Study of Sirolimus in CTD-TP in China

Summary

We have designed a single-arm, open-label study of sirolimus in Chinese patients with refractory CTD-TP unresponsive to, or intolerant of, conventional medications at Peking Union Medical College Hospital (PUMCH). Patients received oral sirolimus at a starting dose of 2 mg per day for 3 days and a sequential dose of 1mg per day, with dose adjusted according to tolerance and to maintain a therapeutic range of 6-15 ng/mL. Patients were treated with sirolimus for more than 6 months. Safety outcomes and the efficacy endpoints were assessed in all patients who received at least one dose of treatment.

Timeline

Start

2017-09-21

Primary completion

2018-12-31

Completion

2019-09-30

Publications

• Wu C, Wang Q, Xu D, Li M, Zeng X. Sirolimus for patients with connective tissue disease-related refractory thrombocytopenia: a single-arm, open-label clinical trial. Rheumatology (Oxford). 2021 Jun 18;60(6):2629-2634. doi: 10.1093/rheumatology/keaa645.

Drugs

Indications