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