Pulse IV cyclophosphamide in ocular inflammatory disease: efficacy and short-term safety

To assess the efficacy and short-term safety of appropriately monitored pulse IV cyclophosphamide therapy in the treatment of patients with severe or treatment-resistant autoimmune ocular inflammatory disease.

Retrospective noncomparative interventional case series.

Thirty-eight patients with severe or recalcitrant ocular inflammation of diverse etiologies.

Charts of patients seen on the Ocular Immunology & Uveitis Service at the Massachusetts Eye & Ear Infirmary were reviewed. Thirty-eight consecutive patients treated with pulse IV cyclophosphamide between January 1995 and March 2002 were analyzed.

The control of inflammation, steroid-sparing effect, visual acuity, and adverse reactions.

A positive response to treatment occurred in 68% of patients during the study period, with 55% achieving complete quiescence. A steroid-sparing effect was achieved in all patients previously on systemic steroid, allowing successful discontinuation of the drug in 41%. Visual acuity was maintained in 66% and improved in 21% of involved eyes. The most common side effects observed were fatigue (63%), nausea (32%), and headache (22%). None required a permanent discontinuation of therapy.

Pulse IV cyclophosphamide is an effective therapeutic modality in patients with severe or treatment-resistant ocular inflammatory disease.