目的 探讨口服西罗莫司治疗儿童微囊型淋巴管畸形的疗效。方法 微囊型淋巴管畸形患儿9例,均给予口服西罗莫司治疗,剂量0.05～0.10 mg/Kg·d,分1～2次口服,连续服用3～6个月。服药期间每个月复诊1次,观察瘤体大小及并发症情况。结果 所有患者随访6～18个月,7例早期治疗的患儿疗效较好,瘤体均有明显萎缩,2例超过10岁开始治疗的患儿疗效欠佳,瘤体无明显缩小。患儿服药期间血常规、血糖、血压、心功能和肝肾功能检查均未发现明显异常,仅1例患儿出现免疫力下降情况,但可耐受。结论 口服西罗莫司治疗儿童微囊型淋巴管畸形安全、有效,且早期治疗十分必要。

Objective To explore the efficacy of sirolimus in treatment of microcystic lymphatic malformation in children.Methods Nine children with microcystic lymphatic malformation were selected and treated with sirolimus orally.The sirolimus was taken orally with the dose of 0.05~0.10 mg/Kg·d in 1-2 times.The treatment lasted for 3-6 months and further consultation was required once a month during the treatment.Tumor size and complications were observed.Results All patients were followed up for 6-18 months.In 7 cases who received early treatment,the curative effect was good with obvious atrophy of the tumor,and in 2 cases who started treatment when they were over 10 years old,the curative effect was poor with no obvious shrinking of the tumor.No obvious abnormalities were found in blood routine,blood glucose,blood pressure,heart function,liver and kidney function.Only 1 patient had immune decline,but it was tolerable.Conclusion Sirolimus is effective and safe for the treatment of microcystic lymphatic malformation in children and early treatment is necessary.