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, Department of Public Health and Clinical Medicine, Umeå University, Umeå, Sweden

Main Body of the Abstract
Background
Diflunisal has been shown to halt disease progression of hereditary transthyretin (ATTR) amyloidosis [Berk et al]. The SveaTTR registry is a longitudinal Swedish quality registry open for patients with ATTR amyloidosis since the 1980’s.
Methods
Data from diflunisal treated ATTR amyloidosis patients registered in SveaTTR as of Dec 2022 were included. Available data on Kumamoto score, PND score, FAP stage, mBMI, NYHA classification and Karnofsky performance status were analyzed using random coefficients mixed effects modelling, and the results were compared with those of the Berk et al pivotal trial.
Results
A total of 118 subjects were included in the analyses, of whom 75% were male, 69% had neuropathy at onset and 93% had a TTR mutation. Mean age at diagnosis was 68.2 years and mean duration of disease since diagnosis was 7.6 years. The estimated mean time of diflunisal therapy was 3.4 years (range 0.1 to 10.2 years). Random coefficients mixed effects modelling demonstrated gradual progression of disease over time by upward trend of Kumamoto score, PND score and FAP stage, and downward trend in Karnofsky score and mBMI while NYHA class score remained flat. A Bayesian augmentation analysis was undertaken in which registry data and pivotal trial data were combined. After 24 months, the probability of superiority for diflunisal over placebo was 99.6% and 97.2% for Kumamoto score and mBMI, respectively.
Conclusions
SveaTTR registry data are highly consistent with the results of the pivotal trial and further support the efficacy of diflunisal for hereditary ATTR amyloidosis.

**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, Department of Public Health and Clinical Medicine, Umeå University, Umeå, Sweden

**Main Body of the Abstract**
**_Background_**
Diflunisal has been shown to halt disease progression of hereditary transthyretin (ATTR) amyloidosis [Berk et al]. The SveaTTR registry is a longitudinal Swedish quality registry open for patients with ATTR amyloidosis since the 1980’s.
**_Methods_**
Data from diflunisal treated ATTR amyloidosis patients registered in SveaTTR as of Dec 2022 were included. Available data on Kumamoto score, PND score, FAP stage, mBMI, NYHA classification and Karnofsky performance status were analyzed using random coefficients mixed effects modelling, and the results were compared with those of the Berk et al pivotal trial.
**_Results_**
A total of 118 subjects were included in the analyses, of whom 75% were male, 69% had neuropathy at onset and 93% had a _TTR_ mutation. Mean age at diagnosis was 68.2 years and mean duration of disease since diagnosis was 7.6 years. The estimated mean time of diflunisal therapy was 3.4 years (range 0.1 to 10.2 years). Random coefficients mixed effects modelling demonstrated gradual progression of disease over time by upward trend of Kumamoto score, PND score and FAP stage, and downward trend in Karnofsky score and mBMI while NYHA class score remained flat. A Bayesian augmentation analysis was undertaken in which registry data and pivotal trial data were combined. After 24 months, the probability of superiority for diflunisal over placebo was 99.6% and 97.2% for Kumamoto score and mBMI, respectively.
**_Conclusions_**
SveaTTR registry data are highly consistent with the results of the pivotal trial and further support the efficacy of diflunisal for hereditary ATTR amyloidosis.

Open-label treatment data from the Swedish SveaTTR registry support the efficacy of diflunisal for hereditary transthyretin amyloidosis. PC100 (#116)

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.