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Aims: Many large-scale population-based surveys, research studies, and clinical care allow for inclusion of proxy reporting as a strategy to collect outcomes when patients are unavailable or unable to provide reliable self-report. Prior work identified an absence of methodological guidelines regarding proxy reporting in adult populations, including who can serve as a proxy, and considerations for data collection, analysis, and reporting. The primary objective of this work by the ISOQOL Proxy Task Force was to review documents and clinical outcome assessment measures with respect to proxy reporting and to develop, through consensus, considerations for proxy reporting. Methods: We assembled an international group with clinically relevant and/or methodological expertise on proxy use in adult populations. We conducted a targeted review of documentation based on regulatory, non-regulatory, professional society, and individual measure sources. Using a standardized collection form, proxy-related information was extracted from each source including definitions of a proxy, characteristics of a proxy, domains addressable or addressed by a proxy, and observer-reporting. Results: The definition of proxy was inconsistent across 39 sources, except regulatory documents which defined a proxy as a person other than the patient who reports on an outcome as if she/he were the patient. While proxy report was discouraged in regulatory documentation, it was acknowledged there were instances where self-report was impossible. Many documentation sources indicated proxies would be well-justified in certain contexts, but did not indicate who could act as a proxy, when proxies could be used, what domains of patient health they could report on, or how data should be reported. Observer-reported outcomes were typically defined as those based on observed behaviors, however there was not a consistent differentiation between proxy and observer reporting. Based on information extracted from these resources, we developed a checklist of considerations when including proxy-reported measures or using proxies in study design, data collection, analysis, interpretation and reporting of proxy reported data. Conclusion: Our targeted review highlights a lack of clarity in capturing, interpreting and reporting data from proxies in adult populations. We provide a checklist of considerations to assist researchers and clinicians with including proxies in research studies and clinical care. Lastly, our review identified areas where further guidance and future research are necessary. [ABSTRACT FROM AUTHOR]

PURPOSE Despite evidence-based guidelines recommending early palliative care, it remains unclear how to identify and refer oncology patients, particularly in settings with constrained access to palliative care. We hypothesize that patient-reported outcome (PRO) data can be used to characterize patients with palliative care needs. To determine if PRO data can identify latent phenotypes that characterize indications for specialty palliative care referral. METHODS We conducted a retrospective study of self-reported symptoms on the Edmonton Symptom Assessment System collected from solid tumor oncology patients (n = 745) referred to outpatient palliative care. Data were collected as part of routine clinical care from October 2012 to March 2018 at eight community and academic sites. We applied latent profile analysis to identify PRO phenotypes and examined the association of phenotypes with clinical and demographic characteristics using multinomial logistic regression. RESULTS We identified four PRO phenotypes: (1) Low Symptoms (n = 295, 39.6%), (2) Moderate Pain/Fatigue + Mood (n = 180, 24.2%), (3) Moderate Pain/Fatigue + Appetite + Dyspnea (n = 201, 27.0%), and (4) High Symptoms (n = 69, 9.3%). In a secondary analysis of 421 patients, we found that two brief items assessing social and existential needs aligned with higher severity symptom and psychological distress phenotypes. CONCLUSION Oncology patients referred to outpatient palliative care in a real-world setting can be differentiated into clinically meaningful phenotypes using brief, routinely collected PRO measures. Latent modeling provides a mechanism to use patient-reported data on a population level to identify distinct subgroups of patients with unmet palliative needs. [ABSTRACT FROM AUTHOR]

Introduction: Scant evidence reveals whether the use of weekly versus daily pain ratings leads to meaningful differences when measuring pain as a clinical trial outcome. We compared the ability of weekly ratings and descriptors of daily ratings to evaluate pain as an endpoint in a randomized phase 3 drug trial. Methods: Participants (n = 119) with metastatic castration-resistant prostate cancer were randomized to treatment arms and rated their pain on the average and at its worst during a baseline week and at weeks 3, 6, and 12 of study treatment. For each reporting period, participants rated their pain daily for 7 days. On day 7, participants rated their pain over the prior 7 days. We estimated mean differences and intraclass correlation coefficients of the weekly ratings and the mean and the maximum daily ratings. We compared the ability of the weekly ratings and the daily rating descriptors to detect change in pain and evaluated the agreement of the weekly rating and the mean daily rating of pain at its worst to detect treatment response. Results: For both pain constructs, the weekly rating was consistently higher than the mean daily rating and lower than the maximum daily rating yet was moderately to highly correlated with both daily rating descriptors (intraclass correlation coefficient range = 0.55–0.94). The weekly rating and the daily rating descriptors consistently detected change in pain for the study sample and participant subgroups. Substantial agreement existed between the weekly rating and the mean daily rating of pain at its worst when used with trial protocol opioid criteria to detect treatment response (Cohen's κ = 0.71). Conclusion: Use of daily over weekly ratings delivered no added benefit in evaluating pain in this clinical trial. This study is the first to compare weekly and daily recall to measure pain as an endpoint in a randomized phase 3 drug trial, and the pattern of differences in ratings that we observed is consistent with other recent evaluations of weekly and daily symptom reporting. [ABSTRACT FROM AUTHOR]

Background: The Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events is an item library designed for eliciting patient-reported adverse events in oncology. For each adverse event, up to three individual items are scored for frequency, severity, and interference with daily activities. To align the Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events with other standardized tools for adverse event assessment including the Common Terminology Criteria for Adverse Events, an algorithm for mapping individual items for any given adverse event to a single composite numerical grade was developed and tested. Methods: A five-step process was used: (1) All 179 possible Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events score combinations were presented to 20 clinical investigators to subjectively map combinations to single numerical grades ranging from 0 to 3. (2) Combinations with <75% agreement were presented to investigator committees at a National Clinical Trials Network cooperative group meeting to gain majority consensus via anonymous voting. (3) The resulting algorithm was refined via graphical and tabular approaches to assure directional consistency. (4) Validity, reliability, and sensitivity were assessed in a national study dataset. (5) Accuracy for delineating adverse events between study arms was measured in two Phase III clinical trials (NCT02066181 and NCT01522443). Results: In Step 1, 12/179 score combinations had <75% initial agreement. In Step 2, majority consensus was reached for all combinations. In Step 3, five grades were adjusted to assure directional consistency. In Steps 4 and 5, composite grades performed well and comparably to individual item scores on validity, reliability, sensitivity, and between-arm delineation. Conclusion: A composite grading algorithm has been developed and yields single numerical grades for adverse events assessed via the Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events, and can be useful in analyses and reporting. [ABSTRACT FROM AUTHOR]

Outcome measures for embedded pragmatic clinical trials (ePCTs) should reflect the lived experience of people living with dementia (PLWD) and their caregivers, yet patient‐ and caregiver‐reported outcomes (PCROs) are rarely available in large clinical and administrative data sources. Although pragmatic methods may lead to use of existing administrative data rather than new data collected directly from PLWD, interventions are truly impactful only when they change outcomes prioritized by PLWD and their caregivers. The Patient‐ and Caregiver‐Reported Outcomes Core (PCRO Core) of the IMbedded Pragmatic Alzheimer's Disease (AD) and AD‐Related Dementias Clinical Trials (IMPACT) Collaboratory aims to promote optimal use of outcomes relevant to PLWD and their caregivers in pragmatic trials. The PCRO Core will address key scientific challenges limiting outcome measurement, such as gaps in existing measures, methodologic constraints, and burdensome data capture. PCRO Core investigators will create a searchable library of AD/AD‐related dementias (ADRD) clinical outcome measures, including measures in existing data sources with potential for AD/ADRD ePCTs, and will support best practices in measure development, including pragmatic adaptation of PCROs. Working together with other Cores and Teams within the IMPACT Collaboratory, the PCRO Core will support investigators to select from existing outcome measures, and to innovate in methods for measurement and data capture. In the future, the work of the IMPACT Collaboratory may galvanize broader embedded use of outcomes that matter to PLWD and their care partners in large health systems. J Am Geriatr Soc 68:S55–S61, 2020. [ABSTRACT FROM AUTHOR]

PURPOSE: Patient-reported outcome measures (PROMs) that assess how patients feel and function have potential for evaluating quality of care. Stakeholder recommendations for PRO-based performance measures (PMs) were elicited, and feasibility testing was conducted at six cancer centers. METHODS: Interviews were conducted with 124 stakeholders to determine priority symptoms and risk adjustment variables for PRO-PMs and perceived acceptability. Stakeholders included patients and advocates, caregivers, clinicians, administrators, and thought leaders. Feasibility testing was conducted in six cancer centers. Patients completed PROMs at home 5-15 days into a chemotherapy cycle. Feasibility was operationalized as ≥ 75% completed PROMs and ≥ 75% patient acceptability. RESULTS: Stakeholder priority PRO-PMs for systemic therapy were GI symptoms (diarrhea, constipation, nausea, vomiting), depression/anxiety, pain, insomnia, fatigue, dyspnea, physical function, and neuropathy. Recommended risk adjusters included demographics, insurance type, cancer type, comorbidities, emetic risk, and difficulty paying bills. In feasibility testing, 653 patients enrolled (approximately 110 per site), and 607 (93%) completed PROMs, which indicated high feasibility for home collection. The majority of patients (470 of 607; 77%) completed PROMs without a reminder call, and 137 (23%) of 607 completed them after a reminder call. Most patients (72%) completed PROMs through web, 17% paper, or 2% interactive voice response (automated call that verbally asked patient questions). For acceptability, > 95% of patients found PROM items to be easy to understand and complete. CONCLUSION: Clinicians, patients, and other stakeholders agree that PMs that are based on how patients feel and function would be an important addition to quality measurement. This study also shows that PRO-PMs can be feasibly captured at home during systemic therapy and are acceptable to patients. PRO-PMs may add value to the portfolio of PMs as oncology transitions from fee-for-service payment models to performance-based care that emphasizes outcome measures. [ABSTRACT FROM AUTHOR]

Aims: We evaluated pedometry as a novel patient-centered outcome because it enables passive continuous assessment of activity and may provide information about the consequences of symptomatic toxicity complementary to self-report.Methods: Adult patients undergoing hematopoietic cell transplant (HCT) wore pedometers and completed PRO assessments during transplant hospitalization (4 weeks) and 4 weeks post-discharge. Patient reports of symptomatic treatment toxicities (single items from PRO-CTCAE, http://healthcaredelivery.cancer.gov/pro-ctcae ) and symptoms, physical health, mental health, and quality of life (PROMIS(®) Global-10, http://nih.promis.org ), assessed weekly with 7-day recall on Likert scales, were compared individually with pedometry data, summarized as average daily steps per week, using linear mixed models.Results: Thirty-two patients [mean age 55 (SD = 14), 63 % male, 84 % white, 56 % autologous, 43 % allogeneic] completed a mean 4.6 (SD = 1.5, range 1-8) evaluable assessments. Regression model coefficients (β) indicated within-person decrements in average daily steps were associated with increases in pain (β = -852; 852 fewer steps per unit increase in pain score, p < 0.001), fatigue (β = -886, p < 0.001), vomiting (β = -518, p < 0.01), shaking/chills (β = -587, p < 0.01), diarrhea (β = -719, p < 0.001), shortness of breath (β = -1018, p < 0.05), reduction in carrying out social activities (β = 705, p < 0.01) or physical activities (β = 618, p < 0.01), and global physical health (β = 101, p < 0.001), but not global mental health or quality of life.Conclusions: In this small sample of HCT recipients, more severe symptoms, impaired physical health, and restrictions in the performance of usual daily activities were associated with statistically significant decrements in objectively measured daily steps. Pedometry may be a valuable outcome measure and validation anchor in clinical research. [ABSTRACT FROM AUTHOR]

Background: PRO-CTCAE is a library of items that measure cancer treatment-related symptomatic adverse events (NCI Contracts: HHSN261201000043C and HHSN 261201000063C). The objective of this study is to examine the equivalence and acceptability of the three data collection modes (Web-enabled touchscreen tablet computer, Interactive voice response system [IVRS], and paper) available within the US National Cancer Institute (NCI) Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE) measurement system.Methods: Participants (n = 112; median age 56.5; 24 % high school or less) receiving treatment for cancer at seven US sites completed 28 PRO-CTCAE items (scoring range 0-4) by three modes (order randomized) at a single study visit. Subjects completed one page (approx. 15 items) of the EORTC QLQ-C30 between each mode as a distractor. Item scores by mode were compared using intraclass correlation coefficients (ICC); differences in scores within the 3-mode crossover design were evaluated with mixed-effects models. Difficulties with each mode experienced by participants were also assessed.Results: 103 (92 %) completed questionnaires by all three modes. The median ICC comparing tablet vs IVRS was 0.78 (range 0.55-0.90); tablet vs paper: 0.81 (0.62-0.96); IVRS vs paper: 0.78 (0.60-0.91); 89 % of ICCs were ≥0.70. Item-level mean differences by mode were small (medians [ranges] for tablet vs. IVRS = -0.04 [-0.16-0.22]; tablet vs paper = -0.02 [-0.11-0.14]; IVRS vs paper = 0.02 [-0.07-0.19]), and 57/81 (70 %) items had bootstrapped 95 % CI around the effect sizes within +/-0.20. The median time to complete the questionnaire by tablet was 3.4 min; IVRS: 5.8; paper: 4.0. The proportion of participants by mode who reported "no problems" responding to the questionnaire was 86 % tablet, 72 % IVRS, and 98 % paper.Conclusions: Mode equivalence of items was moderate to high, and comparable to test-retest reliability (median ICC = 0.80). Each mode was acceptable to a majority of respondents. Although the study was powered to detect moderate or larger discrepancies between modes, the observed ICCs and very small mean differences between modes provide evidence to support study designs that are responsive to patient or investigator preference for mode of administration, and justify comparison of results and pooled analyses across studies that employ different PRO-CTCAE modes of administration.Trial Registration: NCT Clinicaltrials.gov identifier: NCT02158637. [ABSTRACT FROM AUTHOR]

Answer questions and earn CME/CNE Patient-reported outcome (PRO) questionnaires assess topics a patient can report about his or her own health. This includes symptoms (eg, nausea, fatigue, diarrhea, pain, or frequent urination), physical functioning (eg, difficulty climbing stairs or difficulty fastening buttons), and mental health (eg, anxiety, fear, or worry). Electronic PRO (ePRO) systems are used in oncology clinical care because of 1) their ability to enhance clinical care by flagging important symptoms and saving clinicians time; 2) the availability of standardized methods for creating and implementing PROs in clinics; and 3) the existence of user-friendly platforms for patient self-reporting like tablet computers and automated telephone surveys. Many ePRO systems can provide actionable links to clinical care such as summary reports in a patient's electronic medical record and real-time e-mail alerts to providers when patients report acute needs. This review presents 5 examples of ePRO systems currently in use in oncology practice. These systems support multiple clinical activities, including assessment of symptoms and toxicities related to chemotherapy and radiation, postoperative surveillance, and symptom management during palliative care and hospice. Patient self-reporting is possible both at clinical visits and between visits over the Internet or by telephone. The implementation of an ePRO system requires significant resources and expertise, as well as user training. ePRO systems enable regular monitoring of patient symptoms, function, and needs, and can enhance the efficiency and quality of care as well as communication with patients. CA Cancer J Clin 2012. © 2012 American Cancer Society. [ABSTRACT FROM AUTHOR]

Background: Traditional concordance metrics have shortcomings based on dataset characteristics (e.g., multiple attributes rated, missing data); therefore it is necessary to explore supplemental approaches to quantifying agreement between independent assessments. The purpose of this methodological paper is to apply an Item Response Theory (IRT) -based framework to an existing dataset that included unidimensional clinician and multiple attribute patient ratings of symptomatic adverse events (AEs), and explore the utility of this method in patient-reported outcome (PRO) and health-related quality of life (HRQOL) research. Methods: Data were derived from a National Cancer Institute-sponsored study examining the validity of a measurement system (PRO-CTCAE) for patient self-reporting of AEs in cancer patients receiving treatment (N = 940). AEs included 13 multiple attribute patient-reported symptoms that had corresponding unidimensional clinician AE grades. A Bayesian IRT Model was fitted to calculate the latent grading thresholds between raters. The posterior mean values of the model-fitted item responses were calculated to represent model-based AE grades obtained from patients and clinicians. Results: Model-based AE grades showed a general pattern of clinician underestimation relative to patient-graded AEs. However, the magnitude of clinician underestimation was associated with AE severity, such that clinicians' underestimation was more pronounced for moderate/very severe model-estimated AEs, and less so with mild AEs. Conclusions: The Bayesian IRT approach reconciles multiple symptom attributes and elaborates on the patterns of clinician-patient non-concordance beyond that provided by traditional metrics. This IRT-based technique may be used as a supplemental tool to detect and characterize nuanced differences in patient-, clinician-, and proxy-based ratings of HRQOL and patient-centered outcomes. Trial registration: ClinicalTrials.gov NCT01031641. Registered 1 December 2009. [ABSTRACT FROM AUTHOR]

Context. Patient-reported outcomes (PROs) provide a way to understand the effects of hematopoietic cell transplantation (HCT)-related stress on patients' lives. We previously reported that weekly collection of PROs is feasible. Objectives. Here, we report on the feasibility of daily patient-reported symptom collection and examine the relationship between daily vs. weekly symptom reporting over time. Methods. We analyzed data from 32 autologous and allogeneic HCT patients obtained until Day (D) +100. We used questions from the PRO version of the Common Terminology Criteria for Adverse Events to capture symptoms. Results. We found that overall rates of daily survey completion were moderate to high (range 67%-86%). The effect size of the difference between the maximum daily severity score and the weekly severity score ranged from 0.15 to 0.35, and the concordance correlation coefficient ranged from 0.513 to 0.834. Concordance of daily and weekly surveys was higher for maximum daily severity rating and mean daily severity rating than for minimum daily severity rating or most recent daily severity rating. Conclusion. We conclude that a seven-day recall period for symptom severity provides acceptable accuracy and precision in the first 100 days after HCT. Further studies to explore the utility of daily symptom reporting within specific clinical contexts may be warranted. [ABSTRACT FROM AUTHOR]

The objective of this report was to address the use and mixing of data collection modes within and between trials in which patient-reported outcome (PRO) end points are intended to be used to support medical product labeling. The report first addresses the factors that should be considered when selecting a mode or modes of PRO data collection in a clinical trial, which is often when mixing is first considered. Next, a summary of how to "faithfully" migrate instruments is presented followed by a section on qualitative and quantitative study designs used to evaluate measurement equivalence of the new and original modes of data collection. Finally, the report discusses a number of issues that must be taken into account when mixing modes is deemed necessary or unavoidable within or between trials, including considerations of the risk of mixing at different levels within a clinical trial program and mixing between different types of platforms. In the absence of documented evidence of measurement equivalence, it is strongly recommended that a quantitative equivalence study be conducted before mixing modes in a trial to ensure that sufficient equivalence can be demonstrated to have confidence in pooling PRO data collected by the different modes. However, we also strongly discourage the mixing of paper and electronic field-based instruments and suggest that mixing of electronic modes be considered for clinical trials and only after equivalence has been established. If proceeding with mixing modes, it is important to implement data collection carefully in the trial itself in a planned manner at the country level or higher and minimize ad hoc mixing by sites or individual subjects. Finally, when mixing occurs, it must be addressed in the statistical analysis plan for the trial and the ability to pool the data must be evaluated to then evaluate treatment effects with mixed modes data. A successful mixed modes trial requires a "faithful migration," measurement equivalence established between modes, and carefully planned implementation to minimize the risk of increased measurement error impacting the power of the trial to detect a treatment effect. [ABSTRACT FROM AUTHOR]