Ibrutinib Effective for Treating Hairy Cell Leukemia, Study Shows

In the past, ibrutinib has been used to treat patients with a variety of conditions, including chronic lymphocytic leukemia (CLL), mantle cell lymphoma (MCL), and chronic graft-versus-host disease (cGVHD). According to News Medical, a new study shows that the treatment may also benefit patients with high-risk hairy cell leukemia. Within the Phase 2 clinical trial, researchers from The Ohio State University Comprehensive Cancer Center – Arthur G. James Cancer Hospital and Richard J. Solove Research Institute (“OSUCCC – James”) determined that orally administered ibrutinib resulted in durable disease control.

Interested in learning more? Check out the full study findings published in Blood.


In many cases, patients with hairy cell leukemia often have a generally good prognosis following diagnosis. However, the researchers note that purine analogues, the current standard-of-care, are not beneficial for a subset of patients. Because of this inability to tolerate current treatments, or a failure to respond to these treatments, new and effective therapeutic options are needed.

Within this study, researchers evaluated ibrutinib for patients with high-risk hairy cell leukemia. While most patients had previously been treated, some had not; however, this group had a specific form of hairy cell leukemia which traditionally responds poorly to the standard-of-care. During the study, patients received either 420mg or 840mg ibrutinib. According to Chemocare, ibrutinib is a targeted therapy which:

inhibits the function of Bruton’s tyrosine kinase (BTK),…a key signaling molecule of the B-cell receptor signaling complex that plays an important role in the survival of malignant B cells. Ibrutinib blocks signals that stimulate malignant B cells to grow and divide uncontrollably.

Findings from the study include:

  • The overall response rate (ORR) was 24% at 32 weeks and 36% at 48 weeks.
  • Around 36-months, the overall survival rate was 85%, and the progression-free survival rate was 73%, showing efficacy.
  • Altogether, ibrutinib was relatively safe and well-tolerated. Some side effects did occur, which include:
    • Anemia (low red blood cell count)
    • Thrombocytopenia (low platelet count)
    • Neutropenia (low white blood cell count)
    • Diarrhea
    • Muscle pain
    • Fatigue
    • Nausea

Hairy Cell Leukemia

Hairy cell leukemia is a rare and chronic blood cancer in which the bone marrow creates too many lymphocytes, a type of white blood cell. These abnormal cells look “hairy” under a microscope, giving this slow-growing cancer its name. As the bone marrow produces too many leukemic cells, the body fails to produce enough healthy red and white blood cells, as well as platelets. Typically, hairy cell leukemia affects males more than females. It often manifests in middle-aged and older adults. Risk factors include radiation exposure, as well as exposure to certain agricultural or industrial chemicals. Symptoms and complications include:

  • Appetite loss
  • Feeling of abdominal “fullness”
  • Night sweats
  • Easy bruising and bleeding
  • Fatigue
  • Unintended weight loss
  • Frequent or recurrent infections
  • Shortness of breath
  • General weakness
  • Enlarged spleen
  • Anemia (low red blood cell count)

Learn more about hairy cell leukemia.

Jessica Lynn

Jessica Lynn

Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.

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