Reliability and validity of the Huntington's Disease Everyday Functioning (Hi-DEF): A patient-reported measure of cognitive capacity on daily functioning in Huntington's disease.

BackgroundThe Huntington's Disease (HD) Everyday Functioning (Hi-DEF) is a novel patient-reported outcome (PRO) scale developed to assess the impact of cognitive impairment on daily functioning in early HD patients.ObjectiveTo examine the psychometric properties, including reliability and validity, of the Hi-DEF. Findings from psychometric analyses using classical test theory (CTT) approach are presented here.

Measuring Satisfaction with Minimally Invasive Aesthetic Treatments with the SKIN-Q Treatment Outcome Scale.

Background: A key outcome in aesthetic treatments is the patient's view of how their skin looks and feels after a treatment.

Objectives: The aim of this study was to add a Treatment Outcome scale to the SKIN-Q patient-reported outcome measure.

Methods: Concept elicitation interviews were performed with patients in Canada and the United States.

Development and Assessment of a Patient-Reported Outcome Instrument for Gender-Affirming Care.

Importance: There is an urgent need for a validated gender-affirming care-specific patient-reported outcome measure (PROM).

Objective: To field test the GENDER-Q, a new PROM for gender-affirming care, in a large, international sample of transgender and gender diverse (TGD) adults and evaluate its psychometric properties.

Design, Setting, And Participants: This international cross-sectional study was conducted among TGD adults aged 18 years and older who were seeking or had received gender-affirming care within the past 5 years at 21 clinical sites across Canada, the United States, the Netherlands, and Spain; participants were also recruited through community groups (eg, crowdsourcing platform, social media).

A patient-centered qualitative evaluation of meaningful change on the NSAA and PUL in Duchenne Muscular Dystrophy.

Background: Duchenne Muscular Dystrophy (DMD) is a rare X-linked genetic disorder caused by mutations in the dystrophin gene. The North Star Ambulatory Assessment (NSAA) and Performance of Upper Limb (PUL) are used to measure motor performance in ambulatory and non-ambulatory individuals, respectively. There is limited published qualitative evidence on what constitutes a meaningful change on either instrument.

Qualitative research and literature review support the integrated Alzheimer's Disease Rating Scale (iADRS) content validity in early symptomatic AD.

Background And Objectives: The integrated Alzheimer's Disease Rating Scale (iADRS) is a measure of cognition and daily function used to evaluate treatment effects in Alzheimer's disease (AD) clinical trials. This study aimed to assess the content validity of the iADRS in early symptomatic AD, and to determine whether integrating assessment of cognition and function into a single measure of global disease severity is supported by the patients' experience.

Methods: A targeted literature review of qualitative research in AD and qualitative interviews with 25 care partners of individuals with early symptomatic AD were conducted.

Estimation of and clinical consensus on the meaningful motor progression threshold on MDS-UPDRS Part III.

BackgroundTo understand changes in the underlying progression of early Parkinson's disease, it is important to derive estimates of the threshold for meaningful motor progression on the MDS-UPDRS Part III in OFF medication state.ObjectiveTo estimate this threshold using two approaches: anchor-based analyses, and clinical consensus via a modified Delphi panel.MethodsFor the anchor-based analyses, data from a Phase II clinical trial were used.

Cognitive impairment in Huntington's disease and its impact on functioning: Concept elicitation and cognitive debriefing for the Huntington's Disease Everyday Functioning (Hi-DEF) scale.

Background: Cognitive impairment in Huntington's disease (HD) is a key driver of disability that can have deleterious impacts on everyday functioning. Currently available patient-reported outcome measures may not adequately capture the impact of HD-related cognitive impairment on daily life, particularly higher-order executive functioning (i.e.

Patient Factors That Impact FACE-Q Aesthetics Outcomes: An Exploratory Cross-sectional Regression Analysis.

Background: The FACE-Q Aesthetics is a validated tool for assessing patient-reported outcomes related to surgical and nonsurgical facial aesthetic treatments. Recognizing patient-specific variables that may influence FACE-Q scores is essential to control for potential confounders in research.

Objectives: This study aimed to identify factors that predict FACE-Q Aesthetics scale scores.

Establishing Convergent Validity of the FACE-Q Aesthetics Module Scales.

Background: FACE-Q Aesthetics scales can be used to assess patient-important outcomes following both surgical and nonsurgical facial cosmetic interventions. Convergent validity is the degree to which the scores of one measurement relate to another measuring a similar construct.

Objectives: The aim of this study was to establish the convergent validity of 11 FACE-Q Aesthetics appearance scales vs the MERZ Aesthetics (Raleigh, NC) scales.

Plasma p-tau181 and GFAP reflect 7T MR-derived changes in Alzheimer's disease: A longitudinal study of structural and functional MRI and MRS.

Background: Associations between longitudinal changes of plasma biomarkers and cerebral magnetic resonance (MR)-derived measurements in Alzheimer's disease (AD) remain unclear.

Methods: In a study population (n = 127) of healthy older adults and patients within the AD continuum, we examined associations between longitudinal plasma amyloid beta 42/40 ratio, tau phosphorylated at threonine 181 (p-tau181), glial fibrillary acidic protein (GFAP), neurofilament light chain (NfL), and 7T structural and functional MR imaging and spectroscopy using linear mixed models.

Results: Increases in both p-tau181 and GFAP showed the strongest associations to 7T MR-derived measurements, particularly with decreasing parietal cortical thickness, decreasing connectivity of the salience network, and increasing neuroinflammation as determined by MR spectroscopy (MRS) myo-inositol.

Measuring the Impact of Surgical and Non-surgical Facial Cosmetic Interventions Using FACE-Q Aesthetic Module Scales: A Systematic Review and Meta-Analysis.

The FACE-Q Aesthetic module measures patient-important outcomes following surgical and non-surgical facial cosmetic procedures. The primary aim of this systematic review was to summarize the pre- to post-intervention mean differences of facial aesthetic interventions that evaluate outcomes using the FACE-Q Face Overall, Psychological, and Social scales. Ovid Medline, Embase, Cochrane, and Web of Science databases were searched on December 20, 2022 with the assistance of a health-research librarian (CRD42023404238).

Rasch Measurement Theory (RMT) Analyses of the Huntington's Disease Everyday Functioning (Hi-DEF) to Evaluate Item Fit and Performance.

Background: The Huntington's Disease (HD) Everyday Functioning (Hi-DEF) is a new patient-reported outcome (PRO) instrument designed to measure the impact of cognitive impairment on daily functioning in the early stages of HD.

Objective: To assess the measurement properties and finalize item content of the Hi-DEF.

Methods: A cross-sectional, observational psychometric validation study was conducted among individuals with early stages of HD at 9 US centers of excellence.

Improving the Impact of BODY-Q Scores Through Minimal Important Differences in Body Contouring Surgery: An International Prospective Cohort Study.

Background: The BODY-Q is a widely used patient-reported outcome measure for comprehensive assessment of treatment outcomes specific to patients undergoing body contouring surgery (BCS). However, for the BODY-Q to be meaningfully interpreted and used in clinical practice, minimal important difference (MID) scores are needed. A MID is defined as the smallest change in outcome measure score that patients perceive as important.

Development and validation of a patient-reported outcome measure for hair loss treatment: The HAIR-Q.

Background: Patient-reported outcome measures (PROMs) for hair loss focus mainly on Alopecia Areata. We created a PROM (i.e.

Establishing test-retest reliability and the smallest detectable change of FACE-Q Aesthetic Module scales.

Background: The test-retest (TRT) reliability of FACE-Q Aesthetic scales is yet to be assessed. The aim of this study was to establish the TRT reliability of 17 FACE-Q Aesthetic scales and determine the smallest detectable change (SDC) that can be identified using these scales.

Methods: Data were collected from an online international sample platform (Prolific).

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.

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.

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

As the aesthetics field continues to innovate, it is important that outcomes are carefully evaluated. To develop item libraries to measure how skin looks and feels from the patient perspective, that is, SKIN-Q. Concept elicitation interviews were conducted and data were used to draft the SKIN-Q, which was refined with patient and expert feedback.

"I Want It 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 understudied area. We added scales to FACE-Q Aesthetics to provide a means to measure this concept from the patient's perspective.

Objectives: The objective of this study was to develop and validate the FACE-Q Aesthetic Natural module.

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.

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.

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.

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.