**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.**
Nicholas S Streicher
MedStar Health
Georgetown University School of Medicine
Washington, DC, USA

**Main Body of the Abstract**
Introduction: Transthyretin amyloidosis (ATTR) is now widely recognized as a rare spectrum disease manifesting as ATTR polyneuropathy, ATTR cardiomyopathy (ATTR-CM), or a mixed phenotype. High-dose tafamidis (61 mg) is a first-in-class highly specific and selective stabilizer of both wild-type ATTR (ATTRwt) and variant ATTR (ATTRv) approved worldwide to reduce cardiovascular mortality and cardiovascular-related hospitalization in ATTR-CM. A recent exploratory real-world study in the United States demonstrated high-dose tafamidis was beneficial for delaying neurologic disease progression in patients with mixed-phenotype ATTRv-CM exposed to tafamidis for >6 months. The most recent Transthyretin Amyloidosis Outcomes Survey analysis reported that almost half of mixed phenotype ATTR amyloidosis patients in North America were ATTRwt genotype. The current study will assess the benefit of high-dose tafamidis treatment on neurologic disease progression in mixed-phenotype ATTRv-CM or ATTRwt-CM patients treated with tafamidis for >12 months in a real-world setting.
Methods: This retrospective, non-interventional cohort study will include approximately 50 patients from the United States with mixed-phenotype ATTRv-CM or ATTRwt-CM treated with tafamidis 61 mg for at least 12 months. Anonymized patient data will be collected from electronic medical records pre- and post-tafamidis treatment. Assessments will include the neuropathy impairment score (NIS) subscales, polyneuropathy disability (PND) score, Medical Research Council (MRC) Scale for Muscle Strength, and modified body mass index (mBMI). Data analysis will be descriptive.
Results: Available results will be provided at the time of the meeting.
Conclusions: This study will elucidate the benefit of long-term high-dose tafamidis treatment on neurologic progression in mixed phenotype ATTRv-CM and ATTRwt-CM.



Real-World Effectiveness of High-Dose Tafamidis on Neurologic Disease Progression in Mixed-Phenotype Transthyretin Amyloid Cardiomyopathy PC93 (#14)

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.