Thomas Sonu, Bikeyeva Viktoriya, Abdullah Ahmed, Radivojevic Aleksandra, Abu Jad Anas A, Ravanavena Anvesh, Ravindra Chetna, Igweonu-Nwakile Emmanuelar O, Ali Safina, Paul Salomi, Yakkali Shreyas, Teresa Selvin Sneha, Hamid Pousette
Medicine, California Institute of Behavioral Neurosciences & Psychology, Fairfield, USA.
Internal Medicine, California Institute of Behavioral Neurosciences & Psychology, Fairfield, USA.
Cureus. 2022 Aug 13;14(8):e27963. doi: 10.7759/cureus.27963. eCollection 2022 Aug.
As oncology practice is rapidly shifting away from toxic chemotherapy, gene therapy provides a highly specific therapeutic approach for brain tumors. In this systematic review, we investigate gene therapy's status in pediatric brain tumors and future recommendations. The search was conducted systematically using PubMed, Cochrane, Google Scholar, and ClinicalTrials.gov databases. The field search used in the process was selected based on the keywords and Medical Subject Headings (MeSH), depending on the database used. We included cases of neurofibromatosis type 1 (NF1) brain tumors in all age groups with the additional inclusion of English language, free full text, articles published within the last 20 years, randomized controlled trials (RCTs), observational studies, systematic reviews, and meta-analyses. We excluded case reports, case studies, and editorials. The search identified a total of 1,213 articles from the databases. We included 19 studies with 16 narrative reviews, one systematic review, and two randomized clinical trials with 43 patients. After reviewing all data in the articles, we found that gene therapy can improve standard treatment efficacy when used as adjuvant therapy. It can be used to overcome barriers such as chemotherapy resistance by downregulating resistance genes. It is associated with mild toxicity when compared with other available treatment options, but given the overall poor prognosis in pediatric brain tumors, further studies are warranted.
随着肿瘤学实践正迅速从毒性化疗转向其他治疗方法，基因治疗为脑肿瘤提供了一种高度特异性的治疗途径。在这项系统评价中，我们调查了基因治疗在小儿脑肿瘤中的现状以及未来的建议。我们使用PubMed、Cochrane、谷歌学术和ClinicalTrials.gov数据库进行了系统检索。在检索过程中使用的字段搜索是根据关键词和医学主题词（MeSH）来选择的，具体取决于所使用的数据库。我们纳入了所有年龄组的1型神经纤维瘤病（NF1）脑肿瘤病例，另外还纳入了英文、免费全文、过去20年内发表的文章、随机对照试验（RCT）、观察性研究、系统评价和荟萃分析。我们排除了病例报告、病例研究和社论。检索共从数据库中识别出1213篇文章。我们纳入了19项研究，其中包括16篇叙述性综述、1篇系统评价和2项随机临床试验，涉及43名患者。在审查了文章中的所有数据后，我们发现基因治疗作为辅助治疗时可以提高标准治疗的疗效。它可以通过下调耐药基因来克服诸如化疗耐药等障碍。与其他可用的治疗选择相比，它的毒性较轻，但鉴于小儿脑肿瘤的总体预后较差，仍需要进一步研究。
