Treatment of severe thrombocytopenia with IL-11 in children with Wiskott-Aldrich syndrome.

This report examines the safety and efficacy of IL-11 in treating severe thrombocytopenia secondary to Wiskott-Aldrich syndrome in two pediatric patients before allogeneic stem cell transplantation (SCT). Both patients had a substantial increase in their platelet counts and a decrease in bleeding episodes and platelet transfusions. The median platelet count increased from 32,000/mm3 to 64,000/mm3. Each subsequently received allogeneic SCT; 1 year after transplantation, both are reconstituted with 100% donor hematopoietic stem cells with sustained normal platelet counts (>200 K/mm3). Larger studies are required to confirm this observation of the safety and efficacy of IL-11 in this setting.