Childhood Cancer Data Initiative Annual Symposium (Abstract Registration): Submission #47
Submission information
Submission Number: 47
Submission ID: 150060
Submission UUID: eb3be377-e947-4a52-af58-05e13916be10
Submission URI: /nci/ccdisymposium/abstract
Created: Wed, 08/27/2025 - 23:25
Completed: Wed, 08/27/2025 - 23:42
Changed: Wed, 08/27/2025 - 23:42
Remote IP address: 10.208.28.150
Submitted by: Anonymous
Language: English
Is draft: No
Abstract Submission for Poster Presentation
Real-World Experience of Pediatric Precision Oncology Cohort At a Single Institution: An Interim Analysis
Our primary aim is to study the real-world impact of integrative clinical sequencing (ICS) based pediatric precision oncology program on the outcomes of pediatric-AYA cancer patients at a single institution.
Methods: Pediatric-AYA oncology patients (age 0-25yr) were enrolled and sequenced using methodology described previously.1 Longitudinal clinical data extracted from EHR using NCI supported tool EMERSE were entered into a REDCap.
Results: Clinical characteristics are shown in Fig-1. Of 1000 patients enrolled, 925 (92.5%) were sequenced successfully, 235 (23.5%) underwent repeat ICS (Range 1-4). Interim analysis of the first 250 patients revealed 230 patients with full sequencing results. Of those, 133 (57.8%) patients had total 228 actionable alterations (AA) (mean1.71 AA /pt), of which 30 (13%) patients had germline AA. Of the total AA, 64 patients had 84 SNV (Single Nucleotide Variants), 33 patients had 38 actionable fusions, and 46 had 76 actionable CNA’s (Copy Number Alterations). Fifty-one (22.2%) patients received a total of 96 targeted therapies (TT) supported by variant level evidence (VLE)2, 3 (L1=39, L2=5, L3=4, L4=4, Other=44), while 16 (7.0%) patients received 26 TT without AA based on clinician preference. Eighty-two (35.6%) patients with AA did not receive any TT. Sixty-five (28.3%) patients underwent repeat ICS, among which 9.2% (6) had additional AA identified and 4.6% (3) received TT based on repeat ICS.
Conclusion: Interim analyses showed that 67 patients (29.1%) received TT with variable level of evidence. Outcomes of patients receiving TT including, EFS, OS and barrier in receiving TT despite having AA, are under further analyses.
Methods: Pediatric-AYA oncology patients (age 0-25yr) were enrolled and sequenced using methodology described previously.1 Longitudinal clinical data extracted from EHR using NCI supported tool EMERSE were entered into a REDCap.
Results: Clinical characteristics are shown in Fig-1. Of 1000 patients enrolled, 925 (92.5%) were sequenced successfully, 235 (23.5%) underwent repeat ICS (Range 1-4). Interim analysis of the first 250 patients revealed 230 patients with full sequencing results. Of those, 133 (57.8%) patients had total 228 actionable alterations (AA) (mean1.71 AA /pt), of which 30 (13%) patients had germline AA. Of the total AA, 64 patients had 84 SNV (Single Nucleotide Variants), 33 patients had 38 actionable fusions, and 46 had 76 actionable CNA’s (Copy Number Alterations). Fifty-one (22.2%) patients received a total of 96 targeted therapies (TT) supported by variant level evidence (VLE)2, 3 (L1=39, L2=5, L3=4, L4=4, Other=44), while 16 (7.0%) patients received 26 TT without AA based on clinician preference. Eighty-two (35.6%) patients with AA did not receive any TT. Sixty-five (28.3%) patients underwent repeat ICS, among which 9.2% (6) had additional AA identified and 4.6% (3) received TT based on repeat ICS.
Conclusion: Interim analyses showed that 67 patients (29.1%) received TT with variable level of evidence. Outcomes of patients receiving TT including, EFS, OS and barrier in receiving TT despite having AA, are under further analyses.
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