Experience of 2nd line therapy with eltrombopag in patients with immune thrombocytopenia

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Abstract

   Background. Immune thrombocytopenia (ITP), or idiopathic thrombocytopenic purpura, is a hematological autoimmune disease characterized by bleeding and an isolated decrease in platelet count <100 × 109 / l. The decision to start treatment for ITP depends on several factors. The ITP treatment strategy is based on the clinical symptoms, with a focus on reducing the risk of severe bleeding and increasing platelet counts.

   Aim. To evaluate the efficacy of 2nd line therapy with the thrombopoietin receptor agonist eltrombopag in patients with ITP.

   Materials and methods. 490 patients with ITP are under observation at the center for orphan diseases of M. F. Vladimirskiy Moscow Regional Research Clinical Institute. The present study included 186 patients with primary ITP after 1st line glucocorticosteroid therapy. eltrombopag, a thrombopoietin receptor agonist, was prescribed as the 2nd line of therapy.

   Results. The median platelet count prior to eltrombopag therapy in all patients was 27.5 × 109 / l. after eltrombopag therapy, a significant (by 490 %) increase in platelet levels (median 135 × 109 / l) and a complete response according to clinical recommendations were noted.

   Conclusion. Glucocorticosteroids (prednisolone, dexamethasone) remain the drugs of choice for the 1st line of therapy. Treatment with drugs of this group in most cases allows achieving an optimal platelet level and preventing bleeding. In case of inefficiency, intolerance, occurrence of side effects, the appointment of thrombopoietin receptor agonists eltrombopag or romiplostim is recommended. therapy with eltrombopag at a dose of 50 mg daily for several weeks has been able to achieve an increase in platelet levels, correct hemorrhagic syndrome, reduce the number of side effects during first-line glucocorticosteroid therapy, and improve the patient’s quality of life.

About the authors

S. G. Zakharov

M. F. Vladimirskiy Moscow Regional Research Clinical Institute

Author for correspondence.
Email: hematologymoniki@mail.ru
ORCID iD: 0000-0003-2847-4374

Sergey Gennad’evich Zakharov

129110

61 / 2 Shchepkina St.

Moscow

Russian Federation

T. A. Mitina

M. F. Vladimirskiy Moscow Regional Research Clinical Institute

ORCID iD: 0000-0001-7493-0030

129110

61 / 2 Shchepkina St.

Moscow

Russian Federation

R. V. Vardanyan

M. F. Vladimirskiy Moscow Regional Research Clinical Institute

ORCID iD: 0000-0003-3842-4709

129110

61 / 2 Shchepkina St.

Moscow

Russian Federation

Z. R. Tekeeva

M. F. Vladimirskiy Moscow Regional Research Clinical Institute

ORCID iD: 0000-0001-8327-2500

129110

61 / 2 Shchepkina St.

Moscow

Russian Federation

I. N. Kontievskiy

M. F. Vladimirskiy Moscow Regional Research Clinical Institute

ORCID iD: 0000-0001-9337-6784

129110

61 / 2 Shchepkina St.

Moscow

Russian Federation

Yu. Yu. Chuksina

M. F. Vladimirskiy Moscow Regional Research Clinical Institute

ORCID iD: 0000-0002-4393-1759

129110

61 / 2 Shchepkina St.

Moscow

Russian Federation

A. V. Zakharova

United Hospital with Outpatient Department, Administrative Department of the President of the Russian Federation

ORCID iD: 0009-0008-0134-1049

119285

Build. 15, 6 Michurinskiy Prospekt

Moscow

Russian Federation

References

  1. Vrbensky J. The Characterization of CD8+ T cells as a potential mechanism of disease in immune thrombocytopenia. Hamilton, Ontario, Canada: McMaster University, 2022.
  2. Neunert C., Lim W., Crowther M. et al. The American Society of Hematology 2011 evidence­based practice guideline for immune thrombocytopenia. Blood 2011;117(16):4190–207. doi: 10.1182/blood-2010-08-302984
  3. Moulis G., Palmaro A., Montastruk J.L. et al. Epidemiology of incident immune thrombocytopenia: a nationwide population based study in France. Blood 2014;124(22):3308–15. doi: 10.1182/blood-2014-05-578336
  4. Neunert C., Terrell D., Arnold D. et al. American Society of Hematology 2019 guidelines for immune thrombocytopenia. Blood Adv 2019;3(23):3829–66. doi: 10.1182/bloodadvances.2019000966
  5. McKenzie C.G.J., Guo L., Freedman J., Semple J.W. Cellular immune dysfunction in immune thrombocytopenia (ITP). Br J Haematol 2013;163(1):10–23. doi: 10.1111/bjh.12480
  6. Shulman N.R., Marder V.J., Weinrach R.S. Similarities between known antiplatelet antibodies and the factor responsible for thrombocytopenia in idiopathic purpura. Physiologic, serologic and isotopic studies. Ann N Y Acad Sci 2006;124(2):499–542. doi: 10.1111/j.1749-6632.1965.tb18984.x
  7. Rank A., Weigert O., Ostermann H. Management of chronic immune thrombocytopenic purpura: targeting insufficient megakaryopoiesis as a novel therapeutic principle. Biologics 2010;4:139–45. doi: 10.2147/btt.s3436
  8. Ji X., Zhang L., Peng J., Hou M. T cell immune abnormalities in immune thrombocytopenia. J Hematol Oncol 2014;7:72. doi: 10.1186/s13045-014-0072-6
  9. Vrbensky J.R., Nazy I., Clare R. et al. T cell-­mediated autoimmunity in immune thrombocytopenia. Eur J Haematol 2022;108(1):18–27. doi: 10.1111/ejh.13705
  10. D’Orazio J.A., Neely J., Farhoudi N. ITP in Children. J Pediatr Hematol Oncol 2013;35(1):1–13. doi: 10.1097/MPH.0b013e318271f457
  11. Provan D., Arnold D., Bussel J.B. et al. Updated international consensus report on the investigation and management of primary immune thrombocytopenia. Blood Adv 2019;3(22):3780–817. doi: 10.1182/bloodadvances.2019000812
  12. Page L.K., Psaila B., Prowan D. et al. The immune thrombo­ cytopenic purpura (ITP) bleeding score: assessment of bleeding in patients with ITP. Br J Haematol 2007; 138(2):245–8. doi: 10.1111/j.1365-2141.2007.06635.x
  13. Cines D.B., Liebman H.A. The immune thrombocytopenia syndrome: a disorder of diverse pathogenesis and clinical presentation. Hematol Oncol Clin North Am 2009;23(6):1155–61. doi: 10.1016/j.hoc.2009.09.003
  14. Matzdorff A., Meyer O., Ostermann H. et al. Immune throm­ bocytopenia – 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. doi: 10.1159/000492187
  15. Jaime­Pérez J.C., Aguilar­Calderon P., Jimenez­ Castillo R.A. et al. Treatment outcomes and chronicity predictors for primary immune thrombocytopenia: 10­-year data from an academic center. Ann Hematol 2020;99(11):2513–20. doi: 10.1007/s00277-020-04257-2

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