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. 2023 Mar 13:19:255-267.
doi: 10.2147/TCRM.S391476. eCollection 2023.

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

Simone Gambazza #  1   2 , Annalisa Orenti #  2 , Giovanna Pizzamiglio  3 , Anna Zolin  2 , Carla Colombo  4   5 , Dario Laquintana  1 , Federico Ambrogi  2   6 ; ECFSPR
Affiliations

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

Simone Gambazza et al. Ther Clin Risk Manag. .

Abstract

Background: Association between dependence on oxygen therapy (OT) and natural disease progression in people with cystic fibrosis (pwCF) has not been estimated yet. The aim of this study is to understand the prognosis for pwCF on OT, evaluating how the transition probabilities from being alive without lung transplantation (LTx) to LTx and to death, and from being alive after LTx to death change in pwCF with and without OT.

Methods: We used 2008-2017 data from the 35-country European CF Society Patient Registry. A multi-state model was fitted to assess the effects of individual risk factors on transition probabilities.

Results: We considered 48,343 pwCF aged from 6 to 50 years. OT (HR 5.78, 95% CI: 5.32-6.29) and abnormal FEV1 (HR 6.41, 95% CI: 5.28-7.79) were strongly associated with the probability of having LTx; chronic infection with Burkholderia cepacia complex (HR 3.19, 95% CI: 2.78-3.67), abnormal FEV1 (HR 5.00, 95% CI: 4.11-6.08) and the need for OT (HR 4.32, 95% CI: 3.93-4.76) showed the greatest association with the probability of dying without LTx. Once pwCF received LTx, OT (HR 1.75, 95% CI: 1.41-2.16) and abnormal FEV1 (HR 1.63, 95% CI: 1.18-2.25) were the main factors associated with the probability of dying. An association of gross national income with the probability of receiving LTx and with the probability of dying without LTx was also found.

Conclusion: Oxygen therapy is associated with poor survival in pwCF with and without LTx; harmonization of CF care throughout European countries and minimization of the onset of pulmonary gas exchange abnormalities using all available means remains of paramount importance.

Keywords: cystic fibrosis; epidemiology; lung transplantation; mortality; oxygen therapy.

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Conflict of interest statement

The authors report no conflicts of interest in this work.

Figures

Figure 1
Figure 1
Multi-state model for pwCF: arrows denote direction of transition probabilities (T): from alive without LTx to alive with LTx (T1) or to death (T2); and from alive with LTx to death (T3).
Figure 2
Figure 2
Percentage of pwCF on OT, stratified by year of follow-up, age class and economic status. Low income: Albania, Armenia, Bulgaria, Czech Republic, Greece, Croatia, Hungary, Latvia, Lithuania, North Macedonia, Republic of Moldova, Poland, Portugal, Romania, Russian Federation, Serbia, Slovak Republic, Slovenia, Turkey, Ukraine; high income: Austria, Belgium, Denmark, France, Germany, Ireland, Israel, Italy, Luxembourg, The Netherlands, Norway, Spain, Sweden, Switzerland, United Kingdom.
Figure 3
Figure 3
Probabilities of moving from state 1 (alive without LTx) to state 2 (alive with LTx) or state 3 (death) (upper panels) and probabilities for pwCF moving from state 2 to state 3 (lower panels), according to treatment with supplemental oxygen in pwCF at 20 years of age. Adjusting covariates are held constant at the most frequent occurrence.
Figure 4
Figure 4
Probabilities to move from state 1 (alive without LTx) to state 2 (alive with LTx) or state 3 (death) (upper panels) and probabilities for pwCF to move from state 2 to state 3 (lower panels), according to GNI in pwCF at 20 years of age. Adjusting covariates are held constant at the most frequent occurrence.
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References

    1. 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 - DOI - PubMed
    1. Katz ES. Cystic fibrosis and sleep. Clin Chest Med. 2014;35(3):495–504. doi:10.1016/j.ccm.2014.06.005 - DOI - PubMed
    1. 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 - DOI - PubMed
    1. Elphick HE, Mallory G. Oxygen therapy for cystic fibrosis. Cochrane Database Syst Rev. 2009;1:CD003884. doi:10.1002/14651858.CD003884.pub3 - DOI - PubMed
    1. 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 - DOI - PubMed