Department of Pediatric Surgery, Children's Hospital of Fudan University, Shanghai, China.
Department of Pediatric Surgery, Shandong Dezhou People's Hospital, Shandong, China.
J Dermatol. 2019 Nov;46(11):956-961. doi: 10.1111/1346-8138.15076. Epub 2019 Sep 5.
Mammalian target of rapamycin inhibitors have shown promising results in the management of kaposiform hemangioendothelioma (KHE). The purpose of this study was to present our experience involving sirolimus therapy for KHE. A retrospective study was conducted to review the medical documents of 26 patients with KHE who were treated with sirolimus at our hospital between March 2012 and December 2016. Fifteen males and 11 females manifested KHE in infancy with an average age of 2.9 ± 1.8 months. Multiple anatomical sites were involved. Four patients had multifocal lesions, while 22 patients had solitary lesions. Twenty-five patients had Kasabach-Merritt phenomenon (KMP). Twenty patients completed sirolimus therapy in 28.3 ± 12.5 months. Nineteen KHE lesions reduced to small residuals with platelet counts reaching normal levels 3.7 ± 2.8 weeks after treatment; one KHE lesion had no response to therapy. One patient with multifocal lesions died due to a severe infection, although the patient had previously responded to sirolimus. Five patients remained in treatment and had good responses with normal platelet counts. Nineteen patients with anemia had normal hemoglobin levels after 3.5 ± 1.9 weeks of treatment. Mild side effects were observed. The median follow-up time was 32 months (26-60 months), with no evidence of recurrences. Sirolimus was shown to be efficacious in the management of KHE with an average course of 28 months. The time-to-response was variable, with an average of 1 week. After 4 weeks of treatment, the platelet count and hemoglobin level had normalized. Multifocal KHE with KMP is more severe than solitary KHE.
哺乳动物雷帕霉素靶蛋白抑制剂在卡波西样血管内皮细胞瘤（KHE）的治疗中显示出良好的效果。本研究旨在介绍我们使用西罗莫司治疗 KHE 的经验。对 2012 年 3 月至 2016 年 12 月在我院接受西罗莫司治疗的 26 例 KHE 患者的病历进行回顾性分析。男 15 例，女 11 例，发病年龄平均为 2.9±1.8 个月。多发病变 4 例，单发 22 例。25 例患者有卡波西肉瘤-梅里特现象（KMP）。20 例患者完成了 28.3±12.5 个月的西罗莫司治疗。19 例 KHE 病变缩小至小残余，血小板计数在治疗后 3.7±2.8 周内恢复正常；1 例 KHE 病变对治疗无反应。1 例多发病变患者因严重感染死亡，尽管患者此前对西罗莫司有反应。5 例患者仍在治疗中，血小板计数正常，反应良好。19 例贫血患者在治疗 3.5±1.9 周后血红蛋白水平正常。观察到轻微的副作用。中位随访时间为 32 个月（26-60 个月），无复发证据。西罗莫司治疗 KHE 有效，平均疗程为 28 个月。反应时间不同，平均为 1 周。治疗 4 周后，血小板计数和血红蛋白水平恢复正常。伴有 KMP 的多发病变 KHE 比单发 KHE 更严重。
