Background The combination of topical interferon α-2b (IFN α-2b) and all-trans retinoic acid (ATRA) 0.01% has previously been shown to be effective in conjunctival and corneal intraepithelial neoplasia. This combination was incidentally found to be effective in a patient with partial limbal stem cell deficiency (LSCD), a condition which can be challenging to treat if conservative measures fail. This retrospective study evaluates the combination of topical IFN α-2b and ATRA 0.01% in the treatment of partial LSCD.

Methods Five patients from one institution with a clinical and/or histopathological diagnosis of LSCD had failed a period of conservative treatment with cessation of toxic stimuli and use of lubricating eye-drops. These patients were treated with a combination regimen of topical IFN α-2b and ATRA 0.01%.

Results All five patients had partial LSCD, but limbal involvement was significantly worse in one patient who later progressed to total LSCD. Complete clinical resolution of signs of LSCD was achieved in the four patients with partial LSCD after a mean of 9 months of treatment. The one patient who progressed to total LSCD did not respond to treatment. Duration of follow-up after clinical resolution in the four patients with partial LSCD was at least 18 months, with no signs of recurrence seen. Aside from the complaint of ocular irritation in one patient, no other side effects were observed.

Conclusions Combination treatment of topical IFN α-2b and ATRA 0.01% can be considered in partial LSCD, where adjacent parts of the limbus remain intact. This treatment is associated with minimal side effects and no recurrence of signs of LSCD at least 18 months after clinical resolution.