Sirolimus Versus Sirolimus Plus Prednisolone for Kaposiform Hemangioendothelioma
Status:
Recruiting
Trial end date:
2021-12-31
Target enrollment:
Participant gender:
Summary
Kaposiform hemangioendothelioma (KHE) is a rare vascular neoplasm that occurs predominantly
in infancy or early childhood. KHE has a nearly equal sex ratio. The annual incidence of KHE
has been estimated at 0.071 per 100,000 children. KHE presents with intermediate-malignant
and locally aggressive characteristics but without distant metastases.
This pilot trial studies sirolimus versus sirolimus plus pednisolone in treating patients
diagnosed with kaposiform hemangioendothelioma (KHE) and Kasabach-Merritt phenomemon (KMP)
that cannot be removed by surgery. The purpose of this study is to compare the efficacy and
safety of orally administered sirolimus versus sirolimus plus pednisolone in the treatment of
KHE associated with KMP.