Improving treatment for hairy cell leukemia northwesternu
Vemurafenib, an oral BRAF inhibitor, has demonstrated high response rates in relapsed/refractory hairy cell leukemia . However, little is known about long-term outcomes and response to retreatment. Herein, we report the results of 36 patients with R/R HCL treated with vemurafenib from the United States arm of the phase 2 clinical trial . The best overall response rate was 86%, including 33% complete response and 53% partial response .
Fourteen of 21 relapsed patients were retreated with vemurafenib, with 86% achieving complete hematologic response. Two patients acquired resistance to vemurafenib with the emergence of new KRAS and CDKN2A mutations, respectively. Six of 12 responders to vemurafenib retreatment experienced another relapse with a median RFS of 12.7 months. Overall survival was 82% at 4 years, with a significantly shorter OS in patients who relapsed within 1 year of initial treatment with vemurafenib.
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