Prolonged Response Induced by Single Agent Vemurafenib in a Spinal Ganglioglioma: A Case Report and Review of the Literature.
Fiche publication
Date publication
janvier 2019
Journal
Frontiers in oncology
Auteurs
Membres identifiés du Cancéropôle Est :
Dr CURTIT Elsa
Tous les auteurs :
Garnier L, Ducray F, Verlut C, Mihai MI, Cattin F, Petit A, Curtit E
Lien Pubmed
Résumé
Spinal ganglioglioma is a rare low-grade, slow-growing tumor of the central nervous system affecting mostly children and young adults. After surgery, some patients show tumor recurrence and/or malignant transformation. Gangliogliomas harbor molecular deficiencies such as mutations in the B-rapidly accelerated fibrosarcoma () gene, resulting in activation of a downstream signaling pathway and cancer development. Vemurafenib is a inhibitor used to treat patients with -mutated cancer. Although a few studies have reported the clinical responses in gangliogliomas, the sequence and duration of treatment have not been established. We describe a case of an adult with a progressive mutant spinal cord ganglioglioma 9 years after surgery who was treated with vemurafenib. This treatment resulted in a partial response within 2 months, which was sustained for more than a year. The patient then decided to stop treatment because of side effects. Despite this decision, the tumor showed no sign of progression 21 months after treatment discontinuation. This is the first reported case of a response to vemurafenib in an adult with progressive spinal cord -mutated ganglioglioma which was sustained after treatment discontinuation.
Mots clés
BRAF, central nervous system tumor, ganglioglioma, safety, spinal cord, tumor regression, vemurafenib
Référence
Front Oncol. 2019 ;9:177