背景:Kapososiform血管内皮瘤(KHE)是一种局部侵袭性和潜在致死性的婴儿期血管肿瘤。目前的共识建议包括使用长春新碱和/或全身性类固醇作为一线治疗。哺乳动物雷帕霉素靶(mTOR)抑制剂代表了KHE患者的有希望的治疗方法。我们研究的目的是严格评估现有文献中关于使用mTOR抑制剂治疗KHE患者的预后。
方法:我们从2000年1月1日至2022年4月30日进行了文献检索。纳入了报告使用mTOR抑制剂治疗KHE的患者结果的文章。描述性统计用于描述和总结治疗的结果。
结果:我们纳入了327例患者,诊断时平均年龄为9.1个月(SD±9)。患者用mTOR抑制剂治疗平均15.2个月(SD±4.1)。共有315例(96.3%)患者有积极的结果,包括肿瘤大小的改善。227例(85%)症状和/或实验室参数,38例(12%)完全缓解。7名(2%)患者对治疗无反应,7名(2%)患者死于败血症(4),卡萨巴赫-梅里特现象并发症(1),动脉导管引起的心脏和肝脏衰竭(1),或转移性疾病(1)。
结论:本系统综述支持mTOR抑制剂治疗KHE的有效性和安全性。他们的使用导致了积极的结果,减少症状,肿瘤大小的减少和生化指标的改善,死亡率为2%。根据这些发现,我们建议修订KHE联合mTOR抑制剂可能考虑一线治疗的共识治疗指南.
BACKGROUND: Kaposiform hemangioendothelioma (KHE) is a locally aggressive and potentially lethal vascular tumor of infancy. Current consensus recommendations include the use of vincristine and/or systemic steroids as first-line treatment. Mammalian target of rapamycin (mTOR) inhibitors represent a promising therapy for patients with KHE. The goal of our study is to critically assess the existing literature on outcomes of patients with KHE treated with mTOR inhibitors.
METHODS: We conducted a literature search from 1 January 2000, to 30 April 2022. Articles reporting outcomes of patients treated with mTOR inhibitors for KHE were included. Descriptive statistics were used to describe and summarize the outcomes of the treatment.
RESULTS: We included 327 patients with a mean age at diagnosis of 9.1 months (SD ± 9). Patients were treated with an mTOR inhibitor for a mean of 15.2 months (SD ± 4.1). A total of 315 (96.3%) patients had positive outcomes including improvement of the tumor size, symptoms and/or laboratory parameters in 227 (85%) and complete remission in 38 (12%). Seven (2%) patients did not respond to treatment and seven (2%) died of sepsis (4), Kasabach-Merritt phenomenon complications (1), cardiac and liver failure due to ductus arteriosus (1), or metastatic disease (1).
CONCLUSIONS: This systematic review supports the efficacy and safety of mTOR inhibitors for KHE. Their use resulted in positive outcomes in terms of decreased symptoms, reduction in tumor size and improvement in biochemical parameters with a mortality rate of 2%. According to these findings, we suggest revised consensus treatment guidelines for KHE with mTOR inhibitors potentially considered first-line therapy.