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Hematopoietic cell transplantation in chronic lymphocytic leukemia

Robert S Negrin, MD
Kanti R Rai, MD
Section Editor
Richard A Larson, MD
Deputy Editor
Rebecca F Connor, MD


Chronic lymphocytic leukemia (CLL) is one of the chronic lymphoproliferative disorders (lymphoid neoplasms). It is characterized by a progressive accumulation of functionally incompetent lymphocytes, which are monoclonal in origin.

CLL is considered to be identical (ie, one disease at different stages) to the mature (peripheral) B-cell neoplasm small lymphocytic lymphoma (SLL), one of the indolent non-Hodgkin lymphomas. The term CLL is used when the disease manifests primarily in the bone marrow and blood, while the term SLL is used when involvement is primarily nodal. While there are some differences in the treatment of early stage CLL and SLL, the treatment of advanced stage disease is the same. (See "Clinical presentation, pathologic features, diagnosis, and differential diagnosis of chronic lymphocytic leukemia".)

The natural history of CLL is extremely variable. While many cases follow an indolent course that is well controlled with standard therapy, others progress rapidly and die within two to three years after diagnosis from complications or causes directly related to CLL. The initial treatment of symptomatic CLL primarily consists of an immunotherapy-based chemotherapy regimen (eg, rituximab plus chemotherapy). (See "Overview of the treatment of chronic lymphocytic leukemia" and "Staging and prognosis of chronic lymphocytic leukemia" and "Overview of the complications of chronic lymphocytic leukemia".)

The use of either autologous or allogeneic hematopoietic cell transplantation (HCT) in CLL is an emerging therapy for selected patients and is the subject of ongoing clinical trials. Autologous HCT is not curative, but may result in prolonged remissions and has a low treatment-related mortality rate. In comparison, allogeneic HCT may cure a percentage of patients with CLL, but is associated with higher treatment-related mortality. The term "hematopoietic cell transplantation" (HCT) will be used throughout this review as a general term to cover transplantation of progenitor cells from any source (eg, bone marrow, peripheral blood, 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 use of HCT in CLL is reviewed below. Other treatment options are presented separately. (See "Treatment of relapsed or refractory chronic lymphocytic leukemia".)

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Literature review current through: Sep 2017. | This topic last updated: Jan 09, 2017.
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