guest ::
| Home > Publications database > Effective targeting of PDGFRA-altered high-grade glioma with avapritinib. > print |
| Home > Publications database > Effective targeting of PDGFRA-altered high-grade glioma with avapritinib. > print |
| 001 | 300325 | ||
| 005 | 20250416143334.0 | ||
| 024 | 7 | _ | |a 10.1016/j.ccell.2025.02.018 |2 doi |
| 024 | 7 | _ | |a pmid:40086436 |2 pmid |
| 024 | 7 | _ | |a 1535-6108 |2 ISSN |
| 024 | 7 | _ | |a 1878-3686 |2 ISSN |
| 024 | 7 | _ | |a altmetric:175155232 |2 altmetric |
| 037 | _ | _ | |a DKFZ-2025-00771 |
| 041 | _ | _ | |a English |
| 082 | _ | _ | |a 610 |
| 100 | 1 | _ | |a Mayr, Lisa |b 0 |
| 245 | _ | _ | |a Effective targeting of PDGFRA-altered high-grade glioma with avapritinib. |
| 260 | _ | _ | |a Cambridge, Mass. |c 2025 |b Cell Press |
| 336 | 7 | _ | |a article |2 DRIVER |
| 336 | 7 | _ | |a Output Types/Journal article |2 DataCite |
| 336 | 7 | _ | |a Journal Article |b journal |m journal |0 PUB:(DE-HGF)16 |s 1744806768_25806 |2 PUB:(DE-HGF) |
| 336 | 7 | _ | |a ARTICLE |2 BibTeX |
| 336 | 7 | _ | |a JOURNAL_ARTICLE |2 ORCID |
| 336 | 7 | _ | |a Journal Article |0 0 |2 EndNote |
| 500 | _ | _ | |a 2025 Apr 14;43(4):740-756.e8 |
| 520 | _ | _ | |a 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. |
| 536 | _ | _ | |a 312 - Funktionelle und strukturelle Genomforschung (POF4-312) |0 G:(DE-HGF)POF4-312 |c POF4-312 |f POF IV |x 0 |
| 588 | _ | _ | |a Dataset connected to CrossRef, PubMed, , Journals: inrepo02.dkfz.de |
| 650 | _ | 7 | |a PDGFRA alteration |2 Other |
| 650 | _ | 7 | |a PDGFRA amplification |2 Other |
| 650 | _ | 7 | |a PDGFRA inhibitor |2 Other |
| 650 | _ | 7 | |a PDGFRA mutation |2 Other |
| 650 | _ | 7 | |a avapritinib |2 Other |
| 650 | _ | 7 | |a brain penetrance |2 Other |
| 650 | _ | 7 | |a diffuse midline glioma |2 Other |
| 650 | _ | 7 | |a glioblastoma |2 Other |
| 650 | _ | 7 | |a high-grade glioma |2 Other |
| 650 | _ | 7 | |a tyrosine kinase inhibitor |2 Other |
| 700 | 1 | _ | |a Neyazi, Sina |b 1 |
| 700 | 1 | _ | |a Schwark, Kallen |b 2 |
| 700 | 1 | _ | |a Trissal, Maria |b 3 |
| 700 | 1 | _ | |a Beck, Alexander |b 4 |
| 700 | 1 | _ | |a Labelle, Jenna |b 5 |
| 700 | 1 | _ | |a Eder, Sebastian K |b 6 |
| 700 | 1 | _ | |a Weiler-Wichtl, Liesa |b 7 |
| 700 | 1 | _ | |a Marques, Joana G |b 8 |
| 700 | 1 | _ | |a de Biagi-Junior, Carlos A O |b 9 |
| 700 | 1 | _ | |a Lo Cascio, Costanza |b 10 |
| 700 | 1 | _ | |a Chapman, Owen |b 11 |
| 700 | 1 | _ | |a Sridhar, Sunita |b 12 |
| 700 | 1 | _ | |a Kenkre, Rishaan |b 13 |
| 700 | 1 | _ | |a Dutta, Aditi |b 14 |
| 700 | 1 | _ | |a Wang, Shanqing |b 15 |
| 700 | 1 | _ | |a Wang, Jessica |b 16 |
| 700 | 1 | _ | |a Hack, Olivia |b 17 |
| 700 | 1 | _ | |a Nascimento, Andrezza |b 18 |
| 700 | 1 | _ | |a Nguyen, Cuong M |b 19 |
| 700 | 1 | _ | |a Castellani, Sophia |b 20 |
| 700 | 1 | _ | |a Rozowsky, Jacob S |b 21 |
| 700 | 1 | _ | |a Groves, Andrew |b 22 |
| 700 | 1 | _ | |a Panditharatna, Eshini |b 23 |
| 700 | 1 | _ | |a Cruzeiro, Gustavo Alencastro Veiga |b 24 |
| 700 | 1 | _ | |a Haase, Rebecca D |b 25 |
| 700 | 1 | _ | |a Tabatabai, Kuscha |b 26 |
| 700 | 1 | _ | |a Madlener, Sibylle |b 27 |
| 700 | 1 | _ | |a Wadden, Jack |b 28 |
| 700 | 1 | _ | |a Adam, Tiffany |b 29 |
| 700 | 1 | _ | |a Kong, Seongbae |b 30 |
| 700 | 1 | _ | |a Miclea, Madeline |b 31 |
| 700 | 1 | _ | |a Patel, Tirth |b 32 |
| 700 | 1 | _ | |a Bruckner, Katharina |b 33 |
| 700 | 1 | _ | |a Senfter, Daniel |b 34 |
| 700 | 1 | _ | |a Lämmerer, Anna |b 35 |
| 700 | 1 | _ | |a Supko, Jeffrey |b 36 |
| 700 | 1 | _ | |a Guntner, Armin S |b 37 |
| 700 | 1 | _ | |a Palova, Hana |b 38 |
| 700 | 1 | _ | |a Neradil, Jakub |b 39 |
| 700 | 1 | _ | |a Stepien, Natalia |b 40 |
| 700 | 1 | _ | |a Lötsch-Gojo, Daniela |b 41 |
| 700 | 1 | _ | |a Berger, Walter |b 42 |
| 700 | 1 | _ | |a Leiss, Ulrike |b 43 |
| 700 | 1 | _ | |a Rosenmayr, Verena |b 44 |
| 700 | 1 | _ | |a Dorfer, Christian |b 45 |
| 700 | 1 | _ | |a Dieckmann, Karin |b 46 |
| 700 | 1 | _ | |a Peyrl, Andreas |b 47 |
| 700 | 1 | _ | |a Azizi, Amedeo A |b 48 |
| 700 | 1 | _ | |a Baumgartner, Alicia |b 49 |
| 700 | 1 | _ | |a Slaby, Ondrej |b 50 |
| 700 | 1 | _ | |a Pokorna, Petra |b 51 |
| 700 | 1 | _ | |a Clark, Louise M |b 52 |
| 700 | 1 | _ | |a Cameron, Amy |b 53 |
| 700 | 1 | _ | |a Nguyen, Quang-De |b 54 |
| 700 | 1 | _ | |a Wakimoto, Hiroaki |b 55 |
| 700 | 1 | _ | |a Dubois, Frank |b 56 |
| 700 | 1 | _ | |a Greenwald, Noah F |b 57 |
| 700 | 1 | _ | |a Bandopadhayay, Pratiti |b 58 |
| 700 | 1 | _ | |a Beroukhim, Rameen |b 59 |
| 700 | 1 | _ | |a Ligon, Keith |b 60 |
| 700 | 1 | _ | |a Kramm, Christof |b 61 |
| 700 | 1 | _ | |a Bronsema, Annika |b 62 |
| 700 | 1 | _ | |a Bailey, Simon |b 63 |
| 700 | 1 | _ | |a Stucklin, Ana Guerreiro |b 64 |
| 700 | 1 | _ | |a Mueller, Sabine |b 65 |
| 700 | 1 | _ | |a Skrypek, Mary |b 66 |
| 700 | 1 | _ | |a Martinez, Nina |b 67 |
| 700 | 1 | _ | |a Bowers, Daniel C |b 68 |
| 700 | 1 | _ | |a Jones, David |0 P:(DE-He78)551bb92841f634070997aa168d818492 |b 69 |u dkfz |
| 700 | 1 | _ | |a Jones, Chris |b 70 |
| 700 | 1 | _ | |a Jäger, Natalie |0 P:(DE-He78)bff9e3e3d86865d2b0836bb8f3ce98f3 |b 71 |u dkfz |
| 700 | 1 | _ | |a Sterba, Jaroslav |b 72 |
| 700 | 1 | _ | |a Müllauer, Leonhard |b 73 |
| 700 | 1 | _ | |a Haberler, Christine |b 74 |
| 700 | 1 | _ | |a Kumar-Sinha, Chandan |b 75 |
| 700 | 1 | _ | |a Chinnaiyan, Arul |b 76 |
| 700 | 1 | _ | |a Mody, Rajen |b 77 |
| 700 | 1 | _ | |a Chavez, Lukas |b 78 |
| 700 | 1 | _ | |a Furtner, Julia |b 79 |
| 700 | 1 | _ | |a Koschmann, Carl |b 80 |
| 700 | 1 | _ | |a Gojo, Johannes |b 81 |
| 700 | 1 | _ | |a Filbin, Mariella G |b 82 |
| 773 | _ | _ | |a 10.1016/j.ccell.2025.02.018 |g p. S1535610825000704 |0 PERI:(DE-600)2074034-7 |n 4 |p 740-756.e8 |t Cancer cell |v 43 |y 2025 |x 1535-6108 |
| 909 | C | O | |p VDB |o oai:inrepo02.dkfz.de:300325 |
| 910 | 1 | _ | |a Deutsches Krebsforschungszentrum |0 I:(DE-588b)2036810-0 |k DKFZ |b 69 |6 P:(DE-He78)551bb92841f634070997aa168d818492 |
| 910 | 1 | _ | |a Deutsches Krebsforschungszentrum |0 I:(DE-588b)2036810-0 |k DKFZ |b 71 |6 P:(DE-He78)bff9e3e3d86865d2b0836bb8f3ce98f3 |
| 913 | 1 | _ | |a DE-HGF |b Gesundheit |l Krebsforschung |1 G:(DE-HGF)POF4-310 |0 G:(DE-HGF)POF4-312 |3 G:(DE-HGF)POF4 |2 G:(DE-HGF)POF4-300 |4 G:(DE-HGF)POF |v Funktionelle und strukturelle Genomforschung |x 0 |
| 914 | 1 | _ | |y 2025 |
| 915 | _ | _ | |a DBCoverage |0 StatID:(DE-HGF)0200 |2 StatID |b SCOPUS |d 2024-12-20 |
| 915 | _ | _ | |a DBCoverage |0 StatID:(DE-HGF)0300 |2 StatID |b Medline |d 2024-12-20 |
| 915 | _ | _ | |a DBCoverage |0 StatID:(DE-HGF)0199 |2 StatID |b Clarivate Analytics Master Journal List |d 2024-12-20 |
| 915 | _ | _ | |a DBCoverage |0 StatID:(DE-HGF)1050 |2 StatID |b BIOSIS Previews |d 2024-12-20 |
| 915 | _ | _ | |a DBCoverage |0 StatID:(DE-HGF)0160 |2 StatID |b Essential Science Indicators |d 2024-12-20 |
| 915 | _ | _ | |a DBCoverage |0 StatID:(DE-HGF)1030 |2 StatID |b Current Contents - Life Sciences |d 2024-12-20 |
| 915 | _ | _ | |a DBCoverage |0 StatID:(DE-HGF)1190 |2 StatID |b Biological Abstracts |d 2024-12-20 |
| 915 | _ | _ | |a DBCoverage |0 StatID:(DE-HGF)1110 |2 StatID |b Current Contents - Clinical Medicine |d 2024-12-20 |
| 915 | _ | _ | |a WoS |0 StatID:(DE-HGF)0113 |2 StatID |b Science Citation Index Expanded |d 2024-12-20 |
| 915 | _ | _ | |a DBCoverage |0 StatID:(DE-HGF)0150 |2 StatID |b Web of Science Core Collection |d 2024-12-20 |
| 915 | _ | _ | |a JCR |0 StatID:(DE-HGF)0100 |2 StatID |b CANCER CELL : 2022 |d 2024-12-20 |
| 915 | _ | _ | |a DBCoverage |0 StatID:(DE-HGF)0600 |2 StatID |b Ebsco Academic Search |d 2024-12-20 |
| 915 | _ | _ | |a Peer Review |0 StatID:(DE-HGF)0030 |2 StatID |b ASC |d 2024-12-20 |
| 915 | _ | _ | |a IF >= 50 |0 StatID:(DE-HGF)9950 |2 StatID |b CANCER CELL : 2022 |d 2024-12-20 |
| 920 | 1 | _ | |0 I:(DE-He78)B360-20160331 |k B360 |l Pädiatrische Gliomforschung |x 0 |
| 920 | 1 | _ | |0 I:(DE-He78)B062-20160331 |k B062 |l B062 Pädiatrische Neuroonkologie |x 1 |
| 980 | _ | _ | |a journal |
| 980 | _ | _ | |a VDB |
| 980 | _ | _ | |a I:(DE-He78)B360-20160331 |
| 980 | _ | _ | |a I:(DE-He78)B062-20160331 |
| 980 | _ | _ | |a UNRESTRICTED |
| Library | Collection | CLSMajor | CLSMinor | Language | Author |
|---|