# Prospective longitudinal cohort of Argentinean patients with progressive supranuclear palsy and corticobasal syndrome: A platform for epidemiological and translational research

## TL;DR

This paper describes a new research initiative in Argentina to study rare brain disorders called PSP and CBS, aiming to improve understanding and treatment in underrepresented populations.

## Contribution

The paper introduces a prospective cohort study in Argentina for PSP and CBS, highlighting feasibility in a developing country.

## Key findings

- 33 patients with PSP and CBS have been enrolled, with over 60% completing initial assessments and biosample collection.
- Environmental exposures and clinical data are being systematically collected to support translational research.
- One patient donated their brain for pathological confirmation of PSP, supporting clinical diagnosis accuracy.

## Abstract

Progressive supranuclear palsy (PSP) and corticobasal syndrome (CBS) are neurodegenerative disorders with disabling postural instability, falls and cognitive impairment associated with 4-repeat tau protein neuropathology. The low prevalence (6/100,000 inhabitants) is estimated with data only from North American, European countries or Japan.

A multicenter initiative for studying the epidemiology and tau biology of Argentinean patients with PSP and CBS and their ovelaps, Consorcio Argentino de Investigación Traslacional en Tauopatias Primarias (CAITauP).

A prospective observational cohort of people with PSP (PwPSP), CBS and PSP/CBS overlaps will be longitudinally studied aiming at recruitment of 75 patients in 3 years. Deep clinical phenotyping, neurocognitive testing every 12 months, and biosamples collection at baseline are planned. Disease severity will be assessed every 6 months and history of environmental exposures will be assessed at baseline.

33/75 expected patients with PSP (19) and CBS (14) have been enrolled. Female gender is 56%, mean age 76, with estimated disease duration of 4.73 years. More than 60% of enrolled patients have completed first visit and biosamples collection, one patient underwent brain donation, pathological diagnosis of PSP, clinical Richardson syndrome. Environmental exposures of a preliminary subsample are reported.

The CAITauP enrolled a cohort of Argentinean PwPSP for deep clinical phenotyping and biobanking. Despite the rarity of PSP/CBS, we highlight the feasibility of establishing local cohorts in developing countries for translational research. The consortium aims to address underrepresented groups of Argentinean to improve diagnosis, treatment, and to create trial-ready cohorts.