English Polski
Tom 17, Nr 2 (2024)
Wytyczne / stanowisko ekspertów
Opublikowany online: 2024-09-05

dostęp otwarty

Wyświetlenia strony 281
Wyświetlenia/pobrania artykułu 481
Pobierz cytowanie

Eksport do Mediów Społecznościowych

Eksport do Mediów Społecznościowych

Polskie zalecenia postępowania w pierwotnej małopłytkowości immunologicznej u dorosłych opracowane przez Grupę ds. Hemostazy Polskiego Towarzystwa Hematologów i Transfuzjologów — aktualizacja 2024

Krzysztof Chojnowski1, Anna Klukowska2, Magdalena Łętowska3, Paweł Łaguna4, Wojciech Młynarski5, Andrzej Mital6, Jacek Musiał7, Jacek Treliński8, Anetta Undas9, Tomasz Urasiński10, Jerzy Windyga11, Joanna Zdziarska12, Maria Podolak‑Dawidziak13
Journal of Transfusion Medicine 2024;17(2):76-101.

Streszczenie

Pierwotna małopłytkowość immunologiczna (ITP) jest rzadko występującą chorobą, którą się rozpoznaje po wykluczeniu innych przyczyn małopłytkowości. Od opublikowania pierwszego wydania Wytycznych upłynęło prawie 14 lat [1], w ciągu których nastąpił istotny postęp w leczeniu ITP. Głównym celem obecnej pracy jest aktualizacja obowiązujących zasad postępowania w ITP. Niniejsze zalecenia ograniczają się tylko do leczenia dorosłych chorych na ITP, postępowanie u dzieci w tej chorobie zostanie opublikowane oddzielnie. W wytycznych pominięto również wtórną małopłytkowość immunologiczną. Chociaż można w niej uzyskać odpowiedź płytkową, stosując te same leki co w postaci pierwotnej, to podstawą leczenia postaci wtórnej jest usunięcie przyczyny immunizacji. Zalecenia zostały przygotowane na podstawie opublikowanych wyników badań i wytycznych przygotowanych przez ekspertów z innych krajów [2, 3]. Ostateczny kształt nadano dokumentowi podczas konferencji uzgodnieniowych z udziałem członków Grupy ds. Hemostazy Polskiego Towarzystwa Hematologów i Transfuzjologów, które się odbyły w 2023 i 2024 r. Adresatami zaleceń są przede wszystkim lekarze sprawujący bezpośrednią opiekę nad chorymi na ITP, a także lekarze pierwszego kontaktu.

Artykuł dostępny w formacie PDF

Pokaż PDF Pobierz plik PDF

Referencje

  1. Zawilska K, et al. Podolak‑Dawidziak M. Chojnowski K. : skie zalecenia postępowania w pierwotnej małopłytkowości immunologicznej opracowane przez Grupę ds. Hemostazy skiego Towarzystwa Transfuzjologów i Hematologów. Pol Arch Med Wewn. 2010; 120(supl.): 5–28.
  2. 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.
  3. Neunert CE, Arnold DM, Grace RF, et al. American Society of Hematology 2019 guidelines for immune thrombocytopenia. Blood Adv. 2019; 3(23): 3829–3866.
  4. Matzdorff A, et al. Meyer O. Ostermann H. : Immune thrombocytopenia – current diagnostics and therapy: recommendations of a Joint Working Group of DGHO ÖGHO SGH GPOH and DGTI. Oncol Res Treat. 2018; 41(supl. 5): 1–30.
  5. Kühne T, Berchtold W, Michaels LA, et al. Intercontinental Cooperative ITP Study Group. Newly diagnosed immune thrombocytopenia in children and adults: a comparative prospective observational registry of the Intercontinental Cooperative Immune Thrombocytopenia Study Group. Haematologica. 2011; 96(12): 1831–1837.
  6. Frederiksen H, Maegbaek ML, Nørgaard M. Twenty-year mortality of adult patients with primary immune thrombocytopenia: a Danish population-based cohort study. Br J Haematol. 2014; 166(2): 260–267.
  7. Schuff-Werner P, Steiner M, Fenger S, et al. Effective estimation of correct platelet counts in pseudothrombocytopenia using an alternative anticoagulant based on magnesium salt. Br J Haematol. 2013; 162(5): 684–692.
  8. Brighton TA, Evans S, Castaldi PA, et al. Prospective evaluation of the clinical usefulness of an antigen-specific assay (MAIPA) in idiopathic thrombocytopenic purpura and other immune thrombocytopenias. Blood. 1996; 88(1): 194–201.
  9. Kim KJ, Baek IW, Yoon CH, et al. Thrombotic risk in patients with immune thrombocytopenia and its association with antiphospholipid antibodies. Br J Haematol. 2013; 161(5): 706–714.
  10. Pierrot-Deseilligny Despujol C, Michel M, Khellaf M, et al. Antiphospholipid antibodies in adults with immune thrombocytopenic purpura. Br J Haematol. 2008; 142(4): 638–643.
  11. Liebman H. Other immune thrombocytopenias. Semin Hematol. 2007; 44(4 Suppl 5): S24–S34.
  12. Altintas A, Ozel A, Okur N, et al. Prevalence and clinical significance of elevated antinuclear antibody test in children and adult patients with idiopathic thrombocytopenic purpura. J Thromb Thrombolysis. 2007; 24(2): 163–168.
  13. Khellaf M, Chabrol A, Mahevas M, et al. Hydroxychloroquine is a good second-line treatment for adults with immune thrombocytopenia and positive antinuclear antibodies. Am J Hematol. 2014; 89(2): 194–198.
  14. Makar RS, Zhukov OS, Sahud MA, et al. Thrombopoietin levels in patients with disorders of platelet production: diagnostic potential and utility in predicting response to TPO receptor agonists. Am J Hematol. 2013; 88(12): 1041–1044.
  15. Al-Samkari H, Kuter DJ. Thrombopoietin level predicts response to treatment with eltrombopag and romiplostim in immune thrombocytopenia. Am J Hematol. 2018; 93(12): 1501–1508.
  16. Al-Samkari H, Kuter DJ. Immune thrombocytopenia in adults: modern approaches to diagnosis and treatment. Semin Thromb Hemost. 2020; 46(3): 275–288.
  17. Portielje JE, Westendorp RG, Kluin-Nelemans HC, et al. Morbidity and mortality in adults with idiopathic thrombocytopenic purpura. Blood. 2001; 97(9): 2549–2554.
  18. 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; 113(11): 2386–2393.
  19. Butros LJ, Bussel JB. Intracranial hemorrhage in immune thrombocytopenic purpura: a retrospective analysis. J Pediatr Hematol Oncol. 2003; 25(8): 660–664.
  20. Cohen YC, Djulbegovic B, Shamai-Lubovitz O, et al. The bleeding risk and natural history of idiopathic thrombocytopenic purpura in patients with persistent low platelet counts. Arch Intern Med. 2000; 160(11): 1630–1638.
  21. Provan D, Stasi R, Newland AC, et al. International consensus report on the investigation and management of primary immune thrombocytopenia. Blood. 2010; 115(2): 168–186.
  22. Neunert C, Lim W, Crowther M, et al. American Society of Hematology. The American Society of Hematology 2011 evidence-based practice guideline for immune thrombocytopenia. Blood. 2011; 117(16): 4190–4207.
  23. Wei Yu, Ji Xb, Wang Yw, et al. High-dose dexamethasone vs prednisone for treatment of adult immune thrombocytopenia: a prospective multicenter randomized trial. Blood. 2016; 127(3): 296–302; quiz 370.
  24. Matschke J, Müller-Beissenhirtz H, Novotny J, et al. A randomized trial of daily prednisone versus pulsed dexamethasone in treatment-naïve adult patients with immune thrombocytopenia: EIS 2002 study. Acta Haematol. 2016; 136(2): 101–107.
  25. Wong R, Saleh M, Khelif A, et al. Safety and efficacy of long-term treatment of chronic/persistent ITP with eltrombopag: final results of the EXTEND study. Blood. 2017; 130(23): 2527–2536.
  26. Kuter DJ, Bussel JB, Lyons RM, et al. Efficacy of romiplostim in patients with chronic immune thrombocytopenic purpura: a double-blind randomised controlled trial. Lancet. 2008; 371(9610): 395–403.
  27. Jurczak W, Chojnowski K, Mayer J, et al. Phase 3 randomised study of avatrombopag, a novel thrombopoietin receptor agonist for the treatment of chronic immune thrombocytopenia. Br J Haematol. 2018; 183(3): 479–490.
  28. Ghanima W, Cooper N, Rodeghiero F, et al. Thrombopoietin receptor agonists: ten years later. Haematologica. 2019; 104(6): 1112–1123.
  29. González-Porras JR, Godeau B, Carpenedo M. Switching thrombopoietin receptor agonist treatments in patients with primary immune thrombocytopenia. Ther Adv Hematol. 2019; 10: 2040620719837906.
  30. Zhang J, Liang Yi, Ai Y, et al. Eltrombopag versus romiplostim in treatment of adult patients with immune thrombocytopenia: A systematic review incorporating an indirect-comparison meta-analysis. PLoS One. 2018; 13(6): e0198504.
  31. Al-Samkari H, Jiang D, Gernsheimer T, et al. Adults with immune thrombocytopenia who switched to avatrombopag following prior treatment with eltrombopag or romiplostim: A multicentre US study. Br J Haematol. 2022; 197(3): 359–366.
  32. Guillet S, Crickx E, Azzaoui I, et al. Prolonged response after TPO-RA discontinuation in primary ITP: results of a prospective multicenter study. Blood. 2023; 141(23): 2867–2877.
  33. Podda GM, Fiorelli EM, Birocchi S, et al. Thrombopoietin receptor agonists for the treatment of primary immune thrombocytopenia: a meta-analysis and systematic review. Platelets. 2021; 32(2): 216–226.
  34. Cooper N, Hill QA, Grainger J, et al. Tapering and discontinuation of thrombopoietin receptor agonist therapy in patients with immune thrombocytopenia: results from a modified Delphi Panel. Acta Haematol. 2021; 144(4): 418–426.
  35. Chugh S, Darvish-Kazem S, Lim W, et al. Rituximab plus standard of care for treatment of primary immune thrombocytopenia: a systematic review and meta-analysis. Lancet Haematol. 2015; 2(2): e75–e81.
  36. Chapin J, Lee CS, Zhang H, et al. Gender and duration of disease differentiate responses to rituximab-dexamethasone therapy in adults with immune thrombocytopenia. Am J Hematol. 2016; 91(9): 907–911.
  37. Bussel J, Arnold DM, Grossbard E, et al. Fostamatinib for the treatment of adult persistent and chronic immune thrombocytopenia: Results of two phase 3, randomized, placebo-controlled trials. Am J Hematol. 2018; 93(7): 921–930.
  38. Bussel JB, Arnold DM, Boxer MA, et al. Long-term fostamatinib treatment of adults with immune thrombocytopenia during the phase 3 clinical trial program. Am J Hematol. 2019; 94(5): 546–553.
  39. Strich J, Tian X, Samour M, et al. Fostamatinib for the Treatment of Hospitalized Adults With Coronavirus Disease 2019: A Randomized Trial. Clinical Infectious Diseases. 2021; 75(1): e491–e498.
  40. Lucchesi A, Fattizzo B, De Stefano V, et al. Use and positioning of fostamatinib in the management of primary chronic immune thrombocytopenia: an Italian expert opinion. Ther Adv Hematol. 2023; 14: 20406207221147777.
  41. Mishra K, Pramanik S, Sandal R, et al. Safety and efficacy of azathioprine in immune thrombocytopenia. Am J Blood Res. 2021; 11(3): 217–226.
  42. Choudhary DR, Naithani R, Mahapatra M, et al. Efficacy of cyclosporine as a single agent therapy in chronic idiopathic thrombocytopenic purpura. Haematologica. 2008; 93(10): e61–2; discussion e63.
  43. Kappers-Klunne MC, van't Veer MB. Cyclosporin A for the treatment of patients with chronic idiopathic thrombocytopenic purpura refractory to corticosteroids or splenectomy. Br J Haematol. 2001; 114(1): 121–125.
  44. Taylor A, Neave L, Solanki S, et al. Mycophenolate mofetil therapy for severe immune thrombocytopenia. Br J Haematol. 2015; 171(4): 625–630.
  45. Zhang Wg, Ji L, Cao Xm, et al. Mycophenolate mofetil as a treatment for refractory idiopathic thrombocytopenic purpura. Acta Pharmacol Sin. 2005; 26(5): 598–602.
  46. Verlin M. Laros R.K. Penner .A.: Treatment of refractory thrombocytopenic purpura with cyclophosphamine. Am J Hematol. 1976; 1: 97–104.
  47. Pizzuto J, Ambriz R. Therapeutic experience on 934 adults with idiopathic thrombocytopenic purpura: Multicentric Trial of the Cooperative Latin American group on Hemostasis and Thrombosis. Blood. 1984; 64(6): 1179–1183.
  48. Liu W, Gu X, Fu R, et al. The effect of danazol in primary immune thrombocytopenia: an analysis of a large cohort from a single center in China. Clin Appl Thromb Hemost. 2016; 22(8): 727–733.
  49. Godeau B, Durand JM, Roudot-Thoraval F, et al. Dapsone for chronic autoimmune thrombocytopenic purpura: a report of 66 cases. Br J Haematol. 1997; 97(2): 336–339.
  50. Estève C, Samson M, Guilhem A, et al. Efficacy and safety of dapsone as second line therapy for adult immune thrombocytopenia: A retrospective study of 42 patients. PLoS One. 2017; 12(10): e0187296.
  51. Stasi R, Newland A, Thornton P, et al. Should medical treatment options be exhausted before splenectomy is performed in adult ITP patients? A debate. Ann Hematol. 2010; 89(12): 1185–1195.
  52. Mikhael J, Northridge K, Lindquist K, et al. Short-term and long-term failure of laparoscopic splenectomy in adult immune thrombocytopenic purpura patients: a systematic review. Am J Hematol. 2009; 84(11): 743–748.
  53. Kojouri K, Vesely SK, Terrell DR, et al. Splenectomy for adult patients with idiopathic thrombocytopenic purpura: a systematic review to assess long-term platelet count responses, prediction of response, and surgical complications. Blood. 2004; 104(9): 2623–2634.
  54. Kühne T, Blanchette V, Buchanan GR, et al. Intercontinental Childhood ITP Study Group. 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(6): 829–834.
  55. Vianelli N, Palandri F, Polverelli N, et al. Splenectomy as a curative treatment for immune thrombocytopenia: a retrospective analysis of 233 patients with a minimum follow up of 10 years. Haematologica. 2013; 98(6): 875–880.
  56. Mageau A, Terriou L, Ebbo M, et al. Splenectomy for primary immune thrombocytopenia revisited in the era of thrombopoietin receptor agonists: New insights for an old treatment. Am J Hematol. 2022; 97(1): 10–17.
  57. Liu EH, Dilip CK, Yeh TS, et al. Long-term relapse-free rurvival rate and predictive factors of idiopathic thrombocytopenic purpura in adults undergoing splenectomy. Biomed J. 2013; 36(1): 23–27.
  58. Duperier T, Brody F, Felsher J, et al. Predictive factors for successful laparoscopic splenectomy in patients with immune thrombocytopenic purpura. Arch Surg. 2004; 139(1): 61–6; discussion 66.
  59. Aleem A. Durability and factors associated with long term response after splenectomy for primary immune thrombocytopenia (ITP) and outcome of relapsed or refractory patients. Platelets. 2011; 22(1): 1–7.
  60. Juliá A, Araguás C, Rosselló J, et al. Lack of useful clinical predictors of response to splenectomy in patients with chronic idiopathic thrombocytopenic purpura. Br J Haematol. 1990; 76(2): 250–255.
  61. Sarpatwari A, Provan D, Erqou S, et al. Autologous 111 In-labelled platelet sequestration studies in patients with primary immune thrombocytopenia (ITP) prior to splenectomy: a report from the United Kingdom ITP Registry. Br J Haematol. 2010; 151(5): 477–487.
  62. Ejstrud P, Kristensen B, Hansen JB, et al. Risk and patterns of bacteraemia after splenectomy: a population-based study. Scand J Infect Dis. 2000; 32(5): 521–525.
  63. Bisharat N, Omari H, Lavi I, et al. Risk of infection and death among post-splenectomy patients. J Infect. 2001; 43(3): 182–186.
  64. Boyle S, White RH, Brunson A, et al. Splenectomy and the incidence of venous thromboembolism and sepsis in patients with immune thrombocytopenia. Blood. 2013; 121(23): 4782–4790.
  65. Thomsen RW, Schoonen WM, Farkas DK, et al. Risk for hospital contact with infection in patients with splenectomy: a population-based cohort study. Ann Intern Med. 2009; 151(8): 546–555.
  66. Vianelli N, Galli M, de Vivo A, et al. Gruppo Italiano per lo Studio delle Malattie Ematologiche dell'Adulto. Efficacy and safety of splenectomy in immune thrombocytopenic purpura: long-term results of 402 cases. Haematologica. 2005; 90(1): 72–77.
  67. Crary SE, Buchanan GR. Vascular complications after splenectomy for hematologic disorders. Blood. 2009; 114(14): 2861–2868.
  68. Rodeghiero F, Ruggeri M. Short- and long-term risks of splenectomy for benign haematological disorders: should we revisit the indications? Br J Haematol. 2012; 158(1): 16–29.
  69. Arnold DM, Clerici B, Ilicheva E, et al. Refractory immune thrombocytopenia in adults: Towards a new definition. Br J Haematol. 2023; 203(1): 23–27.
  70. Miltiadous O, Hou M, Bussel JB. Identifying and treating refractory ITP: difficulty in diagnosis and role of combination treatment. Blood. 2020; 135(7): 472–490.
  71. Arnold DM, Nazi I, Santos A, et al. Combination immunosuppressant therapy for patients with chronic refractory immune thrombocytopenic purpura. Blood. 2010; 115(1): 29–31.
  72. Lv Y, Shi H, Liu H, et al. Current therapeutic strategies and perspectives in refractory ITP: What have we learned recently? Front Immunol. 2022; 13: 953716.
  73. Carr JM, Kruskall MS, Kaye JA, et al. Efficacy of platelet transfusions in immune thrombocytopenia. Am J Med. 1986; 80(6): 1051–1054.
  74. Salama A, Kiesewetter H, Kalus U, et al. Massive platelet transfusion is a rapidly effective emergency treatment in patients with refractory autoimmune thrombocytopenia. Thromb Haemost. 2008; 100(5): 762–765.
  75. Spahr JE, Rodgers GM. Treatment of immune-mediated thrombocytopenia purpura with concurrent intravenous immunoglobulin and platelet transfusion: a retrospective review of 40 patients. Am J Hematol. 2008; 83(2): 122–125.
  76. Care A, Pavord S, Knight M, et al. Severe primary autoimmune thrombocytopenia in pregnancy: a national cohort study. BJOG. 2018; 125(5): 604–612.
  77. Cines DB, Levine LD. Thrombocytopenia in pregnancy. Blood. 2017; 130(21): 2271–2277.
  78. Loustau V, Debouverie O, Canoui-Poitrine F, et al. Effect of pregnancy on the course of immune thrombocytopenia: a retrospective study of 118 pregnancies in 82 women. Br J Haematol. 2014; 166(6): 929–935.
  79. Guillet S, Loustau V, Boutin E, et al. Immune thrombocytopenia and pregnancy: an exposed/nonexposed cohort study. Blood. 2023; 141(1): 11–21.
  80. Wang X, Xu Y, Luo W, et al. Thrombocytopenia in pregnancy with different diagnoses: Differential clinical features, treatments, and outcomes. Medicine (Baltimore). 2017; 96(29): e7561.
  81. Rottenstreich A, Bussel JB. Treatment of immune thrombocytopenia during pregnancy with thrombopoietin receptor agonists. Br J Haematol. 2023; 203(5): 872–885.
  82. Bussel JB, Cooper N, Lawrence T, et al. Romiplostim use in pregnant women with immune thrombocytopenia. Am J Hematol. 2023; 98(1): 31–40.
  83. Crickx E, Mahévas M, Michel M, et al. Older Adults and Immune Thrombocytopenia: Considerations for the Clinician. Clin Interv Aging. 2023; 18: 115–130.
  84. Waisbren J, Dinner S, Altman J, et al. Disease characteristics and prognosis of myelodysplastic syndrome presenting with isolated thrombocytopenia. Int J Hematol. 2017; 105(1): 44–51.
  85. Piel‐Julian M, Mahévas M, Germain J, et al. Risk factors for bleeding, including platelet count threshold, in newly diagnosed immune thrombocytopenia adults. J Thromb Haemost. 2018; 16(9): 1830–1842.
  86. Palandri F, Catani L, Auteri G, et al. Understanding how older age drives decision-making and outcome in Immune Thrombocytopenia. A single centre study on 465 adult patients. Br J Haematol. 2019; 184(3): 424–430.