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000131762 1001_ $$00000-0001-5274-910X$$avan Tilburg, Cornelis M$$b0$$eFirst author
000131762 245__ $$aResponse in a child with a BRAF V600E mutated desmoplastic infantile astrocytoma upon retreatment with vemurafenib.
000131762 260__ $$aNew York, NY$$bWiley$$c2018
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000131762 520__ $$aInfants with low-grade glioma (LGG) and diencephalic syndrome have a poor outcome. The patient described here had a desmoplastic infantile astrocytoma harboring a BRAF V600E mutation. After relapse following initial standard chemotherapy treatment, he was successfully treated with the BRAF V600E inhibitor vemurafenib at the age of 3 years 11 months and 5 years 0 months. A rapid response was observed on both occasions. This illustrates the possibility of continuous oncogenic addiction and the therapeutic potential of BRAF V600E inhibitor monotherapy in LGG, even in very young severely compromised children. BRAF V600E inhibition in LGG and possible (re-)treatment regimens are briefly discussed.
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000131762 7001_ $$0P:(DE-He78)a23e88cc676489fe05be8c178ceaf58e$$aSelt, Florian$$b1$$udkfz
000131762 7001_ $$0P:(DE-He78)a1f4b408b9155beb2a8f7cba4d04fe88$$aSahm, Felix$$b2$$udkfz
000131762 7001_ $$aBächli, Heidi$$b3
000131762 7001_ $$0P:(DE-He78)f746aa965c4e1af518b016de3aaff5d9$$aPfister, Stefan$$b4$$udkfz
000131762 7001_ $$0P:(DE-He78)143af26de9d57bf624771616318aaf7c$$aWitt, Olaf$$b5$$udkfz
000131762 7001_ $$0P:(DE-He78)0be2f86573954f87e97f8a4dbb05cb0f$$aMilde, Till$$b6$$eLast author$$udkfz
000131762 773__ $$0PERI:(DE-600)2130978-4$$a10.1002/pbc.26893$$gVol. 65, no. 3, p. e26893 -$$n3$$pe26893 -$$tPediatric blood & cancer$$v65$$x1545-5009$$y2018
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