The use of PROs has increased significantly in cystic fibrosis (CF) research and care over the past 20 years [6]. The Cystic Fibrosis Questionnaire-Revised (CFQ-R) is the most commonly used and best validated disease-specific PRO [7,8,9]. The CFQ-R has been validated (i.e. demonstrated reliability, validity, and sensitivity) and has been translated into 38 languages [8], and has been used in clinical trials evaluating medications with differing mechanisms of action (e.g., hypertonic saline, inhaled antibiotics, CFTR correctors-modulators). Importantly, it was developed using the FDA Guidance (2009), including qualitative phases, which elicited patient, parent and provider input. It was designed to systematically measure respiratory and gastrointestinal symptoms, treatment burden, and daily functioning.
Four validated versions of the CFQ-R were adapted for electronic administration in this study: 1) self-report for adolescents and adults, ages 14 years and older (CFQ-R Teen/Adult); 2) self-report for children, ages 12–13; 3) interviewer-administered version for children ages 6 to 11; and 4) parent proxy version for caregivers of children, ages 6 to 13 years.
The code of the application is open source and can be found at https://github.com/CFQR where continuous integration is achieved with Travis CI. The CFQ-R-application is a React web application (HTML, CSS, Javascript) built with ReactJS framework. The CFQ-R application is freely available at https://cfqr.github.io and is made to be run from different environments: It can be used online, downloaded as a desktop application for offline use (Windows, Mac, Linux), and is also available as a mobile application for iOS and Android platforms (from popular application stores).
Besides automatically calculating the scores from the different domains obtained by the CFQ-R, saving time, and avoiding miscalculations, the CFQ-R application has several other functionalities. It can be used to visually represent longitudinal trajectories of CFQ-R scores (Fig. 1), which may be helpful for the clinician. It also allows data export for downstream statistical processing for the researcher. The application can store data in different ways. Data can be stored locally on the tablet or PC used by the user. The user may also wish to connect the device to the Internet to allow automatic backup in a cloud (using the Google service Firebase). The latter function will enable synchronized data capture from multiple devices (e.g. in larger clinical trial). CFQ-R’s in eight languages have currently been included (i.e. Danish, Dutch, English, German, Italian, French, Spanish, and Swedish). Other languages can be added on request.
As an additional perspective, users of non-CF PROs measures may wish to use codes from this application as a template to make similar tools that can be used for other respiratory diseases, such as primary ciliary dyskinesia and non-CF bronchiectasis. Although a simpler desktop tool for the St. George’s Respiratory Questionnaire was previously developed [10], we are not aware of the existence of similar applications for other respiratory PROs.
This application has some limitations. First, the user should be aware that data collection has to comply with the rules established by local ethics committees. Second, although a meta-analysis of PROs has concluded that there is no bias attributable to paper vs. electronic completion of a large number of PROs [11], we have not studied the user experience for this application of the CFQ-R.