An open-label phase 2 study of topical
dexamethasone versus
tacrolimus solutions in new-onset oral
chronic graft-versus-host disease (cGVHD) revealed the superior efficacy of
dexamethasone. The objective of this study was to report long-term patterns of topical
therapy utilization and clinical outcomes in this cohort after completing the 30-day trial. A retrospective record review was performed from the date of study completion to January 2017. Topical
therapies, systemic immunosuppressive therapies, objective measurements (National Institutes of Health severity score, oral mucosal scores), patient- reported outcomes (dryness, sensitivity,
pain), and adverse events were recorded for oral cGVHD-related outpatient visits. Follow-up (FU) periods were defined as FU1 (0-1 month), FU2 (1-3 months), FU3 (3-6 months), FU4 (6-12 months), FU5 (12-18 months), and FU6 (18-24 months). Forty patients (52.5% males, median age, 56 years) completed the clinical trial and were included in the analysis. Topical
therapies used were
dexamethasone,
tacrolimus,
clobetasol, or a combination of these agents. At FU1, all 40 patients were receiving topical
therapy, which decreased to 54.5% (12 out of 22) at FU6. Clinician-reported oral mucosal scores (0-12) and patient-reported sensitivity scores (0-10) decreased over time from FU1 (median mucosal score, 3; sensitivity, 3) to FU6 (mucosal score, 1; sensitivity, 2). Intralesional
steroid therapy was provided to 6 patients for management of refractory oral ulcerations, all within the first year of follow-up. Patients with de novo symptomatic oral cGVHD may require
long-term care with topical
immunomodulatory therapy for up to 2 years, if not longer. Topical
steroid and
tacrolimus therapies are safe and effective in managing symptomatic oral cGVHD. Second-line topical
therapy for refractory oral cGVHD requires further investigation.