Nan Rothrock

The Patient-Reported Outcomes Measurement Information System (PROMIS): progress of an NIH Roadmap cooperative group during its first two years.

Background:The National Institutes of Health (NIH) Patient-Reported Outcomes Measurement Information System (PROMIS) Roadmap initiative (www.nihpromis.org) is a 5-year cooperative group program of research designed to develop, validate, and standardize item banks to measure patient-reported outcomes (PROs) relevant across common medical conditions. In this article, we will summarize the organization and scientific activity of the PROMIS network during its first 2 years. Design:The network consists of 6 primary research sites (PRSs), a statistical coordinating center (SCC), and NIH research scientists. Governed by a steering committee, the network is organized into functional subcommittees and working groups. In the first year, we created an item library and activated 3 interacting protocols: Domain Mapping, Archival Data Analysis, and Qualitative Item Review (QIR). In the second year, we developed and initiated testing of item banks covering 5 broad domains of self-reported health. Results:The domain mapping process is built on the World Health Organization (WHO) framework of physical, mental, and social health. From this framework, pain, fatigue, emotional distress, physical functioning, social role participation, and global health perceptions were selected for the first wave of testing. Item response theory (IRT)-based analysis of 11 large datasets supplemented and informed item-level qualitative review of nearly 7000 items from available PRO measures in the item library. Items were selected for rewriting or creation with further detailed review before the first round of testing in the general population and target patient populations. Conclusions:The NIH PROMIS network derived a consensus-based framework for self-reported health, systematically reviewed available instruments and datasets that address the initial PROMIS domains. Qualitative item research led to the first wave of network testing which began in the second year.

Evaluation of item candidates: The PROMIS qualitative item review

One of the PROMIS (Patient-Reported Outcome Measurement Information System) network’s primary goals is the development of a comprehensive item bank for patient-reported outcomes of chronic diseases. For its first set of item banks, PROMIS chose to focus on pain, fatigue, emotional distress, physical function, and social function. An essential step for the development of an item pool is the identification, evaluation, and revision of extant questionnaire items for the core item pool. In this work, we also describe the systematic process wherein items are classified for subsequent statistical processing by the PROMIS investigators. Six phases of item development are documented: identification of extant items, item classification and selection, item review and revision, focus group input on domain coverage, cognitive interviews with individual items, and final revision before field testing. Identification of items refers to the systematic search for existing items in currently available scales. Expert item review and revision was conducted by trained professionals who reviewed the wording of each item and revised as appropriate for conventions adopted by the PROMIS network. Focus groups were used to confirm domain definitions and to identify new areas of item development for future PROMIS item banks. Cognitive interviews were used to examine individual items. Items successfully screened through this process were sent to field testing and will be subjected to innovative scale construction procedures.

Development of a PROMIS item bank to measure pain interference

&NA; This paper describes the psychometric properties of the PROMIS‐pain interference (PROMIS‐PI) bank. An initial candidate item pool (n = 644) was developed and evaluated based on the review of existing instruments, interviews with patients, and consultation with pain experts. From this pool, a candidate item bank of 56 items was selected and responses to the items were collected from large community and clinical samples. A total of 14,848 participants responded to all or a subset of candidate items. The responses were calibrated using an item response theory (IRT) model. A final 41‐item bank was evaluated with respect to IRT assumptions, model fit, differential item function (DIF), precision, and construct and concurrent validity. Items of the revised bank had good fit to the IRT model (CFI and NNFI/TLI ranged from 0.974 to 0.997), and the data were strongly unidimensional (e.g., ratio of first and second eigenvalue = 35). Nine items exhibited statistically significant DIF. However, adjusting for DIF had little practical impact on score estimates and the items were retained without modifying scoring. Scores provided substantial information across levels of pain; for scores in the T‐score range 50–80, the reliability was equivalent to 0.96–0.99. Patterns of correlations with other health outcomes supported the construct validity of the item bank. The scores discriminated among persons with different numbers of chronic conditions, disabling conditions, levels of self‐reported health, and pain intensity (p < 0.0001). The results indicated that the PROMIS‐PI items constitute a psychometrically sound bank. Computerized adaptive testing and short forms are available.

Relative to the general US population, chronic diseases are associated with poorer health-related quality of life as measured by the Patient-Reported Outcomes Measurement Information System (PROMIS)

OBJECTIVES The Patient-Reported Outcomes Measurement Information System (PROMIS) allows assessment of the impact of chronic conditions on health-related quality of life (HRQL) across diseases. We report on the HRQL impact of individual and comorbid conditions as well as conditions that are described as limiting activity. STUDY DESIGN AND SETTING Data were collected through online and clinic recruitment as part of the PROMIS item calibration sample (n=21,133). Participants reported the presence or absence of 24 chronic health conditions and whether their activity was limited by each condition. RESULTS Across health status domains, the presence of a chronic condition was associated with poorer scores than those without a diagnosis, particularly for those individuals who reported that their condition was disabling. The magnitude of detriment in HRQL was more pronounced for individuals with two or more chronic conditions and could not be explained by sociodemographic factors. Patterns of HRQL deficits varied across disease and comorbidity status. CONCLUSION The impact of chronic conditions, particularly when experienced with comorbid disease, is associated with detriments in HRQL. The negative impact on HRQL varies across symptoms and functional areas within a given condition.

Neuro-QOL Brief measures of health-related quality of life for clinical research in neurology

Objective: To address the need for brief, reliable, valid, and standardized quality of life (QOL) assessment applicable across neurologic conditions. Methods: Drawing from larger calibrated item banks, we developed short measures (8–9 items each) of 13 different QOL domains across physical, mental, and social health and evaluated their validity and reliability. Three samples were utilized during short form development: general population (Internet-based, n = 2,113); clinical panel (Internet-based, n = 553); and clinical outpatient (clinic-based, n = 581). All short forms are expressed as T scores with a mean of 50 and SD of 10. Results: Internal consistency (Cronbach α) of the 13 short forms ranged from 0.85 to 0.97. Correlations between short form and full-length item bank scores ranged from 0.88 to 0.99 (0.82–0.96 after removing common items from banks). Online respondents were asked whether they had any of 19 different chronic health conditions, and whether or not those reported conditions interfered with ability to function normally. All short forms, across physical, mental, and social health, were able to separate people who reported no health condition from those who reported 1–2 or 3 or more. In addition, scores on all 13 domains were worse for people who acknowledged being limited by the health conditions they reported, compared to those who reported conditions but were not limited by them. Conclusion: These 13 brief measures of self-reported QOL are reliable and show preliminary evidence of concurrent validity inasmuch as they differentiate people based upon number of reported health conditions and whether those reported conditions impede normal function.

Stress and symptoms in patients with interstitial cystitis: a life stress model.

Objectives. Stress-related exacerbation of interstitial cystitis (IC) symptoms has frequently been reported. Previous research has found stress-related IC symptom exacerbation in an experimental model. However, this relationship has not been objectively examined with daily life stressors. We used a prospective daily symptom diary method to investigate the relationships among stress and bladder symptoms in patients with IC and age-matched healthy controls.Methods. Forty-five previously diagnosed female patients with IC completed a bladder symptom and stress diary nightly for 1 month; 31 female age-matched healthy controls completed a similar diary for 7 days. The symptom questions were modified from the Interstitial Cystitis Data Base study.Results. Patients reported greater mean daily stress, bladder pain, urgency, and daytime and nocturnal frequency than controls (all P values less than 0.001). Among all patients, a significant relationship between stress and urgency was observed. In addition, a significant relationship between stress and pain was observed among patients with moderate and severe disease. As the disease severity increased, more pronounced relationships between stress and the symptoms of urgency and pain were evidenced. Greater stress was associated with greater nocturnal frequency among patients with more severe disease. These stress-symptom relationships were not observed among the controls.Conclusions. Higher levels of stress were related to greater pain and urgency in patients with IC but not in the controls. In addition, the relationship of stress and these IC symptoms was stronger among patients with more severe disease. The results indicate that life stress is associated with greater IC symptoms, particularly among patients whose disease is not well controlled.

The development of a clinical outcomes survey research application: Assessment CenterSM

IntroductionThe National Institutes of Health sponsored Patient-Reported Outcome Measurement Information System (PROMIS) aimed to create item banks and computerized adaptive tests (CATs) across multiple domains for individuals with a range of chronic diseases.PurposeWeb-based software was created to enable a researcher to create study-specific Websites that could administer PROMIS CATs and other instruments to research participants or clinical samples. This paper outlines the process used to develop a user-friendly, free, Web-based resource (Assessment CenterSM) for storage, retrieval, organization, sharing, and administration of patient-reported outcomes (PRO) instruments.MethodsJoint Application Design (JAD) sessions were conducted with representatives from numerous institutions in order to supply a general wish list of features. Use Cases were then written to ensure that end user expectations matched programmer specifications. Program development included daily programmer “scrum” sessions, weekly Usability Acceptability Testing (UAT) and continuous Quality Assurance (QA) activities pre- and post-release.ResultsAssessment Center includes features that promote instrument development including item histories, data management, and storage of statistical analysis results.ConclusionsThis case study of software development highlights the collection and incorporation of user input throughout the development process. Potential future applications of Assessment Center in clinical research are discussed.

Familial risk for common diseases in primary care: the Family Healthware Impact Trial.

CONTEXT Family history is a risk factor for many common chronic diseases, yet it remains underutilized in primary care practice. BACKGROUND Family Healthware is a self-administered, web-based tool that assesses familial risk for CHD; stroke; diabetes; and colorectal, breast, and ovarian cancer, and provides a personalized prevention plan based on familial risk. The Family Healthware Impact Trial evaluated the tool. DESIGN In this cluster RCT, participants completed baseline and 6-month follow-up surveys. The intervention group used Family Healthware directly after the baseline survey. Controls used the tool after completing the follow-up survey. SETTING/PARTICIPANTS Patients aged 35-65 years with no known diagnosis of these six diseases were enrolled from 41 primary care practices. MAIN OUTCOME MEASURES The prevalence of family-history-based risk for coronary heart disease (CHD); stroke; diabetes; and colorectal, breast, and ovarian cancer was determined in a primary care population. RESULTS From 2005 to 2007, 3786 participants enrolled. Data analysis was undertaken from September 2007 to March 2008. Participants had a mean age of 50.6 years and were primarily white (91%) women (70%). Of the 3585 participants who completed the risk assessment tool, 82% had a strong or moderate familial risk for at least one of the diseases: CHD (strong=33%, moderate=26%); stroke (strong=15%, moderate=34%); diabetes (strong=11%, moderate=26%); colorectal cancer (strong=3%, moderate=11%); breast cancer (strong=10%, moderate=12%); and ovarian cancer (strong=4%, moderate=6%). Women had a significantly (p<0.04) higher familial risk than men for all diseases except colorectal and ovarian cancer. Overweight participants were significantly (p<or=0.02) more likely to have a strong family history for CHD, stroke, and diabetes. Older participants were significantly (p<or=0.02) more likely to report a strong family history for CHD and stroke as well as colorectal and breast cancer. CONCLUSIONS This self-administered, online tool delineated a substantial burden of family-history-based risk for these chronic diseases in an adult, primary care population. TRIAL REGISTRATION NCT00164658.

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.

Comparison of risk perceptions and beliefs across common chronic diseases.

OBJECTIVES Few studies have compared perceptions of risk, worry, severity and control across multiple diseases. This paper examines how these perceptions vary for heart disease, diabetes, stroke, and colon, breast, and ovarian cancers. METHODS The data for this study came from the Family Healthware Impact Trial (FHITr), conducted in the United States from 2005 to 2007. Healthy adults (N=2362) from primary care practices recorded their perceptions at baseline for each disease. Analyses were conducted controlling for study site and personal risk factors. RESULTS Perceived risk was significantly higher for cancers than for other diseases. Men worried most about getting heart disease; women worried most about getting breast cancer, followed by heart disease. Diabetes was perceived to be the least severe condition. Heart disease was perceived to be the most controllable compared to cancers, which were perceived to be the least controllable. Women had higher perceived risk and worry ratings compared to men for several diseases. CONCLUSIONS These data highlight how individuals comparatively view chronic diseases. Addressing prior disease perceptions when communicating multiple disease risks may facilitate an accurate understanding of risk for diseases, and help individuals to effectively identify and engage in relevant behaviors to reduce their risk.