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Background <p>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.</p> Objective <p>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<sup>+</sup>) generalized Myasthenia gravis (gMG).</p> Methods <p>This is an investigator-initiated prospective, multicentric, observational study. Seventy newly diagnosed immunotherapy-naïve AChR<sup>+</sup> 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.</p> Perspective <p>The results will provide evidence for prognostic biomarkers, supporting risk stratification and early individualized treatment for AChR<sup>+</sup> gMG patients.</p> Trial registration <p>The study is registered in the German clinical trial register (DRKS00035457).</p>

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

  • 期刊论文

  • 出版模式：

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  • 发表日期：

    2026年5月12日

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  • Felix Kohle,
  • Franz Felix Konen,
  • Ulrike Grittner,
  • Daniel Schulze,
  • Stefanie Glaubitz,
  • Christopher Nelke,
  • Christina B. Schroeter,
  • Niklas Huntemann,
  • Peter Trillenberg,
  • Julian Großkreutz,
  • Benedikt Schoser,
  • Charlotte Schubert,
  • Jana Zschüntzsch,
  • Michael Schroeter,
  • Tobias Hegelmaier,
  • Thomas Skripuletz,
  • Jörn Peter Sieb,
  • Tobias Ruck,
  • Sven Meuth,
  • Heinz Wiendl,
  • Christian Wolfgang Keller,
  • Jan D. Lünemann,
  • Sophie Lehnerer,
  • Andreas Meisel,
  • Frauke Stascheit

摘要

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).