an effective treatment. Our aim was to analyze clinical characteristics and survival of patients with ATTR-CM aged ≥80 years diagnosed after November 2018, treated with tafamidis 80/61 mg, and compare them with a non-treated group diagnosed before that date.

METHODS

Data from the two groups were extracted from the Healthcare European Amyloidosis Registry (HEAR). Propensity score matching was used to adjust for baseline differences between the groups. Kaplan-Meier survival curves and Cox regression analyses were applied to assess survival outcomes.

RESULTS

Out of 1380 patients, 1194 were treated with tafamidis 80/61 mg. Treated patients were significantly less severe at baseline, with a lower occurrence of NYHA class III-IV compared to the untreated group (24 vs. 46 %, p < 0.001). The median NT-proBNP at baseline was lower in the treated group (2330 vs. 4854 pg/ml, p < 0.001), as was the average level of high-sensitivity troponin T (55 vs. 74 ng/ml, p < 0.001), and the interventricular septal thickness (16 vs. 18 mm, p < 0.001). The 3-year survival rate for treated patients was 57 %, and 40 % for untreated patients. In the treated group, the 3-year survival rate was 68 % for patients aged 80-85 years and 58 % for those over 85 years. Survival rates were confirmed after propensity score analyses.

CONCLUSIONS

This study demonstrates that tafamidis provides significant survival benefits for elderly patients with ATTR-CM, even in those over 85 years old. The findings emphasize the importance of early diagnosis and treatment.

摘要

背景

心脏转甲状腺素蛋白淀粉样变心肌病(ATTR-CM)是一种危及生命的心肌病。已证明塔非酰胺是一种有效的治疗方法。我们的目的是分析2018年11月后诊断的年龄≥80岁、接受80/61mg塔非酰胺治疗的ATTR-CM患者的临床特征和生存率,并将其与该日期之前诊断的未治疗组进行比较。

方法

两组数据均从欧洲医疗淀粉样变注册中心(HEAR)提取。采用倾向评分匹配法来调整两组之间的基线差异。应用Kaplan-Meier生存曲线和Cox回归分析来评估生存结果。

结果

在1380例患者中,1194例接受了80/61mg塔非酰胺治疗。与未治疗组相比,治疗组患者基线时病情明显较轻,纽约心脏协会(NYHA)III-IV级的发生率较低(24%对46%,p<0.001)。治疗组基线时的NT-proBNP中位数较低(2330对4854pg/ml,p<0.001),高敏肌钙蛋白T的平均水平也较低(55对74ng/ml,p<0.001),室间隔厚度也较低(16对18mm,p<0.001)。治疗组患者的3年生存率为57%,未治疗组为40%。在治疗组中,80-85岁患者的3年生存率为68%,85岁以上患者为58%。倾向评分分析后生存率得到证实。

结论

本研究表明,塔非酰胺为老年ATTR-CM患者带来显著的生存益处,即使是85岁以上的患者。这些发现强调了早期诊断和治疗的重要性。

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