Lumacaftor/ivacaftor-associated health stabilisation in adults with severe cystic fibrosis
Introduction: Lumacaftor/ivacaftor (LUM/IVA) has been shown to improve clinical outcomes in cystic fibrosis (CF) patients homozygous for Phe508del with forced expiratory volume in 1 s (FEV1) % pred >40%. We assessed the clinical utility of LUM/IVA in all eligible adult CF patients with FEV1 % pre...
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| description | Introduction: Lumacaftor/ivacaftor (LUM/IVA) has been shown to improve clinical outcomes in cystic fibrosis (CF) patients homozygous for Phe508del with forced expiratory volume in 1 s (FEV1) % pred >40%. We assessed the clinical utility of LUM/IVA in all eligible adult CF patients with FEV1 % pred |
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| fullrecord | <record><control><sourceid>proquest_webof</sourceid><recordid>TN_cdi_webofscience_primary_000625441800008</recordid><sourceformat>XML</sourceformat><sourcesystem>PC</sourcesystem><doaj_id>oai_doaj_org_article_7098879b20a2481d8007c4747cebd423</doaj_id><sourcerecordid>2488569558</sourcerecordid><originalsourceid>FETCH-LOGICAL-c401t-ab10b5f895bf576ce8ab84aa3c33999bc92f1eaa80a5965f464eac3f8293333c3</originalsourceid><addsrcrecordid>eNqNUk1r3DAQNaWlCWl-QaH4WChORl-2dCmUpR-BhV7aSy9iJMtZBa-VSvKG_PvKu5sluVWH0TDz5s1onqrqPYErQiS7poxQEJxcAVBgDS32VXW-RJsl_PqZf1ZdpnQHAERQydv2bXXGmGiVAHpe_VnPW7Q45BCv_e7oNZhSsB6z6-uNwzFv6pTR-NEnzD5MtZ9q7Ocxp_rBL0m3c9HV9jFlb-vBmxiST--qNwOOyV0e74vq97evv1Y_mvXP7zerL-vGciC5QUPAiEEqYQbRtdZJNJIjMsuYUspYRQfiECWgUK0YeMsdWjZIqlg5ll1UNwfePuCdvo9-i_FRB_R6HwjxVmMsg41Od6Ck7JShgJRL0kuAzvKOd9aZnlNWuD4fuO5ns3W9dVOOOL4gfZmZ_Ebfhp3uZEuA0ULw8UgQw9_Zpay3Plk3jji5MCdd2spl90IWKDtAbVlXim44tSGgF5H1k8h6L7JeRC5VH55PeKp5krQAPh0AD86EIVnvJutOsPINWio4J-XpAMsQ8v_RK5_3-q_CPGX2D870w_E</addsrcrecordid><sourcetype>Open Website</sourcetype><iscdi>true</iscdi><recordtype>article</recordtype><pqid>2488569558</pqid></control><display><type>article</type><title>Lumacaftor/ivacaftor-associated health stabilisation in adults with severe cystic fibrosis</title><source>DOAJ Directory of Open Access Journals</source><source>Web of Science - Science Citation Index Expanded - 2021<img src="https://exlibris-pub.s3.amazonaws.com/fromwos-v2.jpg" /></source><source>EZB-FREE-00999 freely available EZB journals</source><source>PubMed Central</source><creator>King, Susannah J. ; Keating, Dominic ; Williams, Elyssa ; Paul, Eldho ; Borg, Brigitte M. ; Finlayson, Felicity ; Button, Brenda M. ; Wilson, John W. ; Kotsimbos, Tom</creator><creatorcontrib>King, Susannah J. ; Keating, Dominic ; Williams, Elyssa ; Paul, Eldho ; Borg, Brigitte M. ; Finlayson, Felicity ; Button, Brenda M. ; Wilson, John W. ; Kotsimbos, Tom</creatorcontrib><description>Introduction: Lumacaftor/ivacaftor (LUM/IVA) has been shown to improve clinical outcomes in cystic fibrosis (CF) patients homozygous for Phe508del with forced expiratory volume in 1 s (FEV1) % pred >40%. We assessed the clinical utility of LUM/IVA in all eligible adult CF patients with FEV1 % pred <40% treated for at least 1 year under a single-centre managed access programme.
Methods: Following clinical optimisation, eligible patients (n=40) with FEV1 % pred <40% were commenced on LUM/IVA and monitored for tolerance and clinical outcomes, including health service utilisation, pulmonary function, weight and body composition. 24 patients reached 1 year of treatment by the time of evaluation. Six patients discontinued due to adverse events (five for increased airways reactivity) and three underwent lung transplantation.
Results: In comparison with the year prior to LUM/IVA commencement, significant reductions (median per year) were observed in the treatment year in the number of pulmonary exacerbations requiring hospitalisation (from 3 to 1.5; p=0.0002), hospitalisation days (from 27 to 17; p=0.0002) and intravenous antibiotic (IVAB) usage days (from 45 to 27; p=0.0007). Mean +/- SD change in FEV1 % pred was -2.10 +/- 1.18% per year in the year prior, with the decline reversed in the year following (+1.45 +/- 1.13% per year; p=0.035), although there was significant heterogeneity in individual responses. Mean +/- SD weight gain at 1 year was 2.5 +/- 4.1 kg (p=0.0007), comprising mainly fat mass (mean 2.2 kg). The proportion of patients severely underweight (body mass index <18.5 kg.m(-2)) decreased from 33% at baseline to 13% at 1 year (p=0.003).
Conclusion: This real-world evaluation study demonstrated benefits over several clinical domains (infective exacerbations requiring hospitalisation, IVABs, pulmonary function decline and nutritional parameters) in CF patients with severe lung disease.</description><identifier>ISSN: 2312-0541</identifier><identifier>EISSN: 2312-0541</identifier><identifier>DOI: 10.1183/23120541.00203-2020</identifier><identifier>PMID: 33569502</identifier><language>eng</language><publisher>SHEFFIELD: European Respiratory Soc Journals Ltd</publisher><subject>Life Sciences & Biomedicine ; Original ; Respiratory System ; Science & Technology</subject><ispartof>ERJ open research, 2021-01, Vol.7 (1), p.203, Article 00203</ispartof><rights>Copyright ©ERS 2021.</rights><rights>Copyright ©ERS 2021 2021</rights><lds50>peer_reviewed</lds50><oa>free_for_read</oa><woscitedreferencessubscribed>true</woscitedreferencessubscribed><woscitedreferencescount>12</woscitedreferencescount><woscitedreferencesoriginalsourcerecordid>wos000625441800008</woscitedreferencesoriginalsourcerecordid><citedby>FETCH-LOGICAL-c401t-ab10b5f895bf576ce8ab84aa3c33999bc92f1eaa80a5965f464eac3f8293333c3</citedby><cites>FETCH-LOGICAL-c401t-ab10b5f895bf576ce8ab84aa3c33999bc92f1eaa80a5965f464eac3f8293333c3</cites><orcidid>0000-0001-6150-6927 ; 0000-0001-6821-9234 ; 0000-0001-5102-5783</orcidid></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><linktopdf>$$Uhttps://www.ncbi.nlm.nih.gov/pmc/articles/PMC7861032/pdf/$$EPDF$$P50$$Gpubmedcentral$$Hfree_for_read</linktopdf><linktohtml>$$Uhttps://www.ncbi.nlm.nih.gov/pmc/articles/PMC7861032/$$EHTML$$P50$$Gpubmedcentral$$Hfree_for_read</linktohtml><link.rule.ids>230,315,728,781,785,865,886,2103,2115,27929,27930,39263,53796,53798</link.rule.ids><backlink>$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/33569502$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink></links><search><creatorcontrib>King, Susannah J.</creatorcontrib><creatorcontrib>Keating, Dominic</creatorcontrib><creatorcontrib>Williams, Elyssa</creatorcontrib><creatorcontrib>Paul, Eldho</creatorcontrib><creatorcontrib>Borg, Brigitte M.</creatorcontrib><creatorcontrib>Finlayson, Felicity</creatorcontrib><creatorcontrib>Button, Brenda M.</creatorcontrib><creatorcontrib>Wilson, John W.</creatorcontrib><creatorcontrib>Kotsimbos, Tom</creatorcontrib><title>Lumacaftor/ivacaftor-associated health stabilisation in adults with severe cystic fibrosis</title><title>ERJ open research</title><addtitle>ERJ OPEN RES</addtitle><addtitle>ERJ Open Res</addtitle><description>Introduction: Lumacaftor/ivacaftor (LUM/IVA) has been shown to improve clinical outcomes in cystic fibrosis (CF) patients homozygous for Phe508del with forced expiratory volume in 1 s (FEV1) % pred >40%. We assessed the clinical utility of LUM/IVA in all eligible adult CF patients with FEV1 % pred <40% treated for at least 1 year under a single-centre managed access programme.
Methods: Following clinical optimisation, eligible patients (n=40) with FEV1 % pred <40% were commenced on LUM/IVA and monitored for tolerance and clinical outcomes, including health service utilisation, pulmonary function, weight and body composition. 24 patients reached 1 year of treatment by the time of evaluation. Six patients discontinued due to adverse events (five for increased airways reactivity) and three underwent lung transplantation.
Results: In comparison with the year prior to LUM/IVA commencement, significant reductions (median per year) were observed in the treatment year in the number of pulmonary exacerbations requiring hospitalisation (from 3 to 1.5; p=0.0002), hospitalisation days (from 27 to 17; p=0.0002) and intravenous antibiotic (IVAB) usage days (from 45 to 27; p=0.0007). Mean +/- SD change in FEV1 % pred was -2.10 +/- 1.18% per year in the year prior, with the decline reversed in the year following (+1.45 +/- 1.13% per year; p=0.035), although there was significant heterogeneity in individual responses. Mean +/- SD weight gain at 1 year was 2.5 +/- 4.1 kg (p=0.0007), comprising mainly fat mass (mean 2.2 kg). The proportion of patients severely underweight (body mass index <18.5 kg.m(-2)) decreased from 33% at baseline to 13% at 1 year (p=0.003).
Conclusion: This real-world evaluation study demonstrated benefits over several clinical domains (infective exacerbations requiring hospitalisation, IVABs, pulmonary function decline and nutritional parameters) in CF patients with severe lung disease.</description><subject>Life Sciences & Biomedicine</subject><subject>Original</subject><subject>Respiratory System</subject><subject>Science & Technology</subject><issn>2312-0541</issn><issn>2312-0541</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>2021</creationdate><recordtype>article</recordtype><sourceid>HGBXW</sourceid><sourceid>DOA</sourceid><recordid>eNqNUk1r3DAQNaWlCWl-QaH4WChORl-2dCmUpR-BhV7aSy9iJMtZBa-VSvKG_PvKu5sluVWH0TDz5s1onqrqPYErQiS7poxQEJxcAVBgDS32VXW-RJsl_PqZf1ZdpnQHAERQydv2bXXGmGiVAHpe_VnPW7Q45BCv_e7oNZhSsB6z6-uNwzFv6pTR-NEnzD5MtZ9q7Ocxp_rBL0m3c9HV9jFlb-vBmxiST--qNwOOyV0e74vq97evv1Y_mvXP7zerL-vGciC5QUPAiEEqYQbRtdZJNJIjMsuYUspYRQfiECWgUK0YeMsdWjZIqlg5ll1UNwfePuCdvo9-i_FRB_R6HwjxVmMsg41Od6Ck7JShgJRL0kuAzvKOd9aZnlNWuD4fuO5ns3W9dVOOOL4gfZmZ_Ebfhp3uZEuA0ULw8UgQw9_Zpay3Plk3jji5MCdd2spl90IWKDtAbVlXim44tSGgF5H1k8h6L7JeRC5VH55PeKp5krQAPh0AD86EIVnvJutOsPINWio4J-XpAMsQ8v_RK5_3-q_CPGX2D870w_E</recordid><startdate>20210101</startdate><enddate>20210101</enddate><creator>King, Susannah J.</creator><creator>Keating, Dominic</creator><creator>Williams, Elyssa</creator><creator>Paul, Eldho</creator><creator>Borg, Brigitte M.</creator><creator>Finlayson, Felicity</creator><creator>Button, Brenda M.</creator><creator>Wilson, John W.</creator><creator>Kotsimbos, Tom</creator><general>European Respiratory Soc Journals Ltd</general><general>European Respiratory Society</general><scope>BLEPL</scope><scope>DTL</scope><scope>HGBXW</scope><scope>NPM</scope><scope>AAYXX</scope><scope>CITATION</scope><scope>7X8</scope><scope>5PM</scope><scope>DOA</scope><orcidid>https://orcid.org/0000-0001-6150-6927</orcidid><orcidid>https://orcid.org/0000-0001-6821-9234</orcidid><orcidid>https://orcid.org/0000-0001-5102-5783</orcidid></search><sort><creationdate>20210101</creationdate><title>Lumacaftor/ivacaftor-associated health stabilisation in adults with severe cystic fibrosis</title><author>King, Susannah J. ; Keating, Dominic ; Williams, Elyssa ; Paul, Eldho ; Borg, Brigitte M. ; Finlayson, Felicity ; Button, Brenda M. ; Wilson, John W. ; Kotsimbos, Tom</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-LOGICAL-c401t-ab10b5f895bf576ce8ab84aa3c33999bc92f1eaa80a5965f464eac3f8293333c3</frbrgroupid><rsrctype>articles</rsrctype><prefilter>articles</prefilter><language>eng</language><creationdate>2021</creationdate><topic>Life Sciences & Biomedicine</topic><topic>Original</topic><topic>Respiratory System</topic><topic>Science & Technology</topic><toplevel>peer_reviewed</toplevel><toplevel>online_resources</toplevel><creatorcontrib>King, Susannah J.</creatorcontrib><creatorcontrib>Keating, Dominic</creatorcontrib><creatorcontrib>Williams, Elyssa</creatorcontrib><creatorcontrib>Paul, Eldho</creatorcontrib><creatorcontrib>Borg, Brigitte M.</creatorcontrib><creatorcontrib>Finlayson, Felicity</creatorcontrib><creatorcontrib>Button, Brenda M.</creatorcontrib><creatorcontrib>Wilson, John W.</creatorcontrib><creatorcontrib>Kotsimbos, Tom</creatorcontrib><collection>Web of Science Core Collection</collection><collection>Science Citation Index Expanded</collection><collection>Web of Science - Science Citation Index Expanded - 2021</collection><collection>PubMed</collection><collection>CrossRef</collection><collection>MEDLINE - Academic</collection><collection>PubMed Central (Full Participant titles)</collection><collection>DOAJ Directory of Open Access Journals</collection><jtitle>ERJ open research</jtitle></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext</fulltext></delivery><addata><au>King, Susannah J.</au><au>Keating, Dominic</au><au>Williams, Elyssa</au><au>Paul, Eldho</au><au>Borg, Brigitte M.</au><au>Finlayson, Felicity</au><au>Button, Brenda M.</au><au>Wilson, John W.</au><au>Kotsimbos, Tom</au><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>Lumacaftor/ivacaftor-associated health stabilisation in adults with severe cystic fibrosis</atitle><jtitle>ERJ open research</jtitle><stitle>ERJ OPEN RES</stitle><addtitle>ERJ Open Res</addtitle><date>2021-01-01</date><risdate>2021</risdate><volume>7</volume><issue>1</issue><spage>203</spage><pages>203-</pages><artnum>00203</artnum><issn>2312-0541</issn><eissn>2312-0541</eissn><abstract>Introduction: Lumacaftor/ivacaftor (LUM/IVA) has been shown to improve clinical outcomes in cystic fibrosis (CF) patients homozygous for Phe508del with forced expiratory volume in 1 s (FEV1) % pred >40%. We assessed the clinical utility of LUM/IVA in all eligible adult CF patients with FEV1 % pred <40% treated for at least 1 year under a single-centre managed access programme.
Methods: Following clinical optimisation, eligible patients (n=40) with FEV1 % pred <40% were commenced on LUM/IVA and monitored for tolerance and clinical outcomes, including health service utilisation, pulmonary function, weight and body composition. 24 patients reached 1 year of treatment by the time of evaluation. Six patients discontinued due to adverse events (five for increased airways reactivity) and three underwent lung transplantation.
Results: In comparison with the year prior to LUM/IVA commencement, significant reductions (median per year) were observed in the treatment year in the number of pulmonary exacerbations requiring hospitalisation (from 3 to 1.5; p=0.0002), hospitalisation days (from 27 to 17; p=0.0002) and intravenous antibiotic (IVAB) usage days (from 45 to 27; p=0.0007). Mean +/- SD change in FEV1 % pred was -2.10 +/- 1.18% per year in the year prior, with the decline reversed in the year following (+1.45 +/- 1.13% per year; p=0.035), although there was significant heterogeneity in individual responses. Mean +/- SD weight gain at 1 year was 2.5 +/- 4.1 kg (p=0.0007), comprising mainly fat mass (mean 2.2 kg). The proportion of patients severely underweight (body mass index <18.5 kg.m(-2)) decreased from 33% at baseline to 13% at 1 year (p=0.003).
Conclusion: This real-world evaluation study demonstrated benefits over several clinical domains (infective exacerbations requiring hospitalisation, IVABs, pulmonary function decline and nutritional parameters) in CF patients with severe lung disease.</abstract><cop>SHEFFIELD</cop><pub>European Respiratory Soc Journals Ltd</pub><pmid>33569502</pmid><doi>10.1183/23120541.00203-2020</doi><tpages>10</tpages><orcidid>https://orcid.org/0000-0001-6150-6927</orcidid><orcidid>https://orcid.org/0000-0001-6821-9234</orcidid><orcidid>https://orcid.org/0000-0001-5102-5783</orcidid><oa>free_for_read</oa></addata></record> |
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| subjects | Life Sciences & Biomedicine Original Respiratory System Science & Technology |
| title | Lumacaftor/ivacaftor-associated health stabilisation in adults with severe cystic fibrosis |
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