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. Availability of a standardized, remote-use version of the ALSFRS-R may promote more inclusive, decentralized clinical trials. A scoping literature review was conducted to identify existing remote-use ALSFRS-R tools, synthesize feasibility and comparability of administration modes, and summarize barriers and facilitators to inform development of a standardized remote-use ALSFRS-R tool. METHODS: Included studies reported comparisons between remote and in-person, clinician-reported, ALSFRS-R administration and were published in English (2002-2022). References were identified by searching peer-reviewed and gray literature. Twelve studies met the inclusion criteria and were analyzed to compare findings within and across modes of administration. RESULTS: Remote modes of ALSFRS-R administration were categorized into 4 nonmutually exclusive categories: telephone (n = 6), videoconferencing (n = 3), computer or online platforms (n = 3), mobile applications and wearables (n = 2), and 1 unspecified telemedicine modality (n = 1). Studies comparing in-person to telephone or videoconferencing administration reported high ALSFRS-R rating correlations and nonsignificant between-mode differences. CONCLUSIONS: There is insufficient information in the ALSFRS-R literature to support remote clinician administration for collecting high quality data. Future research should engage persons living with amyotrophic lateral sclerosis, care partners, and providers to develop a standardized remote-use ALSFRS-R version.

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. Inability to compare subgroups validly is a broad issue and has implications for clinical trials, and it also has specific and important implications for identifying and beginning to address health inequities. We describe this threat to validity (the psychometric term is differential item functioning), why it is so important in heart-failure outcomes, the research that has been conducted thus far in this area, the gaps that remain, and what we can do to avoid this threat to validity. PROs bring unique information to clinical decision making, and the validity of PRO measures is key to interpreting differences in heart failure outcomes.

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). All interviews were conducted over the phone/web and audio recorded. Interviews were transcribed and descriptive qualitative content analysis was used to summarize findings overall and by gender. RESULTS: Twenty-five adults (56% women) diagnosed with HF participated. The average age was 67 years (range: 25-88). Women attributed a wider variety of symptoms to HF than men. Some participants had difficulty differentiating whether their experiences were due to HF, side effects of their medications, or age. We found very little evidence that participants interpreted KCCQ-23 items differently based on gender. CONCLUSIONS: Overall, our findings indicate that interpretation of the KCCQ-23 items were similar in men and women. However, some modifications to items may improve clarity of interpretation for a wide range of patients.

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. PARTICIPANTS: Individuals ≥18 years of age who underwent burn-related surgery and met one of the following criteria: (1) >10% total body surface area (TBSA) burn and ≥65 years of age; (2) >20% TBSA burn and 18 to 64 years of age; (3) electrical high voltage/lightning injury; or (4) burn injury to the hands, face, or feet. The participants (N=378) had data on all variables of interest and were included in the analyses. INTERVENTIONS: Not applicable. MAIN OUTCOME MEASURE: Satisfaction With Life Scale. RESULTS: Growth mixture modeling identified 2 classes with different trajectories of SWL. The mean SWL of the unchanged class (n=224, 60%) was flat over 2 years with high initial SWL scores. The SWL of the dissatisfied class (n=154, 40%) was at the low end of average and got progressively worse over time. CONCLUSIONS: SWL after burn injury can be described by 2 different trajectories with substantially different outcomes. Older age, worse mental health, and unemployment prior to injury predicted membership in the dissatisfied class. Additional services could be provided to those at high risk for low SWL to achieve better outcomes.

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. Ten specific subdomains emerged from the panel discussions, which could be classified into the four overarching categories of physical health, mental health, social function, and financial resources. The caregivers highlighted the impact on the subdomains of sleep and fatigue as most critical. A review of existing caregiver impact measures confirmed that there is no measure currently available that assesses all of these relevant domains, indicating the need for the development of such a measure. The current findings highlight the significant life effects of caring for a child with severe epilepsy and can be used to inform the development of such a tool.

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). METHODS: Multi-group confirmatory factor analysis was used to evaluate successive levels of measurement invariance of the 6-item short form, the UW-SES: (a) configural invariance, i.e., equivalent item-factor structures between groups; (b) metric invariance, i.e., equivalent unstandardized factor loadings between groups; and (c) scalar invariance, i.e., equivalent item intercepts between groups. Responses from the four groups with different diagnostic disorders were compared: MD (n = 172), MS (n = 868), PPS (n = 225), and SCI (n = 242). RESULTS: The results of this study support that the most rigorous form of invariance (i.e., scalar) holds for the 6-item short form of the UW-SES across the four diagnostic subgroups. CONCLUSIONS: The current study suggests that the 6-item short form of the UW-SES has the same meaning across the four diagnostic subgroups. Thus, the 6-item short form is validated for people with MD, MS, PPS, and SCI.

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. RESULTS: We abstracted data on 9 Consensus-based Standards of the selection of health Measurements INstruments (COSMIN) measurement properties and analyzed these data according to COSMIN criteria. Only 3 PROMs provided evidence for at least half of the COSMIN properties: Eight-item Jaw Function Limitations Scale, Oral Health Impact Profile (OHIP)-TMD (OHIP-TMD), and TMJ Ankylosis Specific Quality of Life Questionnaire. Only the OHIP-TMD provided evidence for properties essential to evaluating change over time: reliability, measurement error, and responsiveness. CONCLUSION: We believe these results can alert clinical practitioners to gaps in our knowledge about the most widely used PROMs in TMD practice, and identify topics requiring further study for clinical researchers.

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). Growth mixture modeling (GMM) was used to model PCS scores over time. Covariables included burn size, participant characteristics, and scores from the Community Integration Questionnaire (CIQ)/Social Integration portion, Satisfaction With Life Scale (SWLS), and Satisfaction With Appearance Scale (SWAP). RESULTS: Data from 939 participants were used for complete-case analysis. Participants were 72% male, 64% non-Hispanic White, with an average age of 44 years and an average burn size of 20% of total body surface area (TBSA). The best fitting model suggested three distinct trajectories (Class 1 through 3) for HRQOL. We titled each Class according to the characteristics of their trajectory. Class 1 (recovering; n = 632), Class 2 (static; n = 77), and Class 3 (weakened; n = 205) reported near average HRQOL preinjury, then reported lower scores at discharge, with Class 1 subsequently improving to preinjury levels and Class 3 improving but not reaching their preinjury quality of life. Class 3 experienced the largest decrease in HRQOL. Class 2 reported the lowest preinjury HRQOL and remained low for the next 2 years, showing minimal change in their HRQOL. CONCLUSIONS: These findings emphasize the importance of early universal screening and sustained intervention for those most at risk for low HRQOL following injury. For Class 2 (static), lower than average HRQOL before their injury is a warning. For Class 3 (weakened), if the scores at 6 months show a large decline, then the person is at risk for not regaining their HRQOL by 24 months and thus needs all available interventions to optimize their outcomes. Results of this study provide guidance for how to identify people with burn injury who would benefit from more intensive rehabilitation to help them achieve or regain better HRQOL. (PsycInfo Database Record (c) 2023 APA, all rights reserved).

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). METHODS: We conducted ten focus groups with 40 men and women with mild, moderate, and severe TMD. Focus groups elicited descriptions of symptoms and asked participants to review a list of existing patient-reported outcomes (PROs) from the literature and patient advisor input and speak to how those PROs reflect their own experience, including rating their importance. RESULTS: We identified 52 distinct concepts across six domains: somatic, physical, social, sexual, affective, and sleep. Focus groups identified the ability to chew and eat; clicking, popping, and other jaw noises; jaw pain and headaches; jaw misalignment or dislocation; grinding, clenching, or chewing, including at night; and ear sensations as most important. Participants with severe TMDs more often reported affective concepts like depression and shame than did participants with mild or moderate TMDs. CONCLUSION: Findings support PROM item development for TMDs, including selecting existing PROMs or developing new ones that reflect patients' lived experiences, priorities, and preferred terminology. Such measures are needed to increase understanding of TMDs, promote accurate diagnosis and effective treatment, and help advance research on TMDs.

Patients with temporomandibular joint disorders, or TMDs, have pain and other problems in their jaw, and face and neck areas. We talked to 40 patients with mild, moderate, and severe TMDs to learn about their symptoms. We also asked patients to review a list of TMD symptoms. They then chose the most important ones based on their experience. The data showed 52 TMD symptoms and functions across six domains. The patients chose the ability to chew and eat; clicking, popping, and other jaw noises; and jaw pain and headaches as most important. They also chose jaw misalignment or dislocation; grinding, clenching, or chewing, including at night; and ear feelings as important. Findings support creating patient-reported outcome measures, or PROMs, for TMDs. These PROMs should reflect patients' experiences and what is most important to them. Such measures can help doctors treat TMDs and help advance research on TMDs.

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. Food and Drug Administration's (FDA) Center for Devices and Radiological Health (CDRH) hosted a public meeting in September 2020 discussing how PROs can be used in medical device evaluation throughout the total product life cycle, as well as methods for developing and modifying PRO instruments to ensure they are fit-for-purpose. This commentary presents key points of discussion from the meeting, providing insight into the increased interest in PRO data to support medical product development while also exploring future opportunities of incorporating PRO data throughout healthcare. MAIN BODY: Thoughtful use of fit-for-purpose PRO instruments to integrate the patient's voice into clinical care paradigms, medical device development, regulatory decisions, and reimbursement and coverage decisions were emphasized throughout the meeting. Existing PRO instruments may be used if the context of use is appropriate. Modifications to an existing PRO instrument may also be explored to ensure the instrument is fit-for-purpose in a new context of use. Development of a novel PRO instrument may be necessary to capture attributes in a new patient population or application. Multi-stakeholder collaborations, of which patients are a key component, create efficiencies in the development and modification of PRO instruments. CONCLUSION: Continued multi-stakeholder collaborations bringing together researchers, clinicians, patients, regulators, and payers are critical to further advance the inclusion of the patient voice incorporating PRO instruments throughout the healthcare ecosystem in an efficient manner that is least burdensome to patients.

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. This study examined the incorporation of patient-reported outcome (PRO) instruments in the evaluation of medical devices over a six-year period. PRO instruments used to inform study endpoints were extracted from the summary documents and clinical trial data of premarket authorizations posted on publicly available FDA databases between October 1, 2014-September 30, 2020. RESULTS: PROs were included in 53% of authorizations, with 34% using PROs as primary and secondary endpoints. This study found that PRO instruments were used in each type of marketing authorization and in all medical specialties examined in this study. CONCLUSIONS: Expanding the current collaborative efforts to develop and modify PRO instruments may help to improve use of PROs in medical device evaluations.

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.Gly992Arg and an iPSC line from an unrelated healthy female adult control individual. Human iPSCs were derived from fibroblasts using retroviruses carrying the four reprogramming factors OCT4, SOX2, KLF4 and C-MYC. These lines provide a valuable resource for studying the pathophysiology of NPC and for pharmacological intervention.

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. Historically, integration of patient perspectives into KRT product development has been limited. However, the US Food and Drug Administration recognizes the importance of incorporating patient perspectives into the total product life cycle (i.e., from product conception to postmarket surveillance) and encourages the consideration of patient-reported outcomes in regulatory-focused clinical trials when appropriate. Recognizing the significance of identifying patient-reported outcome measures (PROMs) that capture contemporary patient priorities, the Kidney Health Initiative, a public-private partnership between the American Society of Nephrology and US Food and Drug Administration, convened a workgroup to (1) develop a conceptual framework for a health-related quality of life PROM; (2) identify and map existing PROMs to the conceptual framework, prioritizing them on the basis of their supporting evidence for use in the regulatory environment; and (3) describe next steps for identifying PROMs for use in regulatory clinical trials of transformative KRT devices. This paper summarizes the proposed health-related quality-of-life PROM conceptual framework, maps and prioritizes PROMs, and identifies gaps and future needs to advance the development of rigorous, meaningful PROMS for use in clinical trials of transformative KRT devices.

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). Items were administered to a sample of people with chronic medical conditions commonly associated with disability (N = 1,457) and to a general population sample (N = 300) representative of the Unites States general population with respect to age, gender, race, and ethnicity. RESULTS: The final item bank includes 28 items calibrated to IRT with the scores on a T-metric. A mean of 50 represents the mean resilience in the general population sample. Four and eight item short forms are available, and their scores are highly correlated with the item bank score (r ≥ .94). Reliability is excellent across most of the resilience continuum. Initial analyses provide strong support for validity of the score. CONCLUSIONS: The findings support reliability and validity of the University of Washington Resilience Scale (UWRS) for assessing resilience in any population, including individuals with chronic health conditions or disabilities. It can be administered using computerized adaptive testing or by short forms. (PsycInfo Database Record (c) 2020 APA, all rights reserved).

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. Reliability was adequate from a classical test and IRT perspective. IRT analysis found that the SWLS could be improved by using only five response categories rather than seven and by removing the fifth item, "If I could live my life over, I would change almost nothing." This item functions poorly and reduces instrument reliability. With these revisions, the SWLS is a useful instrument to monitor an important outcome of trauma rehabilitation.

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. METHODS: An expert panel of pain clinicians and researchers was convened to establish construct definitions of pain-related self-efficacy and pain catastrophizing and guide item development. Two patient advisors provided guidance throughout the project. Nineteen people with chronic pain participated in focus groups about their perspectives and experiences related to pain-related self-efficacy and pain catastrophizing. Twenty-two people with chronic pain participated in cognitive interviews to test proposed candidate items. RESULTS: Saturation was reached after three focus groups with no new subdomains identified by participants in the third focus group. Following cognitive interviews, five of the 48 initial pain-related self-efficacy candidate items were dropped and seven required substantial revision resulting in 43 pain-related self-efficacy candidate items. After two rounds of cognitive interviews, ten items were eliminated and ten substantially revised, resulting in a set of 30 from the initial 43 pain catastrophizing candidate items. CONCLUSION: This article summarizes results of the qualitative phase of the development of new measures of pain-related self-efficacy and pain catastrophizing. Candidate items will be field tested with a large sample of people with chronic pain and the data will be used to calibrate items to an item response theory model. Resulting item banks and short forms will be made publicly available to researchers and clinicians.

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

BACKGROUND: New health status instruments can be administered by computerized adaptive test or short forms. The Prosthetic Limb Users Survey of Mobility (PLUS-MTM) 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. OBJECTIVES: To compare scores obtained from computerized adaptive test to scores obtained from fixed-length short forms (7-item and 12-item) in order to provide guidance to researchers and clinicians on how to select the best form of administration for different uses. STUDY DESIGN: Cross-sectional, observational study. METHODS: Individuals with lower limb loss completed the PLUS-M by computerized adaptive test and short forms. Administration time, correlations between the scores, and standard errors were compared. RESULTS: Scores and standard errors from the computerized adaptive test, 7-item short form, and 12-item short form were highly correlated and all forms of administration were efficient. Computerized adaptive test required less time to administer than either paper or electronic short forms; however, time savings were minimal compared to the 7-item short form. CONCLUSION: Results indicate that the PLUS-M computerized adaptive test is most efficient, and differences in scores between administration methods are minimal. The main advantage of the computerized adaptive test was more reliable scores at higher levels of mobility compared to short forms. Clinical relevance Health-related item banks, like the Prosthetic Limb Users Survey of Mobility (PLUS-MTM), can be administered by computerized adaptive testing (CAT) or as fixed-length short forms (SFs). Results of this study will help clinicians and researchers decide whether they should invest in a CAT administration system or whether SFs are more appropriate.

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. Fit values from a one-factor confirmatory factor analysis were acceptable, supporting unidimensionality. Cronbach's α was 0.82, indicating good internal consistency. One item had a corrected item-total score correlation of less than 0.40. Associations between the 4-D and other measures were in the expected direction and magnitude. A negligible number of participants (no more than two) selected the lowest category for all items (ie, minimal floor effect) or the highest category for all items (ie, minimal ceiling effect). 4-D had acceptable psychometric properties in a sample of adult burn injury survivors; however, the scale could be improved by removing the item with a low correlation with the total score.