Off-label cyclosporine: Effective for diseases other than dry eye

The effectiveness of cyclosporine (Restasis, Allergan) is not limited to patients with dry eye. Numerous researchers have reported other uses for the drug, according to Ana Luisa Höfling-Lima, MD, chairwoman and professor, Federal University of São Paulo, Brazil.

"The FDA has approved the use of cyclosporine 0.05% to treat chronic dry eye," Dr. Höfling-Lima said. "An off-label use of the drug is for chronic and acute inflammation of the cornea and conjunctiva. For chronic dry eye, tears help, but cyclosporine helps return the ocular surface to normal with increased goblet cell density."

Cyclosporine also has been used for a number of other diseases on an off-label basis, Dr. Höfling-Lima said. For fungal infections, cyclosporine 0.05% has a moderate effect on fungal growth when used with antifungal drugs, for example. For epidemic keratoconjunctivitis, cyclosporine decreases or eliminates the infiltrates and the visual acuity increases; the mechanism of action results in changes in the local immune reaction of the host. Cyclosporine is also useful in the treatment of stromal keratitis. In Stevens-Johnson syndrome, cyclosporine used in the acute phase inhibits CD8 activation.

When used to prevent graft rejection in patients at low risk for rejection after corneal transplantation, a study published in Ophthalmology in 2006 found that topical cyclosporine 0.05% was not as effective as topical prednisolone in preventing graft rejection.

"There is a lack of randomized studies of cyclosporine," Dr. Höfling-Lima concluded. "Such studies will provide a better understanding of the mechanisms of the drug and result in new diseases that can be treated with cyclosporine."