A Sum Score to Define Therapy-Refractory Myasthenia Gravis: A German Consensus.

BACKGROUND AND PURPOSE

In 2017, eculizumab has been approved for treatment-refractory generalised myasthenia gravis (TRgMG). The German Myasthenia Foundation has published a consensus statement on the use of eculizumab, with a recent update. However, a treatment-refractory state is still ill-defined and the term warrants further clarification. We aimed at developing a sum score to operationalise the definition of a TRgMG status, which is easy- to-handle in clinical decision making.

METHODS

We established a structured consensus process according to the Delphi consensus methodology, with 12 members of the medical advisory board of the German Myasthenia Foundation. Accordingly, 4 consensus rounds were accomplished. Additionally, a literature survey covering the years 2004-2020 was done and relevant information offered to the consensus group. Consensus criteria were predefined. In the consensus process the relative importance of scoring items were to be consented, with a sum score of 20 and above indicating a TRgMG status.

RESULTS

The sum score considers the categories disease severity, inefficiency of antecedent therapies, cessation of therapies due to side effects, and long term stay on the intensive care unit. Categories were specified by a total of 13 scoring items. Eventually, the Delphi process developed an unanimous scoring consensus.

CONCLUSION

We suggest a sum score to define treatment refractory state in generalised myasthenia gravis. Beyond clarifying the indication of eculizumab, this easy-to-handle score facilitates clinical decision making and offers new inclusion criteria for clinical studies that explore new therapeutic perspectives in myasthenia gravis treatment.

Informations de copyright

© The Author(s) 2021.

Déclaration de conflit d'intérêts

Declaration of conflicting interests:The authors declared the following potential conflicts of interest with respect to the research, authorship, and/or publication of this article: MS..: Personal honoraria from Alexion, Biogen, Gilead, Roche, Sanofi. Institutional support: Alexion, Bayer, Biogen, CSL Behring, Grifols, Merck, Sanofi, Teva. B.B.: Received travel grants and/or training expenses from Bayer Vital GmbH, Ipsen Pharma GmbH, Novartis, Biogen GmbH and Genzyme, as well as lecture fees from Ipsen Pharma GmbH, Alexion Pharma GmbH, Merck, Sanofi Genzyme and Roche. T.H.: Received honoraria from Alexion, Biogen, Sanofi-Aventis, Roche, Novartis, CSL Behring. Institutional support: Sanofi-Aventis, Biogen, Roche and Avexis. S.J.: Reports personal honoraria from Alexion, Biogen, Bayer Vital GmbH, Bristol-Myers Squibb, Pfizer, Daiichi Sankyo, and institutional support from Sanofi Genzyme, Alnylam Germany, Santhera, Novartis, CSL Behring, UCB Pharma, Shire. D.H.L.: Received personal honoria from Alexion, Biogen, Bayer Vital GmbH, Novartis, Sanofi Genzyme, Merck, Roche. P.K.: Personal honoraria from Alexion, Biogen, Novatris, Sanofi Genzyme, Merck, Teva, Roche. T.R.: Reports grants from German Ministry of Education, Science, Research and Technology, grants and personal fees from Sanofi-Genzyme and Alexion; personal fees from Biogen, Roche and Teva; personal fees and nonfinancial support from Merck Serono, outside the submitted work. F.B.: Received honoraria from Alexion, Grifols, Sanofi Genzyme. Travel grant from Bayer Vital GmbH. U.S.: Received honoraris for advisory board participation and counselling from Flexion. P.U.: Personal honoraria from Alexion, Grifols, Zambon, Desitin, Abbvie. A.M.: Reports personal honoraria from Alexion, Argnx, Novartis, Bayer, Grifols, Bristol-Myers Squibb, Pfizer, UCB Pharma, Hormosan and Morphosys as well as institutional support from Alexion and Octapharma.