目的探讨医疗保险政策及慈善项目对中国重型血友病A患儿选择规律预防治疗的影响。方法 (1)对2007年11月1日至2013年5月31日期间,在中国12个血友病中心接受过Ⅷ因子规律预防治疗的重型血友病A患儿进行回顾性研究。(2)记录研究期间的各项临床资料。(3)从各中心收集重型血友病A患儿选择或放弃规律预防治疗影响因素的调查问卷。(4)查询12个中心(位于11个城市)历年的血友病相关医保政策,比较不同医保报销比例下患儿年均FⅧ使用量及自付水平。比较参加慈善援助项目及未参加慈善援助项目年均FⅧ使用量的差异。结果 (1)2008年开始出现患儿选择规律预防治疗,并且人数呈逐年增加的趋势。(2)患儿选择规律预防治疗的前三位原因为:既往按需治疗效果不理想、当地医保政策改善、患儿或家属对疾病认识提高。中途放弃规律预防治疗的主要原因为家庭经济支付能力不足。(3)研究中83.4%的患儿享受医保报销,其中80.11%患儿的医保政策年度支付上限不低于15万元,可满足其规律低剂量预防方案的医疗费用。(4)医保报销比例高的患儿年均FⅧ使用量相对较高。医保报销比例低的患儿,自付水平明显较高(P<0.05)。66.4%的患儿自付水平≥50%,其人均可支配收入的一半以上需用于支付预防治疗费用。(5)预防治疗的患儿中79.6%接受了慈善援助项目的支持。(6)2012年患儿实际年FⅧ使用量均值为30376IU(合999IU/kg),仅为全年坚持规律预防治疗理论用量的62.9%,明显低于高收入国家FⅧ使用量。结论(1)医保政策是影响中国儿童选择和坚持预防治疗的重要因素,慈善援助项目对其起到了推动作用。(2)对于低剂量预防治疗方案来讲,医保报销比例,而非年度支付上限,可能是制约患儿坚持长期规律预防治疗的重要因素。(3)目前中国儿童血友病预防治疗为短疗程、低剂量.与中高收入国家FⅧ使用量存在明显差距,医保政策有待进一步改善。 Objective To explore the influence of medical insurance policy and charity project on prevention and treatment of children with severe hemophilia A in China. Methods (1) A retrospective study was conducted on children with severe hemophilia A who were treated with Factor VIII prophylaxis at 12 hemophilia centers in China from November 1, 2007 to May 31, 2013. (2) record the clinical data during the study. (3) Questionnaire of selecting or abandoning the factors influencing the regular prevention and treatment of children with severe hemophilia A collected from the centers. (4) Inquiring into the hemophilia-related health insurance policies in 12 centers (in 11 cities) over the years, and comparing the annual average FⅧ consumption and the level of out-of-pocket children under different health insurance reimbursement rates. Compare the annual average FVIII usage for participating charity projects and not participating in charity projects. Results (1) The prevention and treatment of children’s selection rules began to appear in 2008, and the number of patients showed a trend of increasing year by year. (2) The first three reasons for the prevention and treatment of children’s selection rule are as follows: Previous treatment on demand is not satisfactory, the local health insurance policy is improved, and the awareness of children or their families on the disease is improved. The main reason for abandoning the regular prevention and treatment halfway is the lack of affordability of the family economy. (3) 83.4% of the children in the study enjoy Medicare reimbursement, of which 80.11% of the children’s Medicare policy annual payment cap is not less than 150,000 yuan, to meet its regular low-dose prevention program of medical expenses. (4) Children with high proportion of Medicare reimbursement have relatively high annual usage of FⅧ. Children with a low proportion of reimbursement for medical insurance had significantly higher copay levels (P <0.05). 66.4% of children with co-payment level ≥50%, more than half of their per capita disposable income needed to pay for prevention and treatment costs. (5) 79.6% of children who received prophylactic treatment were supported by the charity project. (6) In 2012, the mean annual FVIII usage of children was 30376 IU (999 IU / kg), which was only 62.9% of the theoretical theoretical amount of preventive treatment in the whole year, which was significantly lower than that of FⅧ in high-income countries. Conclusions (1) Medicare policy is an important factor affecting Chinese children’s choices and adhering to prevention and treatment, and the charity project has played a catalytic role. (2) For low-dose prophylaxis and treatment programs, the proportion of Medicare reimbursement, rather than the annual payment cap, may be an important factor restricting children’s adherence to long-term prevention and treatment. (3) At present, prevention and treatment of hemophilia in children in China is short-course and low-dose, and there is a clear gap between the usage of FⅧ in middle- and high-income countries and the medical insurance policy needs to be further improved.