Vol 55, No 2 (2024)
Original research article
Published online: 2024-03-28

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Eltrombopag and high-dose dexamethasone as first-line treatment in children with newly diagnosed primary immune thrombocytopenia

Monika Richert-Przygońska1, Dominika Kołuda1, Mariusz Wysocki1
Acta Haematol Pol 2024;55(2):108-111.

Abstract

Introduction: Novel treatment strategies for newly diagnosed immune thrombocytopenia (ndITP) pediatric patients are required.

Material and methods: The aim of this study was to analyze the safety and efficacy of eltrombopag and dexamethasone when used as the first-line treatment in children with ndITP. Inclusion criteria: age 5–18 years, and ndITP with bleeding manifestation. Treatment course: 28 days of eltrombopag with oral dexamethasone in three repeated courses.

Results: A complete response was achieved in 90% of patients after the first week of treatment, and in all patients after the end of the treatment course. Durable and sustained platelet response was observed in 90% of patients after 12 months of follow-up.

Conclusions: Our finding support safety and efficacy of eltrombopag and dexamethasone as combined first-line therapy of ndITP in children.

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References

  1. Neunert CE, Cooper N. Evidence-based management of immune thrombocytopenia: ASH guideline update. Hematology Am Soc Hematol Educ Program. 2018; 2018(1): 568–575.
  2. 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.
  3. Neunert C, Terrell DR, Arnold DM, et al. American Society of Hematology 2019 guidelines for immune thrombocytopenia. Blood Adv. 2019; 3(23): 3829–3866.
  4. Shimano KA, Grace RF, Despotovic JM, et al. Phase 3 randomised trial of eltrombopag versus standard first-line pharmacological management for newly diagnosed immune thrombocytopaenia (ITP) in children: study protocol. BMJ Open. 2021; 11(8): e044885.
  5. 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.
  6. Colović M, Suvajdzic N, Colović N, et al. Mycophenolate mophetil therapy for chronic immune thrombocytopenic purpura resistant to steroids, immunosuppressants, and/or splenectomy in adults. Platelets. 2011; 22(2): 153–156.
  7. 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.
  8. Ma J, Fu LL, Chen ZP, et al. [Clinical study of pulsed high- dose dexamethasone treatment in 38 children with primary immune thrombocytopenic purpura]. Zhonghua Xue Ye Xue Za Zhi. 2016; 37(10): 912–915.
  9. Fu L, Ma J, Gu H, et al. An escalating treatment strategy for children with severe chronic immune thrombocytopenia: Preliminary report from a single center. Pediatr Blood Cancer. 2021; 68(6): e29006.
  10. Neunert C, Despotovic J, Haley K, et al. Pediatric ITP Consortium of North America (ICON). Thrombopoietin receptor agonist use in children: data from the pediatric ITP consortium of North America ICON2 study. Pediatr Blood Cancer. 2016; 63(8): 1407–1413.
  11. Oved JH, Lee CSY, Bussel JB. Treatment of children with persistent and chronic idiopathic thrombocytopenic purpura: 4 infusions of rituximab and three 4-day cycles of dexamethasone. J Pediatr. 2017; 191: 225–231.
  12. Youssef MAM, Salah Eldeen E, Elsayh KI, et al. High dose dexamethasone as an alternative rescue therapy for active bleeding in children with chronic ITP: clinical and immunological effects. Platelets. 2019; 30(7): 886–892.
  13. Burness CB, Keating GM, Garnock-Jones KP. Eltrombopag: a review in paediatric chronic immune thrombocytopenia. Drugs. 2016; 76(8): 869–878.
  14. 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; 2(8): e315–e325.
  15. Cuker A. Transitioning patients with immune thrombocytopenia to second-line therapy: Challenges and best practices. Am J Hematol. 2018; 93(6): 816–823.
  16. Mahévas M, Gerfaud-Valentin M, Moulis G, et al. Characteristics, outcome, and response to therapy of multirefractory chronic immune thrombocytopenia. Blood. 2016; 128(12): 1625–1630.
  17. Patel VL, Mahévas 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.
  18. Gómez-Almaguer D, Herrera-Rojas MA, Jaime-Pérez JC, et al. Eltrombopag and high-dose dexamethasone as frontline treatment of newly diagnosed immune thrombocytopenia in adults. Blood. 2014; 123(25): 3906–3908.
  19. Gómez-Almaguer D, Tarín-Arzaga L, Moreno-Jaime B, et al. High response rate to low-dose rituximab plus high-dose dexamethasone as frontline therapy in adult patients with primary immune thrombocytopenia. Eur J Haematol. 2013; 90(6): 494–500.
  20. Buchanan GR. Bleeding signs in children with idiopathic thrombocytopenic purpura. J Pediatr Hematol Oncol. 2003; 25 Suppl 1: S42–S46.
  21. Neunert CE, Buchanan GR, Imbach P, et al. Intercontinental Cooperative ITP Study Group Registry II Participants. Bleeding manifestations and management of children with persistent and chronic immune thrombocytopenia: data from the Intercontinental Cooperative ITP Study Group (ICIS). Blood. 2013; 121(22): 4457–4462.
  22. Bussel JB. Update on eltrombopag for ITP. Oncology (Williston Park). 2009; 23(13): 1177–1178.
  23. Bussel JB, Provan D, Shamsi T, et al. Effect of eltrombopag on platelet counts and bleeding during treatment of chronic idiopathic thrombocytopenic purpura: a randomised, double-blind, placebo-controlled trial. Lancet. 2009; 373(9664): 641–648.
  24. Ghanima W, Godeau B, Cines DB, et al. How I treat immune thrombocytopenia: the choice between splenectomy or a medical therapy as a second-line treatment. Blood. 2012; 120(5): 960–969.
  25. Kuter DJ. The biology of thrombopoietin and thrombopoietin receptor agonists. Int J Hematol. 2013; 98(1): 10–23.
  26. 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.