Treatment practices and response in kaposiform hemangioendothelioma: A multicenter cohort study.
Alexandra J BorstWhitney EngMorgan GriffinKiersten W RicciElissa EngelDenise M AdamsJillian DaynekaSally J Cohen-CutlerSteven M AndreoliMelinda D WuAllison P WheelerKenneth M HeymShelley E CraryTaizo A NakanoRachael R SchulteBhuvana A SettyThomas Williams McLeanKristy S PahlStefanos IntzesIrina PatevaMax TeitelbaumZili ZongYimei LiMichael Raymond JengPublished in: Pediatric blood & cancer (2023)
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.