Modern treatment of craniopharyngioma to improve outcomes: evidence of a change of paradigm

Background: Craniopharyngiomas, rare primary brain tumors of the pituitary-hypothalamic axis, frequently result in sicubstantial morbidity, including compromised quality of life, vision impairment, hypothalamic and endocrine dysfunction, and neuroendocrine disturbances. Of particular importance is t...

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Detalles Bibliográficos
Autores: González-Gallego, Carlos, Molina, Pedro, Hostalot, Cristina, Oliva, Anna, Blanco, Alberto, Puyalto de Pablo, Paloma, Comas, Silvia, Carrato, Cristina, Valassi, Elena, Puig-Domingo, Manel
Tipo de recurso: artículo
Fecha de publicación:2025
País:España
Institución:Varias* (Consorci de Biblioteques Universitáries de Catalunya, Centre de Serveis Científics i Acadèmics de Catalunya)
Repositorio:Recercat. Dipósit de la Recerca de Catalunya
OAI Identifier:oai:recercat.cat:20.500.12328/5061
Acceso en línea:http://hdl.handle.net/20.500.12328/5061
https://dx.doi.org/10.1007/s12020-025-04216-9
Access Level:acceso abierto
Palabra clave:Inhibidors de BRAF-MEK
Craniofaringioma papil·lar
Craniofaringioma adamantinomatós
Mutació BRAF V600E
Wnt/beta-catenina
Tractament dirigit
Inhibidores de BRAF-MEK
Craneofaringioma papilar
Craneofaringioma adamantinomatoso
Mutación BRAF V600E
Tratamiento dirigido
BRAF-MEK inhibitors
Papillary craniopharyngioma
Adamantinomatous craniopharyngioma
BRAF V600E mutation
Wnt/beta-catenin
Targeted treatment
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Descripción
Sumario:Background: Craniopharyngiomas, rare primary brain tumors of the pituitary-hypothalamic axis, frequently result in sicubstantial morbidity, including compromised quality of life, vision impairment, hypothalamic and endocrine dysfunction, and neuroendocrine disturbances. Of particular importance is the development of hypothalamic obesity, which affects up to 25% of patients at diagnosis and increases to 50% after treatment. Genotyping has revealed that over 90% of papillary craniopharyngiomas (PCP) harbor BRAF V600E mutations. Recent studies have demonstrated a significant reduction in tumor size with the use of BRAF-MEK inhibitors in PCP. Methods: We conducted a systematic review of recent literature on pretreatment or neo-adjuvant medical therapies, analyzing their effectiveness, safety, and sequelae following surgical treatment with this new approach. Results: At the time of this review, 15 studies involving more than 50 patients have been published, with a response rate of up to 90%. Conclusion: Based on this evidence, we propose a new treatment paradigm aimed at improving outcomes by maximizing relief from compressive symptoms while minimizing hypothalamic dysfunction.