A longitudinal analysis of respiratory symptoms in people with cystic fibrosis with advanced lung disease on and off ETI
•ETI decreases respiratory symptoms in adults with CF with advanced lung disease over 24-months.•Women on ETI have no significant difference in symptoms than men not on ETI in advanced CF disease.•No ceiling affect noted on CFRSD-CRISS and CFQ-R in adults with CF with advanced lung disease. People w...
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| Veröffentlicht in: | Journal of cystic fibrosis 2024-01, Vol.23 (1), p.161-164 |
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| container_title | Journal of cystic fibrosis |
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| creator | Gill, Eliana R. Bartlett, Lauren E. Milinic, Tijana Burdis, Nora Pilewski, Joseph M. Dunitz, Jordan M. Kapnadak, Siddhartha G. Goss, Christopher H. Ramos, Kathleen J. |
| description | •ETI decreases respiratory symptoms in adults with CF with advanced lung disease over 24-months.•Women on ETI have no significant difference in symptoms than men not on ETI in advanced CF disease.•No ceiling affect noted on CFRSD-CRISS and CFQ-R in adults with CF with advanced lung disease.
People with CF (PwCF), particularly those with advanced lung disease (ALD), experience frequent respiratory symptoms. A major CF breakthrough was the approval of elexacaftor/tezacaftor/ivacaftor (ETI) in 2019, which has been shown to improve symptoms and lung function in the CF population, and decrease pulmonary exacerbations. The purpose of this study was to analyze longitudinal changes in respiratory symptoms over 24 months in ETI-treated and untreated PwCF with ALD Symptoms were measured among CF adults with ppFEV1 < 40% (N = 48, 24 ETI-treated, 24 untreated) using the CFRSD-CRISS and the CFQ-R [respiratory]. Two multilevel growth models assessed the rate of change in symptoms overall and within the ETI-treated and untreated groups. PwCF on ETI had significantly lower symptom severity over 24 months than those not on ETI as measured by the CRISS and CFQ-R. The ETI-treated group maintained an -11.7 and +19.3 point difference(p |
| doi_str_mv | 10.1016/j.jcf.2023.11.008 |
| format | Article |
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People with CF (PwCF), particularly those with advanced lung disease (ALD), experience frequent respiratory symptoms. A major CF breakthrough was the approval of elexacaftor/tezacaftor/ivacaftor (ETI) in 2019, which has been shown to improve symptoms and lung function in the CF population, and decrease pulmonary exacerbations. The purpose of this study was to analyze longitudinal changes in respiratory symptoms over 24 months in ETI-treated and untreated PwCF with ALD Symptoms were measured among CF adults with ppFEV1 < 40% (N = 48, 24 ETI-treated, 24 untreated) using the CFRSD-CRISS and the CFQ-R [respiratory]. Two multilevel growth models assessed the rate of change in symptoms overall and within the ETI-treated and untreated groups. PwCF on ETI had significantly lower symptom severity over 24 months than those not on ETI as measured by the CRISS and CFQ-R. The ETI-treated group maintained an -11.7 and +19.3 point difference(p<0.01) in CRISS and CFQ-R scores over the study compared to the non-ETI group, achieving minimal clinically important differences on average between groups on both instruments. No change in the symptom burden trajectory between groups was observed (p = 0.58). Even with ALD, ETI-treated PwCF have a lower respiratory burden than those not on ETI. This may be confounded by survivorship bias in the non-ETI group. Of note, in this ALD cohort, neither instrument demonstrated ceiling effects. Our results suggest that, while ETI has significantly improved the lived experience, PwCF with ALD are still plagued by respiratory symptoms.</description><identifier>ISSN: 1569-1993</identifier><identifier>ISSN: 1873-5010</identifier><identifier>EISSN: 1873-5010</identifier><identifier>DOI: 10.1016/j.jcf.2023.11.008</identifier><identifier>PMID: 38008684</identifier><language>eng</language><publisher>Netherlands: Elsevier B.V</publisher><subject>Adult ; Aminophenols ; Benzodioxoles - therapeutic use ; Cystic fibrosis ; Cystic Fibrosis - complications ; Cystic Fibrosis - drug therapy ; Cystic Fibrosis Transmembrane Conductance Regulator ; Cystic fibrosis transmembrane conductance regulator modulator ; Elexacaftor/tezacaftor/ivacaftor ; Humans ; Lung ; Mutation ; Pyrazoles ; Pyridines ; Pyrrolidines ; Respiratory symptoms</subject><ispartof>Journal of cystic fibrosis, 2024-01, Vol.23 (1), p.161-164</ispartof><rights>2023 European Cystic Fibrosis Society</rights><rights>Copyright © 2023 European Cystic Fibrosis Society. Published by Elsevier B.V. All rights reserved.</rights><lds50>peer_reviewed</lds50><woscitedreferencessubscribed>false</woscitedreferencessubscribed><cites>FETCH-LOGICAL-c361t-61dd7a1d2da5669e0faaa4820f6401e859716068e4558ea1508fd4e7f17b6abb3</cites><orcidid>0000-0002-8329-3958 ; 0000-0002-5183-9338 ; 0000-0002-1889-9769 ; 0000-0002-7486-4165 ; 0009-0004-4610-4769</orcidid></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><linktohtml>$$Uhttps://www.sciencedirect.com/science/article/pii/S1569199323016788$$EHTML$$P50$$Gelsevier$$H</linktohtml><link.rule.ids>230,314,776,780,881,3537,27901,27902,65534</link.rule.ids><backlink>$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/38008684$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink></links><search><creatorcontrib>Gill, Eliana R.</creatorcontrib><creatorcontrib>Bartlett, Lauren E.</creatorcontrib><creatorcontrib>Milinic, Tijana</creatorcontrib><creatorcontrib>Burdis, Nora</creatorcontrib><creatorcontrib>Pilewski, Joseph M.</creatorcontrib><creatorcontrib>Dunitz, Jordan M.</creatorcontrib><creatorcontrib>Kapnadak, Siddhartha G.</creatorcontrib><creatorcontrib>Goss, Christopher H.</creatorcontrib><creatorcontrib>Ramos, Kathleen J.</creatorcontrib><title>A longitudinal analysis of respiratory symptoms in people with cystic fibrosis with advanced lung disease on and off ETI</title><title>Journal of cystic fibrosis</title><addtitle>J Cyst Fibros</addtitle><description>•ETI decreases respiratory symptoms in adults with CF with advanced lung disease over 24-months.•Women on ETI have no significant difference in symptoms than men not on ETI in advanced CF disease.•No ceiling affect noted on CFRSD-CRISS and CFQ-R in adults with CF with advanced lung disease.
People with CF (PwCF), particularly those with advanced lung disease (ALD), experience frequent respiratory symptoms. A major CF breakthrough was the approval of elexacaftor/tezacaftor/ivacaftor (ETI) in 2019, which has been shown to improve symptoms and lung function in the CF population, and decrease pulmonary exacerbations. The purpose of this study was to analyze longitudinal changes in respiratory symptoms over 24 months in ETI-treated and untreated PwCF with ALD Symptoms were measured among CF adults with ppFEV1 < 40% (N = 48, 24 ETI-treated, 24 untreated) using the CFRSD-CRISS and the CFQ-R [respiratory]. Two multilevel growth models assessed the rate of change in symptoms overall and within the ETI-treated and untreated groups. PwCF on ETI had significantly lower symptom severity over 24 months than those not on ETI as measured by the CRISS and CFQ-R. The ETI-treated group maintained an -11.7 and +19.3 point difference(p<0.01) in CRISS and CFQ-R scores over the study compared to the non-ETI group, achieving minimal clinically important differences on average between groups on both instruments. No change in the symptom burden trajectory between groups was observed (p = 0.58). Even with ALD, ETI-treated PwCF have a lower respiratory burden than those not on ETI. This may be confounded by survivorship bias in the non-ETI group. Of note, in this ALD cohort, neither instrument demonstrated ceiling effects. Our results suggest that, while ETI has significantly improved the lived experience, PwCF with ALD are still plagued by respiratory symptoms.</description><subject>Adult</subject><subject>Aminophenols</subject><subject>Benzodioxoles - therapeutic use</subject><subject>Cystic fibrosis</subject><subject>Cystic Fibrosis - complications</subject><subject>Cystic Fibrosis - drug therapy</subject><subject>Cystic Fibrosis Transmembrane Conductance Regulator</subject><subject>Cystic fibrosis transmembrane conductance regulator modulator</subject><subject>Elexacaftor/tezacaftor/ivacaftor</subject><subject>Humans</subject><subject>Lung</subject><subject>Mutation</subject><subject>Pyrazoles</subject><subject>Pyridines</subject><subject>Pyrrolidines</subject><subject>Respiratory symptoms</subject><issn>1569-1993</issn><issn>1873-5010</issn><issn>1873-5010</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>2024</creationdate><recordtype>article</recordtype><sourceid>EIF</sourceid><recordid>eNp9kc1u1DAUhSMEoqXwAGyQl2wS7s2P44gFqqoClSqxKWvLsa-nHiVxsJMpeXs8TKlgw8a27HM-X52TZW8RCgTkH_bFXtuihLIqEAsA8Sw7R9FWeQMIz9O54V2OXVedZa9i3ANgC614mZ1VIom5qM-zn5ds8NPOLatxkxqYSssWXWTeskBxdkEtPmwsbuO8-DEyN7GZ_DwQe3DLPdNbXJxm1vXBH22_L5U5qEmTYcM67ZhxkVQk5qdENwls2fXdzevshVVDpDeP-0X2_fP13dXX_Pbbl5ury9tcVxyXnKMxrUJTGtVw3hFYpVQtSrC8BiTRdC1y4ILqphGksAFhTU2txbbnqu-ri-zTiTuv_UhG07QENcg5uFGFTXrl5L8vk7uXO3-QCF0tKqgT4f0jIfgfK8VFji5qGgY1kV-jLEVXp1kBuiTFk1SnNGIg-_QPgjxWJvcyVSaPlUlEmVpInnd_D_jk-NNREnw8CSjFdHAUZNSOjvm6QHqRxrv_4H8BKzapxw</recordid><startdate>20240101</startdate><enddate>20240101</enddate><creator>Gill, Eliana R.</creator><creator>Bartlett, Lauren E.</creator><creator>Milinic, Tijana</creator><creator>Burdis, Nora</creator><creator>Pilewski, Joseph M.</creator><creator>Dunitz, Jordan M.</creator><creator>Kapnadak, Siddhartha G.</creator><creator>Goss, Christopher H.</creator><creator>Ramos, Kathleen J.</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><scope>5PM</scope><orcidid>https://orcid.org/0000-0002-8329-3958</orcidid><orcidid>https://orcid.org/0000-0002-5183-9338</orcidid><orcidid>https://orcid.org/0000-0002-1889-9769</orcidid><orcidid>https://orcid.org/0000-0002-7486-4165</orcidid><orcidid>https://orcid.org/0009-0004-4610-4769</orcidid></search><sort><creationdate>20240101</creationdate><title>A longitudinal analysis of respiratory symptoms in people with cystic fibrosis with advanced lung disease on and off ETI</title><author>Gill, Eliana R. ; Bartlett, Lauren E. ; Milinic, Tijana ; Burdis, Nora ; Pilewski, Joseph M. ; Dunitz, Jordan M. ; Kapnadak, Siddhartha G. ; Goss, Christopher H. ; Ramos, Kathleen J.</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-LOGICAL-c361t-61dd7a1d2da5669e0faaa4820f6401e859716068e4558ea1508fd4e7f17b6abb3</frbrgroupid><rsrctype>articles</rsrctype><prefilter>articles</prefilter><language>eng</language><creationdate>2024</creationdate><topic>Adult</topic><topic>Aminophenols</topic><topic>Benzodioxoles - therapeutic use</topic><topic>Cystic fibrosis</topic><topic>Cystic Fibrosis - complications</topic><topic>Cystic Fibrosis - drug therapy</topic><topic>Cystic Fibrosis Transmembrane Conductance Regulator</topic><topic>Cystic fibrosis transmembrane conductance regulator modulator</topic><topic>Elexacaftor/tezacaftor/ivacaftor</topic><topic>Humans</topic><topic>Lung</topic><topic>Mutation</topic><topic>Pyrazoles</topic><topic>Pyridines</topic><topic>Pyrrolidines</topic><topic>Respiratory symptoms</topic><toplevel>peer_reviewed</toplevel><toplevel>online_resources</toplevel><creatorcontrib>Gill, Eliana R.</creatorcontrib><creatorcontrib>Bartlett, Lauren E.</creatorcontrib><creatorcontrib>Milinic, Tijana</creatorcontrib><creatorcontrib>Burdis, Nora</creatorcontrib><creatorcontrib>Pilewski, Joseph M.</creatorcontrib><creatorcontrib>Dunitz, Jordan M.</creatorcontrib><creatorcontrib>Kapnadak, Siddhartha G.</creatorcontrib><creatorcontrib>Goss, Christopher H.</creatorcontrib><creatorcontrib>Ramos, Kathleen J.</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><collection>PubMed Central (Full Participant titles)</collection><jtitle>Journal of cystic fibrosis</jtitle></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext</fulltext></delivery><addata><au>Gill, Eliana R.</au><au>Bartlett, Lauren E.</au><au>Milinic, Tijana</au><au>Burdis, Nora</au><au>Pilewski, Joseph M.</au><au>Dunitz, Jordan M.</au><au>Kapnadak, Siddhartha G.</au><au>Goss, Christopher H.</au><au>Ramos, Kathleen J.</au><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>A longitudinal analysis of respiratory symptoms in people with cystic fibrosis with advanced lung disease on and off ETI</atitle><jtitle>Journal of cystic fibrosis</jtitle><addtitle>J Cyst Fibros</addtitle><date>2024-01-01</date><risdate>2024</risdate><volume>23</volume><issue>1</issue><spage>161</spage><epage>164</epage><pages>161-164</pages><issn>1569-1993</issn><issn>1873-5010</issn><eissn>1873-5010</eissn><abstract>•ETI decreases respiratory symptoms in adults with CF with advanced lung disease over 24-months.•Women on ETI have no significant difference in symptoms than men not on ETI in advanced CF disease.•No ceiling affect noted on CFRSD-CRISS and CFQ-R in adults with CF with advanced lung disease.
People with CF (PwCF), particularly those with advanced lung disease (ALD), experience frequent respiratory symptoms. A major CF breakthrough was the approval of elexacaftor/tezacaftor/ivacaftor (ETI) in 2019, which has been shown to improve symptoms and lung function in the CF population, and decrease pulmonary exacerbations. The purpose of this study was to analyze longitudinal changes in respiratory symptoms over 24 months in ETI-treated and untreated PwCF with ALD Symptoms were measured among CF adults with ppFEV1 < 40% (N = 48, 24 ETI-treated, 24 untreated) using the CFRSD-CRISS and the CFQ-R [respiratory]. Two multilevel growth models assessed the rate of change in symptoms overall and within the ETI-treated and untreated groups. PwCF on ETI had significantly lower symptom severity over 24 months than those not on ETI as measured by the CRISS and CFQ-R. The ETI-treated group maintained an -11.7 and +19.3 point difference(p<0.01) in CRISS and CFQ-R scores over the study compared to the non-ETI group, achieving minimal clinically important differences on average between groups on both instruments. No change in the symptom burden trajectory between groups was observed (p = 0.58). Even with ALD, ETI-treated PwCF have a lower respiratory burden than those not on ETI. This may be confounded by survivorship bias in the non-ETI group. Of note, in this ALD cohort, neither instrument demonstrated ceiling effects. Our results suggest that, while ETI has significantly improved the lived experience, PwCF with ALD are still plagued by respiratory symptoms.</abstract><cop>Netherlands</cop><pub>Elsevier B.V</pub><pmid>38008684</pmid><doi>10.1016/j.jcf.2023.11.008</doi><tpages>4</tpages><orcidid>https://orcid.org/0000-0002-8329-3958</orcidid><orcidid>https://orcid.org/0000-0002-5183-9338</orcidid><orcidid>https://orcid.org/0000-0002-1889-9769</orcidid><orcidid>https://orcid.org/0000-0002-7486-4165</orcidid><orcidid>https://orcid.org/0009-0004-4610-4769</orcidid></addata></record> |
| fulltext | fulltext |
| identifier | ISSN: 1569-1993 |
| ispartof | Journal of cystic fibrosis, 2024-01, Vol.23 (1), p.161-164 |
| issn | 1569-1993 1873-5010 1873-5010 |
| language | eng |
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| source | MEDLINE; Elsevier ScienceDirect Journals Complete |
| subjects | Adult Aminophenols Benzodioxoles - therapeutic use Cystic fibrosis Cystic Fibrosis - complications Cystic Fibrosis - drug therapy Cystic Fibrosis Transmembrane Conductance Regulator Cystic fibrosis transmembrane conductance regulator modulator Elexacaftor/tezacaftor/ivacaftor Humans Lung Mutation Pyrazoles Pyridines Pyrrolidines Respiratory symptoms |
| title | A longitudinal analysis of respiratory symptoms in people with cystic fibrosis with advanced lung disease on and off ETI |
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