van Tilburg, Cornelis M., Pfaff, Elke, Pajtler, Kristian W., Langenberg, Karin P. S., Fiesel, Petra, Jones, Barbara C., Balasubramanian, Gnana Prakash, Stark, Sebastian, Johann, Pascal D., Blattner-Johnson, Mirjam, Schramm, Kathrin, Dikow, Nicola, Hirsch, Steffen, Sutter, Christian, Grund, Kerstin, von Stackelberg, Arend, Kulozik, Andreas E., Lissat, Andrej, Borkhardt, Arndt, Meisel, Roland, Reinhardt, Dirk, Klusmann, Jan-Henning ORCID: 0000-0002-1070-0727, Fleischhack, Gudrun, Tippelt, Stephan, von Schweinitz, Dietrich, Schmid, Irene, Kramm, Christof M., von Bueren, Andre O., Calaminus, Gabriele, Vorwerk, Peter, Graf, Norbert ORCID: 0000-0002-2248-323X, Westermann, Frank, Fischer, Matthias, Eggert, Angelika ORCID: 0000-0003-3476-8184, Burkhardt, Birgit, Woessmann, Wilhelm, Nathrath, Michaela, Hecker-Nolting, Stefanie, Fruehwald, Michael C., Schneider, Dominik T. ORCID: 0000-0001-8153-1601, Brecht, Ines B., Ketteler, Petra, Fulda, Simone ORCID: 0000-0002-0459-6417, Koscielniak, Ewa, Meister, Michael T., Scheer, Monika, Hettmer, Simone, Schwab, Matthias, Tremmel, Roman, Ora, Ingrid, Hutter, Caroline, Gerber, Nicolas U., Lohi, Olli, Kazanowska, Bernarda ORCID: 0000-0002-7198-9122, Kattamis, Antonis, Filippidou, Maria, Goemans, Bianca, Zwaan, C. Michel, Milde, Till, Jaeger, Natalie, Wolf, Stephan, Reuss, David, Sahm, Felix ORCID: 0000-0001-5441-1962, von Deimling, Andreas ORCID: 0000-0002-5863-540X, Dirksen, Uta, Freitag, Angelika, Witt, Ruth, Lichter, Peter, Kopp-Schneider, Annette, Jones, David T. W., Molenaar, Jan J., Capper, David ORCID: 0000-0003-1945-497X, Pfister, Stefan M. and Witt, Olaf (2021). The Pediatric Precision Oncology INFORM Registry: Clinical Outcome and Benefit for Patients with Very High-Evidence Targets. Cancer Discov., 11 (11). S. 2764 - 2780. PHILADELPHIA: AMER ASSOC CANCER RESEARCH. ISSN 2159-8290

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Abstract

INFORM is a prospective, multinational registry gathering clinical and molecular data of relapsed, progressive, or high-risk pediatric patients with cancer. This report describes long-term follow-up of 519 patients in whom molecular alterations were evaluated according to a predefined seven-scale target prioritization algorithm. Mean turnaround time from sample receipt to report was 25.4 days. The highest target priority level was observed in 42 patients (8.1%). Of these, 20 patients received matched targeted treatment with a median progression-free survival of 204 days [95% confidence interval (CI), 99-not applicable], compared with 117 days (95% CI, 106-143; P = 0.011) in all other patients. The respective molecular targets were shown to be predictive for matched treatment response and not prognostic surrogates for improved outcome. Hereditary cancer predisposition syndromes were identified in 7.5% of patients, half of which were newly identified through the study. Integrated molecular analyses resulted in a change or refinement of diagnoses in 8.2% of cases. SIGNIFICANCE: The pediatric precision oncology INFORM registry prospectively tested a target prioritization algorithm in a real-world, multinational setting and identified subgroups of patients benefiting from matched targeted treatment with improved progression-free survival, refinement of diagnosis, and identification of hereditary cancer predisposition syndromes.

Item Type: Journal Article
Creators:
CreatorsEmailORCIDORCID Put Code
van Tilburg, Cornelis M.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Pfaff, ElkeUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Pajtler, Kristian W.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Langenberg, Karin P. S.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Fiesel, PetraUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Jones, Barbara C.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Balasubramanian, Gnana PrakashUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Stark, SebastianUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Johann, Pascal D.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Blattner-Johnson, MirjamUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Schramm, KathrinUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Dikow, NicolaUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Hirsch, SteffenUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Sutter, ChristianUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Grund, KerstinUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
von Stackelberg, ArendUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Kulozik, Andreas E.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Lissat, AndrejUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Borkhardt, ArndtUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Meisel, RolandUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Reinhardt, DirkUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Klusmann, Jan-HenningUNSPECIFIEDorcid.org/0000-0002-1070-0727UNSPECIFIED
Fleischhack, GudrunUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Tippelt, StephanUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
von Schweinitz, DietrichUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Schmid, IreneUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Kramm, Christof M.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
von Bueren, Andre O.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Calaminus, GabrieleUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Vorwerk, PeterUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Graf, NorbertUNSPECIFIEDorcid.org/0000-0002-2248-323XUNSPECIFIED
Westermann, FrankUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Fischer, MatthiasUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Eggert, AngelikaUNSPECIFIEDorcid.org/0000-0003-3476-8184UNSPECIFIED
Burkhardt, BirgitUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Woessmann, WilhelmUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Nathrath, MichaelaUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Hecker-Nolting, StefanieUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Fruehwald, Michael C.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Schneider, Dominik T.UNSPECIFIEDorcid.org/0000-0001-8153-1601UNSPECIFIED
Brecht, Ines B.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Ketteler, PetraUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Fulda, SimoneUNSPECIFIEDorcid.org/0000-0002-0459-6417UNSPECIFIED
Koscielniak, EwaUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Meister, Michael T.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Scheer, MonikaUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Hettmer, SimoneUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Schwab, MatthiasUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Tremmel, RomanUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Ora, IngridUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Hutter, CarolineUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Gerber, Nicolas U.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Lohi, OlliUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Kazanowska, BernardaUNSPECIFIEDorcid.org/0000-0002-7198-9122UNSPECIFIED
Kattamis, AntonisUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Filippidou, MariaUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Goemans, BiancaUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Zwaan, C. MichelUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Milde, TillUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Jaeger, NatalieUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Wolf, StephanUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Reuss, DavidUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Sahm, FelixUNSPECIFIEDorcid.org/0000-0001-5441-1962UNSPECIFIED
von Deimling, AndreasUNSPECIFIEDorcid.org/0000-0002-5863-540XUNSPECIFIED
Dirksen, UtaUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Freitag, AngelikaUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Witt, RuthUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Lichter, PeterUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Kopp-Schneider, AnnetteUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Jones, David T. W.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Molenaar, Jan J.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Capper, DavidUNSPECIFIEDorcid.org/0000-0003-1945-497XUNSPECIFIED
Pfister, Stefan M.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Witt, OlafUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
URN: urn:nbn:de:hbz:38-607888
DOI: 10.1158/2159-8290.CD-21-0094
Journal or Publication Title: Cancer Discov.
Volume: 11
Number: 11
Page Range: S. 2764 - 2780
Date: 2021
Publisher: AMER ASSOC CANCER RESEARCH
Place of Publication: PHILADELPHIA
ISSN: 2159-8290
Language: English
Faculty: Unspecified
Divisions: Unspecified
Subjects: no entry
Uncontrolled Keywords:
KeywordsLanguage
RETROSPECTIVE ANALYSIS; PROGNOSTIC-FACTORS; POOLED ANALYSIS; SOLID TUMORS; CHILDREN; ADOLESCENTS; SURVIVAL; THERAPY; RELAPSE; RISKMultiple languages
OncologyMultiple languages
URI: http://kups.ub.uni-koeln.de/id/eprint/60788

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