Publications

Authors: Luo, N.M.; Fink, A.; Sabadosa, K.

Background: As CFTR modulators are used by increasing numbers of CF patients, our ability to track impact on health and well-being, as well as to understand the most-effective combination of treatments for each individual patient, is limited without systematically collecting patient- and family-reported data. Folia is a cloud-based platform that enables the capture of home-reported outcomes (HROs) by patients and family caregivers (PFC). Folia has been previously piloted in CF programs in Maine Medical Center, Dell Medical School, and University of Vermont Medical Center. Users select the data elements they choose to collect, including symptom presence, severity, characteristics; follow-through with treatments; biometrics; problematic behavior frequency; and wellness indicators. Folia utilizes multiple-choice questions to simplify data capture. Data can be reported to a clinical team in a PDF at or before an appointment.

Objectives: (1) Determine optimal strategies for recruiting study participants; (2) Assess consistency of data entry throughout study; (3) Measure PFC satisfaction with app-based study participation; (4) Understand to what extent current users of CFTR modulators have made other changes to their treatment regimens.

Methods: We will use three arms for study recruitment: 1) current users of Folia; 2) clinic-based enrollment - individuals will be provided information about Folia and this study from their CF care center; 3) ambassador-based enrollment - PFC will share information about Folia and the study on social media. Once individuals are enrolled in Folia and onsent to participate in the study, they will be asked to complete questions regarding use of daily CF treatments, including: airway clearance; CFTR modulators; dornase alfa; hypertonic saline; and inhaled antibiotics. Study follow-up will be six weeks, and data will be linked to the CF Foundation Patient Registry. At the end of the study, each participant will complete a survey to assess their satisfaction with the app, the feasibility of conducting a long-term study using Folia, and whether having this tracked data impacted their behavior and care.

Results: Enrollment metrics will be 1) percentage of current Folia users who agree to provide their data for this research project; 2) percentage of individuals who receive information about Folia from their clinic who enroll in the study; 3) number of users who enroll in Folia from an ambassador. Among individuals enrolled, we will stratify by enrollment type and examine 1) the distribution of the number of days they enter data into Folia; 2) the percentage of people with variability in treatment regimens during the follow-up period; 3) key reasons reported for varying treatment regimens; and 4) satisfaction with Folia

Conclusions: Technology provides an opportunity to enrich our research with data elements provided real-time by individuals with CF and their caregivers. This study will help us understand the feasibility of using Folia to conduct a real-world research study of treatment withdrawal in the era of highly effective CFTR modulators.