Additive value of Transarterial Embolization to systemic Sirolimus treatment in Kaposiform Hemangioendothelioma
Menée en Allemagne à l'aide de données portant sur 17 jeunes patients atteints d'hémangio-endothéliome kaposiforme accompagné d'un syndrome de Kasabach-Merritt, cette étude évalue l'intérêt, du point de vue de la réponse tumorale évaluée par IRM à 6 et 12 mois, d'ajouter une embolisation transartérielle à un traitement par sirolimus
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. 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 grade 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. 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. 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. This article is protected by copyright. All rights reserved.