Long-term outcomes of pediatric ocular myasthenia gravis.
OBJECTIVE
To review the presenting signs, therapeutic interventions, and clinical outcomes of purely ocular myasthenia gravis in a preadolescent population treated primarily with pyridostigmine bromide.


METHODS
Retrospective case series.


METHODS
Twenty-one consecutive patients younger than 12 years with purely ocular myasthenia gravis at initial presentation.


METHODS
The clinical charts were reviewed retrospectively.


METHODS
Time to generalization, stabilization, or resolution; prism cover test results; and visual acuity.


RESULTS
Median age at onset was 26 months. The mean duration of follow-up was 6.5 years (range, 2-15). Presenting signs included ptosis (95%), strabismus (76%), limitation of ductions (17%), and Cogan's lid twitch (76%). The most common form of strabismus was exotropia. Pyridostigmine monotherapy was the initial treatment for all patients. Corticosteroids were added to the therapy for 6 patients. Generalization to systemic disease occurred in 3 patients. Two of those required intravenous immunoglobulin and thymectomy. Complete resolution off of medical therapy occurred in 4 patients. All patients had stabilization of the ocular motor deficits regardless of treatment. Eleven patients were treated for amblyopia; 2 had residual amblyopia.


CONCLUSIONS
Ocular myasthenia in preadolescent children generally presents before age 5 and is clinically distinguished from the disease as it affects adults. Although the presenting signs of strabismus, ptosis, and Cogan's lid twitch are common in children or adults, the response to treatment and eventual outcomes differ. Most children can be safely treated with pyridostigmine alone. Generalization to systemic disease occurs at a much lower rate than in adults. Ocular manifestations stabilize in all children and completely resolve in some.
D001763:Blepharoptosis / Q000175:diagnosis*; D002648:Child; D002675:Child, Preschool; D002800:Cholinesterase Inhibitors / Q000627:therapeutic use; D004359:Drug Therapy, Combination; D005260:Female; D005500:Follow-Up Studies; D005938:Glucocorticoids / Q000627:therapeutic use; D006801:Humans; D016756:Immunoglobulins, Intravenous / Q000627:therapeutic use; D007223:Infant; D008297:Male; D009157:Myasthenia Gravis / Q000175:diagnosis* / Q000188:drug therapy; D015835:Ocular Motility Disorders / Q000175:diagnosis*; D011729:Pyridostigmine Bromide / Q000627:therapeutic use; D012189:Retrospective Studies; D013285:Strabismus / Q000175:diagnosis*; D013934:Thymectomy; D016896:Treatment Outcome; D014792:Visual Acuity
D002800:Cholinesterase Inhibitors; D005938:Glucocorticoids; D016756:Immunoglobulins, Intravenous; D011729:Pyridostigmine Bromide