Next-generation personalised medicine for high-risk paediatric cancer patients

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Next-generation personalised medicine for high-risk paediatric cancer patients - The INFORM pilot study

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作者：Barbara C. Worst^a ; ^b ; ^c ; ¹ ; ^{b.worst@dkfz-heidelberg.de" class="auth_mail" title="E-mail the corresponding author} ; Cornelis M. van Tilburg^b ; ^c ; ^d ; ^e ; ¹ ; ^{cornelis.vantilburg@nct-heidelberg.de" class="auth_mail" title="E-mail the corresponding author} ; Gnana Prakash Balasubramanian^c ; ^f ; ^g ; ¹ ; ^{g.balasubramanian@dkfz-heidelberg.de" class="auth_mail" title="E-mail the corresponding author} ; Petra Fiesel^c ; ^h ; ⁱ ; ¹ ; ^{Petra.Fiesel@med.uni-heidelberg.de" class="auth_mail" title="E-mail the corresponding author} ; Ruth Witt^d ; ^{ruth.witt@nct-heidelberg.de" class="auth_mail" title="E-mail the corresponding author} ; Angelika Freitag^d ; ^{angelika.freitag@nct-heidelberg.de" class="auth_mail" title="E-mail the corresponding author} ; Miream Boudalil^c ; ^h ; ⁱ ; ^{Miream.Boudalil@med.uni-heidelberg.de" class="auth_mail" title="E-mail the corresponding author} ; Christopher Previti^j ; ^{christopher.previti@Dkfz-Heidelberg.de" class="auth_mail" title="E-mail the corresponding author} ; Stephan Wolf^j ; ^{s.wolf@dkfz-heidelberg.de" class="auth_mail" title="E-mail the corresponding author} ; Sabine Schmidt^j ; ^{sabine.schmidt@dkfz-heidelberg.de" class="auth_mail" title="E-mail the corresponding author} ; Sasithorn Chotewutmontri^j ; ^{s.chotewutmontri@Dkfz-Heidelberg.de" class="auth_mail" title="E-mail the corresponding author} ; Melanie Bewerunge-Hudler^j ; ^{M.Hudler@dkfz-heidelberg.de" class="auth_mail" title="E-mail the corresponding author} ; Matthias Schick^j ; ^{m.schick@Dkfz-Heidelberg.de" class="auth_mail" title="E-mail the corresponding author} ; Matthias Schlesner^c ; ^k ; ^{m.schlesner@Dkfz-Heidelberg.de" class="auth_mail" title="E-mail the corresponding author} ; Barbara Hutter^c ; ^f ; ^g ; ^{b.hutter@dkfz-heidelberg.de" class="auth_mail" title="E-mail the corresponding author} ; Lenka Taylor^l ; ^{Lenka.Taylor@med.uni-heidelberg.de" class="auth_mail" title="E-mail the corresponding author} ; Tobias Borst^m ; ^{Tobias.Borst@uk-erlangen.de" class="auth_mail" title="E-mail the corresponding author} ; Christian Sutterⁿ ; ^{C.Sutter@med.uni-heidelberg.de" class="auth_mail" title="E-mail the corresponding author} ; Claus R. Bartramⁿ ; ^{cr_bartram@med.uni-heidelberg.de" class="auth_mail" title="E-mail the corresponding author} ; Till Milde^b ; ^c ; ^e ; ^{t.milde@dkfz-heidelberg.de" class="auth_mail" title="E-mail the corresponding author} ; Elke Pfaff^a ; ^b ; ^c ; ^{e.pfaff@dkfz-heidelberg.de" class="auth_mail" title="E-mail the corresponding author} ; Andreas E. Kulozik^b ; ^{andreas.kulozik@med.uni-heidelberg.de" class="auth_mail" title="E-mail the corresponding author} ; Arend von Stackelberg^o ; ^{Arend.Stackelberg@charite.de" class="auth_mail" title="E-mail the corresponding author} ; Roland Meisel^p ; ^{Meisel@med.uni-duesseldorf.de" class="auth_mail" title="E-mail the corresponding author} ; Arndt Borkhardt^p ; ^{Arndt.Borkhardt@med.uni-duesseldorf.de" class="auth_mail" title="E-mail the corresponding author} ; Dirk Reinhardt^q ; ^{dirk.reinhardt@uk-essen.de" class="auth_mail" title="E-mail the corresponding author} ; Jan-Henning Klusmann^r ; ^{Klusmann.Jan-Henning@mh-hannover.de" class="auth_mail" title="E-mail the corresponding author} ; Gudrun Fleischhack^q ; ^{gudrun.fleischhack@uk-essen.de" class="auth_mail" title="E-mail the corresponding author} ; Stephan Tippelt^q ; ^{stephan.tippelt@uk-essen.de" class="auth_mail" title="E-mail the corresponding author} ; Uta Dirksen^s ; ^{Uta.Dirksen@ukmuenster.de" class="auth_mail" title="E-mail the corresponding author} ; Heribert Jü ; rgens^s ; ^{jurgh@ukmuenster.de" class="auth_mail" title="E-mail the corresponding author} ; Christof M. Kramm^t ; ^{christof.kramm@med.uni-goettingen.de" class="auth_mail" title="E-mail the corresponding author} ; Andre O. von Bueren^t ; ^u ; ^{Andre.vonBuren@hcuge.ch" class="auth_mail" title="E-mail the corresponding author} ; Frank Westermann^c ; ^v ; ^{frank.westermann@Dkfz-Heidelberg.de" class="auth_mail" title="E-mail the corresponding author} ; Matthias Fischer^w ; ^x ; ^y ; ^{matthias.fischer@uk-koeln.de" class="auth_mail" title="E-mail the corresponding author} ; Birgit Burkhardt^s ; ^{birgit.burkhardt@ukmuenster.de" class="auth_mail" title="E-mail the corresponding author} ; Wilhelm Wö ; &szlig ; mann^z ; ^{wilhelm.woessmann@paediat.med.uni-giessen.de" class="auth_mail" title="E-mail the corresponding author} ; Michaela Nathrath^aa ; ^ab ; ^{michaela.nathrath@klinikum-kassel.de" class="auth_mail" title="E-mail the corresponding author} ; Stefan S. Bielack^ac ; ^{s.bielack@klinikum-stuttgart.de" class="auth_mail" title="E-mail the corresponding author} ; Michael C. Frü ; hwald^ad ; ^{Michael.Fruehwald@klinikum-augsburg.de" class="auth_mail" title="E-mail the corresponding author} ; Simone Fulda^c ; ^ae ; ^{Simone.Fulda@kgu.de" class="auth_mail" title="E-mail the corresponding author} ; Thomas Klingebiel^af ; ^{thomas.klingebiel@kgu.de" class="auth_mail" title="E-mail the corresponding author} ; Ewa Koscielniak^ac ; ^{E.Koscielniak@klinikum-stuttgart.de" class="auth_mail" title="E-mail the corresponding author} ; Matthias Schwab^c ; ^ag ; ^ah ; ^{Matthias.Schwab@ikp-stuttgart.de" class="auth_mail" title="E-mail the corresponding author} ; Roman Tremmel^ag ; ^ah ; ^{Roman.Tremmel@ikp-stuttgart.de" class="auth_mail" title="E-mail the corresponding author} ; Pablo Herná ; iz Driever^o ; ^{pablo.hernaiz@charite.de" class="auth_mail" title="E-mail the corresponding author} ; Johannes H. Schulte^o ; ^q ; ^{johannes.schulte@charite.de" class="auth_mail" title="E-mail the corresponding author} ; Benedikt Brors^c ; ^f ; ^g ; ^{b.brors@dkfz-heidelberg.de" class="auth_mail" title="E-mail the corresponding author} ; Andreas von Deimling^c ; ^h ; ⁱ ; ^{andreas.vondeimling@dkfz-heidelberg.de" class="auth_mail" title="E-mail the corresponding author} ; Peter Lichter^c ; ^ai ; ^{Peter.Lichter@Dkfz-Heidelberg.de" class="auth_mail" title="E-mail the corresponding author}
关键词：Personalised medicine ; Oncology ; Paediatrics ; Precision medicine ; Molecular targeted therapy ; Deep sequencing ; Cancer ; Sarcoma ; Brain
刊名：European Journal of Cancer
出版年：2016
出版时间：September 2016
年：2016
卷：65
期：Complete
页码：91-101
全文大小：1139 K

文摘

•: Germany-wide personalised medicine study for high-risk paediatric cancer patients.
•: Comprehensive sequencing and analysis is feasible in clinically relevant time frame.
•: Half of patients across entities harbored a reasonable confidence druggable target.
•: Cases of clinical response to targeted agents demonstrate proof of principle.
•: This model can expand paediatric access to molecular profiling and targeted therapies.