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
{"title":"颅咽管瘤的药物治疗","authors":"Krystallenia I Alexandraki,&nbsp;Paraskevi Xekouki","doi":"10.17925/EE.2021.17.2.121","DOIUrl":null,"url":null,"abstract":"<p><p>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 <i>BRAF-V600E</i> 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.</p>","PeriodicalId":75231,"journal":{"name":"TouchREVIEWS in endocrinology","volume":"17 2","pages":"121-132"},"PeriodicalIF":0.0000,"publicationDate":"2021-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8676107/pdf/touchendo-17-121.pdf","citationCount":"5","resultStr":"{\"title\":\"Medical Therapy for Craniopharyngiomas.\",\"authors\":\"Krystallenia I Alexandraki,&nbsp;Paraskevi Xekouki\",\"doi\":\"10.17925/EE.2021.17.2.121\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><p>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 <i>BRAF-V600E</i> 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.</p>\",\"PeriodicalId\":75231,\"journal\":{\"name\":\"TouchREVIEWS in endocrinology\",\"volume\":\"17 2\",\"pages\":\"121-132\"},\"PeriodicalIF\":0.0000,\"publicationDate\":\"2021-11-01\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8676107/pdf/touchendo-17-121.pdf\",\"citationCount\":\"5\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"TouchREVIEWS in endocrinology\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"https://doi.org/10.17925/EE.2021.17.2.121\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"2021/11/8 0:00:00\",\"PubModel\":\"Epub\",\"JCR\":\"\",\"JCRName\":\"\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"TouchREVIEWS in endocrinology","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.17925/EE.2021.17.2.121","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2021/11/8 0:00:00","PubModel":"Epub","JCR":"","JCRName":"","Score":null,"Total":0}
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。

摘要图片

摘要图片

查看原文
分享 分享
微信好友 朋友圈 QQ好友 复制链接
本刊更多论文
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.

求助全文
通过发布文献求助,成功后即可免费获取论文全文。 去求助
来源期刊
CiteScore
2.40
自引率
0.00%
发文量
0
期刊最新文献
Amyloid Goiter: A Peruvian Case Series. Clinical and Biochemical Profiles of Hospitalized Patients with Hypercalcaemia from a Tertiary Care Centre in North India. Dry Eye in Diabetes: The Indian Diabetic and Endocrine Eye Diseases (INDEED) Review. Efficacy and Safety of Novel Twincretin Tirzepatide, a Dual GIP/GLP-1 Receptor Agonist, as an Anti-obesity Medicine in Individuals Without Diabetes: A Systematic Review and Meta-analysis. Metabolic Dysfunction-associated Steatotic Liver Disease and Type 2 Diabetes: A Deadly Synergy.
×
引用
GB/T 7714-2015
复制
MLA
复制
APA
复制
导出至
BibTeX EndNote RefMan NoteFirst NoteExpress
×
×
提示
您的信息不完整,为了账户安全,请先补充。
现在去补充
×
提示
您因"违规操作"
具体请查看互助需知
我知道了
×
提示
现在去查看 取消
×
提示
确定
0
微信
客服QQ
Book学术公众号 扫码关注我们
反馈
×
意见反馈
请填写您的意见或建议
请填写您的手机或邮箱
已复制链接
已复制链接
快去分享给好友吧!
我知道了
×
扫码分享
扫码分享
Book学术官方微信
Book学术文献互助
Book学术文献互助群
群 号:481959085
Book学术
文献互助 智能选刊 最新文献 互助须知 联系我们:info@booksci.cn
Book学术提供免费学术资源搜索服务,方便国内外学者检索中英文文献。致力于提供最便捷和优质的服务体验。
Copyright © 2023 Book学术 All rights reserved.
ghs 京公网安备 11010802042870号 京ICP备2023020795号-1