Wang Huaijie, Guo Xinkui, Duan Yitao, Zheng Baijun, Gao Ya
Department of Pediatric Surgery, Second Affiliated Hospital, Medical College of Xi'an, JiaoTong University, Xi'an, China.
Pediatr Dermatol. 2018 Sep;35(5):635-638. doi: 10.1111/pde.13600. Epub 2018 Jul 12.
Sirolimus has been used to manage various complex vascular anomalies. Kaposiform hemangioendothelioma and tufted angioma may develop Kasabach-Merritt phenomenon in infancy.
We retrospectively reviewed the clinical and laboratory data of eight patients with kaposiform hemangioendothelioma and tufted angioma who were initially treated using oral sirolimus in our center, including six with Kasabach-Merritt phenomenon.
Five girls and three boys seen between September 2012 and March 2015 were included. Age at initiation of sirolimus ranged from 30 days to 14 weeks (mean±SD 8.6 ± 3.5 weeks). Six of these eight patients had kaposiform hemangioendothelioma, and two had a tufted angioma. Platelet count before start of oral sirolimus ranged from 5 × 10 /L to 189 × 10 /L ((78.8 ± 65.2)×10 /L) and fibrinogen level from 68 to 215 mg/dL (123.1 ± 50.5 mg/dL). All patients received standard doses of sirolimus (0.05 mg/kg orally, twice daily) as initial therapy. All patients with thrombocytopenia or hypofibrinogenemia reached a normal platelet count and a normal fibrinogen level within 3 to 4 weeks after sirolimus treatment. Length of treatment ranged from 12 to 79 weeks (39.9 ± 15.3 weeks). Two patients developed grade 2 oral mucositis during treatment.
Sirolimus as first-line therapy shows great promise in the treatment of kaposiform hemangioendothelioma and tufted angioma.
西罗莫司已被用于治疗各种复杂的血管异常。卡波西样血管内皮瘤和簇状血管瘤在婴儿期可能会发生卡萨巴赫-梅里特现象。
我们回顾性分析了在我们中心最初接受口服西罗莫司治疗的8例卡波西样血管内皮瘤和簇状血管瘤患者的临床和实验室数据，其中6例有卡萨巴赫-梅里特现象。
纳入了2012年9月至2015年3月期间就诊的5名女孩和3名男孩。开始使用西罗莫司时的年龄为30天至14周（平均±标准差8.6±3.5周）。这8例患者中，6例患有卡波西样血管内皮瘤，2例患有簇状血管瘤。开始口服西罗莫司前的血小板计数为5×10⁹/L至189×10⁹/L（(78.8±65.2)×10⁹/L），纤维蛋白原水平为68至215mg/dL（123.1±50.5mg/dL）。所有患者均接受标准剂量的西罗莫司（0.05mg/kg口服，每日2次）作为初始治疗。所有血小板减少或纤维蛋白原血症患者在西罗莫司治疗后3至4周内血小板计数和纤维蛋白原水平恢复正常。治疗时间为12至79周（39.9±15.3周）。2例患者在治疗期间出现2级口腔黏膜炎。
西罗莫司作为一线治疗药物在卡波西样血管内皮瘤和簇状血管瘤的治疗中显示出巨大的前景。
