What type of study is this?

This is a Literature Review study.

September 5, 2025

Threading through the data for chemotherapy-free alternatives in the treatment of hairy cell leukemia

Key Points

Targeted therapies offer a new outlook for hairy cell leukemia, reducing reliance on traditional chemotherapy.
High rates of minimal residual disease negativity have been observed with newer agents, indicating effective disease management.
BRAF and MEK inhibitors, as well as other innovative treatments, show significant potential for relapsed or refractory cases of HCL.
The shift towards chemotherapy-free therapy is driven by the long-term toxicities associated with conventional purine analog treatments.

Abstract

Hairy cell leukemia (HCL) is a rare, indolent lymphoproliferative disorder that is highly responsive to purine analog therapy. While agents such as cladribine and pentostatin remain standard front-line therapies, their long-term toxicities-particularly myelosuppression and immune dysfunction-have led to growing interest in chemotherapy-free agents. Recent advances in molecular characterization of HCL, including the near-universal presence of BRAF V600E mutations, have led to an era of targeted therapeutics for the treatment of this disease. In this review, we summarize existing and emerging treatment strategies for HCL, including BRAF and MEK inhibitors, Bruton tyrosine kinase (BTK) inhibitors, anti-CD20 monoclonal antibodies, and venetoclax. These approaches offer therapeutic promise for patients with relapsed/refractory disease and for those ineligible to receive traditional chemotherapy. These regimens also result in high rates of minimal residual disease (MRD) negativity, potentially bringing us closer to the cure for HCL.

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