Sofia F. Garcia

Standardizing patient-reported outcomes assessment in cancer clinical trials: a patient-reported outcomes measurement information system initiative.

Patient-reported outcomes (PROs), such as symptom scales or more broad-based health-related quality-of-life measures, play an important role in oncology clinical trials. They frequently are used to help evaluate cancer treatments, as well as for supportive and palliative oncology care. To be most beneficial, these PROs must be relevant to patients and clinicians, valid, and easily understood and interpreted. The Patient-Reported Outcomes Measurement Information System (PROMIS) Network, part of the National Institutes of Health Roadmap Initiative, aims to improve appreciably how PROs are selected and assessed in clinical research, including clinical trials. PROMIS is establishing a publicly available resource of standardized, accurate, and efficient PRO measures of major self-reported health domains (eg, pain, fatigue, emotional distress, physical function, social function) that are relevant across chronic illnesses including cancer. PROMIS is also developing measures of self-reported health domains specifically targeted to cancer, such as sleep/wake function, sexual function, cognitive function, and the psychosocial impacts of the illness experience (ie, stress response and coping; shifts in self-concept, social interactions, and spirituality). We outline the qualitative and quantitative methods by which PROMIS measures are being developed and adapted for use in clinical oncology research. At the core of this activity is the formation and application of item banks using item response theory modeling. We also present our work in the fatigue domain, including a short-form measure, as a sample of PROMIS methodology and work to date. Plans for future validation and application of PROMIS measures are discussed.

Measuring social health in the patient-reported outcomes measurement information system (PROMIS): item bank development and testing

PurposeTo develop a social health measurement framework, to test items in diverse populations and to develop item response theory (IRT) item banks.MethodsA literature review guided framework development of Social Function and Social Relationships sub-domains. Items were revised based on patient feedback, and Social Function items were field-tested. Analyses included exploratory factor analysis (EFA), confirmatory factor analysis (CFA), two-parameter IRT modeling and evaluation of differential item functioning (DIF).ResultsThe analytic sample included 956 general population respondents who answered 56 Ability to Participate and 56 Satisfaction with Participation items. EFA and CFA identified three Ability to Participate sub-domains. However, because of positive and negative wording, and content redundancy, many items did not fit the IRT model, so item banks do not yet exist. EFA, CFA and IRT identified two preliminary Satisfaction item banks. One item exhibited trivial age DIF.ConclusionAfter extensive item preparation and review, EFA-, CFA- and IRT-guided item banks help provide increased measurement precision and flexibility. Two Satisfaction short forms are available for use in research and clinical practice. This initial validation study resulted in revised item pools that are currently undergoing testing in new clinical samples and populations.

Environmental Barriers and Supports to Everyday Participation: A Qualitative Insider Perspective From People With Disabilities

OBJECTIVE To describe environmental factors that influence participation of people with disabilities. DESIGN Constant comparative, qualitative analyses of transcripts from 36 focus groups across 5 research projects. SETTING Home, community, work, and social participation settings. PARTICIPANTS Community-dwelling people (N=201) with diverse disabilities (primarily spinal cord injury, traumatic brain injury, and stroke) from 8 states. INTERVENTIONS None. MAIN OUTCOME MEASURES Environmental barriers and supports to participation. RESULTS We developed a conceptual framework to describe how environmental factors influence the participation of people with disabilities, highlighting 8 domains of environmental facilitators and barriers (built, natural, assistive technology, transportation, information and technology access, social support and attitudes, systems and policies, economics) and a transactional model showing the influence of environmental factors on participation at the micro (individual), mesa (community), and macro (societal) levels. Focus group data validated some International Classification of Functioning, Disability and Health environmental categories while also bringing unique factors (eg, information and technology access, economic quality of life) to the fore. Data were used to construct items to enable people with disabilities to assess the impact of environmental factors on everyday participation from their firsthand experience. CONCLUSIONS Participants with disabilities voiced the need to evaluate the impact of the environment on their participation at the immediate, community, and societal levels. The results have implications for assessing environmental facilitators and barriers to participation within rehabilitation and community settings, evaluating outcomes of environmental interventions, and effecting system and policy changes to target environmental barriers that may result in societal participation disparities versus opportunities.

Environmental factors item development for persons with stroke, traumatic brain injury, and spinal cord injury.

OBJECTIVES To describe methods used in operationalizing environmental factors; to describe the results of a research project to develop measures of environmental factors that affect participation; and to define an initial item set of facilitators and barriers to participation after stroke, traumatic brain injury, and spinal cord injury. DESIGN Instrument development included an extensive literature review, item classification and selection, item writing, and cognitive testing following the approach of the Patient-Reported Outcomes Measurement Information System. SETTING Community. PARTICIPANTS Content area and outcome measurement experts (n=10) contributed to instrument development; individuals (n=200) with the target conditions participated in focus groups and in cognitive testing (n=15). INTERVENTIONS None. MAIN OUTCOME MEASURES Environmental factor items were categorized in 6 domains: assistive technology; built and natural environment; social environment; services, systems, and policies; access to information and technology; and economic quality of life. RESULTS We binned 2273 items across the 6 domains, winnowed this pool to 291 items for cognitive testing, and recommended 274 items for pilot data collection. CONCLUSIONS Five of the 6 domains correspond closely to the International Classification of Functioning, Disability and Health taxonomy of environmental factors; the sixth domain, economic quality of life, reflects an important construct that reflects financial resources that affect participation. Testing with a new and larger sample is underway to evaluate reliability, validity, and sensitivity.

Physical, emotional, and social health differences between posttreatment young adults with cancer and matched healthy controls

Young adults (YAs; ages 18‐39 years) with cancer face interrupted developmental milestones and increased stressors that can adversely influence psychosocial adjustment. Transitioning from active treatment to posttreatment survivorship can be particularly challenging. The purpose of this study is to describe the health‐related quality of life (HRQL) and psychological adaptation of YAs after treatment, relative to young adults without cancer.

Health Literacy and Patient-Reported Outcomes: A Cross-Sectional Study of Underserved English- and Spanish-Speaking Patients With Type 2 Diabetes

This study examined associations between patient characteristics, health behaviors, and health outcomes and explored the role of health literacy as a potential mediator of outcomes. English- and Spanish-speaking adults with Type 2 diabetes used a bilingual multimedia touchscreen to complete questionnaires. The behavioral model for vulnerable populations guided multivariable regression and mediation testing. Dependent variables were diabetes self-care, health status, and satisfaction with communication. Independent variables included sociodemographic and clinical characteristics, health literacy, health beliefs, and self-efficacy. Spanish speakers had lower health literacy and poorer physical, mental, and overall health compared to English speakers. Higher health literacy was associated with less social support for diet, fewer diet and medication barriers, younger age, higher diabetes knowledge, and talking with health care professionals to get diabetes information. In contrast to expectations, health literacy was not associated with diabetes self-care, health status, or satisfaction with communication, and it did not mediate the effects of other factors on these outcomes. Diabetes self-efficacy was significantly associated with health behaviors and outcomes. The association between Spanish language preference and poorer health was not mediated by this groups lower health literacy. Increasing health-related self-efficacy might be an important clinical strategy for improving outcomes in underserved patients with Type 2 diabetes.

The psychosocial impact of cancer: Evidence in support of independent general positive and negative components

PurposeConsiderable research has demonstrated the negative psychosocial impact of cancer. Recent work has highlighted positive psychosocial outcomes. Research is now needed to evaluate the relationship between negative and positive impacts. This paper reports the development and validation of a measurement model capturing positive and negative psychosocial illness impacts.MethodsThe sample included 754 cancer patients on- or post-treatment. Item development was informed by literature review, expert input patient interviews and the results of a pilot study of 205 cancer patients, resulting in 43 positive and 46 negative items. Factor analyses were used to evaluate the dimensionality of the item pools. Analysis of variance (ANOVA) was used to examine relationships between psychosocial illness impact and other variables.ResultsUnidimensionality was demonstrated within but not across negative and positive impact items. ANOVA results showed differential relationships between negative and positive impacts, respectively, and patient sociodemographic and clinical variables.ConclusionPositive and negative psychosocial illness impacts are best conceptualized and measured as two independent factors. Computerized adaptive tests and short-form measures developed from this comprehensive psychosocial illness impact item bank may benefit future research and clinical applications.

Literacy-fair measurement of health-related quality of life will facilitate comparative effectiveness research in Spanish-speaking cancer outpatients.

Background:Health-related quality of life (HRQL) assessment is frequently used in comparative effectiveness research, but low-literacy patients are often excluded. Appropriately translated and user-friendly HRQL measures are essential to ensure inclusion of low-literate and non-English-speaking patients in comparative effectiveness research. Objectives:To compare HRQL responses across literacy levels in Spanish-speaking patients with cancer using a multimedia touch screen program. Subjects:A total of 414 adult patients with cancer (213 with low literacy and 201 with high literacy). Research Design:The touch screen system administered 3 questionnaires: The Functional Assessment of Cancer Therapy-General, the Short Form-36 Health Survey, and the Standard Gamble Utility Questionnaire. Measurement bias was evaluated using item response theory. Effects of literacy on HRQL were evaluated using regression models. Results:Patients rated the touch screen easy to use and commented favorably on the multimedia approach. There was statistically significant item response theory measurement bias in 6 of 10 HRQL subscales; however, only 3 showed meaningful bias. Low-literacy patients had significantly lower mean scores on 3 of 4 Functional Assessment of Cancer Therapy-General subscales, before and after adjustment for patient characteristics. Low-literacy patients also had significantly lower mean scores on 5 of 6 Short Form-36 subscales; adjustment for patient characteristics attenuated or eliminated differences. Similar proportions of low- and high-literacy patients valued their current health as equivalent to perfect health. Conclusions:This study demonstrates the feasibility of this multimedia touch screen program for low-literacy patients. The program will provide opportunities to evaluate the effectiveness of interventions in more diverse patient populations.

Development and validation of the functional assessment of chronic illness therapy treatment satisfaction (FACIT TS) measures

PurposeTo develop and validate a new functional assessment of chronic illness therapy (FACIT) measure of satisfaction with treatment for chronic illnesses such as cancer and HIV/AIDS.MethodsTo define domains and generate items, a literature review informed creation of semi-structured interview guides for patients and an international expert panel of clinicians and researchers. Patients and experts also rated 15 areas of satisfaction for relevance. The final list of items underwent further refinement by the original expert panel and a new group of clinical experts. Items were tested in four studies (primarily lung cancer) and data were pooled for analysis. Exploratory and confirmatory factor analyses (CFA), and item response theory modeling were conducted to evaluate dimensionality. Internal consistency reliability and test–retest reliability were both evaluated. Validity was evaluated by correlating the FACIT subscale scores and measures of comparable concepts and by testing the scales’ ability to distinguish people according to their overall treatment satisfaction.ResultsTwo instruments were created: the FACIT TS-general (G), an overall evaluation of current treatment, and the FACIT TS-patient satisfaction (PS), a measure of patient satisfaction. CFA results were not optimal for a five-factor solution for PS. Internal consistency reliability met psychometric standards (≥0.70) for all PS subscales. Construct validity was established for the PS subscales: Physician Communication, Treatment Staff Communication, Technical Competence, Confidence and Trust, and Nurse Communication.ConclusionsThe two instruments generated here offer a new way to assess several key dimensions of patient satisfaction with treatment, especially for people with lung cancer.

Development of Self-Report Measures of Social Attitudes That Act As Environmental Barriers and Facilitators for People With Disabilities

OBJECTIVE To describe the development of new self-report measures of social attitudes that act as environmental facilitators or barriers to the participation of people with disabilities in society. DESIGN A mixed-methods approach included a literature review; item classification, selection, and writing; cognitive interviews and field testing of participants with spinal cord injury (SCI), traumatic brain injury (TBI), or stroke; and rating scale analysis to evaluate initial psychometric properties. SETTING General community. PARTICIPANTS Individuals with SCI, TBI, or stroke participated in cognitive interviews (n=9); community residents with those same conditions participated in field testing (n=305). INTERVENTIONS None. MAIN OUTCOME MEASURE Self-report item pool of social attitudes that act as facilitators or barriers to people with disabilities participating in society. RESULTS An interdisciplinary team of experts classified 710 existing social environment items into content areas and wrote 32 new items. Additional qualitative item review included item refinement and winnowing of the pool prior to cognitive interviews and field testing of 82 items. Field test data indicated that the pool satisfies a 1-parameter item response theory measurement model and would be appropriate for development into a calibrated item bank. CONCLUSIONS Our qualitative item review process supported a social environment conceptual framework that includes both social support and social attitudes. We developed a new social attitudes self-report item pool. Calibration testing of that pool is underway with a larger sample to develop a social attitudes item bank for persons with disabilities.