Validation of the Kansas City Cardiomyopathy Questionnaire in Patients With Tricuspid Regurgitation.

Importance: Improving patients' health status is a key goal of treating tricuspid regurgitation (TR). The Kansas City Cardiomyopathy Questionnaire (KCCQ) is a heart failure disease-specific health status measure used to capture the health status impact of TR and the benefit of transcatheter tricuspid valve intervention (TTVI), but its validity in this clinical setting is unknown.

Objective: To evaluate the psychometric properties of the KCCQ in patients with TR.

Remote-Use Applications of the Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised Clinical Outcome Assessment Tool: A Scoping Review.

Objectives: In 2021, the US Congress passed the Accelerating Access to Critical Therapies for Amyotrophic Lateral Sclerosis Act. The law encourages development of "tools, methods, and processes" to improve clinical trial efficiency for neurodegenerative diseases. The Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) is an outcome measure administered during in-person clinic visits and used to support investigational studies for persons living with amyotrophic lateral sclerosis.

Trajectories of physical health-related quality of life among adults living with burn injuries: A burn model system national database investigation to improve early intervention and rehabilitation service delivery.

Introduction: Understanding trajectories of recovery in key domains can be used to guide patients, families, and caregivers. The purpose of this study was to describe common trajectories of physical health over time and to examine predictors of these trajectories.

Method: Adults with burn injuries completed self-reported assessments of their health-related quality of life (HRQOL) as measured by the SF-12® Physical Component Summary (PCS) score at distinct time points (preinjury via recall, index hospital discharge, and at 6-, 12-, and 24 months after injury).

Focus Groups to Inform the Development of a Patient-Reported Outcome Measure (PROM) for Temporomandibular Joint Disorders (TMDs).

Background: Understanding symptoms of temporomandibular joint disorders (TMDs) can help doctors and patients document, monitor, and manage the disease and help researchers evaluate interventions. Patients with TMDs experience symptoms ranging from mild to severe, primarily in the head and neck region. This study describes findings from formative patient focus groups to capture, categorize, and prioritize symptoms of TMDs towards the development of a patient-reported outcome measure (PROM).

Investigating gender-based differential item functioning on the Kansas City Cardiomyopathy Questionnaire (KCCQ) using qualitative content analysis.

Purpose: The purpose of this study is to evaluate potential gender-based differences in interpreting the Kansas City Cardiomyopathy Questionnaire (KCCQ-23) and to explore if there are aspects of health-related quality of life (HRQOL) not captured by the KCCQ-23 that are important to assess in men and/or women with heart failure (HF).

Methods: Patients ≥ 22 years of age with clinician-diagnosed HF and left ventricular ejection fraction ≤ 40% were recruited from two academic medical centers to participate in semi-structured concept elicitation and cognitive debriefing interviews. Enrollment was stratified by patient-identified gender (half women/half men).

Patient-reported outcome measures for individuals with temporomandibular joint disorders: a systematic review and evaluation.

Objective: We used consensus-based, systematic protocols to evaluate the reliability and validity of patient-reported outcome measures (PROMs) for use in documenting treatment outcomes for temporomandibular disorders (TMDs).

Study Design: Following the Preferred Reporting Items for Systematic Reviews and Meta-Analyses guidelines, we conducted a focused search of 5 literature databases resulting in a yield of 445 articles that qualified for full-text review. Our review identified 124 PROMs, from which we selected 19 directly related to symptoms of TMD including temporomandibular joint (TMJ) function.

Do PRO Measures Function the Same Way for all Individuals With Heart Failure?

Women diagnosed with heart failure report worse quality of life than men on patient-reported outcome (PRO) measures. An inherent assumption of PRO measures in heart failure is that women and men interpret questions about quality of life the same way. If this is not the case, the risk then becomes that the PRO scores cannot be used for valid comparison or to combine outcomes by subgroups of the population.

Patient-reported outcomes and medical device evaluation: from conception to implementation.

Background: The insights gleaned from patient-reported outcomes (PROs) have implications across the healthcare ecosystem, from clinical investigations to evaluate the safety and effectiveness of medical devices to clinical care and reimbursement decisions. The U.S.

Inclusion of patient-reported outcome instruments in US FDA medical device marketing authorizations.

Background: The U.S. Food and Drug Administration encourages the incorporation of the patient voice throughout the medical device total product lifecycle.

Generation of an iPSC line (AKOSi004-A) from fibroblasts of a female adult NPC1 patient, carrying the compound heterozygous mutation p.Val1023Serfs*15/p.Gly992Arg and of an iPSC line (AKOSi005-A) from a female adult control individual.

Niemann-Pick disease Type C (NPC) is a rare progressive neurodegenerative disorder with an incidence of 1:120,000 caused by mutations in the NPC1 or NPC2 gene leading to a massive cholesterol accumulation. Here, we describe the generation of induced pluripotent stem cells (iPSCs) of an affected female adult individual carrying the NPC1 mutation p.Val1023Serfs*15/p.

Toward Patient-Centered Innovation: A Conceptual Framework for Patient-Reported Outcome Measures for Transformative Kidney Replacement Devices.

Individuals with dialysis-dependent kidney failure experience considerable disease- and treatment-related decline in functional status and overall well-being. Despite these experiences, there have been few substantive technological advances in KRT in decades. As such, new federal initiatives seek to accelerate innovation.

Development of a resilience item bank and short forms.

Purpose: The purpose of this study was to develop a publicly available, psychometrically sound item bank and short forms for measuring resilience in any population, but especially resilience in individuals with chronic medical conditions or long-term disability.

Research Methods: A panel of 9 experts including disability researchers, clinical psychologists, and health outcomes researchers developed a definition of resilience that guided item development. The rigorous methodology used focus groups, cognitive interviews, and modern psychometric theory quantitative methods, including item response theory (IRT).

Psychometric Properties of the Satisfaction With Life Scale in People With Traumatic Brain, Spinal Cord, or Burn Injury: A National Institute on Disability, Independent Living, and Rehabilitation Research Model System Study.

This study evaluated the measurement properties of the Satisfaction With Life Scale (SWLS) in a sample of 17,897 people with spinal cord injury (48%, n = 8,566), traumatic brain injury (44%, n = 7,941), and burn injury (8%, n = 1,390), 1 year following injury. We examined measurement invariance across the groups, unidimensionality, local independence, reliability from a classical test and item response theory (IRT) framework, and fit to a unidimensional IRT model. The results support unidimensionality and local independence of the SWLS.

Satisfaction With Life Over Time in People With Burn Injury: A National Institute on Disability, Independent Living, and Rehabilitation Research Burn Model System Study.

Objective: To examine trajectories of satisfaction with life (SWL) of burn survivors over time and their clinical, demographic, and other predictors.

Design: Longitudinal survey.

Setting: Not applicable.

Measuring Pain Catastrophizing and Pain-Related Self-Efficacy: Expert Panels, Focus Groups, and Cognitive Interviews.

Purpose: Pain-related self-efficacy and pain catastrophizing are important psychosocial determinants of pain and can be therapeutic targets for chronic pain management. Advances in psychometric science have made shorter or dynamically administered instruments possible. The aim of this study was to generate and test candidate items for two new patient-reported outcome measures of pain-related self-efficacy and pain catastrophizing.

A comparison of computerized adaptive testing and fixed-length short forms for the Prosthetic Limb Users Survey of Mobility (PLUS-M).

Background: New health status instruments can be administered by computerized adaptive test or short forms. The Prosthetic Limb Users Survey of Mobility (PLUS-M) is a self-report measure of mobility for prosthesis users with lower limb loss. This study used the PLUS-M to examine advantages and disadvantages of computerized adaptive test and short forms.

Life impact of caregiving for severe childhood epilepsy: Results of expert panels and caregiver focus groups.

Severe epilepsy in children and young adults can significantly affect the lives of their caregivers. However, the lack of a reliable and valid measure of caregiver impact has limited our understanding of the scope and correlates of this impact, as well as our ability to measure the effects of treatments that could lessen it. The purpose of this study was to facilitate focus groups and interviews with an international group of clinician experts and caregivers to identify the most important domains that should be assessed in a measure of caregiver impact.

Psychometric Properties of the Modified 5-D Itch Scale in a Burn Model System Sample of People With Burn Injury.

The aim of this study is to evaluate the psychometric properties of the 4-dimension (4-D) itch scale, a modified version of the 5-dimension itch scale, in a sample of individuals with severe burn injury and/or burn injuries to hands, face, and/or feet. Four of the five domains of the 5-dimension itch scale (4-D) were administered to individuals who reported itching (N = 173) in the Burn Injury Model System Centers Program longitudinal study at either 5 or 10 years after injury. Analyses of the scale included evaluation of dimensionality, internal consistency, associations with other symptoms or quality of life measures, and an examination of floor and ceiling effects.

Testing the measurement invariance of the University of Washington Self-Efficacy Scale short form across four diagnostic subgroups.

Purpose: The University of Washington Self-Efficacy Scale (UW-SES) was originally developed for people with multiple sclerosis (MS) and spinal cord injury (SCI). This study evaluates the measurement invariance of the 6-item short form of the UW-SES across four disability subgroups. Evidence of measurement invariance would extend the UW-SES for use in two additional diagnostic groups: muscular dystrophy (MD) and post-polio syndrome (PPS).