Use of leflunomide for the management of Ocular mucous membrane pemphigoid: a multicentric retrospective cohort study

BACKGROUND: Ocular mucous membrane pemphigoid (OcMMP) is a chronic autoimmune scarring disease that may lead to progressive visual impairment. Evidence regarding the use of leflunomide (LFN) in OcMMP is limited. RESEARCH DESIGN AND METHODS: We conducted a retrospective, observational multicenter study including adult patients with OcMMP treated with LFN between May 2006 and June 2025 at rheumatology centers in Argentina. Clinical characteristics, treatment regimens, and outcomes were collected from medical records. Disease severity and progression were assessed using Foster staging. LFN was used as second-line therapy after methotrexate failure, as monotherapy or in combination. Treatment effectiveness at 6 months was defined as suppression of ocular inflammation and achievement of a glucocorticoid-sparing effect. Treatment failure included persistent inflammation, discontinuation due to adverse events, lack of steroid-sparing effect, or need for additional immunomodulatory therapy. RESULTS: Thirty-six patients (mean age 67.1 years) were included. At 6 months, 61.1% achieved inflammatory control with glucocorticoid reduction or discontinuation; overall effectiveness during follow-up was 55.5%. Foster stage progression occurred in 8.5%. Adverse events leading to discontinuation were infrequent. CONCLUSIONS: LFN may be a safe and effective option for selected OcMMP patients, although the retrospective design and limited sample size warrant prospective studies.