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HLA-haploidentical hematopoietic cell transplantation

Ephraim J Fuchs, MD, MBA
Leo Luznik, MD
Section Editor
Nelson J Chao, MD
Deputy Editor
Alan G Rosmarin, MD


Allogeneic hematopoietic cell transplantation (HCT) is a potentially curative therapy for a wide variety of malignant and non-malignant hematologic disorders. 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. Historically, the best results of allogeneic HCT have been obtained when the stem cell donor is a human leukocyte antigen (HLA)-matched sibling.

Given the small family sizes in developed nations and the 25 percent likelihood that any sibling is fully HLA-matched to the patient, an HLA-matched sibling can be found for only approximately 30 percent of patients. For patients who lack an HLA-matched sibling, alternative sources of donor grafts include suitably HLA-matched adult unrelated donors, umbilical cord blood stem cells, and partially HLA-mismatched, or HLA-haploidentical, related donors. Substantial improvements in haploidentical transplantations have occurred in the last decade and there is growing use of haploidentical family donors. The decision of which donor source to utilize depends, to a large degree, upon the clinical situation and the approaches employed at the individual transplant center.

The major challenge of HLA-haploidentical HCT is intense bi-directional alloreactivity leading to high incidences of graft rejection and graft-versus-host disease (GVHD). Advances in graft engineering and in pharmacologic prophylaxis of GVHD have reduced the risks of graft failure and GVHD after HLA-haploidentical HCT, and have made this stem cell source a viable alternative for patients lacking an HLA-matched sibling.

This topic will discuss the advantages and disadvantages of HLA-haploidentical HCT and the selection of an HLA-haploidentical donor. A general approach to donor selection for allogeneic HCT is discussed separately. (See "Donor selection for hematopoietic cell transplantation".)


A HLA-haploidentical donor is one who shares, by common inheritance, exactly one HLA haplotype with the recipient and is mismatched for a variable number of HLA genes, ranging from zero to five, on the unshared haplotype. Potential HLA-haploidentical donors include biological parents; biological children; full or half siblings (figure 1); and even extended family donors such as aunts, uncles, nieces, nephews, cousins, or grandchildren.

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Literature review current through: Oct 2017. | This topic last updated: Jun 15, 2017.
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