scholarly journals Cystic Fibrosis Diagnostic Challenges over 4 Decades: Historical Perspectives and Lessons Learned

2017 ◽  
Vol 181 ◽  
pp. S16-S26 ◽  
Author(s):  
Philip M. Farrell ◽  
Terry B. White ◽  
Nico Derichs ◽  
Carlo Castellani ◽  
Beryl J. Rosenstein
Keyword(s):  
Cystic Fibrosis ◽  
Lessons Learned ◽  
Historical Perspectives

scholarly journals Gene Therapy for Cystic Fibrosis: Lessons Learned and Paths Forward

2021 ◽  
Vol 29 (2) ◽  
pp. 428-430
Author(s):  
Soon H. Choi ◽  
John F. Engelhardt
Keyword(s):  
Cystic Fibrosis ◽  
Gene Therapy ◽  
Lessons Learned

scholarly journals CFTR: cystic fibrosis and beyond

2014 ◽  
Vol 44 (4) ◽  
pp. 1042-1054 ◽  
Author(s):  
Marcus A. Mall ◽  
Dominik Hartl
Keyword(s):  
Cystic Fibrosis ◽  
Lung Disease ◽  
Lessons Learned ◽  
Chronic Obstructive ◽  
Common Mutation ◽  
Transmembrane Conductance Regulator ◽  
Obstructive Pulmonary Disease ◽  
Airway Diseases ◽  
Δf508 Cftr

Cystic fibrosis (CF) remains the most common fatal hereditary lung disease. The discovery of the cystic fibrosis transmembrane conductance regulator (CFTR) gene 25 years ago set the stage for: 1) unravelling the molecular and cellular basis of CF lung disease; 2) the generation of animal models to study in vivo pathogenesis; and 3) the development of mutation-specific therapies that are now becoming available for a subgroup of patients with CF. This article highlights major advances in our understanding of how CFTR dysfunction causes chronic mucus obstruction, neutrophilic inflammation and bacterial infection in CF airways. Furthermore, we focus on recent breakthroughs and remaining challenges of novel therapies targeting the basic CF defect, and discuss the next steps to be taken to make disease-modifying therapies available to a larger group of patients with CF, including those carrying the most common mutation ΔF508-CFTR. Finally, we will summarise emerging evidence indicating that acquired CFTR dysfunction may be implicated in the pathogenesis of chronic obstructive pulmonary disease, suggesting that lessons learned from CF may be applicable to common airway diseases associated with mucus plugging.

scholarly journals Developing a decision aid to facilitate patient-provider conversations about mechanical ventilation and lung transplantation to treat cystic fibrosis: lessons learned from usability testing (Preprint)

2020 ◽  
Author(s):  
Katherine L. Dauber-Decker ◽  
Melissa Basile ◽  
D'Arcy King ◽  
Jennifer Polo ◽  
Karina Calise ◽  
...  
Keyword(s):  
Cystic Fibrosis ◽  
Advance Care Planning ◽  
Decision Aid ◽  
Usability Testing ◽  
Lessons Learned ◽  
Care Planning ◽  
Think Aloud ◽  
System Usability Scale ◽  
Average System ◽  
Advance Care

BACKGROUND Cystic fibrosis (CF) is a life-limiting genetic disease that causes chronic lung infections. We developed an internet-based decision aid (decision aid) to help CF patients make better informed decisions regarding treatments and advance care planning (ACP). We built the decision aid around two major treatment decisions: whether or not to have a lung transplant, and whether or not to agree to intubation. OBJECTIVE To present results from iterative usability testing of the InformedChoices Cystic Fibrosis advance care planning decision aid among adults with cystic fibrosis, their clinicians and family caregivers. METHODS We performed a patient needs assessment and “think aloud” usability testing with CF patients, their surrogates, and clinicians caring for CF patients. “Think aloud” participants provided feedback while navigating the decision aid and answered surveys. Session transcripts and survey results were categorized into common, generalizable themes and optimizations for improving content, comprehension, and navigation. RESULTS Participants gave the decision aid an average System Usability Scale (SUS) score of 88.33 (“excellent”). Comments were coded into three themes: Functionality, Visibility and Navigation, and Content and Usefulness. Areas for improvement included reducing repetition, enhancing comprehension, and changing the flow. CONCLUSIONS Usability testing revealed areas for potential improvement. Testing also yielded positive feedback, suggesting the decision aid’s future success.Integrating changes prior to implementation should improve the decision aid’s comprehension, navigation, and usefulness, and lead to greater adoption.

scholarly journals Lessons learned from the cystic fibrosis pig

2016 ◽  
Vol 86 (1) ◽  
pp. 427-432 ◽  
Author(s):  
David K. Meyerholz
Keyword(s):  
Cystic Fibrosis ◽  
Lessons Learned
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