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| ID | Type | Description | Link |
|---|---|---|---|
| 202301AY070001-226 | Other Grant/Funding Number | the Joint and Special Project of Kunming medical university |
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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.
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Modified transplantation system | Other | Modified transplantation conditioning include: Fludarabine 30mg/m2*6 day, Melphalan 100mg/m2*1 day, cyclophosphamide 50mg/kg*2 day. |
| Measure | Description | Time Frame |
|---|---|---|
| Primary graft falure rate | no apperance or complete loss of donor-derived neutrophils by +28 days | From the day of HSCs transfusion to +100 day after HSCT. |
| aGVHD incidence | proportion of patients who developed aGVHD within 100 days after HSCT | From the day of HSCT transfusion to 100 days after HSCT |
| Measure | Description | Time Frame |
|---|---|---|
| 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 | From the day of HSCs transfusion to 24 months after HSCT |
| cGVHD incidence |
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Inclusion Criteria:
Exclusion Criteria:
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Patients who were diagnosed with severe aplastic anemia by NCCN guidelines, and has received HSCT with this transplantation preconditioning and aGVHD prophylaxis regimen;
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Department of Hematology,920th Hospital of Joint Logistics Support Force | Kunming | Yunnan | 650000 | China |
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| ID | Term |
|---|---|
| D000741 | Anemia, Aplastic |
| ID | Term |
|---|---|
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D000080983 | Bone Marrow Failure Disorders |
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the proportion of patients who developed chronic GVHD within the observation duration
| From +100 days after HSCT to the follow-up date |
| adverse events | the incidence of adverse events during the transplantation preconditioning periods. | 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 | 24 months |
| Disease-free survival | proportion of patients still be alive without GVHD or other diseases. | 24 months |
| D001855 | Bone Marrow Diseases |