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

项目摘要 将基因组分析整合到儿科、青少年和年轻成人（AYA）的护理中 癌症患者已经导致了潜在的靶向改变的鉴定， 分子靶向治疗（MTT）。儿科和AYA癌症患者越来越多地 在临床试验之外用MTT治疗。MTT使用的系统数据收集和发表 缺乏临床试验之外的信息，因此这些现实世界的经验不是最佳的。 有助于目前理解MTT在儿科和AYA癌症中的应用。这个项目 将利用先前提供给儿童癌症数据倡议（CCDI）的基因组数据 以及丹娜-法伯癌症研究所（DFCI）的其他机构队列， 具有靶向基因组改变的患者。然后我们将确定接受靶向治疗的患者 从临床试验中提取关键的治疗数据，包括药物信息，剂量， 给药制剂、联合治疗、治疗持续时间、最佳缓解、至最佳缓解时间 缓解、缓解持续时间、因毒性导致的剂量调整、毒性、给药方式 治疗访问（标签外，单个患者研究性新药（IND）方案）。的 本研究中提出的CCDI患者队列MTT使用的系统性临床数据收集 该项目将作为访问和丰富CCDI数据的概念证明， 有价值的数据，否则被囚禁在电子医疗记录中。该项目将 确定创建现实世界靶向治疗经验的可扩展注册表的可行性 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。

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.