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The application of methotrexate (MTX) in the treatment of autoimmune diseases has been gradually increasing, but reports of MTX treatment for advanced ocular cicatricial pemphigoid (OCP) are extremely rare. This study investigated the efficacy and adverse reactions of low-dose MTX in patients with OCP.This was a retrospective, noncontrolled, case series study. Eleven patients diagnosed with advanced OCP (4 cases in stage III and 7 cases in stage IV) were enrolled. Treatment by oral administration of MTX (7.5 ± 2.5 mg) alone was performed. Visual acuity of the patients, conjunctival inflammation, cicatrization, ocular surface keratinization, and toxic side effects of drugs were evaluated.All patients enrolled in this study were females aged 32 to 83 years. Patients were followed up for 4 to 33 months. Low-dose MTX improved visual acuity of 3 cases (6 eyes, 27.3%). Conjunctival inflammation of 5 patients (10 eyes) rested after treatment, and conjunctival inflammation of 3 cases (6 eyes, 27.3%) was decreased with an effective rate of 72.7%. Cicatrices of 8 cases (15 eyes) showed degeneration after treatment with an effective rate of 71.4% (15/21). Ocular surface keratinization receded in 4 cases with an effective rate of 66.7%. None of the patients discontinued the treatment due to severe toxic side effects. All patients tolerated mild drug-induced gastrointestinal reactions. Three patients terminated the treatment in advance after 4 to 6 months due to no improvement in the disease condition.Observation of clinical efficacy and safety findings demonstrated that low-dose MTX can be used to treat patients with advanced OCP.

Efficacy and adverse reactions of methotrexate in the treatment of ocular cicatricial pemphigoid