The pediatric precision oncology inform registry: Clinical outcome and benefit for patients with very high-evidence targets

Cornelis M. Van Tilburg; Elke Pfaff; Kristian W. Pajtler; Karin P.S. Langenberg; Petra Fiesel; Barbara C. Jones; Gnana Prakash Balasubramanian; Sebastian Stark; Pascal D. Johann; Mirjam Blattner-Johnson; Kathrin Schramm; Nicola Dikow; Steffen Hirsch; Christian Sutter; Kerstin Grund; Arend Von Stackelberg; Andreas E. Kulozik; Andrej Lissat; Arndt Borkhardt; Roland Meisel; Dirk Reinhardt; Jan Henning Klusmann; Gudrun Fleischhack; Stephan Tippelt; Dietrich Von Schweinitz; Irene Schmid; Christof M. Kramm; André O. Von Bueren; Gabriele Calaminus; Peter Vorwerk; Norbert Graf; Frank Westermann; Matthias Fischer; Angelika Eggert; Birgit Burkhardt; Wilhelm Wößmann; Michaela Nathrath; Stefanie Hecker-Nolting; Michael C. Frühwald; Dominik T. Schneider; Ines B. Brecht; Petra Ketteler; Simone Fulda; Ewa Koscielniak; Michael T. Meister; Monika Scheer; Simone Hettmer; Matthias Schwab; Roman Tremmel; Ingrid Øra; Caroline Hutter; Nicolas U. Gerber; Olli Lohi; Bernarda Kazanowska; Antonis Kattamis; Maria Filippidou; Bianca Goemans; C. Michel Zwaan; Till Milde; Natalie Jäger; Stephan Wolf; David Reuss; Felix Sahm; Andreas Von Deimling; Uta Dirksen; Angelika Freitag; Ruth Witt; Peter Lichter; Annette Kopp-Schneider; David T.W. Jones; Jan J. Molenaar; David Capper; Stefan M. Pfister; Olaf Witt

doi:10.1158/2159-8290.CD-21-0094

The pediatric precision oncology inform registry: Clinical outcome and benefit for patients with very high-evidence targets

Cornelis M. Van Tilburg, Elke Pfaff, Kristian W. Pajtler, Karin P.S. Langenberg, Petra Fiesel, Barbara C. Jones, Gnana Prakash Balasubramanian, Sebastian Stark, Pascal D. Johann, Mirjam Blattner-Johnson, Kathrin Schramm, Nicola Dikow, Steffen Hirsch, Christian Sutter, Kerstin Grund, Arend Von Stackelberg, Andreas E. Kulozik, Andrej Lissat, Arndt Borkhardt, Roland MeiselDirk Reinhardt, Jan Henning Klusmann, Gudrun Fleischhack, Stephan Tippelt, Dietrich Von Schweinitz, Irene Schmid, Christof M. Kramm, André O. Von Bueren, Gabriele Calaminus, Peter Vorwerk, Norbert Graf, Frank Westermann, Matthias Fischer, Angelika Eggert, Birgit Burkhardt, Wilhelm Wößmann, Michaela Nathrath, Stefanie Hecker-Nolting, Michael C. Frühwald, Dominik T. Schneider, Ines B. Brecht, Petra Ketteler, Simone Fulda, Ewa Koscielniak, Michael T. Meister, Monika Scheer, Simone Hettmer, Matthias Schwab, Roman Tremmel, Ingrid Øra, Caroline Hutter, Nicolas U. Gerber, Olli Lohi, Bernarda Kazanowska, Antonis Kattamis, Maria Filippidou, Bianca Goemans, C. Michel Zwaan, Till Milde, Natalie Jäger, Stephan Wolf, David Reuss, Felix Sahm, Andreas Von Deimling, Uta Dirksen, Angelika Freitag, Ruth Witt, Peter Lichter, Annette Kopp-Schneider, David T.W. Jones, Jan J. Molenaar, David Capper, Stefan M. Pfister, Olaf Witt

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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.

Original language	English
Pages (from-to)	2764-2779
Number of pages	16
Journal	CANCER DISCOVERY
Volume	11
Issue number	11
DOIs	https://doi.org/10.1158/2159-8290.CD-21-0094
Publication status	Published - 2021
Publication type	A1 Journal article-refereed

Publication forum classification

Publication forum level 3

ASJC Scopus subject areas

Oncology

UN SDGs

This output contributes to the following UN Sustainable Development Goals (SDGs)

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10.1158/2159-8290.CD-21-0094Licence: CC BY-NC-ND

2764.full-1Final published version, 13 MBLicence: CC BY-NC-ND

https://urn.fi/URN:NBN:fi:tuni-202201071123Licence: CC BY-NC-ND

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Van Tilburg, C. M., Pfaff, E., Pajtler, K. W., Langenberg, K. P. S., Fiesel, P., Jones, B. C., Balasubramanian, G. P., Stark, S., Johann, P. D., Blattner-Johnson, M., Schramm, K., Dikow, N., Hirsch, S., Sutter, C., Grund, K., Von Stackelberg, A., Kulozik, A. E., Lissat, A., Borkhardt, A., ... Witt, O. (2021). The pediatric precision oncology inform registry: Clinical outcome and benefit for patients with very high-evidence targets. CANCER DISCOVERY, 11(11), 2764-2779. https://doi.org/10.1158/2159-8290.CD-21-0094

@article{d49e0dc93b6c4409bffaf6d36b721c6c,

title = "The pediatric precision oncology inform registry: Clinical outcome and benefit for patients with very high-evidence targets",

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.",

author = "{Van Tilburg}, {Cornelis M.} and Elke Pfaff and Pajtler, {Kristian W.} and Langenberg, {Karin P.S.} and Petra Fiesel and Jones, {Barbara C.} and Balasubramanian, {Gnana Prakash} and Sebastian Stark and Johann, {Pascal D.} and Mirjam Blattner-Johnson and Kathrin Schramm and Nicola Dikow and Steffen Hirsch and Christian Sutter and Kerstin Grund and {Von Stackelberg}, Arend and Kulozik, {Andreas E.} and Andrej Lissat and Arndt Borkhardt and Roland Meisel and Dirk Reinhardt and Klusmann, {Jan Henning} and Gudrun Fleischhack and Stephan Tippelt and {Von Schweinitz}, Dietrich and Irene Schmid and Kramm, {Christof M.} and {Von Bueren}, {Andr{\'e} O.} and Gabriele Calaminus and Peter Vorwerk and Norbert Graf and Frank Westermann and Matthias Fischer and Angelika Eggert and Birgit Burkhardt and Wilhelm W{\"o}{\ss}mann and Michaela Nathrath and Stefanie Hecker-Nolting and Fr{\"u}hwald, {Michael C.} and Schneider, {Dominik T.} and Brecht, {Ines B.} and Petra Ketteler and Simone Fulda and Ewa Koscielniak and Meister, {Michael T.} and Monika Scheer and Simone Hettmer and Matthias Schwab and Roman Tremmel and Ingrid {\O}ra and Caroline Hutter and Gerber, {Nicolas U.} and Olli Lohi and Bernarda Kazanowska and Antonis Kattamis and Maria Filippidou and Bianca Goemans and Zwaan, {C. Michel} and Till Milde and Natalie J{\"a}ger and Stephan Wolf and David Reuss and Felix Sahm and {Von Deimling}, Andreas and Uta Dirksen and Angelika Freitag and Ruth Witt and Peter Lichter and Annette Kopp-Schneider and Jones, {David T.W.} and Molenaar, {Jan J.} and David Capper and Pfister, {Stefan M.} and Olaf Witt",

note = "Publisher Copyright: {\textcopyright} 2021 The Authors; Published by the American Association for Cancer Research.",

year = "2021",

doi = "10.1158/2159-8290.CD-21-0094",

language = "English",

volume = "11",

pages = "2764--2779",

journal = "CANCER DISCOVERY",

issn = "2159-8274",

publisher = "American Association for Cancer Research",

number = "11",

}

Van Tilburg, CM, Pfaff, E, Pajtler, KW, Langenberg, KPS, Fiesel, P, Jones, BC, Balasubramanian, GP, Stark, S, Johann, PD, Blattner-Johnson, M, Schramm, K, Dikow, N, Hirsch, S, Sutter, C, Grund, K, Von Stackelberg, A, Kulozik, AE, Lissat, A, Borkhardt, A, Meisel, R, Reinhardt, D, Klusmann, JH, Fleischhack, G, Tippelt, S, Von Schweinitz, D, Schmid, I, Kramm, CM, Von Bueren, AO, Calaminus, G, Vorwerk, P, Graf, N, Westermann, F, Fischer, M, Eggert, A, Burkhardt, B, Wößmann, W, Nathrath, M, Hecker-Nolting, S, Frühwald, MC, Schneider, DT, Brecht, IB, Ketteler, P, Fulda, S, Koscielniak, E, Meister, MT, Scheer, M, Hettmer, S, Schwab, M, Tremmel, R, Øra, I, Hutter, C, Gerber, NU, Lohi, O, Kazanowska, B, Kattamis, A, Filippidou, M, Goemans, B, Zwaan, CM, Milde, T, Jäger, N, Wolf, S, Reuss, D, Sahm, F, Von Deimling, A, Dirksen, U, Freitag, A, Witt, R, Lichter, P, Kopp-Schneider, A, Jones, DTW, Molenaar, JJ, Capper, D, Pfister, SM & Witt, O 2021, 'The pediatric precision oncology inform registry: Clinical outcome and benefit for patients with very high-evidence targets', CANCER DISCOVERY, vol. 11, no. 11, pp. 2764-2779. https://doi.org/10.1158/2159-8290.CD-21-0094

TY - JOUR

T1 - The pediatric precision oncology inform registry

T2 - Clinical outcome and benefit for patients with very high-evidence targets

AU - Van Tilburg, Cornelis M.

AU - Pfaff, Elke

AU - Pajtler, Kristian W.

AU - Langenberg, Karin P.S.

AU - Fiesel, Petra

AU - Jones, Barbara C.

AU - Balasubramanian, Gnana Prakash

AU - Stark, Sebastian

AU - Johann, Pascal D.

AU - Blattner-Johnson, Mirjam

AU - Schramm, Kathrin

AU - Dikow, Nicola

AU - Hirsch, Steffen

AU - Sutter, Christian

AU - Grund, Kerstin

AU - Von Stackelberg, Arend

AU - Kulozik, Andreas E.

AU - Lissat, Andrej

AU - Borkhardt, Arndt

AU - Meisel, Roland

AU - Reinhardt, Dirk

AU - Klusmann, Jan Henning

AU - Fleischhack, Gudrun

AU - Tippelt, Stephan

AU - Von Schweinitz, Dietrich

AU - Schmid, Irene

AU - Kramm, Christof M.

AU - Von Bueren, André O.

AU - Calaminus, Gabriele

AU - Vorwerk, Peter

AU - Graf, Norbert

AU - Westermann, Frank

AU - Fischer, Matthias

AU - Eggert, Angelika

AU - Burkhardt, Birgit

AU - Wößmann, Wilhelm

AU - Nathrath, Michaela

AU - Hecker-Nolting, Stefanie

AU - Frühwald, Michael C.

AU - Schneider, Dominik T.

AU - Brecht, Ines B.

AU - Ketteler, Petra

AU - Fulda, Simone

AU - Koscielniak, Ewa

AU - Meister, Michael T.

AU - Scheer, Monika

AU - Hettmer, Simone

AU - Schwab, Matthias

AU - Tremmel, Roman

AU - Øra, Ingrid

AU - Hutter, Caroline

AU - Gerber, Nicolas U.

AU - Lohi, Olli

AU - Kazanowska, Bernarda

AU - Kattamis, Antonis

AU - Filippidou, Maria

AU - Goemans, Bianca

AU - Zwaan, C. Michel

AU - Milde, Till

AU - Jäger, Natalie

AU - Wolf, Stephan

AU - Reuss, David

AU - Sahm, Felix

AU - Von Deimling, Andreas

AU - Dirksen, Uta

AU - Freitag, Angelika

AU - Witt, Ruth

AU - Lichter, Peter

AU - Kopp-Schneider, Annette

AU - Jones, David T.W.

AU - Molenaar, Jan J.

AU - Capper, David

AU - Pfister, Stefan M.

AU - Witt, Olaf

PY - 2021

Y1 - 2021

N2 - 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.

AB - 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.

U2 - 10.1158/2159-8290.CD-21-0094

DO - 10.1158/2159-8290.CD-21-0094

M3 - Article

C2 - 34373263

AN - SCOPUS:85113553839

SN - 2159-8274

VL - 11

SP - 2764

EP - 2779

JO - CANCER DISCOVERY

JF - CANCER DISCOVERY

IS - 11

ER -

The pediatric precision oncology inform registry: Clinical outcome and benefit for patients with very high-evidence targets

Abstract

Publication forum classification

ASJC Scopus subject areas

UN SDGs

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