BACKGROUND: Autoimmune cytopenias are characterized by immune-mediated 
destruction of hematopoietic cell lines with immune thrombocytopenia (ITP) 
affecting platelets and Evans syndrome (ES) affecting platelets and red blood 
cells. For patients with persistent disease, limited options for effective and 
well-tolerated therapies exist.
OBJECTIVES: Our aim is to describe our institution's experience with sirolimus 
as therapy for pediatric patients with persistent ITP and ES.
DESIGN/METHOD: A retrospective analysis was performed in patients with 
persistent ITP and ES treated with sirolimus. Responses were categorized as 
complete response (CR), partial response, modest response, or no response.
RESULTS: Of the 17 patients treated, 12 had ITP and 5 had ES. Seventy-three 
percent of ITP patients achieved a CR, 78% of them by 3 months. Only 2 patients 
did not achieve a durable response. Eighty percent of ES patients had a 
response, with 50% of them achieving CR and the other 50% an asymptomatic 
partial response. One patient with ES achieved modest response, but discontinued 
therapy due to an adverse effect. Of the patients that achieved CR, 90% remain 
off all therapy for a median of 2 years.
CONCLUSIONS: Our data suggest that sirolimus is a safe and effective 
steroid-sparing agent in the treatment of persistent ITP and ES.