The Medical Therapy of Craniopharyngiomas: The Way Ahead.

Alexandraki, Krystallenia I, Kaltsas, Gregory A, Karavitaki, Niki and Grossman, Ashley B (2019) The Medical Therapy of Craniopharyngiomas: The Way Ahead. The Journal of clinical endocrinology and metabolism. ISSN 1945-7197.

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Craniopharyngiomas which are categorized as adamantinomatous (ACP) or papillary (PCP) have traditionally been treated with surgery and/or radiotherapy, although when the tumors progress or recur further therapeutic possibilities are very limited. Following recent advances in their molecular pathogenesis, new medical therapeutic options have emerged.


The search strategy that we selected to identify the appropriate evidence involved the following mesh terms: ("Craniopharyngioma"[Mesh] AND "Craniopharyngioma/drug therapy"[Mesh] NOT ("review"[Publication Type] OR "review literature as topic"[MeSH Terms] OR "review"[All Fields]) AND ("2009/05/01"[PDat]: "2019/04/28"[PDat]).


Mutations of β-catenin causing Wnt activation with alterations of the MEK/ERK pathway are encountered in the great majority of patients with ACP; specific alterations also stratify patients to a more aggressive behavior. In most PCP there is primary activation of the Ras/Raf/MEK/ERK pathway secondary to BRAF-V600E mutations. BRAF inhibitors, such as dabrafenib or vemurafenib, either alone or in combination with the MEK inhibitors trametinib and cobimetinib, have been administered to patients with PCP producing clinically useful and, in some cases, sustained responses. In contrast to PCP, drugs targeting β-catenin and its downstream MAPK pathway in ACP have so far only been used in in vitro studies, but appear to be promising new targets clinically.


The identification of specific genetic alterations in patients with craniopharyngiomas has expanded the therapeutic options, providing evidence for a a customized approach using newer molecular agents. More studies including a larger number of carefully selected patients are required to evaluate the response to currently available and evolving agents alone and in combination.

Item Type: Article
Subjects: WK Endocrine system. Endocrinology
WL Nervous system. Neurology
Divisions: Ambulatory Care
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Depositing User: Mrs Yolande Brookes
Date Deposited: 02 Aug 2019 14:51
Last Modified: 02 Aug 2019 14:51

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