Objective: To investigate the efficacy and safety of tocilizumab in the treatment of critically ill children with acute necrotizing encephalopathy (ANE). Methods: It is a retrospective cohort study. The children with ANE admitted to the pediatric intensive care unit of 4 Chinese tertiary hospitals from December 2022 to November 2023 were divided into conventional treatment group and tocilizumab group, and the comparison between groups was performed by using Mann - Whitney U test or Chi-square test. Results: Among 21 cases of severe ANE, there were 11 males with the onset age of 65 (27, 113) months. The duration from onset to PICU admission was 2 (1, 2) days. There were 13 cases of ultra-high fever (greater than 40 ℃), including 18 cases of convulsions, and 19 cases with a GCS score of less than 8 points. The causative agent was novel coronavirus Omicron in 7 cases and influenza A in 14 cases. All cases had central respiratory failure requiring mechanical ventilation. Of the 21 cases, 18 were shock, 15 were coagulopathy, 10 were kidney injury and 13 were liver dysfunction. Of these hospitalized patients, 8 children with ANE were treated with tocilizumab. Eight cases received continuous blood purification (CBP) treatment, 5 of them were combined with plasmapheresis. Serum cytokine levels were elevated in 21 children with ANE, including (interleukin, IL)-6 and IL-8 (61 (22, 1 513) and 68 (5, 296) ng/L). There were 14 cases (67%) deaths, including 11 cases in the conventional treatment group and 3 cases in the tocilizumab group. There was no significant difference in the mortality rate between the two groups (P=0.056). Tocilizumab-related rash or other adverse events were not observed. Conclusions: The motality of critically ill ANE patients was high. The combination of Tocilizumab with conventional treatment did not reduce the motality of severe ANE patients, and no adverse reactions of tocilizumab were observed.
目的： 探讨托珠单抗治疗儿童危重急性坏死性脑病（ANE）的初步效果与安全性。 方法： 回顾性队列研究。总结国内4家三级甲等医院儿童重症监护病房（PICU）2022年12月至2023年11月收治的21例危重ANE患儿（ANE评分≥5分）的临床资料。根据是否使用托珠单抗分为常规治疗组和托珠单抗组，采用非参数秩和检验、χ2检验或Fisher确切概率法组间比较。 结果： 21例危重ANE患儿中男11例、女10例，发病年龄为65（27，113）月龄。发病至收住PICU时间为2（1，2）d。其中热峰>40 ℃者13例，惊厥18例，格拉斯哥昏迷（GCS）评分<8分19例。ANE患儿中7例病原为新型冠状病毒，14例为甲型流感病毒，均合并中枢性呼吸衰竭需呼吸机支持，休克18例，凝血功能障碍15例，急性肾损伤10例，肝功能障碍13例。8例患儿接受托珠单抗治疗。连续性血液净化（CBP）治疗8例（其中5例联合血浆置换）。21例ANE患儿血清白细胞介素（IL）-6和IL-8均升高［61（22，1 513）和68（5，296）ng/L］。死亡14例（67%），其中常规治疗组死亡11例、托珠单抗组死亡3例，两组病死率差异无统计学意义（P=0.056）。未观察到托珠单抗相关皮疹、休克等不良反应。 结论： 危重ANE患儿病死率高，联合托珠单抗治疗并未降低危重ANE患儿病死率，同时未观察到托珠单抗不良反应。.