Discontinuation of Immunosuppressive Therapy in Patients With Neuromyelitis Optica Spectrum Disorder With Aquaporin-4 Antibodies

Objective To evaluate the outcomes of immunosuppressive therapy (IST) discontinuation in patients with neuromyelitis optica spectrum disorder (NMOSD) after a sustained remission period. Methods We retrospectively reviewed the medical records of 17 patients with antiaquaporin-4 antibodypositive NMOSD who discontinued IST after a relapse-free period of ≥3 years. Results IST was discontinued at a median age of 40 years (interquartile range [IQR], 32–51) after a median relapse-free period of 62 months (IQR, 52–73). Among the 17 enrolled patients, 14 (82%) relapsed at a median interval of 6 months (IQR, 4–34) after IST discontinuation, 3 (18%) of whom experienced severe attacks; notably, all 3 of these patients had a history of severe attack before IST. These 3 patients received steroids, followed by plasma exchange for acute treatment, but 2 exhibited poor recovery and significant disability worsening at 6 months after relapse. Conclusions IST discontinuationmay increase the risk of relapse in seropositive patients with NMOSD even after 5 years of remission. Given the potentially devastating consequence of a single attack of NMOSD, caution is advised with IST discontinuation, particularly in patients with severe attack before IST. From the Department of Neurology, Research Institute and Hospital of National Cancer Center, Goyang, South Korea. Go to Neurology.org/NN for full disclosures. Funding information is provided at the end of the article. The Article Processing Charge was funded by the authors. This is an open access article distributed under the terms of the Creative Commons Attribution-NonCommercial-NoDerivatives License 4.0 (CC BY-NC-ND), which permits downloading and sharing the work provided it is properly cited. The work cannot be changed in any way or used commercially without permission from the journal. Copyright © 2021 The Author(s). Published by Wolters Kluwer Health, Inc. on behalf of the American Academy of Neurology. 1 Neuromyelitis optica spectrum disorder (NMOSD) is a rare and severe inflammatory disorder of the CNS associated with aquaporin-4 (AQP4) antibodies. Because of the high morbidity associated with NMOSD relapse, early initiation of immunosuppressive therapy is recommended. Rituximab, mycophenolate mofetil (MMF), and azathioprine (AZA) are the most commonly prescribed therapeutic agents for preventing relapse in NMOSD. Although these agents are generally well-tolerated, the long-term use of these drugs may lead to an increased risk of opportunistic infections, particularly in elderly patients, resulting in increased economic burden. However, there is no current consensus regarding the optimum duration of immunosuppressive therapy (IST), and a common clinical dilemma is the feasibility of treatment withdrawal in patients who have achieved a sustained period of remission. Here, we aimed to evaluate the outcomes of IST discontinuation in patients with NMOSD with a sustained remission period before discontinuation.