Purpose: To determine the effectiveness of short-term high-dose chlorambucil in the treatment of sight-threatening uveitis and to ascertain the incidence of severe side effects, particularly late malignancy. Design: Retrospective, noncomparative interventional case series. Participants: Fifty-three patients treated at the University of Illinois at Chicago Eye and Ear Infirmary and the private office of one of the authors for severe sight-threatening uveitis. Methods: Treatment with short-term high-dose chlorambucil (2-9 months of therapy). Main Outcome Measure: Visual acuity and degree of inflammation were assessed at every visit. The development of systemic side effects, including malignancy, was assessed using a detailed questionnaire. Results: Total cumulative dose of chlorambucil ranged from 392 to 5200 mg with an average of 1429 mg. The maximum daily dose ranged from 10 to 30 mg with an average of 20 mg. Average duration of treatment was 16 weeks with a range of 7 to 40 weeks. Seventy-seven percent of patients treated were in remission with an average follow-up of 4 years (range: 6 months to 24 years). Forty-seven percent had at least two lines of improvement in Snellen visual acuity after treatment, with an average gain of 3.5 lines. Adverse effects include secondary amenorrhea, nonophthalmic herpes zoster, testicular atrophy, and erectile dysfunction. None of the patients had developed a malignancy as of their last follow-up. Conclusion: Short-term high-dose chlorambucil therapy may be a reasonable option in patients with intractable sight-threatening uveitis.
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