Human Leukocyte Antigen (HLA)-Haploidentical Hematopoietic Stem Cell Transplantation for Patients With Aplastic Anemia

Asan Medical Center

Status and phase

Completed

Phase 2

Phase 1

Conditions

Aplastic Anemia

Treatments

Drug: Cyclophosphamide

Procedure: CD3±CD19 depleted hematopoietic stem cell transplantation

Drug: Fludarabine

Biological: filgrastim

Biological: anti-thymocyte globulin

Study type

Interventional

Funder types

Other

Identifiers

NCT01105273

AMCPHO-SCT0802

Details and patient eligibility

About

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.

Enrollment

12 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