西罗莫司治疗复杂性血管畸形的研究进展

Sirolimus for the treatment of complex vascular malformations

血管畸形是一类良性先天性血管性病变的统称，可累及多个系统和脏器。复杂型血管畸形可以引起严重的并发症，导致毁形性损害、功能障碍甚至危及生命。西罗莫司是一种西罗莫司靶蛋白特异性抑制剂，常规应用于器官移植术后抗移植排斥反应。近年来将其用于治疗复杂性血管畸形取得良好疗效。目前报道，西罗莫司口服治疗最小年龄仅为14周龄，治疗的常用剂量为每天1．5，2mg／m2，所有患者治疗总有效率为20％～80％。常见的生物学不良反应包括血液系统改变，代谢改变。少见且严重的不良反应为间质性肺炎和潜在性免疫抑制。目前研究显示，西罗莫司治疗复杂血管性疾病可能有非常好的前景，但尚需更多循证医学的证据支持。

Vascular malformations are a heterogeneous group of benign congenital vascular disorders, and can involve multiple systems and organs. Complex vascular malformations can cause severe complications, and result in disfigurement, dysfunctions, and even threaten life. Sirolimus, a kind of specific inhibitor of mammalian target of rapamycin （roTOR）, is commonly used to prevent graft rejection after organ transplantation. In recent years, it has shown a favorable therapeutic effect on complex vascular malformations. The minimum age of patients receiving oral sirolimus that has ever been reported is only 14 weeks, the most common dosage is 1.5 - 2 mg/m2 per day, and the total response rate ranges from 20% to 80%. Common adverse biological reactions are hematological and metabolic changes. Interstitial pneumonitis and potential immunosuppression are rare and severe adverse reactions. Studies have shown that sirolimus has good prospects in the treatment of complex vascular malformations, but more evidence is needed to support its use in clinical practice.