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Introduction The Insomnia Daytime Symptoms and Impacts Questionnaire (IDSIQ) is validated to measure daytime functioning in individuals with insomnia disorder. This study assessed the content validity of the IDSIQ through cognitive interviews in individuals with insomnia and comorbid nocturia. Methods This was a cross-sectional, qualitative observational study. Data were collected from 10 adult subjects from the US suffering from insomnia disorder (DSM-5) and comorbid nocturia (≥2 nocturnal voids). One-on-one, web-based interviews were conducted using semi-structured guides and lasted 60–90 minutes. Based on the stage of the study, all participants examined the content validity of the IDSIQ as part of 1) hybrid concept elicitation and cognitive interviews (n=5; Stage 1), or 2) cognitive interviews (n=5; Stage 2). Institutional review board approval and web-based informed consent were obtained prior to the patient interviews. Results The mean age of participants was 59.5 years (SD=4.3) and 62.2 years (SD=8.5) in the hybrid and cognitive interview groups, respectively. All individuals in the hybrid interviews were female, whereas all individuals in the cognitive interviews were male. Participants’ overall impressions were positive (10/10), describing the IDSIQ as straightforward (5/10), good (1/10), simple (1/10), very precise (1/10), very specific (1/10), clear (1/10), and aligned with their experience (3/10). The items were understood by all participants (10/10) except Item 1, which was understood by almost all participants (9/10). One participant suggested that the responses to questions on the IDSIQ may vary depending on the time the survey is administered, and the amount of sleep obtained the previous night. All 10 participants reported that they could answer all questions. Of the nine participants who were asked, all stated that the IDSIQ was relevant to their experience of their condition. Most participants reported that there were no missing items, whereas two participants stated that an item assessing the impact of insomnia on personal relationships (including family) was missing. Conclusion The content of the IDSIQ was easy to understand by patients that suffer from insomnia and comorbid nocturia. The study provides evidence for the content validity of the IDSIQ and its applicability in this population. Support (if any) Funded by Idorsia Pharmaceuticals Ltd.

To assess the reliability and validity of age-specific versions of the Fibrodysplasia Ossificans Progressiva Physical Function Questionnaire (FOP-PFQ), developed to measure the impact of FOP on physical function and activities of daily living.FOP-PFQ development included a literature review, two iterative phases of qualitative work involving individuals with FOP, and clinical expert review. The analysis used pooled FOP-PFQ data from an FOP natural history study (NCT02322255), a patient registry (NCT02745158), and phase II trials (NCT02190747; NCT02279095; NCT02979769). Item-level and factor analysis informed item retention and determined factor structure. Reliability was evaluated using Cronbach's alpha and intraclass correlation coefficients. Convergent validity was assessed by comparing scores with age, the Cumulative Analogue Joint Involvement Scale (CAJIS), the Patient-Reported Outcomes Measurement Information System Global Health Scale (PROMIS), and heterotopic ossification (HO) volume. Known-groups validity assessment used age, CAJIS, and HO volume.Factor analysis confirmed a two-factor solution: Mobility and Upper Extremity. Results reflected high internal consistency and were supportive of test-retest reliability; correlation coefficients0.90 demonstrated FOP-PFQ scores were stable over a one- to three-week period. The majority of scores were moderately (r = 0.30-0.50) to highly (r ≥ 0.50) correlated with CAJIS and HO volume, supporting convergent validity. With the exception of some age-based and functional groups, FOP-PFQ scores were significantly worse in groups with more severe disease, demonstrating known-groups validity.The FOP-PFQ was demonstrated to be a reliable, valid measure that may be responsive to change in individuals with FOP, although some results were inconclusive for pediatric versions.

Geographic atrophy (GA) is an advanced form of age-related macular degeneration characterized by progressive, irreversible visual function loss. This analysis evaluates the psychometric properties of the 25-Item National Eye Institute Visual Function Questionnaire (NEI VFQ-25) composite, near activity, and distance activity scores in patients with GA.Reliability and validity study.Reliability and validity were tested with NEI VFQ-25 data collected from 100 subjects with GA from United States' sites of the phase 2 Mahalo study of lampalizumab (ClinicalTrials.gov identifier: NCT01229215).Strong internal consistency and reproducibility were demonstrated for the NEI VFQ-25 composite (Cronbach's α, 0.95; intraclass correlation coefficient [ICC], 0.86), near activity (Cronbach's α, 0.84; ICC, 0.80), and distance activity (Cronbach's α, 0.84; ICC, 0.84) scores. Convergent validity with the binocular measures, Minnesota Low-Vision Reading Test (MNRead) reading speed and Functional Reading Independence (FRI) index score, was demonstrated for baseline NEI VFQ-25 composite (Pearson correlation [r] = 0.61 and 0.69, respectively), near activities (r = 0.69 and 0.73), and distance activities (r = 0.57 and 0.64) scores. Known-group validity testing for baseline mean NEI VFQ-25 scores (composite, near activities, and distance activities) showed differences between patients with mean maximum MNRead reading speed ≥ 80 vs80 words per minute, and between mean FRI index score ≥ 2.5 vs2.5 (all P.0001).Psychometric evidence supports the NEI VFQ-25 as a reliable and valid cross-sectional measure of the impact of GA on patient visual function and vision-related quality of life.

Purpose Content validity is the extent to which a patient-reported outcome measure evaluates the concepts most relevant to a patient's condition and treatment. The St George's Respiratory Questionnaire (SGRQ) has been validated in a range of respiratory conditions. This study evaluated the content validity of the SGRQ in patients with severe asthma. Methods A qualitative study, guided by a protocol, which included concept elicitation and cognitive debriefing of the SGRQ, was conducted in patients aged ≥18 years with history of severe asthma and blood eosinophil counts of ≥150/μL (past month) or ≥300/μL (past 12 months). Patients were recruited until saturation for concept elicitation was achieved (i.e. no additional concepts identified). Concepts identified by the patients were then mapped to the SGRQ. Results 18 patient interviews provided concept saturation. Concept elicitation confirmed that the SGRQ includes the commonly reported asthma symptoms and their impact on daily life. In total, 89–100% of patients routinely experienced cough, nighttime awakenings, shortness of breath, chest tightness, sleep difficulty, phlegm/mucus, and wheezing. Patients reported asthma impacting daily and physical activities, mood and sleep. Cognitive interviewing confirmed that patients understood the instructions, items and response options in the SGRQ. Nearly half of the concepts in the SGRQ were endorsed by ≥12 patients; of the 17 items with scoring weights ≥85, 11 were mentioned by ≥12 patients. Conclusions This study demonstrates that the SGRQ is a relevant, comprehensive and content-valid instrument to assess health status in patients with severe asthma.

Although multiple lines of evidence suggest a negative impact of secondary hyperparathyroidism on patients with kidney failure treated by hemodialysis, it is uncertain whether patients can detect associated symptoms. The objective was to determine whether changes in parathyroid hormone (PTH) levels are associated with changes in symptoms within this patient population.Prospective cohort.165 adults with hyperparathyroidism secondary to kidney failure diagnosed, a range of dialysis vintages, and receiving regular hemodialysis from a US single-provider organization.Change in PTH levels over 24 weeks.19 putative symptoms of secondary hyperparathyroidism measured up to 4 times using a self-administered questionnaire that assessed severity on a 5-level ordinal scale.Longitudinal associations between changes in PTH levels and symptom severity were assessed using generalized additive models.The 165 participants studied represented 81% of enrollees (N=204) who had sufficiently complete data for analysis. Mean age was 56 years and 54% were women. Increases in PTH levels over time were associated (P0.1) with worsening of bone aches and stiffness, joint aches, muscle soreness, overall pain, itchy skin, and tiredness, and the effects were more pronounced with larger changes in PTH levels.Findings may have been influenced by confounding by unmeasured comorbid conditions, concomitant medications, and multiple testing coupled with a P value threshold of 0.10.In this exploratory study, we observed that among patients with secondary hyperparathyroidism, increases in PTH levels over time were associated with worsening of 1 or more cluster of symptoms. Replication of these findings in other populations is needed before concluding about the magnitude and shape of these associations. If replicated, these findings could inform clinically useful approaches for measuring patient-reported outcomes related to secondary hyperparathyroidism.

IMPORTANCE: There is an unmet need for psychometrically sound instruments to measure pruritus associated with prurigo nodularis (PN). OBJECTIVE: To evaluate the psychometric properties of the itch numeric rating scale (itch NRS), both the Worst Itch Numeric Rating Scale (WI-NRS) and the Average Itch Numeric Rating Scale (AI-NRS). DESIGN, SETTING, AND PARTICIPANTS: This secondary analysis is based on a secondary end point of a phase 2 randomized clinical trial of serlopitant for treatment of pruritus associated with PN. This randomized, double-blind, placebo-controlled study was conducted at 15 sites in Germany. Eligible patients were aged 18 to 80 years and had generalized PN for more than 6 weeks that was refractory to previous antipruritic therapies. Patients were required to have a visual analog scale itch score of 7 or higher at screening. Data were collected from July 2014 to June 2016 and analyzed from June 2016 to January 2017. MAIN OUTCOMES AND MEASURES: The itch NRS (AI-NRS and WI-NRS) was correlated together with the following measures: the electronic verbal rating scale (eVRS) for itch self-categorization, average itch visual analog scale (AI-VAS), worst itch visual analog scale (WI-VAS), the pruritus-specific quality-of-life rating instrument ItchyQoL, Dermatology Life Quality Index (DLQI), and Prurigo Activity and Severity Score (items 7b and 7a: percentage healed prurigo lesions and percentage of prurigo lesions with excoriations). RESULTS: There were 123 participants in this study; the mean (SD) age of participants was 57.3 (11.58) years, and 58 (47.2%) were male. Strong associations (r ≥ 0.5) were observed between itch NRS items (WI-NRS and AI-NRS) and AI-VAS (24 hours) at weeks 2, 4, and 8 (r = 0.72-0.90; P

BACKGROUND Shared decision making (SDM) is a process in which health care providers and patients relate to and influence each other as they collaborate in making decisions about patients’ health care. Hypothesized as a means to improve quality of care, successful applications of SDM in routine cancer care have not been widely documented. OBJECTIVE The objective of this study was to examine the literature to determine if elements of SDM implementation between cancer patients and their clinicians were more or less successful at improving the quality of care and health outcomes. METHODS A systematic literature search of SDM approaches and outcomes in cancer care was conducted using PubMed and EMBASE. An integrative model for SDM was used to classify elements included in SDM intervention studies and the resulting outcomes. RESULTS From 1,018 unique publications, 23 articles meeting eligibility criteria were included. Only three studies addressed elements of patient-clinician interaction as part of the study objectives. Interventions included decision aid (DA) evaluation (n=22) and clinician communication training (n=1). SDM elements commonly included were: defining/explaining the problem (n=23); presenting options (n=19); discussing pros and cons (n=17); assessing patient priorities and preferences (n=17); clinician knowledge and recommendations (n=15); and making or deferring treatment decisions (n=12). The most frequently-measured outcomes were patient-reported outcomes including treatment preference or decision (n=12), decisional conflict (n=10), patient satisfaction (n=10), patient participation (n=9), and patient knowledge (n=7). No clear patterns demonstrating relationships between SDM elements and outcomes were identified. Information on how patients and clinicians utilized DA information to promote SDM was limited. CONCLUSIONS Evaluation of SDM in cancer care has been increasing. However, the term “SDM” was generally applied to studies that focused on the development and/or evaluation of DAs which limited the current analyses to a review of SDM elements as part of the DAs. Most studies did not include a qualitative or quantitative measure of SDM specific to patient-clinician communication and interaction. Instead, there was an underlying assumption that SDM occurred organically with DA implementation. Without a qualitative or quantitative measure of SDM, identification of successful SDM elements and their relationships to patient outcomes remains unclear. Additional research is warranted on SDM implementation and measurement in real-world cancer care settings.

ObjectiveThe objective was to evaluate the psychometric properties of the Rheumatoid Arthritis-Work Instability Scale (RA-WIS) in a clinical trial setting.MethodsSecondary analyses were conducted using data from a 56-week, randomized controlled trial of patients with early rheumatoid arthritis (RA). Patient-reported outcome measures included the RA-WIS, the Health Assessment Questionnaire (HAQ), the Rheumatoid Arthritis Quality of Life Questionnaire, and the Global Assessment of Disease Activity and Pain, data for which were collected at baseline and at weeks 12, 16, 24, and 56. Data were analyzed for reliability, validity, and responsiveness.ResultsAmong 148 patients whose data were analyzed, more than half were women (56.1%) with a mean age of 46.8 years. On average, patients experienced RA symptoms for 8.7 months; the mean 28-Joint Disease Activity Score (DAS28) was 5.9, and the mean HAQ – Disability Index was 1.3. The RA-WIS demonstrated excellent internal consistency and test-retest reliability (α = 0.89 and intraclass correlation coefficient = 0.91, respectively). At baseline and week 24, moderate to strong correlations were seen between RA-WIS total scores and the HAQ, the Global Assessment of Disease Activity, and the Pain Rheumatoid Arthritis Quality of Life Questionnaire, ranging from 0.47 to 0.81 (all P < 0.0001). Mean RA-WIS total scores and work disability risk levels discriminated between clinical severity scores on the DAS28, the HAQ – Disability Index, and the Physician Global Assessment of Disease Activity (all P < 0.05). Mean baseline to week 24 RA-WIS total change scores were significantly different among American College of Rheumatology responder groups (P ≤ 0.0001) and between DAS28 remission status groups (P < 0.001).ConclusionsThese findings provide evidence supporting the reliability, validity, and responsiveness of the RA-WIS for evaluating work disability in patients with RA in a clinical trial setting.

Summary Objectives There is increasing evidence that physical activity (PA) can affect health outcomes, particularly in chronic disease. While pharmacologic therapy and exercise training can improve exercise capacity, increasing PA requires behavior change. This review examined clinical trials testing the effectiveness of behavioral interventions to increase PA in adults with chronic disease to inform future research in COPD. Methods Embase and PubMed searches of studies published in English, 1995–2011. Inclusion criteria: Adults ≥45 years; COPD, diabetes, heart failure, obesity; exercise or PA endpoint; behavioral intervention described in sufficient detail to permit interpretation. Results 932 abstracts screened; 169 articles retrieved; 36 reviewed. Most were randomized trials ( n = 32, 89%); 2 arms ( n = 26, 72%), sample sizes 40–100 ( n = 15, 42%); recruitment through clinical settings ( n = 28, 78%); disease severity as primary eligibility criterion ( n = 23, 64%); mean duration: 10 months (range: 1–84). Exercise intervention: aerobic activity, 30–60 min ( n = 20, 56%), 3–5 times/week ( n = 20, 56%). Behavioral intervention: Counseling ( n = 19, 53%) with personal follow-up ( n = 12, 33%). Control group: Exercise without behavioral intervention ( n = 14, 39%) or usual care ( n = 15, 42%). Significant effects were reported in 15 of 25 (60%) studies testing exercise capacity (6-minute walk, cycle, treadmill), 19 of 26 (73%) testing PA (pedometer, activity log, questionnaire), 11 of 22 (50%) measuring quality of life, and 8 of 13 (62%) capturing behavioral endpoints. Conclusions This review provides insight into the range of designs, interventions, and outcome measures used in studies testing methods to improve PA in chronic disease with implications for designing trials in COPD.

Purpose To develop and validate the Functional Reading Independence (FRI) Index, a new patient-reported outcome measure assessing reading activities in individuals with geographic atrophy (GA) due to age-related macular degeneration. Methods The Index was developed through expert consultation and qualitative patient interviews. Reliability, validity, and responsiveness were tested with data from the Mahalo study (NCT01229215) of lampalizumab in patients with GA. Results Qualitative interviews (n = 40) yielded a 10-item FRI Index, which was refined to seven items in quantitative testing (n = 100). Strong internal consistency (marginal reliability = 0.90) and reproducibility (intraclass correlation coefficient = 0.86) were shown. Known-group validity testing for baseline mean FRI Index scores showed differences (mean [SD]) between patients with Minnesota Low-Vision Reading test reading speed ≥80 vs.

The effect of adalimumab on patient-reported outcomes (PROs) was evaluated in patients with moderate to severe psoriasis during the initial 16-week, double-blind period of a 52-week, Phase III, multicenter trial.Patients were randomized to placebo or adalimumab 80 mg at Week 0 and 40 mg every other week from Week 1 to Week 15. PROs were evaluated throughout the study and included the Dermatology Life Quality Index (DLQI), the Short Form 36 Health Survey (SF-36), the Work Productivity and Activity Impairment Questionnaire-Specific Health Problem (WPAI-SHP), and several patient-rated symptom scales.The adalimumab-treated group reported significantly greater improvements in DLQI total score (p0.001), SF-36 Physical Component Summary score (p0.001), and Mental Component Summary score (p0.001) compared with the placebo-treated group over 16 weeks. Significant differences, favoring adalimumab, were also seen for the DLQI subscale scores (p0.001); SF-36 scale scores (p0.001); WPAI-SHP work impairment (p0.001), activity limitation (p0.001), and overall work impairment scores (p0.001); patient's global assessment of disease severity (p0.001), psoriasis pain (p0.001), and psoriasis-related pruritus (p = 0.002).Adalimumab was efficacious in improving dermatology-specific and general health-related quality of life, work and activity limitations, and psoriasis-related symptoms in patients with moderate to severe psoriasis over a 16-week period.

Objectives Anemia, defined as having low levels of hemoglobin (HGB), is caused by disease-related (e.g., bone marrow suppression, nutritional deficiency) or treatment-related (e.g., chemotherapy, antiretroviral therapy) factors. Although epoetin alfa has been shown to improve HGB outcomes in cancer, HIV/AIDS, and chronic kidney disease (CKD), these results have been viewed in isolation, rather than across populations. The purpose of this article is to review findings from trials that evaluated the impact of epoetin alfa on HGB and health-related quality of life (HRQL) across various populations with different underlying causes of anemia. Methods A review of clinical trials published in English between January 1993 and September 2005. Searches were conducted using MEDLINE and EMBASE. Between- and within-group changes in HGB and HRQL were examined. Results One hundred ten articles were retrieved and 18 were reviewed. Statistically significant improvements in HGB were generally seen (1) between groups for cancer patients receiving epoetin alfa compared with those receiving placebo or standard of care (SOC) (between-group differences in changes from baseline to end point ranging from 1.2 to 1.9 g/dl); and (2) within groups for HIV/AIDS and CKD patients receiving epoetin alfa (changes from baseline to end point of 2.5 and 2.9 g/dl and 2.7 g/dl, respectively). Statistically and clinically significant improvements in HRQL, particularly with regard to fatigue, were seen across chronic conditions based on the Linear Analog Scale Assessment energy scale; where improvements of at least 8 mm—considered clinically relevant—were generally seen (1) between groups for cancer patients receiving epoetin alfa compared with those receiving placebo or SOC (differences in changes from baseline to end point from 0.8 to 19.8 mm); and (2) within groups for HIV/AIDS and CKD patients receiving epoetin alfa (changes from baseline to end point of 23 and 25 mm and 28 mm, respectively). Conclusions Results of published clinical trials suggest that treatment of anemia associated with cancer, HIV/AIDS and CKD can have a significant impact on HRQL, particularly fatigue, and that this impact is both statistically and clinically significant.

To compare the effect of adalimumab plus methotrexate (MTX) versus MTX monotherapy on health-related quality of life (HRQOL) and work activities in patients with early rheumatoid arthritis (RA).Patients in this PREMIER study subanalysis (n = 525) were randomized to adalimumab 40 mg every other week plus MTX or MTX monotherapy. Medical Outcome Study Short-Form 36 Health Survey (SF-36) scores of RA patients were compared with US population norms at Weeks 12, 52, and 104.Physical Component Summary (PCS) scores at Week 12 for both groups improved from baseline and were significantly lower than US population scores (43.5 combination, 39.4 MTX, 49.4 US norm; p0.001). At Week 52, PCS score for adalimumab plus MTX was similar to that of the US population (47.5 vs 48.3; p = 0.25), while the PCS score for MTX was not similar to that of the US population (44.2 vs 48.3; p0.001). Criterion- and content-based interpretations for between-treatment differences in PCS scores suggest that those receiving combination therapy had fewer employment difficulties than those receiving MTX.After 2 years, HRQOL for patients with early RA treated with adalimumab plus MTX improved to US norms. Combination therapy had reduced the influence of RA on work activity.

Objective.—To evaluate the psychometric properties of the revised Patient Perception of Migraine Questionnaire (PPMQ-R), which measures satisfaction with migraine treatment. Background.—The original version of the PPMQ was developed prior to the introduction of newer migraine treatments, which may have attributes that influence treatment satisfaction. Methods.—Literature review, patient focus groups, and one-on-one patient interviews guided revisions to the original PPMQ. The revised questionnaire was tested in 200 migraine patients visiting neurologists and primary care clinics. At baseline, all subjects completed the PPMQ-R, a migraine-specific quality-of-life measure, and a migraine experience questionnaire; and 125 subjects completed assessments 24 hours after each migraine attack and within 30 days post-baseline. Factor analysis was performed to inform the development of a scoring algorithm, and reliability, validity, and responsiveness of the PPMQ-R were evaluated. Results.—Factor analyses confirmed that the revised questionnaire has five domains measuring satisfaction with efficacy, functionality, ease of use, medication cost, and bothersomeness of medication side effects. A total score can be created by taking the average of efficacy, functionality, and ease of use scores. The PPMQ-R scale scores and Total score demonstrated internal consistency reliability (Cronbach's α: 0.80 to 0.98) and test–retest reliability (intra-class correlation coefficient: 0.79 to 0.91). Except for the Cost domain, the PPMQ-R scores discriminated among migraine pain severity levels (all P < .05) and levels of impairment in ability to work and perform usual activities (all P < .05). Except for the Ease of Use and Cost domains, mean PPMQ-R scores were significantly higher among patients with no change in treatment and whose pain improved between two consecutive migraine attacks (all P < .01). Conclusion.—The PPMQ-R is a reliable and valid measure of patient satisfaction with acute migraine treatment in women with frequent migraine attacks.

Objective To develop a questionnaire to evaluate preferences for attributes of intranasal corticosteroids (INSs) in clinical trials with allergic rhinitis (AR) patients. Study design and setting Established questionnaire development practices were used, including performance of a literature review and use of patient and physician focus groups, cognitive debriefing interviews, and pilot testing before validation. Results Findings from patient and physician focus groups suggest that sensory attributes are relevant to AR patients when choosing INSs. Physician focus groups identified the need for 2 distinct preference instruments, a clinical trial patient preference questionnaire (CTPPQ) and a clinical practice preference questionnaire (CPPPQ). A pilot study suggests that the CTPPQ is capable of discriminating between 2 INSs in the clinical trial setting. Conclusions Initial findings suggest that items in the CTPPQ and CPPPQ are easy to understand and relevant to patients. Further validation studies with larger sample sizes are needed to assess the psychometric properties of both questionnaires. EBM rating: B-20.

Treatment satisfaction is used to capture the full impact of disease on patients' lives. Currently, no instruments exist to evaluate satisfaction with pharmacologic therapy in patients with Crohn's disease (CD). The purpose of this study was to evaluate the psychometric properties of a treatment satisfaction questionnaire for CD (TSQ-C). The 36-item questionnaire was completed by CD patients who reported taking 5-aminosalicylic acid derivatives to treat their CD. Measures used in the validation study were the Inflammatory Bowel Disease Questionnaire (IBDQ), Crohn's Work Activity Impairment Index (CWAII), and patient reports of clinical indicators (e.g., number of active flares and medications taken). Exploratory factor analysis was used to evaluate the items and subscale structure. Internal consistency reliability and concurrent and discriminant validity were assessed using Cronbach's alpha, Pearson correlation coefficients, and analysis of variance. A total of 813 CD patients participated, with the majority being Caucasian (95.9%), female (67.0%), and >34 years old (86.1%). Patient-rated severity of CD was mild (49.3%), moderate (41.7%), and severe (7.5%). The final TSQ-C consisted of 32 items, with six subscales (Symptoms, Satisfaction, Expectations, Physician Relationships, Bother, and Cost), with each subscale score ranging from 1 to 6. Cronbach's alpha values ranged from 0.63 (Cost) to 0.94 (Symptoms). Strong correlations were observed among the IBDQ, CWAII, and the Satisfaction and Symptoms subscales of the TSQ-C. TSQ-C subscales, particularly Symptoms and Satisfaction, significantly discriminated among levels of number of flares per year, patient-rated disease severity, and number of medication classes. The TSQ-C demonstrated excellent validity and reliability and appears to be a useful tool for evaluating satisfaction with pharmacologic therapy among patients with CD.

Objective To evaluate the impact of adalimumab on health-related quality of life (HRQOL) for patients with moderate to severe plaque psoriasis. Background Psoriasis is a chronic, inflammatory, immune-mediated disease that has a significant impact on patients' HRQOL. Adalimumab is a fully human monoclonal antibody that blocks tumor necrosis factor, a pro-inflammatory cytokine, and is effective and well-tolerated for patients with moderate to severe psoriasis. Methods Data were obtained for a secondary analysis of patients in a randomized, controlled Phase III trial evaluating the effect of adalimumab in patients with psoriasis (N = 1,205). Patients with moderate to severe psoriasis were randomized in a 2:1 ratio to adalimumab 80 mg (two 40 mg injections administered subcutaneously at baseline followed by one 40 mg injection every other week from Week 1 to Week 15) or placebo. Short Form-36 (SF-36) Health Survey scores of psoriasis patients were used to assess HRQOL and were compared with United States (US) population norms at baseline and Week 16. Results Baseline Physical Component Summary (PCS) scores for the placebo and adalimumab groups were similar to the general US population. Baseline mean Mental Component Summary (MCS) scores were significantly lower for the adalimumab and placebo groups compared with the general population (47.4, 47.7, and 50.8 points, respectively; p < 0.0001). PCS scores at Week 16 for patients receiving adalimumab had improved and were significantly greater than scores for the general US population (52.7 vs 48.9; p < 0.001). Compared with the general US population, MCS scores at Week 16 were similar for patients receiving adalimumab (51.2 vs 50.8; p = 1.000) and lower for patients receiving placebo (50.8 vs 48.7; p < 0.0001). Conclusion Psoriasis has a broad impact on patient functioning and well-being. Improvement in skin lesions and joint symptoms associated with adalimumab treatment was accompanied by improvements in HRQOL to levels that were similar to or greater than those of the general US population. Trial registration Clinicaltrials.gov NCT00237887

Purpose To examine the psychometric properties of the Injection Pen Assessment Questionnaire (IPAQ) including the following: 1) item and scale characteristics (e.g., frequencies, item distributions, and factor structure), 2) reliability, and 3) validity. Methods Focus groups and one-on-one dyad interviews guided the development of the IPAQ. The IPAQ was subsequently tested in 136 parent–child dyads in a Phase 3, 2-month, open-label, multicenter trial for a new Genotropin® disposable pen. Factor analysis was performed to inform the development of a scoring algorithm, and reliability and validity of the IPAQ were evaluated using the data from this two months study. Psychometric analyses were conducted separately for each injection pen. Results Confirmatory factor analysis provides evidence supporting a second order factor solution for four subscales and a total IPAQ score. These factor analysis results support the conceptual framework developed from previous qualitative research in patient dyads using the reusable pen. However, the IPAQ subscales did not consistently meet acceptable internal consistency reliability for some group level comparisons. Cronbach’s alphas for the total IPAQ score for both pens were 0.85, exceeding acceptable levels of reliability for group comparisons. Conclusions The total IPAQ score is a useful measure for evaluating ease of use and preference for injection pens in clinical trials among patient dyads receiving hGH. The psychometric properties of the individual subscales, mainly the lower internal consistency reliability of some of the subscales and the predictive validity findings, do not support the use of subscale scores alone as a primary endpoint.