Prospective management of biopsy-proven ocular mucous membrane pemphigoid using an inflammation-oriented stepladder treatment strategy

Objective/Purpose To prospectively describe the implementation and clinical outcomes of an inflammation-oriented management strategy for biopsy-proven ocular mucous membrane pemphigoid (MMP), in which the maximum conjunctival inflammation grade of the more severely affected eye was used to assign the initial treatment-intensity tier. Design Prospective cohort study. Participants Forty-eight (48) patients (96 eyes) with biopsy-proven ocular MMP. Methods Patients with biopsy-confirmed ocular MMP were prospectively managed between 2018 and 2025 using an inflammation-oriented stepladder treatment protocol. Moderate to severe cases received intravenous cyclophosphamide or rituximab, while milder cases were treated with oral mycophenolate mofetil. Clinical outcomes, treatment response, disease relapse, and treatment-related adverse events were prospectively recorded. Results Disease activity at presentation was minimal in 1 patient (2.1%), mild in 28 (58.3%), moderate in 7 (14.6%), and severe in 12 (25.0%). . Initial treatment corresponded to the intended inflammation-based tier in 46 patients (95.8%). Complete inflammatory control (grade 0 in both eyes) was achieved in 23 patients (47.9%) at a median of 9 months (range, 3–12 months), and 3 patients (6.3%) subsequently achieved drug-free remission. During follow-up, disease relapse occurred in 6 patients (12.5%), all of whom were successfully managed with escalation of systemic immunosuppression. At final follow-up, no eyes demonstrated moderate or severe inflammation. Treatment-related adverse events were manageable, with mycophenolate mofetil dose reduction required in 5 patients (10.4%). No treatment-related deaths occurred. Conclusion This prospective study supports the feasibility of an inflammation-oriented management protocol for biopsy-proven ocular MMP. Future comparative studies are warranted to validate this approach and determine its broader clinical utility.