During the past decade, a number of advances have been made in the treatment or patients sickle cell anemia and thalassemia. Among these is allogeneic bone marrow transplantation, the only current treatment that offers the potential for cure. In sickle cell anemia, transplantation has been performed in patients who have had advanced organ damage while in thalassemia, transplantation has been performed before evidence of iron related tissue damage. Due to concerns over engraftment and graft versus host disease (GVHD), transplants for patients with hemoglobinopathies have been limited to situations where an HLA compatible donor exists. Unfortunately, an HLA matched related donor is often not available. Umbilical cord blood, a recently recognized source of hematopoetic stem cells, has been used to successfully transplant over 500 patients. The potential advantages of cord blood over other donor sources of stem cells is related to the observation that even without complete HLA compatibility, the risk of high-grade GVHD is minimal. The objective of this proposal is to increase the chance to offer transplantation as a therapeutic option for patients with hemoglobinopathies by establishing a genetically related umbilical cord blood bank.
Our specific aims are (1) to establish a cord blood bank containing 2500 cord blood units (CBU) which will serve as a resource to hemoglobinopathy centers, (2) to implement, validate and monitor CBU collection procedures in these centers, (3) to determine if quality CBUs can be collected at multiple sites and shipped to Children's Hospital Oakland Research Institute (CHORI) for processing and cryopreservation, and (4) to establish a research resource for evaluation of the role of CBU transplantation in patients with hemoglobinopathies. Protocols for cord blood collection, processing and data collection have been adapted from the NIH unrelated Cord Blood Transplantation study (COBLT). Statistical evaluation and computerized data collection will be performed by The EMMES, Corp., a group providing similar assistance to the COBLT study. Molecular HLA typing and hemoglobinopathy diagnostic services will be made available to collaborating programs. The related CBU resource described in this application will provide investigators with an opportunity to study the role of CBU for transplantation in patients with hemoglobinopathies, to evaluate at the molecular level relationships between HLA disparity, engraftment and GVHD using CBUs, and to determine if CBUs collection should be offered to all families who either have a child with a hemoglobinopathy or are at risk of having a child with a hemoglobinopathy. The resource provided by this related cord blood bank may offer the possibility of cure to a number of patients who are affected by sickle cell anemia and thalassemia.

National Institute of Health (NIH)
National Heart, Lung, and Blood Institute (NHLBI)
Resource-Related Research Projects--Cooperative Agreements (U24)
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Special Emphasis Panel (ZHL1 (O1))
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Harvath, Liana
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Children's Hospital & Res Ctr at Oakland
United States
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