Nadal Marion, Giraudeau Bruno, Tavernier Elsa, Jonville-Bera Annie-Pierre, Lorette Gerárd, Maruani Annabel
Department of Dermatology, University François Rabelais Tours, CHRU Tours, FR-37044 Tours, France.
Acta Derm Venereol. 2016 May;96(4):448-52. doi: 10.2340/00015555-2300.
Mammalian target of rapamycin (mTOR) inhibitors are a promising new treatment in vascular anomalies, but no published randomized controlled trials are available. The aim of this systematic review of all reported cases was to assess the efficacy and safety of mTOR inhibitors in all vascular anomalies, except cancers, in children and adults. In November 2014 MEDLINE, CENTRAL, LILACS and EMBASE were searched for studies of mTOR inhibitors in any vascular condition, except for malignant lesions, in humans. Fourteen publications and 9 posters, with data on 25 and 59 patients, respectively, all < 18 years old were included. Of these patients, 35.7% (n = 30) had vascular tumours, and 64.3% (n = 54) had malformations. Sirolimus was the most frequent mTOR inhibitor used (98.8%, n = 83). It was efficient in all cases, at a median time of 2 weeks (95% confidence interval 1-10 weeks). Sirolimus was well tolerated, the main side-effect being mouth sores, which led to treatment withdrawal in one case. The dosage of sirolimus was heterogeneous, the most common being 1.6 mg/m2/day.
雷帕霉素哺乳动物靶点（mTOR）抑制剂是一种有前景的治疗血管异常的新方法，但尚无已发表的随机对照试验。本系统综述所有报告病例的目的是评估mTOR抑制剂在儿童和成人中除癌症外的所有血管异常中的疗效和安全性。2014年11月，检索了MEDLINE、CENTRAL、LILACS和EMBASE，以查找有关mTOR抑制剂在除恶性病变外的任何人类血管疾病中的研究。纳入了14篇出版物和9张海报，分别有25例和59例患者的数据，所有患者均<18岁。在这些患者中，35.7%（n = 30）患有血管肿瘤，64.3%（n = 54）患有血管畸形。西罗莫司是最常用的mTOR抑制剂（98.8%，n = 83）。在所有病例中均有效，中位起效时间为2周（95%置信区间1 - 10周）。西罗莫司耐受性良好，主要副作用是口腔溃疡，有1例因此停药。西罗莫司的剂量各不相同，最常见的是1.6 mg/m²/天。
