[Recent Advances in the Treatment of Craniopharyngioma:A Breakthrough in Molecular Targeted Therapy].

Abstract

Craniopharyngiomas are benign sellar and parasellar tumors that present significant clinical challenge because of their close proximity to the optic apparatus, hypothalamus, and pituitary gland. Recent molecular studies have identified distinct driver mutations, namely, CTNNB1 in adamantinomatous craniopharyngiomas and BRAF V600E in papillary craniopharyngiomas, leading to a deeper understanding of tumor biology and treatment strategies. Advances in endoscopic endonasal transsphenoidal surgery have improved surgical safety and expanded the indications for minimally invasive resection, whereas radiotherapy remains an effective option for residual or recurrent disease. Molecular targeted therapy with BRAF and MEK inhibitors has resulted in remarkable tumor shrinkage in papillary craniopharyngiomas harboring the BRAF V600E mutation, and recent clinical studies have established this approach as a promising treatment option. These agents have emerged not only as effective treatment options for recurrent or refractory disease but also as promising options for neoadjuvant treatment and function-preserving management. Future treatment strategies are likely to incorporate molecular subtypes, patient age, and functional outcomes to achieve individualized care. In addition, the development of reliable preoperative molecular diagnostic techniques, including liquid biopsy, may further facilitate the integration of targeted therapies into routine clinical practice.