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Multicenter Study
. 2024 May;30(5):669-678.
doi: 10.1016/j.cardfail.2023.09.016. Epub 2023 Oct 30.

The Cardiac Amyloidosis Registry Study (CARS): Rationale, Design and Methodology

Lily K Stern  1 , Justin L Grodin  2 , Mathew S Maurer  3 , Frederick L Ruberg  4 , Ayan R Patel  5 , Michel G Khouri  6 , Lori R Roth  2 , Mandar A Aras  7 , Anju Bhardwaj  8 , Priyanka Bhattacharya  3 , Yevgeniy Brailovsky  9 , Brian M Drachman  10 , Imo A Ebong  11 , Nowell M Fine  12 , Hanna Gaggin  13 , Deepa Gopal  4 , Jan Griffin  14 , Daniel Judge  15 , Paul Kim  16 , Joshua Mitchell  17 , Sumeet S Mitter  18 , Rajeev C Mohan  19 , Hannia Ramos  15 , Alex Reyentovich  20 , Farooq H Sheikh  21 , Brett Sperry  22 , Spencer Carter  23 , Marcus Urey  16 , Joban Vaishnav  24 , Amanda R Vest  5 , Michelle M Kittleson  1 , Jignesh K Patel  25
Affiliations
Multicenter Study

The Cardiac Amyloidosis Registry Study (CARS): Rationale, Design and Methodology

Lily K Stern et al. J Card Fail. 2024 May.

Abstract

Background: CARS (Cardiac Amyloidosis Registry Study) is a multicenter registry established in 2019 that includes patients with transthyretin (ATTR, wild-type and variant) and light chain (AL) cardiac amyloidosis (CA) evaluated at major amyloidosis centers between 1997 and 2025. CARS aims to describe the natural history of CA with attention to clinical and diagnostic variables at the time of diagnosis, real-world treatment patterns, and associated outcomes of patients in a diverse cohort that is more representative of the at-risk population than that described in CA clinical trials.

Methods and results: This article describes the design and methodology of CARS, including procedures for data collection and preliminary results. As of February 2023, 20 centers in the United States enrolled 1415 patients, including 1155 (82%) with ATTR and 260 (18%) with AL CA. Among those with ATTR, wild-type is the most common ATTR (71%), and most of the 305 patients with variant ATTR have the p.V142I mutation (68%). A quarter of the total population identifies as Black. More individuals with AL are female (39%) compared to those with ATTR (13%).

Conclusions: CARS will answer crucial clinical questions about CA natural history and permit comparison of different therapeutics not possible through current clinical trials. Future international collaboration will further strengthen the validity of observations of this increasingly recognized condition.

Keywords: Cardiac amyloidosis; light chain amyloidosis; registry; transthyretin amyloidosis.

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Conflict of interest statement

Disclosures JLG reports consulting fees from Pfizer, Eidos/BridgeBio, Alnlyam, Intellia, Sarepta, and AstraZeneca; Grant support from Pfizer 67656485, Eidos/BridgeBio, Texas Health Resources Clinical Scholarship, and NHLBI R01 HL160892-01A1. MSM reports consulting fees from AstraZeneca, Akcea, Intellia, and Novo-Nordisk; grant support from NIH R01HL139671, Alnylam, Pfizer, Eidos, Prothena, and Ionis. FLR reports consulting fees from Attralus and grant support from Pfizer, Alnylam, and Akcea/Ionis. ARP reports grant support from Pfizer. MGK reports consulting fees from and advisory board membership in Pfizer, Eidos, Alnylam; Grant support: Pfizer, Alnylam, Eidos, Ionis, and Moleculin Biotech and speaker's bureau with Alnylam. YB reports grant support from Pfizer. BMD reports consulting fees from Anylam and Eidos. NMF reports consulting/speaker's fees and bureau/honoraria from Pfizer, Akcea/Ionis, Sobi, Alnylam, Sanofi-Genzyme, AstraZeneca, and Takeda; grant support from Pfizer, Akcea/Ionis, Servier, Takeda, Novartis, and Eidos. HG reports consulting fees from Amgen, Bayer, Merck, Pfizer, and ExpertConnect and grant support from Roche Diagnostics, Jana Care, Ortho Clinical, Novartis, Pfizer, Alnylam, Akcea/Ionis, and Eidos/BridgeBio and stock ownership in Eko; research payments for clinical endpoint committees from Baim INstitute for Clinical Research for Abbott, Siemens and Beckman Coulter and from ACI Clinical for Abbott Laboratories. DJ reports consulting fees from Alexion, Alleviant, Cytokinetics, Novo-Nordisk, Pfizer, Renovacor/Rocket, and Tenaya. JM reports consultingfees from Myocardial Solutions, Abbott, Longer Life Foundation, Children's Discovery Institue, Pfizer, BridgeBio, and Altathera. SSM reports speaker's fees from and Bureau/Advisory Board member of Alnylam. RCM reports consulting fees/honoraria from Pfizer and AstraZeneca. FHS reports grant support from Alnylam and Ionis. BS reports consulting fees from Alnylam and BridgeBio/Eidos and speaker's bureau fees from Pfizer. MU reports grant support from Ionis; advisory board membership with Alnylam, Cytokinetics, BridgeBio/Eidos; speaker's bureau with Pfizer, Alnylam, Akcea. JKP reports grant support from Alexion, Alnylam, AstraZeneca, BridgeBio/Eidos, Novo-Nordisk, and Pfizer; advisory board/speaker's bureau membership with Alnylam, CareDx, Eidos, Ionis, Natera, and Pfizer. All other authors have no financial relationships to disclose.

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