Abstract
PURPOSE: To evaluate the incidence and clinical characteristics of ocular graft-versus-host disease (oGVHD) associated with tapering of systemic immunosuppression after allogeneic hematopoietic stem cell transplantation (allo-HSCT).
METHODS: This retrospective study included adult allo-HSCT recipients diagnosed with oGVHD during or after immunosuppression tapering at a single tertiary center (1998-2023). Data collected included transplant indications and donor characteristics, GVHD prophylaxis regimens, immunosuppression status at engraftment and at oGVHD onset, and systemic and ocular GVHD manifestations. Cumulative incidence of oGVHD was estimated using Kaplan-Meier survival analyses.
RESULTS: Fifty-six patients (mean age, 56 ± 12 years; 59% male) developed oGVHD at a median of 9 months posttransplant (interquartile range, 6.8-13). The predominant prophylaxis regimen at engraftment was tacrolimus with methotrexate (62.5%). At the time of oGVHD diagnosis, 25.0% of patients were fully tapered off systemic immunosuppression, whereas 28.5% remained on tacrolimus monotherapy. Kaplan-Meier analysis showed a rapid rise in oGVHD incidence within the first year, with 75% of cases diagnosed by month 14. Systemic GVHD preceded ocular involvement in 62% of patients, whereas 18% developed isolated oGVHD. Most patients required escalation of ocular surface therapy-including lubrication, punctal occlusion, and topical antiinflammatory or immunomodulatory agents-although no patient required reinstatement or escalation of systemic immunosuppression solely for ocular disease.
CONCLUSIONS: oGVHD frequently emerges during the late phase of systemic immunosuppression tapering, with many cases occurring after complete discontinuation or reduction to monotherapy. Given the high incidence within the first posttransplant year and the occurrence of isolated ocular disease, these findings support routine ophthalmic evaluation for all allo-HSCT recipients as systemic immunosuppression is withdrawn. Early detection and timely escalation of topical therapy are essential to prevent ocular surface damage while permitting safe continuation of systemic tapering.