Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation
United States6 participantsStarted 2018-06-15
Plain-language summary
In this study, the investigators test 2 dose levels of thiotepa (5 mg/kg and 10 mg/kg) added to the backbone of targeted reduced dose IV busulfan, fludarabine and rabbit anti-thymocyte globulin (rATG) to determine the minimum effective dose required for reliable engraftment for subjects undergoing hematopoietic stem cell transplantation for non-malignant disease.
Who can participate
Age range3 Months – 39 Years
SexALL
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Inclusion Criteria:
* Diagnoses:
* Hemoglobinopathies (e.g. thalassemia or sickle cell disease),
* Cytopenias (e.g.Diamond-Blackfan anemia, congenital or acquired neutropenia, congenital or acquired thrombocytopenia, congenital or acquired anemia, and others, regardless clonality),
* Hemophagocytic lymphohistiocytosis,
* Primary immunodeficiencies (e.g. Wiscott Aldrich Syndrome, chronic granulomatous disease, common variable immune deficiency, X-linked lymphoproliferative disease, NK+ severe combined immune deficiencies),
* Metabolic disorders (Hurler's syndrome, mannosidosis, adrenal leuko-dystrophy)
* Other non-malignant disorders for which there is published evidence that HSCT (hematopoietic stem cell transplant) is a curative therapy.
* Donor Requirements
* Related or unrelated donor who is suitable and willing to donate bone marrow or peripheral blood stem cells. HLA typing should be done by high-resolution typing at A, B, C, DrB1 and DQ loci and the donor should be at a minimum ≥8/10 match (with one antigen/allele mismatch allowed at A, B, or C-loci and other at DQ loci).
* Cord blood units must be matched at a minimum of 6/8 antigens/alleles at A, B, C and DrB1 loci. High resolution typing at all loci is required. The minimum TNC dose pre-cryopreservation must be ≥3.7 x10\^7/kg of recipient's weight, if a single cord blood unit is used, or at least 2x10\^7/kg per unit, if two cord blood units are used. The mismatches cannot be at the same loci (e.g. …
What they're measuring
1
Assessment of Minimum Effective Dose (MED) of Thiotepa