A case report describes the use of low-dose sirolimus (rapamycin) for treating blue rubber bleb nevus syndrome (BRBNS). (Pediatrics 2012; 129: e1080-84). This patient, an 8-year-old girl from Turkey) presented with massive gastrointestinal bleeding had multiple venous malformations all over her body, especially throughout her gastrointestinal tract.
Before instituting sirolimus, patient had failed several agents: prednisolone, interferon-α, propranolol, and aminocaproic acid. Sirolimus was dosed at 0.05-0.1 mg/kg as an antiangiogenic agent with a goal of maintaining a trough level between 1 and 5 ng/mL. The lesions decreased in size but did not disappear. Initially, patient received therapy with propranolol concomitantly. Dramatic improvements in hemoglobin were noted after starting sirolimus therapy.
- -Pediatr Blood Cancer 2011; 57: 1018-24. Use of sirolimus for complicated vascular anomalies in children
- -Pediatrics 2001; 107: 418. Case report of BRBNS
- -JPGN 2001; 33:183-188. Use of octreotide in BRBNS