Karam Salma, Gebreil Ahmad, Alksas Ahmed, Balaha Hossam Magdy, Khalil Ashraf, Ghazal Mohammed, Contractor Sohail, El-Baz Ayman
Bioengineering Department, University of Louisville, Louisville, KY 40292, USA.
College of Technological Innovation, Zayed University, Abu Dhabi 4783, United Arab Emirates.
Biomedicines. 2024 Jun 30;12(7):1455. doi: 10.3390/biomedicines12071455.
Wilms tumor (WT), or nephroblastoma, is the predominant renal malignancy in the pediatric population. This narrative review explores the evolution of personalized care strategies for WT, synthesizing critical developments in molecular diagnostics and treatment approaches to enhance patient-specific outcomes. We surveyed recent literature from the last five years, focusing on high-impact research across major databases such as PubMed, Scopus, and Web of Science. Diagnostic advancements, including liquid biopsies and diffusion-weighted MRI, have improved early detection precision. The prognostic significance of genetic markers, particularly WT1 mutations and miRNA profiles, is discussed. Novel predictive tools integrating genetic and clinical data to anticipate disease trajectory and therapy response are explored. Progressive treatment strategies, particularly immunotherapy and targeted agents such as HIF-2α inhibitors and GD2-targeted immunotherapy, are highlighted for their role in personalized treatment protocols, especially for refractory or recurrent WT. This review underscores the necessity for personalized management supported by genetic insights, with improved survival rates for localized disease exceeding 90%. However, knowledge gaps persist in therapies for high-risk patients and strategies to reduce long-term treatment-related morbidity. In conclusion, this narrative review highlights the need for ongoing research, particularly on the long-term outcomes of emerging therapies and integrating multi-omic data to inform clinical decision-making, paving the way for more individualized treatment pathways.
肾母细胞瘤(WT),即肾胚胎瘤,是儿童群体中主要的肾脏恶性肿瘤。本叙述性综述探讨了WT个性化护理策略的演变,综合了分子诊断和治疗方法的关键进展,以改善针对患者的治疗效果。我们调查了过去五年的近期文献,重点关注了PubMed、Scopus和Web of Science等主要数据库中的高影响力研究。诊断方面的进展,包括液体活检和扩散加权磁共振成像,提高了早期检测的精度。讨论了基因标志物的预后意义,特别是WT1突变和微小RNA谱。探索了整合基因和临床数据以预测疾病发展轨迹和治疗反应的新型预测工具。渐进性治疗策略,特别是免疫疗法以及HIF-2α抑制剂和GD2靶向免疫疗法等靶向药物,因其在个性化治疗方案中的作用而受到关注,尤其是对于难治性或复发性WT。本综述强调了基于基因见解的个性化管理的必要性,局部疾病的生存率提高超过90%。然而,高危患者的治疗以及降低长期治疗相关发病率的策略仍存在知识空白。总之,本叙述性综述强调了持续研究的必要性,特别是关于新兴疗法的长期结果以及整合多组学数据以指导临床决策,为更个性化的治疗途径铺平道路。