Rationale: Chemotherapy with fludarabine, cyclophosphamide and anti-thymocyte globulin may induce the engraftment cross the immunologic barrier in the setting of HLA-haploidentical allogeneic hematopoietic cell transplantation. In addition, depletion CD3±CD19 cells may contribute to prevent developing severe acute graft versus host disease (GVHD) in haploidentical transplantation. Purpose: This phase I/II trial is to evaluate the safety and efficacy of fludarabine, cyclophosphamide and antithymocyte globulin with CD3±CD19 depleted graft from haploidentical donors in treating patients with aplastic anemia.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
12
On days -3 to -1
Beginning on day 4 and continuing until blood counts recover
30mg/M2 once daily IV on days -6 to -2
60 mg/kg IV on day-3 and -2
Immunogenetic depletion on CliniMACS
Asan Medical Center
Seoul, South Korea
To assess the engraftment rate and survival of CD3±CD19 depleted haploidentical peripheral blood stem cell transplantation after conditioning with fludarabine, cyclophosphamide and anti-thymocyte globulin.
Time frame: 2 years post-transplant
To assess engraftment and graft failure
Number of patients who failed to engraft by 28 days.
Time frame: 28 days post-transplant
To estimate the risk of acute GVHD
Number of patients with acute GVHD.
Time frame: 100 days post-transplant
To assess treatment related mortality
Number of death after transplantation
Time frame: 100 days post-transplant
To estimate overall survival
Time frame: 1 year after transplantation
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