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Background: The majority of persons with Huntington disease (HD) experience mental health symptoms. Patient-reported outcome (PRO) measures are capable of capturing unobservable behaviors and feelings relating to mental health. The current study aimed to test the reliability and responsiveness to self-reported and clinician-rated change over time of Neuro-QoL and PROMIS mental health PROs over the course of a 24-month period., Methods: At baseline, 12-months, and 24-months, 362 participants with premanifest or manifest HD completed the Neuro-QoL Depression computer adaptive test (CAT), PROMIS Depression short form (SF), Neuro-QoL Anxiety CAT, PROMIS Anxiety SF, PROMIS Anger CAT and SF, Neuro-QoL Emotional/Behavioral Dyscontrol CAT and SF, Neuro-QoL Positive Affect and Well-Being CAT and SF, and Neuro-QoL Stigma CAT and SF. Participants completed several clinician-administered measures at each time point, as well as several global ratings of change at 12- and 24-months. Reliability (test-retest reliability and measurement error) and responsiveness (using standardized response means and general linear models) were assessed., Results: Test-retest reliability and measurement error were excellent for all PROs (all ICC ≥ .90 for test-retest reliability and all SEM percentages ≤ 6.82%). In addition, 12- and 24-month responsiveness were generally supported for the Neuro-QoL and PROMIS mental health PROs; findings relative to clinician-rated anchors of change (e.g., SRMs for the group with declines ranged from .38 to .91 for 24-month change and .09 to .45, with the majority above .25 for 12-month change) were generally more robust than those relative to self-reported anchors of change (e.g., SRMs for the group with declines ranged from .02 to .75, with the majority above .39 for 24-month change and .09 to .45, with the majority above .16 for 12-month change)., Conclusions: The Neuro-QoL and PROMIS mental health PROs demonstrated strong psychometric reliability, as well as responsiveness to self-reported and clinician-rated change over time in people with HD.

Background: Huntington's disease (HD) is a neurological disorder that causes severe motor symptoms that adversely impact health-related quality of life. Patient-reported physical function outcome measures in HD have shown cross-sectional evidence of validity, but responsiveness has not yet been assessed., Objectives: This study evaluates the responsiveness of the Huntington Disease Health-Related Quality of Life (HDQLIFE) and the Quality of Life in Neurological Disorders (Neuro-QoL) physical function measures in persons with HD., Methods: A total of 347 participants completed baseline and at least 1 follow-up (12-month and 24-month) measure (HDQLIFE Chorea, HDQLIFE Swallowing Difficulties, HDQLIFE Speech Difficulties, Neuro-QoL Upper Extremity Function, and/or Neuro-QoL Lower Extremity Function). Of the participants that completed the baseline assessment, 338 (90.9%) completed the 12-month assessment, and 293 (78.8%) completed the 24-month assessment. Standardized response means and general linear models evaluated whether the physical function measures were responsive to self-reported and clinician-rated change over time., Results: Small to moderate effect sizes for the standardized response means supported 12-month and 24-month responsiveness of the HDQLIFE and Neuro-QoL measures for those with either self-reported or clinician-rated declines in function. General linear models supported 12-month and 24-month responsiveness for all HRQOL measures relative to self-reported declines in health, but generally only 24-month responsiveness was supported relative to clinician-rated declines in function., Conclusions: Longitudinal analyses indicate that the HDQLIFE and the Neuro-QoL physical function measures are sensitive to change over time in individuals with HD. Thus, these scales exhibit evidence of responsiveness and may be useful outcome measures in future clinical trials. © 2019 International Parkinson and Movement Disorder Society., (© 2019 International Parkinson and Movement Disorder Society.)

Background: Social health is an important concern in persons with Huntington's disease (HD); however, there is little literature examining this construct in this population., Objective: While cross-sectional data supports the clinical utility of two Neuro-QoL social health measures in persons with HD, data is still needed to establish their longitudinal validity., Methods: Participants (N = 358) completed baseline and at least one follow-up (12- and 24-month) assessment that included the completion of Neuro-QoL Social Health computer adaptive tests (CATs) and short forms (for Ability to Participate in Social Roles and Activities [SRA] and Satisfaction with SRA). Test-retest reliability was examined using intra class correlations, and one-way ANOVAs with Bonferroni post-hoc contrasts were used to determine whether there were group differences among premanifest, early- and late-stage HD participants on the Social health measures. In addition, standardized response means were used to examine longitudinal responsiveness, and mixed or general linear models were used to examine change over time (relative to self-reported change on an associated anchor item about social health and clinician-rated change based on Total Functional Capacity scores from the UHDRS)., Results: Test-retest reliability of the measures was excellent (ICCs ranged from 0.82 to 0.87 across the different measures) and persons with greater disease burden reported more problems with social health than those at earlier stages in the disease process (all p < 0.0001). Responsiveness was supported for all measures except the Ability to Participate in SRA CAT; participants who had self-reported or clinician-rated declines in health generally had 12- and 24-month declines on the Neuro-QoL measures., Conclusions: Findings indicate that these measures may be useful for studies attempting to assess change in social health over time.

Objective: To develop an item response theory (IRT)-based patient-reported outcome measure of functional communication for adults with traumatic brain injury (TBI)., Setting: Five medical centers that were TBI Model Systems sites., Participants: A total of 569 adults with TBI (28% complicated-mild; 13% moderate; and 58% severe)., Design: Grounded theory-based qualitative item development, large-scale item calibration testing, confirmatory factor analyses, psychometric analyses with graded response model IRT., Main Measure: Traumatic Brain Injury-Quality of Life (TBI-QOL) Communication Item Bank, version 1.0., Results: From an initial pool of 48 items, 31 items were retained in the final instrument based on adequate fit to a unidimensional model and absence of bias across several demographic and clinical subgroupings. The TBI-QOL Communication Item Bank demonstrated excellent score precision (reliability ≥ 0.95) across a wide range of communication impairment levels, particularly for individuals with more severe difficulties. The TBI-QOL Communication Item Bank is available as a full item bank, fixed-length short form, and as a computerized adaptive test., Conclusions: The TBI-QOL Communication Item Bank permits precise measurement of patient-reported functional communication after TBI. Future development will validate the instrument against performance-based, clinician-reported, and surrogate-reported assessments.

Objective: To develop, calibrate, and evaluate the test-retest reliability of a new patient-reported outcome measure of headache pain relevant for individuals with traumatic brain injury (TBI)., Setting: Six TBI Model Systems rehabilitation centers in the United States., Participants: Adults with medically confirmed documentation of TBI., Design: Cross-sectional calibration field testing and test-retest reliability analyses., Main Measures: Traumatic Brain Injury-Quality of Life Headache Pain item bank., Results: Thirteen headache pain items were calibrated as a unidimensional measure using data from 590 participants. The new measure was reliable (α = .98; item-total correlation range: 0.71-0.91). Item parameter estimates were estimated using Samejima's Graded Response Model and a 10-item calibrated short form was created. Simulation testing confirmed that both the computer-adaptive test and the short-form administrations were equivalent to the full item bank. One- to-2-week test-retest reliability of the computer-adaptive test was high (Pearson r and intraclass correlation coefficients = 0.81). Approximately two-thirds of the sample reported at least 1 headache symptom., Conclusion: The Traumatic Brain Injury-Quality of Life Headache Pain item bank and short form provide researchers and clinicians with reliable measures of the subjective experience of headache symptoms for individuals with a history of TBI.

Objective: To develop and calibrate new patient-reported outcome measures of cognitive concerns for individuals with traumatic brain injury (TBI)., Setting: Five TBI model systems rehabilitation centers in the United States., Participants: Adults with medically confirmed history of TBI., Design: Cross-sectional survey in interview format., Main Measures: Traumatic Brain Injury-Quality of Life (TBI-QOL) Executive Function and TBI-QOL Cognition-General Concerns item banks., Results: A total of 569 adults with complicated-mild, moderate, or severe TBI completed preliminary item pools, which included 65 Executive Function items and 56 Cognition-General Concerns items. Confirmatory factor analysis supported the retention of 37 Executive Function and 39 Cognition-General Concerns items. Samejima's graded response model was used to estimate item parameters for associated computer adaptive test administrations, and informed the selection of corresponding static short forms. Data from an independent sample of 77 adults with complicated-mild, moderate, or severe TBI supported the test-retest reliability of these newly developed measures., Conclusion: The TBI-QOL Executive Function and Cognition-General Concerns item banks provide researchers and clinicians with reliable tools for assessing patient-reported post-TBI cognitive difficulties as part of the comprehensive TBI-QOL measurement system.

Purpose: Patient-reported outcomes after radical prostatectomy have focused on erectile function. To date, no studies have validated the Patient-Reported Outcome Measurement Information System (PROMIS) Sexual Interest and Satisfaction single item measures in patients with prostate cancer, nor have studies examined how these measures relate to erectile function. In addition, data are lacking with regard to the clinical responsiveness of these measures to treatment. We sought to validate and examine the clinical utility of these measures in men after radical prostatectomy., Patients and Methods: We identified men who underwent a robotic radical prostatectomy from May 2014 to January 2016 in the Michigan Urological Surgery Improvement Collaborative. A single item from the PROMIS Global Satisfaction With Sex Life subdomain and a single item from the PROMIS Interest in Sexual Activity subdomain were administered. Erectile function was also assessed. Differences between baseline and 24-month T-scores for both PROMIS interest and satisfaction were examined. Multilevel models were constructed to examine change over time., Results: A total of 1,604 patients were included in the analysis. Convergent and discriminant validity of the PROMIS measures was supported. The mean PROMIS interest T-score decreased significantly from baseline to 3 months ( P = .001) and significantly increased from 3 months to 24 months in this cohort, with 24-month scores exceeding baseline scores ( P < .001). The mean PROMIS satisfaction T-score declined from baseline to 3 months and increased from 3 months to 24 months ( P < .002)., Conclusion: PROMIS Global Satisfaction With Sex Life and Interest in Sexual Activity single-item measures are fundamental measures in prostate cancer survivorship. Patients are interested in sex despite functional losses and can salvage satisfaction, thereby giving insight into attainable patient-centered survivorship goals for sexual recovery after radical prostatectomy.

Objective: Nephrotic syndrome (NS) is a kidney disease known to adversely impact health-related quality of life (HRQOL). Patient-reported outcome (PRO) measures are commonly used to characterize HRQOL and the patient disease experience. This study aims to improve the interpretability and clinical utility of the Patient-Reported Outcomes Measurement Information System® (PROMIS®) by identifying distinct meaningful HRQOL profiles in children and adults with NS., Method: Patients were from 2 prospective NS cohort studies (PROMIS-II®: 121 children; NEPTUNE: 40 children and 219 adults) with data from 6 PROMIS® domains. Latent Profile Analysis was used to identify subgroups of patients based on PROMIS® score patterns. A 3-step analysis of latent profile predictors was used to determine how clinical parameters predicted HRQOL profile membership., Results: We identified 3 HRQOL profiles (Good, Average, and Poor) with strong indicators of membership classification (entropy >0.86). Complete proteinuria remission, reduction in symptoms, and shorter disease duration, were significant predictors of better HRQOL profile membership., Conclusions: Patients with NS can be classified by HRQOL into clinically meaningful categories. Integrating this approach into clinic may help in the identification of individuals with poor HRQOL will help clinicians better manage their symptoms and researchers study the causes and possible interventions for these patients. PROMIS® HRQOL profiles were reproducible in replication cohorts. (PsycINFO Database Record (c) 2019 APA, all rights reserved).

Objectives: To establish the reliability and validity of the newly developed TBI-CareQOL patient-reported outcome (PRO) measures in caregivers of civilians and service members/veterans (SMVs) with traumatic brain injury (TBI) so that they can be used with confidence in clinical research and practice., Design: Computer-based surveys delivered through an on-line data capture platform., Setting: Three TBI Model Systems rehabilitation hospitals, an academic medical center, and a military medical treatment facility., Participants: Caregivers (N=560) of individuals with TBI; this included 2 different study samples: 344 caregivers of civilians with TBI and 216 caregivers of SMVs with TBI., Intervention: Not Applicable., Main Outcome Measures: Five Traumatic Brain Injury Caregiver Quality of Life (TBI-CareQOL) item banks., Results: Reliabilities for the TBI-CareQOL measures were excellent (all Cronbach's α >.88); 3-week test-retest reliability ranged from .75 to .90 across the 2 samples. Convergent validity was supported by moderate to high associations among the TBI-CareQOL measures and moderate correlations between the TBI-CareQOL measures and other measures of health-related quality of life (HRQOL) and caregiver burden. Discriminant validity was supported by low correlations between the TBI-CareQOL measures and less-related constructs (eg, caregiver satisfaction). Known-groups validity was supported: caregivers of individuals that were low functioning had worse HRQOL than caregivers of high-functioning individuals., Conclusions: Results provide psychometric support for the new TBI-CareQOL item banks. As such, these measures fill a significant gap in the caregiver literature where sensitive PRO measures that capture changes in HRQOL are needed to detect improvements for interventions designed to assist family caregivers., (Copyright © 2018 American Congress of Rehabilitation Medicine. All rights reserved.)

Objective: To examine the reliability and validity of the short form (SF) and computer adaptive test (CAT) versions of the Patient-Reported Outcomes Measurement Information System (PROMIS) measures of social health of caregivers of civilians and service members/veterans (SMVs) with traumatic brain injury (TBI)., Design: Self-report questionnaires administered through an online data collection platform., Setting: Hospital and community-based outreach at 3 TBI Model Systems rehabilitation hospitals, an academic medical center, and a military medical treatment facility., Participants: Caregivers (N=560) (344 civilians and 216 military) of individuals with a documented TBI., Intervention: Not applicable., Main Outcome Measures: A total of 5 PROMIS social health measures., Results: All 5 PROMIS social health measures exceeded the a priori criterion for internal consistency reliability (≥0.70); most PROMIS measures met the criterion for test-retest reliability (≥0.70) in the civilian sample; in the SMV sample, test-retest reliability was generally below this criterion, except for social isolation. For both samples, convergent validity was supported by moderate correlations between the 5 PROMIS social health measures and related measures, and discriminant validity was supported by low correlations between PROMIS social health measures and measures of dissimilar constructs. Most PROMIS scores indicated significantly worse social health in both samples of those caring for individuals who were low functioning. Finally, impairment rates in social health were elevated for those caring for low-functioning individuals, especially in the SMV sample., Conclusions: The PROMIS CAT and SF social health measures have potential clinical utility for use in caregivers of civilians and SMVs with TBI., (Copyright © 2018 American Congress of Rehabilitation Medicine. All rights reserved.)

Background: Patient-reported outcomes (PROs) for mental health are important for persons with Huntington disease (HD) who commonly experience symptoms of depression, anxiety, irritability, anger, aggression, and apathy. Given this, there is a need for reliable and valid patient-reported outcomes measures of mental health for use as patient-centered outcomes in clinical trials., Objective: Thus, the purpose of this study was to establish the psychometric properties (i.e., reliability and validity) of six Neuro-QoL and PROMIS mental health measures to support their clinical utility in persons with HD., Methods: 294 individuals with premanifest (n = 102) or manifest HD (n = 131 early HD; n = 61 late HD) completed Neuro-QoL/PROMIS measures of Emotional and Behavioral Dyscontrol, Positive Affect and Well-Being, Stigma, Anger, Anxiety, and Depression, legacy measures of self-reported mental health, and clinician-rated assessments of functioning., Results: Convergent validity and discriminant validity for the Neuro-QoL and PROMIS measures of Emotional and Behavioral Dyscontrol, Positive Affect and Well-Being, Stigma, Anger, Anxiety, and Depression, were supported in persons with HD. Neuro-QoL measures of Anxiety and Depression also demonstrated moderate sensitivity and specificity (i.e., they were able to distinguish between individuals with and without clinically significant anxiety and depression)., Conclusions: Findings provide psychometric support for the clinical utility of the Neuro-QoL/PROMIS measures of mental health measures in persons with HD. As such, these measures should be considered for the standardized assessment of health-related quality of life in persons with HD.

Purpose: Symptom progression in Huntington disease (HD) is associated with cognitive decline which may interfere with the self-report of symptoms. Unfortunately, data to support or refute the psychometric reliability of patient-reported outcomes (PROs) as HD progresses are limited. This is problematic given that PROs are increasingly recognized as important measures of efficacy for new treatments., Methods: We examined PRO data from the HDQLIFE Measurement System (Speech Difficulties; Swallowing Difficulties; Chorea) in 509 individuals with premanifest, early-stage, or late-stage HD. Clinician-administered assessments of motor functioning (items from the UHDRS) and standardized objective assessments of cognition (Stroop, Symbol Digit Modalities) were also collected. We examined item bias using differential item functioning (DIF) across HD stage (premanifest, early-, late-) and relative to cognitive performance. We also examined the correlations between self-report and clinician ratings. Regression models that considered total cognitive ability were utilized to determine psychometric reliability of the PROs., Results: Most PRO items were free from DIF for both staging and cognition. There were modest correlations between PROs and clinician report (ranged from - 0.40 to - 0.60). Modeling analyses indicated that psychometric reliability breaks down with poorer cognition and more progressed disease stage; split-half reliability was compromised (i.e., split-half reliability < 0.80) when scores were < 136 for Chorea, < 109 for Speech Difficulties, and < 179 for Swallowing Difficulties., Conclusions: Results indicate that the psychometric reliability of PROs can be compromised as HD symptoms progress and cognition declines. Clinicians should consider PROs in conjunction with other types of assessments when total cognition scores exceed critical thresholds.

Background: Clinician-rated measures of functioning are often used as primary endpoints in clinical trials and other behavioral research in Huntington disease. As study costs for clinician-rated assessments are not always feasible, there is a question of whether patient self-report of commonly used clinician-rated measures may serve as acceptable alternatives in low risk behavioral trials., Aim: The purpose of this paper was to determine the level of agreement between self-report and clinician-ratings of commonly used functional assessment measures in Huntington disease., Design: 486 participants with premanifest or manifest Huntington disease were examined. Total Functional Capacity, Functional Assessment, and Independence Scale assessments from the Unified Huntington Disease Rating scale were completed by clinicians; a self-report version was also completed by individuals with Huntington disease. Cronbach's α was used to examine internal consistency, one-way analysis of variance was used to examine group differences, and paired t tests, kappa agreement coefficients, and intra-class correlations were calculated to determine agreement between raters., Results: Internal consistency for self-reported ratings of functional capacity and ability were good. There were significant differences between those with premanifest, early-, and late-stage disease; those with later-stage disease reported less ability and independence than the other clinical groups. Although self-report ratings were not a perfect match with associated clinician-rated measures, differences were small. Cutoffs for achieving specified levels of agreement are provided., Conclusions: Depending on the acceptable margin of error in a study, self-reported administration of these functional assessments may be appropriate when clinician-related assessments are not feasible.

Social functioning is an essential but poorly understood component of health-related quality of life (HRQOL) for people with Huntington disease (HD). We report on the psychometric properties of 2 Neuro-QoL patient-reported outcome measures to assess social functioning in HD. Persons with prodromal (n = 198) or manifest HD (n = 195 early and n = 117 late) completed Neuro-QoL Ability to Participate in Social Roles and Activities, and Satisfaction with Social Roles and Activities. Items from 2 generic HRQOL patient-reported outcome measures were used to create a social functioning composite score; items from the Unified Huntington's Disease Rating Scale and Problem Behaviors Assessment Scale were used to create a clinician-rated composite score of social function. Internal consistencies for the scores on the Neuro-QoL measures were excellent (> .88). Computer adaptive test administration had some advantages over computer-administered static Short Forms. Validity was supported by significant associations between the scores on the Neuro-QoL measures and other self- and clinician-reports of social function. Individuals with prodromal HD had better social functioning than the manifest HD groups; individuals with late-HD had less satisfaction and ability to participate in social roles and activities than the other 2 groups. Neuro-QoL provides brief, reliable scores of social functioning that measure ability to participate in, and satisfaction with, social roles and activities in persons with prodromal and manifest HD. In addition, test score interpretations of these measures support their validity in people with prodromal and manifest HD. These measurement tools add breadth to treatment outcome measures in HD and can increase understanding of the social implications of living with HD. (PsycINFO Database Record, ((c) 2018 APA, all rights reserved).)

Purpose: Cognitive functioning impacts health-related quality of life (HRQOL) for individuals with Huntington disease (HD). The Neuro-QoL includes two patient-reported outcome (PRO) measures of cognition-Executive Function (EF) and General Concerns (GC). These measures have not previously been validated for use in HD. The purpose of this analysis is to evaluate the reliability and validity of the Neuro-QoL Cognitive Function measures for use in HD., Methods: Five hundred ten individuals with prodromal or manifest HD completed the Neuro-QoL Cognition measures, two other PRO measures of HRQOL (WHODAS 2.0 and EQ5D), and a depression measure (PROMIS Depression). Measures of functioning The Total Functional Capacity and behavior (Problem Behaviors Assessment) were completed by clinician interview. Objective measures of cognition were obtained using clinician-administered Symbol Digit Modalities Test and the Stroop Test (Word, Color, and Interference). Self-rated, clinician-rated, and objective composite scores were developed. We examined the Neuro-QoL measures for reliability, convergent validity, discriminant validity, and known-groups validity., Results: Excellent reliabilities (Cronbach's alphas ≥ 0.94) were found. Convergent validity was supported, with strong relationships between self-reported measures of cognition. Discriminant validity was supported by less robust correlations between self-reported cognition and other constructs. Prodromal participants reported fewer cognitive problems than manifest groups, and early-stage HD participants reported fewer problems than late-stage HD participants., Conclusions: The Neuro-QoL Cognition measures provide reliable and valid assessments of self-reported cognitive functioning for individuals with HD. Findings support the utility of these measures for assessing self-reported cognition.

Purpose: In Huntington disease (HD), motor, cognitive, and psychiatric changes can have a detrimental impact on health-related quality of life (HRQOL). The purpose of this paper is to describe the extent and type of assistance needed to complete online HRQOL surveys, and the impact of assistance on HRQOL scores., Methods: A patient-reported outcome measurement system was developed for HD-specific HRQOL. Individuals across the prodromal and diagnosed disease severity spectrum (n = 532) completed surveys by computer, and reported the amount and type of assistance they received., Results: Some participants (n = 56; 10.5%) did not complete all surveys; this group had larger proportions with late stage disease, racial/ethnic minority status, low education and single marital status, and poorer motor, independence and cognitive function compared to those who completed all surveys (n = 476). Overall, 72% of individuals did not receive assistance, 11% received computer assistance only, and 17% received assistance answering the survey questions. The majority of late stage individuals (78%) received some assistance compared to early stage (29%) and prodromal individuals (< 1%). Those who received assistance had higher proportions with late stage disease, were older, had less education, and had poorer functional and cognitive skills. Before and after adjustment for sociodemographic and clinical characteristics, those who received assistance had poorer scores on some HRQOL outcomes than those who did not receive assistance., Conclusions: Computer-based assessments are feasible for many persons with HD, although other methods may also be needed. Clinicians and researchers should develop strategies to assist people with HD to complete HRQOL surveys.

Background: Nephrotic syndrome represents a condition in pediatric nephrology typified by a relapsing and remitting course, proteinuria and the presence of edema. The PROMIS measures have previously been studied and validated in cross-sectional studies of children with nephrotic syndrome. This study was designed to longitudinally validate the PROMIS measures in pediatric nephrotic syndrome., Methods: One hundred twenty seven children with nephrotic syndrome between the ages of 8 and 17 years participated in this prospective cohort study. Patients completed a baseline assessment while their nephrotic syndrome was active, a follow-up assessment at the time of their first complete proteinuria remission or study month 3 if no remission occurred, and a final assessment at study month 12. Participants completed six PROMIS measures (Mobility, Fatigue, Pain Interference, Depressive Symptoms, Anxiety, and Peer Relationships), the PedsQL version 4.0, and two global assessment of change items., Results: Disease status was classified at each assessment: nephrotic syndrome active in 100% at baseline, 33% at month 3, and 46% at month 12. The PROMIS domains of Mobility, Fatigue, Pain Interference, Depressive Symptoms, and Anxiety each showed a significant overall improvement over time (p < 0.001). When the PROMIS measures were compared to the patients' global assessment of change, the domains of Mobility, Fatigue, Pain Interference, and Anxiety consistently changed in an expected fashion. With the exception of Pain Interference, change in PROMIS domain scores did not correlate with changes in disease activity. PROMIS domain scores were moderately correlated with analogous PedsQL domain scores., Conclusion: This study demonstrates that the PROMIS Mobility, Fatigue, Pain Interference, and Anxiety domains are sensitive to self-reported changes in disease and overall health status over time in children with nephrotic syndrome. The lack of significant anchoring to clinically defined nephrotic syndrome disease active and remission status may highlight an opportunity to improve the measurement of HRQOL in children with nephrotic syndrome through the development of a nephrotic syndrome disease-specific HRQOL measure.

Background: There is a need for patient-reported outcome measures that capture the impact that motor impairments have on health-related quality of life in individuals with Huntington's disease., Objectives: The objectives of this study were to establish the reliability and validity of new physical functioning patient-reported outcome measures in Huntington's disease., Methods: A total of 510 individuals with Huntington's disease completed 2 Quality of Life in Neurological Disorders (Lower Extremity Function and Upper Extremity Function) and 3 Huntington's Disease Health-Related Quality of Life (Chorea, Speech Difficulties, and Swallowing Difficulties) measures. Clinician-rated and generic self-report measures were also administered., Results: Reliabilities for the new patient reported physical functioning measures were excellent (all Cronbach's α > .92). Convergent, discriminant validity and known group validity was supported., Conclusions: The results provide psychometric support for new patient-reported physical functioning measures and the fact that these measures can be used as clinically meaningful endpoints in Huntington's disease research and clinical practice. © 2017 International Parkinson and Movement Disorder Society., (© 2017 International Parkinson and Movement Disorder Society.)

Incorporation of the patient perspective into research and clinical practice will enrich our understanding of the status and management of patients with glomerular disease and may result in therapies that better address patient needs. In recent years, the importance of the patient experience of glomerular disease has become clear, and significant efforts have been undertaken to systematically capture and describe the patient's disease experience. Patient-reported outcome instruments provide a means to assess the patient's experience in a quantitative manner, thus enabling for comparisons within and between patients. Patient-reported outcome assessments are solely on the basis of a patient report about the status of their health without amendment or interpretation by a clinician or others. Patient-reported outcome assessments provide an opportunity to incorporate the patient perspective into clinical care, research, and clinical trials. Our paper provides an overview of terminology and development methods for patient-reported outcomes and reviews (1) currently available patient-reported outcome instruments appropriate for use in glomerular disease, (2) existing patient-reported outcome data in glomerular disease, and (3) opportunities for incorporating patient-reported outcome instruments into clinical care and research., (Copyright © 2016 by the American Society of Nephrology.)

Objectives: To develop a measure of natural environment and human-made change features (Chapter 2 of the international classification of functioning, disability, and health) and evaluate the influence of perceived barriers on health-related quality of life., Methods: A sample of 570 adults with stroke, spinal cord injury, and traumatic brain injury residing in community settings reported their functioning in home, outdoor, and community settings (mean age = 47.0 years, SD = 16.1). They rated 18 items with a 5-point rating scale to describe the influence of barriers to moving around, seeing objects, hearing sounds, hearing conversations, feeling safe, and regulating temperature and indicated whether any difficulties were due to environmental features. We used Rasch analysis to identify misfitting items and evaluate differential item functioning (DIF) across impairment groups. We computed correlations between barriers and patient-reported outcomes measurement information system (PROMIS) social domain measures and community participation indicators (CPI) measures., Results: The 18 items demonstrated person reliability of .70, discriminating nearly three levels of barriers. All items fit the Rasch model; impairment-related DIF was negligible. Ceiling effects were negligible, but 25 % of the respondents were at the floor, indicating that they did not experience barriers that they attributed to the built and natural environment. As anticipated, barriers correlated moderately with PROMIS and CPI variables, suggesting that although this new item bank measures a construct that is related to participation and health-related quality of life, it also captures something unique. Known-groups validity was supported by wheelchair users reporting a higher level of barriers than did ambulatory respondents., Conclusions: Preliminary evidence supports the reliability and validity of this new measure of barriers to the built and natural environment. This measure allows investigators and clinicians to measure perceptions of the natural environment and human-made changes, providing information that can guide interventions to reduce barriers. Moderate relationships between barriers and PROMIS and CPI variables provide support for the measurement and theory of environmental influences on social health and participation.

Objective: To evaluate the extent to which patient-reported outcomes (PROs) (eg, health-related quality of life) are distinct from clinical outcomes following bariatric surgery., Background: Hospital quality measurement often focuses on traditional clinical outcomes (eg, complications). However, PROs may provide a unique perspective regarding performance, particularly for common, low-risk procedures., Methods: We used data from 11,420 patients who underwent bariatric surgery (2008-2012) from the Michigan Bariatric Surgery Collaborative (39 hospitals). We included both short-term (30-day complication rates) and long-term (1-year weight loss and comorbidity resolution) outcomes. For PROs, we used health-related quality of life assessed by the Health and Activities Limitations Index (HALex) and Bariatric Quality of Life (BQL) index preoperatively and at 1 year. We used multivariable linear regression to determine the association between these PROs and both short and long-term clinical outcomes, adjusting for patient factors and the type of surgical procedure., Results: After adjustment for risk factors and surgical procedure, hospital rankings based on PROs (either the average change in HALex or BQL scores) were not correlated with hospital rankings based on complications. In contrast, both PRO measures were correlated with weight loss. Specifically, the average change in HALex score (R = 0.24, P < 0.002) and average change in BQL score (R = 0.44, P < 0.001) were correlated with hospital average percent excess. One PRO measure-BQL score-was correlated with a decline in the need for medications due to associated comorbidities (R = 0.16, P < 0.01). After accounting for short and long-term clinical outcomes, between 15% and 44% of the variation in PROs remained unexplained at the hospital level., Conclusions: Patient-reported outcomes are not correlated with early perioperative events, but are correlated with measures of clinical effectiveness after bariatric surgery. A comprehensive approach to surgical quality should incorporate both clinical events and self-reported measures of health status throughout the short and long-term recovery period.