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.

Additional references:

• -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