StudyU: A Platform for Designing and Conducting Innovative Digital N-of-1 Trials.

N-of-1 trials are the gold standard study design to evaluate individual treatment effects and derive personalized treatment strategies. Digital tools have the potential to initiate a new era of N-of-1 trials in terms of scale and scope, but fully functional platforms are not yet available. Here, we present the open source StudyU platform, which includes the StudyU Designer and StudyU app. With the StudyU Designer, scientists are given a collaborative web application to digitally specify, publish, and conduct N-of-1 trials. The StudyU app is a smartphone app with innovative user-centric elements for participants to partake in trials published through the StudyU Designer to assess the effects of different interventions on their health. Thereby, the StudyU platform allows clinicians and researchers worldwide to easily design and conduct digital N-of-1 trials in a safe manner. We envision that StudyU can change the landscape of personalized treatments both for patients and healthy individuals, democratize and personalize evidence generation for self-optimization and medicine, and can be integrated in clinical practice.

Digital Therapeutic Care and Decision Support Interventions for People With Low Back Pain: Systematic Review.

BACKGROUND: Low back pain (LBP) is the leading cause of worldwide years lost because of disability, with a tremendous economic burden for health care systems. Digital therapeutic care (DTC) programs provide a scalable, universally accessible, and low-cost approach to the multidisciplinary treatment of LBP. Moreover, novel decision support interventions such as personalized feedback messages, push notifications, and data-driven activity recommendations amplify DTC by guiding the user through the program while aiming to increase overall engagement and sustainable behavior change. OBJECTIVE: This systematic review aims to synthesize recent scientific literature on the impact of DTC apps for people with LBP and outline the implementation of add-on decision support interventions, including their effect on user retention and attrition rates. METHODS: We searched bibliographic databases, including MEDLINE, Cochrane Library, Web of Science, and the Physiotherapy Evidence Database, from March 1, 2016, to October 15, 2020, in accordance with the PRISMA (Preferred Reporting Items for Systematic Reviews and Meta-Analyses) guidelines and conducted this review based on related previously published systematic reviews. Besides randomized controlled trials (RCTs), we also included study designs with the evidence level of at least a retrospective comparative study. This enables the consideration of real-world user-generated data and provides information regarding the adoption and effectiveness of DTC apps in a real-life setting. For the appraisal of the risk of bias, we used the Risk of Bias 2 Tool and the Risk of Bias in Non-Randomized Studies of Interventions Tool for the RCTs and nonrandomized trials, respectively. The included studies were narratively synthesized regarding primary and secondary outcome measures, DTC components, applied decision support interventions, user retention, and attrition rates. RESULTS: We retrieved 1388 citations, of which 12 studies are included in this review. Of the 12 studies, 6 (50%) were RCTs and 6 (50%) were nonrandomized trials. In all included studies, lower pain levels and increased functionality compared with baseline values were observed in the DTC intervention group. A between-group comparison revealed significant improvements in pain and functionality levels in 67% (4/6) of the RCTs. The study population was mostly homogeneous, with predominantly female, young to middle-aged participants of normal to moderate weight. The methodological quality assessment revealed moderate to high risks of biases, especially in the nonrandomized trials. CONCLUSIONS: This systematic review demonstrates the benefits of DTC for people with LBP. There is also evidence that decision support interventions benefit overall engagement with the app and increase participants' ability to self-manage their recovery process. Finally, including retrospective evaluation studies of real-world user-generated data in future systematic reviews of digital health intervention trials can reveal new insights into the benefits, challenges, and real-life adoption of DTC programs.

Mobile app requirements for patients with rare liver diseases: A single center survey for the ERN RARE-LIVER‬‬‬.

BACKGROUND: More patient data are needed to improve research on rare liver diseases. Mobile health apps enable an exhaustive data collection. Therefore, the European Reference Network on Hepatological diseases (ERN RARE-LIVER) intends to implement an app for patients with rare liver diseases communicating with a patient registry, but little is known about which features patients and their healthcare providers regard as being useful. AIMS: This study aimed to investigate how an app for rare liver diseases would be accepted, and to find out which features are considered useful. METHODS: An anonymous survey was conducted on adult patients with rare liver diseases at a single academic, tertiary care outpatient-service. Additionally, medical experts of the ERN working group on autoimmune hepatitis were invited to participate in an online survey. RESULTS: In total, the responses from 100 patients with autoimmune (n = 90) or other rare (n = 10) liver diseases and 32 experts were analyzed. Patients were convinced to use a disease specific app (80%) and expected some benefit to their health (78%) but responses differed significantly between younger and older patients (93% vs. 62%, p < 0.001; 88% vs. 64%, p < 0.01). Comparing patients' and experts' feedback, patients more often expected a simplified healthcare pathway (e.g. 89% vs. 59% (p < 0.001) wanted access to one's own medical records), while healthcare providers saw the benefit mainly in improving compliance and treatment outcome (e.g. 93% vs. 31% (p < 0.001) and 70% vs. 21% (p < 0.001) expected the app to reduce mistakes in taking medication and improve quality of life, respectively). CONCLUSION: Our results underline the great desire for disease-specific apps but also the need to involve patients and healthcare providers in the development of such apps in order to achieve long-term use and, thereby, improvements of patient care and research. The results of this study will help tremendously to implement the first cross-country app that communicates with an ERN patient registry.

The pain of being misunderstood: Invalidation of pain complaints in chronic low back pain patients.

A particular form of social pain is invalidation. Therefore, this study (a) investigates whether patients with chronic low back pain experience invalidation, (b) if it has an influence on their pain, and (c) explores whether various social sources (e.g. partner and work) influence physical pain differentially. A total of 92 patients completed questionnaires, and for analysis, Pearson's correlation coefficients and hierarchical linear regression analyses were conducted. They indicated a significant association between discounting and disability due to pain (respective ß = .29, p > .05). Especially, discounting by partner was linked to higher disability ( ß = .28, p > .05).