How do patients interpret and respond to a novel patient-reported eastern cooperative oncology group performance status (ECOG)?

PURPOSE: Performance status is an important concept in oncology, but is typically clinician-reported. Efforts are underway to include patient-reported measures in cancer care, which may improve patient symptoms, quality of life and overall survival. The purpose of this study was to gain a preliminary understanding of how patients determined their physical performance status based on a novel patient-reported version of the Eastern Cooperative Oncology Group Performance Status (ECOG) scale. METHODS: We conducted qualitative interviews, including concept elicitation and cognitive interviewing as part of the Patient Reports of Physical Functioning Study (PROPS) to investigate how participants selected their answers to a novel patient-reported ECOG. Participants were administered the patient-reported ECOG and asked to describe devices and modifications used to keep up with daily activities. RESULTS: Participants generally understood the ECOG as intended. Participants with recent changes in status had some difficulty selecting an answer. Most participants used modifications and assistive devices in their daily lives but did not incorporate these into their rational for the ECOG. CONCLUSION: The potential benefits of a patient-reported ECOG are numerous and this study demonstrates that participants were able to understand and answer the patient-reported ECOG as intended. We recommend future evaluation for the most-appropriate recall period, whether to include modifications in the ECOG instructions, and if increasing the number of response options to the patient-reported ECOG may improve confidence when providing an answer.

Facets of physical function assessed by patient-reported outcome measures in oncology research.

PURPOSE: The U.S. Food & Drug Administration has identified physical functioning (PF) as a core patient-reported outcome (PRO) in cancer clinical trials. The purpose of this study was to identify PF PRO measures (PROMs) in adult cancer populations and classify the PROMs by content covered (facets of PF) in each measure. METHODS: As part of the Patient Reports of Physical Functioning Study (PROPS) research program, we conducted a targeted literature review to identify PROMs that could be used in clinical trials to evaluate PF from the patient perspective. Next, we convened an advisory panel to conduct a modified, reactive, Delphi study to reach consensus on which PF facets are assessed by PROMs identified in the review. The panel engaged in a "card sort" activity to classify PROM items by PF facets. Consensus was reached when 80% of panel members agreed that at least one facet was being measured by each PROM item. RESULTS: The literature review identified 13 PROMs that met inclusion criteria. Eight facets of PF were identified for classification in the Delphi study: ability, completion, difficulty, limitation, quality, frequency, bother, and satisfaction. Through two rounds, the panel documented and classified conceptual approaches for each PRO item presented. The most prevalent PF facets were ability, difficulty, and limitation. CONCLUSION: Classifying PF PROMs by PF facets will promote more consistent communication regarding the aspects of PF represented in each PROM, helping researchers prioritize measures for inclusion in cancer clinical trials.

What facets of physical function are most important to adults diagnosed with cancer?

PURPOSE: Successful patient-focused drug development involves selecting and measuring outcomes in clinical trials that are important to patients. The U.S. Food & Drug Administration's definition of clinical benefit includes how patients feel, function, or survive. Patients are considered the experts in describing how they feel and function. In cancer trials, patient-reported measures of physical function provide insight into how patients function at baseline, benefit from the interventions being studied, and the impact of treatment side effects. We conducted a qualitative study with adults diagnosed with cancer to describe facets of physical function from their perspective and to identify which facets are most important to this patient population. METHODS: Using concept elicitation and cognitive interviewing techniques, we conducted semi-structured interviews with 72 adults ≥ 22 years of age with cancer who received treatment with an anticancer drug or biologic within six months of the interview. We selected participants using purposive sampling with the aim to elicit diverse experiences regarding how they may interpret and respond to questions related to physical function. Participants were presented with patient-reported outcome (PRO) items representative of PRO measures used in cancer and general populations. RESULTS: Five facets of how physical function relates to activities were defined from the patient perspective: ability, difficulty, limitation, satisfaction, and completion. More than half of the participants indicated that ability was the most important facet of physical function. The next most important were satisfaction (18.3%), limitation (14.1%), difficulty (5.6%), and completion (2.8%). CONCLUSION: This study demonstrates that we must be more specific about the facets of physical function that we set out to assess when we use PRO measures to describe the patient experience. These results have implications for the specificity of physical function facets when measured in cancer clinical trials.

Do PRO Measures Function the Same Way for all Individuals With Heart Failure?

Women diagnosed with heart failure report worse quality of life than men on patient-reported outcome (PRO) measures. An inherent assumption of PRO measures in heart failure is that women and men interpret questions about quality of life the same way. If this is not the case, the risk then becomes that the PRO scores cannot be used for valid comparison or to combine outcomes by subgroups of the population. Inability to compare subgroups validly is a broad issue and has implications for clinical trials, and it also has specific and important implications for identifying and beginning to address health inequities. We describe this threat to validity (the psychometric term is differential item functioning), why it is so important in heart-failure outcomes, the research that has been conducted thus far in this area, the gaps that remain, and what we can do to avoid this threat to validity. PROs bring unique information to clinical decision making, and the validity of PRO measures is key to interpreting differences in heart failure outcomes.

Investigating gender-based differential item functioning on the Kansas City Cardiomyopathy Questionnaire (KCCQ) using qualitative content analysis.

PURPOSE: The purpose of this study is to evaluate potential gender-based differences in interpreting the Kansas City Cardiomyopathy Questionnaire (KCCQ-23) and to explore if there are aspects of health-related quality of life (HRQOL) not captured by the KCCQ-23 that are important to assess in men and/or women with heart failure (HF). METHODS: Patients ≥ 22 years of age with clinician-diagnosed HF and left ventricular ejection fraction ≤ 40% were recruited from two academic medical centers to participate in semi-structured concept elicitation and cognitive debriefing interviews. Enrollment was stratified by patient-identified gender (half women/half men). All interviews were conducted over the phone/web and audio recorded. Interviews were transcribed and descriptive qualitative content analysis was used to summarize findings overall and by gender. RESULTS: Twenty-five adults (56% women) diagnosed with HF participated. The average age was 67 years (range: 25-88). Women attributed a wider variety of symptoms to HF than men. Some participants had difficulty differentiating whether their experiences were due to HF, side effects of their medications, or age. We found very little evidence that participants interpreted KCCQ-23 items differently based on gender. CONCLUSIONS: Overall, our findings indicate that interpretation of the KCCQ-23 items were similar in men and women. However, some modifications to items may improve clarity of interpretation for a wide range of patients.

Health-related quality of life by race, ethnicity, and country of origin among cancer survivors.

BACKGROUND: Typical cancer research studies of health-related quality of life (HRQOL) in the United States do not include country of origin when examining race and ethnic group differences. This population-based, cross-sectional study used an innovative methodology to examine how self-reported racial and ethnic groups, by country of origin, report differential HRQOL experiences after adjusting for clinical and demographic characteristics, including socioeconomic status. METHODS: Recruited from 4 cancer registries in California, Louisiana, and New Jersey, cancer survivors completed Patient-Reported Outcomes Measurement Information System measures of fatigue, pain interference, anxiety, depression, sleep disturbance, physical function, ability to participate in social roles, and cognitive function. Latent profile analysis clustered survivors in HRQOL clusters based on including all the Patient-Reported Outcomes Measurement Information System domains. RESULTS: The 5366 participants (60% female; 40% male; average age of 59.8 years) included 17% Asian, 18% Black, 21% Hispanic, and 41% White survivors. Survivors were grouped into 4 clusters: high HRQOL (26%), average HRQOL (34%), low HRQOL (29%), and very low HRQOL (11%). Among many differences by race, ethnicity, and country of origin, Caribbean cancer survivors were more likely to be in the very low HRQOL cluster (odds ratio = 2.67, 95% confidence interval = 1.31 to 5.43) compared with non-Hispanic White survivors. Similarly, American Indian and Alaska Native, Cuban, Dominican, and Puerto Rican cancer survivors had relatively high percentages in the very low HRQOL cluster. CONCLUSIONS: This study found statistically significant differences in HRQOL experience by race, ethnicity, and country of origin, even after adjusting for social determinants of health. These findings inform future HRQOL research to include these self-reported factors.

Psychometric Evaluation of the Kansas City Cardiomyopathy Questionnaire in Men and Women With Heart Failure.

BACKGROUND: The Kansas City Cardiomyopathy Questionnaire (KCCQ) has been psychometrically evaluated in multiple heart failure (HF) populations, but the comparability of its psychometric properties between men and women is unknown. METHODS: Data from 3 clinical trials (1 in stable HF with preserved ejection fraction, 1 each in stable and acute HF with reduced ejection fraction) and 1 prospective cohort study (stable HF with reduced ejection fraction), incorporating 6773 men and 3612 women with HF, were used to compare the construct validity, internal and test-retest reliability, ability to detect change, predict mortality and hospitalizations and minimally important differences between the 2 sexes. Interactions of the KCCQ overall summary and subdomain scores by sex were independently examined. RESULTS: The KCCQ-Overall Summary score correlated well with New York Heart Association functional class in both sexes across patients with stable (correlation coefficient: -0.40 in men versus -0.49 in women) and acute (-0.37 in men versus -0.34 in women) HF. All KCCQ subdomains demonstrated concordant relationships with relevant comparison standards with no significant interactions by sex in 19 of 21 of these construct validity analyses. All KCCQ scores were equally predictive and other psychometric evaluations showed similar results by sex: test-retest reliability (intraclass correlation coefficient 0.94 in men versus 0.92 in women), responsive to change (standardized response mean 1.01 in both sexes), as were the minimally important differences and internal reliability. CONCLUSIONS: The psychometric properties of the KCCQ, in terms of validity, prognosis, reliability, and sensitivity to change, are comparable in men and women with HF with preserved ejection fraction and HF with reduced ejection fraction.

Association Between Public Knowledge About COVID-19, Trust in Information Sources, and Adherence to Social Distancing: Cross-Sectional Survey.

BACKGROUND: The success of behavioral interventions and policies designed to reduce the impact of the COVID-19 pandemic depends on how well individuals are informed about both the consequences of infection and the steps that should be taken to reduce the impact of the disease. OBJECTIVE: The aim of this study was to investigate associations between public knowledge about COVID-19, adherence to social distancing, and public trust in government information sources (eg, the US Centers for Disease Control and Prevention), private sources (eg, FOX and CNN), and social networks (eg, Facebook and Twitter) to inform future policies related to critical information distribution. METHODS: We conducted a cross-sectional survey (N=1243) between April 10 and 14, 2020. Data collection was stratified by US region and other demographics to ensure representativeness of the sample. RESULTS: Government information sources were the most trusted among the public. However, we observed trends in the data that suggested variations in trust by age and gender. White and older populations generally expressed higher trust in government sources, while non-White and younger populations expressed higher trust in private sources (eg, CNN) and social networks (eg, Twitter). Trust in government sources was positively associated with accurate knowledge about COVID-19 and adherence to social distancing. However, trust in private sources (eg, FOX and CNN) was negatively associated with knowledge about COVID-19. Similarly, trust in social networks (eg, Facebook and Twitter) was negatively associated with both knowledge and adherence to social distancing. CONCLUSIONS: During pandemics such as the COVID-19 outbreak, policy makers should carefully consider the quality of information disseminated through private sources and social networks. Furthermore, when disseminating urgent health information, a variety of information sources should be used to ensure that diverse populations have timely access to critical knowledge.