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

点击翻译按钮获取中文摘要

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.