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General principles of hematopoietic cell transplantation for acute lymphoblastic leukemia in adults

Richard A Larson, MD
Section Editor
Robert S Negrin, MD
Deputy Editor
Alan G Rosmarin, MD


The results of treatment of adults with acute lymphoblastic leukemia (ALL) have steadily improved and currently complete remission (CR) can be induced in 75 to 90 percent of patients. However, in contrast to ALL in children, relapse rates remain high and long-term survival rates are approximately 25 to 50 percent, depending upon patient age and disease characteristics [1,2]. (See "Induction therapy for Philadelphia chromosome negative acute lymphoblastic leukemia in adults".)

The potential benefits of allogeneic hematopoietic cell transplantation (allo-HCT) are due to both the myeloablative chemoradiotherapy and the immune-mediated reaction of donor lymphocytes directed against residual ALL cells in the recipient (ie, the graft-versus-leukemia reaction). To be effective, the survival benefit should outweigh the greater expense and higher risk of early toxicity and death, and late complications such as graft versus host disease (GVHD) and sterility. Allo-HCT is commonly used as part of the post-remission therapy of patients with ALL demonstrating high-risk features, such as the presence of the Philadelphia (Ph) chromosome or a Ph-like molecular signature. Results have been best when allo-HCT is performed in first complete remission (CR), but allo-HCT can also cure some patients in second CR.

This topic review will discuss general issues in hematopoietic cell transplantation for ALL in adults including: selection of the donor, preparative chemoradiotherapy, and the graft-versus-leukemia effect. Outcomes with this procedure in different subsets of adults with ALL are discussed separately. (See "Donor selection for hematopoietic cell transplantation" and "Post-remission therapy for Philadelphia chromosome negative acute lymphoblastic leukemia in adults".)

The term "hematopoietic cell transplantation" (HCT) will be used throughout this review as a general term for transplantation of progenitor cells from any source (eg, bone marrow, peripheral blood, umbilical cord blood). Otherwise, the source of such cells will be specified (eg, autologous peripheral blood progenitor cell transplantation). (See "Sources of hematopoietic stem cells".)


The preferred donor for patients undergoing allogeneic hematopoietic cell transplantation (allo-HCT) for ALL is an HLA-matched sibling or matched unrelated donor (MUD). However, fewer than 25 percent of patients in the transplantable age group have an HLA-matched sibling donor. Allo-HCT using a partially matched family member donor or umbilical cord blood is a reasonable option for patients who do not have an HLA-identical matched donor [2,3]. (See "Sources of hematopoietic stem cells".)

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Literature review current through: Nov 2017. | This topic last updated: Feb 06, 2017.
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