Phenotypic Considerations for Tafamidis Use in a Real-World Cohort of Older Patients with Transthyretin Amyloid Cardiomyopathy
9 affiliations
- 1Department of Advanced Medical and Surgical Sciences, University of Campania “Luigi Vanvitelli”
- 2Tuscan Regional Amyloidosis Centre, Careggi University Hospital
- 3Columbia University Irving Medical Center
- 4Department of Cardiology, Angiology and Respiratory Medicine, University Hospital Heidelberg
- 5Division of Hematology, Mayo Clinic
- 6Hospital Universitario Puerta de Hierro de Majadahonda, IDIPHISA, CIBERCV, and Centro Nacional de Investigaciones Cardiovasculares (CNIC)
- 7Department of Cardiovascular Medicine, Amyloidosis Center, Cleveland Clinic
- 8Pfizer Inc
- 9National Referral Center for Cardiac Amyloidosis, Henry Mondor University Hospital
Abstract
Introduction Disease-modifying therapies for transthyretin amyloid cardiomyopathy (ATTR-CM) improve clinical outcomes, yet older patients are undertreated. In this analysis, we evaluated survival and cardiovascular-related hospitalizations (CVH) among patients treated with tafamidis (tafamidis free acid at 61 mg or bioequivalent tafamidis meglumine at 80 mg) aged < 80, 80–85, and > 85 years to assess effects of baseline clinical phenotype on outcomes. Methods Data were pooled from an early access cohort (ClinicalTrials.gov ID: NCT02791230) and the Transthyretin Amyloidosis Outcomes Survey registry (NCT00628745; enrollment 2019–2023; cardiac/mixed phenotype). Outcomes included all-cause mortality (ACM), CVH, and ACM or CVH. Cox proportional hazards and negative binomial regression models were adjusted for age, sex, TTR genotype, phenotype, New York Heart Association (NYHA) class, and estimated glomerular filtration rate (eGFR). Results Among the 1924 patients enrolled in the study, 1188, 505, and 231 were aged < 80, 80–85, and > 85 years, respectively. Patients aged > 85 versus 80–85 and < 80 years had lower baseline mean eGFR (39.7 vs. 49.8 and 64.1 mL/min/1.73 m2, respectively) and lower modified body mass index (mBMI; 1047.1 vs. 1084.0 and 1134.3 kg g/[m2 L]). Also, more patients aged > 85 years versus 80–85 and < 80 years were NYHA class IV (3.3% vs. 0.8% and 0.9%). Kaplan–Meier estimated survival rates were significantly lower in patients aged > 85 and 80–85 years versus those aged < 80 years at months 30 (56.3% and 66.2% vs. 77.0%) and 42 (42.9% and 57.2% vs. 70.9%). ACM risk was higher in patients aged > 85 years than in those aged < 80 years (hazard ratio 1.49, 95% confidence interval [CI] 1.11–2.01). Total annual CVH rates in patients aged > 85, 80–85, and < 80 years were 0.79, 0.65, and 0.59, respectively. CVH and ACM or CVH risks were similar across groups. Patients aged 80–85 and < 80 years with preserved mBMI, eGFR, or NYHA class I/II symptoms had similar survival rates. Across groups, patients with NYHA class III symptoms or eGFR < 45 mL/min/1.73 m2 had similar survival rates. Conclusion Patients aged ≥ 80 years who were treated with tafamidis had outcomes comparable to those of younger patients when clinical vulnerability markers were considered. These findings support individualized treatment decisions based on patient’s overall clinical profile and multidimensional assessment rather than age alone. Trial registration ClinicalTrials.gov identifiers: NCT02791230, NCT00628745.
Related
- Impact of Tafamidis on Transthyretin Concentration and Its Relation to All-Cause Mortality in Patients with Transthyretin Amyloid Cardiomyopathy: An Analysis from ATTR-ACT
- Survival in a Contemporary, Real-World, Matched Cohort of Patients with Transthyretin Amyloid Cardiomyopathy Treated vs Not Treated with Tafamidis
- Cardiovascular-Related Mortality and Hospitalizations in Patients with Transthyretin Amyloid Cardiomyopathy Treated with Tafamidis: A Post Hoc Analysis of the Phase 3 ATTR-ACT and Long-Term Extension
- Sex Differences in a Contemporary, Real-World Cohort of Patients with Transthyretin Amyloid Cardiomyopathy Treated with Tafamidis
- Stage-Specific Cost-Effectiveness of Early vs. Delayed Tafamidis for Transthyretin Amyloid Cardiomyopathy in Japan
BibTeX
@article{fumagalli2026phenotypic,
title = {{Phenotypic Considerations for Tafamidis Use in a Real-World Cohort of Older Patients with Transthyretin Amyloid Cardiomyopathy}},
author = {Carlo Fumagalli and Francesco Cappelli and Mathew S. Maurer and Fabian aus dem Siepen and Angela Dispenzieri and Pablo Garcia-Pavia and Mazen Hanna and Martin Carlsson and Thibaud Damy},
journal = {Cardiology and Therapy},
year = {2026},
month = sep,
doi = {10.1007/s40119-026-00474-4},
url = {https://doi.org/10.1007/s40119-026-00474-4},
note = {CC BY-NC},
}
RIS
TY - JOUR
TI - Phenotypic Considerations for Tafamidis Use in a Real-World Cohort of Older Patients with Transthyretin Amyloid Cardiomyopathy
AU - Carlo Fumagalli
AU - Francesco Cappelli
AU - Mathew S. Maurer
AU - Fabian aus dem Siepen
AU - Angela Dispenzieri
AU - Pablo Garcia-Pavia
AU - Mazen Hanna
AU - Martin Carlsson
AU - Thibaud Damy
PY - 2026
DA - 2026/09/01
JO - Cardiology and Therapy
DO - 10.1007/s40119-026-00474-4
UR - https://doi.org/10.1007/s40119-026-00474-4
AB - Introduction Disease-modifying therapies for transthyretin amyloid cardiomyopathy (ATTR-CM) improve clinical outcomes, yet older patients are undertreated. In this analysis, we evaluated survival and cardiovascular-related hospitalizations (CVH) among patients treated with tafamidis (tafamidis free acid at 61 mg or bioequivalent tafamidis meglumine at 80 mg) aged < 80, 80–85, and > 85 years to assess effects of baseline clinical phenotype on outcomes. Methods Data were pooled from an early access cohort (ClinicalTrials.gov ID: NCT02791230) and the Transthyretin Amyloidosis Outcomes Survey registry (NCT00628745; enrollment 2019–2023; cardiac/mixed phenotype). Outcomes included all-cause mortality (ACM), CVH, and ACM or CVH. Cox proportional hazards and negative binomial regression models were adjusted for age, sex, TTR genotype, phenotype, New York Heart Association (NYHA) class, and estimated glomerular filtration rate (eGFR). Results Among the 1924 patients enrolled in the study, 1188, 505, and 231 were aged < 80, 80–85, and > 85 years, respectively. Patients aged > 85 versus 80–85 and < 80 years had lower baseline mean eGFR (39.7 vs. 49.8 and 64.1 mL/min/1.73 m2, respectively) and lower modified body mass index (mBMI; 1047.1 vs. 1084.0 and 1134.3 kg g/[m2 L]). Also, more patients aged > 85 years versus 80–85 and < 80 years were NYHA class IV (3.3% vs. 0.8% and 0.9%). Kaplan–Meier estimated survival rates were significantly lower in patients aged > 85 and 80–85 years versus those aged < 80 years at months 30 (56.3% and 66.2% vs. 77.0%) and 42 (42.9% and 57.2% vs. 70.9%). ACM risk was higher in patients aged > 85 years than in those aged < 80 years (hazard ratio 1.49, 95% confidence interval [CI] 1.11–2.01). Total annual CVH rates in patients aged > 85, 80–85, and < 80 years were 0.79, 0.65, and 0.59, respectively. CVH and ACM or CVH risks were similar across groups. Patients aged 80–85 and < 80 years with preserved mBMI, eGFR, or NYHA class I/II symptoms had similar survival rates. Across groups, patients with NYHA class III symptoms or eGFR < 45 mL/min/1.73 m2 had similar survival rates. Conclusion Patients aged ≥ 80 years who were treated with tafamidis had outcomes comparable to those of younger patients when clinical vulnerability markers were considered. These findings support individualized treatment decisions based on patient’s overall clinical profile and multidimensional assessment rather than age alone. Trial registration ClinicalTrials.gov identifiers: NCT02791230, NCT00628745.
SN - 2193-8261
SN - 2193-6544
ER -