Zurück zur Übersicht
| Cancer discovery. 2021 Aug 9. pii: 2159-8290.CD-21-0094. doi: 10.1158/2159-8290.CD-21-0094 |
| The Pediatric Precision Oncology INFORM Registry: Clinical Outcome and Benefit for Patients with Very High-Evidence Targets. |
| van Tilburg CM1, Pfaff E2, Pajtler KW3, Langenberg KPS4, Fiesel P5, Jones BC6, Balasubramanian GP7, Stark S8, Johann PD9, Blattner-Johnson M10, Schramm K11, Dikow N12, Hirsch S13, Sutter C14, Grund K15, von Stackelberg A16, Kulozik AE17, Lissat A18, Borkhardt A19, Meisel R20, Reinhardt D21, Klusmann JH22, Fleischhack G23, Tippelt S24, von Schweinitz D25, Schmid I26, Kramm CM27, von Bueren AO28, Calaminus G29, Vorwerk P30, Graf N31, Westermann F32, Fischer M33, Eggert A34, Burkhardt B35, Wößmann W36, Nathrath M37, Hecker-Nolting S38, Frühwald MC39, Schneider DT40, Brecht IB41, Ketteler P42, Fulda S43, Koscielniak E44, Meister MT45, Scheer M46, Hettmer S47, Schwab M48, Tremmel R49, Øra I50, Hutter C51, Gerber NU52, Lohi O53, Kazanowska B54, Kattamis A55, Filippidou M56, Goemans B57, Zwaan CM58, Milde T59, Jäger N60, Wolf S61, Reuss D62, Sahm F63, von Deimling A64, Dirksen U65, Freitag A66, Witt R67, Lichter P68, Kopp-Schneider A69, Jones DTW70, Molenaar JJ71, Capper D72, Pfister SM73, Witt O74 |
 Author information
|
|
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; = 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. |
|
©2021 The Authors; Published by the American Association for Cancer Research. |
|
Publikations ID: 34373263 Quelle: öffnen |
Author information
Schnellinfo
-- Cancer Care
-- Kliniken und Partner
-- CCC Cancer School
-- Young CCC
-- CCC Tumorboards
-- CCC Forschungscluster
-- CCC Units
-- CCC Platforms
-- SOPs / Leitlinien
-- Kontakt
Featured
