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The BRAF V600E mutation is present in tumor cells of hairy cell leukemia (HCL) and is a disease-defining biomarker (NEJM JW Oncol Hemtol Aug 2011 and N Engl J Med 2011; 364:2305). Now, two independent phase II trials, conducted in the U.S. and Italy, have tested the clinical activity of vemurafenib in previously treated HCL patients. Results from the two trials were combined into a single report.
A total of 50 evaluable patients received all planned 16 to 18 weeks of oral vemurafenib (960 mg twice daily). Patients had a median of three prior therapies (range, 1–12); approximately half were refractory to their most recent therapy.
In the 24 U.S. patients, the overall response rate (ORR) was 100%, the complete response rate (CRR) was 42%, and t…