Sirolimus Treatment of Complicated Vascular Anomalies: A Single Center’s Experience Over 2 Years
10.13029/aps.2024.30.1.14
- Author:
Jueun PARK
1
;
Suhyun HA
;
Hyunhee KWON
;
Jung-Man NAMGOONG
;
Hee Mang YOON
;
Pyeong Hwa KIM
;
Dae Yeon KIM
Author Information
1. Division of Pediatric Surgery, Asan Medical Center, University of Ulsan College of Medicine, Seoul, Korea
- Publication Type:Original Article
- From:
Advances in Pediatric Surgery
2024;30(1):14-22
- CountryRepublic of Korea
- Language:English
-
Abstract:
Purpose:Complicated vascular anomalies, characterized by encasing vital organ or diffusely locating unresectable lesion, pose therapeutic challenges with limited response to conventional treatment such as surgical resection or sclerotherapy. Sirolimus, an mammalian target of rapamycin inhibitor, has shown promising therapeutic effects in patients with vascular anomalies by inhibiting vascular endothelial growth factor, as reported in several studies. Here, we analyzed the treatment outcomes of patients who received sirolimus for complicated vascular anomalies at our institution.
Methods:Patients treated with sirolimus at the Department of Pediatric Surgery, Asan Medical Center from January 2018 to December 2021 were included. Sirolimus was administered twice daily at a dose of 0.8 mg per body surface area (BSA), with dose adjustments to achieve a target drug concentration of 8–12 ng/mL. Adverse drug effects and therapeutic responses were periodically assessed. Treatment efficacy was evaluated based on clinical findings pre- and post-sirolimus administration, absolute volume reduction of lesions through imaging tests (magnetic resonance imaging; MRI), and relative volume reduction adjusted to the patient's BSA.
Results:There were 16 females (50.0%) and 16 males (50.0%), with a median follow-up period of 41 months after sirolimus administration. Vascular anomaly types included lymphatic malformations (41%), venous malformations (28%), lymphovenous malformations (19%), and others (12.5%). The most common adverse effect was oral ulcer (6 patients). MRI volumetry revealed volume decreases in 17 patients (53.1%) with 22 patients (71%) exhibited lesion decreases relative to BSA. Notably, 9 patients (28.1%) had markedly decreased volume reduction based on absolute volume, and 12 (38.7%) based on volume compared to BSA.
Conclusion:Over a 2-year follow-up, sirolimus was effective in treating patients with complicated vascular anomalies, when administered with cautious consideration of side effects. A multidisciplinary approach is needed for evaluating treatment outcomes in these patients, necessitating further long-term research on adverse effects.
