ObjectiveTo systematically review the pharmacoeconomics research of coagulation factor Ⅷ for the treatment of hemophilia A. MethodsPubMed, EMbase, Web of Science, The Cochrane Library, CNKI, VIP and WanFang Data databases were electronically searched to collect pharmacoeconomic studies of coagulation factor Ⅷ for the treatment of hemophilia A from inception to February 2022. Two reviewers independently screened literature, extracted data and assessed the risk of bias of included studies; then, qualitative systematic review was carried out from the aspects of research model, research parameters and uncertainty analysis. ResultsA total of 17 pharmacoeconomic studies were included. The overall quality of the included literature was relatively high, and most of them conformed to the basic framework of pharmacoeconomic research; however, there were still differences and deficiencies in model setting and parameter selection. Most results of the study evaluation showed that prophylaxis of coagulation factor Ⅷ had cost-effectiveness advantages over on-demand treatment. ConclusionCurrent evidence shows that the preventive treatment of coagulation factor Ⅷ may have certain cost-effectiveness advantages compared with on-demand treatment; however, the adaptability of this conclusion to China still needs to be analyzed.
【摘要】 目的 探討重組人凝血因子Ⅷ制劑小劑量短程預防性輸注能否有效減少中重度血友病A患兒關節出血問題。 方法 對2008年11月-2009年4月期間就診的13例年齡3~11歲的中重度血友病A患兒，均在為期2個月內接受重組人凝血因子Ⅷ 2次/周、間隔3 d、每次7.5~10.0 U/kg的靜脈預防性輸注，記錄治療前2個月與治療2個月時關節出血次數，以及同一關節反復發生出血的情況。 結果 治療前關節出血的發生次數為（3.77±2.13）次，治療后關節出血的發生次數為（0.46±0.87）次，治療前后比較，差異有統計學意義（Plt;0.01）；治療前靶關節出血的發生率為35.7％，治療后靶關節出血的發生率為0.0%，治療前后比較，差異有統計學意義（Plt;0.01）。患兒治療成本約510~680元/（kg?2個月）。 結論 重組人凝血因子Ⅷ制劑小劑量短療程預防性輸注能有效減少中重度血友病A患兒關節出血次數，同時可有效減少靶關節出血的發生率，從而在一定程度上保護關節的功能。治療費用相對可接受。【Abstract】 Objective To evaluate the efficacy of low-dose short-course infusion of recombinant human factor Ⅷ concentration in treating joint bleeding in children with severe and moderate hemophilia A. Methods Thirteen children aged 3 to 11 years old with severe or moderate hemophilia A were included in the present study from November 2008 to April 2009. For children in the treatment group, they were treated with low-dose short-course infusion of recombinant human factor Ⅷ concentration with a dose of 7.5-10.0 U/kg twice weekly as secondary prophylaxis for two months. The incidence of joint bleeding 2 months before treatment (control group) and during the 2 months of treatment (treatment group) was observed. Moreover, the incidence of their target joint bleeding was measured in both groups. Results Children in the control group had (3.77±2.13) joint bleedings while children in the treatment group had (0.46±0.87) joint bleedings, which was obviously lower than those in the control group (Plt;0.01). Meanwhile, the incidence of target joint bleeding in the treatment group was 0%, which was obviously lower than that in the control group (35.7%) (Plt;0.01). In the treatment group, the costs of treatment were about RMB 510-680 yuan/kg every 2 months. Conclusions Treatment with low-dose short-course infusion of recombinant human factor Ⅷ concentration can effectively decrease joint bleeding in children with severe and moderate hemophilia A, and can effectively decrease the incidence of target joint bleeding. Therefore, this method may play an important role in protection of the joint function in those patients at an acceptable cost.
