长期小剂量凝血因子Ⅷ预防重度血友病A患儿关节出血的疗效与相关因素分析

摘要

Objective To evaluate the efficacy of low-dose long-course infusion of recombinant human factor Ⅷ in treating joint bleeding in children with severe hemophilia. Methods Sixteen children aged from 2 to 16 years with severe hemophilia A were enrolled in the present study. Children in the treatment group were treated with low-dose long-course infusion of recombinant human factor Ⅷ with a dose of 5 ~ 15 U/kg twice-weekly as prophylaxis for one year. The incidence of joint bleeding one year in control group and treatment group was observed. Moreover, the incidence of their target joint bleeding and factor Ⅷ inhibitor formation before and after the treatment were measured in both groups. The changes in safety, including syphilis, AIDS, hepatitis B, hepatitis C, hepatitis D, hepatitis E and liver function were also observed. Results Joint bleedings occurred in the treatment group were significantly less than that in the control group ( P < 0. 01) . Meanwhile, the incidence of target joint bleedings in the treatment group was 14. 6% , which was obviously lower than that in the control group ( 45. 2% ) ( P < 0. 01) . The incidence of inhibitor formation of factor Ⅷ and the antibody titre were lower in the treatment group. Syphilis, AIDS, hepatitis B, hepatitis C, hepatitis D, hepatitis E were all negative, and the liver function was normal after treatment. Conclusions Low-dose long-course infusion of recombinant human factor Ⅷ can effectively decrease joint bleeding in children with severe hemophilia A. It also can effectively decrease the incidence of target joint bleeding. Therefore, this method may play an important role in protecting the joint function in those patients. Besides, this treatment is safe because of the lower antibody titre and inhibitor formation incidence.%目的 探讨重组人凝血因子Ⅷ(FⅧ)长期小剂量次级预防重度血友病A患儿关节出血的疗效与相关因素.方法 对2010年4月1日-2011年4月1日我院16位2～16岁重度血友病A患儿进行FⅧ预防性静脉输注(每次5～15 u/kg,间隔3 d),记录治疗前1年与治疗后1年的关节出血次数,同一关节反复出血的情况,治疗前后FⅧ抑制物产生情况,梅毒、艾滋病、乙肝、丙肝、丁肝、戊肝感染情况及肝功能变化.结果 治疗前1年关节出血次数为(29.69±4.48),治疗后1年关节出血次数为(10.94±3.30)次,治疗前后比较差异有显著性(P＜0.01).治疗前靶关节出血发生率为45.2%,治疗后靶关节出血发生率为14.6%,治疗前后比较差异有显著性(P＜0.01).治疗后FⅧ抑制物产生率为12.5%,产生率低及抗体滴度低,治疗前后梅毒、艾滋病、乙肝、丙肝、丁肝、戊肝均阴性,肝功能正常.结论 FⅧ长期小剂量次级预防输注可有效减少中重度血友病A患儿关节出血次数,同时可有效减少靶关节出血发生率,从而在一定程度上保护关节的功能,且FⅧ抑制物产生率及滴度低,无相关疾病传播,安全可靠.