Home Time Among Older Adults With Acute Myeloid Leukemia Following Chemotherapy.

Importance: Patients with acute myeloid leukemia (AML) recognize days spent at home (home time) vs in a hospital or nursing facility as an important factor in treatment decision making. No study has adequately described home time among older adults with AML. Objective: To describe home time among older adults with AML (aged ≥66 years) and compare home time between 2 common treatments: anthracycline-based chemotherapy and hypomethylating agents (HMAs). Design, Setting, and Participants: A cohort of adults aged 66 years or older with a new diagnosis of AML from the Surveillance, Epidemiology, and End Results (SEER)-Medicare linked database in 2004 to 2016 was identified. Individuals were stratified into anthracycline-based therapy, HMAs, or chemotherapy, not otherwise specified (NOS) using claims. Main Outcomes and Measures: The primary outcome was home time, quantified by subtracting the total number of person-days spent in hospitals and nursing facilities from the number of person-days survived and dividing by total person-days. A weighted multinomial regression model with stabilized inverse probability of treatment weighting to estimate adjusted home time was used. Results: The cohort included 7946 patients with AML: 2824 (35.5%) received anthracyclines, 2542 (32.0%) HMAs, and 2580 (32.5%) were classified as chemotherapy, NOS. Median (IQR) survival was 11.0 (5.0-27.0) months for those receiving anthracyclines and 8.0 (3.0-17.0) months for those receiving HMAs. Adjusted home time for all patients in the first year was 52.4%. Home time was highest among patients receiving HMAs (60.8%) followed by those receiving anthracyclines (51.9%). Despite having a shorter median survival, patients receiving HMAs had more total days at home and 33 more days at home in the first year on average than patients receiving anthracyclines (222 vs 189). Conclusions and Relevance: This retrospective study of older adults with AML using SEER-Medicare data and propensity score weighting suggests that the additional survival afforded by receiving anthracycline-based therapy was entirely offset by admission to the hospital or to nursing facilities.

Clinical Decision Support for Surgery: A Mixed Methods Study on Design and Implementation Perspectives From Urologists.

OBJECTIVE: To assess urologist attitudes toward clinical decision support (CDS) embedded into the electronic health record (EHR) and define design needs to facilitate implementation and impact. With recent advances in big data and artificial intelligence (AI), enthusiasm for personalized, data-driven tools to improve surgical decision-making has grown, but the impact of current tools remains limited. METHODS: A sequential explanatory mixed methods study from 2019 to 2020 was performed. First, survey responses from the 2019 American Urological Association Annual Census evaluated attitudes toward an automatic CDS tool that would display risk/benefit data. This was followed by the purposeful sampling of 25 urologists and qualitative interviews assessing perspectives on CDS impact and design needs. Bivariable, multivariable, and coding-based thematic analysis were applied and integrated. RESULTS: Among a weighted sample of 12,366 practicing urologists, the majority agreed CDS would help decision-making (70.9%, 95% CI 68.7%-73.2%), aid patient counseling (78.5%, 95% CI 76.5%-80.5%), save time (58.1%, 95% CI 55.7%-60.5%), and improve patient outcomes (42.9%, 95% CI 40.5%-45.4%). More years in practice was negatively associated with agreement (P <.001). Urologists described how CDS could bolster evidence-based care, personalized medicine, resource utilization, and patient experience. They also identified multiple implementation barriers and provided suggestions on form, functionality, and visual design to improve usefulness and ease of use. CONCLUSION: Urologists have favorable attitudes toward the potential for clinical decision support in the EHR. Smart design will be critical to ensure effective implementation and impact.

Emotional and functional well-being in long-term breast cancer survivorship.

PURPOSE: Emotional and functional well-being (EWB and FWB) are important components of mental health and quality of life. This study aims to evaluate long-term EWB and FWB in breast cancer (BC) survivors. METHODS: The Carolina Breast Cancer Study Phase 3 oversampled Black and younger (< 50 years in age) women so that they each represent approximately 50% of the study population and assessed participants' EWB and FWB with the Functional Assessment of Cancer Therapy-Breast (FACT-B) at 5- (baseline), 25-, and 84-months post diagnosis. Multinomial logit models were used to estimate adjusted odds ratios (ORs) and 95% confidence intervals (CIs) for associations between demographic and clinical characteristics and well-being change relative to baseline. RESULTS: Among 2,781 participants with BC, average EWB and FWB improved with time since diagnosis. Persistent FWB decrements were associated with Black race [OR 1.4 (95% CI 1.2-1.7) and 1.3 (95% CI 1.1-1.6), at 25-months and 84-months respectively], older age [OR 1.4 (95% CI 1.1-1.7) and 1.5 (95% CI 1.2-1.8), respectively], no chemotherapy, and recurrence [OR 2.9 (95% CI 1.8-4.8) and 3.1 (95% CI 2.1-4.6), respectively]. EWB decrements were associated with advanced stage and recurrence. Decrements in combined (FWB+EWB) well-being were associated with recurrence at both follow-up survey timepoints [ORs 4.7 (95% CI 2.7-8.0) and 4.3 (95% CI 2.8-6.6), respectively]. CONCLUSIONS: Long-term well-being varies by demographics and clinical features, with Black women and women with aggressive disease at greatest risk of long-term decrements.

Benefits, Harms, and Stakeholder Perspectives Regarding Opioid Therapy for Pain in Individuals With Metastatic Cancer: Protocol for a Descriptive Cohort Study.

BACKGROUND: Opioids are a key component of pain management among patients with metastatic cancer pain. However, the evidence base available to guide opioid-related decision-making in individuals with advanced cancer is limited. Patients with advanced cancer or cancer that is unlikely to be cured frequently experience pain. Opioids are a key component of pain management among patients with metastatic cancer pain. Many individuals with advanced cancer are now living long enough to experience opioid-related harm. Emerging evidence from chronic noncancer pain literature suggests that longer-term opioid therapy may have limited benefits for pain and function, and opioid-related harms are also a major concern. However, whether these benefits and harms of opioids apply to patients with cancer-related pain is unknown. OBJECTIVE: This manuscript outlines the protocol for the "Opioid Therapy for Pain in Individuals With Metastatic Cancer: The Benefits, Harms, and Stakeholder Perspectives (BEST) Study." The study aims to better understand opioid decision-making in patients with advanced cancer, along with opioid benefits and harms, through prospective examination of patients' pain experiences and opioid side effects and understanding the decision-making by patients, care partners, and clinicians. METHODS: This is a multicenter, prospective cohort study that aims to enroll 630 patients with advanced cancer, 20 care partners, and 20 clinicians (670 total participants). Patient participants must have an advanced solid cancer diagnosis, defined by the American Cancer Society as cancer that is unlikely to be cured. We will recruit patient participants within 12 weeks after diagnosis so that we can understand opioid benefits, harms, and perspectives on opioid decision-making throughout the course of their advanced cancer (up to 2 years). We will also specifically elicit information regarding long-term opioid use (ie, opioids for ≥90 consecutive days) and exclude patients on long-term opioid therapy before an advanced cancer diagnosis. Lived-experience perspectives related to opioid use in those with advanced cancer will be captured by qualitative interviews with a subset of patients, clinicians, and care partners. Our data collection will be grounded in a behavioral decision research approach that will allow us to develop future interventions to inform opioid-related decision-making for patients with metastatic cancer. RESULTS: Data collection began in October 2022 and is anticipated to end by November 2024. CONCLUSIONS: Upon successful execution of our study protocol, we anticipate the development of a comprehensive evidence base on opioid therapy in individuals with advanced cancer guided by the behavioral decision research framework. The information gained from this study will be used to guide interventions to facilitate opioid decisions among patients, clinicians, and care partners. Given the limited evidence base about opioid therapy in people with cancer, we envision this study will have significant real-world implications for cancer-related pain management and opioid-related clinical decision-making. INTERNATIONAL REGISTERED REPORT IDENTIFIER (IRRID): DERR1-10.2196/54953.

Development of a Lived Experience Panel to inform the design of embedded pragmatic trials of dementia care interventions.

BACKGROUND: The National Institute on Aging (NIA) Imbedded Pragmatic Alzheimer's Disease and Alzheimer's Related Dementia Clinical Trials (IMPACT) Collaboratory convened a Lived Experience Panel (LEP) to inform the development of research priorities and provide input on conducting embedded pragmatic clinical trials (ePCTs) of dementia care interventions. Given the importance of people with lived experience to dementia research, and the unique considerations of engaging people with dementia, we report on our process for the recruitment, selection, and initial convening of the IMPACT LEP. METHODS: The IMPACT Engaging Partners Team, in partnership with the Alzheimer's Association, sought nominations of individuals with mild cognitive impairment or early-stage dementia, care partners of other people living with dementia (PLWD), and proxy representatives for individuals with mid-to-late stage dementia. The 11-member LEP was composed of individuals with diverse personal experiences in part due to their age, race, ethnicity, gender, sexual orientation, geography, disability, or type of dementia. In its first year, the LEP met with IMPACT's Patient and Caregiver Relevant Outcomes Core and Ethics and Regulation Core. RESULTS: LEP members provided valuable insights and nuanced discussion of issues relevant to ePCTs in dementia care from a broad range of personal experience. Panelists identified key research priorities and provided insight on outcomes often studied by researchers. The LEP also informed investigators' approaches to waivers and modifications of written informed consent and evaluation of minimal risk. Summary reports of the LEP meetings with each Core are available on the IMPACT website. At the end of the first year, changes were made to the composition of the LEP, and opportunities were identified for expanding panelist engagement with IMPACT investigators, as were priorities and scope for future input. CONCLUSIONS: The IMPACT LEP provides a model for engaging PLWD and care partners in the research process as collaborators.

Selecting outcomes for pragmatic clinical trials in dementia care: The IMPACT Collaboratory iLibrary.

BACKGROUND: Many interventions improve care and outcomes for people with Alzheimer's Disease and related dementias (ADRD), yet are never disseminated. Pragmatic trials facilitate the adoption and dissemination of best practices, but gaps in pragmatic outcome measurement are a critical obstacle. Our objectives are (1) to describe the development and structure of the IMbedded Pragmatic ADRD Clinical Trials Collaboratory (IMPACT) iLibrary of potential outcome measures for ADRD pragmatic trials, and (2) to assess their pragmatic characteristics. METHODS: We identified potential outcome measures from several sources: a database of administrative and clinical outcome measures from ADRD clinical trials registered in ClinicalTrials.gov, published reviews, and IMPACT pilot pragmatic trial outcome measures. The iLibrary reports (a) number of items, (b) completion time, (c) readability for diverse populations, (d) cost or copyright barriers to use, (e) method of administration, (f) assessor training burden, and (g) feasibility of data capture and interpretation in routine care; a summary of pragmatic characteristics of each outcome measure (high, moderate, low); items or descriptions of items; and links to primary citations regarding development or psychometric properties. RESULTS: We included 140 outcome measures in the iLibrary: 66 administrative (100% were pragmatic) and 74 clinical (52% were pragmatic). The most commonly addressed outcome domains from administrative assessments included physical function, quality of care or communication concerns, and psychological symptoms or distress behaviors. The most commonly addressed outcome domains from clinical assessments were psychological symptoms or distress behaviors, physical function, cognitive function, and health-related quality of life. CONCLUSIONS: Pragmatic outcome measures are brief, meaningful to diverse populations, easily scored and interpreted by clinicians, and available in electronic format for analysis. The iLibrary can facilitate the selection of measures for a wide range of outcomes relevant to people with ADRD and their care partners.

Development of consensus-based considerations for use of adult proxy reporting: an ISOQOL task force initiative.

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.

Dementia and electronic health record phenotypes: a scoping review of available phenotypes and opportunities for future research.

OBJECTIVE: We performed a scoping review of algorithms using electronic health record (EHR) data to identify patients with Alzheimer's disease and related dementias (ADRD), to advance their use in research and clinical care. MATERIALS AND METHODS: Starting with a previous scoping review of EHR phenotypes, we performed a cumulative update (April 2020 through March 1, 2023) using Pubmed, PheKB, and expert review with exclusive focus on ADRD identification. We included algorithms using EHR data alone or in combination with non-EHR data and characterized whether they identified patients at high risk of or with a current diagnosis of ADRD. RESULTS: For our cumulative focused update, we reviewed 271 titles meeting our search criteria, 49 abstracts, and 26 full text papers. We identified 8 articles from the original systematic review, 8 from our new search, and 4 recommended by an expert. We identified 20 papers describing 19 unique EHR phenotypes for ADRD: 7 algorithms identifying patients with diagnosed dementia and 12 algorithms identifying patients at high risk of dementia that prioritize sensitivity over specificity. Reference standards range from only using other EHR data to in-person cognitive screening. CONCLUSION: A variety of EHR-based phenotypes are available for use in identifying populations with or at high-risk of developing ADRD. This review provides comparative detail to aid in choosing the best algorithm for research, clinical care, and population health projects based on the use case and available data. Future research may further improve the design and use of algorithms by considering EHR data provenance.

Electronic Health Record Use and Perceptions among Urologic Surgeons.

OBJECTIVE: Electronic health records (EHRs) have become widely adopted with increasing emphasis on improving care delivery. Improvements in surgery may be limited by specialty-specific issues that impact EHR usability and engagement. Accordingly, we examined EHR use and perceptions in urology, a diverse surgical specialty. METHODS: We conducted a national, sequential explanatory mixed methods study. Through the 2019 American Urological Association Census, we surveyed urologic surgeons on EHR use and perceptions and then identified associated characteristics through bivariable and multivariable analyses. Using purposeful sampling, we interviewed 25 urologists and applied coding-based thematic analysis, which was then integrated with survey findings. RESULTS: Among 2,159 practicing urologic surgeons, 2,081 (96.4%) reported using an EHR. In the weighted sample (n = 12,366), over 90% used the EHR for charting, viewing results, and order entry with most using information exchange functions (59.0-79.6%). In contrast, only 35.8% felt the EHR increases clinical efficiency, whereas 43.1% agreed it improves patient care, which related thematically to information management, administrative burden, patient safety, and patient-surgeon interaction. Quantitatively and qualitatively, use and perceptions differed by years in practice and practice type with more use and better perceptions among more recent entrants into the urologic workforce and those in academic/multispecialty practices, who may have earlier EHR exposure, better infrastructure, and more support. CONCLUSION: Despite wide and substantive usage, EHRs engender mixed feelings, especially among longer-practicing surgeons and those in lower-resourced settings (e.g., smaller and private practices). Beyond reducing administrative burden and simplifying information management, efforts to improve care delivery through the EHR should focus on surgeon engagement, particularly in the community, to boost implementation and user experience.

Real-world treatment patterns and outcomes for patients with advanced melanoma treated with immunotherapy or targeted therapy.

OBJECTIVE: To identify real-world patterns of first line treatment, treatment sequence and outcomes for older adults diagnosed with advanced melanoma who received immunotherapy or targeted therapy. METHODS: The study population included older adults (ages 65+) diagnosed with unresectable or metastatic melanoma between 2012 and 2017 and who received first line immunotherapy or targeted therapy. Using the linked surveillance, epidemiology, and end results-medicare data, we described patterns of first line treatment and treatment sequence through 2018. We used descriptive statistics to report patient and provider characteristics by first line treatment receipt and changes in first line therapy use over calendar time. We also described overall survival (OS) and time to treatment failure (TTF) by first line treatment using the Kaplan-Meier method. For patterns of treatment sequence, we reported commonly observed treatment switch patterns by treatment sub-category and calendar year. RESULTS: The analyses included 584 patients (mean age = 76.3 years). A majority (n = 502) received first line immunotherapy. There was a sustained increase in immunotherapy uptake, most notably from 2015 to 2016. The estimated median OS and TTF were longer with first line immunotherapy than with targeted therapy. Individuals treated with CTLA-4 + PD-1 inhibitors had the longest median OS (28.4 months). The most common treatment switch pattern was from a first line CTLA-4 inhibitor to a second line PD-1 inhibitor. CONCLUSIONS: Our findings inform understanding of treatment patterns of currently used immunotherapies and targeted therapies in older adults with advanced melanoma. Immunotherapy use has increased steadily with PD-1 inhibitors becoming a dominant treatment option since 2015.

Influence of family planning and immunization services integration on contraceptive use and family planning information and knowledge among clients: A cross-sectional analysis in urban Nigeria.

Reproductive autonomy necessitates that women have access to high quality family planning information and services. Additionally, closely spaced pregnancies increase maternal and infant morbidity and mortality. Although integrating family planning into child immunization services may increase access to information and services and postpartum contraceptive use, evidence on how integration affects service delivery and health outcomes is scarce. One limitation of previous studies is the use of binary integration measures. To address this limitation, this study applied Provider and Facility Integration Index scores to estimate associations between integration and contraceptive use, receipt of family planning information, and knowledge of family planning services availability. This study leveraged pooled cross-sectional health facility client exit interview data collected from 2,535 women in Nigeria. Provider and Facility Integration Index scores were calculated (0-10, 0 = low, 10 = high) for each facility (N = 94). The Provider Integration Index score measures provider skills and practices that support integrated service delivery; the Facility Integration Index score measures facility norms that support integrated service delivery. Logistic regression models identified associations between Provider and Facility Integration Index scores and (a) contraceptive use among postpartum women, (b) receipt of family planning information during immunization visits, and (c) correct identification of family planning service availability. Overall, 46% of women were using any method of contraception, 51% received family planning information during the immunization appointment, and 83% correctly identified family planning service availability at the facility. Mean Provider and Facility Integration Index scores were 6.46 (SD = 0.21) and 7.27 (SD = 0.18), respectively. Provider and Facility Integration Index scores were not significantly associated with postpartum contraceptive use. Facility Integration Index scores were negatively associated with receipt of family planning information. Provider Integration Index scores were positively associated with correct identification of family planning service availability. Our results challenge the position that integration provides a clear path to improved outcomes. The presence of facility and provider attributes that support integration may not result in the delivery of integrated care.

An Exploratory Analysis of the "Was It Worth It?" Questionnaire as a Novel Metric to Capture Patient Perceptions of Cancer Treatment.

OBJECTIVES: Asking "Was it worth it?" (WIWI) potentially captures the patient perception of a treatment's benefit weighed against its harms. This exploratory analysis evaluates the WIWI questionnaire as a metric of patients' perspectives on the worthwhileness of cancer treatment. METHODS: A 3-item WIWI questionnaire was assessed at end of treatment in patients with cancer on the COMET-2 trial (NCT01522443). WIWI items were evaluated to determine their association with quality of life (QOL), treatment duration, end-of-treatment reason, patient-reported adverse events (AEs), and disease response. RESULTS: A total of 65 patients completed the questionnaire; 40 (62%), 16 (25%), and 9 (14%) patients replied yes, uncertain, and no to "Was it worthwhile for you to receive the cancer treatment given in this study?" (item 1), respectively; 39 (60%), 12 (18%), and 14 (22%) to "If you had to do it over again, would you choose to have this cancer treatment?"; and 40 (62%), 14 (22%), and 11 (17%) to "Would you recommend this cancer treatment to others?" Patients responding yes to item 1 remained on treatment longer than those responding uncertain or no (mean 23.0 vs 11.3 weeks, P<.001). Patients responding uncertain/no to item 1 discontinued treatment because of AEs more frequently than those responding yes (36% vs 7.5%, P=.004) and demonstrated meaningful decline in QOL from baseline (-2.5 vs -0.2 mean change, P<.001). Associations between WIWI responses and most patient-reported AEs or treatment efficacy did not reach statistical significance. CONCLUSIONS: Patients who responded affirmatively on WIWI items remained on therapy longer, were less likely to stop treatment because of AEs, and demonstrated superior QOL. The WIWI may inform clinical practice, oncology research, and value frameworks.

Effect of Electronic Symptom Monitoring on Patient-Reported Outcomes Among Patients With Metastatic Cancer: A Randomized Clinical Trial.

Importance: Electronic systems that facilitate patient-reported outcome (PRO) surveys for patients with cancer may detect symptoms early and prompt clinicians to intervene. Objective: To evaluate whether electronic symptom monitoring during cancer treatment confers benefits on quality-of-life outcomes. Design, Setting, and Participants: Report of secondary outcomes from the PRO-TECT (Alliance AFT-39) cluster randomized trial in 52 US community oncology practices randomized to electronic symptom monitoring with PRO surveys or usual care. Between October 2017 and March 2020, 1191 adults being treated for metastatic cancer were enrolled, with last follow-up on May 17, 2021. Interventions: In the PRO group, participants (n = 593) were asked to complete weekly surveys via an internet-based or automated telephone system for up to 1 year. Severe or worsening symptoms triggered care team alerts. The control group (n = 598) received usual care. Main Outcomes and Measures: The 3 prespecified secondary outcomes were physical function, symptom control, and health-related quality of life (HRQOL) at 3 months, measured by the European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire (QLQ-C30; range, 0-100 points; minimum clinically important difference [MCID], 2-7 for physical function; no MCID defined for symptom control or HRQOL). Results on the primary outcome, overall survival, are not yet available. Results: Among 52 practices, 1191 patients were included (mean age, 62.2 years; 694 [58.3%] women); 1066 (89.5%) completed 3-month follow-up. Compared with usual care, mean changes on the QLQ-C30 from baseline to 3 months were significantly improved in the PRO group for physical function (PRO, from 74.27 to 75.81 points; control, from 73.54 to 72.61 points; mean difference, 2.47 [95% CI, 0.41-4.53]; P = .02), symptom control (PRO, from 77.67 to 80.03 points; control, from 76.75 to 76.55 points; mean difference, 2.56 [95% CI, 0.95-4.17]; P = .002), and HRQOL (PRO, from 78.11 to 80.03 points; control, from 77.00 to 76.50 points; mean difference, 2.43 [95% CI, 0.90-3.96]; P = .002). Patients in the PRO group had significantly greater odds of experiencing clinically meaningful benefits vs usual care for physical function (7.7% more with improvements of ≥5 points and 6.1% fewer with worsening of ≥5 points; odds ratio [OR], 1.35 [95% CI, 1.08-1.70]; P = .009), symptom control (8.6% and 7.5%, respectively; OR, 1.50 [95% CI, 1.15-1.95]; P = .003), and HRQOL (8.5% and 4.9%, respectively; OR, 1.41 [95% CI, 1.10-1.81]; P = .006). Conclusions and Relevance: In this report of secondary outcomes from a randomized clinical trial of adults receiving cancer treatment, use of weekly electronic PRO surveys to monitor symptoms, compared with usual care, resulted in statistically significant improvements in physical function, symptom control, and HRQOL at 3 months, with mean improvements of approximately 2.5 points on a 0- to 100-point scale. These findings should be interpreted provisionally pending results of the primary outcome of overall survival. Trial Registration: ClinicalTrials.gov Identifier: NCT03249090.

Can Steps per Day Reflect Symptoms in Children and Adolescents Undergoing Cancer Treatment?

BACKGROUND: Multiple symptoms occur in children receiving cancer therapy. Decreased steps per day may be associated with burdensome symptoms. OBJECTIVE: To evaluate associations between self-reported symptoms (pain interference, anxiety, depressive symptoms, psychological stress, and fatigue) and function (physical function-mobility and physical activity) and cumulative symptom count with steps per day. METHODS: Five sites enrolled English-speaking children, 8 to 17 years, receiving treatment for a first cancer diagnosis. Patient-reported outcome (PRO) surveys were administered before (T1) and after (T2) a course of chemotherapy. Garmin VivoFit 3 (Garmin International, Olathe, KS) accelerometers were worn 7 days prior to each data point. Univariate changes in scores over time were evaluated with dependent-sample t tests. Pearson correlations examined associations between PRO domains and step count. Multivariable mixed-effect models examined associations between steps and PROs. RESULTS: Participants' (n = 65) steps per day decreased during treatment (4099 [T1] and 3135 [T2]; P < .01), with larger reductions observed during hospitalization and in younger children compared with adolescents. Steps significantly correlated with PROMIS (Patient-Reported Outcome Measurement Information System) Pediatric physical activity and physical function-mobility. Decreased steps per day were associated with increased fatigue and cumulative symptom count. CONCLUSIONS: In children and adolescents with cancer, steps per day can serve as an indicator of fatigue, cumulative symptom count, physical activity, and physical functioning-mobility. IMPLICATIONS FOR PRACTICE: Child self-reports of physical activity and physical function are valid during cancer therapy and should be captured. In the absence of self-report, decreasing step count may prompt additional assessments related to fatigue or cumulative symptom count and trigger early interventions to support physical activity and physical function-mobility.

Electronic patient-reported outcomes monitoring during lung cancer chemotherapy: A nested cohort within the PRO-TECT pragmatic trial (AFT-39).

OBJECTIVES: Patients with lung cancer have high symptom burden and diminished quality of life. Electronic patient-reported outcome (PRO) platforms deliver repeated longitudinal surveys via web or telephone to patients and alert clinicians about concerning symptoms. This study aims to determine feasibility of electronic PRO monitoring in lung cancer patients receiving treatment in community settings. METHODS: Adults receiving treatment for advanced or metastatic lung cancer at 26 community sites were invited to participate in a prospective trial of weekly electronic PRO symptom monitoring for 12 months (NCT03249090). Surveys assessing patients' satisfaction with the electronic PRO system were administered at 3 months. Descriptive statistics were generated for demographics, survey completion rates, symptom occurrence, and provider PRO alert management approaches. Pairwise relationships between symptom items were evaluated using intra-individual repeated-measures correlation coefficients. RESULTS: Lung cancer patients (n = 118) participating in electronic PROs were older (mean 64.4 vs 61.9 years, p = 0.03), had worse performance status (p = 0.002), more comorbidities (p = 0.02), and less technology experience than patients with other cancers. Of delivered weekly PRO surveys over 12 months, 91% were completed. Nearly all (97%) patients reported concerning (i.e., severe or worsening) symptoms during participation, with 33% of surveys including concerning symptoms. Pain was the most frequent and longest lasting symptom and was associated with reduced activity level. More than half of alerts to clinicians for concerning symptoms led to intervention. The majority (87%) would recommend using electronic PRO monitoring to other lung cancer patients. CONCLUSIONS: Remote longitudinal weekly monitoring of patients with lung cancer using validated electronic PRO surveys was feasible in a multicenter, community-based pragmatic study. A high symptom burden specific to lung cancer was detected and clinician outreach in response to alerts was frequent, suggesting electronic PROs may be a beneficial strategy for identifying actionable symptoms and allow opportunities to optimize well-being in this population.

Facility-level characteristics associated with family planning and child immunization services integration in urban areas of Nigeria: a longitudinal analysis.

BACKGROUND: Unmet need for postpartum contraception is high. Integration of family planning with routine child immunization services may help to satisfy unmet need. However, evidence about the determinants and effects of integration has been inconsistent, and more evidence is required to ascertain whether and how to invest in integration. In this study, facility-level family planning and immunization integration index scores are used to: (1) determine whether integration changes over time and (2) identify whether facility-level characteristics, including exposure to the Nigerian Urban Reproductive Health Initiative (NURHI), are associated with integration across facilities in six urban areas of Nigeria. METHODS: This study utilizes health facility data collected at baseline (n = 400) and endline (n = 385) for the NURHI impact evaluation. Difference-in-differences models estimate the associations between facility-level characteristics, including exposure to NURHI, and Provider and Facility Integration Index scores. The two outcome measures, Provider and Facility Integration Index scores, reflect attributes that support integrated service delivery. These indexes, which range from 0 (low) to 10 (high), were constructed using principal component analysis. Scores were calculated for each facility. Independent variables are (1) time period, (2) whether the facility received the NURHI intervention, and (3) additional facility-level characteristics. RESULTS: Within intervention facilities, mean Provider Integration Index scores were 6.46 at baseline and 6.79 at endline; mean Facility Integration Index scores were 7.16 (baseline) and 7.36 (endline). Within non-intervention facilities, mean Provider Integration Index scores were 5.01 at baseline and 6.25 at endline; mean Facility Integration Index scores were 5.83 (baseline) and 6.12 (endline). Provider Integration Index scores increased significantly (p = 0.00) among non-intervention facilities. Facility Integration Index scores did not increase significantly in either group. Results identify facility-level characteristics associated with higher levels of integration, including smaller family planning client load, family planning training among providers, and public facility ownership. Exposure to NURHI was not associated with integration index scores. CONCLUSION: Programs aiming to increase integration of family planning and immunization services should monitor and provide targeted support for the implementation of a well-defined integration strategy that considers the influence of facility characteristics and concurrent initiatives.

SPIRIT-PRO Extension explanation and elaboration: guidelines for inclusion of patient-reported outcomes in protocols of clinical trials.

Patient-reported outcomes (PROs) are used in clinical trials to provide valuable evidence on the impact of disease and treatment on patients' symptoms, function and quality of life. High-quality PRO data from trials can inform shared decision-making, regulatory and economic analyses and health policy. Recent evidence suggests the PRO content of past trial protocols was often incomplete or unclear, leading to research waste. To address this issue, international, consensus-based, PRO-specific guidelines were developed: the Standard Protocol Items: Recommendations for Interventional Trials (SPIRIT)-PRO Extension. The SPIRIT-PRO Extension is a 16-item checklist which aims to improve the content and quality of aspects of clinical trial protocols relating to PRO data collection to minimise research waste, and ultimately better inform patient-centred care. This SPIRIT-PRO explanation and elaboration (E&E) paper provides information to promote understanding and facilitate uptake of the recommended checklist items, including a comprehensive protocol template. For each SPIRIT-PRO item, we provide a detailed description, one or more examples from existing trial protocols and supporting empirical evidence of the item's importance. We recommend this paper and protocol template be used alongside the SPIRIT 2013 and SPIRIT-PRO Extension paper to optimise the transparent development and review of trial protocols with PROs.

Mapping the Memorial Anxiety Scale for Prostate Cancer to the SF-6D.

PURPOSE: To create a crosswalk that predicts Short Form 6D (SF-6D) utilities from Memorial Anxiety Scale for Prostate Cancer (MAX-PC) scores. METHODS: The data come from prostate cancer patients enrolled in the North Carolina Prostate Cancer Comparative Effectiveness & Survivorship Study (NC ProCESS, N = 1016). Cross-sectional data from 12- to 24-month follow-up were used as estimation and validation datasets, respectively. Participants' SF-12 scores were used to generate SF-6D utilities in both datasets. Beta regression mixture models were used to evaluate SF-6D utilities as a function of MAX-PC scores, race, education, marital status, income, employment status, having health insurance, year of cancer diagnosis and clinically significant prostate cancer-related anxiety (PCRA) status in the estimation dataset. Models' predictive accuracies (using mean absolute error [MAE], root mean squared error [RMSE], Akaike information criterion [AIC] and Bayesian information criterion [BIC]) were examined in both datasets. The model with the highest prediction accuracy and the lowest prediction errors was selected as the crosswalk. RESULTS: The crosswalk had modest prediction accuracy (MAE = 0.092, RMSE = 0.114, AIC = - 2708 and BIC = - 2595.6), which are comparable to prediction accuracies of other SF-6D crosswalks in the literature. About 24% and 52% of predictions fell within ± 5% and ± 10% of observed SF-6D, respectively. The observed mean disutility associated with acquiring clinically significant PCRA is 0.168 (standard deviation = 0.179). CONCLUSION: This study provides a crosswalk that converts MAX-PC scores to SF-6D utilities for economic evaluation of clinically significant PCRA treatment options for prostate cancer survivors.

The development and initial validation of the Breast Cancer Recurrence instrument (BreastCaRe)-a patient-reported outcome measure for detecting symptoms of recurrence after breast cancer.

PURPOSE: Patient-reported outomes (PRO) may facilitate prompt treatment. We describe the development and psychometric properties of the first instrument to monitor for symptoms of breast cancer (BC) recurrence. METHODS: This study is nested in the MyHealth randomized trial of nurse-led follow-up based on electronically-collected PROs. We constructed items assessing symptoms of potential recurrence through expert interviews with six BC specialists in Denmark. Semi-structured cognitive interviews were carried out with a patient panel to assess acceptability and comprehensibility. Items were subsequently tested in a population of 1170 women 1-10 years after completing BC treatment. We carried out multiple-groups confirmatory factor analysis (CFA) and Rasch analysis to test dimensionality, local dependence (LD) and differential item functioning (DIF) according to sociodemographic and treatment-related factors. Clinical data was obtained from the Danish Breast Cancer Group registry. RESULTS: Twenty-two items were generated for the Breast Cancer Recurrence instrument (BreastCaRe). Cognitive testing resulted in clearer items. Seven subscales based on general, bone, liver, lung, brain, locoregional and contralateral recurrence symptoms were proposed. Both CFA and Rasch models confirmed the factor structure. No DIF was identified. Five item pairs showed LD but all items were retained to avoid loss of clinical information. Rasch models taking LD into account were used to generate a standardized scoring table for each subscale. CONCLUSIONS: The BreastCaRe has good content and structural validity, patient acceptability and measurement invariance. We are preparing to examine the predictive validity of this new instrument.

Comparison of weekly and daily recall of pain as an endpoint in a randomized phase 3 trial of cabozantinib for metastatic castration-resistant prostate cancer.

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.