颅咽管瘤的药物治疗

TouchREVIEWS in endocrinology Pub Date : 2021-11-01 Epub Date: 2021-11-08 DOI:10.17925/EE.2021.17.2.121
Krystallenia I Alexandraki, Paraskevi Xekouki
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引用次数: 5

摘要

颅咽管瘤是一种罕见的良性肿瘤,表现为两种不同的类型,金刚腺瘤(ACP)和乳头状瘤(PCP),它们在分子和临床上是不同的。传统的治疗方法包括手术切除和放射治疗,由于肿瘤靠近重要的大脑结构,这伴随着许多使人衰弱的并发症。颅咽管瘤分子发病机制的最新进展为医学治疗选择开辟了视野。acp的主要特征是β-catenin突变,其激活无翼/Int (Wnt),改变丝裂原细胞外激酶(MEK)/细胞外信号调节激酶(ERK)通路,以及炎症、细胞衰老、细胞程序性死亡和sonic hedgehog (SHH)通路。pcp主要以BRAF-V600E突变继发的Ras/Raf/MEK/ERK通路激活为特征。MEK抑制剂(如binimetinib)或抗炎介质(如tocilizumab或干扰素)已被用于ACP患者,且大多数疗效良好。另一方面,BRAF抑制剂,如dabrafenib或vemurafenib,单独使用或与MEK抑制剂trametinib和cobimetinib联合使用,已被用于PCP患者,并产生良好的反应。一些正在进行的试验将揭示新的分子靶向治疗的方案、剂量、联合治疗和安全性问题,通过启动这些罕见肿瘤的个性化医疗时代,改变颅咽管瘤患者的管理。我们进行了一项系统综述,以确定目前接受全身药物治疗的患者的病例系列或病例报告。
本文章由计算机程序翻译,如有差异,请以英文原文为准。

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Medical Therapy for Craniopharyngiomas.

Craniopharyngiomas are rare benign neoplasms presenting in two different types, adamantinomatous (ACP) or papillary (PCP), which are molecularly and clinically distinct. Traditional treatment includes surgical resection and radiotherapy, which are accompanied by a number of debilitating complications because of the tumours' proximity to important brain structures. Recent advances in the understanding of molecular pathogenesis of craniopharyngiomas have opened horizons to medical therapeutic options. ACPs are mainly characterized by mutations of β-catenin, which activate Wingless/Int (Wnt), and alter the mitogen extracellular kinase (MEK)/extracellular signal-regulated kinase (ERK) pathway, as well as inflammatory, cellular senescence, programmed cell death and sonic hedgehog (SHH) pathways. PCPs are mainly characterized by Ras/Raf/MEK/ERK pathway activation secondary to BRAF-V600E mutations. MEK inhibitors, such as binimetinib, or anti-inflammatory mediators, such as tocilizumab or interferon, have been administered to patients with ACP and the efficacy is mostly favourable. On the other hand, 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 PCP resulting in favourable responses. A number of ongoing trials will shed light on schemes, doses, combined treatments and safety issues of the new molecular-targeted treatments, changing the management of patients with craniopharyngiomas by launching the era of personalized medicine in these rare neoplasms. We conducted a systematic review to identify case series or case reports with patients currently treated with systemic medical therapy.

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