Aplastic Anemia
Conditions
Interventions
Sponsors
The First Affiliated Hospital of University of Science and Technology of China (Anhui Provincial Hospital)
Eligibility
Sex/Gender
All
Age
2 Years to 42 Years
Inclusion criteria
Inclusion criteria: 1. A definite diagnosis of severe aplastic anemia (Camitta criteria): (1) Bone marrow cell proliferation =25% normal but =4/6 and (A, B, CW, DRB1) >=5/8 and (A, B, CW, DRB1, DQB1) >=6/10.The total number of nuclear cells (TNC) >=3.0x10^7/kg, the number of CD34+ cells >=1.5x10^7/kg and the tubule recovery rate >=80%; The patients and their family members or their guardians agree to the study and sign the informed consent; 5. No serious organ failure; Within 2 weeks before transplantation, GPT, GGT and bilirubin were less than 2 times of the upper limit of normal, serum creatinine was less than 2 times of the upper limit of normal, and myocardial enzyme was less than 2 times of the upper limit of normal: (1) Heart: at rest, left ventricular ejection fraction (LVEF) was greater than 40%.For patients younger than 13 years of age, echocardiography shortened LFEF by more than 26% in SF; (2) Liver: total bilirubin was less than 3 times of the upper limit of normal for the corresponding age (ULN);Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) were less than 5 times of the corresponding age of ULN.(3) Kidney: for patients aged 13 years or more, serum creatinine was less than 2 ULN; Estimated creatinine clearance > 50ml/min using the Cockcroft-Gault formula and actual body weight .Patients =13 years old: lung carbon monoxide diffusion volume (DLCO) (corrected/adjusted hemoglobin) > 40%, forced expiratory volume 1 second (FEV1) > 50% predicted (without bronchodilator) and forced vital capacity (FVC) > 50% predicted.For patients younger than 13 years of age, pulmonary function tests (PFT) cannot be performed because of age or developmental ability: 1) there is no evidence of dyspnea at rest; 2) no need for oxygen in indoor air; 6. Karnofsky score >=60%; 7. Women of childbearing age must have a negative pregnancy test and agree to use effective contraceptives during treatment and for a subsequent year.
Exclusion criteria
Exclusion criteria: 1. Hereditary bone marrow debilitating diseases; 2. Specific HLA antibodies were positive for HLA-A, B, CW, DRB1, DQB1 and DPB1 sites of the donors (umbilical cord blood and haploid donors), and the mean fluorescence intensity (MFI) > 1000 was measured by solid phase fluorescence method; 3. Previous treatment with allogeneic hematopoietic stem cell transplantation or solid organ transplantation; 4. Uncontrolled bacterial, viral or fungal infections.Uncontrolled is defined as no clinical improvement or progression with adequate anti-infective therapy; 5.HIV infection or active hepatitis B or C; 6. Pregnant or lactating women; 7. A history of primary malignancy (other than basal cell carcinoma resection or orthotopic cervical cancer treatment) within 3 years prior to transplant treatment; 8. Drug dependent;Subjects with uncontrolled mental illness;People with cognitive dysfunction; 9. Join another similar clinical investigator within 3 months;Those whom the investigator considered unsuitable for inclusion (for example, patients who were expected to be unable to maintain treatment due to financial issues).
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 1 year overall survival;Serum ferritin;Hepatic ferromagnetic resonance; | — |
Secondary
| Measure | Time frame |
|---|---|
| 180-day transplant-related mortality;1-year transplant-related mortality;Implantation rate of neutrophils;Implantation time of neutrophils;Platelet implantation rate;Platelet implantation time;Implantation time of reticulocytes;Implantation rate of reticulocytes;II~IV/III~IV Cumulative incidence of acute GVHD;Incidence of bloodstream infection;Immune reconstitution after transplantation (immunoglobulin level, T, B, NK, etc.);Marrow cytology; | — |
Countries
China
Contacts
Public ContactSun Zimin
The First Affiliated Hospital of University of Science and Technology of China (Anhui Provincial Hospital)
Outcome results
None listed