Prospective, Randomized, Double- Masked Clinical Trial Comparing Safety and Efficacy of Topical Tacrolimus vs. Methylprednisolone for Ocular Graft-Versus-Host Disease

PURPOSE: To evaluate the safety and efficacy of topical tacrolimus 0.05% vs. topical methylprednisolone 0.5% in patients with ocular graft‐versus‐host disease. METHODS: Prospective, randomized, double masked clinical trial evaluating 40 patients treated with topical tacrolimus 0.05% (N=24) or methylprednisolone 0.5% (N=16) twice a day for 10 weeks. The following clinical signs were evaluated: corneal fluorescein staining (CFS), tear break‐up time (TBUT) and Schirmer test. Symptoms were evaluated using the Ocular Surface Disease Index (OSDI). Tolerability was assessed based on subjects' reports of discomfort after drop instillation; adverse events and intraocular pressure (IOP) were also assessed. RESULTS: No major adverse events were reported during the study. There was no significant difference in the tolerability scores between the two groups (P=0.053); however, burning sensation upon drug instillation was more frequent in the tacrolimus vs. methylprednisolone (P<0.001). Topical tacrolimus was more effective than methylprednisolone in reducing the CFS score (55% vs. 23%, respectively; P=0.015) and increasing the TBUT (P<0.001) after 10 weeks of treatment. There was a statistically significant reduction of OSDI with tacrolimus (P=0.024) but not with methylprednisolone (P=0.057). The Schirmer scores did not change in either group. Treatment with methylprednisolone modestly increased IOP when compared to baseline values (P=0.04). CONCLUSIONS: Topical tacrolimus 0.05% is safe, effective and generally well tolerated for the treatment of ocular graft‐versus‐host disease, without the hypertensive side effect of topical corticosteroid therapy.