Vol 12, Supp. B (2021)
Case report
Published online: 2021-06-11
Eltrombopag in the treatment of pediatric chronic immune thrombocytopenia — a single-center experience
Abstract
Primary immune thrombocytopenia (ITP) is the most common bleeding disorder in pediatric population, defined as an isolated low platelet count below 100 G/l. ITP is an autoimmune disease, mostly associated with the presence of auto-antibodies against platelet membrane antigens. This mechanism leads to shorten platelets half-life and may also inhibit the platelets production in the bone marrow. Approximately 25% of patients develop chronic ITP (cITP), diagnosed if the thrombocytopenia lasts a minimum 12 months. Management of childhood cITP remains controversial and decision about treatment should be individualized, based on general condition, risk of bleeding and the impact on the quality of life. Therapeutic approach includes intravenous immunoglobulins, corticosteroids, oral immunosuppressants, rituximab and splenectomy. New thrombopoietin agonists seem to be an efficacious and safe therapeutic option in the strategy of management of pediatric cITP, both in monotherapy and in combination with other drugs.
Keywords: childhood chronic primary immunologic thrombocytopeniacITPtreatmenteltrombopag
References
- Dusza A, Matysiak M. Pierwotna małopłytkowość immunologiczna u dzieci. Nowa Pediatr. 2018; 22(4): 122–129.
- Del Vecchio GC, De Santis A, Accettura L, et al. Chronic immune thrombocytopenia in childhood. Blood Coagul Fibrinolysis. 2014; 25(4): 297–9.
- Faki Osman MEl. Childhood immune thrombocytopenia: clinical presentation and management. Sudan J Paediatr. 2012; 12(1): 27–39.
- Kühne T. Diagnosis and management of immune thrombocytopenia in childhood. Hamostaseologie. 2017; 37(1): 36–44.
- Cuker A, Cines DB, Neunert CE. Controversies in the treatment of immune thrombocytopenia. Curr Opin Hematol. 2016; 23(5): 479–485.
- Roganović J. Immune thrombocytopenia in children. Med Sci. 2015; 42: 59–72.
- Despotovic JM, Grimes AB. Pediatric ITP: is it different from adult ITP? Hematology Am Soc Hematol Educ Program. 2018; 2018(1): 405–411.
- Liu APY, Cheuk DKL, Lee AHY, et al. Cyclosporin A for persistent or chronic immune thrombocytopenia in children. Ann Hematol. 2016; 95(11): 1881–1886.
- Samson M, Fraser W, Lebowitz D. Treatments for primary immune thrombocytopenia: a review. Cureus. 2019; 11(10): e5849.
- Kim TO, Despotovic J, Lambert MP. Eltrombopag for use in children with immune thrombocytopenia. Blood Adv. 2018; 2(4): 454–461.
- 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.
- Revolade. Charakterystyka Produktu Leczniczego. https://ec.europa.eu/health/documents/community-register/2017/20170216137166/anx_137166_pl.pdf (November 30, 2020).
- 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.
- 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.
- Kuter DJ, Mufti GJ, Bain BJ, et al. Evaluation of bone marrow reticulin formation in chronic immune thrombocytopenia patients treated with romiplostim. Blood. 2009; 114(18): 3748–3756.
- Brynes RK, Wong R, Thein M, et al. A 2-year, longitudinal, prospective study of the effects of eltrombopag on bone marrow in patients with chronic immune thrombocytopenia. Acta Haematologica. 2016; 137(2): 66–72.
- Wong RSM, 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.
- Gudbrandsdottir S, Leven E, Imahiyerobo A, et al. Combination of thrombopoietin receptor agonists, immunosuppressants and intravenous immunoglobulin as treatment of severe refractory immune thrombocytopenia in adults and children. Br J Haematol. 2020; 189(2): e37–e40.
- Gómez-Almaguer D. Eltrombopag-based combination treatment for immune thrombocytopenia. Ther Adv Hematol. 2018; 9(10): 309–317.
- Gonzalez-Porras JR, Bastida JM. Eltrombopag in immune thrombocytopenia: efficacy review and update on drug safety. Ther Adv Drug Saf. 2018; 9(6): 263–285.
