To evaluate visual and systemic outcomes in pediatric patients with purely ocular myasthenia gravis (OMG) treated at the Children's Hospital of Philadelphia.

Retrospective chart review.

Pediatric patients with OMG seen at a single institution over a 16-year period with a minimum follow-up of 1 year were reviewed. Associations of demographic and clinical characteristics with disease resolution, amblyopia, and development of generalized symptoms of myasthenia gravis were analyzed.

Thirty-nine patients were identified, with a mean age of 5.4 ± 4.8 years and mean follow-up of 4.8 ± 4.3 years. Fifteen patients were treated with pyridostigmine only, 19 (49%) also received steroids, and 15 (38%) underwent thymectomy. Four patients (10%) received steroid-sparing immunosuppressive therapy. Resolution occurred in 10 patients, and generalized symptoms eventually occurred in 9 patients. Although 10 patients were treated for amblyopia, only 1 had amblyopia at the final visit. There was no correlation between sex or age with amblyopia or development of generalized symptoms. Thymectomy, when performed before the onset of generalized symptoms, showed a trend toward protection from the development of generalized symptoms (P = .07).

In our series, 24% of patients had disease resolution and 23% had generalized symptoms. Our larger cohort confirms previous findings that treated and untreated pediatric patients with OMG have a relatively low risk of developing generalized symptoms and that related amblyopia is readily reversible. Although our treatments were more aggressive than those previously reported, our rates of amblyopia and development of generalized symptoms are comparable.