Chinese Journal of Dermatology ›› 2022, Vol. 55 ›› Issue (2): 129-134.doi: 10.35541/cjd.20210629

• Original Articles • Previous Articles     Next Articles

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 Online:2022-02-15 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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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