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1.  The relationships between asthma control, daytime sleepiness, and quality of life among children with asthma: a path analysis 
Sleep medicine  2013;14(7):641-647.
Objectives
We aimed to examine the relationships between asthma control, daytime sleepiness, and asthma-specific health-related quality of life (HRQOL) among children with asthma. Path analyses were conducted to test if daytime sleepiness can mediate the effect of asthma control status on asthma-specific HRQOL.
Methods
160 dyads of asthmatic children and their parents were collected for analyses. The Asthma Control and Communication Instrument (ACCI) was used to categorize adequate and poor asthma control status. The Cleveland Adolescent Sleepiness Questionnaire (CASQ) was used to measure children’s daytime sleepiness, including sleep in school, awake in school, sleep in evening, and sleep during transport. The Patient-Reported Outcomes Measurement Information System (PROMIS) Asthma Impact Scale was used to measure asthma-specific HRQOL.
Results
Poorly controlled asthma was associated with daytime sleepiness and impaired asthma-specific HRQOL. Asthma control status was directly associated with asthma-specific HRQOL (P<.05), whereas sleep in school and sleep in evening domains of daytime sleepiness significantly mediated the relationship between poor asthma control and impaired HRQOL (P<.01).
Conclusions
Asthma control status was associated with pediatric asthma-specific HRQOL, and the association was significantly mediated by daytime sleepiness. Healthcare providers need to address pediatric sleep needs related to poor asthma control to reduce the impact on HRQOL.
doi:10.1016/j.sleep.2013.04.002
PMCID: PMC3715592  PMID: 23684939
Asthma control; children; health-related quality of life; patient-reported outcomes; sleepiness; structural equation modeling
2.  Measurement invariance of the PROMIS pain interference item bank across community and clinical samples 
Purpose
This study examined the measurement invariance of responses to the patient-reported outcomes measurement information system (PROMIS) pain interference (PI) item bank. The original PROMIS calibration sample (Wave I) was augmented with a sample of persons recruited from the American Chronic Pain Association (ACPA) to increase the number of participants reporting higher levels of pain. Establishing measurement invariance of an item bank is essential for the valid interpretation of group differences in the latent concept being measured.
Methods
Multi-group confirmatory factor analysis (MG-CFA) was used to evaluate successive levels of measurement invariance: configural, metric, and scalar invariance.
Results
Support was found for configural and metric invariance of the PROMIS-PI, but not for scalar invariance.
Conclusions and recommendations
Based on our results of MG-CFA, we recommend retaining the original parameter estimates obtained by combining the community sample of Wave I and ACPA participants. Future studies should extend this study by examining measurement equivalence in an item response theory framework such as differential item functioning analysis.
doi:10.1007/s11136-012-0191-x
PMCID: PMC3873645  PMID: 22552604
Factor analysis; Pain interference; Pain measurement; Patient outcome measures; Psychometrics
3.  The case for an international patient-reported outcomes measurement information system (PROMIS®) initiative 
Patient-reported outcomes (PROs) play an increasingly important role in clinical practice and research. Modern psychometric methods such as item response theory (IRT) enable the creation of item banks that support fixed-length forms as well as computerized adaptive testing (CAT), often resulting in improved measurement precision and responsiveness. Here we describe and discuss the case for developing an international core set of PROs building from the US PROMIS® network.
PROMIS is a U.S.-based cooperative group of research sites and centers of excellence convened to develop and standardize PRO measures across studies and settings. If extended to a global collaboration, PROMIS has the potential to transform PRO measurement by creating a shared, unifying terminology and metric for reporting of common symptoms and functional life domains. Extending a common set of standardized PRO measures to the international community offers great potential for improving patient-centered research, clinical trials reporting, population monitoring, and health care worldwide. Benefits of such standardization include the possibility of: international syntheses (such as meta-analyses) of research findings; international population monitoring and policy development; health services administrators and planners access to relevant information on the populations they serve; better assessment and monitoring of patients by providers; and improved shared decision making.
The goal of the current PROMIS International initiative is to ensure that item banks are translated and culturally adapted for use in adults and children in as many countries as possible. The process includes 3 key steps: translation/cultural adaptation, calibration, and validation. A universal translation, an approach focusing on commonalities, rather than differences across versions developed in regions or countries speaking the same language, is proposed to ensure conceptual equivalence for all items. International item calibration using nationally representative samples of adults and children within countries is essential to demonstrate that all items possess expected strong measurement properties. Finally, it is important to demonstrate that the PROMIS measures are valid, reliable and responsive to change when used in an international context.
IRT item banking will allow for tailoring within countries and facilitate growth and evolution of PROs through contributions from the international measurement community. A number of opportunities and challenges of international development of PROs item banks are discussed.
doi:10.1186/1477-7525-11-210
PMCID: PMC3879205  PMID: 24359143
Patient-reported outcomes; Health-related quality of life research; Patients’ experiences; Questionnaires; Cross-cultural equivalence; Health information systems; Clinical decision making; Comparative effectiveness research; Patient empowerment; Cross-national comparisons
4.  Comparison of US Panel Vendors for Online Surveys 
Background
Despite the increasing use of panel surveys, little is known about the differences in data quality across panels.
Objective
The aim of this study was to characterize panel survey companies and their respondents based on (1) the timeliness of response by panelists, (2) the reliability of the demographic information they self-report, and (3) the generalizability of the characteristics of panelists to the US general population. A secondary objective was to highlight several issues to consider when selecting a panel vendor.
Methods
We recruited a sample of US adults from 7 panel vendors using identical quotas and online surveys. All vendors met prespecified inclusion criteria. Panels were compared on the basis of how long the respondents took to complete the survey from time of initial invitation. To validate respondent identity, this study examined the proportion of consented respondents who failed to meet the technical criteria, failed to complete the screener questions, and provided discordant responses. Finally, characteristics of the respondents were compared to US census data and to the characteristics of other panels.
Results
Across the 7 panel vendors, 2% to 9% of panelists responded within 2 days of invitation; however, approximately 20% of the respondents failed the screener, largely because of the discordance between self-reported birth date and the birth date in panel entry data. Although geographic characteristics largely agreed with US Census estimates, each sample underrepresented adults who did not graduate from high school and/or had annual incomes less than US $15,000. Except for 1 vendor, panel vendor samples overlapped one another by approximately 20% (ie, 1 in 5 respondents participated through 2 or more panel vendors).
Conclusions
The results of this head-to-head comparison provide potential benchmarks in panel quality. The issues to consider when selecting panel vendors include responsiveness, failure to maintain sociodemographic diversity and validated data, and potential overlap between panels.
doi:10.2196/jmir.2903
PMCID: PMC3869084  PMID: 24292159
survey methods; community surveys; sampling bias; selection bias; Internet; data sources
5.  Asthma Symptom Utility Index: Reliability, validity, responsiveness and the minimal important difference in adult asthma patients 
Background
The evaluation of asthma symptoms is a core outcome measure in asthma clinical research. The Asthma Symptom Utility Index (ASUI) was developed to assess frequency and severity of asthma symptoms. The psychometric properties of the ASUI are not well characterized and a minimal important difference (MID) is not established.
Objectives
We assessed the reliability, validity, and responsiveness to change of the ASUI in a population of adult asthma patients. We also sought to determine the MID for the ASUI.
Methods
Adult asthma patients (n = 1648) from two previously completed multicenter randomized trials were included. Demographic information, spirometry, ASUI scores, and other asthma questionnaire scores were obtained at baseline and during follow-up visits. Participants also kept a daily asthma diary.
Results
Internal consistency reliability of the ASUI was 0.74 (Cronbach’s alpha). Test-retest reliability was 0.76 (intra-class correlation). Construct validity was demonstrated by significant correlations between ASUI scores and Asthma Control Questionnaire (ACQ) scores (Spearman correlation r = −0.79, 95% CI [−0.85, −0.75], P<0.001) and Mini Asthma Quality of Life Questionnaire (Mini AQLQ) scores (r = 0.59, 95% CI [0.51, 0.61], P<0.001). Responsiveness to change was demonstrated, with significant differences between mean changes in ASUI score across groups of participants differing by 10% in the percent predicted FEV1 (P<0.001), and by 0.5 points in ACQ score (P < 0.001). Anchor-based methods and statistical methods support an MID for the ASUI of 0.09 points.
Conclusions
The ASUI is reliable, valid, and responsive to changes in asthma control over time. The MID of the ASUI (range of scores 0–1) is 0.09.
doi:10.1016/j.jaci.2012.07.058
PMCID: PMC3501248  PMID: 23026499
Asthma Symptom Utility Index; reliability; validity; responsiveness; minimal important difference
6.  Agreement was not found in adolescents’ quality of life rated by parents and adolescents 
Journal of clinical epidemiology  2008;62(3):10.1016/j.jclinepi.2008.06.012.
Objective
Limited evidence examines agreement between the ratings of adolescents’ health-related quality of life (HRQOL) by parents and adolescents especially accounting for measurement properties. This study aimed to test whether agreement exists between the dyads using a methodology of measurement invariance.
Study Design and Setting
We conducted a telephone survey to collect data from parents and their adolescents enrolled in Florida’s Children’s Medical Services (376 pairs) using the PedsQL 4.0. We used multi-group confirmatory factor analysis to test measurement invariance, including configural (equivalent HRQOL construct), metric (equivalent item–domain associations), scalar (equivalent starting value of ratings), and residual (equivalent nonsystematic measurement errors of ratings) invariance. We also investigated correlates of discrepancies in the dyadic ratings.
Results
There were equivalent HRQOL constructs and item–domain associations between the dyads. However, some items show different starting values and nonsystematic errors in the dyadic ratings. After adjusting for noninvariant items, adolescents reported significant higher HRQOL scores than parents in all domains (P < 0.05). Parents’ rating of adolescents’ health significantly contributed to discrepancies in the dyadic ratings (P < 0.05).
Conclusions
Adolescents rated HRQOL higher than their parents. This discrepancy was associated with severe health conditions. Without assuring measurement invariance, comparisons of the dyadic HRQOL ratings can be misleading.
doi:10.1016/j.jclinepi.2008.06.012
PMCID: PMC3812676  PMID: 18834712
Adolescent; Confirmatory factor analysis; Health-related quality of life; Health status; Proxy; Measurement invariance
7.  Using three legacy measures to develop a health-related quality of life tool for young adult survivors of childhood cancer 
Background
Little attention has been paid to selecting and developing health-related quality of life (HRQOL) measurement tools for young adult survivors of childhood cancer (YASCC). The primary purpose of this study was to develop a HRQOL tool for YASCC based on three legacy instruments.
Methods
Data collected from 151 YASCC were analyzed. HRQOL was measured using the Medical Outcomes Study SF-36, Quality of Life in Adult Cancer Survivors, and Quality of Life-Cancer Survivor. We used the following stages to develop our HRQOL tool: mapping items from three instruments into a common HRQOL construct, checking dimensionality using confirmatory factor analyses (CFA), and equating items using Rasch modeling.
Results
We assigned 123 items to a HRQOL construct comprised of six generic and eight survivor-specific domains. CFA retained 107 items that meet the assumptions of unidimensionality and local independence. Rasch analysis retained 68 items that satisfied the indices of information-weighted/outlier-sensitive fit statistic mean square. However, items in most domains possess relatively easy measurement properties, whereas YASCC’s underlying HRQOL was on the middle to higher levels.
Conclusions
Psychometric properties of the established tool for measuring HRQOL of YASCC were not satisfied. Future studies need to refine this tool, especially adding more challenging items.
doi:10.1007/s11136-011-0055-9
PMCID: PMC3328688  PMID: 22101901
Childhood cancer; measurement; quality of life; Rasch analysis; young adult survivor
8.  Determining the clinically important difference in visual analog scale scores in abuse liability studies evaluating novel opioid formulations 
Purpose
This study determined how the magnitude of change in positive subjective responses predicts clinical outcome in a treatment setting. Specifically, we attempted to define what constitutes a clinically important difference (CID) in subjective responses.
Methods
A 100-mm visual analog scale (VAS) measured subjective ratings of drug “high,” calculated via an anchor-based method with published data from participants receiving sustained-release naltrexone (NTX) and heroin in a laboratory setting. The data were then compared to clinical outcomes in a treatment trial with sustained-release naltrexone. A distribution-based method subsequently analyzed data from participants who received ALO-01 (extended-release morphine with sequestered NTX) to predict its abuse liability.
Results
Differences in ratings of drug high of approximately 10 mm on a 100-mm line were clinically significant. By extrapolation, CIDs were also found between crushed or intact ALO-01 and immediate-release morphine sulfate (IRMS). No CIDs were found between intact and crushed ALO-01.
Conclusions
From laboratory and treatment trial data involving naltrexone, calculation of CIDs in subjective ratings of high is possible. Consequently, crushing/swallowing or injecting ALO-01 produces clinically significantly less drug high than oral or intravenous morphine alone, suggesting that ALO-01 has lower abuse liability by those routes than morphine formulations.
doi:10.1007/s11136-011-0012-7
PMCID: PMC3628592  PMID: 21964915
Analgesics; Opioid; Drug formulations; Morphine; Drug high; Abuse liability
9.  Exploring factors influencing asthma control and asthma-specific health-related quality of life among children 
Respiratory Research  2013;14(1):26.
Background
Little is known about factors contributing to children’s asthma control status and health-related quality of life (HRQoL). The study objectives were to assess the relationship between asthma control and asthma-specific HRQoL in asthmatic children, and to examine the extent to which parental health literacy, perceived self-efficacy with patient-physician interaction, and satisfaction with shared decision-making (SDM) contribute to children’s asthma control and asthma-specific HRQoL.
Methods
This cross-sectional study utilized data collected from a sample of asthmatic children (n = 160) aged 8–17 years and their parents (n = 160) who visited a university medical center. Asthma-specific HRQoL was self-reported by children using the National Institutes of Health’s Patient-Reported Outcomes Measurement Information System (PROMIS) Pediatric Asthma Impact Scale. Satisfaction with SDM, perceived self-efficacy with patient-physician interaction, parental health literacy, and asthma control were reported by parents using standardized measures. Structural equation modeling (SEM) was performed to test the hypothesized pathways.
Results
Path analysis revealed that children with better asthma control reported higher asthma-specific HRQoL (β = 0.4, P < 0.001). Parents with higher health literacy and greater perceived self-efficacy with patient-physician interactions were associated with higher satisfaction with SDM (β = 0.38, P < 0.05; β = 0.58, P < 0.001, respectively). Greater satisfaction with SDM was in turn associated with better asthma control (β = −0.26, P < 0.01).
Conclusion
Children’s asthma control status influenced their asthma-specific HRQoL. However, parental factors such as perceived self-efficacy with patient-physician interaction and satisfaction with shared decision-making indirectly influenced children’s asthma control status and asthma-specific HRQoL.
doi:10.1186/1465-9921-14-26
PMCID: PMC3599064  PMID: 23432913
Asthma control; Health-related quality of life; PROMIS; Satisfaction with shared decision-making; Perceived self-efficacy with patient-physician interaction; Structural equation modeling
10.  Patient-reported outcomes in randomized clinical trials: development of ISOQOL reporting standards 
Quality of Life Research  2012;22:1161-1175.
Purpose
To develop expert consensus on a suite of reporting standards for HRQL outcomes of RCTs.
Methods
A Task Force of The International Society of Quality of Life Research (ISOQOL) undertook a systematic review of the literature to identify candidate reporting standards for HRQL in RCTs. Subsequently, a web-based survey was circulated to the ISOQOL membership. Respondents were asked to rate candidate standards on a 4-point Likert scale based on their perceived value in reporting studies in which HRQL was a study outcome (primary or secondary). Results were synthesized into draft reporting guidelines, which were further reviewed by the membership to inform the final guidance.
Results
Forty-six existing candidate standards for reporting HRQL results in RCTs were synthesized to produce a 40 item survey that was completed electronically by 161 respondents. The majority of respondents rated all 40 items to be either ‘essential’ or ‘desirable’ when HRQL was a primary RCT outcome. Ratings changed when HRQL was a secondary study outcome. Feedback on the survey findings resulted in the Task Force generalizing the guidance to include patient-reported outcomes (PROs). The final guidance, which recommends standards for use in reporting PROs generally, and more specifically, for PROs identified as primary study outcomes, was approved by the ISOQOL Board of Directors.
Conclusions
ISOQOL has developed a suite of recommended standards for reporting PRO results of RCTs. Improved reporting of PROs will enable accurate interpretation of evidence to inform patient choice, aid clinical decision making, and inform health policy.
doi:10.1007/s11136-012-0252-1
PMCID: PMC3731511  PMID: 22987144
Reporting; Randomized clinical trials; Quality of life; Patient-reported outcomes; Guidelines
11.  Content validity and test-retest reliability of patient perception of intensity of urgency scale (PPIUS) for overactive bladder 
BMC Urology  2012;12:26.
Background
The Patient Perception of Intensity of Urgency Scale (PPIUS) is a patient-reported outcome instrument intended to measure the intensity of urgency associated with each urinary or incontinence episode. The objectives of this study were to assess the content validity, test-retest reliability, and acclimation effect of the PPIUS in overactive bladder (OAB) patients.
Methods
Patients undergoing treatment for OAB were recruited to participate in a non-interventional study by completing a three-day micturition diary including the PPIUS for three consecutive weeks. Following completion of the three-week study, participants from two select sites also completed a cognitive interview to assess their comprehension of the PPIUS.
Results
Thirty-nine participants successfully completed the three-week test-retest study; twelve of these participants completed the cognitive interview. Test-retest reliability was high based on intra-class correlation coefficient of 0.95. Among stable patients, the difference between the mean ratings of any two weeks was non-significant. Among the twelve interview participants, nine found it simple to choose a PPIUS rating for each of their micturition episodes and most found the urgency rating definitions consistent with their urgency experiences.
Conclusions
The results demonstrated content validity based on qualitative interviews, and excellent test-retest reliability among stable patients. In addition, no acclimation effect was observed among stable patients. These findings support the use of the PPIUS as a reliable measure of urgency in both clinical trial and real life settings. The validity of PPIUS could be further established with future studies investigating the relationship between discretely graded urgency and incontinence continuum.
doi:10.1186/1471-2490-12-26
PMCID: PMC3479079  PMID: 22958621
Over active bladder; OAB; Urinary urgency; Urge incontinence; Patient perception of intensity of urgency scale; PPIUS
12.  Differential item functioning in quality of life measure between children with and without special health care needs 
Objectives
Limited studies consider the effect of differential item functioning (DIF) on health-related quality of life (HRQOL) comparisons between ill and health children. The objective is to assess DIF and compare HRQOL between children with special health care needs (CSHCN) and children without needs.
Methods
Data were collected from 1195 families of children enrolled in Florida’s public insurance programs. HRQOL was measured using physical, emotional, social, and school functioning of the PedsQL. We identified CSHCN using the CSHCN Screener and assessed DIF related to CSHCN using a multiple group-multiple indicator-multiple cause (MG-MIMIC) method. We assessed the impact of DIF by examining expected item/test scores and item/test information function. We tested the discrepancy between underlying HRQOL scores of both groups before and after DIF calibration (allowing parameters of DIF items to be different and DIF-free items to be the same across both groups).
Results
Two (25%) and 3 items (60%) of physical and school functioning, respectively, were identified with non-uniform DIF, and two items (40%) of social functioning were identified with uniform DIF. Expected item/test scores and item/test information function suggest the impact of DIF is minimal. Before DIF calibration, HRQOL in CSHCN was more impaired than children without needs (effect size −1.04, − 0.74, −0.96, and −0.98 for physical, emotional, social and school functioning, respectively). After DIF calibration, the discrepancy was increased slightly.
Conclusions
Although 30% of items in the PedsQL were identified with DIF related to CSHCN and children without needs, the impact of DIF is minimal.
doi:10.1016/j.jval.2011.03.004
PMCID: PMC3173710  PMID: 21914509
Children; differential item functioning; health-related quality of life; item response theory
13.  Review of patient-reported outcome measures in chronic hepatitis C 
Background
Chronic hepatitis C (CHC) and its treatment are associated with a variety of patient-reported symptoms and impacts. Some CHC symptoms and impacts may be difficult to evaluate through objective clinical testing, and more easily measured through patient self-report. This literature review identified concepts raised by CHC patients related to symptoms, impacts, and treatment effects, and evaluated integration of these concepts within patient-reported outcome (PRO) measures. The goal of this work was to provide recommendations for incorporation of PRO measurement of concepts that are relevant to the CHC experience into CHC clinical trial design.
Methods
A three-tiered literature search was conducted. This included searches on concepts of importance, PRO measures used in clinical trials, and existing PRO measures. The PRO Concept Search focused on reviewing issues raised by CHC patients about CHC symptoms, disease impact, and treatment effects. The CHC Trials with PRO Endpoints Search reviewed clinical trials with PRO endpoints to assess differences between treatments over time. The PRO Measure Search reviewed existing PRO measures associated with the concepts of interest.
Results
This multi-tiered approach identified five key concepts of interest: depression/anxiety, fatigue, flu-like symptoms, cognitive function, insomnia. Comparing these five concepts of interest to the PRO measures in published CHC clinical trials showed that, while treatment of CHC may decrease health-related quality of life in a number of mental and physical domains, the PRO measures that were utilized in published clinical trials inadequately covered the concepts of interest. Further review of 18 existing PRO measures of the concepts of interest showed only four of the 18 were validated in CHC populations.
Conclusions
This review identified several gaps in the literature regarding assessment of symptoms and outcomes reported as important by CHC patients. Further research is needed to ensure that CHC clinical trials evaluate concepts that are important to patients and include measures that have evidence supporting content validity, reliability, construct validity, and responsiveness.
doi:10.1186/1477-7525-10-92
PMCID: PMC3547737  PMID: 22871087
Hepatitis C; Patient-reported outcomes; Literature review; Health-related quality of life
14.  Validation of a patient interview for assessing reasons for antipsychotic discontinuation and continuation 
Introduction
The Reasons for Antipsychotic Discontinuation Interview (RAD-I) was developed to assess patients’ perceptions of reasons for discontinuing or continuing an antipsychotic. The current study examined reliability and validity of domain scores representing three factors contributing to these treatment decisions: treatment benefits, adverse events, and distal reasons other than direct effects of the medication.
Methods
Data were collected from patients with schizophrenia or schizoaffective disorder and their treating clinicians. For approximately 25% of patients, a second rater completed the RAD-I for assessment of inter-rater reliability.
Results
All patients (n = 121; 81 discontinuation, 40 continuation) reported at least one reason for discontinuation or continuation (mean = 2.8 reasons for discontinuation; 3.4 for continuation). Inter-rater reliability was supported (kappas = 0.63–1.0). Validity of the discontinuation domain scores was supported by associations with symptom measures (the Positive and Negative Syndrome Scale for Schizophrenia, the Clinical Global Impression – Schizophrenia Scale; r = 0.30 to 0.51; all P < 0.01), patients’ primary reasons for discontinuation, and adverse events. However, the continuation domain scores were not significantly associated with these other indicators.
Discussion
Results support the reliability, convergent validity, and known-groups validity of the RAD-I for assessing patients’ reasons for antipsychotic discontinuation. Further research is needed to examine validity of the RAD-I continuation section.
doi:10.2147/PPA.S25635
PMCID: PMC3413072  PMID: 22879738
discontinuation; antipsychotic; schizophrenia; treatment continuation; patientreported outcomes; instrument development
15.  Health related quality of life outcomes for unresectable stage III or IV melanoma patients receiving ipilimumab treatment 
Background
In an international, randomized Phase III trial ipilimumab demonstrated a significant overall survival benefit in previously treated advanced melanoma patients. This report summarizes health-related quality of life (HRQL) outcomes for ipilimumab with/without gp100 vaccine compared to gp100 alone during the clinical trial’s 12 week treatment induction period.
Methods
The Phase III clinical trial (MDX010-20) was a double-blind, fixed dose study in 676 previously treated advanced unresectable stage III or IV melanoma patients. Patients were randomized 3:1:1 to receive either ipilimumab (3 mg/kg q3w x 4 doses) + gp100 (peptide vaccine; 1 mg q3w x 4 doses; ipilimumab plus gp100, n = 403); gp100 vaccine + placebo (gp100 alone, n = 136); or ipilimumab + placebo (ipilimumab alone, n = 137). The European Organization for Research and Treatment of Cancer Quality of Life Questionnaire (EORTC QLQ-C30) assessed HRQL. Baseline to Week 12 changes in EORTC QLQ-C30 function, global health status, and symptom scores were analyzed for ipilimumab with/without gp100 vaccine compared to gp100 alone. Mean change in scores were categorized “no change” (0–5), “a little” (5–10 points), “moderate” (10–20 points), and “very much” (>20).
Results
In the ipilimumab plus gp100 and ipilimumab alone groups, mean changes from baseline to Week 12 generally indicated “no change” or “a little” impairment across EORTC QLQ-C30 global health status, function, and symptom subscales. Significant differences in constipation, favoring ipilimumab, were observed (p < 0.05). For ipilimumab alone arm, subscales with no or a little impairment were physical, emotional, cognitive, social function, global health, nausea, pain, dyspnea, constipation, and diarrhea subscales. For the gp100 alone group, the observed changes were moderate to large for global health, role function, fatigue, and for pain.
Conclusions
Ipilimumab with/without gp100 vaccine does not have a significant negative HRQL impact during the treatment induction phase relative to gp100 alone in stage III or IV melanoma patients.
Trial registration
Clinicaltrials.gov identification number NCT00094653
doi:10.1186/1477-7525-10-66
PMCID: PMC3426458  PMID: 22694829
Ipilimumab; Randomized clinical trial; EORTC QLQ-C30; Advanced melanoma; Health-related quality of life
16.  The Fatigue Associated with Depression Questionnaire (FAsD): responsiveness and responder definition 
Quality of Life Research  2012;22(2):351-360.
Purpose
The Fatigue Associated with Depression Questionnaire (FAsD) was developed to assess fatigue and its impact among patients with depression. The purpose of this study was to examine the questionnaire’s responsiveness to change and identify a responder definition for interpretation of treatment-related changes.
Methods
Data were collected at baseline and at 6 weeks from patients with depression starting treatment with a new antidepressant.
Results
Of the 96 participants, 55.2% were women, with a mean age of 43.4 years. The total score and both subscales demonstrated statistically significant change with moderate to large effect sizes (absolute values ≥0.76). FAsD change scores were significantly correlated with change on the Brief Fatigue Inventory (r ≥ 0.73; p < 0.001). FAsD mean change scores discriminated among patient subgroups differing by degree of improvement in patient- and clinician-reported fatigue and depression. Responder definition for the two subscales and total score (0.67, 0.57, 0.62) was estimated primarily based on mean change among patients who reported a small but important improvement in fatigue.
Discussion
The FAsD was responsive to change, and the responder definition may be used when interpreting treatment-related change. Results add to previous findings suggesting the FAsD is a useful measure of fatigue among patients with depression.
doi:10.1007/s11136-012-0142-6
PMCID: PMC3576557  PMID: 22403040
Fatigue; Depression; Responsiveness; Responder definition; Minimal important difference; Questionnaire
17.  Initial Adult Health Item Banks and First Wave Testing of the Patient-Reported Outcomes Measurement Information System (PROMIS™) Network: 2005–2008 
Journal of clinical epidemiology  2010;63(11):1179-1194.
Objective
Patient-reported outcomes (PROs) are essential when evaluating many new treatments in health care, yet current measures have been limited by a lack of precision, standardization and comparability of scores across studies and diseases. The Patient-Reported Outcomes Measurement Information System (PROMIS™) provides item banks that offer the potential for PRO measurement that is efficient (minimizes item number without compromising reliability) flexible (enables optional use of interchangeable items), and precise (has minimal error in estimate) measurement of commonly-studied PROs. We report results from the first large-scale testing of PROMIS items.
Study Design and Setting
Fourteen item pools were tested in the U.S. general population and clinical groups using an online panel and clinic recruitment. A scale-setting sub-sample was created reflecting demographics proportional to the 2000 U.S. census.
Results
Using item response theory (graded response model), 11 item banks were calibrated on a sample of 21,133, measuring components of self-reported physical, mental and social health, along with a 10-item global health scale. Short forms from each bank were developed and compared to the overall bank as well as with other well-validated and widely accepted (“legacy”) measures. All item banks demonstrated good reliability across the majority of the score distributions. Construct validity was supported by moderate to strong correlations with legacy measures.
Conclusion
PROMIS item banks and their short forms provide evidence they are reliable and precise measures of generic symptoms and functional reports comparable to legacy instruments. Further testing will continue to validate and test PROMIS items and banks in diverse clinical populations.
doi:10.1016/j.jclinepi.2010.04.011
PMCID: PMC2965562  PMID: 20685078
Outcome Measures; Quality of life; Chronic disease
18.  Quality of Life Measurement for Children with Life-Threatening Conditions: Limitations and a New Framework 
Child indicators research  2011;4(1):145-160.
About 500,000 children are coping with life-threatening conditions (LTC) in the United States every year. Different service programs such as an integrated pediatric palliative care program may benefit health-related quality of life (HRQOL) which is a great concern of this children population and their families. However, evidence is limited about the appropriate HRQOL instruments for use. This study aims to validate psychometric properties of a generic HRQOL instrument, the Pediatric Quality of Life (PedsQL) 4.0, for children with LTC. The parent proxy-report was used. We conducted a telephone interview to collect data of 257 parents whose children had LTC and were enrolled in Medicaid. We used standard psychometric methods to validate the PedsQL: scale reliability, item-domain convergent/discriminant validity, and known-groups validity. We also conducted Rasch analysis to assess construct validity. Results suggest that the PedsQL did not demonstrate valid psychometric properties for measuring HRQOL in this population. Rasch analysis suggests that the contents of the items in all domains did not appropriately cover the latent HRQOL of children with LTC. We document several methodological challenges in using a generic instrument to measuring HRQOL and propose a new framework to improve HRQOL measures for children with LTC. The strategies include revising the content of existing items, designing new items, adding important themes (e.g., financial challenge), and applying computerized adaptive test to better select appropriate items for individual children with LTC.
doi:10.1007/s12187-010-9079-x
PMCID: PMC3133777  PMID: 21760876
Quality of life; Life-threatening conditions; Psychometrics
19.  Quality of life information and trust in physicians among families of children with life-limiting conditions 
Patient related outcome measures  2010;2010(1):141-148.
Purpose
To examine information that parents of children with life-limiting conditions want to discuss with children’s physicians to assist decision-making, and whether the desire for this information is associated with parents’ trust in physicians.
Study design
A cross-sectional study using a telephone survey.
Patients and methods
Subjects comprised a random sample of 266 parents whose children were enrolled in Florida’s Medicaid Program. Parents were asked if they wanted to discuss information related to their children’s treatment, including quality of life (QOL), pain relief, spiritual beliefs, clinical diagnosis/laboratory data, changes in the child’s behavior due to treatment, changes in the child’s appearance due to treatment, chances of recovery, and advice from the physician and family/friends. The Wake Forest Physician Trust Scale was used to measure parents’ trust in physicians. We tested the relationships between parents’ age, race/ethnicity, education, parent-reported children’s health status, and the desired information. We also tested whether the desire for information was associated with greater trust in physicians.
Results
Most parents wanted information on their children’s QOL (95%), followed by chance of recovery (88%), and pain relief (84%). Compared with nonHispanic whites, nonHispanic blacks and Hispanics showed a greater desire for information and a chance to discuss QOL information had greater trust in their children’s physicians than other information after adjusting for covariates (P < 0.05).
Conclusions
Among children with life-limiting conditions, QOL is the most frequently desired information that parents would like to receive from physicians as part of shared decision-making. Parents’ desire for QOL information is associated with greater trust in their children’s physicians.
PMCID: PMC3134229  PMID: 21760753
children; information; life-limiting condition; quality of life; shared decision-making
20.  Development of A Promis Item Bank to Measure Pain Interference 
Pain  2010;150(1):173-182.
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 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 to 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.
doi:10.1016/j.pain.2010.04.025
PMCID: PMC2916053  PMID: 20554116
Quality-of-life outcomes; quality-of-life measurement; pain
21.  Psychometric characteristics of the short form 36 health survey and functional assessment of chronic illness Therapy-Fatigue subscale for patients with ankylosing spondylitis 
Background
We evaluated the psychometric characteristics of the Short Form 36 (SF-36) Health Survey and the Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue subscale in patients with ankylosing spondylitis (AS).
Methods
We analyzed clinical and patient-reported outcome (PRO) data collected during 12-week, double-blind, placebo-controlled periods of two randomized controlled trials comparing adalimumab and placebo for the treatment of active AS. The Bath Ankylosing Spondylitis Disease Activity Index, Bath Ankylosing Spondylitis Functional Index, and other clinical measures were collected during the clinical trial. We evaluated internal consistency/reliability, construct validity, and responsiveness to change for the SF-36 and FACIT-Fatigue.
Results
The SF-36 (Cronbach alpha, 0.74-0.92) and FACIT-Fatigue (Cronbach alpha, 0.82-0.86) both had good internal consistency/reliability. At baseline, SF-36 and FACIT-Fatigue scores correlated significantly with Ankylosing Spondylitis Quality of Life scores (r = -0.36 to -0.66 and r = -0.70, respectively; all p < 0.0001). SF-36 scores varied by indicators of clinical severity, with greater impairment observed for more severe degrees of clinical activity (all p < 0.0001). FACIT-Fatigue scores correlated significantly with SF-36 scores (r = 0.42 to 0.74; all p < 0.0001) and varied by clinical severity (p < 0.05 to p < 0.0001).
Conclusions
The SF-36 is a reliable, valid, and responsive measure of health-related quality of life and the FACIT-Fatigue is a brief and psychometrically sound measure of the effects of fatigue on patients with AS. These PROs may be useful in evaluating effectiveness of new treatments for AS.
Trial Registration
ClinicalTrials.gov: NCT00085644 and NCT00195819
doi:10.1186/1477-7525-9-36
PMCID: PMC3124410  PMID: 21600054
Ankylosing spondylitis; Health-related quality of life; Physical and mental health; Reliability; Validity
22.  Development and Psychometric Analysis of the PROMIS Pain Behavior Item Bank 
Pain  2009;146(1-2):158-169.
The measurement of pain behavior is a key component of the assessment of persons with chronic pain; however few self-reported pain behavior instruments have been developed. We developed a pain behavior item bank as part of the Patient Reported Outcome Measurement Information System (PROMIS). For the Wave I testing, because of the large number of PROMIS items, a complex sampling approach was used where participants were randomly assigned to either respond to two full item banks or to multiple 7-item blocks of items. A web-based survey was designed and completed by 15,528 members of the general population and 967 individuals with different types of chronic pain. Item response theory (IRT) analysis models were used to evaluate item characteristics and to scale both items and individuals on the pain behavior domain. The pain behavior item bank demonstrated good fit to a unidimensional model (Comparative Fit Index = 0.94). Several iterations of IRT analyses resulted in a final 39 item pain behavior bank, and different IRT models were fit to the total sample and to those participants who experienced some pain. The results indicated that these items demonstrated good coverage of the pain behavior construct. Pain behavior scores were strongly related to pain intensity and moderately related to self-reported general health status. Mean pain behavior scores varied significantly by groups based on pain severity and general health status. The PROMIS pain behavior item bank can be used to develop static short-form and dynamic measures of pain behavior for clinical studies.
doi:10.1016/j.pain.2009.07.029
PMCID: PMC2775487  PMID: 19683873
Pain behavior; item response theory analysis; patient reported outcomes; psychometric analysis; chronic pain; item banks
23.  Quality of life information and trust in physicians among families of children with life-limiting conditions 
Purpose:
To examine information that parents of children with life-limiting conditions want to discuss with children’s physicians to assist decision-making, and whether the desire for this information is associated with parents’ trust in physicians.
Study design:
A cross-sectional study using a telephone survey.
Patients and methods:
Subjects comprised a random sample of 266 parents whose children were enrolled in Florida’s Medicaid Program. Parents were asked if they wanted to discuss information related to their children’s treatment, including quality of life (QOL), pain relief, spiritual beliefs, clinical diagnosis/laboratory data, changes in the child’s behavior due to treatment, changes in the child’s appearance due to treatment, chances of recovery, and advice from the physician and family/friends. The Wake Forest Physician Trust Scale was used to measure parents’ trust in physicians. We tested the relationships between parents’ age, race/ethnicity, education, parent-reported children’s health status, and the desired information. We also tested whether the desire for information was associated with greater trust in physicians.
Results:
Most parents wanted information on their children’s QOL (95%), followed by chance of recovery (88%), and pain relief (84%). Compared with nonHispanic whites, nonHispanic blacks and Hispanics showed a greater desire for information and a chance to discuss QOL information had greater trust in their children’s physicians than other information after adjusting for covariates (P < 0.05).
Conclusions:
Among children with life-limiting conditions, QOL is the most frequently desired information that parents would like to receive from physicians as part of shared decision-making. Parents’ desire for QOL information is associated with greater trust in their children’s physicians.
doi:10.2147/PROM.S12564
PMCID: PMC3134229  PMID: 21760753
children; information; life-limiting condition; quality of life; shared decision-making
24.  Development and psychometric assessment of the COPD and Asthma Sleep Impact Scale (CASIS) 
Background
Patients with respiratory disease experience disturbed sleep, but there is no widely accepted measure of sleep impairment due to respiratory disease. We developed and evaluated the psychometric performance of a patient-reported measure to assess the impact on sleep due to respiratory disease, the COPD and Asthma Sleep Impact Scale (CASIS).
Methods
Identification of the items forming the CASIS was guided by patient interviews and focus groups. An observational study involving patients from the US and UK was then conducted to assess the psychometric characteristics of the measure.
Results
Qualitative data from 162 patients were used to develop the CASIS (n = 78 COPD; n = 84 asthma). The observational study included 311 patients with COPD and 324 patients with asthma. The final seven items used in the CASIS were identified based on factor and item response theory analyses. Internal consistency was 0.90 (COPD) and 0.92 (asthma), and test-retest reliability was 0.84 (both groups). In the COPD sample, CASIS scores were significantly correlated with the Saint George's Respiratory Questionnaire scores (all p < 0.0001) and differed significantly by patient-reported disease severity, exacerbation status, and overall health status (all p ≤ 0.005). In the asthma sample, CASIS scores were significantly correlated with the Asthma Quality of Life Questionnaire scores (all p < 0.0001) and differed significantly by clinician and patient-reported disease severity, exacerbation status, and overall health status (all p ≤ 0.0005).
Conclusion
The CASIS shows good internal consistency, test-retest reliability, and construct validity and may be useful in helping to understand the impact that COPD and asthma have on sleep outcomes.
doi:10.1186/1477-7525-7-98
PMCID: PMC2794842  PMID: 19968881
25.  Linking Pain Items from Two Studies onto a Common Scale using Item Response Theory 
This study examined two approaches to linking items from two pain surveys to form a single item bank with a common measurement scale. Secondary analysis of two independent surveys: IMMPACT Survey with Main Survey (959 chronic pain patients; 42 pain items) and Pain Modules (N=148; 36 pain items); and CORE Survey (400 cancer patients; 43 pain items). There were common items included among the three data sets. The two approaches were examined, one in which all items were calibrated to an item response theory (IRT) model simultaneously and another in which items were calibrated separately and then the scales were transformed to a common metric. The two approaches produced similar linking result across the two sets of pain interference items because there was sufficient number of common items and large enough sample size. For pain intensity, simultaneous calibration yielded more stable results. Separated calibration yielded unsatisfactory linking result for pain intensity because of a single common item with small sample size. The results suggested that simultaneous IRT calibration method produced the more stable item parameters across independent samples, hence, is recommended for developing comprehensive item banks. Patient reported health outcome surveys are often limited in sample sizes and the number of items owing to the difficulty of recruitment and the burden to the patients. As a result, the surveys either lack statistical power or limited in scope. Using IRT methodology, surveys data can be pooled to lend strength to each other to expand the scope and to increase the sample sizes.
doi:10.1016/j.jpainsymman.2008.11.016
PMCID: PMC2761512  PMID: 19577422
item response theory; pain intensity; pain interference; linking studies

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