Efficacy and Safety of High- vs Low-Dose Sirolimus in Patients with Kaposiform Hemangioendothelioma: A Randomized Clinical Trial.

IF 12.8 1区医学 Q1 DERMATOLOGY

Journal of the American Academy of Dermatology Pub Date : 2025-03-17 DOI:10.1016/j.jaad.2025.03.023

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

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引用次数: 0

Abstract

Background: It remains unknown whether low-dose sirolimus can replace high-dose sirolimus for the treatment of kaposiform hemangioendothelioma (KHE) without the Kasabach-Merritt phenomenon (KMP).

Objective: To evaluate the noninferiority and safety of low-dose versus high-dose sirolimus in KHE patients.

Methods: This randomized, multicenter, open-label, noninferiority trial was conducted from February 2021 to August 2022. Participants received either a low-dose sirolimus regimen (blood trough concentration 5-8 ng/mL) or a high-dose sirolimus regimen (blood trough concentration 10-15 ng/mL). The primary endpoint was the difference in the proportion of patients between groups who achieved an objective response, defined as a ≥20% reduction in KHE volume at month 12.

Results: In this study, 39 were in the low-dose group, and 40 were in the high-dose group. At 1 year of treatment, 90.0% in the high-dose group and 89.7% in the low-dose group achieved an objective response (difference, 0.3%; 95% confidence interval -13.1 to 13.6). The incidences of total adverse events (AEs) and serious AEs were similar between the two groups, but respiratory, skin and mucosal AEs were less common in the low-dose group.

Limitations: The patients enrolled were not associated with KMP.

Conclusion: Low-dose sirolimus is noninferior to high-dose sirolimus in treating KHE.

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来源期刊

Journal of the American Academy of Dermatology 医学-皮肤病学

CiteScore

8.60

自引率

5.80%

发文量

2023

审稿时长

49 days

期刊介绍： The Journal of the American Academy of Dermatology (JAAD) is the official scientific publication of the American Academy of Dermatology (AAD). Its primary goal is to cater to the educational requirements of the dermatology community. Being the top journal in the field, JAAD publishes original articles that have undergone peer review. These articles primarily focus on clinical, investigative, and population-based studies related to dermatology. Another key area of emphasis is research on healthcare delivery and quality of care. JAAD also highlights high-quality, cost-effective, and innovative treatments within the field. In addition to this, the journal covers new diagnostic techniques and various other topics relevant to the prevention, diagnosis, and treatment of skin, hair, and nail disorders.