Sirolimus for management of GI bleeding in blue rubber bleb nevus syndrome: A case series. Issue 11 (12th September 2022)

• Record Type: Journal Article
• Authors: Duong, Jennifer T.; Geddis, Amy; Carlberg, Katie; Rudzinski, Erin; Len, Mary; Zheng, Hengqi B.
• Is Part Of: Pediatric blood & cancer. Volume 69:Issue 11(2022)
• Page Start: n/a
• Page End: n/a
• Publication Date: 2022-09-12
• Subjects: blue rubber bleb nevus syndrome -- GI bleeding -- sirolimus -- vascular malformation; Tumors in children -- Periodicals; Blood -- Diseases -- Periodicals; Cancer in children -- Periodicals; 618.92
• DOI: 10.1002/pbc.29970
• Languages: English
• ISSNs: 1545-5009

Abstract

Abstract: Blue rubber bleb nevus syndrome (BRBNS) commonly presents with anemia from bleeding gastrointestinal (GI) vascular malformations. Management is highly variable, as no consensus guidelines for medical treatment currently exist. Sirolimus has been used in BRBNS to decrease GI bleeding and seems well tolerated, though questions remain regarding dosing, duration of therapy, and adverse effects. Here, we report our single‐center experience of four pediatric patients with BRBNS who were successfully treated with sirolimus and review the existing literature regarding sirolimus for treatment of GI bleeding in BRBNS. Further prospective studies are needed to establish optimal dosage, drug monitoring, and duration.