Journal of the American Academy of Dermatology · 2025
Efficacy and safety of high-vs low-dose sirolimus in patients with kaposiform hemangioendothelioma: A randomized clinical trial.
СтатьяМногоцентровое исследованиеРандомизированное исследованиеСравнительное исследованиеEquivalence TrialПоддержка исследования: вне правительства СШАНазвание и аннотация: Перевод пока не подготовлен
Обработка статьи · этапы, ошибки и управление
Загрузка состояния…
Ошибки и ограничения
История · было → стало
Состояние обновляется каждые 5 секунд, пока блок открыт. Процент показывается только там, где известен объём работы. Пауза останавливает продолжение этапа; текущий запрос к источнику может завершиться.
Zhou Jiangyuan, Lan Yuru, Qiu Tong, Zhang Zixin, Gong Xue, Zhang Xuepeng, Yang Congxia, Zhou Zilong, Zhang Yujia, Yang Min, Fu Jianlei, He Chunshui, Peng Qiang, Hu Fan, Xia Chunchao, Kong Feiteng, Chen Siyuan, Ji Yi
Аннотация
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.
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 and serious adverse events were similar between the 2 groups, but respiratory, skin, and mucosal adverse events were less common in the low-dose group.
LIMITATIONS: The patients enrolled were not associated with Kasabach-Merritt phenomenon.
CONCLUSION: Low-dose sirolimus is noninferior to high-dose sirolimus in treating KHE.
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 and serious adverse events were similar between the 2 groups, but respiratory, skin, and mucosal adverse events were less common in the low-dose group.
LIMITATIONS: The patients enrolled were not associated with Kasabach-Merritt phenomenon.
CONCLUSION: Low-dose sirolimus is noninferior to high-dose sirolimus in treating KHE.