Association of Oxygen Therapy with the Natural Disease Progression of Cystic Fibrosis: A Multi-State Model of the European Cystic Fibrosis Society Patient Registry

References

• Stephenson AL, Stanojevic S, Sykes J, Burgel PR. The changing epidemiology and demography of cystic fibrosis. Presse Med. 2017;46(6 Pt 2):e87–e95. doi:10.1016/j.lpm.2017.04.012
   PubMed Web of Science ®Google Scholar
• Katz ES. Cystic fibrosis and sleep. Clin Chest Med. 2014;35(3):495–504. doi:10.1016/j.ccm.2014.06.005
   PubMed Web of Science ®Google Scholar
• Schidlow DV, Taussig LM, Knowles MR. Cystic fibrosis foundation consensus conference report on pulmonary complications of cystic fibrosis. Pediatr Pulmonol. 1993;15(3):187–198. doi:10.1002/ppul.1950150311
   PubMed Web of Science ®Google Scholar
• Elphick HE, Mallory G. Oxygen therapy for cystic fibrosis. Cochrane Database Syst Rev. 2009;1:CD003884. doi:10.1002/14651858.CD003884.pub3
   Google Scholar
• Hopkinson NS, Molyneux A, Pink J, Harrisingh MC. Chronic obstructive pulmonary disease: diagnosis and management: summary of updated NICE guidance. BMJ. 2019;366(July):1–7. doi:10.1136/bmj.l4486
   Google Scholar
• Zolin A, Orenti A, Naehrlich L, Jung A, van Rens J. ECFS patient registry annual report 2018; 2020. Available from: https://www.ecfs.eu/projects/ecfs-patient-registry/annual-reports. Accessed March 7, 2023.
   Google Scholar
• Viviani L, Zolin A, Mehta A, Olesen HV. The European cystic fibrosis society patient registry: valuable lessons learned on how to sustain a disease registry. Orphanet J Rare Dis. 2014;9(1):1–14. doi:10.1186/1750-1172-9-81
   PubMedGoogle Scholar
• Zolin A, Orenti A, Naehrlich L, van Rens J. ECFSPR annual report 2017; 2019.
   Google Scholar
• Quanjer PH, Stanojevic S, Cole TJ, et al. Multi-ethnic reference values for spirometry for the 3–95-yr age range: the global lung function 2012 equations. Eur Respir J. 2012;40(6):1324–1343. doi:10.1183/09031936.00080312
   PubMed Web of Science ®Google Scholar
• Turck D, Braegger CP, Colombo C, et al. ESPEN-ESPGHAN-ECFS guidelines on nutrition care for infants, children, and adults with cystic fibrosis. Clin Nutr. 2016;35(3):557–577. doi:10.1016/j.clnu.2016.03.004
   PubMed Web of Science ®Google Scholar
• Kuczmarski RJ, Ogden CL, Guo SS, et al. 2000 CDC growth charts for the United States: methods and development. Vital Health Stat 11. 2002;246:1–190.
   PubMedGoogle Scholar
• Gross National Income per Capita. Atlas method and PPP; 2019. Available from: https://databank.worldbank.org/home. Accessed March 29, 2021.
   Google Scholar
• Meira-Machado LF, de Uña-álvarez J, Cadarso-Suárez C, Andersen PK. Multi-state models for the analysis of time-to-event data. Stat Methods Med Res. 2009;18(2):195–222. doi:10.1177/0962280208092301
   PubMed Web of Science ®Google Scholar
• Stanojevic S, Kaminsky DA, Miller M, et al. ERS/ATS technical standard on interpretive strategies for routine lung function tests. Eur Respir J. 2021:2101499. doi:10.1183/13993003.01499-2021
   Web of Science ®Google Scholar
• de Wreede LC, Fiocco M, Putter H. The mstate package for estimation and prediction in non- and semi-parametric multi-state and competing risks models. Comput Methods Programs Biomed. 2010;99(3):261–274. doi:10.1016/j.cmpb.2010.01.001
   PubMed Web of Science ®Google Scholar
• Keogh RH, Szczesniak R, Taylor-Robinson D, Bilton D. Up-to-date and projected estimates of survival for people with cystic fibrosis using baseline characteristics: a longitudinal study using UK patient registry data. J Cyst Fibros. 2018;17(2):218–227. doi:10.1016/j.jcf.2017.11.019
   PubMed Web of Science ®Google Scholar
• Stephenson AL, Sykes J, Stanojevic S, et al. Survival comparison of patients with cystic fibrosis in Canada and the United States. Ann Intern Med. 2017;166(8):537. doi:10.7326/M16-0858
   PubMed Web of Science ®Google Scholar
• Burgel PR, Bellis G, Olesen HV, et al. Future trends in cystic fibrosis demography in 34 European countries. Eur Respir J. 2015;46(1):133–141. doi:10.1183/09031936.00196314
   PubMed Web of Science ®Google Scholar
• Cystic Fibrosis Foundation. Patient Registry Annual Data Report. Cystic Fibrosis Foundation; 2019.
   Google Scholar
• O’Driscoll BR, Howard LS, Earis J, et al. BTS guideline for oxygen use in adults in healthcare and emergency settings. Thorax. 2017;72(6):i1–i90. doi:10.1136/thoraxjnl-2016-209729
   PubMedGoogle Scholar
• Ramos KJ, Smith PJ, McKone EF, et al. Lung transplant referral for individuals with cystic fibrosis: cystic fibrosis foundation consensus guidelines. J Cyst Fibros. 2019;18(3):321–333. doi:10.1016/j.jcf.2019.03.002
   PubMed Web of Science ®Google Scholar
• Ramos KJ, Quon BS, Heltshe SL, et al. Heterogeneity in survival in adult patients with cystic fibrosis with FEV1 < 30% of predicted in the United States. Chest. 2017;151(6):1320–1328. doi:10.1016/j.chest.2017.01.019
   PubMed Web of Science ®Google Scholar
• Kerem E, Reisman J, Corey M, Canny GJ, Levison H. Prediction of mortality in patients with cystic fibrosis. N Engl J Med. 1992;326(18):1187–1191. doi:10.1056/NEJM199204303261804
   PubMed Web of Science ®Google Scholar
• Liou TG, Adler FR, Cox DR, Cahill BC. Lung transplantation and survival in children with cystic fibrosis. N Engl J Med. 2007;357(21):2143–2152. doi:10.1056/NEJMoa066359
   PubMed Web of Science ®Google Scholar
• Krivchenia K, Tumin D, Tobias JD, Hayes D. Increased mortality in adult cystic fibrosis patients with medicaid insurance awaiting lung transplantation. Lung. 2016;194(5):799–806. doi:10.1007/s00408-016-9927-7
   PubMed Web of Science ®Google Scholar
• Nkam L, Lambert J, Latouche A, Bellis G, Burgel PR, Hocine MN. A 3-year prognostic score for adults with cystic fibrosis. J Cyst Fibros. 2017;16(6):702–708. doi:10.1016/j.jcf.2017.03.004
   PubMed Web of Science ®Google Scholar
• Robinson PD, Latzin P, Ramsey KA, et al. Preschool multiple-breath washout testing. An official American thoracic society technical statement. Am J Respir Crit Care Med. 2018;197(5):e1–e19. doi:10.1164/rccm.201801-0074ST
   PubMed Web of Science ®Google Scholar
• Milla CE, Warwick WJ. Risk of death in cystic fibrosis patients with severely compromised lung function. Chest. 1998;113(5):1230–1234. doi:10.1378/chest.113.5.1230
   PubMed Web of Science ®Google Scholar
• Doershuk CF, Stern RC. Timing of referral for lung transplantation for cystic fibrosis: overemphasis on FEV1 may adversely affect overall survival. Chest. 1999;115(3):782–787. doi:10.1378/chest.115.3.782
   PubMed Web of Science ®Google Scholar
• Stanojevic S, Stocks J, Bountziouka V, et al. The impact of switching to the new global lung function initiative equations on spirometry results in the UK CF registry. J Cyst Fibros. 2014;13(3):319–327. doi:10.1016/j.jcf.2013.11.006
   PubMed Web of Science ®Google Scholar
• Hebestreit H, Hulzebos EHJ, Schneiderman JE, et al. Cardiopulmonary exercise testing provides additional prognostic information in cystic fibrosis. Am J Respir Crit Care Med. 2019;199(8):987–995. doi:10.1164/rccm.201806-1110OC
   PubMed Web of Science ®Google Scholar
• Nixon PA, Orenstein DM, Kelsey SF, Doershuk CF. The prognostic value of exercise testing in patients with cystic fibrosis. N Engl J Med. 1992;327(25):1785–1788. doi:10.1056/NEJM199212173272504
   PubMed Web of Science ®Google Scholar
• Stephenson AL, Ramos KJ, Sykes J, et al. Bridging the survival gap in cystic fibrosis: an investigation of lung transplant outcomes in Canada and the United States. J Heart Lung Transplant. 2021;40(3):201–209. doi:10.1016/j.healun.2020.12.001
   PubMed Web of Science ®Google Scholar
• McKone EF, Ariti C, Jackson A, et al. Survival estimates in European cystic fibrosis patients and the impact of socioeconomic factors: a retrospective registry cohort study. Eur Respir J. 2021;58(3):2002288. doi:10.1183/13993003.02288-2020
   PubMed Web of Science ®Google Scholar
• Davies JC, Sermet-Gaudelus I, Naehrlich L, et al. A Phase 3, double-blind, parallel-group study to evaluate the efficacy and safety of tezacaftor in combination with ivacaftor in participants 6 through 11 years of age with cystic fibrosis homozygous for F508del or heterozygous for the F508del-CFTR mutati. J Cyst Fibros. 2021;20(1):68–77. doi:10.1016/j.jcf.2020.07.023
   PubMed Web of Science ®Google Scholar
• Milla CE, Ratjen F, Marigowda G, Liu F, Waltz D, Rosenfeld M. Lumacaftor/ivacaftor in patients aged 6–11 years with cystic fibrosis and homozygous for F508del-CFTR. Am J Respir Crit Care Med. 2017;195(7):912–920. doi:10.1164/rccm.201608-1754OC
   PubMed Web of Science ®Google Scholar
• Frauchiger BS, Binggeli S, Yammine S, et al. Longitudinal course of clinical lung clearance index in children with cystic fibrosis. Eur Respir J. 2021;58(1):2002686. doi:10.1183/13993003.02686-2020
   PubMed Web of Science ®Google Scholar
• Kurz JM, Ramsey KA, Rodriguez R, et al. Association of lung clearance index with survival in individuals with cystic fibrosis. Eur Respir J. 2021. doi:10.1183/13993003.00432-2021
   PubMed Web of Science ®Google Scholar
• Barry PJ, Taylor-Cousar JL. Triple combination cystic fibrosis transmembrane conductance regulator modulator therapy in the real world - opportunities and challenges. Curr Opin Pulm Med. 2021;27(6):554–566. doi:10.1097/MCP.0000000000000819
   PubMed Web of Science ®Google Scholar
• Zolin A, Orenti A, Naehrlich L, Jung A, van Rens J. ECFSPR annual report 2018; 2020.
   Google Scholar
• EMA/CHMP/SAWP/622564/2018. Qualification opinion on the European Cystic Fibrosis Society Patient Registry (ECFSPR) and CF pharmaco-epidemiology studies. Available from: https://www.ema.europa.eu/en/documents/regulatory-procedural-guideline/qualification-opinion-european-cystic-fibrosis-society-patient-registry-ecfspr-cf-pharmaco_en.pdf. Accessed March 7, 2023.
   Google Scholar
• Naehrlich L, Fox A, Krasynk M, Orenti A, Zolin A, van Rens J. P080 the European Cystic Fibrosis Society Patient Registry (ECFSPR) data validation programme: accuracy and consistency of data. J Cyst Fibros. 2019;18:S80. doi:10.1016/S1569-1993(19)30374-1
   Google Scholar