Allogeneic Stem Cell Transplantation in Pediatric Patients With Malignant and Non-malignant High-risk Diseases

Allogeneic Stem Cell Transplantation With CD3/CD19 Depleted Stem Cells of Related or Unrelated Haploidentical Donors in Pediatric Patients With Malignant and Non-malignant Diseases

The aim of the study is to investigate the feasibility and toxicity of allogeneic haploidentical or unrelated transplantation with CD3/CD19 depleted stem cells associated with a reduced or a standard conditioning regimen in pediatric patients with malignant and non-malignant high-risk diseases, for whom allogeneic stem cell transplantation represents the only possible therapy option and no human leukocyte antigen (HLA) compatible related donors are available.

Inclusion Criteria:

• Patients aged 0 to 30 years

• Written informed consent from patient and/or parents or guardian

• Patients with Karnofsky Index > 60%

• Malignant disease:

  • acute lymphoblastic leukemia

  • acute myeloid leukemia

  • myelodysplastic syndrome

  • chronic myeloid leukemia according to the standard indications

  • solid tumors (e.g. neuroblastoma recurrence, soft-tissue sarcoma, Ewing's sarcoma, osteosarcoma, hepatoblastoma)

    . Non malignant disease:

  • acquired anemias (e.g. severe aplastic anemia, particularly severe Evans syndrome)

  • congenital anemias (e.g. thalassemia and sickle cell anemia)

• Women reliable contraception method when appropriate

Exclusion Criteria:

• Participation in other clinical trials
• Patients, parents, or guardians unable to understand the nature, the importance and the implications of the procedure
• Pregnant or nursing women
• Patients who underwent a stem cell transplantation in the last 250 days
• Patients with kidney, heart or liver insufficiency