Pilomyxoid astrocytoma treated successfully with vemurafenib. Issue 11 (12th May 2014)

• Record Type: Journal Article
• Authors: Skrypek, Mary; Foreman, Nicholas; Guillaume, Daniel; Moertel, Christopher
• Is Part Of: Pediatric blood & cancer. Volume 61:Issue 11(2014:Nov.)
• Page Start: 2099
• Page End: 2100
• Publication Date: 2014-05-12
• Subjects: Tumors in children -- Periodicals; Blood -- Diseases -- Periodicals; Cancer in children -- Periodicals; 618.92
• DOI: 10.1002/pbc.25084
• Languages: English
• ISSNs: 1545-5009

Abstract

<abstract abstract-type="main"> <title> <x xml:space="preserve">Abstract</x> </title> <sec id="pbc25084-sec-0001" sec-type="section"> <p>The BRAF V600E missense mutation is known to be present in a subset of central nervous system tumors. We report a patient with a BRAF V600E mutated pilomyxoid astrocytoma who failed multiple conventional chemotherapy regimens. Treatment with vemurafenib, a molecularly targeted therapy against the mutant BRAF V600E kinase, combined with vinblastine resulted in tumor regression. Furthermore, this patient experienced almost immediate progression of disease after holding vemurafenib for only 2–3 weeks, suggesting that the tumor response is vemurafenib dependent. This population of patients may benefit from targeted therapy and testing of individual tumors for BRAF mutations is justified. Pediatr Blood Cancer 2014;61:2099–2100. © 2014 Wiley Periodicals, Inc.</p> </sec> </abstract>