The pediatric precision oncology INFORM registry : clinical outcome and benefit for patients with very high-evidence targets
(2021) In Cancer Discovery 11(11). p.2764-2779- Abstract
INFORM is a prospective, multi-national registry gathering clinical and molecular data of relapsed, progressive or high-risk pediatric cancer patients. This report describes long-term follow-up of 519 patients in whom molecular alterations were evaluated according to a pre-defined 7-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, twenty patients received matched targeted treatment with a median PFS of 204 days (95% CI 99 - N.A.), 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... (More)
INFORM is a prospective, multi-national registry gathering clinical and molecular data of relapsed, progressive or high-risk pediatric cancer patients. This report describes long-term follow-up of 519 patients in whom molecular alterations were evaluated according to a pre-defined 7-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, twenty patients received matched targeted treatment with a median PFS of 204 days (95% CI 99 - N.A.), 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.
(Less)
- author
- publishing date
- 2021-08-09
- type
- Contribution to journal
- publication status
- published
- subject
- in
- Cancer Discovery
- volume
- 11
- issue
- 11
- article number
- 0094
- pages
- 2764 - 2779
- publisher
- American Association for Cancer Research
- external identifiers
-
- scopus:85113553839
- pmid:34373263
- ISSN
- 2159-8274
- DOI
- 10.1158/2159-8290.CD-21-0094
- language
- English
- LU publication?
- no
- id
- d3a87ed1-f83e-46ea-b347-a8a3458cd22a
- date added to LUP
- 2021-09-09 12:31:02
- date last changed
- 2024-04-20 12:03:45
@article{d3a87ed1-f83e-46ea-b347-a8a3458cd22a,
abstract = {{<p>INFORM is a prospective, multi-national registry gathering clinical and molecular data of relapsed, progressive or high-risk pediatric cancer patients. This report describes long-term follow-up of 519 patients in whom molecular alterations were evaluated according to a pre-defined 7-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, twenty patients received matched targeted treatment with a median PFS of 204 days (95% CI 99 - N.A.), 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.</p>}},
author = {{van Tilburg, Cornelis M and Pfaff, Elke and Pajtler, Kristian W and Langenberg, Karin P S and Fiesel, Petra and Jones, Barbara C and Balasubramanian, Gnana Prakash and Stark, Sebastian and Johann, Pascal D and Blattner-Johnson, Mirjam and Schramm, Kathrin and Dikow, Nicola and Hirsch, Steffen and Sutter, Christian and Grund, Kerstin and von Stackelberg, Arend and Kulozik, Andreas E and Lissat, Andrej and Borkhardt, Arndt and Meisel, Roland and Reinhardt, Dirk and Klusmann, Jan-Henning and Fleischhack, Gudrun and Tippelt, Stephan and von Schweinitz, Dietrich and Schmid, Irene and Kramm, Christof M and von Bueren, Andre O and Calaminus, Gabriele and Vorwerk, Peter and Graf, Norbert and Westermann, Frank and Fischer, Matthias and Eggert, Angelika and Burkhardt, Birgit and Wossmann, Wilhelm and Nathrath, Michaela and Hecker-Nolting, Stefanie and Fruhwald, Michael C and Schneider, Dominik T and Brecht, Ines B and Ketteler, Petra and Fulda, Simone and Koscielniak, Ewa and Meister, Michael T and Scheer, Monika and Hettmer, Simone and Schwab, Matthias and Tremmel, Roman and Ora, Ingrid and Hutter, Caroline and Gerber, Nicolas U and Lohi, Olli and Kazanowska, Bernarda and Kattamis, Antonis and Filippidou, Maria and Goemans, Bianca and Zwaan, C Michel and Milde, Till and Jager, Natalie and Wolf, Stephan and Reuss, David and Sahm, Felix and von Deimling, Andreas and Dirksen, Uta and Freitag, Angelika and Witt, Ruth and Lichter, Peter and Kopp-Schneider, Annette and Jones, David T W and Molenaar, Jan J and Capper, David and Pfister, Stefan M and Witt, Olaf}},
issn = {{2159-8274}},
language = {{eng}},
month = {{08}},
number = {{11}},
pages = {{2764--2779}},
publisher = {{American Association for Cancer Research}},
series = {{Cancer Discovery}},
title = {{The pediatric precision oncology INFORM registry : clinical outcome and benefit for patients with very high-evidence targets}},
url = {{http://dx.doi.org/10.1158/2159-8290.CD-21-0094}},
doi = {{10.1158/2159-8290.CD-21-0094}},
volume = {{11}},
year = {{2021}},
}