Efficacy of plasma exchange in children with severe hemophagocytic syndrome: a prospective randomized controlled trial北大核心CSCD

目的 探讨血浆置换在辅助救治儿童重症噬血细胞综合征（hemophagocytic syndrome，HPS）中的疗效和应用价值。 方法 采用前瞻性随机对照研究方法，选取2018年10月至2020年10月在湖南省儿童医院儿童重症监护室（pediatric intensive care unit，PICU）接受救治的重症HPS患儿40例为研究对象，将患儿随机分为置换组和常规组（n=20），常规组患儿进行病因和对症常规支持治疗，置换组在常规治疗的基础上加以血浆置换辅助治疗。对两组患儿的基本情况、治疗前后的临床症状体征、主要实验室检查指标、疗效和预后情况进行比较分析。 结果 两组患儿治疗前在性别、年龄、入院前病程、病因构成、小儿危重病例评分、器官或系统功能累及情况等指标上比较差异均无统计学意义（P>0.05），在实验室指标的比较上差异亦均无统计学意义（P>0.05）。两组患儿治疗7 d后，临床症状体征均有所缓解和改善。治疗后置换组C反应蛋白、降钙素原、血清铁蛋白、丙氨酸氨基转移酶、总胆红素水平均低于常规组（P<0.05）。置换组的PICU住院时间较常规组明显缩短，总有效率较常规组明显提高（P<0.05），但两组总的住院时间和3个月病死率比较差异无统计学意义（P>0.05）。 结论 血浆置换在辅助治疗儿童重症HPS上的疗效优于常规治疗，能改善患儿的临床症状体征和部分实验室指标，缩短PICU住院时间，可能成为治疗儿童重症HPS的一项有效辅助治疗方法。

Efficacy of plasma exchange in children with severe hemophagocytic syndrome: a prospective randomized controlled trial

Objective To investigate the efficacy and application value of plasma exchange as an adjuvant therapy in children with hemophagocytic syndrome (HPS). Methods A prospective randomized controlled trial was designed. Forty children with severe HPS were enrolled, who were treated in the pediatric intensive care unit (PICU) of Hunan Children's Hospital from October 2018 to October 2020. The children were randomly divided into a plasma exchange group and a conventional treatment group using a random number table, with 20 children in each group. The children in the conventional treatment group received etiological treatment and conventional symptomatic supportive treatment, and those in the plasma exchange group received plasma exchange in addition to the treatment in the conventional treatment group. The two groups were compared in terms of general information, clinical symptoms and signs before and after treatment, main laboratory markers, treatment outcome, and prognosis. Results Before treatment, there were no significant differences between the two groups in gender, age, course of the disease before admission, etiological composition, pediatric critical illness score, involvement of organ or system functions, and laboratory markers (P>0.05). After 7 days of treatment, both groups had remission and improvement in clinical symptoms and signs. After treatment, the plasma exchange group had significantly lower levels of C-reactive protein, procalcitonin, and serum protein levels than the conventional treatment group (P<0.05). The plasma exchange group also had significantly lower levels of alanine aminotransferase and total bilirubin than the conventional treatment group (P<0.05). The length of stay in the PICU in the plasma exchange group was significantly shorter than that in the conventional treatment group (P<0.05). The plasma exchange group had a significantly higher treatment response rate than the conventional treatment group (P<0.05). There were no significant differences between the two groups in the total length of hospital stay and 3-month mortality rate (P>0.05). Conclusions Plasma exchange as an adjuvant therapy is effective for children with severe HPS. It can improve clinical symptoms and signs and some laboratory markers and shorten the length of stay in the PICU, and therefore, it may become an optional adjuvant therapy for children with severe HPS.