Clinical characterization and long‐term outcomes in pediatric epithelioid hemangioendothelioma

Abstract

There is a paucity of information about the clinical characteristics and long‐term outcomes of pediatric epithelioid hemangioendothelioma (EHE), a rare vascular neoplasm commonly presenting in adulthood. In our case series of 24 patients with EHE aged 2‐26 years, the majority presented with multi‐organ disease. Progression was seen in 63% of patients with a mean time to progression of 18.4 months (range: 0‐72). Three patients treated with sirolimus achieved stable disease or partial response for >2.5 years. Longitudinal prospective pediatric studies are needed to develop standardized approaches to surgical and medical management.

Systemic therapies in advanced epithelioid haemangioendothelioma: A retrospective international case series from the World Sarcoma Network and a review of literature

“…To the best of our knowledge, only 2 responses have been reported with doxorubicin in 1997 and 2010, in two metastatic EHE patients, however, the diagnosis was not confirmed by either molecular testing or IHC 26,27 . Cioffi A. et al did not record any radiological responses in 16 advanced EHE patients treated with anthracyclines, with a m‐PFS of 4.8 months 19 . Similarly, no PR were reported by Yousaf N. et al and in most case reports available 13 .…”

“…mTOR inhibitors (i.e. sirolimus) can control the disease in slowly progressive cases, whereas their activity seems to be limited in more aggressive variants, such as those presenting with serosal effusions 5,19,20 . In the absence of any alternative potentially active treatment approved other that pazopanib, EHE patients with advanced progressive disease are treated with conventional chemotherapy, which is assumed to have very limited activity 13,21 …”

Natural history of epithelioid hemangioendothelioma that progressed over 20 years

“…At present, EHE is usually said to be lethal in 50% of cases 5 ; however, the clinical course is quite variable as reported 1,3 . A Japanese study on EHE by Shiba et al.…”

“…We read with great interest the paper by Cournoyer et al. (PBC 2020 Feb; 67(2): e28045) describing 24 cases of epithelioid hemangioendothelioma (EHE); of 15 evaluable patients, overall survival was 73% at 5 years 1 . EHE is a rare sarcoma of vascular origin and uncertain biological behavior, often occurring as an indolent angiocentric vascular tumor at various anatomic sites 2 .…”

“…EHE often involves the liver (81%), lungs (57%), and bones (12%) 2 . Although there are various chemotherapy regimens that show limited efficacy, 1,3 this patient received none until the terminal stage. A promising candidate, a novel agent targeting the EHE‐specific fusion oncogene WWTR1‐CAMTA1, 4 could be developed for future management of EHE.…”