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Radium-223, abiraterone, and enzalutamide have each been shown to significantly improve survival as monotherapy in patients with metastatic castration-resistant prostate cancer. However, effects of combination radium-223 plus abiraterone or enzalutamide on survival and safety remain unclear. This single-center retrospective cohort study used electronic health record data of patients with metastatic castration-resistant prostate cancer and bone metastases who were treated with radium-223 between April 1, 2014 and February 19, 2019. Patients who received radium-223 monotherapy were compared to patients who received a combination of radium-223 plus either abiraterone or enzalutamide. The primary endpoint was overall survival. Secondary endpoints included progression-free survival, time to symptomatic skeletal event, symptomatic skeletal event-free survival, and incidence of drug-related adverse events. Time-to-event analyses were estimated by log rank tests using Kaplan-Meier curves. Hazard ratios and 95% confidence intervals were derived from Cox proportional hazards models. Chi-square tests evaluated difference in serious adverse events between the two arms. A total of 60 patients met inclusion criteria (n = 41 in the monotherapy arm, n = 19 in the combination arm). Differences in median overall survival were not observed (12.7 vs. 12.8 months; HR 1.15, 95% CI 0.59-2.23; P = 0.68), but median progression-free survival was significantly longer in the combination arm (7.6 vs. 4.9 months; HR 1.94, 95% CI 1.11-3.40; P = 0.02). Significant differences were not observed in time to first SSE (P = 0.97), SSE-free survival (P = 0.16), or in the overall incidence of serious adverse events (P = 0.45). Combination radium-223 plus abiraterone or enzalutamide did not improve overall survival, but prolonged progression-free survival without increasing the incidence of serious adverse events in metastatic castration-resistant prostate cancer patients with bone metastases. However, these results are limited by small numbers and patient selection inherent in retrospective analysis.

Background Symptoms in patients with advanced cancer are often inadequately captured during encounters with the healthcare team. Emerging evidence demonstrates that weekly electronic home-based patient-reported symptom monitoring with automated alerts to clinicians reduces healthcare utilization, improves health-related quality of life, and lengthens survival. However, oncology practices have lagged in adopting remote symptom monitoring into routine practice, where specific patient populations may have unique barriers. One approach to overcoming barriers is utilizing resources from value-based payment models, such as patient navigators who are ideally positioned to assume a leadership role in remote symptom monitoring implementation. This implementation approach has not been tested in standard of care, and thus optimal implementation strategies are needed for large-scale roll-out. Methods This hybrid type 2 study design evaluates the implementation and effectiveness of remote symptom monitoring for all patients and for diverse populations in two Southern academic medical centers from 2021 to 2026. This study will utilize a pragmatic approach, evaluating real-world data collected during routine care for quantitative implementation and patient outcomes. The Consolidated Framework for Implementation Research (CFIR) will be used to conduct a qualitative evaluation at key time points to assess barriers and facilitators, implementation strategies, fidelity to implementation strategies, and perceived utility of these strategies. We will use a mixed-methods approach for data interpretation to finalize a formal implementation blueprint. Discussion This pragmatic evaluation of real-world implementation of remote symptom monitoring will generate a blueprint for future efforts to scale interventions across health systems with diverse patient populations within value-based healthcare models. Trial registration NCT04809740 ; date of registration 3/22/2021.

Aims We evaluated pedometry as a novel patient-cen-tered 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 PROCTCAE, 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.

[This corrects the article DOI: 10.1371/journal.pone.0253021.].

Real-world evidence is increasingly valued by regulators and payers. Central to this evidence base is patient-reported outcome data describing the impact of drugs on quality of life, daily activities and symptoms. Here, we highlight key challenges with current real-world, patient-reported outcome data and describe collaborative next steps for international stakeholders to overcome these issues.Real-world evidence is increasingly valued by regulators and payers. Central to this evidence base is patient-reported outcome data describing the impact of drugs on quality of life, daily activities and symptoms. Here, we highlight key challenges with current real-world, patient-reported outcome data and describe collaborative next steps for international stakeholders to overcome these issues.

Purpose The U.S. NCI’s PRO-CTCAE is a library of self-report items for assessing symptomatic adverse events in cancer clinical trials from the patient perspective. The aim of this study was to translate and linguistically validate a Spanish version. Methods PRO-CTCAE’s 124 items were translated from English into Spanish using multiple forward and back translations. Native Spanish speakers undergoing cancer treatment were enrolled at six cancer treatment sites. Participants each completed approximately 50 items and were then interviewed using cognitive probes. The interviews were analyzed at the item level by linguistic themes, and responses were examined for evidence of equivalence to English. Items for which ≥20 % of participants experienced difficulties were reviewed, and phrasing was revised and then retested in subsequent interviews. Items where <20 % of respondents experienced difficulties were also reviewed and were considered for rephrasing and retesting. Results One hundred nine participants from diverse Spanish-speaking countries were enrolled (77 in Round 1 and 32 in Round 2). A majority of items were well comprehended in Round 1. Two items presented difficulties in ≥20 % of participants and were revised/retested without further difficulties. Two items presented difficulties in <20 %, and when retested exhibited no further difficulties. Two items presented difficulties in <20 %, but were not revised due to lack of alternatives. Sixteen items presented difficulties in ≤12 % and were not revised because difficulties were minor. Conclusions The Spanish PRO-CTCAE has been developed and refined for use in Spanish-speaking populations, with high levels of comprehension and equivalence to the English PRO-CTCAE. Trial registration: ClinicalTrials.gov:NCT01436240

Background Good clinical care of prostate cancer patients after radical prostatectomy depends on careful assessment of post-operative morbidities, yet physicians do not always judge patient symptoms accurately. Logistical problems associated with using paper questionnaire limit their use in the clinic. We have implemented a web-interface (\"STAR\") for patient-reported outcomes after radical prostatectomy. Methods We analyzed data on the first 9 months of clinical implementation to evaluate the validity of the STAR questionnaire to assess functional outcomes following radical prostatectomy. We assessed response rate, internal consistency within domains, and the association between survey responses and known predictors of sexual and urinary function, including age, time from surgery, nerve sparing status and co-morbidities. Results Of 1581 men sent an invitation to complete the instrument online, 1235 responded for a response rate of 78%. Cronbach's alpha was 0.84, 0.86 and 0.97 for bowel, urinary and sexual function respectively. All known predictors of sexual and urinary function were significantly associated with survey responses in the hypothesized direction. Conclusions We have found that web-based assessment of functional recovery after radical prostatectomy is practical and feasible. The instrument demonstrated excellent psychometric properties, suggested that validity is maintained when questions are transferred from paper to electronic format and when patients give responses that they know will be seen by their doctor and added to their clinic record. As such, our system allows ready implementation of patient-reported outcomes into routine clinical practice.

INTRODUCTION:Patient-reported outcome (PRO) measures used during cancer care delivery improve communication about symptoms between patients and clinicians and reduce service utilization for uncontrolled symptoms. However, uptake of PROs in routine cancer care has been slow. In this paper, we describe stakeholder engagement activities used to overcome barriers to implementing PROs. Implementation occurred in 2 study settingsPROs completed in the waiting room and reviewed during clinical visits to guide symptom management for multiple myeloma (visit-based PROs); and weekly PROs completed by cancer patients between chemotherapy visits to monitor symptoms at home (remote PROs). METHODS:PRO implementation steps across studies included(1) clinician and patient input on key symptoms, PRO measures, and identifying which PRO responses are clinically concerning to better target nursing actions; (2) developing PRO-based clinical decision support (CDS) for responding to concerning PROs; (3) training clinicians and clinical research assistants to interpret PROs and use software; and (4) describing implementation impact (frequency of concerning PRO responses and nursing actions). DISCUSSION:Clinician and patient input was critical for identifying key symptoms, PRO measures, and clinically concerning response options. For the visit-based PRO observational study, all symptom scores appeared on a clinician dashboard, and those rated ≥1 by patients (on a 0–4 or 0–10 scale) had PRO-based CDS available for access. For the 2 remote PROs trials, stakeholders recommended that the 2 “worst” response options (eg, PRO responses of “often”/“always” or “severe”/“very severe”) would trigger an automated email alert to a nurse along with PRO-based CDS. In each study, PRO-based CDS was tailored based on clinician input. Across studies, the most common nursing response to concerning PROs was counseling patients on (or providing care plans for) self-management of symptoms. In the trials, the percentage of weekly remote PROs generating an alert to a nurse ranged from 13% at an academic center to 36% in community oncology practices. KEY POINTS:Across 3 prospective studies, PROs implemented into cancer care enabled tailored care based on issues identified on PROs. Stakeholder engagement was critical for successful implementation. This paper assists in addressing important PRO implementation challenges by describing a stakeholder-driven approach.

PurposeAs patient-reported outcome measures (PROMs) are increasingly used in clinical practice for screening, monitoring, and management, the potential for response bias has been raised (e.g., over-reporting problems for attention, under-reporting to avoid treatment changes/discontinuation). We investigated whether patients systematically bias their responses when they know clinicians will review their PROM results.MethodsWe conducted secondary analyses of three experimental studies evaluating PROMs in adult and pediatric care. Prior to PROM completion, intervention group patients were informed that the results would be shown to their clinicians (“feedback” arm), whereas control group patients were told that their clinicians would not see their responses (“no feedback” arm). Independent sample t-tests compared the “feedback” and “no feedback” arms’ PROM scores at baseline. Effect sizes and 95% confidence intervals were estimated using Cohen’s d statistics with Hedges’ g correction, and effect sizes > 0.50 were considered clinically relevant.ResultsAcross the 29 domains assessed in the three studies, no between-arm differences reached an effect size of ± 0.50. Only 3/29 effect sizes exceeded ± 0.30. The confidence intervals for 14 domains included ± 0.50, with 4 favoring the “no feedback” arm and 10 favoring the “feedback” arm. Two domains reached statistical significance, one favoring the “no feedback” arm and one favoring the “feedback” arm.ConclusionThis study does not support the hypothesis that patients systematically bias their PROM responses if they know that clinicians will see their results. These findings support using PROMs in clinical practice as a valid mechanism to promote patient-centered care.

Abstract Introduction Standard investigator-based adverse events (AE) assessment is via CTCAE for clinical trials. However, including the patient perspective through PRO (patient-reported outcomes) enhances clinicians’ understanding of patient toxicity and fosters early detection of AEs. We assessed longitudinal integration of PRO-CTCAE within clinical workflow in a phase II trial. Materials and methods As a sub-study in a phase II trial of genotype-directed irinotecan dosing evaluating efficacy in patients with metastatic colorectal cancer receiving FOLFIRI and bevacizumab, patients reported on 13 AEs generating a PRO-CTCAE form. The primary objective was to estimate forms completed by patients and clinicians at least 80% of time. Secondary objectives were estimating concordance and time to first score of specific symptoms between patient and clinician pairs. Results Feasibility of longitudinal PRO-CTCAE integration was met as 96% of patients and clinician-patient pairs completed at least 80% of PRO-CTCAE forms available to them with 79% achieving 100% completion. Concordance between patient and clinician reporting a severe symptom was 73% with 24 disconcordant pairs, 21 involved patients who reported a severe symptom that the clinician did not. Although protocol-mandated dose reductions were guided by CTCAE not PRO-CTCAE responses, the median time to dose reduction of 2.53 months, and the time-to-event curve closely approximated time to patient-reported toxicity. Conclusion Longitudinal integration of PRO-CTCAE paired CTCAE proved feasible. Compared to clinicians, patients reported severe symptoms more frequently and earlier. Patient-reported toxicity more closely aligned with dose decreases indicating incorporation into routine clinical practice may enhance early detection of toxicity improving patient safety and quality of life. This study showed that patient-reported outcomes are feasible, implementable in the context of a clinical trial, and allow patients the opportunity to report toxicity earlier which likely leads to earlier detection of effects on quality of life and improved patient safety.