Real‐world characteristics and treatment of cardiac transthyretin amyloidosis: A multicentre, observational study
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 Amyloid...
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Wiley
2025-04-01
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| Series: | ESC Heart Failure |
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| Online Access: | https://doi.org/10.1002/ehf2.15126 |
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| author | Richard J. Nies Svenja Ney Ingrid Kindermann Yvonne Bewarder Angela Zimmer Fabian Knebel Katrin Hahn Sebastian Spethmann Peter Luedike Lars Michel Tienush Rassaf Maria Papathanasiou Stefan Störk Vladimir Cejka Amin Polzin Fabian Voss Malte Kelm Bernhard Unsöld Christine Meindl Michael Paulus Ali Yilmaz Bishwas Chamling Caroline Morbach Roman Pfister |
| author_facet | Richard J. Nies Svenja Ney Ingrid Kindermann Yvonne Bewarder Angela Zimmer Fabian Knebel Katrin Hahn Sebastian Spethmann Peter Luedike Lars Michel Tienush Rassaf Maria Papathanasiou Stefan Störk Vladimir Cejka Amin Polzin Fabian Voss Malte Kelm Bernhard Unsöld Christine Meindl Michael Paulus Ali Yilmaz Bishwas Chamling Caroline Morbach Roman Pfister |
| author_sort | Richard J. Nies |
| collection | DOAJ |
| description | 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. |
| format | Article |
| id | doaj-art-39c87cb2fbd84d709642f98d878f8f9a |
| institution | DOAJ |
| issn | 2055-5822 |
| language | English |
| publishDate | 2025-04-01 |
| publisher | Wiley |
| record_format | Article |
| series | ESC Heart Failure |
| spelling | doaj-art-39c87cb2fbd84d709642f98d878f8f9a2025-08-20T02:55:48ZengWileyESC Heart Failure2055-58222025-04-011221203121610.1002/ehf2.15126Real‐world characteristics and treatment of cardiac transthyretin amyloidosis: A multicentre, observational studyRichard J. Nies0Svenja Ney1Ingrid Kindermann2Yvonne Bewarder3Angela Zimmer4Fabian Knebel5Katrin Hahn6Sebastian Spethmann7Peter Luedike8Lars Michel9Tienush Rassaf10Maria Papathanasiou11Stefan Störk12Vladimir Cejka13Amin Polzin14Fabian Voss15Malte Kelm16Bernhard Unsöld17Christine Meindl18Michael Paulus19Ali Yilmaz20Bishwas Chamling21Caroline Morbach22Roman Pfister23Faculty of Medicine and University Hospital Cologne, Clinic III for Internal Medicine University of Cologne Cologne GermanyFaculty of Medicine and University Hospital Cologne, Clinic III for Internal Medicine University of Cologne Cologne GermanyKlinik für Innere Medizin III, Universitätsklinikum des Saarlandes Saarland University Homburg GermanyKlinik für Innere Medizin III, Universitätsklinikum des Saarlandes Saarland University Homburg GermanyKlinik für Innere Medizin III, Universitätsklinikum des Saarlandes Saarland University Homburg GermanySana Klinikum Berlin Lichtenberg Berlin GermanyKlinik für Kardiologie, Angiologie und Intensivmedizin Deutsches Herzzentrum der Charité Berlin GermanyKlinik für Kardiologie, Angiologie und Intensivmedizin Deutsches Herzzentrum der Charité Berlin GermanyWest German Heart and Vascular Center, Department of Cardiology and Vascular Medicine University Hospital Essen, University Duisburg‐Essen Essen GermanyWest German Heart and Vascular Center, Department of Cardiology and Vascular Medicine University Hospital Essen, University Duisburg‐Essen Essen GermanyWest German Heart and Vascular Center, Department of Cardiology and Vascular Medicine University Hospital Essen, University Duisburg‐Essen Essen GermanyWest German Heart and Vascular Center, Department of Cardiology and Vascular Medicine University Hospital Essen, University Duisburg‐Essen Essen GermanyDepartment of Clinical Research and Epidemiology, Department of Medicine I, Comprehensive Heart Failure Center University Hospital Würzburg Würzburg GermanyDepartment of Clinical Research and Epidemiology, Department of Medicine I, Comprehensive Heart Failure Center University Hospital Würzburg Würzburg GermanyDivision of Cardiology, Pulmonology, and Vascular Medicine, University Duesseldorf, Medical Faculty Cardiovascular Research Institute Düsseldorf (CARID) Duesseldorf GermanyDivision of Cardiology, Pulmonology, and Vascular Medicine, University Duesseldorf, Medical Faculty Cardiovascular Research Institute Düsseldorf (CARID) Duesseldorf GermanyDivision of Cardiology, Pulmonology, and Vascular Medicine, University Duesseldorf, Medical Faculty Cardiovascular Research Institute Düsseldorf (CARID) Duesseldorf GermanyMedical Clinic I, Cardiology and Angiology Justus‐Liebig‐University Giessen Giessen GermanyDepartment of Internal Medicine II University Hospital Regensburg Regensburg GermanyDepartment of Internal Medicine II University Hospital Regensburg Regensburg GermanyKlinik für Kardiologie I, Sektion für Herzbildgebung Universitätsklinikum Münster Münster GermanyKlinik für Kardiologie I, Sektion für Herzbildgebung Universitätsklinikum Münster Münster GermanyDepartment of Clinical Research and Epidemiology, Department of Medicine I, Comprehensive Heart Failure Center University Hospital Würzburg Würzburg GermanyFaculty of Medicine and University Hospital Cologne, Clinic III for Internal Medicine University of Cologne Cologne GermanyAbstract 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.https://doi.org/10.1002/ehf2.15126Cardiac amyloidosisCardiomyopathyHeart failureTTRTransthyretinTafamidis |
| spellingShingle | Richard J. Nies Svenja Ney Ingrid Kindermann Yvonne Bewarder Angela Zimmer Fabian Knebel Katrin Hahn Sebastian Spethmann Peter Luedike Lars Michel Tienush Rassaf Maria Papathanasiou Stefan Störk Vladimir Cejka Amin Polzin Fabian Voss Malte Kelm Bernhard Unsöld Christine Meindl Michael Paulus Ali Yilmaz Bishwas Chamling Caroline Morbach Roman Pfister Real‐world characteristics and treatment of cardiac transthyretin amyloidosis: A multicentre, observational study ESC Heart Failure Cardiac amyloidosis Cardiomyopathy Heart failure TTR Transthyretin Tafamidis |
| title | Real‐world characteristics and treatment of cardiac transthyretin amyloidosis: A multicentre, observational study |
| title_full | Real‐world characteristics and treatment of cardiac transthyretin amyloidosis: A multicentre, observational study |
| title_fullStr | Real‐world characteristics and treatment of cardiac transthyretin amyloidosis: A multicentre, observational study |
| title_full_unstemmed | Real‐world characteristics and treatment of cardiac transthyretin amyloidosis: A multicentre, observational study |
| title_short | Real‐world characteristics and treatment of cardiac transthyretin amyloidosis: A multicentre, observational study |
| title_sort | real world characteristics and treatment of cardiac transthyretin amyloidosis a multicentre observational study |
| topic | Cardiac amyloidosis Cardiomyopathy Heart failure TTR Transthyretin Tafamidis |
| url | https://doi.org/10.1002/ehf2.15126 |
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