Understanding patient (pt) experiences, including disease- and treatment-related symptoms, physical functioning, and other aspects of daily life, is increasingly important when evaluating the benefits and risks of new therapies. The Journey PRO study is a proof-of-concept study designed to evaluate the feasibility of using a smartphone app to assess pts’ daily experience and its potential applicability in a clinical trial setting. The app captures the burden of chronic anemia and its impact on quality of life (QoL) in pts with MDS, BT, and MF.
To explore the feasibility and user acceptance of using the Journey PRO smartphone app to collect QoL data from pts with chronic anemia.
Pts with anemia due to MDS, BT, or MF were enrolled online and in parallel into 1 of 3 cohorts (n = 100 pts/cohort planned) based on their disease. Using the Journey PRO Apple iPhone app, pts completed a daily 3-question survey, as well as weekly pt-reported outcome (PRO) instruments (SF-12v2®, subsections of FACIT-Fatigue [all pts]; NTDT-PRO© [non-transfusion-dependent BT pts]; Myeloproliferative Neoplasm Symptom Assessment Form [MPN-SAF] total symptom score [TSS] [MF pts]), cognitive tests, and performance outcomes (6-min walk test). Pts also provided health-related information (e.g. transfusions received, medication changes). Passive measurements such as heart rate and daily number of steps completed were collected via the Apple Watch. Once enrolled, pts completed these activities for ≤12 weeks, with the opportunity to continue using the app for their own benefit. Pt feedback was captured using semi-structured interviews and the validated System Usability Scale (SUS) and User Burden Scale (UBS) surveys.
In the first 6 months, 42 pts had enrolled (12 with MDS, 28 with BT, and 2 with MF). Median ages were 68, 37, and 41 years, and 55%, 30%, and 100% were men, respectively. Overall, pts performed 3,343 unique activities, including answering surveys, performing cognitive tests, or contributing passive data. Around 70% of pts provided data in Week 2, 50% in Week 6, and 25% in Week 12; a small group of pts participated beyond Week 12. The daily surveys were the most consistently performed items with 37 pts (88%) completing ≥1 survey by Week 12. Daily compliance with the survey was 45% in Week 1 and decreased to 25% at the end of Week 2. Strong internal correlations were found across PROs collected with the app. Although correlations between survey- and activity-based PROs were not statistically significant due to the limited sample size, consistency across PROs and activity-based measures suggests the validity of the PRO data collected with the app. Pts found the app easy to use and valuable to track symptoms. SUS scores were above the industry average (86/100 vs 68/100, respectively) (Figure). UBS results suggested that the app did not pose a financial, time, or social burden.
The Journey PRO app is designed to provide an in-depth assessment of pts’ experience of their disease and treatment. Our results indicate that the frequent data collection from activities with the app was well received by the pts. This encourages the use of such apps in clinical trials. Limitations of this study were related to pt attrition and reduced compliance with activities, which were probably due to the burden of anemia. Activities to boost continued participation and enrollment fostered through clinical trials in future studies may help to promote the value and benefit of the Journey PRO app from a clinical trial perspective.