不同剂量重组人凝血因子Ⅷ对血友病A患儿关节损害及生活质量的影响

Effects of different doses of recombinant human coagulation factor Ⅷ on joint damage and quality of life in children with hemophilia A

目的探究不同剂量重组人凝血因子Ⅷ对血友病A患儿关节损害及生活质量的影响。方法选取温州市人民医院2019年1月至12月收治的80例血友病A急性出血患儿为研究对象,随机分为小剂量组(每次10U/kg重组人凝血因子Ⅷ,每3天1次)与中剂量组(15~30U/kg重组人凝血因子Ⅷ,每周3次),治疗4个月后比较两组患儿出血情况、关节损害情况、生活质量、不良反应情况与新增关节病变。其中关节损害情况采用血友病功能独立性评分量表(FISH)评价,生活质量采用加拿大血友病儿童生活质量评估工具(CHO-KLAT)评估。结果两组患儿月出血频率较治疗前显著降低,且中剂量组显著低于小剂量组(t=3.574,P<0.05),中剂量组急诊次数、严重出血事件显著少于小剂量组(t/χ^2值为4.986、3.914,均P<0.05);治疗后,两组患儿FISH量表自我照顾、转移功能、移动功能评分及总分较治疗前均提高,且中剂量组显著高于小剂量组(t值分别为2.192、4.760、3.516、5.523,均P<0.05);治疗后,两组患儿CHO-KLAT评分较治疗前均提高,且中剂量组显著高于小剂量组(t=2.496,P<0.05);中剂量组和小剂量组患儿不良反应发生率差异无统计学意义(χ^2=0.125,P>0.05),新增关节病变少于小剂量组,差异有统计学意义(χ^2=4.110,P<0.05)。结论小剂量与中剂量重组人凝血因子Ⅷ均可有效改善血友病A患儿关节损害,提高患儿生活质量,中剂量治疗疗效优于小剂量治疗。

Objective To explore the effects of different doses of recombinant human coagulation factorⅧon joint damage and quality of life in children with hemophilia A.Methods A total of 80 children with hemophilia A in Wenzhou People’s Hospital from January 2019 to December 2019 were selected as the research subjects,and randomly divided into low-dose group(10 U/kg recombinant human coagulation factorⅧ,once every 3 days)and medium-dose group(15~30 U/kg recombinant human coagulation factorⅧ,3 times a week).The bleeding,joint damage,quality of life,adverse reactions and new joint lesions were compared between the two groups after 4 months of treatment.The joint damage was evaluated by the Functional Independence Score in Hemophilia(FISH),and the quality of life was evaluated by the Canadian Hemophilia Outcomes-Kids’Life Assessment Tool(CHO-KLAT).Results The monthly bleeding frequency of the two groups was significantly lower than before treatment,and the medium-dose group was significantly lower than that in low-dose group(t=3.574,P<0.05).The number of emergency frequency and severe bleeding events in medium-dose group was significantly less than those in low-dose group(t/χ^2=4.986 and 3.914,separately,both P<0.05).After treatment,the scores of self-care,transfer function,mobility ability and total score of FISH scale in the two groups were improved compared with before treatment,and the medium-dose group was significantly higher than low-dose group(t=2.192,4.760,3.516 and 5.523,respectively,all P<0.05).The scores of CHO-KLAT of the two groups increased compared to before treatment,and the medium-dose group was significantly higher than low-dose group(t=2.496,P<0.05).There was no significant difference in the incidence of adverse reactions between the medium-dose group and low-dose group(χ^2=0.125,P>0.05),and the number of new joint lesions was less than that in low-dose group,and the difference was statistically significant(χ^2=4.110,P<0.05).Conclusion Both low-dose and medium-dose recombinant human coagulation factorⅧcan effectively improve joint damage and enhance the quality of life of children with hemophilia A.The efficacy of medium-dose therapy is better than low-dose therapy.