ArticleFrontiers in neurology2026
Treatment patterns, intensive treatment, and hospitalization-related burden of juvenile generalized myasthenia gravis in Japan: a retrospective cohort study using a hospital-based claims database.
Article in Frontiers in neurology, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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Abstract
Objective: Juvenile generalized myasthenia gravis (MG) management aims to control symptoms while minimizing corticosteroid-related toxicity. However, large-scale evidence on real-world pediatric treatment patterns remains limited. Methods: We conducted a retrospective cohort study using a large Japanese hospital-based claims database (Medical Data Vision). We identified patients aged 0-17 years with juvenile generalized MG between April 2008 and November 2024. We summarized the use of cholinesterase inhibitors, oral corticosteroids, immunosuppressants, and intensive treatments (plasma exchange, intravenous immunoglobulin [IVIg], or high-dose steroid pulse therapy) during the one-year follow-up period after the first confirmed MG diagnosis recorded in the database. We also compared patient characteristics and treatment patterns by age group (children: 0-9 years vs. adolescents: 10-17 years). Results: We identified 261 patients with juvenile generalized MG, including 125 children and 136 adolescents cases. Cholinesterase inhibitors were the most frequently used medication (69.3% [181/261]). Oral corticosteroids were commonly used (65.9% [172/261]), whereas immunosuppressant use was limited; only 89/261 (34.1%) received tacrolimus. Among the 217 patients with complete one-year follow-up data, 146 received corticosteroid. The mean daily corticosteroid dose, calculated as the annual cumulative corticosteroid dose divided by 365 days, was 8.7 mg/day (SD 7.1) (children: 6.2 mg/day [SD 4.1]; adolescents: 11.4 mg/day [SD 8.6]). Among 261 generalized MG patients, 52 (19.9%) received intensive treatments, with a higher proportion in adolescents than children (25.7% [35/136] vs. 13.6% [17/125]). IVIg was the most common intensive treatment (13.4% [35/261]), followed by steroid pulse therapy (10.3% [27/261]) and plasma exchange (3.8% [10/261]). Adolescent generalized MG had higher intensive care unit hospitalization rates than child generalized MG (8.8% [12/136] vs. 0.8% [1/125]), and all thymectomies occurred in adolescent patients (8.1% [11/136] vs. 0% [0/125]). Conclusion: In Japanese real-world practice, about one in five children with generalized MG required intensive treatments, particularly among adolescents, while steroid-sparing immunosuppression was used in only one-third. These findings support close monitoring of adolescent generalized MG and highlight opportunities to optimize steroid-sparing management in juvenile generalized MG.
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