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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.
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Hematopoietic stem cell product from haploidentical or unrelated donor CD3/CD19 depleted with CliniMACS | Other | The aim is to transplant 7x106 CD34+/kg of recipient body weight. |
| Measure | Description | Time Frame |
|---|---|---|
| To evaluate engraftment after CD3/CD19 depletion of the graft | within 1 year |
| Measure | Description | Time Frame |
|---|---|---|
| To evaluate immunoreconstitution after transplantation by assessing lymphocyte subsets | within 1 year |
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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:
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| Name | Affiliation | Role |
|---|---|---|
| Peter Bader, Professor | University Hospital Frankfurt, Dept. for Children and Adolescents, Division for Stem Cell Transplantation, Goethe University. | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University Hospital Frankfurt, Dept. for Children and Adolescents, Division for Stem Cell Transplantation, Goethe University | Frankfurt am Main | Hesse | 60590 | Germany |
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