Efficacy and safety of eculizumab-combined therapy for patients with anti-GBM disease

Abstract Background The study aimed to evaluate the efficacy and safety of eculizumab in addition to conventional therapy for anti-glomerular basement membrane (anti-GBM) disease presenting rapidly progressive glomerulonephritis (RPGN). Methods 42 patients with anti-GBM disease presenting with RPGN were included in this multicenter retrospective cohort study. All patients received intensive immunosuppressive therapy including intravenous methylprednisolone pulses followed by maintenance prednisone, in combination with at least two of the following: intravenous cyclophosphamide, therapeutic plasma apheresis, rituximab, or eculizumab. The cohort was stratified into two groups: the eculizumab group (n = 13) which received eculizumab (900 mg once weekly for 1–5 doses) in combination with other therapies, and the control group (n = 29) which did not receive eculizumab. Kidney survival at 12 weeks and 24 weeks, and adverse events (infections, metabolic alterations, laboratory abnormalities), ascertained via clinical and laboratory data. Results At 12 weeks, the eculizumab group showed significantly higher kidney survival compared with the control group (69.2% vs. 31.0%, p = 0.021). At 24 weeks, kidney survival remained numerically higher in the eculizumab group (66.7% vs. 40.7%, p = 0.081). Kaplan-Meier curves for renal survival by the Gehan–Breslow–Wilcoxon test, which places greater emphasis on early events, revealed a statistically significant difference between the groups (χ² = 4.137; p = 0.042); however, the difference assessed by the log-rank test did not reach statistical significance (χ² = 3.491; p = 0.062). There were no significant differences in the overall incidence of infections or other adverse events. Conclusion Combined therapy with eculizumab showed improved early kidney survival for anti-GBM nephritis at 12 weeks, and did not increase the risk of adverse events. Prospective, large-sample studies with long-term follow-up are needed to validate its efficacy and safety.