Umbilical cord blood transplantation in adults using myeloablative and nonmyeloablative preparative regimens
- Nelson J Chao, MD
Nelson J Chao, MD
- Section Editor — Bone Marrow Transplantation
- Professor of Medicine
- Duke University School of Medicine
Allogeneic hematopoietic cell transplantation (HCT) is an important and potentially curative treatment option for a wide variety of malignant and nonmalignant diseases. The pluripotent hematopoietic stem cells required for this procedure are usually obtained from the bone marrow or peripheral blood of a related or unrelated donor. Umbilical cord blood (UCB), the blood remaining in the umbilical cord and placenta following the birth of an infant, has emerged as an established alternative source of hematopoietic stem cells in allogeneic HCT.
Engraftment and survival rates following HCT are optimized when the donor and recipient are genetically compatible. Human leukocyte antigen (HLA)-matched sibling donors are generally the preferred donor source for an allogeneic HCT. Unfortunately, finding an HLA-matched sibling is not always possible. Each full sibling potential donor has only a 25 percent chance of being fully HLA-matched with a sibling requiring a transplant. Therefore, most patients do not have an HLA-identical relative. (See "Donor selection for hematopoietic cell transplantation", section on 'Matched sibling donors'.)
When a suitable related donor is not available, a search is conducted to identify a potential unrelated HLA-matched donor. Finding an appropriate donor through a national registry is a lengthy process that is not always successful, especially for individuals who are not of Northern European descent. (See "Donor selection for hematopoietic cell transplantation", section on 'Unrelated donors'.)
In comparison, the relative ease of procurement and the lower than anticipated risk of severe acute graft versus host disease has made unrelated UCB transplantation a possible alternative to unrelated donor bone marrow or mobilized peripheral blood progenitor cell transplant. The increased representation of ethnic minorities and the ability to use partially HLA-matched UCB units significantly expands the donor pool. In addition, the use of reduced-intensity or non-myeloablative preparative regimens to allow engraftment of UCB broadens the scope of patients who may benefit from allogeneic HCT, including elderly and medically infirm patients without an HLA-matched sibling donor. (See "Selection of an umbilical cord blood graft for hematopoietic cell transplantation", section on 'Advantages and limitations of cord blood'.)
The use of UCB transplantation in adults using myeloablative and non-myeloablative preparative regimens will be discussed here. Other issues related to UCB are discussed separately, including the advantages and limitations of using UCB as a graft, the collection, storage, and ethical issues regarding the use of UCB for HCT, and the selection of an UCB graft for a particular recipient. (See "Collection and storage of umbilical cord blood for hematopoietic cell transplantation".)
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- CORD BLOOD AS A RESOURCE
- CORD BLOOD UNIT SELECTION
- PREPARATIVE REGIMEN
- Choice of preparative regimen
- Myeloablative preparative regimens
- - Efficacy
- - Toxicity and transplant-related mortality
- Graft-versus-host disease
- Organ toxicity
- Non-myeloablative preparative regimens
- - Efficacy
- Regimen intensity
- - Toxicity
- Immune reconstitution
- Graft-versus-host disease