Cord Blood Transplant in Children and Young Adults With Blood Cancers and Non-malignant Disorders
Trial Parameters
Brief Summary
This is a single-arm study to investigate 1-year treatment related mortality (TRM) in patients with life threatening non-malignant and malignant hematologic disorders who do not have a matched related donor for allogeneic transplantation.
Eligibility Criteria
Participant Inclusion Criteria: Age and Donor Status: Patients with age ≤ 21 years at time of consent with no available and suitably matched related or unrelated donor in the required time period. Diagnoses : I. Acute myelogenous leukemia (AML) : * Complete first remission (CR1) at high risk for relapse such as any of the following: * Known prior diagnosis of myelodysplasia (MDS) or myeloproliferative disorder (MPS). * Therapy-related AML (t-AML). * White cell count at presentation \> 100,000. * Presence of extramedullary leukemia at diagnosis. * Any unfavorable subtype by FAB or WHO classification. * High-risk cytogenetics (e.g. those associated with MDS, abnormalities of 5, 7, 8, complex karyotype) or high-risk molecular abnormalities. * Requirement for 2 or more inductions to achieve CR1. * Presence of Minimal Residual Disease (MRD+) by cytogenetics, flow cytometry or molecular methods after induction. * Any patient with newly diagnosed AML with intermediate risk cytogenetics who el