(A35) Characteristics, Disease Burden, and Preliminary Outcomes of Patients with Myasthenia Gravis in Real-World Treatment Settings Across the United States
Background: Myasthenia gravis (MG) is a neuromuscular disorder that causes skeletal muscle weakness and fatigability. There are limited data characterizing patients’ diagnosis, disease management and outcomes in real-world clinical practice. PicnicHealth is collaborating with patients with MG to generate a real-world dataset (RWD) to support research and improve patient care.
Objectives: Describe demographics, clinical characteristics and disease burden of a cohort of patients with MG in real-world clinical practice settings across the United States.
Methods: Recruitment began in August 2021 and is ongoing. Patients with MG onboard to PicnicHealth’s research platform and consent to share their de-identified medical records for this IRB-approved observational study. PicnicHealth collects retrospective and prospective medical records from across U.S. health systems. Eligibility requires confirmation of MG diagnosis in medical records. Data are abstracted from structured and unstructured portions of records using human-verified machine learning.
Results: As of June 2023, the 795 confirmed patients were 69% female and 75% white, dispersed across the U.S. in the South (47%), Midwest (22%), West (19%) and Northeast (12%). Median (IQR) age at enrollment was 53 (40-66) years. Median (IQR) age at diagnosis was 40 (28, 54) years for females and 60 (47, 68) for males. Most common symptoms were diplopia (63%), ptosis (61%), dyspnea (53%) and dysphagia (52%). Patients saw a neurologist an average of 3.73 times per year and were treated most commonly with pyridostigmine (93%), prednisone (75%) and immunoglobulin G (52%). Evidence of MG exacerbation and myasthenic crisis were found in 301 and 146 patients, respectively, with a median (IQR) of 2 (1-4) exacerbations and 1 (1-2) crisis. MG-ADL scores were available for 733 (92%) patients with a median (IQR) of 3 (1-6) scores per patient and mean (SD) value of 6.17 (4.03).
Conclusions: This study leverages RWD to generate insights into clinical characteristics, treatment patterns and experiences of patients with MG; the data characterize a diverse cohort of patients with this rare disease. Ongoing efforts will grow cohort size and comprehensive data collection, including supplementation of MG-ADL data through prospective patient-reported outcomes.
