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J Natl Cancer Inst. 2015 Oct 23;108(2). pii: djv310. doi: 10.1093/jnci/djv310. Print 2016 Feb.

Dramatic Response of BRAF V600E Mutant Papillary Craniopharyngioma to Targeted Therapy.

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Department of Medicine (PKB, RS), Department of Neurology (PKB, CMG), Department of Neurosurgery (GMS, PJ, BN, DPC, WTC, FGB), Department of Surgical Oncology (DTF), Department of Pathology (GG, DNL), Cancer Center (PKB, CMG, NN, DNL), Department of Radiology (JR) Massachusetts General Hospital, Harvard Medical School, Boston, MA; Broad Institute (ATW, GG), Department of Pathology, (MA, SS) and Department of Neurosurgery, Brigham and Women's Hospital (IFD), Boston, MA; Department of Medicine, Beth Israel Deaconess Medical Center, Harvard Medical School, Boston, MA (DJP).


We recently reported that BRAF V600E is the principal oncogenic driver of papillary craniopharyngioma, a highly morbid intracranial tumor commonly refractory to treatment. Here, we describe our treatment of a man age 39 years with multiply recurrent BRAF V600E craniopharyngioma using dabrafenib (150mg, orally twice daily) and trametinib (2mg, orally twice daily). After 35 days of treatment, tumor volume was reduced by 85%. Mutations that commonly mediate resistance to MAPK pathway inhibition were not detected in a post-treatment sample by whole exome sequencing. A blood-based BRAF V600E assay detected circulating BRAF V600E in the patient's blood. Re-evaluation of the existing management paradigms for craniopharyngioma is warranted, as patient morbidity might be reduced by noninvasive mutation testing and neoadjuvant-targeted treatment.

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