Ju Yalin, Geng Chang, Guan Hongzhi
Department of Neurology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Beijing, 100730, China.
J Neurol. 2025 Sep 7;272(9):618. doi: 10.1007/s00415-025-13355-y.
To evaluate the clinical efficacy of tocilizumab, a interleukin-6 (IL-6) receptor blocker, for the treatment of acute necrotizing encephalopathy (ANE).
PubMed, Cochrane Library, Embase, and Web of Science were searched for systematic review based on PRISMA guidelines. ANE patients treated with and without tocilizumab were included. Methodological quality was assessed independently by two authors. Data on clinical features, neuroimaging patterns and outcomes were analyzed.
In total, 77 cases from 21 studies were included. Most patients had fever and seizures. Respiratory viruses were common precipitating infection. All patients had bilateral thalamic lesions, and brainstem lesions were more frequently observed in the tocilizumab group (88.0% vs 64.0%, p = 0.04). The length of hospital stay was significantly longer in the tocilizumab group both before (p = 0.01) and after propensity score matching (PSM) (p = 0.02), while the mortality rate was significantly lower in tocilizumab compared with non-tocilizumab group (8.0% vs 33.3%, p = 0.02). Initiation of tocilizumab within 24 h was related to a good outcome both before (p < 0.01) and after PSM (p < 0.01). In multivariate regression analysis, elder age and < 24 h immunotherapy were independently related with good outcome (age: aOR = 1.35; 95% CI 1.06-1.74, p = 0.02; < 24 h immunotherapy: aOR = 8.60; 95% CI 1.08-68.63, p = 0.04). Brainstem lesions was an independent risk factor of poor outcome (aOR = 0.03; 95% CI 0.003-0.29, p < 0.01). Tocilizumab use was independently associated with reduced mortality (aOR = 7.95, 95%CI 1.47-43.03, p = 0.02). No adverse effects were reported.
This review suggests that timely initiation of tocilizumab therapy within 24 h is safe, and may be an effective treatment in ANE, providing a strong rationale for a clinical trial.
评估白细胞介素-6（IL-6）受体阻滞剂托珠单抗治疗急性坏死性脑病（ANE）的临床疗效。
根据PRISMA指南，检索PubMed、Cochrane图书馆、Embase和Web of Science进行系统评价。纳入接受和未接受托珠单抗治疗的ANE患者。由两位作者独立评估方法学质量。分析临床特征、神经影像学表现和结局的数据。
共纳入21项研究中的77例病例。大多数患者有发热和癫痫发作。呼吸道病毒是常见的诱发感染因素。所有患者均有双侧丘脑病变，托珠单抗组脑干病变的发生率更高（88.0%对64.0%，p = 0.04）。在倾向得分匹配（PSM）之前（p = 0.01）和之后（p = 0.02），托珠单抗组的住院时间均显著更长，而托珠单抗组的死亡率显著低于未使用托珠单抗组（8.0%对33.3%，p = 0.02）。在PSM之前（p < 0.01）和之后（p < 0.01），在24小时内开始使用托珠单抗均与良好结局相关。在多因素回归分析中，年龄较大和免疫治疗时间<24小时与良好结局独立相关（年龄：调整后比值比[aOR]=1.35；95%置信区间[CI]1.06 - 1.74，p = 0.02；免疫治疗时间<24小时：aOR = 8.60；95% CI 1.08 - 68.63，p = 0.04）。脑干病变是预后不良的独立危险因素（aOR = 0.03；95% CI 0.003 - 0.29，p < 0.01）。使用托珠单抗与降低死亡率独立相关（aOR = 7.95，95% CI 1.47 - 43.03，p = 0.02）。未报告不良反应。
本综述表明，在24小时内及时开始托珠单抗治疗是安全的，可能是治疗ANE的有效方法，为临床试验提供了有力依据。
