血友病A抑制物产生的危险因素及相关治疗进展

Risk factors in inhibitors development of hemophilia A and advances treatment

血友病A(hemophilia A,HA)是一种由凝血因子Ⅷ (coagulation factorⅧ,FⅧ)活性降低引起的 X 染色体连锁隐性遗传的出血性疾病.临床上根据 FⅧ 活性水平分为重型(FⅧ:C<1%),中间型(1% <FⅧ:C<5%),轻型 (5% <FⅧ:C<40%)^(1).目前HA 仍以替代治疗为主,抑制物产生是其最严重的并发症之一,在重型 HA 中发病率达30%左右,通常发生在第9~12个暴露日^(2) .

Haemophilia A (HA)is an X-linked and inherited bleeding disorder which is resulted from deficien-cy of coagulant factorⅧ (FⅧ).The severity of hemophilia is classified based on plasma levels of FⅧ activity:se-vere if<1%,moderate if between 1and 5%and mild if>5and<40%of normal.Inhibitor formation is among the most severe complications of hemophilia treatment,with a cumulative incidence of 30%in HA patients.And it usu-ally occurs on the 9th-12th exposure day.Inhibitors patients become unresponsive to standard factor treatment,re-sulting in 2-fold the hospitalizations,10-fold the cost,and 3.5-fold mortality of noninhibitor patients.Furthermore,inhibitors are a recognized contraindication to gene therapy.