Pediatric Diffuse Midline Gliomas: An Unfinished Puzzle.
Pediatric Diffuse Midline Gliomas: An Unfinished Puzzle.
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DOI：
10.3390/diagnostics12092064
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发表时间：
2022-08-25
期刊：
影响因子：
3.6
通讯作者：
Mastronuzzi, Angela
中科院分区：
文献类型：
--
作者：
Di Ruscio, Valentina;Del Baldo, Giada;Fabozzi, Francesco;Vinci, Maria;Cacchione, Antonella;de Billy, Emmanuel;Megaro, Giacomina;Carai, Andrea;Mastronuzzi, Angela
关键词：
弥漫性中线神经胶质瘤（DMG）是一组异质性的侵袭性小儿脑肿瘤，具有致命的预后。大部分病例的生物学标志是H3 K27变异。预后仍然很差，从诊断开始的中位生存期为9至12个月。临床和影像学预后因素仅部分改变无进展生存期，但不能改善总生存期。尽管做出了努力，但目前还没有DMG的治愈性疗法。放射治疗仍然是标准治疗，只有短暂的好处。没有发现化疗方案能显著改善预后。在组织学和分子学研究结果更深入整合的新时代，目前正在研究潜在的新方法。整个国际科学界都在试图从不同方面瞄准DMG。治疗策略涉及靶向表观遗传改变，如甲基化和乙酰化状态，以及确定调节致癌增殖的新分子途径;免疫治疗方法也是肿瘤学领域研究的一个有趣点，目前已在几项临床试验中评估了驱动免疫系统对抗肿瘤细胞的可能性，并取得了有希望的初步结果。此外，由于纳米技术的改进，开发创新的递送方法来跨越敌对的肿瘤微环境和几乎完整的血脑屏障可能会改变肿瘤对不同治疗的反应。在这篇综述中，我们提供了一个全面的概述，现有的和潜在的新的治疗方法，是在世界范围内的调查，目的是患者和肿瘤特异性治疗可能会改变这种疾病的生物不祥的历史。
Diffuse midline glioma (DMG) is a heterogeneous group of aggressive pediatric brain tumors with a fatal prognosis. The biological hallmark in the major part of the cases is H3K27 alteration. Prognosis remains poor, with median survival ranging from 9 to 12 months from diagnosis. Clinical and radiological prognostic factors only partially change the progression-free survival but they do not improve the overall survival. Despite efforts, there is currently no curative therapy for DMG. Radiotherapy remains the standard treatment with only transitory benefits. No chemotherapeutic regimens were found to significantly improve the prognosis. In the new era of a deeper integration between histological and molecular findings, potential new approaches are currently under investigation. The entire international scientific community is trying to target DMG on different aspects. The therapeutic strategies involve targeting epigenetic alterations, such as methylation and acetylation status, as well as identifying new molecular pathways that regulate oncogenic proliferation; immunotherapy approaches too are an interesting point of research in the oncology field, and the possibility of driving the immune system against tumor cells has currently been evaluated in several clinical trials, with promising preliminary results. Moreover, thanks to nanotechnology amelioration, the development of innovative delivery approaches to overcross a hostile tumor microenvironment and an almost intact blood–brain barrier could potentially change tumor responses to different treatments. In this review, we provide a comprehensive overview of available and potential new treatments that are worldwide under investigation, with the intent that patient- and tumor-specific treatment could change the biological inauspicious history of this disease.
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影响因子：
12.7
作者：
Buczkowicz P;Bartels U;Bouffet E;Becher O;Hawkins C
通讯作者：
Hawkins C
影响因子：
8.4
作者：
Bailey, S.;Howman, A.;Hargrave, D.
通讯作者：
Hargrave, D.
影响因子：
2.7
作者：
Cacciotti, Chantel;Liu, Kevin X.;Warren, Katherine E.
通讯作者：
Warren, Katherine E.
影响因子：
4.7
作者：
Buczkowicz P;Hawkins C
通讯作者：
Hawkins C
DOI：
10.1016/j.jconrel.2018.05.005
发表时间：
2018-07-10
期刊：
Journal of controlled release : official journal of the Controlled Release Society
影响因子：
--
作者：
Alli S;Figueiredo CA;Golbourn B;Sabha N;Wu MY;Bondoc A;Luck A;Coluccia D;Maslink C;Smith C;Wurdak H;Hynynen K;O'Reilly M;Rutka JT
通讯作者：
Rutka JT