Jing Peng, Miriam Kessi, Fei Yin, Lifen Yang
INTRODUCTION: To perform systematic reviews and meta-analyses on the use of the tocilizumab in the central nervous system (CNS) diseases. METHODS: A systematic search was carried out in the PubMed, Embase and Cochrane databases for all years until August 2025. RESULTS: The review revealed 618 CNS diseases patients treated with tocilizumab. The proportion of neuromyelitis optica spectrum disorder (NMOSD) relapse-free patients after tocilizumab was 79.00% (95% confidence interval (CI): 0.67, 0.89). There was a significant correlation between remission and positive anti-AQP4 antibody status (p = 0.001). Tocilizumab significantly reduced the standardized mean Annualized Relapse Ratio (-1.3 (95% CI: -1.94, -0.66; p = 0.00191), Extended Disability Status Scale score (-0.48; 95% CI: -0.92, -0.04; p = 0.0329), and fatigue score (-1.08; 95% CI: -1.53, -0.63; p < 0.0001). The proportion of new-onset refractory status epilepticus (NORSE), febrile infection-related epilepsy syndrome (FIRES), and refractory status epilepticus patients who improved after tocilizumab was 95.00% (95% CI: 0.78, 1.00). The proportion of the anti-NMDAR encephalitis patients with a favorable outcome after tocilizumab was 73.00% (95% CI: 0.64, 0.81). Although other CNS diseases showed improvement following tocilizumab, they did not meet the criteria for meta-analyses. Tocilizumab altered several pro- and anti-inflammatory markers in humans, animals, and cell models. The pooled proportion of tocilizumab adverse effects was 45.00% (95% CI: 0.26, 0.65). DISCUSSION: This study may guide clinicians in managing patients with CNS diseases and in conducting prospective studies and clinical trials. CONCLUSION: Some of the CNS diseases may be treated with tocilizumab.