PT - JOURNAL ARTICLE AU - Pellkofer, H.L. AU - Krumbholz, M. AU - Berthele, A. AU - Hemmer, B. AU - Gerdes, L.A. AU - Havla, J. AU - Bittner, R. AU - Canis, M. AU - Meinl, E. AU - Hohlfeld, R. AU - Kuempfel, T. TI - Long-term follow-up of patients with neuromyelitis optica after repeated therapy with rituximab AID - 10.1212/WNL.0b013e3182152881 DP - 2011 Apr 12 TA - Neurology PG - 1310--1315 VI - 76 IP - 15 4099 - http://n.neurology.org/content/76/15/1310.short 4100 - http://n.neurology.org/content/76/15/1310.full SO - Neurology2011 Apr 12; 76 AB - Background: Neuromyelitis optica (NMO) is a severe autoimmune disease targeting optic nerves and spinal cord. The monoclonal anti-CD20 B-cell antibody rituximab is an emerging therapeutic option in NMO. However, neither long-term efficacy or safety of rituximab, nor the correlation between B-cell counts, B-cell fostering cytokines, aquaporin-4 antibodies (AQP4-ab), and disease activity in NMO, have been investigated prospectively. Methods: We performed a prospective long-term cohort study of 10 patients with NMO who were treated up to 5 times with rituximab as a second-line therapy. Clinical examinations, B-cell counts, and serum concentrations of BAFF (B-cell activating factor of the TNF family; also called TNFSF13b), APRIL (a proliferation-inducing ligand; also called TNFSF13), AQP4-ab, and immunoglobulin levels were measured every 3 months. Results: Repeated treatment with rituximab led to sustained clinical stabilization in most patients with NMO. Disease activity correlated with B-cell depletion, but not clearly with AQP4-ab or levels of APRIL. BAFF levels increased after application of rituximab and indicated persisting efficacy of the drug but did not correlate with disease activity. Overall, rituximab was well-tolerated even after up to 5 consecutive treatment courses; however, we observed several severe adverse reactions. Conclusion: Our data indicate that long-term therapy with rituximab is effective in NMO as a second-line therapy and has an acceptable safety profile. Retreatment with rituximab should be applied before reappearance of circulating B cells. Classification of evidence: This study provides Class IV evidence that repeated doses of rituximab result in stabilization in most patients.