Clinical effect of sirolimus in treatment of blue rubber bleb nevus syndrome in children: a report of 2 cases and literature review.
- Author:
Xu QUAN
1
;
Chun-Di XU
;
Ping LIU
;
Qing-Qing ZHANG
;
Ye CHEN
;
Le WANG
;
Yuan XIAO
Author Information
1. Department of Pediatrics, Ruijin Hospital North, Shanghai Jiao Tong University, School of Medicine, Shanghai 201821, China. xy11438@rjh.com.cn.
- Publication Type:Case Reports
- MeSH:
Adolescent;
Child;
Child, Preschool;
Gastrointestinal Neoplasms;
drug therapy;
Humans;
Infant;
Infant, Newborn;
Nevus, Blue;
drug therapy;
Prospective Studies;
Retrospective Studies;
Sirolimus;
therapeutic use;
Skin Neoplasms;
drug therapy
- From:
Chinese Journal of Contemporary Pediatrics
2020;22(9):1011-1016
- CountryChina
- Language:Chinese
-
Abstract:
To study the clinical effect of oral sirolimus in the treatment of children with blue rubber bleb nevus syndrome (BRBNS) in the gastrointestinal tract, a retrospective analysis was performed on the clinical data and follow-up results of two children with BRBNS treated by sirolimus. The two children with BRBNS had gastrointestinal bleeding and anemia and were treated with sirolimus at a dose of 1 mg/day as part of treatment. The plasma concentration of the drug was maintained between 2.5-12.0 ng/mL. The children showed disappearance of gastrointestinal bleeding and improvements in anemia and coagulation function, and blood transfusion could be stopped during treatment, with no obvious adverse drug reactions. PubMed, Wanfang Data, and CNKI were searched for related articles on sirolimus in the treatment of BRBNS. A total of 26 cases of children with BRBNS, aged 0-18 years, were obtained. With the addition of the 2 cases in this study, sirolimus treatment achieved a satisfactory clinical effect in all 28 cases. Sirolimus may be effective and safe in the treatment of children with BRBNS, and further prospective studies are needed to evaluate the long-term efficacy of this drug.
