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

Abstract

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.

Keywords: Adamantinomatous craniopharyngioma; BRAF V600E mutation; BRAF-MEK inhibitors; Papillary craniopharyngioma; Targeted treatment; Wnt/beta-catenin.

Fig. 1

Proposed ACP multimodal treatment approach through multidisciplinary committee assessment. NED non evident disease, PR partial response, R response, ACP adamantinomatous Craniopharyngioma, PCP papillary Craniopharyngioma

Fig. 2

Proposed PCP multimodal treatment approach through multidisciplinary committee assessment. NED non evident disease, PR partial response, R response, ACP adamantinomatous Craniopharyngioma PCP papillary Craniopharyngioma