Combined therapy of prednisone and mTOR inhibitor sirolimus for treating retroperitoneal fibrosis.

ABSTRACT:

Objectives

Retroperitoneal fibrosis (RPF) is a rare autoimmune disease with fibrous tissue growth and inflammation in retroperitoneum. Its current treatments involve long-term uptake of glucocorticoids (e.g., prednisone) for controlling inflammation; however, side effects are common. We strived for an improved therapy for fibrosis remission while reducing side effects.

Methods

We surveyed gene-disease-drug databases and discovered that mammalian target of rapamycin (mTOR) was a key signalling protein in RPF and the mTOR inhibitor compound sirolimus affected many RPF pathways. We designed a therapy combining a gradual reduction of prednisone with a long-term, stable dosage of sirolimus. We then implemented a single-arm clinical trial and assessed the effects in eight RPF pati

SUBMITTER: Gao H 

PROVIDER: S-EPMC10176363 | biostudies-literature | 2023 May

REPOSITORIES: biostudies-literature