Article
Response in a child with a BRAF V600E mutated desmoplastic infantile astrocytoma upon retreatment with vemurafenib.
Pediatric blood & cancer - 1 Mar 2018
van Tilburg Cornelis M, Selt Florian, Sahm Felix, Bächli Heidi, Pfister Stefan M, Witt Olaf, Milde Till
Abstract excerpt
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...
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