Accuracy of Established Prognostic Staging Systems for Cardiac Transthyretin Amyloidosis in the Tafamidis Era
Background: Biomarker-based prognostic staging systems, including the National Amyloidosis Centre (NAC) and the Mayo staging systems, are widely-used but have only been validated for treatment-naive patients with cardiac transthyretin amyloidosis (ATTR-CA). Objectives: The purpose of this study was...
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Elsevier
2025-02-01
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| Series: | JACC: Advances |
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| Online Access: | http://www.sciencedirect.com/science/article/pii/S2772963X24008494 |
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| author | Maximilian Leo Müller, MD Sebastian Spethmann, MD Daniel Messroghli, MD Anna Brand, MD Isabel Mattig, MD Katrin Hahn, MD Ulf Landmesser, MD Bettina Heidecker, MD |
| author_facet | Maximilian Leo Müller, MD Sebastian Spethmann, MD Daniel Messroghli, MD Anna Brand, MD Isabel Mattig, MD Katrin Hahn, MD Ulf Landmesser, MD Bettina Heidecker, MD |
| author_sort | Maximilian Leo Müller, MD |
| collection | DOAJ |
| description | Background: Biomarker-based prognostic staging systems, including the National Amyloidosis Centre (NAC) and the Mayo staging systems, are widely-used but have only been validated for treatment-naive patients with cardiac transthyretin amyloidosis (ATTR-CA). Objectives: The purpose of this study was to assess the accuracy of the NAC and Mayo staging systems in patients with ATTR-CA treated with tafamidis. Methods: A retrospective observational study following patients with ATTR-CA from initiation of tafamidis (baseline) to time of all-cause death was conducted. Patients were stratified according to the NAC and an adapted Mayo staging system incorporating high-sensitivity cardiac troponin T. Agreement was assessed using weighted Cohen’s kappa. The staging systems’ ability to identify subgroups with distinct overall survival was assessed using Kaplan-Meier analyses and pairwise log-rank tests. Results: A total of 251 patients with wild-type ATTR-CA treated with tafamidis were followed for a median of 521 (IQR: 262-842) days. There was substantial agreement (weighted kappa = 0.661; P < 0.001) between the NAC and the adapted Mayo staging system. Significant differences in estimated overall survival were observed across all disease stages of the adapted Mayo staging system (I vs II: P = 0.032; I vs III: P < 0.001; II vs III: P = 0.036). Accordingly, estimated overall survival was significantly lower in NAC III compared to NAC I (P < 0.001) and NAC II (P = 0.015). However, there was no significant difference between NAC I and NAC II (P = 0.340). Conclusions: Both staging systems identified a distinct group of patients with ATTR-CA at the highest risk of death but only the adapted Mayo staging system could accurately distinguish between low- and intermediate-risk patients in the setting of disease-modifying treatment with tafamidis. |
| format | Article |
| id | doaj-art-6dd458bfdb194bd79be05f1d5a3ceb7c |
| institution | Kabale University |
| issn | 2772-963X |
| language | English |
| publishDate | 2025-02-01 |
| publisher | Elsevier |
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| series | JACC: Advances |
| spelling | doaj-art-6dd458bfdb194bd79be05f1d5a3ceb7c2025-01-19T06:27:00ZengElsevierJACC: Advances2772-963X2025-02-0142101568Accuracy of Established Prognostic Staging Systems for Cardiac Transthyretin Amyloidosis in the Tafamidis EraMaximilian Leo Müller, MD0Sebastian Spethmann, MD1Daniel Messroghli, MD2Anna Brand, MD3Isabel Mattig, MD4Katrin Hahn, MD5Ulf Landmesser, MD6Bettina Heidecker, MD7Department of Cardiology, Angiology and Intensive Care Medicine, Deutsches Herzzentrum der Charité, Berlin, Germany; Charité–Universitätsmedizin Berlin, Corporate Member of Freie Universität Berlin and Humboldt-Universität Zu Berlin, Berlin, Germany; Amyloidosis Center Charité Berlin (ACCB), Charité–Universitätsmedizin Berlin, Berlin, GermanyDepartment of Cardiology, Angiology and Intensive Care Medicine, Deutsches Herzzentrum der Charité, Berlin, Germany; Charité–Universitätsmedizin Berlin, Corporate Member of Freie Universität Berlin and Humboldt-Universität Zu Berlin, Berlin, Germany; Amyloidosis Center Charité Berlin (ACCB), Charité–Universitätsmedizin Berlin, Berlin, Germany; DZHK (German Centre for Cardiovascular Research), Partner Site Berlin, Berlin, GermanyDepartment of Cardiology, Angiology and Intensive Care Medicine, Deutsches Herzzentrum der Charité, Berlin, Germany; Charité–Universitätsmedizin Berlin, Corporate Member of Freie Universität Berlin and Humboldt-Universität Zu Berlin, Berlin, Germany; Amyloidosis Center Charité Berlin (ACCB), Charité–Universitätsmedizin Berlin, Berlin, Germany; DZHK (German Centre for Cardiovascular Research), Partner Site Berlin, Berlin, Germany; Department of Cardiology, Rhythmology and Angiology, Medizinische Universität Lausitz–Carl Thiem, Cottbus, GermanyDepartment of Cardiology, Angiology and Intensive Care Medicine, Deutsches Herzzentrum der Charité, Berlin, Germany; Charité–Universitätsmedizin Berlin, Corporate Member of Freie Universität Berlin and Humboldt-Universität Zu Berlin, Berlin, Germany; Amyloidosis Center Charité Berlin (ACCB), Charité–Universitätsmedizin Berlin, Berlin, Germany; DZHK (German Centre for Cardiovascular Research), Partner Site Berlin, Berlin, GermanyDepartment of Cardiology, Angiology and Intensive Care Medicine, Deutsches Herzzentrum der Charité, Berlin, Germany; Charité–Universitätsmedizin Berlin, Corporate Member of Freie Universität Berlin and Humboldt-Universität Zu Berlin, Berlin, Germany; Amyloidosis Center Charité Berlin (ACCB), Charité–Universitätsmedizin Berlin, Berlin, Germany; DZHK (German Centre for Cardiovascular Research), Partner Site Berlin, Berlin, Germany; Berlin Institute of Health (BIH) at Charité, Berlin, GermanyCharité–Universitätsmedizin Berlin, Corporate Member of Freie Universität Berlin and Humboldt-Universität Zu Berlin, Berlin, Germany; Amyloidosis Center Charité Berlin (ACCB), Charité–Universitätsmedizin Berlin, Berlin, Germany; Berlin Institute of Health (BIH) at Charité, Berlin, Germany; Department of Neurology and Experimental Neurology, Charité - Universitätsmedizin Berlin, Corporate Member of Freie Universität Berlin and Humboldt-Universität zu Berlin, Berlin, GermanyDepartment of Cardiology, Angiology and Intensive Care Medicine, Deutsches Herzzentrum der Charité, Berlin, Germany; Charité–Universitätsmedizin Berlin, Corporate Member of Freie Universität Berlin and Humboldt-Universität Zu Berlin, Berlin, Germany; DZHK (German Centre for Cardiovascular Research), Partner Site Berlin, Berlin, GermanyDepartment of Cardiology, Angiology and Intensive Care Medicine, Deutsches Herzzentrum der Charité, Berlin, Germany; Charité–Universitätsmedizin Berlin, Corporate Member of Freie Universität Berlin and Humboldt-Universität Zu Berlin, Berlin, Germany; Amyloidosis Center Charité Berlin (ACCB), Charité–Universitätsmedizin Berlin, Berlin, Germany; Berlin Institute of Health (BIH) at Charité, Berlin, Germany; Address for correspondence: Dr Bettina Heidecker, Deutsches Herzzentrum der Charité, Campus Benjamin Franklin, Hindenburgdamm 30, Berlin 12203, Germany.Background: Biomarker-based prognostic staging systems, including the National Amyloidosis Centre (NAC) and the Mayo staging systems, are widely-used but have only been validated for treatment-naive patients with cardiac transthyretin amyloidosis (ATTR-CA). Objectives: The purpose of this study was to assess the accuracy of the NAC and Mayo staging systems in patients with ATTR-CA treated with tafamidis. Methods: A retrospective observational study following patients with ATTR-CA from initiation of tafamidis (baseline) to time of all-cause death was conducted. Patients were stratified according to the NAC and an adapted Mayo staging system incorporating high-sensitivity cardiac troponin T. Agreement was assessed using weighted Cohen’s kappa. The staging systems’ ability to identify subgroups with distinct overall survival was assessed using Kaplan-Meier analyses and pairwise log-rank tests. Results: A total of 251 patients with wild-type ATTR-CA treated with tafamidis were followed for a median of 521 (IQR: 262-842) days. There was substantial agreement (weighted kappa = 0.661; P < 0.001) between the NAC and the adapted Mayo staging system. Significant differences in estimated overall survival were observed across all disease stages of the adapted Mayo staging system (I vs II: P = 0.032; I vs III: P < 0.001; II vs III: P = 0.036). Accordingly, estimated overall survival was significantly lower in NAC III compared to NAC I (P < 0.001) and NAC II (P = 0.015). However, there was no significant difference between NAC I and NAC II (P = 0.340). Conclusions: Both staging systems identified a distinct group of patients with ATTR-CA at the highest risk of death but only the adapted Mayo staging system could accurately distinguish between low- and intermediate-risk patients in the setting of disease-modifying treatment with tafamidis.http://www.sciencedirect.com/science/article/pii/S2772963X24008494ATTR-CAdisease-modifying treatmentestimated glomerular filtration rate (eGFR)high-sensitivity cardiac troponin T (hs-cTnT)N-terminal pro B-type natriuretic peptide (NT-proBNP)risk-stratification |
| spellingShingle | Maximilian Leo Müller, MD Sebastian Spethmann, MD Daniel Messroghli, MD Anna Brand, MD Isabel Mattig, MD Katrin Hahn, MD Ulf Landmesser, MD Bettina Heidecker, MD Accuracy of Established Prognostic Staging Systems for Cardiac Transthyretin Amyloidosis in the Tafamidis Era JACC: Advances ATTR-CA disease-modifying treatment estimated glomerular filtration rate (eGFR) high-sensitivity cardiac troponin T (hs-cTnT) N-terminal pro B-type natriuretic peptide (NT-proBNP) risk-stratification |
| title | Accuracy of Established Prognostic Staging Systems for Cardiac Transthyretin Amyloidosis in the Tafamidis Era |
| title_full | Accuracy of Established Prognostic Staging Systems for Cardiac Transthyretin Amyloidosis in the Tafamidis Era |
| title_fullStr | Accuracy of Established Prognostic Staging Systems for Cardiac Transthyretin Amyloidosis in the Tafamidis Era |
| title_full_unstemmed | Accuracy of Established Prognostic Staging Systems for Cardiac Transthyretin Amyloidosis in the Tafamidis Era |
| title_short | Accuracy of Established Prognostic Staging Systems for Cardiac Transthyretin Amyloidosis in the Tafamidis Era |
| title_sort | accuracy of established prognostic staging systems for cardiac transthyretin amyloidosis in the tafamidis era |
| topic | ATTR-CA disease-modifying treatment estimated glomerular filtration rate (eGFR) high-sensitivity cardiac troponin T (hs-cTnT) N-terminal pro B-type natriuretic peptide (NT-proBNP) risk-stratification |
| url | http://www.sciencedirect.com/science/article/pii/S2772963X24008494 |
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