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. 2026 Jan 26.
doi: 10.1007/s00392-026-02848-z. Online ahead of print.

Current treatment decisions in cardiac transthyretin amyloidosis: a multicentre analysis

Daniel Lavall  1 , Katharina Knoll  2   3 , Sebastian Spethmann  4   5   6   7 , Katrin Hahn  6   8   9 , Gina Barzen  4   5   6 , Ephraim B Winzer  10 , Stefanie Jellinghaus  10 , Lisa K Schöner  10 , Monique Tröbs  11 , Dominik Kauffmann  11 , Nora Donhauser  11 , Lars Michel  12   13 , Julia Vogel  12   13 , Tienush Rassaf  12   13 , Maria Papathanasiou  14 , Lara S Schlender  14 , David M Leistner  14   15 , Birgit Aßmus  16 , Bernhard Unsöld  16 , Larissa Bühner  16 , Fabian Aus dem Siepen  17 , Eva Hofmann  17 , Christian Nagel  17 , Ingrid Kindermann  18 , Angela Zimmer  18 , Roman Pfister  19 , Matthieu Schäfer  19 , Natascha Majunke  20 , Irina Müller-Kozarez  20 , Heribert Schunkert  2   3 , Patrick Fuchs  2 , Stéphanie K Schwarting  17   21 , Yuliyan Metodiev  21 , Selen Alieva  21 , Ali Yilmaz  22 , Alexandru Zlibut  22 , Julian Mustroph  23 , Maria Tafelmeier  23 , Thomas Krammer  23 , Stefan Störk  24   25   26 , Aikaterini Papagianni  25   27 , Maximilian J Steinhardt  25   28 , Vladimir Cejka  24   25   26 , Caroline Morbach  24   25   26 , Teresa Trenkwalder  29   30
Affiliations

Current treatment decisions in cardiac transthyretin amyloidosis: a multicentre analysis

Daniel Lavall et al. Clin Res Cardiol. .

Abstract

Background: The efficacy of transthyretin stabilisation in cardiac transthyretin amyloidosis (ATTR-CM) has been demonstrated in a clinical trial setting, but little is known about treatment decision-making in the real world. Particularly, initiating or discontinuing specific therapy is challenging in early and advanced disease. We evaluated current decision pathways for tafamidis in ATTR-CM.

Methods: This multicentre retrospective study included consecutive patients from 15 tertiary centres in Germany in whom ATTR-CM was newly diagnosed between January and June 2024, as well as patients, in whom tafamidis treatment was discontinued during this period.

Results: Out of 516 patients with newly established ATTR-CM included in the present analysis, tafamidis was initiated in 414 (80%). The 99 patients without recommendation for tafamidis were older (p = 0.002), had a higher amyloidosis disease stage (NAC score), worse NYHA class (both p < 0.001), and higher NT-proBNP levels (p = 0.002) compared to those with tafamidis initiation. During the same observation period, tafamidis therapy was discontinued in 28 ATTR-CM patients. Treatment decisions were mainly taken by an interdisciplinary board (73% of centres). The most frequent reasons for not starting or stopping tafamidis were 'frailty' (47%/61%) and 'life expectancy or comorbidity' (38%/43%), respectively.

Conclusions: In this multicentre analysis, treatment with tafamidis was initiated in about 80% of patients with newly diagnosed ATTR-CM. In most centres, treatment decisions were made by an interdisciplinary board, and the reasons for treatment decisions were similar across centres. Due to the lack of consensus criteria, our data may help to standardise decision pathways for ATTR-CM.

Keywords: ATTR; Amyloidosis; Cardiomyopathy; Transthyretin; Treatment decisions.

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Conflict of interest statement

Declarations. Ethics approval: This retrospective, multicentre study was conducted retrospectively from data obtained for clinical purposes. All study centres obtained local ethical or institutional review board approval for their local registries. The study was performed in line with the principles of the Declaration of Helsinki. Conflict of interest: D.L. received speaker and/or consulting fees from Pfizer, Bayer, Boehringer Ingelheim, Bristol Myers Squibb, Alnylam, and AstraZeneca. K.K. report speaker, consulting fees, and/or travel grants from Pfizer, Alnylam, AstraZeneca, Bayer, Boehringer Ingelheim, and Bristol Myers Squibb. K.H. has received funding by Alnylam Pharmaceuticals Inc., Pfizer Pharmaceuticals, and Berlin Institute of Health (BIH), funding unrelated to this article. K. H. received financial reimbursement for consulting, advisory board activities, speaker fees and/or contributions to congresses, and travel support to attend scientific meetings by Akcea Therapeuticals Inc., Alnylam Pharmaceuticals Inc., Amicus, AstraZeneca, GSK, Hormosan, Takeda Pharmaceutical Inc., Pfizer Pharmaceuticals Inc., and Swedish Orphan Biovitrum Inc., and ViiV Healthcare GmbH. K. H. received financial reimbursement for consulting, advisory board activities, speaker fees and/or contributions to congresses, and travel support to attend scientific meetings by Akcea Therapeuticals Inc., Alnylam Pharmaceuticals Inc., Amicus, AstraZeneca, GSK, Hormosan, Takeda Pharmaceutical Inc., Pfizer Pharmaceuticals Inc., and Swedish Orphan Biovitrum Inc., and ViiV Healthcare GmbH. S.S. received consulting fees, advisory board fees, lecture fees, research funding, and travel grants for participation in scientific meetings from Pfizer Pharma GmbH, Alnylam Netherlands BV, Bayer Vital GmbH, Boehringer Ingelheim Pharma GmbH, Edwards Lifesciences, and AstraZeneca GmbH, which were not related to this project. G.B. has received funding by Alnylam Pharmaceuticals Inc. unrelated to this article. G.B. received lecture fees, research funding, and travel reimbursement by Alnylam Pharmaceuticals, Intellia Therapeutics, and AstraZeneca. E.B.W. reported grants from Boehringer Ingelheim and personal fees from Amarin, Amgen, AstraZeneca, Bayer, Boehringer-Ingelheim, Bristol Myers Squibb, CVRx, Daiichi Sankyo, Novartis, Novo Nordisk, and Pfizer outside the submitted work. L.K.S. reported grants from Pfizer and Alnalym outside the submitted work. S.J. reported personal fees from Amgen, Daiichi Sankyo, Abbott, Medtronic, Abiomed, and Sanofi Aventis outside the submitted work. M.T. received speaker and/or consulting fees from Pfizer, Abbott, Bristol Myers Squibb, Alnylam, and AstraZeneca. L.M.: personal fees outside the submitted work from Bayer, Bristol Myers Squibb, Alnylam, AstraZeneca, IFFM e. V. and from Bund der Niedergelassenen Kardiologen (BNK). J.V.: personal fees outside the submitted work from Eli Lilly, Bayer, Alnylam, and Pfizer. Research funding: UMEA Clinician Scientist Scholarship from the Faculty of Medicine, University of Duisburg-Essen and Bayer for an investigator/institution sponsored collaborative study outside the submitted work. T.R. has received honoraria, lecture fees, and grant support from Edwards Lifesciences, AstraZeneca, Bayer, Novartis, Berlin Chemie, Daiicho-Sankyo, Boehringer Ingelheim, Novo Nordisk, Cardiac Dimensions, and Pfizer, all unrelated to this work. He is co-founder of Bimyo GmbH, a company that develops cardioprotective peptides and a co-founder of myCor GmbH, a company focusing on the development of ECG-algorithms to detect patients with amyloidosis. F.S. reports speaker fees and/or consulting fees from Pfizer, Alnylam, Bayer, Neurimmune, AstraZeneca, and NovoNordisc. I.K. reports consulting fees, advisory, and speakers honoraria as well as travel grants from Pharmacosmos GmbH, Pfizer Pharma GmbH, Bristol- Myers-Squibb, Daiichi Sankyo, Akcea Therapeutics Germany GmbH, Vifor Pharma Deutschland, Novartis Pharma GmbH, Servier Deutschland GmbH, AstraZeneca GmbH, Bayer Vital GmbH, Daiichi Sankyo, Alnylam, SOBI, Diaplan, Boehringer- Ingelheim Pharma GmbH &Co.KG, Orionpharma, Scirac. I.K. serves as principal investigator in trials sponsored by IONIS Pharmaceuticals, Alexion Pharmaceuticals, and AstraZeneca. B.U. reports speaker and consulting fees from Pfizer, Alnylam, Sobi, AstraZeneca und Bayer. R.P. reports speaker fees and travel grants from Pfizer, Alnylam, AstraZeneca, Bayer, and NovoNordisk. Further, he received a research grant by Pfizer. S.K.S. reports speaker and consulting fees from Pfizer, Alnylam, AstraZeneca, and Bayer and serves as principal investigator in trials sponsored by Bayer, NovoNordisk, Intellia, AstraZeneca, and Alexion. A.Y. reports speakers and consulting fees from Alnylam, Bayer, Philips, GE, AstraZeneca, BridgeBio, NovoNordisk, Alexion und Pfizer as well as research cooperations with Philips und Circle. J.M. reports speaker fees for talks related to cardiac amyloidosis and/or hypertrophic cardiomyopathy by Bayer, Bristol Myers Squibb, and AstraZeneca. A.P. report speaker, consulting fees and/or travel grants from Pfizer, Alnylam, AstraZeneca, and SoBi outside the submitted work. V.C. reports sponsoring of attendance and travel to scientific congresses from Lily, Boheringer Ingelheim, and Alnylam. He has received lecturing fees from Pfizer. C. M. reports research cooperation with the University of Würzburg and Tomtec Imaging Systems funded by a research grant from the Bavarian Ministry of Economic Affairs, Regional Development and Energy, German Germany (MED-1811–0011, LSM-2104–0002, and LSM-2403–0005); C.M. further received advisory and speakers honoraria as well as travel grants from Tomtec, Edwards, Alnylam, Pfizer, Boehringer Ingelheim, Eli Lilly, SOBI, AstraZeneca, NovoNordisk, Alexion, Janssen, Bayer, Intellia, and EBR Systems; she serves as principal investigator in trials sponsored by Alnylam, Bayer, NovoNordisk, Intellia, and AstraZeneca. T.T. reports speaker and travel grants: Pfizer, Alnylam, Zoll, Novartis, Astra Zeneca, Alexion, Bayer, Bristol Myers Squibb, Zoll, and Boehringer Ingelheim. B.H. received speaker fees from Pfizer Pharmaceuticals Inc. and AstraZeneca, as well as research grant from Pfizer Pharmaceuticals Inc. unrelated to this project. D.K., N.D., M.P., L.S.S., D.M.L., B.A., L.B., E.H., C.N., A.Z., M.S., N.M., I.M., Y.M., S.A., A.Z., M.T., T.K., and M.J.S. report no conflicts of interest relevant to the submitted work.

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