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@ARTICLE{vanTilburg:131762,
      author       = {C. M. van Tilburg$^*$ and F. Selt$^*$ and F. Sahm$^*$ and
                      H. Bächli and S. Pfister$^*$ and O. Witt$^*$ and T.
                      Milde$^*$},
      title        = {{R}esponse in a child with a {BRAF} {V}600{E} mutated
                      desmoplastic infantile astrocytoma upon retreatment with
                      vemurafenib.},
      journal      = {Pediatric blood $\&$ cancer},
      volume       = {65},
      number       = {3},
      issn         = {1545-5009},
      address      = {New York, NY},
      publisher    = {Wiley},
      reportid     = {DKFZ-2018-00061},
      pages        = {e26893 -},
      year         = {2018},
      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.},
      cin          = {L101 / G340 / G380 / B062},
      ddc          = {610},
      cid          = {I:(DE-He78)L101-20160331 / I:(DE-He78)G340-20160331 /
                      I:(DE-He78)G380-20160331 / I:(DE-He78)B062-20160331},
      pnm          = {317 - Translational cancer research (POF3-317)},
      pid          = {G:(DE-HGF)POF3-317},
      typ          = {PUB:(DE-HGF)16},
      pubmed       = {pmid:29230924},
      doi          = {10.1002/pbc.26893},
      url          = {https://inrepo02.dkfz.de/record/131762},
}