Evaluation of cerebrospinal fluid biomarkers in pediatric patients with spinal muscular atrophy
Spinal muscular atrophy (SMA) is a rare neuromuscular disorder characterised by muscle weakness and muscle atrophy and classified into five known subtypes based on clinical features. The recent development of novel drugs to treat SMA has been encouraging, and nusinersen is the first drug approved to...
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| Veröffentlicht in: | Brain & development (Tokyo. 1979) 2023-01, Vol.45 (1), p.2-7 |
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| container_title | Brain & development (Tokyo. 1979) |
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| creator | Kobayashi, Yoshiyuki Ishikawa, Nobutsune Tateishi, Yuichi Izumo, Hiroki Eto, Syohei Eguchi, Yuta Okada, Satoshi |
| description | Spinal muscular atrophy (SMA) is a rare neuromuscular disorder characterised by muscle weakness and muscle atrophy and classified into five known subtypes based on clinical features. The recent development of novel drugs to treat SMA has been encouraging, and nusinersen is the first drug approved to treat SMA.
To explore cerebrospinal fluid (CSF) biomarkers of SMA and investigate their relationship with symptoms and the treatment response in pediatric patients.
We analyzed the CSF levels of chitotriosidase 1 (CHIT1) and inflammatory cytokines (tumor necrosis factor [TNF]-α and interferon [INF]-γ) using enzyme-linked immunosorbent assays in pediatric SMA patients treated at Hiroshima University Hospital over 2 years.
This study analyzed pediatric SMA patients. While the CSF inflammatory cytokines (TNF-α and INF-γ) in these SMA children were unchanged, the CHIT1 levels decreased significantly from year 1 to 2 of treatment. We also found a trend toward an inverse correlation between the motor function score (HINE-2 scores) and CHIT1 level from year 1 to 2 of treatment.
CHIT1 may be a CSF biomarker of the treatment response in pediatric SMA. |
| doi_str_mv | 10.1016/j.braindev.2022.09.008 |
| format | Article |
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To explore cerebrospinal fluid (CSF) biomarkers of SMA and investigate their relationship with symptoms and the treatment response in pediatric patients.
We analyzed the CSF levels of chitotriosidase 1 (CHIT1) and inflammatory cytokines (tumor necrosis factor [TNF]-α and interferon [INF]-γ) using enzyme-linked immunosorbent assays in pediatric SMA patients treated at Hiroshima University Hospital over 2 years.
This study analyzed pediatric SMA patients. While the CSF inflammatory cytokines (TNF-α and INF-γ) in these SMA children were unchanged, the CHIT1 levels decreased significantly from year 1 to 2 of treatment. We also found a trend toward an inverse correlation between the motor function score (HINE-2 scores) and CHIT1 level from year 1 to 2 of treatment.
CHIT1 may be a CSF biomarker of the treatment response in pediatric SMA.</description><identifier>ISSN: 0387-7604</identifier><identifier>EISSN: 1872-7131</identifier><identifier>DOI: 10.1016/j.braindev.2022.09.008</identifier><identifier>PMID: 36210235</identifier><language>eng</language><publisher>Netherlands: Elsevier B.V</publisher><subject>Biomarker ; Biomarkers - cerebrospinal fluid ; Cerebrospinal fluid ; Child ; CHIT1 ; Cytokines ; Humans ; Interferon-gamma ; Muscular Atrophy ; Muscular Atrophy, Spinal - drug therapy ; Nusinersen ; Spinal Muscular Atrophies of Childhood - diagnosis ; Spinal Muscular Atrophies of Childhood - drug therapy ; Spinal muscular atrophy</subject><ispartof>Brain & development (Tokyo. 1979), 2023-01, Vol.45 (1), p.2-7</ispartof><rights>2022 The Japanese Society of Child Neurology</rights><rights>Copyright © 2022 The Japanese Society of Child Neurology. Published by Elsevier B.V. All rights reserved.</rights><lds50>peer_reviewed</lds50><woscitedreferencessubscribed>false</woscitedreferencessubscribed><citedby>FETCH-LOGICAL-c392t-a3ec5aa38f320e6bf5cd96d5865fce8bc3612795a3289dab6613e1a4e8c7e8883</citedby><cites>FETCH-LOGICAL-c392t-a3ec5aa38f320e6bf5cd96d5865fce8bc3612795a3289dab6613e1a4e8c7e8883</cites></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><linktohtml>$$Uhttps://dx.doi.org/10.1016/j.braindev.2022.09.008$$EHTML$$P50$$Gelsevier$$H</linktohtml><link.rule.ids>314,780,784,3550,27924,27925,45995</link.rule.ids><backlink>$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/36210235$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink></links><search><creatorcontrib>Kobayashi, Yoshiyuki</creatorcontrib><creatorcontrib>Ishikawa, Nobutsune</creatorcontrib><creatorcontrib>Tateishi, Yuichi</creatorcontrib><creatorcontrib>Izumo, Hiroki</creatorcontrib><creatorcontrib>Eto, Syohei</creatorcontrib><creatorcontrib>Eguchi, Yuta</creatorcontrib><creatorcontrib>Okada, Satoshi</creatorcontrib><title>Evaluation of cerebrospinal fluid biomarkers in pediatric patients with spinal muscular atrophy</title><title>Brain & development (Tokyo. 1979)</title><addtitle>Brain Dev</addtitle><description>Spinal muscular atrophy (SMA) is a rare neuromuscular disorder characterised by muscle weakness and muscle atrophy and classified into five known subtypes based on clinical features. The recent development of novel drugs to treat SMA has been encouraging, and nusinersen is the first drug approved to treat SMA.
To explore cerebrospinal fluid (CSF) biomarkers of SMA and investigate their relationship with symptoms and the treatment response in pediatric patients.
We analyzed the CSF levels of chitotriosidase 1 (CHIT1) and inflammatory cytokines (tumor necrosis factor [TNF]-α and interferon [INF]-γ) using enzyme-linked immunosorbent assays in pediatric SMA patients treated at Hiroshima University Hospital over 2 years.
This study analyzed pediatric SMA patients. While the CSF inflammatory cytokines (TNF-α and INF-γ) in these SMA children were unchanged, the CHIT1 levels decreased significantly from year 1 to 2 of treatment. We also found a trend toward an inverse correlation between the motor function score (HINE-2 scores) and CHIT1 level from year 1 to 2 of treatment.
CHIT1 may be a CSF biomarker of the treatment response in pediatric SMA.</description><subject>Biomarker</subject><subject>Biomarkers - cerebrospinal fluid</subject><subject>Cerebrospinal fluid</subject><subject>Child</subject><subject>CHIT1</subject><subject>Cytokines</subject><subject>Humans</subject><subject>Interferon-gamma</subject><subject>Muscular Atrophy</subject><subject>Muscular Atrophy, Spinal - drug therapy</subject><subject>Nusinersen</subject><subject>Spinal Muscular Atrophies of Childhood - diagnosis</subject><subject>Spinal Muscular Atrophies of Childhood - drug therapy</subject><subject>Spinal muscular atrophy</subject><issn>0387-7604</issn><issn>1872-7131</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>2023</creationdate><recordtype>article</recordtype><sourceid>EIF</sourceid><recordid>eNqFkLtOxDAQRS0EguXxC8glTcLY3jhOB0K8pJVooLYcZyK8JHGwk0X8PV7tQks1zblzdQ8hlwxyBkxer_M6GDc0uMk5cJ5DlQOoA7JgquRZyQQ7JAsQqsxKCcsTchrjGgAYZ3BMToRMl4tiQfT9xnSzmZwfqG-pxYB18HF0g-lo282uobXzvQkfGCJ1Ax2xcWYKztIxpXCYIv1y0zvdR_o52rkzgSbGj-_f5-SoNV3Ei_09I28P9693T9nq5fH57naVWVHxKTMCbWGMUK3ggLJuC9tUsimULFqLqrZCMl5WhRFcVY2ppWQCmVmisiUqpcQZudr9HYP_nDFOunfRYteZAf0cNS-5WCoFEhIqd6hNQ2PAVo_BpYXfmoHeytVr_StXb-VqqHSSm4KX-4657rH5i_3aTMDNDsC0dOMw6GiTIpuUBbSTbrz7r-MHSxeQsQ</recordid><startdate>202301</startdate><enddate>202301</enddate><creator>Kobayashi, Yoshiyuki</creator><creator>Ishikawa, Nobutsune</creator><creator>Tateishi, Yuichi</creator><creator>Izumo, Hiroki</creator><creator>Eto, Syohei</creator><creator>Eguchi, Yuta</creator><creator>Okada, Satoshi</creator><general>Elsevier B.V</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>202301</creationdate><title>Evaluation of cerebrospinal fluid biomarkers in pediatric patients with spinal muscular atrophy</title><author>Kobayashi, Yoshiyuki ; Ishikawa, Nobutsune ; Tateishi, Yuichi ; Izumo, Hiroki ; Eto, Syohei ; Eguchi, Yuta ; Okada, Satoshi</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-LOGICAL-c392t-a3ec5aa38f320e6bf5cd96d5865fce8bc3612795a3289dab6613e1a4e8c7e8883</frbrgroupid><rsrctype>articles</rsrctype><prefilter>articles</prefilter><language>eng</language><creationdate>2023</creationdate><topic>Biomarker</topic><topic>Biomarkers - cerebrospinal fluid</topic><topic>Cerebrospinal fluid</topic><topic>Child</topic><topic>CHIT1</topic><topic>Cytokines</topic><topic>Humans</topic><topic>Interferon-gamma</topic><topic>Muscular Atrophy</topic><topic>Muscular Atrophy, Spinal - drug therapy</topic><topic>Nusinersen</topic><topic>Spinal Muscular Atrophies of Childhood - diagnosis</topic><topic>Spinal Muscular Atrophies of Childhood - drug therapy</topic><topic>Spinal muscular atrophy</topic><toplevel>peer_reviewed</toplevel><toplevel>online_resources</toplevel><creatorcontrib>Kobayashi, Yoshiyuki</creatorcontrib><creatorcontrib>Ishikawa, Nobutsune</creatorcontrib><creatorcontrib>Tateishi, Yuichi</creatorcontrib><creatorcontrib>Izumo, Hiroki</creatorcontrib><creatorcontrib>Eto, Syohei</creatorcontrib><creatorcontrib>Eguchi, Yuta</creatorcontrib><creatorcontrib>Okada, Satoshi</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>Brain & development (Tokyo. 1979)</jtitle></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext</fulltext></delivery><addata><au>Kobayashi, Yoshiyuki</au><au>Ishikawa, Nobutsune</au><au>Tateishi, Yuichi</au><au>Izumo, Hiroki</au><au>Eto, Syohei</au><au>Eguchi, Yuta</au><au>Okada, Satoshi</au><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>Evaluation of cerebrospinal fluid biomarkers in pediatric patients with spinal muscular atrophy</atitle><jtitle>Brain & development (Tokyo. 1979)</jtitle><addtitle>Brain Dev</addtitle><date>2023-01</date><risdate>2023</risdate><volume>45</volume><issue>1</issue><spage>2</spage><epage>7</epage><pages>2-7</pages><issn>0387-7604</issn><eissn>1872-7131</eissn><abstract>Spinal muscular atrophy (SMA) is a rare neuromuscular disorder characterised by muscle weakness and muscle atrophy and classified into five known subtypes based on clinical features. The recent development of novel drugs to treat SMA has been encouraging, and nusinersen is the first drug approved to treat SMA.
To explore cerebrospinal fluid (CSF) biomarkers of SMA and investigate their relationship with symptoms and the treatment response in pediatric patients.
We analyzed the CSF levels of chitotriosidase 1 (CHIT1) and inflammatory cytokines (tumor necrosis factor [TNF]-α and interferon [INF]-γ) using enzyme-linked immunosorbent assays in pediatric SMA patients treated at Hiroshima University Hospital over 2 years.
This study analyzed pediatric SMA patients. While the CSF inflammatory cytokines (TNF-α and INF-γ) in these SMA children were unchanged, the CHIT1 levels decreased significantly from year 1 to 2 of treatment. We also found a trend toward an inverse correlation between the motor function score (HINE-2 scores) and CHIT1 level from year 1 to 2 of treatment.
CHIT1 may be a CSF biomarker of the treatment response in pediatric SMA.</abstract><cop>Netherlands</cop><pub>Elsevier B.V</pub><pmid>36210235</pmid><doi>10.1016/j.braindev.2022.09.008</doi><tpages>6</tpages></addata></record> |
| fulltext | fulltext |
| identifier | ISSN: 0387-7604 |
| ispartof | Brain & development (Tokyo. 1979), 2023-01, Vol.45 (1), p.2-7 |
| issn | 0387-7604 1872-7131 |
| language | eng |
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| source | MEDLINE; ScienceDirect Journals (5 years ago - present) |
| subjects | Biomarker Biomarkers - cerebrospinal fluid Cerebrospinal fluid Child CHIT1 Cytokines Humans Interferon-gamma Muscular Atrophy Muscular Atrophy, Spinal - drug therapy Nusinersen Spinal Muscular Atrophies of Childhood - diagnosis Spinal Muscular Atrophies of Childhood - drug therapy Spinal muscular atrophy |
| title | Evaluation of cerebrospinal fluid biomarkers in pediatric patients with spinal muscular atrophy |
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