TY - JOUR
T1 - Tafamidis in women with wild-type transthyretin cardiac amyloidosis
T2 - an international cohort study
AU - Debonnaire, Philippe
AU - L'Hoyes, Wouter
AU - Dujardin, Karl
AU - Donal, Erwan
AU - Verheyen, Nicolas
AU - Dupont, Matthias
AU - De Sutter, Johan
AU - Timmermans, Philippe
AU - Pouleur, Anne-Catherine
AU - Droogmans, Steven
AU - Issa, Victor Sarli
AU - Dulgheru, Raluca
AU - Regeer, Madelien
AU - Jurcut, Ruxandra
AU - Bondue, Antoine
AU - Bohyn, Alexandre
AU - Bogaerts, Kris
AU - Christiaen, Emma
AU - Wyseure, Nicolas
AU - Bezard, Melanie
AU - Zach, David
AU - Schwegel, Nora
AU - Knapen, Robbe
AU - Buytaert, Lars
AU - de Marneffe, Nils
AU - Ajmone Marsan, Nina
AU - Adam, Robert
AU - Tavernier, Rene
AU - Buysschaert, Ian
AU - Trenson, Sander
N1 - © The Author(s) 2026. Published by Oxford University Press on behalf of the European Society of Cardiology. All rights reserved. For commercial re-use, please contact [email protected] for reprints and translation rights for reprints. All other permissions can be obtained through our RightsLink service via the Permissions link on the article page on our site—for further information please contact [email protected].
PY - 2026/5/19
Y1 - 2026/5/19
N2 - AIMS: The natural history and response to tafamidis treatment in women with wild-type transthyretin amyloid cardiomyopathy (ATTRwt-CM) remain insufficiently characterised. Current study aimed to explore sex-differences in clinical presentation, natural course and tafamidis treatment efficacy, focusing on women with ATTRwt-CM.METHODS AND RESULTS: An international, multicentric cohort of ATTRwt-CM subjects was evaluated, including for all-cause mortality. In total 1454 patients were studied (mean age 81±7y), including 307 (21.1%) females. At presentation, females were ∼3 years older than males with slightly worse phenotype, including higher indexed left ventricular wall thickness and National Amyloidosis Centre (NAC) disease stage (p<0.050). Heart failure with preserved ejection fraction and hypertension coincided more often in women (p=0.001). Natural disease course was poor without sex-difference, even when age-corrected (p=0.210). Tafamidis was initiated in 1055 patients, 12% less in females (p<0.001), although reasons for non-initiation and discontinuation did not show heterogeneity by sex (p=0.116 and p=0.304, respectively), indicating structural undertreatment. After 1.9 (0.9-3.3) years of median follow-up, 409 (28.1%) patients died. Tafamidis related to lower mortality in the overall and propensity score-matched cohort (n=742, HR 0.44, 95%CI 0.32-0.61, p<0.001), without sex-difference (female HR 0.76, 95%CI 0.52-1.11, p=0.150) nor sex-based treatment efficacy interaction (p=0.381). NAC disease stages strongly related to mortality under tafamidis treatment (HR 2.13%, 95%, 1.81-2.50, p<0.001), but female sex did not (HR 0.82, 95%CI 0.53-1.27, p=0.365).CONCLUSIONS: Women with ATTRwt-CM are prone to underdiagnosis and undertreatment, despite similar poor natural course and tafamidis treatment efficacy. Initiatives to increase diagnostic awareness and disease modifying treatment initiation in women are urgently needed.
AB - AIMS: The natural history and response to tafamidis treatment in women with wild-type transthyretin amyloid cardiomyopathy (ATTRwt-CM) remain insufficiently characterised. Current study aimed to explore sex-differences in clinical presentation, natural course and tafamidis treatment efficacy, focusing on women with ATTRwt-CM.METHODS AND RESULTS: An international, multicentric cohort of ATTRwt-CM subjects was evaluated, including for all-cause mortality. In total 1454 patients were studied (mean age 81±7y), including 307 (21.1%) females. At presentation, females were ∼3 years older than males with slightly worse phenotype, including higher indexed left ventricular wall thickness and National Amyloidosis Centre (NAC) disease stage (p<0.050). Heart failure with preserved ejection fraction and hypertension coincided more often in women (p=0.001). Natural disease course was poor without sex-difference, even when age-corrected (p=0.210). Tafamidis was initiated in 1055 patients, 12% less in females (p<0.001), although reasons for non-initiation and discontinuation did not show heterogeneity by sex (p=0.116 and p=0.304, respectively), indicating structural undertreatment. After 1.9 (0.9-3.3) years of median follow-up, 409 (28.1%) patients died. Tafamidis related to lower mortality in the overall and propensity score-matched cohort (n=742, HR 0.44, 95%CI 0.32-0.61, p<0.001), without sex-difference (female HR 0.76, 95%CI 0.52-1.11, p=0.150) nor sex-based treatment efficacy interaction (p=0.381). NAC disease stages strongly related to mortality under tafamidis treatment (HR 2.13%, 95%, 1.81-2.50, p<0.001), but female sex did not (HR 0.82, 95%CI 0.53-1.27, p=0.365).CONCLUSIONS: Women with ATTRwt-CM are prone to underdiagnosis and undertreatment, despite similar poor natural course and tafamidis treatment efficacy. Initiatives to increase diagnostic awareness and disease modifying treatment initiation in women are urgently needed.
UR - https://www.webofscience.com/api/gateway?GWVersion=2&SrcApp=medunigraz_woslite&SrcAuth=WosAPI&KeyUT=WOS:001769532500001&DestLinkType=FullRecord&DestApp=WOS_CPL
U2 - 10.1093/ehjqcco/qcag074
DO - 10.1093/ehjqcco/qcag074
M3 - Research article
C2 - 42035239
SN - 2058-5225
JO - European Heart Journal - Quality of Care and Clinical Outcomes
JF - European Heart Journal - Quality of Care and Clinical Outcomes
ER -