Whelan Ros, Hengartner Astrid, Folzenlogen Zach, Prince Eric, Hankinson Todd C
Department of Neurosurgery, University of Colorado Hospital, Aurora, CO, USA.
Division of Pediatric neurosurgery, Children's Hospital Colorado, University of Colorado, Aurora, CO, USA.
Childs Nerv Syst. 2020 Aug;36(8):1635-1642. doi: 10.1007/s00381-020-04677-5. Epub 2020 May 22.
Pediatric adamantinomatous craniopharyngiomas (ACPs) are histologically benign brain tumors that often follow an aggressive clinical course. Arising in the sellar/suprasellar region, they grow in close proximity to critical neurological and vascular structures and can result in significant neuroendocrine morbidity. First-line treatment often involves surgical resection with or without radiotherapy and has been associated with significant morbidity and poor quality of life outcomes. As a result, the discovery of alternative effective and safe treatments is clearly desirable. In recent years, laboratory studies have harnessed sophisticated techniques to identify the upregulation of several markers that may represent potential therapeutic targets. These targets include IL-6, PD1/PD-L1, MEK, IDO-1, and others. Agents that target these pathways exist, and there is an opportunity to investigate their potential efficacy in the treatment of ACP. Trials investigating some of these agents as monotherapy and in combination for the treatment of pediatric ACP are underway or in development. If positive, these trials may result in a paradigm shift in treatment that will hopefully result in reduced morbidity and better outcomes for patients.
小儿造釉细胞瘤型颅咽管瘤（ACPs）是组织学上的良性脑肿瘤，但临床过程往往具有侵袭性。它们起源于鞍区/鞍上区域，生长在关键的神经和血管结构附近，可导致严重的神经内分泌功能障碍。一线治疗通常包括手术切除，可联合或不联合放疗，且与显著的发病率和不良的生活质量结果相关。因此，显然需要发现替代的有效且安全的治疗方法。近年来，实验室研究利用先进技术确定了几种可能代表潜在治疗靶点的标志物上调。这些靶点包括白细胞介素-6（IL-6）、程序性死亡蛋白1（PD1）/程序性死亡配体1（PD-L1）、丝裂原活化蛋白激酶/细胞外信号调节激酶（MEK）、吲哚胺2,3-双加氧酶1（IDO-1）等。针对这些途径的药物已经存在，有机会研究它们在治疗ACPs中的潜在疗效。正在进行或正在开展一些试验，研究这些药物作为单一疗法以及联合治疗小儿ACPs的效果。如果试验结果为阳性，这些试验可能会导致治疗模式的转变，有望降低发病率并为患者带来更好的治疗效果。
