Efficacy and safety of sirolimus in the treatment of blue rubber bleb naevus syndrome in paediatric patients

• DOI: 10.1111/ced.14003
• Publication Date: 2019-05-10T10:50:23Z
• Authors: B. Zhang; L. Li; N. Zhang; M. Zhao; Y. Liu; L. Wei; L. Ma; Z. Xu
• Keywords: Sirolimus

Blue rubber bleb naevus syndrome (BRBNS) is an extremely rare venous malformation that often manifests as multiple haemangioma‐like lesions in the skin and gastrointestinal tract. The drug sirolimus plays a key role signalling pathway of angiogenesis subsequent development BRBNS its use has been described several case reports. We present series four patients with who exhibited good treatment response to sirolimus. All were administered oral at doses 1.0–1.5 mg/m2/day target level 5–10 ng/mL median duration 20 months. had reduction size normalization coagulopathy tolerable adverse reactions follow‐up. Sirolimus may be effective safe paediatric BRBNS. Further prospective studies are suggested evaluate long‐term effectiveness this drug.