Effects of immunotherapies and prognostic predictors in neuromyelitis optica spectrum disorder: a prospective cohort study
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Neuromyelitis optica spectrum disorder (NMOSD), a relapsing autoimmune demyelinating disease of the CNS, often leads to severe visual and/or motor disability. This study aimed to evaluate the long-term effects of the first-line immunotherapies on relapse and disability, and identify the prognostic predictors in NMOSD.
In this prospective cohort study, we enrolled patients with NMOSD from Southwest China and performed a long-term follow-up. We compared no immunotherapies (NIT) versus treatment of mycophenolate mofetil (MMF), azathioprine (AZA), or only corticosteroid (CS). Cox proportional-hazards model was used to explore the prognostic predictors in NMOSD.
Ultimately, 281 patients were enrolled during 2009 to 2017. The proportions of relapse, motor disability, and mortality were significantly lower in treatments of MMF and AZA than in NIT (all P < 0.001), while no significant difference was found between the CS and NIT groups. The multivariate Cox analyses indicated that onset with optic neuritis and increased age at onset were risk predictors of visual disability and motor disability, respectively. Comparing with NIT, MMF and AZA were remarkably reduced risk of relapse and motor disability but not visual disability. Additionally, median time to first relapse and motor disability was significantly longer in treatments of MMF and AZA than in NIT (both P < 0.001). Furthermore, we estimated the risk of relapse and disability for AQP4-Abs positive NMOSD in 1–5 years based on prognostic predictors identified above.
Our study revealed the potential predictors of relapse and disability, and strengthened evidence that early immunosuppressive treatments, such as MMF and AZA, could effectively reduce the risk of relapse and disability, and delayed progression of NMOSD.
KeywordsNueromyelitis optica spectrum disorders NMOSD Mycophenolate mofetil Azathioprine Outcomes Predictors
This work was supported by the National Key R&D Program of China (Grant No. 2017YFC0907704), the National Natural Science Foundation of China (Grant No. 81801202), and the Department of Science and Technology of Sichuan Province (Grant No. 2018SZ0388). We are very grateful to Mr. Chennan Wang (University of Electronic Science and Technology of China, Chengdu, China) and Dr. Xiaomeng Liu (Sichuan Cancer Hospital and Research Institute, Sichuan Cancer Center, Chengdu, China) for their important contributions to the statistical analysis and revision of our manuscript. We also thank Dr. Zhiyun Lian (Chongqing City Hospital of Traditional Chinese Medicine, Chongqing, China), Dr. Ju Liu (the First Affiliated Hospital of Zhengzhou University, Zhengzhou, Henan, China), and Dr. Huiru Feng (Hospital of Chengdu Office of People's Government of Tibetan Autonomous Region, Chengdu, Sichuan, China) for their contributions to data collection and follow-up of patients. Finally, we appreciate all of participants in this study for their supports.
Compliance with ethical standards
Conflicts of interest
The authors declare that they have no conflicts of interest, including relevant financial interests, activities, relationships, or affiliations.
This study was approved by the Medical Ethics Committee of West China Hospital, Sichuan University and was performed in accordance with the ethical standards of the Declaration of Helsinki (approval number: 2018-29). All participants provided informed consent prior to their inclusion in this study.
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