transfusion-dependent thalassemia
Conditions
Sponsors
The 923rd Hospital of the Joint Logistics Support Force of the People's Liberation Army
Eligibility
Sex/Gender
All
Age
3 Years to 25 Years
Inclusion criteria
Inclusion criteria: 1. The patient was clinically and genetically diagnosed as a TDT patient; 2. Age range from 3 to 25 years old (including 3 and 25 years old), male and female; ECOG physical strength score 0~2; 3. Signed written informed consent before the test. 4. Good organ function: i. Left ventricular ejection fraction (LVEF) =40% indicated by echocardiography; ii. Serum total bilirubin 60 mL/min. 5. The patient was in stable condition, and the investigators judged that it was suitable to receive cyclophosphamide, cestipide, and butoxamide pretreatment;
Exclusion criteria
Exclusion criteria: 1. History of uncontrollable epilepsy, mental disorders or other mental diseases; 2. Pregnant and lactating women and subjects of childbearing age who do not want to take contraceptive measures; 3. Patients with severe cardiopulmonary disease or abnormal liver function; Complicated with cerebrovascular, cardiovascular, liver, kidney, tumor and other serious primary diseases; Anemia caused by other causes; 4. Patients receiving any other investigational drug; 5. History of malignant tumor (other than skin cancer or cervical intraepithelial neoplasia) or family history of hereditary familial cancer syndrome; 6. Previously received allogeneic bone marrow transplantation or gene therapy; 7. There is bleeding disease that cannot be corrected; 8. There are other diseases or causes that interfere with the research process; 9. The researcher decides that it is not suitable to participate in this study.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The 1-year survival rate after transplantation;The 2-year survival rate after transplantation; | — |
Secondary
| Measure | Time frame |
|---|---|
| Implantation rate at 28 days after transplantation;Incidence of aGVHD 100 days after transplantation;Incidence of cGVHD 1 year and 2 years after transplantation; | — |
Countries
China
Contacts
Public ContactYin xiaolin
The 923rd Hospital of the Joint Logistics Support Force of the People's Liberation Army
Outcome results
None listed