Sirolimus Combined With Low-dose Warfarin for the Treatment of Refractory PNH

Summary

Classical paroxysmal nocturnal hemoglobinuria(PNH) is mainly characterized by hemolysis and thrombosis, which reduced patients ' quality of life(QoL) greatly and even lead to death. Glucocorticoids and symptomatic supportive therapy are traditional treatments and the response rate is far from satisfactory. Eculizumab is an effective therapy but it is expensive and not available in China mainland.The investigators aim to explore the efficacy and safety of sirolimus for refractory classic PNH.

Timeline

Start

2019-04-01

Primary completion

2021-03-01

Completion

2022-04-01

Drugs

Indications