guest ::
| Home > Publications database > Next-generation personalised medicine for high-risk paediatric cancer patients - The INFORM pilot study. > print |
| Home > Publications database > Next-generation personalised medicine for high-risk paediatric cancer patients - The INFORM pilot study. > print |
| 001 | 130973 | ||
| 005 | 20240228143504.0 | ||
| 024 | 7 | _ | |a 10.1016/j.ejca.2016.06.009 |2 doi |
| 024 | 7 | _ | |a pmid:27479119 |2 pmid |
| 024 | 7 | _ | |a 0014-2964 |2 ISSN |
| 024 | 7 | _ | |a 0959-8049 |2 ISSN |
| 024 | 7 | _ | |a 1879-0852 |2 ISSN |
| 024 | 7 | _ | |a 1879-2995 |2 ISSN |
| 024 | 7 | _ | |a altmetric:10207157 |2 altmetric |
| 037 | _ | _ | |a DKFZ-2017-06049 |
| 041 | _ | _ | |a eng |
| 082 | _ | _ | |a 610 |
| 100 | 1 | _ | |a Worst, Barbara |0 P:(DE-He78)fae1bf941c5fd76cf5356ecfa1243cc4 |b 0 |e First author |u dkfz |
| 245 | _ | _ | |a Next-generation personalised medicine for high-risk paediatric cancer patients - The INFORM pilot study. |
| 260 | _ | _ | |a Amsterdam [u.a.] |c 2016 |b Elsevier |
| 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 1524485188_2971 |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 |
| 520 | _ | _ | |a The Individualized Therapy for Relapsed Malignancies in Childhood (INFORM) precision medicine study is a nationwide German program for children with high-risk relapsed/refractory malignancies, which aims to identify therapeutic targets on an individualised basis. In a pilot phase, reported here, we developed the logistical and analytical pipelines necessary for rapid and comprehensive molecular profiling in a clinical setting. Fifty-seven patients from 20 centers were prospectively recruited. Malignancies investigated included sarcomas (n = 25), brain tumours (n = 23), and others (n = 9). Whole-exome, low-coverage whole-genome, and RNA sequencing were complemented with methylation and expression microarray analyses. Alterations were assessed for potential targetability according to a customised prioritisation algorithm and subsequently discussed in an interdisciplinary molecular tumour board. Next-generation sequencing data were generated for 52 patients, with the full analysis possible in 46 of 52. Turnaround time from sample receipt until first report averaged 28 d. Twenty-six patients (50%) harbored a potentially druggable alteration with a prioritisation score of intermediate or higher (level 4 of 7). Common targets included receptor tyrosine kinases, phosphoinositide 3-kinase-mammalian target of rapamycin pathway, mitogen-activated protein kinase pathway, and cell cycle control. Ten patients received a targeted therapy based on these findings, with responses observed in some previously treatment-refractory tumours. Comparative primary relapse analysis revealed substantial tumour evolution as well as one case of unsuspected secondary malignancy, highlighting the importance of re-biopsy at relapse. This study demonstrates the feasibility of comprehensive, real-time molecular profiling for high-risk paediatric cancer patients. This extended proof-of-concept, with examples of treatment consequences, expands upon previous personalised oncology endeavors, and presents a model with considerable interest and practical relevance in the burgeoning era of personalised medicine. |
| 536 | _ | _ | |a 317 - Translational cancer research (POF3-317) |0 G:(DE-HGF)POF3-317 |c POF3-317 |f POF III |x 0 |
| 588 | _ | _ | |a Dataset connected to CrossRef, PubMed, |
| 700 | 1 | _ | |a van Tilburg, Cornelis Martinus |0 P:(DE-He78)a6b5fcabf661bef95109dbee87dc5271 |b 1 |u dkfz |
| 700 | 1 | _ | |a Balasubramanian, Gnana Prakash |0 P:(DE-HGF)0 |b 2 |
| 700 | 1 | _ | |a Fiesel, Petra |0 P:(DE-He78)8de7015af76bd61fb0bc13bb3facb1df |b 3 |u dkfz |
| 700 | 1 | _ | |a Witt, Ruth |b 4 |
| 700 | 1 | _ | |a Freitag, Angelika |b 5 |
| 700 | 1 | _ | |a Boudalil, Miream |0 P:(DE-He78)679116f340f0d6abd96deb6b0c9e6f0f |b 6 |u dkfz |
| 700 | 1 | _ | |a Previti, Christopher |0 P:(DE-He78)1bae3784eb554a693b0e7ce03a20bdb8 |b 7 |u dkfz |
| 700 | 1 | _ | |a Wolf, Stephan |0 P:(DE-He78)1efb774993effe7a66a6ffc1b1cf9ccb |b 8 |u dkfz |
| 700 | 1 | _ | |a Schmidt, Sabine |0 P:(DE-He78)d6e2a991c1003a27b302b66a5477e6cd |b 9 |u dkfz |
| 700 | 1 | _ | |a Chotewutmontri, Sasithorn |0 P:(DE-He78)c843b772402b21e0544db687fbc8824a |b 10 |u dkfz |
| 700 | 1 | _ | |a Bewerunge-Hudler, Melanie |0 P:(DE-He78)7999346780553d7fab7ba69d5afdfa71 |b 11 |u dkfz |
| 700 | 1 | _ | |a Schick, Matthias |0 P:(DE-He78)c954da35a243d177fdc4a3924f4f8a5c |b 12 |u dkfz |
| 700 | 1 | _ | |a Schlesner, Matthias |0 P:(DE-He78)f2a782242acf94a3114d75c45dc75b37 |b 13 |u dkfz |
| 700 | 1 | _ | |a Hutter, Barbara |0 P:(DE-He78)135e8c8d1dd1b66b8127c3d1e3a9b6a0 |b 14 |u dkfz |
| 700 | 1 | _ | |a Taylor, Lenka |b 15 |
| 700 | 1 | _ | |a Borst, Tobias |b 16 |
| 700 | 1 | _ | |a Sutter, Christian |b 17 |
| 700 | 1 | _ | |a Bartram, Claus R |b 18 |
| 700 | 1 | _ | |a Milde, Till |0 P:(DE-He78)0be2f86573954f87e97f8a4dbb05cb0f |b 19 |u dkfz |
| 700 | 1 | _ | |a Pfaff, Elke |0 P:(DE-He78)c0538fae462bd98e3cd8d54ed885b0eb |b 20 |u dkfz |
| 700 | 1 | _ | |a Kulozik, Andreas E |b 21 |
| 700 | 1 | _ | |a von Stackelberg, Arend |b 22 |
| 700 | 1 | _ | |a Meisel, Roland |b 23 |
| 700 | 1 | _ | |a Borkhardt, Arndt |b 24 |
| 700 | 1 | _ | |a Reinhardt, Dirk |b 25 |
| 700 | 1 | _ | |a Klusmann, Jan-Henning |b 26 |
| 700 | 1 | _ | |a Fleischhack, Gudrun |b 27 |
| 700 | 1 | _ | |a Tippelt, Stephan |b 28 |
| 700 | 1 | _ | |a Dirksen, Uta |b 29 |
| 700 | 1 | _ | |a Jürgens, Heribert |b 30 |
| 700 | 1 | _ | |a Kramm, Christof M |b 31 |
| 700 | 1 | _ | |a von Bueren, Andre O |b 32 |
| 700 | 1 | _ | |a Westermann, Frank |0 P:(DE-He78)91f32735ee876c579d63c05a7f4778dd |b 33 |u dkfz |
| 700 | 1 | _ | |a Fischer, Matthias |b 34 |
| 700 | 1 | _ | |a Burkhardt, Birgit |b 35 |
| 700 | 1 | _ | |a Wößmann, Wilhelm |b 36 |
| 700 | 1 | _ | |a Nathrath, Michaela |b 37 |
| 700 | 1 | _ | |a Bielack, Stefan S |b 38 |
| 700 | 1 | _ | |a Frühwald, Michael C |b 39 |
| 700 | 1 | _ | |a Fulda, Simone |0 P:(DE-HGF)0 |b 40 |
| 700 | 1 | _ | |a Klingebiel, Thomas |b 41 |
| 700 | 1 | _ | |a Koscielniak, Ewa |b 42 |
| 700 | 1 | _ | |a Schwab, Matthias |0 P:(DE-He78)0321c153233e619c095d93d9d5546c9d |b 43 |u dkfz |
| 700 | 1 | _ | |a Tremmel, Roman |b 44 |
| 700 | 1 | _ | |a Driever, Pablo Hernáiz |b 45 |
| 700 | 1 | _ | |a Schulte, Johannes H |b 46 |
| 700 | 1 | _ | |a Brors, Benedikt |0 P:(DE-He78)fc949170377b58098e46141d95c72661 |b 47 |u dkfz |
| 700 | 1 | _ | |a von Deimling, Andreas |0 P:(DE-He78)a8a10626a848d31e70cfd96a133cc144 |b 48 |u dkfz |
| 700 | 1 | _ | |a Lichter, Peter |0 P:(DE-He78)e13b4363c5fe858044ef8a39c02c870c |b 49 |u dkfz |
| 700 | 1 | _ | |a Eggert, Angelika |b 50 |
| 700 | 1 | _ | |a Capper, David |0 P:(DE-He78)51bf9ae9cb5771b30c483e5597ef606c |b 51 |u dkfz |
| 700 | 1 | _ | |a Pfister, Stefan |0 P:(DE-He78)f746aa965c4e1af518b016de3aaff5d9 |b 52 |u dkfz |
| 700 | 1 | _ | |a Jones, David |0 P:(DE-He78)551bb92841f634070997aa168d818492 |b 53 |u dkfz |
| 700 | 1 | _ | |a Witt, Olaf |0 P:(DE-He78)143af26de9d57bf624771616318aaf7c |b 54 |e Last author |u dkfz |
| 773 | _ | _ | |a 10.1016/j.ejca.2016.06.009 |g Vol. 65, p. 91 - 101 |0 PERI:(DE-600)1468190-0 |p 91 - 101 |t European journal of cancer |v 65 |y 2016 |x 0959-8049 |
| 909 | C | O | |o oai:inrepo02.dkfz.de:130973 |p VDB |
| 910 | 1 | _ | |a Deutsches Krebsforschungszentrum |0 I:(DE-588b)2036810-0 |k DKFZ |b 0 |6 P:(DE-He78)fae1bf941c5fd76cf5356ecfa1243cc4 |
| 910 | 1 | _ | |a Deutsches Krebsforschungszentrum |0 I:(DE-588b)2036810-0 |k DKFZ |b 1 |6 P:(DE-He78)a6b5fcabf661bef95109dbee87dc5271 |
| 910 | 1 | _ | |a Deutsches Krebsforschungszentrum |0 I:(DE-588b)2036810-0 |k DKFZ |b 2 |6 P:(DE-HGF)0 |
| 910 | 1 | _ | |a Deutsches Krebsforschungszentrum |0 I:(DE-588b)2036810-0 |k DKFZ |b 3 |6 P:(DE-He78)8de7015af76bd61fb0bc13bb3facb1df |
| 910 | 1 | _ | |a Deutsches Krebsforschungszentrum |0 I:(DE-588b)2036810-0 |k DKFZ |b 6 |6 P:(DE-He78)679116f340f0d6abd96deb6b0c9e6f0f |
| 910 | 1 | _ | |a Deutsches Krebsforschungszentrum |0 I:(DE-588b)2036810-0 |k DKFZ |b 7 |6 P:(DE-He78)1bae3784eb554a693b0e7ce03a20bdb8 |
| 910 | 1 | _ | |a Deutsches Krebsforschungszentrum |0 I:(DE-588b)2036810-0 |k DKFZ |b 8 |6 P:(DE-He78)1efb774993effe7a66a6ffc1b1cf9ccb |
| 910 | 1 | _ | |a Deutsches Krebsforschungszentrum |0 I:(DE-588b)2036810-0 |k DKFZ |b 9 |6 P:(DE-He78)d6e2a991c1003a27b302b66a5477e6cd |
| 910 | 1 | _ | |a Deutsches Krebsforschungszentrum |0 I:(DE-588b)2036810-0 |k DKFZ |b 10 |6 P:(DE-He78)c843b772402b21e0544db687fbc8824a |
| 910 | 1 | _ | |a Deutsches Krebsforschungszentrum |0 I:(DE-588b)2036810-0 |k DKFZ |b 11 |6 P:(DE-He78)7999346780553d7fab7ba69d5afdfa71 |
| 910 | 1 | _ | |a Deutsches Krebsforschungszentrum |0 I:(DE-588b)2036810-0 |k DKFZ |b 12 |6 P:(DE-He78)c954da35a243d177fdc4a3924f4f8a5c |
| 910 | 1 | _ | |a Deutsches Krebsforschungszentrum |0 I:(DE-588b)2036810-0 |k DKFZ |b 13 |6 P:(DE-He78)f2a782242acf94a3114d75c45dc75b37 |
| 910 | 1 | _ | |a Deutsches Krebsforschungszentrum |0 I:(DE-588b)2036810-0 |k DKFZ |b 14 |6 P:(DE-He78)135e8c8d1dd1b66b8127c3d1e3a9b6a0 |
| 910 | 1 | _ | |a Deutsches Krebsforschungszentrum |0 I:(DE-588b)2036810-0 |k DKFZ |b 19 |6 P:(DE-He78)0be2f86573954f87e97f8a4dbb05cb0f |
| 910 | 1 | _ | |a Deutsches Krebsforschungszentrum |0 I:(DE-588b)2036810-0 |k DKFZ |b 20 |6 P:(DE-He78)c0538fae462bd98e3cd8d54ed885b0eb |
| 910 | 1 | _ | |a Deutsches Krebsforschungszentrum |0 I:(DE-588b)2036810-0 |k DKFZ |b 33 |6 P:(DE-He78)91f32735ee876c579d63c05a7f4778dd |
| 910 | 1 | _ | |a Deutsches Krebsforschungszentrum |0 I:(DE-588b)2036810-0 |k DKFZ |b 40 |6 P:(DE-HGF)0 |
| 910 | 1 | _ | |a Deutsches Krebsforschungszentrum |0 I:(DE-588b)2036810-0 |k DKFZ |b 43 |6 P:(DE-He78)0321c153233e619c095d93d9d5546c9d |
| 910 | 1 | _ | |a Deutsches Krebsforschungszentrum |0 I:(DE-588b)2036810-0 |k DKFZ |b 47 |6 P:(DE-He78)fc949170377b58098e46141d95c72661 |
| 910 | 1 | _ | |a Deutsches Krebsforschungszentrum |0 I:(DE-588b)2036810-0 |k DKFZ |b 48 |6 P:(DE-He78)a8a10626a848d31e70cfd96a133cc144 |
| 910 | 1 | _ | |a Deutsches Krebsforschungszentrum |0 I:(DE-588b)2036810-0 |k DKFZ |b 49 |6 P:(DE-He78)e13b4363c5fe858044ef8a39c02c870c |
| 910 | 1 | _ | |a Deutsches Krebsforschungszentrum |0 I:(DE-588b)2036810-0 |k DKFZ |b 51 |6 P:(DE-He78)51bf9ae9cb5771b30c483e5597ef606c |
| 910 | 1 | _ | |a Deutsches Krebsforschungszentrum |0 I:(DE-588b)2036810-0 |k DKFZ |b 52 |6 P:(DE-He78)f746aa965c4e1af518b016de3aaff5d9 |
| 910 | 1 | _ | |a Deutsches Krebsforschungszentrum |0 I:(DE-588b)2036810-0 |k DKFZ |b 53 |6 P:(DE-He78)551bb92841f634070997aa168d818492 |
| 910 | 1 | _ | |a Deutsches Krebsforschungszentrum |0 I:(DE-588b)2036810-0 |k DKFZ |b 54 |6 P:(DE-He78)143af26de9d57bf624771616318aaf7c |
| 913 | 1 | _ | |a DE-HGF |l Krebsforschung |1 G:(DE-HGF)POF3-310 |0 G:(DE-HGF)POF3-317 |2 G:(DE-HGF)POF3-300 |v Translational cancer research |x 0 |4 G:(DE-HGF)POF |3 G:(DE-HGF)POF3 |b Gesundheit |
| 914 | 1 | _ | |y 2016 |
| 915 | _ | _ | |a Nationallizenz |0 StatID:(DE-HGF)0420 |2 StatID |
| 915 | _ | _ | |a DBCoverage |0 StatID:(DE-HGF)0200 |2 StatID |b SCOPUS |
| 915 | _ | _ | |a DBCoverage |0 StatID:(DE-HGF)0300 |2 StatID |b Medline |
| 915 | _ | _ | |a DBCoverage |0 StatID:(DE-HGF)0310 |2 StatID |b NCBI Molecular Biology Database |
| 915 | _ | _ | |a JCR |0 StatID:(DE-HGF)0100 |2 StatID |b EUR J CANCER : 2015 |
| 915 | _ | _ | |a DBCoverage |0 StatID:(DE-HGF)0600 |2 StatID |b Ebsco Academic Search |
| 915 | _ | _ | |a Peer Review |0 StatID:(DE-HGF)0030 |2 StatID |b ASC |
| 915 | _ | _ | |a DBCoverage |0 StatID:(DE-HGF)0199 |2 StatID |b Thomson Reuters Master Journal List |
| 915 | _ | _ | |a WoS |0 StatID:(DE-HGF)0110 |2 StatID |b Science Citation Index |
| 915 | _ | _ | |a DBCoverage |0 StatID:(DE-HGF)0150 |2 StatID |b Web of Science Core Collection |
| 915 | _ | _ | |a WoS |0 StatID:(DE-HGF)0111 |2 StatID |b Science Citation Index Expanded |
| 915 | _ | _ | |a DBCoverage |0 StatID:(DE-HGF)1110 |2 StatID |b Current Contents - Clinical Medicine |
| 915 | _ | _ | |a DBCoverage |0 StatID:(DE-HGF)1030 |2 StatID |b Current Contents - Life Sciences |
| 915 | _ | _ | |a DBCoverage |0 StatID:(DE-HGF)1050 |2 StatID |b BIOSIS Previews |
| 915 | _ | _ | |a IF >= 5 |0 StatID:(DE-HGF)9905 |2 StatID |b EUR J CANCER : 2015 |
| 920 | 1 | _ | |0 I:(DE-He78)B062-20160331 |k B062 |l Pädiatrische Neuroonkologie |x 0 |
| 920 | 1 | _ | |0 I:(DE-He78)G040-20160331 |k G040 |l Klinische Studienzentrale |x 1 |
| 920 | 1 | _ | |0 I:(DE-He78)G200-20160331 |k G200 |l Angewandte Bioinformatik |x 2 |
| 920 | 1 | _ | |0 I:(DE-He78)G380-20160331 |k G380 |l KKE Neuropathologie |x 3 |
| 920 | 1 | _ | |0 I:(DE-He78)B080-20160331 |k B080 |l Theoretische Bioinformatik |x 4 |
| 920 | 1 | _ | |0 I:(DE-He78)G340-20160331 |k G340 |l KKE Pädiatrische Onkologie |x 5 |
| 920 | 1 | _ | |0 I:(DE-He78)B087-20160331 |k B087 |l Neuroblastom Genomik |x 6 |
| 920 | 1 | _ | |0 I:(DE-He78)W110-20160331 |k W110 |l Microarray Unit |x 7 |
| 920 | 1 | _ | |0 I:(DE-He78)B060-20160331 |k B060 |l Molekulare Genetik |x 8 |
| 920 | 1 | _ | |0 I:(DE-He78)L101-20160331 |k L101 |l DKTK Heidelberg |x 9 |
| 920 | 1 | _ | |0 I:(DE-He78)L501-20160331 |k L501 |l DKTK Frankfurt |x 10 |
| 920 | 1 | _ | |0 I:(DE-He78)L201-20160331 |k L201 |l DKTK Berlin |x 11 |
| 920 | 1 | _ | |0 I:(DE-He78)L401-20160331 |k L401 |l DKTK Essen |x 12 |
| 980 | _ | _ | |a journal |
| 980 | _ | _ | |a VDB |
| 980 | _ | _ | |a I:(DE-He78)B062-20160331 |
| 980 | _ | _ | |a I:(DE-He78)G040-20160331 |
| 980 | _ | _ | |a I:(DE-He78)G200-20160331 |
| 980 | _ | _ | |a I:(DE-He78)G380-20160331 |
| 980 | _ | _ | |a I:(DE-He78)B080-20160331 |
| 980 | _ | _ | |a I:(DE-He78)G340-20160331 |
| 980 | _ | _ | |a I:(DE-He78)B087-20160331 |
| 980 | _ | _ | |a I:(DE-He78)W110-20160331 |
| 980 | _ | _ | |a I:(DE-He78)B060-20160331 |
| 980 | _ | _ | |a I:(DE-He78)L101-20160331 |
| 980 | _ | _ | |a I:(DE-He78)L501-20160331 |
| 980 | _ | _ | |a I:(DE-He78)L201-20160331 |
| 980 | _ | _ | |a I:(DE-He78)L401-20160331 |
| 980 | _ | _ | |a UNRESTRICTED |
| Library | Collection | CLSMajor | CLSMinor | Language | Author |
|---|