Successful treatment of a BRAF V600E-mutant extracranial metastatic anaplastic oligoastrocytoma with vemurafenib and everolimus

References

• Schindler G, Capper D, Meyer J, Janzarik W, Omran H, Herold-Mende C, Schmieder K, Wesseling P, Mawrin C, Hasselblatt M, et al. Analysis of BRAF V600E mutation in 1,320 nervous system tumors reveals high mutation frequencies in pleomorphic xanthoastrocytoma, ganglioglioma and extra-cerebellar pilocytic astrocytoma. Acta Neuropathol. 2011;121:397–405. doi:10.1007/s00401-011-0802-6.
   PubMed Web of Science ®Google Scholar
• Rosove MH, Peddi PF, Glaspy JA. BRAF V600E inhibition in anaplastic thyroid cancer. N Engl J Med. 2013;368:684–685. doi:10.1056/NEJMc1215697.
   PubMed Web of Science ®Google Scholar
• Wen PY, Lee EQ, Reardon DA, Ligon KL, Alfred Yung WK. Current clinical development of PI3K pathway inhibitors in glioblastoma. Neuro Oncol. 2012;14:819–829. doi:10.1093/neuonc/nos117.
   PubMed Web of Science ®Google Scholar
• Turner SG, Peters KB, Vredenburgh JJ, Desjardins A, Friedman HS, Reardon DA. Everolimus tablets for patients with subependymal giant cell astrocytoma. Expert Opin Pharmacother. 2011;12:2265–2269. doi:10.1517/14656566.2011.601742.
   PubMed Web of Science ®Google Scholar
• Flaherty KT, Puzanov I, Kim KB, Ribas A, McArthur GA, Sosman JA, O'Dwyer PJ, Lee RJ, Grippo JF, Nolop K, et al. Inhibition of mutated, activated BRAF in metastatic melanoma. N Engl J Med. 2010;363:809–819. doi:10.1056/NEJMoa1002011.
   PubMed Web of Science ®Google Scholar
• Flaherty KT, Infante JR, Daud A, Gonzalez R, Kefford RF, Sosman J, Hamid O, Schuchter L, Cebon J, Ibrahim N, etal. Combined BRAF and MEK inhibition in melanoma with BRAF V600 mutations. N Engl J Med. 2012;367:1694–1703. doi:10.1056/NEJMoa1210093.
   PubMed Web of Science ®Google Scholar
• Hauschild A, Grob JJ, Demidov LV, Jouary T, Gutzmer R, Millward M, Rutkowski P, Blank CU, Miller WH Jr, Kaempgen E, et al. Dabrafenib in BRAF-mutated metastatic melanoma: a multicentre, open-label, phase 3 randomised controlled trial. Lancet. 2012;380:358–365. doi:10.1016/S0140-6736(12)60868-X.
   PubMed Web of Science ®Google Scholar
• Robinson GW, Orr BA, Gajjar A. Complete clinical regression of a BRAF V600E-mutant pediatric glioblastoma multiforme after BRAF inhibitor therapy. BMC Cancer. 2014;14:258. doi:10.1186/1471-2407-14-258.
   PubMed Web of Science ®Google Scholar
• Rush S, Foreman N, Liu A. Brainstem ganglioglioma successfully treated with vemurafenib. J Clin Oncol. 2013;31:e159–160. doi:10.1200/JCO.2012.44.1568.
   PubMed Web of Science ®Google Scholar
• Lee EQ, Ruland S, Leboeuf NR, Wen PY, Santagata S. Successful treatment of a progressive BRAF V600E-mutated anaplastic pleomorphic xanthoastrocytoma with vemurafenib monotherapy. J Clin Oncol. 2016;34:e87–89. doi:10.1200/JCO.2013.51.1766.
   PubMed Web of Science ®Google Scholar
• Louis DN, Perry A, Reifenberger G, von DeimlingA, Figarella-Branger D, Cavenee WK, Ohgaki H, Wiestler OD, Kleihues P, Ellison DW. The 2016 World Health Organization classification of tumors of the central nervous system: a summary. Acta Neuropathol. 2016;131:803–820. doi:10.1007/s00401-016-1545-1.
   PubMed Web of Science ®Google Scholar
• Chakravarti A, Zhai G, Suzuki Y, Sarkesh S, Black PM, Muzikansky A, Loeffler JS. The prognostic significance of phosphatidylinositol 3-kinase pathway activation in human gliomas. J Clin Oncol. 2004;22:1926–1933. doi:10.1200/JCO.2004.07.193.
   PubMed Web of Science ®Google Scholar
• Sarkaria JN, Galanis E, Wu W, Peller PJ, Giannini C, Brown PD, Uhm JH, McGraw S, Jaeckle KA, Buckner JC. North central cancer treatment group phase I trial N057K of everolimus (RAD001) and temozolomide in combination with radiation therapy in patients with newly diagnosed glioblastoma multiforme. Int J Radiat Oncol Biol Phys. 2011;81:468–475. doi:10.1016/j.ijrobp.2010.05.064.
   PubMed Web of Science ®Google Scholar
• Mason WP, Macneil M, Kavan P, Easaw J, Macdonald D, Thiessen B, Urva S, Lwin Z, McIntosh L, Eisenhauer E. A phase I study of temozolomide and everolimus (RAD001) in patients with newly diagnosed and progressive glioblastoma either receiving or not receiving enzyme-inducing anticonvulsants: an NCIC CTG study. Invest New Drugs. 2012;30:2344–2351. doi:10.1007/s10637-011-9775-5.
   PubMed Web of Science ®Google Scholar
• Chinnaiyan P, Won M, Wen PY, Rojiani AM, Wendland M, Dipetrillo TA, Corn BW, Mehta MP. RTOG 0913: a phase 1 study of daily everolimus (RAD001) in combination with radiation therapy and temozolomide in patients with newly diagnosed glioblastoma. Int J Radiat Oncol Biol Phys. 2013;86:880–884. doi:10.1016/j.ijrobp.2013.04.036.
   PubMed Web of Science ®Google Scholar
• Hainsworth JD, Shih KC, Shepard GC, Tillinghast GW, Brinker BT, Spigel DR. Phase II study of concurrent radiation therapy, temozolomide, and bevacizumab followed by bevacizumab/everolimus as first-line treatment for patients with glioblastoma. Clin Adv Hematol Oncol. 2012;10:240–246.
   PubMedGoogle Scholar
• Solit DB, Garraway LA, Pratilas CA, Sawai A, Getz G, Basso A, Ye Q, Lobo JM, She Y, Osman I, et al. BRAF mutation predicts sensitivity to MEK inhibition. Nature. 2006 Jan 19;439(7074):358–362. Epub 2005 Nov 6. doi:10.1038/nature04304
   PubMed Web of Science ®Google Scholar
• Di Nicolantonio F, Arena S, Tabernero J, Grosso S, Molinari F, Macarulla T, Russo M, Cancelliere C,  Zecchin D, Mazzucchelli L, et al. Deregulation of the PI3K and KRAS signaling pathways in human cancer cells determines their response to everolimus. J Clin Invest. 2010 Aug;120(8):2858–2866. doi:10.1172/JCI37539.
   PubMed Web of Science ®Google Scholar
• Brennan CW, Verhaak RG, Mckenna A, Campos B, Noushmehr H, Salama SR, Zheng S, Chakravarty D, Sanborn JZ, Berman SH, et al. The Somatic Genomic Landscape of Glioblastoma. Cell. 2013;155:462–477. doi:10.1016/j.cell.2013.09.034.
   PubMed Web of Science ®Google Scholar
• Patel AP, Tirosh I, Trombetta JJ, Shalek AK, Gillespie SM, Wakimoto H, Cahill DP, Nahed BV, Curry WT, Martuza RL, et al. Single-cell RNA-seq highlights intratumoral heterogeneity in primary glioblastoma. Science. 2014;344:1396–1401. doi:10.1126/science.1254257.
   PubMed Web of Science ®Google Scholar
• Sottoriva A, Spiteri I, Piccirillo SG, Touloumis A, Collins VP, Marioni JC, Curtis C, Watts C, Tavaré S. Intratumor heterogeneity in human glioblastoma reflects cancer evolutionary dynamics. Proc Natl Acad Sci USA. 2013;110:4009–4014. doi:10.1073/pnas.1219747110.
   PubMed Web of Science ®Google Scholar
• Abou-El-Ardat K, Seifert M, Becker K, Eisenreich S, Lehmann M, Hackmann K, Rump A, Meijer G, Carvalho B, Temme A, et al. Comprehensive molecular characterization of multifocal glioblastoma proves its monoclonal origin and reveals novel insights into clonal evolution and heterogeneity of glioblastomas. Neuro Oncol. 2017;19:546–557. doi:10.1093/neuonc/now231.
   PubMed Web of Science ®Google Scholar