**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.**
Jonas Wixner, Umeå University, Umeå, Sweden

**Main Body of the Abstract**
Background: Tafamidis is the only approved treatment for hereditary and wild-type transthyretin amyloid cardiomyopathy (ATTR-CM) to reduce mortality and cardiovascular-related hospitalizations. Many patients with ATTR-CM present with a mixed phenotype, and real-world effectiveness studies of tafamidis in this cohort are lacking. This analysis examined real-world survival in tafamidis-treated and tafamidis-untreated patients with mixed phenotype transthyretin amyloidosis (ATTR amyloidosis).
Methods: The Transthyretin Amyloidosis Outcomes Survey (THAOS; NCT00628745) was the largest longitudinal, observational, phase 4 study of patients with ATTR amyloidosis (treatment-naïve or receiving tafamidis). Patients from THAOS with a mixed phenotype who received any tafamidis dose at, prior, or after enrollment (tafamidis-treated) or never received tafamidis in THAOS (tafamidis-untreated) were included. Results are based on the completed THAOS dataset (data cutoff: July 24, 2023).
Results: There were 672 tafamidis-treated (median enrollment age, 69.2 years; 68.2% male) and 963 tafamidis-untreated (median enrollment age, 69.6 years; 71.9% male) patients; 71.7% and 77.3% had a variant transthyretin genotype (most commonly V30M), and 29.6% (126/425) and 39.5% (264/668) were New York Heart Association Class III-IV. Median symptom duration at enrollment was 5.1 years in both groups. Survival rates (95% CI) at 30 months for the combined, wild-type, and variant cohorts, respectively, were 87.7% (84.6-90.3), 85.1% (75.1-91.3), and 88.0% (84.5-90.8) in tafamidis-treated patients, and 72.4% (69.2-75.4), 62.8% (55.3-69.4), and 75.3% (71.6-78.5) in tafamidis-untreated patients (Figure).
Conclusions: This real-world analysis of patients with mixed phenotype ATTR amyloidosis showed increased survival in tafamidis-treated patients compared with prior reports. These data support the effectiveness of tafamidis in this population.



**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.**
Jonas Wixner, Umeå University, Umeå, Sweden

**Main Body of the Abstract**
**Background:** Tafamidis is the only approved treatment for hereditary and wild-type transthyretin amyloid cardiomyopathy (ATTR-CM) to reduce mortality and cardiovascular-related hospitalizations. Many patients with ATTR-CM present with a mixed phenotype, and real-world effectiveness studies of tafamidis in this cohort are lacking. This analysis examined real-world survival in tafamidis-treated and tafamidis-untreated patients with mixed phenotype transthyretin amyloidosis (ATTR amyloidosis).
**Methods:** The Transthyretin Amyloidosis Outcomes Survey (THAOS; NCT00628745) was the largest longitudinal, observational, phase 4 study of patients with ATTR amyloidosis (treatment-naïve or receiving tafamidis). Patients from THAOS with a mixed phenotype who received any tafamidis dose at, prior, or after enrollment (tafamidis-treated) or never received tafamidis in THAOS (tafamidis-untreated) were included. Results are based on the completed THAOS dataset (data cutoff: July 24, 2023).
**Results:** There were 672 tafamidis-treated (median enrollment age, 69.2 years; 68.2% male) and 963 tafamidis-untreated (median enrollment age, 69.6 years; 71.9% male) patients; 71.7% and 77.3% had a variant transthyretin genotype (most commonly V30M), and 29.6% (126/425) and 39.5% (264/668) were New York Heart Association Class III-IV. Median symptom duration at enrollment was 5.1 years in both groups. Survival rates (95% CI) at 30 months for the combined, wild-type, and variant cohorts, respectively, were 87.7% (84.6-90.3), 85.1% (75.1-91.3), and 88.0% (84.5-90.8) in tafamidis-treated patients, and 72.4% (69.2-75.4), 62.8% (55.3-69.4), and 75.3% (71.6-78.5) in tafamidis-untreated patients (**Figure**).
**Conclusions:** This real-world analysis of patients with mixed phenotype ATTR amyloidosis showed increased survival in tafamidis-treated patients compared with prior reports. These data support the effectiveness of tafamidis in this population.



Survival in a real-world cohort of patients with mixed phenotype transthyretin amyloidosis treated with tafamidis: an analysis from the Transthyretin Amyloidosis Outcomes Survey PC107 (#10)

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.