paroxysmal nocturnal hemoglobinuria
Conditions
Interventions
experimental group:Eculizumab
Sponsors
West China Hospitai,Sichuan University
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Age>= 18years. 2. Flow cytometry evaluation of PNH clone size>=10%. 3. LDH level>=2×ULN. 4. Hb=30mL/min.
Exclusion criteria
Exclusion criteria: 1. Current or previous treatment with a complement inhibitor. 2. History of N.meningitidis infection within 6 months prior to screening and up to first drug administration. 3. Hereditary complement deficiency. 4. HIV infection. 5. Active systemic bacterial, viral, or fungal infection within 14 days before first drug administration. 6. Immunized with a live attenuated vaccine within 1 month before first drug administration. 7. History of malignancy within 5 years prior to screening and up to the first drug administration. 8. History of myelodysplastic syndrome with Revised International Prognostic Scoring System (IPSS-R) prognostic risk categories of intermediate, high, and very high. 9. History of hypersensitivity, allergic, or anaphylactic reactions to any ingredient contained in eculizumab, including hypersensitivity to human,humanized,or murine monoclonal antibodies or known hypersensitivity to any constituent of the product. 10. Concurrent disease, treatment, procedure or surgery, or abnormality in clinical laboratory tests that could interfere with the conduct of the study, may pose any additional risk for the patient, or would, in the opinion of the investigator, preclude the patients safe participation in and completion of the study.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Mean proportion of patients with hemolysis control;The proportion of patients who achieve transfusion avoidance; | — |
Secondary
| Measure | Time frame |
|---|---|
| Proportion of patients with Breakthrough hemolysis;Proportion of patients with stabilized hemoglobin;Mean change in fatigue as assessed through the use of the FACIT-Fatigue; | — |
Countries
China
Contacts
Public ContactChuan He
West China Hospitai,Sichuan University
Outcome results
None listed