HLH-2004方案治疗儿童噬血细胞综合征40例临床分析

Clinical analysis of 40 children with HLH diagnosed and treated by HLH-2004 regime

目的探讨儿童HLH的临床特征、发病危险因素、实验室特点以及采用HLH-2004方案治疗的疗效和预后情况。方法回顾分析2011年1月至2014年12月收治的40例HLH患儿的临床资料。诊断标准和治疗方案按照国际组织细胞协会HLH-2004方案。结果40例HLH患儿中,男24例、女16例,发病中位年龄1岁(4个月~10.5岁);2例有明确家族史,27例有明确诱因。主要临床表现为发热、肝脾肿大、出血、肺浸润、胸腔积液、中枢神经系统病变、皮疹等。实验室检查血细胞减少、肝功能异常、凝血异常、高三酰甘油血症、铁蛋白升高,骨髓可见噬血细胞。35例患儿采用HLH-2004方案治疗。存活26例,死亡14例。死亡原因主要为感染、弥散性血管内凝血和多脏器衰竭。结论儿童HLH病情凶险,临床表现复杂,须尽早采用HLH-2004方案诊疗。

Objective To investigate the clinical characteristics, family history, as well as the ef ficiency and prognosis of children with HLH diagnosed and treated by HLH-2004 regime. Methods A total of 40 children with HLH diagnosed in Xuzhou Children’s Hospital from January 2011 to December 2014 were enrolled. Clinical data including family history, clinical characteristics, laboratory results, treatment and prognosis were retrospectively analyzed. Results Among the 40 children with HLH, 24 were males and 16 were females. The median age was 1 year old (range from 4 months to 10.5 years old). Two cases had positive family history, 27 cases had known primary diseases, while 13 cases had no clear reasons. Main clinical manifestations included fever, hepatosplenomegaly, bleeding, pulmonary in filtration, pleural effusion, central nervous system lesions, rash and so on. Main laboratory abnormalities were cytopenia, abnormal liver function, coagulation abnormalities, hypertriglyceridemia, elevated ferritin, hemophagocytosis in bone marrow. There were 35 cases treated with HLH-2004 regimen, 26 cases survived and 14 cases died. The main causes of death were infection, disseminated intravascular coagulation and multiple organ failure. Conclusion HLH in Chinese children is an aggressive syndrome with complicated manifestations. Treatment with HLH-2004 regime resulted in a relatively good prognosis.