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Response in a child with a BRAF V600E mutated desmoplastic infantile astrocytoma upon retreatment with vemurafenib

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 inh...

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Bibliographic Details
Main Authors: Tilburg, Cornelis M. van (Author) , Selt, Florian (Author) , Sahm, Felix (Author) , Bächli, Heidi (Author) , Pfister, Stefan (Author) , Witt, Olaf (Author) , Milde, Till (Author)
Format: Article (Journal)
Language:English
Published: 2018
In: Pediatric blood & cancer
Year: 2017, Volume: 65, Issue: 3, Pages: 1-4
ISSN:1545-5017
DOI:10.1002/pbc.26893
Online Access:Verlag, Volltext: http://dx.doi.org/10.1002/pbc.26893
Verlag, Volltext: https://onlinelibrary.wiley.com/doi/abs/10.1002/pbc.26893
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Author Notes:Cornelis M. van Tilburg, Florian Selt, Felix Sahm, Heidi Bächli, Stefan M. Pfister, Olaf Witt, Till Milde
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Summary: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.
Item Description:First published: 12 December 2017
Gesehen am 27.09.2018
Physical Description:Online Resource
ISSN:1545-5017
DOI:10.1002/pbc.26893

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