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中、低剂量重组人凝血因子Ⅷ预防治疗重型血友病A成年患者的疗效对比研究 被引量:1

Comparison of efficacy of intermediate- and low-dose recombinant human coagulation factor Ⅷ prophylaxis in adult patients with severe hemophilia A
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摘要 目的探讨中、低剂量重组人凝血因子Ⅷ(rhFⅧ)预防治疗重型血友病A成年患者的疗效。方法选择2016年10月至2018年12月,于成都市第三人民医院血液科门诊接受rhFⅧ低剂量预防治疗1年后,继续接受中剂量rhFⅧ预防治疗1年的16例重型血友病A成年患者为研究对象。患者均为男性,中位年龄为30岁(20~37岁)。中剂量预防治疗方案为静脉注射rhFⅧ15~18 IU/(kg·次)、1~3次/周,中位rhFⅧ剂量及频次分别为17 U/(kg·次)和2次/周;低剂量预防治疗方案为静脉注射rhFⅧ10~12 IU/(kg·次)、1~3次/周,中位rhFⅧ剂量及频次分别为10 U/(kg·次)和2次/周。采用回顾性分析方法,收集患者于低、中剂量rhFⅧ预防治疗期间的年总出血次数(ABR),年关节出血次数(AJBR),双侧肘关节、膝关节、踝关节的超声总评分,血友病关节健康评分(HJHS),以及中剂量预防治疗期间FⅧ抑制物发生情况。患者低、中剂量rhFⅧ预防治疗期间的AJBR、关节超声总评分及HJHS等比较,采用配对t检验;ABR比较,采用Wilcoxon符号秩和检验。本研究遵循的程序符合成都市第三人民医院人体试验委员会制定的标准,经过该伦理委员会批准(批准文号:2021-S-23),并且与患者签署临床研究知情同意书。结果①本组16例重型血友病A成年患者接受低剂量rhFⅧ预防治疗期间,其中位ABR、AJBR和HJHS分别为20.5次(2.0~38.0次)、(17.9±8.6)次和(43.7±17.3)分,分别高于中剂量预防治疗期间的10.5次(3.0~36.0次)、(9.8±6.7)次和(37.3±15.3)分,并且差异均有统计学意义(Z=3.132、P=0.002,t=3.820、P=0.002,t=3.315、P=0.005)。②本组16例患者接受低、中剂量rhFⅧ预防治疗期间,其关节超声总评分分别为(26.9±14.1)分和(31.8±11.8)分,二者比较,差异无统计学意义(t=-4.88,P=0.064)。③中剂量rhFⅧ预防期间,患者的FⅧ抑制物均呈阴性。结论重型血友病A成年患者采用中剂量rhFⅧ预防治疗方案较低剂量预防治疗,可以明显减少患者ABR和AJBR,改善患者的关节功能,并且不增加其FⅧ抑制物的产生。 Objective To explore efficacy of intermediate-and low-dose recombinant human coagulation factorⅧ(rhFⅧ)prophylaxis in adult patients with severe hemophilia A.Methods From October 2016 to December 2018,a total of 16 adult patients with severe hemophilia A who received low-dose rhFⅧprophylaxis for 1 year then transited to intermediate-dose rhFⅧfor 1 year in Hemophilia Clinic of the Third People′s Hospital of Chengdu were selected as research subjects.All patients were male with median age of 30 years(20-37 years).Regimen of intermediate-dose prophylaxis was intravenous injection of rhFⅧ15-18 IU/(kg·time),1-3 times per week,with median dosage of 17 IU/(kg·time)and median frequency of 2 times per week,and low-dose prophylaxis was intravenous injection of rhFⅧ10-12 IU/(kg·time),1-3 times per week,with median dosage of 10 IU/(kg·time)and median frequency of 2 times per week.Betwenn two regimens,adult patients with servere hemophilia A′s annual bleeding rate(ABR),annual joint bleeding rate(AJBR),total joints ultrasonic score and hemophilia joint health score(HJHS)of bilateral elbow joints,knee joints and ankle joints,as well as FⅧinhibitor test results were collected retrospectively.The paired t test was conducted for comparing of AJBR,total joints ultrasonic score and HJHS of patients between prophylaxis periods of intermediate-and low-dose rhFⅧ,and Wilcoxon signed rank test was conducted for comparing of ABR of patients between two periods,too.The procedures followed in this study were in accordance with the standards established by the Medical Ethics Committee of Chengdu Third People′s Hospital,and this study was approved by the committee(Approval No.2021-S-23).All the subjects signed the informed consents for clinical trials.Results①In this study,the median ABR,AJBR and HJHS of 16 adult patients with severe hemophilia A during low-dose rhFⅧprophylaxis period were 20.5 times(2.0-38.0 times),(17.9±8.6)times and(43.7±17.3)scores respectively,which were higher than those of 10.5 times(3.0-36.0 times),(9.8±6.7)times and(37.3±15.3)scores during intermediate-dose period,and the differences were statistically significant(Z=3.132,P=0.002;t=3.820,P=0.002;t=3.315,P=0.005).②The total joints ultrasonic score of these 16 patients were(26.9±14.1)and(31.8±11.8)scores respectively during low-and intermediate-dose rhFⅧprophylaxis period,and there were no significant difference between them(t=-4.88,P=0.064).③During intermediate-dose rhFⅧprophylaxis period,all these 16 patient′s FⅧinhibitors were negative.Conclusions Intermediate-dose rhFⅧprophylaxis could significantly reduce ABR and AJBR,and improve joint function in adult patients with severe hemophilia A compared with low-dose prophylaxis.And intermediate-dose rhFⅧprophylaxis would not increase the risk of FⅧinhibitors in patients with severe hemophilia A.
作者 陈蓉 高华 周世秋 刘佳 谭竞 Chen Rong;Gao Hua;Zhou Shiqiu;Liu Jia;Tan Jing(Affiliated Hospital of Southwest Jiaotong University,Chengdu Second Clinical College Affiliated to Chongqing Medical University,Third People′s Hospital of Chengdu,Department of Hematology,Chengdu 610031,Sichuan Province,China)
出处 《国际输血及血液学杂志》 2021年第3期207-212,共6页 International Journal of Blood Transfusion and Hematology
关键词 血友病A 出血 关节 成年人 对比研究 Hemophilia A Hemorrhage Joints Adult Comparative study
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