Minimal important difference in weight loss following bariatric surgery: Enhancing BODY-Q interpretability.

BODY-Q is a patient-reported outcome measure for comprehensive assessment of outcomes specific to patients undergoing bariatric surgery. The clinical utility of BODY-Q is hampered by the lack of guidance on score interpretation. This study aimed to determine minimal important difference (MID) for assessment of BODY-Q. Prospective BODY-Q data from Denmark and the Netherlands pre- and post-bariatric surgery were collected. Two distribution-based methods were used to estimate MID by 0.2 standard deviations of baseline scores and the mean standardized response change of scores from baseline to 3-years postoperatively. In total, 5476 assessments from 2253 participants were included of which 1628 (72.3%) underwent Roux-en-Y gastric bypass, 586 (26.0%) sleeve gastrectomy, 33 (1.5%) gastric banding, and 6 (0.03%) other surgeries. The mean age was 45.1 ± 10.9 with a mean BMI of 46.6 ± 9.6. Baseline MID ranged from 1 to 4 in health-related quality of life (HRQL) and from 2 to 8 in appearance scales. The mean change of scores ranged from 4 to 5 in HRQL and from 4 to 7 in the appearance scales. The estimated MID for the change in BODY-Q HRQL and appearance scales ranged from 3 to 8 and is recommended for use to interpret BODY-Q scores and assess treatment effects in bariatric surgery.

Development and initial validation of a new self-report measure to assess perceived dependence on tobacco and nicotine products.

How nicotine is administered has evolved from cigarettes to various delivery systems. Assessing perceived dependence on nicotine-containing products now requires accounting for product specificity while allowing comparisons across products and users. This study aims to develop a new self-report measure to assess perceived dependence on tobacco and nicotine products (TNPs) among exclusive and poly-TNP users. A draft version of the new measure, the ABOUT-Dependence, was constructed based on literature review, qualitative research, and expert opinion. Data for scale formation and psychometric assessment was obtained through a US-based web survey (n = 2334) that included additional dependence measures for convergent validity assessment. Qualitative research confirmed a preliminary conceptual framework with seven sub-concepts. Following a cognitive debriefing, 19 items were considered to best represent the different sub-concepts. Psychometric findings supported a three-domain structure [i.e., behavioral impact (five items), signs and symptoms (five items), and extent/timing of use (two items)] and an overall total composite score. The data confirmed convergent and known-group validity, as well as test-retest reliability. The ABOUT-Dependence is a 12-item, psychometrically sound, self-report measure that may be used as a tool for research and further understanding of perceived dependence across the spectrum of TNP and TNP users.

Further psychometric evaluation of the WOUND-Q: A responsiveness study.

The WOUND-Q is a modular patient-reported outcome measure (PROM) with 13 scales measuring constructs across 4 domains (i.e., wound characteristics, health related quality of life, experience of care and wound treatment). The psychometrics of the WOUND-Q were previously assessed and the 13 scales evidenced good validity and reliability. However, the responsiveness (i.e., ability to detect clinical change) of the WOUND-Q has yet to be assessed. The objective of this study was to evaluate responsiveness for 9 WOUND-Q scales that assess outcomes, in a sample of people 18 years of age or older with chronic wounds that were present for at least 3 months. This study conducted a 4 month follow-up of 421 participants who completed the WOUND-Q as part of a previous psychometric study. Participants completed an online survey answering questions about their current wound state (e.g., number, type, size, smell, drainage), anchor questions about change, as well as the WOUND-Q scales that they had completed in their initial assessment. Pre-defined hypotheses were tested with a 75% acceptance threshold indicating sufficient evidence of responsiveness. Minimally important differences (MIDs) were also calculated using both anchor-based and distribution-based methods. Of 390 invited participants, 320 provided responses, ranging in age from 19 to 84 years. Acceptance of hypotheses ranged from 60% to 100%, with only the Symptom scale not meeting the 75% threshold. The findings of this study provide evidence that the WOUND-Q can validly measure clinical change in patients with chronic wounds.

Extending the Range of Measurement for Minimally Invasive Treatments by Adding New Concepts to FACE-Q Aesthetics Scales.

Background: The Satisfaction with Face Overall and Psychological Function scales are the most frequently used FACE-Q Aesthetics module scales. This study aimed to extend their range of measurement by adding and testing new concepts. We aimed to create FACE-Q Aesthetics item libraries. Methods: In-depth concept elicitation interviews were conducted. Concepts were formed into items and refined through multiple rounds of patient and expert input. The items were tested with people living in the United States, Canada, and the United Kingdom who had minimally invasive facial aesthetic treatments. Participants were recruited through an online platform (ie, Prolific). Psychometric properties were examined using Rasch measurement theory analysis, test-retest reliability, and construct validity. Results: We conducted 26 interviews. New concepts were developed into items and refined with input from 12 experts, 11 clinic patients, and 184 Prolific participants. A sample of 1369 Prolific participants completed 52 appearance and 22 psychological items. After removing 10 and 2 items respectively, the psychometric tests provided evidence of reliability with the person separation index, Cronbach alpha, and test-retest reliability values without extremes of 0.88 or more. For validity, lower scores were associated with looking older than one's age, being more bothered by facial skin laxity, treatment wearing off, and having deeper lines on Merz Assessment scales. Short-form scales formed from the 42 appearance items provide examples of item library application. Conclusions: This study provides an innovative means to customize scales to measure appearance and psychological function that maximizes content validity and minimizes respondent burden in the context of minimally invasive treatments.

The SKIN-Q: An Innovative Patient-Reported Outcome Measure for Evaluating Minimally Invasive Skin Treatments for the Face and Body.

Background: As the aesthetics field continues to innovate, it is important that outcomes are carefully evaluated. Objectives: To develop item libraries to measure how skin looks and feels from the patient perspective, that is, SKIN-Q. Methods: Concept elicitation interviews were conducted and data were used to draft the SKIN-Q, which was refined with patient and expert feedback. An online sample (i.e., Prolific) provided field-test data. Results: We conducted 26 qualitative interviews (88% women; 65% ≥ 40 years of age). A draft of the SKIN-Q item libraries were formed and revised with input from 12 experts, 11 patients, and 174 online participants who provided 180 survey responses. The psychometric sample of 657 participants (82% women; 36% aged ≥40 years) provided 713 completed surveys (facial, n = 595; body, n = 118). After removing 14 items, the psychometric analysis provided evidence of reliability (≥0.85) and validity for a 20-item set that measures how skin feels and a 46-item set that measures how skin looks. Short-form scales were tested to provide examples for how to utilize the item sets. Conclusion: The SKIN-Q represents an innovative way to measure satisfaction with skin (face and body) in the context of minimally invasive treatments.

"I Want to Look Natural": Development and Validation of the FACE-Q Aesthetics Natural Module.

BACKGROUND: The concept of natural after a facial aesthetic treatment represents an under-studied area. We added scales to FACE-Q Aesthetics to provide a means to measure this concept from the patient's perspective. OBJECTIVES: To develop and validate FACE-Q natural scales. METHODS: Concept elicitation interviews with people having minimally invasive treatments were conducted to explore the natural concept and develop scales. Patient and expert input was used to refine scale content. An online sample (i.e., Prolific) of people who had a facial aesthetic treatment to examine psychometric properties using Rasch Measurement Theory analysis. A test-retest reliability study was performed, and construct validity examined. RESULTS: Interviews with 26 people were conducted. Three scales were developed and refined with input from 12 experts, 11 patients, and 184 online survey participants. Data from 1358 online participants provided evidence of scale reliability and validity. Reliability was high with Person Separation Index, Cronbach alpha and intraclass correlation coefficients values without extremes >0.82. Tests of construct validity confirmed that the scales functioned as hypothesized. Higher scores on the Expectations scale were associated with wanting a more natural look and movement after treatment. In addition, higher scores on the Natural Appearance and Natural Outcome scales correlated with better scores on other FACE-Q Aesthetics scales, and were associated with the face looking and feeling natural. CONCLUSIONS: Many people seeking facial aesthetic treatments want to look natural. These new FACE-Q Aesthetics scales provide a means to measure the concept of 'natural' from the patient's perspective.

Further psychometric validation and test-retest reproducibility of the WOUND-Q.

WOUND-Q is a condition-specific patient-reported outcome measure developed for all types of chronic wounds, located anywhere on the body. To establish reliability and validity of a patient-reported outcome measure, multiple pieces of evidence are required. The purpose of this study was to examine the measurement properties of 9 of the 13 WOUND-Q scales and perform a test-retest reproducibility study in an international sample. In August 2022, we invited members of an international online community (Prolific.com) with any type of chronic wound to complete a survey containing the WOUND-Q scales, the Wound-QoL and EQ-5D. A test-retest survey was performed 7 days after the first survey. It was possible to examine the reliability and validity of eight of the nine WOUND-Q scales by Rasch Measurement Theory (RMT). To examine test-retest reproducibility intraclass correlation coefficients (ICCs), the standard error of the measurement and the smallest detectable change were calculated. In total, 421 patients from 22 different countries with 11 different types of chronic wounds took part in this study. Our analyses provided further evidence of the reliability and validity of the scales measuring wound characteristics (assessment, drainage, smell), health-related quality of life (life impact, psychological, sleep, social) and wound treatment (dressing).

BODY-Q Normative Scores: Psychometric Validation of the BODY-Q in the General Population in Europe and North America.

Background: BODY-Q is a rigorously developed patient-reported outcome measure designed to measure outcomes of weight loss and body contouring patients. To allow interpretation and comparison of BODY-Q scores across studies, normative BODY-Q values were generated from the general population. The aim of this study was to examine the psychometric properties of BODY-Q in the normative population. Methods: Data were collected using two crowdsourcing platforms (Prolific and Amazon Mechanical Turk) in 12 European and North American countries. Rasch measurement theory (RMT) was used to examine reliability and validity of BODY-Q scales. Results: RMT analysis supported the psychometric properties of BODY-Q in the normative sample with ordered thresholds in all items and nonsignificant chi-square values for 167 of 176 items. Reliability was high with person separation index of greater than or equal to 0.70 in 20 of 22 scales and Cronbach alpha values of greater than or equal to 0.90 in 17 of 22 scales. Mean scale scores measuring appearance, health-related quality of life, and eating-related concerns scales varied as predicted across subgroups with higher scores reported by participants who were more satisfied with their weight. Analysis to explore differential item functioning by sample (normative versus field-test) flagged some potential issues, but subsequent comparison of adjusted and unadjusted person estimates provided evidence that the scoring algorithm worked equivalently for the normative sample as in the field-test samples. Conclusions: The BODY-Q scales showed acceptable reliability and validity in the normative sample. The normative values can be used as reference in research and clinical practice in combination with local estimates for parallel analysis and comparison.

Does the Multiple Sclerosis Impact Scale-29 (MSIS-29) have the range to capture the experience of fully ambulatory multiple sclerosis patients? Learnings from the ASCLEPIOS studies.

Background: Trials of disease-modifying therapies (DMTs) for multiple sclerosis (MS) often include patients with minimal disability. Patient-reported outcome instruments used in these trials have often not captured physical and psychological treatment effects concomitant with observed clinical benefits. Objective: To examine whether the Multiple Sclerosis Impact Scale-29 (MSIS-29) captures changes in the impact of MS in a sample of patients enrolled in the Phase 3 ASCLEPIOS studies (ofatumumab vs. teriflunomide). Methods: Measurement properties (i.e. item fit, reliability, and targeting) of the MSIS-29 were analyzed using Rasch measurement theory (RMT) in data from two phase 3 ofatumumab clinical trials including patients with relapsing-remitting or secondary progressive MS (N = 1882). Targeting of the MSIS-29 items to the patient population was explored within groups categorized by Expanded Disability Status Scale (EDSS) scores. Results: Under RMT analyses, both the Physical and Psychological Impact scales of the MSIS-29 were not appropriately targeted to the overall sample of patients. In particular, 49% and 30% of patients with an EDSS score ≤ 2.5 had fewer physical and psychological impacts, respectively, than would typically be captured by these MSIS-29 items compared to patients with EDSS scores of ≥ 3. Conclusion: The MSIS-29 is commonly used to evaluate the patient-reported physical and psychological impact of MS. However, it may be limited in evaluating changes associated with DMTs in patients with minimal disability.

A Mixed-method Approach to Develop an Ambulatory Module of the SMA Independence Scale.

BACKGROUND: Limited qualitative data exist on the symptoms and impacts of spinal muscular atrophy (SMA) experienced by ambulant individuals. An ambulant module of the SMA Independence Scale (SMAIS) was developed to quantify the assistance required to perform everyday mobility-related activities. OBJECTIVE: The objective of this study was to develop a patient-centered module that provides key insights into what constitutes independence for ambulant and near-ambulant individuals with SMA. METHODS: A stepwise, mixed-method approach was used. Semi-structured interviews were conducted in three waves with individuals with SMA and caregivers of children with SMA who were ambulant or near-ambulant (can walk ≥5 steps with support). Wave 1 interviews (n = 20) focused on concept elicitation. Wave 2 and 3 interviews (n = 15, both) involved completion and cognitive debriefing of items generated based on Wave 1 interviews. Therapeutic area experts were consulted throughout all key steps of the study. In particular, feedback was provided for item refinement and response option decisions. A macro-level preliminary, exploratory analysis, using Rasch Measurement Theory (RMT), provided insight on measurement properties. RESULTS: Wave 1 resulted in 42 mobility and 11 instrumental activity of daily living (iADL) items. During Wave 2, participants defined independence as completing a task with supportive aids but without help from another person, leading to item refinement and modifications to the response scale. Lack of conceptual relevance and ceiling effects led to the removal of all iADL items after Wave 2, and 41 mobility items were tested in Wave 3. Final exploratory RMT and item refinement to reduce overlap led to a 27-item set related to mobility tasks. CONCLUSIONS: Our study provides preliminary support for using the 27-item SMAIS-Ambulatory Module for ambulant or near-ambulant individuals with SMA. Larger-scale analyses to further assess the psychometric properties of the scale are warranted.

Best Practices for FACE-Q Aesthetics Research: A Systematic Review of Study Methodology.

BACKGROUND: The FACE-Q Aesthetics module is a validated patient-reported outcome measure (PROM) that evaluates perspectives on facial aesthetic treatments. Improper administration and poor study methodology can compromise the validity and interpretation of this PROM. OBJECTIVES: This systematic review sought to evaluate the administration and scoring of the FACE-Q Aesthetics scales within the literature. METHODS: A search of Ovid Medline, Embase, Cochrane, and Web of Science was performed on December 20, 2022, with the assistance of a health-research librarian (CRD42022383676). Studies that examined facial aesthetic interventions using the FACE-Q Aesthetics module as a primary or secondary outcome measure were included for analysis. RESULTS: There were 114 studies included. The Face Overall (n = 52, 45.6%), Psychological (n = 45, 39.4%), and Social (n = 43, 37.7%) scales were most frequently reported. Errors in FACE-Q administration were identified in 30 (26.3%) studies. The most common error was the presentation of raw ordinal scores rather than the converted Q score (n = 23). Most studies reported a time horizon for their primary analysis (n = 76, 66.7%); however, only 4 studies provided a rationale for this selection. Sample size calculations for the primary outcome were rarely performed (n = 9, 7.9%). CONCLUSIONS: There continues to be limitations in PROM administration and the quality of articles that report FACE-Q Aesthetic scale data. The authors suggest that future investigators using the FACE-Q refer to the User's Guide regarding administration and scoring of this scale, report a rationale for the study time horizon, and provide an a priori sample size calculation for the primary outcome of interest.

Development and early qualitative evidence of two novel patient-reported outcome instruments to assess daily functioning in people with early-stage Parkinson's.

BACKGROUND: Previous research on concepts that are important to people living with early-stage Parkinson's indicated that 'functional' slowness, fine motor skills, and subtle gait abnormalities are cardinal concepts that are not comprehensively captured by existing patient-reported outcome (PRO) instruments that are used in clinical practice and research to assess symptoms and daily functioning within this patient population. We sought to develop novel PRO instruments to address this unmet need. METHODS: PRO instrument development was led by a multidisciplinary research group, including people living with Parkinson's (termed 'patient experts'), as well as patient engagement and involvement, regulatory science, clinical, and outcome measurement experts. A first set of PRO instruments, termed Early Parkinson's Function Slowness (42 items) and Early Parkinson's Mobility (26 items), were drafted to capture 'functional' slowness, fine motor skills, and subtle gait abnormalities. These PRO instruments were used in cognitive debriefing interviews with people living with early-stage Parkinson's (who were not involved with the multidisciplinary research group) to identify issues with relevance, clarity, ease of completion, conceptual overlap, or missing concepts. RESULTS: Sixty people living with early-stage Parkinson's were interviewed, which led to refining the items to 45 for the Early Parkinson's Functional Slowness and 23 for the Early Parkinson's Mobility PRO instruments. Refinement included rewording items to address clarity issues, merging or splitting items to address overlap issues, and adding new items to address missing concepts. The Early Parkinson's Function Slowness PRO instrument resulted in a multidimensional instrument covering upper limb, complex/whole body, general activity, and cognitive functional slowness. The Early Parkinson's Mobility PRO instrument resulted in comprehensive coverage of everyday mobility tasks, with a focus on gait concepts, plus complex/whole body, balance, and lower limb mobility. CONCLUSIONS: The Early Parkinson's Function Slowness and Early Parkinson's Mobility PRO instruments aim to address gaps in existing PRO instruments to measure meaningful symptoms and daily functioning in people living with early-stage Parkinson's. Utilizing a meticulous study design led by a multidisciplinary research group that included patient experts helped to ensure that the PRO instruments were patient-centric, content valid, and meaningful from a clinical and measurement perspective.

The Development, Deployment, and Evaluation of the CLEFT-Q Computerized Adaptive Test: A Multimethods Approach Contributing to Personalized, Person-Centered Health Assessments in Plastic Surgery.

BACKGROUND: Routine use of patient-reported outcome measures (PROMs) and computerized adaptive tests (CATs) may improve care in a range of surgical conditions. However, most available CATs are neither condition-specific nor coproduced with patients and lack clinically relevant score interpretation. Recently, a PROM called the CLEFT-Q has been developed for use in the treatment of cleft lip or palate (CL/P), but the assessment burden may be limiting its uptake into clinical practice. OBJECTIVE: We aimed to develop a CAT for the CLEFT-Q, which could facilitate the uptake of the CLEFT-Q PROM internationally. We aimed to conduct this work with a novel patient-centered approach and make source code available as an open-source framework for CAT development in other surgical conditions. METHODS: CATs were developed with the Rasch measurement theory, using full-length CLEFT-Q responses collected during the CLEFT-Q field test (this included 2434 patients across 12 countries). These algorithms were validated in Monte Carlo simulations involving full-length CLEFT-Q responses collected from 536 patients. In these simulations, the CAT algorithms approximated full-length CLEFT-Q scores iteratively, using progressively fewer items from the full-length PROM. Agreement between full-length CLEFT-Q score and CAT score at different assessment lengths was measured using the Pearson correlation coefficient, root-mean-square error (RMSE), and 95% limits of agreement. CAT settings, including the number of items to be included in the final assessments, were determined in a multistakeholder workshop that included patients and health care professionals. A user interface was developed for the platform, and it was prospectively piloted in the United Kingdom and the Netherlands. Interviews were conducted with 6 patients and 4 clinicians to explore end-user experience. RESULTS: The length of all 8 CLEFT-Q scales in the International Consortium for Health Outcomes Measurement (ICHOM) Standard Set combined was reduced from 76 to 59 items, and at this length, CAT assessments reproduced full-length CLEFT-Q scores accurately (with correlations between full-length CLEFT-Q score and CAT score exceeding 0.97, and the RMSE ranging from 2 to 5 out of 100). Workshop stakeholders considered this the optimal balance between accuracy and assessment burden. The platform was perceived to improve clinical communication and facilitate shared decision-making. CONCLUSIONS: Our platform is likely to facilitate routine CLEFT-Q uptake, and this may have a positive impact on clinical care. Our free source code enables other researchers to rapidly and economically reproduce this work for other PROMs.

Re-examining content validity of the BREAST-Q more than a decade later to determine relevance and comprehensiveness.

PURPOSE: The BREAST-Q is the most used patient-reported outcome measure (PROM) in breast cancer surgery. The purposes of this study were to re-examine the content validity of BREAST-Q cancer modules (mastectomy, lumpectomy and reconstruction) and to determine the need for new scales. METHODS: Interviews were conducted with women with breast cancer (Stage 0-4, any treatment), and were audio-recorded and transcribed verbatim. Deductive (based on original BREAST-Q conceptual framework) and inductive (new codes from the data) content analysis approaches were used to analyze the data. The number of codes that mapped to BREAST-Q were recorded. RESULTS: Dataset included 3948 codes from 58 participants. Most of the breast (n = 659, 96%) and all psychosocial (n = 127, 100%), sexual (n = 179, 100%) and radiation-related (n = 79, 100%) codes mapped to BREAST-Q Satisfaction with Breast, Psychosocial Wellbeing, Sexual Wellbeing and Adverse Effects of Radiation scales, respectively. For the physical wellbeing codes (n = 939) for breast/chest and arm, 34% (n = 321) mapped to the Physical Wellbeing-Chest scale. Most of the abdomen codes (n = 311) mapped to Satisfaction with Abdomen (n = 90, 76%) and Physical Wellbeing-Abdomen (n = 171, 89%) scales. Codes that did not map (n = 697, 30%) covered breast sensation and lymphedema. Concerns related to fatigue, cancer worry, and work impact were most reported and did not map to BREAST-Q. CONCLUSION: The BREAST-Q, which was developed using extensive patient input more than a decade ago, is still relevant. To ensure the BREAST-Q remains comprehensive, new scales for upper extremity lymphedema, breast sensation, fatigue, cancer worry, and work impact were developed.

Body Contouring Surgery Improves Long-Term Satisfaction with Appearance and Health-Related Quality of Life after Bariatric Surgery.

BACKGROUND: Patient-reported outcomes are crucial in bariatric surgery (BaS) and body contouring surgery (BC) because patients' goals include improvement in appearance and health-related quality of life (HR-QOL). The BODY-Q is a patient-reported outcome measure developed to measure change in satisfaction with appearance and HR-QOL in BaS and BC patients. The aim of this study was to examine BODY-Q scores over the entire weight loss journey, and to investigate the impact of BC after BaS. METHODS: Patients completed the BODY-Q before and after BaS and BC at four hospital departments in Denmark between 2015 and 2019. Cross-sectional scores were analyzed by phase of weight loss journey using one-way analysis of variance. Scores for patients who provided longitudinal assessments were analyzed using repeated measures analysis of variance and paired t test. The impact of BC was examined over time after BaS, using an independent t test from before BaS through more than 7 years after BaS. RESULTS: The study included 1527 patients who provided 2285 BODY-Q assessments. The cross-sectional analysis by phase of weight loss journey showed higher scores after BaS, lower scores before BC, and highest-level scores after BC. The longitudinal analysis showed higher postoperative mean scores compared with preoperative scores for both BaS and BC. The analysis over time after BaS revealed lower mean scores in patients who did not receive BC. CONCLUSION: The authors' results provide evidence of the positive impact of BaS and BC on patients' lives and emphasize the importance of considering BC to finalize the weight loss journey, as it helps to maintain improvements in appearance and HR-QOL. CLINICAL QUESTION/LEVEL OF EVIDENCE: Therapeutic, IV.

A Mixed-Methods Study to Better Measure Patient-Reported Pain and Fatigue in Soft Tissue Sarcoma.

INTRODUCTION: Pain and fatigue are commonly reported by patients with soft tissue sarcoma (STS) as distressing symptoms, yet no patient-reported outcome (PRO) measures have been validated or developed specifically for STS. This study aimed to develop novel PRO scales using existing item banks to measure pain and fatigue in STS. METHODS: A three-stage mixed-methods approach was used. Stage 1: a literature review examined the development and validation of the European Organization for Research and Treatment of Cancer (EORTC) library, Patient-Reported Outcomes Measurement Information System (PROMIS) pain/fatigue item banks, Functional Assessment of Cancer Therapy-General, and FACIT-Fatigue. Conceptual models were developed for pain and fatigue. Stage 2: semi-structured interviews were conducted with clinical experts (n = 3) and STS patients (n = 28) to ensure conceptual coverage and cognitively debrief the selected PRO items. Stage 3: exploratory Rasch measurement theory (RMT) analyses were performed to examine the measurement properties of the proposed scales. RESULTS: Stage 1: The conceptual model for fatigue was organized into two overarching domains: fatigability and fatigue, further split into two subdomains: symptoms and impact. The conceptual model for pain had one overarching domain split into two subdomains: descriptors and impact. Pain (n = 56) and fatigue (n = 40) items were selected from the EORTC item library. Stage 2: qualitative findings ensured conceptual coverage, provided insight into the relevance and comprehension of the items, and informed subsequent item reduction. Stage 3: The total item number was reduced to 43 (pain n = 18, fatigue n = 25). Exploratory RMT analyses supported the final scales' psychometric properties. CONCLUSIONS: This mixed-methods research generated important information on the experience of pain and fatigue in specific subtypes of STS. Five novel PRO scales have been developed through careful item selection in consultation with experts and supported by qualitative and quantitative evidence. These scales may be of value to future clinical trials for STS.

Validation of the Dutch Version of the BODY-Q Measuring Appearance, Health-Related Quality of Life, and Experience of Healthcare in Patients Undergoing Bariatric and Body Contouring Surgery.

BACKGROUND: The BODY-Q is a patient-reported outcome measure developed for use in bariatric and body contouring surgery. OBJECTIVES: The objective of this study was to examine the validity and reliability of the Dutch version of the BODY-Q. METHODS: The BODY-Q consists of 163 items in 21 independently functioning scales that measure appearance, health-related quality of life, and experience of care. The data used to validate the Dutch BODY-Q were provided by 2 prospective multicenter cohort studies across 3 hospitals in the Netherlands. The BODY-Q was administered before and after surgery at 3 or 4 months and 12 months. Rasch measurement theory (RMT) analysis was used to evaluate the BODY-Q for targeting, category threshold order, Rasch model fit, Person Separation Index, and differential item functioning by language (original English data vs Dutch data). RESULTS: Data were collected between January 2016 and May 2019. The study included 876 participants, who provided 1614 assessments. Validity was supported by 3 RMT findings: most scales showed good targeting, 160 out of 163 items (98.2%) evidenced ordered thresholds, and 142 out of 163 items (87.1%) fitted the RMT model. Reliability was high with Person Separation Index values >0.70 for 19 out of 21 scales. There was negligible influence of differential item functioning by language on person item locations and the scale scoring. CONCLUSIONS: This study provides evidence for the reliability and validity of the Dutch BODY-Q for use in bariatric and body contouring patients in the Netherlands. The Dutch BODY-Q can be used in (inter)national research and clinical practice.

The Patient and Clinician Assessment of Gastrointestinal (GI) Related Adverse Events Associated with Oral Disease-Modifying Therapies in Multiple Sclerosis: A Qualitative Study.

INTRODUCTION: Current guidelines for relapsing-remitting multiple sclerosis (RRMS) call for treatment with disease-modifying therapies (DMTs) early in the disease to prevent relapses and accumulation of neurologic impairment and disability. However, patients taking certain oral DMTs may experience gastrointestinal (GI)-related adverse events (AEs), particularly at dose titration. We conducted qualitative research with healthcare professionals (HCPs) and patients in Canada to contextualize their experiences with three oral DMTs: dimethyl fumarate (Tecfidera®), fingolimod (Gilenya®), and teriflunomide (Aubagio®). The objectives of this study were to (1) gather qualitative data to better understand the patient and HCP experience of GI AEs in oral MS DMT treatment in Canada and (2) determine to what extent two patient-reported outcome (PRO) instruments used in recent oral DMT trials capture what is important to patients regarding GI AEs in oral MS DMT treatment (content validity) and to provide qualitative data to help interpret PRO scores. METHODS: This was a qualitative, non-interventional, descriptive, cross-sectional study comprising HCP and patient interviews conducted in English and French, using a 1:1 semi-structured interview approach. RESULTS: Patients reported 16 unique GI AE concepts related to oral DMTs. The most commonly reported symptoms were diarrhea, indigestion, and nausea. While patients acknowledged the negative impact associated with GI-related AEs, most characterized the treatment experience as positive, focusing on preference for oral administration, perceived efficacy of DMTs in terms of lack of MS relapses, slowed progression of their disease, and improvement in MS symptoms. Results supported the content validity (relevance, comprehension, and comprehensiveness) of the two PROs assessed. HCP feedback reinforced patient perspectives on both GI concepts and the two PRO instruments. CONCLUSION: Outcomes of these research activities include experiential data on the symptom and impact experience of oral DMTs in MS from both patients and HCPs that contribute to the process of determining therapeutic value.

The Role of Entropy in Construct Specification Equations (CSE) to Improve the Validity of Memory Tests: Extension to Word Lists.

Metrological methods for word learning list tests can be developed with an information theoretical approach extending earlier simple syntax studies. A classic Brillouin entropy expression is applied to the analysis of the Rey's Auditory Verbal Learning Test RAVLT (immediate recall), where more ordered tasks-with less entropy-are easier to perform. The findings from three case studies are described, including 225 assessments of the NeuroMET2 cohort of persons spanning a cognitive spectrum from healthy older adults to patients with dementia. In the first study, ordinality in the raw scores is compensated for, and item and person attributes are separated with the Rasch model. In the second, the RAVLT IR task difficulty, including serial position effects (SPE), particularly Primacy and Recency, is adequately explained (Pearson's correlation R=0.80) with construct specification equations (CSE). The third study suggests multidimensionality is introduced by SPE, as revealed through goodness-of-fit statistics of the Rasch analyses. Loading factors common to two kinds of principal component analyses (PCA) for CSE formulation and goodness-of-fit logistic regressions are identified. More consistent ways of defining and analysing memory task difficulties, including SPE, can maintain the unique metrological properties of the Rasch model and improve the estimates and understanding of a person's memory abilities on the path towards better-targeted and more fit-for-purpose diagnostics.

Patient Experience in Early-Stage Parkinson's Disease: Using a Mixed Methods Analysis to Identify Which Concepts Are Cardinal for Clinical Trial Outcome Assessment.

INTRODUCTION: Qualitative research on patient experiences in early-stage Parkinson's disease (PD) is limited. It is increasingly acknowledged that clinical outcome assessments used in trials do not fully capture the range of symptoms/impacts that are meaningful to people with early-stage PD. We aimed to conceptualize the patient experience in early-stage PD and identify, from the patient perspective, those cardinal symptoms/impacts which might be more useful to measure in clinical trials. METHODS: In a mixed-methods analysis, 50 people with early-stage PD and nine relatives were interviewed. Study design and results interpretation were led by a multidisciplinary group of patient, clinical, regulatory, and outcome measurements experts, and patient organization representatives. Identification of the cardinal concepts was informed by the relative frequency of reported concepts combined with insights from patient experts and movement disorder specialists. RESULTS: A conceptual model of the patient experience of early-stage PD was developed. Concept elicitation generated 145 unique concepts mapped across motor and non-motor symptoms, function, and impacts. Bradykinesia/slowness (notably in the form of "functional slowness"), tremor, rigidity/stiffness, mobility (particularly fine motor dexterity and subtle gait abnormalities), fatigue, depression, sleep/dreams, and pain were identified as cardinal in early-stage PD. "Functional slowness" (related to discrete tasks involving the upper limbs, complex mobility tasks, and general activities) was deemed to be more relevant than "difficulty" to patients with early-stage PD, who report being slower at completing tasks rather than encountering significant impairment with task completion. CONCLUSION: Patient experiences in early-stage PD are complex and wide-ranging, and the currently available patient-reported outcome (PRO) instruments do not evaluate many early-stage PD concepts such as functional slowness, fine motor skills, and subtle gait abnormalities. The development of a new PRO instrument, created in conjunction with people with PD, that fully assesses symptoms and the experience of living with early-stage PD, is required.

We conducted research to find out about the experiences and symptoms that have the greatest impact on everyday living for people with early-stage Parkinson's disease. This research also looked at which symptoms patients think are important to be tracked in clinical trials. The research team running this study included people living with Parkinson's disease (called "patient experts"). The team also included technical experts and representatives of patient organizations. To begin with, people living with early-stage Parkinson's disease and relatives were interviewed. The interviews collected their thoughts on the impact of early-stage Parkinson's disease on their daily lives. These insights revealed which experiences and symptoms were most important. The research team analyzed ideas and quotes from the interviews to create a picture of early-stage Parkinson's disease. The symptoms that mattered the most to people living with early-stage Parkinson's disease were tremor, rigidity/stiffness, fatigue, depression, sleep/dreams, and pain. Another important symptom was slowness of movement (which is called "bradykinesia/slowness"), and in particular "functional slowness," which included tasks involving the upper limbs, complicated movement tasks, and general activities. Effects on mobility were also important, particularly fine motor skills and subtle walking abnormalities. This research shows the wide-ranging effects that early-stage Parkinson's disease has on patients from their perspective. It also shows which effects are important to capture in trials of therapies aimed at this patient group.