中国大陆血友病患者治疗现状和经济负担的系统评价

目的了解中国大陆血友病患者（people with hemophilia，PWH）的治疗现状及经济负担，并探讨血友病在中国的最佳治疗方案。方法于2011年6月系统检索《中国生物医学文献数据库》（CBM）、《中国期刊全文数据库》（CNKI）、《中国科技期刊数据库》（VIP）、万方数据库等中文数据库，以及PubMed、EMbase和The Cochrane Library（2011年第6期）等英文数据库中收录的1980—2011年发表的有关PWH治疗及经济负担的研究。结果中国PWH诊断、治疗滞后；超30％的PWH不治疗或偶尔治疗，不足10％的PWH接受预防治疗；仍有相当一部分患者使用新鲜冷冻血浆（FFP）或冷沉淀等容易导致血源性病毒传播疾病的传统血制品；大于50％的PWH家庭只能支付很少或完全支付不起医疗费用。低剂量预防治疗相对于足量按需治疗更经济。以目前中国PWH实际治疗情况计算，每例PWH每年多投入53844元即可实现低剂量预防治疗．从而减少80％的出血。结论中国大陆PWH的治疗现状差，经济负担重，推行综合关怀模式，采用低剂量预防治疗是最佳治疗方案。

Treatment Status and Economic Burden of People with Hemophilia in Mainland China: A Systematic Review

Objective To be aware of the treatment status and economic burden of people with hemophilia （PWH） in mainland China, so as to seek the optimal therapy for them. Methods The relevant Chinese and English databases such as CBM, CNKI, VIP, WanFang Database, PubMed, EMbase and The Cochrane Library （Issue 6 of 12, ]une 2011） were searched in June, 2011. The economic analyses and studies on PWH treatment and economic burden published from 1980 to 2011 were collected. Results The diagnosis and treatment of PWH in mainland China lagged behind. More than 30% of PWH did not receive or occasionally received treatment, and less than 10% received prophylactic therapy. Lots of PWHs still used FFP or cryoglobulin which were easily to cause blood-borne viral diseases. More than half of PWH families could afford a little or completely could not afford the therapy. Low dose prophylactic therapy was cost-efficient than on-demand therapy. Based on the therapy status, it was estimated that approximately RMB 53 844 yuan per year per patient should be put into practice in order to have PWH received low-dose prophylactic therapy, and to prevent 80% of bleeding. Conclusion PWH in mainland China is poor in treatment status and heavy in economic burden, so it is an optimal way to adopt comprehensive care model and low-dose prophylactic therapy in mainland China.