Patient experiences in ulcerative colitis: conceptual model and review of patient-reported outcome measures.

PURPOSE: To identify symptoms and their impacts on daily functioning and health-related quality of life (HRQoL) experienced by adult patients with ulcerative colitis (UC) and evaluate patient-reported outcome (PRO) measures for UC clinical studies. METHODS: A conceptual model of symptoms and impacts of UC were developed from a literature review. PRO measures were identified from the literature, clinical trials databases, health technology assessment submissions, and regulatory label claims, and were selected for conceptual analysis based on disease specificity and use across information sources. PRO measures covering the most concepts when mapped against the conceptual model were assessed for gaps in psychometric properties using Food and Drug Administration (FDA) guidance and consensus-based standards for the selection of health measurement instruments (COSMIN) criteria. RESULTS: The conceptual model grouped the 52 symptom concepts and 72 proximal and distal impacts into eight, two, and five dimensions, respectively. Of 65 PRO measures identified, eight underwent conceptual analysis. Measures covering the most concepts and assessed for psychometric properties were the Inflammatory Bowel Disease Questionnaire, Symptoms and Impacts Questionnaire for UC, UC-PRO symptoms modules, UC-PRO impact modules, and Crohn's and UC Questionnaire; all had good or excellent support for content validity. The UC-PRO Signs and Symptoms fully met FDA guidance and COSMIN criteria for content validity and most psychometric properties. CONCLUSION: Existing PRO measures assess concepts relevant to patients with UC, but all PRO measures reviewed require further psychometric evaluation to demonstrate they are fit for purpose.

Psychometric validation of the Psoriasis Symptom Scale, Functional Assessment of Chronic Illness Therapy-Fatigue and pain-Visual Analogue Scale in patients with generalized pustular psoriasis.

BACKGROUND: Generalized pustular psoriasis (GPP) is a rare, chronic, inflammatory skin disease associated with considerable patient burden. The Psoriasis Symptom Scale (PSS), Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) and pain-Visual Analogue Scale (pain-VAS) are patient-reported outcomes (PROs) that have not yet been validated in patients with GPP. OBJECTIVES: To evaluate the psychometric properties of the PSS, FACIT-Fatigue and pain-VAS using data from Effisayil 1, a randomised trial of spesolimab in patients with moderate-to-severe GPP. METHODS: Inter-item correlations and confirmatory factor analysis (CFA) were performed using Week 1 data. Internal consistency was assessed with Cronbach's α coefficient using baseline and Week 1 data. Test-retest reliability was assessed using intraclass correlation coefficients (ICCs); change data for the GPP Physician Global Assessment total score and pustulation subscore were used to define a stable population. Convergent validity was assessed at baseline and Week 1 using Spearman's rank-order correlations. Known-groups validity was measured by analysis of variance using Week 1 data. Ability to detect change from baseline to Week 1 was evaluated by analysis of covariance. RESULTS: Inter-item and item-to-total correlations were moderate or strong for most PSS and FACIT-Fatigue items. CFA demonstrated the unidimensionality of the PSS and FACIT-Fatigue, with high factor loadings for most items (PSS range, 0.75-0.94; FACIT-Fatigue range, 0.11-0.93) and acceptable fit statistics. Both scores demonstrated internal consistency (Cronbach's α, 0.71 and 0.95, respectively). The PSS, FACIT-Fatigue and pain-VAS demonstrated test-retest reliability (ICCs ≥0.70) and good evidence of convergent validity. Furthermore, the PROs could differentiate between known groups of varying symptom severity (range, p < 0.0001-0.0225) and detect changes in symptom severity from baseline to Week 1 (range, p < 0.0001-0.0002). CONCLUSIONS: Overall, these results support the reliability, validity and ability to detect change of the PSS, FACIT-Fatigue and pain-VAS as PROs in patients with GPP.

Psychometric validation of the generalized pustular psoriasis physician global assessment (GPPGA) and generalized pustular psoriasis area and severity index (GPPASI).

BACKGROUND: Generalized pustular psoriasis (GPP) is a rare and life-threatening skin disease often accompanied by systemic inflammation. There are currently no standardized or validated GPP-specific measures for assessing severity. OBJECTIVE: To evaluate the reliability, validity and responder definitions of the Generalized Pustular Psoriasis Physician Global Assessment (GPPGA) and Generalized Pustular Psoriasis Area and Severity Index (GPPASI). METHODS: The GPPGA and GPPASI were validated using outcome data from Week 1 of the Effisayil™ 1 study. The psychometric analyses performed included confirmatory factor analysis, item-to-item/item-to-total correlations, internal consistency reliability, test-retest reliability, convergent validity, known-groups validity, responsiveness analysis and responder definition analysis. RESULTS: Using data from this patient cohort (N = 53), confirmatory factor analysis demonstrated unidimensionality of the GPPGA total score (root mean square error of approximation <0.08), and GPPGA item-to-item and item-to-total correlations ranged from 0.58 to 0.90. The GPPGA total score, pustulation subscore and GPPASI total score all demonstrated good test-retest reliability (intraclass correlation coefficient: 0.70, 0.91 and 0.95 respectively), and good evidence of convergent validity. In anchor-based analyses, all three scores were able to detect changes in symptom and disease severity over time; reductions of -1.4, -2.2 and - 12.0 were suggested as clinically meaningful improvement thresholds for the GPPGA total score, GPPGA pustulation subscore and GPPASI total score respectively. Anchor-based analyses also supported the GPPASI 50 as a clinically meaningful threshold for improvement. CONCLUSIONS: Overall, our findings indicate that the GPPGA and GPPASI are valid, reliable and responsive measures for the assessment of GPP disease severity, and support their use in informing clinical endpoints in trials in GPP.

The Development of a Patient-Reported Outcome Measure for Assessment of Genital Psoriasis Symptoms: The Genital Psoriasis Symptoms Scale (GPSS).

INTRODUCTION: Patient-reported outcome measures (PROs) specific for genital psoriasis (GenPs) have not been described. METHODS: In this cross-sectional, qualitative study in patients with moderate-to-severe GenPs, we sought to develop a PRO useful for GenPs symptom assessment. A literature review was performed to identify relevant psoriasis or GenPs symptoms and existing PROs that may be useful in the evaluation of symptom severity in GenPs patients. The literature review findings were discussed with clinicians, and then patients with GenPs. RESULTS: Relevant psoriasis or GenPs symptoms from the literature review included itch, pain, scaling, redness/erythema, and stinging/burning. The validity of these symptoms for GenPs and potentially relevant PROs was corroborated by clinical experts. After gap analysis, a draft symptom scale consisting of Numeric Rating Scale (NRS) items was constructed. We then conducted interviews with GenPs patients (n = 20) to support content validity and use of the draft symptom NRS items in routine practice and in clinical trials. Participants identified and confirmed relevant symptoms and evaluated the utility of the draft PRO. A new PRO was developed: the Genital Psoriasis Symptoms Scale (GPSS). Cognitive debriefing and cultural adaptation/translation interviews with a second group of patients confirmed cultural appropriateness of the GPSS. CONCLUSION: The GPSS may be useful for assessing symptoms before, during, and after treatment in routine clinical practice and in clinical trials involving patients with GenPs. FUNDING: Eli Lilly & Company. Plain language summary available for this article.

Priorities of Municipal Policy Makers in Relation to Physical Activity and the Built Environment: A Latent Class Analysis.

OBJECTIVE: To examine policy makers' public policy priorities related to physical activity and the built environment, identify classes of policy makers based on priorities using latent class analysis, and assess factors associated with class membership. DESIGN: Cross-sectional survey data from municipal officials in 94 cities and towns across 6 US states were analyzed. PARTICIPANTS: Participants (N = 423) were elected or appointed municipal officials spanning public health, planning, transportation/public works, community and economic development, parks and recreation, and city management. MAIN OUTCOME MEASURES: Participants rated the importance of 11 policy areas (public health, physical activity, obesity, economic development, livability, climate change, air quality, natural resource conservation, traffic congestion, traffic safety, and needs of vulnerable populations) in their daily job responsibilities. Latent class analysis was used to determine response patterns and identify distinct classes based on officials' priorities. Logistic regression models assessed participant characteristics associated with class membership. RESULTS: Four classes of officials based on policy priorities emerged: (1) economic development and livability; (2) economic development and traffic concerns; (3) public health; and (4) general (all policy areas rated as highly important). Compared with class 4, officials in classes 1 and 3 were more likely to have a graduate degree, officials in class 2 were less likely to be in a public health job/department, and officials in class 3 were more likely to be in a public health job/department. CONCLUSIONS: Findings can guide public health professionals in framing discussions with policy makers to maximize physical activity potential of public policy initiatives, particularly economic development.

Short and precise patient self-assessment of heart failure symptoms using a computerized adaptive test.

BACKGROUND: Assessment of dyspnea, fatigue, and physical disability is fundamental to the monitoring of patients with heart failure (HF). A plethora of patient-reported measures exist, but most are too burdensome or imprecise to be useful in clinical practice. New techniques used for computer adaptive tests (CATs) may be able to address these problems. The purpose of this study was to build a CAT for patients with HF. METHODS AND RESULTS: Item banks of 74 queries ("items") were developed to assess self-reported physical disability, fatigue, and dyspnea. All queries were administered to 658 adults with HF to build 3 item banks. The resulting HF-CAT was administered to 100 patients with ancillary HF (New York Heart Association I, 11%; II, 53%; III and IV, 36%). In addition, the physical function and vitality domains of the SF-36 Health Survey questionnaire, an established shortness-of-breath scale, and the Minnesota Living with Heart Failure Questionnaire were applied. The HF-CAT assessment took 3:09±1:52 minutes to complete and score. All HF-CAT scales demonstrated good construct validity through high correlations with the corresponding SF-36 Health Survey physical function (r=-0.87), vitality (r=-0.85), and shortness-of-breath (r=0.84) scales. Simulation studies showed a more precise measurement of all HF-CAT scales over a larger range than comparable static tools. The HF-CAT scales identified significant differences between patients classified by the New York Heart Association symptom criteria, similar to the Minnesota Living with Heart Failure Questionnaire. CONCLUSIONS: A new CAT for patients with HF was built using modern psychometric methods. Initial results demonstrate its potential to increase the feasibility and precision of patient self-assessments of symptoms of HF with minimized respondent burden. CLINICAL TRIAL REGISTRATION- URL: http://www.projectreporter.nih.gov. Unique identifier: 1R43HL083622-01.

Health and role functioning: the use of focus groups in the development of an item bank.

BACKGROUND: Role functioning is an important part of health-related quality of life. However, assessment of role functioning is complicated by the wide definition of roles and by fluctuations in role participation across the life-span. The aim of this study is to explore variations in role functioning across the lifespan using qualitative approaches, to inform the development of a role functioning item bank and to pilot test sample items from the bank. METHODS: Eight focus groups were conducted with a convenience sample of 38 English-speaking adults recruited in Rhode Island. Participants were stratified by gender and four age groups. Focus groups were taped, transcribed, and analyzed for thematic content. RESULTS: Participants of all ages identified family roles as the most important. There was age variation in the importance of social life roles, with younger and older adults rating them as more important. Occupational roles were identified as important by younger and middle-aged participants. The potential of health problems to affect role participation was recognized. Participants found the sample items easy to understand, response options identical in meaning and preferred five response choices. CONCLUSIONS: Participants identified key aspects of role functioning and provided insights on their perception of the impact of health on their role participation. These results will inform item bank generation.

Development and preliminary testing of a computerized adaptive assessment of chronic pain.

UNLABELLED: The aim of this article is to report the development and preliminary testing of a prototype computerized adaptive test of chronic pain (CHRONIC PAIN-CAT) conducted in 2 stages: (1) evaluation of various item selection and stopping rules through real data-simulated administrations of CHRONIC PAIN-CAT; (2) a feasibility study of the actual prototype CHRONIC PAIN-CAT assessment system conducted in a pilot sample. Item calibrations developed from a US general population sample (N = 782) were used to program a pain severity and impact item bank (kappa = 45), and real data simulations were conducted to determine a CAT stopping rule. The CHRONIC PAIN-CAT was programmed on a tablet PC using QualityMetric's Dynamic Health Assessment (DYHNA) software and administered to a clinical sample of pain sufferers (n = 100). The CAT was completed in significantly less time than the static (full item bank) assessment (P < .001). On average, 5.6 items were dynamically administered by CAT to achieve a precise score. Scores estimated from the 2 assessments were highly correlated (r = .89), and both assessments discriminated across pain severity levels (P < .001, RV = .95). Patients' evaluations of the CHRONIC PAIN-CAT were favorable. PERSPECTIVE: This report demonstrates that the CHRONIC PAIN-CAT is feasible for administration in a clinic. The application has the potential to improve pain assessment and help clinicians manage chronic pain.