Vol 11, No 2 (2020)
Review paper
Published online: 2020-09-02

open access

Page views 927
Article views/downloads 2426
Get Citation

Connect on Social Media

Connect on Social Media

Pharmacological treatment of primary immune thrombocytopenia in adults

Krzysztof Chojnowski1
Hematologia 2020;11(2):73-81.


The treatment goal in patients with primary immune thrombocytopenia (ITP) is to prevent severe bleeding, the risk of which is increased if the platelet count (PLT) remains below 20–30 G/L. First-line treatment includes corticosteroids and/or intravenous immunoglobulins, which are effective in 70–90% of patients. In most cases, initial treatment will not lead to a sustained increase in PLT. Further treatment may include pharmacotherapy or splenectomy. Of all the therapeutic options, splenectomy offers the best chance of a permanent cure. Moreover a lot of ITP patients do not agree to splenectomy and some patients have contraindications to the operation. These patients should be treated with a thrombopoietin receptor agonist or rituximab. Unfortunately, in Poland, these agents are not reimbursed by the National Health Fund in adult ITP patients with preserved spleen. For this reason, less effective and more toxic drugs are still used as a second-line treatment.

Article available in PDF format

View PDF (Polish) Download PDF file


  1. Al-Samkari H, Kuter DJ. Immune thrombocytopenia in adults: modern approaches to diagnosis and treatment. Semin Thromb Hemost. 2020; 46(3): 275–288.
  2. Portielje JE, Westendorp RG, Kluin-Nelemans HC, et al. Morbidity and mortality in adults with idiopathic thrombocytopenic purpura. Blood. 2001; 97(9): 2549–2554.
  3. Rodeghiero F, Michel M, 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.
  4. Butros LJ, Bussel JB. Intracranial hemorrhage in immune thrombocytopenic purpura: a retrospective analysis. J Pediatr Hematol Oncol. 2003; 25(8): 660–664.
  5. 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.
  6. Matzdorff A, Meyer O, Ostermann H, et al. Immune thrombocytopenia — current diagnostics and therapy: recommendations of a Joint Working Group of DGHO, ÖGHO, SGH, GPOH, and DGTI. Oncol Res Treat. 2018; 41(Suppl 5): 1–30.
  7. Neunert C, Terrell DR, Arnold DM, et al. American Society of Hematology 2019 guidelines for immune thrombocytopenia. Blood Adv. 2019; 3(23): 3829–3866.
  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. Mithoowani S, Gregory-Miller K, Goy J, et al. High-dose dexamethasone compared with prednisone for previously untreated primary immune thrombocytopenia: a systematic review and meta-analysis. Lancet Haematol. 2016; 3(10): e489–e496.
  10. 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.
  11. Wong RSM, Saleh MN, 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.
  12. 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.
  13. Ghanima W, Cooper N, Rodeghiero F, et al. Thrombopoietin receptor agonists: ten years later. Haematologica. 2019; 104(6): 1112–1123.
  14. Cooper KL, Fitzgerald P, Dillingham K, et al. Romiplostim and eltrombopag for immune thrombocytopenia: methods for indirect comparison. Int J Technol Assess Health Care. 2012; 28(3): 249–258.
  15. Cantoni S, Carpenedo M, Mazzucconi MG, et al. Alternate use of thrombopoietin receptor agonists in adult primary immune thrombocytopenia patients: A retrospective collaborative survey from Italian hematology centers. Am J Hematol. 2018; 93(1): 58–64.
  16. Birocchi S, Podda GM, Manzoni M, et al. Thrombopoietin receptor agonists for the treatment of primary immune thrombocytopenia: a meta-analysis and systematic review. Platelets. 2020 [Epub ahead of print]: 1–11.
  17. 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.
  18. 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.
  19. 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.
  20. 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.
  21. 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.
  22. Taylor A, Neave L, Solanki S, et al. Mycophenolate mofetil therapy for severe immune thrombocytopenia. Br J Haematol. 2015; 171(4): 625–630.
  23. 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.
  24. Verlin M, Laros RK, Penner JA. Treatment of refractory thrombocytopenic purpura with cyclophosphamine. Am J Hematol. 1976; 1(1): 97–104.
  25. 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.
  26. 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.
  27. 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.
  28. 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.

Hematology in Clinical Practice