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Tafamidis has been shown to provide increased life expectancy and quality of life in patients with ATTR-CM. This study further quantified these findings.

The Tafamidis in Transthyretin Cardiomyopathy Clinical Trial (ATTR-ACT) successfully illustrated that tafamidis reduced mortality and cardiovascular-related hospitalizations in patients with transthyretin amyloid cardiomyopathy (ATTR-CM). A recent study, published in the European Heart Journal – Quality of Care and Clinical Outcomes, used a multi-state cohort Markov model to simulate the disease course of ATTR-CM throughout a lifetime, in order to gain a better understanding of the impact that tafamidis can have on survival and quality-adjusted life-years for patients with ATTR-CM.

In order to extrapolate survival rates, survival curves were fitted by treatment arm and New York Heart Association Class I, II, and III cohorts using the individual patient-level data from both the ATTR-ACD and the long-term extension study. The predicted mean survival for the total population was 6.73 years for tafamidis and 2.85 years for the standard of care. This resulted in an incremental mean survival of 3.88 years. Of the 6.73 life-years, patients on tafamidis spend an average of 4.82 years in NYHA classes I and II, while patients on the standard of care spend an average of 1.6 life-years in these classes. 

The combination of longer survival time spent in lower NYHA classes produces an overall quality-adjusted life-year increase of 5.39 for tafamidis, and 2.11 for the standard of care. The total incremental quality-adjusted life-years are 3.29 in favor of tafamidis. The researchers note that although this number seems small at first glance, tafamidis is expected to more than double the life expectancy and quality-adjusted life-years of patients with ATTR-CM, according to the disease simulation model. Longer-term follow-up data is expected to inform these findings and provide more context.

 

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Rozenbaum, M. H., Garcia, A., Grima, D., Tran, D., Bhambri, R., Stewart, M., Li, B., Heeg, B., Postma, M., & Masri, A. (2022). 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. Eur Heart J Qual Care Clin Outcomes, 8(5), 529-538. https://doi.org/10.1093/ehjqcco/qcab031