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Hairy cell leukemia (HCL) is a rare chronic lymphoid malignancy characterized by cytopenia, splenomegaly, the BRAF V600E kinase-activating mutation, and high cell-surface expression of the B-cell marker CD20. Investigators report a phase 2 single-center trial of the oral BRAF inhibitor (BRAFi) vemurafenib combined with the anti-CD20 monoclonal antibody rituximab in patients with relapsed or refractory BRAF-mutated HCL.
Eligible patients had one or more cytopenias and primary treatment resistance, early progression, or severe side effects after purine nucleoside analogue chemotherapy. Patients with prior BRAFi therapy and those with active infection were eligible. Treatment included vemurafenib twice daily for 4 weeks with rituximab on days 1…