pediatric acquired severe aplastic anemia
Conditions
Interventions
Experimental group:Anti-thymocyte globulin+Ciclosporin A+Mesenchymal stem cell
Control group:Anti-thymocyte globulin+Ciclosporin A
Sponsors
Institute of Hematology & Blood Disease Hospital Chinese Academy of Medical Sciences
Eligibility
Sex/Gender
All
Age
1 Years to 16 Years
Inclusion criteria
Inclusion criteria: To be included in the study, patients must meet all of the following inclusion criteria: 1.Aged > 1 and = 25% normal but < 50%, the remaining hematopoietic cells should be < 30%; Blood images need to specify two of the following three items: Absolute value of neutrophils < 0.5 x 10^9/L; Platelet count < 20 x 10^9/L; Absolute value of reticulocyte < 20 x 10^9/L. 3.Unsuitable or unwilling to perform hematopoietic stem cell transplantation (HSCT) directly; 4.The guardians are willing to let the pupillus participate and sign the informed consent.
Exclusion criteria
Exclusion criteria: Patients with any of the following were not included in the study: 1.Previous history of hematopoietic stem cell transplantation; 2.Patients with uncontrolled bleeding and/or infection after standard treatment before enrollment; 3.Previous HIV infection or carrier or hepatitis C antibody positive at the time of screening; 4.Previous history of cirrhosis or portal hypertension; 5.Subjects who have developed malignant solid tumors of any organ system within the previous 5 years, regardless of whether they have been treated, metastatic or recurrent; Subjects found to have hematologic tumors in the past or during screening; 6.Participated in other clinical trials within the previous 3 months; 7.The Investigator considers that there are any other conditions which may cause the subject to be unable to complete the study or which pose a significant risk to the subject; 8.Patients with congenital aplastic anemia.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Complete response rate (CR) at 6 months of administration; | — |
Secondary
| Measure | Time frame |
|---|---|
| Partial hematologic response rate (PR) and total hematologic response rate (OR) at 6 months of administration;Time required for the first hematologic response (CR or PR) within 6 months of administration;Proportion of subjects who were removed from red blood cell infusion at 3 and 6 months of administration;Proportion of subjects disengaged from platelet transfusion at 3 and 6 months of administration; | — |
Countries
China
Contacts
Public ContactZhu Xiaofan
Institute of Hematology & Blood Disease Hospital Chinese Academy of Medical Sciences
Outcome results
None listed