Effect of long‐term tafamidis treatment on health‐related quality of life in patients with transthyretin amyloid cardiomyopathy.

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. [ABSTRACT FROM AUTHOR]