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Neuromyelitis Optica Spectrum Disorder With Anti-Aquaporin-4 Antibody: Outcome Prediction Models.

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机构: [1]Department of Neurology, Huashan Hospital, Shanghai Medical College, Fudan University, Shanghai, China. [2]National Center for Neurological Disorders (NCND), Shanghai, China. [3]Department of Neurology, The First Affiliated Hospital of Xinjiang Medical University, Urumqi, China. [4]Department of Rehabilitation Medicine, Jing'an District Centre Hospital of Shanghai, Fudan University, Shanghai, China. [5]Department of Rehabilitation Medicine, Huashan Hospital, Shanghai Medical College, Fudan University, Shanghai, China. [6]Department of Neurology, Jing'an District Centre Hospital of Shanghai, Fudan University, Shanghai, China. [7]Department of Neurology, The Second Affiliated Hospital of Guangzhou University of Chinese Medicine, Guangzhou, China. [8]Department of Neurology, The First People's Hospital of Yunnan Province, Kunming, China. [9]Department of Neurology, Sir Run Run Shaw Hospital, School of Medicine, Zhejiang University, Hangzhou, China. [10]Department of Neurology, The First Affiliated Hospital of Wenzhou Medical University, Wenzhou, China. [11]Department of Neurology, The Fifth Affiliated Hospital of Zhengzhou University, Zhengzhou, China. [12]Department of Neurology, Wuhan No.1 Hospital, Wuhan, China. [13]Department of Neurology, Central Hospital Affiliated to Shandong First Medical University, Jinan, China. [14]Department of Ophthalmology and Vision Science, Eye and ENT Hospital, Shanghai Medical College, Fudan University, Shanghai, China.
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关键词: neuromyelitis optica spectrum disorder anti-aquaporin-4 antibody maintenance therapy outcome prediction model

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Recognizing the predictors of disease relapses in patients with anti-aquaporin-4 antibody (AQP4-ab)-positive neuromyelitis optica spectrum disorder (NMOSD) is essential for individualized treatment strategy. We aimed to identify the factors that predicted relapses among patients with AQP4-ab-positive NMOSD, develop outcome prediction models, and validate them in a multicenter validation cohort.Between January 2015 and December 2020, 820 patients with NMOSD were registered at Huashan Hospital. We retrospectively reviewed their medical records, and included 358 AQP4-ab-positive patients with 1135 treatment episodes. Univariate and multivariate analyses were used to explore the predictors of relapse, severe visual or motor disability during follow-up. A model predicting the 1- and 2-year relapse-free probability was developed and validated in an external validation cohort of 92 patients with 213 treatment episodes.Lower serum AQP4-ab titer (< 1:100), higher Expanded Disability Status Scale (EDSS) score at onset (≥ 2.5), and use of intravenous methylprednisolone (IVMP) at the first attack predicted an overall lower annualized relapse rate. Older age (> 48 years), optic neuritis at onset, and higher onset EDSS score (≥ 2.5) significantly increased the risk for blindness, while IVMP at the first attack and maintenance therapy reduced the risk for blindness. Myelitis at onset increased the possibility of motor disability (EDSS ≥ 6.0), severe motor disability or death (EDSS ≥ 8.0), while maintenance therapy reduced these possibilities. Anderson and Gill model identified that the risk factors predicting recurrent relapses under certain treatment status were female gender, high AQP4-ab titer (≥ 1:100), previous attack under same therapy, lower EDSS score at treatment initiation (< 2.5), and no maintenance therapy or oral prednisone lasting less than 6 months. A nomogram using the above factors showed good discrimination and calibration abilities. The concordance indexes in the primary and validation cohort were 0.66 and 0.65, respectively.This study reports the demographic, clinical and therapeutic predictors of relapse, and severe visual or motor disability in NMOSD. Early identification of patients at risk of unfavorable outcomes is of paramount importance to inform treatment decisions.Copyright © 2022 Wang, Du, Li, Li, Wang, Zhao, Meng, Li, Pan, Xia, Wu, Yang, Li, Ma, ZhangBao, Huang, Chang, Tan, Yu, Zhou, Lu, Wang, Dong, Lu, Zhao and Quan.

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大类 | 2 区 医学
小类 | 2 区 免疫学
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大类 | 2 区 医学
小类 | 2 区 免疫学
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Q1 IMMUNOLOGY
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Q1 IMMUNOLOGY

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第一作者机构: [1]Department of Neurology, Huashan Hospital, Shanghai Medical College, Fudan University, Shanghai, China. [2]National Center for Neurological Disorders (NCND), Shanghai, China.
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通讯机构: [1]Department of Neurology, Huashan Hospital, Shanghai Medical College, Fudan University, Shanghai, China. [2]National Center for Neurological Disorders (NCND), Shanghai, China.
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