title: Profound clinical and radiological response to BRAF inhibition in a 2-month-old diencephalic child with hypothalamic/chiasmatic glioma.
abstract: Infants with low-grade glioma (LGG) have a poor survival. BRAFV600E mutation has been identified in pediatric LGG; however, the use of BRAF inhibitors in infants has never been reported. A 2-month-old with V600E mutant hypothalamic/chiasmatic glioma progressed on chemotherapy resulting in profound visual loss, massive ascites, and diencephalic syndrome. Initiation of dabrafenib resulted in rapid and sustained disappearance of clinical symptoms and a profound sustained cytoreduction. BRAF inhibition was safely tolerated with dramatic clinicoradiological response, suggesting early targeted therapy is a viable option in infants with LGG. A re-evaluation of current management paradigms in this population is warranted to leverage the potential benefit of upfront-targeted therapies.
authors.affiliation: Division of Haematology/Oncology, The Hospital for Sick Children, Toronto, Ontario, Canada.|The Arthur and Sonia Labatt Brain Tumour Research Centre, The Hospital for Sick Children, Toronto, Ontario, Canada.
authors.forename: Alvaro
authors.initials: A
authors.lastname: Lassaletta
authors.affiliation: Division of Haematology/Oncology, The Hospital for Sick Children, Toronto, Ontario, Canada.|The Arthur and Sonia Labatt Brain Tumour Research Centre, The Hospital for Sick Children, Toronto, Ontario, Canada.|Developmental & Stem Cell Biology Program, The Hospital for Sick Children, Toronto, Ontario, Canada.
authors.forename: Ana
authors.initials: A
authors.lastname: Guerreiro Stucklin
authors.affiliation: Division of Haematology/Oncology, The Hospital for Sick Children, Toronto, Ontario, Canada.|The Arthur and Sonia Labatt Brain Tumour Research Centre, The Hospital for Sick Children, Toronto, Ontario, Canada.|Developmental & Stem Cell Biology Program, The Hospital for Sick Children, Toronto, Ontario, Canada.
authors.forename: Vijay
authors.initials: V
authors.lastname: Ramaswamy
authors.affiliation: Division of Haematology/Oncology, The Hospital for Sick Children, Toronto, Ontario, Canada.|The Arthur and Sonia Labatt Brain Tumour Research Centre, The Hospital for Sick Children, Toronto, Ontario, Canada.|Developmental & Stem Cell Biology Program, The Hospital for Sick Children, Toronto, Ontario, Canada.
authors.forename: Michal
authors.initials: M
authors.lastname: Zapotocky
authors.affiliation: Division of Haematology/Oncology, The Hospital for Sick Children, Toronto, Ontario, Canada.
authors.forename: Tara
authors.initials: T
authors.lastname: McKeown
authors.affiliation: The Arthur and Sonia Labatt Brain Tumour Research Centre, The Hospital for Sick Children, Toronto, Ontario, Canada.|Developmental & Stem Cell Biology Program, The Hospital for Sick Children, Toronto, Ontario, Canada.|Department of Laboratory Medicine and Pathobiology, University of Toronto, Toronto, Ontario, Canada.
authors.forename: Cynthia
authors.initials: C
authors.lastname: Hawkins
authors.affiliation: Division of Haematology/Oncology, The Hospital for Sick Children, Toronto, Ontario, Canada.|The Arthur and Sonia Labatt Brain Tumour Research Centre, The Hospital for Sick Children, Toronto, Ontario, Canada.|Developmental & Stem Cell Biology Program, The Hospital for Sick Children, Toronto, Ontario, Canada.
authors.forename: Eric
authors.initials: E
authors.lastname: Bouffet
authors.affiliation: Division of Haematology/Oncology, The Hospital for Sick Children, Toronto, Ontario, Canada. uri.tabori@sickkids.ca.|The Arthur and Sonia Labatt Brain Tumour Research Centre, The Hospital for Sick Children, Toronto, Ontario, Canada. uri.tabori@sickkids.ca.|Developmental & Stem Cell Biology Program, The Hospital for Sick Children, Toronto, Ontario, Canada. uri.tabori@sickkids.ca.
authors.forename: Uri
authors.initials: U
authors.lastname: Tabori
mesh_terms: D001932:Brain Neoplasms / Q000000981:diagnostic imaging / Q000188:drug therapy*; D005260:Female; D005910:Glioma / Q000000981:diagnostic imaging / Q000188:drug therapy*; D006801:Humans; D007027:Hypothalamic Diseases / Q000188:drug therapy*; D007223:Infant; D008279:Magnetic Resonance Imaging; D048493:Proto-Oncogene Proteins B-raf / Q000037:antagonists & inhibitors*
chemical_list: D048493:Proto-Oncogene Proteins B-raf
keywords: dabrafenib; glioma; hypothalamic-chiasmatic; infant; low grade