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 Ji

Description

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

Funding

National Natural Science Foundation of China

82473553

National Natural Science Foundation of China

82273556

Science and Technology Department of Sichuan Province

2022YFS0233

Science and Technology Department of Sichuan Province

2022YFS0225

Science and Technology Department of Sichuan Province

2022NSFSC1480

West China Hospital of Sichuan University

2020HXFH048, 2023HXFH004, and ZYJC21060

Sichuan University

2022SCUH0033 and YGJC004

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