What is the initial treatment recommendation for hairy cell leukemia?

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From the Guidelines

The initial treatment recommendation for hairy cell leukemia is a purine analog, with cladribine (2-CdA) being the preferred first-line agent, as it induces durable and unmaintained response in 87%–100% patients, including complete response in 85%–91%, after a single course of therapy 1.

Key Considerations

  • Cladribine is typically administered as a single course of 0.1 mg/kg/day by continuous intravenous infusion for 7 days, or alternatively as a subcutaneous injection of the same dose for 5-7 days 1.
  • Pentostatin is an alternative purine analog that can be used at a dose of 4 mg/m² intravenously every other week for 3-6 months 1.
  • Treatment is indicated when patients develop significant cytopenias (hemoglobin <10 g/dL, platelets <100,000/μL, absolute neutrophil count <1,000/μL), recurrent infections, symptomatic splenomegaly, or constitutional symptoms 1.

Patient Assessment

  • Before initiating treatment, patients should have baseline blood counts, bone marrow examination, and assessment for infections, as hairy cell leukemia patients are susceptible to infections due to neutropenia and monocytopenia 1.
  • Prophylactic antibiotics may be necessary during treatment 1.

Watch-and-Wait Approach

  • Not all patients require immediate treatment; asymptomatic patients with adequate blood counts can be monitored with a watch-and-wait approach 1.
  • Clinical judgement is required in the decision to initiate therapy, because not all patients with newly diagnosed HCL will require immediate treatment 1.

From the FDA Drug Label

At initiation of treatment, most patients in the clinical studies had hematologic impairment as a manifestation of active Hairy Cell Leukemia. Following treatment with cladribine, further hematologic impairment occurred before recovery of peripheral blood counts began The myelosuppressive effects of cladribine were most notable during the first month following treatment Careful hematologic monitoring, especially during the first 4 to 8 weeks after treatment with cladribine, is recommended

The initial treatment recommendation for hairy cell leukemia is cladribine.

  • The recommended treatment regimen is 0.09 mg/kg/day for 7 consecutive days by continuous intravenous infusion.
  • Careful hematologic monitoring is recommended, especially during the first 4 to 8 weeks after treatment with cladribine.
  • Patients should be monitored for signs of myelosuppression, including neutropenia, anemia, and thrombocytopenia 2.

From the Research

Hairy Cell Leukemia Treatment

  • Hairy cell leukemia (HCL) is an uncommon, indolent, chronic B-cell lymphoproliferative disorder involving the marrow and spleen 3.
  • The initial treatment recommendation for hairy cell leukemia includes cladribine, which is a purine nucleoside analogue 4.

Cladribine Treatment

  • Cladribine is administered at a dose of 0.1 mg/kg/d for a total of 7 days as a continuous intravenous infusion 3, 5.
  • A single course of cladribine can induce complete remission (CR) in the majority of patients, with an overall response rate of 100% 3, 5.
  • The median first-response duration for all responders is around 98 months, and the overall survival rate is around 97% at 108 months 3.

Combination Therapy

  • Recent studies have explored the use of cladribine in combination with rituximab, which can enhance the achievement of minimal residual disease (MRD)-free complete remission 6.
  • Concurrent rituximab with cladribine has been shown to lead to higher rates of MRD-free CR compared to delayed rituximab or cladribine monotherapy 6.

Relapse and Retreatment

  • Relapse is not uncommon, with around 20-30% of patients experiencing relapse at a median of 42-48 months 3, 5, 7.
  • Patients who relapse can be successfully retreated with cladribine, with around 80-90% achieving CR or partial response 3, 5, 7.

References

Guideline

Guideline Directed Topic Overview

Dr.Oracle Medical Advisory Board & Editors, 2025

Research

Extended follow-up of patients with hairy cell leukemia after treatment with cladribine.

Journal of clinical oncology : official journal of the American Society of Clinical Oncology, 2003

Research

Cladribine in the treatment of hairy-cell leukaemia.

Best practice & research. Clinical haematology, 2003

Research

Treatment of hairy-cell leukemia with cladribine: response, toxicity, and long-term follow-up.

Journal of clinical oncology : official journal of the American Society of Clinical Oncology, 1997

Research

Randomized Phase II Study of First-Line Cladribine With Concurrent or Delayed Rituximab in Patients With Hairy Cell Leukemia.

Journal of clinical oncology : official journal of the American Society of Clinical Oncology, 2020

Professional Medical Disclaimer

This information is intended for healthcare professionals. Any medical decision-making should rely on clinical judgment and independently verified information. The content provided herein does not replace professional discretion and should be considered supplementary to established clinical guidelines. Healthcare providers should verify all information against primary literature and current practice standards before application in patient care. Dr.Oracle assumes no liability for clinical decisions based on this content.

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