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  • Title: Sirolimus in blue rubber bleb naevus syndrome: A systematic review.
    Author: Wong XL, Phan K, Rodríguez Bandera AI, Sebaratnam DF.
    Journal: J Paediatr Child Health; 2019 Feb; 55(2):152-155. PubMed ID: 30565378.
    Abstract:
    The aim of this study is to review sirolimus as a treatment for blue rubber bleb naevus syndrome (BRBNS). A literature search of Medline, Embase, CINAHL, SCOPUS and Google Scholar was conducted for publications reporting treatment of patients with BRBNS with sirolimus. Of 46 articles identified, 17 studies reporting 23 patients met inclusion criteria. Sirolimus was well tolerated in all but one patient who required treatment cessation; 17/18 patients noted an improvement in gastrointestinal disease where this was reported; 21/22 patients noted an improvement in cutaneous disease where this was reported. Based on these results, sirolimus may be considered a first-line treatment of BRBNS depending on patient morbidity.
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