Early detection of rapid cystic fibrosis disease progression tailored to point of care: A proof-of-principle study

Abstract

Slowing cystic fibrosis (CF) lung disease progression is crucial to survival, but point-of-care technologies aimed at early detection—and possibly prevention—of rapid lung function decline are limited. This proof-of-principle study leverages a rich national patient registry and follow-up data on a local CF cohort to build an algorithm and prototype prognostic tool aimed at early detection of rapid lung function decline. The algorithm was developed using a novel longitudinal analysis of lung function (measured as forced expiratory volume in 1 s of % predicted, FEV1). Covariates included clinical and demographic characteristics selected from the registry based on information criterion. Preliminary assessment of algorithm performance suggested excellent predictive accuracy and earlier detection of rapid decline than standard of care being applied at a local center. Graphical displays were presented and evaluated for clinical utility. Predictions from the algorithms and chosen graphical displays were translated into a prototype web application using RShiny and underwent iterative development based on clinician feedback. This paper suggests that the algorithm and its translation could offer a means for earlier detection and treatment of rapid decline, providing clinicians with a viable point-of-care technology to intervene prior to irreversible lung damage.

Improving Detection of Rapid Cystic Fibrosis Disease Progression–Early Translation of a Predictive Algorithm Into a Point-of-Care Tool

“…Clinician feedback and prototype refinements were performed iteratively based on application mock-ups of clinical dashboards. Earlier developments were presented and reported as conference proceedings [19].…”

Assessment of Distinct Electrophysiological Parameters in Rectal Biopsies for the Choice of the Best Diagnosis/Prognosis Biomarkers for Cystic Fibrosis

“…The hallmark of the clinical course of CF disease is progressive loss of lung function with eventual respiratory failure. In the context of CF, lung function is perhaps the most prominent measure of disease severity, progression, and therapeutic efficacy ( Szczesniak et al, 2017 ). The primary spirometry result of interest in CF is FEV 1 , an index of airway obstruction that has played a critical role in both clinical care and research.…”

“…The primary spirometry result of interest in CF is FEV 1 , an index of airway obstruction that has played a critical role in both clinical care and research. Over the past 50 years, FEV 1 decline has been associated with morbidity and mortality among PwCF ( Liou et al, 2001 ), as well as with greater risk of pulmonary exacerbation, hospitalizations and with P. aeruginosa colonization ( Szczesniak et al, 2017 ), and therefore correlations with FEV 1 , as well as with FVC, were analyzed in this work. Our data show that the previously validated parameter I’ sc–I/F/C evidenced a lower correlation with FEV 1 measurements than V I/F+I/F/C (27 vs. 32%) ( Table 1 ).…”

Stakeholder feedback informs investigations for chronic rhinosinusitis in cystic fibrosis

“…Incorporation of stakeholder feedback supports engaging individuals representative of the population of interest and other relevant stakeholders in ways that can include study design, outcome measure selection, subject recruitment strategies, and interpretation and/or distribution of study findings 7 . Stakeholder engagement and incorporation of feedback has been successfully employed in multiple fields, including detecting pulmonary disease progression in CF, 8 developing a protocol to improve pediatric asthma outcomes, 9 and engaging patients and family members to improve outcomes of healthcare information technology initiatives 10 …”