Multicentric prognostic observational study on biomarker profile in immunotherapy-naïve patients with highly active generalized myasthenia gravis (PROGNO-MG): a study protocol.

BACKGROUND

Myasthenia gravis has a variable disease course. Early identification of patients with a highly active disease is crucial to prevent myasthenic exacerbations and crisis, necessitating more aggressive immunotherapy. However, no biomarkers currently exist to identify patients at risk for highly active disease course.

OBJECTIVE

The PROGNO-MG study aims to identify blood-based biomarker signatures associated with a highly active disease course in immunotherapy-naïve patients with acetylcholine-receptor-antibody positive (AChR+) generalized Myasthenia gravis (gMG).

METHODS

This is an investigator-initiated prospective, multicentric, observational study. Seventy newly diagnosed immunotherapy-naïve AChR+ gMG patients will be enrolled and followed up for 24 months. Blood samples and clinical data will be collected biannually. Biomarkers (calprotectin, neurofilament light chain, ITIH3, complement activation, kappa free light chains, and exploratory proteome-analyses and immune cell phenotyping) will be measured and their association with clinical outcomes will be evaluated.

PERSPECTIVE: The results will provide evidence for prognostic biomarkers, supporting risk stratification and early individualized treatment for AChR+ gMG patients.

TRIAL REGISTRATION: The study is registered in the German clinical trial register (DRKS00035457).