Radiographic and visual response to the type II RAF inhibitor tovorafenib in children with relapsed/refractory optic pathway glioma in the FIREFLY-1 trial
Tovorafenib demonstrated anti-tumor activity in relapsed/refractory BRAF-altered OPG across radiological assessment criteria and was generally well tolerated. Importantly, vision remained stable or improved in most patients.
Authors
- Geoffrey McCowage (ORCID: https://orcid.org/0000-0003-3471-8443)
- Daniel Landi (ORCID: https://orcid.org/0000-0002-1487-1136)
- Darren Hargrave (ORCID: https://orcid.org/0000-0001-8219-9807)
- Nicholas G. Gottardo (ORCID: https://orcid.org/0000-0002-1082-6776)
- Jordan R. Hansford (ORCID: https://orcid.org/0000-0001-7733-383X)
- Pablo Hernáiz Driever (ORCID: https://orcid.org/0000-0003-3135-3872)
- Peter Manley (ORCID: https://orcid.org/0000-0003-3485-4517)
- Devorah Segal (ORCID: https://orcid.org/0000-0002-9740-1286)
- Jung Woo Han (ORCID: https://orcid.org/0000-0001-8936-1205)
- Sarah Leary (ORCID: https://orcid.org/0000-0003-0225-6184)
- Lindsay Kilburn (ORCID: https://orcid.org/0000-0003-1478-3619)
- David S. Ziegler (ORCID: https://orcid.org/0000-0001-7451-7916)
- Evelien de Vos‐Kerkhof (ORCID: https://orcid.org/0000-0002-4828-7069)
- Nicholas Whipple (ORCID: https://orcid.org/0000-0001-9503-2002)
- Tim Hassall (ORCID: https://orcid.org/0000-0002-7883-3710)
- Daniel Da Costa (ORCID: https://orcid.org/0000-0001-7971-6287)
- Andrea T. Franson (ORCID: https://orcid.org/0000-0002-5361-7683)
- Cassie Kline (ORCID: https://orcid.org/0000-0001-7765-7690)
- Dong‐Anh Khuong‐Quang
- Susan Chi (ORCID: https://orcid.org/0000-0001-7753-878X)
- Nada Jabado (ORCID: https://orcid.org/0000-0003-2485-3692)
- Michal Oren
- Karsten Nysom (ORCID: https://orcid.org/0000-0003-2935-0058)
- Sébastien Perreault (ORCID: https://orcid.org/0000-0002-4417-7698)
- Patricia Baxter (ORCID: https://orcid.org/0000-0003-3703-8742)
- Simon Bailey (ORCID: https://orcid.org/0000-0003-4763-4329)
- Jiaheng Qiu
- Sandya Govinda Raju
- Olaf Witt
Institutions
- Royal Children's Hospital (AU)
- Primary Children's Hospital (US)
- Children's Hospital of Philadelphia (US)
- Children's National (US)
- Duke University (US)
- The Kids Research Institute Australia (AU)
- The University of Western Australia (AU)
- German Cancer Research Center (DE)
- Baylor College of Medicine (US)
- University of Utah (US)
- Heidelberg University (DE)
- University of Michigan (US)
- Sheba Medical Center (IL)
- Montreal Children's Hospital (CA)
- Severance Hospital (KR)
- Great Ormond Street Hospital (GB)
- Princess Margaret Hospital for Children (AU)
- University Health System (US)
- Women's and Children's Hospital (AU)
- McGill University Health Centre (CA)
- Cancer Institute of New South Wales (AU)
- University Hospital Heidelberg (DE)
- Centre Hospitalier Universitaire Sainte-Justine (CA)
- Australian Institute of Business (AU)
- Copenhagen University Hospital (DK)
- Rigshospitalet (DK)
- UNSW Sydney (AU)
- Humboldt-Universität zu Berlin (DE)
- NYU Langone Health (US)
- Children’s Health Queensland Hospital and Health Service (AU)
- Center for Cancer and Blood Disorders (US)
- National Center for Tumor Diseases (DE)
- Hopp Children's Cancer Center Heidelberg (DE)
- NGM Biopharmaceuticals (United States) (US)
- Sydney Children's Hospital (AU)
- Princess Máxima Center (NL)
- South Australian Health and Medical Research Institute (AU)
- Great North Children's Hospital (GB)
- Centre for Cancer Biology (AU)
- Texas Children's Hospital (US)
- Sydney Children’s Hospitals Network (AU)
- Children's Cancer Center (US)
- C. S. Mott Children's Hospital (US)
- Duke University Hospital (US)
- Yonsei University Health System (KR)
- Dana-Farber/Boston Children's Cancer and Blood Disorders Center (US)
- Perth Children's Hospital
- Deutsches Konsortium für Translationale Krebsforschung (DE)
- University College London (GB)
- The University of Adelaide (AU)
- Université de Montréal (CA)
- Freie Universität Berlin (DE)
- Charité - Universitätsmedizin Berlin (DE)
- University of Pennsylvania (US)
- Newcastle University (GB)
- Philadelphia University (US)
Publication Details
- Journal
- Neuro-Oncology
- Published
- 2024-12-19
- DOI
- https://doi.org/10.1093/neuonc/noae274
- Citations
- 8
- Primary Topic
- Glioma Diagnosis and Treatment
- Type
- article
- Field-Weighted Citation Impact
- 3.50
Funders
- Children's Hospital of Philadelphia
- University of Pennsylvania
- Texas Children's Hospital
- McGill University
- Deutsches Krebsforschungszentrum
- Seattle Children's Research Institute
- McGill University Health Centre
- Children's National Hospital
- National Institute for Health and Care Research
- Department of Health and Social Care
- Newcastle University
- Cancer Institute NSW
- University of New South Wales
- Yonsei University
- Gentofte Hospital
- Great Ormond Street Hospital for Children
- Rigshospitalet
- Université de Montréal
- Region Hovedstaden
- Humboldt-Universität zu Berlin
- Freie Universität Berlin
- Deutschen Konsortium für Translationale Krebsforschung
- Universitätsklinikum Heidelberg
- Perelman School of Medicine, University of Pennsylvania
- Medical School, University of Michigan
- Medical Research Council
- National Health and Medical Research Council
- Great Ormond Street Institute of Child Health