Additive value of transarterial embolization to systemic sirolimus treatment in kaposiform hemangioendothelioma

Abstract

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 whether additional transarterial embolization is of benefit for children with KHE and KMP. Seventeen 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 × 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.

Cutaneous Vascular Neoplasms of Uncertain Biological Behavior

“…Occasionally, the spindled tumor cells can demonstrate a swirling growth pattern, invoking a somewhat glomeruloid appearance. Tumor cells have uniform hyperchromatic spindled nuclei arranged in short fascicles with slit-like vascular lumina surrounded by a dense collagenous stroma ( Figure 2 ) [ 6 , 7 , 8 , 9 , 10 , 11 ]. Additionally, most cases show extravasated red blood cells, single cells with lumina containing RBCs, dilated lymphatic vessels, eosinophilic bodies, and hemosiderin amidst vascular cells and fibrin thrombi.…”

“…In contrast, deeper and more extensively infiltrative tumors not amenable to a conservative excision continue to present a conundrum. A review of the current literature has demonstrated several studies in which conservative radiotherapy [ 63 ] or the use of targeted drugs (e.g., Telatnib and Sirolimus) [ 7 , 11 ] provides excellent outcomes for patients, demonstrated by high regression rates.…”

“…Additionally, a recent study conducted by Brill et al demonstrated the utilization of transarterial embolization in combination with systemic sirolimus therapy in non-surgical candidates with KMP. The use of both therapies together showed rapid resolution of life-threatening KMP, engendering improved survival for patients with KHE [11]. The first description of the Dabska tumor was recorded in 1969 by Maria D ąbska in a case report series of six children with various cutaneous lesions.…”

“…Recently the field of cutaneous vascular neoplasms has recognized a significant number of unique and challenging entities [ 1 , 2 , 3 , 4 , 5 , 6 , 7 , 8 , 9 ]. While tumors have been classified into benign or malignant categories, others remain poorly characterized and thus are referred to as „cutaneous neoplasms of uncertain biological potential” [ 10 , 11 , 12 ]. These cases, while not the most common in daily practice, present quite a diagnostic and prognostic conundrum for both dermatopathologists and treating clinicians.…”

Transarterial Embolization in Neonatal Kasabach–Merritt Syndrome

“…However, the long treatment course and high rate of side effects prompt us into seeking other therapeutic alternatives ( 6 – 8 ). Transarterial embolization (TAE) has been introduced to manage KMS for about 20 years, with decent results in experienced hands ( 9 , 10 ). We have previously analyzed the clinical features of 16 neonates with KMS and found that TAE had a good effect in some neonates ( 11 ).…”