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Retrospective Comparison of Targeted Anticancer Drugs Predicted by the CNS-TAP Tool Versus Those Selected by a Molecularly Driven Tumor Board in Children With DIPG

Authors :
Roberts, Holly J.
Ravi, Karthik
Marini, Bernard L.
Schepers, Allison
Kline, Cassie
Kilburn, Lindsay
Prados, Michael
Byron, Sara A.
Sturza, Julie
Mueller, Sabine
Koschmann, Carl
Franson, Andrea T.
Source :
Journal of Pediatric Hematology / Oncology; January 2025, Vol. 47 Issue: 1 p19-30, 12p
Publication Year :
2025

Abstract

The recent trial Pediatric Neuro-Oncology Consortium 003 (PNOC003) utilized a molecular tumor board to recommend personalized treatment regimens based on tumor sequencing results in children with DIPG. We separately developed the Central Nervous System Targeted Agent Prediction (CNS-TAP) tool, which numerically scores targeted anticancer agents using preclinical, clinical, and patient-specific data. We hypothesized that highly scored agents from CNS-TAP would overlap with the PNOC003 tumor board’s recommendations. For each of the 28 participants, actionable genetic alterations were derived from PNOC003 genomic reports and input to CNS-TAP to identify the highest scoring agents. These agents were then compared with PNOC003 recommendations, with a resultant concordance percentage calculated. Overall, 38% of the total agents recommended by the tumor board were also selected by CNS-TAP, with higher concordance (63%) in a subanalysis including only targeted anticancer agents. Furthermore, nearly all patients (93%) had at least 1 drug chosen by both methods. We demonstrate overlap between agents recommended by CNS-TAP and PNOC003 tumor board, though this does not appear to improve survival. We do observe some discordance, highlighting strengths and limitations of each method. We propose that a combination of expert opinion and data-driven tools may improve targeted treatment recommendations for children with DIPG.

Details

Language :
English
ISSN :
10774114 and 15363678
Volume :
47
Issue :
1
Database :
Supplemental Index
Journal :
Journal of Pediatric Hematology / Oncology
Publication Type :
Periodical
Accession number :
ejs68456745
Full Text :
https://doi.org/10.1097/MPH.0000000000002964