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.