Electronic Patient-reported Outcomes Research Articles

P22.03.A FEASIBILITY OF AND COMPLIANCE TO ELECTRONIC PATIENT-REPORTED OUTCOME ASSESSMENT IN THE MOLECULAR TUMOR BOARD IN (NEURO-)ONCOLOGICAL PATIENTS UNDER TARGETED THERAPY (TRACE)

Abstract BACKGROUND Comprehensive genomic profiling and biomarker-based therapies are increasingly used in clinical trials and in innovative health care systems including the center for personalized medicine network. Systematic assessments of patient-reported outcomes (PRO) are warranted to gain insight into the perspective of patients during biomarker-based therapies. In the present study, we focused on the feasibility of and compliance to an electronic PRO assessment in patients treated at our center. MATERIAL AND METHODS Based on a retrospective evaluation of symptom burden in 265 neurooncology patients in the Molecular Tumor Board (MTB), we developed an app by 14 expert rounds including validated assessments of health-related quality of life (HRQoL), burden of symptoms und psychological stress, and tested it in a pilot study. After informed consent, patients were introduced to the app by study staff and completed subsequently patient-reported outcomes at several time points (weekly, monthly, and quarterly). We conducted a structured interview with health care personnel and the patients as users after 3 months to assess the app’s usability and feasibility. The interview was transcribed and analyzed according to Mayring´s qualitative content analysis regarding handling, feasibility and applicability in daily routine.Furthermore, we assessed compliance and descriptive data of the ePROs. RESULTS So far, 14 patients and caregivers have been enrolled, (9 female, 5 male). Median age was 50 years (range 28 -79), 3 out of 14 patients dropped out due to death. A total of 4 study staff members, 9 patients and one caregiver were interviewed regarding usability /feasibility. Both evaluated as good (study staff members in total n=40 quotes, patients/caregivers n=112 quotes). Main advantages were the possibility to complete questionnaires at home and the easy implementation in daily life. Compliance to questionnaires was high, e.g., 91% of weekly distributed NCCN Distress Thermometer were answered in time (range 65-100%, assessment duration 5-56 weeks, 262 answers), we observed a median Distress score of 4 (range 0-9, n=262, weekly assessed) and a median Global health score of 66.7 (range 16.7-100, n=69, EORTC QLQ-C30, question 29 and 30, monthly assessed, compliance 94%). CONCLUSION This pilot study proved feasibility and acceptance of the app and might enable PRO assessment during biomarker-guided therapies.

Feasibility of Weekly Electronic Health Record-Embedded Patient-Reported Outcomes for Patients Starting Oral Cancer-Directed Therapy.

To examine the feasibility of integrating a symptom management platform into the electronic health record (EHR) using electronic patient-reported outcomes (ePROs) during oral cancer-directed therapy (OCDT) and explore the impact of prompting oncology nurse navigators (ONNs) to respond to severe symptomatic adverse events (SAEs). Adults prescribed OCDT at Dana-Farber Cancer Institute were consecutively invited to participate. Participants received weekly messages to complete ePROs. The first half enrolled in a passive (P) group where ePROs responses could be viewed anytime, but outreach was not expected. The second half enrolled in an active (A) group where severe SAEs prompted emails to ONNs for outreach within 1 business day. Feasibility was the proportion of participants completing ≥2 ePROs during the first 30 days. Participants were followed for up to 90 days. From June 25, 2019, to August 18, 2021, 100 participants enrolled, and 96 remained enrolled for at least 30 days. Overall, average age was 59 years, 80% female, and 9% used the platform in Spanish. Twenty-two A (45%) and 27 P (57%) participants met the feasibility threshold (P = .26). ePROs returned at 30 days were similar (P = .50): 0 ePROs 17 A, 13 P; 1 ePRO 10 A, 7 P; 2 ePROs 3 A, 5 P; 3 ePROs 1 A, 4 P; 4 ePROs 7 A, 8 P; and 5 ePROs 11 A, 10 P. Documented telephone encounters at 30 days were similar (109 A, 101 P; P = .86). EHR-embedded ePROs administered weekly for people on OCDT was feasible, although many went incomplete. ePRO completion was not clearly affected by nursing calls for severe SAEs. Future efforts will investigate improving engagement and addressing symptoms proactively.

Protocol for the ASTRO study (SSOP-01): a multicentre prospective cohort study investigating adverse events based on electronic patient-reported outcomes in patients with breast cancer after adjuvant chemotherapy

IntroductionSymptom-related adverse events associated with perioperative chemotherapy in patients with breast cancer include short-term adverse events such as nausea and vomiting. However, changes in the severity and duration of prolonged...

Study within a trial of electronic versus paper-based Patient-Reported oUtcomes CollEction (SPRUCE): study protocol for a partially randomised patient preference study

IntroductionPatient-reported outcomes (PRO) are currently collected from trial participants using paper questionnaires by the Clinical Trials and Statistics Unit at The Institute of Cancer Research (ICR-CTSU). Streamlining PRO collection using...

Electronic Patient-Reported Outcome Data Collection Systems in Oncology Clinical Trials: A Survey of Clinical Research Professionals (an Alliance Study).

To describe clinical research professionals (CRPs)' experiences with electronic patient-reported outcome (ePRO) data collection systems in oncology clinical trials and identify correlates of CRPs' attitude toward technology. An online survey was conducted among 210 CRPs from 125 National Cancer Institute-funded research sites. Measures included CRPs' demographic characteristics, working years, employment locations, and previous experiences with various types of ePROs. Their attitude toward technology was measured by the Technology Attitude Scale-Adapted. The Wilcoxon signed-rank test was used to compare two subdomains of attitude (perceived usefulness [PU] and perceived ease of use [PEU]). Multiple linear regression was used to explore correlates of (1) overall attitude, (2) PU, and (3) PEU. The significance level was 5%. Participants' median age was 41 years (range, 21-67). Most were female (90%) and White (82%). More than half of the participants had previous experiences with web-based ePROs using patients' own devices (72%) or site-/sponsor-provided on-site devices (eg, kiosks or tablets; 64%). CRPs who were 60 years or older (β = -0.32, P < .05) or worked for 10-20 years (β = -0.11, P < .05) had relatively negative attitudes, controlling for other factors. Previous experiences with more ePRO types were associated with more positive attitudes (β = 0.08, P = .02). Similar correlates were found with PU but not with PEU. This study revealed that CRPs had various experiences with ePRO systems and attitudes toward technology. Age, working years, and previous experiences with ePROs were correlates of overall attitude toward technology and PU. These findings suggest necessary targeted training to facilitate ePRO use in oncology clinical trials by improving CRPs' awareness and attitude toward technology.

Electronic Patient-Reported Outcome Monitoring to Improve Quality of Life After Joint Replacement

Although remote patient-reported outcome measure (PROM) monitoring has shown promising results in cancer care, there is a lack of research on PROM monitoring in orthopedics. To determine whether PROM monitoring can improve health outcomes for patients with joint replacement compared with the standard of care. A 2-group, patient-level randomized clinical trial (PROMoting Quality) across 9 German hospitals recruited patients aged 18 years or older with primary hip or knee replacement from October 1, 2019, to December 31, 2020, with follow-up until March 31, 2022. Intervention and control groups received the standard of care and PROMs at hospital admission, discharge, and 12 months after surgery. In addition, the intervention group received PROMs at 1, 3, and 6 months after surgery. Based on prespecified PROM score thresholds, at these times, an automated alert signaled critical recovery paths to hospital study nurses. On notification, study nurses contacted patients and referred them to their physicians if necessary. The prespecified outcomes were the mean change in PROM scores (European Quality of Life 5-Dimension 5-Level version [EQ-5D-5L; range, -0.661 to 1.0, with higher values indicating higher levels of health-related quality of life (HRQOL)], European Quality of Life Visual Analogue Scale [EQ-VAS; range, 0-100, with higher values indicating higher levels of HRQOL], Hip Disability and Osteoarthritis Outcome Score-Physical Function Shortform [HOOS-PS; range, 0-100, with lower values indicating lower physical impairment] or Knee Injury and Osteoarthritis Outcome Score-Physical Function Shortform [KOOS-PS; range, 0-100, with lower values indicating lower physical impairment], Patient-Reported Outcomes Measurement Information System [PROMIS]-fatigue [range, 33.7-75.8, with lower values indicating lower levels of fatigue], and PROMIS-depression [range, 41-79.4, with lower values indicating lower levels of depression]) from baseline to 12 months after surgery. Analysis was on an intention-to-treat basis. The study included 3697 patients with hip replacement (mean [SD] age, 65.8 [10.6] years; 2065 women [55.9%]) and 3110 patients with knee replacement (mean [SD] age, 66.0 [9.2] years; 1669 women [53.7%]). Exploratory analyses showed significantly better health outcomes in the intervention group on all PROMs except the EQ-5D-5L among patients with hip replacement, with a 2.10-point increase on the EQ-VAS in the intervention group compared with the control group (HOOS-PS, -1.86 points; PROMIS-fatigue, -0.69 points; PROMIS-depression, -0.57 points). Patients in the intervention group with knee replacement had a 1.24-point increase on the EQ-VAS, as well as significantly better scores on the KOOS-PS (-0.99 points) and PROMIS-fatigue (-0.84 points) compared with the control group. Mixed-effect models showed a significant difference in improvement on the EQ-VAS (hip replacement: effect estimate [EE], 1.66 [95% CI, 0.58-2.74]; knee replacement: EE, 1.71 [95% CI, 0.53-2.90]) and PROMIS-fatigue (hip replacement: EE, -0.65 [95% CI, -1.12 to -0.18]; knee replacement: EE, -0.71 [95% CI, -1.23 to -0.20]). The PROMIS-depression score was significantly reduced in the hip replacement group (EE, -0.60 [95% CI, -1.01 to -0.18]). In this randomized clinical trial, the PROM-based monitoring intervention led to a small improvement in HRQOL and fatigue among patients with hip or knee replacement, as well as in depression among patients with hip replacement. Deutsches Register Klinischer Studien ID: DRKS00019916.

Real-World Use of Electronic Patient-Reported Outcome (ePRO) Tools Integrated in the Electronic Medical Record During Radiation Therapy for Head and Neck Cancer: Feasibility Study.

Purpose Use of electronic patient-reported outcome (ePRO) tools in routine oncology practice can be challenging despite evidence showing they can improve survival, improve patient and practitioner satisfaction, and reduce medical resource utilization. Head and neck cancer (HNC) patients receiving radiation therapy (RT) may be a group that would particularly benefit from interventions focused on early symptom management. Methods Patients undergoing definitive RT for HNC were enrolled in a feasibility study and received ePRO surveys integrated within the electronic medical record (EMR) on a weekly basis during RT. After completion of each ePRO survey, a radiation oncology registered nurse documented the findings and subsequent interventions within the EMR. Results Thirty-four patients with HNC who received curative RT at a single center were enrolled. The total number of surveys completed was 194 with a median of 7 surveys per patient (range 1-8). There was a total of 887 individual abnormal findings reported on the ePROs, and the authors found that all 887 had a corresponding documented intervention. Post-treatment practitioner questionnaires highlighted that ePROs were felt to be helpful for the care team in providing care to HNC patients. Conclusion For patients with HNC receiving RT, ePROs can be effectively utilized to address patient symptoms within an integrated health care system. Creating an infrastructure for the use of ePROs integrated within the EMR in routine care requires an approach that accounts for local workflows and buy-in from patients and the entire care team.

Diversified caregiver input to upgrade the Young Children’s Participation and Environment Measure for equitable pediatric re/habilitation practice

BackgroundPractitioner and family experiences of pediatric re/habilitation can be inequitable. The Young Children’s Participation and Environment Measure (YC-PEM) is an evidence-based and promising electronic patient-reported outcome measure that was designed with and for caregivers for research and practice. This study examined historically minoritized caregivers’ responses to revised YC-PEM content modifications and their perspectives on core intelligent virtual agent functionality needed to improve its reach for equitable service design.MethodsCaregivers were recruited during a routine early intervention (EI) service visit and met five inclusion criteria: (1) were 18 + years old; (2) identified as the parent or legal guardian of a child 0–3 years old enrolled in EI services for 3 + months; (3) read, wrote, and spoke English; (4) had Internet and telephone access; and (5) identified as a parent or legal guardian of a Black, non-Hispanic child or as publicly insured. Three rounds of semi-structured cognitive interviews (55–90 min each) used videoconferencing to gather caregiver feedback on their responses to select content modifications while completing YC-PEM, and their ideas for core intelligent virtual agent functionality. Interviews were transcribed verbatim, cross-checked for accuracy, and deductively and inductively content analyzed by multiple staff in three rounds.ResultsEight Black, non-Hispanic caregivers from a single urban EI catchment and with diverse income levels (Mdn = $15,001–20,000) were enrolled, with children (M = 21.2 months, SD = 7.73) enrolled in EI. Caregivers proposed three ways to improve comprehension (clarify item wording, remove or simplify terms, add item examples). Environmental item edits prompted caregivers to share how they relate and respond to experiences with interpersonal and institutional discrimination impacting participation. Caregivers characterized three core functions of a virtual agent to strengthen YC-PEM navigation (read question aloud, visual and verbal prompts, more examples and/or definitions).ConclusionsResults indicate four ways that YC-PEM content will be modified to strengthen how providers screen for unmet participation needs and determinants to design pediatric re/habilitation services that are responsive to family priorities. Results also motivate the need for user-centered design of an intelligent virtual agent to strengthen user navigation, prior to undertaking a community-based pragmatic trial of its implementation for equitable practice.

The effectiveness of symptom management interventions based on electronic patient-reported outcomes (ePROs) for symptom burden, quality of life, and overall survival among patients with cancer: A meta-analysis of randomized controlled trials

ObjectiveTo explore the effectiveness of ePRO-based symptom management interventions on symptom burden, quality of life, and overall survival among patients with cancer for the first time and to explore the effects of different types of these interventions. BackgroundSince advances in screening and treatment have transformed cancer into a chronic illness rather than a fatal disease, symptom management has become increasingly critical in oncology nursing. In recent decades, ePROs have been increasingly used in the symptom management of cancer patients to improve their symptom burden, quality of life and overall survival, but the existing findings are still inconsistent and equivocal. MethodsA literature search was conducted in PubMed, Web-of-Science, CENTRAL, and CINAHL-Plus-with-Full-Text from inception to January 31, 2023. The quality of methodology and evidence were evaluated by the revised Cochrane risk-of-bias tool and the Grading of Recommendations, Assessment, Development, and Evaluation framework. All data were analyzed using R within the RStudio platform, and the effects of interventions were determined by calculating SMD, HR and 95 %CI. Subgroup analysis, sensitivity analysis and cumulative meta-analysis were performed, and statistical heterogeneity was examined by I2 statistic, P value, and Egger's or arcsine test. Statistical significance was defined as a two-tailed P value <0.05. ResultsA total of 23 randomized controlled trials with 7231 patients were included. The results indicated that ePRO-based symptom management interventions could improve the symptom burden (SMD = −0.19, 95 % CI [−0.33, −0.05], P < 0.01), quality of life (SMD = 0.16, 95 % CI [0.06, 0.25], P < 0.01) and overall survival (HR = 0.84, 95 % CI [0.73, 0.97], P = 0.02) of cancer patients. Subgroup analysis showed that targeted interventions for patients undergoing specific treatments were effective in relieving the symptom burden and enhancing quality of life. Short-term (≤3 months) interventions or reporting via telephone call contributed to alleviating the symptom burden, while quality of life improved when the intervention was more than three months in duration or not reported by telephone call. The pooled results of symptom burden and quality of life were stable, and the beneficial trends of all three outcomes were steady. The overall quality of methodology and evidence was moderate. ConclusionsWe found that ePRO-based symptom management interventions are conducive to improving symptom burden, quality of life, and overall survival of cancer patients. In addition to encouraging the integration of ePRO-based interventions into routine oncology care, interventions with tailored plans, proper intensity and multidimensional supports need to be developed in the future to optimize the symptom management of cancer patients. RegistrationCRD42023393330.

Delivery of the HIV Service and Telemedicine Through Effective Patient-Reported Outcomes (+STEP) Intervention to Increase Screening and Treatment of Mental Health and Substance Use Disorders for People Living With HIV in Alabama: Protocol for an Effectiveness-Implementation Study.

The syndemic of mental health (MH) and substance use disorders (SUDs) is common among persons living with HIV and jeopardizes HIV treatment adherence, engagement in care, and viral load suppression. Electronic patient-reported outcomes (ePROs), completed through tablet or computer, and telemedicine are evidence- and technology-based interventions that have been used to successfully increase screening and treatment, respectively, a model that holds promise for persons living with HIV. To date, there is limited guidance on implementing ePROs and telemedicine into HIV clinical practice even though it is well known that these evidence-based tools improve diagnosis and access to care. To address this, we aim to conduct a multicomponent intervention for persons living with HIV, including the delivery of HIV services and telemedicine through effective ePROs (+STEP), to increase screening and treatment of MH and SUD in Ryan White HIV/AIDS Program (RWHAP)-funded clinics in Alabama. Through this intervention, we will conduct a readiness, acceptability, and accessibility assessment and implement +STEP to improve the diagnosis and treatment of MH and SUD at RWHAP clinics in Alabama. To describe implementation strategies that address barriers to the uptake of +STEP in RWHAP clinics, we will conduct qualitative interviews in years 1 (early implementation), 2 (scale up), and 4 (maintenance) with patients and key staff to evaluate barriers, facilitators, and implementation strategies. Our Results will enable us to modify strategies to enhance +STEP penetration over time and inform the implementation blueprint, which we will develop for both RWHAP clinics in Alabama and future sites. We will assess the impact of implementing +STEP on diagnoses, referrals, and health care use related to MH, SUD, and HIV by comparing clinical outcomes from patients receiving these interventions (ePROs and telemedicine) with historical controls. The first study site began implementation in April 2022. A total of 2 additional sites have initiated ePROs. Final results are expected in 2026. The results of this study will provide a foundation for future research expanding access to ePROs for improved diagnosis linked to telemedicine access to accelerate patients along the continuum of care from MH and SUD diagnosis to treatment. Achieving the end of the HIV epidemic in the United States necessitates programs that accelerate movement across the MH and SUD care continuum from diagnosis to treatment for persons living with HIV. Scaling these services represents a path toward improved treatment outcomes with both individual health and population-level prevention benefits of sustained HIV viral suppression in the era of undetectable=untransmittable (U=U). This study will address this evidence gap through the evaluation of the implementation of +STEP to establish the necessary systems and processes to screen, identify, and better treat substance use and MH for people living with HIV. DERR1-10.2196/40470.

Electronic patient-reported outcome (ePRO) application for patients with prostate cancer.

Cancer patients experience different complications and outcomes during or after medical treatments. Electronic reporting of the outcomes by patients is a solution that facilitates communication with physicians and improve patient health status. The aim of this study was to develop a smartphone-based application for electronic reporting of outcomes by patients with prostate cancer. The present research was conducted in 2021 in two phases. In the first phase, initially, users' requirements were identified based on reviewing the related literature, existing applications, and guidelines. Then, a questionnaire was designed and the specialists' opinions about the users' requirements were investigated. The specialties included urologists, hemato-oncologists, uro-oncologists, and radiotherapists (n = 15). In the second phase, the application was designed, and patients with prostate cancer (n = 21) and specialists (n = 10) evaluated it using the post-study system usability questionnaire (PSSUQ). Data were analyzed using descriptive statistics. The findings of the first phase of the research showed that out of 108 data elements and functions proposed for the application, 91 items were found essential by the specialists. Data elements were categorized into the patient data, general complications of prostate cancer and side effects of drug therapy, surgery, chemotherapy, cryotherapy, radiation therapy, and hormone therapy. Necessary functions for the application included presenting a patient care summary, communication between the patient and the specialist, free text explanation for complications and sides effects, generating reports, reminder and alert, completing quality of life questionnaire, and calculating the score for the questionnaire. In the second phase of the research, the application was developed and evaluated. The mean value for user satisfaction was (5.95 ± 0.55) out of 7. The developed application can help to accelerate communication with the specialists. It can improve quality of care, reduce unnecessary treatment visits and side effects, and improve timely data collection for a variety of research purposes. However, further research on the cost-effectiveness and usefulness of the collected data is recommended.

Evaluating the Usability of Electronic Patient-Reported Outcome Apps: Comment on a Symptom Management Platform for Outpatients With Advanced Cancer.

Evaluating the Usability of Electronic Patient-Reported Outcome Apps: Comment on a Symptom Management Platform for Outpatients With Advanced Cancer.

Barriers and facilitators of electronic patient-reported outcome measures (e-PROMs) for patients in home palliative cancer care: a qualitative study of healthcare professionals’ perceptions

BackgroundPatient-reported outcomes in palliative care enable early monitoring and management of symptoms that most impact patients’ daily lives; however, there are several barriers to adopting electronic Patient-reported Outcome Measures (e-PROMs) in daily practice. This study explored the experiences of health care professionals (HCPs) regarding potential barriers and facilitators in implementing e-PROMs in palliative cancer care at home.MethodsThis was a qualitative descriptive study. The data were collected from two focus groups structured according to the conceptual framework of Grol. HCPs involved in home palliative cancer care of Fondazione IRCCS Istituto Nazionale dei Tumori of Milan were enrolled. Data were analyzed using a reflexive thematic analysis.ResultsA total of 245 codes were generated, 171 for the first focus group and 74 for the second focus group. The results were subdivided into subthemes according to Grol’s themes: Innovation, Individual professional, Patient, Social context, Organizational context, except Economic Political context. Nine HCPs attended the first focus group, and ten attended the second. According to these participants, e-PROMs could be integrated into clinical practice after adequate training and support of HCPs at all stages of implementation. They identified barriers, especially in the social and organizational contexts, due to the uniqueness of the oncological end-of-life setting and the intangible care interventions, as well as many facilitators for the innovation that these tools bring and for improved communication with the patient and the healthcare team.Conclusionse-PROMs are perceived by HCPs as adding value to patient care and their work; however, barriers remain especially related to the fragility of these patients, the adequacy of technological systems, lack of education, and the risk of low humanization of care.

Digital tool to identify and monitor regorafenib-associated hand-foot skin reactions: A proof-of-concept study protocol.

The FACET study is a prospective, open-label, low risk interventional clinical trial aiming at exploring the fitness-for-purpose and usability of an electronic device suite for the detection of hand-foot skin reaction symptoms in patients with metastatic colorectal cancer treated with regorafenib. 38 patients with metastatic colorectal cancer are being selected in 6 centers in France, and will be followed for 2 regorafenib treatment cycles, or for approximately 56 days. The electronic device suite includes connected insoles and a mobile device equipped with a camera and a companion application with electronic patient-reported outcomes questionnaires and educational material. The FACET study is intended to provide information useful for the improvement of the electronic device suite and its usability before the testing of its robustness in a larger follow-up study. This paper describes the protocol of the FACET study and discusses the limitations to consider for the implementation of digital devices in real-life practice.

Development and validation of a novel personalized electronic patient-reported outcome measure to assess quality of life (Q-LIFE): a prospective observational study in people with Cystic Fibrosis

Generic and disease-specific patient-reported outcome measures (PROMs) may lack relevance and sensitivity on a patient-level in chronic diseases with differential disease expression and high individual variability, such as Cystic Fibrosis (CF). This study aimed to develop and validate a novel personalized electronic PROM (ePROM) that captures relevant aspects of quality of life in individuals with CF. The Q-Life app was developed as a short personalized ePROM to assess individual quality of life. Psychometric properties were assessed in a single-center cross-sectional study between September 2019 and September 2021 and in a prospective cohort study between September 2021 and September2022. Combined studies included 223 participants (median age: 24 years, IQR: 19.0-32.5 years, range: 12.0-58.0 years). Internal consistency (Cronbach's alpha: 0.83-0.90) and test-retest reliability (intraclass correlation coefficient: 0.90; 95% CI: 0.65-0.92; p<0.001) of quality of life (Q-Life) scores were strong. Q-Life scores were associated with overall Cystic Fibrosis Questionnaire-Revised (CFQ-R) scores (ρ=0.71; p<0.001), CFQ-R respiratory domain scores (ρ=0.57; p<0.001) and forced expiratory volume in 1s (ρ=0.41; p<0.001). Furthermore, Q-Life scores improved from 65.0 (IQR: 45.0-63.3) at baseline to 84.2 (IQR: 75.0-95.0) and 87.5 (IQR: 75.0-100.0) after 3 and 6 months of elexacaftor/tezacaftor/ivacaftor treatment (change: 20.8; 95% CI: 17.5-25.0; p<0.001), comparable to CFQ-R respiratory domain scores (change: 22.2, 95% CI: 19.4-25.0, p<0.001). The Q-Life app is a reliable, valid and sensitive personalized ePROM to measure all aspects of quality of life that really matter to individuals with Cystic Fibrosis. This patient-centered approach could provide important advantages over generic and disease-specific PROMs in the era of personalized medicine and value-based healthcare. Dutch Cystic Fibrosis Foundation, Health-Holland.

Development of an eHealth-enhanced model of care for the monitoring and management of immune-related adverse events in patients treated with immune checkpoint inhibitors

PurposeThe use of electronic patient-reported outcome (ePRO) data in routine care has been tied to direct patient benefits such as improved quality of care and symptom control and even overall survival. The modes of action behind such benefits are seldom described in detail. Here, we describe the development of a model of care leveraging ePRO data to monitor and manage symptoms of patients treated with immune checkpoint inhibitors.MethodsDevelopment was split into four stages: (1) identification of an underlying theoretical framework, (2) the selection of an ePRO measure (ePROM), (3) the adaptation of an electronic application to collect ePRO data, and (4) the description of an ePRO-oriented workflow. The model of care is currently evaluated in a bicentric longitudinal randomized controlled phase II trial, the IePRO study.ResultsThe IePRO model of care is grounded in the eHealth Enhanced Chronic Care Model. Patients are prompted to report symptoms using an electronic mobile application. Triage nurses are alerted, review the reported symptoms, and contact patients in case of a new or worsening symptom. Nurses use the UKONS 24-hour telephone triage tool to issue patient management recommendations to the oncology team. Adapted care coordinating procedures facilitate team collaboration and provide patients with timely feedback.ConclusionThis report clarifies how components of care are created and modified to leverage ePRO to enhance care. The model describes a workflow that enables care teams to be proactive and provide patients with timely, multidisciplinary support to manage symptoms.

Psychometric evaluation of a patient-reported outcomes instrument for congenital thrombotic thrombocytopenic purpura

BackgroundCongenital thrombotic thrombocytopenic purpura (cTTP) is an ultra-rare, life-threatening hereditary disorder that causes patients to experience significant morbidity and decreased health-related quality of life (HRQoL). A cTTP disease-specific patient-reported outcome (PRO) instrument that is reflective of patients’ experiences with the disorder does not currently exist. The objective of this study was to evaluate and validate the psychometric properties of the Congenital Thrombotic Thrombocytopenic Purpura–Patient Experience Questionnaire (cTTP-PEQ), developed using a literature review, interviews with expert clinicians, and qualitative concept elicitation and cognitive debriefing interviews.MethodsThis prospective, observational study (NCT03519672) was conducted with patients diagnosed with cTTP currently receiving treatment. Patients were enrolled through investigator sites and direct-to-patient recruitment. Individuals completed electronic self-administered PRO measures, including the cTTP-PEQ, at baseline and Day 14 (+ up to 10 days). The cTTP-PEQ consisted of five multi-item domains (Pain/Bruising, Cognitive Impairment, Visual Impairment, Mood, Treatment Burden) and three single-item domains (Fatigue, Headache, Activity Limitation), and assessed symptoms and impact of cTTP in the previous 24 h, 7 days, and 2 weeks. Convergent and discriminant validity were evaluated using Spearman’s rank correlation coefficients. Known-groups validity was assessed between patient groups separated by Patient Global Impression of Severity (PGI-S; normal vs. mild/moderate/severe). Internal reliability was assessed using Cronbach’s alpha. Test–retest reliability was assessed using intraclass correlation coefficients (ICCs).ResultsThirty-six patients participated in this study. Convergent validity was confirmed with high-to-moderate correlations (r ≥ 0.4) for 12/15 hypothesized relationships between pairs of domains and/or total scores. Discriminant validity was confirmed with low correlations (r < 0.3) observed for 5/7 hypothesized relationships. Known-groups validity was confirmed with significant differences (p ≤ 0.05) in mean cTTP-PEQ scores between the two PGI-S groups for most domains and items at both timepoints. Cronbach’s alpha was 0.88 at baseline and 0.91 at Day 14, confirming internal consistency of the instrument. Test–retest reliability was also confirmed with a high ICC (0.96).ConclusionThis study validates the psychometric properties of the novel cTTP-PEQ for use in research and clinical practice to assess HRQoL among patients with cTTP. This instrument will be particularly useful when assessing cTTP disease burden and the impact of new treatments.

Evaluating the Usability and Equivalence of Electronic Patient-Reported Outcome Measures for Individuals with a Lower-Limb Amputation.

ABSTRACT Introduction Electronic versions of patient-reported outcome measures (PROMs) seem to have a clear administrative logging advantage to traditional paper versions. However, most of them have not been formally evaluated for their suitability to replace paper outcome measures for assessment of individuals with lower-limb amputations. The aim of this study is to examine the usability and equivalence of electronic to paper versions of PROMs suitable for use in prosthetic clinical care and research for persons with lower-limb loss. Methods In this cross-sectional study, 10 participants remotely completed the following PROMs online and then on paper: Orthotic and Prosthetic User Survey (OPUS), Modified Falls Efficacy Scale (MFES), Prosthetic Evaluation Questionnaire (PEQ), Patient Health Questionnaire–9 (PHQ-9), and Community Participation Indicators (CPI). Participants also answered open-ended and standardized questions regarding the usability of the electronic surveys. Wilcoxon signed rank tests, comparisons to minimum detectable change, intraclass correlation coefficients, and Bland-Altman plots were used to evaluate differences between the two survey versions, meaningful changes in scores, reliability, and systematic biases, respectively. Results Electronic surveys had fewer missing or ambiguous responses than paper surveys; however, the PEQ Social Burdens subscale could not be evaluated due to error in the creation of the electronic survey. No significant differences were found between scores of the two versions for any of the measures, but multiple participants had meaningful changes in the Appearance and Sounds PEQ subscales. All measures demonstrated acceptable reliability between versions, except the Appearance, Perceived Response, and Sounds subscales of the PEQ. No systematic biases in scores or usability concerns were found for any measures. Conclusions This study analysis showed that most of the electronic PROMs studied are easily used and demonstrate equivalence to the paper versions. However, the PEQ Appearance, Perceived Response, Sounds, and Social Burden subscales require further evaluation. Clinical Relevance Except for the PEQ, electronic versions of the PROMs in this study can likely be used interchangeably with paper versions among individuals with lower-limb loss.

Disconnected: Electronic Patient-Reported Outcome Measure Collection In Orthopaedic Patients at an Urban Safety-Net Hospital.

Collection of patient-reported outcome measures (PROMs) in orthopaedic patients at safety-net hospitals is challenging. The purpose of this study was to evaluate the success of electronic PROM (E-PROM) collection in this setting. A retrospective review identified 207 consecutive orthopaedic patients undergoing 77 elective arthroplasty procedures and 130 trauma procedures. E-PROMs were collected through automated e-mails from an online patient engagement platform (PatientIQ) at 2 weeks, 6 weeks, and 3 months postoperatively. Patients with trauma received the percentage of normal Single Assessment Numerical Evaluation (SANE) and Patient-Reported Outcomes Measurement Information System-Physical Function (PROMIS-PF). Arthroplasty patients received the Hip/Knee SANE, Hip/Knee Disability and Osteoarthritis Outcome Score-Joint Replacement (HOOS Jr/KOOS Jr), PROMIS Global Physical Health (PROMIS-G-PH), and Veterans RAND 12-Item (VR-12) Health Survey. Compared with patients with trauma, arthroplasty patients were older (median difference 18.0 years; 95% confidence interval [CI] 12.0-22.0; P < 0.0001), more likely to be Hispanic/Black (proportional difference 16.9%; CI 2.8-30.3%; P = 0.02), more likely to have noncommercial or no insurance (proportional difference 34.0%; CI 23.2-43.0%; P < 0.001), and did not differ in Area Deprivation Index or E-PROM completion at each time point. E-PROMs were completed at 2 weeks, 6 weeks, and 3 months by 25.1% (52 of 207), 24.6% (51 of 207), and 21.7% (45 of 207) of all patients, respectively. Trauma and arthroplasty patients had a similar rate of partial E-PROM completion. Patients who completed 3-month E-PROMs were less likely to be Hispanic/Black (PD -16.4%; CI -31.0 to -0.2%; P < 0.04); less likely to have noncommercial/no insurance (PD -20.0%; CI -35.5 to -4.5%; P = 0.01); and did not differ in age, sex, Area Deprivation Index, or procedure type. The low collection rate of E-PROMs from orthopaedic patients at safety-net hospitals should be weighed against their costs. E-PROM collection may exacerbate disparities in PROM collection among certain patient populations. Diagnostic Level III.

Electronic patient-reported outcome (e-PRO) monitoring for adverse event management during cabozantinib treatment in patients with advanced renal cell carcinoma: protocol for a three-arm, randomised, multicentre phase II trial (e-PRO vs paper-PRO or usual care)

IntroductionCabozantinib monotherapy is an option for treatment of advanced renal cell carcinoma (RCC). However, cabozantinib dose modification and discontinuation due to symptomatic adverse events (AEs) remains a challenge. The use...