摘要目的 总结本院非典型溶血尿毒综合征(aHUS)患儿的临床资料,对治疗及预后进行分析.方法 对2011年1月至2017年12月首都医科大学附属北京儿童医院确诊的66例患儿进行前瞻性队列研究,根据抗H因子抗体检测结果分为阳性组和阴性组,比较两组患儿的临床特点、治疗方案及预后.结果 符合入组标准的66例患儿中,抗H因子抗体阳性组43例(65.2％),平均(8.0±2.9)岁;抗体阴性组23例(34.8％),平均(3.0±2.6)岁.在血浆治疗基础上,抗体阳性组激素使用率为83.3％(35/42),免疫抑制剂使用率为42.9％(18/42);抗体阴性组激素使用率为63.6％(14/22),免疫抑制剂使用率为13.6％(3/22).平均随访19.3个月,两组中各有1例患儿失访.抗体阳性组8例复发(19.0％),复发间期平均为16.1个月;抗体阴性组7例复发(31.8％),复发间期平均为9.3个月,抗体阳性组复发间期较抗体阴性组长(P＜0.05).85.9％(55/64)的患儿溶血完全控制、肾功能完全恢复,其中抗体阳性组为85.7％(36/42),抗体阴性组为86.4％(19/22);7.8％(5/64)的患儿肾功能异常,其中抗体阳性组为9.5％ (4/42),抗体阴性组为4.5％(1/22);4.7％ (3/64)患儿死亡,其中抗体阳性组为2.4％(1/42),抗体阴性组为9.1％(2/22);1.6％(1/64)患儿透析依赖,为抗体阳性患儿.多因素Cox回归分析提示,年龄小于3岁为预后不良的危险因素(HR=4.651,95％CI 0.988 ～ 21.898,P=0.047).结论 抗H因子抗体阳性在aHUS患儿中所占比例高,发病年龄较大.根据抗H因子抗体结果加用免疫抑制剂的个体化治疗对于疾病缓解、预防复发及改善预后具有重要意义.年龄小于3岁为aHUS患儿预后不良的危险因素.

abstractsObjective To summarize the clinical data of atypical hemolytic uremic syndrome (aHUS) and analyze the treatment and prognosis.Methods A prospective cohort study was conducted on 66 cases in Beijing Children's Hospital affiliated to Capital Medical University from January 2011 to December 2017.The children were divided into positive and negative auto-antibody groups according to the results of anti-factor H autoantibody test.The clinical characteristics,treatment plan and prognosis of the two groups were compared.Results Among the 66 children who met the inclusion criteria,there were 43 cases (65.2％) in the positive group,with an average onset age of (8.0±2.9) years.There were 23 eases (34.8％) in the negative group,with an average onset age of (3.0± 2.6) years.On the basis of plasma treatment,in the positive group,the usage rate of hormone was 83.3％ (35/42) and the usage rate of immunosuppressive agents was 42.9％(18/42),while in the negative group,the rates were 63.6％(14/22) and 13.6％(3/22) respectively.The average follow-up time was 19.3 months.One child in each group was lost to follow-up.In the positive group,8 cases recurred (19.0％)and the average recurrence interval time was 16.1 months.In the negative group,7 cases recurred (31.8％) and the average recurrence interval time was 9.3 months.And the recurrent interval time in the positive group was more longer than the negative group (P ＜ 0.05).A total of 85.9％(55/64) children had complete hemolysis control and complete recovery of renal function,in which the positive group was 85.7％(36/42) and negative group was 86.4％(19/22).However,7.8％(5/64) children had abnormal renal function,in which the positive group was 9.5％(4/42) and the negative group was 4.5％(1/22).And 4.7％(3/64) children died,in which the positive group was 2.4％(1/42) and the negative group was 9.1％ (2/22).The one left (1.6％) showed dialysis dependence,which was positive for the auto-antibody.Multifactor Cox regression analysis showed that the age of less than 3 years old was the risk factor of poor prognosis (HR=4.651,95％CI 0.988-21.898,P=0.047).Conclusions The positive proportion of anti-factor H autoantibody in children with aHUS is high.The age of these children is older.Individualized therapy based on anti-factor H autoantibody and immunosuppressive therapy is of great significance for disease remission,preventing recurrence and improving the prognosis.Age less than 3 years old is the risk factor for poor prognosis.