American Association for Cancer Research
Browse

sorry, we can't preview this file

cd-21-0094_table_s1_suppst1.xlsx (11.97 kB)

Supplementary Table from The Pediatric Precision Oncology INFORM Registry: Clinical Outcome and Benefit for Patients with Very High-Evidence Targets

Download (11.97 kB)
dataset
posted on 2023-04-03, 23:41 authored by 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
Supplementary Table from The Pediatric Precision Oncology INFORM Registry: Clinical Outcome and Benefit for Patients with Very High-Evidence Targets

Funding

German Cancer Aid

German Childhood Oncology Foundation

German Federal Ministry of Health

German Federal Ministry of Education and Research

German Cancer Research Center

History

ARTICLE 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. 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.See related commentary by Eggermont et al., p. 2677.This article is highlighted in the In This Issue feature, p. 2659

Usage metrics

    Cancer Discovery

    Categories

    Keywords

    Licence

    Exports

    RefWorks
    BibTeX
    Ref. manager
    Endnote
    DataCite
    NLM
    DC