Profound clinical and radiological response to BRAF inhibition in a 2-month-old diencephalic child with hypothalamic/chiasmatic glioma

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• 作者：Alvaro Lassaletta ; Ana Guerreiro Stucklin ; Vijay Ramaswamy ; Michal Zapotocky ; Tara McKeown ; Cynthia Hawkins ; Eric Bouffet and Uri Tabori
• 刊名：Pediatric Blood & Cancer
• 出版年：2016
• 出版时间：November 2016
• 年：2016
• 卷：63
• 期：11
• 页码：2038-2041
• 全文大小：308K
• ISSN：1545-5017

文摘

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.