{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,6]],"date-time":"2026-10-06T14:50:39Z","timestamp":1791298239005,"version":"4.1.0"},"reference-count":11,"publisher":"Journal of Neurosurgery Publishing Group (JNSPG)","issue":"3","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["PED"],"published-print":{"date-parts":[[2017,3]]},"abstract":"<jats:p>Brain tumors are the most common solid tumor in childhood, and astrocytomas account for the largest proportion of these tumors. Increasing sophistication in genetic testing has allowed for the detection of specific mutations within tumor subtypes that may represent targets for individualized tumor treatment. The mitogen-activating protein kinase (MAPK) pathway and, more specifically, <jats:italic>BRAF<\/jats:italic> mutations have been shown to be prevalent in pediatric pilocytic astrocytomas and may represent one such area to target. Herein, the authors describe 2 cases of inoperable, chemotherapy-resistant pediatric pilocytic astrocytomas with a documented response to trametinib, an MAPK pathway inhibitor. While these cases were not treated in the setting of a clinical trial, their data support further ongoing clinical trial investigation to evaluate the safety and efficacy of this agent in pediatric low-grade gliomas.<\/jats:p>","DOI":"10.3171\/2016.9.peds16328","type":"journal-article","created":{"date-parts":[[2016,12,23]],"date-time":"2016-12-23T15:02:03Z","timestamp":1482505323000},"page":"319-324","source":"Crossref","is-referenced-by-count":43,"title":["Report of effective trametinib therapy in 2 children with progressive hypothalamic optic pathway pilocytic astrocytoma: documentation of volumetric response"],"prefix":"10.3171","volume":"19","author":[{"given":"Catherine","family":"Miller","sequence":"first","affiliation":[{"name":"Departments of Neurosurgery,"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Daniel","family":"Guillaume","sequence":"additional","affiliation":[{"name":"Departments of Neurosurgery,"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kathryn","family":"Dusenbery","sequence":"additional","affiliation":[{"name":"Radiation Oncology,"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"H. Brent","family":"Clark","sequence":"additional","affiliation":[{"name":"Pathology, and"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Christopher","family":"Moertel","sequence":"additional","affiliation":[{"name":"Pediatric Hematology\/Oncology, University of Minnesota, Minneapolis, Minnesota"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"1754","reference":[{"key":"b1-peds16328","series-title":"Oncogene","doi-asserted-by":"publisher","first-page":"2019","DOI":"10.1038\/onc.2013.148","article-title":"The molecular and cell biology of pediatric low-grade gliomas","volume":"33","author":"Chen YH,","year":"2014","unstructured":"Chen YH, Gutmann DH: The molecular and cell biology of pediatric low-grade gliomas. 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Br J Cancer 101:722\u2013733, 200910.1038\/sj.bjc.6605179"},{"key":"b4-peds16328","series-title":"Cell Mol Life Sci","doi-asserted-by":"publisher","first-page":"1799","DOI":"10.1007\/s00018-011-0898-9","article-title":"MAPK pathway activation in pilocytic astrocytoma","volume":"69","author":"Jones DT,","year":"2012","unstructured":"Jones DT, Gronych J, Lichter P, Witt O, Pfister SM: MAPK pathway activation in pilocytic astrocytoma. Cell Mol Life Sci 69:1799\u20131811, 20122215958610.1007\/s00018-011-0898-9"},{"key":"b5-peds16328","series-title":"Neuro Oncol","doi-asserted-by":"publisher","first-page":"1408","DOI":"10.1093\/neuonc\/nou059","article-title":"Phase II study of sorafenib in children with recurrent or progressive low-grade astrocytomas","volume":"16","author":"Karajannis MA,","year":"2014","unstructured":"Karajannis MA, Legault G, Fisher MJ, Milla SS, Cohen KJ, Wisoff JH, : Phase II study of sorafenib in children with recurrent or progressive low-grade astrocytomas. 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