Showing papers in "Quality of Life Research in 2015"

Sample size for pre-tests of questionnaires

Abstract: Purpose To provide guidance regarding the desirable size of pre-tests of psychometric questionnaires, when the purpose of the pre-test is to detect misunderstandings, ambiguities, or other difficulties participants may encounter with instrument items (called «problems»). Methods We computed (a) the power to detect a problem for various levels of prevalence and various sample sizes, (b) the required sample size to detect problems for various levels of prevalence, and (c) upper confidence limits for problem prevalence in situations where no problems were detected. Results As expected, power increased with problem prevalence and with sample size. If problem prevalence was 0.05, a sample of 10 participants had only a power of 40 % to detect the problem, and a sample of 20 achieved a power of 64 %. To achieve a power of 80 %, 32 participants were necessary if the prevalence of the problem was 0.05, 16 participants if prevalence was 0.10, and 8 if prevalence was 0.20. If no problems were observed in a given sample, the upper limit of a two-sided 90 % confidence interval reached 0.26 for a sample size of 10, 0.14 for a sample size of 20, and 0.10 for a sample of 30 participants. Conclusions Small samples (5‐15 participants) that are common in pre-tests of questionaires may fail to uncover even common problems. A default sample size of 30 participants is recommended.

Conceptual and practical foundations of patient engagement in research at the patient-centered outcomes research institute

Abstract: To provide an overview of PCORI’s approach to engagement in research. The Patient-Centered Outcomes Research Institute (PCORI) was established in 2010 to fund patient-centered comparative effectiveness research. Requirements for research funding from PCORI include meaningful engagement of patients and other stakeholders in the research. PCORI’s approach to engagement in research is guided by a conceptual model of patient-centered outcomes research (PCOR), that provides a structure for understanding engagement in research. To understand and improve engagement in research PCORI is learning from awardees and other stakeholders. Those efforts are described along with PCORI’s capacity building and guidance to awardees via the Engagement Rubric. PCORI’s unique model of engaging patients and other stakeholders in merit review of funding applications is also described. Additional support for learning about engagement in research is provided through specific research funding and through PCORI’s major infrastructure initiative, PCORnet. PCORI requires engagement of stakeholders in the research it funds. In addition PCORI engages stakeholders in activities including review of funding applications and establishment of CER research infrastructure through PCORnet. The comprehensive approach to engagement is being evaluated to help guide the field toward promising practices in research engagement.

Estimating the minimum important change in the 15D scores

Abstract: To facilitate the interpretation of empirical results produced by the 15D, a generic, preference-based instrument for measuring health-related quality of life (HRQoL), a subjective five-category global assessment scale (GAS) was used as an external anchor to determine the minimum important change (MIC) in the 15D scores. Altogether 4,903 hospital patients representing sixteen disease entities and having the 15D score at baseline repeated the HRQoL assessment at 6 months after treatment and answered the question: compared to the situation before treatment, my overall health status is now (1) much better, (2) slightly better, (3) much the same, (4) slightly worse, (5) much worse. Regression analysis was used to estimate the MIC for improvement/deterioration, defined as the lower/upper limit of 99.9 % confidence interval of the regression coefficient, standardized for baseline HRQoL, for categories (2) and (4), respectively, and confirmed by ROC curve analysis. The limits or intervals for classifying the changes of 15D scores into GAS categories were >.035 for (1), .015–.035 for (2),>−.015 and<.015 for (3), −.035–−.015 for (4) and <−.035 for (5). The lower/upper limits of ±.015 for categories (2) and (4) can be regarded as the generic MIC thresholds for improvement/deterioration, respectively. The generic MICs for the change of 15D scores are ±.015. Follow-up studies using the 15D should report the mean change in the 15D score, its statistical significance, relationship to the MIC, and the distribution of the changes of the 15D scores into the five categories.

PROMIS(®) pediatric self-report scales distinguish subgroups of children within and across six common pediatric chronic health conditions

Abstract: To conduct a comparative analysis of eight pediatric self-report scales for ages 8–17 years from the National Institutes of Health (NIH) Patient Reported Outcomes Measurement Information System (PROMIS®) in six pediatric chronic health conditions, using indicators of disease severity. Pediatric patients (N = 1454) with asthma, cancer, chronic kidney disease, obesity, rheumatic disease, and sickle cell disease completed items from the PROMIS pediatric mobility, upper extremity functioning, depressive symptoms, anxiety, anger, peer relationships, pain interference, and fatigue self-report scales. Comparisons within the six pediatric chronic health conditions were conducted by examining differences in groups based on the disease severity using markers of severity that were specific to characteristics of each disease. A comparison was also made across diseases between children who had been recently hospitalized and those who had not. In general, there were differences in self-reported health outcomes within each chronic health condition, with patients who had higher disease severity showing worse outcomes. Across health conditions, when children with recent hospitalizations were compared with those who had not been hospitalized in the past 6 months, we found significant differences in the expected directions for all PROMIS domains, except anger. PROMIS measures discriminate between different clinically meaningful subgroups within several chronic illnesses. Further research is needed to determine the responsiveness of the PROMIS pediatric scales to change over time.

Common patterns of morbidity and multi-morbidity and their impact on health-related quality of life: evidence from a national survey.

Abstract: Background There is limited evidence about the impact of specific patterns of multi-morbidity on health-related quality of life (HRQoL) from large samples of adult subjects.

Validity of EQ-5D-5L in stroke

Abstract: Purpose To assess EQ-5D-5L (5L) validity in patients with acute stroke, in comparison with EQ-5D-3L (3L).

Validation of the PROMIS physical function measures in a diverse US population-based cohort of cancer patients

Abstract: To evaluate the validity of the Patient-Reported Outcomes Measurement Information System (PROMIS) physical function measures in a diverse, population-based cancer sample. Cancer patients 6–13 months post-diagnosis (n = 4840) were recruited for the Measuring Your Health study. Participants were diagnosed between 2010 and 2013 with non-Hodgkin lymphoma or cancers of the colorectum, lung, breast, uterus, cervix, or prostate. Four PROMIS physical function short forms (4a, 6b, 10a, and 16) were evaluated for validity and reliability across age and race–ethnicity groups. Covariates included gender, marital status, education level, cancer site and stage, comorbidities, and functional status. PROMIS physical function short forms showed high internal consistency (Cronbach’s α = 0.92–0.96), convergent validity (fatigue, pain interference, FACT physical well-being all r ≥ 0.68), and discriminant validity (unrelated domains all r ≤ 0.3) across survey short forms, age, and race–ethnicity. Known-group differences by demographic, clinical, and functional characteristics performed as hypothesized. Ceiling effects for higher-functioning individuals were identified on most forms. This study provides strong evidence that PROMIS physical function measures are valid and reliable in multiple race–ethnicity and age groups. Researchers selecting specific PROMIS short forms should consider the degree of functional disability in their patient population to ensure that length and content are tailored to limit response burden.

Training clinicians in how to use patient-reported outcome measures in routine clinical practice.

Abstract: Introduction Patient-reported outcome measures (PROs) were originally developed for comparing groups of people in clinical trials and population studies, and the results were used to support treatment recommendations or inform health policy, but there was not direct benefit for the participants providing PROs data. However, as the experience in using those measures increased, it became obvious the clinical value in using individual patient PROs profiles in daily practice to identify/monitor symptoms, evaluate treatment outcomes and support shared decision-making. A key issue limiting successful implementation is clinicians’ lack of knowledge on how to effectively utilize PROs data in their clinical encounters.

Normative data for the Hospital Anxiety and Depression Scale

Abstract: The Hospital Anxiety and Depression Scale (HADS) is widely used in both research and clinical contexts. However, UK normative data from HADS remain limited. In our recent review of the literature, only six reports from four studies were identified as reporting UK normative data and all had limitations. The aim of our study was to use a large population-based dataset to address this. The Epidemiology of Functional Disorders Study is a large longitudinal population-based study carried out in Northwest England. All adults aged between 25 and 65 years registered with three general practices were sent a self-completion questionnaire which contained the HADS and other health-related instruments. Scores were calculated for participants completing all items on each sub-scale (anxiety 6,189 participants and depression 6,198 participants). Scores are presented by gender and by 5-year age groups. Percentile scores were also generated. The median anxiety score was higher in women [6, interquartile range (IQR) 4–9] than in men (5, IQR 2–8) and increased with age in both groups. The median depression score for both women and men was 3 (IQR 1–6). Our study is the largest population-based study providing UK normative data from the HADS. While our data confirm some of the normative data reported previously, subtle and important differences emerged, particularly at the upper end of the percentile scores. Due to the nature of our study design and the number of participants sampled, we believe that our data are likely to be more representative of the UK population than existing published normative values.

Reliability and validity of the EQ-5D-5L compared to the EQ-5D-3L in patients with osteoarthritis referred for hip and knee replacement

Abstract: To assess the test–retest reliability of the EQ-5D-5L (5L) and compare the validity of the 5L and EQ-5D-3L (3L) in osteoarthritis patients referred to an orthopaedic surgeon for total joint replacement. We mailed questionnaires to 306 consecutive patients following referral and a second questionnaire after 2 weeks to assess reliability. Questionnaires included the 5L, EQ-VAS, Short Form-12, Oxford hip and knee scores, pain VAS, and the 3L. We compared the ceiling effect, redistribution properties, convergent and discriminant validity, and discriminatory power of the 5L and 3L. We obtained 176 respondents (response rate 58 %), 60 % female, 64 % knee patients, mean age 65 years (SD 11), with no significant differences between responders versus non-responders. Intraclass correlation coefficients were 0.61–0.77 for the 5L dimensions and 0.87 for the 5L index. For the 3L, most patients used level 2 (some/moderate problems) for mobility (87 %), usual activities (78 %), and pain/discomfort (71 %). In comparison, 5L responses were spread out with only 52, 42, and 50 %, respectively, using the middle level. All convergent validity coefficients were stronger with the 5L (Spearman coefficients 0.51–0.75). Absolute informativity (Shannon’s index) showed higher results for all dimensions of the 5L compared with the 3L (average difference 0.74). Relative informativity (Shannon’s evenness index) showed an increase from the 3L to the 5L in mobility, usual activities, and pain/discomfort. The 5L provided stronger validity evidence than the 3L, especially for dimensions relevant to this patient population—mobility, usual activities, and pain/discomfort.

Equivalence of electronic and paper-based patient-reported outcome measures

Abstract: Aim Electronic formats (ePROs) of paper-based patient-reported outcomes (PROs) should be validated before they can be reliably used. This review aimed to examine studies investigating measurement equivalence between ePROs and their paper originals to identify methodologies used and to determine the extent of such validation. Methods Three databases (OvidSP, Web of Science and PubMed) were searched using a set of keywords. Results were examined for compliance with inclusion criteria. Articles or abstracts that directly compared screen-based electronic versions of PROs with their validated paper-based originals, with regard to their measurement equivalence, were included. Publications were excluded if the only instruments reported were stand-alone visual analogue scales or interactive voice response formats. Papers published before 2007 were excluded, as a previous meta-analysis examined papers published before this time. Results Fifty-five studies investigating 79 instruments met the inclusion criteria. 53 % of the 79 instruments studied were condition specific. Several instruments, such as the SF-36, were reported in more than one publication. The most frequently reported formats for ePROs were Web-based versions. In 78 % of the publications, there was evidence of equivalence or comparability between the two formats as judged by study authors. Of the 30 publications that provided preference data, 87 % found that overall participants preferred the electronic format. Conclusions When examining equivalence between paper and electronic versions of PROs, formats are usually judged by authors to be equivalent. Participants prefer electronic formats. This literature review gives encouragement to the further widespread development and use of ePROs.

Comparing responsiveness of the EQ-5D-5L, EQ-5D-3L and EQ VAS in stroke patients

Abstract: Aims To date, evidence to support the construct validity of the EQ-5D-5L has primarily focused on cross-sectional data. The aims of this study were to examine the responsiveness of EQ-5D-5L in patients with stroke and to compare it with responsiveness of EQ-5D-3L and visual analogue scale (EQ VAS).

Time to health-related quality of life score deterioration as a modality of longitudinal analysis for health-related quality of life studies in oncology: do we need RECIST for quality of life to achieve standardization?

Abstract: Purpose Longitudinal analysis of health-related quality of life (HRQoL) remains unstandardized and compromises comparison of results between trials. In oncology, despite available statistical approaches, results are poorly used to change standards of care, mainly due to lack of standardization and the ability to propose clinical meaningful results. In this context, the time to deterioration (TTD) has been proposed as a modality of longitudinal HRQoL analysis for cancer patients. As for tumor response and progression, we propose to develop RECIST criteria for HRQoL.

Why do multi-attribute utility instruments produce different utilities: the relative importance of the descriptive systems, scale and ‘micro-utility’ effects

Abstract: Purpose Health state utilities measured by the major multi-attribute utility instruments differ. Understanding the reasons for this is important for the choice of instrument and for research designed to reconcile these differences. This paper investigates these reasons by explaining pairwise differences between utilities derived from six multi-attribute utility instruments in terms of (1) their implicit measurement scales; (2) the structure of their descriptive systems; and (3) ‘micro-utility effects’, scale-adjusted differences attributable to their utility formula.

A qualitative study of the impact of severe asthma and its treatment showing that treatment burden is neglected in existing asthma assessment scales.

Abstract: People with severe asthma experience significant respiratory symptoms and suffer adverse effects of oral corticosteroids (OCS), including disturbed mood and physical symptoms. OCS impacts on health-related quality of life (HRQoL) have not been quantified. Asthma HRQoL scales are valid as outcome measures for patients requiring OCS only if they assess the deficits imposed by OCS. The aim of this study was to compare the burden of disease and treatment in patients with severe asthma with items in eight asthma-specific HRQoL scales. Twenty-three patients with severe asthma recruited from a severe asthma clinic were interviewed about the impact of their respiratory symptoms and the burden of their treatment. The domains from a thematic analysis of these interviews were compared with the items of eight asthma-specific HRQoL scales. In addition to the burden caused by symptoms, ten domains of OCS impact on HRQoL were identified: depression, irritability, sleep, hunger, weight, skin, gastric, pain, disease anxiety, and medication anxiety. Some patients experienced substantial HRQoL deficits attributed to OCS. Although all HRQoL scales include some OCS-relevant items, all eight scales fail to adequately assess the several types of burden experienced by some patients while on OCS. The burden of OCS in severe asthma is neglected in policy and practice because it is not assessed in outcome studies. Existing asthma HRQoL scales provide an overly positive estimation of HRQoL in patients with frequent exposure to OCS and underestimate the benefit of interventions that reduce OCS exposure. Changes to existing measurement procedures are needed.

Living with oropharyngeal dysphagia: effects of bolus modification on health-related quality of life--a systematic review.

Abstract: Difficulty swallowing, oropharyngeal dysphagia, is widespread among many patient populations (such as stroke and cancer groups) and aged community-dwelling individuals. It is commonly managed with bolus modification: altering food (usually cutting, mashing or pureeing) or fluids (typically thickening) to make them easier or safer to swallow. Although this treatment is ubiquitous, anecdotal evidence suggests patients dislike this management, and this may affect compliance and well-being. This review aimed to examine the impact of bolus modification on health-related quality of life. A systematic review of the literature was conducted by speech pathologists with experience in oropharyngeal dysphagia. The literature search was completed with electronic databases, PubMed and Embase, and all available exclusion dates up to September 2012 were used. The search was limited to English-language publications which were full text and appeared in peer-reviewed journals. Eight studies met the inclusion criteria. Generally, bolus modification was typically associated with worse quality of life. Modifications to foods appeared to be more detrimental than modifications to fluids, but this may be due to the increased severity of dysfunction that is implied by the necessity for significant alterations to foods. The number of studies retrieved was quite small. The diverse nature of methodologies, terminologies and assessment procedures found in the studies makes the results difficult to generalise. Overall, even though the severity of dysphagia may have been a confounding factor, the impact of bolus modification on health-related quality of life in patients with oropharyngeal dysphagia appears to be negative, with increased modification of food and fluids often correlating to a decreased quality of life. Further, associated disease factors, such as decreased life expectancy, may also have affected health-related quality of life. More research is needed.

A methodological review of the Short Form Health Survey 36 (SF-36) and its derivatives among breast cancer survivors

Abstract: Purpose A systematic review of the validity, reliability and sensitivity of the Short Form (SF) health survey measures among breast cancer survivors.

Communicating patient-reported outcome scores using graphic formats: results from a mixed-methods evaluation

Abstract: Background Patient-reported outcomes (PROs) promote patient-centered care by using PRO research results (“group-level data”) to inform decision making and by monitoring individual patient’s PROs (“individual-level data”) to inform care. We investigated the interpretability of current PRO data presentation formats.

Building new roles and relationships in research: a model of patient engagement research.

Abstract: Purpose Patient engagement is influenced by institutional ideologies, professional attitudes and patient readiness to accept new, engaged roles. This article provides an opportunity to consider a new role for patients who are trained to conduct patient experience research using qualitative methods.

Health-related quality of life of Asian patients with end-stage renal disease (ESRD) in Singapore.

Abstract: This study aimed to identify factors associated with the health-related quality of life (HRQOL) of multiethnic Asian end-stage renal disease (ESRD) patients treated with dialysis. The role of dialysis modality was also explored. Data used in this study were from two cross-sectional surveys of Singaporean ESRD patients on haemodialysis (HD) or peritoneal dialysis (PD). In both surveys, participants were assessed using the kidney disease quality of life (KDQOL) instrument and questions assessing socio-demographic characteristics. Clinical data including co-morbidity (measured by Charlson comorbidity index [CCI]), albumin level, haemoglobin level, and dialysis-related variables (e.g. dialysis vintage and dialysis adequacy) were retrieved from medical records. The 36-item KDQOL (KDQOL-36) was used to generate three summary scores (physical component summary [PCS], mental component summary [MCS] and kidney disease component summary [KDCS]) and two health utility scores (Short Form 6-dimension [SF-6D] and EuroQol 5-dimension [EQ-5D]). Linear regression analysis was performed to examine the association of factors with each of the HRQOL scale scores. Five hundred and two patients were included in the study (mean age 57.1 years; male 52.4 %; HD 236, PD 266). Mean [standard deviation (SD)] PCS, MCS and KDCS scores were 37.9 (9.7), 46.4 (10.8) and 57.6 (18.1), respectively. Mean (SD) health utility score was 0.66 (0.12) for SF-6D and 0.60 (0.21) for EQ-5D. In multivariate regression analysis, factors found to be significantly associated with better HRQOL included: young ( 60 years), low CCI (<5), high albumin (≥37 g/l) and high haemoglobin (≥11 g/dl) with PCS; long dialysis vintage (≥3.5 years) with MCS; old age, Malay ethnicity and PD modality with KDCS; low CCI, high albumin and high haemoglobin with EQ-5D and high albumin with SF-6D. Clinical characteristics are better predictors of HRQOL in ESRD patients than socio-demographics in Singapore. Dialysis modality has no impact on the health utility of those patients.

Clinical improvement and satisfaction after total joint replacement: a prospective 12-month evaluation on the patients' perspective.

Abstract: To determine short-term improvements, satisfaction rates and the patient acceptable symptom state (PASS) after total joint replacement (TJR) for different patient-reported outcome measures (PROMs). This prospective cohort study included 426 consecutive patients undergoing total hip (n = 193) or knee arthroplasty (n = 233). The following PROMs were completed before TJR, and at 3, 6 and 12 months after surgery, respectively: WOMAC, Oxford Hip or Knee Score, Lower Extremity Functional Scale, University of California at Los Angeles (UCLA) activity scale and EuroQol-5 dimension (EQ-5D). Satisfaction rates and the PASS thresholds were also assessed. THA patients improved quicker and achieved higher outcome scores than TKA patients. Comorbidities according to the Sangha score were moderately correlated with all PROM values in an inverse direction at all time points (r = −0.27 to −0.47, p < 0.01) in both groups. Satisfaction with the result of surgery improved over time. At 12 months, more than 90 % of the patients were satisfied or very satisfied with the achieved result. The THA group showed a higher proportion of very satisfied patients than the TKA group at all time points. PASS thresholds increased over time for all PROMs except for the UCLA and the EQ-5D in TKA patients. More than 90 % of the patients will be satisfied 1 year after TJR. THA patients recover faster than TKA patients, i.e., they achieve higher PROM values at earlier follow-up time points. Cutoff values defining a successful result in terms of the PASS could be defined for all PROMs at different time points and can serve as reference for future studies and patient-oriented follow-ups.

The use of the EQ-5D as a measure of health-related quality of life in people with dementia and their carers

Abstract: Purpose To assess the acceptability, validity and inter-rater agreement of self- and family carer proxy ratings of the EQ-5D as a generic health-related quality of life (HRQOL) measure, in people with mild to moderate dementia (PwD) living in the community. A secondary aim was to identify the most important factors influencing self- and family carer proxy ratings of HRQOL, distinguishing between spouse and adult child caregiver ratings.

The STOP-BANG questionnaire: reliability and validity of the Persian version in sleep clinic population.

Abstract: The snoring, tiredness, observed apnea, blood pressure, body mass index, age, neck circumference, gender (STOP-BANG) is a concise and effective obstructive sleep apnea (OSA) screening tool, part questionnaire (STOP), and part demographic or anthropometric measurements (BANG). The main purpose of this study was to translate this well-liked questionnaire into Persian and assess its reliability and validity in sleep clinic population. Standard forward–backward method was used for translation. A sample of 603 patients, who admitted to the sleep clinic, completely answered to the STOP questionnaire and underwent in-laboratory polysomnography, included in this study. Height, weight, and neck circumference were measured by technicians for calculating BANG score. The apnea–hypopnea index (AHI) on the polysomnography was used as gold standard for OSA diagnosis: none (AHI < 5), mild (5 ≤ AHI < 15), moderate (15 ≤ AHI < 30), and severe (AHI ≥ 30). One hundred and forty one patients were answered to the STOP questionnaire twice at a time interval of 2–4 weeks for test–retest analysis. In reliability analysis, 124 (87.9 %) patients had same STOP score and 130 (92.2 %) patients were classified in same risk of OSA. Based on the polysomnography, 438 patients (72.6 %) had mild (n = 124, 20.4 %), moderate (n = 114, 18.9 %), and severe (n = 201, 33.3 %) OSA, whereas according to the STOP-BANG, 502 patients (83.3 %) were at high risk of OSA. The sensitivity and specificity of the STOP-BANG were found to be 91.6 and 45.2 %, respectively, at AHI ≥ 5, 97.1 and 35.2 %, respectively, at AHI ≥ 15, and 98 and 29.4 %, respectively, at AHI ≥ 30. The area under the curve of the STOP-BANG for identifying mild, moderate, and severe OSA was 0.805, 0.779, and 0.755, respectively. Persian version of the STOP-BANG performs similar to its original version and is an easy-to-use questionnaire which could be considered as a reliable and valid tool for OSA screening.

Engaging patients to recover life projectuality: an Italian cross-disease framework.

Abstract: Chronic disease is recognized as having a large impact on patient quality of life (QoL), which can be defined as an individual’s satisfaction or happiness with life in domains he or she considers important. Policy makers and clinicians recognize increasingly that patients can safeguard their QoL by making healthy lifestyle choices and being actively engaged in their health care. However, in the emphasis on promoting patient engagement to enhance patients’ QoL, there is no consensus regarding the relationship between QOL and patient engagement, resulting in a lack of shared guidelines among clinicians on interventions. Furthermore, no studies have provided an in-depth exploration of the perspective of patients with chronic conditions who are engaged in their health care and their requirements to achieve an improved QoL. Given this theoretical gap, the present study attempted to explore the patient engagement experience and its relationship with patient QoL in the context of the Italian healthcare system and in relation to different chronic diseases. In-depth qualitative interviews on a sample of 99 patients with a wide variety of chronic conditions (heart failure, chronic obstructive pulmonary disease, stroke, diabetes, and cancer). Patient engagement in health care can be defined as a context-based and cross-disease process that appears to enable patients to recover their life projectuality, which had been impaired by the onset of chronic disease. Successful patient engagement may also be related to a positive shift in the ways in which patients perceive self and life and experience empowerment to realize their life potential, thus improving quality of life. Patient engagement is a powerful concept capable of reflecting significant psychosocial changes that promote patient QoL along the care process. There appears to be theoretical and empirical justification for a broad definition of QoL. QoL deeply depends on the patient ability to engage in their care and on the health expectations they have. We propose a model of the relation between patient engagement and patients’ trajectories in critical event responses and use it to illustrate a new perspective on QoL. This research showed the heuristic value patient engagement as a is a key concept in the promotion of a patients’ experience-sensitive QoL interventions and assessment measures.

Item-level informant discrepancies between children and their parents on the PROMIS® pediatric scales

Abstract: The study objective was to describe the individual item-level discrepancies between children ages 8–17 years and their parents for the PROMIS® pediatric scales. Contextual effects on item-level informant discrepancies for the pediatric pain interference items were further analyzed conditional on whether the child, the parent, or anyone else in the household experienced chronic pain. Parallel pediatric self-report and parent proxy-report items were completed by approximately 300 parent–child dyads depending on form assignment and individual nonresponse. Agreement between parent and child responses to individual items was measured using the polychoric correlation coefficient and weighted κ. The Chi-square test of symmetry was utilized for a comparison of the pattern of parent–child item discrepancies on the response scales, and the differences between the child and parent responses on the 1–5 item response scale are summarized . A continuum of higher item-level parent–child discrepancies was demonstrated starting with peer relationships, anger, anxiety, and depressive symptoms, followed by progressively lower parent–child discrepancies for energy, fatigue, asthma impact, pain interference, upper extremity, and mobility items. Parent–child discrepancies for pain interference items were lower in the context of chronic pain either in the child or in the parent. Parent–child item-level discrepancies were lower for more objective or visible items than for items measuring internal states or less observable items measuring latent variables such as peer relationships and fatigue. Future research should focus on the child and parent characteristics that influence domain-specific item-level discrepancies, and under what conditions item-level parent–child discrepancies predict child health outcomes.

Patient and public engagement in health-related quality of life and patient-reported outcomes research: what is important and why should we care? Findings from the first ISOQOL patient engagement symposium

Abstract: Recent years have witnessed growing international interest in the active involvement, or engagement [patient engagement (PE)], of patients and the public in health services research. However, there is limited evidence of the extent or impact of PE in health-related quality of life (HRQL) and patient-reported outcomes (PRO) research. Therefore, in October 2013, the International Society for Quality of Life research (ISOQOL) hosted its first symposium, which sought to explore the potential for PE in this field. A ‘World Cafe’ format encouraged the exploration of three ‘menu’ questions’ in small groups at round tables. Views, opinions and concerns were captured. A thematic analysis was undertaken, and key themes listed. Sixty conference attendees participated in the ‘PE Cafe’, which lasted for 90 min. A diversity of experience was communicated, with most participants positive about the potential for PE. Similarities and differences in approaches, barriers and solutions were identified. However, a key message focused on the uncertainty about how to effectively engage with patients throughout the research process. Moreover, the lack of evidence-base demonstrating the impact of PE was a significant concern. No patient partners participated in the meeting. This study describes the first international exploration of PE in HRQL and PRO research. Discussions highlighted that, in the absence of good practice guidelines, a framework or toolkit of how to embed PE within HRQL and PRO research is required. Moreover, this framework should support the rigorous evaluation of PE impact. ISOQOL should be instrumental in taking these ideas forward, actively engaging with patient partners towards shaping a future ISOQOL PE strategy.

Establishing a common metric for self-reported pain: linking BPI Pain Interference and SF-36 Bodily Pain Subscale scores to the PROMIS Pain Interference metric

Abstract: The study purposes were to mathematically link scores of the Brief Pain Inventory Pain Interference Subscale and the Short Form-36 Bodily Pain Subscale (legacy pain interference measures) to the NIH Patient-Reported Outcome Measurement Information System (PROMIS®) Pain Interference (PROMIS-PI) metric and evaluate results. Linking was accomplished using both equipercentile and item response theory (IRT) methods. Item parameters for legacy items were estimated on the PROMIS-PI metric to allow for pattern scoring. Crosswalk tables also were developed that associated raw scores (summed or average) on legacy measures to PROMIS-PI scores. For each linking strategy, participants’ actual PROMIS-PI scores were compared to those predicted based on their legacy scores. To assess the impact of different sample sizes, we conducted random resampling with replacement across 10,000 replications with sample sizes of n = 25, 50, and 75. Analyses supported the assumption that all three scales were measuring similar constructs. IRT methods produced marginally better results than equipercentile linking. Accuracy of the links was substantially affected by sample size. The linking tools (crosswalks and item parameter estimates) developed in this study are robust methods for estimating the PROMIS-PI scores of samples based on legacy measures. We recommend using pattern scoring for users who have the necessary software and score crosswalks for those who do not.

Psychometric properties of the EQ-5D-5L in Thai patients with chronic diseases

Abstract: Due to the problem of high ceiling effects of the EQ-5D-3L, the EQ-5D-5L was developed. However, little was known about the full psychometric properties of the EQ-5D-5L. Thus, this study aimed to evaluate its practicality, reliability, validity, and responsiveness in Thai patients with chronic diseases. One thousand one hundred and fifty-six adults taking a medicine at least 3 months were identified from three university hospitals in Bangkok, Thailand, between July 2014 and March 2015. Practicality was evaluated by administration times and ceiling effects. Test–retest reliability was assessed using weighted kappa and intraclass correlation coefficients (ICCs). Validity was tested with correlations between the EQ-5D-5L and WHOQoL-BREF and SF-12v2, and known-groups validity. Responsiveness was measured with standardized effect sizes (SES). The mean administration time was approximately 2 min, and the ceiling effect of the EQ-5D-5L index was 13.6 %. The weighted kappa values and ICC of the EQ-5D-5L were 0.48–0.61 and 0.82, respectively. Similar dimensions of the EQ-5D-5L had higher correlations with those of WHOQoL-BREF and SF-12v2. As expected, elderly, female, low-educated, unemployed, higher number of comorbidities and medicines, patients’ perception of poor disease control, and having an adverse drug reaction tended to have poorer EQ-5D-5L scores. The SES of EQ-5D-5L index and EQ-VAS were considered small (0.33–0.42) for the improved group. For the worsened group, the SES of the EQ-5D-5L index were considered small (−0.29) but that of the EQ-VAS considered large (−0.82). The EQ-5D-5L was practical, reliable, valid, and responsive in Thai patients with chronic diseases.

Creating meaningful cut-scores for Neuro-QOL measures of fatigue, physical functioning, and sleep disturbance using standard setting with patients and providers

Abstract: To establish clinically relevant classifications of health outcome scores for four Neuro-QOL measures (lower extremity function, upper extremity function, fatigue, and sleep disturbance). We employed a modified educational standard-setting methodology to identify cut-scores for symptom severity. Clinical vignettes were developed to represent graduated levels of symptom severity. A clinician panel and a panel of persons with multiple sclerosis (PwMS) were recruited, and, in separate, 1-day meetings, the panelists identified adjacent vignettes they judged to represent the threshold between two levels of severity for a given domain (e.g., threshold between a vignette that indicated “no problems” with sleep and the adjacent one that represented “mild problems” with sleep). Working independently, each panel (PwMS and clinicians) reached consensus on its recommended thresholds for each of the four targeted measures. Cut-scores were defined as the mean location for each pair of threshold vignettes. PwMS and clinician panels derived identical thresholds for severity levels of lower extremity function and sleep disturbance, but slightly different ones for upper extremity function and fatigue. In every case of divergence, PwMS set higher thresholds for more severe classifications of symptoms (by 0.5 SDs) than did clinicians. The modified bookmarking method is effective for defining thresholds for symptom severity based on self-reported outcome scores and consensus judgments. Derived cut-scores and severity levels provide an interpretative context for Neuro-QOL scores. Future studies should explore whether these findings can be replicated and evaluate the validity of the classifications compared to external criteria.

The EQ-5D-5L index score is more discriminative than the EQ-5D-3L index score in diabetes patients

Abstract: To compare the discriminative power of the index scores of EQ-5D-5L (5L) and EQ-5D-3L (3L) in diabetes patients in China. A consecutive sample of type 2 diabetes mellitus (T2DM) patients in the clinics self-completed the two versions of EQ-5D. The 3L index score was obtained from the Chinese 3L value set; the 5L index score was predicted from the 3L index score using an interim scoring. Relative efficiency (RE) of the 5L and 3L index scores was calculated to compare their ability in differentiating between T2DM patients with and without one of ten clinical conditions. The efficiency of the 5L and 3L health state classification systems was assessed using the Shannon index (H′) and in terms of ceiling effects. A total of 289 T2DM patients participated in this study. The 5L score was systematically lower than the 3L score for T2DM patients with and without a condition (range −0.36 to −0.06). The 5L score exhibited higher discriminative power in nine of ten conditions, with the mean RE value being 1.92. 5L had higher H′ values than 3L in all the five EQ-5D dimensions: mobility (1.14 vs. 0.70), self-care (0.44 vs. 0.33), usual activities (0.72 vs. 0.47), pain/discomfort (1.58 vs. 1.10), and anxiety/depression (1.03 vs. 0.67). The overall ceiling effects decreased from 56.7 % (3L) to 36.7 % (5L). The 5L index score is more discriminative than the 3L index score in T2DM patients and therefore is preferable for use in this population.