To evaluate the effectiveness and toxicity of a stepladder immunosuppression strategy, including the use of mycophenolate mofetil and combination therapy, in the treatment of ocular mucous membrane pemphigoid.

Retrospective, noncomparative, interventional case series.

Two hundred twenty-three eyes of 115 patients.

Patients with a diagnosis of ocular mucous membrane pemphigoid commencing immunosuppression between January 1994 and July 2005 were identified. A treatment episode was defined by the use of a particular therapy or combination of therapies.

For each treatment episode, success of immunosuppressive therapy in controlling ocular inflammation was graded as a success (S), qualified success (QS), or failure (F). Initial and final visual acuities (VAs), stage of cicatrization (Foster, Mondino), grade of conjunctival inflammation, and side effects were recorded.

In 70% (80/115) of patients, inflammation was controlled by the end of the study. At least 6 months remission off treatment occurred in 16 patients (14%). Of the 388 treatment episodes, 50% were classified as S; 27%, QS; and 23%, F. The most successful therapies were based on cyclophosphamide (S, 69%; QS, 21%; F, 10%), followed by mycophenolate (S, 59%; QS, 22%; F, 19%), azathioprine (S, 47%; QS, 24%; F, 29%), dapsone (S, 47%; QS, 30%; F, 23%), and sulfapyridine (S, 38%; QS, 27%; F, 35%). Combination sulfa–steroid–myelosuppressive agent therapy increased the response from 73% with single-agent therapy to 87%. Side effects were the reason for 29% of changes in therapy. These were most prominent with azathioprine (40%) and least with mycophenolate (15%). Initial best-corrected VA (BCVA) was 6/60 or less in 17% (37/223) of eyes, pemphigoid being the cause in 13% (29/223). Final BCVA was 6/60 or less in 34% (76/223) of eyes, pemphigoid being the cause in 26% (57/223). By the end of the study, Mondino stage cicatrization had progressed in 41% (92/223) of eyes and 53% (61/115) of patients.

Mycophenolate mofetil seems to be an effective and well-tolerated immunosuppressant for moderately active ocular mucous membrane pemphigoid. Combination sulfa–steroid–myelosuppressive agent therapy in a stepladder regimen is a useful strategy to improve disease control. Cicatrization and VA may still progress and worsen despite adequate control of inflammation.