Cardiovascular Morbidity and Mortality Among ATTR-CM Patients Treated with Tafamidis in the US
Author(s)
Ankur Patel, MS1, Teresa Kauf, PhD1, Dave Danese, MBA1, John Berk, MD2;
1Alnylam Pharmaceuticals Inc, Cambridge, MA, USA, 2Boston University, Boston, MA, USA
1Alnylam Pharmaceuticals Inc, Cambridge, MA, USA, 2Boston University, Boston, MA, USA
Presentation Documents
OBJECTIVES: There continues to be a significant unmet therapeutic need in transthyretin-mediated amyloidosis with cardiomyopathy (ATTR-CM) despite current disease management strategies. Characterization of cardiovascular (CV) morbidity and mortality in patients receiving tafamidis in the real-world setting can provide valuable insight into the current unmet need in ATTR-CM. The objective of this analysis is to describe CV-related morbidity and mortality in ATTR-CM patients treated with tafamidis.
METHODS: A retrospective analysis of US insurance claims from January 2017 to May 2024 was conducted. ATTR-CM patients identified by ICD10-CM codes were included in the analysis if they received tafamidis on or after ATTR-CM diagnosis and had 6 months continuous enrollment before tafamidis initiation. Patients were followed from tafamidis initiation (index date) to the end of continuous enrollment in the health plan, death, or study end date, whichever came first. Demographic and clinical characteristics were reported for the baseline period. CV-related morbidity and mortality events in the follow-up period were reported as absolute frequencies and as event incidence rates per 1000 person-years.
RESULTS: Of 2,710 ATTR-CM patients included in the analysis, mean (standard deviation; SD) age was 78.1 (8.5), 79.7% were male, 83.1% had Medicare insurance; and 872 (32.2%) had evidence of polyneuropathy. 24.1% of patients experienced at least one CV event in the baseline period. Over an average (SD) follow-up period of 485.3 (433.4) days following tafamidis initiation, 1,060 (39.1%) of ATTR-CM patients receiving tafamidis experienced at least one CV event: 936 (34.5%) had CV-related hospitalization, 235 (8.7%) had myocardial infarction, 50 (1.9%) had stroke, and 329 (12.1) % died. Overall, the incidence of CV events was 405.8 and mortality rate of 91.3 per 1000 person-years.
CONCLUSIONS: Patients treated with tafamidis for ATTR-CM experience substantial CV-related morbidity and mortality. There remains an unmet need for effective treatments that reduce the burden of ATTR-CM.
METHODS: A retrospective analysis of US insurance claims from January 2017 to May 2024 was conducted. ATTR-CM patients identified by ICD10-CM codes were included in the analysis if they received tafamidis on or after ATTR-CM diagnosis and had 6 months continuous enrollment before tafamidis initiation. Patients were followed from tafamidis initiation (index date) to the end of continuous enrollment in the health plan, death, or study end date, whichever came first. Demographic and clinical characteristics were reported for the baseline period. CV-related morbidity and mortality events in the follow-up period were reported as absolute frequencies and as event incidence rates per 1000 person-years.
RESULTS: Of 2,710 ATTR-CM patients included in the analysis, mean (standard deviation; SD) age was 78.1 (8.5), 79.7% were male, 83.1% had Medicare insurance; and 872 (32.2%) had evidence of polyneuropathy. 24.1% of patients experienced at least one CV event in the baseline period. Over an average (SD) follow-up period of 485.3 (433.4) days following tafamidis initiation, 1,060 (39.1%) of ATTR-CM patients receiving tafamidis experienced at least one CV event: 936 (34.5%) had CV-related hospitalization, 235 (8.7%) had myocardial infarction, 50 (1.9%) had stroke, and 329 (12.1) % died. Overall, the incidence of CV events was 405.8 and mortality rate of 91.3 per 1000 person-years.
CONCLUSIONS: Patients treated with tafamidis for ATTR-CM experience substantial CV-related morbidity and mortality. There remains an unmet need for effective treatments that reduce the burden of ATTR-CM.
Conference/Value in Health Info
2025-05, ISPOR 2025, Montréal, Quebec, CA
Value in Health, Volume 28, Issue S1
Code
RWD106
Topic
Real World Data & Information Systems
Disease
SDC: Cardiovascular Disorders (including MI, Stroke, Circulatory), SDC: Rare & Orphan Diseases