**Author Listing - Lead/ Submitting Author (we will be communicating with this individual): Name, Institution, City and State where the work was done. Co-authors will be added in a separate section.**
Pablo Garcia-Pavia, Hospital Universitario Puerta de Hierro Majadahonda, Madrid, Spain

**Main Body of the Abstract**
Background: In the pivotal Tafamidis in Transthyretin Cardiomyopathy Clinical Trial (ATTR-ACT), tafamidis significantly reduced mortality and cardiovascular-related hospitalizations, leading to its approval worldwide for the treatment of transthyretin amyloid cardiomyopathy (ATTR-CM). Real-world evidence on survival following tafamidis treatment of ATTR-CM has not been reported extensively. This analysis examined real-world survival in ATTR-CM patients treated with tafamidis.
Methods: The Transthyretin Amyloidosis Outcomes Survey (THAOS; NCT00628745) was the largest longitudinal, observational, phase 4 study of patients with transthyretin amyloidosis (treatment-naïve or receiving tafamidis), and asymptomatic patients carrying pathogenic transthyretin variants. This analysis included patients from THAOS with a predominantly cardiac phenotype who received any dose of tafamidis at, prior, or after enrollment (tafamidis-treated) or never received tafamidis in THAOS (tafamidis-untreated). Results are based on the completed THAOS dataset (data cutoff: July 24, 2023).
Results: There were 587 tafamidis-treated and 854 tafamidis-untreated patients included in this analysis. In the respective groups, median age at enrollment was 77.7 and 76.4 years, 91.8% and 90.0% were male, and 91.8% and 83.8% had wild-type disease. Survival rates (95% CI) at 30 and 42 months, respectively, were 84.4% (80.5–87.7) and 76.8% (70.9–81.7) in tafamidis-treated patients, and 70.0% (66.4­–73.2) and 59.3% (55.2–63.0) in tafamidis-untreated patients (Figure). No new safety signals were identified.
Conclusions: In this real-world cohort of ATTR-CM patients, survival rates were higher than in ATTR-ACT and consistent with more recent reports. These results provide further evidence supporting tafamidis’ safety and effectiveness.



**Author Listing - Lead/ Submitting Author (we will be communicating with this individual): Name, Institution, City and State where the work was done. Co-authors will be added in a separate section.**
Pablo Garcia-Pavia, Hospital Universitario Puerta de Hierro Majadahonda, Madrid, Spain

**Main Body of the Abstract**
**Background:** In the pivotal Tafamidis in Transthyretin Cardiomyopathy Clinical Trial (ATTR-ACT), tafamidis significantly reduced mortality and cardiovascular-related hospitalizations, leading to its approval worldwide for the treatment of transthyretin amyloid cardiomyopathy (ATTR-CM). Real-world evidence on survival following tafamidis treatment of ATTR-CM has not been reported extensively. This analysis examined real-world survival in ATTR-CM patients treated with tafamidis.
**Methods:** The Transthyretin Amyloidosis Outcomes Survey (THAOS; NCT00628745) was the largest longitudinal, observational, phase 4 study of patients with transthyretin amyloidosis (treatment-naïve or receiving tafamidis), and asymptomatic patients carrying pathogenic transthyretin variants. This analysis included patients from THAOS with a predominantly cardiac phenotype who received any dose of tafamidis at, prior, or after enrollment (tafamidis-treated) or never received tafamidis in THAOS (tafamidis-untreated). Results are based on the completed THAOS dataset (data cutoff: July 24, 2023).
**Results:** There were 587 tafamidis-treated and 854 tafamidis-untreated patients included in this analysis. In the respective groups, median age at enrollment was 77.7 and 76.4 years, 91.8% and 90.0% were male, and 91.8% and 83.8% had wild-type disease. Survival rates (95% CI) at 30 and 42 months, respectively, were 84.4% (80.5–87.7) and 76.8% (70.9–81.7) in tafamidis-treated patients, and 70.0% (66.4­–73.2) and 59.3% (55.2–63.0) in tafamidis-untreated patients (**Figure**). No new safety signals were identified.
**Conclusions:** In this real-world cohort of ATTR-CM patients, survival rates were higher than in ATTR-ACT and consistent with more recent reports. These results provide further evidence supporting tafamidis’ safety and effectiveness.



Survival in a real-world cohort of patients with transthyretin amyloid cardiomyopathy treated with tafamidis: an analysis from the Transthyretin Amyloidosis Outcomes Survey PC149 (#8)

We invite you to attend the XIX International Symposium on Amyloidosis to be held in Rochester, MN.

This symposium is organized as a hybrid meeting (with the focus on in-person) held from Sunday, May 26 through Thursday, May 30, 2024.

Together with the ISA Board and members of Mayo Clinic, a very attractive scientific hybrid program is being planned. The program will cover a variety of types of systemic amyloidosis: ATTR (hereditary and wild-type), AL (light-chain), AA and less frequent forms. There will be key lectures, specific symposia including those company sponsored, oral presentations and poster sessions of accepted abstracts.

COURSE DESCRIPTION
This program will provide a comprehensive review of state-of-the-art education in systemic amyloidosis: basic science, pathophysiology, diagnosis, current and emerging treatments. Scientific presentations will include competitive oral and poster abstracts. The rapid evolution of scientific discovery and expanding treatment options has created tremendous interest in the field and this meeting will fill and important gap in educating scientists and clinicians.