Abstract 125P
Background
Vascular anomalies (VA) are endothelial disorders that affect blood vessels, veins, lymph nodes, and capillaries. There are a few treatment alternatives available for VA. Sirolimus, an mTOR inhibitor, inhibits cell growth and proliferation by controlling ribosome biogenesis and protein synthesis in VA and vascular cancers. However, although most sirolimus studies on VA were in the pediatric age group, its efficacy in adults is limited. The aim of this study was to convey our real-world clinical experience with sirolimus in treating adult patients with VA.
Methods
We retrospectively identified VA patients over 16 years who were followed at Hacettepe University Cancer Institute from January 2013 to September 2022. Demographic and clinical characteristics of the patients were recorded. The primary outcome measure was the efficacy of sirolimus at the time of best response. The secondary endpoint was related to toxicity and safety.
Results
A total of 38 patients were analyzed. The mean age was 26.77 (16–66). The median follow-up was 18,56 (95% CI: 6,23–31,10) months. The disease control rate was found to be 92.1% (35/38). VA with head and neck location was statistically significant with an increased partial response rate (p = 0,001). Sirolimus was well tolerated. Grade 1 or 2 oral mucositis (n = 4) and skin rash (n = 3) were the most common side effects.
Conclusions
In this study, we found sirolimus was efficacious and well tolerated in adult patients with VA.
Clinical trial identification
Editorial acknowledgement
Legal entity responsible for the study
Hacettepe University, Ethics Boards and Commissions.
Funding
Has not received any funding.
Disclosure
All authors have declared no conflicts of interest.