Successful re-treatment of a relapsed V600E mutated HCL patient with low-dose vemurafenib

Caroline Bailleux1,*, Guillaume Robert2,3,*, Clémence Ginet2,3, Daniel Re1, Antoine Thyss1, Isabelle Sudaka4, Isabelle Peyrottes5, Paul Hofman6, Patrick Auberger2,3,**and Frederic Peyrade1,**

1 Department of Medical Oncology, Centre Antoine Lacassagne, Nice, France

2 INSERM, U1065, TEAM 2, Nice, France

3 University of Nice, Nice, France

4 Laboratory of Hematology, CHU Nice, Nice, France

5 Department of Pathology, Centre Antoine Lacassagne, Nice, France

6 Laboratory of Clinical and Experimental Pathology, Pasteur Hospital, CHU Nice

* Co-first authors

** Co-last authors


Guillaume Robert, email:

Keywords: hairy-cell leukemia, V600E mutation, relapse, vemurafenib, re-treatment

Received: October 10, 2014 Accepted: December 22, 2014 Published: December 26, 2014


Hairy cell leukemia (HCL) is chronic B-cell lymphoproliferative disorder that accounts for 2% of all leukemia. Recent identification of the recurrent V600E BRAF mutation in a majority of HCL patients has led some teams to evaluate the clinical potential of vemurafenib, a BRAF V600 specific inhibitor in a limited number of refractory HCL patients. Recently, we published the case of an HCL patient successfully treated with a low dose of vemurafenib. Eight months after the ending of treatment this patient relapsed. We present here the successful retreatment of this patient with a second line of vemurafenib. Our data suggest for the first time that vemurafenib at the dose of 240 mg once a day could be sufficient to maintain a complete hematological remission after an initial induction treatment with low-dose vemurafenib (2 x 240 mg) daily without inducing major toxicity.

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