Trial reportNature medicine2024
The type II RAF inhibitor tovorafenib in relapsed/refractory pediatric low-grade glioma: the phase 2 FIREFLY-1 trial.
Erratum issuedOpen access · hybridAbstract readClinical Trial, Phase II
In one paragraph
Trial report in Nature medicine, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. Cited by 139 papers, 4 of them syntheses that pooled it.
0numbers the graph read from it
0cells of the map it votes in
139citing papers in PubMed, 4 pooled it
45.7field-weighted citation impact, top 1% 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
139 citing papers in PubMed, 4 syntheses or guidelines pooled it, 159 citations in OpenAlex.
- BRAF-targeted therapy in non-melanomatous BRAF-mutant tumours: a systematic review of broad but histology-modulated efficacy.International journal of clinical oncology · 2026Pooled it
- Intratumoral and intracranial hemorrhage associated with MAPK-pathway targeted therapy: a systematic review and mechanistic synthesis.Journal of neuro-oncology · 2026Pooled it
- Diagnostic and predictive molecular biomarkers in brain tumors across the lifespan: an age-stratified consensus statement.Journal of neuro-oncology · 2025Pooled it
- Updated EANO guideline on rational molecular testing of gliomas, glioneuronal, and neuronal tumors in adults for targeted therapy selection-Update 1.Neuro-oncology · 2025Guideline
- Taste Profile and Relative Bioavailability of Tovorafenib Powder for Oral Suspension and Food Effect of the Tovorafenib Tablet in Healthy Participants.Clinical pharmacology in drug development · 2025Trial
- Radiographic and visual response to the type II RAF inhibitor tovorafenib in children with relapsed/refractory optic pathway glioma in the FIREFLY-1 trial.Neuro-oncology · 2025Trial
- FDA Approval Summary: Tovorafenib for Relapsed or Refractory BRAF-Altered Pediatric Low-Grade Glioma.Clinical cancer research : an official journal of the American Association for Cancer Research · 2025Trial
- Practical management of BRAF inhibitors in glioma: toxicity and resistance.CNS oncology · 2026Review
- Rapid Whole Genome Sequencing Enhances Pediatric Cancer Diagnosis.Nature communications · 2026Observational
- PMDA regulatory update on approval and revision of the precautions for use of anticancer drugs in Japan; tovorafenib and dordaviprone for glioma, mirvetuximab soravtansine and pembrolizumab for ovarian cancer, tislelizumab for nasopharyngeal cancer, trastuzumab deruxtecan, palbociclib and datopotamab deruxtecan for breast cancer, epcoritamab for lymphoma, teclistamab for multiple myeloma.International journal of clinical oncology · 2026Article
- RAF-Kinase Inhibitor Associated Hair Hypopigmentation.International journal of dermatology · 2026Article
- Rigorous, transparent and inconclusive: what Europe's first joint clinical assessment reveals about a framework trying to serve everyone.Journal of comparative effectiveness research · 2026Review
- Bridging pediatric and adult neuro-oncology: Insights into adolescents and young adults (AYA) central nervous system tumors.Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2026Review
- Clinical Outcomes and New Treatments in Pediatric Brain Tumors.Current oncology (Toronto, Ont.) · 2026Article
- Next-generation therapeutics for glioblastoma: Challenges and future directions.Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2026Review
- Efficacy Study of Vemurafenib in Children With Recurrent or Progressive BRAFJCO precision oncology · 2026Article
- Histiocytosis development and clinical variation through the lens of genomics.The Journal of pathology · 2026Review
- Tempered optimism: Advances in the precision medicine era for pediatric low-grade glioma.Neuro-oncology · 2026Review
- Exploring the landscape of targetable alterations in patients with glioblastoma.Nature reviews. Clinical oncology · 2026Review
- A synergistic deep learning and machine learning framework for screening heterocyclic compounds against ALDH1A1.Molecular diversity · 2026Article
79 more citing papers are in PubMed but not listed here.
Corrections and comments
- Erratum issued
- Erratum issued
Authors and funding
42 authors at 20 institutions in 11 countries.
Lindsay B KilburnChildren's National Hospital, Washington, DC, USA. lkilburn@childrensnational.org.ORCID 0000-0003-1478-3619
Dong-Anh Khuong-QuangChildren's Cancer Centre, Royal Children's Hospital Melbourne, Melbourne, Victoria, Australia.ORCID 0000-0001-6305-7790
Jordan R HansfordMichael Rice Centre for Hematology and Oncology, Women's and Children's Hospital, Adelaide, South Australia, Australia.ORCID 0000-0001-7733-383X
Daniel LandiDuke University, Durham, NC, USA.
Jasper van der LugtPrincess Máxima Center for Pediatric Oncology, Utrecht, The Netherlands.ORCID 0000-0002-8186-338X
Sarah E S LearyCancer and Blood Disorders Center, Seattle Children's, Seattle, WA, USA.ORCID 0000-0003-0225-6184
Pablo Hernáiz DrieverCharité Universitätsmedizin Berlin, corporate member of Freie Universität Berlin and Humboldt-Universität Berlin, German HIT-LOGGIC-Registry for LGG in Children and Adolescents, Berlin, Germany.ORCID 0000-0003-3135-3872
Simon BaileyGreat North Children's Hospital and Newcastle University Centre for Cancer, Newcastle-upon-Tyne, UK.ORCID 0000-0003-4763-4329
Sébastien PerreaultCHU Sainte-Justine, Université de Montréal, Montréal, Quebec, Canada.
Geoffrey McCowageSydney Children's Hospitals Network, Westmead, New South Wales, Australia.
Angela J WaandersAnn & Robert H. Lurie Children's Hospital, Chicago, IL, USA.
David S ZieglerKids Cancer Centre, Sydney Children's Hospital, Randwick, New South Wales, Australia.ORCID 0000-0001-7451-7916
Olaf WittHopp Children's Cancer Center Heidelberg (KiTZ), Heidelberg, Germany.
Patricia A BaxterTexas Children's Cancer Center, Texas Children's Hospital, Baylor College of Medicine, Houston, TX, USA.
Hyoung Jin KangDepartment of Pediatrics, Seoul National University College of Medicine, Seoul National University Cancer Research Institute, Seoul National University Children's Hospital, Seoul, Republic of Korea.ORCID 0000-0003-1009-6002
Timothy E HassallChildren's Health Queensland Hospital and Health Service, South Brisbane, QLD, Australia.
Jung Woo HanSeverance Hospital, Yonsei University Health System, Seoul, Republic of Korea.ORCID 0000-0001-8936-1205
Darren HargraveUCL Great Ormond Street Institute of Child Health and Great Ormond Street Hospital for Children, London, UK.ORCID 0000-0001-8219-9807
Andrea T FransonC.S. Mott Children's Hospital, University of Michigan Medical School, Ann Arbor, MI, USA.ORCID 0000-0002-5361-7683
Michal Yalon OrenPediatric Hemato-Oncology, Sheba Medical Center, Ramat Gan, Israel.
Helen ToledanoDepartment of Pediatric Oncology, Schneider Children's Medical Center, Petach Tikva, and Faculty of Medicine, Tel Aviv University, Tel Aviv, Israel.
Valérie LaroucheDepartment of Pediatrics, Centre Mère-Enfant Soleil du CHU de Québec-Université Laval, Quebec City, Quebec, Canada.ORCID 0000-0003-4082-7267
Cassie KlineDivision of Oncology, Department of Pediatrics, Children's Hospital of Philadelphia, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, USA.ORCID 0000-0001-7765-7690
Mohamed S AbdelbakiDivision of Hematology and Oncology, Department of Pediatrics, School of Medicine, Washington University, St. Louis, MO, USA.
Nada JabadoMcGill University Health Centre (MUHC), Montreal Children's Hospital (MCH), Montreal, Quebec, Canada.ORCID 0000-0003-2485-3692
Nicholas G GottardoDepartment of Pediatric and Adolescent Oncology and Hematology, Perth Children's Hospital, Perth, Australia, and Brain Tumor Research Program, Telethon Kids Cancer Centre, Telethon Kids Institute, University of Western Australia, Perth, Western Australia, Australia.ORCID 0000-0002-1082-6776
Nicolas U GerberDepartment of Oncology, University Children's Hospital, Zurich, Switzerland.ORCID 0000-0002-1783-631X
Nicholas S WhipplePrimary Children's Hospital and University of Utah, Salt Lake City, UT, USA.
Devorah SegalNYU Langone Health, New York, NY, USA.ORCID 0000-0002-9740-1286
Susan N ChiPediatric Neuro-Oncology, Department of Pediatrics, Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Boston, MA, USA.
Liat OrenDepartment of Hematology & Oncology, Rambam Healthcare Campus, Haifa, Israel.
Enrica E K TanHaematology/Oncology Service, KK Women's and Children's Hospital, Singapore, Singapore.
Sabine MuellerDepartment of Neurology, Neurosurgery and Pediatrics, University of California, San Francisco, San Francisco, CA, USA.ORCID 0000-0002-8216-9357
Izzy CornelioDay One Biopharmaceuticals, Brisbane, CA, USA.
Lisa McLeodDay One Biopharmaceuticals, Brisbane, CA, USA.
Xin ZhaoDay One Biopharmaceuticals, Brisbane, CA, USA.
Ashley WalterDay One Biopharmaceuticals, Brisbane, CA, USA.
Daniel Da CostaDay One Biopharmaceuticals, Brisbane, CA, USA.
Peter ManleyDay One Biopharmaceuticals, Brisbane, CA, USA.
Samuel C BlackmanDay One Biopharmaceuticals, Brisbane, CA, USA.
Roger J PackerDivision of Neurology, Brain Tumor Institute, Center for Neuroscience and Behavioral Medicine, Children's National Hospital, Washington, DC, USA.
Karsten NysomDepartment of Pediatrics and Adolescent Medicine, Copenhagen University Hospital - Rigshospitalet, Copenhagen, Denmark.ORCID 0000-0003-2935-0058
NGM Biopharmaceuticals (United States) · USChildren's National · USBaylor College of Medicine · USCancer Institute of New South Wales · AUCenter for Cancer and Blood Disorders · USCentre Hospitalier Universitaire Sainte-Justine · CAChildren’s Health Queensland Hospital and Health Service · AUChildren's Hospital of Philadelphia · USCopenhagen University Hospital · DKDana-Farber/Boston Children's Cancer and Blood Disorders Center · USDuke University · USGerman Cancer Research Center · DEGreat North Children's Hospital · GBGreat Ormond Street Hospital · GBHumboldt-Universität zu Berlin · DEKK Women's and Children's Hospital · SGLurie Children's Hospital · USMontreal Children's Hospital · CANYU Langone Health · USPrimary Children's Hospital · US
Funding
No grant is acknowledged in the PubMed record.
Abstract
BRAF genomic alterations are the most common oncogenic drivers in pediatric low-grade glioma (pLGG). Arm 1 (n = 77) of the ongoing phase 2 FIREFLY-1 (PNOC026) trial investigated the efficacy of the oral, selective, central nervous system-penetrant, type II RAF inhibitor tovorafenib (420 mg m
Indexed as
FirefliesGliomaAnimalsChildHumansProto-Oncogene Proteins B-rafProto-Oncogene Proteins B-raf
Identifiers
What OpenQuestion holds
Textmetadata
LicenceCC BY
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.