Journal Article

DKFZ-2018-00061

http://join2-wiki.gsi.de/foswiki/pub/Main/Artwork/join2_logo100x88.png Response in a child with a BRAF V600E mutated desmoplastic infantile astrocytoma upon retreatment with vemurafenib.

van Tilburg, C. M. (First author)DKFZ* ; Selt, F.DKFZ* ; Sahm, F.DKFZ* ; Bächli, H. ; Pfister, S.DKFZ* ; Witt, O.DKFZ* ; Milde, T. (Last author)DKFZ*

2018
Wiley New York, NY

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Please use a persistent id in citations: doi:10.1002/pbc.26893

Abstract: Infants with low-grade glioma (LGG) and diencephalic syndrome have a poor outcome. The patient described here had a desmoplastic infantile astrocytoma harboring a BRAF V600E mutation. After relapse following initial standard chemotherapy treatment, he was successfully treated with the BRAF V600E inhibitor vemurafenib at the age of 3 years 11 months and 5 years 0 months. A rapid response was observed on both occasions. This illustrates the possibility of continuous oncogenic addiction and the therapeutic potential of BRAF V600E inhibitor monotherapy in LGG, even in very young severely compromised children. BRAF V600E inhibition in LGG and possible (re-)treatment regimens are briefly discussed.

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Contributing Institute(s):

1. DKTK Heidelberg (L101)
2. KKE Pädiatrische Onkologie (G340)
3. KKE Neuropathologie (G380)
4. B062 Pädiatrische Neuroonkologie (B062)

Research Program(s):

1. 317 - Translational cancer research (POF3-317) (POF3-317)

Appears in the scientific report 2018

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Database coverage:
; BIOSIS Previews ; Current Contents - Clinical Medicine ; IF < 5 ; JCR ; NCBI Molecular Biology Database ; SCOPUS ; Science Citation Index ; Science Citation Index Expanded ; Thomson Reuters Master Journal List ; Web of Science Core Collection

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