Sarju S. Patel, MD, MPH,1,2 Emilio M. Dodds, MD,3 Laura V. Echandi, MD,3 Cristobal A. Couto, MD,4 Ariel Schlaen, MD,4 Howard H. Tessler, MD,1 Debra A. Goldstein, MD1,5
Objective: To evaluate the safety and effectiveness of short-term, high-dose chlorambucil therapy in achieving long-term, drug-free remission in the treatment of sympathetic ophthalmia (SO).
Design: Retrospective case series.
Participants: Sixteen patients with SO treated with high-dose, short-term chlorambucil therapy between 1970 and 2010.
Methods: Descriptive and bivariate analyses were used to characterize disease and outcomes.
Main Outcome Measures: Months of disease-free remission, prevalence rate of relapse, and prevalence of serious treatment-related adverse events.
Results: Sixteen patients with SO treated with short-term, high-dose chlorambucil were identified. Patients were treated with chlorambucil for a median of 14.0 weeks (mean, 14.5 weeks; range, 12.0e19.0 weeks). Median follow-up was 98.5 months (mean, 139.1 months; range, 48e441 months) from initiation of chlorambucil therapy. Control of inflammation was achieved in 100% of patients. Thirteen patients (81.3%) maintained vision of 20/40 or better in the sympathizing eye. Four patients (25%) relapsed after a median of 83 months (mean, 131 months) after cessation of systemic therapy. Seventy-five percent of relapses were controlled with topical therapy only. Conjunctival Kaposi’s sarcoma developed in 1 patient. No patient demonstrated systemic malignancy.
Conclusions: Short-term, high-dose chlorambucil therapy provides sustained periods of drug-free remis- sion. With median follow-up of more than 8 years (mean, 11.6 years; range, 4e37 years), there was a low rate of recurrence and minimal long-term serious health consequences or adverse events. Because SO may be a lifelong condition and because chlorambucil therapy may offer long-term, drug-free remission, this treatment may be worth considering early in the decision-making process for severe sight-threatening disease.
Financial Disclosure(s): The author(s) have no proprietary or commercial interest in any materials discussed in this article. Ophthalmology 2013;-:1e7 a 2013 by the American Academy of Ophthalmology.