真实世界中不同止血方案治疗血友病A伴抑制物患者轻中度出血的成本与效果

Pharmacoeconomic Analysis of Different Regimes in the Treatment of Minor-to-Mild Bleeds in Hemophilia Patients with Inhibitors:a Real World Study

目的比较真实世界中不同止血方案治疗血友病A伴抑制物患者轻中度出血的成本与效果。方法选取7家医院的26例血友病A伴抑制物患者进行线上问卷调查,收集患者过去一年内的用药治疗情况,并对不同止血方案治疗情况的成本和效果进行统计分析。结果 26例患者平均年龄为(20.4±13.2)岁,严重血友病患者21例,高滴度抑制物患者25例;对患者共计111次轻中度出血治疗用药情况进行分析显示:先使用基因重组活化凝血因子Ⅶ(rFⅦa),再使用凝血酶原复合物(PCC)方案止血有效的比例最高,为76.9%,其次为仅使用rFⅦa方案(69.6%)、使用重组FⅧ或血源性FⅧ等其他药物方案(68.8%)、先使用PCC再使用rFⅦa方案(63.2%)、仅使用PCC方案止血有效的比例最低,为52.5%;先使用rFⅦa再使用PCC,12 h内重复出血次数最少,12 h内重复出血的比例最低,为1次(7.7%)。患者单次出血花费差异较大,仅使用PCC花费中位数最少为9911元,先使用PCC再使用rFⅦa治疗的花费最多为47280元。结论 "先使用rFⅦa再使用PCC"比"先使用PCC再使用rFⅦa"的成本更低,效果更好,建议出血后尽早使用r FⅦa。

Objective To compare the cost and effectiveness of different regimes treatment of minor-to-mild bleeds in hemophilia patients with inhibitors in real world. Methods We applied the online questionnaire collecting patients’ treatment information over the past year from 7 hospitals in China. Descriptive statistics were used to compare cost and effectiveness of different regimes. Results Overall, 111 bleeds occurred in 26 hemophilia patients with inhibitors(26 adults;mean age=20.4±13.2 years;21 patients with severe hemophilia;25 patients with high response inhibitors). The first use of genetic recombination to activate coagulation factor Ⅶ(rFⅦa), and then the use of prothrombin complex(PCC) program had the highest hemostatic effect,which was 76.9%, followed by the use of only rFⅦa program(69.6%), using recombinant FⅧ or blood-derived FⅧ and other drug regimens(68.8%), using PCC first and then using rFⅦa regimen(63.2%), using only the PCC regimen was the lowest effective in hemostasis, at 52.5%;using rFⅦa first and then using PCC, the number of repeated bleeding was the least within 12 hours, and the rate of repeated bleeding was the lowest within 12 hours, which was 1 time(7.7%). The cost of a single bleed varies greatly among patients, with the median cost of bleed treated with PCC only at least CNY 9 911, and median cost of bleed treated with PCC-rFⅦa at most CNY 47 280. Conclusion The rFⅦa-PCC treatment regimen is cheaper and more effective than PCC-rFⅦa. It is recommended to use rFⅦa as soon as possible after bleeding.