Treatment Goals in Myasthenia Gravis: Expert Consensus Recommendations Using the RAND/UCLA Appropriateness Method
Pushpa Narayanaswami1, Renato Mantegazza2, Carolina Barnett Tapia3, Elena Cortes-Vicente4, Miriam Freimer5, Nils Gilhus6, Ali A. Habib7, James F. Howard Jr.8, Anna Kostera-Pruszczyk9, M. Isabel Leite10, Andreas Meisel11, Mamatha Pasnoor12, Sabrina Sacconi13, Nicholas Silvestri14, Shigeaki Suzuki16, Akiyuki Uzawa17, Gil I. Wolfe15, Kimiaki Utsugisawa18
1Beth Israel Deaconess Medical Center, 2Fondazione IRCCS, Istituto Nazionale Neurologico Carlo Besta, 3Ellen and Martin Prosserman Centre for Neuromuscular Diseases, General Hospital, University of Toronto, 4Hospital de la Santa Creu i Sant Pau, 5The Ohio State University Wexner Medical Center, 6Haukeland University Hospital, 7University of California, Irvine, 8The University of North Carolina at Chapel Hill, 9Department of Neurology,, Medical University of Warsaw, 10Nuffield Department of Clinical Neurosciences - University of Oxford, 11Charité Universitätsmedizin Berlin, 12University of Kansas Medical Center, 13Université Côte d'Azur, Peripheral Nervous System & Muscle Department, Pasteur 2 Hospital, Centre Hospitalier Universitaire de Nice, 14Department of Neurology, Jacobs School of Medicine and Biomedical Sciences, University at Buffalo, 15University at Buffalo, 16Department of Neurology, Tokyo Metropolitan Neurological Hospital, 17Department of Neurology, Graduate School of Medicine, Chiba University, 18Hanamaki General Hospital
Objective:

To develop globally relevant consensus recommendations to define and operationalize treatment goals for patients with myasthenia gravis (MG).

Background:

Treatment goals are important in guiding individualized treatment selection and therapeutic adjustments in MG, but limited guidance is available on how to define, implement and achieve goals.

Design/Methods:

An international panel of MG experts was formed. A review of the literature was conducted to inform the development of consensus recommendations. Relevant topics were selected by the steering committee and draft recommendations developed for each topic by the methodologist. The RAND/UCLA appropriateness method was used to achieve formal consensus. All panel members rated the draft recommendations online on a scale of 1 (extremely inappropriate) to 9 (extremely appropriate). Panelists provided comments and suggestions for modifications. The methodologist modified recommendations for further rating rounds, based on summary scores and qualitative panel feedback. Recommendations that achieved agreement as appropriate by four rounds of voting were accepted.

Results:

Seventeen MG experts from North America, Europe and Asia rated the recommendations. Overall, 21 recommendations reached agreement as appropriate. Recommendation 1 defined the ongoing treatment goal in MG as “to work towards, achieve and sustain minimal symptoms and treatment-related adverse events, with a patient-acceptable quality of life (using validated measures)”. Subsequent recommendations described how to implement and achieve this goal for individual patients, across six areas: establishing and sustaining a treatment goal; working towards early symptom control; infection screening and vaccination; pregnancy/family planning; fatigue and comorbidity management; and managing impending crisis and crisis. Recommendations emphasise the importance of patient engagement and education, reassessing and changing treatment to achieve goals, and care coordination with other specialists. The recommendations will be presented in full.

Conclusions:
Expert consensus was reached on a range of globally relevant recommendations for treatment goals in MG. These recommendations will inform patient care for optimal disease outcomes.
10.1212/WNL.0000000000215185
Disclaimer: Abstracts were not reviewed by Neurology® and do not reflect the views of Neurology® editors or staff.