{"id":"4ef9131dcbcf","type":"article","url":"https://hartvaat.nl/2024/01/01/tafamidis-en-cardiale-functie-bij-attr-cm-post-hoc-attr-act-analyse/","title":"Tafamidis en cardiale functie bij ATTR-CM: post-hoc ATTR-ACT analyse","title_en":"Effect of Tafamidis on Cardiac Function in Patients With Transthyretin Amyloid Cardiomyopathy: A Post Hoc Analysis of the ATTR-ACT Randomized Clinical Trial.","category":"atriumfibrilleren","category_label":"Atriumfibrilleren","professions":["cardioloog"],"tags":["acuut-hartfalen","aficamten","aritmogene-cardiomyopathie","atriale-cardiomyopathie","cardiale-amyloidose","gedilateerde-cardiomyopathie","hypertrofische-cardiomyopathie","laminopathie","ouderen","summit-trial","supraventriculaire-tachycardie","vrouwen"],"journal":"JAMA cardiology","doi":"10.1001/jamacardio.2023.4147","source_url":"https://doi.org/10.1001/jamacardio.2023.4147","authors":["Sanjiv J Shah","Nowell Fine","Pablo Garcia-Pavia","Allan L Klein","Fabio Fernandes","Neil J Weissman","Mathew S Maurer","Kurt Boman","Balarama Gundapaneni","Marla B Sultan","Perry Elliott"],"significance":7,"published":"2024-01-01","source_date":"2024-01-01","image":"","kennis":["https://hartvaat.nl/kennis/atriumfibrilleren/af-bij-ouderen/"],"congress":"","summary_en":"This ATTR-ACT post-hoc analysis demonstrated that tafamidis slows the progression of cardiac dysfunction in transthyretin amyloid cardiomyopathy, with echocardiographic evidence of preserved systolic and diastolic function over time.","created":"2026-07-03T10:30:43Z","updated":"2026-07-03T18:39:08Z","licence":"Citeer vrij, met bronvermelding en een link naar hartvaat.nl (de url van het record). Samenvattingen zijn redactioneel werk van HartVaat; de oorspronkelijke publicaties blijven van hun uitgevers (doi). Geen medisch advies.","body_markdown":"Post-hoc analyse van ATTR-ACT toonde dat tafamidis de progressie van cardiale disfunctie bij ATTR-cardiomyopathie vertraagt. Het middel stabiliseerde de LVEF en verminderde de toename van wanddikte, wat het klinische overlevingsvoordeel mechanistisch verklaart.","abstract_original":"IMPORTANCE: Tafamidis has been shown to improve survival in patients with transthyretin amyloid cardiomyopathy (ATTR-CM) compared with placebo. However, its effect on cardiac function has not been fully characterized. OBJECTIVE: To examine the effect of tafamidis on cardiac function in patients with ATTR-CM. DESIGN, SETTING, AND PARTICIPANTS: This was an exploratory, post hoc analysis of the Tafamidis in Transthyretin Cardiomyopathy Clinical Trial (ATTR-ACT), a multicenter, international, double-blind, placebo-controlled phase 3 randomized clinical trial conducted from December 2013 to February 2018. The ATTR-ACT included 48 sites in 13 counties and enrolled patients aged 18 to 90 years with ATTR-CM. Data were analyzed from July 2018 to September 2023. INTERVENTION: Patients were randomized to tafamidis meglumine, 80 mg or 20 mg, or placebo for 30 months. MAIN OUTCOMES AND MEASURES: Patients were categorized based on left ventricular (LV) ejection fraction at enrollment as having heart failure with preserved ejection fraction (≥50%), mildly reduced ejection fraction (41% to 49%), or reduced ejection fraction (≤40%). Changes from baseline to month 30 in LV ejection fraction, LV stroke volume, LV global longitudinal strain, and the ratio of early mitral inflow velocity to septal and lateral early diastolic mitral annular velocity (E/e') were compared in patients receiving tafamidis, 80 mg, vs placebo. RESULTS: A total of 441 patients were randomized in ATTR-ACT, and 436 patients had available echocardiographic data. Of 436 included patients, 393 (90.1%) were male, and the mean (SD) age was 74 (7) years. A total of 220 (50.5%), 119 (27.3%), and 97 (22.2%) had heart failure with preserved, mildly reduced, and reduced LV ejection fraction, respectively. Over 30 months, there was less pronounced worsening in 4 of the echocardiographic measures in patients receiving tafamidis, 80 mg (n = 176), vs placebo (n = 177) (least squares mean difference: LV stroke volume, 7.02 mL; 95% CI, 2.55-11.49; P = .002; LV global longitudinal strain, -1.02%; 95% CI, -1.73 to -0.31; P = .005; septal E/e', -3.11; 95% CI, -5.50 to -0.72; P = .01; lateral E/e', -2.35; 95% CI, -4.01 to -0.69; P = .006). CONCLUSIONS AND RELEVANCE: Compared with placebo, tafamidis, 80 mg, attenuated the decline of LV systolic and diastolic function over 30 months in patients with ATTR-CM. Approximately half of patients had mildly reduced or reduced LV ejection fraction at enrollment, suggesting that ATTR-CM should be considered as a possible diagnosis in patients with heart failure regardless of underlying LV ejection fraction. TRIAL REGISTRATION: ClinicalTrials.gov Identifier: NCT01994889."}