Vincristine with or without steroid therapy

The most common treatment option for complicated kaposiform hemangioendotheliomas with or without Kasabach-Merritt phenomenon has traditionally been steroid therapy with or without vincristine or other agents.[21-26] However, many institutions are now using the mTOR inhibitor sirolimus, with or without steroid therapy, as primary treatment for high-risk patients.[27-31] Steroid therapy has not been effective as a single agent for complicated kaposiform hemangioendotheliomas, even at high doses. Patients treated with steroid therapy have a response rate of 10% to 20% and a significant number of side effects.[21]

Vincristine was shown to have a hematologic response and reduction in tumor volume in patients with high-risk kaposiform hemangioendotheliomas.[22] Furthermore, in a retrospective review of 37 children with kaposiform hemangioendotheliomas whose lesions did not respond to steroids, 26 of the lesions achieved complete remission, with platelet counts reaching normal levels within 7.6 (± 5.2) weeks after vincristine treatment.[24][Level of evidence C3] Vincristine monotherapy in other studies has not been shown to be effective.[27,31] Successful management of patients with kaposiform hemangioendotheliomas who were treated with vincristine and ticlopidine has also been reported.[32]

In 2013, consensus guidelines for the management of complicated kaposiform hemangioendotheliomas proposed the use of vincristine with or without steroids as first-line therapy. This recommendation was based on available evidence.[21]