Haploidentical Stem Cell Transplantation for Children With Acquired Severe Aplastic Anemia

Asan Medical Center10 enrolled

Overview

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

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Acquired Aplastic Anemia

Interventions

Eligibility

Sex: ALLMax age: 21 Years

Medical Language ↔ Plain English

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

Outcomes

Primary Outcomes

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

Time frame: 2 years posttransplant

Secondary Outcomes

To assess engraftment and graft failure

Number of patients who failed to engraft by 28 days

Time frame: 28 days posttransplant

To estimate the risk of acute GVHD

Number of patients with acute GVHD.

Time frame: 100 days posttransplant

To assess treatment related mortality

Number of death after transplantation

Time frame: 100 days posttransplant

To estimate overall survival and failure free survival

Time frame: 1 year posttransplant