Objective  To evaluate the efficacy, safety and cost-effectiveness of avatrombopag for treating primary immune thrombocytopenia (ITP) by rapid health technology assessment (rHTA), to provide evidence-based basis for clinical decision-making.

Methods  PubMed, Cochrane Library, Web of Science, Embase, CNKI, WanFang Data and the official website of health technology assessment (HTA) agency were electronically searched to collect systematic reviews/Meta-analyses, pharmacoeconomic literature, and HTA reports of avatrombopag for the treatment of ITP from inception to May 31, 2025. Two reviewers independently screened studies, extracted data, assessed the quality of included studies, and qualitatively analyzed the result.

Results  A total of 12 studies were included, including 10 systematic reviews/Meta-analyses, 1 pharmacoeconomic study and 1 HTA report. In terms of effectiveness, compared with placebo, treatment with avatrombopag significantly elevated platelet response, durable platelet response, early platelet response, and platelet count, reduction in use of concomitant ITP medication (P<0.05), and there was no significant difference in the need for remedial measures during the treatment period (P>0.05). Avatrombopag was also superior to eltrombopag, romiplostim, rituximab alone or in combination with recombinant human thrombopoietin (rhTPO) for platelet response (P<0.05). In terms of safety, there was no significant difference in the incidence of adverse events, serious adverse events, bleeding events and thrombotic events when comparing avatrombopag to placebo, eltrombopag, romiplostim and rituximab for the treatment of ITP (P>0.05). Economic study showed that avatrombopag has an economic advantage compared to eltrombopag and romiplostim, but it does not have economic advantages compared with observation and rescue.

Conclusion  Compared with placebo or other second-line treatments for ITP, avatrombopag has good efficacy and no significant differences in safety. Regarding economics, avatrombopag demonstrates greater cost-effectiveness than eltrombopag and romiplostim. However, due to its higher price, it does not have economic advantages compared to observation and rescue. Future economic research should be conducted domestically.

1.Bussel J, Cooper N, Boccia R, et al. Immune thrombocytopenia[J]. Expert Rev Hematol, 2021, 14(11): 1013-1025. DOI: 10.1080/17474086.2021.1995347.

2.Schifferli A, Moulis G, Godeau B et al. Adolescents and young adults with newly diagnosed primary immune thrombocytopenia[J]. Haematologica, 2023, 108 (10): 2783-2793. DOI: 10.3324/haematol.2022.282524.

3.中华医学会血液学分会血栓与止血学组. 成人原发免疫性血小板减少症诊断与治疗中国指南（2020年版）[J]. 中华血液学杂志, 2020, 41(8): 617-623. [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)[J]. Chinese Journal of Hematology, 2020, 41(8): 617-623.] DOI: 10.3760/cma.j.issn.0253-2727.2020.08.001.

4.陈灿, 张其坤, 许青. 新型血小板生成素受体激动剂: 阿伐曲泊帕[J]. 中国新药与临床杂志, 2020, 39(11): 652-656. [Chen C, Zhang QK, Xu Q. A novel thrombopoietin receptor agonist: avatrombopag[J]. Chinese Journal of New Drugs and Clinical Remedies, 2020, 39(11): 652-656.] DOI: 10.14109/j.cnki.xyylc.2020.11.03.

5.Satapathy SK, Sundaram V, Shiffman ML, et al. Real-world use of avatrombopag in patients with chronic liver disease and thrombocytopenia undergoing a procedure[J]. Medicine, 2023, 102(40): e35208. DOI: 10.1097/MD.0000000000035208.

6.彭惠芳, 洪玮莉, 陈曼娜, 等. 阿伐曲泊帕治疗免疫性血小板减少症超说明书用药的循证评价[J]. 海峡药学, 2024, 36(11): 76-80. [Peng HF, Hong WL, Chen MN, et al. Evidence-based evaluation of the use of avatrombopag in patients with primary immune thrombocytopenia[J]. Strait Pharmaceutical Journal, 2024, 36(11): 76-80.] DOI: 10.3969/j.issn.1006-3765.2024.11.021.

7.Carpenedo M, Baldacci E, Baratè C, et al. Second-line administration of thrombopoietin receptor agonists in immune thrombocytopenia: Italian Delphi-based consensus recommendations[J]. Ther Adv Hematol, 2021, 12: 20406207211 048361. DOI: 10.1177/20406207211048361.

8.Fogarty PF, Tarantino MD, Brainsky A, et al. Selective validation of the WHO Bleeding Scale in patients with chronic immune thrombocytopenia[J]. Curr Med Res Opin, 2012, 28(1): 79-87. DOI: 10.1185/03007995.2011.644849.

9.Shea BJ, Reeves BC, Wells G, et al. AMSTAR 2: a critical appraisal tool for systematic reviews that include randomised or non-randomised studies of healthcare interventions, or both[J]. BMJ, 2017, 358: j4008. DOI: 10.1136/bmj.j4008.

10.邢质聪, 孙福东, 薛青霞, 等. 4种静脉铁剂治疗缺铁性贫血的快速卫生技术评估[J]. 药物流行病学杂志, 2024, 33(9): 1030-1043. [Xing ZC, Sun FD, Xue QX, et al. Four intravenous iron formulations in the treatment of iron deficiencyanemia:a rapid health technology assessment[J]. Chinese Journal of Pharmacoepidemiology, 2024, 33(9): 1030-1043.] DOI: 10.12173/j.issn.1005-0698.202406030.

11.Husereau D, Drummond M, Augustovski F, et al. Consolidated health economic evaluation reporting standards 2022 (CHEERS 2022) statement: updated reporting guidance for health economic evaluations[J]. Value Health, 2022, 25(1): 3-9. DOI: 10.1016/j.jval.2021.11.1351.

12.陶立元, 甘戈, 刘珏. 卫生经济学评价报告标准2022解读 [J]. 中华流行病学杂志, 2023, 44(4): 667-672. [Tao LY, Gan G, Liu J. Interpretation of consolidated health economic evaluation reporting standards 2022[J]. Chinese Journal of Epidemiology, 2023, 44(4): 667-672.] DOI: 10.3760/cma.j.cn112338-20221127-01002.

13.Hailey D. Toward transparency in health technology assessment: a checklist for HTA reports[J]. Int J Technol Assess Health Care, 2003, 19(1): 1-7. DOI: 10.1017/s0266462303000011.

14.吴亦航, 陆艳丽, 罗鲜丹, 等. 艾曲泊帕、阿伐曲泊帕、海曲泊帕治疗免疫性血小板减少症的疗效与安全性的网状Meta分析[J]. 中国新药杂志, 2025, 34(9): 1000-1008. [Wu YH, L YL, Luo XD, et al. Efficacy and safety of eltrombopag, avatrombopag and hetrombopag in patients with chronic immune thrombocytopenia: a systematic literature review and network Meta-analysis[J]. Chinese Journal of New Drugs, 2025, 34(9): 1000-1008.] DOI: 10.20251/j.cnki.1003-3734.2025.09.015.

15.Liu Y, Zhang N, Jiang T, et al. Efficacy and safety and analysis of thrombopoietin receptor agonists for the treatment of immune thrombocytopenia in adults: analysis of a systematic review and network Meta-analysis of randomized controlled trials and results of real-world safety data[J]. Front Med (Lausanne), 2025, 12: 1531824. DOI: 10.3389/fmed.2025.1531824.

16.彭进城, 夏泉, 刘昌伟, 等. 阿伐曲泊帕治疗血小板减少症的有效性和安全性Meta分析[J]. 中国临床研究, 2025, 38(1): 93-99. [Peng JC, Xia Q, Liu CW, et al. Efficacy and safety of avatrombopag in the treatment of thrombocytopenia: a Meta-analysis[J]. Chinese Journal of Clinical Research, 2025, 38(1): 93-99.] DOI: 10.13429/j.cnki.cjcr.2025.01.019.

17.Dong Y, Xia Z, Zhou J, et al. Risk of thrombotic events in immune thrombocytopenia patients treated with thrombopoietic agents: a systematic review and Meta-analysis[J]. Thromb J, 2023, 21(1): 69. DOI: 10.1186/s12959-023-00509-z.

18.Li T, Liu Q, Pu T, et al. Efficacy and safety of thrombopoietin receptor agonists in children and adults with persistent and chronic immune thrombocytopenia: a Meta-analysis[J]. Expert Opin Pharmacother, 2023, 24(6): 763-774. DOI: 10.1080/14656566.2023.2198089.

19.Liu Y, Zhang HX, Su J, et al. Efficacy and incidence of treatment-related adverse events of thrombopoietin receptor agonists in adults with immune thrombocytopenia: a systematic review and network Meta-analysis of randomized controlled study[J]. Acta Haematol, 2023, 146(3): 173-184. DOI: 10.1159/000528642.

20.Deng J, Hu H, Huang F, et al. Comparative efficacy and safety of thrombopoietin receptor agonists in adults with thrombocytopenia: a systematic review and network Meta-analysis of randomized controlled trial[J]. Front Pharmacol, 2021, 12: 704093. DOI: 10.3389/fphar.2021.704093.

21.Wojciechowski P, Wilson K, Nazir J, et al. Efficacy and safety of avatrombopag in patients with chronic immune thrombocytopenia: a systematic literature review and network Meta-analysis[J]. Adv Ther, 2021, 38(6): 3113-3128. DOI: 10.1007/s12325-021-01752-4.

22.Yang R, Lin L, Yao H, et al. Therapeutic options for adult patients with previously treated immune thrombocytopenia-a systematic review and network Meta-analysis[J]. Hematology, 2019, 24(1): 290-299. DOI: 10.1080/16078454.2019.1568659.

23.Arai Y, Matsui H, Jo T, et al. Comparison of treatments for persistent/chronic immune thrombocytopenia: a systematic review and network Meta-analysis[J]. Platelets, 2019, 30(8): 946-956. DOI: 10.1080/09537104.2018.1543864.

24.Cooper N, Guterres S, Pochopień M, et al. The cost-effectiveness of avatrombopag versus eltrombopag and romiplostim in the treatment of patients with immune thrombocytopenia in the UK[J]. J Mark Access Health Policy, 2025, 13(2): 11. DOI: 10.3390/jmahp13020011.

25.Canada's Drug and Health Technology Agency (CADTH). Avatrombopag (Doptelet): Therapeutic area: chronic immune thrombocytopenia: CADTH reimbursement review[R/OL]. (2024-05-08) [2025-05-06]. https://www.cadth.ca/ avatrombopag.

26.Aiello A, Mariano EE, Prada M, et al. Budget impact analysis for avatrombopag in the treatment of chronic primary immune thrombocytopenia in adult patients refractory to other treatments[J]. J Mark Access Health Policy, 2023, 11(1): 2230663. DOI: 10.1080/20016689.2023.2230663.

27.Dranitsaris G, Peevyhouse A, Wood T, et al. Fostamatinib or thrombopoietin for the treatment of Chronic immune thrombocytopenia in adult patients: a real-world assessment of safety, effectiveness and cost[J]. Acta Haematol, 2023, 147(3): 333-343. DOI: 10.1159/000533175.