Mycophenolate mofetil for the treatment of cicatrizing conjunctivitis secondary to ocular pemphigoid

•Most patients demonstrated a clinical response to mycophenolate mofetil.•Side effects were common but generally mild.•Discontinuation was most commonly due to side effects or inadequate response. To characterize the use of mycophenolate mofetil (MMF) in a large cohort of patients undergoing treatment for ocular mucous membrane pemphigoid (ocMMP). This was a retrospective consecutive observational chart review of patients with cicatrizing conjunctivitis associated with pemphigoid (ocMMP) at the University of Texas Southwestern Medical Center in Dallas, TX from 2016 to 2023 who underwent treatment with MMF. Data collection included patient demographics, medical and ocular history, clinical findings, slit lamp photographs, biopsy results, additional testing, disease stage, time on MMF, patient response to treatment, and side effects of treatment. The data was analyzed with logistic regression using IBM SPSS (version 29). The dependent variable was remission status. A total of 52 patients with cicatrizing conjunctivitis associated with pemphigoid were treated with MMF, of which 37 (71.2 %) patients had biopsy proven disease. Of the cohort, 29 (55.8 %) patients were female and 23 (44.2 %) patients were male. The mean age at initiation of MMF therapy was 65.1 years (range 35–93). The best degree of response achieved by each patient in our cohort was as follows: no response (4, 7.7 %), partial response (28, 53.8 %), clinical remission with steroids (4, 7.7 %), steroid-free remission (14, 26.9 %), and durable drug-free remission (2, 3.8 %). The mean time of treatment with MMF was 35.5 months (range 2–118), with a mean follow up time of 42.7 months (range 3–123). The mean time to clinical remission was 6.7 months (range 1–18), while the mean time to steroid-free remission was 7.2 months (range 2–18). Of the entire cohort, 16 (30.8 %) patients reported side effects. The most common side effect was nausea (7 patients). There was one adverse event (anemia, 1, 1.9 %). Overall, 30 (57.7 %) patients discontinued MMF, most commonly due to side effects (11 patients) or inadequate disease control (11 patients). Mycophenolate mofetil (MMF) can be beneficial in ocMMP as a steroid-sparing alternative. Although effective, side effects and discontinuation of treatment were common.