Eight years tumor control with pazopanib for a metastatic resistant epithelioid hemangioendothelioma

Abstract

Epithelioid hemangioendothelioma is a rare connective tissue tumor of vascular origin. It is most commonly found in young to middle aged women, and its clinical behavior is remakably variable from an indolent metastatic tumor to an aggressive rapidly growing neoplasm. Most tumors are diagnosed in an advanced unresectable phase and when clinically aggressive, require systemic cytotoxic treatment of sarcoma. Then, the 5-year survival rate after chemotherapy does not exceed 30%. Antiangiogenics are active in selected sarcoma subtypes: pazopanib, the only anti angiogenic registered agent for sarcoma provides a median PFS of 4.5 months only in the pivotal study. Their activity in EHE has been reported but long term outcome of these patients remain unreported. We report a case of a female patient with HEH who was treated with pazopanib for almost 8 years. Pazopanib therapy resulted in clinical improvement of symptoms and durable stabilization of liver tumors and lung lesions.Conclusion: Pazopanib is a promising therapeutic option in patients with HEH.

Treatment patterns and clinical outcomes with pazopanib in patients with advanced soft tissue sarcomas in a compassionate use setting: results of the SPIRE study

“…It should also be noted that fibromatosis (n ¼ 2) and sarcomatoid carcinoma (n ¼ 1) are not strictly sarcomas, fibromatosis because it is a clonal connective tissue tumor that does not metastasize, but can be locally aggressive and occasionally fatal, and sarcomatoid carcinoma because it has epithelial differentiation but nevertheless may behave like a sarcoma and is often treated as one. However, these results are in accordance with previous publications in several case series of patients which have shown some patients with certain subtypes benefit from using pazopanib, including LMS, synovial sarcoma, angiosarcoma, UPS, MPNST, SFT, epithelioid hemangioendothelioma, hemangiopericytoma, rhabdomyosarcoma, desmoplastic small round cell and desmoid tumor/aggressive fibromatosis [15][16][17][18][19][20][21][22][23][24][25][26][27]. Overall, in this study, 46% of patients experienced clinical benefit at any time while on pazopanib.…”

Epithelioid hemangioendotheliomas of the liver and lung in children and adolescents

“…The pleiotropic cytokine interferon‐alpha yielded a CR in one, PR in one, and SD in two of six cases reported; two patients progressed on interferon‐alpha . Finally, the multityrosine kinase inhibitor pazopanib achieved one PR and one SD in two cases reported . Therapy was overall well tolerated.…”

“…[49][50][51][52][53][54] Finally, the multityrosine kinase inhibitor pazopanib achieved one PR and one SD in two cases reported. 55,56 Therapy was overall well tolerated. Finally, EHE diagnosis in the index patient was incidental following a workup for lymphedema of the left lower extremity.…”

Molecular characterization of hepatic epithelioid hemangioendothelioma reveals alterations in various genes involved in DNA repair, epigenetic regulation, signaling pathways, and cell cycle control

“…Therefore, clinicians need to focus on other therapeutic options. Until now, reliable options for target therapy approaches in larger patient cohorts do not exist although case reports 28,29 or smaller cohort studies 17 have been published especially on the successful usage of mTOR inhibitors. 30 In accordance to COSMIC several of the mutations found in our cohort might have the potential for targeted therapy but it is difficult to predict whether targeting these mutations might be an option for a larger cohort of patients.…”