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.
Mia Papas, AstraZeneca, Wilmington DE

Main Body of the Abstract
Background/Aim
Transthyretin (ATTR) amyloidosis is a rare, progressive disorder characterized by systemic ATTR amyloid deposition due to hereditary (ATTRv) or non-hereditary (wild-type [ATTRwt]) transthyretin protein instability. Dominant phenotypes include the heart (ATTR-cardiomyopathy [CM]), peripheral nerves (ATTR-polyneuropathy [PN]), or both (mixed). US ATTR amyloidosis prevalence is increasing, and true ATTRwt prevalence may be as high as 300,000, yet US-specific ATTR amyloidosis patient demographics and treatment utilization are not well-characterized.
Methods
This study used 2018-2020 data from Milliman’s Consolidated Health Cost Guidelines Sources Database (commercial – individual, small/large group) and the 100% Research Identifiable Files (Medicare Fee-for-Service [FFS]) from the Centers for Medicare and Medicaid Services (CMS) to characterize patients with amyloidosis. The population was segmented into genotypes and clinical phenotypes using ATTR-specific drugs (tafamidis, patisiran, inotersen), amyloidosis diagnosis codes, and diagnosis codes for cardiovascular and neurologic disease. Patients with evidence of primary amyloidosis, secondary amyloidosis, or non-segmented amyloidosis were excluded, yielding the ATTR amyloidosis cohorts.
Results
Patients with ATTR amyloidosis are covered by Medicare at a rate of 8:1 versus commercial. Patients with ATTRwt are predominantly male, while ATTRv is more evenly split. Except for ATTRv-CM, the average patient age is over 50. Patients with ATTRwt use ATTR-specific drugs more frequently than ATTRv patients.
Conclusion
Although all segments include patients who may not be candidates for treatment (e.g., asymptomatic or late-stage), only 8% to 36% of diagnosed patients receive any ATTR-specific drugs. The observed use of ATTR-specific treatments highlights the need for further investigation into factors driving treatment initiation and continuation.

**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.**
Mia Papas, AstraZeneca, Wilmington DE

**Main Body of the Abstract**
**Background/Aim**
Transthyretin (ATTR) amyloidosis is a rare, progressive disorder characterized by systemic ATTR amyloid deposition due to hereditary (ATTRv) or non-hereditary (wild-type [ATTRwt]) transthyretin protein instability. Dominant phenotypes include the heart (ATTR-cardiomyopathy [CM]), peripheral nerves (ATTR-polyneuropathy [PN]), or both (mixed). US ATTR amyloidosis prevalence is increasing, and true ATTRwt prevalence may be as high as 300,000, yet US-specific ATTR amyloidosis patient demographics and treatment utilization are not well-characterized.
**Methods**
This study used 2018-2020 data from Milliman’s Consolidated Health Cost Guidelines Sources Database (commercial – individual, small/large group) and the 100% Research Identifiable Files (Medicare Fee-for-Service [FFS]) from the Centers for Medicare and Medicaid Services (CMS) to characterize patients with amyloidosis. The population was segmented into genotypes and clinical phenotypes using ATTR-specific drugs (tafamidis, patisiran, inotersen), amyloidosis diagnosis codes, and diagnosis codes for cardiovascular and neurologic disease. Patients with evidence of primary amyloidosis, secondary amyloidosis, or non-segmented amyloidosis were excluded, yielding the ATTR amyloidosis cohorts.
**Results**
Patients with ATTR amyloidosis are covered by Medicare at a rate of 8:1 versus commercial. Patients with ATTRwt are predominantly male, while ATTRv is more evenly split. Except for ATTRv-CM, the average patient age is over 50. Patients with ATTRwt use ATTR-specific drugs more frequently than ATTRv patients.
**Conclusion**
Although all segments include patients who may not be candidates for treatment (e.g., asymptomatic or late-stage), only 8% to 36% of diagnosed patients receive any ATTR-specific drugs. The observed use of ATTR-specific treatments highlights the need for further investigation into factors driving treatment initiation and continuation.

Descriptive characteristics of patients diagnosed with transthyretin amyloidosis (ATTR) in the Medicare fee-for-service and commercial populations PC142 (#518)

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.