Rapid health technology assessment of avatrombopag for the treatment of primary immune thrombocytopenia in adults
10.12173/j.issn.1005-0698.202508005
- VernacularTitle:阿伐曲泊帕治疗成人免疫性血小板减少症的快速卫生技术评估
- Author:
Zhou LI
1
;
Ya LING
1
;
Jie ZHANG
1
;
Ling ZHU
1
Author Information
1. 苏州大学附属第一医院药学部(江苏 苏州 215006)
- Publication Type:Journal Article
- Keywords:
Avatrombopag;
Primary immune thrombocytopenia;
Efficacy;
Safety;
Economy;
Rapid health technology assessment
- From:
Chinese Journal of Pharmacoepidemiology
2025;34(11):1311-1319
- CountryChina
- Language:Chinese
-
Abstract:
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.