Skip to main navigation Skip to search Skip to main content

A Delphi consensus on integrating novel therapies into the management of generalized myasthenia gravis

  • John Vissing*
  • , Fritz Zimprich
  • , Sari Atula
  • , Andrew Chan
  • , Henning Andersen
  • , Raffi Topakian
  • , Martijn R. Tannemaat
  • , Konrad P. Weber
  • , Hakan Cetin
  • , Christoph Neuwirth
  • , Hanna Kuusisto
  • , Wolfgang N. Loescher
  • *Corresponding author for this work

Research output: Contribution to journalArticleScientificpeer-review

4 Downloads (Pure)

Abstract

Background: The treatment landscape for myasthenia gravis (MG) has evolved with the introduction of novel therapies. An international consensus on patient selection criteria and optimal time to initiate these therapies could improve clinical outcomes and reduce delays for likely beneficiaries. Objective: This Delphi consensus was undertaken by MG specialists from selected European countries to explore gaps in the application of national guidelines and elicit expert opinion in practice. Design: A mixed-method approach was used; qualitative and quantitative study phases were combined to explore key concepts and reach consensus. Methods: The qualitative first phase involved seven healthcare professionals (HCPs) and two patient advocacy group representatives who participated in idea generation. Findings from this phase supported the development of a Delphi survey, which was completed by 16 HCPs in two rounds. This constituted the quantitative second phase of the study. Consensus was defined as ⩾70% agreement or disagreement on a 6-point Likert scale. Results: In total, 65% of statements achieved consensus. Key findings include—HCPs highly regard international guidelines but find critical discrepancies between the “ideal” scenario and current clinical practices. Consensus was achieved on the importance of incorporating patient-related quality of life in decision-making, despite limited current methods. Consensus was obtained on steroid tapering and treatment-switch criteria based on steroid dose and duration. Consensus was also achieved on suitable patient profiles, including those with persistent symptoms, severe side effects, or needing rapid control. Conclusion: This study recognized that guidelines offer valuable direction but do not replace individualized treatment decisions. This study identified the areas of alignment and opportunities to refine patient selection criteria and treatment-switch categories, particularly to integrate novel therapy use in MG management, highlighting a path to a more patient-centric approach.

Original languageEnglish
Number of pages1
JournalTherapeutic Advances in Neurological Disorders
Volume19
DOIs
Publication statusPublished - 2026
Publication typeA1 Journal article-refereed

Funding

The authors disclosed receipt of the following financial support for the research, authorship, and/or publication of this article: This study was supported by sponsorship from Alexion and AstraZeneca Rare Disease. The authors acknowledge and thank Katrin Moser-Schirr and Luis Zurkirchen of Alexion, AstraZeneca Rare Disease, for their recommendation to conduct this study. The authors also thank Vartika Savarna and Alexandra Starry of Cytel for their support in the execution, coordination, and management of this consensus project, including editorial and administrative assistance through the manuscript submission phase. This assistance was funded by Alexion and AstraZeneca Rare Disease. The authors confirm that they authorized the submission of this manuscript via a third party and that they have reviewed and approved the final content of the manuscript, as well as all statements and declarations, including those related to conflicts of interest and funding.

Keywords

  • Delphi consensus
  • Europe
  • generalized
  • innovative therapies
  • myasthenia gravis

Publication forum classification

  • Publication forum level 1

ASJC Scopus subject areas

  • Pharmacology
  • Neurology
  • Clinical Neurology

Fingerprint

Dive into the research topics of 'A Delphi consensus on integrating novel therapies into the management of generalized myasthenia gravis'. Together they form a unique fingerprint.

Cite this