Effectiveness and Safety of Cystic Fibrosis Transmembrane Conductance Regulator Modulators in Children With Cystic Fibrosis: A Meta-Analysis

Background and AimCystic fibrosis (CF) is a genetic disease that is difficult to treat and caused by dysfunction of the cystic fibrosis transmembrane conductance regulator (CFTR) protein. Small molecules have been used to treat the symptom caused by CFTR mutations by restoring CFTR protein function....
Ausführliche Beschreibung

Gespeichert in:

Autor*in:	Qiyu Li [verfasserIn] Siyuan Liu [verfasserIn] Xuemei Ma [verfasserIn] Jiaping Yu [verfasserIn]

Format:	E-Artikel
Sprache:	Englisch

Erschienen:	2022

Schlagwörter:	CFTR (cystic fibrosis transmembrane conductance regulator) cystic fibrosis – CF corrector potentiator cystic fibrosis transmembrane conductance regulator

Übergeordnetes Werk:	In: Frontiers in Pediatrics - Frontiers Media S.A., 2013, 10(2022)
Übergeordnetes Werk:	volume:10 ; year:2022

Links:	Link aufrufen Link aufrufen Link aufrufen Journal toc

DOI / URN:	10.3389/fped.2022.937250

Katalog-ID:	DOAJ02763762X

Internformat


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520			\|a Background and AimCystic fibrosis (CF) is a genetic disease that is difficult to treat and caused by dysfunction of the cystic fibrosis transmembrane conductance regulator (CFTR) protein. Small molecules have been used to treat the symptom caused by CFTR mutations by restoring CFTR protein function. However, the data on children with CF are scarce. This meta-analysis aimed to evaluate the effectiveness and safety of this therapy in children diagnosed with CF.Materials and MethodsRelevant studies were identified through searching medical databases before April 1, 2022. The primary outcomes of ppFEV1, lung clearance index2.5 (LCI2.5), sweat chloride concentration (SwCI), and Cystic Fibrosis Questionnaire-Revised (CFQ-R) score were pooled and analyzed. The secondary outcomes were nutritional status (weight, BMI, stature, and their z-score) and adverse events under therapy.ResultsA total of twelve studies were included. Compared with the placebo group, the pooled outcome of the ppFEV1, LCI2.5, SwCI, and CFQ-R score were improved by 7.91 {[95% confidence interval (CI), 3.71–12.12], –1.00 (95% CI, –1.38 to –0.63), –35.22 (95% CI, –55.51 to –14.92), and 4.45 (95% CI, 2.31–6.59), respectively}. Compared with the placebo group, the pooled result of the change in weight was improved by 1.53 (95% CI, 0.42–2.63). All the aforementioned results were also improved in single-arm studies. No clear differences in adverse events were found between CFTR modulator therapy and the placebo group.ConclusionCFTR modulators could improve multiaspect function in children with CF and result in comparable adverse events.
650		4	\|a CFTR (cystic fibrosis transmembrane conductance regulator)
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allfields	10.3389/fped.2022.937250 doi (DE-627)DOAJ02763762X (DE-599)DOAJ479b9ffa5c4e4e27a494259f96bf00b0 DE-627 ger DE-627 rakwb eng RJ1-570 Qiyu Li verfasserin aut Effectiveness and Safety of Cystic Fibrosis Transmembrane Conductance Regulator Modulators in Children With Cystic Fibrosis: A Meta-Analysis 2022 Text txt rdacontent Computermedien c rdamedia Online-Ressource cr rdacarrier Background and AimCystic fibrosis (CF) is a genetic disease that is difficult to treat and caused by dysfunction of the cystic fibrosis transmembrane conductance regulator (CFTR) protein. Small molecules have been used to treat the symptom caused by CFTR mutations by restoring CFTR protein function. However, the data on children with CF are scarce. This meta-analysis aimed to evaluate the effectiveness and safety of this therapy in children diagnosed with CF.Materials and MethodsRelevant studies were identified through searching medical databases before April 1, 2022. The primary outcomes of ppFEV1, lung clearance index2.5 (LCI2.5), sweat chloride concentration (SwCI), and Cystic Fibrosis Questionnaire-Revised (CFQ-R) score were pooled and analyzed. The secondary outcomes were nutritional status (weight, BMI, stature, and their z-score) and adverse events under therapy.ResultsA total of twelve studies were included. Compared with the placebo group, the pooled outcome of the ppFEV1, LCI2.5, SwCI, and CFQ-R score were improved by 7.91 {[95% confidence interval (CI), 3.71–12.12], –1.00 (95% CI, –1.38 to –0.63), –35.22 (95% CI, –55.51 to –14.92), and 4.45 (95% CI, 2.31–6.59), respectively}. Compared with the placebo group, the pooled result of the change in weight was improved by 1.53 (95% CI, 0.42–2.63). All the aforementioned results were also improved in single-arm studies. No clear differences in adverse events were found between CFTR modulator therapy and the placebo group.ConclusionCFTR modulators could improve multiaspect function in children with CF and result in comparable adverse events. CFTR (cystic fibrosis transmembrane conductance regulator) cystic fibrosis – CF corrector potentiator cystic fibrosis transmembrane conductance regulator Pediatrics Siyuan Liu verfasserin aut Xuemei Ma verfasserin aut Jiaping Yu verfasserin aut In Frontiers in Pediatrics Frontiers Media S.A., 2013 10(2022) (DE-627)742738744 (DE-600)2711999-3 22962360 nnns volume:10 year:2022 https://doi.org/10.3389/fped.2022.937250 kostenfrei https://doaj.org/article/479b9ffa5c4e4e27a494259f96bf00b0 kostenfrei https://www.frontiersin.org/articles/10.3389/fped.2022.937250/full kostenfrei https://doaj.org/toc/2296-2360 Journal toc kostenfrei GBV_USEFLAG_A SYSFLAG_A GBV_DOAJ GBV_ILN_11 GBV_ILN_20 GBV_ILN_22 GBV_ILN_23 GBV_ILN_24 GBV_ILN_39 GBV_ILN_40 GBV_ILN_60 GBV_ILN_62 GBV_ILN_63 GBV_ILN_65 GBV_ILN_69 GBV_ILN_73 GBV_ILN_74 GBV_ILN_95 GBV_ILN_105 GBV_ILN_110 GBV_ILN_151 GBV_ILN_161 GBV_ILN_170 GBV_ILN_206 GBV_ILN_213 GBV_ILN_230 GBV_ILN_285 GBV_ILN_293 GBV_ILN_602 GBV_ILN_2003 GBV_ILN_2014 GBV_ILN_4012 GBV_ILN_4037 GBV_ILN_4112 GBV_ILN_4125 GBV_ILN_4126 GBV_ILN_4249 GBV_ILN_4305 GBV_ILN_4306 GBV_ILN_4307 GBV_ILN_4313 GBV_ILN_4322 GBV_ILN_4323 GBV_ILN_4324 GBV_ILN_4325 GBV_ILN_4338 GBV_ILN_4367 GBV_ILN_4700 AR 10 2022
spelling	10.3389/fped.2022.937250 doi (DE-627)DOAJ02763762X (DE-599)DOAJ479b9ffa5c4e4e27a494259f96bf00b0 DE-627 ger DE-627 rakwb eng RJ1-570 Qiyu Li verfasserin aut Effectiveness and Safety of Cystic Fibrosis Transmembrane Conductance Regulator Modulators in Children With Cystic Fibrosis: A Meta-Analysis 2022 Text txt rdacontent Computermedien c rdamedia Online-Ressource cr rdacarrier Background and AimCystic fibrosis (CF) is a genetic disease that is difficult to treat and caused by dysfunction of the cystic fibrosis transmembrane conductance regulator (CFTR) protein. Small molecules have been used to treat the symptom caused by CFTR mutations by restoring CFTR protein function. However, the data on children with CF are scarce. This meta-analysis aimed to evaluate the effectiveness and safety of this therapy in children diagnosed with CF.Materials and MethodsRelevant studies were identified through searching medical databases before April 1, 2022. The primary outcomes of ppFEV1, lung clearance index2.5 (LCI2.5), sweat chloride concentration (SwCI), and Cystic Fibrosis Questionnaire-Revised (CFQ-R) score were pooled and analyzed. The secondary outcomes were nutritional status (weight, BMI, stature, and their z-score) and adverse events under therapy.ResultsA total of twelve studies were included. Compared with the placebo group, the pooled outcome of the ppFEV1, LCI2.5, SwCI, and CFQ-R score were improved by 7.91 {[95% confidence interval (CI), 3.71–12.12], –1.00 (95% CI, –1.38 to –0.63), –35.22 (95% CI, –55.51 to –14.92), and 4.45 (95% CI, 2.31–6.59), respectively}. Compared with the placebo group, the pooled result of the change in weight was improved by 1.53 (95% CI, 0.42–2.63). All the aforementioned results were also improved in single-arm studies. No clear differences in adverse events were found between CFTR modulator therapy and the placebo group.ConclusionCFTR modulators could improve multiaspect function in children with CF and result in comparable adverse events. CFTR (cystic fibrosis transmembrane conductance regulator) cystic fibrosis – CF corrector potentiator cystic fibrosis transmembrane conductance regulator Pediatrics Siyuan Liu verfasserin aut Xuemei Ma verfasserin aut Jiaping Yu verfasserin aut In Frontiers in Pediatrics Frontiers Media S.A., 2013 10(2022) (DE-627)742738744 (DE-600)2711999-3 22962360 nnns volume:10 year:2022 https://doi.org/10.3389/fped.2022.937250 kostenfrei https://doaj.org/article/479b9ffa5c4e4e27a494259f96bf00b0 kostenfrei https://www.frontiersin.org/articles/10.3389/fped.2022.937250/full kostenfrei https://doaj.org/toc/2296-2360 Journal toc kostenfrei GBV_USEFLAG_A SYSFLAG_A GBV_DOAJ GBV_ILN_11 GBV_ILN_20 GBV_ILN_22 GBV_ILN_23 GBV_ILN_24 GBV_ILN_39 GBV_ILN_40 GBV_ILN_60 GBV_ILN_62 GBV_ILN_63 GBV_ILN_65 GBV_ILN_69 GBV_ILN_73 GBV_ILN_74 GBV_ILN_95 GBV_ILN_105 GBV_ILN_110 GBV_ILN_151 GBV_ILN_161 GBV_ILN_170 GBV_ILN_206 GBV_ILN_213 GBV_ILN_230 GBV_ILN_285 GBV_ILN_293 GBV_ILN_602 GBV_ILN_2003 GBV_ILN_2014 GBV_ILN_4012 GBV_ILN_4037 GBV_ILN_4112 GBV_ILN_4125 GBV_ILN_4126 GBV_ILN_4249 GBV_ILN_4305 GBV_ILN_4306 GBV_ILN_4307 GBV_ILN_4313 GBV_ILN_4322 GBV_ILN_4323 GBV_ILN_4324 GBV_ILN_4325 GBV_ILN_4338 GBV_ILN_4367 GBV_ILN_4700 AR 10 2022
allfields_unstemmed	10.3389/fped.2022.937250 doi (DE-627)DOAJ02763762X (DE-599)DOAJ479b9ffa5c4e4e27a494259f96bf00b0 DE-627 ger DE-627 rakwb eng RJ1-570 Qiyu Li verfasserin aut Effectiveness and Safety of Cystic Fibrosis Transmembrane Conductance Regulator Modulators in Children With Cystic Fibrosis: A Meta-Analysis 2022 Text txt rdacontent Computermedien c rdamedia Online-Ressource cr rdacarrier Background and AimCystic fibrosis (CF) is a genetic disease that is difficult to treat and caused by dysfunction of the cystic fibrosis transmembrane conductance regulator (CFTR) protein. Small molecules have been used to treat the symptom caused by CFTR mutations by restoring CFTR protein function. However, the data on children with CF are scarce. This meta-analysis aimed to evaluate the effectiveness and safety of this therapy in children diagnosed with CF.Materials and MethodsRelevant studies were identified through searching medical databases before April 1, 2022. The primary outcomes of ppFEV1, lung clearance index2.5 (LCI2.5), sweat chloride concentration (SwCI), and Cystic Fibrosis Questionnaire-Revised (CFQ-R) score were pooled and analyzed. The secondary outcomes were nutritional status (weight, BMI, stature, and their z-score) and adverse events under therapy.ResultsA total of twelve studies were included. Compared with the placebo group, the pooled outcome of the ppFEV1, LCI2.5, SwCI, and CFQ-R score were improved by 7.91 {[95% confidence interval (CI), 3.71–12.12], –1.00 (95% CI, –1.38 to –0.63), –35.22 (95% CI, –55.51 to –14.92), and 4.45 (95% CI, 2.31–6.59), respectively}. Compared with the placebo group, the pooled result of the change in weight was improved by 1.53 (95% CI, 0.42–2.63). All the aforementioned results were also improved in single-arm studies. No clear differences in adverse events were found between CFTR modulator therapy and the placebo group.ConclusionCFTR modulators could improve multiaspect function in children with CF and result in comparable adverse events. CFTR (cystic fibrosis transmembrane conductance regulator) cystic fibrosis – CF corrector potentiator cystic fibrosis transmembrane conductance regulator Pediatrics Siyuan Liu verfasserin aut Xuemei Ma verfasserin aut Jiaping Yu verfasserin aut In Frontiers in Pediatrics Frontiers Media S.A., 2013 10(2022) (DE-627)742738744 (DE-600)2711999-3 22962360 nnns volume:10 year:2022 https://doi.org/10.3389/fped.2022.937250 kostenfrei https://doaj.org/article/479b9ffa5c4e4e27a494259f96bf00b0 kostenfrei https://www.frontiersin.org/articles/10.3389/fped.2022.937250/full kostenfrei https://doaj.org/toc/2296-2360 Journal toc kostenfrei GBV_USEFLAG_A SYSFLAG_A GBV_DOAJ GBV_ILN_11 GBV_ILN_20 GBV_ILN_22 GBV_ILN_23 GBV_ILN_24 GBV_ILN_39 GBV_ILN_40 GBV_ILN_60 GBV_ILN_62 GBV_ILN_63 GBV_ILN_65 GBV_ILN_69 GBV_ILN_73 GBV_ILN_74 GBV_ILN_95 GBV_ILN_105 GBV_ILN_110 GBV_ILN_151 GBV_ILN_161 GBV_ILN_170 GBV_ILN_206 GBV_ILN_213 GBV_ILN_230 GBV_ILN_285 GBV_ILN_293 GBV_ILN_602 GBV_ILN_2003 GBV_ILN_2014 GBV_ILN_4012 GBV_ILN_4037 GBV_ILN_4112 GBV_ILN_4125 GBV_ILN_4126 GBV_ILN_4249 GBV_ILN_4305 GBV_ILN_4306 GBV_ILN_4307 GBV_ILN_4313 GBV_ILN_4322 GBV_ILN_4323 GBV_ILN_4324 GBV_ILN_4325 GBV_ILN_4338 GBV_ILN_4367 GBV_ILN_4700 AR 10 2022
allfieldsGer	10.3389/fped.2022.937250 doi (DE-627)DOAJ02763762X (DE-599)DOAJ479b9ffa5c4e4e27a494259f96bf00b0 DE-627 ger DE-627 rakwb eng RJ1-570 Qiyu Li verfasserin aut Effectiveness and Safety of Cystic Fibrosis Transmembrane Conductance Regulator Modulators in Children With Cystic Fibrosis: A Meta-Analysis 2022 Text txt rdacontent Computermedien c rdamedia Online-Ressource cr rdacarrier Background and AimCystic fibrosis (CF) is a genetic disease that is difficult to treat and caused by dysfunction of the cystic fibrosis transmembrane conductance regulator (CFTR) protein. Small molecules have been used to treat the symptom caused by CFTR mutations by restoring CFTR protein function. However, the data on children with CF are scarce. This meta-analysis aimed to evaluate the effectiveness and safety of this therapy in children diagnosed with CF.Materials and MethodsRelevant studies were identified through searching medical databases before April 1, 2022. The primary outcomes of ppFEV1, lung clearance index2.5 (LCI2.5), sweat chloride concentration (SwCI), and Cystic Fibrosis Questionnaire-Revised (CFQ-R) score were pooled and analyzed. The secondary outcomes were nutritional status (weight, BMI, stature, and their z-score) and adverse events under therapy.ResultsA total of twelve studies were included. Compared with the placebo group, the pooled outcome of the ppFEV1, LCI2.5, SwCI, and CFQ-R score were improved by 7.91 {[95% confidence interval (CI), 3.71–12.12], –1.00 (95% CI, –1.38 to –0.63), –35.22 (95% CI, –55.51 to –14.92), and 4.45 (95% CI, 2.31–6.59), respectively}. Compared with the placebo group, the pooled result of the change in weight was improved by 1.53 (95% CI, 0.42–2.63). All the aforementioned results were also improved in single-arm studies. No clear differences in adverse events were found between CFTR modulator therapy and the placebo group.ConclusionCFTR modulators could improve multiaspect function in children with CF and result in comparable adverse events. CFTR (cystic fibrosis transmembrane conductance regulator) cystic fibrosis – CF corrector potentiator cystic fibrosis transmembrane conductance regulator Pediatrics Siyuan Liu verfasserin aut Xuemei Ma verfasserin aut Jiaping Yu verfasserin aut In Frontiers in Pediatrics Frontiers Media S.A., 2013 10(2022) (DE-627)742738744 (DE-600)2711999-3 22962360 nnns volume:10 year:2022 https://doi.org/10.3389/fped.2022.937250 kostenfrei https://doaj.org/article/479b9ffa5c4e4e27a494259f96bf00b0 kostenfrei https://www.frontiersin.org/articles/10.3389/fped.2022.937250/full kostenfrei https://doaj.org/toc/2296-2360 Journal toc kostenfrei GBV_USEFLAG_A SYSFLAG_A GBV_DOAJ GBV_ILN_11 GBV_ILN_20 GBV_ILN_22 GBV_ILN_23 GBV_ILN_24 GBV_ILN_39 GBV_ILN_40 GBV_ILN_60 GBV_ILN_62 GBV_ILN_63 GBV_ILN_65 GBV_ILN_69 GBV_ILN_73 GBV_ILN_74 GBV_ILN_95 GBV_ILN_105 GBV_ILN_110 GBV_ILN_151 GBV_ILN_161 GBV_ILN_170 GBV_ILN_206 GBV_ILN_213 GBV_ILN_230 GBV_ILN_285 GBV_ILN_293 GBV_ILN_602 GBV_ILN_2003 GBV_ILN_2014 GBV_ILN_4012 GBV_ILN_4037 GBV_ILN_4112 GBV_ILN_4125 GBV_ILN_4126 GBV_ILN_4249 GBV_ILN_4305 GBV_ILN_4306 GBV_ILN_4307 GBV_ILN_4313 GBV_ILN_4322 GBV_ILN_4323 GBV_ILN_4324 GBV_ILN_4325 GBV_ILN_4338 GBV_ILN_4367 GBV_ILN_4700 AR 10 2022
allfieldsSound	10.3389/fped.2022.937250 doi (DE-627)DOAJ02763762X (DE-599)DOAJ479b9ffa5c4e4e27a494259f96bf00b0 DE-627 ger DE-627 rakwb eng RJ1-570 Qiyu Li verfasserin aut Effectiveness and Safety of Cystic Fibrosis Transmembrane Conductance Regulator Modulators in Children With Cystic Fibrosis: A Meta-Analysis 2022 Text txt rdacontent Computermedien c rdamedia Online-Ressource cr rdacarrier Background and AimCystic fibrosis (CF) is a genetic disease that is difficult to treat and caused by dysfunction of the cystic fibrosis transmembrane conductance regulator (CFTR) protein. Small molecules have been used to treat the symptom caused by CFTR mutations by restoring CFTR protein function. However, the data on children with CF are scarce. This meta-analysis aimed to evaluate the effectiveness and safety of this therapy in children diagnosed with CF.Materials and MethodsRelevant studies were identified through searching medical databases before April 1, 2022. The primary outcomes of ppFEV1, lung clearance index2.5 (LCI2.5), sweat chloride concentration (SwCI), and Cystic Fibrosis Questionnaire-Revised (CFQ-R) score were pooled and analyzed. The secondary outcomes were nutritional status (weight, BMI, stature, and their z-score) and adverse events under therapy.ResultsA total of twelve studies were included. Compared with the placebo group, the pooled outcome of the ppFEV1, LCI2.5, SwCI, and CFQ-R score were improved by 7.91 {[95% confidence interval (CI), 3.71–12.12], –1.00 (95% CI, –1.38 to –0.63), –35.22 (95% CI, –55.51 to –14.92), and 4.45 (95% CI, 2.31–6.59), respectively}. Compared with the placebo group, the pooled result of the change in weight was improved by 1.53 (95% CI, 0.42–2.63). All the aforementioned results were also improved in single-arm studies. No clear differences in adverse events were found between CFTR modulator therapy and the placebo group.ConclusionCFTR modulators could improve multiaspect function in children with CF and result in comparable adverse events. CFTR (cystic fibrosis transmembrane conductance regulator) cystic fibrosis – CF corrector potentiator cystic fibrosis transmembrane conductance regulator Pediatrics Siyuan Liu verfasserin aut Xuemei Ma verfasserin aut Jiaping Yu verfasserin aut In Frontiers in Pediatrics Frontiers Media S.A., 2013 10(2022) (DE-627)742738744 (DE-600)2711999-3 22962360 nnns volume:10 year:2022 https://doi.org/10.3389/fped.2022.937250 kostenfrei https://doaj.org/article/479b9ffa5c4e4e27a494259f96bf00b0 kostenfrei https://www.frontiersin.org/articles/10.3389/fped.2022.937250/full kostenfrei https://doaj.org/toc/2296-2360 Journal toc kostenfrei GBV_USEFLAG_A SYSFLAG_A GBV_DOAJ GBV_ILN_11 GBV_ILN_20 GBV_ILN_22 GBV_ILN_23 GBV_ILN_24 GBV_ILN_39 GBV_ILN_40 GBV_ILN_60 GBV_ILN_62 GBV_ILN_63 GBV_ILN_65 GBV_ILN_69 GBV_ILN_73 GBV_ILN_74 GBV_ILN_95 GBV_ILN_105 GBV_ILN_110 GBV_ILN_151 GBV_ILN_161 GBV_ILN_170 GBV_ILN_206 GBV_ILN_213 GBV_ILN_230 GBV_ILN_285 GBV_ILN_293 GBV_ILN_602 GBV_ILN_2003 GBV_ILN_2014 GBV_ILN_4012 GBV_ILN_4037 GBV_ILN_4112 GBV_ILN_4125 GBV_ILN_4126 GBV_ILN_4249 GBV_ILN_4305 GBV_ILN_4306 GBV_ILN_4307 GBV_ILN_4313 GBV_ILN_4322 GBV_ILN_4323 GBV_ILN_4324 GBV_ILN_4325 GBV_ILN_4338 GBV_ILN_4367 GBV_ILN_4700 AR 10 2022
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Small molecules have been used to treat the symptom caused by CFTR mutations by restoring CFTR protein function. However, the data on children with CF are scarce. This meta-analysis aimed to evaluate the effectiveness and safety of this therapy in children diagnosed with CF.Materials and MethodsRelevant studies were identified through searching medical databases before April 1, 2022. The primary outcomes of ppFEV1, lung clearance index2.5 (LCI2.5), sweat chloride concentration (SwCI), and Cystic Fibrosis Questionnaire-Revised (CFQ-R) score were pooled and analyzed. The secondary outcomes were nutritional status (weight, BMI, stature, and their z-score) and adverse events under therapy.ResultsA total of twelve studies were included. Compared with the placebo group, the pooled outcome of the ppFEV1, LCI2.5, SwCI, and CFQ-R score were improved by 7.91 {[95% confidence interval (CI), 3.71–12.12], –1.00 (95% CI, –1.38 to –0.63), –35.22 (95% CI, –55.51 to –14.92), and 4.45 (95% CI, 2.31–6.59), respectively}. Compared with the placebo group, the pooled result of the change in weight was improved by 1.53 (95% CI, 0.42–2.63). All the aforementioned results were also improved in single-arm studies. No clear differences in adverse events were found between CFTR modulator therapy and the placebo group.ConclusionCFTR modulators could improve multiaspect function in children with CF and result in comparable adverse events.</subfield></datafield><datafield tag="650" ind1=" " ind2="4"><subfield code="a">CFTR (cystic fibrosis transmembrane conductance regulator)</subfield></datafield><datafield tag="650" ind1=" " ind2="4"><subfield code="a">cystic fibrosis – CF</subfield></datafield><datafield tag="650" ind1=" " ind2="4"><subfield code="a">corrector</subfield></datafield><datafield tag="650" ind1=" " ind2="4"><subfield code="a">potentiator</subfield></datafield><datafield tag="650" ind1=" " ind2="4"><subfield code="a">cystic fibrosis transmembrane conductance regulator</subfield></datafield><datafield tag="653" ind1=" " ind2="0"><subfield code="a">Pediatrics</subfield></datafield><datafield tag="700" ind1="0" ind2=" "><subfield code="a">Siyuan Liu</subfield><subfield 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callnumber-first	R - Medicine
author	Qiyu Li
spellingShingle	Qiyu Li misc RJ1-570 misc CFTR (cystic fibrosis transmembrane conductance regulator) misc cystic fibrosis – CF misc corrector misc potentiator misc cystic fibrosis transmembrane conductance regulator misc Pediatrics Effectiveness and Safety of Cystic Fibrosis Transmembrane Conductance Regulator Modulators in Children With Cystic Fibrosis: A Meta-Analysis
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topic_title	RJ1-570 Effectiveness and Safety of Cystic Fibrosis Transmembrane Conductance Regulator Modulators in Children With Cystic Fibrosis: A Meta-Analysis CFTR (cystic fibrosis transmembrane conductance regulator) cystic fibrosis – CF corrector potentiator cystic fibrosis transmembrane conductance regulator
topic	misc RJ1-570 misc CFTR (cystic fibrosis transmembrane conductance regulator) misc cystic fibrosis – CF misc corrector misc potentiator misc cystic fibrosis transmembrane conductance regulator misc Pediatrics
topic_unstemmed	misc RJ1-570 misc CFTR (cystic fibrosis transmembrane conductance regulator) misc cystic fibrosis – CF misc corrector misc potentiator misc cystic fibrosis transmembrane conductance regulator misc Pediatrics
topic_browse	misc RJ1-570 misc CFTR (cystic fibrosis transmembrane conductance regulator) misc cystic fibrosis – CF misc corrector misc potentiator misc cystic fibrosis transmembrane conductance regulator misc Pediatrics
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title	Effectiveness and Safety of Cystic Fibrosis Transmembrane Conductance Regulator Modulators in Children With Cystic Fibrosis: A Meta-Analysis
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title_full	Effectiveness and Safety of Cystic Fibrosis Transmembrane Conductance Regulator Modulators in Children With Cystic Fibrosis: A Meta-Analysis
author_sort	Qiyu Li
journal	Frontiers in Pediatrics
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author_browse	Qiyu Li Siyuan Liu Xuemei Ma Jiaping Yu
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doi_str_mv	10.3389/fped.2022.937250
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title_sort	effectiveness and safety of cystic fibrosis transmembrane conductance regulator modulators in children with cystic fibrosis: a meta-analysis
callnumber	RJ1-570
title_auth	Effectiveness and Safety of Cystic Fibrosis Transmembrane Conductance Regulator Modulators in Children With Cystic Fibrosis: A Meta-Analysis
abstract	Background and AimCystic fibrosis (CF) is a genetic disease that is difficult to treat and caused by dysfunction of the cystic fibrosis transmembrane conductance regulator (CFTR) protein. Small molecules have been used to treat the symptom caused by CFTR mutations by restoring CFTR protein function. However, the data on children with CF are scarce. This meta-analysis aimed to evaluate the effectiveness and safety of this therapy in children diagnosed with CF.Materials and MethodsRelevant studies were identified through searching medical databases before April 1, 2022. The primary outcomes of ppFEV1, lung clearance index2.5 (LCI2.5), sweat chloride concentration (SwCI), and Cystic Fibrosis Questionnaire-Revised (CFQ-R) score were pooled and analyzed. The secondary outcomes were nutritional status (weight, BMI, stature, and their z-score) and adverse events under therapy.ResultsA total of twelve studies were included. Compared with the placebo group, the pooled outcome of the ppFEV1, LCI2.5, SwCI, and CFQ-R score were improved by 7.91 {[95% confidence interval (CI), 3.71–12.12], –1.00 (95% CI, –1.38 to –0.63), –35.22 (95% CI, –55.51 to –14.92), and 4.45 (95% CI, 2.31–6.59), respectively}. Compared with the placebo group, the pooled result of the change in weight was improved by 1.53 (95% CI, 0.42–2.63). All the aforementioned results were also improved in single-arm studies. No clear differences in adverse events were found between CFTR modulator therapy and the placebo group.ConclusionCFTR modulators could improve multiaspect function in children with CF and result in comparable adverse events.
abstractGer	Background and AimCystic fibrosis (CF) is a genetic disease that is difficult to treat and caused by dysfunction of the cystic fibrosis transmembrane conductance regulator (CFTR) protein. Small molecules have been used to treat the symptom caused by CFTR mutations by restoring CFTR protein function. However, the data on children with CF are scarce. This meta-analysis aimed to evaluate the effectiveness and safety of this therapy in children diagnosed with CF.Materials and MethodsRelevant studies were identified through searching medical databases before April 1, 2022. The primary outcomes of ppFEV1, lung clearance index2.5 (LCI2.5), sweat chloride concentration (SwCI), and Cystic Fibrosis Questionnaire-Revised (CFQ-R) score were pooled and analyzed. The secondary outcomes were nutritional status (weight, BMI, stature, and their z-score) and adverse events under therapy.ResultsA total of twelve studies were included. Compared with the placebo group, the pooled outcome of the ppFEV1, LCI2.5, SwCI, and CFQ-R score were improved by 7.91 {[95% confidence interval (CI), 3.71–12.12], –1.00 (95% CI, –1.38 to –0.63), –35.22 (95% CI, –55.51 to –14.92), and 4.45 (95% CI, 2.31–6.59), respectively}. Compared with the placebo group, the pooled result of the change in weight was improved by 1.53 (95% CI, 0.42–2.63). All the aforementioned results were also improved in single-arm studies. No clear differences in adverse events were found between CFTR modulator therapy and the placebo group.ConclusionCFTR modulators could improve multiaspect function in children with CF and result in comparable adverse events.
abstract_unstemmed	Background and AimCystic fibrosis (CF) is a genetic disease that is difficult to treat and caused by dysfunction of the cystic fibrosis transmembrane conductance regulator (CFTR) protein. Small molecules have been used to treat the symptom caused by CFTR mutations by restoring CFTR protein function. However, the data on children with CF are scarce. This meta-analysis aimed to evaluate the effectiveness and safety of this therapy in children diagnosed with CF.Materials and MethodsRelevant studies were identified through searching medical databases before April 1, 2022. The primary outcomes of ppFEV1, lung clearance index2.5 (LCI2.5), sweat chloride concentration (SwCI), and Cystic Fibrosis Questionnaire-Revised (CFQ-R) score were pooled and analyzed. The secondary outcomes were nutritional status (weight, BMI, stature, and their z-score) and adverse events under therapy.ResultsA total of twelve studies were included. Compared with the placebo group, the pooled outcome of the ppFEV1, LCI2.5, SwCI, and CFQ-R score were improved by 7.91 {[95% confidence interval (CI), 3.71–12.12], –1.00 (95% CI, –1.38 to –0.63), –35.22 (95% CI, –55.51 to –14.92), and 4.45 (95% CI, 2.31–6.59), respectively}. Compared with the placebo group, the pooled result of the change in weight was improved by 1.53 (95% CI, 0.42–2.63). All the aforementioned results were also improved in single-arm studies. No clear differences in adverse events were found between CFTR modulator therapy and the placebo group.ConclusionCFTR modulators could improve multiaspect function in children with CF and result in comparable adverse events.
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title_short	Effectiveness and Safety of Cystic Fibrosis Transmembrane Conductance Regulator Modulators in Children With Cystic Fibrosis: A Meta-Analysis
url	https://doi.org/10.3389/fped.2022.937250 https://doaj.org/article/479b9ffa5c4e4e27a494259f96bf00b0 https://www.frontiersin.org/articles/10.3389/fped.2022.937250/full https://doaj.org/toc/2296-2360
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up_date	2024-07-04T02:25:09.742Z
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Small molecules have been used to treat the symptom caused by CFTR mutations by restoring CFTR protein function. However, the data on children with CF are scarce. This meta-analysis aimed to evaluate the effectiveness and safety of this therapy in children diagnosed with CF.Materials and MethodsRelevant studies were identified through searching medical databases before April 1, 2022. The primary outcomes of ppFEV1, lung clearance index2.5 (LCI2.5), sweat chloride concentration (SwCI), and Cystic Fibrosis Questionnaire-Revised (CFQ-R) score were pooled and analyzed. The secondary outcomes were nutritional status (weight, BMI, stature, and their z-score) and adverse events under therapy.ResultsA total of twelve studies were included. Compared with the placebo group, the pooled outcome of the ppFEV1, LCI2.5, SwCI, and CFQ-R score were improved by 7.91 {[95% confidence interval (CI), 3.71–12.12], –1.00 (95% CI, –1.38 to –0.63), –35.22 (95% CI, –55.51 to –14.92), and 4.45 (95% CI, 2.31–6.59), respectively}. Compared with the placebo group, the pooled result of the change in weight was improved by 1.53 (95% CI, 0.42–2.63). All the aforementioned results were also improved in single-arm studies. No clear differences in adverse events were found between CFTR modulator therapy and the placebo group.ConclusionCFTR modulators could improve multiaspect function in children with CF and result in comparable adverse events.</subfield></datafield><datafield tag="650" ind1=" " ind2="4"><subfield code="a">CFTR (cystic fibrosis transmembrane conductance regulator)</subfield></datafield><datafield tag="650" ind1=" " ind2="4"><subfield code="a">cystic fibrosis – CF</subfield></datafield><datafield tag="650" ind1=" " ind2="4"><subfield code="a">corrector</subfield></datafield><datafield tag="650" ind1=" " ind2="4"><subfield code="a">potentiator</subfield></datafield><datafield tag="650" ind1=" " ind2="4"><subfield code="a">cystic fibrosis transmembrane conductance regulator</subfield></datafield><datafield tag="653" ind1=" " ind2="0"><subfield code="a">Pediatrics</subfield></datafield><datafield tag="700" ind1="0" ind2=" "><subfield code="a">Siyuan Liu</subfield><subfield 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