CAR-T（CAR-T）细胞疗法在非肿瘤性疾病中的应用
Chimeric antigen receptor T (CAR-T) cell therapy in non-oncological diseases.
CELL INTELLIGENCE · 肿瘤细胞治疗研究
肿瘤细胞治疗研究
用于将真实世界淋巴瘤登记数据转换为 CDISC 标准的可复用流程：使用淋巴瘤流行病学结局联盟真实世界证据的实施与验证
英文原题：Reusable Pipeline for Converting Real-World Lymphoma Registry Data to CDISC Standards: Implementation and Validation Using Lymphoma Epidemiology of Outcomes Consortium for Real World Evidence.
查看英文原题
Reusable Pipeline for Converting Real-World Lymphoma Registry Data to CDISC Standards: Implementation and Validation Using Lymphoma Epidemiology of Outcomes Consortium for Real World Evidence.
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研究概要
本研究展示了一个实用、可复用的流程，用于淋巴瘤中 CDISC 标准化的 RWD。
研究思路结论见上方概要
目的
癌症登记数据的异质性限制了真实世界数据（RWD）与临床试验一起用于支持监管决策的可重用性。利妥昔单抗、吉西他滨和奥沙利铂（R-GemOx）通常是大B细胞淋巴瘤（LBCL）试验中的对照方案并在实践中使用，但RWD的结构不适合用于试验比较。我们开发了一个可重用的流程，通过构建一个通用的、覆盖整个登记数据库的研究数据列表模型（SDTM）并衍生出可直接用于分析的分析数据模型（ADaM）数据集，将淋巴瘤流行病学结局联盟真实世界证据（LEO CReWE）登记数据库转换为符合临床数据交换标准联盟（CDISC）标准的数据集。
方法
使用R 4.5.2及sdtm.oak、admiral和admiralonco包，我们为多中心LEO队列中接受R-GemOx治疗的183例复发/难治性LBCL患者生成了ADaM。
结果
ADaM 衍生的汇总统计显示与该队列已发表结果完全一致：中位年龄 68 岁，完全缓解率 29%，中位总生存期 13.5 个月，中位无事件生存期 2.3 个月。使用 CDISC Open Rules Engine（CORE）评估 CDISC 符合性；未发现任何代表拒绝级结构性不符合的发现。通用化 SDTM 使 ADaM 能够在队列定义变化时快速重新生成，包括一个接受 axicabtagene ciloleucel（一种CAR-T 细胞）治疗的独立队列，并支持各站点保留对患者级数据控制权的联邦分析。
展开英文摘要原文
PURPOSE
Heterogeneity in cancer registries limits reuse of real-world data (RWD) alongside clinical trials to support regulatory decisions. Rituximab, gemcitabine, and oxaliplatin (R-GemOx) is commonly a comparator in large B-cell lymphoma (LBCL) trials and used in practice, but RWD are not structured for trial comparison. We developed a reusable pipeline to convert the Lymphoma Epidemiology of Outcomes Consortium for Real World Evidence (LEO CReWE) registry into Clinical Data Interchange Standards Consortium (CDISC)-compliant data sets by constructing a generalized, registry-wide Study Data Tabulation Model (SDTM) and deriving analysis-ready Analysis Data Model (ADaM) data sets.
METHODS
Using R 4.5.2 with the sdtm.oak, admiral, and admiralonco packages, we generated ADaM for 183 patients with relapsed/refractory LBCL treated with R-GemOx across the multicenter LEO cohort.
RESULTS
ADaM-derived summary statistics showed exact concordance with published results for this cohort: median age 68 years, complete response rate 29%, median overall survival 13.5 months, and median event-free survival 2.3 months. CDISC conformance was assessed using the CDISC Open Rules Engine (CORE); no finding represented rejection-level structural nonconformance. The generalized SDTM enabled rapid regeneration of ADaM as cohort definitions changed, including a separate cohort treated with axicabtagene ciloleucel (a chimeric antigen receptor T-cell), and supports federated analyses in which sites retain control of patient-level data.
CONCLUSION
This work demonstrates a practical, reusable pipeline for CDISC-standardized RWD in lymphoma.
论文信息
- 作者
- Kane MJ、Chihara D、Ayers A、Flowers CR
- 单位
- Department of Lymphoma and Myeloma, The University of Texas MD Anderson Cancer Center, Houston, TX.United States
- 期刊
- JCO clinical cancer informatics2026 Jul-Sep
- 原文标识
- PubMed 42726020 · DOI 10.1200/CCI-26-00027