Kaposiform hemangioendotheliomas and tufted angiomas: Sirolimus for treating children

Abstract

Introduction. Kaposiform hemangioendothelioma (KHE) and tufted angioma (TA) are extremely rare vascular tumors characterized by pathologically progressive angiogenesis and lymphangiogenesis. They are also associated with the development of the KazabachMerritt Phenomenon (KMP; Kasabach-Merritt Phenomenon).Material and methods. 8 patients (5 boys, 3 girls) with kaposiform hemangioendothelioma (two out of them with Kazabakh-Merritt syndrome) and 4 patients (3 boys, 1 girl) with tufted angioma were treated at the Russian Children's Clinical Hospital of Pirogov Russian National Research Medical University (Moscow, Russia) . All patients were prescribed Sirolimus therapy for antiproliferative purpose.Results. In all children, positive changes were registered: reduction of vascular tumor volume, gradual improvement of clinical picture and control of complications: pain syndrome, KMP, improvement or restoration of functions in affected organs /systems (increase/restoration of movement volume in joints, compensation for length discrepancy in lower legs). Complete resorption of vascular tumors and clinical symptoms were registered in 3 patients.Conclusion. The obtained encouraging results after Sirolimus therapy prescribed for treating KME and TA allow the authors to recommend this therapy to patients with KME with or without KMP and TA. However, further researches with a large sample of participants is required to confirm the obtained results.