儿童噬血细胞综合征的临床特征及预后分析

Analysis of clinical characteristics and prognostic factors of hemophagocytic lymphohistiocytosis in children

目的分析噬血细胞综合征(HLH)患儿的临床特点、实验室检查、疗效评估及影响预后的因素,提高对HLH认识并评估危险因素。方法回顾性分析2016年12月—2021年5月湖南省儿童医院收治的142例HLH患儿的临床资料,分析临床特征、实验室检查结果、转归和危险因素。结果142例患儿中,男性78例(54.9%),女性64例(45.1%),中位年龄2.4(1.4,4.3)岁。截止至随访时间,死亡34例(24.0%),存活100例(70.4%),失访8例(5.6%)。缓解组5年OS明显高于非缓解组(83.3%vs.38.9%),差异有统计学意义(P<0.001),多器官功能受累组5年OS明显低于少器官功能受累组为(63.8%vs.85.7%),差异有统计学意义(P=0.026),CNS受累组5年OS明显低于非CNS受累组(29.1%vs.88.5%),差异有统计学意义(P<0.001)。单因素Cox回归分析,血小板<30×10^(9)/L、多器官功能障碍、CNS受累、年龄≤3岁、消化道出血是影响患儿预后的危险因素(P值分别为P<0.001、P=0.020、P<0.001、P=0.004,和P=0.026),2周疗效评估获得缓解是影响患儿预后的保护因素(P<0.001)。多因素Cox回归分析,多器官功能障碍、CNS受累、血小板<30×10^(9)/L是影响患儿预后的独立危险因素(P值分别为0.007、<0.001和0.006),2周疗效评估获得缓解是影响患儿预后的独立保护因素(P<0.001)。结论HLH病死率高,临床表现多样,以多脏器功能障碍常见,多器官功能障碍、CNS受累、血小板<30×10^(9)/L是影响患儿预后的独立危险因素;2周疗效评估获得缓解是影响患儿预后的独立保护因素。

Objective To analyze the clinical features,laboratory examination,therapeutic effect and prognostic factors in children with hemophagocytic lymphohistiocytosis(HLH),so as to improve the understanding of HLH and to evaluate the risk factors.Methods Clinical data of 142 children with HLH admitted to Hunan Children′s Hospital from December 2016 to May 2021 were collected retrospectively,and their clinical characteristics,laboratory findings,prognosis and risk factors were analyzed.Results Among 142 patients,there were 78 males(54.9%)and 64 females(45.1%).The median age was 2.4(1.4,4.3)years old.By the date of last follow-up,34 cases(24.0%)died,100 cases(70.4%)survived,and 8 cases(5.6%)were lost to follow-up.The 5-year overall survival(OS)rate in the remission group was significantly higher than that in the non-remission group(83.3%vs.38.9%,P<0.001).The 5-year OS rate in the multiple organ dysfunction group was significantly lower than those in the minor organ dysfunction group(63.8%vs.85.7%,P=0.026).The 5-year OS rate in the central nerve system(CNS)involved group were significantly lower than those in the non-CNS involved group(29.1%vs.88.5%,P<0.001).Univariate Cox regression analysis showed that platelet<30×10^(9)/L,multiple organ dysfunction,CNS involvement,age≤3 years old and gastrointestinal bleeding were the risk factors that affected the prognosis of the children(P<0.001,P=0.020,P<0.001,P=0.004,and P=0.026)and 2-week remission was a protective factor affecting the prognosis(P<0.001).Multivariate Cox regression analysis showed that multiple organ dysfunction,CNS involvement and platelet<30×10^(9)/L were the independent risk factors affecting the prognosis of children(P=0.007,P<0.001 and P=0.006)and 2-week remission was an independent protective factor affecting the prognosis(P<0.001).Conclusions HLH has high mortality and various clinical manifestations,multiple organ dysfunction syndrome is common in HLH patients.Multiple organ dysfunction syndrome,CNS involvement and platelets<30×10^(9)/L were the independent risk factors affecting the prognosis of children with HLH,The remission of two-week was an independent protective factor for the children.