rapamycin + methylprednisolone

Rapamycin (sirolimus) in combination with methylprednisolone is a therapeutic regimen being investigated for the treatment of moderate to severe active Graves' ophthalmopathy (GO). Rapamycin is a small molecule immunosuppressant that acts as an mTOR inhibitor; it binds to the intracellular protein FKBP12 to form a complex that inhibits the mechanistic target of rapamycin complex 1 (mTORC1), thereby suppressing T-cell activation and orbital fibroblast proliferation. Methylprednisolone is a synthetic glucocorticoid that acts as an agonist of the glucocorticoid receptor, providing potent anti-inflammatory and immunosuppressive effects. This combination is being studied by the First Affiliated Hospital of Xi'an Jiaotong University to determine if adding oral rapamycin to standard intravenous methylprednisolone pulse therapy improves clinical outcomes, such as the Clinical Activity Score (CAS) and proptosis, in patients with active GO.