- Research Article
- 10.2147/opth.s514118
Tear Biomarkers of Topical Sirolimus in Meibomian Gland Dysfunction: A Randomized Trial
- Mar 27, 2026
- Clinical Ophthalmology (Auckland, N.Z.)
- Lei Zhou + 7 more +7
PurposePharmacodynamic biomarkers of sirolimus were investigated using omics analysis of tear fluids from Japanese patients with meibomian gland dysfunction (MGD).MethodsIn a Phase 2a trial, sirolimus or vehicle eyedrops were administered twice daily for 12 weeks. Tear samples from 29 patients (15 sirolimus, 14 vehicle) were collected pre- and post-treatment. LC-MS/MS-based proteomics and lipidomics were performed. Within-group changes were analyzed, followed by differential expression and pathway analysis. Key candidates were evaluated using estimation plots (Registration ID: UMIN000049186).ResultsOver 3000 proteins and 55 lipids were quantified. mTOR signaling components (ATP6V1D, RRAGC, DEPTOR) were significantly modulated. ATP6V1D showed a significant decrease in the sirolimus group (p = 0.0024), but not in the vehicle group (p = 0.528). Lipids 12-HETE and 13-HpODE significantly increased post-treatment in the sirolimus group.ConclusionResults suggested that sirolimus inhibited the mTOR pathway. ATP6V1D, 12-HETE, and 13-HpODE were suggested to be pharmacodynamic biomarkers for sirolimus. These findings may facilitate pharmacodynamic monitoring of mTOR-targeted therapies for ocular surface disorders.
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