低剂量重组FⅧ预防治疗血友病A儿童的临床效果

CLINICAL EFFECT OF LOW-DOSE RECOMBINANT COAGULATION FACTORⅧPROPHYLAXIS TREATMENT IN CHILDREN WITH HEMOPHILIA A

目的探讨低剂量重组凝血因子Ⅷ(rFⅧ)预防治疗血友病A儿童的临床效果。方法回顾性分析2016年9月1日—2018年2月1日于我院接受低剂量rFⅧ预防治疗的32例血友病A患儿的临床资料,收集患儿低剂量rFⅧ预防治疗前后月出血次数、月关节出血次数、关节功能评分、日常活动能力评分、疾病家庭负担评分等指标,并对各指标治疗前后进行分析比较。结果低剂量rFⅧ预防治疗后患儿月出血次数及月关节出血次数较治疗前明显减少(t=5.028、3.919,P<0.05)。患儿治疗后关节功能总评分、滑膜增生评分、软骨评分及骨评分与治疗前比较差异无统计学意义(P>0.05)。患儿治疗后进行日常活动评分及参加学校活动的评分较治疗前显著增高(t=2.239、2.482,P<0.05)。患儿(年龄≥6岁且排除预防治疗前已辍学)上学出勤率较治疗前明显提高(UC=2.576,P<0.05)。治疗后患儿的疾病家庭负担评分相关指标(除家庭经济负担评分)较治疗前明显降低(t=2.135~3.888,P<0.05)。治疗后患儿的家庭经济负担评分与治疗前比较差异无统计学意义(P>0.05)。结论低剂量rFⅧ预防治疗后,血友病A儿童关节出血次数明显减少,日常活动能力提高,家庭负担减轻,生活质量提高,值得在我国推广实施。

Objective To investigate the clinical effect of low-dose recombinant coagulation factorⅧ(rFⅧ)prophylaxis treatment in children with hemophilia A.Methods A retrospective analysis was performed for the clinical data of 32 children with hemophilia A who received low-dose rFⅧprophylaxis treatment in our hospital from September 1,2016 to February 1,2018.Related indices were collected,including the number of monthly bleeding times,the number of monthly joint bleeding times,joint function score,activities of daily living score,and family burden scale of disease(FBSD)score before and after low-dose rFⅧprophylaxis treatment,and analysis and comparison were performed.Results There were significant reductions in the number of monthly bleeding times and the number of monthly joint bleeding times after low-dose rFⅧprophylaxis treatment(t=5.028,3.919,P<0.05),while there were no significant changes in total joint function score,synovial hyperplasia score,cartilage score,and bone score after treatment(P>0.05).After treatment,there were significant increases in the scores of daily activities and school activities(t=2.239,2.482,P<0.05),and there was also a significant increase in school attendance rate of children aged≥6 years(who had dropped out of school before prophylaxis treatment were excluded)(UC=2.576,P<0.05).After treatment,there were significant decreases in the indicators associated with FBSD(except the score of family economic burden)(t=2.135-3.888,P<0.05),while there was no significant change in the score of family economic burden after treatment(P>0.05).Conclusion Low-dose rFⅧprophylaxis treatment can effectively reduce the number of joint bleeding times,improve the activities of daily living,reduce family burden,and improve the quality of life,and thus it holds promise for clinical application in China.