RT Journal Article SR Electronic T1 Long-term follow-up of patients with neuromyelitis optica after repeated therapy with rituximab JF Neurology JO Neurology FD Lippincott Williams & Wilkins SP 1310 OP 1315 DO 10.1212/WNL.0b013e3182152881 VO 76 IS 15 A1 H.L. Pellkofer A1 M. Krumbholz A1 A. Berthele A1 B. Hemmer A1 L.A. Gerdes A1 J. Havla A1 R. Bittner A1 M. Canis A1 E. Meinl A1 R. Hohlfeld A1 T. Kuempfel YR 2011 UL http://n.neurology.org/content/76/15/1310.abstract 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.