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Umbilical-cord blood transplantation for the treatment of cancer

Key Points

  • Allogeneic bone-marrow transplantation is the best treatment for patients with haematological cancers. This procedure, however, is limited by lack of suitable donors, as well as the complications of graft-versus-host disease (GVHD) and opportunistic infection.

  • Umbilical-cord blood is an excellent source of haematopoietic stem cells. It has the advantages of speedy availability and reduced incidence of causing GVHD. This allows for transplantation of grafts with limited HLA disparity, and thereby extends the donor pool.

  • The main determinants of successful umbilical-cord blood transplant are the number of cells that are available for transplantation and HLA matching.

  • Cord blood transplantation has been validated as an alternative to bone-marrow transplantation for the treatment of leukaemia in children.

  • A number of approaches are being developed to improve the efficacy of cord blood transplantation in adults, such as multiple-unit transplantation and reduced intensity of chemo-radiation therapy.

Abstract

Haematopoietic stem-cell transplantation is used to treat many haematological cancers, but is limited by the lack of suitable bone-marrow donors, the risk of graft-versus-host disease (GVHD) and slow immune reconstitution. Umbilical-cord blood is an alternative source of haematopoietic stem cells that has recently been tested in both child and adult cancer patients. These studies have identified several advantages to umbilical-cord cell transplantation, including a lower incidence of GVHD. Umbilical-cord blood is therefore a promising alternative to bone-marrow-derived stem cells.

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Figure 1: Treatment regimen and factors influencing the outcome of haematopoietic stem-cell transplantation.

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Acknowledgements

Supported by grants from the National Institutes of Health and the Children's Cancer Research Fund.

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Authors and Affiliations

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Corresponding author

Correspondence to Juliet N. Barker.

Related links

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DATABASES

Cancer.gov

leukaemia

renal-cell carcinoma

LocusLink

CD4

CD8

CD34

DRB1

G-CSF

HLA-A

HLA-B

HLA-C

OMIM

Fanconi anaemia

FURTHER INFORMATION

Cord blood banks

Cord blood-bank operating procedures

Cord blood transplantation

Pre-implantation genetic diagnosis

Stem-cell research

Glossary

AUTOLOGOUS TISSUE

Haematopoietic stem cells that are derived from the patient.

ALLOGENEIC TISSUE

Haematopoietic stem cells that are from an antigenically distinct individual of the same species — from a sibling or unrelated donor.

GRAFT-VERSUS-LEUKAEMIA (GVL) EFFECT

The ability of the immune cells that are derived from transplanted haematopoietic stem cells to recognize and destroy leukaemia cells. This partially accounts for the mechanism of action of allogeneic transplantation as treatment for leukaemia. The term has been broadened to 'graft-versus-malignancy', to acknowledge that this effect is seen in patients with non-leukaemic diseases, such as myeloma or lymphoma.

GRAFT-VERSUS-HOST DISEASE (GVHD).

A complication of allogeneic haematopoietic transplantation. It occurs when the immune system of the donor graft recognizes the tissues of the recipient (host) as foreign and attacks them. This can be lethal in its most severe form.

MAJOR HISTOCOMPATIBILITY ANTIGENS

Antigens that are encoded by genes in the major histocompatibility complex on the short arm of chromosome 6. They are highly polymorphic and determine the immunological identity of the cell. In humans, they are also known as human leukocyte antigens (HLAs).

CLASS 1 MATCHING

Matching patients for HLA-A and HLA-B antigens.

MYELOABLATIVE

High-dose chemotherapy or chemotherapy with radiation that destroys (ablates) the haematopoietic system of the patient.

CD34+ CELLS

Immature haematopoietic progenitors.

TRANSPLANT-RELATED MORTALITY

Death due to transplant complications such as GVHD or infection (in contrast to death from relapse of leukaemia). Also known as 'non-relapse mortality'.

EVENT-FREE SURVIVAL

The length of time after treatment that a person remains free of certain negative events, such as severe treatment side effects, cancer recurrence or progression, or death (from treatment side effects or from the cancer itself).

PRE-IMPLANTATION GENETIC DIAGNOSIS

The technique of testing for certain diseases or other attributes, such as HLA type, in an embryo.

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Barker, J., Wagner, J. Umbilical-cord blood transplantation for the treatment of cancer. Nat Rev Cancer 3, 526–532 (2003). https://doi.org/10.1038/nrc1125

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