RT Journal Article SR Electronic T1 Interleukin-6 Receptor Blockade in Treatment-Refractory MOG-IgG–Associated Disease and Neuromyelitis Optica Spectrum Disorders JF Neurology - Neuroimmunology Neuroinflammation JO Neurol Neuroimmunol Neuroinflamm FD Lippincott Williams & Wilkins SP e1100 DO 10.1212/NXI.0000000000001100 VO 9 IS 1 A1 Ringelstein, Marius A1 Ayzenberg, Ilya A1 Lindenblatt, Gero A1 Fischer, Katinka A1 Gahlen, Anna A1 Novi, Giovanni A1 Hayward-Könnecke, Helen A1 Schippling, Sven A1 Rommer, Paulus S. A1 Kornek, Barbara A1 Zrzavy, Tobias A1 Biotti, Damien A1 Ciron, Jonathan A1 Audoin, Bertrand A1 Berthele, Achim A1 Giglhuber, Katrin A1 Zephir, Helene A1 Kümpfel, Tania A1 Berger, Robert A1 Röther, Joachim A1 Häußler, Vivien A1 Stellmann, Jan-Patrick A1 Whittam, Daniel A1 Jacob, Anu A1 Kraemer, Markus A1 Gueguen, Antoine A1 Deschamps, Romain A1 Bayas, Antonios A1 Hümmert, Martin W. A1 Trebst, Corinna A1 Haarmann, Axel A1 Jarius, Sven A1 Wildemann, Brigitte A1 Grothe, Matthias A1 Siebert, Nadja A1 Ruprecht, Klemens A1 Paul, Friedemann A1 Collongues, Nicolas A1 Marignier, Romain A1 Levy, Michael A1 Karenfort, Michael A1 Deppe, Michael A1 Albrecht, Philipp A1 Hellwig, Kerstin A1 Gold, Ralf A1 Hartung, Hans-Peter A1 Meuth, Sven G. A1 Kleiter, Ingo A1 Aktas, Orhan A1 , YR 2022 UL http://nn.neurology.org/content/9/1/e1100.abstract AB Background and Objectives To evaluate the long-term safety and efficacy of tocilizumab (TCZ), a humanized anti–interleukin-6 receptor antibody in myelin oligodendrocyte glycoprotein–IgG–associated disease (MOGAD) and neuromyelitis optica spectrum disorders (NMOSD).Methods Annualized relapse rate (ARR), Expanded Disability Status Scale score, MRI, autoantibody titers, pain, and adverse events were retrospectively evaluated in 57 patients with MOGAD (n = 14), aquaporin-4 (AQP4)-IgG seropositive (n = 36), and seronegative NMOSD (n = 7; 12%), switched to TCZ from previous immunotherapies, particularly rituximab.Results Patients received TCZ for 23.8 months (median; interquartile range 13.0–51.1 months), with an IV dose of 8.0 mg/kg (median; range 6–12 mg/kg) every 31.6 days (mean; range 26–44 days). For MOGAD, the median ARR decreased from 1.75 (range 0.5–5) to 0 (range 0–0.9; p = 0.0011) under TCZ. A similar effect was seen for AQP4-IgG+ (ARR reduction from 1.5 [range 0–5] to 0 [range 0–4.2]; p < 0.001) and for seronegative NMOSD (from 3.0 [range 1.0–3.0] to 0.2 [range 0–2.0]; p = 0.031). During TCZ, 60% of all patients were relapse free (79% for MOGAD, 56% for AQP4-IgG+, and 43% for seronegative NMOSD). Disability follow-up indicated stabilization. MRI inflammatory activity decreased in MOGAD (p = 0.04; for the brain) and in AQP4-IgG+ NMOSD (p < 0.001; for the spinal cord). Chronic pain was unchanged. Regarding only patients treated with TCZ for at least 12 months (n = 44), ARR reductions were confirmed, including the subgroups of MOGAD (n = 11) and AQP4-IgG+ patients (n = 28). Similarly, in the group of patients treated with TCZ for at least 12 months, 59% of them were relapse free, with 73% for MOGAD, 57% for AQP4-IgG+, and 40% for patients with seronegative NMOSD. No severe or unexpected safety signals were observed. Add-on therapy showed no advantage compared with TCZ monotherapy.Discussion This study provides Class III evidence that long-term TCZ therapy is safe and reduces relapse probability in MOGAD and AQP4-IgG+ NMOSD.ADEM=acute disseminated encephalomyelitis; AQP4=aquaporin-4; ARR=annualized relapse rate; AZA=azathioprine; EDSS=Expanded Disability Status Scale; HDS=high-dose steroid; IL-6=interleukin-6; IQR=interquartile range; IVIG=IV immunoglobulin; LDS=low-dose steroid; MMF=mycophenolate mofetil; MOG=myelin oligodendrocyte glycoprotein; MOGAD=myelin oligodendrocyte glycoprotein–IgG–associated disease; MTX=methotrexate; NMOSD=neuromyelitis optica spectrum disorder; ON=optic neuritis; RTX=rituximab; SLE=systemic lupus erythematosus; TCZ=tocilizumab; UTI=urinary tract infection