英国新诊断骨肉瘤患者的前瞻性观察队列研究:标志性研究初步结果。
A Prospective Observational Cohort Study for Newly Diagnosed Osteosarcoma Patients in the UK: ICONIC Study Initial Results.
发表日期:2024 Jun 27
作者:
Alexa Childs, Craig Gerrand, Bernadette Brennan, Robin Young, Kenneth S Rankin, Michael Parry, Jonathan Stevenson, Adrienne M Flanagan, Rachel M Taylor, Lorna Fern, Dominique Heymann, Filipa Vance, Jenny Sherriff, Saurabh Singh, Rubina Begum, Sharon L Forsyth, Krystyna Reczko, Kate Sparksman, William Wilson, Sandra J Strauss
来源:
Bone & Joint Journal
摘要:
过去 25 年里,骨肉瘤 (OS) 的标准治疗几乎没有变化,如果要改善结果,就需要确定新的生物标志物和新的治疗方法。此外,关于 OS 治疗对患者报告结果 (PRO) 影响的证据有限。 ICONIC(通过骨肉瘤合作改善结果;NCT04132895)是一项前瞻性观察队列研究,招募了英国 (UK) 新诊断的 OS 患者,并纵向收集了匹配的临床、生物学和 PRO 数据。第一阶段评估了招募和数据收集的可行性,在 22 个地点招募了 102 名患者,其中代表来自 OS 研究中经常排除的患者群体,包括 50 岁以上的患者和原发部位不太常见的患者。除了 PRO 数据之外,收集临床和生物样本的可行性已经确定,并且作为第二阶段的一部分,正在对这些数据进行持续分析。ICONIC 将提供代表英国患者的独特的前瞻性队列,由新诊断的 OS 患者组成人群,具有与分子特征生物样本相关的完整注释的临床结果,允许进行全面分析,以更好地了解生物学并开发新的生物标志物和新颖的治疗方法。
There has been little change to the standard treatment for osteosarcoma (OS) over the last 25 years and there is an unmet need to identify new biomarkers and novel therapeutic approaches if outcomes are to improve. Furthermore, there is limited evidence on the impact of OS treatment on patient-reported outcomes (PROs). ICONIC (Improving Outcomes through Collaboration in Osteosarcoma; NCT04132895) is a prospective observational cohort study recruiting newly diagnosed OS patients across the United Kingdom (UK) with matched longitudinal collection of clinical, biological, and PRO data. During Stage 1, which assessed the feasibility of recruitment and data collection, 102 patients were recruited at 22 sites with representation from patient groups frequently excluded in OS studies, including patients over 50 years and those with less common primary sites. The feasibility of collecting clinical and biological samples, in addition to PRO data, has been established and there is ongoing analysis of these data as part of Stage 2. ICONIC will provide a unique, prospective cohort of newly diagnosed OS patients representative of the UK patient population, with fully annotated clinical outcomes linked to molecularly characterised biospecimens, allowing for comprehensive analyses to better understand biology and develop new biomarkers and novel therapeutic approaches.