Impact of Tafamidis on Health-Related Quality of Life in Patients With Transthyretin Amyloid Cardiomyopathy (from the Tafamidis in Transthyretin Cardiomyopathy Clinical Trial).

Abstract

In the Tafamidis in Transthyretin Cardiomyopathy Clinical Trial, tafamidis significantly reduced all-cause mortality and cardiovascular-related hospitalizations in patients with transthyretin amyloid cardiomyopathy (ATTR-CM). ATTR-CM is associated with a significant burden of disease; further analysis of patient-reported quality of life will provide additional data on the efficacy of tafamidis. In the Tafamidis in Transthyretin Cardiomyopathy Clinical Trial, 441 adult patients with ATTR-CM were randomized (2:1:2) to tafamidis 80 mg, tafamidis 20 mg, or placebo for 30 months, with pooled tafamidis (80 mg and 20 mg) compared with placebo. Change in Kansas City Cardiomyopathy Questionnaire Overall Summary (KCCQ-OS) domain scores, EQ-5D-3L scores, and patient global assessment, were prespecified exploratory end points. A greater proportion of patients improved KCCQ-OS score at month 30 with tafamidis (41.8%) versus placebo (21.4%). Tafamidis significantly reduced the decline in all 4 KCCQ-OS domains (p <0.0001 for all), and in EQ-5D-3L utility (0.09 [confidence interval 0.05 to 0.12]; p <0.0001) and EQ visual analog scale (9.11 [confidence interval 5.39 to 12.83]; p <0.0001) scores at month 30 versus placebo. A larger proportion of tafamidis-treated patients reported their patient global assessment improved at month 30 (42.3% vs 23.8% with placebo). In conclusion, tafamidis effectively reduced the decline in patient-reported outcomes, providing further insight into its efficacy in health-related quality of life in patients with ATTR-CM.

• Authors: Mazen Hanna, Thibaud Damy, Martha Grogan, Michelle Stewart, Balarama Gundapaneni, Terrell A Patterson, Jeffrey H Schwartz, Marla B Sultan, Mathew S Maurer
• Journal: The American journal of cardiology (Am J Cardiol) Vol. 141 Pg. 98-105 (02 15 2021) ISSN: 1879-1913 [Electronic] United States
• PMID: 33220323 (Publication Type: Journal Article, Randomized Controlled Trial, Research Support, Non-U.S. Gov't)
• Copyright: Copyright © 2020. Published by Elsevier Inc.

Chemical References

• Benzoxazoles
• tafamidis

Topics

• Activities of Daily Living
• Aged
• Aged, 80 and over
• Amyloid Neuropathies, Familial (drug therapy, physiopathology)
• Benzoxazoles (therapeutic use)
• Cardiomyopathies (drug therapy, physiopathology)
• Cost of Illness
• Female
• Humans
• Male
• Patient Reported Outcome Measures
• Quality of Life
• Self Efficacy
• Social Participation