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Pediatr Blood Cancer. 2018 Mar;65(3). doi: 10.1002/pbc.26893. Epub 2017 Dec 12.

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

van Tilburg CM1,2,3, Selt F1,2,3,4, Sahm F3,5,6, Bächli H7, Pfister SM1,2,3,8, Witt O1,2,3,4, Milde T1,2,3,4.

Author information

1
Department of Pediatric Hematology and Oncology, Hopp Children's Cancer Center at the NCT Heidelberg (KiTZ), Heidelberg, Germany.
2
Department of Pediatric Hematology and Oncology, Heidelberg University Hospital, Heidelberg, Germany.
3
German Cancer Research Center (DKFZ) and German Consortium for Translational Cancer Research (DKTK), Heidelberg, Germany.
4
Clinical Cooperation Unit Pediatric Oncology, German Cancer Research Center (DKFZ), Heidelberg, Germany.
5
Department of Neuropathology, Institute of Pathology, University Hospital Heidelberg, Heidelberg, Germany.
6
Clinical Cooperation Unit Neuropathology, German Consortium for Translational Cancer Research (DKTK), German Cancer Research Center (DKFZ), Heidelberg, Germany.
7
Department of Neurosurgery, Heidelberg University Hospital, Heidelberg, Germany.
8
Division of Pediatric Neurooncology, German Cancer Research Center (DKFZ), Heidelberg, Germany.

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.

KEYWORDS:

BRAF V600E inhibitor; child; desmoplastic infantile astrocytoma; infant; low-grade glioma; vemurafenib

PMID:
29230924
DOI:
10.1002/pbc.26893
[Indexed for MEDLINE]

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