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Immunotherapy for the prevention and treatment of relapse following hematopoietic cell transplantation

Robert S Negrin, MD
Section Editor
Nelson J Chao, MD
Deputy Editor
Alan G Rosmarin, MD


The majority of patients with malignancy who undergo hematopoietic cell transplantation (HCT) will attain a complete remission. However, many of these patients will have residual disease that is too small to detect using conventional techniques (ie, minimal residual disease) that puts them at risk of eventual relapse. Relapse ultimately occurs in 40 to 75 percent of patients who undergo an autologous HCT and 10 to 40 percent of those who undergo an allogeneic HCT. Discontinuation of immunosuppression followed by immunotherapy with donor lymphocyte infusion (DLI) may be used to treat relapse after allogeneic HCT. Other methods of immunotherapy (eg, interleukin-2, interferon alpha, dendritic cell vaccination) are under investigation for the prevention of or treatment of relapse following autologous or allogeneic HCT.

The rationale for using immunotherapy to prevent and/or treat the reemergence of malignancy is based in part upon the following observations:

Rates of leukemia relapse are much higher in patients who have received identical twin (syngeneic) transplants compared with HLA-identical sibling transplants administered with identical cytotoxic treatment [1]. This suggests that a graft-versus-tumor (GVT) effect plays a major role in reducing the risk of relapse following an allogeneic HCT. (See "Donor selection for hematopoietic cell transplantation", section on 'Identical twin donors'.)

Patients who develop acute or chronic graft-versus-host disease (GVHD), particularly chronic GVHD, have a lower risk of relapse [2,3].

The withdrawal of immunosuppression among patients with relapsing chronic myeloid leukemia after HCT may rarely result in cytogenetic remission occurring in association with GVHD [4]. A similar effect has been described in a patient with relapsing chronic lymphocytic leukemia [5].


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Literature review current through: Sep 2016. | This topic last updated: Dec 23, 2015.
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