Development and validation of the HDSIM™ assessment system: a measure of hemorrhoid disease symptom impact.

BACKGROUND: Hemorrhoid disease (HD) affects 10 million people in the US at any given time, and 50% of the US population will develop symptomatic hemorrhoids during their lifetime. Approximately 60% of people with hemorrhoids experience symptoms. Despite its prevalence and impact on quality of life, the existing patient-reported outcome measures of HD have not been validated using standard psychometric methods. The present study thus aimed to develop the Hemorrhoid Disease Symptom Impact Measure™ (HDSIM™) assessment system, a patient-reported measure of HD symptoms and impact for use in HD clinical research. METHODS: On the basis of results from qualitative cognitive interviews, we generated the conceptual model and item pool. A cross-sectional web-based survey (n = 1066) was done, including a randomly selected retest subsample (n = 100) 1-2 weeks later. The survey sample was selected to be evenly distributed across mild, moderate, and severe levels of disease and to be nationally representative of the general United States population in terms of race, gender, and region. Existing disease-specific measures of symptoms and generic measures of quality of life and well-being were compared to the new tool for construct validation. RESULTS: The HDSIM system includes 38 items representing six conceptual-model-driven subscales, aligning with the conceptual model: Symptoms at Worst, Symptoms at Best, Bowel Health Impact, Life Impact, Mental Health Impact, and Manageability. Psychometric analyses documented that the subscales had excellent internal consistency reliability, cross-sectional construct validity (i.e., convergent and divergent validity, known groups validity), test-retest stability, and longitudinal construct validity (i.e., responsiveness). CONCLUSION: The HDSIM system is fit for purpose in hemorrhoid disease clinical trials research. Since measures are validated in an iterative manner over many studies and over time, the present study results should be considered preliminary.

Disabled from work and depressed: cognitive factors associated with exacerbated or attenuated depression over the COVID-19 pandemic.

BACKGROUND: People who were disabled from working reported substantially worse depression in recent research [1] despite adjustment for demographic covariates, cognitive-appraisal processes, and COVID-specific stressors, thus motivating the present work. OBJECTIVE: This study sought to "drill down" to understand employment-group differences (employed, retired, unemployed, disabled) in cognitive factors, and how these factors played into paths to depression during COVID early in the pandemic and depression trajectories over 15.5 months of follow-up. METHODS: This longitudinal cohort study (n = 771) included chronically ill and general-population samples in the United States, characterized into the same depression-trajectory groups as the earlier study [1]. The Quality-of-Life Appraisal Profilev2 Short-Form assessed cognitive-appraisal processes. COVID-specific scales assessed hardship, worry, and social support. Chi-square, Analysis of Variance, classification and regression tree, and random effects modeling investigated factors associated with reported depression over time specifically by employment group, rather than in the whole sample which was the focus of the earlier study. RESULTS: Disabled participants were disproportionately represented in the stably depressed trajectory group, reporting more hardship and worry, and lower social support than employed and retired participants (p < 0.0001). They were more likely to focus on health goals, problem goals, and emphasizing the negative (p < 0.001). They had different paths and cut-points to depression than employed/unemployed/retired participants. Even mild endorsement of emphasizing the negative and recent changes predicted higher depression. COVID-specific stressors and cognitive-appraisal processes were less implicated in depression among disabled participants compared to others. CONCLUSIONS: Disabled participants were at greater risk of stable depression during the COVID pandemic. Small increases in emphasizing the negative were a path to worse depression, and disabled participants' depression may be less reactive to external circumstances or ways of thinking.

When better is the enemy of good: two cautionary tales of conceptual validity versus parsimony in clinical psychometric research.

This paper presents an empirical challenge to the assumption that an item-response theory analysis always yields a better measure of a clinical construct. We summarize results from two measurement development studies that showed that such an analysis lost important content reflecting the conceptual model ("conceptual validity"). The cost of parsimony may thus be too high. Conceptual models that form the foundation of QOL measurement reflect the patient's experience. This experience may include concepts and items that are psychometrically "redundant" but capture distinct features of the concept. Good measurement is likely a balance between relying on IRT's quantitative metrics and recognizing the importance of conceptual validity and clinical utility.

Individual differences in the long-term impact of the pandemic: moderators of COVID-related hardship, worry, and social support.

PURPOSE: Understanding people's response to the pandemic needs to consider individual differences in priorities and concerns. The present study sought to understand how individual differences in cognitive-appraisal processes might moderate the impact of three COVID-specific factors-hardship, worry, and social support-on reported depression. METHODS: This longitudinal study of the psychosocial impact of the COVID-19 pandemic included 771 people with data at three timepoints over 15.5 months. Participants were recruited from panels of chronically ill or general population samples. Depression was measured by an item response theory validated depression index created using items from existing measures that reflected similar content to the Patient Health Questionnaire-8. COVID-specific factors of hardship, worry, and social support were assessed with items compiled by the National Institutes of Health. The Quality of Life Appraisal Profilev2 Short-Form assessed cognitive appraisal processes. A series of random effects models examined whether appraisal moderated the effects of hardship, worry, and social support on depression over time. RESULTS: Over time the association between low social support and depression was greater (p = 0.0181). Emphasizing the negative was associated with exacerbated depression, in particular for those with low social support (p = 0.0007). Focusing on demands and habituation was associated with exacerbated depression unless one experienced greater hardship (p = 0.0074). There was a stronger positive connection between recent changes and depression for those people with higher worry scores early in the pandemic as compared to later, but a stronger positive correlation for those with lower worry scores later in the pandemic (p = 0.0015). Increased endorsement of standards of comparison, emphasizing the negative, problem goals, and health goals was associated with worse depression scores (all p < 0.0001). People who were younger, disabled, or had greater difficulty paying bills also reported worse depression (p < 0.0001, 0.0001, and 0.002, respectively). CONCLUSION: At the aggregate level, COVID-specific stressors changed over the course of the pandemic, whereas depression and social-support resources seemed stable. However, deeper analysis revealed substantial individual differences. Cognitive-appraisal processes showed considerable variability across individuals and moderated the impact of COVID-specific stressors and resources over time. Future work is needed to investigate whether coaching individuals away from maladaptive cognitive-appraisal processes can reduce depression and lead to better overall well-being.

Response-shift effects in childhood cancer survivors: A prospective study.

BACKGROUND: Treatment-related late effects can worsen over time among cancer survivors. Such worsening health states may trigger changes in internal standards, values, or conceptualization of quality-of-life (QOL). This "response-shift" phenomenon can jeopardize the validity of QOL assessment, and misrepresent QOL comparisons over time. This study tested response-shift effects in reporting future-health concerns among childhood cancer survivors who experienced progression in chronic health conditions (CHCs). METHODS: 2310 adult survivors of childhood cancer from St. Jude Lifetime Cohort Study completed a survey and clinical assessment at two or more timepoints. Based on 190 individual CHCs graded for adverse-event severity, global CHC burden was classified as "progression" or "non-progression". QOL was assessed using the SF-36TM eight domains and physical- and mental-component summary scores (PCS, MCS). A single global item measured concerns about future health. Random-effects models comparing survivors with and without progressive global CHC burden (progressors vs. non-progressors) evaluated response-shift effects (recalibration, reprioritization, reconceptualization) in reporting future-health concerns. RESULTS: Compared with non-progressors, progressors were more likely to de-emphasize (or downplay) overall physical and mental health in evaluating future-health concerns (p-values<0.05), indicating recalibration response-shift, and more likely to de-emphasize physical health earlier rather than later in follow-up (p-value<0.05), indicating reprioritization response-shift. There was evidence for a reconceptualization response-shift with progressor classification associated with worse-than-expected future-health concerns and physical health, and better-than-expected pain and role-emotional functioning (p-values<0.05). CONCLUSION: We identified three types of response-shift phenomena in reporting concerns about future health among childhood cancer survivors. Survivorship care or research should consider response-shift effects when interpreting changes in QOL over time.

Predictors of hospital-acquired pressure injuries in patients with complete spinal cord injury: a retrospective case-control study.

BACKGROUND: Despite current best practices, pressure injuries (PI) remain a devastating and prevalent hospital-acquired complication for patients with acute traumatic spinal cord injuries (SCIs). This study examined associations between risk factors for PI development in patients with complete SCI, such as norepinephrine dose and duration, and other demographic factors or lesion characteristics. METHODS: This case-control study included adults with acute complete SCIs ASIA-A, who were admitted to a level-one trauma center between 2014-18. A retrospective review was implement using data on patient and injury characteristics, including age, gender, level of SCI (cervical vs. thoracic), Injury Severity Score (ISS), length of stay (LOS) and mortality; presence/absence of PI during their acute hospital stay; and treatment factors such as spinal surgery, mean arterial pressure (MAP) targets, and vasopressor treatment. Multivariable logistic regression evaluated associations with PI. RESULTS: Eighty-two out of 103 eligible patients had complete data, and 30 (37%) developed PIs. Patient and injury characteristics, including age (Mean: 50.6; SD:21.3), location of SCI (48 cervical, 59%) and ISS (Mean 33.1; SD:11.8), did not differ between PI and non-PI groups. Logistic regression analysis revealed that male gender (OR:34.1; CI95:2.3-506.5, p = 0.010) and increased LOS (log-transformed; OR:20.5, CI95:2.8-149.9, p = 0.003) were associated with increased risk of PI. Having an order for a MAP > 80mmg (OR:0.05; CI95:0.01-0.30, p = 0.001) was associated with a reduced risk of PI. There were no significant associations between PI and duration of norepinephrine treatment. CONCLUSIONS: Norepinephrine treatment parameters were not associated with development of PI, suggesting that MAP targets should be a focus for future investigations for SCI management. Increasing LOS should highlight the need for high-risk PI prevention and vigilance.

If it's information, it's not "bias": a scoping review and proposed nomenclature for future response-shift research.

BACKGROUND: The growth in response-shift methods has enabled a stronger empirical foundation to investigate response-shift phenomena in quality-of-life (QOL) research; but many of these methods utilize certain language in framing the research question(s) and interpreting results that treats response-shift effects as "bias," "noise," "nuisance," or otherwise warranting removal from the results rather than as information that matters. The present project will describe the various ways in which researchers have framed the questions for investigating response-shift issues and interpreted the findings, and will develop a nomenclature for such that highlights the important information about resilience reflected by response-shift findings. METHODS: A scoping review was done of the QOL and response-shift literature (n = 1100 articles) from 1963 to 2020. After culling only empirical response-shift articles, raters characterized how investigators framed and interpreted study research questions (n = 164 articles). RESULTS: Of 10 methods used, papers using four of them utilized terms like "bias" and aimed to remove response-shift effects to reveal "true change." Yet, the investigators' reflections on their own conclusions suggested that they do not truly believe that response shift is error to be removed. A structured nomenclature is proposed for discussing response-shift results in a range of research contexts and response-shift detection methods. CONCLUSIONS: It is time for a concerted and focused effort to change the nomenclature of those methods that demonstrated this misinterpretation. Only by framing and interpreting response shift as information, not bias, can we improve our understanding and methods to help to distill outcomes with and without response-shift effects.

"Looking Under the Hood" of Anchor-Based Assessment of Clinically Important Change: A Machine Learning Approach.

OBJECTIVES: The Global Assessment of Change (GAC) item has facilitated the interpretation of change in patient-reported outcomes, providing an anchor for computing minimally important differences. Construct validity has been documented via disease-specific patient-reported outcomes change. We examined what domains, sociodemographic characteristics, attributions of change, and cognitive-appraisal processes are reflected in GAC ratings. METHODS: This secondary analysis examined data from 1,481 chronically ill patients and caregivers surveyed at baseline and 17 months. Items queried change since baseline in overall disease symptoms (GAC) and in physical, emotional, and social functioning. Candidate predictors included sociodemographic factors, health-related quality-of-life domains, change attributions, and quality-of-life appraisal processes. Least absolute shrinkage and selection operator and bootstrapping tested 77 predictors' effectiveness and stability. RESULTS: GAC worsening was notably associated with being disabled (ß = -0.24) and having difficulty paying bills (ß = -0.13). GAC was better explained by the physical domain than the emotional or social (ß = 0.67, 0.10, and 0.03, respectively; R2adj = 0.63) after sociodemographic-covariate adjustment. In a separate model (R2adj = 0.18), GAC variance was explained by attributions about changing health and changing response of one's health team, goals related to solving healthcare problems and maintaining activities, and appraisal about things getting better (ß = -0.14, 0.08, -0.07, 0.05, 0.21, respectively; prange ~0.0005-0.05) after adjustment. CONCLUSIONS: The GAC primarily reflects the physical domain, and the GAC reflects attributions, goals, and patterns of emphasis related to change in health and healthcare. Commonly unmeasured factors have some bearing on GAC scores and can facilitate the interpretation of change.

No impact of Asian ethnicity on EORTC QLQ-C30 scores: Group differences and differential item functioning in paroxysmal nocturnal hemoglobinuria.

BACKGROUND: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, life-threatening terminal-complement-mediated disease resulting in intravascular hemolysis and thrombosis with significant morbidity and premature mortality. There exists no disease-specific quality-of-life (QOL) measure for PNH. Its QOL effects resemble those of hematologic cancers, which supports the use of cancer-specific QOL measures in PNH clinical trials. The European Organisation for Research and Treatment of Cancer (EORTC) QLQ-C30 has published norms for many European and North American countries, but not for Asian countries. We investigated differences by Asian ethnicity in scores and item function on the EORTC QLQ-C30. METHODS: This secondary analysis focused on two non-inferiority PNH trials (301 and 302) comparing eculizumab and ravulizumab (n = 441). Analysis of covariance examined the main effect of Asian ethnicity on baseline EORTC QLQ-C30 scores, after adjusting for propensity scores encompassing trial, demographic and clinical factors. Mixed modeling of longitudinal data compared subscale scores in Asian vs. non-Asian patients, after propensity adjustment. Differential item function (DIF) was examined using ordinal regression models at baseline and longitudinally, to predict item score from total score, ethnicity, and their interaction to test for uniform DIF (significant main effect for Asian) and non-uniform DIF (significant Asian-by-total-score interaction). RESULTS: Of the 15 baseline domains, Asian patients scored slightly better on role and emotional functioning and slightly worse on constipation and diarrhea (0.22 < Cohen's d < 0.36). In longitudinal models, Asians reported slightly higher appetite loss, diarrhea, and financial difficulties than non-Asians (R2 increment < 0.0005). There was negligible uniform and non-uniform DIF, i.e., R2 0 to 0.018, far below Zumbo's (1999) criterion of 0.13. On average there were larger differences from norms for Asians (mean = 0.05, sd = 0.44) than non-Asians (mean = -0.07, sd = 0.36), but the size and direction of the differences varied considerably by domain, age, and gender. CONCLUSION: When compared to norms, Asian patients showed no systematic biases. DIF results supported this finding. We conclude that Asian ethnicity does not impact interpretation of EORTC QLQ-C30 scores.

Response-shift effects in neuromyelitis optica spectrum disorder: a secondary analysis of clinical trial data.

BACKGROUND: Researchers have long posited that response-shift effects may obfuscate treatment effects. The present work investigated possible response-shift effects in a recent clinical trial testing a new treatment for Neuromyelitis Optica Spectrum Disorder (NMOSD). This pivotal trial provided impressive support for the drug Eculizumab in preventing relapse, but less strong or null results as the indicators became more subjective or evaluative. This pattern of results suggests that response-shift effects are present. METHODS: This secondary analysis utilized data from a randomized, double-blind trial evaluating the impact of Eculizumab in preventing relapses in 143 people with NMOSD. Treatment arm and then relapse status were hypothesized 'catalysts' of response shift in two series of analyses. We devised a "de-constructed" version of Oort structural-equation modeling using random-effects modeling for use in small samples. This method begins by testing an omnibus response-shift hypothesis and then, pending a positive result, implements a series of random-effects models to elucidate specific response-shift effects. RESULTS: In the omnibus test, the 'standard quality-of-life (QOL) model' captured substantially less well the experience of placebo as compared to Eculizumab group. Recalibration and reconceptualization response-shift effects were detected. Detected relapse-related response shifts included recalibration, reprioritization, and reconceptualization. CONCLUSIONS: Trial patients experienced response shifts related to treatment- and relapse-related experiences. Published trial results likely under-estimated Eculizumab vs. Placebo differences due to recalibration and reconceptualization, and relapse effects due to recalibration, reprioritization, and reconceptualization. This novel random-effects- model application builds on response-shift theory and provides a small-sample method for better estimating treatment effects in clinical trials.

Response-shift effects in neuromyelitis optica spectrum disorder: estimating response-shift-adjusted scores using equating.

BACKGROUND: In our companion paper, random intercept models (RIMs) investigated response-shift effects in a clinical trial comparing Eculizumab to Placebo for people with neuromyelitis optica spectrum disorder (NMOSD). RIMs predicted Global Health using the EQ-5D Visual Analogue Scale item (VAS) to encompass broad criteria that people might consider. The SF36™v2 mental and physical component scores (MCS and PCS) helped us detect response shift in VAS. Here, we sought to "back-translate" the VAS into the MCS/PCS scores that would have been observed if response shift had not been present. METHODS: This secondary analysis utilized NMOSD clinical trial data evaluating the impact of Eculizumab in preventing relapses (n = 143). Analyses began by equating raw scores from the VAS, MCS, and PCS, and computing scores that removed response-shift effects. Correlation analysis and descriptive displays provided a more comprehensive examination of response-shift effects. RESULTS: MCS and PCS crosswalks with VAS equated the scores that include and exclude response-shift effects. These two sets of scores had low shared variance for MCS for both groups, suggesting that corresponding mental health constructs were substantially different. The shared variance contrast for physical health was distinct only for the Placebo group. The larger MCS response-shift effects were found at end of study for Placebo only and were more prominent at extremes of the MCS score distribution. CONCLUSIONS: Our results reveal notable treatment group differences in MCS but not PCS response shifts, which can explain null results detected in previous work. The method introduced herein provides a way to provide further information about response-shift effects in clinical trial data.

Patient Factors That Matter in Predicting Hip Arthroplasty Outcomes: A Machine-Learning Approach.

BACKGROUND: Despite the success of total hip arthroplasty (THA), approximately 10%-15% of patients will be dissatisfied with their outcome. Identifying patients at risk of not achieving meaningful gains postoperatively is critical to pre-surgical counseling and clinical decision support. Machine learning has shown promise in creating predictive models. This study used a machine-learning model to identify patient-specific variables that predict the postoperative functional outcome in THA. METHODS: A prospective longitudinal cohort of 160 consecutive patients undergoing total hip replacement for the treatment of degenerative arthritis completed self-reported measures preoperatively and at 3 months postoperatively. Using four types of independent variables (patient demographics, patient-reported health, cognitive appraisal processes and surgical approach), a machine-learning model utilizing Least Absolute Shrinkage Selection Operator (LASSO) was constructed to predict postoperative Hip Disability and Osteoarthritis Outcome Score (HOOS) at 3 months. RESULTS: The most predictive independent variables of postoperative HOOS were cognitive appraisal processes. Variables that predicted a worse HOOS consisted of frequent thoughts of work (ß = -0.34), frequent comparison to healthier peers (ß = -0.26), increased body mass index (ß = -0.17), increased medical comorbidities (ß = -0.19), and the anterior surgical approach (ß = -0.15). Variables that predicted a better HOOS consisted of employment at the time of surgery (ß = 0.17), and thoughts related to family interaction (ß = 0.12), trying not to complain (ß = 0.13), and helping others (ß = 0.22). CONCLUSIONS: This clinical prediction model in THA revealed that the factors most predictive of outcome were cognitive appraisal processes, demonstrating their importance to outcome-based research. LEVEL OF EVIDENCE: Prognostic Level 1.

Moving toward better health: exercise practice is associated with improved outcomes after spine surgery in people with degenerative lumbar conditions.

Background: Recovery and rehabilitation following surgery can take many months. Understanding what patients can do to facilitate recovery would be beneficial for spinal surgeons. This study sought to evaluate the impact of exercise practice, before and after surgery, on long-term outcomes of spine surgery in a robust clinical sample. Methods: This prospective longitudinal cohort study included adult patients undergoing spinal surgery for degenerative spinal conditions. Patients were administered a survey that included preoperative and postoperative exercise practices and the following patient-reported outcome measures: the physical component score (PCS) and mental component score (MCS) of the Medical Outcomes Study 36-Item Short Form Survey (Rand-36), the Oswestry Disability Index (ODI) score, the Numeric Rating Scale (NRS) score for pain and the Patient-Reported Outcome Measurement Information System (PROMIS) Pain Interference Short Form score. Random effects models investigated the relationship of exercise, follow-up time and their interaction in predicting each patient-reported outcome measure over time, with and without sociodemographic covariates. Results: There were 168 patients in the study sample with up to 12 months of follow-up data. Analysis revealed modest significant main effects of exercise on PCS, MCS, ODI and PROMIS scores and main effects of time on all outcomes. The exercise-by-time interaction was significant in predicting the trajectories of the ODI and MCS scores. When full models were adjusted for education and employment status, interaction effects were no longer significant, but exercise main effects remained significant for ODI score. Conclusion: Patients who engage in exercise before and after spine surgery have better mental health and spine-specific recovery trajectories than those who do not. All health care providers should encourage patients to exercise while they are waiting for surgery within preoperative limitations and as soon as they are able after surgery and to continue this over the long term.

Contexte: Le rétablissement et la réadaptation postopératoires s'échelonnent parfois sur plusieurs mois. Comprendre ce que les patients peuvent faire pour faciliter leur rétablissement serait utile aux spécialistes de la chirurgie de la colonne vertébrale. Cette étude a voulu évaluer l'impact de la pratique d'exercices avant et après une chirurgie de la colonne vertébrale sur son issue à long terme dans un solide échantillon clinique. Méthodes: Cette étude de cohorte longitudinale prospective a regroupé des patients adultes qui devaient subir une chirurgie de la colonne vertébrale pour des maladies dégénératives. Les patients ont été invités à répondre à un questionnaire qui portait entre autre sur la pratique d'exercices pré- et postopératoires et sur les paramètres autorapportés suivants : scores aux composantes physique (PCS) et mentale (MCS) du questionnaire SF-36 (Medical Outcomes Study 36-Item Short Form Survey [Rand-36]), à l'échelle d'incapacité d'Oswestry (ODI), à une échelle d'évaluation numérique (ÉÉN) de la douleur et au questionnaire court PROMIS (Patient-Reported Outcome Measurement Information System) sur l'interférence de la douleur. Des modèles à effets aléatoires ont permis d'analyser les liens entre l'exercice, la durée du suivi et leur interaction pour ce qui est de prédire chacun des paramètres autorapportés au fil du temps, avec et sans les covariables sociodémographiques. Résultats: L'étude a regroupé 168 patients et les données pour un suivi allant jusqu'à 12 mois. L'analyse a fait état d'effets majeurs significatifs modestes de l'exercice sur les paramètres PCS, MCS, ODI et PROMIS et d'effets majeurs du temps sur tous les paramètres. L'interaction entre exercices et durée du suivi a été significative pour prédire la trajectoire des scores ODI et MCS. Lorsque les modèles intégraux ont été ajustés pour tenir compte du niveau de scolarité et du statut professionnel, les effets de l'interaction n'étaient plus significatifs, mais les effets majeurs de l'exercice sont demeurés significatifs pour le score ODI. Conclusion: Les patients qui ont fait des exercices avant et après une chirurgie de la colonne vertébrale ont présenté des trajectoires plus favorables au plan de la santé mentale et du rétablissement de leur colonne vertébrale comparativement à ceux qui n'avaient pas fait d'exercices. Tous les professionnels de la santé devraient encourager les patients à faire des exercices pendant qu'ils attendent leur chirurgie en tenant compte de leurs limites préopératoires, à les reprendre le plus rapidement possible après la chirurgie et à les maintenir à long terme.

Positive emotions despite substantial burden: The alchemy of haemophilia caregiving.

INTRODUCTION: Qualitative interviews when developing the haemophilia caregiver impact measure© (HCI) documented the importance of capturing the positive aspects of caregiving, not just the negative. AIM: The present study thus investigates the construct underlying the positive emotions HCI subscale and tests models proposing a more comprehensive way of thinking about this construct. METHODS: Secondary analysis was implemented on longitudinal web-based survey data (n = 323) from haemophilia A or haemophilia B caregivers. Person-reported outcomes (PROs) included the HCI, the PROMIS-10 and Ryff psychological well-being subscales. Predictors included caregiver demographics; patient haemophilia characteristics; exercise; adherence; and quality-of-life (QOL) appraisal processes as measured by the brief appraisal inventory (BAI) which yields composite scores assessing awareness of challenges, fulfillment and growth, stay positive, social comparison and interpersonal problem-solving. Second-order factor analysis, structural equation modelling and residual modelling were implemented. RESULTS: A structural equation model fit the data well that contained bifactor representation of well-being with a general factor comprised of environmental mastery, positive relations with others, physical functioning and emotional functioning. Positive emotions was modelled as a component of well-being, with a unique component ('Alchemy') characterized by its associations with stay positive, and awareness of challenges appraisals, and difficulty paying bills. Alchemy had positive linear relationships with the first two, and a positive quadratic relationship with difficulty paying bills. CONCLUSIONS: Adopting positive-focused ways of thinking about one's life limitations may transform the negatives of haemophilia caregiving into something positive. Such cognitive habits reflect an awareness and acceptance of the limitations imposed by haemophilia caregiving.

Lung transplant referral practice patterns: a survey of cystic fibrosis physicians and general pulmonologists.

BACKGROUND: Many individuals with cystic fibrosis (CF) die from respiratory failure without referral for lung transplant. Physician practices that may expedite, delay, or preclude referral, are poorly understood. METHODS: Two parallel, web-based surveys focusing on lung transplant referral triggers and barriers, as well as pre-referral evaluation, were emailed to pulmonologists practicing in the New England region. One questionnaire was sent to CF providers (n = 61), and the second to general pulmonary providers practicing at the same institutions (n = 61). RESULTS: There were 43 (70%) responses to the CF provider survey, and 25 (41%) responses to the general pulmonary ('non-CF') provider survey. Primary reasons for CF providers to refer their patients included: rapidly declining lung function (91%) and a forced expiratory volume in 1 s (FEV1) below 30% predicted (74%). The greatest barriers to referral for both CF and non-CF providers included active tobacco use (65 and 96%, respectively, would not refer), and active alcohol or other substance use or dependence (63 and 80%). Furthermore, up to 42% of CF providers would potentially delay their referral if triple-combination therapy or other promising new, disease-specific therapy were anticipated. In general, non-CF providers perform a more robust pre-referral medical work-up, while CF providers complete a psychosocial evaluation in higher numbers. Across both groups, communication with lung transplant programs was reported to be inadequate. CONCLUSIONS: Physician-level barriers to timely lung transplant referral exist and need to be addressed. Enhanced communication between lung transplant programs and pulmonary providers may reduce these barriers.

Perceptions of Response Burden Associated with Completion of Patient-Reported Outcome Assessments in Oncology.

BACKGROUND: Patient response burden is often raised as a human subject concern in consideration of the length or complexity of patient-reported outcome (PRO) instruments used in oncology. OBJECTIVES: To quantify patient response burden and identify its predictive factors. METHODS: Data were collected presurgically during a prospective trial that used a comprehensive symptom and health-related quality-of-life (HRQOL) PRO assessment. A subset of patients also completed HRQOL interviews. Response burden was captured using an internally developed six-item instrument. Demographic and clinical characteristics as well as HRQOL scores were examined as potential predictors using hierarchical regression. Response burden was used to predict participant dropout at the first follow-up interval. RESULTS: A total of 275 patients (mean age 67.5 years; 23.6% female) completed surveys (n = 126) or surveys in addition to interviews (n = 149). Patients experienced low response burden (mean 12.19 ± 11.65). Repetitive questions were identified by 60 patients (21.8%), whereas 31.6% indicated that additional information should be gathered; 35 patients (12.7%) identified repetitive questions and expressed a desire for additional items. Low self-reported cognitive function was a significant predictor of higher response burden (ß = -0.20; t(270) = -3.38; P = 0.01; model-adjusted R2 = 0.04). Response burden was not a significant predictor of study dropout. CONCLUSIONS: Despite completing a large battery of PRO measures and interviews, patients reported minimal response burden, with nearly one-third expressing that more questions should have been asked. Patients with lower cognitive function are more likely to report higher response burden when completing PRO measures. Further examination of patient characteristics related to response burden may reveal useful pathways for tailoring patient-centered interventions.

Advancing quality-of-life research by deepening our understanding of response shift: a unifying theory of appraisal.

Studies of response-shift phenomena in quality-of-life (QOL) research have grown steadily in the more than two decades of research. As a field, we have been calling a lot of different approaches "response shift" over the years, but the only approach that fully embodies the foundational (Sprangers and Schwartz, Soc Sci Med 48(11):1507-1515, 1999) theoretical model is appraisal. According to the definition proposed in this model, response shift is about individual cognitive change. This paper presents the argument that all response-shift research models need to be grounded in an understanding of cognitive appraisal. We present a reasoned argument for why appraisal is fundamental to QOL response-shift research. We describe current measurement and analytic methods for working with appraisal, and how these methods can be integrated into the current response-shift statistical 'tool box.' We propose future research directions on theory, methods, and cross-calibration of group- and individual-level methods. There are currently three tools available in multiple languages for assessing QOL appraisal processes. They have been tested and used to assess response shift in empirical studies of ~ 7000 people with chronic illness. The study findings illustrate how appraisal theory and methods can facilitate methodological investigations of and to enhance other response-shift detection methods. Future research directions are proposed to enrich QOL theory, response-shift methods, and interpretation of QOL change over time. Appraisal theory and methods are the closest approximation to a response-shift 'gold standard.' They provide the foundation for understanding response shift and point to a unified theory of QOL.

Longitudinal validity of the hemophilia caregiver impact measure.

INTRODUCTION: The hemophilia caregiver impact (HCI) measure is a 36-item self-report tool that has documented reliability and validity in a large cross-sectional study, but its longitudinal construct validity is unknown. This study's objective was to evaluate the responsiveness of the HCI to clinically important change, and to provide interpretation guidelines. METHODS: This web-based study invited 458 hemophilia caregivers involved in the HCI's validation study to provide follow-up data. Measures included the HCI, and a Likert item querying Global Assessment of Change (GAC) for caregiver burden. Responsiveness was estimated using anchor- and distribution-based methods. The anchor-based method computed the minimally important difference (MID) by computing the mean change separately for those who reported lesser or more caregiver burden on the GAC. The distribution-based method computed the Modified Standardized Response Mean (MSRM) separately for people who reporting reduced or increased burden as compared to the 'same' groups. RESULTS: The study sample included 323 caregivers (71% response rate), with mean follow-up of 21.9 months. The HCI Burden Summary score and all negative-burden subscales but not the Positive Emotions subscale evidenced responsiveness to clinically important differences, showing statistically significant differences by transition group. The MIDs were relatively small mean changes over time (e.g., Burden Summary MID ranged from - 2.2 to 2.6, for reduced versus increased burden), and the MSRMs were small effect sizes. The Burden Summary score was equally sensitive to reduced versus increased burden (MSRM of - 0.32 and 0.35, respectively). CONCLUSIONS: The HCI demonstrated longitudinal construct validity. The HCI shows promise for clinical hemophilia studies as a caregiver-based tool for evaluating treatments.

Adherence in Youth With Multiple Sclerosis: A Qualitative Assessment of Habit Formation, Barriers, and Facilitators.

Rates of medication nonadherence in youth with multiple sclerosis (MS) range from 10% to 60%. Qualitative studies of adherence can provide insight into children's own perspectives about barriers and facilitators to their adherence and inform future interventions. This qualitative longitudinal descriptive study included children with MS ( n = 28) participating in a randomized controlled trial focused on medication adherence ( clinicaltrials.gov : NCT02234713). Following established methods, three independent reviewers coded transcripts of motivational interviewing (MI) sessions (three interviews per subject, performed monthly over a 3-month period) for relevant themes. They were subsequently categorized using inductive content analysis. Youth described medication adherence as being dependent on the ability to build and maintain healthy habits related to medication use, including embodiment of these habits. Barriers and facilitators included remembering/forgetting, experiences with fatigue, and experiences with medication. These themes were maintained through the second and third interviews. Future research focus on barriers and facilitators to habit maintenance in this population.

Risk factors for non-adherence to disease-modifying therapy in pediatric multiple sclerosis.

BACKGROUND: Adherence to disease-modifying therapies (DMTs) in pediatric multiple sclerosis (MS) is not well understood. We examined the prevalence and risk factors for poor adherence in pediatric MS. METHODS: This cross-sectional study recruited youth with MS from 12 North American pediatric MS clinics. In addition to pharmacy-refill data, patients and parents completed self-report measures of adherence and quality of life. Additionally, patients completed measures of self-efficacy and well-being. Factor analysis and linear regression methods were used. RESULTS: A total of 66 youth (mean age, 15.7 years) received MS DMTs (33% oral, 66% injectable). Estimates of poor adherence (i.e. missing >20% of doses) varied by source: pharmacy 7%, parent 14%, and patient 41%. Factor analysis yielded two composites: adherence summary and parental involvement in adherence. Regressions revealed that patients with better self-reported physical functioning were more adherent. Parents were more likely to be involved in adherence when their child had worse parent-reported PedsQL School Functioning and lower MS Self-Efficacy Control. Oral DMTs were associated with lesser parental involvement in adherence. CONCLUSION: Rates of non-adherence varied by information source. Better self-reported physical functioning was the strongest predictor of adherence. Parental involvement in adherence was associated with worse PedsQL School Functioning and lower MS Self-Efficacy-measured confidence in controlling MS.