Thongngam N, Sathitsamitphong L, Morakote W, Chanthong S, Maneekesorn S, Natesirinilkul R, Charoenkwan P, Choed-Amphai C. Sirolimus for the treatment of vascular tumors and malformations in children: A 10-year single-institution retrospective study. World J Clin Pediatr 2026; 15(3): 119506 [DOI: 10.5409/wjcp.119506]
Corresponding Author of This Article
Chane Choed-Amphai, MD, PhD, Assistant Professor, Department of Pediatrics, Faculty of Medicine, Chiang Mai University, No. 110 Intawarorot Road, Sriphum, Muang, Chiang Mai 50200, Thailand. chane.c@cmu.ac.th
Research Domain of This Article
Pediatrics
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research-article
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Thongngam N, Sathitsamitphong L, Morakote W, Chanthong S, Maneekesorn S, Natesirinilkul R, Charoenkwan P, Choed-Amphai C. Sirolimus for the treatment of vascular tumors and malformations in children: A 10-year single-institution retrospective study. World J Clin Pediatr 2026; 15(3): 119506 [DOI: 10.5409/wjcp.119506]
Nattaporn Thongngam, Lalita Sathitsamitphong, Supapitch Chanthong, Supawadee Maneekesorn, Rungrote Natesirinilkul, Pimlak Charoenkwan, Chane Choed-Amphai, Department of Pediatrics, Faculty of Medicine, Chiang Mai University, Chiang Mai 50200, Thailand
Lalita Sathitsamitphong, Supapitch Chanthong, Supawadee Maneekesorn, Rungrote Natesirinilkul, Pimlak Charoenkwan, Chane Choed-Amphai, Thalassemia and Hematology Center, Faculty of Medicine, Chiang Mai University, Chiang Mai 50200, Thailand
Wipawee Morakote, Department of Radiology, Faculty of Medicine, Chiang Mai University, Chiang Mai 50200, Thailand
Author contributions: Thongngam N and Choed-Amphai C contributed to conceptualization, methodology, and statistical analysis; Thongngam N contributed to data collection and writing; Morakote W contributed to data collection and radiologic review; Choed-Amphai C contributed to supervision, reviewing, and editing; Sathitsammitpong L, Chanthong S, Manikasorn S, Netsirinilkul R, and Charoenkwan P contributed to methodology, reviewing, and editing; and all authors contributed to the article and approved the submitted version.
Institutional review board statement: This study was approved by the Institutional Review Board/Ethics Committee of the Faculty of Medicine, Chiang Mai University, Chiang Mai, Thailand (approval No. PED-2568-0835).
Informed consent statement: The requirement for written informed consent was waived by the Institutional Review Board/Ethics Committee of the Faculty of Medicine, Chiang Mai University, Chiang Mai, Thailand, due to the retrospective nature of the study and the use of de-identified data.
Conflict-of-interest statement: All the authors report no relevant conflicts of interest for this article.
Data sharing statement: The datasets used and analyzed during the current study are available from the corresponding author on reasonable request.
Corresponding author: Chane Choed-Amphai, MD, PhD, Assistant Professor, Department of Pediatrics, Faculty of Medicine, Chiang Mai University, No. 110 Intawarorot Road, Sriphum, Muang, Chiang Mai 50200, Thailand. chane.c@cmu.ac.th
Received: January 30, 2026 Revised: March 13, 2026 Accepted: April 15, 2026 Published online: September 9, 2026 Processing time: 183 Days and 16.6 Hours
Abstract
BACKGROUND
Vascular anomalies, including vascular tumors and vascular malformations, are common pediatric conditions that may cause significant morbidity. Although standard treatments are effective in many cases, a subset of patients experiences inadequate responses or has unresectable disease, necessitating alternative therapeutic strategies. Sirolimus has emerged as a targeted treatment option for selected vascular anomalies.
AIM
To evaluate treatment outcomes and the safety of sirolimus in children with vascular anomalies.
METHODS
We conducted a retrospective review of medical records of pediatric patients with vascular anomalies who received sirolimus at Chiang Mai University Hospital between 2016 and 2025. Clinical characteristics, treatment regimens, therapeutic responses, and adverse events were analyzed.
RESULTS
Eleven children were included in the study. Five patients had vascular tumors, including two kaposiform hemangioendotheliomas, one composite hemangioendothelioma, and two hemangiomas, while six patients had vascular malformations. Sirolimus was administered as salvage therapy in nine patients. After sirolimus treatment, 10 patients (90.9%) achieved partial response, and one patient had stable disease. At a median follow-up of 30 months, all patients were alive with disease. Sirolimus-related adverse events occurred in four patients, with proteinuria being the most common. Other events included hypokalemia, elevated serum creatinine, and hypercholesterolemia. No serious infections were observed.
CONCLUSION
Sirolimus demonstrated a high response rate and an acceptable safety profile in children with vascular anomalies. These findings support the use of sirolimus as an effective treatment option for refractory or unresectable cases, including complex entities such as composite hemangioendothelioma.
Core Tip: Sirolimus is increasingly used as targeted therapy for pediatric vascular anomalies, yet real-world clinical data remain limited. In this retrospective study of children with vascular tumors and malformations, sirolimus demonstrated a high response rate and an acceptable safety profile across diverse disease subtypes. Rapid resolution of Kasabach-Merritt phenomenon was observed in patients with kaposiform hemangioendothelioma receiving combination therapy. However, disease progression occurred after treatment discontinuation, highlighting the need for careful post-therapy monitoring. These findings support sirolimus as an effective treatment option in refractory or unresectable pediatric vascular anomalies and emphasize the importance of optimizing treatment duration and follow-up strategies.
