小儿卡波西型血管内皮瘤的个体化治疗分析

目的总结分析小儿卡波西型血管内皮瘤（KHE）的临床特点，探讨其个体化治疗策略。 方法回顾性分析2016年1月至2020年3月江西省儿童医院收治的20例KHE患儿的临床资料，总结病例的临床特征、肿瘤性状、是否合并卡萨巴赫-梅里特现象（KMP）、治疗策略及转归等特点。 结果20例KHE患儿中女6例，男14例。年龄<1岁的患儿16例（80.0%），其中13例（65.0%）出生时即发现。合并KMP的患儿有8例，合并KMP与不合并KMP患儿在瘤体大小（P=0.009）、是否侵犯深部肌肉（P=0.003）的差异有统计学意义。合并KMP患儿中3例病灶行网状缝扎治疗，5例口服西罗莫司治疗；1例网状缝扎治疗无效后联合口服西罗莫司治疗。不合并KMP的12例患儿中，7例行肿瘤完全切除，2例行肿瘤部分切除后联合口服激素，2例病灶行网状缝扎治疗，1例口服西罗莫司；1例网状缝扎治疗无效后联合口服西罗莫司治疗。随访2~5年，合并KMP的患儿中1例实现瘤体接近消失，7例带瘤生存；不合并KMP的患儿中7例肿瘤完全切除，未见复发，5例无症状带瘤生存。 结论大多数KHE为婴儿期发病，肿瘤大小、病灶累及深部肌肉情况与患儿是否合并KMP相关。对于合并KMP的患儿，初步予以激素冲击治疗或输注血液制品稳定病情，病灶合适病例可先单纯进行网状缝扎治疗；如病灶不适合网状缝扎术或治疗无效，可单用或联合口服西罗莫司治疗。

Analysis of individualized treatment of Kaposiform hemangioendothelioma in infants

ObjectiveTo summarize and analyze the clinical characteristics of Kaposiform hemangioendothelioma (KHE) in infants and explore the individualized treatment strategies. MethodsThe clinical data of 20 infants with KHE treated in Jiangxi Provincial Children's Hospital from January 2016 to March 2020 were selected and analyzed. The general demographics, tumor characteristics, presence or absence of Kasabach-Merritt phenomenon (KMP), treatment strategies and outcome of the cases were retrospectively analyzed. The differences in clinical features between children with KMP and without KMP were analyzed. ResultsA total of 20 infants with KHE were enrolled, including 6 females (30.0%) and 14 males (70.0%). Sixteen infants (80.0%) were younger than 1 year old, and 13 of them (65.0%) were found at birth. There were 8 infants with KMP, and there were statistically significant differences in tumor size (P=0.009) and invasion of deep muscle (P=0.003) between children with and without KMP. Among the children with KMP, 3 cases were treated with mesh suture, and the other 5 were given oral sirolimus. 1 case was treated with oral sirolimus after mesh suture therapy failed. Among the 12 infants without KMP, 7 received complete tumor resection, 2 received partial tumor resection combined with oral glucocorticoid, 2 received mesh suture therapy, and 1 received oral sirolimus. 1 case was treated with oral sirolimus after mesh suture therapy failed. The patients were followed up for 2-5 years after treatment. Among the infants with KMP, the tumor was nearly disappeared in 1 case, and 7 cases survived with tumor. In the infants without KMP, 7 cases of complete tumor resection were effective and no recurrence was observed, and 5 cases achieved asymptomatic survival with tumor. ConclusionsMost of KHE cases are in infancy, and the size of tumor and the involvement of the lesion in the deep muscle are related to KMP. For infants complicated with KMP, initial high-dose glucocorticoid implosive therapy or infusion of blood products to stabilize the disease, suitable lesions can be treated with mesh suture first. If the lesion is not suitable for mesh suture or does not respond to treatment, oral sirolimus alone or in combination may be used.