Haploidentical Stem Cell Transplantation for Children With Acquired Severe Aplastic Anemia

Asan Medical Center

Status and phase

Unknown

Phase 2

Conditions

Acquired Aplastic Anemia

Treatments

Procedure: CD3-depleted hematopoietic cell transplantation

Drug: Fludarabine

Biological: anti-thymocyte globulin

Drug: Cyclophosphamide

Biological: filgrastim

Radiation: Total body irradiation

Study type

Interventional

Funder types

Other

Identifiers

NCT01759732

AMCPHO-SCT1202

Details and patient eligibility

About

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

Enrollment

10 estimated patients

Sex

All

Ages

Under 21 years old

Volunteers

No Healthy Volunteers

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