Background Neuromyelitis optica spectrum disorders (NMOSD) and myelin oligodendrocyte glycoprotein-associated disease (MOGAD) are autoimmune antibody-mediated diseases. Efgartigimod is a neonatal Fc receptor-targeting therapeutic that causes a reduction in the antibody titers. The efficacy and the safety of efgartigimod as an add-on therapy with intravenous methylprednisolone (IVMP) in patients with NMOSD and MOGAD were assessed in this study. Methods In total, 27 adult patients diagnosed with NMOSD or MOGAD were enrolled, with 13 patients treated with IVMP plus efgartigimod and 14 comparable controls treated with IVMP alone. Efgartigimod was administered intravenously at 10 mg/kg at four doses or 20 mg/kg at two doses. The Expanded Disability Status Scale (EDSS) scores, the serum immunoglobulin G (IgG) levels, and the pathogenic antibody titers were evaluated before and after therapy. Results Compared with IVMP alone, the efgartigimod group achieved better outcome with EDSS reduction of 1.3 ± 0.6 ( p 0.05) compared with the control group (0.5 ± 0.5). In the efgartigimod-treated group, the serum IgG levels decreased by 69.8% after therapy ( p 0.001), and nine patients (69.2%) showed a reduction in antibody titers. Moreover, the EDSS and antibody titers showed a rapid downward trend in the intensive therapy cohort (20 mg/kg, two doses). Conclusions In this preliminary study, efgartigimod add-on therapy showed better trends than IVMP alone in accelerating short-term recovery in patients with NMOSD and MOGAD at the acute phase.
Yin et al. (Tue,) studied this question.