Transplants With Unlicensed Preserved Cord Blood

• To examine the incidence of neutrophil recovery of greater than or equal to 500/mm (3) after unlicensed CBU transplant.
  

• Assess incidence of graft rejection, transmission of infection, serious infusion reactions.
  
• Determine 1 year survival.
  
• Assess cumulative incidence of acute GVHD (grades II to IV), (grades III to IV), and incidence of chronic GVHD.
  

Procedure: Cord Blood Transplant
N/A

Background

The requirement for licensure became part of the FDA final rules for Human Cells, Tissues, and Tissue-Based Products (HCT/Ps) (March 29, 2004 and May 25, 2005) in an effort to prevent transmission of communicable disease, minimize contamination and preserved integrity and function during processing, outline safety and effectiveness requirements for cells from unrelated donors or when HCT/Ps are more than minimally manipulated, assure labeling is clear accurate and not misleading and monitor and communicate with industry via establishment registration. However, as of October 20, 2011, those units that do not meet the manufacturing requirements for licensure can only be distributed for transplantation if the transplant will occur under an IND research protocol. These units are in current and future inventory at domestic and international cord blood banks that cannot be demonstrated to meet licensing requirements. In addition to the licensure guidance, the FDA published a guidance in August 2011 entitled Investigational New Drug Applications (INDs) for Minimally Manipulated, Unrelated Allogeneic Placental/Umbilical Cord Blood Intended for Hematopoietic Reconstitution for Specified Indications.

This study is an access and distribution protocol conducted by the National Marrow Donor Program(Registered Trademark) (NMDP) for unlicensed cryopreserved cord blood units (CBUs) in pediatric and adult patients, in which sites wishing to receive NMDP unrelated cord blood units for treatment or research must participate with an locally IRB- approved protocol.

Primary Objectives

The primary objective of this study is to examine the incidence of neutrophil recovery of greater than or equal to 500/mm(3) 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 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 CBU-derived engraftment

Eligibility Criteria

Inclusion Criteria

• Patients of any age with FDA-specified indications
• Signed informed consent (and assent when applicable)

Exclusion Criteria

• Patients who have licensed CBUs available
• Cord blood transplant recipients at international transplant centers

Design

-Treatment description, including pre-transplant conditioning and GVHD prophylaxis, will occur per each transplant center's specification.