Efficacy and Safety of High- vs Low-Dose Sirolimus in Patients with Kaposiform Hemangioendothelioma: A Randomized Clinical Trial
Published: 10 March 2025| Version 1 | DOI: 10.17632/vp8vrj8m72.1
Contributors:
Jiangyuan Zhou, Yuru Lan, Tong Qiu, Zixin Zhang, Xue Gong, Xuepeng Zhang, Congxia Yang, Zilong Zhou, Yujia Zhang, Min Yang, Jianlei Fu, Chunshui He, Qiang Peng, Fan Hu, Chunchao Xia, Feiteng Kong, Siyuan Chen, Yi JiDescription
Sirolimus is recommended for the treatment of kaposiform hemangioendothelioma (KHE). However, the optimal sirolimus dose for the treatment of KHE remains unknown. A prospective multicenter study was conducted to evaluate the noninferiority and safety of low-dose versus high-dose sirolimus in patients exhibiting KHE without Kasabach–Merritt phenomenon (KMP). Among the 79 participants included in the study, low-dose sirolimus is noninferior to high-dose sirolimus, suggesting that it may be an effective and safe option for KHE without KMP.
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Institutions
Sichuan University West China Hospital
Categories
Hemangioma, Sirolimus, Safety, Pediatrics Patient, Efficacy Assessment