Data from The Pediatric Precision Oncology INFORM Registry: Clinical Outcome and Benefit for Patients with Very High-Evidence Targets
Authors: 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 , Fleischhack Gudrun , Tippelt Stephan , von Schweinitz Dietrich , Schmid Irene , Kramm Christof M. , von Bueren André O. , Calaminus Gabriele , Vorwerk Peter , Graf Norbert , Westermann Frank , Fischer Matthias , Eggert Angelika , Burkhardt Birgit , Wößmann Wilhelm , Nathrath Michaela , Hecker-Nolting Stefanie , Frühwald Michael C. , Schneider Dominik T. , Brecht Ines B. , Ketteler Petra , Fulda Simone , Koscielniak Ewa , Meister Michael T. , Scheer Monika , Hettmer Simone , Schwab Matthias , Tremmel Roman , Øra Ingrid , Hutter Caroline , Gerber Nicolas U. , Lohi Olli , Kazanowska Bernarda , Kattamis Antonis , Filippidou Maria , Goemans Bianca , Zwaan C. Michel , Milde Till , Jäger Natalie , Wolf Stephan , Reuss David , Sahm Felix , von Deimling Andreas , Dirksen Uta , Freitag Angelika , Witt Ruth , Lichter Peter , Kopp-Schneider Annette , Jones David T.W. , Molenaar Jan J. , Capper David , Pfister Stefan M. , Witt Olaf 
Published: April 2023

<div>Abstract<p>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; <i>P</i> = 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.</p>Significance:<p>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.</p><p><i><a href="https://aacrjournals.org/cancerdiscovery/article/doi/10.1158/2159-8290.CD-21-1069" target="_blank">See related commentary by Eggermont et al., p. 2677</a></i>.</p><p><i><a href="https://aacrjournals.org/cancerdiscovery/article/doi/10.1158/2159-8290.CD-11-11-ITI" target="_blank">This article is highlighted in the In This Issue feature, p. 2659</a></i></p></div>