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PURPOSE To compare taxane maintenance chemotherapy, paclitaxel (P) and paclitaxel poliglumex (PP), with surveillance (S) in women with ovarian, peritoneal, or fallopian tube (O/PC/FT) cancer who attained clinical complete response after first-line platinum-taxane therapy. METHODS Women diagnosed with O/PC/FT cancer who attained clinical complete response after first-line platinum-taxane–based chemotherapy were randomly allocated 1:1:1 to S or maintenance, P 135 mg/m2 once every 28 days for 12 cycles, or PP at the same dose and schedule. Overall survival (OS) was the primary efficacy end point. RESULTS Between March 2005 and January 2014, 1,157 individuals were enrolled. Grade 2 or worse GI adverse events were more frequent among those treated with taxane (PP: 20%, P: 27% v S: 11%). Grade 2 or worse neurologic adverse events occurred more often with taxane treatment (PP: 46%, P: 36% v S: 14%). At the fourth scheduled interim analysis, both taxane regimens passed the OS futility boundary and the Data Monitoring Committee approved an early release of results. With a median follow-up of 8.1 years, 653 deaths were reported; none were attributed to the study treatment. Median survival durations were 58.3, 56.8, and 60.0 months for S, P, and PP, respectively. Relative to S, the hazard of death for P was 1.091 (95% CI, 0.911 to 1.31; P = .343) and for PP, it was 1.033 (95% CI, 0.862 to 1.24; P = .725). The median times to first progression or death (PFS) were 13.4, 18.9, and 16.3 months for S, P, and PP, respectively. Hazard ratio = 0.801; 95% CI, 0.684 to 0.938; P = .006 for P and hazard ratio = 0.854; 95% CI, 0.729 to 1.00; P = .055 for PP. CONCLUSION Maintenance therapy with P and PP did not improve OS among patients with newly diagnosed O/tubal/peritoneal cancer, but may modestly increase PFS. GI and neurologic toxicities were more frequent in the taxane treatment arms.

Fear of recurrence (FoR) is prevalent among breast cancer survivors (BCS) and may be exacerbated by avoidance coping. This study examined BCS with avoidance coping and their engagement in a FoR eHealth intervention (FoRtitude).BCS (N = 196) with elevated FoR participated in FoRtitude. Patient-reported measures assessed avoidance coping with FoR and baseline emotional and behavioral health. Intervention engagement was measured quantitatively (e.g., website logins, telecoaching attendance) and qualitatively (i.e., telecoaching notes).38 BCS (19%) endorsed avoidance coping, which was associated with more severe post-traumatic anxiety-related symptoms and worse global mental health (ps .05), but not anxiety (p = .19), depression (p = .11), physical health (p = .12), alcohol consumption (p = .85), or physical activity (p = .39). Avoidance coping was not associated with engagement levels (ps .05) but did characterize engagement-related motivators and barriers.Avoidance coping was not a barrier to FoRtitude engagement. eHealth delivery is a promising modality for engaging survivors with avoidance coping in FoR interventions.

PURPOSE Platinum-based chemotherapy is the standard of care for platinum-sensitive ovarian cancer, but complications from repeated platinum therapy occur. We assessed the activity of two all-oral nonplatinum alternatives, olaparib or olaparib/cediranib, versus platinum-based chemotherapy. PATIENTS AND METHODS NRG-GY004 is an open-label, randomized, phase III trial conducted in the United States and Canada. Eligible patients had high-grade serous or endometrioid platinum-sensitive ovarian cancer. Patients were randomly assigned 1:1:1 to platinum-based chemotherapy, olaparib, or olaparib/cediranib. The primary end point was progression-free survival (PFS) in the intention-to-treat population. Secondary end points included activity within germline BRCA-mutated or wild-type subgroups and patient-reported outcomes (PROs). RESULTS Between February 04, 2016, and November 13, 2017, 565 eligible patients were randomly assigned. Median PFS was 10.3 (95% CI, 8.7 to 11.2), 8.2 (95% CI, 6.6 to 8.7), and 10.4 (95% CI, 8.5 to 12.5) months with chemotherapy, olaparib, and olaparib/cediranib, respectively. Olaparib/cediranib did not improve PFS versus chemotherapy (hazard ratio [HR] 0.86; 95% CI, 0.66 to 1.10; P = .077). In women with germline BRCA mutation, the PFS HR versus chemotherapy was 0.55 (95% CI, 0.32 to 0.94) for olaparib/cediranib and 0.63 (95% CI, 0.37 to 1.07) for olaparib. In women without a germline BRCA mutation, the PFS HR versus chemotherapy was 0.97 (95% CI, 0.73 to 1.30) for olaparib/cediranib and 1.41 (95% CI, 1.07 to 1.86) for olaparib. Hematologic adverse events occurred more commonly with chemotherapy; however, nonhematologic adverse events were higher with olaparib/cediranib. In 489 patients evaluable for PROs, patients receiving olaparib/cediranib scored on average 1.1 points worse on the NFOSI-DRS-P subscale (97.5% CI, –2.0 to –0.2, P = .0063) versus chemotherapy; no difference between olaparib and chemotherapy was observed. CONCLUSION Combination olaparib/cediranib did not improve PFS compared with chemotherapy and resulted in reduced PROs. Notably, in patients with a germline BRCA mutation, both olaparib and olaparib/cediranib had significant clinical activity.

OBJECTIVES: This study aimed to determine whether responses to Patient-Reported Outcomes Measurement Information System Short Form v2.0 - Physical Function 8c (PROMIS PF8c) items differed when the use of a 7-day recall period was compared with no specified recall period.METHODS: Using a within-subject design, we surveyed 1810 individuals from the US general population, administering PROMIS PF8c at survey beginning and end. The order of measure presentation was randomly assigned. We calculated recall difference scores (RDSs) as no recall score minus 7-day recall score using both item response theory-based T scores and raw summed scores. We examined the distribution and created Bland-Altman plots for both RDS Tscore and RDS Raw. We also calculated correlations between no recall versus 7-day recall T score and raw scores. Finally, we determined whether differences in no recall versus 7-day recall scores were associated with patient-reported PF. RESULTS: RDS Tscore and RDS Raw had means (root mean square differences) of 0.00 (5.43) and -0.04 (3.79), respectively. The vast majority (%) of RDS Tscore and RDS Raw values fell between the Bland-Altman limits of agreement (-10.65 to 10.66 and -7.46 to 7.38, respectively). Pearson's correlations between no recall and 7-day recall for T scores and raw scores were 0.88 and 0.87, respectively. Effect sizes for mean RDS Tscore and RDS Raw compared across level of Eastern Oncology Cooperative Group performance status, patient global impression of PF severity, and single PF items were near 0. CONCLUSIONS: We did not find any significant recall period effect on PF8c responses. Therefore, we recommend the use of the PROMIS physical function standard, with no specified recall time period.

PURPOSE Distant metastases are present in 6% or more of patients with newly diagnosed breast cancer. In this context, locoregional therapy for the intact primary tumor has been hypothesized to improve overall survival (OS), but clinical trials have reported conflicting results. METHODS Women presenting with metastatic breast cancer and an intact primary tumor received systemic therapy for 4-8 months; if no disease progression occurred, they were randomly assigned to locoregional therapy for the primary site (surgery and radiotherapy per standards for nonmetastatic disease) or continuing sysmetic therapy. The primary end point was OS; locoregional control and quality of life were secondary end points. The trial design provided 85% power to detect a 19.3% absolute difference in the 3-year OS rate in randomly assigned patients. The stratified log-rank test and Cox proportional hazards model were used to compare OS between arms. Cumulative incidence of locoregional progression was compared using Gray's test. Quality-of-life assessment used standard instruments. RESULTS Of 390 participants enrolled, 256 were randomly assigned: 131 to continued systemic therapy and 125 to early locoregional therapy. The 3-year OS was 67.9% without and 68.4% with early locoregional therapy (hazard ratio = 1.11; 90% CI, 0.82 to 1.52; P = .57). The median OS was 53.1 months (95% CI, 47.9 to not estimable) in the systemic therapy arm and 54.9 months (95% CI, 46.7 to not estimable) in the locoregional therapy arm. Locoregional progression was less frequent in those randomly assigned to locoregional therapy (3-year rate: 16.3% v 39.8%; P < .001). Quality-of-life measures were largely similar between arms. CONCLUSION Early locoregional therapy for the primary site did not improve survival in patients presenting with metastatic breast cancer. Although it was associated with improved locoregional control, this had no overall impact on quality of life.

OBJECTIVES The purpose of this study was to examine the prevalence and factors associated with objective and subjective cognitive dysfunction in older breast cancer survivors (BCS). MATERIALS AND METHODS This cross-sectional descriptive study leveraged previously collected data from older BCS (n = 335). Separate linear regression models were used to determine relationships between demographic factors (age, education), medical factors (comorbidities), disease factors (time since diagnosis, cancer stage), cancer-related symptoms (depressive symptoms, anxiety, fatigue, sleep disturbance) and cognitive dysfunction measures, including objective learning, delayed recall, attention, executive function-working memory, verbal fluency and subjective attentional function. RESULTS Cognitive dysfunction was prevalent with up to 18.6% of older BCS experiencing mild-moderate dysfunction (1.5 standard deviations below mean of non-cancer controls) in at least one cognitive domain. Poor to moderate subjective attentional function was reported by 26% of older BCS. More depressive symptoms were significantly related to poorer cognitive function including learning (p < .01), delayed recall (p < .05), verbal fluency (p < .001), and subjective attentional function (p < .001) but not attention and executive function-working memory. Age, education, anxiety, and fatigue were also negatively associated with cognitive function in some models (p < .05-0.001). CONCLUSION Cognitive dysfunction is common among older BCS and depressive symptoms, anxiety, and fatigue are related factors. Importantly, depressive symptoms were not only related to self-report, but also to cognitive performance. Healthcare providers should be aware of and assess for related factors and cognitive dysfunction itself in older BCS even years after diagnosis and treatment thorough geriatric assessment. Future longitudinal research is needed to discern these relationships.

BACKGROUND The benefits of moderate to vigorous physical activity (MVPA) for breast cancer survivors are well established. However, most are insufficiently active. Fit2Thrive used the Multiphase Optimization Strategy methodology to determine the effect of 5 intervention components on MVPA in this population. METHODS Two hundred sixty-nine participants (mean age, 52.5 years; SD, 9.9 years) received a core intervention (the Fit2Thrive self-monitoring app and Fitbit) and were randomly assigned to 5 intervention components set to on/off in a full factorial experiment: support calls, deluxe app, buddy, online gym, and text messages. The intervention was delivered over 12 weeks with a 12-week follow-up. MVPA was measured via accelerometry at the baseline (T1), at 12 weeks (T2), and at 24 weeks (T3). The main effects and interaction effects at each time point were examined for all components. RESULTS Trial retention was high: 91.8% had valid accelerometer data at T2 or T3. Across all conditions, there were significant increases in MVPA (+53.6 min/wk; P < .001) and in the proportion of survivors meeting MVPA guidelines (+22.3%; P < .001) at T2 that were maintained but attenuated at T3 (MVPA, +24.6 min/wk; P < .001; meeting guidelines, +12.6%; P < .001). No individual components significantly improved MVPA, although increases were greater for the on level versus the off level for support calls, buddy, and text messages at T2 and T3. CONCLUSIONS The Fit2Thrive core intervention (the self-monitoring app and Fitbit) is promising for increasing MVPA in breast cancer survivors, but the components provided no additional increases in MVPA. Future research should evaluate the core intervention in a randomized trial and determine what components optimize MVPA behaviors in breast cancer survivors.

Objective: To evaluate psychometric performance of the NCCN-FACT Ovarian Cancer Symptom Index-18 (NFOSI-18) in advanced ovarian cancer. Methods: Cross-sectional, observational data from patients receiving treatment for ovarian cancer. Other measures included European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire core (EORTC QLQ-C30) and associated ovarian cancer module (EORTC QLQ-OV28) and Work Productivity and Activity Impairment. Internal consistency reliability, construct validity and anchor-based clinically important differences were assessed. Results: 897 patients were analyzed. Reliability was acceptable for all NFOSI-18 scores; construct validity was supported. Twelve anchors sufficiently correlated with NFOSI-18 scores and suggested clinically important differences: NFOSI-18 total score (5–7), disease-related symptoms – physical (3–4), disease-related symptoms – emotional (1), treatment side effects (2) and functional well-being (1–2). Conclusions: Results provide evidence of reliability and validity of NFOSI-18 scores. Generated CIDs will help improve interpretation of between-group treatment differences in clinical trials.

Objective Previous work in Huntington’s disease (HD) has shown that a sense of meaning and purpose (M&P) is positively associated with positive affect and well‐being (PAW); however, it was unknown whether HD‐validated patient‐reported outcomes (PROs) influence this association and how M&P impacts PROs in the future. Our study was designed to examine if HD‐validated PROs moderate the relationship between M&P and PAW and to evaluate if baseline M&P predicts 12‐ and 24‐month changes in HD‐validated PROs. Methods This was a longitudinal, multicenter study to develop several PROs (e.g., specific for the physical, emotional, cognitive, and social domains) for people with HD (HDQLIFE). The sample consisted of 322 people with HD (n = 50 prodromal, n = 171 early‐stage manifest, and n = 101 late‐stage manifest HD). A single, multivariate linear mixed‐effects model was performed with PAW as the outcome predicted by main effects for M&P and several moderators (i.e., an HD‐validated PRO) and interactions between M&P and a given PRO. Linear‐mixed models were also used to assess if baseline M&P predicted HD‐validated PROs at 12 and 24 months. Results Higher M&P was positively associated with higher PAW regardless of the magnitude of symptom burden, as represented by HD‐validated PROs, and independent of disease stage. In our primary analysis, baseline M&P predicted increased PAW and decreased depression, anxiety, anger, emotional/behavioral disruptions, and cognitive decline at 12 and 24 months across all disease stages. Interpretation These findings parallel those seen in the oncology population and have implications for adapting and developing psychotherapeutic and palliative HD interventions.

BACKGROUND: Financial toxicity is commonly reported by cancer patients, but few studies have assessed caregiver perceptions. We aimed to validate the modified Comprehensive Score for Financial Toxicity (COST) in cancer caregivers, identify factors associated with financial toxicity in both patients and caregivers, and assess the association of caregiver financial toxicity with patient and caregiver outcomes. METHODS: Using a convenience sampling method, 100 dyads of adult cancer patients and a primary caregiver visiting outpatient oncology clinics (Jan–Sep 2019) were recruited. We assessed the internal consistency and convergent and divergent validity of the modified COST. Multivariable analyses identified correlates of financial toxicity. Association of financial toxicity with care non-adherence, lifestyle-altering behaviors (e.g., home refinance/sale, retirement/saving account withdrawal), and quality of life (QOL) was investigated. RESULTS: Recruited patient vs. caregiver characteristics were as follows: mean age: 60.6 vs. 56.5; 34% vs. 46.4% female; 79% vs. 81.4% white. The caregiver COST measure demonstrated high internal consistency (Cronbach α = 0.91). In patients, older age (B, 0.3 [95% CI, 0.1–0.4]) and higher annual household income (B, 14.3 [95% CI, 9.3–19.4]) correlated with lower financial toxicity (P < 0.05). In caregivers, lower patient financial toxicity (B, 0.4 [95% CI, 0.2–0.6]) and cancer stages 1–3 (compared to stage 4) (B, 4.6 [95% CI, 0.4–8.8]) correlated with lower financial toxicity (P < 0.05). Increased caregiver financial toxicity correlated with higher care non-adherence in patients, increased lifestyle-altering behaviors, and lower QOL in patients and caregivers (P < 0.05). CONCLUSION: The COST measure can also be used to assess caregiver financial toxicity. Caregivers’ financial toxicity was associated with negative outcomes for both dyad members.

Purpose Younger breast cancer survivors (BCS) often report cognitive impairment and poor quality of life (QoL), which could be interrelated. The purpose of this study was to examine the association of cognitive impairment and breast cancer status (BCS versus healthy control (HC)), with QoL, which included psychological (depressive symptoms, well-being, perceived stress, and personal growth) and physical well-being (physical functioning and fatigue). Methods Four hundred ninety-eight BCS (≤45 years at diagnosis) who were 3 to 8 years post-chemotherapy treatment and 394 HC completed subjective questionnaires and a one-time neuropsychological assessment, including tests of attention, memory, processing speed, and verbal fluency. For each test, cognitive impairment was defined as scoring 1.5 and 2.0 standard deviations below the mean of the HC group. Separate linear regression models for each outcome were ran controlling for known covariates. Results BCS reported significantly more memory problems than HC (p < 0.0001), with up to 23% having significant impairment. Cognitive performance did not differ significantly between BCS and HCs. BCS vs. HCs had greater depression and fatigue, yet more personal growth. Objective and subjective cognitive impairment were significantly related to greater depressive symptoms and perceived stress and lower well-being and physical functioning; whereas, objective impairment was related to less personal growth and subjective impairment was related to greater fatigue. Conclusions Younger BCS report significant cognitive impairment years after treatment which may relate to greater decrements in QoL. Implications to Cancer Survivors Assessment and interventions to address cognitive concerns may also influence QoL outcomes in younger BCS.

Objectives To develop a cancer-specific multi-attribute utility instrument derived from the Functional Assessment of Cancer Therapy - General (FACT-G) health-related quality of life (HRQL) questionnaire. Methods We derived a descriptive system based on a subset of the 27-item FACT-G. Item selection was informed by psychometric analyses of existing FACT-G data (n = 6912) and by patient input (n = 82). We then conducted an online valuation survey, with participants recruited via an Australian general population online panel. A discrete choice experiment (DCE) was used, with attributes being the HRQL dimensions of the descriptive system and survival duration, and 16 choice-pairs per participant. Utility decrements were estimated with conditional logit and mixed logit modeling. Results Eight HRQL dimensions were included in the descriptive system: pain, fatigue, nausea, sleep, work, social support, sadness, and future health worry; each with 5 levels. Of 1737 panel members who accessed the valuation survey, 1644 (95%) completed 1 or more DCE choice-pairs and were included in analyses. Utility decrements were generally monotonic; within each dimension, poorer HRQL levels generally had larger utility decrements. The largest utility decrements were for the highest levels of pain (-0.40) and nausea (-0.28). The worst health state had a utility of -0.54, considerably worse than dead. Conclusions A descriptive system and preference-based scoring approach were developed for the FACT-8D, a new cancer-specific multi-attribute utility instrument derived from the FACT-G. The Australian value set is the first of a series of country-specific value sets planned that can facilitate cost-utility analyses based on items from the FACT-G and related FACIT questionnaires containing FACT-G items.

BACKGROUND: Early discontinuation is a substantial barrier to the delivery of endocrine therapies (ETs) and may influence recurrence and survival. The authors investigated the association between early discontinuation of ET and social determinants of health, including insurance coverage and the neighborhood deprivation index (NDI), which was measured on the basis of patients' zip codes, in breast cancer. METHODS: In this retrospective analysis of a prospective randomized clinical trial (Trial Assigning Individualized Options for Treatment), women with hormone receptor-positive, human epidermal growth factor receptor 2-negative breast cancer who started ET within a year of study entry were included. Early discontinuation was calculated as stopping ET within 4 years of its start for reasons other than distant recurrence or death via Kaplan-Meier estimates. A Cox proportional hazards joint model was used to analyze the association between early discontinuation of ET and factors such as the study-entry insurance and NDI, with adjustments made for other variables. RESULTS: Of the included 9475 women (mean age, 55.6 years; White race, 84%), 58.0% had private insurance, whereas 11.7% had Medicare, 5.8% had Medicaid, 3.8% were self-pay, and 19.1% were treated at international sites. The early discontinuation rate was 12.3%. Compared with those with private insurance, patients with Medicaid (hazard ratio [HR], 1.53; 95% confidence interval [CI], 1.23-1.92) and self-pay patients (HR, 1.65; 95% CI, 1.25-2.17) had higher early discontinuation. Participants with a first-quartile NDI (highest deprivation) had a higher probability of discontinuation than those with a fourth-quartile NDI (lowest deprivation; HR, 1.34; 95% CI, 1.11-1.62). CONCLUSIONS: Patients' insurance and zip code at study entry play roles in adherence to ET, with uninsured and underinsured patients having a high rate of treatment nonadherence. Early identification of patients at risk may improve adherence to therapy. LAY SUMMARY: In this retrospective analysis of 9475 women with breast cancer participating in a clinical trial (Trial Assigning Individualized Options for Treatment), Medicaid and self-pay patients (compared with those with private insurance) and those in the highest quartile of neighborhood deprivation scores (compared with those in the lowest quartile) had a higher probability of early discontinuation of endocrine therapy. These social determinants of health assume larger importance with the expected increase in unemployment rates and loss of insurance coverage in the aftermath of the coronavirus disease 2019 pandemic. Early identification of patients at risk and enrollment in insurance optimization programs may improve the persistence of therapy.

OBJECTIVE The Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-F) is validated for measuring fatigue in rheumatoid arthritis (RA). However, 10 of 13 FACIT-F items are identified as relevant to patients with RA. The Patient-Reported Outcomes Measurement Information System (PROMIS) uses an item response theory-calibrated T score metric. The PROMIS Fatigue item bank includes the FACIT-F items, enabling score conversion. The performance of converted PROMIS Fatigue scores has not been evaluated in RA populations or clinical trials. Our objective was to assess the performance of converted PROMIS Fatigue scores in 2 RA clinical trials of baricitinib. METHODS Crosswalk tables and pattern-scoring methods converted FACIT-F scores to PROMIS Fatigue for both the 13-item FACIT-F and the 10-item RA-optimized FACIT-F instrument, in 2 RA clinical trials evaluating baricitinib, RA-BEAM, and RA-BEACON. RA-BEAM patients had an inadequate response to methotrexate. RA-BEACON patients had an inadequate response or intolerance to ≥1 tumor necrosis factor inhibitor. Baricitinib was compared to all treatment arms via analysis of covariance on PROMIS Fatigue score conversions. RESULTS Baseline FACIT-F-derived PROMIS Fatigue scores reflected severe fatigue across treatment groups and were similar using different scoring methods. At week 24 in both studies, baricitinib was associated with clinically meaningful improvements in PROMIS Fatigue scores. PROMIS Fatigue scores were consistent for conversion methods and for the 13-item or 10-item FACIT-F. CONCLUSION All 4 conversion methods showed differentiation of active treatment compared with placebo from week 12, supporting the use of the PROMIS Fatigue and converting the 10-item FACIT-F to assess fatigue and demonstrate treatment benefit in RA clinical trials on a standardized metric.

Objectives To evaluate (a) the prevalence of genitourinary symptoms, (b) which demographic and clinical factors predict genitourinary symptoms, and (c) the association between genitourinary symptoms and sexual functioning in breast cancer survivors. Sample & setting A secondary analysis of cross-sectional, patient-reported outcomes data from 1,085 breast cancer survivors was conducted. Methods & variables Prevalence and correlations with demographics, clinical factors, and sexual functioning were identified using descriptive analysis, multivariable logistic regression analysis, chi-square tests, t tests, and Pearson correlation coefficients. Results Symptoms included vaginal/vulvar irritation, pelvic discomfort, problems with urinary control, vaginal infection, and vaginal bleeding. Younger age, more comorbidities, and taking treatment for menopausal symptoms were significantly related to reporting genitourinary symptoms. Experiencing more symptoms was associated with lower sexual functioning. Implications for nursing The prevalence, correlates, and relationship of genitourinary symptoms with sexual functioning supports the assessment and treatment of these symptoms as part of routine care for breast cancer survivors.

Objective To propose postpartum recovery domains. Design Concept elicitation study. Setting Semi-structured interviews. Population Ten writing committee members and 50 stakeholder interviews (23 postpartum women, nine general obstetricians, five maternal and fetal medicine specialists, eight nurses and five obstetric anaesthetists). Methods Alternating interviews and focus group meetings until concept saturation was achieved (no new themes discussed in three consecutive interviews). Interviews were digitally recorded and transcribed, and an iterative coding process was used to identify domains. Main outcome measures The primary outcome was to identify recovery domains. We also report key symptoms and concerns. Discussion frequency and importance scores (0-100; 0 = not important; 100 = vitally important to recovery) were used to rank domains. Discussion frequency was used to rank factors helping and hindering recovery, and to determine the greatest challenges experienced postpartum. Results Thirty-four interviews and two focus group meetings were performed. The 13 postpartum recovery domains identified, (ranked highest to lowest) were: psychosocial distress, surgical/medical factors, infant feeding and breast health, psychosocial support, pain, physical function, sleep, motherhood experience, infant health, fatigue, appearance, sexual function and cognition. The most frequently discussed factors facilitating postpartum recovery were: family support, lactation/breastfeeding support and partner support. The most frequently discussed factor hindering recovery was inadequate social support. The most frequent challenges reported were: breastfeeding (week 1), breastfeeding (week 3) and sleep (week 6). Conclusions We propose 13 domains that comprehensively describe recovery in women delivering in a single centre within the USA. This provides a novel framework to study the postpartum recovery process. Tweetable abstract We propose 13 postpartum recovery domains that provide a framework to study the recovery process following childbirth.

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

To the authors' knowledge, few studies to date have examined associations between moderate to vigorous physical activity (MVPA) and sitting time with quality of life in cancer survivors compared with a cancer-free group. The current study examined differences in global mental health (GMH) and global physical health (GPH) across levels of MVPA and sitting among cancer survivors and cancer-free participants.Cancer Prevention Study II participants (59.9% of whom were female with an age of 77.8 ± 5.8 years) were grouped as: 1) survivors who were 1 to 5 years after diagnosis (3718 participants); 2) survivors who were 6 to 10 years after diagnosis (4248 participants); and 3) cancer-free participants (ie, no history of cancer; 69,860 participants). In 2009, participants completed MVPA, sitting, and Patient-Reported Outcomes Measurement Information System GMH/GPH surveys. Mean differences in GMH and GPH T scores across MVPA (none, 0 to7.5, 7.5 to15, 15 to22.5, and ≥22.5 metabolic equivalent [MET]-hours/week) and sitting (0 to3, 3 to6, and ≥6 hours/day) were assessed using multivariate generalized linear models.The mean GMH and GPH scores were statistically significantly higher in cancer-free participants compared with cancer survivor groups, although the differences were not clinically meaningful (mean difference of 0.52 for GMH and 0.88 for GPH). More MVPA was associated with higher GMH and GPH scores for all 3 groups (P for trend.001), and differences between the least and most active participants were found to be clinically meaningful (mean differences of ≥4.34 for GMH and ≥6.39 for GPH). Similarly, a lower duration of sitting was associated with higher GMH and GPH scores for all groups (P for trend.001), with clinically meaningful differences observed between the least and most sedentary participants (mean differences of ≥2.74 for GMH and ≥3.75 for GPH).The findings of the current study provide evidence of the importance of increased MVPA and decreased sitting for improved health in older adults with or without a prior cancer diagnosis.

Background Patients with advanced head and neck cancer have identified pain, fatigue, and difficulties swallowing, breathing, and communicating as high‐priority disease‐related symptoms. The Functional Assessment of Cancer Therapy‐Head and Neck Symptom Index‐10 (FHNSI‐10) assesses these symptoms. We sought to validate the FHNSI‐10, another brief symptom index (FHNSI‐7), and individual symptom endpoints representing these high‐rated priority disease symptoms among patients with recurrent or metastatic squamous cell carcinoma of the head and neck (SCCHN). Methods Patients (N = 239) were enrolled in a phase III randomized clinical trial (E1302) and completed the FHNSI‐10 at multiple time points. We assessed the internal consistencies and test–retest reliabilities of the FHNSI‐10 and FHNSI‐7 scores, and the known‐groups validity, predictive criterion validity, and responsiveness‐to‐change of the symptom indexes and individual symptom endpoint scores. Results The FHNSI‐10 and FHNSI‐7 indexes showed satisfactory internal consistencies (Cronbach's alpha coefficient range 0.60‐0.75) and acceptable test–retest reliabilities (intraclass correlation coefficients = 0.75 and 0.74, respectively). The FHNSI‐10, FHNSI‐7, and the pain, fatigue, swallowing, and breathing symptom scores showed evidence of known‐groups validity by performance status at baseline. The FHNSI‐10, FHNSI‐7, and the pain, fatigue, and breathing symptom scores at baseline showed evidence of predictive criterion validity for overall survival, but not time‐to‐progression (TTP). Changes in the symptom indexes and individual symptom scores were not associated with changes in performance status over 4 weeks, though most patients had stable performance status. Conclusions There is initial evidence of validity for the FHNSI‐10 and FHNSI‐7 indexes and selected individual symptom endpoints as brief disease‐related symptom assessments for patients with recurrent or metastatic SCCHN., The patient‐centered FHNSI‐10 and FHNSI‐7 symptom indexes show evidence of validity and reliability for use among patients with recurrent or metastatic squamous cell carcinoma of the head and neck. Individual symptom endpoints (1‐2 items each) are also valid for assessing pain, fatigue, swallowing, and breathing in this population.

Objective: To develop a symptom-focused index to evaluate representative symptoms, treatment side effects, and emotional and functional well-being of patients with carcinoid syndrome (CS). Methods: The development of the Functional Assessment of Cancer Therapy-Carcinoid Syndrome Symptom Index (FACT-CSI) followed US Food and Drug Administration guidelines for the development of patient-reported outcome (PRO) measures and involved the following: (a) literature review; (b) interviews with 14 CS patients; (c) interviews with 9 clinicians; and (d) instrument development involving input from a range of PRO measure development and CS experts. The resulting draft instrument underwent cognitive interviews with 7 CS patients. Results: Forty-six CS sources were reviewed. Analysis of patient interviews produced 23 patient-reported symptoms. The most frequently endorsed physical symptoms were flushing, diarrhea, abdominal pain, fatigue, and food sensitivity/triggers. Seven priority CS emotional and functional themes were also identified by patients. Expert interviews revealed 12 unique priority symptoms – the most common being diarrhea, flushing, wheezing, edema, abdominal pain/cramping, fatigue, and 8 emotional and functional concerns. Through an iterative process of team and clinical collaborator meetings, data review, item reduction and measure revision, 24 items were selected for the draft symptom index representing symptoms, emotional concerns, global assessment of treatment side effects, and functional well-being. Cognitive interview results demonstrated strong content validity, including positive endorsement of item clarity (>86% across items), symptom relevance (>70% for most items), and overall measure content (86%). Conclusions: The FACT-CSI is a content-relevant, symptom-focused index reflecting the highest priority and clinically relevant symptoms and concerns of people with CS.

Aims Measurement of self-reported lower urinary tract symptoms (LUTS) typically uses a recall period, for example, "In the past 30 days…." Compared to averaged daily reports, 30-day recall is generally unbiased, but recall bias varies by item. We examined the associations between personal characteristics (eg, age, symptom bother) and 30-day recall of LUTS using items from the Symptoms of Lower Urinary Tract Dysfunction Research Network Comprehensive Assessment of Self-reported Urinary Symptoms questionnaire. Methods Participants (127 women and 127 men) were recruited from 6 US tertiary care sites. They completed daily assessments for 30 days and a 30-day recall assessment at the end of the study month. For each of the 18 tested items, representing 10 LUTS, the average of the participant's daily responses was modeled as a function of their 30-day recall, the personal characteristic, and the interaction between the 30-day recall and the characteristic in separate general linear regression models, adjusted for sex. Results Nine items representing 7 LUTS exhibited under- or overreporting (recall bias) for at least 25% of participants. Bias was associated with personal characteristics for six LUTS. Underreporting of incontinence was associated with older age, lower anxiety, and negative affect; overreporting of other LUTS was associated with, symptom bother, symptom variability, anxiety, and depression. Conclusions We identified under- or overreporting that was associated with personal characteristics for six common LUTS. Some cues (eg, less bother and lower anxiety) were related to recall bias in an unexpected direction. Thus, providers should exercise caution when making judgments about the accuracy of a patient's symptom recall based on patient demographic and psychosocial characteristics.

Background: CheckMate 025 has shown superior efficacy for nivolumab over everolimus in patients with advanced renal cell carcinoma (aRCC) along with improved safety and tolerability. This analysis assesses the long-term clinical benefits of nivolumab versus everolimus. Methods: The randomized, open-label, phase 3 CheckMate 025 trial (NCT01668784) included patients with clear cell aRCC previously treated with 1 or 2 antiangiogenic regimens. Patients were randomized to nivolumab (3 mg/kg every 2 weeks) or everolimus (10 mg once a day) until progression or unacceptable toxicity. The primary endpoint was overall survival (OS). The secondary endpoints were the confirmed objective response rate (ORR), progression-free survival (PFS), safety, and health-related quality of life (HRQOL). Results: Eight hundred twenty-one patients were randomized to nivolumab (n = 410) or everolimus (n = 411); 803 patients were treated (406 with nivolumab and 397 with everolimus). With a minimum follow-up of 64 months (median, 72 months), nivolumab maintained an OS benefit in comparison with everolimus (median, 25.8 months [95% CI, 22.2-29.8 months] vs 19.7 months [95% CI, 17.6-22.1 months]; hazard ratio [HR], 0.73; 95% CI, 0.62-0.85) with 5-year OS probabilities of 26% and 18%, respectively. ORR was higher with nivolumab (94 of 410 [23%] vs 17 of 411 [4%]; P

The Patient-Reported Outcomes Measurement Information System (PROMIS) Itch Questionnaire (PIQ) was recently developed.To validate PIQ short forms in adults with AD.Self-administered questionnaires and skin examinations were performed in 239 adults with atopic dermatitis (AD) in a dermatology practice setting.PIQ items had good content validity. PIQ item bank T-scores strongly correlated with each other, moderately correlated with numeric and verbal rating scales for worst or average itch and with itch frequency, moderately to strongly correlated with patient-oriented eczema measure, and weakly to moderately correlated with the Eczema Area and Severity Index and Objective-Scoring AD (Spearman correlations, P .0001). There were significant and stepwise increases of T-scores for all item banks with increasing patient-reported global severity (Wilcoxon rank sum test, P .0001). However, there was limited ability to discriminate between the lowest or highest 2 levels of AD or itch severity. Item banks showed good internal consistency (Cronbach α, 0.91-0.95). No differential item functioning was identified by age, sex, race/ethnicity, or educational level. There were floor effects for total scores, particularly in almost clear/mild AD or itch.Single-center study.PIQ item bank short forms showed good content and construct validity and are feasible for potential use in clinical trials and practice.

Introduction Somatostatin analogues are used to treat symptoms and slow tumour progression in patients with neuroendocrine tumours (NETs) and carcinoid syndrome and to reduce hormone secretion and pituitary tumour volume in patients with acromegaly. A new syringe for lanreotide autogel/depot (LAN) was developed following feedback from a human factors study to improve ease of injection compared with previous syringes. PRESTO aimed to assess preferences of nurses between the LAN new syringe and the octreotide long-acting release (LAR) syringe. Methods PRESTO, a multinational, multicentre, prospective, noninterventional, simulated-use study, enrolled nurses with ≥ 2 years’ experience injecting LAN and/or octreotide LAR in patients with NETs and/or acromegaly. Nurses administered injections into pads using the LAN new syringe and octreotide LAR syringe in a randomised sequence. In an anonymous web-based questionnaire, nurses reported their overall preference (‘strong’ or ‘slight’; primary endpoint) and rated and ranked the importance of nine attributes for each syringe (1 [not at all] to 5 [very much]). Results Overall, 90 nurses attended sessions and completed valid questionnaires. Most nurses (97.8%) expressed a preference (85.6% ‘strong’, 12.2% ‘slight’) for the LAN new syringe versus the octreotide LAR syringe (P

BACKGROUND: Social health is an important concern in persons with Huntington’s disease (HD); however, there is little literature examining this construct in this population. OBJECTIVE: While cross-sectional data supports the clinical utility of two Neuro-QoL social health measures in persons with HD, data is still needed to establish their longitudinal validity. METHODS: Participants (N = 358) completed baseline and at least one follow-up (12- and 24-month) assessment that included the completion of Neuro-QoL Social Health computer adaptive tests (CATs) and short forms (for Ability to Participate in Social Roles and Activities [SRA] and Satisfaction with SRA). Test-retest reliability was examined using intra class correlations, and one-way ANOVAs with Bonferroni post-hoc contrasts were used to determine whether there were group differences among premanifest, early- and late-stage HD participants on the Social health measures. In addition, standardized response means were used to examine longitudinal responsiveness, and mixed or general linear models were used to examine change over time (relative to self-reported change on an associated anchor item about social health and clinician-rated change based on Total Functional Capacity scores from the UHDRS). RESULTS: Test-retest reliability of the measures was excellent (ICCs ranged from 0.82 to 0.87 across the different measures) and persons with greater disease burden reported more problems with social health than those at earlier stages in the disease process (all p < 0.0001). Responsiveness was supported for all measures except the Ability to Participate in SRA CAT; participants who had self-reported or clinician-rated declines in health generally had 12- and 24-month declines on the Neuro-QoL measures. CONCLUSIONS: Findings indicate that these measures may be useful for studies attempting to assess change in social health over time.

OBJECTIVES The family stress model proposes economic hardship results in caregiver distress and relational problems, which negatively impact youth outcomes. We extend this model to evaluate the impact of coronavirus disease 2019 pandemic-related family hardships on caregiver and youth stress, and, in turn, youth’s psychological well-being. We also investigate how social supports moderate this relationship. METHODS We used 2 samples of cross-sectional survey data collected between May 2020 and May 2021: children aged 2 to 12 years (n = 977) and adolescents aged 11 to 17 years (n = 669). Variables included pandemic-related family hardships, stress, social support, and youth life satisfaction. Data were analyzed using structural equation modeling. RESULTS Experiencing more pandemic-related family hardships was associated with increased caregiver and youth stress (b = 0.04 to 0.21, SE = 0.01–0.02) and, in turn, decreased youth life satisfaction (b = –0.36 to –0.38, SE = 0.04–0.07). Social connectedness (b^ = 0.11–0.17, SE = 0.04) and family engagement (b^ = 0.12–0.18, SE = 0.05–0.06) had direct positive associations with life satisfaction; for children aged 2 to 12 years, greater family engagement was associated with decreased effect of child stress on life satisfaction (b^ = 0.15, SE = 0.05). For adolescents, females had higher levels of stress compared with males (b^ = 0.40, SE = 0.6), and having anxiety and/or depression was associated with decreased life satisfaction (b^ = –0.24, SE = 0.11). CONCLUSIONS Caregivers and youth who experienced more coronavirus disease 2019 pandemic hardships had higher levels of stress, particularly adolescent females. Although stress negatively impacted life satisfaction across all ages, family engagement was a protective factor for children aged 2 to 12 years, whereas having anxiety and/or depression was a risk factor for adolescents. For all youth, however, being more socially connected and engaged with family promoted life satisfaction.

IMPORTANCE: Racial disparities in survival outcomes among Black women with hormone receptor–positive breast cancer have been reported. However, the association between individual-level and neighborhood-level social determinants of health on such disparities has not been well studied. OBJECTIVE: To evaluate the association between race and clinical outcomes (ie, relapse-free interval and overall survival) adjusting for individual insurance coverage and neighborhood deprivation index (NDI), measured using zip code of residence, in women with breast cancer. DESIGN, SETTING, AND PARTICIPANTS: This was a post hoc analysis of 9719 women with breast cancer in the Trial Assigning Individualized Options for Treatment, a randomized clinical trial conducted from April 7, 2006, to October 6, 2010. All participants received a diagnosis of hormone receptor–positive, ERBB2-negative, axillary node–negative breast cancer. The present data analysis was conducted from April 1 to October 22, 2021. MAIN OUTCOMES AND MEASURES: A multivariate model was developed to evaluate the association between race and relapse-free interval and overall survival adjusting for insurance and NDI level at study entry, early discontinuation of endocrine therapy 4 years after initiation, and clinicopathologic characteristics of cancer. Median follow-up for clinical outcomes was 96 months. RESULTS: A total of 9719 women (4.2% [n = 405] Asian; 7.1% [n = 693] Black; 84.3% [n = 8189] White; 4.4% [n = 403] others/not specified) were included; 9.1% of included women [n = 889] were Hispanic or Latino. Median (SD) age was 56 (9.2) years. In multivariate models, Black race compared with White race was associated with statistically significant shorter relapse-free interval (hazard ratio [HR], 1.39; 95% CI, 1.05-1.84; P = .02) and overall survival (HR, 1.49; 95% CI, 1.10-2.99; P = .009), adjusting for insurance and NDI level at study entry and other factors. Although uninsured status was not associated with clinical outcomes, patients with Medicare (HR, 1.30; 95% CI, 1.01-1.68; P = .04) and Medicaid (HR, 1.44; 95% CI, 1.01-2.05; P = .05) had shorter overall survival compared with those with private insurance. Participants living in neighborhoods in the highest NDI quartile experienced shorter overall survival compared with those in the lowest quartile (HR, 1.34; 95% CI, 1.01-1.77; P = .04), regardless of self-identified race. CONCLUSIONS AND RELEVANCE: The findings of this post hoc analysis of a randomized clinical trial suggest that Black women with breast cancer have significantly shorter relapse-free interval and overall survival compared with White women. Early discontinuation of endocrine therapy, clinicopathologic characteristics, insurance coverage, and NDI do not fully explain the observed disparity. TRIAL REGISTRATION: ClinicalTrials.gov Identifier: NCT00310180

Health-related quality of life (HRQOL) is increasingly recognized as important when evaluating cancer treatments. The use, reporting, and analysis of patient-reported outcome measures (PROMs), however, are not standardized in clinical trials and are often poorly implemented in clinical practice. We report the results of a systematic literature review (PubMed search: January 1, 2000 to August 15, 2020) of PROM use, reporting, and analysis in phase 3 clinical trials of hormone receptor–positive (HR+) advanced breast cancer (ABC). Further inspection of cyclin-dependent kinase 4/6 (CDK4/6) inhibitor publications was performed to examine PROMs in the HR+/human epidermal growth factor receptor 2–negative (HER2-) setting. A total of 88 results were identified in the initial search; 32 were included in the final analysis. Among included studies, most (66%) had been published in the last 5 years (2015 to 2020). CDK4/6 inhibitors (38%) were the most common agents reported. No clear standard for PROM use, reporting, or analysis was found. The most common PROMs were European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ-C30) (59%) and the Functional Assessment of Cancer Therapy–Breast (FACT-B; 34%). Important differences, among studies that reported them, ranged from 5 to 10 points for the EORTC QLQ-C30 and 8 points for the FACT-B total score. This review showed that a lack of clear consistency remains for PROM use, reporting, and analysis in phase 3 clinical trials of HR+ ABC. However, HRQOL is of high interest in the literature, including for CDK4/6 inhibitors.

BACKGROUND: An increased focus on patient-reported outcome measures (PROMs) has led to a proliferation of these measures in orthopaedic surgery. Mandating a single PROM in clinical and research orthopaedics is not feasible given the breadth of data already collected with older measures and the emergence of psychometrically superior measures. Creating crosswalk tables for scores between measures allows providers to maintain control of measure choice. Furthermore, crosswalk tables permit providers to compare scores collected with older outcome measures with newly collected ones. Given the widespread use of the newer Patient-reported Outcome Measure Information System Physical Function (PROMIS PF) and the established Knee Outcome and Osteoarthritis Score (KOOS), it would be clinically useful to link these two measures.QUESTION/PURPOSE: Can the KOOS Function in Activities of Daily Living (ADL) subscale be robustly linked to the PROMIS PF to create a crosswalk table of equivalent scores that accurately reflects a patient's reported physical function level on both scales?METHODS: We sought to establish a common standardized metric for collected responses to the PROMIS PF and the KOOS ADL to develop equations for converting a PROMIS PF score to a score for the KOOS-ADL subscale and vice versa. To do this, we performed a retrospective, observational study at two academic medical centers and two community hospitals in an urban and suburban healthcare system. Patients 18 years and older who underwent TKA were identified. Between January 2017 and July 2020, we treated 8165 patients with a TKA, 93% of whom had a diagnosis of primary osteoarthritis. Of those, we considered patients who had completed a full KOOS and PROMIS PF 10a on the same date as potentially eligible. Twenty-one percent (1708 of 8165) of patients were excluded because no PROMs were collected at any point, and another 67% (5454 of 8165) were excluded because they completed only one of the required PROMs, leaving 12% (1003 of 8165) for analysis here. PROMs were collected each time they visited the health system before and after their TKAs. Physical function was measured by the PROMIS PF version 1.0 SF 10a and KOOS ADL scale. Analyses to accurately create a crosswalk of equivalent scores between the measures were performed using the equipercentile linking method with both unsmoothed and log linear smoothed score distributions.RESULTS: Crosswalks were created, and adequate validation results supported their validity; we also created tables to allow clinicians and clinician scientists to convert individual patients' scores easily. The mean difference between the observed PROMIS PF scores and the scores converted by the crosswalk from the KOOS-ADL scores was -0.08 ± 4.82. A sensitivity analysis was conducted, confirming the effectiveness of these crosswalks to link the scores of two measures from patients both before and after surgery.CONCLUSION: The PROMIS PF 10a can be robustly linked to the KOOS ADL measure. The developed crosswalk table can be used to convert PROMIS PF scores from KOOS ADL and vice versa.CLINICAL RELEVANCE: The creation of a crosswalk table between the KOOS Function in ADL subscale and PROMIS PF allows clinicians and researchers to easily convert scores between the measures, thus permitting greater choice in PROM selection while preserving comparability between patient cohorts and PROM data collected from older outcome measures. Creating a crosswalk, or concordance table, between the two scales will facilitate this comparison, especially when pooling data for meta-analyses.

Background TAILORx (Trial Assigning Individualized Options for Treatment) prospectively assessed fatigue and endocrine symptoms among women with early-stage hormone receptor-positive breast cancer and a midrange risk of recurrence who were randomized to endocrine therapy (E) or chemotherapy followed by endocrine therapy (CT+E). Methods Participants completed the Functional Assessment of Chronic Illness Therapy-Fatigue, the Patient-Reported Outcomes Measurement Information System-Fatigue Short Form, and the Functional Assessment of Cancer Therapy-Endocrine Symptoms at the baseline and at 3, 6, 12, 24, and 36 months. Linear regression was used to model outcomes on baseline symptoms, treatment, and other factors. Results Participants (n = 458) in both treatment arms reported greater fatigue and endocrine symptoms at early follow-up in comparison with the baseline. The magnitude of change in fatigue was significantly greater for the CT+E arm than the E arm at 3 and 6 months but not at 12, 24, or 36 months. The CT+E arm reported significantly greater changes in endocrine symptoms from the baseline to 3 months in comparison with the E arm; change scores were not significantly different at later time points. Endocrine symptom trajectories by treatment differed by menopausal status, with the effect larger and increasing for postmenopausal patients. Conclusions Adjuvant CT+E was associated with greater increases in fatigue and endocrine symptoms at early time points in comparison with E. These differences lessened over time, and this demonstrated early chemotherapy effects more than long-term ones. Treatment arm differences in endocrine symptoms were more evident in postmenopausal patients. Lay summary Participants in TAILORx (Trial Assigning Individualized Options for Treatment) with early-stage hormone receptor-positive breast cancer and an intermediate risk of recurrence were randomly assigned to endocrine or chemoendocrine therapy. Four hundred fifty-eight women reported fatigue and endocrine symptoms at the baseline and at 3, 6, 12, 24, and 36 months. Both groups reported greater symptoms at early follow-up versus the baseline. Increases in fatigue were greater for the chemoendocrine group than the endocrine group at 3 and 6 months but not later. The chemoendocrine group reported greater changes in endocrine symptoms in comparison with the endocrine group at 3 months but not later.

ObjectivesIn a prospective study of platinum-resistant ovarian cancer patients, we examined whether the Disease-related Symptoms-Physical (DRS--P) scale of the NCCN/FACT-Ovarian Cancer Symptom Index-18 (NFOSI-18) is responsive to clinical change in patients estimated by their provider to survive at least six months.MethodsThe NFOSI-18, and other FACT measures, was collected at study entry and 3 and 6months post-enrollment. Measures were compared for those who died or dropped off study prior to 3months or prior to 6months (assumed as health deterioration over time), or those who stayed on study through 6months (presumed as stable disease over time). Statistical analyses included a fitted linear mixed model for estimating the group differences over time, Cox regression to assess the probability of survival with patient-reported outcomes, and effect size.ResultsDRS-P scores of patients who completed only one assessment were significantly lower compared to patients who were able to complete two assessments [5.9 points lower (2.0-9.8); p&nbsp

In the CheckMate 9ER trial, patients with advanced renal cell carcinoma who received first-line nivolumab plus cabozantinib had significantly better progression-free survival compared with those given sunitinib. In this study, we aimed to describe the patient-reported outcome (PRO) results from CheckMate 9ER.In this open-label, randomised, phase 3 trial done in 125 cancer centres, urology centres, and hospitals across 18 countries, patients aged 18 years or older with previously untreated advanced renal cell carcinoma with a clear-cell component, a Karnofsky performance status of 70% or more, and available tumour tissue were randomly assigned (1:1) via interactive response technology to nivolumab 240 mg intravenously every 2 weeks plus oral cabozantinib 40 mg per day, or oral sunitinib 50 mg per day monotherapy for 4 weeks in 6-week cycles. The primary endpoint of progression-free survival was reported previously. PROs were analysed as prespecified exploratory endpoints at common timepoints (at baseline and every 6 weeks) until week 115. Disease-related symptoms were evaluated using the 19-item Functional Assessment of Cancer Therapy-Kidney Symptom Index (FKSI-19), and global health status was assessed with the three-level EQ-5D (EQ-5D-3L) visual analogue scale (VAS) and UK utility index. PRO analyses were done in the intention-to-treat population. Change from baseline was assessed using mixed-model repeated measures. A time-to-deterioration analysis was done for first and confirmed deterioration events. This study is registered with ClinicalTrials.gov, NCT03141177, and is closed to recruitment.Between Sept 11, 2017, and May 14, 2019, 323 patients were randomly assigned to nivolumab plus cabozantinib and 328 to sunitinib. Median follow-up was 23·5 months (IQR 21·0-26·5). At baseline, patients in both groups reported low symptom burden (FKSI-19 disease-related symptoms version 1 mean scores at baseline were 30·24 [SD 5·19] for the nivolumab plus cabozantinib group and 30·06 [5·03] for the sunitinib group). Change from baseline in PRO scores indicated that nivolumab plus cabozantinib was associated with more favourable outcomes versus sunitinib (treatment difference 2·38 [95% CI 1·20-3·56], nominal p0·0001, effect size 0·33 [95% CI 0·17-0·50] for FKSI-19 total score; 1·33 [0·84-1·83], nominal p0·0001, 0·45 [0·28-0·61] for FKSI-19 disease-related symptoms version 1; 3·48 [1·58-5·39], nominal p=0·0004, 0·30 [0·14-0·47] for EQ-5D-3L VAS; and 0·04 [0·01-0·07], nominal p=0·0036, 0·25 [0·08-0·41] for EQ-5D-3L UK utility index), reaching significance at most timepoints. Nivolumab plus cabozantinib was associated with decreased risk of clinically meaningful deterioration for FKSI-19 total score compared with sunitinib (first deterioration event hazard ratio 0·70 [95% CI 0·56-0·86], nominal p=0·0007; confirmed deterioration event 0·63 [0·50-0·80], nominal p=0·0001).PROs were maintained or improved with nivolumab plus cabozantinib versus sunitinib. Compared with sunitinib, nivolumab plus cabozantinib significantly delayed time to deterioration of patient-reported outcome scores. These results suggest a benefit for nivolumab plus cabozantinib compared with sunitinib in the treatment of patients with advanced renal cell carcinoma.Bristol Myers Squibb.

Increased moderate and vigorous physical activity (MVPA) is associated with better health outcomes in breast cancer survivors; yet, most are insufficiently active. Smartphone applications (apps) to promote MVPA have high scalability potential, but few evidence-based apps exist. The purpose is to describe the testing and usability of Fit2Thrive, a MVPA promotion app for breast cancer survivors. A user-centered, iterative design process was utilized on three independent groups of participants. Two groups of breast cancer survivors (group 1 n = 8; group 2: n = 14) performed app usability field testing by interacting with the app for ≥3 days in a free-living environment. App refinements occurred following each field test. The Post-Study System Usability Questionnaire (PSSUQ) and the User Version Mobile Application Rating Scale (uMARS) assessed app usability and quality on a 7- and 5-point scale, respectively, and women provided qualitative written feedback. A third group (n = 15) rated potential app notification content. Quantitative data were analyzed using descriptive statistics. Qualitative data were analyzed using a directed content analysis. The PSSUQ app usability score (M1= 3.8; SD = 1.4 vs. M2= 3.2; SD = 1.1; lower scores are better) and uMARS app quality score (M1 = 3.4; SD = 1.3 vs. M2= 3.4; SD = 0.6; higher scores are better) appeared to improve in Field Test 2. Group 1 participants identified app “clunkiness,” whereas group 2 participants identified issues with error messaging/functionality. Group 3 “liked” 53% of the self-monitoring, 71% of the entry reminder, 60% of the motivational, and 70% of the goal accomplishment notifications. Breast cancer survivors indicated that the Fit2Thrive app was acceptable and participants were able to use the app. Future work will test the efficacy of this app to increase MVPA.

Background Patients with cancer who are treated with immune checkpoint modulators (ICMs) have their health-related quality of life (HRQOL) measured using general patient-reported outcome (PRO) tools. To the authors' knowledge, no instrument has been developed to date specifically for patients treated with ICMs. The objective of the current study was to develop a toxicity subscale PRO instrument for patients treated with ICMs to assess HRQOL. Methods Input was collected from a systematic review as well as patients and physicians experienced with ICM treatment. Descriptive thematic analysis was used to evaluate the qualitative data obtained from patient focus groups and interviews, which informed an initial list of items that described ICM side effects and their impact on HRQOL. These inputs informed item generation and/or reduction to develop a toxicity subscale. Results Focus groups and individual interviews with 37 ICM-treated patients generated an initial list of 176 items. After a first round of item reduction that produced a shortened list of 76 items, 16 physicians who care for patients who are treated with ICMs were surveyed with a list of 49 patient-reported side effects and 11 physicians participated in follow-up interviews. A second round of item reduction was informed by the physician responses to produce a list of 25 items. Conclusions To the authors' knowledge, this 25-item list is the first HRQOL-focused toxicity subscale for patients treated with ICMs and was developed in accordance with US Food and Drug Administration guidelines, which prioritize patient input in developing PRO tools. The subscale will be combined with the Functional Assessment of Cancer Therapy-General (FACT-G) to form the FACT-ICM. Prior to recommending the formal use of this PRO instrument, the authors will evaluate its validity and reliability in longitudinal studies involving substantially more patients.

PURPOSE: Lower urinary tract symptoms (LUTS) are common in men and women. The Lower Urinary Tract Dysfunction Research Network (LURN) sought to create a brief, clinically relevant tool to improve upon existing measurements of LUTS in both men and women. MATERIALS AND METHODS: Using a modified Delphi methodology during an expert consensus meeting, we reduced the LURN Comprehensive Assessment of Self-Reported Urinary Symptoms (CASUS) questionnaire to a very brief set of clinically-relevant items measuring LUTS. The sum score of these items was evaluated by comparing to the American Urological Association Symptom Index (AUA-SI), the Urinary Distress Inventory Short Form (UDI-6; in women only), and LUTS screening questions from CASUS, using Pearson correlations, regression analysis, and receiver operating characteristic (ROC) curves. RESULTS: The 10-item LURN-Symptom Index (LURN SI-10) assesses urinary frequency, nocturia, urgency, incontinence, bladder pain, voiding, and post-micturition symptoms (score range: 0–38). The correlation between the LURN SI-10 score and the AUA-SI was 0.77 in men and 0.70 in women. The UDI-6 and LURN SI-10 were highly correlated in women (r=0.76). The LURN SI-10 showed good accuracy in predicting both moderate and severe LUTS as defined by the AUA-SI (area under the ROC curve [AUC] range 0.82–0.90). Similar accuracy was shown in predicting different levels of symptom status using the UDI (AUC range 0.84–0.86). CONCLUSIONS: The LURN SI-10 correlates well with the AUA-SI and UDI-6. It includes items related to a broader spectrum of LUTS, particularly incontinence, bladder pain, and post-micturition symptoms, and applies to both men and women.

BACKGROUND: Data are limited regarding the use of poly(adenosine diphosphate [ADP]-ribose) polymerase inhibitors, such as veliparib, in combination with chemotherapy followed by maintenance as initial treatment in patients with high-grade serous ovarian carcinoma. METHODS: In an international, phase 3, placebo-controlled trial, we assessed the efficacy of veliparib added to first-line induction chemotherapy with carboplatin and paclitaxel and continued as maintenance monotherapy in patients with previously untreated stage III or IV high-grade serous ovarian carcinoma. Patients were randomly assigned in a 1:1:1 ratio to receive chemotherapy plus placebo followed by placebo maintenance (control), chemotherapy plus veliparib followed by placebo maintenance (veliparib combination only), or chemotherapy plus veliparib followed by veliparib maintenance (veliparib throughout). Cytoreductive surgery could be performed before initiation or after 3 cycles of trial treatment. Combination chemotherapy was 6 cycles, and maintenance therapy was 30 additional cycles. The primary end point was investigator-assessed progression-free survival in the veliparib-throughout group as compared with the control group, analyzed sequentially in the BRCA-mutation cohort, the cohort with homologous-recombination deficiency (HRD) (which included the BRCA-mutation cohort), and the intention-to-treat population. RESULTS: A total of 1140 patients underwent randomization. In the BRCA-mutation cohort, the median progression-free survival was 34.7 months in the veliparib-throughout group and 22.0 months in the control group (hazard ratio for progression or death, 0.44; 95% confidence interval [CI], 0.28 to 0.68; P

PURPOSE: Individuals with Huntington disease (HD) experience progressive cognitive decline that may appear years before motor manifestations of the disease. These declines have a profound effect on health-related quality of life (HRQOL) over the disease course, and thus it is important that self-report measures of cognitive function are validated for use in longitudinal studies. METHODS: 359 individuals with premanifest or manifest HD completed baseline and at least one follow-up (12- and 24-month) assessment. Neuro-QoL™ Cognitive Function was administered at each time-point. Participants completed a self-reported global rating of cognitive change, as well as performance-based cognitive changes (using the Symbol Digit Modalities Test). Standardized response means (SRMs) and general linear models evaluated whether Neuro-QoL™ Cognitive Function was responsive to change over time with respect to self-reported and performance-based anchors. Test–retest reliability and known-group validity were also examined. RESULTS: Responsiveness was supported by effect sizes that were small in magnitude, but in the expected direction relative to self-reported and performance-based change. General linear models generally supported 12- and 24-month responsiveness relative to self-reported cognitive change and 12-month responsiveness relative to performance-based change. Test–retest reliability was excellent, and the measure exhibited known-group validity. CONCLUSION: Longitudinal analyses generally indicate that the Neuro-QoL™ Cognitive Function measure is sensitive to change over time in individuals with HD. Neuro-QoL Cognitive Function changes reflect self-reported cognitive change at 12 and 24 months and performance-based change at 12 months. This measure may be useful in clinical trials or longitudinal observation studies.

Background Huntington's disease (HD) is a neurological disorder that causes severe motor symptoms that adversely impact health-related quality of life. Patient-reported physical function outcome measures in HD have shown cross-sectional evidence of validity, but responsiveness has not yet been assessed. Objectives This study evaluates the responsiveness of the Huntington Disease Health-Related Quality of Life (HDQLIFE) and the Quality of Life in Neurological Disorders (Neuro-QoL) physical function measures in persons with HD. Methods A total of 347 participants completed baseline and at least 1 follow-up (12-month and 24-month) measure (HDQLIFE Chorea, HDQLIFE Swallowing Difficulties, HDQLIFE Speech Difficulties, Neuro-QoL Upper Extremity Function, and/or Neuro-QoL Lower Extremity Function). Of the participants that completed the baseline assessment, 338 (90.9%) completed the 12-month assessment, and 293 (78.8%) completed the 24-month assessment. Standardized response means and general linear models evaluated whether the physical function measures were responsive to self-reported and clinician-rated change over time. Results Small to moderate effect sizes for the standardized response means supported 12-month and 24-month responsiveness of the HDQLIFE and Neuro-QoL measures for those with either self-reported or clinician-rated declines in function. General linear models supported 12-month and 24-month responsiveness for all HRQOL measures relative to self-reported declines in health, but generally only 24-month responsiveness was supported relative to clinician-rated declines in function. Conclusions Longitudinal analyses indicate that the HDQLIFE and the Neuro-QoL physical function measures are sensitive to change over time in individuals with HD. Thus, these scales exhibit evidence of responsiveness and may be useful outcome measures in future clinical trials. © 2019 International Parkinson and Movement Disorder Society.

PURPOSE: In the Patient-Reported Outcomes Measurement Information System (PROMIS), seven domains (Physical Function, Anxiety, Depression, Fatigue, Sleep Disturbance, Social Function, and Pain Interference) are packaged together as profiles. Each of these domains can also be assessed using computer adaptive tests (CATs) or short forms (SFs) of varying length (e.g., 4, 6, and 8 items). We compared the accuracy and number of items administrated of CAT versus each SF.METHODS: PROMIS instruments are scored using item response theory (IRT) with graded response model and reported as T scores (mean = 50, SD = 10). We simulated 10,000 subjects from the normal distribution with mean 60 for symptom scales and 40 for function scales, and standard deviation 10 in each domain. We considered a subject's score to be accurate when the standard error (SE) was less than 3.0. We recorded range of accurate scores (accurate range) and the number of items administrated.RESULTS: The average number of items administrated in CAT was 4.7 across all domains. The accurate range was wider for CAT compared to all SFs in each domain. CAT was notably better at extending the accurate range into very poor health for Fatigue, Physical Function, and Pain Interference. Most SFs provided reasonably wide accurate range.CONCLUSIONS: Relative to SFs, CATs provided the widest accurate range, with slightly more items than SF4 and less than SF6 and SF8. Most SFs, especially longer ones, provided reasonably wide accurate range.

Self-reported measurement tools often provide a recall period, eg "In the past 7 days…" For lower urinary tract symptoms the concordance of end of day (daily) reports with 7 and 30-day recalled reports is unknown to our knowledge. We evaluated how accurately 7 or 30-day recall questions capture lower urinary tract symptoms.The 261 female and 254 male participants were recruited from a total of 6 United States tertiary care sites. We evaluated 18 items representing 7 symptoms covering storage, voiding and post-micturition symptoms. Item responses on the daily forms were averaged for a 7 or a 30-day period and compared to the corresponding 7 or 30-day recall version of the item. Analyses were item and gender specific. Within person concordance was assessed using the Pearson correlation. Bias (systematic overreporting or underreporting) was calculated as the difference between the recalled item and the averaged daily item score, and reported as a percent of the item scale.All correlations exceeded 0.60. Correlations between averaged daily reports and recalled reports ranged from 0.72 to 0.89 for 7 days and from 0.71 to 0.91 for 30 days among women, and from 0.68 to 0.90 and 0.68 to 0.95, respectively, among men. Most items did not show systematic bias and the median percent bias did not exceed 10% for any item. However, bias exceeding ±10% for some items was observed in a subset of individuals.Recalled reports during the 7 and 30 days tracked well with averaged daily reports for men and women. Systematic bias was minimal, suggesting that 7 and 30-day recall periods for self-reported lower urinary tract symptoms are reasonable.

The EORTC QLU-C10D is a new multi-attribute utility instrument derived from the widely used cancer-specific quality of life questionnaire, EORTC QLQ-C30. It contains 10 dimensions (physical functioning, role functioning, social functioning, emotional functioning, pain, fatigue, sleep, appetite, nausea, bowel problems), each with four levels. The aim of this study was to provide U.K. general population utility weights for the QLU-C10D. A U.K. online panel was quota-sampled to align the sample to the general population proportions of sex and age (≥18 years). The online valuation survey included a discrete choice experiment (DCE). Each participant was asked to complete 16 choice-pairs, each comprising two QLU-C10D health states plus duration. DCE data were analysed using conditional logistic regression to generate utility weights. Data from 2,187 respondents who completed at least one choice set were included in the DCE analysis. The final U.K. QLU-C10D utility weights comprised decrements for each level of each health dimension. For nine of the 10 dimensions (all except appetite), the expected monotonic pattern was observed across levels: Utility decreased as severity increased. For the final model, consistent monotonicity was achieved by merging inconsistent adjacent levels for appetite. The largest utility decrements were associated with physical functioning and pain. The worst possible health state (the worst level of each dimension) is -0.083, which is considered slightly worse than being dead. The U.K.-specific utility weights will enable cost-utility analysis (CUA) for the economic evaluation of new oncology therapies and technologies in the United Kingdom, where CUA is commonly used to inform resource allocation.

AIMS: General self-efficacy is associated with adaptive coping and positive health outcomes. The Patient-Reported Outcomes Measurement Information System (PROMIS®) has developed self-efficacy item banks for managing chronic conditions, but lacks a general self-efficacy measure. We sought to refine and validate an item-response theory (IRT)-based measure of general self-efficacy for PROMIS®.METHODS: Ten items were modified from the NIH Toolbox® Self-Efficacy Item Bank by creating "confidence" response options, and administered to a general population sample (n = 1000) with the Toolbox® Self-Efficacy Item Bank, Life Orientation Test-Revised (LOT-R), and Generalized Expectancy for Success Scale (GESS). We split the sample in half for exploratory factor analysis (EFA) followed by confirmatory factor analysis (CFA). IRT analyses included evaluation of differential item functioning (DIF).RESULTS: Participants had a mean age of 47.8 years and 50.3% were male. EFA showed all items loaded onto one dominant factor and CFA yielded a good fitting model for a general self-efficacy bank with confidence response options (CFI = 0.987, TLI = 0.984, RMSEA = 0.090). Items showed no evidence of DIF by gender, age, education, or race. Internal consistency reliability was α = .94 and .88 for a new 10-item general self-efficacy bank and 4-item short form, respectively. The new bank was correlated with the LOT-R (r = .58), the GESS (r = .55), and the Toolbox® Self-Efficacy Item Bank (r = .87).CONCLUSIONS: The PROMIS® General Self-Efficacy measure demonstrated sufficient unidimensionality and displayed good internal consistency reliability, model fit, and convergent validity. Further psychometric testing of the PROMIS® General Self-Efficacy Item Bank and Short Form can evaluate its utility in people with chronic health conditions.

Financial distress has been established as a clinically relevant patient-reported outcome associated with worse mortality and quality of life. Our goal was to define factors associated with financial burden (FB) in a public health care system.Patients with advanced lung cancer were recruited from outpatient clinics at the Princess Margaret Cancer Centre (Toronto, Canada). FB was measured with the validated Comprehensive Score for Financial Toxicity (COST) instrument, a 12-item survey scored from 0 to 44, with lower scores reflecting worse financial well-being. Data on patient and treatment characteristics, total out-of-pocket costs (OOP), and private insurance coverage were collected. Multivariable logistic regression models were fit for COST score and each variable, to determine factors associated with greater FB (COST 21).Of 251 patients approached, 200 (80%) participated. The median age of the cohort was 65 years; 56% were female. The median total OOP ranged between $1000 and $5000 CAD. The median COST score was 21 (range, 0-44). FB was associated with age, with patients 65 years reporting greater FB than older patients (COST, 18.0 vs. 24.0; P .0001). In multivariable logistic regression analysis, younger age was associated with greater FB, when adjusting for income, employment status, OOP, and private insurance coverage (odds ratio, 3.6; 95% confidence interval, 1.5-9.1; P .0001).Age is significantly associated with FB in the Canadian (Ontario) public health care system, with younger patients with lung cancer reporting greater financial distress. This study highlights priority patient populations where FB should be routinely assessed and appropriate resources for support offered.

BACKGROUND: The All of Us Research Program is building a national longitudinal cohort and collecting data from multiple information sources (e.g., biospecimens, electronic health records (EHRs), and mobile/wearable technologies) to advance precision medicine. Participant-provided information, collected via surveys, will complement and augment these information sources. We report the process used to develop and refine the initial three surveys for this program. METHODS: The All of Us survey development process included: (1) prioritization of domains for scientific needs, (2) examination of existing validated instruments, (3) content creation, (4) evaluation and refinement via cognitive interviews and online testing, (5) content review by key stakeholders, and (6) launch in the All of Us electronic participant portal. All content was translated into Spanish. RESULTS: We conducted cognitive interviews in English and Spanish with 169 participants, and 573 individuals completed online testing. Feedback led to over 40 item content changes. Lessons learned included: (1) validated survey instruments performed well in diverse populations reflective of All of Us; (2) parallel evaluation of multiple languages can ensure optimal survey deployment; (3) recruitment challenges in diverse populations required multiple strategies; and (4) key stakeholders improved integration of surveys into larger Program context. CONCLUSIONS: This efficient, iterative process led to successful testing, refinement, and launch of three All of Us surveys. Reuse of All of Us surveys, available at http://researchallofus.org, may facilitate large consortia targeting diverse populations in English and Spanish to capture participant-provided information to supplement other data, such as genetic, physical measurements, or data from EHRs.

Aims To develop a representative, self-report assessment of lower urinary tract symptoms (LUTS) for men and women, the symptoms of Lower Urinary Tract Dysfunction Research Network Symptom Index-29 (LURN SI-29). Methods Women and men seeking treatment for LUTS at one of six academic medical centers in the US were assessed at baseline, 3-month and 12-month intervals. Twelve-month data on 78 LURN SI-29 items were analyzed among 353 women and 420 men using exploratory factor analysis (EFA), with factor structure confirmed using confirmatory factor analysis (CFA). Internal consistency, reliability, and validity of the five developed scales were evaluated by assessing correlations with the American Urological Association Symptom Index (AUA-SI), the genitourinary pain index (GUPI), and the Pelvic Floor Distress Inventory-20 (PFDI-20), and by examining expected sex differences in scores. Results EFA results (n = 150 women; 150 men) produced an interpretable eight-factor solution, with three of the factors comprised of dichotomous items addressing LUTS-associated sensations. The remaining five factors, confirmed with CFA in an independent sample of 473 participants, produced five scales: incontinence, urgency, voiding difficulty, bladder pain, and nocturia. Subscales and total LURN SI-29 scores were correlated as expected with AUA-SI, GUPI, and PFDI-20. LURN SI-29 scores also performed as expected in differentiating men from women based upon clinically expected differences, with men reporting more voiding difficulties and nocturia, and women reporting more urgency and incontinence. Conclusions The LURN SI-29 questionnaire has the potential to improve research and clinical outcome measurement for both men and women with LUTS.

BACKGROUND AND PURPOSE. Huntington disease (HD) is a progressive neurodegenerative disorder. There are no HD-specific measures to assess for end-of-life (EOL) preferences that have been validated for clinical use. The purpose of this study is to demonstrate reliability and validity of three HD-specific EOL measures for use in and clinical research settings. METHODS. We examined internal reliability, test-retest reliability, floor and ceiling effects, convergent and discriminant validity, known-groups validity, measurement error, and change over time to systematically examine reliability and validity of the HDQLIFE EOL measures. RESULTS. Internal consistency and test-retest reliability were >0.70. The measures were generally free of floor and ceiling effects and measurement error was minimal. Convergent and discriminant validity were consistent with well-known constructs in the field. Hypotheses for known groups validity were partially supported (there were generally group differences for the EOL Planning measures, but not for Meaning and Purpose or Concern with Death and Dying). Measurement error was acceptable and there were minimal changes over time across the EOL measures. CONCLUSIONS. Results support the clinical utility of the HDQLIFE EOL measures in persons with HD.

To improve the potential for finding clinically important subtypes of patients with lower urinary tract symptoms we developed the CASUS (Comprehensive Assessment of Self-reported Urinary Symptoms). We used it to present data on the experiences of lower urinary tract symptoms in treatment seeking women and men from a prospective observational cohort.We created an initial list of lower urinary tract symptoms that were confirmed in 22 qualitative interviews with providers, and 88 qualitative interviews with care seeking and noncare seeking women and men with lower urinary tract symptoms. Items from extant measures were adopted and revised, and new items were developed. All items were evaluated for understanding in 64 cognitive interviews. Items were administered to a prospective cohort of female and male patients with lower urinary tract symptoms who were seeking care. Analyses were done to describe item response distributions and correlations among item responses separately for women and men.A total of 444 males and 372 females provided responses to the CASUS. Several sets of items showed different relationships for women compared to men. In particular the associations between sensation related items and incontinence related items were generally positive among females but often negative among males.After using an intensive development process the CASUS addresses a wide range of lower urinary tract symptoms. It should help identify clinically important subtypes of patients. Further, item collection can provide the foundation for shorter measures for use in the clinic and as trial end points.

The Patient-Reported Outcomes Measurement Information System® (PROMIS®) is a collection of person-centered measures of physical, mental, and social health in children and adults. Developed using state-of-the-art psychometric methods, these measures are offered as fixed-length questionnaires and computerized adaptive tests, with translations available in Spanish and other languages. This special issue presents articles that illustrate the use of PROMIS® measures to advance health behavior research across a wide range of health studies. (PsycINFO Database Record (c) 2019 APA, all rights reserved).

Background The purpose of this study is to evaluate the content validity of the National Comprehensive Cancer Network – Functional Assessment of Cancer Therapy – Breast Cancer Symptom Index (NFBSI-16) and the Patient-Reported Outcomes Measurement Information System (PROMIS) Physical Function Short Form 10b among patients with hormone receptor positive (HR+)/human epidermal growth factor receptor 2 negative (HER2-) advanced breast cancer. Methods Cognitive debriefing interviews sought to evaluate patients’ ability to read, understand, and meaningfully respond to the questionnaires, as well as to evaluate the questionnaires’ relevance in the target patient population. Interviews were conducted by telephone and lasted approximately 90 min. Audio recordings were transcribed, anonymized, and analyzed using qualitative data analysis software. Results Fifteen cognitive debriefing interviews were conducted with women (mean age 66.0 years [standard deviation = 12.4]). Patients reported metastases in the bone (86.7%), liver (20.0%), lung (13.3%), skin (6.7%), and lymph node (6.7%) (not mutually exclusive). All patients for whom data were available demonstrated understanding of the instructions and the recall period of the NFBSI-16 (n = 14/14, 100.0%) and the PROMIS (n = 14/14, 100.0%). Greater than 90% of patients demonstrated understanding of each of the items in the NFBSI-16 and the PROMIS. Greater than 70% of patients demonstrated understanding of the response options of the NFBSI-16, > 90% understood response options of PROMIS Items 1–6, and ≥ 50% understood response options of PROMIS Items 7–10. Conceptual relevance was supported for most items in both questionnaires based on patients’ reports of experiencing the concepts as part of their breast cancer experience. Conclusions The results of the cognitive debriefing interviews provide evidence that the NFBSI-16 and PROMIS Physical Function Short Form 10b have content validity in the HR+/HER2- advanced breast cancer patient population. Patients may benefit from additional instructions at the point the response options reverse direction in the PROMIS.