2020 Fiscal Year Final Research Report
Research for immunological background in patients with childhood-onset myasthenia gravis (preliminary step toward novel immunosuppressive therapy)
Project/Area Number |
18K15703
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Research Category |
Grant-in-Aid for Early-Career Scientists
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Allocation Type | Multi-year Fund |
Review Section |
Basic Section 52050:Embryonic medicine and pediatrics-related
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Research Institution | Shinshu University |
Principal Investigator |
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Project Period (FY) |
2018-04-01 – 2021-03-31
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Keywords | 重症筋無力症 / B細胞活性化因子 / 小児 |
Outline of Final Research Achievements |
Blood samples were obtained from eight patients with generalized myasthenia gravis (GMG), seventeen with ocular myasthenia gravis (OMG), and thirteen age-matched control subjects. I assayed serum concentrations of B cell-activating factor (BAFF). Serum BAFF levels were significantly higher before immune-suppressive therapy in patients with childhood-onset OMG than in controls and decreased after immunosuppressive treatment. On the other hand, there were no significant differences between GMG patients with no immune-suppressive therapy and control subjects. These results suggested that BAFF may play a key role in the pathogenesis of pediatric OMG as well as adult OMG. Furthermore, unlike adult patients with GMG, the elevation of circulating BAFF levels was not correlated with pathophysiology of childhood-onset GMG.
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Free Research Field |
小児神経学
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Academic Significance and Societal Importance of the Research Achievements |
小児OMGでは血清BAFFが病態・病勢に関与していることが再確認された一方で、小児GMGでは血清BAFFが病態・病勢に関与している可能性が低いことを新たに示した。今後、小児MGに対する抗BAFF抗体など新規分子標的治療の導入を検討していく上で、重要な知見が得られた。
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