Effects of immunotherapies and prognostic predictors in neuromyelitis optica spectrum disorder: a prospective cohort study

Background 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 p...

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Veröffentlicht in:Journal of neurology 2020-04, Vol.267 (4), p.913-924
Hauptverfasser: Shi, Ziyan, Du, Qin, Chen, Hongxi, Zhang, Ying, Qiu, Yuhan, Zhao, Zhengyang, Wang, Jiancheng, Yan, Chao, Zhang, Qin, Yang, Mu, Zhou, Hongyu
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container_end_page 924
container_issue 4
container_start_page 913
container_title Journal of neurology
container_volume 267
creator Shi, Ziyan
Du, Qin
Chen, Hongxi
Zhang, Ying
Qiu, Yuhan
Zhao, Zhengyang
Wang, Jiancheng
Yan, Chao
Zhang, Qin
Yang, Mu
Zhou, Hongyu
description Background 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. Methods 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. Results 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  
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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. Methods 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. Results 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  &lt; 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  &lt; 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. Conclusions 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.</description><identifier>ISSN: 0340-5354</identifier><identifier>EISSN: 1432-1459</identifier><identifier>DOI: 10.1007/s00415-019-09649-7</identifier><identifier>PMID: 31776721</identifier><language>eng</language><publisher>Berlin/Heidelberg: Springer Berlin Heidelberg</publisher><subject>Adrenal Cortex Hormones - administration &amp; dosage ; Adrenal Cortex Hormones - pharmacology ; Aquaporin 4 ; Autoimmune diseases ; Azathioprine ; Azathioprine - administration &amp; dosage ; Azathioprine - pharmacology ; Cohort analysis ; Corticosteroids ; Demyelinating diseases ; Demyelination ; Disabled Persons ; Follow-Up Studies ; Health risk assessment ; Humans ; Immunosuppressive Agents - administration &amp; dosage ; Immunosuppressive Agents - pharmacology ; Immunotherapy ; Long-term effects ; Medicine ; Medicine &amp; Public Health ; Mycophenolate mofetil ; Mycophenolic acid ; Mycophenolic Acid - administration &amp; dosage ; Mycophenolic Acid - pharmacology ; Neuritis ; Neurology ; Neuromyelitis ; Neuromyelitis Optica - drug therapy ; Neuromyelitis Optica - physiopathology ; Neuroradiology ; Neurosciences ; Optic neuritis ; Original Communication ; Outcome Assessment, Health Care ; Prognosis ; Proportional Hazards Models ; Prospective Studies ; Risk Factors ; Secondary Prevention ; Sensorimotor integration</subject><ispartof>Journal of neurology, 2020-04, Vol.267 (4), p.913-924</ispartof><rights>Springer-Verlag GmbH Germany, part of Springer Nature 2019</rights><rights>Journal of Neurology is a copyright of Springer, (2019). All Rights Reserved.</rights><lds50>peer_reviewed</lds50><woscitedreferencessubscribed>false</woscitedreferencessubscribed><citedby>FETCH-LOGICAL-c441t-b58ac6eb1df7ebb38cb6f495610f124eef9d27e7db1576e716c875cfed0127673</citedby><cites>FETCH-LOGICAL-c441t-b58ac6eb1df7ebb38cb6f495610f124eef9d27e7db1576e716c875cfed0127673</cites><orcidid>0000-0001-6959-6947</orcidid></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><linktopdf>$$Uhttps://link.springer.com/content/pdf/10.1007/s00415-019-09649-7$$EPDF$$P50$$Gspringer$$H</linktopdf><linktohtml>$$Uhttps://link.springer.com/10.1007/s00415-019-09649-7$$EHTML$$P50$$Gspringer$$H</linktohtml><link.rule.ids>314,776,780,27901,27902,41464,42533,51294</link.rule.ids><backlink>$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/31776721$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink></links><search><creatorcontrib>Shi, Ziyan</creatorcontrib><creatorcontrib>Du, Qin</creatorcontrib><creatorcontrib>Chen, Hongxi</creatorcontrib><creatorcontrib>Zhang, Ying</creatorcontrib><creatorcontrib>Qiu, Yuhan</creatorcontrib><creatorcontrib>Zhao, Zhengyang</creatorcontrib><creatorcontrib>Wang, Jiancheng</creatorcontrib><creatorcontrib>Yan, Chao</creatorcontrib><creatorcontrib>Zhang, Qin</creatorcontrib><creatorcontrib>Yang, Mu</creatorcontrib><creatorcontrib>Zhou, Hongyu</creatorcontrib><title>Effects of immunotherapies and prognostic predictors in neuromyelitis optica spectrum disorder: a prospective cohort study</title><title>Journal of neurology</title><addtitle>J Neurol</addtitle><addtitle>J Neurol</addtitle><description>Background 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. Methods 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. Results 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  &lt; 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  &lt; 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. Conclusions 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.</description><subject>Adrenal Cortex Hormones - administration &amp; dosage</subject><subject>Adrenal Cortex Hormones - pharmacology</subject><subject>Aquaporin 4</subject><subject>Autoimmune diseases</subject><subject>Azathioprine</subject><subject>Azathioprine - administration &amp; dosage</subject><subject>Azathioprine - pharmacology</subject><subject>Cohort analysis</subject><subject>Corticosteroids</subject><subject>Demyelinating diseases</subject><subject>Demyelination</subject><subject>Disabled Persons</subject><subject>Follow-Up Studies</subject><subject>Health risk assessment</subject><subject>Humans</subject><subject>Immunosuppressive Agents - administration &amp; dosage</subject><subject>Immunosuppressive Agents - pharmacology</subject><subject>Immunotherapy</subject><subject>Long-term effects</subject><subject>Medicine</subject><subject>Medicine &amp; 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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. Methods 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. Results 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  &lt; 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  &lt; 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. Conclusions 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.</abstract><cop>Berlin/Heidelberg</cop><pub>Springer Berlin Heidelberg</pub><pmid>31776721</pmid><doi>10.1007/s00415-019-09649-7</doi><tpages>12</tpages><orcidid>https://orcid.org/0000-0001-6959-6947</orcidid></addata></record>
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subjects Adrenal Cortex Hormones - administration & dosage
Adrenal Cortex Hormones - pharmacology
Aquaporin 4
Autoimmune diseases
Azathioprine
Azathioprine - administration & dosage
Azathioprine - pharmacology
Cohort analysis
Corticosteroids
Demyelinating diseases
Demyelination
Disabled Persons
Follow-Up Studies
Health risk assessment
Humans
Immunosuppressive Agents - administration & dosage
Immunosuppressive Agents - pharmacology
Immunotherapy
Long-term effects
Medicine
Medicine & Public Health
Mycophenolate mofetil
Mycophenolic acid
Mycophenolic Acid - administration & dosage
Mycophenolic Acid - pharmacology
Neuritis
Neurology
Neuromyelitis
Neuromyelitis Optica - drug therapy
Neuromyelitis Optica - physiopathology
Neuroradiology
Neurosciences
Optic neuritis
Original Communication
Outcome Assessment, Health Care
Prognosis
Proportional Hazards Models
Prospective Studies
Risk Factors
Secondary Prevention
Sensorimotor integration
title Effects of immunotherapies and prognostic predictors in neuromyelitis optica spectrum disorder: a prospective cohort study
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