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.
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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.