Lee, Minji K.

Background/Aims: The Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE ® ) was developed to capture symptomatic adverse events from the patient perspective. We aim to describe statistical properties of PRO-CTCAE items and summary scores and to provide evidence for recommendations regarding PRO-CTCAE administration and reporting. Methods: Using data from the PRO-CTCAE validation study (NCT02158637), prevalence, means, and standard deviations of PRO-CTCAE items, composite scores, and mean and maximum scores across attributes (frequency, severity, and/or interference) of symptomatic adverse events were calculated. For each adverse event, correlations and agreement between attributes, correlations between attributes and composite scores, and correlations between composite, mean, and maximum scores were estimated. Results: PRO-CTCAE items were completed by 899 patients with various cancer types. Most patients reported experiencing one or more adverse events, with the most prevalent being fatigue (87.7%), sad/unhappy feelings (66.0%), anxiety (63.6%), pain (63.2%), insomnia (61.8%), and dry mouth (60.0%). Attributes were moderately to strongly correlated within an adverse event ( r = 0.53 to 0.77, all p < 0.001) but not fully concordant (κ weighted = 0.26 to 0.60, all p < 0.001), with interference demonstrating lowest mean scores and prevalence among attributes of the same adverse event. Attributes were moderately to strongly correlated with composite scores ( r = 0.67 to 0.97, all p < 0.001). Composite scores were moderately to strongly correlated with mean and maximum scores for the same adverse event ( r = 0.69 to 0.94, all p < 0.001). Correlations between composite scores of different adverse events varied widely ( r = 0.04 to 0.68) but were moderate to strong for conceptually related adverse events. Conclusions: Results provide evidence for PRO-CTCAE administration and reporting recommendations that the full complement of attributes be administered for each adverse event, and that attributes as well as summary scores be reported.

Abstract Patient-reported outcomes (PROs) are often collected in cancer clinical trials. Data obtained from trials with PROs are essential in evaluating participant experiences relating to symptoms, financial toxicity, or health-related quality of life. Although most features of clinical trial design, implementation, and analyses apply to trials with PROs, several considerations are unique. In this paper, we focus on specific issues such as selection of the tool, timing and frequency of assessments, and data collection methods. We discuss how the estimand framework can be used in connection with PROs, properties of common estimation methods, and handling of missing outcomes. With a plethora of literature available, we aim to summarize best practices and pragmatic approaches to the design and analysis of the studies incorporating PROs.

5546 Background: Advanced ovarian cancer (OC) is managed with cytoreductive surgery and platinum-based chemotherapy. Prehabilitation is a multimodal intervention to improve physical and mental health prior to oncologic surgery. This study evaluates baseline differences in clinical characteristics, functional status, and patient-reported outcomes (PROs) between patients undergoing primary cytoreductive surgery (PCS) versus neoadjuvant chemotherapy (NACT) who enrolled in a prospective prehabilitation trial. Methods: This was a single-institution, non-randomized prospective behavioral intervention trial of patients with suspected advanced stage (IIIC/IV) OC. Baseline functional testing included the Short Physical Performance Battery (SPPB), Fried Frailty Index (FFI), gait speed, and grip strength. Patient-Reported Outcomes Measurement Information System (PROMIS) metrics, the Mindfulness Attention Awareness Scale (MAAS), and the Perceived Stress Scale (PSS-10) were used to evaluate PROs. Comparisons between cohorts utilized two-sample t-test or Kruskal Wallis test for continuous variables and chi-square or Fisher’s exact test for categorical variables. T-scores were obtained for PROMIS data using the HealthMeasures Scoring Service; validated cut points for PROMIS, MAAS, and PSS-10 were used for categorization. Results: Amongst the 53 enrolled patients, physical and emotional distress was higher amongst OC patients compared to population means. When compared to PCS patients, NACT patients were more likely to report “severe” in at least one PROMIS domain (emotional distress and anxiety, pain interference, physical function, and fatigue, p<0.01). NACT patients reported significantly higher levels of perceived stress (p<0.01). NACT patients were more likely to have stage IV disease, albumin <3.5 g/dL, and lower gait speed and grip strength (all p<0.01). Despite differences in clinical variables and PROs including physical function, there were no measurable differences in physical function based on SPPB and FFI. Conclusions: Advanced OC patients have high psychosocial needs, with NACT patients reporting severe perceived symptoms on PRO measures. The PSS-10 may be a valuable screening tool for patients undergoing NACT to prioritize supportive care services. Clinical trial information: NCT05047926 . [Table: see text]

Health insurance literacy (HIL) constructs are related to financial toxicity in patients with cancer.

Objectives Determine whether there are different longitudinal patterns of treatment burden in people living with multiple chronic conditions (MCC) and, if so, explore predictors that might reveal potential routes of intervention. Methods We analyzed data from a prospective mailed survey study of 396 adults living with MCC in southeastern Minnesota, USA. Participants completed a measure of treatment burden, the Patient Experience with Treatment and Self-management (PETS), and valid measures of health-related and psycho-social concepts at baseline, 6, 12, and 24 months. Latent class growth mixture modeling (LCGM) determined trajectories of treatment burden in two summary index scores of the PETS: Workload and Impact. Multivariable logistic regressions were used to identify independent predictors of the trajectories. Results LCGM supported a 2-class model for PETS Workload, including a group of consistently high workload ( N = 69) and a group of consistently low workload ( N = 311) over time. A 3-class model was supported for PETS Impact, including groups of consistently high impact ( N = 62), consistently low impact ( N = 278), and increasing impact ( N = 51) over time. Logistic regression analyses showed that the following factors were associated with patterns of consistently high or increasing treatment burden over time: lower health literacy, lower self-efficacy, more interpersonal challenges with others, and worse subjective reports of physical and mental health (all p < .05). Conclusions Different longitudinal patterns of treatment burden exist among people with MCC. Raising health literacy, enhancing self-efficacy, and lessening the effects of negative social interactions might help reduce treatment burden.

Background In clinical trials and clinical practice, patient-reported outcomes are almost always assessed using multiple patient-reported outcome measures at the same time. This raises concerns about whether patient responses are affected by the order in which the patient-reported outcome measures are administered. Methods This questionnaire-based study of order effects included adult cancer patients from five cancer centers. Patients were randomly assigned to complete questionnaires via paper booklets, interactive voice response system, or tablet web survey. Linear Analogue Self-Assessment, Patient-Reported Outcomes Version of the Common Terminology Criteria for Adverse Events, and Patient-Reported Outcomes Measurement Information System assessment tools were each used to measure general health, physical function, social function, emotional distress/anxiety, emotional distress/depression, fatigue, sleep, and pain. The order in which the three tools, and domains within tools, were presented to patients was randomized. Rates of missing data, scale scores, and Cronbach’s alpha coefficients were compared by the order in which they were assessed. Analyses included Cochran–Armitage trend tests and mixed models adjusted for performance score, age, sex, cancer type, and curative intent. Results A total of 1830 patients provided baseline patient-reported outcome assessments. There were no significant trends in rates of missing values by whether a scale was assessed earlier or later. The largest order effect for scale scores was due to a large mean score at one assessment time point. The largest difference in Cronbach’s alpha between the versions for the Patient-Reported Outcomes Measurement Information System scales was 0.106. Conclusion The well-being of a cancer patient has many different aspects such as pain, fatigue, depression, and anxiety. These are assessed using a variety of surveys often collected at the same time. This study shows that the order in which the different aspects are collected from the patient is not important.

Abstract Background The symptom burden associated with cancer and its treatment can negatively affect patients’ quality of life and survival. Symptom-focused collaborative care model (CCM) interventions can improve outcomes, but only if patients engage with them. We assessed the receptivity of severely symptomatic oncology patients to a remote nurse-led CCM intervention. Methods In a pragmatic, cluster-randomized, stepped-wedge trial conducted as part of the National Cancer Institute IMPACT Consortium (E2C2, NCT03892967), patients receiving cancer care were asked to rate their sleep disturbance, pain, anxiety, emotional distress, fatigue, and limitations in physical function. Patients reporting at least 1 severe symptom (≥7/10) were offered phone consultation with a nurse symptom care manager (RN SCM). Initially, patients had to “opt-in” to receive a call, but the protocol was later modified so they had to “opt-out” if they did not want a call. We assessed the impact of opt-in vs opt-out framing and patient characteristics on receptiveness to RN SCM calls. All statistical tests were 2-sided. Results Of the 1204 symptom assessments (from 864 patients) on which at least 1 severe symptom was documented, 469 (39.0%) indicated receptivity to an RN SCM phone call. The opt-out period (odds ratio [OR] = 1.61, 95% confidence interval [CI] = 1.12 to 2.32, P = .01), receiving care at a tertiary care center (OR = 3.59, 95% CI = 2.18 to 5.91, P &lt; .001), and having severe pain (OR = 1.80, 95% CI = 1.24 to 2.62, P = .002) were associated with statistically significantly greater willingness to receive a call. Conclusions Many severely symptomatic patients were not receptive to an RN SCM phone call. Better understanding of reasons for refusal and strategies for improving patient receptivity are needed.

Background: Earlier studies report a direct association between diseases of the skin—particularly those on the face—and depression. However, to our knowledge, such associations have not been examined in patients with non-squamous, non-basal call skin cancers. Methods: The primary goal was to assess whether malignant skin disease—specifically on the face as opposed to other sites—was associated with depression. The medical records of patients with cutaneous cancer (either primary or metastatic but non-squamous, non-basal cell) were reviewed for the relevant data. Results: One hundred and sixty-five patients were studied. Only 23 patients (14%) had metastases to the face, and 115 (70%) had a readily viewable skin cancer. Twenty-one patients (13%) developed depression after a diagnosis of cutaneous cancer (of note, the rate of missing data for depression was 37%). Only one patient with facial cutaneous cancer manifested depression, yielding an odds ratio for not developing depression (95% confidence interval (CI)) of 4.4 (0.5,35); p = 0.13. Depression appeared to occur more often in women (62% versus 43%), patients with a history of depression (52% versus 6%), and younger patients (median age with and without depression 55 years and 67 years, respectively). Conclusion: In contrast to other cutaneous diseases, no association was found between cutaneous cancer to the face and depression. Nonetheless, high rates of missing data underscore the need to focus on depression in patients with cutaneous cancers in the future.

Objective.: The Dizziness Handicap Inventory (DHI) is a 25-item self-report questionnaire developed to measure the disabling and handicapping impact of dizziness. The present investigation was conducted in an effort to re-assess the factor structure of the DHI. Study Design.: Retrospective study. Setting.: Tertiary care center. Patients.: Subjects were 1,991 patients who were evaluated in the Mayo Clinic-Rochester Vestibular and Balance Laboratory. Main Outcome Measures.: Exploratory factor analysis: an exploratory bifactor analysis (EFA) with bifactor rotation was used to analyze a random sample of 999 patients. Analyses were used to determine the dominance of the general factor (i.e., total score) relative to the group factor (i.e., subscales). Confirmatory factor analysis: a confirmatory bifactor graded response model was fit with appropriate item-to-group relationships that was discovered by our exploratory analyses. To validate the bifactor model that was identified with the exploratory analyses, a bifactor model with three grouping factors (i.e., Physical manifestations, Catastrophic impact of dizziness, and the Emotional impact of dizziness) were fit to a different random sample of 992 patients using the new item-to-group factor specifications. Results.: In the confirmatory analyses, all items had a positive factor loading on the general factor. There were 14 items that loaded on the general factor only. The rest of the items (n = 11) loaded on both the general factor and one of three group factors. Conclusions.: Conclusions of the study revealed several findings: 1) reporting the result as a total score (i.e., a single general factor) is warranted, and, 2) there is statistical support for the existence of three subscales representing: the Physical manifestations, Catastrophic impact, and Emotional impact of dizziness and vertigo.