中华皮肤科杂志 ›› 2022, Vol. 55 ›› Issue (2): 129-134.doi: 10.35541/cjd.20210629

• 论著 • 上一篇    下一篇

外用西罗莫司治疗儿童浅表脉管畸形19例的临床疗效及安全性分析

何瑞1    张斌1，2    李丽1    尉莉1    张楠3    于彤4    阴捷5    徐子刚1    马琳1   

  1. 1首都医科大学附属北京儿童医院  国家儿童医学中心皮肤科，北京  100045；2郑州大学附属儿童医院  河南省儿童医院  郑州儿童医院皮肤科，郑州  450000；3首都医科大学附属北京儿童医院  国家儿童医学中心病理科，北京  100045；4首都医科大学附属北京儿童医院  国家儿童医学中心影像科，北京  100045；5首都医科大学附属北京儿童医院 国家儿童医学中心介入科，北京  100045 
  • 收稿日期:2021-08-27 修回日期:2021-12-01 发布日期:2022-01-27
  • 通讯作者: 马琳；徐子刚；张斌 E-mail:bch_maleen@aliyun.com；zigangxu@yahoo.com；dr.binzhang@163.com
  • 基金资助:
    北京医院管理局儿科学科协同发展中心专项（XTZD20180502）

Clinical efficacy and safety of topical sirolimus in the treatment of 19 cases of superficial vascular malformation in children

He Rui1, Zhang Bin1,2, Li Li1, Wei Li1, Zhang Nan3, Yu Tong4, Yin Jie5, Xu Zigang1, Ma Lin1#br#   

  1. 1Department of Dermatology, Beijing Children′s Hospital, Capital Medical University, National Center for Children′s Health, Beijing 100045, China; 2Department of Dermatology, Children′s Hospital Affiliated of Zhengzhou University, Henan Children′s Hospital, Zhengzhou Children′s Hospital, Zhengzhou 450000, China; 3Department of Pathology, Beijing Children′s Hospital, Capital Medical University, National Center for Children′s Health, Beijing 100045, China; 4Department of Radiology, Beijing Children′s Hospital, Capital Medical University, National Center for Children′s Health, Beijing 100045, China; 5Department of Intervention, Beijing Children′s Hospital, Capital Medical University, National Center for Children′s Health, Beijing 100045, China
  • Received:2021-08-27 Revised:2021-12-01 Published:2022-01-27
  • Contact: Ma Lin; Xu Zigang; Zhang Bin E-mail:bch_maleen@aliyun.com；zigangxu@yahoo.com；dr.binzhang@163.com
  • Supported by:
    Special Fund of the Pediatric Medical Coordinated Development Center of Beijing Hospitals Authority（XTZD20180502）

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摘要： 【摘要】 目的 研究外用西罗莫司乳膏治疗儿童浅表脉管畸形的有效性与安全性。方法 采用单中心前瞻性设计，纳入2019年9月至2020年9月就诊于首都医科大学附属北京儿童医院皮肤科脉管性疾病专业门诊的浅表脉管畸形患儿，予0.1%西罗莫司乳膏外用，通过影像学、皮肤镜、主观评定等指标，根据国际通用四级分类法评估疗效，同时监测用药期间的不良反应。采用t检验或单因素方差分析、Fisher精确检验法进行统计学分析。结果 纳入19例浅表脉管畸形患儿，男12例，女7例，年龄1 ~ 11.5岁，14例诊断为血管淋巴管畸形，3例淋巴管畸形，2例静脉畸形。16例发生在下肢，8例伴随疼痛，2例破溃，6例有既往治疗史。治疗6个月时疗效为Ⅰ级3例，Ⅱ级4例，Ⅲ级4例，Ⅳ级8例，有效16例，显效12例。6例同一病灶治疗6个月后长、厚、宽度（1.83 ± 0.84、1.00 ± 0.55、2.25 ± 1.25 cm）均低于治疗前（2.40 ± 0.95、1.35 ± 0.61、2.50 ± 1.34 cm），t值分别为5.22、10.25、3.73，均P < 0.05。患儿的性别、年龄、病史以及是否疼痛对疗效的影响差异无统计学意义（均P > 0.05），而脉管畸形的诊断分类不同对疗效的影响差异有统计学意义（P = 0.008）。19例患儿中，2例用药后局部有轻度烧灼感。治疗后1个月及6个月时西罗莫司血药浓度均低于1.0 ng/ml。结论 外用西罗莫司治疗儿童浅表脉管畸形疗效好且不良反应少见。

关键词: 血管畸形, 淋巴管畸形, 儿童, 西罗莫司

Abstract: 【Abstract】 Objective To investigate efficacy and safety of topical sirolimus cream in the treatment of superficial vascular malformation in children. Methods A single-center prospective study was carried out. Children with superficial vascular malformation were enrolled into this study from Vascular Anomalies Clinic, Beijing Children′s Hospital, Capital Medical University from September 2019 to September 2020, and treated with 0.1% sirolimus cream. The efficacy was evaluated according to an international four-level classification system through imaging examination, dermoscopy and subjective evaluation, and adverse reactions during the treatment were monitored. Statistical analysis was carried out by t test, univariate analysis of variance or Fisher′s exact test. Results A total of 19 children with superficial vascular malformations were enrolled, including 12 males and 7 females, aged 1 - 11.5 years. Fourteen children were diagnosed with vascular and lymphatic malformations, 3 with lymphatic malformations, and 2 with venous malformations. Sixteen children presented with lesions on the lower extremities, 8 were accompanied by pain, 2 presented with ulceration, and 6 had previous treatment history. After 6-month treatment, 3 patients achieved improvement of level Ⅰ, 4 of level Ⅱ, 4 of level Ⅲ, and 8 of level Ⅳ; 16 achieved improvement, and 12 achieved marked improvement. Six patients showed significantly decreased length, thickness and width of lesions after 6 months of treatment （1.83 ± 0.84 cm, 1.00 ± 0.55 cm, 2.25 ± 1.25 cm, respectively） compared with those before treatment （2.40 ± 0.95 cm, 1.35 ± 0.61 cm, 2.50 ± 1.34 cm, t = 5.22, 10.25, 3.73, respectively, all P < 0.05）. Gender, age, medical history and pain sensation did not significantly affect the therapeutic effect （all P > 0.05）, while diagnostic classification of vascular malformations significantly affected the therapeutic effect （P = 0.008）. Among the 19 children, 2 had mild local burning sensation after the treatment. After 1- and 6-month treatment, the blood concentrations of sirolimus were both below 1.0 ng/ml. Conclusion Topical sirolimus is effective and safe in the treatment of superficial vascular malformation in children.

Key words: Vascular malformations, Lymphatic abnormalities, Child, Sirolimus