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Editorials
have a major impact on the way in which pediatric hema-
tologists approach the long standing “To treat or not to
treat” question when evaluating children with suspected 545.
ITP at the time of or soon after presentation22 (Figure 1). This study also highlights a compelling need for careful study of children now receiving TRA for other indica- tions, including hereditary thrombocytopenia and associ- ated qualitative platelet disorders, neonatal thrombocy- topenia, and thrombocytopenia following chemotherapy or bone marrow transplantation (reviewed by Neunert and Rose23).
References
1. Tarantino MD, Bussel JB, Blanchette VS, et al. Long-term treatment with romiplostim and treatment-free platelet responses in children with chronic immune thrombocytopenia. Haematologica. 2019; 104(11):2283-2291.
2. Bennett CM, Neunert C, Grace RF, et al. Predictors of remission in children with newly diagnosed immune thrombocytopenia: Data from the Intercontinental Cooperative ITP Study Group Registry II participants. Pediatr Blood Cancer. 2018;65(1).
3. Neunert CE, Buchanan GR, Imbach P, et al. Bleeding manifestations and management of children with persistent and chronic immune thrombocytopenia: data from the Intercontinental Cooperative ITP Study Group (ICIS). Blood. 2013;121(22):4457-4462.
4. Shaw J, Kilpatrick K, Eisen M, Tarantino M. The incidence and clin- ical burden of immune thrombocytopenia in pediatric patients in the United States. Platelets. 2019:1-8.
5. Cooper N. A review of the management of childhood immune thrombocytopenia: how can we provide an evidence-based approach? Br J Haematol. 2014;165(6):756-767.
6. ProvanD,StasiR,NewlandAC,etal.Internationalconsensusreport on the investigation and management of primary immune thrombo- cytopenia. Blood. 2010;115(2):168-186.
7. Neunert C, Lim W, Crowther M, Cohen A, Solberg L Jr, Crowther MA; American Society of Hematology. The American Society of Hematology 2011 evidence-based practice guideline for immune thrombocytopenia. Blood. 2011;117(16):4190-4207.
8. Klaassen RJ, Blanchette V, Burke TA, et al. Quality of life in child- hood immune thrombocytopenia: international validation of the kids' ITP tools. Pediatr Blood Cancer. 2013;60(1):95-100.
9. Ghanima W, Cooper N, Rodeghiero F, Godeau B, Bussel JB. Thrombopoietin receptor agonists: ten years later. Haematologica. 2019;104(6):1112-1123.
11. CinesDB,GernsheimerT,WasserJ,etal.Integratedanalysisoflong- term safety in patients with chronic immune thrombocytopaenia (ITP) treated with the thrombopoietin (TPO) receptor agonist romi- plostim. Int J Hematol. 2015;102(3):259-270.
12. Bussel JB, Buchanan GR, Nugent DJ, et al. A randomized, double- blind study of romiplostim to determine its safety and efficacy in children with immune thrombocytopenia. Blood. 2011;118(1):28-36.
13. Tarantino MD, Bussel JB, Blanchette VS, et al. Romiplostim in chil- dren with immune thrombocytopenia: a phase 3, randomised, dou- ble-blind, placebo-controlled study. Lancet. 2016;388(10039):45-54.
14. MathiasSD,LiX,EisenM,CarpenterN,CrosbyRD,BlanchetteVS. A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Determine the Effect of Romiplostim on Health-Related Quality of Life in Children with Primary Immune Thrombocytopenia and Associated Burden in Their Parents. Pediatr Blood Cancer. 2016;63(7):1232-1237.
15. Tumaini Massaro J, Chen Y, Ke Z. Efficacy and safety of throm- bopoietin receptor agonists in children with chronic immune throm- bocytopenic purpura: meta-analysis. Platelets. 2019;30(7):828-835.
16. KimTO,DespotovicJ,LambertMP.Eltrombopagforuseinchildren with immune thrombocytopenia. Blood Adv. 2018;2(4):454-461.
17. Neunert C, Despotovic J, Haley K, et al. Thrombopoietin Receptor
Agonist Use in Children: Data From the Pediatric ITP Consortium of North America ICON2 Study. Pediatr Blood Cancer. 2016;63 (8):1407-1413.
18. Grainger JD, Blanchette VS, Grotzinger KM, Roy A, Bussel JB. Health-related quality of life in children with chronic immune thrombocytopenia treated with eltrombopag in the PETIT study. Br J Haematol. 2019;185(1):102-106.
19. Lambert MP, Witmer CM, Kwiatkowski JL. Therapy induced iron deficiency in children treated with eltrombopag for immune throm- bocytopenia. Am J Hematol. 2017;92(6):E88-E91.
20. Kao YR, Chen J, Narayanagari SR, et al. Thrombopoietin receptor- independent stimulation of hematopoietic stem cells by eltrom- bopag. Sci Transl Med. 2018;10(458).
21. Grace RF, Shimano KA, Bhat R, et al. Second-line treatments in chil- dren with immune thrombocytopenia: Effect on platelet count and patient-centered outcomes. Am J Hematol. 2019;94(7):741-750.
22. Neunert CE, Cooper N. Evidence-based management of immune thrombocytopenia: ASH guideline update. Hematology Am Soc Hematol Educ Program. 2018;2018(1):568-575.
23. NeunertCE,RoseMJ.Romiplostimforthemanagementofpediatric immune thrombocytopenia: drug development and current practice. Blood Adv. 2019;3(12):1907-1915.
10. Saleh MN, Bussel JB, Cheng G, et al. Safety and efficacy of eltrom- bopag for treatment of chronic immune thrombocytopenia: results of the long-term, open-label EXTEND study. Blood. 2013;121(3):537-
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