An observational cohort study on pulmonary function in adult patients with 5q-spinal muscular atrophy under nusinersen therapy
Background Few studies assessed the effect of nusinersen on respiratory function in adult patients with spinal muscular atrophy (SMA). The aim of this single-center study was to analyze pulmonary function and its association with muscle function and quality of life (QoL) in adult patients with 5q-SM...
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| Veröffentlicht in: | Journal of neurology 2023-07, Vol.270 (7), p.3616-3622 |
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| creator | Bjelica, Bogdan Wohnrade, Camilla Osmanovic, Alma Schreiber-Katz, Olivia Petri, Susanne |
| description | Background
Few studies assessed the effect of nusinersen on respiratory function in adult patients with spinal muscular atrophy (SMA). The aim of this single-center study was to analyze pulmonary function and its association with muscle function and quality of life (QoL) in adult patients with 5q-SMA under nusinersen.
Methods
We recorded forced vital capacity (FVC), forced expiratory volume in the first second (FEV1) and peak expiratory flow (PEF) during nusinersen treatment in 38 adult SMA patients. Revised Upper Limb Module (RULM), Hammersmith Functional Motor Scale Expanded (HFMSE), 36-Item Short Form Health Survey (SF-36) questionnaire and Fatigue Severity Scale (FSS) were recorded and correlations between muscle function, QoL, fatigue and respiratory parameters were analyzed.
Results
No differences were detected between mean FVC, FEV1, PEF at different timepoints versus baseline. Ambulatory patients showed significant improvement in mean PEF at month 30, compared to non-ambulatory patients (+ 0.8 ± 0.5 vs. − 0.0 ± 0.5,
p
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| doi_str_mv | 10.1007/s00415-023-11711-4 |
| format | Article |
| fullrecord | <record><control><sourceid>proquest_pubme</sourceid><recordid>TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_10106012</recordid><sourceformat>XML</sourceformat><sourcesystem>PC</sourcesystem><sourcerecordid>2825641319</sourcerecordid><originalsourceid>FETCH-LOGICAL-c475t-85ea95471cac4f11ca67a888e7d265a9d5fdb939e0925deef2820ae07fded383</originalsourceid><addsrcrecordid>eNp9kTtvFTEQhS0EIpfAH6BAlmhoFsavfVQoikhAikST3vJdz2Y32rU3fgTdht-OlxvCo6Ca4nzneMaHkNcM3jOA5kMEkExVwEXFWMNYJZ-QHZOCV0yq7inZgZBQKaHkCXkR4y0AtEV4Tk5EAzUH1u7I9zNH_T5iuDdp8s7MtPejD4nGlO2BekfXPC9FCAc6ZNdvEJ0cNTbPia7FhC5F-m1KI1V3VVynLWPJsc-zCdSk4NfxQLOzGKjLcXIYIjqaRgxmPbwkzwYzR3z1ME_J9cWn6_PP1dXXyy_nZ1dVLxuVqlah6ZRsWG96ObAy6sa0bYuN5bUynVWD3XeiQ-i4sogDbzkYhGawaEUrTsnHY-ya9wvavuwczKzXMC3lMO3NpP9W3DTqG3-vGTCogfGS8O4hIfi7jDHpZYo9zrNx6HPUvAXWNXUHXUHf_oPe-hzKt2wUV7Vkgm0UP1J98DEGHB63YaC3evWxXl3q1T_r1bKY3vx5x6PlV58FEEcgFsndYPj99n9ifwDyQbRu</addsrcrecordid><sourcetype>Open Access Repository</sourcetype><iscdi>true</iscdi><recordtype>article</recordtype><pqid>2825641319</pqid></control><display><type>article</type><title>An observational cohort study on pulmonary function in adult patients with 5q-spinal muscular atrophy under nusinersen therapy</title><source>MEDLINE</source><source>SpringerLink Journals - AutoHoldings</source><creator>Bjelica, Bogdan ; Wohnrade, Camilla ; Osmanovic, Alma ; Schreiber-Katz, Olivia ; Petri, Susanne</creator><creatorcontrib>Bjelica, Bogdan ; Wohnrade, Camilla ; Osmanovic, Alma ; Schreiber-Katz, Olivia ; Petri, Susanne</creatorcontrib><description>Background
Few studies assessed the effect of nusinersen on respiratory function in adult patients with spinal muscular atrophy (SMA). The aim of this single-center study was to analyze pulmonary function and its association with muscle function and quality of life (QoL) in adult patients with 5q-SMA under nusinersen.
Methods
We recorded forced vital capacity (FVC), forced expiratory volume in the first second (FEV1) and peak expiratory flow (PEF) during nusinersen treatment in 38 adult SMA patients. Revised Upper Limb Module (RULM), Hammersmith Functional Motor Scale Expanded (HFMSE), 36-Item Short Form Health Survey (SF-36) questionnaire and Fatigue Severity Scale (FSS) were recorded and correlations between muscle function, QoL, fatigue and respiratory parameters were analyzed.
Results
No differences were detected between mean FVC, FEV1, PEF at different timepoints versus baseline. Ambulatory patients showed significant improvement in mean PEF at month 30, compared to non-ambulatory patients (+ 0.8 ± 0.5 vs. − 0.0 ± 0.5,
p
< 0.05). Patients with fatigue at baseline showed significant improvement in mean PEF at month 10, compared to patients without fatigue at baseline (+ 0.6 ± 0.9 vs. − 0.4 ± 0.5,
p
< 0.05). Physical domains of SF-36 positively correlated with the change in FVC and FEV1. FSS negatively correlated with the change in mean PEF.
Conclusion
Mean pulmonary function remained stable during nusinersen treatment over a period of up to 30 months. Improvement in pulmonary function was associated with improvement in motor function, fatigue and QoL, early after nusinersen initiation.</description><identifier>ISSN: 0340-5354</identifier><identifier>EISSN: 1432-1459</identifier><identifier>DOI: 10.1007/s00415-023-11711-4</identifier><identifier>PMID: 37062018</identifier><language>eng</language><publisher>Berlin/Heidelberg: Springer Berlin Heidelberg</publisher><subject>Adult ; Cohort analysis ; Cohort Studies ; Fatigue ; Humans ; Medicine ; Medicine & Public Health ; Muscle function ; Muscular Atrophy, Spinal - drug therapy ; Neurology ; Neuromuscular diseases ; Neuroradiology ; Neurosciences ; Observational studies ; Original Communication ; Quality of Life ; Respiratory function ; Spinal Muscular Atrophies of Childhood - drug therapy ; Spinal muscular atrophy</subject><ispartof>Journal of neurology, 2023-07, Vol.270 (7), p.3616-3622</ispartof><rights>The Author(s) 2023</rights><rights>2023. The Author(s).</rights><rights>The Author(s) 2023. This work is published under http://creativecommons.org/licenses/by/4.0/ (the “License”). Notwithstanding the ProQuest Terms and Conditions, you may use this content in accordance with the terms of the License.</rights><lds50>peer_reviewed</lds50><oa>free_for_read</oa><woscitedreferencessubscribed>false</woscitedreferencessubscribed><citedby>FETCH-LOGICAL-c475t-85ea95471cac4f11ca67a888e7d265a9d5fdb939e0925deef2820ae07fded383</citedby><cites>FETCH-LOGICAL-c475t-85ea95471cac4f11ca67a888e7d265a9d5fdb939e0925deef2820ae07fded383</cites><orcidid>0000-0002-1783-703X</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/s00415-023-11711-4$$EPDF$$P50$$Gspringer$$Hfree_for_read</linktopdf><linktohtml>$$Uhttps://link.springer.com/10.1007/s00415-023-11711-4$$EHTML$$P50$$Gspringer$$Hfree_for_read</linktohtml><link.rule.ids>230,314,780,784,885,27924,27925,41488,42557,51319</link.rule.ids><backlink>$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/37062018$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink></links><search><creatorcontrib>Bjelica, Bogdan</creatorcontrib><creatorcontrib>Wohnrade, Camilla</creatorcontrib><creatorcontrib>Osmanovic, Alma</creatorcontrib><creatorcontrib>Schreiber-Katz, Olivia</creatorcontrib><creatorcontrib>Petri, Susanne</creatorcontrib><title>An observational cohort study on pulmonary function in adult patients with 5q-spinal muscular atrophy under nusinersen therapy</title><title>Journal of neurology</title><addtitle>J Neurol</addtitle><addtitle>J Neurol</addtitle><description>Background
Few studies assessed the effect of nusinersen on respiratory function in adult patients with spinal muscular atrophy (SMA). The aim of this single-center study was to analyze pulmonary function and its association with muscle function and quality of life (QoL) in adult patients with 5q-SMA under nusinersen.
Methods
We recorded forced vital capacity (FVC), forced expiratory volume in the first second (FEV1) and peak expiratory flow (PEF) during nusinersen treatment in 38 adult SMA patients. Revised Upper Limb Module (RULM), Hammersmith Functional Motor Scale Expanded (HFMSE), 36-Item Short Form Health Survey (SF-36) questionnaire and Fatigue Severity Scale (FSS) were recorded and correlations between muscle function, QoL, fatigue and respiratory parameters were analyzed.
Results
No differences were detected between mean FVC, FEV1, PEF at different timepoints versus baseline. Ambulatory patients showed significant improvement in mean PEF at month 30, compared to non-ambulatory patients (+ 0.8 ± 0.5 vs. − 0.0 ± 0.5,
p
< 0.05). Patients with fatigue at baseline showed significant improvement in mean PEF at month 10, compared to patients without fatigue at baseline (+ 0.6 ± 0.9 vs. − 0.4 ± 0.5,
p
< 0.05). Physical domains of SF-36 positively correlated with the change in FVC and FEV1. FSS negatively correlated with the change in mean PEF.
Conclusion
Mean pulmonary function remained stable during nusinersen treatment over a period of up to 30 months. Improvement in pulmonary function was associated with improvement in motor function, fatigue and QoL, early after nusinersen initiation.</description><subject>Adult</subject><subject>Cohort analysis</subject><subject>Cohort Studies</subject><subject>Fatigue</subject><subject>Humans</subject><subject>Medicine</subject><subject>Medicine & Public Health</subject><subject>Muscle function</subject><subject>Muscular Atrophy, Spinal - drug therapy</subject><subject>Neurology</subject><subject>Neuromuscular diseases</subject><subject>Neuroradiology</subject><subject>Neurosciences</subject><subject>Observational studies</subject><subject>Original Communication</subject><subject>Quality of Life</subject><subject>Respiratory function</subject><subject>Spinal Muscular Atrophies of Childhood - drug therapy</subject><subject>Spinal muscular atrophy</subject><issn>0340-5354</issn><issn>1432-1459</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>2023</creationdate><recordtype>article</recordtype><sourceid>C6C</sourceid><sourceid>EIF</sourceid><sourceid>ABUWG</sourceid><sourceid>AFKRA</sourceid><sourceid>BENPR</sourceid><sourceid>CCPQU</sourceid><recordid>eNp9kTtvFTEQhS0EIpfAH6BAlmhoFsavfVQoikhAikST3vJdz2Y32rU3fgTdht-OlxvCo6Ca4nzneMaHkNcM3jOA5kMEkExVwEXFWMNYJZ-QHZOCV0yq7inZgZBQKaHkCXkR4y0AtEV4Tk5EAzUH1u7I9zNH_T5iuDdp8s7MtPejD4nGlO2BekfXPC9FCAc6ZNdvEJ0cNTbPia7FhC5F-m1KI1V3VVynLWPJsc-zCdSk4NfxQLOzGKjLcXIYIjqaRgxmPbwkzwYzR3z1ME_J9cWn6_PP1dXXyy_nZ1dVLxuVqlah6ZRsWG96ObAy6sa0bYuN5bUynVWD3XeiQ-i4sogDbzkYhGawaEUrTsnHY-ya9wvavuwczKzXMC3lMO3NpP9W3DTqG3-vGTCogfGS8O4hIfi7jDHpZYo9zrNx6HPUvAXWNXUHXUHf_oPe-hzKt2wUV7Vkgm0UP1J98DEGHB63YaC3evWxXl3q1T_r1bKY3vx5x6PlV58FEEcgFsndYPj99n9ifwDyQbRu</recordid><startdate>20230701</startdate><enddate>20230701</enddate><creator>Bjelica, Bogdan</creator><creator>Wohnrade, Camilla</creator><creator>Osmanovic, Alma</creator><creator>Schreiber-Katz, Olivia</creator><creator>Petri, Susanne</creator><general>Springer Berlin Heidelberg</general><general>Springer Nature B.V</general><scope>C6C</scope><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>3V.</scope><scope>7TK</scope><scope>7X7</scope><scope>7XB</scope><scope>88E</scope><scope>8AO</scope><scope>8FI</scope><scope>8FJ</scope><scope>8FK</scope><scope>ABUWG</scope><scope>AFKRA</scope><scope>BENPR</scope><scope>CCPQU</scope><scope>FYUFA</scope><scope>GHDGH</scope><scope>K9.</scope><scope>M0S</scope><scope>M1P</scope><scope>PQEST</scope><scope>PQQKQ</scope><scope>PQUKI</scope><scope>7X8</scope><scope>5PM</scope><orcidid>https://orcid.org/0000-0002-1783-703X</orcidid></search><sort><creationdate>20230701</creationdate><title>An observational cohort study on pulmonary function in adult patients with 5q-spinal muscular atrophy under nusinersen therapy</title><author>Bjelica, Bogdan ; Wohnrade, Camilla ; Osmanovic, Alma ; Schreiber-Katz, Olivia ; Petri, Susanne</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-LOGICAL-c475t-85ea95471cac4f11ca67a888e7d265a9d5fdb939e0925deef2820ae07fded383</frbrgroupid><rsrctype>articles</rsrctype><prefilter>articles</prefilter><language>eng</language><creationdate>2023</creationdate><topic>Adult</topic><topic>Cohort analysis</topic><topic>Cohort Studies</topic><topic>Fatigue</topic><topic>Humans</topic><topic>Medicine</topic><topic>Medicine & Public Health</topic><topic>Muscle function</topic><topic>Muscular Atrophy, Spinal - drug therapy</topic><topic>Neurology</topic><topic>Neuromuscular diseases</topic><topic>Neuroradiology</topic><topic>Neurosciences</topic><topic>Observational studies</topic><topic>Original Communication</topic><topic>Quality of Life</topic><topic>Respiratory function</topic><topic>Spinal Muscular Atrophies of Childhood - drug therapy</topic><topic>Spinal muscular atrophy</topic><toplevel>peer_reviewed</toplevel><toplevel>online_resources</toplevel><creatorcontrib>Bjelica, Bogdan</creatorcontrib><creatorcontrib>Wohnrade, Camilla</creatorcontrib><creatorcontrib>Osmanovic, Alma</creatorcontrib><creatorcontrib>Schreiber-Katz, Olivia</creatorcontrib><creatorcontrib>Petri, Susanne</creatorcontrib><collection>Springer Nature OA Free Journals</collection><collection>Medline</collection><collection>MEDLINE</collection><collection>MEDLINE (Ovid)</collection><collection>MEDLINE</collection><collection>MEDLINE</collection><collection>PubMed</collection><collection>CrossRef</collection><collection>ProQuest Central (Corporate)</collection><collection>Neurosciences Abstracts</collection><collection>Health & Medical Collection</collection><collection>ProQuest Central (purchase pre-March 2016)</collection><collection>Medical Database (Alumni Edition)</collection><collection>ProQuest Pharma Collection</collection><collection>Hospital Premium Collection</collection><collection>Hospital Premium Collection (Alumni Edition)</collection><collection>ProQuest Central (Alumni) (purchase pre-March 2016)</collection><collection>ProQuest Central (Alumni Edition)</collection><collection>ProQuest Central UK/Ireland</collection><collection>ProQuest Central</collection><collection>ProQuest One Community College</collection><collection>Health Research Premium Collection</collection><collection>Health Research Premium Collection (Alumni)</collection><collection>ProQuest Health & Medical Complete (Alumni)</collection><collection>Health & Medical Collection (Alumni Edition)</collection><collection>Medical Database</collection><collection>ProQuest One Academic Eastern Edition (DO NOT USE)</collection><collection>ProQuest One Academic</collection><collection>ProQuest One Academic UKI Edition</collection><collection>MEDLINE - Academic</collection><collection>PubMed Central (Full Participant titles)</collection><jtitle>Journal of neurology</jtitle></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext</fulltext></delivery><addata><au>Bjelica, Bogdan</au><au>Wohnrade, Camilla</au><au>Osmanovic, Alma</au><au>Schreiber-Katz, Olivia</au><au>Petri, Susanne</au><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>An observational cohort study on pulmonary function in adult patients with 5q-spinal muscular atrophy under nusinersen therapy</atitle><jtitle>Journal of neurology</jtitle><stitle>J Neurol</stitle><addtitle>J Neurol</addtitle><date>2023-07-01</date><risdate>2023</risdate><volume>270</volume><issue>7</issue><spage>3616</spage><epage>3622</epage><pages>3616-3622</pages><issn>0340-5354</issn><eissn>1432-1459</eissn><abstract>Background
Few studies assessed the effect of nusinersen on respiratory function in adult patients with spinal muscular atrophy (SMA). The aim of this single-center study was to analyze pulmonary function and its association with muscle function and quality of life (QoL) in adult patients with 5q-SMA under nusinersen.
Methods
We recorded forced vital capacity (FVC), forced expiratory volume in the first second (FEV1) and peak expiratory flow (PEF) during nusinersen treatment in 38 adult SMA patients. Revised Upper Limb Module (RULM), Hammersmith Functional Motor Scale Expanded (HFMSE), 36-Item Short Form Health Survey (SF-36) questionnaire and Fatigue Severity Scale (FSS) were recorded and correlations between muscle function, QoL, fatigue and respiratory parameters were analyzed.
Results
No differences were detected between mean FVC, FEV1, PEF at different timepoints versus baseline. Ambulatory patients showed significant improvement in mean PEF at month 30, compared to non-ambulatory patients (+ 0.8 ± 0.5 vs. − 0.0 ± 0.5,
p
< 0.05). Patients with fatigue at baseline showed significant improvement in mean PEF at month 10, compared to patients without fatigue at baseline (+ 0.6 ± 0.9 vs. − 0.4 ± 0.5,
p
< 0.05). Physical domains of SF-36 positively correlated with the change in FVC and FEV1. FSS negatively correlated with the change in mean PEF.
Conclusion
Mean pulmonary function remained stable during nusinersen treatment over a period of up to 30 months. Improvement in pulmonary function was associated with improvement in motor function, fatigue and QoL, early after nusinersen initiation.</abstract><cop>Berlin/Heidelberg</cop><pub>Springer Berlin Heidelberg</pub><pmid>37062018</pmid><doi>10.1007/s00415-023-11711-4</doi><tpages>7</tpages><orcidid>https://orcid.org/0000-0002-1783-703X</orcidid><oa>free_for_read</oa></addata></record> |
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| issn | 0340-5354 1432-1459 |
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| source | MEDLINE; SpringerLink Journals - AutoHoldings |
| subjects | Adult Cohort analysis Cohort Studies Fatigue Humans Medicine Medicine & Public Health Muscle function Muscular Atrophy, Spinal - drug therapy Neurology Neuromuscular diseases Neuroradiology Neurosciences Observational studies Original Communication Quality of Life Respiratory function Spinal Muscular Atrophies of Childhood - drug therapy Spinal muscular atrophy |
| title | An observational cohort study on pulmonary function in adult patients with 5q-spinal muscular atrophy under nusinersen therapy |
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