Mycophenolate Mofetil in the Treatment of Neuromyelitis Optica Spectrum Disorder
IMPORTANCE: Neuromyelitis optica spectrum disorder (NMOSD) is an autoimmune inflammatory disorder of the central nervous system. Recently, various immunosuppressant medications were introduced as therapeutic options for preventing relapse of NMOSD. However, our understanding of the effectiveness of...
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| Veröffentlicht in: | JAMA neurology 2014-11, Vol.71 (11), p.1372-1378 |
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| creator | Huh, So-Young Kim, Su-Hyun Hyun, Jae-Won Joung, Ae-Ran Park, Min Su Kim, Byung-Jo Kim, Ho Jin |
| description | IMPORTANCE: Neuromyelitis optica spectrum disorder (NMOSD) is an autoimmune inflammatory disorder of the central nervous system. Recently, various immunosuppressant medications were introduced as therapeutic options for preventing relapse of NMOSD. However, our understanding of the effectiveness of mycophenolate mofetil (MMF) in treating patients with NMOSD is based on only a small number of studies. OBJECTIVE: To evaluate the efficacy and safety of MMF treatment in patients with NMOSD. DESIGN, SETTING, AND PARTICIPANTS: A 3-center retrospective review of our experiences, examining results from 59 patients with NMOSD (24 with neuromyelitis optica and 35 with a limited form of the disease) who were treated with MMF (1000-2000 mg/d). MAIN OUTCOMES AND MEASURES: Patients’ annualized relapse rate, disability as measured by the Expanded Disability Status Scale, and experience of adverse effects due to MMF were assessed. RESULTS: Of the 59 patients, 1 with NMOSD who had to discontinue MMF use in the first month due to a rash was excluded. The remaining 58 patients were included in the drug-efficacy analysis. The median post-MMF annualized relapse rate was significantly lower than the pre-MMF annualized relapse rate (0.0 vs 1.5; P |
| doi_str_mv | 10.1001/jamaneurol.2014.2057 |
| format | Article |
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Recently, various immunosuppressant medications were introduced as therapeutic options for preventing relapse of NMOSD. However, our understanding of the effectiveness of mycophenolate mofetil (MMF) in treating patients with NMOSD is based on only a small number of studies. OBJECTIVE: To evaluate the efficacy and safety of MMF treatment in patients with NMOSD. DESIGN, SETTING, AND PARTICIPANTS: A 3-center retrospective review of our experiences, examining results from 59 patients with NMOSD (24 with neuromyelitis optica and 35 with a limited form of the disease) who were treated with MMF (1000-2000 mg/d). MAIN OUTCOMES AND MEASURES: Patients’ annualized relapse rate, disability as measured by the Expanded Disability Status Scale, and experience of adverse effects due to MMF were assessed. RESULTS: Of the 59 patients, 1 with NMOSD who had to discontinue MMF use in the first month due to a rash was excluded. The remaining 58 patients were included in the drug-efficacy analysis. The median post-MMF annualized relapse rate was significantly lower than the pre-MMF annualized relapse rate (0.0 vs 1.5; P < .001). The Expanded Disability Status Scale scores also significantly decreased after MMF treatment (3.0 vs 2.5; P = .005). Thirty-five patients (60%) were relapse free, and Expanded Disability Status Scale scores were stabilized or improved in 53 patients (91%). Fourteen patients discontinued MMF treatment owing to ongoing relapse (10 patients), rash (1 patient), pregnancy (1 patient), and financial problems (2 patients), but MMF was generally well tolerated. CONCLUSIONS AND RELEVANCE: In this observational study, MMF treatment induced reduction of relapse frequency, stabilized or improved disability, and was well tolerated in patients with NMOSD.</description><identifier>ISSN: 2168-6149</identifier><identifier>EISSN: 2168-6157</identifier><identifier>DOI: 10.1001/jamaneurol.2014.2057</identifier><identifier>PMID: 25199960</identifier><language>eng</language><publisher>United States: American Medical Association</publisher><subject>Adolescent ; Adult ; Disability Evaluation ; Female ; Follow-Up Studies ; Humans ; Male ; Middle Aged ; Mycophenolic Acid - administration & dosage ; Mycophenolic Acid - adverse effects ; Mycophenolic Acid - analogs & derivatives ; Mycophenolic Acid - therapeutic use ; Neuromyelitis Optica - drug therapy ; Recurrence ; Retrospective Studies ; Treatment Outcome ; Young Adult</subject><ispartof>JAMA neurology, 2014-11, Vol.71 (11), p.1372-1378</ispartof><lds50>peer_reviewed</lds50><woscitedreferencessubscribed>false</woscitedreferencessubscribed><citedby>FETCH-LOGICAL-a392t-7cc169ca2632ca0a638df4759a3fcc13e2323e61b76802fd8b973be502249c203</citedby></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><linktopdf>$$Uhttps://jamanetwork.com/journals/jamaneurology/articlepdf/10.1001/jamaneurol.2014.2057$$EPDF$$P50$$Gama$$H</linktopdf><linktohtml>$$Uhttps://jamanetwork.com/journals/jamaneurology/fullarticle/10.1001/jamaneurol.2014.2057$$EHTML$$P50$$Gama$$H</linktohtml><link.rule.ids>64,314,776,780,3327,27901,27902,76231,76234</link.rule.ids><backlink>$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/25199960$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink></links><search><creatorcontrib>Huh, So-Young</creatorcontrib><creatorcontrib>Kim, Su-Hyun</creatorcontrib><creatorcontrib>Hyun, Jae-Won</creatorcontrib><creatorcontrib>Joung, Ae-Ran</creatorcontrib><creatorcontrib>Park, Min Su</creatorcontrib><creatorcontrib>Kim, Byung-Jo</creatorcontrib><creatorcontrib>Kim, Ho Jin</creatorcontrib><title>Mycophenolate Mofetil in the Treatment of Neuromyelitis Optica Spectrum Disorder</title><title>JAMA neurology</title><addtitle>JAMA Neurol</addtitle><description>IMPORTANCE: Neuromyelitis optica spectrum disorder (NMOSD) is an autoimmune inflammatory disorder of the central nervous system. Recently, various immunosuppressant medications were introduced as therapeutic options for preventing relapse of NMOSD. However, our understanding of the effectiveness of mycophenolate mofetil (MMF) in treating patients with NMOSD is based on only a small number of studies. OBJECTIVE: To evaluate the efficacy and safety of MMF treatment in patients with NMOSD. DESIGN, SETTING, AND PARTICIPANTS: A 3-center retrospective review of our experiences, examining results from 59 patients with NMOSD (24 with neuromyelitis optica and 35 with a limited form of the disease) who were treated with MMF (1000-2000 mg/d). MAIN OUTCOMES AND MEASURES: Patients’ annualized relapse rate, disability as measured by the Expanded Disability Status Scale, and experience of adverse effects due to MMF were assessed. RESULTS: Of the 59 patients, 1 with NMOSD who had to discontinue MMF use in the first month due to a rash was excluded. The remaining 58 patients were included in the drug-efficacy analysis. The median post-MMF annualized relapse rate was significantly lower than the pre-MMF annualized relapse rate (0.0 vs 1.5; P < .001). The Expanded Disability Status Scale scores also significantly decreased after MMF treatment (3.0 vs 2.5; P = .005). Thirty-five patients (60%) were relapse free, and Expanded Disability Status Scale scores were stabilized or improved in 53 patients (91%). Fourteen patients discontinued MMF treatment owing to ongoing relapse (10 patients), rash (1 patient), pregnancy (1 patient), and financial problems (2 patients), but MMF was generally well tolerated. CONCLUSIONS AND RELEVANCE: In this observational study, MMF treatment induced reduction of relapse frequency, stabilized or improved disability, and was well tolerated in patients with NMOSD.</description><subject>Adolescent</subject><subject>Adult</subject><subject>Disability Evaluation</subject><subject>Female</subject><subject>Follow-Up Studies</subject><subject>Humans</subject><subject>Male</subject><subject>Middle Aged</subject><subject>Mycophenolic Acid - administration & dosage</subject><subject>Mycophenolic Acid - adverse effects</subject><subject>Mycophenolic Acid - analogs & derivatives</subject><subject>Mycophenolic Acid - therapeutic use</subject><subject>Neuromyelitis Optica - drug therapy</subject><subject>Recurrence</subject><subject>Retrospective Studies</subject><subject>Treatment Outcome</subject><subject>Young Adult</subject><issn>2168-6149</issn><issn>2168-6157</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>2014</creationdate><recordtype>article</recordtype><sourceid>EIF</sourceid><recordid>eNpFkMtOwzAQRS0EolXpDyCEvGST4kfixEtUnlJLkSjryHEmaqqkDraz6N_jUGhnMTPS3HtHOgjdUDKjhND7rWrVDnprmhkjNA4tSc_QmFGRRYIm6flxj-UITZ3bklAZITGPL9GIJVRKKcgYfSz32nQb2JlGecBLU4GvG1zvsN8AXltQvoWdx6bC78O_dg9N7WuHV52vtcKfHWhv-xY_1s7YEuwVuqhU42D6Nyfo6_lpPX-NFquXt_nDIlJcMh-lWlMhtWKCM62IEjwrqzhNpOJVOHFgnHEQtEhFRlhVZoVMeQEJYSyWmhE-QXeH3M6a7x6cz9vaaWiawMX0LqciJGRZzHiQxgeptsY5C1Xe2bpVdp9Tkg848xPOfMCZDziD7fbvQ1-0UB5N__CC4PogCO5TpPzlzH8ARTN73g</recordid><startdate>20141101</startdate><enddate>20141101</enddate><creator>Huh, So-Young</creator><creator>Kim, Su-Hyun</creator><creator>Hyun, Jae-Won</creator><creator>Joung, Ae-Ran</creator><creator>Park, Min Su</creator><creator>Kim, Byung-Jo</creator><creator>Kim, Ho Jin</creator><general>American Medical Association</general><scope>CGR</scope><scope>CUY</scope><scope>CVF</scope><scope>ECM</scope><scope>EIF</scope><scope>NPM</scope><scope>AAYXX</scope><scope>CITATION</scope><scope>7X8</scope></search><sort><creationdate>20141101</creationdate><title>Mycophenolate Mofetil in the Treatment of Neuromyelitis Optica Spectrum Disorder</title><author>Huh, So-Young ; Kim, Su-Hyun ; Hyun, Jae-Won ; Joung, Ae-Ran ; Park, Min Su ; Kim, Byung-Jo ; Kim, Ho Jin</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-LOGICAL-a392t-7cc169ca2632ca0a638df4759a3fcc13e2323e61b76802fd8b973be502249c203</frbrgroupid><rsrctype>articles</rsrctype><prefilter>articles</prefilter><language>eng</language><creationdate>2014</creationdate><topic>Adolescent</topic><topic>Adult</topic><topic>Disability Evaluation</topic><topic>Female</topic><topic>Follow-Up Studies</topic><topic>Humans</topic><topic>Male</topic><topic>Middle Aged</topic><topic>Mycophenolic Acid - administration & dosage</topic><topic>Mycophenolic Acid - adverse effects</topic><topic>Mycophenolic Acid - analogs & derivatives</topic><topic>Mycophenolic Acid - therapeutic use</topic><topic>Neuromyelitis Optica - drug therapy</topic><topic>Recurrence</topic><topic>Retrospective Studies</topic><topic>Treatment Outcome</topic><topic>Young Adult</topic><toplevel>peer_reviewed</toplevel><toplevel>online_resources</toplevel><creatorcontrib>Huh, So-Young</creatorcontrib><creatorcontrib>Kim, Su-Hyun</creatorcontrib><creatorcontrib>Hyun, Jae-Won</creatorcontrib><creatorcontrib>Joung, Ae-Ran</creatorcontrib><creatorcontrib>Park, Min Su</creatorcontrib><creatorcontrib>Kim, Byung-Jo</creatorcontrib><creatorcontrib>Kim, Ho Jin</creatorcontrib><collection>Medline</collection><collection>MEDLINE</collection><collection>MEDLINE (Ovid)</collection><collection>MEDLINE</collection><collection>MEDLINE</collection><collection>PubMed</collection><collection>CrossRef</collection><collection>MEDLINE - Academic</collection><jtitle>JAMA neurology</jtitle></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext</fulltext></delivery><addata><au>Huh, So-Young</au><au>Kim, Su-Hyun</au><au>Hyun, Jae-Won</au><au>Joung, Ae-Ran</au><au>Park, Min Su</au><au>Kim, Byung-Jo</au><au>Kim, Ho Jin</au><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>Mycophenolate Mofetil in the Treatment of Neuromyelitis Optica Spectrum Disorder</atitle><jtitle>JAMA neurology</jtitle><addtitle>JAMA Neurol</addtitle><date>2014-11-01</date><risdate>2014</risdate><volume>71</volume><issue>11</issue><spage>1372</spage><epage>1378</epage><pages>1372-1378</pages><issn>2168-6149</issn><eissn>2168-6157</eissn><abstract>IMPORTANCE: Neuromyelitis optica spectrum disorder (NMOSD) is an autoimmune inflammatory disorder of the central nervous system. Recently, various immunosuppressant medications were introduced as therapeutic options for preventing relapse of NMOSD. However, our understanding of the effectiveness of mycophenolate mofetil (MMF) in treating patients with NMOSD is based on only a small number of studies. OBJECTIVE: To evaluate the efficacy and safety of MMF treatment in patients with NMOSD. DESIGN, SETTING, AND PARTICIPANTS: A 3-center retrospective review of our experiences, examining results from 59 patients with NMOSD (24 with neuromyelitis optica and 35 with a limited form of the disease) who were treated with MMF (1000-2000 mg/d). MAIN OUTCOMES AND MEASURES: Patients’ annualized relapse rate, disability as measured by the Expanded Disability Status Scale, and experience of adverse effects due to MMF were assessed. RESULTS: Of the 59 patients, 1 with NMOSD who had to discontinue MMF use in the first month due to a rash was excluded. The remaining 58 patients were included in the drug-efficacy analysis. The median post-MMF annualized relapse rate was significantly lower than the pre-MMF annualized relapse rate (0.0 vs 1.5; P < .001). The Expanded Disability Status Scale scores also significantly decreased after MMF treatment (3.0 vs 2.5; P = .005). Thirty-five patients (60%) were relapse free, and Expanded Disability Status Scale scores were stabilized or improved in 53 patients (91%). Fourteen patients discontinued MMF treatment owing to ongoing relapse (10 patients), rash (1 patient), pregnancy (1 patient), and financial problems (2 patients), but MMF was generally well tolerated. CONCLUSIONS AND RELEVANCE: In this observational study, MMF treatment induced reduction of relapse frequency, stabilized or improved disability, and was well tolerated in patients with NMOSD.</abstract><cop>United States</cop><pub>American Medical Association</pub><pmid>25199960</pmid><doi>10.1001/jamaneurol.2014.2057</doi><tpages>7</tpages></addata></record> |
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| subjects | Adolescent Adult Disability Evaluation Female Follow-Up Studies Humans Male Middle Aged Mycophenolic Acid - administration & dosage Mycophenolic Acid - adverse effects Mycophenolic Acid - analogs & derivatives Mycophenolic Acid - therapeutic use Neuromyelitis Optica - drug therapy Recurrence Retrospective Studies Treatment Outcome Young Adult |
| title | Mycophenolate Mofetil in the Treatment of Neuromyelitis Optica Spectrum Disorder |
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