Baseline-dependent improvement in CF studies, plausibility of bias
It has been commonly reported that therapeutic treatments in cystic fibrosis (CF) have ceiling effects, such that their efficacy is diminished for persons with high pre-treatment health (Montgomery et al., 2012 and Newsome et al., 2019). Floor effects have also been reported where decline is of lowe...
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| Published in: | Contemporary clinical trials communications Vol. 42; p. 101378 |
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| Abstract | It has been commonly reported that therapeutic treatments in cystic fibrosis (CF) have ceiling effects, such that their efficacy is diminished for persons with high pre-treatment health (Montgomery et al., 2012 and Newsome et al., 2019). Floor effects have also been reported where decline is of lower magnitude in those with below-average pre-treatment health (Harun et al., 2016; Konstan et al., 2012 and Szczesniak et al., 2017). When measurement error is present, the statistical literature has warned of exaggerated or spurious associations between pre-treatment measures and subsequent change (Chambless and Davis, 2003 and Yanez et al., 1998). Measurement error, equivalently described as day-to-day variation, has been described to occur in CF outcome measurements such as forced expiratory volume in 1 s taken by spirometry (FEV1pp) (Magaret et al., 2024; Stanojevic et al., 2020 and Thornton et al., 2023).
We conducted a simulation study to assess the potential for spurious floor or ceiling effects in studies of CF therapeutics. We considered uncontrolled or single-arm studies, and evaluated estimated association between pre-treatment FEV1pp and treatment-induced change: post-versus pre-treatment.
When day-to-day variation was present in FEV1pp, at levels equivalent to those reported in large studies measuring spirometry both at home and in clinic, naive analytic approaches found spurious associations of change with baseline (Paynter et al., 2022 and Saiman et al., 2003). Type I error ranged from 31.9% to 98.3% for day-to-day variation as high as 3% to 15% relative to biological variation. Incorporating known day-to-day variation, the regression calibration approach corrected bias and controlled type I error (Chambless and Davis, 2003).
Exaggerated ceiling effects are possible. Further studies could provide meaningful confirmation of ceiling effects in CF, perhaps reducing day-to-day variation by incorporating multiple pre- and post-treatment measurements. |
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| AbstractList | It has been commonly reported that therapeutic treatments in cystic fibrosis (CF) have ceiling effects, such that their efficacy is diminished for persons with high pre-treatment health (Montgomery et al., 2012 and Newsome et al., 2019). Floor effects have also been reported where decline is of lower magnitude in those with below-average pre-treatment health (Harun et al., 2016; Konstan et al., 2012 and Szczesniak et al., 2017). When measurement error is present, the statistical literature has warned of exaggerated or spurious associations between pre-treatment measures and subsequent change (Chambless and Davis, 2003 and Yanez et al., 1998). Measurement error, equivalently described as day-to-day variation, has been described to occur in CF outcome measurements such as forced expiratory volume in 1 s taken by spirometry (FEV1pp) (Magaret et al., 2024; Stanojevic et al., 2020 and Thornton et al., 2023).
We conducted a simulation study to assess the potential for spurious floor or ceiling effects in studies of CF therapeutics. We considered uncontrolled or single-arm studies, and evaluated estimated association between pre-treatment FEV1pp and treatment-induced change: post-versus pre-treatment.
When day-to-day variation was present in FEV1pp, at levels equivalent to those reported in large studies measuring spirometry both at home and in clinic, naive analytic approaches found spurious associations of change with baseline (Paynter et al., 2022 and Saiman et al., 2003). Type I error ranged from 31.9% to 98.3% for day-to-day variation as high as 3% to 15% relative to biological variation. Incorporating known day-to-day variation, the regression calibration approach corrected bias and controlled type I error (Chambless and Davis, 2003).
Exaggerated ceiling effects are possible. Further studies could provide meaningful confirmation of ceiling effects in CF, perhaps reducing day-to-day variation by incorporating multiple pre- and post-treatment measurements. It has been commonly reported that therapeutic treatments in cystic fibrosis (CF) have ceiling effects, such that their efficacy is diminished for persons with high pre-treatment health (Montgomery et al., 2012 and Newsome et al., 2019). Floor effects have also been reported where decline is of lower magnitude in those with below-average pre-treatment health (Harun et al., 2016; Konstan et al., 2012 and Szczesniak et al., 2017). When measurement error is present, the statistical literature has warned of exaggerated or spurious associations between pre-treatment measures and subsequent change (Chambless and Davis, 2003 and Yanez et al., 1998). Measurement error, equivalently described as day-to-day variation, has been described to occur in CF outcome measurements such as forced expiratory volume in 1 s taken by spirometry (FEV pp) (Magaret et al., 2024; Stanojevic et al., 2020 and Thornton et al., 2023). We conducted a simulation study to assess the potential for spurious floor or ceiling effects in studies of CF therapeutics. We considered uncontrolled or single-arm studies, and evaluated estimated association between pre-treatment FEV pp and treatment-induced change: post-versus pre-treatment. When day-to-day variation was present in FEV pp, at levels equivalent to those reported in large studies measuring spirometry both at home and in clinic, naive analytic approaches found spurious associations of change with baseline (Paynter et al., 2022 and Saiman et al., 2003). Type I error ranged from 31.9% to 98.3% for day-to-day variation as high as 3% to 15% relative to biological variation. Incorporating known day-to-day variation, the regression calibration approach corrected bias and controlled type I error (Chambless and Davis, 2003). Exaggerated ceiling effects are possible. Further studies could provide meaningful confirmation of ceiling effects in CF, perhaps reducing day-to-day variation by incorporating multiple pre- and post-treatment measurements. AbstractBackground:It has been commonly reported that therapeutic treatments in cystic fibrosis (CF) have ceiling effects, such that their efficacy is diminished for persons with high pre-treatment health (Montgomery et al., 2012 and Newsome et al., 2019). Floor effects have also been reported where decline is of lower magnitude in those with below-average pre-treatment health (Harun et al., 2016; Konstan et al., 2012 and Szczesniak et al., 2017). When measurement error is present, the statistical literature has warned of exaggerated or spurious associations between pre-treatment measures and subsequent change (Chambless and Davis, 2003 and Yanez et al., 1998). Measurement error, equivalently described as day-to-day variation, has been described to occur in CF outcome measurements such as forced expiratory volume in 1 s taken by spirometry (FEV 1pp) (Magaret et al., 2024; Stanojevic et al., 2020 and Thornton et al., 2023). Methods:We conducted a simulation study to assess the potential for spurious floor or ceiling effects in studies of CF therapeutics. We considered uncontrolled or single-arm studies, and evaluated estimated association between pre-treatment FEV 1pp and treatment-induced change: post-versus pre-treatment. Results:When day-to-day variation was present in FEV 1pp, at levels equivalent to those reported in large studies measuring spirometry both at home and in clinic, naive analytic approaches found spurious associations of change with baseline (Paynter et al., 2022 and Saiman et al., 2003). Type I error ranged from 31.9% to 98.3% for day-to-day variation as high as 3% to 15% relative to biological variation. Incorporating known day-to-day variation, the regression calibration approach corrected bias and controlled type I error (Chambless and Davis, 2003). Conclusion:Exaggerated ceiling effects are possible. Further studies could provide meaningful confirmation of ceiling effects in CF, perhaps reducing day-to-day variation by incorporating multiple pre- and post-treatment measurements. It has been commonly reported that therapeutic treatments in cystic fibrosis (CF) have ceiling effects, such that their efficacy is diminished for persons with high pre-treatment health (Montgomery et al., 2012 and Newsome et al., 2019). Floor effects have also been reported where decline is of lower magnitude in those with below-average pre-treatment health (Harun et al., 2016; Konstan et al., 2012 and Szczesniak et al., 2017). When measurement error is present, the statistical literature has warned of exaggerated or spurious associations between pre-treatment measures and subsequent change (Chambless and Davis, 2003 and Yanez et al., 1998). Measurement error, equivalently described as day-to-day variation, has been described to occur in CF outcome measurements such as forced expiratory volume in 1 s taken by spirometry (FEV 1 pp) (Magaret et al., 2024; Stanojevic et al., 2020 and Thornton et al., 2023).BackgroundIt has been commonly reported that therapeutic treatments in cystic fibrosis (CF) have ceiling effects, such that their efficacy is diminished for persons with high pre-treatment health (Montgomery et al., 2012 and Newsome et al., 2019). Floor effects have also been reported where decline is of lower magnitude in those with below-average pre-treatment health (Harun et al., 2016; Konstan et al., 2012 and Szczesniak et al., 2017). When measurement error is present, the statistical literature has warned of exaggerated or spurious associations between pre-treatment measures and subsequent change (Chambless and Davis, 2003 and Yanez et al., 1998). Measurement error, equivalently described as day-to-day variation, has been described to occur in CF outcome measurements such as forced expiratory volume in 1 s taken by spirometry (FEV 1 pp) (Magaret et al., 2024; Stanojevic et al., 2020 and Thornton et al., 2023).We conducted a simulation study to assess the potential for spurious floor or ceiling effects in studies of CF therapeutics. We considered uncontrolled or single-arm studies, and evaluated estimated association between pre-treatment FEV 1 pp and treatment-induced change: post-versus pre-treatment.MethodsWe conducted a simulation study to assess the potential for spurious floor or ceiling effects in studies of CF therapeutics. We considered uncontrolled or single-arm studies, and evaluated estimated association between pre-treatment FEV 1 pp and treatment-induced change: post-versus pre-treatment.When day-to-day variation was present in FEV 1 pp, at levels equivalent to those reported in large studies measuring spirometry both at home and in clinic, naive analytic approaches found spurious associations of change with baseline (Paynter et al., 2022 and Saiman et al., 2003). Type I error ranged from 31.9% to 98.3% for day-to-day variation as high as 3% to 15% relative to biological variation. Incorporating known day-to-day variation, the regression calibration approach corrected bias and controlled type I error (Chambless and Davis, 2003).ResultsWhen day-to-day variation was present in FEV 1 pp, at levels equivalent to those reported in large studies measuring spirometry both at home and in clinic, naive analytic approaches found spurious associations of change with baseline (Paynter et al., 2022 and Saiman et al., 2003). Type I error ranged from 31.9% to 98.3% for day-to-day variation as high as 3% to 15% relative to biological variation. Incorporating known day-to-day variation, the regression calibration approach corrected bias and controlled type I error (Chambless and Davis, 2003).Exaggerated ceiling effects are possible. Further studies could provide meaningful confirmation of ceiling effects in CF, perhaps reducing day-to-day variation by incorporating multiple pre- and post-treatment measurements.ConclusionExaggerated ceiling effects are possible. Further studies could provide meaningful confirmation of ceiling effects in CF, perhaps reducing day-to-day variation by incorporating multiple pre- and post-treatment measurements. Background:: It has been commonly reported that therapeutic treatments in cystic fibrosis (CF) have ceiling effects, such that their efficacy is diminished for persons with high pre-treatment health (Montgomery et al., 2012 and Newsome et al., 2019). Floor effects have also been reported where decline is of lower magnitude in those with below-average pre-treatment health (Harun et al., 2016; Konstan et al., 2012 and Szczesniak et al., 2017). When measurement error is present, the statistical literature has warned of exaggerated or spurious associations between pre-treatment measures and subsequent change (Chambless and Davis, 2003 and Yanez et al., 1998). Measurement error, equivalently described as day-to-day variation, has been described to occur in CF outcome measurements such as forced expiratory volume in 1 s taken by spirometry (FEV1pp) (Magaret et al., 2024; Stanojevic et al., 2020 and Thornton et al., 2023). Methods:: We conducted a simulation study to assess the potential for spurious floor or ceiling effects in studies of CF therapeutics. We considered uncontrolled or single-arm studies, and evaluated estimated association between pre-treatment FEV1pp and treatment-induced change: post-versus pre-treatment. Results:: When day-to-day variation was present in FEV1pp, at levels equivalent to those reported in large studies measuring spirometry both at home and in clinic, naive analytic approaches found spurious associations of change with baseline (Paynter et al., 2022 and Saiman et al., 2003). Type I error ranged from 31.9% to 98.3% for day-to-day variation as high as 3% to 15% relative to biological variation. Incorporating known day-to-day variation, the regression calibration approach corrected bias and controlled type I error (Chambless and Davis, 2003). Conclusion:: Exaggerated ceiling effects are possible. Further studies could provide meaningful confirmation of ceiling effects in CF, perhaps reducing day-to-day variation by incorporating multiple pre- and post-treatment measurements. |
| ArticleNumber | 101378 |
| Author | Heltshe, Sonya L. Graham, Ellen Magaret, Amalia S. |
| Author_xml | – sequence: 1 givenname: Ellen orcidid: 0000-0002-3238-3769 surname: Graham fullname: Graham, Ellen email: graham18@uw.edu organization: MS University of Washington, Department of Biostatistics, United States of America – sequence: 2 givenname: Sonya L. surname: Heltshe fullname: Heltshe, Sonya L. email: sonya.heltshe@seattlechildrens.org organization: University of Washington, Departments of Pediatrics and Biostatistics Seattle Children’s Research Institute, Center for Respiratory Biology and Therapeutics, United States of America – sequence: 3 givenname: Amalia S. orcidid: 0000-0002-1221-3746 surname: Magaret fullname: Magaret, Amalia S. email: amag@uw.edu organization: University of Washington, Departments of Pediatrics and Biostatistics Seattle Children’s Research Institute, Center for Respiratory Biology and Therapeutics, United States of America |
| BackLink | https://www.ncbi.nlm.nih.gov/pubmed/39678155$$D View this record in MEDLINE/PubMed |
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| Cites_doi | 10.1002/(SICI)1097-0258(19981130)17:22<2597::AID-SIM940>3.0.CO;2-G 10.1056/NEJMoa1908639 10.1002/sim.5910 10.1002/ppul.23227 10.1002/sim.3725 10.1016/S1047-2797(01)00280-0 10.1164/rccm.201404-0703OC 10.1056/NEJMoa1105185 10.1001/jama.290.13.1749 10.1016/S0140-6736(19)32597-8 10.1002/sim.1352 10.1164/rccm.201608-1754OC 10.1016/S2213-2600(20)30517-8 10.1164/rccm.201610-2172OC 10.1016/j.jcf.2012.03.009 10.1371/journal.pone.0152428 10.1016/j.jcf.2024.07.005 10.1093/aje/kwi187 10.1164/rccm.202102-0509OC 10.1002/sim.3639 10.1016/j.jcf.2017.01.002 10.1002/sim.2682 10.1164/rccm.201710-2046LE 10.1136/thoraxjnl-2020-214817 10.1016/j.jcf.2018.08.004 10.1037/h0025105 10.1016/j.jcf.2021.08.013 10.1056/NEJMoa1409547 10.1089/jamp.2011.0911 |
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| References | Szczesniak, Heltshe, Stanojevic, Mayer-Hamblett (b4) 2017; 16 Glymour, Weuve, Berkman, Kawachi, Robins (b31) 2005; 162 Wainwright, Elborn, Ramsey, Marigowda, Huang, Cipolli, Colombo, Davies, De Boeck, Flume, Konstan, McColley, McCoy, McKone, Munck, Ratjen, Rowe, Waltz, Boyle (b7) 2015; 373 Stanojevic, Filipow, Ratjen (b19) 2020; 75 Newsome, Daniel, Carr, Bilton, Keogh (b5) 2019; 18 Thornton, Magaret, Carmody, Kalikin, Simon, LiPuma, Caverly (b20) 2023 Yanez, Kronmal, Shemanski (b14) 1998; 17 Zemanick, Taylor-Cousar, Davies, Gibson, Mall, McKone, McNally, Ramsey, Rayment, Rowe, Tullis, Ahluwalia, Chu, Ho, Moskowitz, Noel, Tian, Waltz, Weinstock, Xuan, Wainwright, McColley (b12) 2021; 203 Montgomery, Abuan, Yeager (b2) 2012; 25 Therapeutics Development Network Coordinating Center Lord (b28) 1967; 68 Rowe, Heltshe, Gonska, Donaldson, Borowitz, Gelfond, Sagel, Khan, Mayer-Hamblett, Van Dalfsen, Joseloff, Ramsey, GOAL Investigators of the Cystic Fibrosis Foundation Therapeutics Development Network (b17) 2014; 190 Braun, Held, Ledergerber, Swiss HIV Cohort Study (b27) 2014; 33 Senn (b29) 2006; 25 Magaret, Graham, Caverly, Cromwell, Paynter, Rosenfeld, Thornton, Goss (b13) 2024; 23 Paynter, Khan, Heltshe, Goss, Lechtzin, Hamblett (b22) 2022; 21 Heijerman, McKone, Downey, Van Braeckel, Rowe, Tullis, Mall, Welter, Ramsey, McKee, Marigowda, Moskowitz, Waltz, Sosnay, Simard, Ahluwalia, Xuan, Zhang, Taylor-Cousar, McCoy, VX17-445-103 Trial Group (b9) 2019; 394 Chambless, Davis (b15) 2003; 22 Konstan, Wagener, Vandevanter, Pasta, Yegin, Rasouliyan, Morgan (b1) 2012; 11 Jones, Lonergan, Henley, Pearson, Hattersley, Shields (b18) 2016; 11 Liu, Lu, Mogg, Mallick, Mehrotra (b30) 2009; 28 Heltshe, Rowe, Skalland, Baines, Jain (b32) 2018; 197 Saiman, Marshall, Mayer-Hamblett, Burns, Quittner, Cibene, Coquillette, Fieberg, Accurso, Campbell (b21) 2003; 290 . Harun, Wainwright, Klein, Hennig (b3) 2016; 20 Hamblett (b25) 2022 Ramsey, Davies, McElvaney, Tullis, Bell, Dřevínek, Griese, McKone, Wainwright, Konstan, Moss, Ratjen, Sermet-Gaudelus, Rowe, Dong, Rodriguez, Yen, Ordoñez, Elborn (b6) 2011; 365 Rosenfeld, VanDevanter, Ren, Elkin, Pasta, Konstan, Morgan, Investigators of Coordinators of the Epidemiologic Study of Cystic Fibrosis (b24) 2015; 50 Chilvers, Davies, Milla, Tian, Han, Cornell, Owen, Ratjen (b11) 2021; 9 Harrison, Dunn, Green, Copas (b26) 2009; 28 Milla, Ratjen, Marigowda, Liu, Waltz, Rosenfeld, VX13-809-011 Part B Investigator (b8) 2017; 195 Middleton, Mall, Dřevínek, Lands, McKone, Polineni, Ramsey, Taylor-Cousar, Tullis, Vermeulen, Marigowda, McKee, Moskowitz, Nair, Savage, Simard, Tian, Waltz, Xuan, Rowe, Jain (b10) 2019; 381 Lechtzin, Mayer-Hamblett, West, Allgood, Wilhelm, Khan, Aitken, Ramsey, Boyle, Mogayzel, Gibson, Orenstein, Milla, Clancy, Antony, Goss, eICE Study Team (b16) 2017; 196 Yanez, Kronmal, Shemanski, Psaty, Cardiovascular Health Study (b33) 2002; 12 Rosenfeld (10.1016/j.conctc.2024.101378_b24) 2015; 50 Montgomery (10.1016/j.conctc.2024.101378_b2) 2012; 25 Yanez (10.1016/j.conctc.2024.101378_b33) 2002; 12 Lord (10.1016/j.conctc.2024.101378_b28) 1967; 68 Ramsey (10.1016/j.conctc.2024.101378_b6) 2011; 365 Rowe (10.1016/j.conctc.2024.101378_b17) 2014; 190 Zemanick (10.1016/j.conctc.2024.101378_b12) 2021; 203 Glymour (10.1016/j.conctc.2024.101378_b31) 2005; 162 Liu (10.1016/j.conctc.2024.101378_b30) 2009; 28 Newsome (10.1016/j.conctc.2024.101378_b5) 2019; 18 Chilvers (10.1016/j.conctc.2024.101378_b11) 2021; 9 Heltshe (10.1016/j.conctc.2024.101378_b32) 2018; 197 Yanez (10.1016/j.conctc.2024.101378_b14) 1998; 17 Harun (10.1016/j.conctc.2024.101378_b3) 2016; 20 Chambless (10.1016/j.conctc.2024.101378_b15) 2003; 22 Saiman (10.1016/j.conctc.2024.101378_b21) 2003; 290 Stanojevic (10.1016/j.conctc.2024.101378_b19) 2020; 75 10.1016/j.conctc.2024.101378_b23 Paynter (10.1016/j.conctc.2024.101378_b22) 2022; 21 Wainwright (10.1016/j.conctc.2024.101378_b7) 2015; 373 Milla (10.1016/j.conctc.2024.101378_b8) 2017; 195 Jones (10.1016/j.conctc.2024.101378_b18) 2016; 11 Harrison (10.1016/j.conctc.2024.101378_b26) 2009; 28 Hamblett (10.1016/j.conctc.2024.101378_b25) 2022 Magaret (10.1016/j.conctc.2024.101378_b13) 2024; 23 Thornton (10.1016/j.conctc.2024.101378_b20) 2023 Konstan (10.1016/j.conctc.2024.101378_b1) 2012; 11 Lechtzin (10.1016/j.conctc.2024.101378_b16) 2017; 196 Middleton (10.1016/j.conctc.2024.101378_b10) 2019; 381 Heijerman (10.1016/j.conctc.2024.101378_b9) 2019; 394 Senn (10.1016/j.conctc.2024.101378_b29) 2006; 25 Szczesniak (10.1016/j.conctc.2024.101378_b4) 2017; 16 Braun (10.1016/j.conctc.2024.101378_b27) 2014; 33 |
| References_xml | – volume: 16 start-page: 318 year: 2017 end-page: 326 ident: b4 article-title: Use of FEV1 in cystic fibrosis epidemiologic studies and clinical trials: A statistical perspective for the clinical researcher publication-title: J. Cyst. Fibros. – volume: 195 start-page: 912 year: 2017 end-page: 920 ident: b8 article-title: Lumacaftor/ivacaftor in patients aged 6-11 years with cystic fibrosis and homozygous for F508del-CFTR publication-title: Am. J. Respir. Clin. Care Med. – year: 2023 ident: b20 article-title: Quantifying variation in home spirometry in people with cystic fibrosis during baseline health, and associations with clinical outcomes publication-title: J. Cyst. Fibros. – volume: 196 start-page: 1144 year: 2017 end-page: 1151 ident: b16 article-title: Home monitoring of patients with cystic fibrosis to identify and treat acute pulmonary exacerbations eICE study results publication-title: Am. J. Respir. Clin. Care Med. – volume: 68 start-page: 304 year: 1967 end-page: 305 ident: b28 article-title: A paradox in the interpretation of group comparisons publication-title: Psychol. Bull. – year: 2022 ident: b25 article-title: A Prospective Study to Evaluate Biological and Clinical Effects of Significantly Corrected CFTR Function (The PROMISE Study) – volume: 190 start-page: 175 year: 2014 end-page: 184 ident: b17 article-title: Clinical mechanism of the cystic fibrosis transmembrane conductance regulator potentiator ivacaftor in G551D-mediated cystic fibrosis publication-title: Am. J. Respir. Crit. Care Med. – volume: 9 start-page: 721 year: 2021 end-page: 732 ident: b11 article-title: Long-term safety and efficacy of lumacaftor-ivacaftor therapy in children aged 6-11 years with cystic fibrosis homozygous for the F508del CFTR mutation: a phase 3, open-label, extension study publication-title: Lancet Respir. Med. – volume: 290 start-page: 1749 year: 2003 end-page: 1756 ident: b21 article-title: Azithromycin in patients with cystic fibrosis chronically infected with publication-title: JAMA – volume: 21 start-page: 78 year: 2022 end-page: 83 ident: b22 article-title: A comparison of clinic and home spirometry as longtudinal outcomes in cystic fibrosis publication-title: J. Cyst. Fibros. – volume: 25 start-page: 4334 year: 2006 end-page: 4344 ident: b29 article-title: Change from baseline and analysis of covariance revisited publication-title: Stat. Med. – volume: 11 year: 2016 ident: b18 article-title: Should Studies of Diabetes Treatment Stratification Correct for Baseline HbA1c? publication-title: PLoS One – volume: 18 start-page: 110 year: 2019 end-page: 117 ident: b5 article-title: Investigating the effects of long-term dornase alfa use on lung function using registry data publication-title: J. Cyst. Fibros. – volume: 203 start-page: 1522 year: 2021 end-page: 1532 ident: b12 article-title: A Phase 3 Open-Label Study of Elexacaftor/Tezacaftor/Ivacaftor in Children 6 through 11 Years of Age with Cystic Fibrosis and at Least One F508del Allele publication-title: Am. J. Respir. Crit. Care Med. – volume: 394 start-page: 1940 year: 2019 end-page: 1948 ident: b9 article-title: Efficacy and safety of the elexacaftor plus tezacaftor plus ivacaftor combination regimen in people with cystic fibrosis homozygous for the F508del mutation: a double-blind, randomised, phase 3 trial publication-title: Lancet (London, England) – volume: 12 start-page: 34 year: 2002 end-page: 38 ident: b33 article-title: A regression model for longitudinal change in the presence of measurement error publication-title: Ann. Epidemiol. – reference: Therapeutics Development Network Coordinating Center, – volume: 28 start-page: 3260 year: 2009 end-page: 3275 ident: b26 article-title: Modelling the association between patient characteristics and the change over time in a disease measure using observational cohort data publication-title: Stat. Med. – volume: 25 start-page: 198 year: 2012 end-page: 203 ident: b2 article-title: Regulatory aspects of Phase 3 endpoints for new inhaled antibiotics for cystic fibrosis patients with chronic publication-title: J. Aerosol. Med. Pulm Drug Deliv. – volume: 50 start-page: 856 year: 2015 end-page: 862 ident: b24 article-title: Decline in lung function does not predict future decline in lung function in cystic fibrosis patients publication-title: Pediatr. Pulmonol. – volume: 365 start-page: 1663 year: 2011 end-page: 1672 ident: b6 article-title: A CFTR Potentiator in Patients with Cystic Fibrosis and the G551D Mutation publication-title: New Engl. J. Med. – volume: 22 start-page: 1041 year: 2003 end-page: 1067 ident: b15 article-title: Analysis of associations with change in a multivariate outcome variable when baseline is subject to measurement error publication-title: Stat. Med. – volume: 33 start-page: 2 year: 2014 end-page: 16 ident: b27 article-title: Accounting for baseline differences and measurement error in the analysis of change over time publication-title: Stat. Med. – volume: 197 start-page: 1483 year: 2018 end-page: 1486 ident: b32 article-title: Ivacaftor-treated patients with cystic fibrosis derive long-term benefit despite no short-term clinical improvement publication-title: Am. J. Respir. Clin. Care Med. – volume: 23 start-page: 943 year: 2024 end-page: 946 ident: b13 article-title: Impact of day-to-day variation in FEV1 on measures of change: a conceptual description publication-title: J. Cyst. Fibros. – volume: 28 start-page: 2509 year: 2009 end-page: 2530 ident: b30 article-title: Should baseline be a covariate or dependent variable in analyses of change from baseline in clinical trials? publication-title: Stat. Med. – volume: 373 start-page: 220 year: 2015 end-page: 231 ident: b7 article-title: Lumacaftor–Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del CFTR publication-title: New Engl. J. Med. – volume: 75 start-page: 891 year: 2020 end-page: 896 ident: b19 article-title: Paediatric reproducibility limits for the forced expiratory volume in 1 s publication-title: Thorax – volume: 381 start-page: 1809 year: 2019 end-page: 1819 ident: b10 article-title: Elexacaftor–Tezacaftor–Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele publication-title: New Engl. J. Med. – reference: . – volume: 162 start-page: 267 year: 2005 end-page: 278 ident: b31 article-title: When is baseline adjustment useful in analyses of change? An example with education and cognitive change publication-title: Am. J. Epidemiol. – volume: 17 start-page: 2597 year: 1998 end-page: 2606 ident: b14 article-title: The effects of measurement error in response variables and tests of association of explanatory variables in change models publication-title: Stat. Med. – volume: 11 start-page: 405 year: 2012 end-page: 411 ident: b1 article-title: Risk factors for rate of decline in FEV1 in adults with cystic fibrosis publication-title: J. Cyst. Fibros. – volume: 20 start-page: 55 year: 2016 end-page: 66 ident: b3 article-title: A systematic review of studies examining the rate of lung function decline in patients with cystic fibrosis publication-title: Paediatr. Respir. Rev. – volume: 17 start-page: 2597 issue: 22 year: 1998 ident: 10.1016/j.conctc.2024.101378_b14 article-title: The effects of measurement error in response variables and tests of association of explanatory variables in change models publication-title: Stat. Med. doi: 10.1002/(SICI)1097-0258(19981130)17:22<2597::AID-SIM940>3.0.CO;2-G – volume: 381 start-page: 1809 issue: 19 year: 2019 ident: 10.1016/j.conctc.2024.101378_b10 article-title: Elexacaftor–Tezacaftor–Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele publication-title: New Engl. J. Med. doi: 10.1056/NEJMoa1908639 – volume: 33 start-page: 2 issue: 1 year: 2014 ident: 10.1016/j.conctc.2024.101378_b27 article-title: Accounting for baseline differences and measurement error in the analysis of change over time publication-title: Stat. Med. doi: 10.1002/sim.5910 – volume: 50 start-page: 856 issue: 9 year: 2015 ident: 10.1016/j.conctc.2024.101378_b24 article-title: Decline in lung function does not predict future decline in lung function in cystic fibrosis patients publication-title: Pediatr. Pulmonol. doi: 10.1002/ppul.23227 – volume: 28 start-page: 3260 issue: 26 year: 2009 ident: 10.1016/j.conctc.2024.101378_b26 article-title: Modelling the association between patient characteristics and the change over time in a disease measure using observational cohort data publication-title: Stat. Med. doi: 10.1002/sim.3725 – volume: 12 start-page: 34 issue: 1 year: 2002 ident: 10.1016/j.conctc.2024.101378_b33 article-title: A regression model for longitudinal change in the presence of measurement error publication-title: Ann. Epidemiol. doi: 10.1016/S1047-2797(01)00280-0 – volume: 190 start-page: 175 issue: 2 year: 2014 ident: 10.1016/j.conctc.2024.101378_b17 article-title: Clinical mechanism of the cystic fibrosis transmembrane conductance regulator potentiator ivacaftor in G551D-mediated cystic fibrosis publication-title: Am. J. Respir. Crit. Care Med. doi: 10.1164/rccm.201404-0703OC – volume: 365 start-page: 1663 issue: 18 year: 2011 ident: 10.1016/j.conctc.2024.101378_b6 article-title: A CFTR Potentiator in Patients with Cystic Fibrosis and the G551D Mutation publication-title: New Engl. J. Med. doi: 10.1056/NEJMoa1105185 – volume: 290 start-page: 1749 issue: 13 year: 2003 ident: 10.1016/j.conctc.2024.101378_b21 article-title: Azithromycin in patients with cystic fibrosis chronically infected with Pseudomonas aeruginosa: a randomized controlled trial publication-title: JAMA doi: 10.1001/jama.290.13.1749 – volume: 20 start-page: 55 year: 2016 ident: 10.1016/j.conctc.2024.101378_b3 article-title: A systematic review of studies examining the rate of lung function decline in patients with cystic fibrosis publication-title: Paediatr. Respir. Rev. – volume: 394 start-page: 1940 issue: 10212 year: 2019 ident: 10.1016/j.conctc.2024.101378_b9 article-title: Efficacy and safety of the elexacaftor plus tezacaftor plus ivacaftor combination regimen in people with cystic fibrosis homozygous for the F508del mutation: a double-blind, randomised, phase 3 trial publication-title: Lancet (London, England) doi: 10.1016/S0140-6736(19)32597-8 – volume: 22 start-page: 1041 issue: 7 year: 2003 ident: 10.1016/j.conctc.2024.101378_b15 article-title: Analysis of associations with change in a multivariate outcome variable when baseline is subject to measurement error publication-title: Stat. Med. doi: 10.1002/sim.1352 – volume: 195 start-page: 912 issue: 7 year: 2017 ident: 10.1016/j.conctc.2024.101378_b8 article-title: Lumacaftor/ivacaftor in patients aged 6-11 years with cystic fibrosis and homozygous for F508del-CFTR publication-title: Am. J. Respir. Clin. Care Med. doi: 10.1164/rccm.201608-1754OC – volume: 9 start-page: 721 issue: 7 year: 2021 ident: 10.1016/j.conctc.2024.101378_b11 article-title: Long-term safety and efficacy of lumacaftor-ivacaftor therapy in children aged 6-11 years with cystic fibrosis homozygous for the F508del CFTR mutation: a phase 3, open-label, extension study publication-title: Lancet Respir. Med. doi: 10.1016/S2213-2600(20)30517-8 – year: 2023 ident: 10.1016/j.conctc.2024.101378_b20 article-title: Quantifying variation in home spirometry in people with cystic fibrosis during baseline health, and associations with clinical outcomes publication-title: J. Cyst. Fibros. – ident: 10.1016/j.conctc.2024.101378_b23 – volume: 196 start-page: 1144 issue: 9 year: 2017 ident: 10.1016/j.conctc.2024.101378_b16 article-title: Home monitoring of patients with cystic fibrosis to identify and treat acute pulmonary exacerbations eICE study results publication-title: Am. J. Respir. Clin. Care Med. doi: 10.1164/rccm.201610-2172OC – volume: 11 start-page: 405 issue: 5 year: 2012 ident: 10.1016/j.conctc.2024.101378_b1 article-title: Risk factors for rate of decline in FEV1 in adults with cystic fibrosis publication-title: J. Cyst. Fibros. doi: 10.1016/j.jcf.2012.03.009 – volume: 11 issue: 4 year: 2016 ident: 10.1016/j.conctc.2024.101378_b18 article-title: Should Studies of Diabetes Treatment Stratification Correct for Baseline HbA1c? publication-title: PLoS One doi: 10.1371/journal.pone.0152428 – volume: 23 start-page: 943 issue: 5 year: 2024 ident: 10.1016/j.conctc.2024.101378_b13 article-title: Impact of day-to-day variation in FEV1 on measures of change: a conceptual description publication-title: J. Cyst. Fibros. doi: 10.1016/j.jcf.2024.07.005 – volume: 162 start-page: 267 issue: 3 year: 2005 ident: 10.1016/j.conctc.2024.101378_b31 article-title: When is baseline adjustment useful in analyses of change? An example with education and cognitive change publication-title: Am. J. Epidemiol. doi: 10.1093/aje/kwi187 – volume: 203 start-page: 1522 issue: 12 year: 2021 ident: 10.1016/j.conctc.2024.101378_b12 article-title: A Phase 3 Open-Label Study of Elexacaftor/Tezacaftor/Ivacaftor in Children 6 through 11 Years of Age with Cystic Fibrosis and at Least One F508del Allele publication-title: Am. J. Respir. Crit. Care Med. doi: 10.1164/rccm.202102-0509OC – year: 2022 ident: 10.1016/j.conctc.2024.101378_b25 – volume: 28 start-page: 2509 issue: 20 year: 2009 ident: 10.1016/j.conctc.2024.101378_b30 article-title: Should baseline be a covariate or dependent variable in analyses of change from baseline in clinical trials? publication-title: Stat. Med. doi: 10.1002/sim.3639 – volume: 16 start-page: 318 issue: 3 year: 2017 ident: 10.1016/j.conctc.2024.101378_b4 article-title: Use of FEV1 in cystic fibrosis epidemiologic studies and clinical trials: A statistical perspective for the clinical researcher publication-title: J. Cyst. Fibros. doi: 10.1016/j.jcf.2017.01.002 – volume: 25 start-page: 4334 issue: 24 year: 2006 ident: 10.1016/j.conctc.2024.101378_b29 article-title: Change from baseline and analysis of covariance revisited publication-title: Stat. Med. doi: 10.1002/sim.2682 – volume: 197 start-page: 1483 issue: 11 year: 2018 ident: 10.1016/j.conctc.2024.101378_b32 article-title: Ivacaftor-treated patients with cystic fibrosis derive long-term benefit despite no short-term clinical improvement publication-title: Am. J. Respir. Clin. Care Med. doi: 10.1164/rccm.201710-2046LE – volume: 75 start-page: 891 issue: 10 year: 2020 ident: 10.1016/j.conctc.2024.101378_b19 article-title: Paediatric reproducibility limits for the forced expiratory volume in 1 s publication-title: Thorax doi: 10.1136/thoraxjnl-2020-214817 – volume: 18 start-page: 110 issue: 1 year: 2019 ident: 10.1016/j.conctc.2024.101378_b5 article-title: Investigating the effects of long-term dornase alfa use on lung function using registry data publication-title: J. Cyst. Fibros. doi: 10.1016/j.jcf.2018.08.004 – volume: 68 start-page: 304 issue: 5 year: 1967 ident: 10.1016/j.conctc.2024.101378_b28 article-title: A paradox in the interpretation of group comparisons publication-title: Psychol. Bull. doi: 10.1037/h0025105 – volume: 21 start-page: 78 issue: 1 year: 2022 ident: 10.1016/j.conctc.2024.101378_b22 article-title: A comparison of clinic and home spirometry as longtudinal outcomes in cystic fibrosis publication-title: J. Cyst. Fibros. doi: 10.1016/j.jcf.2021.08.013 – volume: 373 start-page: 220 issue: 3 year: 2015 ident: 10.1016/j.conctc.2024.101378_b7 article-title: Lumacaftor–Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del CFTR publication-title: New Engl. J. Med. doi: 10.1056/NEJMoa1409547 – volume: 25 start-page: 198 issue: 4 year: 2012 ident: 10.1016/j.conctc.2024.101378_b2 article-title: Regulatory aspects of Phase 3 endpoints for new inhaled antibiotics for cystic fibrosis patients with chronic Pseudomonas aeruginosa infections publication-title: J. Aerosol. Med. Pulm Drug Deliv. doi: 10.1089/jamp.2011.0911 |
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| Title | Baseline-dependent improvement in CF studies, plausibility of bias |
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