{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,7,30]],"date-time":"2026-07-30T03:48:44Z","timestamp":1785383324601,"version":"3.55.0"},"reference-count":7,"publisher":"American Medical Association (AMA)","issue":"3","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["JAMA Cardiol"],"published-print":{"date-parts":[[2023,3,1]]},"abstract":"<jats:sec><jats:title>Importance<\/jats:title><jats:p>Tafamidis reduced all-cause mortality and cardiovascular-related hospitalizations and minimized patient-reported health status deterioration at 30 months in patients with transthyretin (ATTR) amyloidosis. However, the clinical significance of health status changes remains unclear, particularly in patients with New York Heart Association (NYHA) class III symptoms who experienced more cardiovascular-related hospitalizations than those with NYHA class I-II symptoms.<\/jats:p><\/jats:sec><jats:sec><jats:title>Objective<\/jats:title><jats:p>To evaluate the health status of patients taking tafamidis with baseline NYHA class III symptoms.<\/jats:p><\/jats:sec><jats:sec><jats:title>Design, Setting, and Participants<\/jats:title><jats:p>This randomized clinical trial post hoc analysis evaluated data for patients with transthyretin (ATTR) cardiac amyloidosis and NYHA class I-III symptoms at baseline who were enrolled in ATTR-ACT, a placebo-controlled study of tafamidis held at 48 sites in 13 countries.<\/jats:p><\/jats:sec><jats:sec><jats:title>Interventions<\/jats:title><jats:p>Tafamidis meglumine, 80 mg or 20 mg (pooled cohort), vs placebo.<\/jats:p><\/jats:sec><jats:sec><jats:title>Main Outcomes and Measures<\/jats:title><jats:p>Established thresholds for clinical benefit on the Kansas City Cardiomyopathy Questionnaire Overall Summary (KCCQ-OS) were used to define response groups (very large decline to very large improvement); the proportion of patients in each group was calculated within each baseline NYHA class.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>Among 441 patients (264 tafamidis, 177 placebo), the mean (SD) age was 74.3 (7.0) years; 398 (90%) were male and 43 (10%) were female. Mean (SD) baseline KCCQ-OS scores were 67.3 (21.4) in the tafamidis group and 65.9 (21.7) in the placebo group (range: 0-100, with 100 indicating the best health). There was a significant shift toward better KCCQ-OS scores in patients receiving tafamidis (odds ratio for 10-point improvement 2.4; 95% CI, 1.6-3.4; <jats:italic>P<\/jats:italic>\u2009&amp;amp;lt;\u2009.001). More patients taking tafamidis were alive and not worse at all time points (37% vs 15% at month 30). These findings were similar in patients with NYHA class III symptoms. In patients with NYHA class III symptoms alive at 30 months, improvements in health status were more common (35% vs 10%) and declines were less common (38% vs 57%) with tafamidis vs placebo.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusions and Relevance<\/jats:title><jats:p>In ATTR-ACT, although patients with baseline NYHA class III symptoms had worse overall outcomes, treatment with tafamidis yielded better health status compared with placebo.<\/jats:p><\/jats:sec><jats:sec><jats:title>Trial Registration<\/jats:title><jats:p>ClinicalTrials.gov Identifier: <jats:ext-link xmlns:xlink=\"http:\/\/www.w3.org\/1999\/xlink\" ext-link-type=\"uri\" xlink:href=\"https:\/\/clinicaltrials.gov\/ct2\/show\/NCT01994889\">NCT01994889<\/jats:ext-link><\/jats:p><\/jats:sec>","DOI":"10.1001\/jamacardio.2022.5251","type":"journal-article","created":{"date-parts":[[2023,2,1]],"date-time":"2023-02-01T16:05:39Z","timestamp":1675267539000},"page":"275","source":"Crossref","is-referenced-by-count":23,"title":["Association of Tafamidis With Health Status in Patients With ATTR Cardiac Amyloidosis"],"prefix":"10.1001","volume":"8","author":[{"given":"Brett W.","family":"Sperry","sequence":"first","affiliation":[{"name":"Saint Luke\u2019s Mid America Heart Institute, Kansas City, Missouri"},{"name":"University of Missouri\u2013Kansas City, Kansas City"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mazen","family":"Hanna","sequence":"additional","affiliation":[{"name":"Cleveland Clinic, Cleveland, Ohio"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mathew S.","family":"Maurer","sequence":"additional","affiliation":[{"name":"Columbia University Irving Medical Center, New York, New York"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jose","family":"Nativi-Nicolau","sequence":"additional","affiliation":[{"name":"Mayo Clinic Jacksonville, Jacksonville, Florida"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lysbeth","family":"Floden","sequence":"additional","affiliation":[{"name":"Clinical Outcomes Solutions, Chicago, Illinois"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michelle","family":"Stewart","sequence":"additional","affiliation":[{"name":"Pfizer, New York, New York"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kathleen W.","family":"Wyrwich","sequence":"additional","affiliation":[{"name":"Pfizer, New York, New York"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alexandra I.","family":"Barsdorf","sequence":"additional","affiliation":[{"name":"Clinical Outcomes Solutions, Chicago, Illinois"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Heli","family":"Kapadia","sequence":"additional","affiliation":[{"name":"Clinical Outcomes Solutions, Chicago, Illinois"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"John A.","family":"Spertus","sequence":"additional","affiliation":[{"name":"Saint Luke\u2019s Mid America Heart Institute, Kansas City, Missouri"},{"name":"University of Missouri\u2013Kansas City, Kansas City"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"10","reference":[{"issue":"11","key":"hbr220012r1","doi-asserted-by":"publisher","first-page":"1765","DOI":"10.1016\/j.amjcard.2019.08.045","article-title":"Increasing rate of hospital admissions in patients with amyloidosis (from the National Inpatient Sample).","volume":"124","author":"Sperry","year":"2019","journal-title":"Am J Cardiol"},{"issue":"38","key":"hbr220012r2","doi-asserted-by":"publisher","first-page":"2585","DOI":"10.1093\/eurheartj\/ehv338","article-title":"Wild-type transthyretin amyloidosis as a cause of heart failure with preserved ejection fraction.","volume":"36","author":"Gonz\u00e1lez-L\u00f3pez","year":"2015","journal-title":"Eur Heart J"},{"issue":"11","key":"hbr220012r3","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"},{"key":"hbr220012r4","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":"6","key":"hbr220012r5","doi-asserted-by":"publisher","DOI":"10.1161\/CIRCHEARTFAILURE.116.003815","article-title":"Design and rationale of the phase 3 ATTR-ACT clinical trial (Tafamidis in Transthyretin Cardiomyopathy Clinical Trial).","volume":"10","author":"Maurer","year":"2017","journal-title":"Circ Heart Fail"},{"issue":"20","key":"hbr220012r6","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":"hbr220012r7","doi-asserted-by":"publisher","first-page":"529","DOI":"10.1093\/ehjqcco\/qcab031","article-title":"Health impact of tafamidis in transthyretin amyloid cardiomyopathy patients: an analysis from the Tafamidis in Transthyretin Cardiomyopathy Clinical Trial (ATTR-ACT) and the open-label long-term extension studies.","volume":"8","author":"Rozenbaum","year":"2022","journal-title":"Eur Heart J Qual Care Clin Outcomes"}],"container-title":["JAMA Cardiology"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/jamanetwork.com\/journals\/jamacardiology\/articlepdf\/2801004\/jamacardiology_sperry_2023_br_220012_1678219731.40482.pdf","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2023,3,8]],"date-time":"2023-03-08T16:10:08Z","timestamp":1678291808000},"score":1,"resource":{"primary":{"URL":"https:\/\/jamanetwork.com\/journals\/jamacardiology\/fullarticle\/2801004"}},"subtitle":["A Post Hoc Analysis of the ATTR-ACT Randomized Clinical Trial"],"short-title":[],"issued":{"date-parts":[[2023,3,1]]},"references-count":7,"journal-issue":{"issue":"3","published-print":{"date-parts":[[2023,3,1]]}},"URL":"https:\/\/doi.org\/10.1001\/jamacardio.2022.5251","relation":{},"ISSN":["2380-6583"],"issn-type":[{"value":"2380-6583","type":"print"}],"subject":[],"published":{"date-parts":[[2023,3,1]]}}}