Treatment practices and response in kaposiform hemangioendothelioma: A multicenter cohort study.
Document Type
Article
Publication Date
3-1-2024
Publication Title
Pediatric blood & cancer
Keywords
washington; spokane; pshmc; Child; Humans; Kasabach-Merritt Syndrome; Vincristine; Vascular Neoplasms; Prospective Studies; Hemangioendothelioma; Sarcoma, Kaposi; Sirolimus; Hemangioma; Skin Neoplasms
Abstract
BACKGROUND AND OBJECTIVES: Kaposiform hemangioendothelioma (KHE) and tufted angioma (TA) are rare vascular tumors in children historically associated with significant morbidity and mortality. This study was conducted to determine first-line therapy in the absence of available prospective clinical trials.
METHODS: Patients from 17 institutions diagnosed with KHE/TA between 2005 and 2020 with more than 6 months of follow-up were included. Response rates to sirolimus and vincristine were compared at 3 and 6 months. Durability of response and response to other treatment modalities were also evaluated.
RESULTS: Of 159 unique KHE/TA subjects, Kasabach-Merritt phenomenon (KMP) was present in 64 (40.3%), and only two patients were deceased (1.3%). Over 60% (n = 96) demonstrated treatment response at 3 months, and more than 70% (n = 114) by 6 months (no significant difference across groups). The vincristine group had higher radiologic response at 3 months compared to sirolimus (72.7% vs. 20%, p = .03), but there were no differences between these groups at 6 months. There were no differences in rates of recurrent or progressive disease between vincristine and sirolimus.
CONCLUSIONS: In this large, multicenter cohort of 159 patients with KHE/TA, rates of KMP were consistent with historical literature, but the mortality rate (1.3%) was much lower. Overall treatment response rates were high (>70%), and there was no significant difference in treatment response or durability of disease comparing sirolimus to vincristine. Our results support individualized treatment decision plans depending on clinical scenario and patient/physician preferences. Response criteria and response rates reported here will be useful for guiding future treatment protocols for vascular tumors.
Clinical Institute
Women & Children
Clinical Institute
Cancer
Department
Hematology
Department
Pediatrics
Department
Oncology
Recommended Citation
Borst, Alexandra J; Eng, Whitney; Griffin, Morgan; Ricci, Kiersten W; Engel, Elissa; Adams, Denise M; Dayneka, Jillian; Cohen-Cutler, Sally J; Andreoli, Steven M; Wu, Melinda D; Wheeler, Allison P; Heym, Kenneth M; Crary, Shelley E; Nakano, Taizo A; Schulte, Rachael R; Setty, Bhuvana A; McLean, Thomas W; Pahl, Kristy S; Intzes, Stefanos; Pateva, Irina; Teitelbaum, Max; Zong, Zili; Li, Yimei; and Jeng, Michael R, "Treatment practices and response in kaposiform hemangioendothelioma: A multicenter cohort study." (2024). Articles, Abstracts, and Reports. 8187.
https://digitalcommons.providence.org/publications/8187
