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

• Status: Not yet recruiting
• Trial end date: 2022-04-01
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 4
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Peking Union Medical College Hospital
• Treatments: Everolimus; Sirolimus; Warfarin

Criteria

Inclusion Criteria:

• age 18-70 years

• diagnosed with PNH and no thrombosis

• ineffective, relapsed or intolerant to conventional treatment (eg glucocorticoids, • • iron, folic acid, androgen, etc.)

• not available for hematopoietic stem cell transplantation

• ECOG≤2

• agreed to sign the consent forms

Exclusion Criteria:

• severe heart, liver and kidney dysfunction

• combined with thrombotic complications

• people who are pregnant and breastfeeding

• history of other immunosuppressive agents in recent 3 months

• Patients who are not eligible to participate in this trial due to any reason based • •
• on the consideration of investigator