Abstract
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.