{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,11]],"date-time":"2026-10-11T14:26:17Z","timestamp":1791728777928,"version":"4.3.4"},"reference-count":30,"publisher":"American Medical Association (AMA)","issue":"9","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["JAMA Cardiol"],"published-print":{"date-parts":[[2026,9,1]]},"abstract":"<jats:sec>\n                    <jats:title>Importance<\/jats:title>\n                    <jats:p>In patients with transthyretin amyloid cardiomyopathy (ATTR-CM), acoramidis achieves near-complete (\u226590%) transthyretin stabilization and reduces mortality and cardiovascular-related hospitalizations; however, its effect on patient-reported health status has not been comprehensively described.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Objective<\/jats:title>\n                    <jats:p>To evaluate the effect of acoramidis on heart failure (HF)\u2013related health status as assessed by the Kansas City Cardiomyopathy Questionnaire Overall Summary score (KCCQ-OS) in patients with ATTR-CM.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Design, Setting, and Participants<\/jats:title>\n                    <jats:p>ATTRibute-CM was a phase 3, multicenter, international, placebo-controlled randomized clinical trial conducted from April 2019 through May 2023. Adults with ATTR-CM were eligible for inclusion. Data were analyzed from July 2023 through November 2023.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Interventions<\/jats:title>\n                    <jats:p>Acoramidis hydrochloride (800 mg) or placebo twice daily for 30 months.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Main Outcomes and Measures<\/jats:title>\n                    <jats:p>The prespecified secondary outcome was least-squares mean (LSM) difference in KCCQ-OS over 30 months, analyzed using a mixed-effects model for repeated measures. Post hoc analysis at month 30 included being \u201calive and not worse\u201d (KCCQ-OS &amp;amp;lt;5-point decrease from baseline), \u201calive and well\u201d (KCCQ-OS &amp;amp;gt;60 and &amp;amp;lt;10-point decrease from baseline), and \u201dalive and better\u201d (KCCQ-OS &amp;amp;gt;5-point increase from baseline).<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>\n                      Among 632 adults with ATTR-CM enrolled, 611 were included in the modified intention-to-treat population. Overall mean (SD) age was 77.2 (6.6) years, and 56 participants (9.2%) were female. Baseline mean (SD) KCCQ-OSs were 71.7 (19.4) and 70.5 (20.7) in the acoramidis (n\u2009=\u2009409) and placebo (n\u2009=\u2009202) groups, respectively. At month 30, a statistically significant, clinically meaningful treatment benefit was observed for acoramidis vs placebo (LSM difference, 9.9; 95% CI, 6.0-13.9;\n                      <jats:italic>P<\/jats:italic>\n                      \u2009&amp;amp;lt;\u2009.001). At month 30 (acoramidis: 367; placebo: 188), 171 acoramidis recipients (47%) were \u201calive and not worse\u201d vs 56 placebo recipients (30%) (odds ratio, 2.1; 95% CI, 1.4-3.1;\n                      <jats:italic>P<\/jats:italic>\n                      \u2009&amp;amp;lt;\u2009.001; number needed to treat [NNT]\u2009=\u20096). More acoramidis recipients (168 [46%]) were \u201calive and well\u201d vs placebo (59 [31%]) (odds ratio, 1.9; 95% CI, 1.3-2.8;\n                      <jats:italic>P<\/jats:italic>\n                      \u2009&amp;amp;lt;\u2009.001; NNT\u2009=\u20097). Similarly, 93 acoramidis recipients (25%) were \u201calive and better\u201d vs 26 placebo recipients (14%) (odds ratio, 2.1; 95% CI, 1.3-3.4;\n                      <jats:italic>P<\/jats:italic>\n                      \u2009=\u2009.002; NNT\u2009=\u20099).\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusions and Relevance<\/jats:title>\n                    <jats:p>In this secondary analysis of the ATTRibute-CM randomized clinical trial, in patients with ATTR-CM, acoramidis significantly attenuated the decline in HF-related health status compared with placebo. These results suggest meaningful patient-centered benefits and clinically relevant modification of disease trajectory with acoramidis.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Trial Registration<\/jats:title>\n                    <jats:p>\n                      ClinicalTrials.gov Identifier:\n                      <jats:ext-link xmlns:xlink=\"http:\/\/www.w3.org\/1999\/xlink\" ext-link-type=\"uri\" xlink:href=\"https:\/\/clinicaltrials.gov\/study\/NCT03860935?cond=NCT03860935&amp;amp;amp;viewType=Card&amp;amp;amp;rank=1\">NCT03860935<\/jats:ext-link>\n                    <\/jats:p>\n                  <\/jats:sec>","DOI":"10.1001\/jamacardio.2026.2413","type":"journal-article","created":{"date-parts":[[2026,7,29]],"date-time":"2026-07-29T15:30:31Z","timestamp":1785339031000},"page":"857","source":"Crossref","is-referenced-by-count":1,"title":["Effect of Acoramidis on Heart Failure\u2013Related Health Status"],"prefix":"10.1001","volume":"11","author":[{"suffix":"IV","given":"Charles F.","family":"Sherrod","sequence":"first","affiliation":[{"name":"Healthcare Institute for Innovations in Quality, University of Missouri\u2013Kansas City"},{"name":"Saint Luke\u2019s Mid America Heart Institute, Kansas City, Missouri"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marianna","family":"Fontana","sequence":"additional","affiliation":[{"name":"National Amyloidosis Centre, Royal Free Hospital, University College London, London, United Kingdom"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Julian D.","family":"Gillmore","sequence":"additional","affiliation":[{"name":"National Amyloidosis Centre, Royal Free Hospital, University College London, London, United Kingdom"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Efstathios","family":"Kastritis","sequence":"additional","affiliation":[{"name":"Department of Clinical Therapeutics, National and Kapodistrian University of Athens, Athens, Greece"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Joshua D.","family":"Mitchell","sequence":"additional","affiliation":[{"name":"Cardiovascular Division, Washington University School of Medicine, St Louis, Missouri"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jan","family":"Krejci","sequence":"additional","affiliation":[{"name":"Department of Cardiovascular Diseases, St Anne\u2019s University Hospital and Masaryk University Brno, Brno, Czech Republic"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"James M.","family":"Tauras","sequence":"additional","affiliation":[{"name":"Montefiore Medical Center, Bronx, New York"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Peter","family":"van der Meer","sequence":"additional","affiliation":[{"name":"Department of Cardiology, University Medical Center Groningen, University of Groningen, Groningen, the Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Chris","family":"Chen","sequence":"additional","affiliation":[{"name":"BridgeBio Pharma, San Francisco, California"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Heather","family":"Falvey","sequence":"additional","affiliation":[{"name":"BridgeBio Pharma, San Francisco, California"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Suresh","family":"Siddhanti","sequence":"additional","affiliation":[{"name":"BridgeBio Pharma, San Francisco, California"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jean-Fran\u00e7ois","family":"Tamby","sequence":"additional","affiliation":[{"name":"BridgeBio Pharma, San Francisco, California"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jonathan C.","family":"Fox","sequence":"additional","affiliation":[{"name":"BridgeBio Pharma, San Francisco, California"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Francesco","family":"Cappelli","sequence":"additional","affiliation":[{"name":"Tuscan Regional Amyloidosis Centre, Careggi University Hospital, Florence, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ahmad","family":"Masri","sequence":"additional","affiliation":[{"name":"Division of Cardiology, Oregon Health and Science University, Portland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Pablo","family":"Garcia-Pavia","sequence":"additional","affiliation":[{"name":"Department of Cardiology, Hospital Universitario Puerta de Hierro Majadahonda, Instituto de Investigaci\u00f3n Sanitaria Puerta de Hierro-Segovia de Arana (IDIPHIM), Centro de Investigaci\u00f3n Biom\u00e9dica en Red de Enfermedades Cardiovasculares (CIBERCV), and Centro Nacional de Investigaciones Cardiovasculares, Madrid, Spain"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mazen","family":"Hanna","sequence":"additional","affiliation":[{"name":"Cleveland Clinic, Cleveland, Ohio"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Brett W.","family":"Sperry","sequence":"additional","affiliation":[{"name":"Healthcare Institute for Innovations in Quality, University of Missouri\u2013Kansas City"},{"name":"Saint Luke\u2019s Mid America Heart Institute, Kansas City, Missouri"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"10","reference":[{"issue":"1","key":"hoi260035r1","doi-asserted-by":"publisher","first-page":"59","DOI":"10.5334\/gh.1262","article-title":"World Heart Federation consensus on transthyretin amyloidosis cardiomyopathy (ATTR-CM).","volume":"18","author":"Brito","year":"2023","journal-title":"Glob Heart"},{"issue":"4","key":"hoi260035r2","doi-asserted-by":"publisher","first-page":"512","DOI":"10.1002\/ejhf.2140","article-title":"Diagnosis and treatment of cardiac amyloidosis. a position statement of the European Society of Cardiology Working Group on Myocardial and Pericardial Diseases.","volume":"23","author":"Garcia-Pavia","year":"2021","journal-title":"Eur J Heart Fail"},{"issue":"1","key":"hoi260035r3","doi-asserted-by":"publisher","first-page":"e7","DOI":"10.1161\/CIR.0000000000000792","article-title":"Cardiac amyloidosis: evolving diagnosis and management: a scientific statement from the American Heart Association.","volume":"142","author":"Kittleson","year":"2020","journal-title":"Circulation"},{"issue":"1","key":"hoi260035r4","doi-asserted-by":"publisher","first-page":"16","DOI":"10.1161\/CIRCULATIONAHA.118.038169","article-title":"Natural history, quality of life, and outcome in cardiac transthyretin amyloidosis.","volume":"140","author":"Lane","year":"2019","journal-title":"Circulation"},{"issue":"3","key":"hoi260035r5","doi-asserted-by":"publisher","first-page":"1871","DOI":"10.1002\/ehf2.14350","article-title":"Health-related quality of life among transthyretin amyloid cardiomyopathy patients.","volume":"10","author":"Eldhagen","year":"2023","journal-title":"ESC Heart Fail"},{"issue":"20","key":"hoi260035r6","doi-asserted-by":"publisher","first-page":"2379","DOI":"10.1016\/j.jacc.2020.09.542","article-title":"Interpreting the Kansas City Cardiomyopathy Questionnaire in clinical trials and clinical care: JACC State-of-the-Art Review.","volume":"76","author":"Spertus","year":"2020","journal-title":"J Am Coll Cardiol"},{"issue":"5","key":"hoi260035r7","doi-asserted-by":"publisher","first-page":"1245","DOI":"10.1016\/S0735-1097(00)00531-3","article-title":"Development and evaluation of the Kansas City Cardiomyopathy Questionnaire: a new health status measure for heart failure.","volume":"35","author":"Green","year":"2000","journal-title":"J Am Coll Cardiol"},{"issue":"2","key":"hoi260035r9","doi-asserted-by":"publisher","first-page":"132","DOI":"10.1056\/NEJMoa2305434","article-title":"Efficacy and safety of acoramidis in transthyretin amyloid cardiomyopathy.","volume":"390","author":"Gillmore","year":"2024","journal-title":"N Engl J Med"},{"issue":"11","key":"hoi260035r10","doi-asserted-by":"publisher","first-page":"1007","DOI":"10.1056\/NEJMoa1805689","article-title":"Tafamidis treatment for patients with transthyretin amyloid cardiomyopathy.","volume":"379","author":"Maurer","year":"2018","journal-title":"N Engl J Med"},{"issue":"1","key":"hoi260035r11","doi-asserted-by":"publisher","first-page":"33","DOI":"10.1056\/NEJMoa2409134","article-title":"Vutrisiran in patients with transthyretin amyloidosis with cardiomyopathy.","volume":"392","author":"Fontana","year":"2025","journal-title":"N Engl J Med"},{"issue":"17","key":"hoi260035r12","doi-asserted-by":"publisher","first-page":"7862","DOI":"10.1021\/acs.jmedchem.8b00817","article-title":"Enthalpy-driven stabilization of transthyretin by AG10 mimics a naturally occurring genetic variant that protects from transthyretin amyloidosis.","volume":"61","author":"Miller","year":"2018","journal-title":"J Med Chem"},{"issue":"10","key":"hoi260035r13","doi-asserted-by":"publisher","first-page":"1003","DOI":"10.1016\/j.jacc.2024.11.042","article-title":"Efficacy of acoramidis on all-cause mortality and cardiovascular hospitalization in transthyretin amyloid cardiomyopathy.","volume":"85","author":"Judge","year":"2025","journal-title":"J Am Coll Cardiol"},{"issue":"20","key":"hoi260035r14","doi-asserted-by":"publisher","first-page":"2191","DOI":"10.1001\/jama.2013.281053","article-title":"World Medical Association Declaration of Helsinki: ethical principles for medical research involving human subjects.","volume":"310","author":"World Medical Association","year":"2013","journal-title":"JAMA"},{"issue":"6","key":"hoi260035r15","doi-asserted-by":"publisher","first-page":"999","DOI":"10.1002\/ejhf.1810","article-title":"Minimal clinically important difference in quality of life scores for patients with heart failure and reduced ejection fraction.","volume":"22","author":"Butler","year":"2020","journal-title":"Eur J Heart Fail"},{"issue":"1","key":"hoi260035r16","doi-asserted-by":"publisher","first-page":"1","DOI":"10.1016\/j.jacc.2023.10.008","article-title":"Health status after transcatheter tricuspid-valve repair in patients with severe tricuspid regurgitation.","volume":"83","author":"Arnold","year":"2024","journal-title":"J Am Coll Cardiol"},{"issue":"19","key":"hoi260035r17","doi-asserted-by":"publisher","first-page":"1773","DOI":"10.1016\/j.jacc.2024.08.014","article-title":"Effect of aficamten on health status outcomes in obstructive hypertrophic cardiomyopathy: results from SEQUOIA-HCM.","volume":"84","author":"Sherrod","year":"2024","journal-title":"J Am Coll Cardiol"},{"issue":"25","key":"hoi260035r18","doi-asserted-by":"publisher","first-page":"2682","DOI":"10.1161\/CIRCULATIONAHA.113.007477","article-title":"Predictors of poor outcomes after transcatheter aortic valve replacement: results from the PARTNER (Placement of Aortic Transcatheter Valve) trial.","volume":"129","author":"Arnold","year":"2014","journal-title":"Circulation"},{"issue":"17","key":"hoi260035r19","doi-asserted-by":"publisher","first-page":"2123","DOI":"10.1016\/j.jacc.2019.02.010","article-title":"Health status after transcatheter mitral-valve repair in heart failure and secondary mitral regurgitation: COAPT trial.","volume":"73","author":"Arnold","year":"2019","journal-title":"J Am Coll Cardiol"},{"issue":"17","key":"hoi260035r20","doi-asserted-by":"publisher","first-page":"1868","DOI":"10.1016\/j.jacc.2016.07.762","article-title":"Prediction of poor outcome after transcatheter aortic valve replacement.","volume":"68","author":"Arnold","year":"2016","journal-title":"J Am Coll Cardiol"},{"issue":"10","key":"hoi260035r21","doi-asserted-by":"publisher","DOI":"10.1161\/CIRCOUTCOMES.121.008072","article-title":"Risk adjustment model for preserved health status in patients with heart failure and reduced ejection fraction: the CHAMP-HF Registry.","volume":"14","author":"Tran","year":"2021","journal-title":"Circ Cardiovasc Qual Outcomes"},{"issue":"1","key":"hoi260035r22","doi-asserted-by":"publisher","first-page":"32","DOI":"10.1001\/jamacardio.2024.4470","article-title":"Interpreting population mean treatment effects in the Kansas City Cardiomyopathy Questionnaire: a patient-level meta-analysis.","volume":"10","author":"Abdel Jawad","year":"2025","journal-title":"JAMA Cardiol"},{"key":"hoi260035r23","doi-asserted-by":"publisher","first-page":"98","DOI":"10.1016\/j.amjcard.2020.10.066","article-title":"Impact of tafamidis on health-related quality of life in patients with transthyretin amyloid cardiomyopathy (from the Tafamidis in Transthyretin Cardiomyopathy Clinical Trial).","volume":"141","author":"Hanna","year":"2021","journal-title":"Am J Cardiol"},{"issue":"3","key":"hoi260035r24","doi-asserted-by":"publisher","first-page":"275","DOI":"10.1001\/jamacardio.2022.5251","article-title":"Association of tafamidis with health status in patients with ATTR cardiac amyloidosis: a post hoc analysis of the ATTR-ACT randomized clinical trial.","volume":"8","author":"Sperry","year":"2023","journal-title":"JAMA Cardiol"},{"issue":"20","key":"hoi260035r25","doi-asserted-by":"publisher","first-page":"1943","DOI":"10.1016\/j.jacc.2025.03.454","article-title":"Impact of vutrisiran on functional capacity and quality of life in transthyretin amyloidosis with cardiomyopathy.","volume":"85","author":"Sheikh","year":"2025","journal-title":"J Am Coll Cardiol"},{"issue":"5","key":"hoi260035r26","doi-asserted-by":"publisher","first-page":"591","DOI":"10.1161\/CIRCOUTCOMES.113.000354","article-title":"How to define a poor outcome after transcatheter aortic valve replacement: conceptual framework and empirical observations from the Placement of Aortic Transcatheter Valve (PARTNER) trial.","volume":"6","author":"Arnold","year":"2013","journal-title":"Circ Cardiovasc Qual Outcomes"},{"issue":"6","key":"hoi260035r27","doi-asserted-by":"publisher","DOI":"10.1161\/CIRCOUTCOMES.118.005420","article-title":"Integrating quality of life and survival outcomes in cardiovascular clinical trials.","volume":"12","author":"Spertus","year":"2019","journal-title":"Circ Cardiovasc Qual Outcomes"},{"issue":"9","key":"hoi260035r28","doi-asserted-by":"publisher","first-page":"2825","DOI":"10.3390\/jcm9092825","article-title":"Gender differences in health-related quality of life in patients with systolic heart failure: results of the VIDA multicenter study.","volume":"9","author":"Garay","year":"2020","journal-title":"J Clin Med"},{"issue":"4","key":"hoi260035r29","doi-asserted-by":"publisher","first-page":"567","DOI":"10.1002\/ejhf.2154","article-title":"Quality of life in men and women with heart failure: association with outcome, and comparison between the Kansas City Cardiomyopathy Questionnaire and the EuroQol 5 Dimensions Questionnaire.","volume":"23","author":"Ravera","year":"2021","journal-title":"Eur J Heart Fail"},{"issue":"5","key":"hoi260035r30","doi-asserted-by":"publisher","first-page":"3875","DOI":"10.1002\/ehf2.13541","article-title":"Natural history and progression of transthyretin amyloid cardiomyopathy: insights from ATTR-ACT.","volume":"8","author":"Nativi-Nicolau","year":"2021","journal-title":"ESC Heart Fail"},{"key":"hoi260035r8","unstructured":"U.S. Food and Drug Administration. Qualification of the Kansas City Cardiomyopathy Questionnaire Clinical Summary Score and its component scores: a patient-reported outcome instrument for use in clinical investigations in heart failure. April 9, 2020. Accessed February 5, 2026. https:\/\/www.fda.gov\/media\/136862\/download"}],"container-title":["JAMA Cardiology"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/jamanetwork.com\/journals\/jamacardiology\/articlepdf\/2851852\/jamacardiology_sherrod_2026_oi_260035_1788536069.76529.pdf","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,9,9]],"date-time":"2026-09-09T15:00:39Z","timestamp":1788966039000},"score":1,"resource":{"primary":{"URL":"https:\/\/jamanetwork.com\/journals\/jamacardiology\/fullarticle\/2851852"}},"subtitle":["A Secondary Analysis of the ATTRibute-CM Randomized Clinical Trial"],"short-title":[],"issued":{"date-parts":[[2026,9,1]]},"references-count":30,"journal-issue":{"issue":"9","published-print":{"date-parts":[[2026,9,1]]}},"URL":"https:\/\/doi.org\/10.1001\/jamacardio.2026.2413","relation":{},"ISSN":["2380-6583"],"issn-type":[{"value":"2380-6583","type":"print"}],"subject":[],"published":{"date-parts":[[2026,9,1]]}}}