ArticleNeuro-oncology2023
Clinical outcome of pediatric medulloblastoma patients with Li-Fraumeni syndrome.
Open access · bronzeAbstract readMulticenter Study
In one paragraph
Article in Neuro-oncology, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 14 papers.
0numbers the graph read from it
0cells of the map it votes in
14citing papers in PubMed
4.4field-weighted citation impact, top 5% 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
14 citing papers in PubMed, 19 citations in OpenAlex.
- High-dose chemotherapy with autologous stem cell rescue in children under 5 years of age with central nervous system embryonal tumors: results from a prospective cohort in an upper-middle-income country.Child's nervous system : ChNS : official journal of the International Society for Pediatric Neurosurgery · 2026Article
- Pediatric medulloblastoma.Child's nervous system : ChNS : official journal of the International Society for Pediatric Neurosurgery · 2026Review
- Article
- PC3/Tis21/BTG2 and BTG1 genes: regulators of the cell cycle and neurogenesis, as well as tumor suppressors in malignant brain tumors.Frontiers in cell and developmental biology · 2026Review
- Radiotherapy results in decreased time to second cancer in children with Li Fraumeni syndrome.Journal of the National Cancer Institute · 2025Article
- Salvage therapies for first relapse of SHH medulloblastoma in early childhood.Neuro-oncology · 2025Article
- Treatment Outcomes and Prognostic Factors for Patients With Medulloblastoma Having Defined Molecular Subtypes.Advances in radiation oncology · 2025Article
- Targeting synthetic lethality between non-homologous end joining and radiation in very-high-risk medulloblastoma.Cell reports. Medicine · 2025Article
- Re-envisioning genetic predisposition to childhood and adolescent cancers.Nature reviews. Cancer · 2025Review
- Application of Circulating Tumor DNA in the Auxiliary Diagnosis and Prognosis Prediction of Glioma.Cellular and molecular neurobiology · 2024Review
- Medulloblastoma in children with Fanconi anemia: Association with FA-D1/FA-N, SHH type and poor survival independent of treatment strategies.Neuro-oncology · 2024Article
- Update on Cancer Predisposition Syndromes and Surveillance Guidelines for Childhood Brain Tumors.Clinical cancer research : an official journal of the American Association for Cancer Research · 2024Review
- Evolution of Systemic Therapy in Medulloblastoma Including Irradiation-Sparing Approaches.Diagnostics (Basel, Switzerland) · 2023Review
- Medulloblastomas withNeuro-oncology advancesArticle
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
73 authors at 20 institutions in 18 countries.
Anna S KolodziejczakHopp Children's Cancer Center (KiTZ), Heidelberg, Germany.ORCID 0000-0002-1866-7116
Lea Guerrini-RousseauDepartment of Children and Adolescents Oncology, Gustave Roussy, Université Paris-Saclay, 94805 Villejuif, France.
Julien Masliah PlanchonDepartment of Diagnostic and Theranostic Medicine, Somatic Genetics Unit, Institut Curie, Paris-Science Lettres University, Paris, France.
Jonas EckerHopp Children's Cancer Center (KiTZ), Heidelberg, Germany.
Florian SeltHopp Children's Cancer Center (KiTZ), Heidelberg, Germany.ORCID 0000-0001-9717-6712
Martin MynarekPediatric Hematology and Oncology, University Medical Center Hamburg-Eppendorf, Hamburg, Germany.ORCID 0000-0003-3302-2719
Denise ObrechtPediatric Hematology and Oncology, University Medical Center Hamburg-Eppendorf, Hamburg, Germany.
Martin SillHopp Children's Cancer Center (KiTZ), Heidelberg, Germany.ORCID 0000-0001-7616-7665
Robert J AutryHopp Children's Cancer Center (KiTZ), Heidelberg, Germany.
Eric Stutheit-ZhaoHopp Children's Cancer Center (KiTZ), Heidelberg, Germany.
Steffen HirschHopp Children's Cancer Center (KiTZ), Heidelberg, Germany.ORCID 0000-0002-4920-0930
Elsa AmouyalSIREDO Pediatric Oncology Center, Institut Curie, Paris-Science Lettres University, Paris, France.
Christelle DufourDepartment of Children and Adolescents Oncology, Gustave Roussy, Université Paris-Saclay, 94805 Villejuif, France.
Olivier AyraultInstitut Curie, PSL Research University, CNRS UMR, INSERM, Orsay, France Université Paris Sud, Université Paris-Saclay, CNRS UMR 3347, INSERM U1021, Orsay, France.
Jacob TorrejonInstitut Curie, PSL Research University, CNRS UMR, INSERM, Orsay, France Université Paris Sud, Université Paris-Saclay, CNRS UMR 3347, INSERM U1021, Orsay, France.
Sebastian M WaszakCentre for Molecular Medicine Norway (NCMM), Nordic EMBL Partnership, University of Oslo and Oslo University Hospital, Oslo, Norway.
Vijay RamaswamyDivision of Neurosurgery, Program in Developmental and Stem Cell Biology, Arthur and Sonia Labatt Brain Tumour Research Centre, Hospital for Sick Children, Toronto, Ontario, Canada.ORCID 0000-0002-6557-895X
Virve PentikainenDivision of Hematology-Oncology and Stem Cell Transplantation, Children's Hospital, Helsinki University Hospital, Helsinki, Finland.
Haci Ahmet DemirDepartment of Pediatric Hematology-Oncology, Private Memorial Ankara Hospital, Ankara, Turkey.
Steven C CliffordWolfson Childhood Cancer Research Centre, Newcastle University Centre for Cancer, Newcastle upon Tyne, United Kingdom.
Ed C SchwalbeWolfson Childhood Cancer Research Centre, Newcastle University Centre for Cancer, Newcastle upon Tyne, United Kingdom.
Luca MassimiPediatric Neurosurgery, Fondazione Policlinico Universitario A. Gemelli IRCCS, Catholic University Medical School, Rome, Italy.
Matija SnuderlDepartment of Pathology, New York University Langone Health, New York City, NY, USA.
Kristyn GalbraithDepartment of Pathology, New York University Langone Health, New York City, NY, USA.ORCID 0000-0002-5660-866X
Matthias A KarajannisDepartment of Pediatrics, Memorial Sloan Kettering Cancer Center, New York City, NY, USA.ORCID 0000-0002-7151-6528
Katherine HillDepartment of Pediatrics, Memorial Sloan Kettering Cancer Center, New York City, NY, USA.
Bryan K LiDepartment of Pediatrics, Memorial Sloan Kettering Cancer Center, New York City, NY, USA.
Mike WalshDepartment of Pediatrics, Memorial Sloan Kettering Cancer Center, New York City, NY, USA.
Christine L WhiteVictorian Clinical Genetics Services, Parkville, Australia.
Shelagh RedmondChildhood Cancer Registry, Institute of Social and Preventive Medicine, University of Bern, Bern, Switzerland.
Loizou LoizosPediatric Oncology/Hematology/Immunology at the Medical School of the University of Nicosia, Nicosia, Cyprus.
Marcus JakobDepartment of Paediatric Haematology, Oncology and Stem-Cell Transplantation, University Hospital Regensburg, Regensburg, Germany.
Uwe R KordesPediatric Hematology and Oncology, University Medical Center Hamburg-Eppendorf, Hamburg, Germany.
Irene SchmidPaediatric Haematology and Oncology, Dr. von Hauner Children's Hospital, Ludwig Maximilians University Munich, Munich, Germany.
Julia HauerPediatric Haematology and Oncology, University Hospital Carl Gustav Carus, Dresden, Germany.
Claudia BlattmannPaediatric Haematology, Oncology and Immunology, Olgahospital, Klinikum Stuttgart, Stuttgart, Germany.
Maria FilippidouDivision of Pediatric Hematology-Oncology, First Department of Pediatrics, National and Kapodistrian University of Athens, "Aghia Sophia" Children's Hospital, Athens, Greece.
Gianluca PiccoloNeuro-Oncology Unit, IRCCS Istituto Giannina Gaslini, Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health, University of Genoa, Genoa, Italy.
Wolfram ScheurlenPaediatric Haematology and Oncology, Cnopfsche Paediatric Clinic, Nurnberg, Germany.
Ahmed FarragDepartment of Paediatric Haematology, Oncology and Stem-Cell Transplantation, Paediatric Clinic, University Hospital Aachen, Aachen, Germany.
Kerstin GrundInstitute of Human Genetics, University Hospital Heidelberg, Heidelberg, Germany.
Christian SutterInstitute of Human Genetics, University Hospital Heidelberg, Heidelberg, Germany.
Torsten PietschInstitute of Neuropathology, DGNN Brain Tumor Reference Center, University of Bonn Medical Center, Bonn, Germany.
Stephan FrankDivision of Neuropathology, Institute of Pathology, Basel University Hospital, Basel, Switzerland.
Denis M ScheweDepartment of Pediatrics, Otto-von-Guericke University Magdeburg, Magdeburg, Germany.
David MalkinDivision of Hematology/Oncology, Department of Pediatrics, The Hospital for Sick Children, University of Toronto, Toronto, Ontario, Canada.
Myriam Ben-ArushPediatric Hematology Oncology, Rambam Medical Center, Haifa, Israel.
Astrid SehestedDepartment of Paediatrics and Adolescent Medicine, Juliane Marie Centre, Copenhagen University Hospital, Copenhagen, Denmark.
Tai-Tong WongDivision of Pediatric Neurosurgery, Department of Neurosurgery, Taipei Medical University Hospital, Taipei Medical University, Taipei 110, Taiwan.
Kuo-Sheng WuGraduate Institute of Clinical Medicine, College of Medicine, Taipei Medical University, Taipei, Taiwan.
Yen-Lin LiuDepartment of Pediatrics, School of Medicine, College of Medicine, Taipei Medical University, Taipei, Taiwan.
Fernando CarcellerPaediatric and Adolescent Oncology Drug Development Team, Children and Young People's Unit, The Royal Marsden NHS Foundation Trust and The Institute of Cancer Research, Sutton, United Kingdom.
Sabine MuellerDepartment of Neurology, Neurosurgery and Pediatrics, University of California, San Francisco, USA.
Schuyler StollerDepartment of Neurology, University of California, San Francisco, USA.ORCID 0000-0002-1931-7521
Michael D TaylorDivision of Neurosurgery, Program in Developmental and Stem Cell Biology, Arthur and Sonia Labatt Brain Tumour Research Centre, Hospital for Sick Children, Toronto, Ontario, Canada.
Uri TaboriDivision of Hematology and Oncology, Hospital for Sick Children, Toronto, Ontario, Canada.ORCID 0000-0002-5019-2683
Eric BouffetDivision of Hematology and Oncology, Hospital for Sick Children, Toronto, Ontario, Canada.ORCID 0000-0002-6832-6539
Marcel KoolHopp Children's Cancer Center (KiTZ), Heidelberg, Germany.
Felix SahmHopp Children's Cancer Center (KiTZ), Heidelberg, Germany.ORCID 0000-0001-5441-1962
Andreas von DeimlingDepartment of Neuropathology, Institute of Pathology, Heidelberg University Hospital, and CCU Neuropathology, German Cancer Institute (DKF), Heidelberg, Germany.
Andrey KorshunovDepartment of Neuropathology, Institute of Pathology, Heidelberg University Hospital, and CCU Neuropathology, German Cancer Institute (DKF), Heidelberg, Germany.
Katja von HoffDepartment of Pediatric Oncology and Hematology, Charité - Universitätsmedizin Berlin, Berlin, Germany.ORCID 0000-0002-5669-8546
Christian P KratzPediatric Hematology and Oncology, Hannover Medical School, Hannover, Germany.
Dominik SturmHopp Children's Cancer Center (KiTZ), Heidelberg, Germany.
David T W JonesHopp Children's Cancer Center (KiTZ), Heidelberg, Germany.
Stefan RutkowskiPediatric Hematology and Oncology, University Medical Center Hamburg-Eppendorf, Hamburg, Germany.
Cornelis M van TilburgHopp Children's Cancer Center (KiTZ), Heidelberg, Germany.
Olaf WittHopp Children's Cancer Center (KiTZ), Heidelberg, Germany.
Gaëlle BougeardUniv Rouen Normandie, Inserm U1245 and CHU Rouen, Department of Genetics, F-76000 Rouen, France.
Kristian W PajtlerHopp Children's Cancer Center (KiTZ), Heidelberg, Germany.ORCID 0000-0002-3562-6121
Stefan M PfisterHopp Children's Cancer Center (KiTZ), Heidelberg, Germany.ORCID 0000-0002-5447-5322
Franck BourdeautSIREDO Pediatric Oncology Center, Institut Curie, Paris-Science Lettres University, Paris, France.ORCID 0000-0001-9489-6781
Till MildeHopp Children's Cancer Center (KiTZ), Heidelberg, Germany.ORCID 0000-0002-7267-1052
German Cancer Research Center · DEHospital for Sick Children · CAMemorial Sloan Kettering Cancer Center · USUniversität Hamburg · DEInserm · FRInstitut Curie · FRHeidelberg University · DENYU Langone Health · USTaipei Medical University · TWUniversité Paris-Sud · FRUniversity of California, San Francisco · USAarhus University Hospital · DKAgostino Gemelli University Polyclinic · ITCnopf´sche Kinderklinik · DECopenhagen University Hospital · DKHelsinki University Hospital · FIHudson Institute of Medical Research · AUIstituto Giannina Gaslini · ITLudwig-Maximilians-Universität München · DEMedizinische Hochschule Hannover · DE
Funding
X-RAY CRYSTALLOGRAPHYP30CA008748 · NCI · SLOAN-KETTERING INSTITUTE FOR CANCER RES · PI Hongbiao Carl Lekaye · 1985 to 2026
$347.4MInducing Neural Maturation in Medulloblastoma by Targeting EZH2R01NS122987 · NINDS · NEW YORK UNIVERSITY SCHOOL OF MEDICINE · PI RAJU, PRAVEEN B., SNUDERL, MATIJA · 2022 to 2025
$2.8MInducing neural maturation in medulloblastoma by targeting EZH2R56NS122987 · NINDS · NEW YORK UNIVERSITY SCHOOL OF MEDICINE · PI RAJU, PRAVEEN B., SNUDERL, MATIJA · 2021 to 2021
$461kCancer Research UK DRCRPG-NOV22/100002NCI NIH HHS P30 CA008748NINDS NIH HHS R01 NS122987NINDS NIH HHS R56 NS122987
Abstract
backgroundThe prognosis for Li-Fraumeni syndrome (LFS) patients with medulloblastoma (MB) is poor. Comprehensive clinical data for this patient group is lacking, challenging the development of novel therapeutic strategies. Here, we present clinical and molecular data on a retrospective cohort of pediatric LFS MB patients.
methodsIn this multinational, multicenter retrospective cohort study, LFS patients under 21 years with MB and class 5 or class 4 constitutional TP53 variants were included. TP53 mutation status, methylation subgroup, treatment, progression free- (PFS) and overall survival (OS), recurrence patterns, and incidence of subsequent neoplasms were evaluated.
resultsThe study evaluated 47 LFS individuals diagnosed with MB, mainly classified as DNA methylation subgroup "SHH_3" (86%). The majority (74%) of constitutional TP53 variants represented missense variants. The 2- and 5-year (y-) PFS were 36% and 20%, and 2- and 5y-OS were 53% and 23%, respectively. Patients who received postoperative radiotherapy (RT) (2y-PFS: 44%, 2y-OS: 60%) or chemotherapy before RT (2y-PFS: 32%, 2y-OS: 48%) had significantly better clinical outcome then patients who were not treated with RT (2y-PFS: 0%, 2y-OS: 25%). Patients treated according to protocols including high-intensity chemotherapy and patients who received only maintenance-type chemotherapy showed similar outcomes (2y-PFS: 42% and 35%, 2y-OS: 68% and 53%, respectively).
conclusionsLFS MB patients have a dismal prognosis. In the presented cohort use of RT significantly increased survival rates, whereas chemotherapy intensity did not influence their clinical outcome. Prospective collection of clinical data and development of novel treatments are required to improve the outcome of LFS MB patients.
Indexed as
Cerebellar NeoplasmsLi-Fraumeni SyndromeMedulloblastomaChildGerm-Line MutationHumansProspective StudiesRetrospective StudiesTumor Suppressor Protein p53Tumor Suppressor Protein p53Li–Fraumeni syndromemedulloblastomasurvivalTP53
Identifiers
What OpenQuestion holds
Textmetadata
Read underepoch 390
Registered trials
None linked
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.