Sirolimus Combined With Low-dose Warfarin for the Treatment of Refractory PNH
Status:
Not yet recruiting
Trial end date:
2022-04-01
Target enrollment:
Participant gender:
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.