The type II RAF inhibitor tovorafenib in relapsed/refractory pediatric low-grade glioma: the phase 2 FIREFLY-1 trial.

Journal

Nature medicine
ISSN: 1546-170X
Titre abrégé: Nat Med
Pays: United States
ID NLM: 9502015

Informations de publication

Date de publication:
17 Nov 2023
Historique:
received: 15 09 2023
accepted: 25 10 2023
pubmed: 18 11 2023
medline: 18 11 2023
entrez: 18 11 2023
Statut: aheadofprint

Résumé

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

Identifiants

DOI: 10.1038/s41591-023-02668-y PMID: 37978284
pubmed: 37978284
doi: 10.1038/s41591-023-02668-y
pii: 10.1038/s41591-023-02668-y
doi:

Banques de données

ClinicalTrials.gov
['NCT04775485']

Types de publication

Journal Article

Langues

eng

Sous-ensembles de citation

IM

Informations de copyright

© 2023. The Author(s).

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Auteurs

Lindsay B Kilburn (LB)

Children's National Hospital, Washington, DC, USA. lkilburn@childrensnational.org.
ORCID: 0000-0003-1478-3619

Dong-Anh Khuong-Quang (DA)

Children's Cancer Centre, Royal Children's Hospital Melbourne, Melbourne, Victoria, Australia.
ORCID: 0000-0001-6305-7790

Jordan R Hansford (JR)

Michael Rice Centre for Hematology and Oncology, Women's and Children's Hospital, Adelaide, South Australia, Australia.
South Australia Health and Medical Research Institute, Adelaide, Australia; South Australian Immunogenomics Cancer Institute, University of Adelaide, Adelaide, South Australia, Australia.
ORCID: 0000-0001-7733-383X

Daniel Landi (D)

Duke University, Durham, NC, USA.

Jasper van der Lugt (J)

Princess Máxima Center for Pediatric Oncology, Utrecht, The Netherlands.
ORCID: 0000-0002-8186-338X

Sarah E S Leary (SES)

Cancer and Blood Disorders Center, Seattle Children's, Seattle, WA, USA.
ORCID: 0000-0003-0225-6184

Pablo Hernáiz Driever (PH)

Charité 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 Bailey (S)

Great North Children's Hospital and Newcastle University Centre for Cancer, Newcastle-upon-Tyne, UK.
ORCID: 0000-0003-4763-4329

Sébastien Perreault (S)

CHU Sainte-Justine, Université de Montréal, Montréal, Quebec, Canada.

Geoffrey McCowage (G)

Sydney Children's Hospitals Network, Westmead, New South Wales, Australia.

Angela J Waanders (AJ)

Ann & Robert H. Lurie Children's Hospital, Chicago, IL, USA.

David S Ziegler (DS)

Kids Cancer Centre, Sydney Children's Hospital, Randwick, New South Wales, Australia.
Children's Cancer Institute, Lowy Cancer Research Centre, University of New South Wales, Sydney, New South Wales, Australia.
School of Clinical Medicine, University of New South Wales, Sydney, New South Wales, Australia.
ORCID: 0000-0001-7451-7916

Olaf Witt (O)

Hopp Children's Cancer Center Heidelberg (KiTZ), Heidelberg, Germany.
Clinical Cooperation Unit, Pediatric Oncology, German Cancer Research Center (DKFZ), Heidelberg, Germany.
Department of Pediatric Oncology, Hematology, Immunology and Pulmonology, Heidelberg University Hospital, Heidelberg, Germany.
German Cancer Consortium (DKTK), Heidelberg, Germany.
National Center for Tumor Diseases (NCT), Heidelberg, Germany.

Patricia A Baxter (PA)

Texas Children's Cancer Center, Texas Children's Hospital, Baylor College of Medicine, Houston, TX, USA.

Hyoung Jin Kang (HJ)

Department 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 Hassall (TE)

Children's Health Queensland Hospital and Health Service, South Brisbane, QLD, Australia.

Jung Woo Han (JW)

Severance Hospital, Yonsei University Health System, Seoul, Republic of Korea.
ORCID: 0000-0001-8936-1205

Darren Hargrave (D)

UCL Great Ormond Street Institute of Child Health and Great Ormond Street Hospital for Children, London, UK.
ORCID: 0000-0001-8219-9807

Andrea T Franson (AT)

C.S. Mott Children's Hospital, University of Michigan Medical School, Ann Arbor, MI, USA.
ORCID: 0000-0002-5361-7683

Michal Yalon Oren (M)

Pediatric Hemato-Oncology, Sheba Medical Center, Ramat Gan, Israel.

Helen Toledano (H)

Department of Pediatric Oncology, Schneider Children's Medical Center, Petach Tikva, and Faculty of Medicine, Tel Aviv University, Tel Aviv, Israel.

Valérie Larouche (V)

Department of Pediatrics, Centre Mère-Enfant Soleil du CHU de Québec-Université Laval, Quebec City, Quebec, Canada.
ORCID: 0000-0003-4082-7267

Cassie Kline (C)

Division 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 Abdelbaki (MS)

Division of Hematology and Oncology, Department of Pediatrics, School of Medicine, Washington University, St. Louis, MO, USA.

Nada Jabado (N)

McGill University Health Centre (MUHC), Montreal Children's Hospital (MCH), Montreal, Quebec, Canada.
ORCID: 0000-0003-2485-3692

Nicholas G Gottardo (NG)

Department 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 Gerber (NU)

Department of Oncology, University Children's Hospital, Zurich, Switzerland.
ORCID: 0000-0002-1783-631X

Nicholas S Whipple (NS)

Primary Children's Hospital and University of Utah, Salt Lake City, UT, USA.

Devorah Segal (D)

NYU Langone Health, New York, NY, USA.
ORCID: 0000-0002-9740-1286

Susan N Chi (SN)

Pediatric Neuro-Oncology, Department of Pediatrics, Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Boston, MA, USA.

Liat Oren (L)

Department of Hematology & Oncology, Rambam Healthcare Campus, Haifa, Israel.

Enrica E K Tan (EEK)

Haematology/Oncology Service, KK Women's and Children's Hospital, Singapore, Singapore.

Sabine Mueller (S)

Department of Neurology, Neurosurgery and Pediatrics, University of California, San Francisco, San Francisco, CA, USA.
ORCID: 0000-0002-8216-9357

Izzy Cornelio (I)

Day One Biopharmaceuticals, Brisbane, CA, USA.

Lisa McLeod (L)

Day One Biopharmaceuticals, Brisbane, CA, USA.

Xin Zhao (X)

Day One Biopharmaceuticals, Brisbane, CA, USA.

Ashley Walter (A)

Day One Biopharmaceuticals, Brisbane, CA, USA.

Daniel Da Costa (D)

Day One Biopharmaceuticals, Brisbane, CA, USA.

Peter Manley (P)

Day One Biopharmaceuticals, Brisbane, CA, USA.

Samuel C Blackman (SC)

Day One Biopharmaceuticals, Brisbane, CA, USA.

Roger J Packer (RJ)

Division of Neurology, Brain Tumor Institute, Center for Neuroscience and Behavioral Medicine, Children's National Hospital, Washington, DC, USA.

Karsten Nysom (K)

Department of Pediatrics and Adolescent Medicine, Copenhagen University Hospital - Rigshospitalet, Copenhagen, Denmark.
ORCID: 0000-0003-2935-0058

Classifications MeSH