Real-World Molecularly Targeted Treatment Registry (MaTTeR): a Pilot Study to Enrich CCDI Data Utilizing Directed Electronic Medical Record (EMR) Extraction

真实世界分子靶向治疗登记处 (MaTTeR)：利用定向电子病历 (EMR) 提取丰富 CCDI 数据的试点研究

• 批准号: 10878384
• 负责人: LAURIE Hollis GLIMCHER
• 金额: $ 49.59万
• 依托单位: DANA-FARBER CANCER INST
• 依托单位国家: 美国
• 财政年份: 2023
• 资助国家: 美国
• 起止时间: 2023-09-01 至 2026-11-30
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/10878384
• 关键词: Adolescent; Adolescent and Young Adult; Adolescent and young adult cancer patients; Adult; Brain Neoplasms; Caring; Cephalic; Child; Childhood; Childhood Brain Neoplasm; Clinical; Clinical Data; Clinical Oncology; Clinical Trials; Combined Modality Therapy; Communities; Community Clinical Oncology Program; Computerized Medical Record; DNA Sequence Alteration; Dana-Farber Cancer Institute; Data; Data Collection; Data Element; Data Set; Databases; Dose; Drug usage; Ecosystem; Enrollment; Exclusion; Formulation; Future; Genes; Genomics; Goals; Health Services Accessibility; Individual; Institution; Investigational Drugs; Label; Malignant Childhood Neoplasm; Malignant Neoplasms; Modification; Mutation; Oncologist; Oncology; Outcome; Patient-Focused Outcomes; Patients; Pediatric Oncology; Pharmaceutical Preparations; Pilot Projects; Principal Investigator; Protocols documentation; Publications; Refractory; Registries; Relapse; Reporting; Research; Resources; Role; Solid Neoplasm; Testing; Time; Toxic effect; United States Food and Drug Administration; Work; Writing; cBioPortal; cancer genomics; clinical care; clinically relevant; cohort; data submission; data visualization; experience; feasibility testing; first-in-human; genomic data; genomic profiles; high risk; leukemia; molecular targeted therapies; novel therapeutics; off-label use; oncology trial; pediatric patients; response; search engine; targeted treatment; tool; treatment and outcome; treatment duration; tumor; young adult

Project Summary Abstract Integration of genomic profiling into the care of pediatric, adolescent and young adult (AYA) patients with cancer has resulted in the identification of potentially targetable alterations and the use of molecularly targeted therapies (MTT). Pediatric and AYA cancer patients are increasingly treated with MTT outside of clinical trials. Systematic data collection and publication of MTT use outside of clinical trials are lacking such that these real-world experiences are not optimally contributing to the current understanding of MTT use in pediatric and AYA cancers. This project will utilize genomic data previously contributed to the Children’s Cancer Data Initiative (CCDI) as well as additional Dana-Farber Cancer Institute (DFCI) institutional cohorts to identify patients with targetable genomic alterations. We will then identify patients who received targeted therapies off of a clinical trial and extract key treatment data, including drug information, dose, dosing formulation, combination treatment, duration of treatment, best response, time to best response, duration of response, dose modifications due to toxicity, toxicities, manner of treatment access (off-label, single patient investigational new drug (IND) protocol). The systematic clinical data collection of MTT use of the CCDI cohort of patients proposed in this project will serve as the proof of concept of accessing and enriching CCDI data with highly valuable data that is otherwise imprisoned within the electronic medical record. This project will establish the feasibility of creating a scalable registry of real-world targeted therapy experience that can be expanded to additional CCDI datasets and serve as an important future resource for the pediatric and AYA cancer community.

项目概要 摘要 将基因组分析整合到儿科、青少年和年轻人 (AYA) 的护理中 癌症患者的研究已导致潜在可靶向改变的识别和 儿童和 AYA 癌症患者越来越多地使用分子靶向治疗 (MTT)。 在临床试验之外使用 MTT 进行治疗 系统性地收集和发布 MTT 使用情况。 缺乏临床试验之外的经验，因此这些现实世界的经验并不是最佳的 有助于当前对 MTT 在儿科和 AYA 癌症中的应用的了解。 将利用之前为儿童癌症数据倡议 (CCDI) 贡献的基因组数据 以及其他丹纳法伯癌症研究所 (DFCI) 机构队列来确定 然后，我们将确定接受靶向基因组改变的患者。 临床试验中的疗法并提取关键治疗数据，包括药物信息、剂量、 剂量配方、联合治疗、治疗持续时间、最佳反应、最佳时间 反应、反应持续时间、由于毒性而改变的剂量、毒性、作用方式 治疗途径（标签外、单一患者研究性新药 (IND) 方案）。 本报告中提出的 CCDI 患者队列 MTT 使用的系统临床数据收集 项目将作为访问和丰富 CCDI 数据的概念证明，具有高度 该项目将保存电子医疗记录中的有价值的数据。 建立创建真实世界靶向治疗经验的可扩展注册库的可行性 可以扩展到其他 CCDI 数据集，并作为未来的重要资源 儿科和 AYA 癌症社区。