Providing Access to Cord Blood Units for Transplants

Study Design: This study is an access and distribution protocol for unlicensed cryopreserved cord blood units (CBUs) in pediatric and adult patients with hematologic malignancies and other indications. Primary Objective: The primary aim of this study is to examine the incidence of neutrophil recovery of (Bullet)500/mm3 after cord blood transplantation in a multi-institution setting using CBUs that are not Food and Drug Administration (FDA) licensed. Secondary Objectives: In patients receiving a non-licensed CBU: * Assess incidence of graft rejection * Assess incidence of transmission of infection * Assess incidence of serious infusion reaction * Determine 1 year survival after cord blood transplantation * Assess cumulative incidence of acute graft vs. host disease (GVHD) grades II to IV and grades III to IV * Assess cumulative incidence of chronic GVHD * Determine platelet engraftment of \>20,000 mcL and \>50,000 mcL * Determine CBU-derived engraftment Eligibility Criteria: Inclusion Criteria * Patients with FDA-specified indications (see Appendix B for further details): * Hematological malignancies * Certain lysosomal storage and peroxisomal enzyme deficiency disorders * Hurler syndrome (MPS I) * Krabbe Disease (Globoid Leukodystrophy) * X-linked Adrenoleukodystrophy * Primary immunodeficiency diseases * Bone marrow failure * Beta-thalassemia * Signed informed consent (and signed assent, if applicable) * Pediatric and adult patients of any age Exclusion Criteria * Patients who are receiving only licensed CBUs * Cord blood transplant recipients at international transplant centers Treatment Description: Treatment, including pre-transplant conditioning and GVHD prophylaxis, will occur per each transplant center s specifications. Accrual Objective: In this access and distribution protocol, U.S. patients undergoing transplant using unlicensed CBUs will be enrolled and there is no accrual maximum. Accrual Period: The accrual period is open ended.