Single-center experience with sirolimus therapy for vascular malformations

Area of Science:

• Vascular Surgery
• Pediatric Oncology
• Dermatology

Background:

• Vascular malformations (VMs) are congenital anomalies of blood vessels causing significant morbidity.
• Treatment options for extensive VMs are limited, with interventional procedures having decreased success rates.
• Medical management for VMs has historically been challenging.

Purpose of the Study:

• To evaluate the efficacy and safety of oral sirolimus in pediatric patients with extensive vascular malformations.
• To assess the response to sirolimus based on the type and extent of vascular malformations.

Main Methods:

• Retrospective case series of 6 pediatric patients with extensive VMs treated with oral sirolimus.
• Patients had previously failed various treatments at other centers.
• Treatment duration averaged 13 months, with ongoing follow-up.

Main Results:

• Five out of six patients achieved partial response to sirolimus treatment.
• Treatment response correlated positively with the lymphatic component of the VM.
• Sirolimus was well-tolerated, with acceptable side effects in all patients.

Conclusions:

• Oral sirolimus is a safe and effective medical treatment for widely distributed pediatric VMs, especially those with significant lymphatic involvement.
• Sirolimus provides a viable therapeutic option for complex VMs when local treatments are exhausted.
• Further research into sirolimus for vascular malformations is warranted.