Characterizing and quantifying disease impacts of generalized myasthenia gravis (gMG) in the United States: insights from patient and caregiver interviews and surveys.

Abstract

Background: Treatment value is traditionally assessed through clinical benefits, health-related quality of life, and healthcare utilization. However, rare diseases such as generalized myasthenia gravis (gMG) that heavily affect families and/or caregivers require a more holistic approach that centers around impacted individuals across all areas of daily life. Here, key domains impacted by gMG were identified through individual interviews with both patients and caregivers and these impacts were further assessed through a survey in an independent sample.

Methods: Interviews with patients with gMG and caregivers identified 25 patient-centered "impact elements" across 8 life domains (occupation, financial, emotional, physical, sleep, social, planning and autonomy, and safety). Independently recruited patients and caregivers who completed a subsequent electronic, quantitative survey rated the relevance of impact elements on a scale of 1 (lowest) to 5 (highest).

Results: Respondents included 239 patients with gMG (63%, aged 18-49 y; 69%, female) and 81 caregivers (58%, aged 18-49 y; 42% female). The 5 domains most impacted by gMG were occupational (mean domain score [SD]: 4.0 [1.2]), planning and autonomy (4.0 [1.1]), financial (3.9 [1.2]), physical (3.9 [1.1]), and sleep (3.9 [1.2]) for patients and financial (3.7 [1.2]), sleep (3.7 [1.1]), occupational (3.6 [1.2]), planning and autonomy (3.6 [1.1]), and safety (3.6 [1.1]) for caregivers. Numerically higher impact scores were observed among patients with more severe disease, younger patients, and caregivers who were unemployed, retired, or disabled.

Conclusions: These findings provide additional insight into the comprehensive burden of gMG that extends beyond symptoms and medical costs.