Assessment of the Clinical Use and Outcomes of Sirolimus Treatment for Complex Vascular Anomalies

Overview

Sirolimus is a drug that was previously approved to prevent organ rejection after kidney transplant. In recent years it has increasingly been used for the treatment of vascular anomalies in children and adults. Because treating vascular anomalies with sirolimus is not FDA approved, there are no guidelines that healthcare providers can follow to determine how to treat these conditions effectively and how to avoid this medication’s potential side effects. It is important to learn directly from providers at different vascular anomaly centers about how they use sirolimus in treating their patients, including: the dose, duration, clinical response, side effects, and long-term outcomes. We plan to collect this and other relevant information in a database, to analyze it, and to help provide guidelines on how to safely treat vascular malformations using sirolimus, especially in children. Once the database is established, it can be updated in the future with additional patients and therapies.

Status

This project was funded through a 2023 PeDRA Research Fellowship Grant.