Immune thrombocytopenia – a single centre experience
Authors:
V. Fiamoli 1; J. Blatný 1; P. Ovesná 2
Authors‘ workplace:
Oddělení dětské hematologie a biochemie, Fakultní nemocnice Brno
1; Institut biostatistiky a analýz s. r. o., Brno
2
Published in:
Čes-slov Pediat 2021; 76 (8): 431-439.
Category:
Original Papers
Overview
Objective: Current treatment options and their efficacy in patients with immune thrombocytopenia – a single centre experience.
Patients and methods: Between years 2009 and 2019, 153 children with immune thrombocytopenia aged 4 months to 17 years (79 boys and 74 girls) were treated at our department. Early diagnosis of immune thrombocytopenia was made in 101 (66%) children, whereas 21 (14%) patients developed a persistent form and 31 (20%) chronic form.
Results: In 52 (34%) patients, „watch and wait“ strategy was sufficient. At the time of diagnosis, 72 (47%) patients were treated with intravenous immunoglobulins, 20 (13%) with steroids only, and 9 (6%) required both intravenous immunoglobulins and steroids. A complete response was achieved in 128 (84%) patients, 12 (8%) patients achieved partial response, and 13 (8%) had no response at all. There were no significant differences in the best achieved response between boys and girls or children of different ages. When evaluating the efficacy of steroid versus intravenous immunoglobulins treatment, there was no significant difference in the best response between the two groups (p=0.793). At least one relapse of immune thrombocytopenia occurred in 38 (25%) patients.
Patients with persistent or chronic immune thrombocytopenia were commenced on thrombopoietin analogues – 14 (9%), rituximab – 4 (3%), mycophenolate mofetil – 2 (1%), and sirolimus – 2 (1%). Splenectomy had to be performed only in three (2%) patients with chronic immune thrombocytopenia who failed all other previous treatment attempts.
Conclusion: Immune thrombocytopenia is relatively frequent haematological diagnosis in children. Most patients have achieved at least partial remission after the first-line treatment. For children with chronic immune thrombocytopenia, there is currently relatively wide range of treatment options available – thrombopoietin analogues, rituximab, sirolimus, mycophenolate mofetil and other immunosuppressives. Splenectomy, on the other hand, is considered as a last option, mainly for its potential risk of severe infections.
Keywords:
treatment options – children – immune thrombocytopenia
Sources
1. Consolini R, legitimo A, Caparello MC. The cebtenary of imune thrombocytopeniea – Part 1: Revising nomenclature and pathogenesis. Front Pediatr 2016; 4: 102.
2. Kuwana M. Dysregulated negative immune regulators in immune thrombocytopenia. VOX 2014; 9: 217–222.
3. Cooper N, Bussel J. The pathogenesis of immune thrombocytopaenic purpura. Br J Haematol 2006; 133 (4): 364–374.
4. Johnsen J. Pathogenesis in immune thrombocytopenia: new insights. Hematology Am Soc Hematol Educ Program 2012; 2012: 306–312.
5. Olsson B, Andersson PO, Jernas M. T-cell mediated cytotoxicity towards platelets in chronic idiopathic thrombocytopenic purpura. Nat Med 2003; 9 (9): 1123–1124.
6. Yenicesu I, Yetgin S, Ozyurek E, Aslan D. Virus-associated immune thrombocytopenic purpura in childhood. Pediatr Hematol Oncol 2002; 19 (6): 433–437.
7. Smalisz-Skrzypczyk K, Romiszewski M, Matyasiak M, et al. The influence of primary cytomegfalovirus or Epstein-Barr virus infection on course of idiopathic trombocytopenic purpura. Adv Exp Med Biol 2016; 878: 83–88.
8. Shah I. Immune thrombocytopenic purpura: a presentation of HIV infection. J Int Assoc Provid AIDS Care 2013; 12 (2): 95–97.
9. Pacifico L, Osborn JF, Tromba V, et al. Helicobacter pylori infection and extragastric disorders in children: a critical update. Worl J Gastroenterol 2014; 20 (6): 1379–1401.
10. Sakuraya N, Murakami H, Uchiumi H, et al. Steroid-refractory chronic idiopathic thrombocytopenic purpura associated with hepatitis C virus infection. Eur J Haematol 2002; 68 (1): 49–53.
11. Reese JA, Nguyen LP, Buchanan GR, et al. Drug-induced thrombocytopenia in children. Pediatr Blood Cancer 2013; 60 (12): 1975–1981.
12. Notarangelo LD. Primary immunodeficiencies (PIDs) presenting with cytopenias. Haematology Am Soc Hematol Educ Program 2009; 1: 139–143.
13. Liu Y, Chen S, Sun Y, et al. Clinical characteristic of immune thrombocytopenia associated with autoimmune disease: a restrospective study. Medicine 2016; 95 (50): e5565.
14. Stern M, Buser AS, Lohri A, et al. Autoimmunity and malignancy in hematology – more than an association. Crit Rev Oncol Hematol 2007; 63 (2): 100–110.
15. Kashyap E, Venkatesh S, Chouldry VP. Post transfusion purpura in thalassemic child. Indian Pediatr 1997; 34 (3): 246–247.
16. Jaime-Perez JC, Aguilar-Calderon PE, Salazar-Cavazos L, Gomez-Almaguer D. Evans syndrome: clinical perspectives, biological insights and treatment modalities. J Blood Med 2018; 9: 171–184; 3: 158–169.
17. Consolini R, Legitimo A, Caparello MC. The centenary of immune thrombocytopenia – Part 2: Revising diagnostic and therapeutic approach. Front Pediatr 2017; 5:179.
18. Neunert C, Buchanan GR, Imbach P, et al. Intercontinental childhood ITP study Group Registry II Participants. Severe hemorrage in children with newly diagnosed immune thrombocytopenic purpura. Blood 2008; 112 (10): 4003–4008.
19. Imbach P, Kuhne T, Muller D, et al. Childhood ITP: 12 months follow-up data from prospective registry I of the Intercontinental Childhood ITP Study Group (ICIS) Pediatr Blood Cancer 2006; 46 (3): 351–356.
20. Rosthoj S, Rajante J, Treutiger I, et al. NOPHO ITP working group. Duration and morbidity of chronic immune thrombocytopenic purpura in children: five years follow-up of a Nordic cohort. Acta Paediatr 2012; 101 (7): 761–766.
21. Kozák T, Čermák J, Červinek L, et al. Doporučení ČHS pro diagnostiku a léčbu imunitní trombocytopenie. Transfuze Hematol Dnes 2017; 3: 158–169.
22. 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.
23. Neunert C, Terrell DR, Arnold AM, et al. American Society of Hematology 2019 guidelines for immune thrombocytopenia. Blood Adv 2019; 3 (23): 3829–3866.
24. Terell 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; 85 (3): 174–180.
25. Heitink-Polle 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; 124 (22): 3295.
26. Kühne T. Diagnosis and management of immune thrombocytopenia in childhood. Hämostaseologie 2017; 37: 36–44.
27. Celik M, Bulbul A, Aydogan G, et al. Comparison of anti-D immunoglobulin, methylprednisolone, or intravenous immunoglobulin therapy in newly diagnosed pediatric immune thrombocytopenic purpura. J Thromb Thrombolysis 2013; 35 (2): 228–233.
28. Bumess CB, Keating GM, Garnock-Jones KP. Eltrombopag: A review in pediatric chronic immune thrombocytopenia. Drugs 2016; 76 (8): 869–878.
29. 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 (10004): 1649–1658.
30. Bussel JB, Hsieh L, Buchanan GR, et al. Long-term use of thrombopoietin mimetic romiplostim in children with severe chronic thrombocytopenia (ITP). Pediatr Blood Cancer 2015; 62: 208–213.
31. Grace RF, Bennett CM, Ritchey AK, et al. Response to steroids predicts response to rituximab in pediatric chronic immune thrombocytopenia. Pediatr Blood Cancer 2012; 58 (2): 221–225.
32. 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): 5 52–558.
33. Mueller BU, Bennett CM, Feldman HA, et al. Glaser Pediatric Research Network. One year follow-up of children and adolescents with chronic immune thrombocytopenic purpura (ITP) treated with rituximab. Pediatr Blood Cancer 2009; 52 (2): 259–262.
34. 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 (25): 5989–5995.
35. Miano M, Ramenghi U, Russo G, et al. Mycophenolat mofetil for the treatment of children with immune thrombocytopenia and Evans syndrome. A retrospective data review from the Italian Association of Paeditatric Haematology/Oncology. Br J Haematol 2016; 175: 490–495.
36. 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; 39 (6): 420–424.
37. Miano M, Scalzone M, Perri K, et al. Mycophenolate mofetil and sirolimus as second or further line treatment in children with chronic refractory primitive or secondary autoimmune cytopenias: a single centre experience. Br J Haematol. 2015; 171: 247–253.
38. Rudolf K, Malý J. Kortikosteroidy a imunosupresiva v chronické léčbě. Prakt Lékáren 2011; 7 (5): 228–231.
39. Bennet CM, Rogers ZR, Kinnamon DD, et al. Prospective phase ½ study of rituximab in childhood and adolescent chronic immune thrombocytopenic purpura. Blood 2006; 107: 2639–2642.
40. Bussel JB, de Miguel PG, Despotovic JM, et al. Eltrombopag for the treatment of children with persistent and chronic immune thrombocytopenia (PETIT): a randomised, multicentre, placebo- controlled study. Lancet Haematol 2015 Aug; 2 (8): e315–e325.
41. Rubin LG, Schaffner W. Clinical practice. Care of asplenic patient. N Engl J Med 2014; 371 (4): 349–356.
Labels
Neonatology Paediatrics General practitioner for children and adolescentsArticle was published in
Czech-Slovak Pediatrics
2021 Issue 8
Most read in this issue
- Current views on the pathophysiology, diagnosis and treatment of immune thrombocytopenia in children
- Immune thrombocytopenia – a single centre experience
- The history of invasive blood pressure measurement
- Genu recurvatum congenitum