Pediatric Autoimmune Ocular Myasthenia Gravis: Evaluation of Presentation and Treatment Outcomes in a Large Cohort.

Abstract Objective Investigate clinical presentation, course, and treatment response of pediatric ocular myasthenia gravis (OMG) in a large single-center cohort. Background In autoimmune myasthenia gravis (MG), autoantibodies target the neuromuscular junction. OMG is localized, affecting only extraocular and/or levator palpebrae muscles. OMG presents across all ages, varying in presentation, treatment modalities, and outcomes. Recently, there have been advances in MG/OMG treatment; their utilization and effectiveness an important part of optimal disease management. Design /Methods: Retrospective chart review of children ≤18 years of age with confirmed diagnosis of OMG presenting from 2002-2019. Results Forty-two patients included with mean age at presentation of 8.5 years (2 – 18 years). Twenty-one patients (50%) had positive antibodies; 90% acetylcholine receptor antibodies. Ten patients developed generalized symptoms, with mean time to generalization of 13.6 months. Multiple logistic regression showed that older age of onset was a trend predictive factor (P = 0.054; odds ratio 1.17) for generalized disease. All patients were treated with pyridostigmine. Immunomodulating agents included steroids (15), mycophenolate mofetil (4), and intravenous immunoglobulin (1). Three patients underwent thymectomy. Twenty patients reached minimal manifestation status, 12 achieved remission. Gender, race, positive antibody status were not statistically significant predictors for advanced immunosuppressive therapy. Conclusions This study reports one of the largest cohorts of pediatric OMG patients who have undergone up-to-date diagnostic and therapeutic regimens. The predictors of outcome and treatment pathway for OMG patients suggested by this report may be further elucidated by future prospective studies.