Purpose: To evaluate the effectiveness of immunosuppressive drug therapy in the treatment of ocular mucous membrane pemphigoid (MMP). Design: Retrospective cohort study. Participants: Ninety-four patients with biopsy-proven ocular MMP seen at the Pemphigoid Clinic at Wilmer Eye Institute from July 1984 through November 2006. Methods: Data recorded included demographics, use and doses of immunosuppressive drugs, response to therapy, and side effects associated with drug use. Main Outcome Measures: Outcome measures included: (1) ocular control, defined as resolution of inflammation and cessation of cicatrization of the conjunctiva; (2) ocular remission, defined as ocular control for 3 months or more after the cessation of immunosuppressive drug therapy; and (3) ocular relapse, defined as the recurrence of ocular disease in either eye after a remission. Results: By 1 year of treatment, 82.9% of patients had complete control of the inflammation, and of these, 86.3% achieved a remission at some point during follow-up. The incidences of ocular control, remission, and relapse were 1.03 (95% confidence interval [CI], 0.78-1.33), 0.50 (95% CI, 0.37-0.67), and 0.04 (95% CI, 0.02-0.09) events per person-years (PY), respectively. Among patients initially treated with prednisone and cyclophosphamide (n = 44), 91% of patients achieved a remission within 2 years after the initiation of immunosuppressive drug therapy. Characteristics at presentation associated with failing to achieve remission in the univariate analysis were trichiasis (relative risk [RR], 0.28; 95% CI, 0.08-097), prior eyelid surgery (RR, 0.11; 95% CI, 0.02-0.78), and esophageal involvement (RR, 0.29; 95% CI, 0.10-0.83). After adjusting for confounding, an initial treatment regimen containing cyclophosphamide and prednisone was associated with a greater likelihood of achieving ocular remission (RR, 8.53; 95% CI, 2.53-28.86; P = 0.001) when compared with other initial treatment regimens. Infections, hematuria, and anemia were the most common side effects observed in patients receiving cyclophosphamide therapy. The rate of discontinuing cyclophosphamide resulting from side effects was 0.20/PY; however, 74% of these patients still achieved remission despite early discontinuation of cyclophosphamide. Conclusions: In patients with ocular MMP, most achieved ocular disease control with immunosuppressive drug therapy. Treatment with cyclophosphamide and prednisone was strongly associated with the development of ocular remission. Financial Disclosure(s): The author(s) have no proprietary or commercial interest in any materials discussed in this article.
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