Cord Blood Transplantation in Hematological and Metabolic Diseases

Umbilical cord blood (CB), as a source of hematopoietic stem and progenitor cells for hematopoietic reconstitution, has been used for over two decades in allogeneic transplantation to treat patients with hematological malignancies, bone marrow failure, immunodeficiency syndromes, hemoglobinopathies, and certain inherited metabolic disorders. CB banks have been established worldwide, and regulatory pathways for licensure of publicly banked units are in place in the USA and other countries around the world. CB is immunologically tolerant thereby expanding access to transplantation for patients unable to find a fully matched donor and, because of rapid availability, is also a unique option for treatment of children with otherwise lethal inherited metabolic diseases affecting the brain. CB is likely to be an important source of cells for use in the emerging fields of cellular therapies and regenerative medicine.

Duke Scholars

Author Jessica Muller Sun Pediatrics, Hematology-Oncology

Author Joanne Kurtzberg Pediatrics, Transplant and Cellular Therapy