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@ARTICLE{Mayr:300325,
      author       = {L. Mayr and S. Neyazi and K. Schwark and M. Trissal and A.
                      Beck and J. Labelle and S. K. Eder and L. Weiler-Wichtl and
                      J. G. Marques and C. A. O. de Biagi-Junior and C. Lo Cascio
                      and O. Chapman and S. Sridhar and R. Kenkre and A. Dutta and
                      S. Wang and J. Wang and O. Hack and A. Nascimento and C. M.
                      Nguyen and S. Castellani and J. S. Rozowsky and A. Groves
                      and E. Panditharatna and G. A. V. Cruzeiro and R. D. Haase
                      and K. Tabatabai and S. Madlener and J. Wadden and T. Adam
                      and S. Kong and M. Miclea and T. Patel and K. Bruckner and
                      D. Senfter and A. Lämmerer and J. Supko and A. S. Guntner
                      and H. Palova and J. Neradil and N. Stepien and D.
                      Lötsch-Gojo and W. Berger and U. Leiss and V. Rosenmayr and
                      C. Dorfer and K. Dieckmann and A. Peyrl and A. A. Azizi and
                      A. Baumgartner and O. Slaby and P. Pokorna and L. M. Clark
                      and A. Cameron and Q.-D. Nguyen and H. Wakimoto and F.
                      Dubois and N. F. Greenwald and P. Bandopadhayay and R.
                      Beroukhim and K. Ligon and C. Kramm and A. Bronsema and S.
                      Bailey and A. G. Stucklin and S. Mueller and M. Skrypek and
                      N. Martinez and D. C. Bowers and D. Jones$^*$ and C. Jones
                      and N. Jäger$^*$ and J. Sterba and L. Müllauer and C.
                      Haberler and C. Kumar-Sinha and A. Chinnaiyan and R. Mody
                      and L. Chavez and J. Furtner and C. Koschmann and J. Gojo
                      and M. G. Filbin},
      title        = {{E}ffective targeting of {PDGFRA}-altered high-grade glioma
                      with avapritinib.},
      journal      = {Cancer cell},
      volume       = {43},
      number       = {4},
      issn         = {1535-6108},
      address      = {Cambridge, Mass.},
      publisher    = {Cell Press},
      reportid     = {DKFZ-2025-00771},
      pages        = {740-756.e8},
      year         = {2025},
      note         = {2025 Apr 14;43(4):740-756.e8},
      abstract     = {PDGFRA is crucial to tumorigenesis and frequently
                      genomically altered in high-grade glioma (HGG). In a
                      comprehensive dataset of pediatric HGG (n = 261), we detect
                      PDGFRA mutations and/or amplifications in $15\%$ of cases,
                      suggesting PDGFRA as a therapeutic target. We reveal that
                      the PDGFRA/KIT inhibitor avapritinib shows (1) selectivity
                      for PDGFRA inhibition, (2) distinct patterns of subcellular
                      effects, (3) in vitro and in vivo activity in
                      patient-derived HGG models, and (4) effective blood-brain
                      barrier penetration in mice and humans. Furthermore, we
                      report preliminary clinical real-world experience using
                      avapritinib in pediatric and young adult patients with
                      predominantly recurrent/refractory PDGFRA-altered HGG (n =
                      8). Our early data demonstrate that avapritinib is well
                      tolerated and results in radiographic response in 3/7 cases,
                      suggesting a potential role for avapritinib in the treatment
                      of HGG with specific PDGFRA alterations. Overall, these
                      translational results underscore the therapeutic potential
                      of PDGFRA inhibition with avapritinib in HGG.},
      keywords     = {PDGFRA alteration (Other) / PDGFRA amplification (Other) /
                      PDGFRA inhibitor (Other) / PDGFRA mutation (Other) /
                      avapritinib (Other) / brain penetrance (Other) / diffuse
                      midline glioma (Other) / glioblastoma (Other) / high-grade
                      glioma (Other) / tyrosine kinase inhibitor (Other)},
      cin          = {B360 / B062},
      ddc          = {610},
      cid          = {I:(DE-He78)B360-20160331 / I:(DE-He78)B062-20160331},
      pnm          = {312 - Funktionelle und strukturelle Genomforschung
                      (POF4-312)},
      pid          = {G:(DE-HGF)POF4-312},
      typ          = {PUB:(DE-HGF)16},
      pubmed       = {pmid:40086436},
      doi          = {10.1016/j.ccell.2025.02.018},
      url          = {https://inrepo02.dkfz.de/record/300325},
}