Transplantation of hematopoietic stem cells (HSCs) collected from bone marrow (BM) or peripheral blood (PB) of related and unrelated donors has become a successful treatment option for several malignant and nonmalignant disorders (Table 97.1). Further success has been hampered by the limited number of suitable HLA-matched donors, the prolonged length of donor unit procurement, and significant transplant-related morbidity. Over the past 15 years, in efforts to overcome these obstacles, allogeneic transplantation using umbilical cord blood (UCB) as an alternative source of HSCs has been investigated.

The use of UCB as a source of HSCs was proposed in the early 1980s. UCB cells have been shown in vitro to demonstrate both self-renewal and hematopoietic differentiation capabilities.1-4 Umbilical cord blood transplantation (UCBT) entered the clinical arena in 1988 when a child with severe Fanconi anemia was successfully transplanted using UCB harvested from an HLA-matched sibling.5 The Placental Blood Project was launched in 1993 to evaluate the clinical utility of expanding UCB as a source of HSCs. Shortly thereafter, mismatched related and unrelated UCBTs in children with high-risk leukemia were performed.6 Since 1998, more than 2500 UCBTs have been completed—75% performed in children and the remaining 25% in adults.7 Potential advantages of UCB as an alternative source of HSCs include (1) negligible risk to donor, (2) absence of donor attrition, (3) ease of procurement and rapid allocation, (4) reduced risk of graft-versus-host disease (GVHD), and (5) less stringent HLA-matching restrictions.

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