Lay Abstract
Doxorubicin is a drug that works well against Desmoid tumors, but it can be harmful to the rest of the body when given through the bloodstream. DEE (Doxorubicin Eluting Embolization) is a treatment method which delivers the drug directly into the blood vessels feeding the tumor, so the tumor gets a high dose while the rest of the body gets much less. This is done by navigating a tube into the blood vessels and releasing tiny drug-filled beads that get stuck within the tumor’s blood supply and slowly release the medication. The technique was first used in children in 2018, expanded to more patients by 2022, and was officially included in treatment guidelines by 2024. This new study aims to collect data from seven hospitals using the same treatment plan to see how well it works and how it affects patients’ quality of life. Showing consistent results across many hospitals is helpful in supporting physicians who want to adopt this new technique to treat their patients locally.
Scientific Abstract
Doxorubicin is an effective treatment for Desmoid Fibromatosis but its use is limited by systemic toxicity. Selective arterial Doxorubicin Eluting Embolization (DEE) can be employed to achieve a high doxorubicin concentration in the target tissue while maintaining low systemic exposure. In this technique, biocompatible microparticles loaded with doxorubicin are released via catheter navigation into blood vessels that supply desmoid tumors. These particles, which are typically 100–300 μm in diameter, lodge within (embolize) the small arteries within a tumor and allow the doxorubicin to penetrate via elution. We initially described this technique in a pediatric case series in 2018 and in 2022 reported on a larger series of 24 patients treated with 52 DEE sessions. In 2024, DEE was incorporated as a therapeutic option in the NCCN guidelines and within the Desmoid Tumor Working Group Expert Consensus Paper. The purpose of this study is to document real-world results using DEE in seven centers using a standardized treatment protocol and follow-up, including patient-reported quality of life metrics.
