(P 189) CLEAR CELL SARCOMA - REAL WORLD DATA ANALYSIS

Objective: Clear Cell Sarcoma (CCS) is an ultra-rare and aggressive soft tissue sarcoma which typically develops in tendons or aponeuroses and affects mostly teens and young adults. In most cases, CCS is caused by a translocation resulting in the fusion of the genes for activating transcription factor 1 (ATF1) and Ewing sarcoma breakpoint region 1 (EWSR1), resulting in the EWSR1-ATF1 fusion protein. Since CCS is so rare, there is a paucity of research, leading to few treatment options that are generally based on weak scientific evidence. The CCS patient community is eager to share real world evidence (RWE) data through a proprietary platform that collected de-identified electronic health records (EHRs) to establish a natural history research study with clinicians, researchers and patients working together for the purpose of improving patient outcomes.

Methods: CCS patients consent, through an IRB approved process, to have their EHRs extracted and annotated into a searchable platform. The information obtained from the EHRs is analyzed to establish patterns in efficacy of various treatment regimens, as well as to identify other patterns of CCS disease progression related to treatment choices. A symposium to discus results and synthesize a consensus paper will be held with CCS patients and caregivers along with clinicians and researchers focused on CCS.

Results: Preliminary results indicate automated EHR collections are incomplete and require human intervention to locate and extract missing data for a complete understanding of the natural history of the disease. In some cases, patients with similar therapeutic treatment history share similar outcomes. Results indicate that pediatric patients are often prescribed different treatment regimens than adult patients with CCS.

Conclusion: Based on the results of analysis of CCS patients’ RWE, the CCSF patients, clinicians and researchers are working together to form and publish a consensus on the information collected for CCS. This will be done with the intent to help guide treatment toward promising options and away from options that RWE shows to be ineffective or worse. The intent is also to expose the areas of the greatest need to focus future research.