Development and Internal Validation of a Prognostic Model of the Probability of Death or Lung Transplantation Within 2 Years for Patients With Cystic Fibrosis and FEV1 ≤ 50% Predicted

Ramos, Kathleen J.; Wai, Travis Hee; Stephenson, Anne L.; Sykes, Jenna; Stanojevic, Sanja; Rodriguez, Patricia; Bansal, Aasthaa; Mayer-Hamblett, Nicole; Goss, Christopher H.; Kapnadak, Siddhartha G.

doi:10.1016/j.chest.2022.05.021

Cited by 7 publications

(5 citation statements)

References 37 publications

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“…Prognostic factors for death or transplant included lower FEV1pp, oxygen therapy (continuous or during exacerbation), and number of PEx requiring IV antibiotics. Overall, this model was superior compared to using FEV1 < 30% alone to predicting outcomes 82 …”

Section: Pulmonologymentioning

confidence: 92%

“…Overall, this model was superior compared to using FEV1 < 30% alone to predicting outcomes. 82 Pretransplant characteristics affect transplant outcomes. In an analysis of the CFFPR, PwCF with hemoptysis were at increased risk of progression to lung transplant/death, with further increased risk with massive hemoptysis.…”

Section: Transplantmentioning

confidence: 99%

“…In France, lung transplants for other indications increased when pandemic restrictions eased, though not specifically for PwCF 81 . For PwCF, the CF Foundation recommends having discussions regarding lung transplant at a FEV1pp of 50%, thus a prognostic model to evaluate 2‐year outcomes of death or lung transplant was developed using the CFFPR and UNOS (United Network for Organ Sharing) data 82 . Of the approximately 3700 PwCF, death was more likely in those with Medicaid and less likely in White PwCF.…”

Section: Pulmonologymentioning

confidence: 99%

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Cystic fibrosis year in review 2022

Kim,

Lyman,

Savant

2023

Pediatric Pulmonology

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Remarkable medical advancements have been made for people with cystic fibrosis (CF) in recent years, with an abundance of research continuing to be conducted worldwide. With concern for limitations in access to highly effective CFTR modulators, as well as the recent Coronavirus Disease‐19 pandemic, there has been a consistent effort to understand and improve CF screening, disease burden, diagnosis, and management. Our aim in this review is to present articles from 2022 with an emphasis on clinically relevant studies. We hope this will serve as a broad overview of the research published in the past year.

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Section: Pulmonologymentioning

confidence: 92%

Section: Transplantmentioning

confidence: 99%

Section: Pulmonologymentioning

confidence: 99%

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Cystic fibrosis year in review 2022

Kim,

Lyman,

Savant

2023

Pediatric Pulmonology

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“…A probabilistic linkage was done between CFFPR and OPTN to update the CFFPR with transplant date and to ensure complete vital statistics. Additional details concerning the record linkage between the CFFPR and OPTN are published 1,13–15 . A unified Canada‐US‐OPTN data set was created after harmonizing data definitions and data collection methods within each registry 1 .…”

Section: Methodsmentioning

confidence: 99%

Cystic fibrosis survival outcomes following second lung transplant: The north American experience

Alshehri

Ramos

Sykes

et al. 2023

Clinical Transplantation

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IntroductionRe‐transplant is an option for those who develop end‐stage lung disease due to rejection; however, little data exist following re‐transplantation in cystic fibrosis (CF).MethodsData from the Canadian CF Registry and US CF Foundation Patient Registry supplemented with data from United Network for Organ Sharing were used. Individuals who underwent a 2nd lung transplant between 2005 and 2019 were included. The Kaplan‐Meier method was used to estimate the probability of survival post‐second transplant at 1, 3, and 5‐years.ResultsOf those people who were waitlisted for a second transplant (N = 818), a total of 254 (31%) died waiting, 395 (48%) were transplanted and 169 (21%) people were alive on the waitlist. Median survival time after 2nd lung transplant was 3.3 years (95% CI: 2.8‐4.1). The 1‐, 3‐ and 5‐year survival rates were 77.4% (95% CI: 73.1‐82%), 52% (95% CI: 46.7‐58%) and 39.4% (95% CI: 34.1‐45.6%).ConclusionsSurvival following second lung transplant in CF patients is lower than estimates following the first transplant. Over half of subjects who are potentially eligible for a second transplant die without receiving a second organ. This warrants further investigation.

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“…Lung transplantation (LTX) is a treatment undertaken to improve the quality of life and survival in carefully selected patients with advanced CF lung disease (ACFLD) ( 2 , 3 ). Various predictors of LTX have been identified in CF, including poor lung function (forced expiratory volume in 1 s [FEV 1 ]), low body mass index (BMI), hypoxemia, infection with Burkholderia cepacia complex, oxygen supplementation, number of hospitalizations for pulmonary exacerbations, and functional exercise capacity ( 4 – 10 ). The changing demographics in CF and remarkable improvements in overall survival ( 11 , 12 ), also seen in those with ACFLD ( 9 ), pose challenges to clinicians with respect to the optimal timing of LTX evaluation and candidate selection.…”

mentioning

confidence: 99%

Cardiopulmonary Exercise Testing Provides Prognostic Information in Advanced Cystic Fibrosis Lung Disease

Radtke,

Urquhart,

Braun

et al. 2024

Annals ATS

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Rationale Cardiopulmonary exercise testing (CPET) provides prognostic information in cystic fibrosis (CF); however, its prognostic value for patients with advanced CF lung disease is unknown. Objectives To determine the prognostic value of CPET on the risk of death or lung transplant (LTX) within 2 years. Methods We retrospectively collected data from 20 CF centers in Asia, Australia, Europe, and North America on patients with a forced expiratory volume in 1 second (FEV 1 ) ⩽ 40% predicted who performed a cycle ergometer CPET between January 2008 and December 2017. Time to death/LTX was analyzed using mixed Cox proportional hazards regression. Conditional inference trees were modeled to identify subgroups with increased risk of death/LTX. Results In total, 174 patients (FEV 1 , 30.9% ± 5.8% predicted) were included. Forty-four patients (25.5%) died or underwent LTX. Cox regression analysis adjusted for age, sex, and FEV 1 revealed percentage predicted peak oxygen uptake ( o 2peak ) and peak work rate (W peak ) as significant predictors of death/LTX: adjusted hazard ratios per each additional 10% predicted were 0.60 (95% confidence interval, 0.43–0.90; P = 0.008) and 0.60 (0.48–0.82; P < 0.001). Tree-structured regression models, including a set of 11 prognostic factors for survival, identified W peak to be most strongly associated with 2-year risk of death/LTX. Probability of death/LTX was 45.2% for those with a W peak ⩽ 49.2% predicted versus 10.9% for those with a W peak > 49.2% predicted ( P < 0.001). Conclusions CPET provides prognostic information in advanced CF lung disease, and W peak appears to be a promising marker for LTX referral and candidate selection.

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Development and Internal Validation of a Prognostic Model of the Probability of Death or Lung Transplantation Within 2 Years for Patients With Cystic Fibrosis and FEV1 ≤ 50% Predicted

Cited by 7 publications

References 37 publications

Cystic fibrosis year in review 2022

Cystic fibrosis year in review 2022

Cystic fibrosis survival outcomes following second lung transplant: The north American experience

Cardiopulmonary Exercise Testing Provides Prognostic Information in Advanced Cystic Fibrosis Lung Disease

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