Evidence map›Paper›PMID 31693904›Full record

ArticleCell reports2019

Genomic Profiling of Childhood Tumor Patient-Derived Xenograft Models to Enable Rational Clinical Trial Design.

Jo Lynne Rokita, Komal S Rathi, Maria F Cardenas, Kristen A Upton, Joy Jayaseelan, Katherine L Cross, Jacob Pfeil, Laura E Egolf, Gregory P Way, Alvin Farrel and 59 more

Open access · goldAbstract read
In one paragraph

Article in Cell reports, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 127 papers.

0numbers the graph read from it
0cells of the map it votes in
127citing papers in PubMed
13.6field-weighted citation impact, top 1% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

127 citing papers in PubMed, 163 citations in OpenAlex.

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  11. Choosing the right animal model for sarcoma research.Cellular and molecular life sciences : CMLS · 2026
    Review
  12. D3-GPC2-Directed CAR T Cells Are Safe and Efficacious in Preclinical Models of Neuroblastoma and Small Cell Lung Cancer.Clinical cancer research : an official journal of the American Association for Cancer Research · 2025
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  17. The Open Pediatric Cancer Project.bioRxiv : the preprint server for biology · 2025
    Article
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67 more citing papers are in PubMed but not listed here.

4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

69 authors at 18 institutions in 2 countries.

Jo Lynne RokitaDivision of Oncology, Children's Hospital of Philadelphia, and Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104-4318, USA; Department of Bioinformatics and Health Informatics, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA; Center for Data-Driven Discovery in Biomedicine, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA.
Komal S RathiDepartment of Bioinformatics and Health Informatics, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA; Center for Data-Driven Discovery in Biomedicine, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA.
Maria F CardenasHuman Genome Sequencing Center, Baylor College of Medicine, Houston, TX 77030, USA.
Kristen A UptonDivision of Oncology, Children's Hospital of Philadelphia, and Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104-4318, USA.
Joy JayaseelanHuman Genome Sequencing Center, Baylor College of Medicine, Houston, TX 77030, USA.
Katherine L CrossGuardian Forensic Sciences, Abington, PA 19001, USA.
Jacob PfeilUC Santa Cruz Genomics Institute, University of California, Santa Cruz, Santa Cruz, CA 95064, USA.
Laura E EgolfDivision of Oncology, Children's Hospital of Philadelphia, and Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104-4318, USA; Cell and Molecular Biology Graduate Group, University of Pennsylvania, Philadelphia, PA 19104, USA.
Gregory P WayGenomics and Computational Biology Graduate Group, University of Pennsylvania, Philadelphia, PA 19104, USA.
Alvin FarrelDepartment of Bioinformatics and Health Informatics, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA.
Nathan M KendserskyDivision of Oncology, Children's Hospital of Philadelphia, and Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104-4318, USA; Department of Systems Pharmacology and Translational Therapeutics, University of Pennsylvania, Philadelphia, PA 19104, USA.
Khushbu PatelDepartment of Bioinformatics and Health Informatics, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA.
Krutika S GaonkarDepartment of Bioinformatics and Health Informatics, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA; Center for Data-Driven Discovery in Biomedicine, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA.
Apexa ModiDivision of Oncology, Children's Hospital of Philadelphia, and Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104-4318, USA; Genomics and Computational Biology Graduate Group, University of Pennsylvania, Philadelphia, PA 19104, USA.
Esther R BerkoDivision of Oncology, Children's Hospital of Philadelphia, and Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104-4318, USA.
Gonzalo LopezDivision of Oncology, Children's Hospital of Philadelphia, and Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104-4318, USA; Department of Bioinformatics and Health Informatics, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA.
Zalman VaksmanDepartment of Bioinformatics and Health Informatics, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA.
Chelsea MayohChildren's Cancer Institute, School of Women's and Children's Health, UNSW Sydney, Sydney, NSW, Australia.
Jonas NanceCancer Center, Texas Tech University Health Sciences Center School of Medicine, Lubbock, TX 79430, USA.
Kristyn McCoyCancer Center, Texas Tech University Health Sciences Center School of Medicine, Lubbock, TX 79430, USA.
Michelle HaberChildren's Cancer Institute, School of Women's and Children's Health, UNSW Sydney, Sydney, NSW, Australia.
Kathryn EvansChildren's Cancer Institute, School of Women's and Children's Health, UNSW Sydney, Sydney, NSW, Australia.
Hannah McCalmontChildren's Cancer Institute, School of Women's and Children's Health, UNSW Sydney, Sydney, NSW, Australia.
Katerina BendakChildren's Cancer Institute, School of Women's and Children's Health, UNSW Sydney, Sydney, NSW, Australia.
Julia W BöhmChildren's Cancer Institute, School of Women's and Children's Health, UNSW Sydney, Sydney, NSW, Australia.
Glenn M MarshallChildren's Cancer Institute, School of Women's and Children's Health, UNSW Sydney, Sydney, NSW, Australia; Sydney Children's Hospital, Sydney, NSW, Australia.
Vanessa TyrrellChildren's Cancer Institute, Kensington, NSW, Australia.
Karthik KalletlaDepartment of Bioinformatics and Health Informatics, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA; Center for Data-Driven Discovery in Biomedicine, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA.
Frank K BraunTexas Children's Cancer and Hematology Center, Department of Pediatrics, Baylor College of Medicine, Houston, TX 77030, USA.
Lin QiPreclinical Neurooncology Research Program, Texas Children's Cancer Research Center, Texas Children's Hospital, Houston, TX 77030, USA; Department of Pediatrics, Baylor College of Medicine, Houston, TX 77030, USA.
Yunchen DuPreclinical Neurooncology Research Program, Texas Children's Cancer Research Center, Texas Children's Hospital, Houston, TX 77030, USA; Department of Pediatrics, Baylor College of Medicine, Houston, TX 77030, USA.
Huiyuan ZhangPreclinical Neurooncology Research Program, Texas Children's Cancer Research Center, Texas Children's Hospital, Houston, TX 77030, USA; Department of Pediatrics, Baylor College of Medicine, Houston, TX 77030, USA.
Holly B LindsayPreclinical Neurooncology Research Program, Texas Children's Cancer Research Center, Texas Children's Hospital, Houston, TX 77030, USA; Department of Pediatrics, Baylor College of Medicine, Houston, TX 77030, USA.
Sibo ZhaoPreclinical Neurooncology Research Program, Texas Children's Cancer Research Center, Texas Children's Hospital, Houston, TX 77030, USA; Department of Pediatrics, Baylor College of Medicine, Houston, TX 77030, USA.
Jack ShuPreclinical Neurooncology Research Program, Texas Children's Cancer Research Center, Texas Children's Hospital, Houston, TX 77030, USA; Department of Pediatrics, Baylor College of Medicine, Houston, TX 77030, USA.
Patricia BaxterPreclinical Neurooncology Research Program, Texas Children's Cancer Research Center, Texas Children's Hospital, Houston, TX 77030, USA; Department of Pediatrics, Baylor College of Medicine, Houston, TX 77030, USA.
Christopher MortonDepartment of Surgery, St. Jude Children's Research Hospital, Memphis, TN 38105, USA.
Dias KurmashevGreehey Children's Cancer Research Institute, University of Texas Health Science Center, San Antonio, TX 78229, USA.
Siyuan ZhengGreehey Children's Cancer Research Institute, University of Texas Health Science Center, San Antonio, TX 78229, USA.
Yidong ChenGreehey Children's Cancer Research Institute, University of Texas Health Science Center, San Antonio, TX 78229, USA.
Jay BowenThe Research Institute at Nationwide Children's Hospital, Columbus, OH 43205, USA.
Anthony C BryanThe Research Institute at Nationwide Children's Hospital, Columbus, OH 43205, USA.
Kristen M LeraasThe Research Institute at Nationwide Children's Hospital, Columbus, OH 43205, USA.
Sara E CoppensThe Research Institute at Nationwide Children's Hospital, Columbus, OH 43205, USA.
HarshaVardhan DoddapaneniHuman Genome Sequencing Center, Baylor College of Medicine, Houston, TX 77030, USA.
Zeineen MominHuman Genome Sequencing Center, Baylor College of Medicine, Houston, TX 77030, USA.
Wendong ZhangDivision of Pediatrics, The University of Texas MD Anderson Cancer Center, Houston, TX 77030, USA.
Gregory I SacksDivision of Oncology, Children's Hospital of Philadelphia, and Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104-4318, USA.
Lori S HartDivision of Oncology, Children's Hospital of Philadelphia, and Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104-4318, USA.
Kateryna KrytskaDivision of Oncology, Children's Hospital of Philadelphia, and Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104-4318, USA.
Yael P MosseDivision of Oncology, Children's Hospital of Philadelphia, and Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104-4318, USA.
Gregory J GattoDepartment of Global Health Technologies, RTI International, Research Triangle Park, NC 27709, USA.
Yolanda SanchezDepartment of Molecular and Systems Biology, Geisel School of Medicine at Dartmouth, Hanover, NH 03755, USA; Norris Cotton Cancer Center, Lebanon, NH 03766, USA.
Casey S GreeneChildhood Cancer Data Lab, Alex's Lemonade Stand Foundation, Philadelphia, PA 19102, USA; Department of Systems Pharmacology and Translational Therapeutics, University of Pennsylvania, Philadelphia, PA 19104, USA.
Sharon J DiskinDivision of Oncology, Children's Hospital of Philadelphia, and Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104-4318, USA; Department of Bioinformatics and Health Informatics, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA.
Olena Morozova VaskeDepartment of Molecular, Cell and Developmental Biology, University of California, Santa Cruz, Santa Cruz, CA 95064, USA; UC Santa Cruz Genomics Institute, University of California, Santa Cruz, Santa Cruz, CA 95064, USA.
David HausslerUC Santa Cruz Genomics Institute, University of California, Santa Cruz, Santa Cruz, CA 95064, USA; Howard Hughes Medical Institute, University of California, Santa Cruz, Santa Cruz, CA 95064, USA.
Julie M Gastier-FosterThe Research Institute at Nationwide Children's Hospital, Columbus, OH 43205, USA; The Ohio State University College of Medicine, Departments of Pathology and Pediatrics, Columbus, OH 43210, USA.
E Anders KolbDepartment of Pediatrics, Sidney Kimmel Medical College at Thomas Jefferson University, Philadelphia, PA 19107, USA; Nemours Center for Cancer and Blood Disorders, Nemours/Alfred I. duPont Hospital for Children, Wilmington, DE 19803, USA.
Richard GorlickDivision of Pediatrics, The University of Texas MD Anderson Cancer Center, Houston, TX 77030, USA.
Xiao-Nan LiPreclinical Neurooncology Research Program, Texas Children's Cancer Research Center, Texas Children's Hospital, Houston, TX 77030, USA; Department of Pediatrics, Baylor College of Medicine, Houston, TX 77030, USA; Division of Hematology, Oncology, Neuro-oncology and Stem Cell Transplant, Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, IL 60611, USA; Department of Pediatrics, Northwestern University Feinberg School of Medicine, Chicago, IL 60611, USA.
C Patrick ReynoldsCancer Center, Texas Tech University Health Sciences Center School of Medicine, Lubbock, TX 79430, USA.
Raushan T KurmashevaGreehey Children's Cancer Research Institute, University of Texas Health Science Center, San Antonio, TX 78229, USA.
Peter J HoughtonGreehey Children's Cancer Research Institute, University of Texas Health Science Center, San Antonio, TX 78229, USA.
Malcolm A SmithNational Cancer Institute, NIH, Bethesda, MD 20814, USA.
Richard B LockChildren's Cancer Institute, Kensington, NSW, Australia.
Pichai RamanDepartment of Bioinformatics and Health Informatics, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA; Center for Data-Driven Discovery in Biomedicine, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA.
David A WheelerHuman Genome Sequencing Center, Baylor College of Medicine, Houston, TX 77030, USA.
John M MarisDivision of Oncology, Children's Hospital of Philadelphia, and Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104-4318, USA. Electronic address: maris@email.chop.edu.
Children's Hospital of Philadelphia · USBaylor College of Medicine · USUNSW Sydney · AUNationwide Children's Hospital · USThe University of Texas Health Science Center at San Antonio · USTexas Tech University · USChildren's Cancer Institute Australia · AUThe University of Texas MD Anderson Cancer Center · USUniversity of California, Santa Cruz · USAlfred I. duPont Hospital for Children · USDartmouth College · USHoward Hughes Medical Institute · USNational Cancer Institute · USNorthwestern University · USRTI International · USSt. Jude Children's Research Hospital · USTranslational Therapeutics (United States) · USUniversity of Pennsylvania · US

Funding

Comprehensive Sequencing and Analysis of Variation in NHLBI CohortsUC2HL102925 · NHLBI · BROAD INSTITUTE, INC. · PI ALTSHULER, DAVID, GABRIEL, STACEY · 2010 to 2010
$14.7M
Northwest Genomics CenterUC2HL102926 · NHLBI · UNIVERSITY OF WASHINGTON · PI GREEN, PHILIP P, NICKERSON, DEBORAH A · 2010 to 2010
$14.0M
Northwest Genomics CenterRC2HL102926 · NHLBI · UNIVERSITY OF WASHINGTON · PI GREEN, PHILIP P, NICKERSON, DEBORAH A · 2009 to 2009
$11.0M
CANCER RESEARCH TRAINING PROGRAMT32CA009615 · NCI · UNIVERSITY OF PENNSYLVANIA · PI Gregory L Beatty, MICHAEL D HOGARTY · 1988 to 2026
$10.4M
Comprehensive Sequencing and Analysis of Variation in NHLBI CohortsRC2HL102925 · NHLBI · BROAD INSTITUTE, INC. · PI ALTSHULER, DAVID, GABRIEL, STACEY · 2009 to 2009
$10.2M
Discovering mechanisms of neuroblastoma tumorigenesis to improve patient outcomesR35CA220500 · NCI · CHILDREN'S HOSP OF PHILADELPHIA · PI JOHN M MARIS · 2017 to 2026
$8.7M
Patient-derived orthotopic xenograft models for cancer disparity studies of pediatric brain tumorsU01CA199288 · NCI · LURIE CHILDREN'S HOSPITAL OF CHICAGO · PI Xiaonan Li · 2015 to 2026
$6.8M
Pediatric Preclinical Testing Consortium - LeukemiaU01CA199000 · NCI · UNIVERSITY OF NEW SOUTH WALES · PI Richard B Lock · 2015 to 2026
$4.8M
Osteosarcoma: Patient Derived Xenograft Preclinical TestingU01CA199221 · NCI · UNIVERSITY OF TX MD ANDERSON CAN CTR · PI Richard G. Gorlick · 2015 to 2026
$3.8M
Pediatric Preclinical Testing Consortium: Research Programs Non-CNS (U01)U01CA199297 · NCI · UNIVERSITY OF TEXAS HLTH SCIENCE CENTER · PI HOUGHTON, PETER J · 2015 to 2021
$3.5M
A Testing Program to Identify Novel Agents for Treatment of Pediatric and AYA High-Risk Sarcoma, Kidney and Liver CancerU01CA263981 · NCI · UNIVERSITY OF TEXAS HLTH SCIENCE CENTER · PI Raushan Kurmasheva · 2021 to 2026
$3.2M
Targeting Tumors with NF1 LossR01NS095411 · NINDS · DARTMOUTH COLLEGE · PI HOOPES, JACK, RATNER, NANCY · 2015 to 2019
$2.7M
NCI NIH HHS R01 CA221957NCI NIH HHS R35 CA220500NCI NIH HHS T32 CA009615NCI NIH HHS U01 CA199000NCI NIH HHS U01 CA199221NCI NIH HHS U01 CA199222NCI NIH HHS U01 CA199287NCI NIH HHS U01 CA199288NCI NIH HHS U01 CA199297NCI NIH HHS U01 CA263981NHLBI NIH HHS RC2 HL102923NHLBI NIH HHS RC2 HL102924NHLBI NIH HHS RC2 HL102925NHLBI NIH HHS RC2 HL102926NHLBI NIH HHS RC2 HL103010NHLBI NIH HHS UC2 HL102923NHLBI NIH HHS UC2 HL102924NHLBI NIH HHS UC2 HL102925NHLBI NIH HHS UC2 HL102926NHLBI NIH HHS UC2 HL103010NINDS NIH HHS R01 NS095411
6 · The paper itself

Abstract

Accelerating cures for children with cancer remains an immediate challenge as a result of extensive oncogenic heterogeneity between and within histologies, distinct molecular mechanisms evolving between diagnosis and relapsed disease, and limited therapeutic options. To systematically prioritize and rationally test novel agents in preclinical murine models, researchers within the Pediatric Preclinical Testing Consortium are continuously developing patient-derived xenografts (PDXs)-many of which are refractory to current standard-of-care treatments-from high-risk childhood cancers. Here, we genomically characterize 261 PDX models from 37 unique pediatric cancers; demonstrate faithful recapitulation of histologies and subtypes; and refine our understanding of relapsed disease. In addition, we use expression signatures to classify tumors for TP53 and NF1 pathway inactivation. We anticipate that these data will serve as a resource for pediatric oncology drug development and will guide rational clinical trial design for children with cancer.

Indexed as

Xenograft Model Antitumor AssaysAnimalsCell Line, TumorCentral Nervous System NeoplasmsChildClinical Trials as TopicDisease Models, AnimalExome SequencingGenomicsHumansMiceMutationNeuroblastomaNeurofibromin 1OsteosarcomaPrecursor Cell Lymphoblastic Leukemia-LymphomaNeurofibromin 1NF1 protein, humanTP53 protein, humanTumor Suppressor Protein p53classifiercopy number profilingpatient-derived xenograftpediatric cancerpreclinical testingrelapsetranscriptome sequencingwhole-exome sequencing

Identifiers

PMID31693904
PMCPMC6880934
OpenAlexW2986723641

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Registered trials

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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.