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

Research output: Contribution to journalArticlepeer-review


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

Original languageEnglish
Pages (from-to)2764-2779
Number of pages16
JournalCancer discovery
Issue number11
Publication statusPublished - Nov 2021


  • Child
  • Humans
  • Neoplasms/diagnosis
  • Precision Medicine
  • Progression-Free Survival
  • Prospective Studies
  • Registries


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