title: Successful Retreatment of a Child with a Refractory Brainstem Ganglioglioma with Vemurafenib.
abstract: A child with brainstem ganglioglioma underwent subtotal resection and focal radiation. Magnetic resonance imaging confirmed tumor progression 6 months later. Another partial resection revealed viable BRAF V600E-positive residual tumor. Vemurafenib (660 mg/m(2) /dose) was administered twice daily, resulting in >70% tumor reduction with sustained clinical improvement for 1 year. Vemurafenib was then terminated, but significant tumor progression occurred 3 months later. Vemurafenib was restarted, resulting in partial response. Toxicities included Grade I pruritus and Grade II rash. Vemurafenib was effectively crushed and administered in solution via nasogastric tube. We demonstrate benefit from restarting vemurafenib therapy.
authors.affiliation: Aflac Cancer & Blood Disorders Center, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, Georgia.
authors.forename: Dolly
authors.initials: D
authors.lastname: Aguilera
authors.affiliation: Aflac Cancer & Blood Disorders Center, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, Georgia.
authors.forename: Anna
authors.initials: A
authors.lastname: Janss
authors.affiliation: Aflac Cancer & Blood Disorders Center, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, Georgia.
authors.forename: Claire
authors.initials: C
authors.lastname: Mazewski
authors.affiliation: Aflac Cancer & Blood Disorders Center, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, Georgia.
authors.forename: Robert Craig
authors.initials: RC
authors.lastname: Castellino
authors.affiliation: Department of Pathology, Emory University School of Medicine, Atlanta, Georgia.
authors.forename: Matthew
authors.initials: M
authors.lastname: Schniederjan
authors.affiliation: Department of Radiology, Children's HealthCare of Atlanta at Scottish Rite, Atlanta, Georgia.
authors.forename: Laura
authors.initials: L
authors.lastname: Hayes
authors.affiliation: Department of Neurosurgery, Children's Health Care of Atlanta, Emory University, School of Medicine, Atlanta, Georgia.
authors.forename: Barunashish
authors.initials: B
authors.lastname: Brahma
authors.affiliation: Aflac Cancer & Blood Disorders Center, Children's Healthcare of Atlanta, Atlanta, Georgia.
authors.forename: Lauren
authors.initials: L
authors.lastname: Fogelgren
authors.affiliation: Aflac Cancer & Blood Disorders Center, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, Georgia.
authors.forename: Tobey J
authors.initials: TJ
authors.lastname: MacDonald
mesh_terms: D001932:Brain Neoplasms / Q000188:drug therapy* / Q000601:surgery; D001933:Brain Stem; D002648:Child; D018303:Ganglioglioma / Q000188:drug therapy* / Q000601:surgery; D006801:Humans; D007211:Indoles / Q000008:administration & dosage / Q000627:therapeutic use*; D007441:Intubation, Gastrointestinal; D008297:Male; D013449:Sulfonamides / Q000008:administration & dosage / Q000627:therapeutic use*; D000077484:Vemurafenib
chemical_list: D007211:Indoles; D013449:Sulfonamides; D000077484:Vemurafenib
keywords: BRAF V600E; ganglioglioma; vemurafenib