Sirolimus Versus Sirolimus Plus Prednisolone for Kaposiform Hemangioendothelioma

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.

Timeline

Start

2017-06-01

Primary completion

2021-12-31

Completion

2021-12-31

Publications

• Background Ji Y, Chen S, Xiang B, Li K, Xu Z, Yao W, Lu G, Liu X, Xia C, Wang Q, Li Y, Wang C, Yang K, Yang G, Tang X, Xu T, Wu H. Sirolimus for the treatment of progressive kaposiform hemangioendothelioma: A multicenter retrospective study. Int J Cancer. 2017 Aug 15;141(4):848-855. doi: 10.1002/ijc.30775. Epub 2017 May 26.
• Background Wang C, Li Y, Xiang B, Li F, Chen S, Li L, Ji Y. Successful Management of Pancreatic Kaposiform Hemangioendothelioma With Sirolimus: Case Report and Literature Review. Pancreas. 2017 May/Jun;46(5):e39-e41. doi: 10.1097/MPA.0000000000000801. No abstract available.
• Background Reichel A, Hamm H, Wiegering V, Wiewrodt B, Neubauer H, Ernestus K, Winkler B. Kaposiform hemangioendothelioma with Kasabach-Merritt syndrome: successful treatment with sirolimus. J Dtsch Dermatol Ges. 2017 Mar;15(3):329-331. doi: 10.1111/ddg.12987. Epub 2017 Feb 21. No abstract available.
• Background Alaqeel AM, Alfurayh NA, Alhedyani AA, Alajlan SM. Sirolimus for treatment of kaposiform hemangioendothelioma associated with Kasabach-Merritt phenomenon. JAAD Case Rep. 2016 Dec 5;2(6):457-461. doi: 10.1016/j.jdcr.2016.06.005. eCollection 2016 Nov. No abstract available.
• Background Mahajan P, Margolin J, Iacobas I. Kasabach-Merritt Phenomenon: Classic Presentation and Management Options. Clin Med Insights Blood Disord. 2017 Mar 16;10:1179545X17699849. doi: 10.1177/1179545X17699849. eCollection 2017.
• Ji Y, Chen S, Zhou J, Yang K, Zhang X, Xiang B, Qiu T, Gong X, Zhang Z, Lan Y, Hu F, Kong F, Qiu Q, Zhang Y. Sirolimus plus prednisolone vs sirolimus monotherapy for kaposiform hemangioendothelioma: a randomized clinical trial. Blood. 2022 Mar 17;139(11):1619-1630. doi: 10.1182/blood.2021014027.

Drugs

Indications