April N. Naegeli

The Worst Itch Numeric Rating Scale for patients with moderate to severe plaque psoriasis or psoriatic arthritis

Plaque psoriasis (PP) and psoriatic arthritis (PsA) are autoinflammatory chronic conditions associated with skin involvement. Pruritus, or itching, is a prevalent and bothersome symptom in patients with PP and is associated with reduced health‐related quality of life. The Worst Itch Numeric Rating Scale (WI‐NRS) has been developed as a simple, single item with which to assess the patient‐reported severity of this symptom at its most intense during the previous 24‐hour period. Qualitative research was undertaken to assess the content validity of the WI‐NRS. Patients with moderate to severe PP and patients with PsA were recruited from clinical sites in the USA. The qualitative research entailed two‐part interviews, which began with concept elicitation to gain understanding of patients’ experiences of itching, followed by cognitive debriefing of the WI‐NRS to assess the instruments understandability, clarity, and degree of appropriateness from the patients perspective. Twelve patients with PP and 22 with PsA participated in the study. Patients reported that itching was an important and relevant symptom of their psoriatic disease. The WI‐NRS was reported to be complete and easy to understand; the recall period was considered appropriate, the response scale was familiar, and, overall, the instrument was found to be appropriate for assessing itching severity. Patient responses support the content validity of the WI‐NRS. The psychometric properties of the tool will be evaluated in future studies.

Psychometric properties of the Itch Numeric Rating Scale in patients with moderate-to-severe plaque psoriasis

Itching is a profoundly distressing symptom for many patients with psoriasis, but it has not been rigorously studied using validated tools for this condition.

The patient experience with fatigue and content validity of a measure to assess fatigue severity: qualitative research in patients with ankylosing spondylitis (AS)

BackgroundAnkylosing spondylitis (AS) is an autoimmune disorder characterized by inflammation of the spine and large joints. Fatigue is a common symptom that many AS patients find significantly impacts their health-related quality of life. The Worst Fatigue – Numeric Rating Scale (WF-NRS) assesses the severity of this symptom during the previous 24-hour period. The objective of this study was to perform qualitative research to support the development and content validity of the WF-NRS.MethodsPatients with AS were recruited from clinical sites in the U.S. for a qualitative study which first entailed concept elicitation interviews to gain understanding of the patients’ experience with AS and fatigue. Subsequently, cognitive debriefing interviews were undertaken to assess the understandability, clarity, and appropriateness from the patient’s perspective, of the content of a measure of fatigue severity.ResultsThirteen patients with AS participated in concept elicitation interviews and cognitive debriefing of the Brief Fatigue Inventory (BFI) fatigue severity subscale. The WF-NRS was developed from the worst fatigue item of the BFI as patients generally reported it to be understandable and covered an important concept, the completion instructions were modified, but the response scale remained as it was familiar and readily completed, and the recall period was appropriate.ConclusionsPatient responses resulted in the development of and supported the content validity of the WF-NRS. Further quantitative evaluation of the WF-NRS is warranted in order to assess its psychometric properties and confirm its usefulness as a clinical trial tool.

The Contribution of Pretreatment Expectations and Expectation-Perception Difference to Change in Treatment Satisfaction and End Point Treatment Satisfaction in the Context of Initiation of Inhaled Insulin Therapy in Patients with Type 2 Diabetes

BACKGROUND This study explored relationships among pretreatment expectations, treatment perceptions, expectation-perception difference, change in diabetes treatment satisfaction, and end point diabetes treatment satisfaction in individuals with type 2 diabetes. METHODS Multivariate models were used to evaluate clinical and patient-reported outcomes (PRO) data from a randomized clinical trial comparing treatment algorithms for an inhaled insulin. PRO measures--Diabetes Symptom Checklist-Revised subscales, Well-Being Questionnaire 12, Diabetes Treatment Satisfaction Questionnaire, and Self-Efficacy Scale--were administered at baseline and end point. Expectations about Insulin Therapy (EITQ) and Perceptions of Insulin Therapy Questionnaires (PITQ) were administered at baseline and end point, respectively. Change scores for hemoglobin A1C (A1C), PRO (i.e., during-trial effects), and expectation-perception change were calculated. Perceptions, expectation-perception difference, and change in and end point treatment satisfaction served as dependent variables for statistical models. RESULTS Complete EITQ and PITQ data were available for 240 insulin-naive individuals with type 2 diabetes (mean age, 58 years; 61% male; 78% white; mean baseline A1C, 8.4%). Treatment perceptions were significantly (P < 0.01) more positive than expectations. After controlling for baseline patient assessments and during-trial effects, regression analyses showed that pretreatment expectations independently contributed to perceptions (beta = 0.18, P = 0.001) and expectation-perception change (beta = -0.71, P < 0.001). Acting as a suppressor variable, pretreatment expectations enhanced the ability of expectation-perception difference to predict both change in (beta = 0.39, P < 0.001) and end point (beta = 0.57, P < 0.001) diabetes treatment satisfaction. CONCLUSIONS Pretreatment expectations and expectation-perception difference play an important role in the amount of change that occurs in satisfaction with new treatment as compared to that of previous treatment, as well as satisfaction with new treatment.

Patients’ Perspectives on the Impact of Genital Psoriasis: A Qualitative Study

IntroductionPlaque psoriasis is a chronic skin disease where genital involvement is relatively common. Yet health care providers do not routinely evaluate psoriasis patients for genital involvement and patients do not readily initiate discussion of it.MethodsA qualitative study of 20 US patients with dermatologist-confirmed genital psoriasis (GenPs) and self-reported moderate-to-severe GenPs at screening was conducted to identify key GenPs symptoms and their impacts on health-related quality of life (HRQoL).ResultsPatients had a mean age of 45 years, 55% were female, and patients had high rates of current/recent moderate-to-severe overall (65%) and genital (70%) psoriasis. Patients reported the following GenPs symptoms: genital itch (100%), discomfort (100%), redness (95%), stinging/burning (95%), pain (85%), and scaling (75%). Genital itching (40%) and stinging/burning (40%) were the most bothersome symptoms. Impacts on sexual health included impaired sexual experience during sexual activity (80%), worsening of symptoms after sexual activity (80%), decreased frequency of sexual activity (80%), avoidance of sexual relationships (75%), and reduced sexual desire (55%). Negative effects on sexual experience encompassed physical effects such as mechanical friction, cracking, and pain as well as psychosocial effects such as embarrassment and feeling stigmatized. Males reported a higher burden of symptoms and sexual impacts. Other HRQoL impacts were on mood/emotion (95%), physical activities (70%), daily activities (60%), and relationships with friends and family (45%). These impacts significantly affected daily activities. Physical activities were affected by symptoms and flares, and increased sweat and friction worsened symptoms. Patients reported daily practices to control outcomes.ConclusionThe high level of reported symptoms and sexual and nonsexual impacts reflects the potential burden of moderate-to-severe GenPs. GenPs can impact many facets of HRQoL and providers should evaluate their patients for the presence of genital psoriasis and its impact on their quality of life.FundingEli Lilly and Company.Plain Language SummaryBackgroundPsoriasis is a skin disease that can cause itchy, raised red patches of skin. Currently, psoriasis cannot be cured but medicines can make the patches smaller or go away completely. The patches can occur anywhere on the body. Sometimes people get them in their genital area. However, people are sensitive about this area and may not tell their doctor. Their doctor may not look or ask either.What We DidWe interviewed 20 men and women who had moderate-to-severe genital psoriasis. We asked about their health-related quality of life, including their sex life.What We LearnedAll 20 people said they had symptoms of itching and discomfort in their genital area. Most people also had symptoms of redness, stinging or burning, pain, and scaling (flaky skin). Most people said symptoms affected their sex life. Sexual activity was less comfortable. People had sexual activity less often. Physical reasons, such as pain, bothered some people. Emotional reasons, such as being embarrassed, bothered other people more. People said the genital psoriasis affected how they felt. For example, it made them stressed, angry, or sad. Genital psoriasis made physical activities such as walking and running more uncomfortable for many people, especially when symptoms “acted up.” Sweating a lot, wearing tight underwear, or working a long day could make symptoms worse too. About half the people spent less time with their family and friends because of their symptoms. People also did things to try to reduce their symptoms. Some people wore loose clothes or soaked in a bathtub every night or after sex. Other people carried cream (to stop the itch) with them all the time.ConclusionOther people may not experience what these 20 people did. However, having genital psoriasis can significantly impact someone’s life. Patients and doctors should talk about it.

Psychometric validation of the Brief Pain Inventory-Short Form in patients with systemic lupus erythematosus in the United States:

This study evaluated the Brief Pain Inventory-Short Form (BPI-SF) in patients with moderate-to-severe systemic lupus erythematosus (SLE). Patients ≥18 years old who self-reported a physician diagnosis of SLE (confirmed by medical record review) and active SLE (Systemic Lupus Activity Questionnaire (SLAQ) score of ≥11) were included. The BPI-SF and Short Form Health Survey version 2 (SF-36v2) were administered electronically at baseline, week 2 and week 12. Psychometric properties of the BPI-SF were evaluated. Cronbach alphas were >0.9 for all BPI-SF items, domains and total score. Test-retest reliability correlations for responses between baseline and week 2 of the BPI-SF had intraclass correlation coefficients (ICCs) ≥0.5. The BPI-SF domains and total score were moderately positively correlated to the SLAQ score (r ≥ 0.4), but negatively correlated to the SF-36v2 bodily pain domain (r ≤ –0.6). The BPI-SF domains and total score were moderately negatively correlated to the SF-36v2 physical functioning domain and physical component summary (r ≤ –0.4), with low correlations between the BPI-SF severity domain and SF-36v2 mental component summary (r = −0.16). Assessment of pain, as measured by the BPI-SF, demonstrated validity and reliability in a sample of patients with moderate-to-severe SLE.

Test–Retest, Responsiveness, and Minimal Important Change of the Ability to Perform Physical Activities of Daily Living Questionnaire in Individuals with Type 2 Diabetes and Obesity

BACKGROUND The Ability to Perform Physical Activities of Daily Living Questionnaire (APPADL) measures the self-reported ability of individuals with type 2 diabetes mellitus (T2DM) and obesity to perform daily physical activities. The primary objective of this study was to estimate APPADL test-retest reliability, responsiveness, and minimal important change (MIC). SUBJECTS AND METHODS Study participants were individuals with T2DM and body mass index ≥30 kg/m(2) enrolled in clinical weight loss programs in the United States. Data were obtained for clinical measures, APPADL, and other patient-reported instruments. APPADL test-retest reliability was estimated with intraclass correlation coefficient. To estimate responsiveness in a subgroup of participants, baseline and 6-month data were analyzed using paired t test and calculation of responsiveness indices (e.g., effect size [ES]). To estimate MIC, both distribution-based and anchor-based methods were used. RESULTS Test-retest data for 106 study participants (mean age, 52 years; 69% female; 31% white; mean body mass index, 38 kg/m(2)) yielded an intraclass correlation coefficient of 0.91. In the subgroup (n = 40) used to estimate responsiveness, weight was significantly less at end point than at baseline (mean, 222.0 vs. 231.9 pounds; P < 0.001, ES = 0.24), and APPADL scores were significantly better than at baseline (mean, 77.0 vs. 70.8; P = 0.01, ES = 0.32). Results of distribution- and anchor-based methods to establish MIC suggest values of 6-14 points (0-100 scale). CONCLUSIONS The APPADL has demonstrated reliability and validity. In addition, it has demonstrated responsiveness to weight loss in individuals with T2DM and obesity, thereby making it a potentially valuable tool in the evaluation of weight loss interventions (e.g., antihyperglycemic medications that produce weight loss) targeted toward patients with T2DM.

A psychometric evaluation of the Diabetes Symptom Checklist-Revised (DSC-R) cognitive distress, fatigue, hyperglycemia, and hypoglycemia subscales in patients with type 1 and type 2 diabetes.

Objective To explore the use of Diabetes Symptom Checklist-Revised (DSC-R) Cognitive Distress, Fatigue, Hyperglycemia, and Hypoglycemia subscales as measures of acute diabetes-associated symptoms in patients with both type 1 and 2 diabetes. Research design and methods Our study was conducted in context of two international, multicenter, randomized clinical trials for inhaled insulin. Confirmatory factor analyses and assessments of reliability and construct validity were performed. Results Study participants were 371 patients with type 2 (56% male; mean age, 57 years) and 481 with type 1 diabetes (57% male, mean age, 40 years). In both populations a four-factor model was the best fit. Cronbach’s α ≥ 0.79 and intraclass correlation coefficient ≥ 0.63; subscales correlated (P ≤ 0.05) with measures of well-being and satisfaction (0.12 ≤ r ≤ 0.71). In patients with type 1 diabetes, three subscales correlated (P < 0.05) with A1C. Conclusions The psychometric properties of the DSC-R Cognitive Distress, Fatigue, Hyperglycemia, and Hypoglycemia suggest they may be utilized in clinical trials as reliable and valid measures of acute symptoms of diabetes.

The caregiver burden in lupus: findings from UNVEIL, a national online lupus survey in the United States.

Objectives: Lupus imposes a substantial burden on patients; however, little is known about its impact on those caring for patients with the disease. In this study, we examined the impact ‘caring for patients with lupus’ has on caregivers from their own perspective. Methods: UNVEIL was a one-time online national cross-sectional survey developed in partnership with the Lupus Foundation of America and fielded targeting the US Lupus Foundation of America constituents in 2014. Eligible caregivers were adults who self-identified as unpaid caregivers of patients with lupus. Eligible caregivers had to complete a series of sociodemographic questions as well as a series of well established outcome measures, such as the Short Form 12v2 Health Survey, the Work Productivity and Activity Index, the Caregiver Burden Inventory, and the Perceived Benefits of Caregiving Scale. Results: A total of 253 caregivers completed the survey. The majority of caregivers (90.1%) were aged 60 years or younger, more than half (54.2%) were men, and more than half (59.7%) identified themselves as either a spouse or a partner to the patient with lupus they were caring for. Overall health-related quality of life was close to the norm mean of the general US population. Caregivers who were employed missed an average of 12.8% of paid work time due to caregiving responsibilities and reported a 33.5% reduction in on-the-job effectiveness. Nearly half of the caregivers surveyed (49.4%) indicated that their caregiving responsibilities impacted their ability to socialize with friends, and almost all caregivers (97.6%) reported experiencing increased anxiety and stress in relation to their caregiving role. Conclusions: Caregiving for patients with lupus has a substantial impact on the work productivity and the social and emotional functioning of caregivers. Healthcare professionals and policymakers should continually assess the impact of healthcare decisions on the well-being of those caring for patients with lupus.

Interpreting change from patient reported outcome (PRO) endpoints: patient global ratings of concept versus patient global ratings of change, a case study among osteoporosis patients

BackgroundRegulatory guidance recommends anchor-based methods for interpretation of treatment effects measured by PRO endpoints. Methodological pros and cons of patient global ratings of change vs. patient global ratings of concept have been discussed but empirical evidence in support of either approach is lacking. This study evaluated the performance of patient global ratings of change and patient global ratings of concept for interpreting patient stability and patient improvement.MethodsPatient global ratings of change and patient global ratings of concept were included in a psychometric validation study of an osteoporosis-targeted PRO instrument (the OPAQ-PF) to assess its ability to detect change and to derive responder definitions. 144 female osteoporosis patients with (n = 37) or without (n = 107) a recent (within 6 weeks) fragility fracture completed the OPAQ-PF and global items at baseline, 2 weeks (no recent fracture), and 12 weeks (recent fracture) post-baseline.ResultsResults differed between the two methods. Recent fracture patients reported more improvement while patients without recent fracture reported more stability on ratings of change than ratings of concept. However, correlations with OPAQ-PF score change were stronger for ratings of concept than ratings of change (both groups). Effect sizes for OPAQ-PF score change increased consistently with level of change in ratings of concept but inconsistently with ratings of change, with the mean AUC for prediction of a one-point change being 0.72 vs. 0.56.ConclusionsThis study provides initial empirical support for methodological and regulatory recommendations to use patient global ratings of concept rather than ratings of change when interpreting change captured by PRO instruments in studies evaluating treatment effects. These findings warrant being confirmed in a purpose-designed larger scale analysis.