Targeted therapy in BRAF mutated aggressive papillary craniopharyngioma: a case report and overview of the literature

Karavitaki N, Cudlip S, Adams CB, Wass JA (2006) Craniopharyngiomas. Endocr Rev 27(4):371–397. https://doi.org/10.1210/er.2006-0002

Article  PubMed  Google Scholar 

Mortini P, Losa M, Pozzobon G, Barzaghi R, Riva M, Acerno S, Angius D, Weber G, Chiumello G, Giovanelli M (2011) Neurosurgical treatment of craniopharyngioma in adults and children: early and long-term results in a large case series. J Neurosurg 114(5):1350–1359. https://doi.org/10.3171/2010.11.JNS10670

Article  PubMed  Google Scholar 

Petito CK, De Girolami U, Earle K (1976) Craniopharyngiomas. A clinical and pathological review. Cancer 37(4):1944–1952. https://doi.org/10.1002/1097-0142(197604)37. (4<1944::aid-cncr2820370446>3.0.co;2-#)

Article  CAS  PubMed  Google Scholar 

Fernandez-Miranda JC, Gardner PA, Snyderman CH, Devaney KO, Strojan P, Suarez C, Genden EM, Rinaldo A, Ferlito A (2012) Craniopharyngioma: a pathologic, clinical, and surgical review. Head Neck 34(7):1036–1044. https://doi.org/10.1002/hed.21771

Article  PubMed  Google Scholar 

Weiner HL, Wisoff JH, Rosenberg ME, Kupersmith MJ, Cohen H, Zagzag D, Shiminski-Maher T, Flamm ES, Epstein FJ, Miller DC (1994) Craniopharyngiomas: a clinicopathological analysis of factors predictive of recurrence and functional outcome. Neurosurgery 35(6):1001–1010. https://doi.org/10.1227/00006123-199412000-00001. (discussion 1010-1011)

Article  CAS  PubMed  Google Scholar 

Mortini P, Gagliardi F, Boari N, Losa M (2013) Surgical strategies and modern therapeutic options in the treatment of craniopharyngiomas. Crit Rev Oncol Hematol 88(3):514–529. https://doi.org/10.1016/j.critrevonc.2013.07.013

Article  PubMed  Google Scholar 

Bülow B, Attewell R, Hagmar L, Malmström P, Nordström CH, Erfurth EM (1998) Postoperative prognosis in craniopharyngioma with respect to cardiovascular mortality, survival, and tumor recurrence. J Clin Endocrinol Metab 83(11):3897–3904. https://doi.org/10.1210/jcem.83.11.5240

Article  PubMed  Google Scholar 

Losa M, Pieri V, Bailo M, Gagliardi F, Barzaghi LR, Gioia L, Del Vecchio A, Bolognesi A, Mortini P (2018) Single fraction and multisession Gamma Knife radiosurgery for craniopharyngioma. Pituitary 21(5):499–506. https://doi.org/10.1007/s11102-018-0903-5

Article  PubMed  Google Scholar 

Brastianos PK, Taylor-Weiner A, Manley PE, Jones RT, Dias-Santagata D, Thorner AR, Lawrence MS, Rodriguez FJ, Bernardo LA, Schubert L, Sunkavalli A, Shillingford N, Calicchio ML, Lidov HG, Taha H, Martinez-Lage M, Santi M, Storm PB, Lee JY, Palmer JN, Adappa ND, Scott RM, Dunn IF, Laws ER Jr, Stewart C, Ligon KL, Hoang MP, Van Hummelen P, Hahn WC, Louis DN, Resnick AC, Kieran MW, Getz G, Santagata S (2014) Exome sequencing identifies BRAF mutations in papillary craniopharyngiomas. Nat Genet 46(2):161–165. https://doi.org/10.1038/ng.2868

Article  CAS  PubMed  PubMed Central  Google Scholar 

Alexandraki KI, Kaltsas GA, Karavitaki N, Grossman AB (2019) The medical therapy of craniopharyngiomas: the way ahead. J Clin Endocrinol Metab 104(12):5751–5764. https://doi.org/10.1210/jc.2019-01299

Article  PubMed  Google Scholar 

Brastianos PK, Shankar GM, Gill CM, Taylor-Weiner A, Nayyar N, Panka DJ, Sullivan RJ, Frederick DT, Abedalthagafi M, Jones PS, Dunn IF, Nahed BV, Romero JM, Louis DN, Getz G, Cahill DP, Santagata S, Curry WT Jr, Barker FG 2nd (2015) Dramatic response of BRAF V600E mutant papillary craniopharyngioma to targeted therapy. J Natl Cancer Inst 108(2):djv310. https://doi.org/10.1093/jnci/djv310

Article  CAS  PubMed  PubMed Central  Google Scholar 

Aylwin SJ, Bodi I, Beaney R (2016) Pronounced response of papillary craniopharyngioma to treatment with vemurafenib, a BRAF inhibitor. Pituitary 19(5):544–546. https://doi.org/10.1007/s11102-015-0663-4

Article  PubMed  Google Scholar 

Roque A, Odia Y (2017) BRAF-V600E mutant papillary craniopharyngioma dramatically responds to combination BRAF and MEK inhibitors. CNS Oncol 6(2):95–99. https://doi.org/10.2217/cns-2016-0034

Article  CAS  PubMed  PubMed Central  Google Scholar 

Rostami E, Witt Nyström P, Libard S, Wikström J, Casar-Borota O, Gudjonsson O (2017) Recurrent papillary craniopharyngioma with BRAFV600E mutation treated with neoadjuvant -targeted therapy. Acta Neurochir (Wien) 159(11):2217–2221. https://doi.org/10.1007/s00701-017-3311-0

Article  PubMed  Google Scholar 

Himes BT, Ruff MW, Van Gompel JJ, Park SS, Galanis E, Kaufmann TJ, Uhm JH (2018) Recurrent papillary craniopharyngioma withBRAF V600E mutation treated with dabrafenib: case report. J Neurosurg. https://doi.org/10.3171/2017.11.JNS172373

Article  PubMed  Google Scholar 

Juratli TA, Jones PS, Wang N, Subramanian M, Aylwin SJB, Odia Y, Rostami E, Gudjonsson O, Shaw BL, Cahill DP, Galanis E, Barker FG 2nd, Santagata S, Brastianos PK (2019) Targeted treatment of papillary craniopharyngiomas harboring BRAF V600E mutations. Cancer 125(17):2910–2914. https://doi.org/10.1002/cncr.32197

Article  PubMed  Google Scholar 

Rao M, Bhattacharjee M, Shepard S, Hsu S (2019) Newly diagnosed papillary craniopharyngioma with BRAF V600E mutation treated with single-agent selective BRAF inhibitor dabrafenib: a case report. Oncotarget 10(57):6038–6042. https://doi.org/10.18632/oncotarget.27203

Article  PubMed  PubMed Central  Google Scholar 

Bernstein A, Mrowczynski OD, Greene A, Ryan S, Chung C, Zacharia BE, Glantz M (2019) Dual BRAF/MEK therapy in BRAF V600E-mutated primary brain tumors: a case series showing dramatic clinical and radiographic responses and a reduction in cutaneous toxicity. J Neurosurg 144(6):1704–1709. https://doi.org/10.3171/2019.8.JNS19643

Article  Google Scholar 

Di Stefano AL, Guyon D, Sejean K, Feuvret L, Villa C, Berzero G, Desforges Bullet V, Halimi E, Boulin A, Baussart B, Gaillard S (2020) Medical debulking with BRAF/MEK inhibitors in aggressive BRAF-mutant craniopharyngioma. Neurooncol Adv 2(1):vdaa141. https://doi.org/10.1093/noajnl/vdaa141

Article  PubMed  PubMed Central  Google Scholar 

Khaddour K, Chicoine MR, Huang J, Dahiya S, Ansstas G (2020) Successful use of BRAF/MEK inhibitors as a neoadjuvant approach in the definitive treatment of papillary craniopharyngioma. J Natl Compr Canc Netw 18(12):1590–1595. https://doi.org/10.6004/jnccn.2020.7624

Article  CAS  PubMed  Google Scholar 

Chik CL, van Landeghem FKH, Easaw JC, Mehta V (2021) Aggressive childhood-onset papillary craniopharyngioma managed with Vemurafenib, a BRAF inhibitor. J Endocr Soc 5(5):bvab043. https://doi.org/10.1210/jendso/bvab043

Article  PubMed  PubMed Central  Google Scholar 

Fasano M, Della Corte CM, Caterino M, Pirozzi M, Rauso R, Troiani T, Martini G, Napolitano S, Morgillo F, Ciardiello F (2022) Dramatic therapeutic response to dabrafenib plus trametinib in BRAF V600E mutated papillary craniopharyngiomas: a case report and literature review. Front Med (Lausanne) 8:652005. https://doi.org/10.3389/fmed.2021.652005

Article  PubMed  Google Scholar 

Nussbaum PE, Nussbaum LA, Torok CM, Patel PD, Yesavage TA, Nussbaum ES (2022) Case report and literature review of BRAF-V600 inhibitors for treatment of papillary craniopharyngiomas: a potential treatment paradigm shift. J Clin Pharm Ther. https://doi.org/10.1111/jcpt.13600

Article  PubMed  Google Scholar 

Calvanese F, Jacquesson T, Manet R, Vasiljevic A, Lasolle H, Ducray F, Raverot G, Jouanneau E (2022) Neoadjuvant B-RAF and MEK inhibitor targeted therapy for adult papillary craniopharyngiomas: a new treatment paradigm. Front Endocrinol (Lausanne) 13:882381. https://doi.org/10.3389/fendo.2022.882381

Article  PubMed  Google Scholar 

Brastianos PK, Twohy E, Geyer S, Gerstner ER, Kaufmann TJ, Tabrizi S, Kabat B, Thierauf J, Ruff MW, Bota DA, Reardon DA, Cohen AL, De La Fuente MI, Lesser GJ, Campian J, Agarwalla PK, Kumthekar P, Mann B, Vora S, Knopp M, Iafrate AJ, Curry WT Jr, Cahill DP, Shih HA, Brown PD, Santagata S, Barker FG 2nd, Galanis E (2023) BRAF-MEK inhibition in newly diagnosed papillary craniopharyngiomas. N Engl J Med 389(2):118–126. https://doi.org/10.1056/NEJMoa2213329

Article  CAS  PubMed  PubMed Central  Google Scholar 

Welsh SJ, Corrie PG (2015) Management of BRAF and MEK inhibitor toxicities in patients with metastatic melanoma. Ther Adv Med Oncol 7(2):122–136. https://doi.org/10.1177/1758834014566428

Article  CAS  PubMed  PubMed Central  Google Scholar 

Heinzerling L, Eigentler TK, Fluck M, Hassel JC, Heller-Schenck D, Leipe J, Pauschinger M, Vogel A, Zimmer L, Gutzmer R (2019) Tolerability of BRAF/MEK inhibitor combinations: adverse event evaluation and management. ESMO Open 4(3):e000491. https://doi.org/10.1136/esmoopen-2019-000491

Article  PubMed  PubMed Central  Google Scholar 

Comments (0)

No login
gif