The investigators retrospectively evaluted the safety and efficacy of the modified transplantation conditioning and aGVHD prophylaxis in severe aplastic anemia in 4 transplantation centers.
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.
Study Type
OBSERVATIONAL
Enrollment
72
Modified transplantation conditioning include: Fludarabine 30mg/m2\*6 day, Melphalan 100mg/m2\*1 day, cyclophosphamide 50mg/kg\*2 day.
Department of Hematology,920th Hospital of Joint Logistics Support Force
Kunming, Yunnan, China
Primary graft falure rate
no apperance or complete loss of donor-derived neutrophils by +28 days
Time frame: From the day of HSCs transfusion to +100 day after HSCT.
aGVHD incidence
proportion of patients who developed aGVHD within 100 days after HSCT
Time frame: From the day of HSCT transfusion to 100 days after HSCT
Poor graft function rate
the presence of at least two cytopenic counts beyond +28 days with a transfusion requirement associated with hypoplastic-aplastic bone marrow
Time frame: From the day of HSCs transfusion to 24 months after HSCT
cGVHD incidence
the proportion of patients who developed chronic GVHD within the observation duration
Time frame: From +100 days after HSCT to the follow-up date
adverse events
the incidence of adverse events during the transplantation preconditioning periods.
Time frame: From the first day of preconditioning to +100 days after hematopoietic stem cell Transfusion
2-year Overall survival rate
proportioin of patients who still be alive within 24 months after HSCT
Time frame: 24 months
Disease-free survival
proportion of patients still be alive without GVHD or other diseases.
Time frame: 24 months
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