Childhood Cancer Data Initiative Annual Symposium (Abstract Registration)
54 submissions
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| 87 | Star/flag Childhood Cancer Data Initiative Annual Symposium (Abstract Registration): Submission #87 | Lock Childhood Cancer Data Initiative Annual Symposium (Abstract Registration): Submission #87 | Add notes to Childhood Cancer Data Initiative Annual Symposium (Abstract Registration): Submission #87 | Fri, 09/04/2026 - 16:53 | Anonymous | 10.208.24.244 | Neuroblastoma Patient-Derived Xenografts and Cell Lines from Postmortem Blood as Models to Understand and Reverse Therapy Resistance | Background: Patient-derived models of neuroblastoma that recapitulate therapy resistance in patients are essential for defining resistance mechanisms. We established and characterized patient derived xenografts (PDXs) and patient derived cell lines (PDCLs) from neuroblastoma patients with progressive disease established post-mortem (PD-PM). Methods: Tumor and blood samples were cultured and/or xenografted in NOD SCIDγ mice. PDCLs and PDXs were validated by STR profiling. Mutations were identified by whole-exome sequencing and telomere maintenance mechanisms by TERT qPCR, C-circle assay, and TERT break-apart FISH. Therapeutic responses were evaluated in subcutaneous xenografts. Results: PD-PM specimens showed higher engraftment rates as xenografts (68%; 21/31 specimens, 83% for PD-PM blood specimens) and had higher take rates as PDCLs (54%, 25/46 specimens) compared to diagnosis (Dx, 17%) or progressive disease (PD, 11%, P<0.001) specimens. PD-PM PDXs had higher mutation burdens than Dx PDXs (P=0.026); 33% harbored activating ALK mutations and 33% mutations in other RAS-MAPK genes. Of 20 high TERT-expressing PD-PM PDXs, 13 had MYCN amplification and 4 MYCN-non-amplified PDXs had TERT rearrangements. One PD-PM PDX was ALT-positive. Temozolomide + irinotecan responses were shorter in 3 PD-PM than Dx and PD PDXs (P<0.001). In a PD-PM PDX, O6-BG reversed temozolomide + irinotecan resistance (P=0.002), while nanoliposomal irinotecan extended event-free survival compared to irinotecan (P=0.003). Conclusions: PD-PM PDXs activate ALK or RAS-MAPK signaling, manifest high levels of chemoresistance, and provide models to study reversing neuroblastoma drug resistance. The Children’s Oncology Group (COG) panel of PDXs is freely available from the Alex's Lemonade Stand Foundation (ALSF)/COG Childhood Cancer Repository (https://cccells.org). |
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BALAKRISHNA KONERU | Texas Tech University Health Sciences Center | balakrishna.koneru@ttuhsc.edu | |
| 85 | Star/flag Childhood Cancer Data Initiative Annual Symposium (Abstract Registration): Submission #85 | Lock Childhood Cancer Data Initiative Annual Symposium (Abstract Registration): Submission #85 | Add notes to Childhood Cancer Data Initiative Annual Symposium (Abstract Registration): Submission #85 | Fri, 09/04/2026 - 16:31 | Anonymous | 10.208.28.41 | The Children’s Oncology Group and Alex’s Lemonade Stand Foundation Childhood Cancer Repository | The Children’s Oncology Group (COG) childhood cancer repository (www.CCcells.org), supported by ALSF, establishes, validates, and banks patient-derived cell lines (PDCLs) and patient-derived xenografts (PDXs) from childhood cancers. Viable tumor, blood, or bone marrow samples obtained under informed consent via COG protocols are sent to a centralized resource lab to establish PDCLs and PDXs. PDCLs/PDXs are validated by short tandem repeat assay to patient material, verified free of human and mouse pathogens, tumor type validated by biomarkers, and telomere maintenance mechanism assessed. The repository has available PDCLs/PDXs from 578/87 neuroblastomas, 16/20 leukemias, 7/5 lymphomas, 20/3 Ewing sarcomas, 13/3 soft tissue sarcomas. 32/1 retinoblastomas, 1/ 5 osteosarcomas, 2/5 Wilm’s tumors, and 11 PDCLs from brain tumors. Hypoxic culture conditions are used to establish PDCLs, enhancing success rates and minimizing selection against cells by hyperoxia. Cultures in hypoxia are more often able to generate a PDCL and low-passage PDCLs in hypoxia generate xenografts comparable in RNA expression and drug response profiles to PDXs. Neuroblastoma PDCLs and PDXs are being established from patients enrolled on COG phase III studies at diagnosis and from disease persisting or progressing during and after therapy to enable future studies comparing genomics and drug response of PDCLs and PDXs to patient data. To date 56 PDCLs and 19 PDXs have been established from patients on the ANBL1531 phase III trial and 41 PDCLs and 4 PDXs from 41 patients on the ANBL2131 phase III study. The repository distributes PDCLs and PDXs to > 700 laboratories in 30 countries. |
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Min H. Kang, PharmD | Texas Tech University Health Sciences Center School of Medicine Lubbock, TX | Min.Kang@ttuhsc.edu | |
| 84 | Star/flag Childhood Cancer Data Initiative Annual Symposium (Abstract Registration): Submission #84 | Lock Childhood Cancer Data Initiative Annual Symposium (Abstract Registration): Submission #84 | Add notes to Childhood Cancer Data Initiative Annual Symposium (Abstract Registration): Submission #84 | Fri, 09/04/2026 - 15:54 | Anonymous | 10.208.24.244 | Nanopore Sequencing for Pediatric CNS Tumor Classification | Central nervous system (CNS) tumors are the most common solid tumors diagnosed in children, making up about 17% of diagnosed childhood cancer cases. Most pediatric CNS tumor cases require a combination of imaging, histopathological, and molecular diagnoses to determine the tumor (sub)type. My project utilizes an emerging molecular diagnostic technology -- nanopore sequencing -- to quickly and accurately generate high-depth whole genome DNA methylation profiles for a diverse cohort of 150 pediatric CNS tumors, and I am currently using this data to train and validate a pediatric-specific deep learning classifier. As part of a clinical research collaboration, I tested the previously-developed machine learning classifier Sturgeon on high-depth nanopore DNA methylation data for 7 matched pairs of standard patient tissue and surgical tumor aspirate (n=14). For each sample, I demonstrated between 7-34x genomic coverage, classified the sample’s tumor (sub)type, and used in-house software to validate key subtype-defining features and identify tumor-specific copy number variation(s). Through this initial analysis, I was able to identify key shortcomings in existing classifier(s), including inappropriate labeling for pediatric samples, insufficient adjustment for low tumor purity samples, and confident misclassification of rare/unique tumors. After making adjustments to existing preprocessing methods, all 14 samples were then classified correctly with high confidence. Matched samples also showed strong pairwise similarities, reflective of interchangeable use of the materials. Together, I used nanopore DNA methylation profiling to demonstrate fast and accurate classification of CNS tumor aspirate and to highlight areas of improvement for the development of a pediatric-specific CNS tumor classifier. |
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Allison A Murray | University of North Carolina Chapel Hill | amurray1@unc.edu | |
| 86 | Star/flag Childhood Cancer Data Initiative Annual Symposium (Abstract Registration): Submission #86 | Lock Childhood Cancer Data Initiative Annual Symposium (Abstract Registration): Submission #86 | Add notes to Childhood Cancer Data Initiative Annual Symposium (Abstract Registration): Submission #86 | Fri, 09/04/2026 - 15:45 | Anonymous | 10.208.24.244 | The Integrated Oncogenomics Portal (IOCP): A Cloud-Native Pipeline and Database for Processing and Sharing CCDI & other Childhood Cancer Genomics Data | NCI’s Childhood Cancer Data Initiative(CCDI) is assembling high-value pediatric cancer datasets, creating a critical need for scalable, reproducible infrastructure to transform them into harmonized, interpretable, and reusable results. The Integrated Oncogenomics Portal(IOCP) addresses this need through a cloud-native analysis pipeline and data portal that identify clinically and biologically relevant genomic alterations and enable interactive exploration at the patient, cohort, and cancer-type levels. The first component is a nextflow pipeline, with parallel hg19/hg38 builds, covering RNA and DNA analysis: somatic-germline variant calling, copy-number, fusions, TMB, MSI, mutational signatures, HLA/neoantigen prediction. It is version-controlled and portable across cloud platforms; genomic datasets can be read directly from their source location without the need to transfer. We are currently processing the PediatricMATCH cohort(phs002883), followed by BeatAML(phs002599), MCI(phs002790), and Oncokids(phs002518). The second component disseminates results through cloud oncogenomics database enabling case and cohort-level exploration, interactive-IGV, survival-analyses, and AVIA annotation integrating genomic, population, functional, cancer-specific, and clinical evidence for variant interpretation and prioritization. Pipeline results are also published to CCDI cBioPortal Cancer Data Explore for standardized OncoPrint visualization and cross-cohort queries. By integrating CCDI cohorts with published pediatric cancer genomic datasets, IOCP combines analytical depth with broad interoperability. IOCP provides a reproducible framework for advancing CCDI’s goal of interoperable pediatric cancer research through harmonized and discoverable genomic results. Beyond CCDI cohorts, the database integrates published pediatric cancer genomics data and offers an LLM-enabled conversational interface letting users query in natural language and receive responses grounded in IOCP data. |
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Vineela Gangalapudi | ABCS, FNLCR, NCI Genetics Branch | vineela.gangalapudi@nih.gov | |
| 83 | Star/flag Childhood Cancer Data Initiative Annual Symposium (Abstract Registration): Submission #83 | Lock Childhood Cancer Data Initiative Annual Symposium (Abstract Registration): Submission #83 | Add notes to Childhood Cancer Data Initiative Annual Symposium (Abstract Registration): Submission #83 | Thu, 09/03/2026 - 19:43 | Anonymous | 10.208.24.244 | Single-Cell Multiome Sequencing Reveals Distinct Transcriptional Programs Associated with ecDNA Amplifications in Medulloblastoma | Extrachromosomal DNA (ecDNA) is a key driver of intratumoral heterogeneity and has been linked to therapeutic resistance and poor patient outcomes. However, technical limitations in identifying, isolating, and analyzing ecDNA at the single-cell level have hindered our understanding of its role in tumor development and treatment response. We have previously developed a single-cell multiome (RNA + ATAC) sequencing approach that enables the simultaneous analyses of ecDNA and its gene expression profiles. Initial application of this method in a medulloblastoma tumor of the sonic hedgehog subgroup revealed distinct transcriptional signatures in ecDNA-positive compared to ecDNA-negative and non-malignant cells. Building on these findings, we performed single-cell multiome sequencing on an additional cohort of medulloblastoma specimens harboring ecDNA amplifications, obtained from Rady Children's Hospital and the Children's Brain Tumor Network (CBTN). Initial analyses revealed distinct functional characteristics between ecDNA-positive and ecDNA-negative tumor cells. For example, ecDNA-positive tumor cells show up-regulation of DNA replication, recombination, and damage repair pathways, while ecDNA-negative cells demonstrated increased expression of synaptic activity, cell signaling, and morphogenesis pathways. Our ongoing analysis focuses on further defining the transcriptional programs that distinguish ecDNA-positive from ecDNA-negative tumor cells, ultimately informing future functional genetic and pharmacological studies targeting transcriptional dependencies in ecDNA-driven medulloblastoma tumors. |
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Hui Hui | Sanford Burnham Prebys Medical Discovery Institute | ahui@sbpdiscovery.org | |
| 82 | Star/flag Childhood Cancer Data Initiative Annual Symposium (Abstract Registration): Submission #82 | Lock Childhood Cancer Data Initiative Annual Symposium (Abstract Registration): Submission #82 | Add notes to Childhood Cancer Data Initiative Annual Symposium (Abstract Registration): Submission #82 | Thu, 09/03/2026 - 13:15 | Anonymous | 10.208.24.244 | Real-World Molecularly Targeted Treatment Registry (MaTTeR): a Pilot Study to Enrich CCDI Data Utilizing Directed EMR Extraction | The incorporation of genomic profiling into the care of pediatric, adolescent and young adult (AYA) cancer patients has resulted in identification of targetable alterations and use of molecularly targeted therapy (MTT), necessitating collection and reporting of outcomes of real-world use of MTT. Using genomic data contributed to the CCDI and our institutional cohorts, we identified a cohort of patients who received MTT outside of clinical trials. We included patients with cancer seen at the Dana-Farber/Boston Children’s, who enrolled in a clinical sequencing/banking study and had next-generation sequencing (NGS) performed (OncoPanel, Rapid Heme and Fusion Panels). Sequencing results were used to identify patients with potentially targetable alterations in a list of 66 genes. Patients who received MTT were identified, and data including dosing, toxicity and response were extracted from the medical record. Between 2013 and 2024, 2163 patients had tumor profiling via targeted NGS. 35% (762/2163) had an actionable alteration detected for which MTT could have been administered. 112 patients received at least one regimen that included an MTT (70 patients with brain tumors, 28 with solid tumors, 14 with hematologic malignancies). Alterations in BRAF, NF1, ALK, FLT3, and PIK3CA led to the most MTT use. Of the 112 patients who received MTT, 81% (91/112) received at least one targeted therapy regimen outside of a clinical trial. This project will create a registry of MTT use within the CCDI that can be expanded to include additional patients and be a resource for MTT use for the pediatric and AYA cancer community. |
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Katherine A. Janeway | Dana-Farber/Boston Children's Cancer and Blood Disorder Center | Suzanne_forrest@dfci.harvard.edu | |
| 81 | Star/flag Childhood Cancer Data Initiative Annual Symposium (Abstract Registration): Submission #81 | Lock Childhood Cancer Data Initiative Annual Symposium (Abstract Registration): Submission #81 | Add notes to Childhood Cancer Data Initiative Annual Symposium (Abstract Registration): Submission #81 | Thu, 09/03/2026 - 11:42 | Anonymous | 10.208.28.41 | AI-driven multimodal analysis integrating WSI, Methyl-Seq, and OncoKids Cancer Panel to improve diagnostic precision of pediatric tumors. | Previously, we added clinical and genomic data from more than 1,000 pediatric cancer patients from our racially and ethnically diverse patient population in Southern California to the CCDI (dbGaP: phs002518). The goal of our current P30 Supplement Project is to augment the CHLA dataset with new types of data from our unique patient population, and to develop tools for integrating the diverse datasets in CCDI to improve diagnosis and treatment of all children with cancer. To date, we have identified, screened, and selected the key whole slide images (WSI) from almost 700 CNS and non-CNS solid tumors in our cohort. We have developed a computational pipeline to access and organize metadata for each WSI. Additionally, our pipeline includes an analytical workflow to quantitatively assess WSI slides. In parallel, we have also generated whole-genome enzymatic methyl-seq data from 170 CNS tumors and developed a methyl-seq bioinformatics pipeline with a CNS tumor classifier compatible with methyl-seq data. We are actively developing tools to leverage these multimodal data for improved diagnosis and characterization of the tumors. Our pipeline is fully automated from raw bioinformatics processing to a classification report detailing predicted classification, classification score, UMAP clustering analysis, copy number profiling, and quality control metrics. Additionally, we have developed AI/ML tools to converge results from WSI analysis, methylation values, and OncoKids Cancer Panel results to enhance diagnostic decision-making. Our long-term goals are to incorporate additional data formats into our suite of multimodal analytic tools and to make these tools available to the CCDI community. |
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Alexander L. Markowitz | Children's Hospital Los Angeles, Keck School of Medicine, University of Southern California | amarkowitz@chla.usc.edu | |
| 80 | Star/flag Childhood Cancer Data Initiative Annual Symposium (Abstract Registration): Submission #80 | Lock Childhood Cancer Data Initiative Annual Symposium (Abstract Registration): Submission #80 | Add notes to Childhood Cancer Data Initiative Annual Symposium (Abstract Registration): Submission #80 | Wed, 09/02/2026 - 12:08 | Anonymous | 10.208.28.41 | Progress-to-date toward building a multicenter study comparing second cancer risks after radiotherapy: the Pediatric Proton and Photon Therapy Comparison Cohort | Proton radiotherapy has emerged as the preferred radiotherapy modality for many cancers, especially in children. Proton radiotherapy is expected to reduce the risk of subsequent cancer and other adverse long-term health effects compared to photon radiotherapy because the physical properties of protons allow for lower radiation exposure to surrounding normal tissues. However, the magnitude of the purported reduction in risk remains uncertain, and no randomized clinical trials have compared the two radiotherapy types in children, who are more susceptible than adults to the late effects of radiation. Observational studies, while generally reassuring, have had important methodological limitations. CCDI funding enabled establishment of the NCI Pediatric Proton and Photon Therapy Comparison Cohort, a large-scale multicenter study with the primary aim to compare the risk of subsequent cancers in pediatric cancer patients treated with proton versus photon radiotherapy. Patient and treatment data, including electronic radiotherapy records, are being collected for eligible patients treated 2006-2025 at 17 participating centers. Long-term follow-up for incident second cancers will be conducted via linkage with state cancer registries. State-of-the-art radiation dose reconstruction methods developed for application in this cohort will allow for assessment of radiation dose-response and dose volume effects. Our poster will update the status of ongoing clinical and radiotherapy data collection and will describe the currently available study population by demographic and treatment factors. Research from this cohort is expected to inform clinical practice for pediatric cancer patients by providing the first large-scale systematic comparison of subsequent cancer risk after proton compared to photon therapy. |
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Todd M Gibson | National Cancer Institute | todd.gibson@nih.gov | |
| 79 | Star/flag Childhood Cancer Data Initiative Annual Symposium (Abstract Registration): Submission #79 | Lock Childhood Cancer Data Initiative Annual Symposium (Abstract Registration): Submission #79 | Add notes to Childhood Cancer Data Initiative Annual Symposium (Abstract Registration): Submission #79 | Wed, 09/02/2026 - 04:13 | Anonymous | 10.208.28.41 | DATA-DRIVEN CHARACTERIZATION OF ANGIOGENESIS AND TREATMENT RESISTANCE IN PEDIATRIC HIGH-GRADE GLIOMA | Pediatric high-grade gliomas (pHGG) are aggressive and molecularly diverse brain tumors with limited treatment options and poor outcomes. Their diffuse growth and marked molecular heterogeneity contribute to frequent treatment failure. In addition to tumor-intrinsic factors, the tumor microenvironment and its interaction with the vasculature are increasingly recognized as important contributors to disease progression. Angiogenesis, vascular remodeling, hypoxia, and tumor–vascular interactions may influence tumor growth and treatment response, but their molecular links to treatment resistance in pHGG. The CCDI provides an opportunity to investigate these relationships using integrated pediatric molecular and clinical datasets. To identify angiogenesis-associated molecular signatures linked to aggressive tumor biology and treatment resistance in pHGG and to prioritize biologically relevant and potentially actionable vascular targets. Publicly available CCDI molecular and clinical datasets will be analyzed using an integrated computational framework. Transcriptomic data will undergo standardized preprocessing and differential-expression analysis, followed by functional and pathway enrichment focused on angiogenesis, vascular remodeling, hypoxia, tumor–vascular interactions, and treatment resistance. Protein–protein interaction and network analyses will be used to identify functionally connected genes and potential regulatory hubs. Where clinical and treatment-response information is available, prioritized candidates will be assessed for associations with disease characteristics and clinical outcomes. Candidate selection will integrate differential expression, pathway involvement,clinical relevance, biological plausibility, and therapeutic tractability. The study is expected to identify angiogenesis-related signatures and vascular regulatory nodes associated with aggressive and treatment-resistant pHGG. By examining vascular biology within the broader tumor molecular landscape, this approach may generate clinically relevant and testable hypotheses for future functional validation. |
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Priya Rithika Vella | Continental Hospital | Priyarithikavella@gmail.com | |
| 78 | Star/flag Childhood Cancer Data Initiative Annual Symposium (Abstract Registration): Submission #78 | Lock Childhood Cancer Data Initiative Annual Symposium (Abstract Registration): Submission #78 | Add notes to Childhood Cancer Data Initiative Annual Symposium (Abstract Registration): Submission #78 | Fri, 08/28/2026 - 08:53 | Anonymous | 10.208.28.32 | Increasing the National Childhood Cancer Registry (NCCR) user base through the development and deployment of educational materials | As one of the tools in the Childhood Cancer Data Initiative’s (CCDI) data ecosystem, the National Childhood Cancer Registry (NCCR), provides comprehensive, high-value data resources critical for advancing childhood cancer research. NCCR is undertaking a strategic initiative to broaden its user base. We are developing and piloting a suite of educational materials designed to increase effective engagement with NCCR data products. Initial consultation with graduate-level educators revealed strong interest in accessible childhood cancer data resources for use in workshops, graduate courses, and clinician-researcher training. In response, we convened an interdisciplinary NCCR Educational Materials Working Group (EDWG) comprised of experienced instructors from leading institutions, NCCR subject matter experts, and NCI project managers. The EDWG is creating a modular instructional unit featuring customizable session formats, student handouts, grading rubrics, data access instructions, compliance tools, case studies, exams, and introductory video content. These materials will be piloted in academic courses and professional workshops. Feedback from students will be collected by professors, and professors will guide refinement of materials before broader dissemination to schools of public health, medicine, epidemiology, and health services research, as well as cancer centers and relevant professional organizations. This effort may be a model for other efforts to increase the user base of CCDI tools and platforms. By expanding access to high-quality training resources, this initiative aims to strengthen data literacy, foster greater engagement with NCCR, and support research capacity-building across the childhood cancer research community. |
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Johanna Goderre | NCI | johanna.goderrejones@nih.gov | |
| 77 | Star/flag Childhood Cancer Data Initiative Annual Symposium (Abstract Registration): Submission #77 | Lock Childhood Cancer Data Initiative Annual Symposium (Abstract Registration): Submission #77 | Add notes to Childhood Cancer Data Initiative Annual Symposium (Abstract Registration): Submission #77 | Thu, 08/27/2026 - 14:20 | Anonymous | 10.208.24.244 | Radiation dosimetry for the first large-scale systemic comparison of the risk of second cancers in children treated with proton versus photon therapy | The Pediatric Proton/Photon Therapy Comparison (PPTC) cohort study is the first large-scale study comparing the risk of second cancers in children treated with proton versus photon radiotherapy. The study has collected treatment records for more than 10,000 pediatric cancer patients from 17 hospitals. Because the cohort data are both large and distributed across multiple databases, we have deployed a cloud-based system to streamline data collection, monitoring, validation, and transfer to a high-performance computing (HPC) cluster. Data for ~7,500 patients have been collected to date in an industry-standard medical imaging format (DICOM), including radiation field parameters, planning computed tomography (CT) images with clinician-delineated anatomical structures, and dose distributions from the treatment planning system (TPS). Another critical component is the development of scalable methods for estimating individualized, organ-level radiation dose for epidemiological dose-response analyses. Planning CT scans typically cover only the treatment region, often omitting organs of interest for late effects research. To address this, we developed a method to extend partial-body CT images using a library of surrogate anatomies. In addition, due to variability and inconsistency in organ delineation and naming, we utilize a deep learning–based automatic segmentation tool to standardize organ delineation. Finally, we address limitations of TPS dose estimates by performing advanced Monte Carlo radiation transport simulations of both modalities. These simulations integrate patient data and detailed physics modeling and are efficiently executed on the NIH HPC cluster. This poster presents our efforts to implement a state-of-the-art dosimetry platform—from data collection to individualized dose calculations. |
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Matthew M. Mille | Division of Cancer Epidemiology and Genetics, National Cancer Institute, National Institutes of Health, Bethesda, MD | matthew.mille@nih.gov | |
| 76 | Star/flag Childhood Cancer Data Initiative Annual Symposium (Abstract Registration): Submission #76 | Lock Childhood Cancer Data Initiative Annual Symposium (Abstract Registration): Submission #76 | Add notes to Childhood Cancer Data Initiative Annual Symposium (Abstract Registration): Submission #76 | Wed, 08/26/2026 - 15:12 | Anonymous | 10.208.24.244 | Characterizing the Children’s Oncology Group panel of neuroblastoma patient-derived xenografts for response to induction and salvage chemotherapy | Tumor biopsies are often not obtained at the time of progressive disease (PD) in neuroblastoma (NB). Patient-derived xenografts (PDXs) established from bone marrow or blood of NB patients enable studies to define molecular mechanisms and approaches to overcome therapy resistance. Tumor, marrow, or peripheral blood samples from 40 high-risk patients, 19 at diagnosis (Dx) and 21 at PD (12 of 21 at post-mortem), were received via Children’s Oncology Group (COG) protocol ANBL00B1 and established as PDXs. PDXs were classified by the response to chemotherapy (Cyclo/Topo, cyclophosphamide + topotecan, 3 x 21-day cycles, or TMZ/IRI, relapse chemo, temozolomide + irinotecan, 2 x 21-day cycles) as non-responders (NR), stable disease (SD), partial responders (PR), and complete responders (CR). Engraftment rates were 17% for Dx and 24% for PD samples. Based on an algorithm we developed for PDX response evaluation, Cyclo/Topo responses were NR (n=11), SD (n=2), PR (n=9), and CR (n=18), and 9 of 11 showed CR to TMZ/IRN. Nine of 13 (69%) of the NR+SD PDXs were established from post-mortem PD (PD-PM) samples, while 12 of 18 (67%) of the CR group were from Dx samples. The response of PDXs to Cyclo/Topo was greater for pretherapy (Dx) PDXs than for PD-PM or PD PDXs; six of the 11 PD models showed significantly better EFS for TMZ/IRN relative to Cyclo/Topo (P<0.05, n=6 PDXs). This panel of 40 NB PDXs, characterized for their response to chemotherapy, will enable studies to define the molecular mechanisms of NB therapy resistance and is available at www.CCcells.org. |
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Min H. Kang | Texas Tech University Health Sciences Center | min.kang@ttuhsc.edu | |
| 75 | Star/flag Childhood Cancer Data Initiative Annual Symposium (Abstract Registration): Submission #75 | Lock Childhood Cancer Data Initiative Annual Symposium (Abstract Registration): Submission #75 | Add notes to Childhood Cancer Data Initiative Annual Symposium (Abstract Registration): Submission #75 | Wed, 08/26/2026 - 12:30 | Anonymous | 10.208.24.244 | Leveraging the ExtractEHR+ Toolkit to Enhance Medication Data in the Children’s Brain Tumor Network and Childhood Cancer Data Initiative | Background: The National Cancer Institute (NCI) Childhood Cancer Data Initiative (CCDI) has enabled the distribution of previously unshared data. A critical component to maximize the utility of these data is to have longitudinal clinical data, such as treatment data. Manual abstraction has typically been used to capture treatment data, but this process is time consuming and subject to human error. This study aimed to use automated extraction and processing of electronic health record (EHR) data using the ExtractEHR+ Toolkit to describe chemotherapy exposures for children enrolled in the Children’s Brain Tumor Network (CBTN) who have contributed data to the CCDI. Methods: Patients enrolled in CBTN at two hospitals were included. ExtractEHR extracted all medication orders and administrations, including outpatient prescriptions, heights, and weights. Medication order and administration data were merged and MedCleanEHR centrally cleaned these data to identify unique chemotherapy exposures and dose amounts. Height and weight data were used to calculate body surface area and merged with medication data. For prescriptions where a discrete dose field was not included, regular expressions were used to identify doses in free text fields. MedCleanEHR calculated cumulative chemotherapy doses each patient received. Results: The cohort included 1628 patients. ExtractEHR successfully pulled 3,849,099 medication administrations and 839,736 medication orders. Once cleaned, there were 98,877 unique chemotherapy administrations and 8,781 chemotherapy prescriptions. Conclusions: The ExtractEHR+ Toolkit can efficiently ascertains chemotherapy exposures and dosing for patients in the CBTN cohort. These data will be transferred to the CCDI to enhance clinical data in the CCDI ecosystem. |
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Tamara P. Miller, MD, MSCE | Emory University/Children's Healthcare of Atlanta | tamara.miller@emory.edu | |
| 74 | Star/flag Childhood Cancer Data Initiative Annual Symposium (Abstract Registration): Submission #74 | Lock Childhood Cancer Data Initiative Annual Symposium (Abstract Registration): Submission #74 | Add notes to Childhood Cancer Data Initiative Annual Symposium (Abstract Registration): Submission #74 | Wed, 08/26/2026 - 00:12 | Anonymous | 10.208.28.32 | Graph Artificial Intelligence for Pediatric Oncology (GAIPO): a graph AI platform for precision oncology using pediatric clinical and omics data | Advances in artificial intelligence (AI) are shifting the paradigm in precision medicine for pediatric cancer, including biomarker identification, drug discovery, and survival analysis. The Childhood Cancer Data Initiative (CCDI) ecosystem provides essential clinicogenomic data for deep learning in pediatric cancer research. To enable the efficient use of the CCDI resource for AI model training and implementation, we developed a generic graph AI platform, Graph Artificial Intelligence for Pediatric Oncology (GAIPO), as well as the standards of data, models, and pipelines, to streamline the use of CCDI clinicogenomics data across various data modalities from bulk and single-cell omics data to clinical information in AI development. GAIPO provides a comprehensive workflow: (1) Data fetching through CCDI Data Federation Resource API and cBioPortal API for clinical metadata, multi-omics, and spatial transcriptomics data; (2) Data modeling through mapping and harmonizing the fetched clinical and genomics data according to the CCDI data model; (3) Graph construction functionalities through GraphML from NetworkX; (4) Graph AI implementation of existing models; and (5) Post-analysis, such as vital feature selection and survival analysis. We demonstrate the capabilities of our GAIPO in treating two pediatric cancers, glioma and Wilms tumor, with potential applicability to other pediatric cancers. We apply graph AI models, MOGONET and PCGS, to integrate multi-omics data using explainable graph convolutional networks, allowing patient classification and biomarker identification. Notably, the new PCGS model identifies background-specific key features for biomarker discovery, risk group identification, and survival analysis in glioma and Wilms tumor, with potential applicability to other pediatric cancers. |
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Jing Su | Indiana University School of Medicine, IU Simon Comprehensive Cancer Center | su1@iu.edu | |
| 73 | Star/flag Childhood Cancer Data Initiative Annual Symposium (Abstract Registration): Submission #73 | Lock Childhood Cancer Data Initiative Annual Symposium (Abstract Registration): Submission #73 | Add notes to Childhood Cancer Data Initiative Annual Symposium (Abstract Registration): Submission #73 | Tue, 08/25/2026 - 13:02 | Anonymous | 10.208.24.128 | Machine-Readable Metadata and AI-Assisted Discovery for the National Childhood Cancer Registry Data Platform | The NCCR Data Platform provides linked, de-identified cancer data for patients ages 0-39 across 9 datasources including tumor registry (1.4M patients, 21 states), pharmacy claims (12.6M records), medical claims (116M records), clinical trials, radiation oncology, and socioeconomic measures. Researchers face barriers discovering available data before investing in IRB-approved access requests. We developed a machine-readable metadata layer using semantic web standards, consisting of: dataset catalog records conforming to NLM's DATMM 6.0.0 with 9 standalone Dataset entries; a lightweight OWL ontology defining classes for variables, value sets, cohort filters, and portable cohort definitions; and an instance data layer containing 42,067 RDF triples representing 533 variables, 3,715 coded values with observed frequencies, and 51 cohort filter definitions linked to 14 biomedical vocabularies. We additionally developed a CLI tool for metadata-driven cohort discovery and a portable cohort definition format. All artifacts are open source. The metadata enables data discovery without patient-level access. Researchers can query which variables exist, what values are permissible, and how many records each value contains. The cohort definition format supports reproducible, shareable patient selection criteria. Critically, the structured metadata enables AI-assisted discovery: when provided to a large language model, it allows grounded answers about data availability — including record counts, filter criteria, and cross-datasource relationships — without hallucination. Publishing NCCR metadata as linked data addresses FAIR compliance, NIH CADR requirements, researcher education, and AI-powered discovery simultaneously. The approach generalizes to other Controlled Access Data Repositories and represents a novel pathway for making complex research platforms discoverable to a broader community. |
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Radu Robotin | NCI | radu.robotin@nih.gov | |
| 72 | Star/flag Childhood Cancer Data Initiative Annual Symposium (Abstract Registration): Submission #72 | Lock Childhood Cancer Data Initiative Annual Symposium (Abstract Registration): Submission #72 | Add notes to Childhood Cancer Data Initiative Annual Symposium (Abstract Registration): Submission #72 | Tue, 08/25/2026 - 09:28 | Anonymous | 10.208.24.128 | GAIPO enables spatial omics on Childhood Cancer Data Initiative ecosystem for precision pediatric oncology | The Childhood Cancer Data Initiative (CCDI) strives to accelerate pediatric cancer research by uniting clinical records with emerging spatial omics data but lacks a unified framework for joint analysis. Although CCDI and public repositories provide rich patient records and high-resolution spatial profiles, no unified data standards and framework exist to extract, harmonize, and process these multimodal datasets for interpretable discovery. We developed the GAIPO (Graph Artificial Intelligence for Pediatric Oncology) clinicospatial data standard and platform, enabling graph artificial intelligence (AI) on the CCDI ecosystem. The GAIPO clinicospatial standard unifies spatial assay formats, spatial resolutions, and registration of spatial data across modalities. To promote interoperability and reproducibility, we define a spatial data model that encompasses data standards, formats, and interfaces to integrate imaging and omics data through spatial graph representation, thereby enabling the implementation of explainable graph AI models. The GAIPO platform provides: flexible interfaces to CCDI APIs and public portals for retrieval of clinical metadata, spatial omics data, and associated histopathology images; a preprocessing pipeline for omics and imaging data; a graph AI module that integrates multiple modalities for downstream analysis; and an interpretation module to identify important features and cells and reveal critical enrichment pathways and cell-cell interactions. We demonstrated GAIPO’s functionalities by using xSiGra as the explainable graph AI model, graph Grad-CAM as the interpretability module, ScPCA as the CCDI-participating resource, and Wilms tumor as the pediatric cancer. The GAIPO standards and platform facilitate graph AI on clinicospatial data in precision oncology for pediatric cancers. |
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Aishwarya Budhkar | Indiana University Bloomington | abudhkar@iu.edu | |
| 71 | Star/flag Childhood Cancer Data Initiative Annual Symposium (Abstract Registration): Submission #71 | Lock Childhood Cancer Data Initiative Annual Symposium (Abstract Registration): Submission #71 | Add notes to Childhood Cancer Data Initiative Annual Symposium (Abstract Registration): Submission #71 | Tue, 08/25/2026 - 00:42 | Anonymous | 10.208.28.32 | Next-generation models to advance pediatric solid cancer treatments. | The high attrition rate in oncology is attributed to an over-reliance on 2D cell cultures and animal models, which often fail to accurately replicate patient tumor biology. The adoption of advanced, clinically relevant preclinical models is essential for better identifying and prioritizing agents with a higher likelihood of success in clinical trials. The Human Cancer Models Initiative (HCMI) is a global initiative founded by the National Cancer Institute (NCI). The mission is to generate patient-derived next-generation cancer models from diverse tumor types as a community resource. Unlike traditional cancer models, the new models are cultured under optimized, predominantly 3D conditions that better preserve the characteristics of the parental tumors than historical culture conditions. This preservation is validated through phenotypic and molecular analyses of tumor tissue and models, which are shared alongside associated clinical and molecular data. To contribute towards the goal of the HCMI the Stanford CDMC is dedicated to models of pediatric solid tumors, emphasizing central nervous system (CNS) tumors, the leading cause of cancer-related death in children. We have generated <85 pediatric cancer models along with case-associated clinical and biospecimen data, as well as internal QC data validating the derived cancer models, for further characterization and distribution via the HCMI pipeline. Our next-generation cancer models partially capture the heterogeneity of pediatric CNS tumors, neuroblastoma, hepatoblastoma, Wilms tumor, and brain metastases from neuroblastoma and rare sarcoma-related cancers. Longitudinal biobanking has identified and characterized novel onco-fusion proteins, rare tumor entities, and recurrences, and therapeutic vulnerabilities through multi-omics. |
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Claudia Katharina Petritsch | Stanford University | cpetri@stanford.edu | |
| 70 | Star/flag Childhood Cancer Data Initiative Annual Symposium (Abstract Registration): Submission #70 | Lock Childhood Cancer Data Initiative Annual Symposium (Abstract Registration): Submission #70 | Add notes to Childhood Cancer Data Initiative Annual Symposium (Abstract Registration): Submission #70 | Wed, 08/19/2026 - 18:04 | Anonymous | 10.208.24.128 | Pediatric Cancer Registries and Data Systems in Sub-Saharan Africa: A Scoping Review of Coverage, Interoperability, and Data-Sharing Readiness. | Background Pediatric cancer registries are essential for surveillance, outcomes research, health-system planning, and participation in collaborative data ecosystems.However, readiness of existing systems in sub-Saharan Africa (SSA) for sustainable cross-institutional data sharing remains unclear. Aim To map pediatric cancer registries and data systems in SSA and assess their coverage,data elements and standardization,interoperability,governance,and readiness for sustainable cross-institutional data sharing. Methods Following PRISMA-ScR principles, bibliographic and supplementary sources were systematically searched through August 2026 for primary studies describing patient-level pediatric cancer registries, databases, or registry-development initiatives in SSA. Eighteen eligible publications were charted across predefined domains: coverage/case ascertainment, data elements/standardization, interoperability, governance/data access, institutional capacity, and sustainability.Interoperability was assessed across semantic, technical, organizational, and patient-linkage dimensions. Results Population- and hospital-based registries, multicountry networks, and prospective digital databases were identified. ICCC-3, ICD-O-3, and Toronto staging demonstrated semantic standardization, including multicountry implementation (Liu et al., 2023; Mallon et al., 2023). Selected systems supported multicentre data aggregation, staging, longitudinal follow-up, survival, and research-ready clinical and socioeconomic data (Mallon et al., 2022; Parkin et al., 2021; Davidson et al., 2022). However, population coverage was uneven, while patient-level linkage and technical interoperability across clinical, pathology, registry, and national information systems were limited or insufficiently reported. Governance, workforce capacity, and sustainable financing were heterogeneous. Conclusion SSA has important foundations for a shareable pediatric cancer data ecosystem, but readiness remains uneven. Priorities include common data elements, patient-level linkage, technical interoperability, longitudinal outcome capture, and governed cross-institutional sharing to strengthen the contribution of SSA data to collaborative ecosystems such as CCDI. |
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Korede Akindele | The Dorcas Cancer Foundation | araireakindele@gmail.com | |
| 69 | Star/flag Childhood Cancer Data Initiative Annual Symposium (Abstract Registration): Submission #69 | Lock Childhood Cancer Data Initiative Annual Symposium (Abstract Registration): Submission #69 | Add notes to Childhood Cancer Data Initiative Annual Symposium (Abstract Registration): Submission #69 | Wed, 08/19/2026 - 13:40 | Anonymous | 10.208.28.32 | Expanding In Vivo Genotyping Across the Clinical Spectrum of Retinoblastoma: Aqueous Humor Liquid Biopsy Enables Comprehensive Biallelic RB1 Characterization Without Tumor Tissue | We previously established aqueous humor (AH) as a tumor-proximal liquid biopsy provides access to tumor-derived molecular information in retinoblastoma, a cancer in which diagnostic tumor biopsy is contraindicated. The critical next question was whether this signal could reconstruct the genotype of the living tumor—and whether molecular detectability persists when tumors are small and eye-sparing. We analyzed diagnostic AH from 65 eyes of 53 patients using LBSeq4Kids, a clinically validated assay optimized for AH specimens. RB1 sequence variants, deletions, promoter alterations, and loss of heterozygosity were assessed, with germline RB1 status determined by blood testing when available. At least one RB1 alteration was identified in 57 of 65 eyes (87.7%), while complete biallelic molecular characterization was achieved in 53 eyes (81.5%). Importantly, molecular detectability was preserved across disease stage: complete characterization was 81.8% in B/C eyes and 81.1% in D/E eyes. At the individual alteration level, B/C eyes achieved 90.0% detection, comparable to published tumor-tissue cohorts (83.1–94.8%). AH captured the clinically relevant spectrum of RB1 alterations, including sequence variants, promoter alterations, deletions, and loss of heterozygosity. These findings advance AH liquid biopsy from detecting tumor-derived molecular signal to reconstructing the genotype of the living tumor. The preserved detectability in small, eye-sparing tumors indicates that AH can provide molecular access even when tumor tissue is unavailable. Because B–E disease represents the vast majority of eyes in large international clinical cohorts, this approach has the potential to expand molecular genotyping beyond tissue-accessible patients and enable multicenter studies linking RB1 genotype with clinical phenotype. |
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Liya Xu | Children's Hospital Los Angeles | liyaxu@usc.edu | |
| 68 | Star/flag Childhood Cancer Data Initiative Annual Symposium (Abstract Registration): Submission #68 | Lock Childhood Cancer Data Initiative Annual Symposium (Abstract Registration): Submission #68 | Add notes to Childhood Cancer Data Initiative Annual Symposium (Abstract Registration): Submission #68 | Tue, 08/18/2026 - 20:01 | Anonymous | 10.208.28.32 | Hybrid Semantic–Lexical Retrieval for Source-to-CDE Mapping in the NCI caDSR | Accurate mapping of source data elements to standardized common data elements (CDEs) is essential for biomedical data integration, but short, abbreviated, and heterogeneous source descriptions make both lexical and semantic matching unreliable. We constructed a benchmark of approximately 69,000 expert-linked source-to-CDE mappings from the National Cancer Institute’s caDSR and used it to evaluate bi-encoders, text representations, and two-stage fine-tuning. We then developed a hybrid system that combines semantic retrieval, CDE Match-inspired keyword retrieval, cross-encoder scoring, and supervised reranking. Across an internal test set and five distribution-shifted holdouts, the system achieved Recall@5 of 0.971 internally and 0.802–0.972 externally. It achieved the highest Recall@5 among evaluated methods on five of six datasets; on GDC, performance was near ceiling at 70/72, within one query of the official NCI CDE Match service and its Python approximation at 71/72. |
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James H Tanis | NCI | james.tanis@nih.gov |