TY - JOUR
T1 - Survival and Lung Transplant Outcomes for Individuals With Advanced Cystic Fibrosis Lung Disease Living in the United States and Canada
T2 - An Analysis of National Registries
AU - Ramos, Kathleen J.
AU - Sykes, Jenna
AU - Stanojevic, Sanja
AU - Ma, Xiayi
AU - Ostrenga, Joshua S.
AU - Fink, Aliza
AU - Quon, Bradley S.
AU - Marshall, Bruce C.
AU - Faro, Albert
AU - Petren, Kristofer
AU - Elbert, Alexander
AU - Goss, Christopher H.
AU - Stephenson, Anne L.
N1 - Funding Information:
FUNDING/SUPPORT: K. J. R. receives funding from the CFF [RAMOS17A0, RAMOS20A0-KB] and the National Institutes of Health [ K23HL138154 ]. C. H. G. was supported by grants from the CFF, the NIH [UM1 HL119073, P30 DK089507, U01 HL114589, UL1 TR000423], and the FDA [R01 FD003704, R01 FD006848].
Funding Information:
FUNDING/SUPPORT: K. J. R. receives funding from the CFF [RAMOS17A0, RAMOS20A0-KB] and the National Institutes of Health [K23HL138154]. C. H. G. was supported by grants from the CFF, the NIH [UM1 HL119073, P30 DK089507, U01 HL114589, UL1 TR000423], and the FDA [R01 FD003704, R01 FD006848].Author contributions: K. J. R. is the guarantor of the content of the manuscript, including the data and analysis. K. J. R. J. S. S. S. A. Fink, B. S. Q. B. C. M. A. Faro, C. H. G. and A. L. S. made substantial contributions to the conception and design of the work; K. J. R. J. S. X. M. J. S. O. A. Fink, K. P. A. E. C. H. G. and A. L. S. were responsible for acquisition of the data; J. S. S. S. and X. M. were responsible for analysis of the data; all of the co-authors made substantial contributions to the interpretation of data. K. J. R. and A. L. S. wrote the first draft of the manuscript, and all of the co-authors critically revised it for important intellectual content. All of the co-authors approved the final version submitted for publication and agree to be accountable for all aspects of the work. All of the co-authors ensure that questions related to the accuracy or integrity of any part of the work are appropriately investigated and resolved. Financial/nonfinancial disclosures: The authors have reported to CHEST the following: J. S. O. A. Fink, B. C. M. A. Faro, K. P. and A. E. work at the Cystic Fibrosis Foundation (CFF) in the United States. K. J. R. C. H. G. and A. L. S. receive grant funding from the CFF, and A. L. S. receives funding from CF Canada. These financial relationships did not influence the interpretation or reporting of the current study. K. J. R. receives funding from the CFF [RAMOS17A0, RAMOS20A0-KB] and the National Institutes of Health [K23HL138154]. C. H. G. was supported by grants from the Cystic Fibrosis Foundation, the NIH [UM1 HL119073, P30 DK089507, U01 HL114589, UL1 TR000423], and the FDA [R01 FD003704, R01 FD006848]. Role of sponsors: The sponsors had no role in the design of the study, the collection and analysis of the data, or the preparation of the manuscript. Disclaimer: Some of the data reported here have been supplied by UNOS as the contractor for the Organ Procurement and Transplantation Network (OPTN). The interpretation and reporting of these data are the responsibility of the author(s) and in no way should be seen as an official policy of or interpretation by the OPTN or the US government. Other contributions: The authors thank the individuals with cystic fibrosis, care providers, and clinic coordinators at CF centers throughout the United States and Canada for their contributions to their respective cystic fibrosis patient registries. Additional information: The e-Appendix, e-Tables, and e-Figures can be found in the Supplemental Materials section of the online article.
Funding Information:
Financial/nonfinancial disclosures: The authors have reported to CHEST the following: J. S. O., A. Fink, B. C. M., A. Faro, K. P., and A. E. work at the Cystic Fibrosis Foundation (CFF) in the United States. K. J. R., C. H. G., and A. L. S. receive grant funding from the CFF, and A. L. S. receives funding from CF Canada. These financial relationships did not influence the interpretation or reporting of the current study. K. J. R. receives funding from the CFF [RAMOS17A0, RAMOS20A0-KB] and the National Institutes of Health [ K23HL138154 ]. C. H. G. was supported by grants from the Cystic Fibrosis Foundation, the NIH [UM1 HL119073, P30 DK089507, U01 HL114589, UL1 TR000423], and the FDA [R01 FD003704, R01 FD006848].
Publisher Copyright:
© 2021 American College of Chest Physicians
PY - 2021/9
Y1 - 2021/9
N2 - Background: Understanding how health outcomes differ for patients with advanced cystic fibrosis (CF) lung disease living in the United States compared with Canada has health policy implications. Research Question: What are rates of lung transplant (LTx) and rates of death without LTx in the United States and Canada among individuals with FEV1 < 40% predicted? Study Design and Methods: This was a retrospective population-based cohort study, 2005 to 2016, using the US CF Foundation, United Network for Organ Sharing, and Canadian CF registries. Individuals with CF and at least two FEV1 measurements < 40% predicted within a 5-year period, age ≥ 6 years, without prior LTx were included. Multivariable competing risk regression for time to death without LTx (LTx as a competing risk) and time to LTx (death as a competing risk) was performed. Results: There were 5,899 patients (53% male) and 905 patients (54% male) with CF with FEV1 < 40% predicted living in the United States and Canada, respectively. Multivariable competing risk regression models identified an increased risk of death without LTx (hazard ratio [HR], 1.79; 95% CI, 1.52-2.1) and decreased LTx (HR, 0.66; 95% CI, 0.58-0.74) among individuals in the United States compared with Canada. More pronounced differences were seen in the patients in the United States with Medicaid/Medicare insurance compared with Canadians (multivariable HR for death without LTx, 2.24 [95% CI, 1.89-2.64]; multivariable HR for LTx, 0.54 [95% CI, 0.47-0.61]). Patients of nonwhite race were also disadvantaged (multivariable HR for death without LTx, 1.56 [95% CI, 1.32-1.84]; multivariable HR for LTx, 0.47 [95% CI, 0.36-0.62]). Interpretation: There are lower rates of LTx and an increased risk of death without LTx for US patients with CF with FEV1 < 40% predicted compared with Canadian patients. Findings are more striking among US patients with CF with Medicaid/Medicare health insurance, and nonwhite patients in both countries, raising concerns about underuse of LTx among vulnerable populations.
AB - Background: Understanding how health outcomes differ for patients with advanced cystic fibrosis (CF) lung disease living in the United States compared with Canada has health policy implications. Research Question: What are rates of lung transplant (LTx) and rates of death without LTx in the United States and Canada among individuals with FEV1 < 40% predicted? Study Design and Methods: This was a retrospective population-based cohort study, 2005 to 2016, using the US CF Foundation, United Network for Organ Sharing, and Canadian CF registries. Individuals with CF and at least two FEV1 measurements < 40% predicted within a 5-year period, age ≥ 6 years, without prior LTx were included. Multivariable competing risk regression for time to death without LTx (LTx as a competing risk) and time to LTx (death as a competing risk) was performed. Results: There were 5,899 patients (53% male) and 905 patients (54% male) with CF with FEV1 < 40% predicted living in the United States and Canada, respectively. Multivariable competing risk regression models identified an increased risk of death without LTx (hazard ratio [HR], 1.79; 95% CI, 1.52-2.1) and decreased LTx (HR, 0.66; 95% CI, 0.58-0.74) among individuals in the United States compared with Canada. More pronounced differences were seen in the patients in the United States with Medicaid/Medicare insurance compared with Canadians (multivariable HR for death without LTx, 2.24 [95% CI, 1.89-2.64]; multivariable HR for LTx, 0.54 [95% CI, 0.47-0.61]). Patients of nonwhite race were also disadvantaged (multivariable HR for death without LTx, 1.56 [95% CI, 1.32-1.84]; multivariable HR for LTx, 0.47 [95% CI, 0.36-0.62]). Interpretation: There are lower rates of LTx and an increased risk of death without LTx for US patients with CF with FEV1 < 40% predicted compared with Canadian patients. Findings are more striking among US patients with CF with Medicaid/Medicare health insurance, and nonwhite patients in both countries, raising concerns about underuse of LTx among vulnerable populations.
KW - access to transplantation
KW - advanced lung disease
KW - cystic fibrosis
KW - lung transplantation
UR - http://www.scopus.com/inward/record.url?scp=85111989279&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=85111989279&partnerID=8YFLogxK
U2 - 10.1016/j.chest.2021.04.010
DO - 10.1016/j.chest.2021.04.010
M3 - Article
C2 - 33878343
AN - SCOPUS:85111989279
VL - 160
SP - 843
EP - 853
JO - Diseases of the chest
JF - Diseases of the chest
SN - 0012-3692
IS - 3
ER -