Objective  To evaluate the efficacy, safety and economics of romiplostim for treating primary immune thrombocytopenia (ITP) by rapid health technology assessment, and to provide an evidence-based basis for policy makers and clinical practice.

Methods  PubMed, Cochrane Library, Embase, CNKI, WanFang Data and VIP databases and the official websites of health technology assessment agency were electronically searched to collect high-quality clinical evidence and pharmacoeconomics evaluation literature of romiplostim for the treatment of ITP from inception to January 18, 2024. Two researchers independently screened the literature, extracted information, and accessed the quality of included the literature, the extracted results were categorized and evaluated.

Results  A total of 14 literature were included, in which 8 systematic reviews/Meta-analysis and 6 pharmacoeconomic studies. In terms of efficacy, treatment with romiplostim significantly elevated platelet response rate, sustained platelet response rate, and mean platelet count in patients with ITP compared with placebo (P<0.05). Romiplostim did not show a significant advantage in elevating patients' platelet response rate and sustained platelet response rate compared with other agents used to treat ITP (P>0.05). In terms of safety, the incidence of serious adverse events was statistically lower with romiplostim compared to placebo (P<0.05), while no significant differences were seen in the incidence of adverse events, bleeding events and thrombotic events (P>0.05). There were no significant differences in the incidence of adverse events, serious adverse events, bleeding events, or thrombotic events when comparing romiplostim to other drugs for the treatment of ITP (P>0.05). From an economic standpoint, most studies considered eltrombopag to be more economic advantages than romiplostim.

Conclusion  Romiplostim has good efficacy and safety in the treatment of ITP, and no advantage was shown in terms of economy.

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