Long-Term Management and Clinical Trials in Adamantinomatous Craniopharyngioma

Müller, Hermann L.

doi:10.1007/978-3-319-51890-9_10

Basic Research and Clinical Aspects of Adamantinomatous Craniopharyngioma 2017

DOI: 10.1007/978-3-319-51890-9_10

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Long-Term Management and Clinical Trials in Adamantinomatous Craniopharyngioma

Hermann L. Müller

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Cited by 3 publications

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“…14 Overall, the current approved management of CP is far from ideal, since a number of complications can alter the quality of life of patients with CP. 10 Therefore, neoadjuvant treatment may be of great use in shrinking the initial lesion, allowing for a GTR and minimizing the postoperative complications.…”

mentioning

confidence: 99%

Medical Therapy for Craniopharyngiomas

Alexandraki

Xekouki

2021

touchREVIEWS in Endocrinology

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mentioning

confidence: 99%

Medical Therapy for Craniopharyngiomas

Alexandraki

Xekouki

2021

touchREVIEWS in Endocrinology

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Optic tract edema in craniopharyngioma as a predictor of BRAFV600E mutation presence

Taguchi

Kinoshita

Kojima

et al. 2023

Japanese Journal of Clinical Oncology

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Objective the advent of BRAF inhibitors for preoperative treatment of craniopharyngioma has necessitated the identification of BRAFV600E status. Hence, we investigated predictors of BRAFV600E mutation in craniopharyngiomas. Methods this retrospective study utilized data from 30 patients who were newly diagnosed with craniopharyngioma between 2011 and 2021. Magnetic resonance imaging (MRI) and computed tomography were performed within 1 week prior to surgery. Genetic analysis for BRAF mutation was performed using the Oncomine next-generation sequencing panel or Sanger sequencing. The relationship between BRAF mutation and demographic data, endocrinological function and tumour characteristics on imaging was assessed. Results tumour tissue carried the BRAFV600E mutation in nine patients. There was no significant difference in age, sex, or presence of hormonal dysfunction amongst patients with and without the BRAFV600E mutation in the tumour. Most tumours with the BRAFV600E mutation were histologically categorized as papillary craniopharyngioma (P = 0.0005), and were solid (P = 0.0002) and supra-diaphragmatic (P = 0.0033) on MRI. BRAFV600E tumours were more frequently associated with optic tract edema than wild-type tumour s (55.6 vs. 0%, P = 0.0009) and all tumour s with optic tract edema carried the BRAFV600E mutation. Optic tract edema was not associated with tumour volume, cysts, or preoperative pituitary function. Conclusions in craniopharyngiomas, the presence of optic tract edema can predict the presence of BRAFV600E mutation with a positive predictive value of 100%. The finding should be verified in larger prospective cohorts and multivariate regression analysis.

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The Medical Therapy of Craniopharyngiomas: The Way Ahead

Alexandraki

Kaltsas

Karavitaki

et al. 2019

The Journal of Clinical Endocrinology &Amp; Metabolism

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ContextCraniopharyngiomas, which are categorized as adamantinomatous (ACPs) or papillary (PCPs), have traditionally been treated with surgery and/or radiotherapy, although when the tumors progress or recur, therapeutic possibilities are very limited. Following recent advances in their molecular pathogenesis, new medical therapeutic options have emerged.Evidence AcquisitionThe search strategy that we selected to identify the appropriate evidence involved the following medical subject headings (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]).Evidence SynthesisMutations of β-catenin causing Wnt activation with alterations of the MEK/ERK pathway are encountered in the great majority of patients with ACPs; specific alterations also stratify patients to a more aggressive behavior. In most PCPs 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 PCPs producing clinically useful and, in some cases, sustained responses. In contrast to PCPs, drugs targeting β-catenin and its downstream MAPK pathway in ACPs have so far only been used in in vitro studies, but there appear to be promising new targets clinically.ConclusionsThe identification of specific genetic alterations in patients with craniopharyngiomas has expanded the therapeutic options, providing evidence for 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.

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Long-Term Management and Clinical Trials in Adamantinomatous Craniopharyngioma

Cited by 3 publications

References 206 publications

Medical Therapy for Craniopharyngiomas

Medical Therapy for Craniopharyngiomas

Optic tract edema in craniopharyngioma as a predictor of BRAFV600E mutation presence

The Medical Therapy of Craniopharyngiomas: The Way Ahead

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