Acquired Aplastic Anemia
Conditions
Keywords
Aplastic anemia, T cell dose, CD3 depletion, Haploidentical hematopoietic stem cell transplantation
Brief summary
Rationale: Fludarabine, cyclophosphamide, anti-thymocyte globulin and low-dose total body irradiation (LD-TBI) may induce the engraftment cross the immunologic barrier in the setting of HLA-haploidentical allogeneic hematopoietic cell transplantation. In addition, depletion of CD3 cells may contribute to prevent developing severe acute graft versus host disease (GVHD) in haploidentical transplantation. Purpose: Phase II trials to evaluate the efficacy of haploidentical stem cell transplantation with fixed dose of T cells after in vitro T cell depletion using CD3 monoclonal antibody for children with acquired severe aplastic anemia
Interventions
DRUGFludarabine
40mg/M2 once daily IV on days -7 to -4
DRUGCyclophosphamide
60 mg/kg IV on day-3 and -2
BIOLOGICALanti-thymocyte globulin
BIOLOGICALfilgrastim
RADIATIONTotal body irradiation
200 cGy per day on D-5 & -4
PROCEDURECD3-depleted hematopoietic cell transplantation
Immunogenetic depletion on CliniMACS
Sponsors
Asan Medical Center
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
No minimum to 21 Years
Healthy volunteers
No
Inclusion criteria
* Diagnosis of life-threatening marrow failure (severe aplastic anemia) of nonmalignant etiology meeting 2 of the following criteria: * Granulocyte count \< 500/mm3, * Corrected reticulocyte count \< 1%, * Platelet count \< 20,000/mm3 * No HLA-identical family member or closely matched (8 of 8 HLA-locus match) unrelated marrow donor available * HLA-haploidentical related donor available
Exclusion criteria
* Paroxysmal nocturnal hemoglobinuria or Fanconi anemia * Clonal cytogenetic abnormalities or myelodysplastic syndromes * Active fungal infections * HIV positive * Severe disease other than aplastic anemia that would severely limit the probability of survival during the graft procedure * Pregnant or nursing
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| To assess engraftment rate and survival of haploidentical stem cell transplantation with fixed dose of T cells after in vitro T cell depletion using CD3 monoclonal antibody for children with acquired severe aplastic anemia | 2 years posttransplant |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| To assess engraftment and graft failure | 28 days posttransplant | Number of patients who failed to engraft by 28 days |
| To estimate the risk of acute GVHD | 100 days posttransplant | Number of patients with acute GVHD. |
| To assess treatment related mortality | 100 days posttransplant | Number of death after transplantation |
| To estimate overall survival and failure free survival | 1 year posttransplant | — |
Countries
South Korea
Contacts
Primary ContactHo Joon Im, MD, PhD
Outcome results
None listed