O Tezol, M Alakaya, B Gundogan, EC Citak

Abstract

mTOR inhibitors have become a new and valid treatment choice for patients with refractory vascular anomalies. We aimed to present paediatric cases with benign vascular anomalies treated with sirolimus. The clinical data of 30 patients were reviewed retrospectively: 46.7% (n=14) had infantile haemangioma and 53.3% (n=16) had vascular malformation. Response to treatment was defined as improvement in radiologic imaging and/or reduction of lesion size and symptoms. The median age of the patients was 2.6 (1.2-17) years. The dosage of oral sirolimus was 0.8 mg/m²/12 hours. The overall successful response rate was 66.7% (n=20) at a median duration of 5 months. Three patients with infantile haemangioma and four patients with venous, one patient with lymphatic, one patient with capillary, and one patient with arteriovenous malformation had no response. There was neither complete response nor worsening on therapy. Sirolimus was well tolerated with no patient required premature termination of treatment. We conclude that sirolimus may be a valid treatment choice for paediatric patients with refractory benign vascular anomalies. Further prospective studies on the optimal therapeutic regimen including dosage and duration should be performed.