Current status and cost burden of non-first-line treatment in ITP: A multicenter study based on real-world data in China

Abstract

Background: Eltrombopag, the first TPO-RA approved for the treatment of ITP, resulted in rapid and sustained increases in platelet counts, significant reductions in bleeding events and concomitant medications, and an acceptable tolerability profile. This study aimed to explore the current status of ITP treatment with eltrombopag as the target drug and the utilization of medical resources from the perspective of hospitals.

Methods: Data collection was based on two parts: real-world data and expert interviews. Real-world data of outpatients with ITP who received second-line treatment or other treatment from 1 year before to 1 year after eltrombopag admission were collected to analyze the current status of non-first-line treatment in ITP outpatients. Ten specialist physicians were interviewed to collect parameters such as efficacy, safety, and medical cost of ITP treatment regimens.

Result: The real-worlddata showed that among non-first-line treatment, market share of second-line treatment was lower than that of other treatment before the admission of eltrombopag, and the market share of second-line treatment was the higher than that of other treatment after the admission of eltrombopag in both adult and pediatric ITP patients. After the admission of eltrombopag, the annual outpatient drug-related treatment costs for adults with ITP increased by ￥13717.05, and the annual outpatient drug-related treatment costs for children with ITP increased by ￥7762.90. The results of expert interviews showed that more patients switched from other treatment to second-line treatment after the admission of eltrombopag in clinical. Eltrombopag demonstrated superior efficacy, safety and higher adherence in second-line treatment.

Conclusions: The new oral TPO-RA drugs have improved the treatment options for ITP patients, and more patients accept eltrombopag for non-first-line treatment. After eltrombopag admission, the annual outpatient drug-related treatment costs increased for both adult and pediatric patients. Nevertheless, by reason of advantages of its efficacy, tolerance, medication adherence and other aspects, eltrombopag has become an important drug in the non-first-line treatment of ITP.Additional comprehensive research is needed to verify the results.

Keywords

Immune thrombocytopenia / Eltrombopag / Real world data

References

[1] Song F , Al-Samkari H . Management of adult patients with immune thrombocytopenia (ITP): a review on current guidance and experience from clinical practice. Hematol Res Rev. 2021 Jul 26;12:653–664.

[2] Neunert C , Terrell DR , Arnold DM , et al. American Society of Hematology 2019 guidelines for immune thrombocytopenia. Blood Adv. 2019 Dec 10;3(23): 3829–3866.

[3] DeSouza S , Angelini D . Updated guidelines for immune thrombocytopenic purpura: expanded management options. Cleve Clin J Med. 2021; 88 (12): 664- 668.

[4] Danese MD , Lindquist K , Gleeson M , et al. Cost and mortality associated with hospitalizations in patients with immune thrombocytopenic purpura. Am J Hematol. 2009; 84 (10): 631- 635.

[5] Audia S , Mahévas M , Samson M , et al. Pathogenesis of immune thrombocytopenia. Autoimmun Rev. 2017 Jun;16(6):620–632.

[6] Cooper N , Kruse A , Kruse C , et al. Immune thrombocytopenia (ITP) world impact survey (I-WISh): impact of ITP on health-related quality of life. Am J Hematol. 2021 Feb 1;96(2):199–207.

[7] Mei H , Xu M , Yuan G , et al. A multicentre double-blind, double-dummy, randomised study of recombinant human thrombopoietin versus eltrombopag in the treatment of immune thrombocytopenia in Chinese adult patients. Br J Haematol. 2021; 195 (5): 781- 789.

[8] Yu Y , Wang M , Hou Y , et al. High-dose dexamethasone plus recombinant human thrombopoietin vs high-dose dexamethasone alone as frontline treatment for newly diagnosed adult primary immune thrombocytopenia: a prospective, multicenter, randomized trial. Am J Hematol. 2020; 95 (12): 1542- 1552.

[9] Kapur R , Zufferey A , Boilard E , et al. Nouvelle cuisine:platelets served with inflammation. J Immunol. 2015; 194 (12): 5579- 5587.

[10] Cecinati V , Principi N , Brescia L , et al. Vaccine administration and the development of immune thrombo-cytopenic purpura in children. Hum Vaccines Immunother. 2013; 9 (5): 1158- 1162.

[11] Thrombosis and Hemostasis Group, Chinese Society of Hematology, Chinese Medical Association. [Chinese guideline on the diagnosis and management of adult primary immune thrombocytopenia (version 2020)]. Zhonghua Xue Ye Xue Za Zhi. 2020 Aug 14;41(8):617-623.

[12] 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.

[13] Gilreath J , Lo M , Bubalo J . Thrombopoietin receptor agonists (TPO-RAs): drug class considerations for pharmacists. Drugs. 2021 Jul;81(11):1285–1305.

[14] Gomez-Almaguer D . Eltrombopag-based combination treatment for immune thrombocytopenia. Ther Adv Hematol. 2018; 9 (10): 309- 317.

[15] Gilreath J , Lo M , Bubalo J . Thrombopoietin receptor agonists (TPO-RAs): drug class considerations for pharmacists. Drugs. 2021; 81 (11): 1285- 1305.

[16] Fattizzo B , Levati G , Cassin R , et al. Eltrombopag in immune thrombocytopenia, aplastic anemia, and myelodysplastic syndrome: from megakaryopoiesis to immunomodulation. Drugs. 2019 Aug;79(12):1305–1319.

[17] 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.

[18] Garside J , Healy N , Besson H , et al. PHEDRA: using real-world data to analyze treatment patterns and ibrutinib effectiveness hematological malignancies. J Comp Eff Res. 2018 Jan;7(1):29–38.

[19] Park H , Park CM , Woo JM , et al. Real-world data analysis of the clinical and economic burden and risk factors in patients with major depressive disorder with an inadequate response to initial antidepressants. J Med Econ. 2021 Jan-Dec;24(1): 589–597.

[20] Moulis G , Palmaro A , Montastruc J , et al. Epidemiology of incident immune thrombocytopenia: a nationwide population-based study in France. Blood. 2014; 124 (22): 3308- 3315.

[21] Zufferey A , Kapur R , Semple JW . Pathogenesis and therapeutic mechanisms in immune thrombocytopenia (ITP). J Clin Med. 2017 Feb 9;6(2):16.

[22] Ozelo MC , Colella MP , Paula ED , et al. Guideline on immune thrombocytopenia in adults: associao Brasileira de Hematologia, Hemoterapia e Terapia Celular. Project guidelines: associao Médica Brasileira – 2018. Hematol Trans Cell Ther. 2018;40(1): 50–74.

[23] Liu WJ , Bai J , Guo QL , et al. Role of platelet function and platelet membrane glycoproteins in children with primary immune thrombocytopenia. Mol Med Rep. 2016; 14 (3): 2052- 2060.

[24] 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[J]. Blood. 2009; 113 (11): 2386- 2393.

[25] Song F , Al-Samkari H . Management of adult patients with immune thrombocytopenia (ITP): a review on current guidance and experience from clinical practice. Hematol Res Rev. 2021 Jul 26;12:653–664.

[26] Liu XG , Hou Y , Hou M . How we treat primary immune thrombocytopenia in adults. J Hematol Oncol. 2023 Jan 19;16(1):4.

[27] Moulis G , Germain J , Comont T , et al. Newly diagnosed immune thrombocytopenia adults: clinical epidemiology, exposure to treatments, and evolution. Results of the CARMEN multicenter prospective cohort. Am J Hematol. 2017;92(6):493–500.

[28] Kuter DJ . The structure, function, and clinical use of the thrombopoietin receptor agonist avatrombopag. Blood Rev. 2022 May;53:100909.

[29] Kim TO , Despotovic J , Lambert MP . Eltrombopag for use in children with immune thrombocytopenia. Blood Adv. 2018 Feb 27;2(4):454–461.

[30] Kuter DJ . The biology of thrombopoietin and thrombopoietin receptor agonists. Int J Hematol. 2013; 98 (1): 10- 23.

[31] Liu X , Hou M , Li J , et al. Efficacy and safety of eltrombopag in Chinese patients with chronic immune thrombocytopenia: stage 2 results from a multicenter phase III study. Platelets. 2022; 33 (1): 82- 88.

[32] Cheng X , Fu L , Ma J , et al. Spotlight on eltrombopag in pediatric ITP in China: a longterm observational study in real-world practice. Blood Adv. 2021; 5 (19): 3799- 3806.

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