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

Autor: Stern LK; Department of Cardiology, Cedars-Sinai Smidt Heart Institute, Los Angeles, CA, USA., Grodin JL; Division of Cardiology, Department of Internal Medicine, UT Southwestern Medical Center, Dallas, TX, USA., Maurer MS; Division of Cardiology, Columbia University Medical Center, New York-Presbyterian Hospital, New York, NY, USA., Ruberg FL; Section of Cardiovascular Medicine, Department of Medicine and Amyloidosis Center, Boston University Chobanian & Avedisian School of Medicine, Boston Medical Center, Boston, MA, USA., Patel AR; Division of Cardiology, The CardioVascular Center, Tufts Medical Center, Boston, MA, USA., Khouri MG; Division of Cardiology, Duke University Hospital, Durham, NC, USA., Roth LR; Division of Cardiology, Department of Internal Medicine, UT Southwestern Medical Center, Dallas, TX, USA., Aras MA; Division of Cardiology, Department of Medicine, Division of Cardiology, University of California, San Francisco, CA, USA., Bhardwaj A; Department of Advanced Cardiopulmonary Therapies and Transplantation, McGovern Medical School, University of Texas-Houston, Houston, TX, USA., Bhattacharya P; Division of Cardiology, Columbia University Medical Center, New York-Presbyterian Hospital, New York, NY, USA., Brailovsky Y; Division of Cardiology, Thomas Jefferson University Hospital, Philadelphia, PA, USA., Drachman BM; Division of Cardiology, Department of Medicine, Penn Presbyterian Medical Center, University of Pennsylvania Health System, Philadelphia, PA, USA., Ebong IA; Division of Cardiology, Department of Medicine, Division of Cardiology, University of California, Davis, CA, USA., Fine NM; Libin Cardiovascular Institute of Alberta, University of Calgary, Calgary, Alberta, Canada., Gaggin H; Division of Cardiology, Department of Medicine, Massachusetts General Hospital, Boston, MA, USA., Gopal D; Section of Cardiovascular Medicine, Department of Medicine and Amyloidosis Center, Boston University Chobanian & Avedisian School of Medicine, Boston Medical Center, Boston, MA, USA., Griffin J; Division of Cardiology, Columbia University Medical Center, New York-Presbyterian Hospital, New York, NY, USA; Division of Cardiology, Medical University of South Carolina, Charleston, SC, USA., Judge D; Division of Cardiology, Medical University of South Carolina, Charleston, SC, USA., Kim P; Division of Cardiology, Department of Medicine, University of California, San Diego, San Diego, CA, USA., Mitchell J; Cardiovascular Division, John T. Milliken Department of Internal Medicine, Cardio-Oncology Center of Excellence, Washington University in St Louis, St Louis, MO, USA., Mitter SS; Zena and Michael A. Wiener Cardiovascular Institute, Icahn School of Medicine at Mount Sinai, New York, NY, USA., Mohan RC; Division of Cardiology, Scripps Clinic, San Diego, CA, USA., Ramos H; Division of Cardiology, Medical University of South Carolina, Charleston, SC, USA., Reyentovich A; The Leon H. Charney Division of Cardiology, NYU Langone Health, New York, New York, USA., Sheikh FH; Infiltrative Cardiomyopathy and Advanced Heart Failure Programs, MedStar Heart and Vascular Institute, Georgetown University, Washington, DC, USA., Sperry B; Saint Luke's Mid America Heart Institute and the University of Kansas City-Missouri, Kansas City, Missouri, USA., Carter S; Division of Cardiovascular Medicine, University of Utah School of Medicine, Salt Lake City, UT, USA., Urey M; Division of Cardiology, Department of Medicine, University of California, San Diego, San Diego, CA, USA., Vaishnav J; Division of Cardiology, Department of Medicine, Johns Hopkins University, Baltimore, MD 21205, USA., Vest AR; Division of Cardiology, The CardioVascular Center, Tufts Medical Center, Boston, MA, USA., Kittleson MM; Department of Cardiology, Cedars-Sinai Smidt Heart Institute, Los Angeles, CA, USA., Patel JK; Department of Cardiology, Cedars-Sinai Smidt Heart Institute, Los Angeles, CA, USA. Electronic address: Jignesh.Patel@cshs.org.
Jazyk: angličtina
Zdroj: Journal of cardiac failure [J Card Fail] 2024 May; Vol. 30 (5), pp. 669-678. Date of Electronic Publication: 2023 Oct 30.
DOI: 10.1016/j.cardfail.2023.09.016
Abstrakt: 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.
Competing Interests: 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.
(Copyright © 2023. Published by Elsevier Inc.)
Databáze: MEDLINE
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