摘要：

颅咽管瘤属WHOⅠ级肿瘤，但是由于其位于鞍区，毗邻垂体柄、下丘脑等重要结构，亦有少部分肿瘤具有恶性生长特性，无论开颅手术或神经内镜手术常难以取得良好疗效，放射治疗仅对部分患者有效，且常合并垂体、视神经放射损伤等并发症，因此，颅咽管瘤的治疗仍是神经外科和内分泌科的重要挑战之一。目前颅咽管瘤的分子病理分型已取得一定进展，2016年WHO中枢神经系统肿瘤分类第四版修订版将其分为釉质表皮型和乳头型，两种分型在临床易发人群、病理组织学特征和遗传改变方面均有显著差异，这些差异既有助于明确颅咽管瘤的分子病理分型，更为其分子靶向药物治疗提供了理论基础。但尚待更多的转化研究和多中心临床药物试验，以研发更有效的分子靶向治疗药物。

关键词: 颅咽管瘤, 分子靶向治疗, 综述

Abstract:

Craniopharyngioma is a benign tumor of WHO grade Ⅰ. As the location of craniopharyngioma is deep, around which the peripheral structure is complex, the tumor adheres closely to the pituitary stalk and hypothalamus, and a small number of craniopharyngioma have the growth characteristics of malignant tumor, the total resection of craniopharyngioma is hard to achieve. Radiotherapy is only effective in some patients and is often associated with complications such as radiation damage to the pituitary and optic nerve. So, the treatment of craniopharyngioma is still challenging for neurosurgeons and endocrinologists. With the development of molecular biology technology, some new findings are made ragarding the pathological classification of craniopharyngioma and its mechanism. The revised 4th edition of 2016 WHO classification of central nervous system tumors classifies craniopharyngioma into adamantinomatous and papillary craniopharyngioma. There are significant differences in clinical susceptibility, histopathological characteristics and genetic changes between two types. These differences not only help to clarify molecular pathological classification of craniopharyngioma, but also provide a theoretical basis for its molecular targeted therapy. However, more transformational studies and multicenter clinical trials are needed to develop more effective molecular targeted therapy drugs.

Key words: Craniopharyngioma, Molecular targeted therapy, Review