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S. Pujari, J. H. Kempen, C. W. Newcomb, E. B. Suhler, J. E. Thorne, D. A. Jabs, G. A. Levy-Clarke, R. B. Nussenblatt, J. T. Rosenbaum, C. S. Foster; Cyclophosphamide for Ocular Inflammatory Diseases. Invest. Ophthalmol. Vis. Sci. 2009;50(13):2020.
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To evaluate the effectiveness and safety of cyclophosphamide as an immunosuppressive treatment for non-infectious ocular inflammation.
A retrospective cohort study of 251 patients with non-infectious ocular inflammation started on cyclophosphamide between 1979-2005 inclusive at five tertiary uveitis clinics was conducted by chart review. Dose of cyclophosphamide, response to therapy, corticosteroid-sparing effectiveness at every visit were noted. The frequency of discontinuation of cyclophosphamide and reasons for discontinuation also were noted. Proportions with success or failure at 6 and 12 months were estimated using Kaplan-Meier methods, and risk factors assessed using Cox regression.
Among the 251 patients (452 eyes) meeting inclusion criteria, 3.6%, 4%, 18.3%, 23.9%, 40.2%, and 10% had anterior uveitis, intermediate uveitis, posterior or panuveitis, scleritis, ocular mucous membrane pemphigoid, and other forms of ocular inflammation respectively. Among those active initially, control of inflammation ≥2 visits spanning ≥28 days was estimated to occur within ≤12 months in 65% overall. Among those on >10 mg of prednisone initially, sustained control of inflammation on a dose of ≤10 mg/day of prednisone within 12 months was 54%. Cyclophosphamide was discontinued in 193 patients (77%), most commonly due to side effects (81 patients, 32%) usually of a reversible nature, although life-threatening side effects were observed rarely. Sixty-one patients (32%) discontinued treatment because of disease remission.
Our data suggest that cyclophosphamide as a single immunosuppressive agent was effective for controlling ocular inflammation in approximately two-thirds of patients within one year, and effective in meeting corticosteroid-sparing objectives in the majority of patients by one year. However, results were substantially better using less stringent success criteria. A minority of patients were able to discontinue therapy in disease remission. Side effects observed during follow-up at the clinics usually were reversible with dose adjustment, but require a high level of attention because life-threatening side effects occur occasionally.
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