Cindy Neunert1, Jenny Despotovic2, Kristina Haley3, Michele P Lambert4, Kerri Nottage5, Kristin Shimano6, Carolyn Bennett7, Robert Klaassen8, Kimo Stine9, Alexis Thompson10, Yves Pastore11, Travis Brown12, Peter W Forbes13, Rachael F Grace12. 1. 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. 2. Texas Children's Hematology Center, Baylor College of Medicine, Houston, Texas. 3. Division of Pediatric Hematology/Oncology, Oregon Health and Sciences University, Portland, Oregon. 4. Department of Pediatrics and The Children's Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania. 5. St. Jude Children's Research Hospital, Memphis, Tennessee. 6. Division of Pediatric Hematology/Oncology and Benioff Children's Hospital, University of California San Francisco, San Francisco, California. 7. Aflac Cancer Center and Blood Disorders Service, Emory University School of Medicine and Children's Healthcare of Atlanta, Atlanta, Georgia. 8. Division of Pediatric Hematology/Oncology, University of Ottawa, Ottawa, Ontario. 9. Division of Pediatric Hematology/Oncology, University of Arkansas for Medical Sciences at Arkansas Children's Hospital, Little Rock, Arkansas. 10. Division of Hematology/Oncology, Feinberg School of Medicine, Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois. 11. CHU Ste-Justine, Montreal University, Montreal, Quebec, Canada. 12. Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Boston, Massachusetts. 13. Clinical Research Center, Boston Children's Hospital, Boston, Massachusetts.
Abstract
BACKGROUND: Data 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. PROCEDURE: We performed a multicenter retrospective study of pediatric ITP patients followed at ITP Consortium of North America (ICON) sites to characterize TPO-RA use. RESULTS: Seventy-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. CONCLUSIONS: Our 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.
BACKGROUND: Data 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. PROCEDURE: We performed a multicenter retrospective study of pediatric ITP patients followed at ITP Consortium of North America (ICON) sites to characterize TPO-RA use. RESULTS: Seventy-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. CONCLUSIONS: Our 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.
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