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Observational Study
. 2025 Apr;12(2):1203-1216.
doi: 10.1002/ehf2.15126. Epub 2024 Nov 6.

Real-world characteristics and treatment of cardiac transthyretin amyloidosis: A multicentre, observational study

Richard J Nies  1 , Svenja Ney  1 , Ingrid Kindermann  2 , Yvonne Bewarder  2 , Angela Zimmer  2 , Fabian Knebel  3   4 , Katrin Hahn  4   5   6 , Sebastian Spethmann  4   6   7 , Peter Luedike  8 , Lars Michel  8 , Tienush Rassaf  8 , Maria Papathanasiou  8   9 , Stefan Störk  10 , Vladimir Cejka  10 , Amin Polzin  11 , Fabian Voss  11 , Malte Kelm  11 , Bernhard Unsöld  12   13 , Christine Meindl  13 , Michael Paulus  13 , Ali Yilmaz  14 , Bishwas Chamling  14   15 , Caroline Morbach  10 , Roman Pfister  1
Affiliations
Observational Study

Real-world characteristics and treatment of cardiac transthyretin amyloidosis: A multicentre, observational study

Richard J Nies et al. ESC Heart Fail. 2025 Apr.

Abstract

Aims: Data on the clinical profiles of patients with transthyretin amyloidosis cardiomyopathy (ATTR-CM) in the post-approval era of tafamidis 61 mg are lacking. Study aims were characterization of contemporary ATTR-CM patients, analysis of potential eligibility for the 'Transthyretin Amyloidosis Cardiomyopathy Clinical Trial' (ATTR-ACT) and identification of factors associated with the decision on tafamidis 61 mg treatment.

Methods and results: This retrospective study analysed ATTR-CM patients seen at eight University Hospitals in the first year after approval of tafamidis 61 mg for ATTR-CM in Germany (April 2020 to March 2021). The cohort comprised 366 patients (median age 79 [74; 82] years, 84% male), with 47% and 45% of the cohort being in National Amyloidosis Centre ATTR stage ≥ II and NYHA class ≥ III, respectively. Sixty-four per cent of patients met key eligibility criteria of the pivotal ATTR-ACT. In recently diagnosed patients (58% with diagnosis ≤6 months), the rate of variant ATTR was significantly lower than in patients diagnosed more than 6 months ago (9.3% vs. 19.7%). Of the 293 patients without prior ATTR specific treatment, tafamidis 61 mg was newly initiated in 77%. Patients with tafamidis 61 mg treatment were significantly younger, were more often eligible for ATTR-ACT, had lower NYHA class and higher serum albumin levels. These variables explained 16% of the variance of treatment decision. Unadjusted survival was higher in patients with than those without treatment (1-year survival 98.6% vs. 87.3%, P < 0.001).

Conclusions: Wild-type ATTR was the primary aetiology amongst contemporary ATTR-CM patients and almost two-thirds of patients were in an advanced disease stage. Clinical profiles of 64% of patients in routine care matched those of the ATTR-ACT. Further effort is needed to detect patients at an earlier disease stage and to validate criteria justifying treatment initiation.

Keywords: Cardiac amyloidosis; Cardiomyopathy; Heart failure; TTR; Tafamidis; Transthyretin.

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Conflict of interest statement

AZ, BU, FK, IK, KH, LM, MP, PL, RP, SSp, SSt, and TR report personal fees from Pfizer. Additionally, KH, RP, and SSp received grants, while RN and VC declare travel support from Pfizer. BU, FK, IK, KH, LM, RP, SSp, SSt, and VC disclose personal fees from Alnylam. Furthermore, VC also reports travel support from Alnylam. Personal fees from AstraZeneca are documented from FK, IK, KH, LM, SSp, and TR. IK, LM, TR, and YB received personal fees from Bayer. Furthermore, IK, LM, and TR declare personal fees from Bristol Myers Squibb. IK, TR, and YB report personal fees from Novartis, and BU, IK and KH received personal fees from Sobi. Personal fees from Daiichi Sankyo are disclosed by IK and TR. IK and YB declare personal fees, and VC reports grants and travel support from Boehringer Ingelheim. Personal fees from Akcea Therapeutics Germany are reported by IK and KH. Additionally, IK documents personal fees from Vifor Pharma Deutschland, Servier Deutschland, AIM, Delab and Diplan. KH declares personal fees from Amicus, GSK, Hormosan, ViiV and the German Society of Neurology. Personal fees from Roche and Berlin Chemie are declared by YB. VC reports grants and travel support from Lili. TR received grants and personal fees from Edwards. FK documents personal fees from BridgeBio, and LM discloses personal fees from IFFM e.V.. SSt reports personal fees from IONIS. AY performed consultant activities for Pfizer, Alnylam, AstraZeneca, GE, BridgeBio, NovoNordisk and Alexion. All of the above‐mentioned conflicts of interest were outside the submitted work.

KH and SSp also received grants and personal fees from Alnylam during the conduct of the study. Additionally, KH discloses grants from Berlin Institute of Health within the study period. AY reports an ongoing research cooperation with Philips and Circle Cardiovascular Imaging at their institution. The other authors have no conflicts of interest to declare.

Figures

Figure 1
Figure 1
Flowchart depicting frequency of criteria defining a potential participation in the ATTR‐ACT trial. *More than one criterion possible.
Figure 2
Figure 2
Distribution of wt‐/v‐ATTR, NYHA class and NAC stage by time since diagnosis. *Only patients with available genotype considered.
Figure 3
Figure 3
Unadjusted Kaplan–Meier survival estimates by new administration of tafamidis 61 mg.
See this image and copyright information in PMC

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