Efficacy and safety of sirolimus in the treatment of blue rubber bleb naevus syndrome in paediatric patients
B. Zhang L. Li N. Zhang M. Zhao Y. Liu L. Wei L. Ma Z. Xu
First published: 10 May 2019 https://doi.org/10.1111/ced.14003
Conflict of interest: the authors declare that they have no conflicts of interest.
LM and ZX contributed equally to this work and should be considered joint corresponding authors.
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Summary
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 in the signalling pathway of angiogenesis and subsequent development of BRBNS and its use has been described in several case reports. We present a case series of four patients with BRBNS who exhibited good treatment response to sirolimus. All four patients were administered oral sirolimus at doses of 1.0–1.5 mg/m2/day with a target drug level of 5–10 ng/mL and median treatment duration of 20 months. All patients had a reduction in the size of the lesions and a normalization of coagulopathy with tolerable drug adverse reactions at follow‐up. Sirolimus may be effective and safe in paediatric patients with BRBNS. Further prospective studies are suggested to evaluate the long‐term effectiveness of this drug.
B. Zhang L. Li N. Zhang M. Zhao Y. Liu L. Wei L. Ma Z. Xu
First published: 10 May 2019 https://doi.org/10.1111/ced.14003
Conflict of interest: the authors declare that they have no conflicts of interest.
LM and ZX contributed equally to this work and should be considered joint corresponding authors.
Read the full text
ePDFPDFTOOLS SHARE
Summary
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 in the signalling pathway of angiogenesis and subsequent development of BRBNS and its use has been described in several case reports. We present a case series of four patients with BRBNS who exhibited good treatment response to sirolimus. All four patients were administered oral sirolimus at doses of 1.0–1.5 mg/m2/day with a target drug level of 5–10 ng/mL and median treatment duration of 20 months. All patients had a reduction in the size of the lesions and a normalization of coagulopathy with tolerable drug adverse reactions at follow‐up. Sirolimus may be effective and safe in paediatric patients with BRBNS. Further prospective studies are suggested to evaluate the long‐term effectiveness of this drug.
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