A SIOPEN pragmatic clinical trial to MOnitor NeuroblastomA relapse with LIquid biopsy Sensitive Analysis (MONALISA)

SIOPEN 通过液体活检敏感性分析 (MONALISA) 监测神经母细胞瘤复发的实用临床试验

• 批准号: 10110442
• 金额: $ 33.83万
• 依托单位: INSTITUTE OF CANCER RESEARCH
• 依托单位国家: 英国
• 项目类别: EU-Funded
• 财政年份: 2024
• 资助国家: 英国
• 起止时间: 2024 至 无数据
• 项目状态: 未结题
• 来源: https://gtr.ukri.org/projects?ref=10110442
• 关键词: SIOPEN; pragmatic; clinical; trial; MOnitor

High-risk neuroblastoma accounts for 15% of cancer related-deaths in children. Half of the >1500 patients yearly diagnosed with neuroblastoma in the EU have high-risk disease, which will relapse or progress in half these cases after first-line treatment. Relapsed neuroblastoma is aggressive and often therapy-resistant. Monitoring for disease relapse and therapy response is crucial for the survival chance of these patients. The current standard-of-care for monitoring are imaging technologies and bone marrow assessment, which are costly, invasive and a burden for children, who often require anesthesia. These drawbacks limit how often is monitored. More sensitive, less invasive and less toxic monitoring techniques are needed. The mutational spectrum often changes in recurring tumors, which may explain therapy resistance and provide additional druggable targets. Imaging, however, provides no information about molecular characteristics. Liquid biopsy tests are minimally invasive, allow frequent sampling and sensitively detect tumor molecular markers in tumor-derived DNA and messenger RNA circulating in peripheral blood. MONALISA aims to close existing gaps and establish liquid biopsies as standard-of-care to monitor relapsed/refractory neuroblastoma, as a blueprint for other pediatric cancers. Reliable, early assessment of molecular progression or relapse is the main aim of the pragmatic randomized clinical trial proposed in MONALISA. We develop a digital decision support tool to help oncologists use the new monitoring and apply patient-reported outcomes to integrate patient viewpoints and assess the effect of minimally invasive, liquid biopsy diagnostics on quality of life. We will establish whether events can be detected earlier using liquid biopsy monitoring, and whether better overall survival is enabled by earlier diagnosis and treatment interventions. This essential step towards personalized medicine will support reliable disease monitoring under treatment. “This action is part of the Cancer Mission cluster of projects on ‘‘Diagnostics and Treatment (diagnostics).”

高危神经母细胞瘤占儿童癌症相关死亡的 15%。在欧盟，每年诊断出的超过 1500 名神经母细胞瘤患者中有一半患有高危疾病，其中一半病例在一线治疗后会复发或进展。复发性神经母细胞瘤具有侵袭性，并且通常具有治疗耐药性，监测疾病复发和治疗反应对于这些患者的生存机会至关重要，目前的监测标准是影像技术和骨髓评估。这些缺点限制了需要更灵敏、侵入性更小、毒性更小的监测技术，这可以解释复发性肿瘤的突变谱。然而，成像技术无法提供有关分子特征的信息，因此可以频繁取样并灵敏地检测外周血中循环的肿瘤来源 DNA 和信使 RNA 中的肿瘤分子标记。缩小现有差距并建立液体活检作为监测复发/难治性神经母细胞瘤的护理标准，作为其他儿科癌症的蓝图 对分子进展或复发进行可靠的早期评估是 MONALISA 提出的实用随机临床试验的主要目标。决策支持工具可帮助肿瘤学家使用新的监测并应用患者报告的结果来整合患者的观点并评估微创液体活检诊断对生活质量的影响我们将确定事件是否可以。使用液体活检监测可以更早地检测到，以及通过早期诊断和治疗干预是否可以实现更好的总体生存率，这一迈向个性化医疗的重要步骤将支持治疗过程中可靠的疾病监测。 “诊断和治疗（诊断）”。