Trial reportCancer discovery2021
Matched Targeted Therapy for Pediatric Patients with Relapsed, Refractory, or High-Risk Leukemias: A Report from the LEAP Consortium.
Open access · bronzeAbstract readClinical TrialMulticenter Study
In one paragraph
Trial report in Cancer discovery, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 25 papers.
0numbers the graph read from it
0cells of the map it votes in
25citing papers in PubMed
2.7field-weighted citation impact, top 7% of its field
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.
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.
Who cites it
25 citing papers in PubMed, 38 citations in OpenAlex.
- First-year results of the International Leukemia/ Lymphoma Target Board for pediatric relapsed and refractory hematological malignancies.Haematologica · 2026Article
- Combining menin and MEK inhibition to target poor prognosis KMT2A-rearranged RAS pathway-mutant acute myeloid leukemia.Blood advances · 2026Article
- Article
- Multi-selective RAS(ON) Inhibition Targets Oncogenic RAS Mutations and Overcomes RAS/MAPK-Mediated Resistance to FLT3 and BCL2 Inhibitors in Acute Myeloid Leukemia.bioRxiv : the preprint server for biology · 2025Article
- A germline FLT3 variant in aplastic anemia.Biomarker research · 2025Article
- A structure-based tool to interpret the significance of kinase mutations in clinical next generation sequencing in cancer.Frontiers in oncology · 2025Article
- Precision Medicine for Acute Lymphoblastic Leukemia in Children: A Review.Children (Basel, Switzerland) · 2024Review
- Nucleotide depletion promotes cell fate transitions by inducing DNA replication stress.Developmental cell · 2024Article
- Oncogenic and immunological targets for matched therapy of pediatric blood cancer patients: Dutch iTHER study experience.HemaSphere · 2024Article
- Acute lymphoblastic leukaemia.Nature reviews. Disease primers · 2024Review
- Splicing modulators impair DNA damage response and induce killing of cohesin-mutant MDS and AML.Science translational medicine · 2024Article
- Integration of Genomic Sequencing Drives Therapeutic Targeting of PDGFRA in T-Cell Acute Lymphoblastic Leukemia/Lymphoblastic Lymphoma.Clinical cancer research : an official journal of the American Association for Cancer Research · 2023Article
- Children's Oncology Group blueprint for research: Acute lymphoblastic leukemia.Pediatric blood & cancer · 2023Article
- Relapsed pediatric acute myeloid leukaemia: state-of-the-art in 2023.Haematologica · 2023Review
- Layered immunity and layered leukemogenicity: Developmentally restricted mechanisms of pediatric leukemia initiation.Immunological reviews · 2023Review
- The relapsed acute lymphoblastic leukemia network (ReALLNet): a multidisciplinary project from the spanish society of pediatric hematology and oncology (SEHOP).Frontiers in pediatrics · 2023Article
- B-cell lymphoma 2 family members and sarcomas: a promising target in a heterogeneous disease.Exploration of targeted anti-tumor therapy · 2023Review
- Ex Vivo Drug Sensitivity Correlates with Clinical Response and Supports Personalized Therapy in Pediatric AML.Cancers · 2022Article
- Pharmacogenomic Profiling of Pediatric Acute Myeloid Leukemia to Identify Therapeutic Vulnerabilities and Inform Functional Precision Medicine.Blood cancer discovery · 2022Article
- How Genetics and Genomics Advances Are Rewriting Pediatric Cancer Research and Clinical Care.Medicina (Kaunas, Lithuania) · 2022Review
Corrections and comments
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Authors and funding
53 authors at 20 institutions in 4 countries.
Yana PikmanDepartment of Pediatric Oncology, Dana-Farber Cancer Institute, Boston, Massachusetts. Kimberly_stegmaier@dfci.harvard.edu Yana_pikman@dfci.harvard.edu.ORCID 0000-0002-5336-0216
Sarah K TasianDivision of Oncology and Center for Childhood Cancer Research, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania.
Maria Luisa SulisDivision of Pediatric Hematology/Oncology/Stem Cell Transplantation, Columbia University Irving Medical Center, New York, New York.
Kristen StevensonDepartment of Biostatistics and Computational Biology, Dana-Farber Cancer Institute, Boston, Massachusetts.
Traci M BlonquistDepartment of Biostatistics and Computational Biology, Dana-Farber Cancer Institute, Boston, Massachusetts.
Beth Apsel WingerDepartment of Pediatrics, Division of Hematology/Oncology, Benioff Children's Hospital and the Helen Diller Family Comprehensive Cancer Center, University of California, San Francisco, San Francisco, California.
Todd M CooperSeattle Children's Hospital, Cancer and Blood Disorders Center, Seattle, Washington.
Melinda PaulyDivision of Hematology/Oncology, Emory University, Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Atlanta, Georgia.ORCID 0000-0001-8906-5836
Kelly W MaloneyChildren's Hospital Colorado, University of Colorado Cancer Center, Aurora, Colorado.
Michael J BurkeMedical College of Wisconsin, Children's Hospital of Wisconsin, Milwaukee, Wisconsin.
Patrick A BrownJohns Hopkins Kimmel Cancer Center, Baltimore, Maryland.ORCID 0000-0002-8653-1069
Nathan GossaiCenter for Cancer and Blood Disorders, Children's Minnesota, Minneapolis, Minnesota.
Jennifer L McNeerUniversity of Chicago, Comer Children's Hospital, Chicago, Illinois.
Neerav N ShuklaDepartment of Pediatrics, Memorial Sloan Kettering Cancer Center, New York, New York.
Peter D ColeChildren's Hospital at Montefiore, Bronx, New York.ORCID 0000-0002-3525-8015
Justine M KahnDivision of Pediatric Hematology/Oncology/Stem Cell Transplantation, Columbia University Irving Medical Center, New York, New York.ORCID 0000-0003-3330-6754
Jing ChenDivision of Pediatric Hematology/Oncology/Stem Cell Transplantation, Columbia University Irving Medical Center, New York, New York.
Matthew J BarthRoswell Park Comprehensive Cancer Center, Buffalo, New York.ORCID 0000-0001-5263-9509
Jeffrey A MageeDivision of Pediatric Hematology/Oncology, Washington University/St. Louis Children's Hospital, St. Louis, Missouri.
Lisa GennariniChildren's Hospital at Montefiore, Bronx, New York.
Asmani A AdhavDepartment of Pediatric Oncology, Dana-Farber Cancer Institute, Boston, Massachusetts.
Catherine M ClintonDepartment of Pediatric Oncology, Dana-Farber Cancer Institute, Boston, Massachusetts.
Nicole Ocasio-MartinezDepartment of Pediatric Oncology, Dana-Farber Cancer Institute, Boston, Massachusetts.
Giacomo GottiDepartment of Pediatric Oncology, Dana-Farber Cancer Institute, Boston, Massachusetts.
Yuting LiDepartment of Pediatric Oncology, Dana-Farber Cancer Institute, Boston, Massachusetts.
Shan LinDepartment of Pediatric Oncology, Dana-Farber Cancer Institute, Boston, Massachusetts.
Alma ImamovicBroad Institute of Massachusetts Institute of Technology and Harvard University, Cambridge, Massachusetts.ORCID 0000-0003-4454-6868
Cristina E TognonDivision of Hematology and Medical Oncology, Knight Cancer Institute, Oregon Health and Science University, Portland, Oregon.
Tasleema PatelDivision of Oncology and Center for Childhood Cancer Research, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania.ORCID 0000-0002-7927-6071
Haley L FaustDivision of Oncology and Center for Childhood Cancer Research, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania.
Cristina F ContrerasDivision of Oncology and Center for Childhood Cancer Research, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania.ORCID 0000-0002-3867-8840
Anjali CremerDepartment of Pediatric Oncology, Dana-Farber Cancer Institute, Boston, Massachusetts.
Wilian A CortopassiDepartment of Pharmaceutical Chemistry, University of California, San Francisco, San Francisco, California.
Diego Garrido RuizDepartment of Pharmaceutical Chemistry, University of California, San Francisco, San Francisco, California.ORCID 0000-0002-2441-385X
Matthew P JacobsonDepartment of Pharmaceutical Chemistry, University of California, San Francisco, San Francisco, California.
Neekesh V DhariaDepartment of Pediatric Oncology, Dana-Farber Cancer Institute, Boston, Massachusetts.ORCID 0000-0001-5048-067X
Angela SuINSERM UMR 944, IRSL, St Louis Hospital, Paris, France.
Amanda L RobichaudDepartment of Pediatric Oncology, Dana-Farber Cancer Institute, Boston, Massachusetts.
Amy Saur ConwayDepartment of Pediatric Oncology, Dana-Farber Cancer Institute, Boston, Massachusetts.
Katherine TarlockSeattle Children's Hospital, Cancer and Blood Disorders Center, Seattle, Washington.
Elliot StieglitzDepartment of Pediatrics, Division of Hematology/Oncology, Benioff Children's Hospital and the Helen Diller Family Comprehensive Cancer Center, University of California, San Francisco, San Francisco, California.ORCID 0000-0001-7032-4623
Andrew E PlaceDepartment of Pediatric Oncology, Dana-Farber Cancer Institute, Boston, Massachusetts.
Alexandre PuissantINSERM UMR 944, IRSL, St Louis Hospital, Paris, France.
Stephen P HungerDivision of Oncology and Center for Childhood Cancer Research, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania.ORCID 0000-0002-5492-3957
Annette S KimDepartment of Pathology, Brigham and Women's Hospital, Boston, Massachusetts.ORCID 0000-0002-8699-2439
Neal I LindemanDepartment of Pathology, Brigham and Women's Hospital, Boston, Massachusetts.
Lia GoreChildren's Hospital Colorado, University of Colorado Cancer Center, Aurora, Colorado.ORCID 0000-0002-2546-4616
Katherine A JanewayDepartment of Pediatric Oncology, Dana-Farber Cancer Institute, Boston, Massachusetts.
Lewis B SilvermanDepartment of Pediatric Oncology, Dana-Farber Cancer Institute, Boston, Massachusetts.
Jeffrey W TynerDivision of Hematology and Medical Oncology, Knight Cancer Institute, Oregon Health and Science University, Portland, Oregon.ORCID 0000-0002-2133-0960
Marian H HarrisDepartment of Pathology, Boston Children's Hospital, Boston, Massachusetts.
Mignon L LohDepartment of Pediatrics, Division of Hematology/Oncology, Benioff Children's Hospital and the Helen Diller Family Comprehensive Cancer Center, University of California, San Francisco, San Francisco, California.
Kimberly StegmaierDepartment of Pediatric Oncology, Dana-Farber Cancer Institute, Boston, Massachusetts. Kimberly_stegmaier@dfci.harvard.edu Yana_pikman@dfci.harvard.edu.
Dana-Farber Cancer Institute · USBoston Children's Hospital · USChildren's Hospital of Philadelphia · USBroad Institute · USCenter for Cancer and Blood Disorders · USUCSF Benioff Children's Hospital · USUniversity of California, San Francisco · USBrigham and Women's Hospital · USInserm · FRMemorial Sloan Kettering Cancer Center · USOregon Health & Science University · USUniversity of Colorado Cancer Center · USChildren's Healthcare of Atlanta · USChildren's Hospital at Montefiore · USChildren's Hospital of Wisconsin · USColumbia University Irving Medical Center · USComer Children's Hospital · USGoethe University Frankfurt · DEHackensack University Medical Center · USRoswell Park Comprehensive Cancer Center · US
Funding
X-RAY CRYSTALLOGRAPHYP30CA008748 · NCI · SLOAN-KETTERING INSTITUTE FOR CANCER RES · PI Hongbiao Carl Lekaye · 1985 to 2026
$347.4MUniversity of Colorado Cancer Center Support Grant - Lung Cancer Patient-Derived Xenografts with Autologous Human Immune SystemsP30CA046934 · NCI · UNIVERSITY OF COLORADO DENVER · PI James V Degregori · 1988 to 2026
$117.0MTargeting SYK Kinase in AMLP50CA206963 · NCI · DANA-FARBER CANCER INST · PI STEGMAIER, KIMBERLY · 2017 to 2021
$10.6MIntegrating Transforming Approaches to Identify and Target New Vulnerabilities in CancerR35CA210030 · NCI · DANA-FARBER CANCER INST · PI STEGMAIER, KIMBERLY · 2016 to 2022
$7.3MResearch Training in Childhood CancerT32CA128583 · NCI · UNIVERSITY OF CALIFORNIA, SAN FRANCISCO · PI Eric Alejandro Sweet-Cordero · 2007 to 2026
$6.0MMultispecific targeting incorporating cytokine receptor pathways in high risk pediatric acute leukemias to improve durability of adoptive cell therapy-induced remissionsU01CA232486 · NCI · UNIVERSITY OF COLORADO DENVER · PI FRY, TERRY J., TASIAN, SARAH KATHLEEN · 2018 to 2018
$4.0MAn Academic-Industry Partnership to Advance Functional Genomics for Personalized Oncology.R01CA214428 · NCI · FRED HUTCHINSON CANCER RESEARCH CENTER · PI KEMP, CHRISTOPHER J · 2018 to 2022
$3.0MTargeting mitochondrial one carbon folate metabolism for novel T-cell acute lymphoblastic leukemia therapyK08CA222684 · NCI · DANA-FARBER CANCER INST · PI PIKMAN, YANA · 2018 to 2022
$886kPI3K Pathway Inhibition for Philadelphia-Like Acute Lymphoblastic LeukemiaK08CA184418 · NCI · CHILDREN'S HOSP OF PHILADELPHIA · PI TASIAN, SARAH KATHLEEN · 2014 to 2018
$848kNCI NIH HHS K08 CA184418NCI NIH HHS K08 CA222684NCI NIH HHS P30 CA008748NCI NIH HHS P30 CA046934NCI NIH HHS P50 CA206963NCI NIH HHS R01 CA214428NCI NIH HHS R35 CA210030NCI NIH HHS T32 CA128583NCI NIH HHS U01 CA232486
Abstract
Despite a remarkable increase in the genomic profiling of cancer, integration of genomic discoveries into clinical care has lagged behind. We report the feasibility of rapid identification of targetable mutations in 153 pediatric patients with relapsed/refractory or high-risk leukemias enrolled on a prospective clinical trial conducted by the LEAP Consortium. Eighteen percent of patients had a high confidence Tier 1 or 2 recommendation. We describe clinical responses in the 14% of patients with relapsed/refractory leukemia who received the matched targeted therapy. Further, in order to inform future targeted therapy for patients, we validated variants of uncertain significance, performed
Indexed as
Biomarkers, TumorChildCohort StudiesDisease ProgressionFeasibility StudiesFemaleHumansLeukemiaMaleMolecular Targeted TherapyNeoplasm Recurrence, LocalProspective StudiesUnited StatesBiomarkers, Tumor
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