Abstract
BACKGROUND: Autoimmune nodopathy (AN) is a distinct CIDP-like entity defined by its poor response to standard treatments, including IVIG. The efficacy and safety of anti-CD20 monoclonal antibodies, a potential mechanism-based therapy, have not been quantitatively synthesized. OBJECTIVE: To systematically evaluate and quantitatively synthesize the efficacy and safety of anti-CD20 monoclonal antibody therapy in patients with AN. METHODS: A comprehensive literature search was conducted across PubMed, Web of Science, Cochrane Library, Embase, and ClinicalTrials.gov from inception to August 4, 2025. Studies reporting clinical outcomes of AN patients treated with anti-CD20 agents were included. A generalized linear mixed model (GLMM) was employed to estimate pooled response rates. RESULTS: Twenty-nine studies comprising 118 patients were included. In the descriptive synthesis, most reports described physician-assessed clinical improvement after anti-CD20 therapy. For quantitative pooling, we restricted the meta-analysis to studies reporting standardized, objective scale-based outcomes (n = 100), yielding a pooled clinical response rate of 92.0% (95% CI, 84.8-95.9%, I(2) = 0%). Subgroup analyses demonstrated sustained responsiveness in patients with anti-NF155 (95.2%) and anti-CNTN1 (88.9%) autoantibodies. Adverse events were recorded in 8.5% of patients (10/118), primarily consisting of mild infusion-related reactions. However, two fatalities (1.7%) associated with severe infection or comorbidities were noted. CONCLUSION: Anti-CD20 therapy has shown high efficacy in treating AN that is refractory to conventional treatments. However, due to the observational nature of the available data and the lack of randomized controlled trials, these results should be interpreted with caution and are not yet practice-changing. Further prospective, controlled studies are needed to better define the treatment's efficacy, optimal dosing strategies, and long-term safety.