Sever aplastic anemia
Conditions
Interventions
Trial group:Standard immunosuppressive therapy (rabbit ATG 3mg/kg/d for 5 days + cyclosporine starting at 5mg/kg/d adjusted to blood drug concentration 150-250ug/L + heltrombopag 15mg/d for 3 months)
Sponsors
Blood Disease Hospital, Chinese Academy of Medical Sciences (Institute of Hematology, Chinese Academy of Medical Sciences)
Eligibility
Sex/Gender
All
Age
18 Years to 60 Years
Inclusion criteria
Inclusion criteria: 1. 18-60 years old, regardless of gender; 2. After routine blood tests, bone marrow puncture, and biopsy, the patient was diagnosed with acquired SAA and ruled out congenital and other acquired or secondary bone marrow hematopoietic failure (BMF); 3. Have not received ATG treatment in the past, or have not been treated with cyclosporine (CsA) for more than 6 months; 4. No HLA matched blood donors; 5. ECOG score for physical condition (Appendix 2) 0-2 levels; 6. No significant abnormalities in liver and kidney function: ALT and AST are within 2.5 times the normal upper limit, while serum Cr and BUN are within 1.25 times the normal upper limit; 7. The electrocardiogram shows no serious abnormalities, and according to the New York Heart Association (NYHA) grading criteria (Appendix 3), the heart function is <= grade 2; 8. No severe infection in the lungs, normal function; 9. Sign an informed consent form based on the patient's personal wishes with full knowledge.
Exclusion criteria
Exclusion criteria: 1. Aplastic anemia caused by malignant tumor treatment, or patients with concurrent malignant tumors; 2. The patient tested positive for four infectious diseases; 3. Patients with severe or difficult to control infections; 4. The patient has a highly allergic constitution or is severely allergic to blood products/biologics; 5. Pregnant or lactating women; 6. The patient clearly does not have compliance to complete the experimental plan (such as suffering from uncontrollable mental illness); 7. The patient also participated in other clinical studies; 8. Other researchers consider patients unsuitable for inclusion.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The overall response rate (ORR) at 4 weeks, 8 weeks, and 12 weeks after IST;The complete response rate (CRR) at 4 weeks, 8 weeks, and 12 weeks after IST; | — |
Secondary
| Measure | Time frame |
|---|---|
| Recovery time of neutrophils, hemoglobin and platelets;Complication rate (including seropathy, infection, bleeding, liver and kidney function damage, etc.);Recurrence rate; | — |
Countries
China
Contacts
Public ContactLi Jianping
Blood Disease Hospital, Chinese Academy of Medical Sciences (Institute of Hematology, Chinese Academy of Medical Sciences)
Outcome results
None listed