Sirolimus for the treatment of children with various complicated vascular anomalies

摘要: Vascular anomalies include a heterogeneous group of disorders that are categorized as vascular tumors or malformations. Treatment options resection, embolization, laser therapy, and sclerotherapy medical treatment such propranolol, steroids, interferon, cytostatic chemotherapy. Mammalian target rapamycin seems to play key role in the signal pathway angiogenesis subsequently development anomalies. Recently, successful use sirolimus has been reported children with lymphatic malformations kaposiform hemangioendotheliomas. We report on six patients different (kaposiform hemangioendothelioma n = 2, combined lymphatico-venous malformation pulmonary lymphangiectasia n = 1, orbital n = 1) who were treated peroral sirolimus. Three initially presented Kasabach-Merrit phenomenon. Median duration was 10 months; two still treatment. each achieved complete partial remission. phenomenon resolved within 1 month all patients. tolerated well; only mild reversible leukopenia observed. Conclusion: Sirolimus proved be effective complicated well acceptable side effects. The optimum length possible long-term effects have evaluated.