| Literature DB >> 34373263 |
Cornelis M van Tilburg1,2,3,4,5, Elke Pfaff6,3,4,5,7, Kristian W Pajtler6,3,4,5,8, Karin P S Langenberg9, Jan J Molenaar9,10, David Capper4,5,11, Stefan M Pfister6,3,4,5,8, Olaf Witt6,2,3,4,5, Petra Fiesel4,5,12,13, Barbara C Jones6,3,4,5,7, Gnana Prakash Balasubramanian6,4,5,8, Sebastian Stark6,3,4,5,7, Pascal D Johann6,3,4,5,8,14, Mirjam Blattner-Johnson6,4,5,7, Kathrin Schramm6,4,5,7, Nicola Dikow15, Steffen Hirsch6,15, Christian Sutter15, Kerstin Grund15, Arend von Stackelberg4,5,16, Andreas E Kulozik6,3,4,5,17, Andrej Lissat4,5,16, Arndt Borkhardt4,18, Roland Meisel4,19, Dirk Reinhardt4,5,20, Jan-Henning Klusmann21, Gudrun Fleischhack4,5,20, Stephan Tippelt4,5,20, Dietrich von Schweinitz4,22, Irene Schmid23, Christof M Kramm24, André O von Bueren25, Gabriele Calaminus26, Peter Vorwerk27, Norbert Graf28, Frank Westermann4,5,29, Matthias Fischer5,30, Angelika Eggert4,5,16, Birgit Burkhardt31, Wilhelm Wößmann32, Michaela Nathrath4,33,34, Stefanie Hecker-Nolting5,35, Michael C Frühwald5,14, Dominik T Schneider36, Ines B Brecht4,5,37, Petra Ketteler4,5,20, Simone Fulda4,38, Ewa Koscielniak5,35, Michael T Meister4,9, Monika Scheer4,5,16, Simone Hettmer4,39, Matthias Schwab4,40,41, Roman Tremmel40, Ingrid Øra42, Caroline Hutter43, Nicolas U Gerber44, Olli Lohi45, Bernarda Kazanowska46, Antonis Kattamis47, Maria Filippidou6,8,47, Bianca Goemans9, C Michel Zwaan9,48, Till Milde6,2,3,4,5, Natalie Jäger6,4,5,8, Stephan Wolf4,5,49, David Reuss4,5,12,13, Felix Sahm4,5,12,13, Andreas von Deimling4,5,12,13, Uta Dirksen4,5,20, Angelika Freitag50, Ruth Witt6,2,4,5, Peter Lichter4,5,51, Annette Kopp-Schneider4,5,52, David T W Jones6,4,5,7.
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. ©2021 The Authors; Published by the American Association for Cancer Research.Entities:
Mesh：
Year: 2021 PMID： 34373263 DOI： 10.1158/2159-8290.CD-21-0094
Source DB: PubMed Journal: Cancer Discov ISSN： 2159-8274 Impact factor: 39.397