Stem Cell Transplant From Donors After Alpha Beta Cell Depletion in Children and Young Adults

Eligibility Criteria

• 1. Age \< 60 years and \> 1 month;
• 2. Life expectancy \> 10 weeks;
• 3. Patients deemed eligible for allogeneic HSCT per institutional guidelines;
• 4. Patients with life-threatening hematological malignancies and non-malignant disorders that could benfit from HSCT;
• 5. A minimum genotypic identical match of 5/10 is required;
• 6. The donor and recipient must be identical, as determined by high resolution typing, in at least one allele of each of the following genetic loci: HLA-A, HLA-B, HLA-Cw, and HLA-DRB1;
• 7. Lansky/Karnofsky score \> 50;
• 8. Signed written informed consent;
• 9. Male and female subjects of childbearing potential must agree to use an effective means of birth control to avoid pregnancy throughout the transplant procedure, while on immunosuppression, and if the subject experiences any chronic GvHD.
• 1. Pregnant or lactating females;
• 2. Greater than Grade II acute GvHD or severe, unmanaged chronic extensive GvHD due to a previous allograft at the time of inclusion;
• 3. Dysfunction of liver (ALT/AST \> 10 times upper normal value, or direct bilirubin \> 3 times upper normal value), or unmanageable dysfunction of renal function;
• 4. Severe cardiovascular disease (arrhythmias requiring chronic treatment, congestive heart failure or left ventricular ejection fraction \< 30%);
• 5. Current active infectious disease (including positive HIV serology or viral RNA);
• 6. Serious concurrent uncontrolled medical disorders;
• 7. Lack of patient's/parents'/guardian's informed consent;
• 8. Any severe concurrent disease which, in the judgement of the sponsor-investigator, would place the patient at increased risk during participation in the study.