Lifitegrast ophthalmic solution for treatment of ocular chronic graft-versus-host disease.

Ocular chronic graft-versus-host disease (oGVHD) is a relatively common complication that occurs following allogeneic hematopoietic cell transplantation. Keratoconjunctivitis sicca (KCS) is the most common manifestation of oGVHD. Lifitegrast is a lymphocyte function-associated antigen-1 antagonist approved to reduce inflammation and symptoms in patients with dry eye disease. We evaluated the efficacy and safety of lifitegrast (5% ophthalmic solution) in patients with ocular GVHD in a single-institution retrospective cohort study of eighteen allogeneic transplant recipients who received topical lifitegrast for oGVHD treatment. The outcome of interest was improvement in oGVHD severity score by National Institutes of Health (NIH) criteria. Lifitegrast was well-tolerated and no serious adverse events were observed. Lifitegrast significantly improved NIH severity scores in 8 (44%) patients. The findings of this study suggest lifitegrast is safe, well-tolerated and is an effective option for oGVHD manifesting as KCS. Prospective evaluation is warranted to confirm efficacy of lifitegrast in this population.HighlightsIn this single-institution retrospective cohort study of eighteen patients who received allogeneic transplant between 2013 and 2018, and received topical lifitegrast for treatment of ocular GVHD, the results demonstrate that lifitegrast eye drops were well-tolerated and led to improvement in symptoms of KCS in 8 (44%) patients.Lifitegrast has the potential to fulfill an unmet need in allogeneic transplant patients with ocular GVHD. Further prospective study is warranted for confirmation.