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Immune Thrombocytopenia in Children: Consensus and Controversies

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Abstract

Newly diagnosed immune thrombocytopenia (ITP) is a relatively common disorder of childhood that does not require an exhaustive laboratory workup for diagnosis. A history and physical exam with a review of the peripheral smear are crucial for excluding secondary causes of thrombocytopenia. Several guidelines have been published to guide physicians in the management of ITP. However, the decision for treatment can be arduous. The management strategy should not be focussed on the platelet count but the severity of bleeding symptoms. Agents for treating acute ITP, including corticosteroids, immunoglobulin and anti-D immunoglobulin, do not seem to have a significant impact on the natural history of the disease. The majority of children with ITP do not need therapy and have a spontaneous resolution of the disease. Some children can develop chronic ITP that is not commonly life-threatening but can lead to impaired quality of life. Traditional therapies such as rituximab and splenectomy for chronic ITP are not without significant adverse effects. Thrombopoietin receptor agonists are newer agents for the treatment of chronic ITP and hold promise, however, their cost currently precludes use in most of the patients in low-middle-income countries. This review compares and contrasts the specific treatments available for the treatment of ITP to help the reader make a balanced choice. This review, based on a series of case examples, will help physicians in making decisions about choosing a practical management strategy for patients with newly diagnosed as well as chronic ITP.

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References

  1. Yong M, Schoonen WM, Li L, et al. Epidemiology of paediatric immune thrombocytopenia in the general practice research database. Br J Haematol. 2010;149:855–64.

    PubMed  Google Scholar 

  2. 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;117:4190–207.

    CAS  PubMed  Google Scholar 

  3. Imbach P, Kuhne T, Muller D, et al. Childhood ITP: 12 months follow-up data from the prospective registry I of the intercontinental childhood ITP study group (ICIS). Pediatr Blood Cancer. 2006;46:351–6.

    CAS  PubMed  Google Scholar 

  4. Arnold DM. Bleeding complications in immune thrombocytopenia. Hematology Am Soc Hematol Educ Program. 2015;2015:237–42.

    PubMed  Google Scholar 

  5. Friedman JN, Beck CE. Diagnosis and management of typical, newly diagnosed primary immune thrombocytopenia (ITP) of childhood. Paediatr Child Health. 2019;24:54–5.

    PubMed  PubMed Central  Google Scholar 

  6. Geddis AE, Balduini CL. Diagnosis of immune thrombocytopenic purpura in children. Curr Opin Hematol. 2007;14:520–5.

    PubMed  Google Scholar 

  7. Consolini R, Costagliola G, Spatafora D. The centenary of immune thrombocytopenia-part 2: revising diagnostic and therapeutic approach. Front Pediatr. 2017;5:179.

    PubMed  PubMed Central  Google Scholar 

  8. British Committee for Standards in Haematology General Haematology Task Force. Guidelines for the investigation and management of idiopathic thrombocytopenic purpura in adults, children and in pregnancy. Br J Haematol. 2003;120:574–96.

    Google Scholar 

  9. Bryant N, Watts R. Thrombocytopenic syndromes masquerading as childhood immune thrombocytopenic purpura. Clin Pediatr (Phila). 2011;50:225–30.

    Google Scholar 

  10. Provan D, Stasi R, Newland AC, et al. International consensus report on the investigation and management of primary immune thrombocytopenia. Blood. 2010;115:168–86.

    CAS  PubMed  Google Scholar 

  11. Calpin C, Dick P, Poon A, Feldman W. Is bone marrow aspiration needed in acute childhood idiopathic thrombocytopenic purpura to rule out leukemia? Arch Pediatr Adolesc Med. 1998;152:345–7.

    CAS  PubMed  Google Scholar 

  12. Bolton-Maggs PH, Moon I. Assessment of UK practice for management of acute childhood idiopathic thrombocytopenic purpura against published guidelines. Lancet. 1997;350:620–3.

    CAS  PubMed  Google Scholar 

  13. Neunert CE, Buchanan GR, Imbach P, et al. Severe hemorrhage in children with newly diagnosed immune thrombocytopenic purpura. Blood. 2008;112:4003–8.

    CAS  PubMed  PubMed Central  Google Scholar 

  14. Treutiger I, Rajantie J, Zeller B, Henter JI, Elinder G, Rosthoj S, NOPHO ITP Study Group. Does treatment of newly diagnosed idiopathic thrombocytopenic purpura reduce morbidity? Arch Dis Child. 2007;92:704–7.

    PubMed  PubMed Central  Google Scholar 

  15. Heitink-Polle KMJ, Uiterwaal CSPM, Porcelijn L, et al. Intravenous immunoglobulin vs observation in childhood immune thrombocytopenia: a randomized controlled trial. Blood. 2018;132:883–91.

    CAS  PubMed  Google Scholar 

  16. Buchanan GR, Adix L. Grading of hemorrhage in children with idiopathic thrombocytopenic purpura. J Pediatr. 2002;141:683–8.

    PubMed  Google Scholar 

  17. Carcao MD, Zipursky A, Butchart S, Leaker M, Blanchette VS. Short-course oral prednisone therapy in children presenting with acute immune thrombocytopenic purpura (ITP). Acta Paediatr Suppl. 1998;424:71–4.

    Google Scholar 

  18. Zhang J, Liang Y, Ai Y, et al. Eltrombopag versus romiplostim in treatment of children with persistent or chronic immune thrombocytopenia: a systematic review incorporating an indirect-comparison meta-analysis. Sci Rep. 2018;8:576.

  19. Bansal D, Rajendran A, Singhi S. Newly diagnosed immune thrombocytopenia: update on diagnosis and management. Indian J Pediatr. 2014;81:1033–41.

    PubMed  Google Scholar 

  20. Lioger B, Maillot F, Ternant D, Passot C, Paintaud G, Bejan-Angoulvant T. Efficacy and safety of anti-D immunoglobulins versus intravenous immunoglobulins for immune thrombocytopenia in children: systematic review and meta-analysis of randomized controlled trials. J Pediatr. 2019;204:225–33.e8.

    PubMed  Google Scholar 

  21. Despotovic JM, Lambert MP, Herman JH, et al. RhIG for the treatment of immune thrombocytopenia: consensus and controversy (CME). Transfusion. 2012;52:1126–36; quiz 1125.

    PubMed  PubMed Central  Google Scholar 

  22. Fujisawa K, Iyori H, Ohkawa H, et al. A prospective, randomized trial of conventional, dose-accelerated corticosteroids and intravenous immunoglobulin in children with newly diagnosed idiopathic thrombocytopenic purpura. Int J Hematol. 2000;72:376–83.

  23. Kuhne T. Diagnosis and management of immune thrombocytopenia in childhood. Hamostaseologie. 2017;37:36–44.

    PubMed  Google Scholar 

  24. Beck CE, Nathan PC, Parkin PC, Blanchette VS, Macarthur C. Corticosteroids versus intravenous immune globulin for the treatment of acute immune thrombocytopenic purpura in children: a systematic review and meta-analysis of randomized controlled trials. J Pediatr. 2005;147:521–7.

    CAS  PubMed  Google Scholar 

  25. Despotovic JM, Neunert CE. Is anti-D immunoglobulin still a frontline treatment option for immune thrombocytopenia? Hematology Am Soc Hematol Educ Program. 2013;2013:283–5.

    PubMed  Google Scholar 

  26. O'Brien SH, Ritchey AK, Smith KJ. A cost-utility analysis of treatment for acute childhood idiopathic thrombocytopenic purpura (ITP). Pediatr Blood Cancer. 2007;48:173–80.

    PubMed  Google Scholar 

  27. Mayer B, Salama A. Successful treatment of bleeding with tranexamic acid in a series of 12 patients with immune thrombocytopenia. Vox Sang. 2017;112:767–72.

    CAS  PubMed  Google Scholar 

  28. Qin YH, Zhou TB, Su LN, Lei FY, Zhao YJ, Huang WF. The efficacy of different dose intravenous immunoglobulin in treating acute idiopathic thrombocytopenic purpura: a meta-analysis of 13 randomized controlled trials. Blood Coagul Fibrinolysis. 2010;21:713–21.

    CAS  PubMed  Google Scholar 

  29. Psaila B, Petrovic A, Page LK, Menell J, Schonholz M, Bussel JB. Intracranial hemorrhage (ICH) in children with immune thrombocytopenia (ITP): study of 40 cases. Blood. 2009;114:4777–83.

    CAS  PubMed  PubMed Central  Google Scholar 

  30. 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;121:4457–62.

    CAS  PubMed  Google Scholar 

  31. Grainger JD, Bolton-Maggs PH, Godeau B, et al. Diagnosis and management of chronic ITP: comments from an ICIS expert group. Ann Hematol. 2010;89:11–7.

    PubMed  Google Scholar 

  32. Rodeghiero F. A critical appraisal of the evidence for the role of splenectomy in adults and children with ITP. Br J Haematol. 2018;181:183–95.

    CAS  PubMed  Google Scholar 

  33. Kuhne T, Blanchette V, Buchanan GR, et al. Splenectomy in children with idiopathic thrombocytopenic purpura: a prospective study of 134 children from the Intercontinental Childhood ITP Study Group. Pediatr Blood Cancer. 2007;49:829–34.

    Google Scholar 

  34. Ahmed R, Devasia AJ, Viswabandya A, et al. Long-term outcome following splenectomy for chronic and persistent immune thrombocytopenia (ITP) in adults and children: splenectomy in ITP. Ann Hematol. 2016;95:1429–34.

    CAS  PubMed  Google Scholar 

  35. Crary SE, Buchanan GR. Vascular complications after splenectomy for hematologic disorders. Blood. 2009;114:2861–8.

    CAS  PubMed  PubMed Central  Google Scholar 

  36. Bhatt NS, Bhatt P, Donda K, et al. Temporal trends of splenectomy in pediatric hospitalizations with immune thrombocytopenia. Pediatr Blood Cancer. 2018;65:e27072.

    PubMed  Google Scholar 

  37. Lucchini E, Zaja F, Bussel J. Rituximab in the treatment of immune thrombocytopenia: what is the role of this agent in 2019? Haematologica. 2019;104:1124–35.

    PubMed  PubMed Central  Google Scholar 

  38. Liang Y, Zhang L, Gao J, Hu D, Ai Y. Rituximab for children with immune thrombocytopenia: a systematic review. PLoS One. 2012;7:e36698.

    CAS  PubMed  PubMed Central  Google Scholar 

  39. Grace RF, Shimano KA, Bhat R, et al. Second-line treatments in children with immune thrombocytopenia: effect on platelet count and patient-centered outcomes. Am J Hematol. 2019;94:741–50.

    CAS  PubMed  PubMed Central  Google Scholar 

  40. Patel VL, Mahevas M, Lee SY, et al. Outcomes 5 years after response to rituximab therapy in children and adults with immune thrombocytopenia. Blood. 2012;119:5989–95.

    CAS  PubMed  PubMed Central  Google Scholar 

  41. Tarantino MD, Bussel JB, Blanchette VS, et al. Romiplostim in children with immune thrombocytopenia: a phase 3, randomised, double-blind, placebo-controlled study. Lancet. 2016;388:45–54.

    PubMed  Google Scholar 

  42. 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;104:2283–91.

    PubMed  PubMed Central  Google Scholar 

  43. 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;63:1407–13.

    CAS  Google Scholar 

  44. Tremblay G, Dolph M, Bhor M, et al. Cost-consequence model comparing eltrombopag and romiplostim in pediatric patients with chronic immune thrombocytopenia. Clinicoecon Outcomes Res. 2018;10:715–21.

    Google Scholar 

  45. Kim TO, Despotovic J, Lambert MP. Eltrombopag for use in children with immune thrombocytopenia. Blood Adv. 2018;2:454–61.

    PubMed  PubMed Central  Google Scholar 

  46. Damodar S, Viswabandya A, George B, Mathews V, Chandy M, Srivastava A. Dapsone for chronic idiopathic thrombocytopenic purpura in children and adults--a report on 90 patients. Eur J Haematol. 2005;75:328–31.

    CAS  PubMed  Google Scholar 

  47. Patel AP, Patil AS. Dapsone for immune thrombocytopenic purpura in children and adults. Platelets. 2015;26:164–7.

    CAS  PubMed  Google Scholar 

  48. Neunert CE, Rose MJ. Romiplostim for the management of pediatric immune thrombocytopenia: drug development and current practice. Blood Adv. 2019;3:1907–15.

    PubMed  PubMed Central  Google Scholar 

  49. Grainger JD, Locatelli F, Chotsampancharoen T, et al. Eltrombopag for children with chronic immune thrombocytopenia (PETIT2): a randomised, multicentre, placebo-controlled trial. Lancet. 2015;386:1649–58.

    CAS  Google Scholar 

  50. Bansal D, Bhamare TA, Trehan A, Ahluwalia J, Varma N, Marwaha RK. Outcome of chronic idiopathic thrombocytopenic purpura in children. Pediatr Blood Cancer. 2010;54:403–7.

    PubMed  Google Scholar 

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GS drafted the initial manuscript. DB and NAMW provided critical guidance and review of the manuscript. NAMW is the guarantor for this paper.

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Correspondence to Nicola A. M. Wright.

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Singh, G., Bansal, D. & Wright, N.A.M. Immune Thrombocytopenia in Children: Consensus and Controversies. Indian J Pediatr 87, 150–157 (2020). https://doi.org/10.1007/s12098-019-03155-4

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  • DOI: https://doi.org/10.1007/s12098-019-03155-4

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