外用西罗莫司治疗儿童体表微囊型淋巴管畸形20例临床观察  

Clinical Observation on 20 Cases of Children's Microcystic Lymphatic Malformation Treated with Sirolimus

作　　者：徐伟洋 解芳[1] 赵磊[1] 皮梦奇 徐淼 丁语[1] XU Weiyang;XIE Fang;ZHAO Lei;PI Mengqi;XU Miao;DING Yu(Interventional Hemangioma&Vascular Surgery Department,Children's Hospital Affiliated to Zhengzhou University,Zhengzhou 450000,China)

机构地区：[1]郑州大学附属儿童医院介入血管瘤&,血管外科,河南郑州450000

出　　处：《中国皮肤性病学杂志》2023年第5期546-550,556,共6页The Chinese Journal of Dermatovenereology

摘　　要：目的探讨外用西罗莫司治疗儿童体表微囊型淋巴管畸形的有效性与安全性。方法2020年9月—2022年9月郑州大学附属儿童医院收治的20例经过传统手术、硬化治疗等无明显效果的体表微囊型淋巴管畸形患儿,男9例,女11例,年龄4~15岁。均予以西罗莫司制剂外用,疗程为12周,期间监测随访影像学及用药前后的病情转归等,并依据国家通用四级分类法评估疗效,同时监测治疗期间的不良反应。结果20例患儿外用西罗莫司治疗后病灶均有不同程度缩小,有效率95.00%(19/20),治疗12周时,疗效评价等级Ⅳ级7例,Ⅲ级7例,Ⅱ级5例,Ⅰ级1例。20例患儿中9例有疼痛症状,治疗后有明显缓解,治疗12周后疼痛评分0.56±0.53均较治疗前(2.33±0.50)相比,差异有统计学意义(P<0.05)。7例患儿同一病灶治疗12周后最大直径(1.54±0.86)cm均低于治疗前(2.61±0.89)cm,差异有统计学意义(P<0.05)。患儿的性别、年龄、病程、伴随症状、病灶部位、大小(7例患儿病变前直径)对疗效的影响差异无统计学意义(均P>0.05)。20例患儿,1例用药后有轻度瘙痒感,1例出现红斑,1例出现烧灼感,外用过程中均未出现破溃、渗出、色素沉着等严重不良反应。治疗4周及12周时西罗莫司血药浓度均低于2.0 ng/mL。结论外用西罗莫司治疗儿童体表微囊型淋巴管畸形疗效好,安全性高;但仍需继续探索准确浓度并积累更多用药经验。Objective To explore the efficacy and safety of external application of sirolimus in children with microcystic lymphatic malformation.Methods From September 2020 to September 2022,the Children's Hospital affiliated to Zhengzhou University treated 20 children with microcystic lymphatic malformation on the body surface who had no obvious effect after traditional surgery and sclerotherapy.There were 9 males and 11 females,aged from 4 to 15 years.The course of treatment of sirolimus was 12 weeks,and the prepared sirolimus preparation was used externally.Through imaging,subjective evaluation,and the prognosis of the disease before and after medication,etc.After the treatment,the curative effect was evaluated according to the national general four-level classification,and the adverse reactions during the treatment were monitored.Results The tumor size of 20 children treated with sirolimus was reduced to some extent,and the effective rate was 95.00%(19/20).After 12 weeks of treatment,7 cases were rated as GradeⅣ,7 cases as GradeⅢ,5 cases as GradeⅡand 1 case as GradeⅠ.In all children,9 patients had pain symptoms,which were significantly relieved after treatment.After 12 weeks of treatment,the pain score 0.56±0.53 was significantly higher than that before treatment(2.33±0.50)(P<0.05).The maximum diameter of the same lesion in 7 patients after 12 weeks of treatment(1.54±0.86)cm was lower than that before treatment(2.61±0.89)cm,(P<0.05).Gender,age,course of disease,accompanying symptoms,location and size of the lesion(diameter before lesion in 7 children)had no significant difference on the curative effect(P>0.05).Among the 20 children who took the drug,one patient developed mild pruritus,one erythema,and one a burning sensation.There were no serious adverse reactions such as ulceration,exudation and pigmentation during external use.The blood concentration of sirolimus was lower than 2.0 ng/mL at 4 weeks and 12 weeks after treatment.Conclusion Sirolimus for external use has good efficacy and safety in the treatm

关 键 词：淋巴管畸形 微囊型 儿童 西罗莫司 

分 类 号：R726.5[医药卫生—儿科]