Patient- and Caregiver-Reported Burden and Quality of Life in Generalised Myasthenia Gravis: A Multinational...

Understanding patient- and caregiver-reported burden of generalised myasthenia gravis (gMG) is essential to inform region-specific management and policy decisions. However, real-world data from the Asia-Pacific region remain limited.

Data were drawn from the Adelphi Real World gMG Disease Specific Programme™, a cross-sectional survey of neurologists and their patients with gMG conducted in Australia, China, Japan, Malaysia, South Korea, and Taiwan between July 2023 and January 2024. Neurologists reported patient demographics, clinical characteristics, and perceived patient quality of life (QoL). Patients, and patients' caregivers provided answers to self-reported outcome measures, including the EQ-5D-5L, corresponding to status at the time of survey. Analyses were descriptive other than weighted kappa analyses used to measure neurologist-patient agreement on subjective patient QoL at the time of survey.

Overall, 163 neurologists reported data for 434 patients, of whom 205 self-reported data alongside 151 caregivers. Most patients were female (58.5%), with a mean (SD) age of 51.4 (15.2) years, and received caregiver support (71.4%). Patients reported deficits across all EQ-5D-5L domains, including usual activities (79.4%), and moderate to extreme anxiety/depression (29.4%), with a mean (SD) index score of 0.72 (0.23). Most patients (54.6%) had a FACIT-Fatigue score < 30, indicating severe fatigue, and 45.0% of patients had reduced employment due to gMG. A kappa value of 0.2692 indicated a fair agreement between neurologist and patient perceived patient QoL. Mean (SD) patient absenteeism was 11.8% (20.8%), presenteeism 39.2% (22.5%), overall work productivity loss of 42.9% (23.9%), and activity impairment of 51.3% (23.9%). Caregivers reported a mean (SD) 27.6% (19.6%) overall work productivity loss and 47.0% (24.1%) activity impairment.

Patients and their caregivers reported substantial burden to QoL, including impaired work productivity, limitations in activities of daily living, fatigue, and anxiety/depression. Further research into health-policy initiatives is warranted to more effectively address these unmet needs in the Asia-Pacific.