Current views on the pathophysiology, diagnosis and treatment of immune thrombocytopenia in children
Authors:
D. Pospíšilová 1; P. Smíšek 2
Authors place of work:
Dětská klinika Fakultní nemocnice a Lékařské fakulty Univerzity Palackého, Olomouc
1; Klinika dětské hematologie a onkologie 2. LF UK a FN Motol, Praha
2
Published in the journal:
Čes-slov Pediat 2021; 76 (8): 460-473.
Category:
Review
Summary
The review summarizes the most recent knowledge of the epidemiology, pathophysiology, current diagnostic aproach and treatment strategies in immune thrombocytopenia in children.
Keywords:
splenectomy – Quality of life – Molecular mimicry – immune thrombocytopenia – antiplatelet antibodies – T-cell mediated platelet destruction – platelet desialylation – immunosupressive treatment – TPO mimetics
Zdroje
1. Terrell DR, Beebe LA, Vesely SK, et al. The incidence of immune thrombocytopenic purpura in children and adults: A critical review of published reports. Am J Hematol 2010 Mar; 85 (3): 174–180.
2. Moulis G, Palmaro A, Montastruc JL, et al. Epidemiology of incident immune thrombocytopenia: a nationwide population- based study in France. Blood 2014 Nov 20; 124 (22): 3308–3315.
3. Yong M, Schoonen WM, Li L, et al. Epidemiology of paediatric immune thrombocytopenia in the General Practice Research Database. Br J Haematol 2010 Jun; 149 (6): 855–864.
4. Kühne T. Diagnosis and management of immune thrombocytopenia in childhood. Hamostaseologie 2017 Jan 31; 37 (1): 36–44.
5. Rodeghiero F, Stasi R, Gernsheimer T, et al. Standardization of terminology, definitions and outcome criteria in immune thrombocytopenic purpura of adults and children: report from an international working group. Blood 2009 Mar 12; 113 (11): 2386–2393.
6. Neunert C, Lim W, Crowther M, et al. The American Society of Hematology 2011 evidence-based practice guideline for immune thrombocytopenia. Blood 2011 Apr 21; 117 (16): 4190–4207.
7. Provan D, Stasi R, Newland AC, et al. International consensus report on the investigation and management of primary immune thrombocytopenia. Blood 2010 Jan 14; 115 (2): 168–186.
8. Provan D, Arnold DM, Bussel JB, et al. Updated international consensus report on the investigation and management of primary immune thrombocytopenia. Blood Adv 2019; 3 (22): 3780–3817.
9. Neunert C, Terrell DR, Arnold DM, et al. American Society of Hematology 2019 guidelines for immune thrombocytopenia. Blood Adv 2019 Dec 10; 3 (23): 3829–3866.
10. Smalisz-Skrzypczyk K, Romiszewski M, Matyasiak M, et al. The influence of primary cytomegalovirus or Epstein-Barr virus infection on course of idiopathic trombocytopenic purpura. Adv Exp Med Biol 2016; 878: 83–88.
11. Shah I. Immune thrombocytopenic purpura: a presentation of HIV infection. J Int Assoc Provid AIDS Care 2013; 12 (2): 95–97.
12. Zhang W, Nardi MA, Borkowsky W, et al. Role of molecular mimicry of hepatitis C virus protein with platelet GPIIIa in hepatitis C-related immunologic thrombocytopenia. Blood 2009 Apr 23; 113 (17): 4086–4093.
13. Elalfy MS, Nugent D. Viruses, anti-viral therapy, and viral vaccines in children with immune thrombocytopenia. Semin Hematol 2016 Apr; 53 (Suppl 1): S70–S72.
14. O’Leary ST, Glanz JM, McClure DL, et al. The risk of immune thrombocytopenic purpura after vaccination in children and adolescents. Pediatrics 2012; 129 (2): 248–255.
15. Russo G, Miraglia V, Branciforte F, et al; AIEOP-ITP Study Group. Effect of eradication of Helicobacter pylori in children with chronic immune thrombocytopenia: a prospective, controlled, multicenter study. Pediatr Blood Cancer 2011; 56 (2): 273–278.
16. Treepongkaruna S, Sirachainan N, Kanjanapongkul S, et al. Absence of platelet recovery following Helicobacter pylori eradication in childhood chronic idiopathic thrombocytopenic purpura: a multi-center randomized controlled trial. Pediatr Blood Cancer 2009; 53 (1): 72–77.
17. Al-Samkari H, Kuter DJ. Antiplatelet antibody testing in immune thrombocytopenia and Evan’s syndrome: longitudinal serological evolution and relation to clinical features. Blood 2018; 132 (Suppl 1): 1137.
18. Al-Samkari H, Rosovsky RP, Karp Leaf RS, et al. A modern reassessment of glycoprotein-specific direct platelet autoantibody testing in immune thrombocytopenia. Blood Adv 2020; 4 (1): 9–18.
19. Chang M, Nakagawa PA, Williams SA, et al. Immune thrombocytopenic purpura (ITP) plasma and purified ITP monoclonal autoantibodies inhibit megakaryocytopoiesis in vitro. Blood 2003 Aug 1; 102 (3): 887–895.
20. Olsson B, Andersson PO, Jernas M, et al. T-cell-mediated cytotoxicity toward platelets in chronic idiopathic thrombocytopenic purpura. Nat Med 2003 Sep; 9 (9): 1123–1124.
21. Aslam R, Segel GB, Burack R et al. Splenic lymphocyte subtypes in immune thrombocytopenia: increased presence of a subtype of B-regulatory cells. Br J Haematol 2016; 173: 159–160.
22. Talaat RM, Elmaghraby AM, Barakat SS, et al. Alterations in immune cell subsets and their cytokine secretion profile in childhood idiopathic thrombocytopenic purpura (ITP). Clin Exp Immunol 2014 May; 176 (2): 291–300.
23. Morodomi Y, Kanaji S, Won E, et al. Mechanisms of anti-GPIbα antibody-induced thrombocytopenia in mice. Blood 2020 Jun 18; 135 (25): 2292–2301.
24. Li J, van der Wal DE, Zhu G, et al. Desialylation is a mechanism of Fc-independent platelet clearance and a therapeutic target in immune thrombocytopenia. Nat Commun 2015 Jul 17; 6: 7737.
25. Tao L, Zeng Q, Li J, et al. Platelet desialylation correlates with efficacy of first-line therapies for immune thrombocytopenia. J Hematol Oncol 2017 Feb 8; 10 (1): 46.
26. Neunert CE, Buchanan GR, Imbach P, et al. Severe hemorrhage in children with newly diagnosed immune thrombocytopenic purpura. Blood 2008 Nov 15; 112 (10): 4003–4008.
27. 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 May 30; 121 (22): 4457–4462.
28. Neunert C, Noroozi N, Norman G, et al. Severe bleeding events in adults and children with primary immune thrombocytopenia: a systematic review. J Thromb Haemost 2015 Mar; 13 (3): 457–464.
29. Psaila B, Petrovic A, Page LK, et al. Intracranial hemorrhage (ICH) in children with immune thrombocytopenia (ITP): study of 40 cases. Blood 2009 Nov 26; 114 (23): 4777–4783.
30. Elalfy M, Elbarbary N, Khaddah N, et al. Intracranial hemorrhage in acute and chronic childhood immune thrombocytopenic purpura over a ten-year period: an Egyptian multicenter study. Acta Haematol 2010; 123 (1): 59–63.
31. Hill QA, Newland AC. Fatigue in immune thrombocytopenia. Br J Haematol 2015 Jul; 170 (2): 141–149.
32. Blatt J, Weston G, Gold C. Fatigue as marker of thrombocytopenia in childhood idiopathic thrombocytopenic purpura. Pediatr Hematol Oncol 2011 Feb; 27 (1): 65–67.
33. Noris P, Klersy C, Gresele P, et al. Platelet size for distinguishing between inherited thrombocytopenias and immune thrombocytopenia: a multicentric, real life study. Br J Haematol 2013; 162 (1): 112–119.
34. Cybulska A, Meintker L, Ringwald J, et al. Measurements of immature platelets with haematology analysers are of limited value to separate immune thrombocytopenia from bone marrow failure. Br J Haematol 2017; 177 (4): 612–619.
35. Almazni I, Stapley R, Morgan NV. Inherited thrombocytopenia: Update on genes and genetic variants which may be associated with bleeding. Front Cardiovasc Med 2019 Jun 19; 6: 80.
36. Heitink-Pollé KM, Nijsten J, Boonacker CW, et al. Clinical and laboratory predictors of chronic immune thrombocytopenia in children: a systematic review and meta-analysis. Blood 2014 Nov 20; 124 (22): 3295–3307.
37. Beck CE, Nathan PC, Parkin PC, et al. 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 Oct; 147 (4): 521–527.
38. Heitink-Pollé KMJ, Uiterwaal CSPM, Porcelijn L, et al. Intravenous immunoglobulin vs observation in childhood immune thrombocytopenia: a randomized controlled trial. Blood 2018; 132 (9): 883–891.
39. Lioger B, Maillot F, Ternant D, et al. 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 Jan; 204: 225–233.
40. 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): e26736.
41. 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 Jul 7; 118 (1): 28–36.
42. 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 Jul 2; 388 (10039): 45–54.
43. Li H, Yang H, Liu WJ. Efficacy of romiplostim in the treatment of ITP in children: a meta-analysis. Eur Rev Med Pharmacol Sci 2018 Sep; 22 (18): 6162–6169.
44. Wong RSM, Saleh MN, Khelif A, et al. Safety and efficacy of longterm treatment of chronic/persistent ITP with eltrombopag: final results of the EXTEND study. Blood 2017; 130 (23): 2527–2536.
45. Grainger JD, Locatelli F, Chotsampancharoen T, et al. Eltrombopag for children with chronic immune thrombocytopenia (PETIT2): a randomised, multicentre, placebo-controlled trial. Lancet 2015 Oct 24; 386 (10004): 1649–1658.
46. Elgebaly AS, Ashal GE, Elfil M, et al. Tolerability and efficacy of Eltrombopag in chronic immune thrombocytopenia: Metaanalysis of randomized controlled trials. Clin Appl Thromb Hemost 2017 Nov; 23 (8): 928–937.
47. Marshall AL, Scarpone R, De Greef M, et al. Remissions after long-term use of romiplostim for immune thrombocytopenia. Haematologica 2016; 101 (12): e476–e478.
48. González-López TJ, Pascual C, Álvarez-Román MT, et al. Successful discontinuation of eltrombopag after complete remission in patients with primary immune thrombocytopenia. Am J Hematol 2015; 90 (3): E40–E43.
49. Mathias SD, Li X, Eisen M, et al. 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.
50. Grainger JD, Blanchette VS, Grotzinger KM, et al. Healthrelated quality of life in children with chronic immune thrombocytopenia treated with eltrombopag in the PETIT study. Br J Haematol 2019; 185 (1): 102–106.
51. Liang Y, Zhang L, Gao J, et al. Rituximab for children with immune thrombocytopenia: a systematic review. PLoS One 2012; 7 (5): e36698.
52. Qu M, Zhou J, Yang SJ, et al. Efficacy and safety of rituximab for minors with immune thrombocytopenia: a systematic review and meta-analysis. J Int Med Res 2020 Oct; 48 (10): 300060520962348.
53. Dai WJ, Zhang RR, Yang XC, et al. Efficacy of standard dose rituximab for refractory idiopathic thrombocytopenic purpura in children. Eur Rev Med Pharmacol Sci 2015 Jul; 19 (13): 2379–2383.
54. Parodi E, Rivetti E, Amendola G, et al. Long-term follow-up analysis after rituximab therapy in children with refractory symptomatic ITP: identification of factors predictive of a sustained response. Br J Haematol 2009; 144 (4): 552–558.
55. Miano M, Ramenghi U, Russo G, et al. Mycophenolate mofetil for the treatment of children with immune thrombocytopenia and Evans syndrome. A retrospective data review from the Italian Association of Paediatric Haematology/Oncology. Br J Haematol. 2016 Nov; 175 (3): 490–495.
56. Panigrahi A, Clark A, Myers J, et al. A novel immunomodulatory treatment involving mycophenolate mofetil and corticosteroids for pediatric autoimmune cytopenias. Pediatr Blood Cancer 2017 Feb; 64 (2): 287–293.
57. Mousavi-Hasanzadeh M, Bagheri B, Mehrabi S, et al. Sirolimus versus cyclosporine for the treatment of pediatric chronic immune thrombocytopenia: A randomized blinded trial. Int Immunopharmacol 2020 Nov; 88: 106895.
58. Jasinski S, Weinblatt ME, Glasser CL. Sirolimus as an effective agent in the treatment of immune thrombocytopenia (ITP) and Evans syndrome (ES): A single institution‘s experience. J Pediatr Hematol Oncol 2017 Aug; 39 (6): 420–424.
59. Liu AP, Cheuk DK, Lee AH, et al. Cyclosporin A for persistent or chronic immune thrombocytopenia in children. Ann Hematol 2016 Oct; 95 (11): 1881–1886.
60. Rodrigo C, Gooneratne L. Dapsone for primary immune thrombocytopenia in adults and children: an evidence-based review. J Thromb Haemost 2013 Nov; 11 (11): 1946–1953.
61. Rodeghiero F. A critical appraisal of the evidence for the role of splenectomy in adults and children with ITP. Br J Haematol 2018 Apr; 181 (2): 183–195.
62. Chaturvedi S, Arnold DM, McCrae KR. Splenectomy for immune thrombocytopenia: down but not out. Blood 2018 Mar 15; 131 (11): 1172–1182.
63. Heitink-Pollé KM, Haverman L, Annink KV, et al. Health-related quality of life in children with newly diagnosed immune thrombocytopenia. Haematologica 2014; 99 (9): 1525–1533.
64. Cooper N, Kruse A, Kruse C, et al. Immune thrombocytopenia (ITP) World Impact Survey (I-WISh): Impact of ITP on health-related quality of life. Am J Hematol 2021 Feb 1; 96 (2): 199–207.
65. Grainger JD, Young NL, Blanchette VS, et al. Quality of life in immune thrombocytopenia following treatment. Arch Dis Child 2013; 98 (11): 895–897.
66. Grainger JD, Blanchette VS, Grotzinger KM, et al. Healthrelated quality of life in children with chronic immune thrombocytopenia treated with eltrombopag in the PETIT study. Br J Haematol 2019; 185 (1): 102–106.
67. Kumar M, Lambert MP, Breakey V, et al; ITP Consortium of North America. Sports participation in children and adolescents with immune thrombocytopenia (ITP). Pediatr Blood Cancer 2015; 62 (12): 2223–2225.
68. Howell C, Scott K, Patel DR. Sports participation recommendations for patients with bleeding disorders. Transl Pediatr 2017; 6 (3): 174–180.
Štítky
Neonatology Paediatrics General practitioner for children and adolescentsČlánok vyšiel v časopise
Czech-Slovak Pediatrics
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