Longitudinal changes in BMD in adults with cystic fibrosis.

Improved survival in people with cystic fibrosis (pwCF) presents new complexities of care, including CF-related bone disease, a common complication in older pwCF. The trajectory of bone loss with age in this population remains unclear. The objective of this study was to estimate the average rate of change in BMD in adults with CF.

Impact of COVID-19 infection on lung function and nutritional status amongst individuals with cystic fibrosis: A global cohort study.

Background: Factors associated with severe COVID-19 infection have been identified; however, the impact of infection on longer-term outcomes is unclear. The objective of this study was to examine the impact of COVID-19 infection on the trajectory of lung function and nutritional status in people with cystic fibrosis (pwCF).

Methods: This is a retrospective global cohort study of pwCF who had confirmed COVID-19 infection diagnosed between January 1, 2020 and December 31, 2021.

Comparative Safety Analysis of Nabilone Versus Opioids: A Population-Based Cohort Study.

Background: Some have advocated that nabilone be used rather than opioids to manage chronic, noncancer pain, since the former drug may have a better safety profile.

Objective: We compared the safety of incident nabilone use relative to incident opioid use with respect to multiple clinically important outcomes.

Design: A population-based, retrospective cohort study.

Socioeconomic burden of cystic fibrosis in Canada.

Background: Cost of illness studies are important tools to summarise the burden of disease for individuals, the healthcare system and society. The lack of standardised methods for reporting costs for cystic fibrosis (CF) makes it difficult to quantify the total socioeconomic burden. In this study, we aimed to comprehensively report the socioeconomic burden of CF in Canada.

Clinical outcomes after liver transplant in people with cystic fibrosis: A systematic review and meta-analysis.

Background: Data on the impact of liver transplantation (LT) in cystic fibrosis (CF) on lung function and exacerbations are limited. The objective of this study was to summarize the literature on lung function, nutritional status, survival, and complications following LT in people with CF.

Methods: Three databases were searched until September 2023, to identify the impact of LT in CF.

Measuring the burden of cystic fibrosis: A scoping review.

Background: Cystic fibrosis (CF) contributes a significant economic burden on individuals, healthcare systems, and society. Understanding the economic impact of CF is crucial for planning resource allocation.

Methods: We conducted a scoping review of literature published between 1990 and 2022 that reported the cost of illness, and/or economic burden of CF.

Global disparities in cystic fibrosis outcomes prior to CFTR modulators: A CF registries cohort study in South Africa and Canada.

Background: Outcomes of cystic fibrosis (CF) differ between low-middle income and high-income countries, but comparative data are lacking. We compared South African (SA) and Canadian CF outcomes to explore what disparities existed prior to access of CFTR modulators in Canada.

Methods: A cross-sectional study of SA and Canadian CF registries data for period 1 January to 31 December 2018.

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

Introduction: Re-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).

Methods: Data 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.

Canadian Cystic Fibrosis-related Diabetes Clinical Practice Survey: Analysis of Current Practices and Gaps in Clinical Care.

Objectives: Our aim in this study was to identify challenges and gaps in Canadian practices in screening, diagnosis, and treatment of cystic fibrosis-related diabetes (CFRD), with the goal of informing a Canadian-specific guideline for CFRD.

Methods: We conducted an online survey of health-care professionals (97 physicians and 44 allied health professionals) who care for people living with CF (pwCF) and/or CFRD (pwCFRD).

Results: Most pediatric centres followed <10 pwCFRD and adult centres followed >10 pwCFRD.

Comparison of quadriceps muscle size and quality in adults with cystic fibrosis with different severities of cystic fibrosis transmembrane conductance regulator protein dysfunction.

Background: Cystic fibrosis (CF) is characterized by CF transmembrane conductance regulator (CFTR) dysfunction. CFTR protein is expressed in human skeletal muscle; however, its impact on skeletal muscle is unknown. The objectives of this study were to compare quadriceps muscle size and quality between adults with various severities of CFTR protein dysfunction.

Contemporary cystic fibrosis incidence rates in Canada and the United States.

Background: The availability of new diagnostic algorithms for cystic fibrosis (CF), changing population demographics and programs that impact family planning decisions can influence incidence rates. Thus, previously reported incidence rates in Canada and the United States (US) may be outdated. The objectives of this study were to estimate contemporary CF incidence rates in Canada and the US and to determine if the incidence rate has changed over time.

Genomic epidemiology of Mycobacterium abscessus in a Canadian cystic fibrosis centre.

The Mycobacterium abscessus complex causes significant morbidity and mortality among patients with Cystic Fibrosis (CF). It has been hypothesized that these organisms are transmitted from patient to patient based on genomics. However, few studies incorporate epidemiologic data to confirm this hypothesis.

Protocol for establishing a core outcome set for evaluation in studies of pulmonary exacerbations in people with cystic fibrosis.

Introduction: Pulmonary exacerbations are associated with increased morbidity and mortality in people with cystic fibrosis (CF). There is no consensus about which outcomes should be evaluated in studies of pulmonary exacerbations or how these outcomes should be measured. Outcomes of importance to people with lived experience of the disease are frequently omitted or inconsistently reported in studies, which limits the value of such studies for informing practice and policy.

Beyond borders: cystic fibrosis survival between Australia, Canada, France and New Zealand.

Background: Life expectancy for people with cystic fibrosis (CF) varies considerably both within and between countries. The objective of this study was to compare survival among countries with single-payer healthcare systems while accounting for markers of disease severity.

Methods: This cohort study used data from established national CF registries in Australia, Canada, France and New Zealand from 2015 to 2019.

Frailty predicts outcomes in cystic fibrosis patients listed for lung transplantation.

Background: Survival predictors are not established for cystic fibrosis (CF) patients listed for lung transplantation (LT). Using the deficit accumulation approach, we developed a CF-specific frailty index (FI) to allow risk stratification for adverse waitlist and post-LT outcomes.

Methods: We studied adult CF patients listed for LT in the Toronto LT Program (development cohort 2005-2015) and the Swiss LT centres (validation cohort 2008-2017).

Hemoptysis and the Risk for Lung Transplant or Death without Transplant in Individuals with Cystic Fibrosis in the United States.

Hemoptysis is a common and important complication in persons with cystic fibrosis (PwCF). Despite this, there is limited literature on the impact of hemoptysis on contemporary cystic fibrosis (CF) outcomes. Evaluate whether hemoptysis increases the risk of lung transplant or death without a transplant in PwCF.

Factors associated with clinical progression to severe COVID-19 in people with cystic fibrosis: A global observational study.

Background: This international study aimed to characterise the impact of acute SARS-CoV-2 infection in people with cystic fibrosis and investigate factors associated with severe outcomes. Methods Data from 22 countries prior to 13 December 2020 and the introduction of vaccines were included. It was de-identified and included patient demographics, clinical characteristics, treatments, outcomes and sequalae following SARS-CoV-2 infection.

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 FEV ≤ 50% Predicted.

Background: Improved methods are needed to risk-stratify patients with cystic fibrosis (CF) and reduced FEV.

Research Questions: What are the predictors of death or lung transplantation (LTx) within 2 years among patients with CF whose FEV ≤ 50% predicted? Do these markers similarly predict outcomes among G551D patients taking ivacaftor since 2012?

Study Design And Methods: Patients with CF, age ≥ 6 years with FEV ≤ 50% predicted as of December 31, 2014, were identified in a data set that merged Cystic Fibrosis Foundation and United Network for Organ Sharing (UNOS) registries. The least absolute shrinkage and selection operator (LASSO) method was applied to a randomly selected training set to select important prognostic variables.

Caution advised in the use of CFTR modulator treatment for individuals harboring specific CFTR variants.

In December 2020, the U.S. Food and Drug Administration (FDA) expanded the list of CFTR variants approved for treatment with CFTR modulators drugs from 39 to 183.