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.