Grogan, MarthaDavis, Margot K.Crespo-Leiro, María GenerosaSultan, Marla B.Gundapaneni, BalaramaHanna, MazenStedile Angeli, Franca2024-06-102024-06-102024-03-04Grogan M, Davis MK, Crespo-Leiro MG, Sultan MB, Gundapaneni B, Stedile Angeli F, Hanna M. Effect of long-term tafamidis treatment on health-related quality of life in patients with transthyretin amyloid cardiomyopathy. Eur J Heart Fail. 2024 Mar;26(3):612-615.1388-9842http://hdl.handle.net/2183/36848Short report[Abstract] Aims: To evaluate the effect of long-term tafamidis treatment on health-related quality of life (HRQoL) in patients with transthyretin amyloid cardiomyopathy (ATTR-CM) enrolled in the Tafamidis in Transthyretin Cardiomyopathy Clinical Trial (ATTR-ACT) and long-term extension (LTE) study. Methods and results: We examined change from baseline in Kansas City Cardiomyopathy Questionnaire overall summary (KCCQ-OS) and clinical summary (KCCQ-CS) scores in patients who received tafamidis meglumine 80 mg for 30 months in ATTR-ACT and tafamidis (meglumine 80 mg or bioequivalent free acid 61 mg) for 30 months in the LTE study, and in patients who received placebo for 30 months in ATTR-ACT and tafamidis for 30 months in the LTE study. In ATTR-ACT, 176 and 177 patients were randomized to tafamidis 80 mg and placebo, respectively. Patients who continuously received tafamidis had a 6- to 7-point reduction in least squares (LS) mean (standard error) KCCQ-OS and KCCQ-CS scores at month 30 (-6.25 [1.53] and -7.48 [1.39]), with little or no further decline over the next 30 months (-5.92 [1.77] and -9.21 [1.88] at month 60). Patients who received placebo in ATTR-ACT had a 20-point reduction in LS mean KCCQ-OS and KCCQ-CS scores at month 30 (-19.60 [1.94] and -19.90 [2.01]), but the decline slowed after initiating tafamidis (-24.70 [3.04] and -25.30 [3.36] at month 60). Conclusion: Tafamidis reduced HRQoL decline in patients with ATTR-CM. Patients continuously treated with tafamidis for 60 months demonstrated stabilized HRQoL. In patients who initially received placebo in ATTR-ACT, tafamidis reduced the decline in HRQoL during the LTE study.spaCreative Commons Attribution-NonCommercial-NoDerivs 4.0 International License (CC-BY-NC-ND 4.0)http://creativecommons.org/licenses/by-nc-nd/4.0/Health‐related quality of lifeKansas City Cardiomyopathy QuestionnaireTafamidisTransthyretin amyloid cardiomyopathyjournal articleopen access10.1002/ejhf.3190