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Long-term Efficacy of Single-agent Vemurafenib for Pleomorphic Xanthoastrocytoma

Finch, Elizabeth A., MD*; Elton, Scott W., MD, FAANS, FAAP†,¶; Huang, Benjamin Y., MD∥,¶; Trembath, Dimitri G., MD, PhD‡,§,¶; Blatt, Julie, MD*,‡,¶

Journal of Pediatric Hematology/Oncology: December 27, 2018 - Volume Publish Ahead of Print - Issue - p
doi: 10.1097/MPH.0000000000001395
Clinical and Laboratory Observations: PDF Only

Pleomorphic xanthoastrocytoma is a malignant brain tumor that has a good prognosis with complete resection but does not respond well to chemotherapy if there is residual tumor. BRAFV600E mutations are common in pleomorphic xanthoastrocytomas and provide an additional means for treatment when excision is not possible. Monotherapy with the BRAF V600E inhibitor vemurafenib has only been reported in a small number of cases and mostly in adults. We present the case of a 16-year-old male who responded to vemurafenib monotherapy initially and had an additional response to vemurafenib following progression after a brief time off the medication.

Departments of *Pediatrics, Division of Pediatric Hematology-Oncology

Neurosurgery, Division of Pediatric Neurosurgery

§Pathology and Lab Medicine


The Lineberger Comprehensive Cancer Center

The University of North Carolina School of Medicine, Chapel Hill, NC

This case was included in the large international cohort of patients with BRAFV600E mutated pediatric low-grade gliomas written by Lassaletta et al, however, it was only briefly mentioned.

The authors declare no conflict of interest.

Reprints: Elizabeth A. Finch, MD, c/o the Division of Pediatric Hematology-Oncology, 170 Manning Dr. CB2736 POB1185A Chapel Hill, NC 27599-7236 (e-mail:

Received May 18, 2018

Accepted November 6, 2018

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