凝血因子Ⅷ治疗血友病A药物经济学研究的系统评价

Pharmacoeconomic studies on coagulation factorⅧfor the treatment of hemophilia A:a systematic review

目的系统评价凝血因子Ⅷ治疗血友病A的药物经济学研究。方法计算机检索PubMed、EMbase、Web of Science、The Cochrane Library、CNKI、VIP和WanFang Data数据库,搜集凝血因子Ⅷ治疗血友病A的药物经济学研究,检索时限均为建库至2022年2月。由2名研究者独立筛选文献、提取资料并评价纳入研究的偏倚风险后,从研究模型、研究参数和不确定性分析等方面进行定性系统评价。结果最终纳入17个药物经济学研究。纳入研究总体质量较高,绝大多数符合药物经济学研究的基本框架,但仍存在模型设定、参数选择等方面的不足。大部分研究结果显示凝血因子Ⅷ对血友病A的预防治疗与按需治疗相比具有成本-效果优势。结论当前证据显示,凝血因子Ⅷ对血友病A的预防治疗相较于按需治疗可能具有一定的成本-效果优势,但仍需在中国环境下验证。

Objective To systematically review the pharmacoeconomics research of coagulation factorⅧfor the treatment of hemophilia A.Methods PubMed,EMbase,Web of Science,The Cochrane Library,CNKI,VIP and WanFang Data databases were electronically searched to collect pharmacoeconomic studies of coagulation factorⅧfor 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.Results A 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 factorⅧhad costeffectiveness advantages over on-demand treatment.Conclusion Current evidence shows that the preventive treatment of coagulation factorⅧmay have certain cost-effectiveness advantages compared with on-demand treatment;however,the adaptability of this conclusion to China still needs to be analyzed.