Division of Oncology, Department of Paediatric Surgery, West China Hospital of Sichuan University, Chengdu, China.
Paediatric Intensive Care Unit, Department of Critical Care Medicine, West China Hospital of Sichuan University, Chengdu, China.
J Paediatr Child Health. 2019 Sep;55(9):1119-1124. doi: 10.1111/jpc.14370. Epub 2019 Jan 2.
Kaposiform haemangioendothelioma (KHE) is a rare, potentially life-threatening vascular tumour that is often associated with thrombocytopenia and coagulopathy, known as the Kasabach-Merritt phenomenon (KMP). Because of the rarity and complexity of KHE, the optimal paradigm for treating KHE has yet to be elucidated. We aim to assess the efficacy and safety of vincristine and sirolimus for the treatment of KHE.
A comprehensive review of the literature was conducted from January 1993 to June 2018. A total of 15 studies were selected for the meta-analysis. Five studies included 75 individuals and reported the response and side effects to vincristine in the treatment of KHE with or without KMP. A total of 10 studies that included 127 individuals reported the response and safety of sirolimus for treating KHE with or without KMP.
The pooled odds ratio (OR) for the effectiveness of vincristine was 0.72. The pooled OR for the effectiveness of sirolimus was 0.91. The side effects associated with vincristine during the treatment included neuropathy, abdominal pain, loss of appetite and mild elevations of aspartate aminotransferase (AST) and alanine aminotransferase (ALT). The side effects associated with sirolimus therapy included bronchitis; lymphopenia; elevated AST, ALT and platelets; hyperlipidaemia; opportunistic infection; mild reversible leukopenia; mucositis; fever; pain and skin rash/vomiting and diarrhoea.
This systematic review showed a high efficacy of vincristine and sirolimus in the treatment of KHE. Based on the available data in the literature, it appears that sirolimus is potentially an efficacious and safe treatment option for KHE. Further randomised, controlled trials are recommended.
卡波西样血管内皮细胞瘤（KHE）是一种罕见的、潜在威胁生命的血管肿瘤，常伴有血小板减少和凝血功能障碍，即卡-梅现象（KMP）。由于 KHE 的罕见性和复杂性，其最佳治疗模式尚未阐明。本研究旨在评估长春新碱和西罗莫司治疗 KHE 的疗效和安全性。
从 1993 年 1 月至 2018 年 6 月进行了全面的文献回顾。共选择了 15 项研究进行荟萃分析。其中 5 项研究共纳入 75 例患者，报告了 KHE 合并或不合并 KMP 患者应用长春新碱治疗的反应和不良反应。10 项研究共纳入 127 例患者，报告了西罗莫司治疗 KHE 合并或不合并 KMP 的反应和安全性。
长春新碱治疗效果的合并优势比（OR）为 0.72。西罗莫司治疗效果的合并 OR 为 0.91。长春新碱治疗相关的不良反应包括神经病变、腹痛、食欲下降和天门冬氨酸氨基转移酶（AST）、丙氨酸氨基转移酶（ALT）轻度升高。西罗莫司治疗相关的不良反应包括支气管炎、淋巴细胞减少、AST、ALT 和血小板升高、高脂血症、机会性感染、轻度可逆性白细胞减少、黏膜炎、发热、疼痛、皮疹/呕吐和腹泻。
本系统评价显示长春新碱和西罗莫司治疗 KHE 的疗效较高。基于现有文献数据，西罗莫司似乎是一种有效的、安全的 KHE 治疗选择。建议进一步开展随机对照试验。
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