**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.**
Agathe COSTE, Pfizer medical advisor , Paris, France, agathe.coste@pfizer.com

**Main Body of the Abstract**
Objective:
We aimed to describe characteristics of patients with transthyretin amyloid variant (ATTRv) polyneuropathy (PN) and ATTRv-mixed (with an association of polyneuropathy and cardiomyopathy) and assess the real-world use and safety profile of tafamidis meglumine 20 mg.
Methods:
Twenty-five French hospitals contributed to the study. Neurology patient files were reviewed to identify clinical manifestations, diagnostic methods, and treatment compliance.
Results:
Four hundred and thirteen patients (296 ATTRv-PN, 117 ATTRv-mixed) were analyzed. Patients were predominantly male (68.0%) with a mean age of 57.2±17.2 years. Median interval between first symptom(s) and diagnosis was 2.1 years (range: 0-21.9). First symptoms included sensory complaints (85.9%), dysautonomia (38.5%), motor deficits (26.4%), carpal tunnel syndrome (31.5%), shortness of breath (13.3%), and unexplained weight loss (16.0%). Mini-invasive accessory salivary gland or punch skin and nerve biopsies looking for amyloidosis were most common, with a performance of 78.8–100%. TTR genetic sequencing, performed in all patients, revealed 31 transthyretin variants. Tafamidis meglumine was initiated in 156(72.9%) ATTRv-PN patients at an early disease stage. Median treatment duration was 6.0 years in ATTRv-PN and 3.4 years in ATTRv-mixed patients. Tafamidis was well tolerated, with 20 adverse events likely related to study drug among the 336 patients.
Conclusion:
In France, ATTRv patients are usually identified early thanks to the national network. This study on real-world clinical ATTR cases further elucidated the characteristics of ATTRv-PN and ATTRv-mixed. Tafamidis meglumine was prescribed to most patients with confirmed expected effectiveness and good safety.



**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.**
Agathe COSTE, Pfizer medical advisor , Paris, France, agathe.coste@pfizer.com

**Main Body of the Abstract**
**Objective:**
We aimed to describe characteristics of patients with transthyretin amyloid variant (ATTRv) polyneuropathy (PN) and ATTRv-mixed (with an association of polyneuropathy and cardiomyopathy) and assess the real-world use and safety profile of tafamidis meglumine 20 mg.
**Methods:**
Twenty-five French hospitals contributed to the study. Neurology patient files were reviewed to identify clinical manifestations, diagnostic methods, and treatment compliance.
**Results:**
Four hundred and thirteen patients (296 ATTRv-PN, 117 ATTRv-mixed) were analyzed. Patients were predominantly male (68.0%) with a mean age of 57.2±17.2 years. Median interval between first symptom(s) and diagnosis was 2.1 years (range: 0-21.9). First symptoms included sensory complaints (85.9%), dysautonomia (38.5%), motor deficits (26.4%), carpal tunnel syndrome (31.5%), shortness of breath (13.3%), and unexplained weight loss (16.0%). Mini-invasive accessory salivary gland or punch skin and nerve biopsies looking for amyloidosis were most common, with a performance of 78.8–100%. TTR genetic sequencing, performed in all patients, revealed 31 transthyretin variants. Tafamidis meglumine was initiated in 156(72.9%) ATTRv-PN patients at an early disease stage. Median treatment duration was 6.0 years in ATTRv-PN and 3.4 years in ATTRv-mixed patients. Tafamidis was well tolerated, with 20 adverse events likely related to study drug among the 336 patients.
**Conclusion:**
In France, ATTRv patients are usually identified early thanks to the national network. This study on real-world clinical ATTR cases further elucidated the characteristics of ATTRv-PN and ATTRv-mixed. Tafamidis meglumine was prescribed to most patients with confirmed expected effectiveness and good safety.



Transthyretin amyloid polyneuropathy in France: a cross-sectional study with 413 patients and real-world tafamidis meglumine use (2009-2019) PB36 (#335)

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.