Topical Sirolimus in Treating Patients with Refractory Oral Chronic Graft-versus-Host Disease

Inclusion Criteria

• While there will be no restrictions on concurrent systemic medications, all subjects must be on a stable immunomodulatory medication regimen for 7 days prior to beginning the study without plans to adjust doses during the following four-week study period; dose modifications to maintain therapeutic drug levels of immunosuppressants (i.e. tacrolimus and sirolimus) during the study intervention period are allowed and do not constitute a trial violation; changes in medications for non-cGVHD medical conditions will not affect eligibility
• Patients with symptomatic oral chronic graft-versus-host disease (sensitivity score >= 4)
• Stable topical steroid therapy with dexamethasone, clobetasol, or budesonide oral solutions (5 min, four times a day) for seven days prior to study enrollment
• Stable systemic cGVHD medication regimen for seven days prior to study enrollment; dose modifications to maintain therapeutic drug levels of immunosuppressants (i.e. tacrolimus and sirolimus) for the month prior and during the study intervention period are allowed and do not constitute a trial violation
• Women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry and for the duration of study participation; should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately; men treated or enrolled on this protocol must also agree to use adequate contraception prior to the study, for the duration of study participation, and 4 months after completion of topical sirolimus administration
• Ability to understand and the willingness to sign a written informed consent document

Exclusion Criteria

• Patients already using topical sirolimus therapy
• Patients who have an allergy/intolerance to sirolimus
• Sensitivity score =< 3
• Inability to comply with study instructions
• Pregnant or breastfeeding
• Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements
• Pregnant women are excluded; breastfeeding should be discontinued if the mother is treated with sirolimus
• Human immunodeficiency (HIV)-positive participants on combination antiretroviral therapy are ineligible