单中心成人重型血友病A低/中剂量凝血因子Ⅷ预防治疗30例疗效分析  

Analysis of the effects of low/intermediate dose of coagulation factor Ⅷ on 30 adult patients with severe hemophilia A in a single center

作　　者：袁燕慧[1] 许佩佩[1] 徐岳一[1] 刘莎 邵晓雁[1] 张玮婧 龚黎[2] 周敏[1] 陈兵[1] 周荣富[1] Yuan Yanhui;Xu Peipei;Xu Yueyi;Liu Sha;Shao Xiaoyan;Zhang Weijing;Gong Li;Zhou Min;Chen Bing;Zhou Rongfu(Department of Hematology,the Affiliated Drum Tower Hospital of Nanjing University Medical School,Nanjing 210008,China;Department of Ulrasound,the Afiliated Drum Tower Hospital of Nanjing University Medical School,Nanjing 210008,China)

机构地区：[1]南京大学医学院附属南京鼓楼医院血液科,南京210008 [2]南京大学医学院附属南京鼓楼医院超声科,南京210008

出　　处：《中华血液学杂志》2023年第1期38-42,共5页Chinese Journal of Hematology

摘　　要：目的评估低/中剂量凝血因子Ⅷ(FⅧ)预防治疗在成人重型血友病A患者中的疗效。方法纳入在南京大学医学院附属南京鼓楼医院接受低/中剂量FⅧ预防治疗的30例成人重型血友病A患者(低剂量预防组20例、中剂量预防组10例),对预防治疗前、后的年出血次数(ABR)、年关节出血次数(AJBR)、靶关节数量、血友病功能独立性评分(FISH)、生活质量评分及健康状况评分(SF-36)进行回顾性分析、比较。结果中位随访48个月。与预防前按需治疗时相比,低、中剂量预防治疗组ABR、AJBR、靶关节数量均明显降低(P<0.05),且中剂量预防治疗组改善优于低剂量预防治疗组(P<0.05)。低、中剂量预防治疗后与按需治疗时相比,患者的FISH评分、生活质量评分及SF-36评分均明显改善(P>0.05),但低剂量预防组和中剂量预防组之间以上指标差异无统计学意义(P>0.05)。结论低、中剂量FⅧ预防治疗较按需治疗可明显降低重型血友病A患者出血频率,延缓关节病变的进展,提高患者生活质量。中剂量预防组临床出血改善方面优于低剂量预防组。Objective To evaluate the clinical effects of low-and intermediate-dose factorⅧ(FⅧ)prophylaxis in Chinese adult patients with severe hemophilia A.Methods Thirty adult patients with severe hemophilia A who received low-(n=20)/intermediate-dose(n=10)FⅧprophylaxis at Nanjing Drum Tower Hospital affiliated with Nanjing University Medical College were included in the study.The annual bleeding rate(ABR),annual joint bleeding rate(AJBR),number of target joints,functional independence score of hemophilia(FISH),quality of life score,and health status score(SF-36)before and after preventive treatment were retrospectively analyzed and compared.Results The median follow-up was 48 months.Compared with on-demand treatment,low-and intermediate-dose prophylaxis significantly reduced ABR,AJBR,and the number of target joints(P<0.05);the improvement in the intermediate-dose prophylaxis group was better than that in the low-dose prophylaxis group(P<0.05).Compared with on-demand treatment,the FISH score,quality of life score,and SF-36 score significantly improved in both groups(P<0.05),but there was no significant difference between the two groups(P>0.05).Conclusion In Chinese adults with severe hemophilia A,low-and intermediate-dose prophylaxis can significantly reduce bleeding frequency,delay the progression of joint lesions,and improve the quality of life of patients as compared with on-demand treatment.The improvement in clinical bleeding was better with intermediate-dose prophylaxis than low-dose prophylaxis.

关 键 词：血友病A 预防治疗 凝血因子Ⅷ 

分 类 号：R554.1[医药卫生—血液循环系统疾病]