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. 2025 Nov 20;4(12 Pt 2):102354.
doi: 10.1016/j.jacadv.2025.102354. Online ahead of print.

Characteristics and Prognosis of Wild-Type Transthyretin Amyloid Cardiomyopathy Patients Diagnosed Before 65 Years Old

Damien Guijarro  1 , Jean-Christophe Eicher  2 , Mélanie Bézard  3 , Nicolas Piriou  4 , François Sauer  5 , François Roubille  6 , Jérôme Costa  7 , Patricia Réant  8 , Erwan Donal  9 , Fabrice Bauer  10 , Arnaud Bisson  11 , Océane Bouchot  12 , Eve Cariou  13 , Olivier Lairez  13 , Pierre-Yves Courand  14 , Charlotte Dagrenat  15 , Jean-Pierre Gueffet  16 , Gilbert Habib  17 , Julien Jeanneteau  18 , Léa Margerit  19 , Silvia Oghina  20 , Romain Trésorier  21 , Mounira Kharoubi  22 , Thibaud Damy  23
Affiliations
Free article

Characteristics and Prognosis of Wild-Type Transthyretin Amyloid Cardiomyopathy Patients Diagnosed Before 65 Years Old

Damien Guijarro et al. JACC Adv. .
Free article

Abstract

Background: Guidelines recommend screening for transthyretin amyloid cardiomyopathy (ATTR-CM) after age 65 years, yet some patients are diagnosed earlier.

Objectives: The purpose of this study was to compare the proportion, clinical characteristics, and prognosis of wild-type ATTR-CM diagnosed ≤65 years (ATTRwt-Yy) with those diagnosed >65 years (ATTRwt-O).

Methods: Data from the HEAR (Healthcare European Amyloidosis Registry), a multicenter, noninterventional, longitudinal registry, were analyzed. Patients were categorized by age at diagnosis: ATTRwt-Yy (≤65 years) and ATTRwt-O (>65 years). ATTRwt-O patients were further classified by onset of first cardiac symptom: ATTRwt-Oy (≤65 years) and ATTRwt-Oo (>65 years).

Results: From July 2021 to May 2024, 3,980 ATTR patients were enrolled; 1,417 had ATTRwt-CM with documented symptom onset. Among them, 67 (4.7%) were ATTRwt-Yy, 111 (7.8%) ATTRwt-Oy, and 1,239 (87.4%) ATTRwt-Oo. Diagnostic delays were 0.65, 20.58, and 0.77 years, respectively (P < 0.001). Heart failure signs at presentation were seen in 34.9% of ATTRwt-Yy, 8.1% of ATTRwt-Oy, and 30.2% of ATTRwt-Oo (P < 0.001). ATTRwt-Yy patients had more extracardiac manifestations, notably osteoarticular disease, whereas rhythm disturbances predominated in ATTRwt-Oy. Median follow-up from diagnosis was 36.2, 23.6, and 22.4 months, respectively. ATTRwt-Yy patients had better survival after diagnosis compared to ATTRwt-O patients.

Conclusions: ATTRwt-CM diagnosed before 65 years shows a distinct phenotype highlighting the need for tailored diagnostic and management strategies.

Keywords: amyloidosis; diagnosis; prognosis; transthyretin.

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Conflict of interest statement

Funding support and author disclosures Dr Guijarro has received consultancy fees from Alnylam and Pfizer. Dr Piriou has received honoraria, consulting fees, and support for attending meeting and/or travel from Alnylam and Pfizer. Dr Sauer has received honoraria from Bristol Myers Squibb and Pfizer. Dr Roubille has received grants/contracts from Abbott and Air Liquide; has received consulting fees from Abbott, Air Liquide, Bayer, and Pfizer; has received honoraria and/or support for attending meetings/travel from Abbott, Air Liquide, Alnylam, AstraZeneca, Bayer, Boehringer, Bristol Myers Squibb, GSK, Implicity, MSD, Newcard, Novartis, Novo Nordisk, Pfizer, QuidelOrtho, Sanofi, Servier, Vifor, and Zoll; has participated in a board for Carmat; has a leadership or fiduciary role in a board/committee/society for Boehringer Ingelheim, Novartis, and Vifor; and has received support for medical writing from Pfizer. Dr Réant has received honoraria, consulting fees, and support for attending meetings/travel from Alnylam and Pfizer. Dr Donal has received grants from Alnylam and consulting fees from Alnylam, AstraZeneca, and Pfizer. Dr Lairez has received speaking fees from Alnylam Pharmaceuticals, Amicus Therapeutics, AstraZeneca, BMS, Bridgebio, Pfizer, Sanofi-Genzyme, and Siemens Healthiners; consulting fees from Alnylam Pharmaceuticals, Amicus Therapeutics, AstraZeneca, and Pfizer; and educational support to his institution from Pfizer. Dr Courand has received consulting fees and honoraria (as an author) from Abbvie, AstraZeneca, Amgen, Astellas, Boehringer Ingelheim/Lilly, Bristol Myers Squibb, Medtronic, Organon, and Pfizer. Dr Dagrenat has received grants, consulting fees, honoraria and/or support for attending meetings/travel from Alnylam, AstraZeneca, Boehringer Ingelheim, Bristol Myers Squibb, and Pfizer. Dr Gueffet has received honoraria and/or support for attending meetings or for travel from Abbott, AstraZeneca, Bayer, Boehringer Ingelheim, CMD Health, GSK, Janssen Cilag, Medtronic, MSD, Novartis, Pfizer, Sanofi, and Viatris. Dr Jeanneteau has received honoraria from AstraZeneca, Bristil Myers Squibb, Novartis, Novo Nordisk, and Pfizer. Dr Oghina has received consulting fees and honoraria from Alnylam, AstraZeneca, Bayer, and Pfizer. Dr Damy has received research grant or consultancy fees from Alnylam, Alexion, AstraZeneca, Bayer, Bridge-Bio, Pfizer, and Neurimmune. Dr Costa has received honoraria, consulting fees from Alnylam, Pfizer, Boehringer, Novartis, Servier, Amgen and Bristol Myers Squibb and support for attending meetings and/or travel from Novartis, Pfizer, Servier, Bristol Myers Squibb, AstraZeneca and Alnylam, has participated on a Data Safety Monitoring Board or Advisory Board for AstraZeneca, Pfizer, Novartis, Novonordisk and Sanofi Genzyme. All other authors have reported that they have no relationships relevant to the contents of this paper to disclose. HEAR is promoted by the HEART foundation.

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