Assessment of Physical Condition in Cystic Fibrosis Patients during 12-month Treatment with Cystic Fibrosis Transmembrane Conductance Regulator Modulators

Assessment of Physical Condition in Cystic Fibrosis Patients during 12-month Treatment with Cystic Fibrosis Transmembrane Conductance Regulator Modulators

Objective − To investigate the effect of cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapy on physical condition in children with cystic fibrosis (CF) during a 12month period.Materials and Methods − This is a retrospective cohort study including children aged ≥5 years trea...

Ausführliche Beschreibung

Gespeichert in:
Bibliographische Detailangaben
Veröffentlicht in:Central European journal of paediatrics (Online) 2024-04, Vol.20 (1)
Hauptverfasser: Hrvoić, Lovro, Tješić-Drinković, Duška, Bambir, Ivan, Slapničar, Andrej, Omerza, Lana, Todorić, Ivana, Vukić Dugac, Andrea
Format: Artikel
Sprache:eng
Online-Zugang:Volltext
Tags: Tag hinzufügen
Keine Tags, Fügen Sie den ersten Tag hinzu!
container_end_page
container_issue 1
container_start_page
container_title Central European journal of paediatrics (Online)
container_volume 20
creator Hrvoić, Lovro
Tješić-Drinković, Duška
Bambir, Ivan
Slapničar, Andrej
Omerza, Lana
Todorić, Ivana
Vukić Dugac, Andrea
description Objective − To investigate the effect of cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapy on physical condition in children with cystic fibrosis (CF) during a 12month period.Materials and Methods − This is a retrospective cohort study including children aged ≥5 years treated with Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) or Lumacaftor/ Ivacaftor (LUM/IVA). A six-minute walk test (6MWT) was performed at baseline and at the 3-month follow-ups. Changes in spirometry and sweat chlorides, and body mass index (BMI) were also observed. Collected data were analysed for changes in 6MWT results in correlation with other parameters. The 6MWT results were compared to those predicted for healthy peers. Missing values were replaced using imputation.Results − Study includes 34 patients (median age 14 years); 28 received ELX/TEZ/ IVA, and 6 LUM/IVA. The average 6MWT walking distance (6MWD) increased from 519.3±107.7 m at baseline to 620.0±99.5 m after 12 months (P
doi_str_mv 10.5457/p2005-114.354
format Article
fullrecord <record><control><sourceid>crossref</sourceid><recordid>TN_cdi_crossref_primary_10_5457_p2005_114_354</recordid><sourceformat>XML</sourceformat><sourcesystem>PC</sourcesystem><sourcerecordid>10_5457_p2005_114_354</sourcerecordid><originalsourceid>FETCH-LOGICAL-c1474-9aa213608c6f6bb8f316e06426e6f8b9cb7e3eb2dcb1a7a04cdc0d95c906e70a3</originalsourceid><addsrcrecordid>eNpdkM9KAzEYxIMoWGqP3vMCqfm32eZYFqtCxSL1vCTZbBvpJiVJkb6KT2vaehBPM99hZj5-ANwTPK14VT_sKcYVIoRPWcWvwIhyiRETNbn-42_BJKVPjDGVlFayHoHveUo2pcH6DEMPV9tjckbtYBN857ILHjoPm2PKzsCF0zEkl-BKZVcCCXaH6PwGEoqG4PMWrqNV-dz15cr5P7eOypepQRe154mDycobC9_t5rBTOUT4GrqLS3fgple7ZCe_OgYfi8d184yWb08vzXyJDOE1R1IpSpjAMyN6ofWsZ0RYLDgVVvQzLY2uLbOadkYTVSvMTWdwJysjsbA1VmwM0KXXlC9TtH27j25Q8dgS3J7Ytme2bWHbFrbsB_QMcFg</addsrcrecordid><sourcetype>Aggregation Database</sourcetype><iscdi>true</iscdi><recordtype>article</recordtype></control><display><type>article</type><title>Assessment of Physical Condition in Cystic Fibrosis Patients during 12-month Treatment with Cystic Fibrosis Transmembrane Conductance Regulator Modulators</title><source>Alma/SFX Local Collection</source><creator>Hrvoić, Lovro ; Tješić-Drinković, Duška ; Bambir, Ivan ; Slapničar, Andrej ; Omerza, Lana ; Todorić, Ivana ; Vukić Dugac, Andrea</creator><creatorcontrib>Hrvoić, Lovro ; Tješić-Drinković, Duška ; Bambir, Ivan ; Slapničar, Andrej ; Omerza, Lana ; Todorić, Ivana ; Vukić Dugac, Andrea</creatorcontrib><description>Objective − To investigate the effect of cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapy on physical condition in children with cystic fibrosis (CF) during a 12month period.Materials and Methods − This is a retrospective cohort study including children aged ≥5 years treated with Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) or Lumacaftor/ Ivacaftor (LUM/IVA). A six-minute walk test (6MWT) was performed at baseline and at the 3-month follow-ups. Changes in spirometry and sweat chlorides, and body mass index (BMI) were also observed. Collected data were analysed for changes in 6MWT results in correlation with other parameters. The 6MWT results were compared to those predicted for healthy peers. Missing values were replaced using imputation.Results − Study includes 34 patients (median age 14 years); 28 received ELX/TEZ/ IVA, and 6 LUM/IVA. The average 6MWT walking distance (6MWD) increased from 519.3±107.7 m at baseline to 620.0±99.5 m after 12 months (P&lt;0.0001). The 12 month 6MWD values matched those expected for healthy peers. The increase in BMI z-score (P=0.019) and ppFEV1 (P&lt;0.0001) visible at the 3-month followup was sustained throughout the rest of the year. Sweat chloride concentration decreased (P&lt; 0.0001); after 12 months 13/34 subjects had values below 60 mmol/L. No correlation between 6MWD and other parameters was observed.Conclusion − Significant improvement in 6MWT results was already visible at three months following the initiation of CFTR modulator treatment. After 12 months patients performed 6MWT at a level indistinguishable from that expected of healthy peers.</description><identifier>ISSN: 2490-3671</identifier><identifier>EISSN: 2490-3671</identifier><identifier>DOI: 10.5457/p2005-114.354</identifier><language>eng</language><ispartof>Central European journal of paediatrics (Online), 2024-04, Vol.20 (1)</ispartof><oa>free_for_read</oa><woscitedreferencessubscribed>false</woscitedreferencessubscribed></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><link.rule.ids>314,780,784,27923,27924</link.rule.ids></links><search><creatorcontrib>Hrvoić, Lovro</creatorcontrib><creatorcontrib>Tješić-Drinković, Duška</creatorcontrib><creatorcontrib>Bambir, Ivan</creatorcontrib><creatorcontrib>Slapničar, Andrej</creatorcontrib><creatorcontrib>Omerza, Lana</creatorcontrib><creatorcontrib>Todorić, Ivana</creatorcontrib><creatorcontrib>Vukić Dugac, Andrea</creatorcontrib><title>Assessment of Physical Condition in Cystic Fibrosis Patients during 12-month Treatment with Cystic Fibrosis Transmembrane Conductance Regulator Modulators</title><title>Central European journal of paediatrics (Online)</title><description>Objective − To investigate the effect of cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapy on physical condition in children with cystic fibrosis (CF) during a 12month period.Materials and Methods − This is a retrospective cohort study including children aged ≥5 years treated with Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) or Lumacaftor/ Ivacaftor (LUM/IVA). A six-minute walk test (6MWT) was performed at baseline and at the 3-month follow-ups. Changes in spirometry and sweat chlorides, and body mass index (BMI) were also observed. Collected data were analysed for changes in 6MWT results in correlation with other parameters. The 6MWT results were compared to those predicted for healthy peers. Missing values were replaced using imputation.Results − Study includes 34 patients (median age 14 years); 28 received ELX/TEZ/ IVA, and 6 LUM/IVA. The average 6MWT walking distance (6MWD) increased from 519.3±107.7 m at baseline to 620.0±99.5 m after 12 months (P&lt;0.0001). The 12 month 6MWD values matched those expected for healthy peers. The increase in BMI z-score (P=0.019) and ppFEV1 (P&lt;0.0001) visible at the 3-month followup was sustained throughout the rest of the year. Sweat chloride concentration decreased (P&lt; 0.0001); after 12 months 13/34 subjects had values below 60 mmol/L. No correlation between 6MWD and other parameters was observed.Conclusion − Significant improvement in 6MWT results was already visible at three months following the initiation of CFTR modulator treatment. After 12 months patients performed 6MWT at a level indistinguishable from that expected of healthy peers.</description><issn>2490-3671</issn><issn>2490-3671</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>2024</creationdate><recordtype>article</recordtype><recordid>eNpdkM9KAzEYxIMoWGqP3vMCqfm32eZYFqtCxSL1vCTZbBvpJiVJkb6KT2vaehBPM99hZj5-ANwTPK14VT_sKcYVIoRPWcWvwIhyiRETNbn-42_BJKVPjDGVlFayHoHveUo2pcH6DEMPV9tjckbtYBN857ILHjoPm2PKzsCF0zEkl-BKZVcCCXaH6PwGEoqG4PMWrqNV-dz15cr5P7eOypepQRe154mDycobC9_t5rBTOUT4GrqLS3fgple7ZCe_OgYfi8d184yWb08vzXyJDOE1R1IpSpjAMyN6ofWsZ0RYLDgVVvQzLY2uLbOadkYTVSvMTWdwJysjsbA1VmwM0KXXlC9TtH27j25Q8dgS3J7Ytme2bWHbFrbsB_QMcFg</recordid><startdate>20240424</startdate><enddate>20240424</enddate><creator>Hrvoić, Lovro</creator><creator>Tješić-Drinković, Duška</creator><creator>Bambir, Ivan</creator><creator>Slapničar, Andrej</creator><creator>Omerza, Lana</creator><creator>Todorić, Ivana</creator><creator>Vukić Dugac, Andrea</creator><scope>AAYXX</scope><scope>CITATION</scope></search><sort><creationdate>20240424</creationdate><title>Assessment of Physical Condition in Cystic Fibrosis Patients during 12-month Treatment with Cystic Fibrosis Transmembrane Conductance Regulator Modulators</title><author>Hrvoić, Lovro ; Tješić-Drinković, Duška ; Bambir, Ivan ; Slapničar, Andrej ; Omerza, Lana ; Todorić, Ivana ; Vukić Dugac, Andrea</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-LOGICAL-c1474-9aa213608c6f6bb8f316e06426e6f8b9cb7e3eb2dcb1a7a04cdc0d95c906e70a3</frbrgroupid><rsrctype>articles</rsrctype><prefilter>articles</prefilter><language>eng</language><creationdate>2024</creationdate><toplevel>online_resources</toplevel><creatorcontrib>Hrvoić, Lovro</creatorcontrib><creatorcontrib>Tješić-Drinković, Duška</creatorcontrib><creatorcontrib>Bambir, Ivan</creatorcontrib><creatorcontrib>Slapničar, Andrej</creatorcontrib><creatorcontrib>Omerza, Lana</creatorcontrib><creatorcontrib>Todorić, Ivana</creatorcontrib><creatorcontrib>Vukić Dugac, Andrea</creatorcontrib><collection>CrossRef</collection><jtitle>Central European journal of paediatrics (Online)</jtitle></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext</fulltext></delivery><addata><au>Hrvoić, Lovro</au><au>Tješić-Drinković, Duška</au><au>Bambir, Ivan</au><au>Slapničar, Andrej</au><au>Omerza, Lana</au><au>Todorić, Ivana</au><au>Vukić Dugac, Andrea</au><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>Assessment of Physical Condition in Cystic Fibrosis Patients during 12-month Treatment with Cystic Fibrosis Transmembrane Conductance Regulator Modulators</atitle><jtitle>Central European journal of paediatrics (Online)</jtitle><date>2024-04-24</date><risdate>2024</risdate><volume>20</volume><issue>1</issue><issn>2490-3671</issn><eissn>2490-3671</eissn><abstract>Objective − To investigate the effect of cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapy on physical condition in children with cystic fibrosis (CF) during a 12month period.Materials and Methods − This is a retrospective cohort study including children aged ≥5 years treated with Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) or Lumacaftor/ Ivacaftor (LUM/IVA). A six-minute walk test (6MWT) was performed at baseline and at the 3-month follow-ups. Changes in spirometry and sweat chlorides, and body mass index (BMI) were also observed. Collected data were analysed for changes in 6MWT results in correlation with other parameters. The 6MWT results were compared to those predicted for healthy peers. Missing values were replaced using imputation.Results − Study includes 34 patients (median age 14 years); 28 received ELX/TEZ/ IVA, and 6 LUM/IVA. The average 6MWT walking distance (6MWD) increased from 519.3±107.7 m at baseline to 620.0±99.5 m after 12 months (P&lt;0.0001). The 12 month 6MWD values matched those expected for healthy peers. The increase in BMI z-score (P=0.019) and ppFEV1 (P&lt;0.0001) visible at the 3-month followup was sustained throughout the rest of the year. Sweat chloride concentration decreased (P&lt; 0.0001); after 12 months 13/34 subjects had values below 60 mmol/L. No correlation between 6MWD and other parameters was observed.Conclusion − Significant improvement in 6MWT results was already visible at three months following the initiation of CFTR modulator treatment. After 12 months patients performed 6MWT at a level indistinguishable from that expected of healthy peers.</abstract><doi>10.5457/p2005-114.354</doi><oa>free_for_read</oa></addata></record>
fulltext fulltext
identifier ISSN: 2490-3671
ispartof Central European journal of paediatrics (Online), 2024-04, Vol.20 (1)
issn 2490-3671
2490-3671
language eng
recordid cdi_crossref_primary_10_5457_p2005_114_354
source Alma/SFX Local Collection
title Assessment of Physical Condition in Cystic Fibrosis Patients during 12-month Treatment with Cystic Fibrosis Transmembrane Conductance Regulator Modulators
url https://sfx.bib-bvb.de/sfx_tum?ctx_ver=Z39.88-2004&ctx_enc=info:ofi/enc:UTF-8&ctx_tim=2025-01-12T03%3A39%3A55IST&url_ver=Z39.88-2004&url_ctx_fmt=infofi/fmt:kev:mtx:ctx&rfr_id=info:sid/primo.exlibrisgroup.com:primo3-Article-crossref&rft_val_fmt=info:ofi/fmt:kev:mtx:journal&rft.genre=article&rft.atitle=Assessment%20of%20Physical%20Condition%20in%20Cystic%20Fibrosis%20Patients%20during%2012-month%20Treatment%20with%20Cystic%20Fibrosis%20Transmembrane%20Conductance%20Regulator%20Modulators&rft.jtitle=Central%20European%20journal%20of%20paediatrics%20(Online)&rft.au=Hrvoi%C4%87,%20Lovro&rft.date=2024-04-24&rft.volume=20&rft.issue=1&rft.issn=2490-3671&rft.eissn=2490-3671&rft_id=info:doi/10.5457/p2005-114.354&rft_dat=%3Ccrossref%3E10_5457_p2005_114_354%3C/crossref%3E%3Curl%3E%3C/url%3E&disable_directlink=true&sfx.directlink=off&sfx.report_link=0&rft_id=info:oai/&rft_id=info:pmid/&rfr_iscdi=true