• Cindy Neunert, Jenny Despotovic, Kristina Haley, Michele P Lambert, Kerri Nottage, Kristin Shimano, Carolyn Bennett, Robert Klaassen, Kimo Stine, Alexis Thompson, Yves Pastore, Travis Brown, Peter W Forbes, Rachael F Grace, and Pediatric ITP Consortium of North America (ICON).
• Division of Pediatric Hematology/Oncology/Stem Cell Transplantation and New York Presbyterian Morgan Stanley Children's Hospital, Columbia University Medical Center, New York, New York.
• Pediatr Blood Cancer. 2016 Aug 1; 63 (8): 1407-13.

BackgroundData on second-line treatment options for pediatric patients with immune thrombocytopenia (ITP) are limited. Thrombopoietin receptor agonists (TPO-RA) provide a nonimmunosuppressive option for children who require an increased platelet count.ProcedureWe performed a multicenter retrospective study of pediatric ITP patients followed at ITP Consortium of North America (ICON) sites to characterize TPO-RA use.ResultsSeventy-nine children had a total of 87 treatments (28 eltrombopag, 43 romiplostim, and eight trialed on both). The majority had primary ITP (82%) and most (60.8%) had chronic ITP. However, 22% had persistent ITP and 18% had newly diagnosed ITP. During the first 3 months of treatment, 89% achieved a platelet count ≥ 50 × 10(9) /l (86% romiplostim, 81% eltrombopag, P = 0.26) at least once in the absence of rescue therapy. The average time to a response was 6.4 weeks for romiplostim and 7.0 weeks for eltrombopag (P = 0.83). Only 40% of patients demonstrated a stable response with consistent dosing over time. An intermittent response with constant dose titration was seen in 15%, and an initial response that waned to no response was seen in 13%. Significant adverse events were minimal with the exception of two patients with thrombotic events and one who developed a neutralizing antibody.ConclusionsOur results demonstrate that TPO-RA agents are being used in children with ITP of varying duration and severity. The response was similar to clinical trials, but the sustainability of response varied. Future studies need to focus on the ideal timing and rationale for these medications in pediatric patients.© 2016 Wiley Periodicals, Inc.

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