Additive value of Transarterial Embolization to systemic Sirolimus
treatment in Kaposiform Hemangioendothelioma
Abstract
Purpose: Kaposiform Hemangioendothelioma (KHE) is a rare vascular tumor
in children, which can be accompanied by life-threatening
thrombocytopenia, referred to as Kasabach-Merritt Phenomenon (KMP). The
mTOR inhibitor sirolimus is emerging as targeted therapy in KHE. As the
sirolimus effect on KHE occurs only after several weeks we aimed to
evaluate if additional transarterial embolization is of benefit for
children with KHE and KMP. Methods: 17 patients with KHE and KMP
acquired from 11 hospitals in Germany were retrospectively divided into
two cohorts. Children being treated with adjunct transarterial
embolization and systemic sirolimus, and those being treated with
sirolimus without additional embolization. Bleeding rate as defined by
WHO was determined for all patients. Response of the primary tumor at 6
and 12 months assessed by Magnetic Resonance Imaging (MRI), time to
response of KMP defined as thrombocyte increase >150 x
103/µl, as well as rebound rates of both after cessation of sirolimus
were compared. Results: N= 8 patients had undergone additive
embolization to systemic sirolimus therapy, sirolimus in this group was
started after a mean of 6.5 ± 3 days following embolization. N=9
patients were identified who had received sirolimus without additional
embolization. Adjunct embolization induced a more rapid resolution of
KMP within a median of 7 days vs 3 months, however tumor response as
well as rebound rates were similar between both groups. Conclusion:
Additive embolization may be of value for a more rapid rescue of
consumptive coagulopathy in children with KHE and KMP compared to
systemic sirolimus only.