Skip to Content
Home > Wellness > Health Library > Hairy Cell Leukemia Treatment (PDQ®): Treatment - Health Professional Information [NCI]
This information is produced and provided by the National Cancer Institute (NCI). The information in this topic may have changed since it was written. For the most current information, contact the National Cancer Institute via the Internet web site at http://cancer.gov or call 1-800-4-CANCER.
Hairy cell leukemia is an indolent, low-grade, B-cell lymphoma usually characterized by the following:
In addition to the B-cell antigens CD19, CD20, and CD22, the cells coexpress CD11c, CD25, and CD103. The BRAF-V600E mutation is a hairy cell leukemia–defining genetic lesion that can be used diagnostically.[1,2] The decision to treat is based on symptomatic cytopenias, massive splenomegaly, or the presence of other complications. About 10% of all patients will never require therapy.
No generally accepted staging system is useful for both prognosis and therapy.
For the purpose of treatment decisions, it is best to consider this disease in the following two broad categories:
Untreated hairy cell leukemia
Untreated hairy cell leukemia is characterized by splenomegaly, varying degrees of leukopenia (occasionally leukocytosis) and/or pancytopenia, and bone marrow infiltration by an atypical cell with prominent cytoplasmic projections (i.e., hairy cells). The bone marrow is usually fibrotic and is not easily aspirated. Bone marrow biopsies are, therefore, required for diagnosis and evaluation of the degree of hairy cell infiltration.
Progressive hairy cell leukemia
Progressive hairy cell leukemia, postsplenectomy (or following any systemic therapy) is characterized by progressive bone marrow replacement by hairy cells with pancytopenia refractory to treatment. For patients with advanced hairy cell leukemia treated with cladribine (2-chlorodeoxyadenosine, 2-CdA), pentostatin, or interferon-alpha, the survival rate appears to be more than 85% at 5 years following the initiation of any one of these therapies.[1,2]
The initial therapies of choice are either cladribine (2-chlorodeoxyadenosine, 2-CdA) or pentostatin.[1,2] These drugs have comparable response rates but have not been compared in phase III trials. Cladribine is administered as a one-time continuous infusion or series of subcutaneous injections and is associated with a high rate of febrile neutropenia.[3,4,5,6] Rarely, more than one course of treatment is required to induce a desirable response. Treatment should be discontinued once complete remission or stable partial remission with normalization of peripheral blood counts is reached. The presence of residual disease may be predictive of relapse but does not seem to affect survival.[5,7]
The role of consolidation or maintenance therapy in preventing relapse or progression of the disease following treatment with purine analogs has not been evaluated and remains unproven. Pentostatin is administered intermittently for a longer treatment duration but may result in a lower incidence of febrile complications.[8,9] While most patients remain disease free 10 years after treatment with these purine analogs, no patient has been followed long enough to assess cure.[10,11] Both nucleoside analogs cause profound suppression of CD4 counts, which may last for a year, and a potential increased risk of second malignancies has been reported.[5,12]
A study of 3,104 survivors of hairy cell leukemia from the SEER database showed an increased risk of second cancers (standardized incidence ratio, 1.24; 95% CI, 1.11–1.37), especially for Hodgkin and non-Hodgkin lymphomas. The increased risk for second cancers was seen even in the 2 decades before the introduction of purine nucleosides. With the use of cladribine, an increased risk of second malignancies is possible among patients with hairy cell leukemia (observed to expected ratio of about 1.8 in several series after 6 years).[5,12] Several series using pentostatin did not report an increased risk of second malignancies.[8,10,14] For a few patients, such as those with severe thrombocytopenia, splenectomy might be considered. After splenectomy, 50% of patients will require no additional therapy, and long-term survivors are common. Therapy with interferon-alpha is another treatment option, especially for patients with intercurrent infection.[9,16]
The hairy cell leukemia variant has a distinctive phenotype and typically presents with leukocytosis instead of leukopenia. Patients with this variant have poorer responses to initial cladribine, shorter durations of response, and typically do not respond again to purine analogues after relapse. Combinations of rituximab and purine analogues are under evaluation and further studies are required to define optimal therapies.[18,19]
Untreated Hairy Cell Leukemia
Hairy cell leukemia is a highly treatable disease. Since it is easily controlled, many patients have prolonged survival with sequential therapies. The decision to treat is based on cytopenias (especially if symptomatic), increasing splenomegaly, indications that the disease is progressing, or the presence of other, usually infectious complications. It is reasonable to offer no therapy if the patient is asymptomatic and blood counts are maintained in an acceptable range.
Progressive Hairy Cell Leukemia
Standard treatment options:
Ongoing trials are studying combinations of cladribine plus the monoclonal antibody rituximab.
Current Clinical Trials
Check for U.S. clinical trials from NCI's list of cancer clinical trials that are now accepting patients with untreated hairy cell leukemia and progressive hairy cell leukemia, initial treatment. The list of clinical trials can be further narrowed by location, drug, intervention, and other criteria.
General information about clinical trials is also available from the NCI Web site.
Cladribine (2-chlorodeoxyadenosine, 2-CdA) and pentostatin are both highly efficacious in the treatment of patients with disease refractory to interferon-alpha.[1,2,3,4] Patients who relapse after the first course of cladribine or pentostatin often respond well to retreatment with the same or another purine analog.[5,6,7,8,9,10,11] Rituximab can induce durable complete remissions with minimal toxic effects in patients with multiple relapsing or refractory disease after purine analog therapy or after interferon.[12,13,14,15][Level of evidence: 3iiiDiv] The lack of subsequent immunosuppression with rituximab has made this treatment a common choice among relapsing patients in the absence of a clinical trial. Combinations of rituximab with either cladribine or pentostatin are effective in achieving complete remission and are under clinical evaluation.[10,16,17] Both anti-CD25 and anti-CD22 recombinant immunotoxins under clinical evaluation can induce complete remissions in patients whose disease is resistant to retreatment with purine analogs or rituximab.[18,19]
Trials (including the ongoing CAT-8015-1001 [NCT00462189] study and NCI-04-C-0014, which is now completed) are in the process of evaluating, or have evaluated, new therapies for this group of patients.
Aggressive, high-dose chemotherapy has been beneficial in some cases, but the associated morbidity and mortality are high. It should not be considered unless other, more frequently effective, therapies have been exhausted.
Check for U.S. clinical trials from NCI's list of cancer clinical trials that are now accepting patients with refractory hairy cell leukemia. The list of clinical trials can be further narrowed by location, drug, intervention, and other criteria.
The PDQ cancer information summaries are reviewed regularly and updated as new information becomes available. This section describes the latest changes made to this summary as of the date above.
Editorial changes were made to this summary.
This summary is written and maintained by the PDQ Adult Treatment Editorial Board, which is editorially independent of NCI. The summary reflects an independent review of the literature and does not represent a policy statement of NCI or NIH. More information about summary policies and the role of the PDQ Editorial Boards in maintaining the PDQ summaries can be found on the About This PDQ Summary and PDQ NCI's Comprehensive Cancer Database pages.
Purpose of This Summary
This PDQ cancer information summary for health professionals provides comprehensive, peer-reviewed, evidence-based information about the treatment of hairy cell leukemia. It is intended as a resource to inform and assist clinicians who care for cancer patients. It does not provide formal guidelines or recommendations for making health care decisions.
Reviewers and Updates
This summary is reviewed regularly and updated as necessary by the PDQ Adult Treatment Editorial Board, which is editorially independent of the National Cancer Institute (NCI). The summary reflects an independent review of the literature and does not represent a policy statement of NCI or the National Institutes of Health (NIH).
Board members review recently published articles each month to determine whether an article should:
Changes to the summaries are made through a consensus process in which Board members evaluate the strength of the evidence in the published articles and determine how the article should be included in the summary.
The lead reviewers for Hairy Cell Leukemia Treatment are:
Any comments or questions about the summary content should be submitted to Cancer.gov through the Web site's Contact Form. Do not contact the individual Board Members with questions or comments about the summaries. Board members will not respond to individual inquiries.
Levels of Evidence
Some of the reference citations in this summary are accompanied by a level-of-evidence designation. These designations are intended to help readers assess the strength of the evidence supporting the use of specific interventions or approaches. The PDQ Adult Treatment Editorial Board uses a formal evidence ranking system in developing its level-of-evidence designations.
Permission to Use This Summary
PDQ is a registered trademark. Although the content of PDQ documents can be used freely as text, it cannot be identified as an NCI PDQ cancer information summary unless it is presented in its entirety and is regularly updated. However, an author would be permitted to write a sentence such as "NCI's PDQ cancer information summary about breast cancer prevention states the risks succinctly: [include excerpt from the summary]."
The preferred citation for this PDQ summary is:
National Cancer Institute: PDQ® Hairy Cell Leukemia Treatment. Bethesda, MD: National Cancer Institute. Date last modified <MM/DD/YYYY>. Available at: http://cancer.gov/cancertopics/pdq/treatment/hairy-cell-leukemia/HealthProfessional. Accessed <MM/DD/YYYY>.
Images in this summary are used with permission of the author(s), artist, and/or publisher for use within the PDQ summaries only. Permission to use images outside the context of PDQ information must be obtained from the owner(s) and cannot be granted by the National Cancer Institute. Information about using the illustrations in this summary, along with many other cancer-related images, is available in Visuals Online, a collection of over 2,000 scientific images.
Based on the strength of the available evidence, treatment options may be described as either "standard" or "under clinical evaluation." These classifications should not be used as a basis for insurance reimbursement determinations. More information on insurance coverage is available on Cancer.gov on the Coping with Cancer: Financial, Insurance, and Legal Information page.
More information about contacting us or receiving help with the Cancer.gov Web site can be found on our Contact Us for Help page. Questions can also be submitted to Cancer.gov through the Web site's Contact Form.
For more information, U.S. residents may call the National Cancer Institute's (NCI's) Cancer Information Service toll-free at 1-800-4-CANCER (1-800-422-6237) Monday through Friday from 8:00 a.m. to 8:00 p.m., Eastern Time. A trained Cancer Information Specialist is available to answer your questions.
The NCI's LiveHelp® online chat service provides Internet users with the ability to chat online with an Information Specialist. The service is available from 8:00 a.m. to 11:00 p.m. Eastern time, Monday through Friday. Information Specialists can help Internet users find information on NCI Web sites and answer questions about cancer.
Write to us
For more information from the NCI, please write to this address:
Search the NCI Web site
The NCI Web site provides online access to information on cancer, clinical trials, and other Web sites and organizations that offer support and resources for cancer patients and their families. For a quick search, use the search box in the upper right corner of each Web page. The results for a wide range of search terms will include a list of "Best Bets," editorially chosen Web pages that are most closely related to the search term entered.
There are also many other places to get materials and information about cancer treatment and services. Hospitals in your area may have information about local and regional agencies that have information on finances, getting to and from treatment, receiving care at home, and dealing with problems related to cancer treatment.
The NCI has booklets and other materials for patients, health professionals, and the public. These publications discuss types of cancer, methods of cancer treatment, coping with cancer, and clinical trials. Some publications provide information on tests for cancer, cancer causes and prevention, cancer statistics, and NCI research activities. NCI materials on these and other topics may be ordered online or printed directly from the NCI Publications Locator. These materials can also be ordered by telephone from the Cancer Information Service toll-free at 1-800-4-CANCER (1-800-422-6237).
Last Revised: 2014-12-04
How this information was developed to help you make better health decisions.
Healthwise, Healthwise for every health decision, and the Healthwise logo are trademarks of Healthwise, Incorporated.
250 Pleasant Street
Concord, NH 03301
Contact Concord Hospital
View Quality Data
© 2015 Concord Hospital