Aplastic Anaemia, Transplantation Conditioning
Conditions
Keywords
modified transplantation conditioning, aplastic anemia, aGVHD prophylaxis
Brief summary
The investigators retrospectively evaluted the safety and efficacy of the modified transplantation conditioning and aGVHD prophylaxis in severe aplastic anemia in 4 transplantation centers.
Detailed description
Aplastic anemia (AA) is a group of myelo-hemopoietic failure syndromes caused by a variety of etiologies. If not intervened, the average expected survival time is less than half a year.Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is one of the possible cures for this disease. The success rate of treatment for this disease can be further improved under the previous regimen system. The survival rate reported in the literature is 60%-90%. From Jun. 2020 to Dec. 2023, 72 patients in 4 transplantation center received this modified transplantation system, The investigators designed this clinical trial to retrospectively evaluted the safety and efficacy of the modified transplantation conditioning and aGVHD prophylaxis in severe aplastic anemia in the 4 clinical center.
Interventions
Modified transplantation conditioning include: Fludarabine 30mg/m2\*6 day, Melphalan 100mg/m2\*1 day, cyclophosphamide 50mg/kg\*2 day.
Sponsors
Hematology department of the 920th hospital
Study design
Observational model
CASE_ONLY
Time perspective
RETROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
3 Years to 65 Years
Healthy volunteers
No
Inclusion criteria
1. Patients who were diagnosed with severe aplastic anemia by NCCN guidelines, and has received HSCT with this transplantation preconditioning and aGVHD prophylaxis regimen; 2. Age 3-65 years old; 3. Weight 10Kg-100Kg; 4. Eastern Cooperative Oncology Group (ECOG) score ≤3; 5. No major organ injury (ECG ejection fraction \>45%; bilirubin \< 2 times the upper limit of normal value; AST and ALT \< 3 times the upper limit of normal value; serum creatinine \< 2 times the upper limit of normal value); 6. No severe infection; 7. Subjects voluntarily participated in this clinical trial and signed the informed consent.
Exclusion criteria
1. Patients with other hematologic diseases who are not eligible for transplantation or who do not wish to receive transplantation; 2. Patients with an expected survival of less than 1 month; 3. Patients with previous autologous or allogeneic hematopoietic stem cell transplantation; 4. pregnant patients; 5. Patients with severe mental or neurological disorders that would affect the ability to provide informed consent and/or to report or observe adverse events; 6. Other conditions that the investigator determines to be inappropriate for enrollment.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Primary graft falure rate | From the day of HSCs transfusion to +100 day after HSCT. | no apperance or complete loss of donor-derived neutrophils by +28 days |
| aGVHD incidence | From the day of HSCT transfusion to 100 days after HSCT | proportion of patients who developed aGVHD within 100 days after HSCT |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| adverse events | From the first day of preconditioning to +100 days after hematopoietic stem cell Transfusion | the incidence of adverse events during the transplantation preconditioning periods. |
| Poor graft function rate | From the day of HSCs transfusion to 24 months after HSCT | the presence of at least two cytopenic counts beyond +28 days with a transfusion requirement associated with hypoplastic-aplastic bone marrow |
| Disease-free survival | 24 months | proportion of patients still be alive without GVHD or other diseases. |
| 2-year Overall survival rate | 24 months | proportioin of patients who still be alive within 24 months after HSCT |
| cGVHD incidence | From +100 days after HSCT to the follow-up date | the proportion of patients who developed chronic GVHD within the observation duration |
Countries
China
Outcome results
None listed