To evaluate the safety and efficacy of Single Umbilical Cord blood transplantation (sUCBT) containing low dose ATG based conditioning regimen in the treatment of acquired Severe Aplastic Anemia (SAA).
Hematopoietic stem cell transplantation (HSCT) from HLA-compatible sibling donors is the standard preferred therapy for Severe Aplastic Anemia (SAA). At present, HLA-compatible donors in China fail to meet the needs of patients for transplantation. Umbilical Cord blood transplantation (UCBT) is an alternative treatment for patients with emergency conditions who lack compatible sibling donors, have failed immunosuppressant therapies or cannot wait for unrelated fully compatible donors. However, implantation failure is one of the main problems in UCBT for SAA due to the low cord blood stem cells. Nowadays, there is no consensus on the optimal conditioning regimen for UCBT treatment of SAA, and whether ATG should be added to the conditioning regimen is still controversial. Early clinical studies in UCBT have shown that the sufficient ATG in the conditioning regimen will lead to a decrease rate of implantation rate and affect survival of UCBT ultimately. However, a range of recent studies have found that with the addition of low-dose ATG, T cell reconstitution seems to outperform bone marrow or peripheral blood stem cell transplantation. Therefore, how to properly use ATG to both retain its effective GVHD prevention effect and reduce its impact on immune reconstruction has important clinical significance for improving the transplantation efficacy. In this study, a multicenter clinical study will be conducted to observe the safety and efficacy of UCBT with low dose ATG in the treatment of SAA and the long-term quality of life of patients.
Study Type
OBSERVATIONAL
Enrollment
72
Indications for trial stopping: subjects occur pregnancy events, severe organ function impairments, or emergency events related or unrelated to treatment; or subjects are unwilling to continue participating in the clinical trial.
The First Affiliated Hospital of University of Science and Technology of China (Anhui Provincial Hospital)
Hefei, Anhui, China
RECRUITINGThe one-year probability of GRFS
GRFS is defined as survival without grade III-IV acute GVHD immunosuppression requiring chronic GVHD or relapse.
Time frame: one year
The cumulative incidence of platelet engraftment at 28 days after transplantation
Platelet engraftment is defined as independent from platelet transfusion for at least 7 days with a platelet count of more than 20 × 10\^9/L.
Time frame: 28 days
The cumulative incidence of neutrophil engraftment at 28 days after transplantation
Neutrophil engraftment is defined as neutrophil count achieves 0.5 ×10\^9/L for three consecutive days.
Time frame: 28 days
Time of platelet engraftment
Platelet engraftment is defined as independent from platelet transfusion for at least 7 days with a platelet count of more than 20 × 10\^9/L.
Time frame: 28 days
Time of neutrophil engraftment
Neutrophil engraftment is defined as neutrophil count achieves 0.5 ×10\^9/L for three consecutive days.
Time frame: 28 days
The cumulative incidence of acute GVHD at 100 days after transplantation
Acute GVHD is defined that occurs within 100 days after transplantation
Time frame: 100 days
The cumulative incidence of chronic GVHD at 100 days after transplantation
Chronic GVHD is defined that occurs 100 days after transplantation
Time frame: one year
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Immune reconstitution after transplantation
The level of immunoglobulin, T cells, B cells and NK cells reconstitution after transplantation
Time frame: one year
The 180-day probability of TRM
TRM(transplant-related mortality) is defined that death due to any transplant-related cause within 180 days after transplantation
Time frame: 180 days
The incidence of infection
Infection is defined as various post-transplant associated infections
Time frame: one year
The one-year probability of OS
OS (overall survival) is defined as the time which begins at diagnosis and up to the time of death
Time frame: one year
The one-year probability of DFS
DFS (disease free survival) is defined as the time from treatment until the recurrence of disease (or death) after transplantation
Time frame: one year