Clinical events and health care costs among pediatric and young adult patients with generalized myasthenia gravis in the United States.
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BACKGROUND: Management of children and young adults with generalized myasthenia gravis (gMG) is currently informed by research conducted largely in older adults. OBJECTIVES: To (1) describe sociodemographic/clinical characteristics and disease burden (treatments, occurrence of acute events [myasthenia gravis (MG) exacerbation or myasthenic crisis], and health care costs) for pediatric/young adult patients with gMG in the United States and (2) estimate the incremental health care cost burden of gMG for this group compared with controls without MG. A secondary objective was to explore the relationship between selected variables-including prior acute events-and the occurrence of subsequent acute events. METHODS: Longitudinal, retrospective cohorts of pediatric/young adult patients were identified from the Merative MarketScan Commercial Claims and Encounters Database (CCAE) and the Multi-State Medicaid Database (MDCD). Patients in the gMG cohort had at least 1 qualified MG event (January 1, 2010-June 30, 2025) and were aged from 2 to 25 years. Patients in the control cohort were selected at random from patients without MG (0.2% of patients with no MG diagnosis codes during study period; index dates from patients with gMG were randomly assigned) and had at least 6 months of continuous health plan enrollment and age range corresponding to the gMG cohort. Baseline characteristics, treatments during follow-up, and rates of acute events during follow-up were reported for each age category. Health care costs from the payer perspective per person per month (PPPM; following inverse probability of treatment weighting) for patients with gMG were compared with those for patients without MG. The association between selected variables of interest on the occurrence of acute events during follow-up was assessed using case controls with regression models. RESULTS: In the CCAE population with gMG (aged 2-11, 12-17, and 18-25 years, respectively [n = 226, n = 282, n = 853]), 25.2%, 34.8%, and 23.9% of patients had at least 1 MG exacerbation and 15.5%, 16.7%, and 13.6% had at least 1 myasthenic crisis (rates 18.2, 22.8, and 19.3/100 person-years [PY] and 9.3, 7.3, and 8.7/100 PY, respectively). Mean total health care costs were $6,104 PPPM for patients with gMG vs $257 PPPM for non-MG controls (n = 14,632)-a difference (95% CI) of $5,847 ($4,875-$6,819) PPPM. Mean costs were $13,401 PPPM for patients with MG exacerbation (vs $3,715 PPPM with no MG exacerbation) and $15,565 PPPM for those with myasthenic crisis (vs $4,734 with no myasthenic crisis). In the MDCD population with gMG (aged 2-11, 12-17, and 18-25 years, respectively [n = 218, n = 179, n = 284]), 29.8%, 40.8%, and 34.5% of patients had at least 1 MG exacerbation, and 13.3%, 23.6%, and 22.5% had at least 1 myasthenic crisis (rates 13.3, 26.9, and 30.1/100 PY and 5.8, 12.0, and 15.8/100 PY, respectively). Mean total health care costs were $2,605 PPPM for patients with gMG vs $162 PPPM for non-MG controls (n = 6,908)-a difference (95% CI) of $2,443 ($1,713-$3,172) PPPM. Mean costs were $4,934 PPPM for those with MG exacerbation (vs $1,535 with no MG exacerbation) and $5,538 PPPM for those with myasthenic crisis (vs $2,045 with no myasthenic crisis). In both populations, previous MG exacerbation was associated with recurrence of acute events during follow-up. CONCLUSIONS: For pediatric and young adult patients with gMG in the United States, health care costs were higher than in controls. Many patients experienced acute events, health care costs were higher for patients with acute events, and the occurrence of acute events was strongly associated with prior events. There is a need for improved gMG management to reduce the frequency of costly acute events.