Monitoring Of Patient-reported Outcomes Research Articles

Electronic Patient-Reported Outcome System Implementation in Outpatient Cardiovascular Care

The integration of patient-reported outcome (PRO) assessments in cardiovascular care has encountered considerable obstacles despite their established clinical relevance. To assess the impact of a physician- and patient-friendly electronic PRO (ePRO) monitoring system on the quality of cardiovascular care in clinical practice. This open-label, multicenter, pilot randomized clinical trial was phase 2 of a multiphase study that was conducted from October 2022 to October 2023 and focused on the implementation and evaluation of an ePRO monitoring system in outpatient clinics in Japan. During phase 1 (conducted from December 2021 to March 2022), this ePRO monitoring system was developed at a single outpatient center using feedback from patients and physicians. Eligible patients were older than 18 years with a clinical diagnosis of heart failure (HF), atrial fibrillation (AF), or coronary artery disease (CAD). Participants were randomly assigned 1:1 to either the ePRO or control group. Statistical analysis was based on the intention-to-treat approach. Patients in the ePRO group were asked to complete the 12-item Kansas City Cardiomyopathy Questionnaire (KCCQ-12), Atrial Fibrillation Effect on Quality-of-Life Questionnaire (AFEQT), or Seattle Angina Questionnaire (SAQ) on smart tablets before their clinical examination. Results were reviewed by physicians at each outpatient visit. The control group received usual care consisting of medication management, lifestyle modification, and invasive procedures in accordance with clinical guidelines. Both groups had 5 monthly outpatient visits. Patient satisfaction, quality of information (QOI) provided by physicians, and disease knowledge were assessed using questionnaires (eg, Patient Satisfaction Questionnaire [PSQ]; score range: 5-25, with higher scores indicating greater patient satisfaction) at baseline and the fifth visit, over approximately 4 months. Of the 50 patients included, 48 (median [SD] age, 71.0 [62.3-75.0] years; 28 males [58.3%]) completed follow-up. The prevalence was 56.2% (n = 27) for HF, 75.0% (n = 36) for AF, and 10.4% (n = 5) for CAD. The distribution of PRO measures used was 21 patients (43.8%) for KCCQ-12, 24 (50.0%) for AFEQT, and 3 (6.2%) for SAQ. Compared with the control group, the ePRO group showed significant improvements in mean (SD) PSQ score (0.16 [2.06] vs 1.61 [1.75]; P = .01) and QOI score regarding treatment (-0.12 [0.53] vs 0.35 [0.71]; P = .01). The ePRO group exhibited a greater increase in the PSQ score regarding communication (-0.12 [0.53] vs 0.43 [0.90]; P = .01). In this randomized clinical trial, implementation of the ePRO monitoring system significantly enhanced patient-physician communication and the clarity of physicians' explanations about treatment. These findings suggest that the ePRO monitoring system is capable of supporting patient-centered cardiovascular care. University Hospital Medical Information Network Identifier: UMIN000049251.

Changes in Recovery Capital Among Patients Receiving Buprenorphine Treatment for Opioid Use Disorder in a Telehealth Setting.

Medications for the treatment of opioid use disorder (MOUD) such as buprenorphine are the most effective treatment available for OUD; yet, beyond drug testing results and retention in care, systematically measured clinical outcomes have proven elusive. There is growing interest in integrating systematic monitoring of patient-reported outcomes and measurement-based care as strategies to improve patients' success in treatment. We analyzed changes in recovery capital assessed via the Brief Assessment of Recovery Capital (BARC-10) from baseline to 30-120 days post-intake among patients initiating buprenorphine treatment from May to October 2023 at Ophelia, a telehealth MOUD provider, who were retained for ≥90 days. Differences in baseline characteristics were assessed between patients with and without high "remission-predictive" baseline scores (≥47) using chi-squared and t-tests. Changes in scores from baseline to follow-up were assessed using paired t-tests. In all, 791 patients initiated treatment during the study period, 742 (93.8%) of whom had a baseline BARC-10 score, 542 (73.0%) of whom were retained in treatment for ≥90 days, and 477 of whom (88.0%) had a follow-up BARC-10 score and represent the analysis sample. Older patients, those not requiring buprenorphine induction, and those not using heroin or fentanyl at intake were more likely to have remission-predictive baseline BARC-10 scores (P < .05). Patients with remission-predictive baseline scores (n = 257) had a mean increase of 1.4 (SD = 5.9) from 52.7 (SD = 4.1) (P < .001), and 234 (91%) sustained remission-predictive scores throughout the assessment period. Patients without remission-predictive baseline scores (n = 220) had a mean increase of 9.2 (SD = 8.2) from 38.4 (SD = 6.6) (P < .001), and 129 (59.0%) achieved a remission-predictive score at follow-up. Most patients had increased or sustained already high levels of recovery capital, an established predictor of sustained remission. Further research is required to better understand variability across patients and how it may relate to long-term outcomes.

An electronic patient-reported outcome symptom monitor: the Chinese experience with rapid development of a ready-to-go symptom monitor

BackgroundMonitoring symptoms is crucial for the early detection of disease progression and timely intervention, which is essential for reducing severe cases and mortality rates in rapidly spreading pandemics, such as COVID-19. Therefore, during infectious disease pandemics, the rapid development of real-time symptom monitoring platforms is essential. This study aimed to explore the urgent development process of an electronic system for patient-reported outcome monitoring in emergency situations.MethodsThe development of the electronic patient-reported outcome COVID-19 symptom monitoring platform (ePRO-CoV-SM) included the following steps: (1) modifying an electronic patient-reported outcome symptom-reporting platform to assess patients with COVID-19 and validating its feasibility and sensitivity for longitudinal symptom measurement; (2) updating the system to accommodate the newly emerged severe acute respiratory syndrome coronavirus 2 BA.2.2 variant; and (3) applying it in real-world settings. Literature review, expert consultation, and subject-group discussions were used to develop symptom items. Response rate and missing item rate were used as validation indicators for ePRO-CoV-SM.ResultsThe ePRO-CoV-SM (2.0) consists of a core set of symptom items, a WeChat mini program, an online project design backend, a management and communication front, and a database. During the 2020 verification, the response rate of ePRO symptom monitoring reached 89.47% and the item missing rate was 0.33%, the monitoring revealed that a considerable number of asymptomatic patients were experiencing undesirable symptoms during the isolation period. In its real-world application in 2022, the response rate was 85.93% and the item missing rate was 4.84%, the monitoring found the symptom burden was higher in the younger group (18–40 years old) than in the older group (40–67 years old), and over 30% of patients reported symptoms such as cough (36.08%), dry mouth (35.67%), sleep disorders (32.27%), appetite loss (32.17%), and sputum (30.79%) during the isolation period.ConclusionsElectronic patient-reported outcome measurement was demonstrated to be sensitive and feasible for monitoring symptoms in patients with COVID-19. By integrating smartphone-based data collection with real-time online data transmission and secure data storage using Secure Sockets Layer encryption, an electronic platform for monitoring critical symptoms can be rapidly established in emergency situations.

WP1.15 - Advances in remote electronic monitoring of patient-reported outcomes following discharge after surgery: a systematic review

Abstract Aims Standardising post-discharge care is critical to improving surgical recovery. The COVID-19 pandemic caused major changes to in-person visits and surgical follow-up. An earlier review examined the pre-pandemic capture of electronic patient-reported outcomes (ePRO) post-discharge after surgery. This updated review explores how the pandemic has influenced the use of ePRO systems. Methods Databases (Medline/Embase/PsycINFO/CINAHL/Cochrane) were searched from July 2019-August 2023 using terms for ‘patient-reported outcomes’, ‘electronic’, ‘surgery’ and ‘at home’. Primary research studies collecting ePROs in adult surgical patients post-discharge were included. Data extraction/analyses characterised study design, patient characteristics, ePRO system features, the scope within which ePROs are used post-discharge, what PRO data is collected, adherence to ePRO monitoring, impact on primary/secondary outcomes and the patient and clinician experience of using ePRO systems. Results 12,805 articles were identified, of which 81 were included. Emerging findings indicate an increase in research examining ePRO systems in the post-discharge care of surgical patients, with ∼20 articles/year published post-pandemic, compared to ∼3/year in the 5 years pre-pandemic. Usage of ePROs seems to be most prevalent in orthopaedic and gastrointestinal surgery. Full findings will be available for presentation at the conference. Conclusions Interest in using ePROs to support post-discharge care of surgical patients is growing, in response to service provision changes. Important advances in the capabilities of ePRO systems include real-time clinician feedback and integration with hospital electronic records. The findings of this review will highlight important directions for improving the design of ePRO systems and future research examining the effectiveness of ePRO systems at improving post-surgical recovery.

Using a modified Delphi procedure to select a PRO-CTCAE-based subset for patient-reported symptomatic toxicity monitoring in rectal cancer patients.

Standardized patient-reported outcomes (PRO) monitoring during and after rectal cancer treatment provides insight into treatment-related toxicities patients experience and improves health-related quality-of-life as well as overall survival. We aimed to select a subset of the PRO version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE) for standardized monitoring of treatment-related symptomatic toxicities in rectal cancer. We used a mixed methods approach including a literature review, and semi-structured interviews with health care providers (HCPs) involved in rectal cancer care and rectal cancer patients. Results from literature and interviews were summarized and used in a modified Delphi procedure to select a PRO-CTCAE subset specific for rectal cancer. Twenty-six PRO-CTCAE symptomatic toxicities were identified from literature. Fifteen HCPs from multiple disciplines (medical, radiation and surgical oncology), and a heterogeneous group of fifteen rectal cancer patients treated with chemotherapy and/or radiotherapy and/or surgery, participated in semi-structured interviews. Ten HCPs (67%) and nine patients (90%) participated in the first Delphi round. The final selected PRO-CTCAE core-subset contained 16 symptomatic toxicities: 'diarrhea', 'fecal incontinence', 'constipation','bloating of the abdomen', 'pain in the abdomen', 'vomiting', 'decreased libido', 'pain during vaginal sex', 'ability to achieve and maintain erection', 'fatigue', 'anxiety', 'feeling that nothing could cheer you up', 'urinary incontinence', 'painful urination', 'general pain', and 'hand-foot syndrome'. Based on a comprehensive mixed methods study, a PRO-CTCAE subset for standardized treatment-related symptomatic toxicity monitoring in rectal cancer was identified. Assessment of the effectiveness and compliance of symptomatic toxicity monitoring using this subset is recommended.

PRO-WAVE1: Monitoring patient reported outcomes (PROs) in prostate cancer through Wave Health, a novel ePRO health platform.

192 Background: It is well established that the monitoring and evaluation of PROs in cancer patients has a positive impact in both quality of life and overall survival [Basch E., JAMA 2017]. In parallel, the widespread use of electronic devices such as smart phones in the general population have increased significantly over recent years [Statista, 2023], representing a unique opportunity to operationalize ePROs. However, there is uncertainty about the feasibility and applicability of these technologies in certain patient population such as prostate cancer (PC) patients. PRO-WAVE1 aims to determine the acceptability, engagement and satisfaction of the Wave Health patient app and healthcare provider portal, enhanced for PC patients. Methods: 98 patients across 8 sites in Spain enrolled in the study, and 82 of those patients actively engaged with the Wave Health App over a 13-week period. Each week, patients completed validated ePRO questionnaires (generating 93.3% compliance) consisting of: a weekly symptom review (WSR), FACT-P and CHLT-30-Dkspa. A report summary of the patients’ WSR was generated for care team view in the Wave Health Connect platform – specifically to conduct weekly check ins on patients. Further, an optional in-app feedback survey was administered once patients completed the study to measure overall app satisfaction, information usefulness, reduction in anxiety, and effectiveness of communication with medical teams. Results: In addition to unusually high compliance and perceived usefulness, there was a particularly high response rate for an optional end of study survey (78%), whereby 64 patients who completed the feedback survey reported remarkable experiences, including: 90% reported being satisfied with the app to track their activities and keep their medical team informed; 95% of patients described the information provided by Wave Health as “useful"; 6 in 10 patients indicated that using Wave Health helped them feel less anxious about their treatment experience; 75% of patients reported that Wave Health helped them track their health and keep their medical team informed. Correspondingly, patients without a caregiver rated the platform higher than those with a caregiver across all areas (information useful/relevancy, communication with care team, likeliness to recommend and overall satisfaction). Additionally, 90% of HCPs positively perceived the platform’s impact on the quality of communication with patients. Conclusions: The high levels of patient compliance, satisfaction, perceived usefulness, and anxiety reduction associated with the app, along with the high HCP perceived usefulness of the portal, underscore the opportunity to enable better treatment management between HCPs and patients - particularly for those without caregivers. A larger study with a comparator arm will help further evaluate impact on costs and clinical outcomes.

Patient-reported outcomes-the role of the patient's subjective perspective for research and clinical care

Because only patients can adequately assess symptoms, disability, and quality of life, concordance between apatient's and physician's assessment is often low. Accordingly, patient-reported outcomes (PROs) are increasingly used in research and routine clinical care. In daily practice, PROs are not only applied to measure the patient's perceived outcome of medical treatments, but also to assess their health status before intervention starts. Typically, several patient-reported outcome measures (PROMs), which are reliable and valid, are available for the assessment of the most important PROMs. In daily clinical practice, the integration of PROs can be useful for clinical assessment and treatment planning or for quality management. Currently, the most promising application is routine patient monitoring using digital PROMs (ePROMs). Systematic reviews have revealed that the routine use of PROMs in daily clinical care is associated with, among others, improved physician-patient communication, higher patient satisfaction, reduced symptom burden, higher quality of life, and improved survival. This effect is especially strong if health care professionals continuously receive the results of the PRO monitoring. Patients are usually inclined to disclose their health status, and the positive effects of routine patient monitoring are widely recognized. However, several barriers to using PROs and PROMs still exist.

PRO-WAVE1: Monitoring patient reported outcomes (PROs) in prostate cancer through Wave Health, a novel ePRO health platform.

e23190 Background: It is well established that the monitoring and evaluation of PROs in cancer patients has a positive impact in both quality of life and overall survival [Basch E., JAMA 2017]. In parallel, the widespread use of electronic devices such as smart phones in the general population have increased significantly over recent years [Statista, 2023], representing a unique opportunity to operationalize ePROs. However, there is uncertainty about the feasibility and applicability of these technologies in certain patient population such as prostate cancer (PC) patients. PRO-WAVE1 aims to determine the acceptability and engagement of the Wave Health patient app and healthcare provider portal, enhanced for PC patients. Specific endpoints include measuring longitudinal use of varying ePROs to impact care management, patient health literacy, and ability to identify and manage emerging risks. Methods: Patients across eight sites in Spain engaged with the Wave Health App over a 13-week period. Each week, patients completed a validated weekly symptom review (WSR) assessing 16 Prostate cancer-specific symptoms, including severity and/or frequency of each; as well as a health-related QoL (FACT-P) and health literacy survey (CHLT-30-Dkspa). A report summary of the patients’ WSR was generated for care team view in the Wave Health Connect platform – specifically to conduct weekly check ins on patients, including verifying patient completion; evaluating report content; indicating plans for follow up per patient; and rating their perceived value of the platform. Results: 98 patients were enrolled in the study – of which 82 actively participated in the study (84% acceptance). The compliance rate for the ePRO questionnaires was 93.3%, while during their participation, patients used the app for an average of 12.2 weeks, and approximately two-thirds (67.4%) used the app for all 13 weeks. Patient satisfaction with the platform was 93%, with 100% of respondents feeling the information provided in the app was relevant to their personal experience. Correspondingly, almost all HCPs (98%) rated the platform as useful – with the majority willing to recommend it to their peers and future patients; 90% of HCPs positively perceived the platform’s impact on the quality of communication with patients. Conclusions: Study results exceeded expectations for patient acceptability and engagement with the app; as well as perceived usefulness of the portal among HCPs – suggesting the platform as an impactful tool for patients working with HCPs to manage treatment journeys. A larger study using a comparator arm will help further evaluate the impact on costs and clinical outcomes.

A randomized study comparing electronic patient-reported outcome (ePRO) monitoring with routine follow-up during trastuzumab deruxtecan treatment in patients with metastatic breast cancer (PRO-DUCE study).

1504 Background: Trastuzumab deruxtecan (T-DXd) is a highly effective agent for HER2-positive metastatic breast cancer (MBC), but is associated with adverse events such as nausea, fatigue, and interstitial lung disease. Based on recent studies showing that symptom monitoring and alert notifications via ePROs could improve patient quality of life (QoL) and prognosis, we hypothesized that employing this method may diminish the treatment burden for patients receiving T-DXd. Methods: In this multicenter, randomized controlled exploratory study (jRCTs031200387), we randomized patients with HER2-positive MBC eligible for T-DXd to either the ePRO monitoring (ePROm) group or the usual care (UC) group. ePROm involved weekly symptom and daily body temperature/SpO2 reports via a smartphone or computer at home. If any symptoms exceeded the predetermined thresholds, an email alert was sent to the medical staff, the ePRO was evaluated, and, if necessary, a phone consultation was provided. The primary endpoint was the change in the Global QoL score of EORTC QLQ-C30 from baseline at week 24. Secondary endpoints included score changes in the functional and symptom scales of EORTC QLQ-C30, and the time to deterioration of Global QoL (defined as a 10-point decrease from baseline). For a score difference between groups of 10 points and a standard deviation of 24, the required number of patients was 55 in each group, with a two-sided alpha error of 10% and a power of 87%. We analyzed the primary endpoint using a mixed-effects model for repeated measures. Results: Between March 2021 and January 2023, 111 patients were enrolled at 38 hospitals in Japan, with 56 assigned to ePROm and 55 to UC; the full-analysis set for QoL assessment included 54 and 52 patients, respectively. During the 24-week period, there were 1223 ePRO reports (95% compliance rate) in the ePROm group, including 427 alert notifications (7.9 per patient) to healthcare providers. Overall compliance with the questionnaire for QoL was 97%. Mean changes from baseline scores in Global QoL at week 24 showed that ePROm was significantly better than UC (mean difference 8.0 [90% CI 0.2, 15.8]; p=0.091). Role, cognitive, and social functioning were better in ePROm, with mean differences of 10.0 (95% CI 1.1, 18.9; p=0.028), 6.3 (1.1, 11.5; p=0.017), and 10.9 (3.9, 18.0; p=0.003), respectively. There was no difference in nausea/vomiting (0.5 [- 6.2, 7.1]; p=0.889), while fatigue was significantly better in ePROm (-8.4 [-16.1, -0.6]; p=0.034). Median time to first deterioration in Global QoL score was 3.9 months (95% CI 2.5, 13.9) in ePROm and 3.0 months (95% CI 1.6, 6.7) in UC (hazard ratio 0.73 [95% CI 0.45, 1.17]; p=0.159). Conclusions: ePROm systems, especially for symptom and SpO2 tracking in HER2-positive MBC patients treated with T-DXd, are promising to enhance patient QoL. Clinical trial information: jRCTs031200387 .

A randomized trial of the impact of symptom monitoring using an electronic patient-reported outcome app on health-related quality of life in postmenopausal breast cancer patients receiving adjuvant endocrine therapy.

Electronic patient-reported outcomes (ePRO) monitoring is a useful communication tool for cancer patients and healthcare providers. In this study, we examined the impact of symptom monitoring using an ePRO app on quality of life (QoL) in postmenopausal breast cancer patients receiving adjuvant endocrine therapy. The free app "Welby My Carte ONC" was used in the study. Patients with breast cancer starting adjuvant endocrine therapy were randomly assigned in a 1:1 ratio to ePRO monitoring (ONC) and control groups. The ONC group reported five symptoms extracted from the Patient-Reported Outcome-Common Terminology Criteria for Adverse Events (PRO-CTCAE) (insomnia, joint pain, headache, anxiety, and hot flashes) weekly for 3months through the app. Reported symptoms were shared with medical personnel. When serious symptoms were reported, these personnel ascertained the patient's health status and provided advice over the phone. The primary endpoint was QoL measured by the Functional Assessment of Cancer Therapy-Breast (FACT-B) at 3months from enrollment. Differences between groups were tested using analysis of covariance. The study included 125 subjects with mean age of 64years in the ONC group (n = 61) and 63years in the control group (n = 64). In the ONC group, the response rate to PRO-CTCAE was about 70% or higher until week 10. The item missing rate was 0. The ONC group reported more symptoms related to joint pain and insomnia. The difference in FACT-B total score between the groups was -1.55 (95% confidence interval: -5.91, 2.81), indicating no significant difference. Symptom monitoring using ePRO early after initiation of adjuvant endocrine therapy after surgery did not improve QoL of breast cancer patients.

Association of Remote Patient-Reported Outcomes and Step Counts With Hospitalization or Death Among Patients With Advanced Cancer Undergoing Chemotherapy: Secondary Analysis of the PROStep Randomized Trial.

Patients with advanced cancer undergoing chemotherapy experience significant symptoms and declines in functional status, which are associated with poor outcomes. Remote monitoring of patient-reported outcomes (PROs; symptoms) and step counts (functional status) may proactively identify patients at risk of hospitalization or death. The aim of this study is to evaluate the association of (1) longitudinal PROs with step counts and (2) PROs and step counts with hospitalization or death. The PROStep randomized trial enrolled 108 patients with advanced gastrointestinal or lung cancers undergoing cytotoxic chemotherapy at a large academic cancer center. Patients were randomized to weekly text-based monitoring of 8 PROs plus continuous step count monitoring via Fitbit (Google) versus usual care. This preplanned secondary analysis included 57 of 75 patients randomized to the intervention who had PRO and step count data. We analyzed the associations between PROs and mean daily step counts and the associations of PROs and step counts with the composite outcome of hospitalization or death using bootstrapped generalized linear models to account for longitudinal data. Among 57 patients, the mean age was 57 (SD 10.9) years, 24 (42%) were female, 43 (75%) had advanced gastrointestinal cancer, 14 (25%) had advanced lung cancer, and 25 (44%) were hospitalized or died during follow-up. A 1-point weekly increase (on a 32-point scale) in aggregate PRO score was associated with 247 fewer mean daily steps (95% CI -277 to -213; P<.001). PROs most strongly associated with step count decline were patient-reported activity (daily step change -892), nausea score (-677), and constipation score (524). A 1-point weekly increase in aggregate PRO score was associated with 20% greater odds of hospitalization or death (adjusted odds ratio [aOR] 1.2, 95% CI 1.1-1.4; P=.01). PROs most strongly associated with hospitalization or death were pain (aOR 3.2, 95% CI 1.6-6.5; P<.001), decreased activity (aOR 3.2, 95% CI 1.4-7.1; P=.01), dyspnea (aOR 2.6, 95% CI 1.2-5.5; P=.02), and sadness (aOR 2.1, 95% CI 1.1-4.3; P=.03). A decrease in 1000 steps was associated with 16% greater odds of hospitalization or death (aOR 1.2, 95% CI 1.0-1.3; P=.03). Compared with baseline, mean daily step count decreased 7% (n=274 steps), 9% (n=351 steps), and 16% (n=667 steps) in the 3, 2, and 1 weeks before hospitalization or death, respectively. In this secondary analysis of a randomized trial among patients with advanced cancer, higher symptom burden and decreased step count were independently associated with and predictably worsened close to hospitalization or death. Future interventions should leverage longitudinal PRO and step count data to target interventions toward patients at risk for poor outcomes. ClinicalTrials.gov NCT04616768; https://clinicaltrials.gov/study/NCT04616768. RR2-10.1136/bmjopen-2021-054675.

Digital Remote Monitoring Using an mHealth Solution for Survivors of Cancer: Protocol for a Pilot Observational Study.

Healthy lifestyle interventions have a positive impact on multiple disease trajectories, including cancer-related outcomes. Specifically, appropriate habitual physical activity, adequate sleep, and a regular wholesome diet are of paramount importance for the wellness and supportive care of survivors of cancer. Mobile health (mHealth) apps have the potential to support novel tailored lifestyle interventions. This observational pilot study aims to assess the feasibility of mHealth multidimensional longitudinal monitoring in survivors of cancer. The primary objective is to test the compliance (user engagement) with the monitoring solution. Secondary objectives include recording clinically relevant subjective and objective measures collected through the digital solution. This is a monocentric pilot study taking place in Bangor, Wales, United Kingdom. We plan to enroll up to 100 adult survivors of cancer not receiving toxic anticancer treatment, who will provide self-reported behavioral data recorded via a dedicated app and validated questionnaires and objective data automatically collected by a paired smartwatch over 16 weeks. The participants will continue with their normal routine surveillance care for their cancer. The primary end point is feasibility (eg, mHealth monitoring acceptability). Composite secondary end points include clinically relevant patient-reported outcome measures (eg, the Edmonton Symptom Assessment System score) and objective physiological measures (eg, step counts). This trial received a favorable ethical review in May 2023 (Integrated Research Application System 301068). This study is part of an array of pilots within a European Union funded project, entitled "GATEKEEPER," conducted at different sites across Europe and covering various chronic diseases. Study accrual is anticipated to commence in January 2024 and continue until June 2024. It is hypothesized that mHealth monitoring will be feasible in survivors of cancer; specifically, at least 50% (50/100) of the participants will engage with the app at least once a week in 8 of the 16 study weeks. In a population with potentially complex clinical needs, this pilot study will test the feasibility of multidimensional remote monitoring of patient-reported outcomes and physiological parameters. Satisfactory compliance with the use of the app and smartwatch, whether confirmed or infirmed through this study, will be propaedeutic to the development of innovative mHealth interventions in survivors of cancer. PRR1-10.2196/52957.

Remote Between Visit Monitoring in Inflammatory Bowel Disease Care: A Qualitative Study of CAPTURE-IBD Participants and Care Team Members.

We recently showed that CAPTURE-inflammatory bowel disease (IBD)-a care coordination intervention comprised of routine remote monitoring of patient-reported outcomes (PRO) and a care coordinator-triggered care pathway-was more effective at reducing symptom burden for patients with IBD compared to usual care. We aimed to understand how patients and care team providers experienced the intervention and evaluate purported mechanisms of action to plan for future implementation. In this study, 205 patients were randomized to CAPTURE-IBD (n = 100) or usual care(n = 105). We conducted semi-structured interviews with 16 of the 100 participants in the CAPTURE-IBD arm and 5 care team providers to achieve thematic saturation. We used qualitative rapid analysis to generate a broad understanding of experiences, perceived impact, the coordinator role, and suggested improvements. Findings highlight that the intervention was acceptable and user-friendly, despite concerns regarding increased nursing workload. Both participants and care team providers perceived the intervention as valuable in supporting symptom monitoring, psychosocial care, and between-visit action plans to improve IBD care and health outcomes. However, few participants leveraged the care coordinator as intended. Finally, participants reported that the intervention could be better tailored to capture day-to-day symptom changes and to meet the needs of patients with specific comorbid conditions (eg, ostomies). Remote PRO monitoring is acceptable and may be valuable in improving care management, promoting tight control, and supporting whole health in IBD. Future efforts should focus on testing and implementing refined versions of CAPTURE-IBD tailored to different clinical settings.

Testing an Electronic Patient-Reported Outcome Platform in the Context of Traumatic Brain Injury: PRiORiTy Usability Study.

Traumatic brain injury (TBI) is a significant public health issue and a leading cause of death and disability globally. Advances in clinical care have improved survival rates, leading to a growing population living with long-term effects of TBI, which can impact physical, cognitive, and emotional health. These effects often require continuous management and individualized care. Traditional paper-based assessments can be cumbersome, potentially impeding regular monitoring of patient-reported outcomes (PROs). Electronic PROs (ePROs) offer a promising alternative by enabling real-time symptom tracking, which can facilitate early identification of issues, support shared decision-making, and improve outcomes for patients with TBI. This study evaluates the usability of an ePRO platform-Atom5-for individuals with TBI. By analyzing how patients use the system to report their symptoms, the study aims to identify usability issues, assess user satisfaction, and determine the potential of Atom5 to support ongoing patient-centered care. Atom5 was customized to enable individuals with TBI to report their symptoms. Usability testing was conducted through one-on-one sessions with participants recruited from Headway UK-an organization supporting brain injury survivors. Each participant took part in cognitive interviews using with the "Think Aloud" method, encouraging them to verbalize their thoughts and experiences while using the platform. This approach provided qualitative insights into areas of difficulty, usability strengths, and accessibility barriers. User satisfaction was quantitatively assessed with a brief 4-item questionnaire based on the System Usability Scale. Usability outcomes were analyzed for critical and noncritical errors, focusing on user experience and overall satisfaction. In total, 9 participants completed a single usability testing session using Atom5, including 4 men, 4 women, and 1 nonbinary individual; 4 participants were under 55 years old, and 6 had their TBI <10 years ago. Finally, 8 participants used an Android device. The platform included measures for anxiety (Generalized Anxiety Disorder-2 item), depression (Patient Health Questionnaire-2), posttraumatic stress disorder (Posttraumatic Stress Disorder checklist 2), and TBI-specific quality of life (Traumatic Brain Injury - Quality of Life Short form) and a total of 26 questions. Overall, all participants were satisfied with the system, noting that it was easy to navigate and accessible despite difficulties in understanding some questions. Further, 6 participants encountered no errors, while 1 participant reported one critical error and 2 others reported one noncritical error each. The participants rated their overall satisfaction with the platform at an average score of 3.9 (SD 0.49) out of 5. This usability study suggests that individuals living with TBI can effectively report symptoms using the Atom5 ePRO platform, with generally high satisfaction and few usability issues, thereby enabling continuous monitoring and proactive symptom management. Future ePRO development should focus on inclusivity and adaptability to address the diverse needs of patients with TBI, ensuring these tools can effectively support a wide range of users.

Statistical analysis plan for the PRO B study: open-label, superiority randomised controlled trial of alarm-based patient-reported outcome monitoring in patients with metastatic breast cancer

BackgroundWith an increasing collection of patient-reported outcomes (PROs) to measure health-related quality of life (HRQoL) in oncological patients, there is still a lack of standardised strategies on how to interpret and use these data in patient care. Prior research has shown support for the use of digital PRO monitoring together with alarm systems to notify clinicians when the PRO values are deteriorating. This system has demonstrated advantages in improving HRQoL and increasing survival rates among oncology patients. Hence, we designed the PRO B study, a superiority multi-centre randomised controlled trial, to investigate the effects of alarm-based monitoring in metastatic breast cancer patients in Germany. The study protocol for the PRO B study was published in September 2021, and this manuscript describes a formal statistical analysis plan (SAP) for the PRO B study to improve the transparency and quality of this trial.Methods and designThe trial aimed to recruit 1000 patients with metastatic breast cancer. However, as of the completion of recruitment on June 15, 2023, we have successfully enrolled 924 patients from 52 breast cancer centres. Patients were 1:1 stratified randomised to the intervention and control groups. App-based PRO questionnaires are sent weekly to the intervention group and every 3 months to the control group. Only patients in the intervention group trigger an alarm if their PRO scores deteriorate, and they are subsequently contacted by the local care team within 48 h. The primary outcome is the fatigue score at 6 months, and secondary outcomes are other HRQoL and overall survival. Evaluation of the superiority of the intervention will be done using a linear mixed model with random intercepts for study centres.ConclusionThis detailed SAP defines the main components of the statistical analysis for the PRO B study to assist the statistician and prevent bias in selecting analysis and reporting findings. Version 1 of the SAP was finalised on January 18, 2024.Trial registrationDRKS (German Clinical Trials Register) DRKS00024015. Registered on February 15, 2021.

PRO-WAVE1: Monitoring patient reported outcomes (PROs) in prostate cancer through Wave Health, a novel ePRO health platform.

114 Background: It is well established that the monitoring and evaluation of PROs in cancer patients has a positive impact in both quality of life and overall survival.1 In parallel, the widespread use of electronic devices such as smart phones in the general population have increased significantly over recent years,2 representing a unique opportunity to operationalize ePROs. However, there is uncertainty about the feasibility and applicability of these technologies in certain patient population such as prostate cancer (PC) patients. PRO-WAVE1 aims to determine the acceptability and engagement of the Wave Health patient app and healthcare provider portal, enhanced for PC patients. Specific endpoints include measuring longitudinal use of varying ePROs to impact care management, patient health literacy, and ability to identify and manage emerging risks. Methods: The study is underway across eight sites in Spain. Patient ePRO engagement is being evaluated through the administration of 19 ePRO questionnaires over the course of 13 weeks, including: health-related QoL (FACT-P); health literacy (CHLT-30-Dkspa); and weekly reviews of symptoms (WSR). Compliance rates per patient and per cohort will be reported at assessment time-points and for the entire study period. Secondary objectives evaluated include engagement with Wave Health App; changes in HRQoL; changes in health literacy; and the perceived value of the platform by patients and HCPs. Results: 98 patients were enrolled in the study. Patient cohort representation included: 44.9% mHSPC, 36.7% mCRPC, and 18.3% nmPC. Of the 98 patients, 84 successfully became active in the study and participated (85.7% acceptance). As of September 20, 2023, among the completed patients (N=46), the compliance rate for the ePRO questionnaires was 93.7%. During their participation, these patients used the app for an average of 12.2 weeks, and approximately two-thirds (67.4%) used the app for all 13 weeks. Weekly “usefulness” ratings among the HCPS averaged 3.6 (scale: 1 “not at all useful” to 5 “very useful”). Conclusions: Study results exceeded expectations for patient acceptability and engagement with the app; as well as perceived usefulness of the portal among HCPs – suggesting the platform as an impactful tool for patients working with HCPs to manage treatment journeys. A larger study using a comparator arm will help further evaluate the impact on costs and clinical outcomes. 1. Basch E., JAMA 2017. 2. Statista, 2023.

P625 Electronic patient-reported outcomes in Inflammatory Bowel Diseases on vedolizumab therapy: Interim analysis of the non-interventional German LISTEN II study

Abstract Background Integrating systematic monitoring of patient-reported outcomes (PROs) with clinical information may enhance disease management and improve patient outcomes. Vedolizumab (VDZ) is approved for treating patients with ulcerative colitis (UC) and Crohn’s disease (CD). The German, multicentre, prospective, non-interventional LISTEN II study evaluates patient characteristics and effectiveness of VDZ treatment in real-world clinical practice, focusing on PROs. Methods Adult UC or CD patients, who either initiate VDZ intravenously (IV) or switch from IV maintenance to subcutaneous (SC) formulation, are enrolled. Data on patient characteristics, modalities of VDZ use and disease activity are recorded quarterly over one year. PROs are recorded via weekly questionnaires in app (ePRO). Sustained ePRO use was defined as proportion of patients reporting at least one PRO for ≥80% of available questionnaires. Interim data up to 9 months follow-up are presented. Results Data of 299 UC and 174 CD patients (51% and 59% female, respectively) with a mean age of 40 years were analysed. VDZ treatment was newly initiated IV (start) in 259 UC and 151 CD patients and switched from IV to SC application (switch) by 40 UC and 23 CD patients at baseline. Total mean pMayo score decreased in UC start-patients from 5.0 to 1.7 and from 1.1 to 0.8 in switch-patients. In CD start-patients HBI score decreased from 5.5 to 2.5 and from 3.1 to 2.3 in switch-patients. The ePRO was initially utilized by 226 UC and 124 CD patients (51% and 61% female, 57% and 52% sustained ePRO users, respectively) with a mean age of 39 years; 86% were start- and 14% switch-patients. Disease activity at baseline was comparable to the overall population, showing a total mean pMayo score of 4.5 for UC and a HBI of 5.3 for CD patients. Most UC (82%) and CD patients (88%) used a patient support program for the first time. ePRO use declined to approx. 40% usage within the second quarter and to 30% during further follow-up. Overall, mean symptom scores reported via the ePRO declined during the 9-month follow-up period. The most notable improvements (baseline to month 9) were reported for the symptom impact on work productivity (from 3.9 to 1.7 points) in UC patients and for sick leave (from 1.3 to 0.4 points) in CD patients (Table). For most PROs reduction in symptom scores was apparent within the first week of ePRO-use (Figure). Conclusion LISTEN II real-world data confirm VDZ effectiveness in UC and CD patients, with reduction in disease activity in IV starters and maintained effectiveness in IV-to-SC switchers. Both UC and CD patients reported sustained symptom improvement under VDZ treatment, with scores indicating improvement within the first week of ePRO-use.

Pilot trial of an electronic patient-reported outcome monitoring system in patients with metastatic breast cancer undergoing chemotherapy.

Electronic patient-reported outcomes monitoring (ePROM) is a useful communication tool for patients and healthcare providers in cancer chemotherapy. In this study, we examined the feasibility of our newly developed ePROM system, which we refer to as "Hibilog". An ePROM app was developed by extracting 18 items from the Patient-Reported Outcome-Common Terminology Criteria for Adverse Events (PRO-CTCAE). Symptom monitoring was conducted every two weeks for patients with metastatic breast cancer undergoing chemotherapy. The primary outcome was the response rate to the ePROM system. The secondary outcomes were response time, item missing rate, and distribution of responses for each symptom. A total of 71 cases (mean age 52.6years) were analyzed. Performance status was 0 in 76% of the cases and 1 or higher in 24%. First-line treatment was being administered in 30% of cases, second-line treatment in 17%, and third-line or higher treatment in 53%. The response rate to the ePROM system from registration to week 40 remained high at around 80%, indicating good compliance. The average response time was 5.5min and the missing rate for each item was below 0.4%. Among 1,093 responses, the top 3 symptoms causing interference with daily life were Fatigue (63%), Numbness and tingling (48%), and General pain (46%). Our developed ePROM system was able to capture symptoms accurately in patients with metastatic breast cancer undergoing chemotherapy while maintaining a high response compliance.

Behavioural digital biomarkers enable real-time monitoring of patient-reported outcomes: a substudy of the multicentre, prospective observational SafeHeart study.

Patient-reported outcome measures (PROMs) serve multiple purposes, including shared decision-making and patient communication, treatment monitoring, and health technology assessment. Patient monitoring using PROMs is constrained by recall and non-response bias, respondent burden, and missing data. We evaluated the potential of behavioural digital biomarkers obtained from a wearable accelerometer to achieve personalized predictions of PROMs. Data from the multicentre, prospective SafeHeart study conducted at Amsterdam University Medical Center in the Netherlands and Copenhagen University Hospital, Rigshospitalet in Copenhagen, Denmark, were used. The study enrolled patients with an implantable cardioverter defibrillator between May 2021 and September 2022 who then wore wearable devices with raw acceleration output to capture digital biomarkers reflecting physical behaviour. To collect PROMs, patients received the Kansas City Cardiomyopathy Questionnaire (KCCQ) and EuroQoL 5-Dimensions 5-Level (EQ5D-5L) questionnaire at two instances: baseline and after six months. Multivariable Tobit regression models were used to explore associations between digital biomarkers and PROMs, specifically whether digital biomarkers could enable PROM prediction. The study population consisted of 303 patients (mean age 62.9±10.9 years, 81.2% male). Digital biomarkers showed significant correlations to patient-reported physical and social limitations, severity and frequency of symptoms, and quality of life. Prospective validation of the Tobit models indicated moderate correlations between the observed and predicted scores for KCCQ [concordance correlation coefficient (CCC) = 0.49, mean difference: 1.07 points] and EQ5D-5L (CCC=0.38, mean difference: 0.02 points). Wearable digital biomarkers correlate with PROMs, and may be leveraged for real-time prediction. These findings hold promise for monitoring of PROMs through wearable accelerometers.

Monitoring Patient Reported Outcomes in PNH: Interim Results of a Market Research with florio ® PNH

Background: Patients withparoxysmal nocturnal hemoglobinuria (PNH), an ultra-rare, acquired disorder, report various cardiovascular, gastrointestinal, neurological or hematological symptoms that can severely impact their physical functioning and health-related quality of life. Patients may require long term treatment with anti-complement therapies to alleviate PNH symptoms or complications. Whilst blood biomarkers can aid the monitoring of disease progression, patient reported outcomes (PRO) and related symptoms are also of value in assessing response to therapy, but they are not always continuously tracked by patients. Hence florio PNH, a software medical device (smartphone app), was designed to allow easy monitoring of PRO and tracking of medication by patients. It allows documentation of therapy and common symptoms as well as hemoglobin and LDH levels. florio PNH can send treatment reminders and compiles the entered data into a patient report. Patients were invited to participate in market research of florio PNH to better understand monitoring habits and expectations towards an app in this area. Methods: Twenty patients were invited to participate in market research sponsored by Florio GmbH. The participants came from the UK PNH service centre in Leeds and were selected by the coordinating clinical team at routine clinic visits. The research was submitted for ethics approval and written informed consent was provided. The market research consisted of a one hour entry interview assessing current behavior and expectations towards monitoring of outcomes, followed by an eight-week period of field-testing florio PNH app. The subsequent one hour exit interview explored the user experience with the app, and which app features were seen of value, of less value or were missing. A total of 20 patients are to be enrolled in the research, of which 17 had completed the initial interview at the time of this report. Results: The majority of participants were 50 years of age or older (59%) and described themselves as “not tech savvy”. Six participants rated the impact of PNH on their life as significant, 6 as somewhat and 5 as little to none. Fatigue was named by 10 patients unprompted as the prominent symptom affecting their social and work life. Before the research, three participants neither tracked symptoms nor reported medication, five tracked both, seven only tracked their anti-complement infusions and two only major events when they happened. Forty seven percent of participants (8 of 17) did not prepare information at all for clinical consultations in advance, as they either experienced few events or relied on their memory. All users managed to install the app, set up their account and log events without problem. The app was described as easy to use, straightforward and participants appreciated that all data was in one location and possible to summarize into a report. Sixteen (94%) patients said that they believe the app will help them track events and that they would be enthused to use it in the future. Conclusions: Monitoring applications are employed in several rare bleeding disorders and have been shown to be valuable assets for collecting patient reported outcomes, especially in the outpatient setting. Participants of this research had no experience with using disease specific apps for collecting outcomes, and usually rely on the calendar functions provided by their smartphones or paper diaries. 94% concluded that the app will help them organize their PNH data, share data with their treating clinical team, better plan their future activities and be reminded when the next treatments are due.