Phone: 1-800-226-2379

SIR-DA-1202: A Randomized Phase 2 Study of Vincristine Versus Sirolimus -to Treat High Risk Kaposiform Hemangioendothelioma (KHE)


Kaposiform Hemangioendothelioma (vascular tumor)


Kaposiform hemangioendotheliomas (KHE) are extremely rare life threatening tumors that can be associated with Kasabach-Merritt Phenomenon consisting of profound thrombocytopenia and hypofibrinogenemia causing a significant risk of bleeding and an associated mortality rate as high as 20% to 30%. Despite the severity of potential complications, uniform guidelines are lacking both for the treatment and response to treatment of children and young adults with these tumors. The overall goal of this trial is to objectively assess the efficacy of sirolimus compared to vincristine for the treatment of patients with high risk KHE.

This is a multi-site trial, with 7 participating centers (NCT 02110069):

Boston Children’s Hospital, Boston, MA

Cincinnati Children’s Hospital Medical Center, Cincinnati, OH

The Johns Hopkins University, Baltimore, MD

Stanford University, Palo Alto, CA

Texas Children’s Hospital, Houston, TX

Medical College of Wisconsin, Milwaukee, WI

Emory University, Atlanta, GA

Eligibility Criteria

Ages Eligible for Study: Children and Adults 0 to 31 years of age

Sexes Eligible for Study: All

Other inclusion and exclusion criteria as listed on


Ionela Iacobas, MD  
Principal Investigator