Low-dose pulsed intravenous cyclophosphamide for severe ocular cicatricial pemphigoid in elderly patients

Ocular cicatricial pemphigoid (OCP) is a relatively rare autoimmune disease affecting elderly patients and causing severe symptoms that may culminate in blindness. Treatment is based on immunosuppression, but optimal regimens have not been established.

A prospective unmasked case series of all patients with severe OCP who gave consent and completed treatment and ≥6 months of follow-up in the cornea and immunomodulation outpatient clinics of an academic hospital. Monthly pulses of intravenous cyclophosphamide (IVC; 500 mg) were administered with ondansetron and adjusted according to response.

Over 14 years, 13 patients (median age 77 years) met inclusion criteria. All had bilateral OCP (2 eyes were previously blind), but extraocular involvement was rare (1/13). Three to 28 pulses were given, and the patients were followed up for a median of 32 (range, 6-167) months. Remission of inflammation in both eyes was achieved in 12 patients (92%). Vision improved in 5 of 13 patients, stabilized in another 5 (combined, 77%), and worsened in only 3 patients. One patient's condition flared up during treatment that responded to steroids and increasing IVC frequency. In 4 patients and 6 eyes (25%), cicatrization progressed (usually, Foster stages 1-3). Two late relapses occurred and responded to retreatment. IVC was generally well tolerated, although nausea led to modification in 2 patients. One patient developed candida keratitis.

Compared with other treatment modalities, low-dose monthly pulse IVC is found to be a relatively safe, simple, and usually effective alternative immunosuppressive treatment in severe OCP.