Patient Preferences for Generalized Myasthenia Gravis Treatment Profiles: Results of a Web-Based Survey


Clinical Management

Poster Number: M259


Karen Yee, Alexion, AstraZeneca Rare Disease, Christine Poulos, RTI International, Cooper Bussberg, RTI International, Kelley Myers, RTI International

Background: Generalized myasthenia gravis (gMG) is a rare, chronic autoimmune disorder. Key therapeutic approaches differ among the available treatments for anti-acetylcholine receptor antibody-positive (AChR Ab+) gMG, but no studies on patient treatment preferences have been published to date.

Objectives: To better understand treatment preferences of patients with AChR Ab+ gMG, and to estimate the relative importance of preferred treatment attributes.

Methods: US adults with a self-reported physician diagnosis of AChR Ab+ gMG completed a web-based survey. Two object-case, best-worst scaling (BWS) exercises were analyzed. The first BWS exercise assessed preferences across 4 different treatment profiles (eg, similar to: ravulizumab, eculizumab, efgartigimod intravenous and subcutaneous, or zilucoplan). The second BWS exercise obtained preferences for the individual attributes used to define the treatment profiles. Profile scenarios were defined by mode of administration and dosing frequency only, followed by the addition of consistent disease control, and meningococcal vaccination requirements. Additionally, the most important gMG treatment attribute was identified.

Results: Of 153 respondents, mean age was 49 years, 77% female, 84% white, 54% with college degree or higher, 41% employed, and 27% had been diagnosed for <3 years. Mean MG-Activities of Daily Living score was 8.0 (min-max: 0-17). Respondents preferred the ravulizumab-like profile vs all 3 other profile-based scenarios: 35% vs 10%-22% when considering mode and dosing frequency only, 44% vs 3%-31% when considering addition of consistent disease control, and 39% vs 5%-29% when considering all 4 attributes. Consistent disease control was selected as the most important attribute when choosing a gMG treatment (82%), followed by mode of administration (10%), dosing frequency (6%), and meningococcal vaccination requirements (3%). Conclusions: Patients with gMG preferred treatments with less frequent dosing schedules and consistent disease control, and consistent disease control was considered the most important attribute when choosing a therapy.