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.
Braithwaite
Kiery
K
Department of Pediatrics and the Lombardi Cancer Center, Georgetown University Medical Center, Washington, DC, USA.
Abu-Ghosh
Amal
A
Anderson
Lauren
L
Cairo
Mitchell S
MS
D002675:Child, Preschool; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D007223:Infant; D017370:Interleukin-11 / Q000627:therapeutic use*; D008297:Male; D010976:Platelet Count; D013921:Thrombocytopenia / Q000473:pathology / Q000628:therapy*; D014923:Wiskott-Aldrich Syndrome / Q000097:blood / Q000175:diagnosis / Q000628:therapy*
D017370:Interleukin-11