Predictors of Early Death in Patients With Wild‐Type Transthyretin Cardiac Amyloidosis
Background For the time being, tafamidis is the only approved treatment for wild‐type transthyretin cardiac amyloidosis. However, benefits on all‐cause death only emerge after ≈18 months. The current available staging systems are unable to specifically discriminate patients at high risk of death wit...
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2025-01-01
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| Series: | Journal of the American Heart Association: Cardiovascular and Cerebrovascular Disease |
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| Online Access: | https://www.ahajournals.org/doi/10.1161/JAHA.124.036755 |
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| author | Paolo Milani Giuseppe Damiano Sanna Roberta Mussinelli Marco Basset Gianluigi Guida Andrea Attanasio Martina Nanci Francesca Fabris Claudia Bellofiore Alessandro Fogliani Elisa Novello Francesca Benigna Laura Obici Pietro Benvenuti Martina Ciardo Mario Nuvolone Manuela Averaimo Gavino Casu Andrea Foli Stefano Perlini Giampaolo Merlini Giovanni Palladini |
| author_facet | Paolo Milani Giuseppe Damiano Sanna Roberta Mussinelli Marco Basset Gianluigi Guida Andrea Attanasio Martina Nanci Francesca Fabris Claudia Bellofiore Alessandro Fogliani Elisa Novello Francesca Benigna Laura Obici Pietro Benvenuti Martina Ciardo Mario Nuvolone Manuela Averaimo Gavino Casu Andrea Foli Stefano Perlini Giampaolo Merlini Giovanni Palladini |
| author_sort | Paolo Milani |
| collection | DOAJ |
| description | Background For the time being, tafamidis is the only approved treatment for wild‐type transthyretin cardiac amyloidosis. However, benefits on all‐cause death only emerge after ≈18 months. The current available staging systems are unable to specifically discriminate patients at high risk of death within 18 months from diagnosis, and the selection of patients who are expected to benefit from tafamidis is left to the clinical judgment of treating physicians, being often based primarily (and sometimes only) on age. We searched baseline variables able to discriminate patients at risk of death within 18 months. Methods and Results We randomly divided our prospectively maintained database in 2 cohorts, a testing (two thirds) and a validating (one third) set, respectively. We used the Eastern Cooperative Oncology Group–Performance Status as a simple scoring of frailty. We analyzed the clinical, laboratory and instrumental features of 691 consecutive wild‐type transthyretin cardiac amyloidosis patients diagnosed between 2006 and 2021. Median follow‐up was 43 months, and 367 patients (53%) died. Eighteen‐month death was predicted by NT‐proBNP (N‐terminal pro‐B‐type natriuretic peptide; cutoff 4200 ng/L), cardiac troponin I (cutoff 92 ng/L), and age (cutoff 80 years). At multivariable analysis, Eastern Cooperative Oncology Group–Performance Status >1 along with the selected variables were independent prognostic determinants and outperformed New York Heart Association functional class. The combination of these variables identified standard‐ and high‐risk patients (all variables above the cutoffs) with a median survival of 57 and 17 months, respectively (P<0.001). This was confirmed in the validation set. Conclusions A simple scoring system including age, cardiac biomarkers, and Eastern Cooperative Oncology Group–Performance Status identifies subjects with cardiac wild‐type transthyretin cardiac amyloidosis at high risk of early death. |
| format | Article |
| id | doaj-art-73a8bda1733c4eb59abafb9446535c36 |
| institution | OA Journals |
| issn | 2047-9980 |
| language | English |
| publishDate | 2025-01-01 |
| publisher | Wiley |
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| series | Journal of the American Heart Association: Cardiovascular and Cerebrovascular Disease |
| spelling | doaj-art-73a8bda1733c4eb59abafb9446535c362025-08-20T02:02:50ZengWileyJournal of the American Heart Association: Cardiovascular and Cerebrovascular Disease2047-99802025-01-0114110.1161/JAHA.124.036755Predictors of Early Death in Patients With Wild‐Type Transthyretin Cardiac AmyloidosisPaolo Milani0Giuseppe Damiano Sanna1Roberta Mussinelli2Marco Basset3Gianluigi Guida4Andrea Attanasio5Martina Nanci6Francesca Fabris7Claudia Bellofiore8Alessandro Fogliani9Elisa Novello10Francesca Benigna11Laura Obici12Pietro Benvenuti13Martina Ciardo14Mario Nuvolone15Manuela Averaimo16Gavino Casu17Andrea Foli18Stefano Perlini19Giampaolo Merlini20Giovanni Palladini21Department of Molecular Medicine University of Pavia ItalyDepartment of Molecular Medicine University of Pavia ItalyAmyloidosis Research and Treatment Center Foundation IRCCS Policlinico San Matteo Pavia ItalyAmyloidosis Research and Treatment Center Foundation IRCCS Policlinico San Matteo Pavia ItalyCardiology Universitary Department IRCCS Policlinico San Donato San Donato Milanese ItalyCardiology Universitary Department IRCCS Policlinico San Donato San Donato Milanese ItalyDepartment of Molecular Medicine University of Pavia ItalyInstitute of Cardiology, Maggiore Hospital Crema ItalyDepartment of Molecular Medicine University of Pavia ItalyAmyloidosis Research and Treatment Center Foundation IRCCS Policlinico San Matteo Pavia ItalyAmyloidosis Research and Treatment Center Foundation IRCCS Policlinico San Matteo Pavia ItalyAmyloidosis Research and Treatment Center Foundation IRCCS Policlinico San Matteo Pavia ItalyAmyloidosis Research and Treatment Center Foundation IRCCS Policlinico San Matteo Pavia ItalyAmyloidosis Research and Treatment Center Foundation IRCCS Policlinico San Matteo Pavia ItalyDepartment of Molecular Medicine University of Pavia ItalyDepartment of Molecular Medicine University of Pavia ItalyCardiocentro Bellinzona ItalyClinical and Interventional Cardiology Sassari University Hospital Sassari ItalyAmyloidosis Research and Treatment Center Foundation IRCCS Policlinico San Matteo Pavia ItalyAmyloidosis Research and Treatment Center Foundation IRCCS Policlinico San Matteo Pavia ItalyDepartment of Molecular Medicine University of Pavia ItalyDepartment of Molecular Medicine University of Pavia ItalyBackground For the time being, tafamidis is the only approved treatment for wild‐type transthyretin cardiac amyloidosis. However, benefits on all‐cause death only emerge after ≈18 months. The current available staging systems are unable to specifically discriminate patients at high risk of death within 18 months from diagnosis, and the selection of patients who are expected to benefit from tafamidis is left to the clinical judgment of treating physicians, being often based primarily (and sometimes only) on age. We searched baseline variables able to discriminate patients at risk of death within 18 months. Methods and Results We randomly divided our prospectively maintained database in 2 cohorts, a testing (two thirds) and a validating (one third) set, respectively. We used the Eastern Cooperative Oncology Group–Performance Status as a simple scoring of frailty. We analyzed the clinical, laboratory and instrumental features of 691 consecutive wild‐type transthyretin cardiac amyloidosis patients diagnosed between 2006 and 2021. Median follow‐up was 43 months, and 367 patients (53%) died. Eighteen‐month death was predicted by NT‐proBNP (N‐terminal pro‐B‐type natriuretic peptide; cutoff 4200 ng/L), cardiac troponin I (cutoff 92 ng/L), and age (cutoff 80 years). At multivariable analysis, Eastern Cooperative Oncology Group–Performance Status >1 along with the selected variables were independent prognostic determinants and outperformed New York Heart Association functional class. The combination of these variables identified standard‐ and high‐risk patients (all variables above the cutoffs) with a median survival of 57 and 17 months, respectively (P<0.001). This was confirmed in the validation set. Conclusions A simple scoring system including age, cardiac biomarkers, and Eastern Cooperative Oncology Group–Performance Status identifies subjects with cardiac wild‐type transthyretin cardiac amyloidosis at high risk of early death.https://www.ahajournals.org/doi/10.1161/JAHA.124.036755amyloidosisbiomarkersprognosisstaging |
| spellingShingle | Paolo Milani Giuseppe Damiano Sanna Roberta Mussinelli Marco Basset Gianluigi Guida Andrea Attanasio Martina Nanci Francesca Fabris Claudia Bellofiore Alessandro Fogliani Elisa Novello Francesca Benigna Laura Obici Pietro Benvenuti Martina Ciardo Mario Nuvolone Manuela Averaimo Gavino Casu Andrea Foli Stefano Perlini Giampaolo Merlini Giovanni Palladini Predictors of Early Death in Patients With Wild‐Type Transthyretin Cardiac Amyloidosis Journal of the American Heart Association: Cardiovascular and Cerebrovascular Disease amyloidosis biomarkers prognosis staging |
| title | Predictors of Early Death in Patients With Wild‐Type Transthyretin Cardiac Amyloidosis |
| title_full | Predictors of Early Death in Patients With Wild‐Type Transthyretin Cardiac Amyloidosis |
| title_fullStr | Predictors of Early Death in Patients With Wild‐Type Transthyretin Cardiac Amyloidosis |
| title_full_unstemmed | Predictors of Early Death in Patients With Wild‐Type Transthyretin Cardiac Amyloidosis |
| title_short | Predictors of Early Death in Patients With Wild‐Type Transthyretin Cardiac Amyloidosis |
| title_sort | predictors of early death in patients with wild type transthyretin cardiac amyloidosis |
| topic | amyloidosis biomarkers prognosis staging |
| url | https://www.ahajournals.org/doi/10.1161/JAHA.124.036755 |
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