Sirolimus as initial therapy for kaposiform hemangioendothelioma and tufted angioma

Abstract

Sirolimus as first-line therapy shows great promise in the treatment of kaposiform hemangioendothelioma and tufted angioma.

Childhood Vascular Tumors

“…Though medical management predominates in the treatment of tufted angiomas and kaposiform hemangioendotheliomas, if a lesion is localized and well-circumscribed, surgery may be an option. Embolization can also be used to stabilize very large lesions until medical therapy can be initiated (34)(35)(36).…”

“…KMP shows a variable response to treatment; both IV vincristine (combined with antiplatelet therapies) and oral sirolimus have shown promising results, though they have not been compared in a study. The conventional standard treatment was previously systemic corticosteroids which may be used initially, but should not delay the initiation of sirolimus or vincristine if indicated (35,37). Other therapies for these lesions have included embolization, surgical excision, pulse dye laser, low-dose aspirin, and radiation therapy.…”

Destructive (Erosive) Tufted Angioma/Kaposiform Hemangioendothelioma of the Eyelid

“…For patients who develop KMP, the first-line therapy consists of vincristine plus prednisone or sirolimus (rapamycin). Aspirin may also help to control the platelet interaction, pain, and growth of TA/KHE [11], [12]. Topical timolol maleate is an option for limited lesions [13].…”

Low dose sirolimus treatment for refractory tufted angioma and congenital kaposiform hemangioendothelioma, both with Kasabach‐Merritt phenomenon

“…6,9,10 Currently, sirolimus alone or combined with a short course of oral corticosteroids appears to be the best therapeutic option. [11][12][13][14][15][16][17][18] The optimal sirolimus dose in patients with KMF has not been established, but serum levels should be maintained between 5 and 15 ng/mL. 5,11,15 Low-dose sirolimus, using levels of 2-3 ng/mL, is associated with low toxicity.…”

“…A 2013 consensus recommended steroids associated with vincristine for KMP . Currently, sirolimus alone or combined with a short course of oral corticosteroids appears to be the best therapeutic option . The optimal sirolimus dose in patients with KMF has not been established, but serum levels should be maintained between 5 and 15 ng/mL.…”