1. The Pediatric Precision Oncology INFORM Registry: Clinical Outcome and Benefit for Patients with Very High-Evidence Targets
- Author
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Steffen Hirsch, Caroline Hutter, Bianca Goemans, Kristian W. Pajtler, David Reuss, Gabriele Calaminus, Gnana Prakash Balasubramanian, Nicola Dikow, C. Michel Zwaan, Arndt Borkhardt, David T.W. Jones, Birgit Burkhardt, Matthias Schwab, Ingrid Øra, Ines B. Brecht, Uta Dirksen, Christian Sutter, Kathrin Schramm, Roman Tremmel, Bernarda Kazanowska, Peter Lichter, M. Scheer, Gudrun Fleischhack, André O. von Bueren, Mirjam Blattner-Johnson, David Capper, Felix Sahm, Simone Fulda, Natalie Jäger, Nicolas U. Gerber, Olaf Witt, Jan-Henning Klusmann, Irene Schmid, Petra Fiesel, Matthias Fischer, Roland Meisel, Stefan M. Pfister, Michaela Nathrath, Cornelis M. van Tilburg, Angelika Eggert, Sebastian Stark, Christof M. Kramm, Elke Pfaff, Kerstin Grund, Arend von Stackelberg, Andrej Lissat, Peter Vorwerk, Petra Ketteler, Angelika Freitag, Olli Lohi, Michael T. Meister, Ruth Witt, Norbert Graf, Annette Kopp-Schneider, Pascal D. Johann, Dominik T. Schneider, Karin P.S. Langenberg, Simone Hettmer, Dirk Reinhardt, Antonis Kattamis, Andreas E. Kulozik, Andreas von Deimling, Jan J. Molenaar, Wilhelm Wößmann, Maria Filippidou, Stephan Tippelt, Frank Westermann, Stephan Wolf, Michael C. Frühwald, Barbara C. Jones, Ewa Koscielniak, Till Milde, Stefanie Hecker-Nolting, Dietrich von Schweinitz, Pediatrics, Tampere University, Department of Paediatrics, and BioMediTech
- Subjects
Prioritization ,medicine.medical_specialty ,Treatment response ,3122 Cancers ,Medizin ,MEDLINE ,03 medical and health sciences ,0302 clinical medicine ,SDG 3 - Good Health and Well-being ,3123 Gynaecology and paediatrics ,Internal medicine ,Neoplasms ,medicine ,Humans ,Prospective Studies ,Registries ,Medical diagnosis ,Precision Medicine ,Child ,030304 developmental biology ,0303 health sciences ,ddc:618 ,business.industry ,Cancer ,medicine.disease ,Confidence interval ,Progression-Free Survival ,ddc ,3. Good health ,Oncology ,Precision oncology ,030220 oncology & carcinogenesis ,Molecular targets ,3111 Biomedicine ,business - 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. See related commentary by Eggermont et al., p. 2677 . This article is highlighted in the In This Issue feature, p. 2659
- Published
- 2021