摘要
目的 探讨四川省血友病A患儿凝血因子Ⅷ(FⅧ)抑制物发生的危险因素、治疗、转归以及抑制物对血友病A患儿出血特征及疾病家庭负担的影响.方法 回顾性分析2015年6月1日至2018年4月1日在我院血友病中心登记注册并长期随访抑制物的113例血友病A患儿病例资料.采用病例对照研究方法分析抑制物发生的危险因素.结果 在纳入研究的113例血友病A患儿中,重型31例(27.44%),中间型77例(68.14%),轻型5例(4.42%).抑制物阳性18例(15.93%),其中重型患儿12例,中间型6例.低滴度抑制物10例,高滴度抑制物8例,抑制物产生后4例高滴度患儿采用免疫耐受诱导治疗,目前中位随访时间7(7~14)个月,抑制物滴度较治疗前均有降低.其余14例出血时选择加大剂量凝血因子Ⅷ(FⅧ)、凝血酶原复合物(PCC)或基因重组活化凝血因子Ⅶ(rFⅦa)治疗,抑制物中位随访时间15.5(2-32)个月,10例低滴度转阴.抑制物产生前后患儿关节年出血率、疾病家庭负担差异无统计学意义.相关性分析显示抑制物形成与疾病程度、首次暴露原因、累积暴露日相关(r分别为0.369、-0.332、-0.528),与血友病家族史、首次暴露月龄、严重出血事件无相关性(r分别为-0.178、0.007、-0.131).结论 疾病程度、首次暴露原因和累积暴露日与抑制物形成相关,抑制物产生后治疗难度加大,但不会增加关节年出血率及疾病家庭负担.
Objective To analyze the risk factors,characteristics and treatment for children hemophilia A(HA)with factorⅧinhibitor,and to investigate the effect of inhibitors with bleeding phenotype and disease burden of family.Methods The clinical data of 113 HA patients from June 2015 to April 2018 were collected.A case control study was performed to investigate the risk factors for inhibitor development.Results A total of 113 HA children were enrolled.31(27.44%)were severe,77(68.14%)moderate,5(4.42%)mild cases.18(15.93%)patients developed inhibitors,12 were severe HA,6 moderate HA and 10 with low-titer inhibitors,8 with high-titer inhibitors.After inhibitor formation,four patients received ITI therapy,and showed an inhibitors titer decrease but remained positive after a median of 7(7-14)months.The other patients received FⅧ、PCC or rFⅦa treatment to stop the bleeding,10 patients with low titer inhibitors showed gradual disappearance after a median of 15.5(2-32)months.The annual joints bleeding rate and disease burden of family showed no significantly difference after inhibitor formation.Correlation Analysis showed that the degree of disease,the reason of first factorⅧtreatment and the cumulative exposure days were risk factors of inhibitor development(r=0.369、-0.332,0.528).whereas,family history,age at first exposure to factorⅧand severe bleeding episodes were not(r=-0.178,0.007,-0.131).Conclusion Our data suggested that the degree of disease,the reason of first factorⅧtreatment and the cumulative exposure days were risk factors of inhibitor development.Although treatment for the patients with inhibitors became more difficult,the annual joints bleeding rate and the disease burden of family did not increase.
作者
张庆
杨小兰
刘晓丽
张秋
蔡晓燕
李晓静
周敏
ZHANG Qing;YANG Xiao-lan;LIU Xiao-li;ZHANG Qiu;CAI Xiao-yan;LI Xiao-jing;ZHOU Min(Depanment of Hematology,and 0ncology,Chendu women and Children’s Central Hospital;Chend u New Century Women’s and Children’s Hospital,Chengdu,610091,China)
出处
《血栓与止血学》
2019年第1期4-7,共4页
Chinese Journal of Thrombosis and Hemostasis
