Published Research in Hairy Cell Leukemia

We fund promising research with an eye toward strengthening understanding of hairy cell leukemia and improving outcomes for patients with the disease. Our research grantees have published their findings in leading journals, advancing knowledge of hairy cell leukemia and bringing us closer to a cure. In 2017, we supported the publication of consensus guidelines for the diagnosis and treatment of hairy cell leukemia. This article was co-authored by 41 HCL experts from around the world and is a resource for both doctors and patients regarding standard of care.

Treatment for hairy cell leukemia

Classic hairy cell leukemia complicated by pancytopenia and severe infection: a report of 3 cases treated with vemurafenib

Journal: Blood Advances (January 22, 2019)

Authors: Daniel P. Shenoi, Leslie A. Andritsos, James S. Blachly, Kerry A. Rogers, Mollie E. Moran, Mirela Anghelina, Jeffrey A. Jones and Michael R. Grever

Key Points

  • Infections are a major cause of morbidity and mortality in HCL patients, and myelosuppressive therapies increase the risk of poor outcomes.

  • Vemurafenib achieves rapid hematologic improvement in HCL and may facilitate management during life-threatening infection.


Consensus Guidelines for the Diagnosis and Management of Patients with Classic Hairy Cell Leukemia

Journal: Blood (February 2, 2017)

Authors: Michael R. Grever, Omar Abdel-Wahab, Leslie A. Andritsos, Versha Banerji, Jacqueline Barrientos, James S. Blachly, Timothy G. Call, Daniel Catovsky, Claire Dearden, Judit Demeter, Monica Else, Francesco Forconi, Alessandro Gozzetti, Anthony D. Ho, James B. Johnston, Jeffrey Jones, Gunnar Juliusson, Eric Kraut, Robert J. Kreitman, Loree Larratt, Francesco Lauria, Gerard Lozanski, Emili Montserrat, Sameer A. Parikh, Jae H. Park, Aaron Polliack, Graeme R. Quest, Kanti R. Rai, Farhad Ravandi, Tadeusz Robak, Alan Saven, John F. Seymour, Tamar Tadmor, Martin S. Tallman, Constantine Tam, Enrico Tiacci, Xavier Troussard, Clive S. Zent, Thorsten Zenz, Pier Luigi Zinzani and Brunangelo Falini


Hairy cell leukemia is an uncommon hematologic malignancy characterized by pancytopenia and marked susceptibility to infection. Tremendous progress in the management of patients with this disease has resulted in high response rates and improved survival, yet relapse and an appropriate approach to re-treatment present continuing areas for research. The disease and its effective treatment are associated with immunosuppression. Because more patients are being treated with alternative programs, comparison of results will require general agreement on definitions of response, relapse, and methods of determining minimal residual disease. The development of internationally accepted, reproducible criteria is of paramount importance in evaluating and comparing clinical trials to provide optimal care. Despite the success achieved in managing these patients, continued participation in available clinical trials in the first-line and particularly in the relapse setting is highly recommended. The Hairy Cell Leukemia Foundation convened an international conference to provide common definitions and structure to guide current management. There is substantial opportunity for continued research in this disease. In addition to the importance of optimizing the prevention and management of the serious risk of infection, organized evaluations of minimal residual disease and treatment at relapse offer ample opportunities for clinical research. Finally, a scholarly evaluation of quality of life in the increasing number of survivors of this now manageable chronic illness merits further study. The development of consensus guidelines for this disease offers a framework for continued enhancement of the outcome for patients.


Proceedings and Papers from the International Hairy Cell Leukemia Consortium Meeting: “Treatment of Hairy Cell Leukemia in its Second Half Century”, held at the NIH, Bethesda, Maryland, USA April 26-27, 2010

Journal: Leukemia & Lymphoma (June 2011)

Guest Editors: Robert Kreitman, Aaron Polliack & Michael Grever

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Hairy Cell Leukemia: A 50 Year Report on Clinical Research

Leukemia & Lymphoma (October 2009)

Guest Editor: Michael Grever

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variant hairy cell leukemia

Cladribine with Immediate Rituximab for the Treatment of Patients with Variant Hairy Cell Leukemia

Journal: Clinical Cancer Research (December 2013)

Authors: Robert J. Kreitman, Wyndham Wilson, Katherine R. Calvo, et al.


Purpose: In contrast with the classic form, variant hairy cell leukemia (HCLv) responds poorly to single-agent purine analogs, expresses unmutated BRAF, has shorter overall survival, and lacks effective standard therapy. No treatment has achieved a high complete remission (CR) rate even in small series, and of 39 reported cases from six studies, overall response rate after cladribine was 44% with 8% CRs. Rituximab has been found to increase the sensitivity of malignant cells to cladribine, suggesting that combination with cladribine might improve response in HCLv. To test this hypothesis, patients with HCLv were treated with simultaneous cladribine and rituximab.

Conclusion: Although cladribine alone lacks effectiveness for early or relapsed HCLv, cladribine with immediate rituximab achieves complete remission without minimal residual disease and is feasible to administer.

BRAF Mutation

Targeting Mutant BRAF in Relapsed or Refractory Hairy-Cell Leukemia

Journal: The New England Journal of Medicine (October 29, 2015)

Authors: Enrico Tiacci, M.D., Jae H. Park, M.D., Luca De Carolis, M.D., Stephen S. Chung, M.D., Alessandro Broccoli, M.D., Sasinya Scott, M.P.H., Francesco Zaja, M.D., Sean Devlin, Ph.D., Alessandro Pulsoni, M.D., Young R. Chung, M.S., Michele Cimminiello, M.D., Eunhee Kim, Ph.D., et al.


Background: BRAF V600E is the genetic lesion underlying hairy cell leukemia. We assessed the safety and activity of the oral BRAF inhibitor vemurafenib in patients with hairy cell leukemia that had relapsed after treatment with a purine analogue or who had disease that was refractory to purine analogues.

Conclusions: A short oral course of vemurafenib was highly effective in patients with relapsed or refractory hairy cell leukemia.


BRAF Mutations in Hairy-Cell Leukemia

Journal: The New England Journal of Medicine (June 16, 2011)

Authors: Enrico Tiacci, M.D., Vladimir Trifonov, Ph.D., Gianluca Schiavoni, Ph.D., Antony Holmes, Ph.D., Wolfgang Kern, M.D., Maria Paola Martelli, M.D., Alessandra Pucciarini, Ph.D., Barbara Bigerna, B.Sc., Roberta Pacini, B.Sc., Victoria A. Wells, B.Sc., Paolo Sportoletti, M.D., Valentina Pettirossi, Ph.D., et al.


Background: Hairy cell leukemia (HCL) is a well-defined clinicopathological entity whose underlying genetic lesion is still obscure. We searched for HCL-associated mutations by performing massively parallel sequencing of the whole exome of leukemic and matched normal cells purified from the peripheral blood of an index patient with HCL. Findings were validated by Sanger sequencing in 47 additional patients with HCL.

Conclusions: The BRAF V600E mutation was present in all patients with HCL who were evaluated. This finding may have implications for the pathogenesis, diagnosis, and targeted therapy of HCL.