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开发一个使用多学科真实世界数据的集成平台,以促进医疗产品的生物标志物发现。

Development of an Integrated Platform Using Multidisciplinary Real-World Data to Facilitate Biomarker Discovery for Medical Products.

机构信息

Center for Devices and Radiological Health, US Food and Drug Administration (CDRH/FDA), Silver Spring, Maryland, USA.

High-Performance Integrated Virtual Environment (HIVE), US Food and Drug Administration (FDA), Silver Spring, Maryland, USA.

出版信息

Clin Transl Sci. 2020 Jan;13(1):98-109. doi: 10.1111/cts.12685. Epub 2019 Sep 12.

Abstract

Translational multidisciplinary research is important for the Center for Devices and Radiological Health's efforts for utilizing real-world data (RWD) to enhance predictive evaluation of medical device performance in patient subpopulations. As part of our efforts for developing new RWD-based evidentiary approaches, including in silico discovery of device-related risk predictors and biomarkers, this study aims to characterize the sex/race-related trends in hip replacement outcomes and identify corresponding candidate single nucleotide polymorphisms (SNPs). Adverse outcomes were assessed by deriving RWD from a retrospective analysis of hip replacement hospital discharge data from the National Inpatient Sample (NIS). Candidate SNPs were explored using pre-existing data from the Personalized Medicine Research Project (PMRP). High-Performance Integrated Virtual Environment was used for analyzing and visualizing putative associations between SNPs and adverse outcomes. Ingenuity Pathway Analysis (IPA) was used for exploring plausibility of the sex-related candidate SNPs and characterizing gene networks associated with the variants of interest. The NIS-based epidemiologic evidence showed that periprosthetic osteolysis (PO) was most prevalent among white men. The PMRP-based genetic evidence associated the PO-related male predominance with rs7121 (odds ratio = 4.89; 95% confidence interval = 1.41-17.05) and other candidate SNPs. SNP-based IPA analysis of the expected gene expression alterations and corresponding signaling pathways suggested possible role of sex-related metabolic factors in development of PO, which was substantiated by ad hoc epidemiologic analysis identifying the sex-related differences in metabolic comorbidities in men vs. women with hip replacement-related PO. Thus, our in silico study illustrates RWD-based evidentiary approaches that may facilitate cost/time-efficient discovery of biomarkers for informing use of medical products.

摘要

转化多学科研究对于设备和放射健康中心利用真实世界数据(RWD)来增强医疗器械在患者亚群中性能的预测评估非常重要。作为我们开发新的基于 RWD 的证据方法的努力的一部分,包括通过计算发现与器械相关的风险预测因子和生物标志物,本研究旨在描述髋关节置换术结果中的性别/种族相关趋势,并确定相应的候选单核苷酸多态性(SNP)。通过从国家住院患者样本(NIS)的髋关节置换术住院数据的回顾性分析中获取 RWD 来评估不良结果。使用个性化医学研究计划(PMRP)中的现有数据来探索候选 SNP。高性能集成虚拟环境用于分析和可视化 SNP 与不良结果之间的潜在关联。通路分析(IPA)用于探索与性别相关的候选 SNP 的合理性,并描述与感兴趣变体相关的基因网络。基于 NIS 的流行病学证据表明,假体周围骨溶解(PO)在白人男性中最为普遍。基于 PMRP 的遗传证据将与 PO 相关的男性优势与 rs7121(优势比=4.89;95%置信区间=1.41-17.05)和其他候选 SNP 相关联。对预期基因表达改变和相应信号通路的 SNP 进行 IPA 分析表明,性别相关的代谢因素可能在 PO 的发展中起作用,这一点通过专门的流行病学分析得到了证实,该分析确定了与髋关节置换相关 PO 的男性与女性在代谢合并症方面的性别差异。因此,我们的计算研究说明了基于 RWD 的证据方法,这些方法可能有助于以具有成本效益的方式发现用于告知医疗器械使用的生物标志物。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/810e/6951466/cbfc041f2b78/CTS-13-98-g001.jpg

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