Cystic fibrosis year in review 2023.

This past year, there were many important advances for patients with cystic fibrosis (CF). Of the many publications related to CF in 2023, there was further evaluation of highly effective modulator therapy, new assessments and guidelines for clinical manifestations and therapies for CF, advances in newborn screening and diagnosis, and evaluation of outcomes for people with CF transmembrane conductance regulator-related metabolic syndrome/CF screen positive, inconclusive diagnosis. The aim of this review article is not to provide a full assessment of the wide range of articles published in 2023, but to provide a brief review of publication that may lead to changes in clinical care.

Cystic fibrosis year in review 2022.

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.

Cystic fibrosis year in review 2021.

People with cystic fibrosis (CF) have an amazing outlook with the treatment availability of highly effective modulators. Unfortunately, not all people with CF are eligible for modulators leading to continued pulmonary exacerbations and advanced lung disease. Additionally, optimizing diagnosis and evaluation for CF in the newborn period continues to be an area of focus for research.

Review of CFTR modulators 2020.

Cystic fibrosis transmembrane conductance regulator (CFTR) modulators are small molecules that directly impact the CFTR protein, improving the function of the CFTR chloride and bicarbonate channel. Beginning in 2012 with the Food and Drug Administration approval of the first CFTR modulator, ivacaftor, this class of medications has had largely positive effects on many outcomes in people with cystic fibrosis (PwCF), including lung function, growth, and other clinical parameters. There have been continued exciting developments in the current research on CFTR modulators, expanding beyond original studies.

Year in Review 2020: Multisystemic impact of cystic fibrosis.

Clinical care in cystic fibrosis (CF) has continued to advance over the last several years, particularly with the widespread eligibility and use of highly effective modulator therapy. Improved outcomes and longevity of persons with CF (PwCF) have increased recognition of the multisystem impact of the disease on the daily lives of PwCF. This review will cover a broad array of topics, from diagnosis to multisystem effects related to mental health, endocrine, palliative care, reproductive health, otolaryngology, and cardiac issues.

Year in review 2020: Nutrition and gastrointestinal disease in cystic fibrosis.

The multisystemic manifestations of cystic fibrosis (CF) involve all parts of the gastrointestinal (GI) system, including the pancreas, intestine, and liver. As providers who care for people with CF, knowledge of the manifestations, treatment, and research related to nutrition and GI disease are important. This review is the last installment of the CF year in review 2020 series, focusing on nutritional, GI, and hepatobiliary articles from Pediatric Pulmonology and other journals of particular interest to clinicians.

Cystic fibrosis year in review 2020: Section 2 pulmonary disease, infections, and inflammation.

The outlook for those with cystic fibrosis (CF) has never been brighter with ever increasing life expectancy and the approval of the highly effective CFTR modulators, such as elexacaftor/tezacaftor/ivacaftor. With that being said, the progressive pulmonary decline and importance of lung health, infection, and inflammation in CF remains. This review is the second part in a three-part CF Year in Review 2020.

Systematic depression and anxiety screening for patients and caregivers: implementation and process improvement in a cystic fibrosis clinic.

Introduction: Depression and anxiety are common. Rates are significantly higher in cystic fibrosis (CF), and impact health outcomes. Screening is recommended, but is difficult to implement/sustain annually in a busy CF centre.

Cystic fibrosis year in review 2019: Section 3 multisystem-based care and research.

During the year 2019, numerous research studies and other reports were published that are interesting and instructive to professionals who care for people with cystic fibrosis (CF) and their families.  This report is the third of 3 CF year in review articles and focuses on the multisystem manifestations of CF care. Previous articles have addressed cystic fibrosis transmembrane regulator modulators and reports on CF pulmonary disease and airway infections.

Cystic fibrosis year in review 2019: Section 2 pulmonary disease and infections.

During the year 2019, research and case reports or series in the field of cystic fibrosis (CF) were in abundance. To adequately address the large body of CF research published during 2019, the CF year in review will be divided into three sections. This report is the second section, focusing specifically on new research related to pulmonary disease and infections.

Cystic fibrosis year in review 2019: Section 1 CFTR modulators.

During the year 2019, research and case reports/series in the field of cystic fibrosis (CF) were in abundance. To adequately address the large body of CF research published during 2019, the CF year in review will be divided into three sections. This report is the first section, focusing specifically on new research related to cystic fibrosis transmembrane conductance regulator modulator therapy.

Improved outcomes in cystic fibrosis using modified e-ducation of irway learance echnique (REACT) programme.

Background And Objectives: Cystic fibrosis (CF) is known to reduce lung function as measured by per cent predicted for the forced expiratory volume in the first second (ppFEV) over time. Our paediatric CF programme demonstrated significant gaps in benchmarked ppFEV predicted compared with the national median. Our objective was to assess whether the implementation of a modified e-ducation of irway learance echniques (REACT) programme could lead to an improvement in lung function as measured by ppFEV.

Cystic fibrosis diagnosed by state newborn screening: Or is it?

Newborn screening for cystic fibrosis is universal across the United States; however, each state chooses the method by which they screen. Illinois employs a two-step process which includes the measurement of the immunoreactive trypsinogen followed by an assay designed to detect 74 of the most common genetic mutations in the cystic fibrosis transmembrane conductance regulator protein. We report the case of an infant born in Illinois with a positive cystic fibrosis newborn screening with an elevated immunoreactive trypsinogen and two genetic mutations identified (F508del/F508del).

Tolerance of 7% Hypertonic Saline in Pediatric Cystic Fibrosis Patients.

Clinical trials of 7% hypertonic saline (HTS) in cystic fibrosis (CF) show short- and long-term benefits, including improved pulmonary function and reduced exacerbation risk. Adverse effects of HTS include bronchospasm, and guidelines recommend tolerance be tested in a clinical environment before prescribing. We evaluated the rate of, and risk factors for, intolerance of HTS at a single pediatric CF program.

Formative evaluation of a dashboard to support coproduction of healthcare services in cystic fibrosis.

Background: Healthcare coproduction engages patients and clinicians to design and execute services, yet little is known about tools that facilitate coproduction. Our objective was to understand uptake, experiences, benefits, and limitations of a dashboard to support patient-clinician partnerships within the cystic fibrosis (CF) community.

Methods: People living with CF (PwCF) and clinicians co-designed a dashboard that displayed patient-reported and clinical data.

Perceptions of safety monitoring in CF clinical studies and potential impact on future study participation.

Background: Individuals with CF and their parents cite safety concerns as barriers to participating in clinical studies. We assessed whether a brochure/infographic describing patient safety monitoring processes could reduce knowledge and attitude barriers regarding safety monitoring. We also identified factors associated with likely participation in future CF studies.

Cystic fibrosis year in review 2018, part 2.

Cystic fibrosis (CF) research and case reports were robust in the year 2018. This report summarizes publications related the multisystem effects of CF, pulmonary exacerbations, new and expanded therapies other than cystic fibrosis transmembrane conductance regulator modulator studies, and patient-reported priorities and outcomes.

Cystic fibrosis year in review 2018, part 1.

Cystic fibrosis research and case reports were robust in the year 2018. This report summarizes research and cases related to Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) modulator therapies, inflammation and infection, epidemiology and the physiologic, and imaging assessment of disease.

Cystic fibrosis year in review 2017.

In this article, we highlight cystic fibrosis (CF) reports published in Pediatric Pulmonology during 2017. We also include articles from a variety of journals that are related or are of special interest to clinicians.

Cystic fibrosis year in review 2016.

In this article, we highlight cystic fibrosis (CF) research and case reports published in Pediatric Pulmonology during 2016. We also include articles from a variety of journals that are thematically related to these articles, or are of special interest to clinicians.

Pediatric Pulmonology year in review 2015: Part 4.

In this article, we highlight cystic fibrosis (CF) research published in Pediatric Pulmonology during 2015. Articles from other journals that reflect similar themes, and those of special importance, are also included. Pediatr Pulmonol.

2014 year in review: Cystic fibrosis.

In this article, we highlight cystic fibrosis (CF) research published in Pediatric Pulmonology during 2014, as well as related articles published in other journals.