Use Of Patient-reported Outcome Measures Research Articles

Abstract 11518: Patient Reported Outcomes Measures Are Infrequently Used in Clinical Studies of Heart Transplant Recipients

Introduction / Background: The goals of heart transplantation (HT) are to improve symptoms, restore quality of life, and extend survival. Clinical trials in HT recipients have largely focused on objective outcomes such as measures of cardiac function and survival; however, a paucity of data exists regarding the use of patient reported outcome measures (PROMs) as outcomes in these studies. Aims: We aimed to characterize the use of PROMs in registered clinical trials of HT recipients. Methods: All clinical studies of adult HT recipients were queried from ClinicalTrials.gov, and studies were stratified by inclusion of PROMs. Studies reporting PROMs were identified via specific search terms using the “outcomes measures” field. Summary statistics compared characteristics of studies that did versus did not report PROMs. Results/Data: Between November 1999 and August 2022, 227 studies of HT recipients were registered on ClinicalTrials.gov. At least one PROM was reported in 11% (n=24/227) of registered studies. Studies reporting PROMs were more likely to be conducted outside of the United States (91.7% vs 54.2%, p<0.001) and report a greater number of total primary/secondary outcomes (PROMs: median 7 [IQR: 4, 9] vs. No PROMs: median 3 [IQR: 2, 6]; p<0.001). The majority of studies reporting PROMs (58.3%) were initiated after 2020. Twenty-one distinct PROM tools/domains were reported as outcome measures, with the Short Form Health Survey 36 being most frequently used (n=10/24). Thirteen of the 21 PROMs included questions assessing mental health, whereas only three PROM tools were cardiac-specific (Figure). Conclusions: Only about one in ten registered clinical trials of HT recipients include PROMs, and mental health was the most commonly assessed domain when a PROM was included. Development and validation of PROM tools is needed to assess the multifaceted nature of health-related quality of life in HT recipients.

Patient-Reported Outcome Measures for Patients With CKD: The Case for Patient-Reported Outcomes Measurement Information System (PROMIS) Tools

Chronic kidney disease (CKD), kidney failure, and kidney replacement therapies are associated with high symptom burden and impaired health-related quality of life (HRQOL). Symptoms change with disease progression or transition between treatment modalities and frequently go unreported and unmanaged. Tools that reliably monitor symptoms may improve the management of patients with CKD. Patient-reported outcome measures (PROMs) assess symptom severity; physical, psychological, social, and cognitive functioning; treatment-related side effects; and HRQOL. Systematic use of PROMs can improve patient-provider communication, patient satisfaction, clinical outcomes, and HRQOL. Potential barriers to their use include a lack of engagement, response burden, and limited guidance about PROM collection, score interpretation, and workflow integration. Well-defined, acceptable, and effective clinical response pathways are essential for implementing PROMs. PROMs developed by the Patient-Reported Outcomes Measurement Information System (PROMIS) address some challenges and may be suitable for clinical use among patients with CKD. PROMIS tools assess multiple patient-valued, clinically actionable symptoms and functions. They can be administered as fixed-length, customized short forms or computer adaptive tests, offering precise measurement across a range of symptom severities or function levels, tailored questions to individuals, and reduced question burden. Here we provide an overview of the potential use of PROMs in CKD care, with a focus on PROMIS.

Frequency of standardized patient-reported outcome measures in routine clinical care of patients with cancer.

342 Background: Patient-reported outcomes (PROs) assess a patient’s self-reported health status, including disease symptoms, treatment experience, functional outcomes, and quality of life. Incorporation of PROs has been linked to improved patient symptom control and increased shared decision making. Standardized PRO measures (PROMs) are becoming more frequent as an endpoint for clinical trials, however their use is less frequent in routine care for patients with cancer. The goal of this study was to assess the utilization of PROMs and identify barriers to their use. Methods: US-licensed oncologists/hematologists convened at one of three live meetings in 2023 to review recent clinical updates. Respondent demographics were collected ahead via an online survey. During the meetings, participants responded to queries on their use and perceptions of PROs and PROMs. Responses were captured via audience response system technology; not all participants answered every question. Pooled data were summarized using descriptive statistics. Results: Of the 146 respondents, 77.4% self-identified as community-setting providers, spent 84.3% of their time seeing patients, saw an average of 19.8 patients/day, and averaged 17.7 years of experience. Respondents found PROs to be most useful in early detection/management of treatment toxicities and symptoms (63.8%) and tailoring of clinical/supportive care (56%). The majority of PROs are typically recorded through informal verbal inquiries, while 18.1% and 10.9% of respondents use validated and non-validated PROMs, respectively. Few respondents (11.3%) administer PROMs to every patient at every visit, while over one-third (38.3%) never administer PROMs. Within the practice, medical assistants and nurses are most likely to be the provider administering PROMs. PROMs are most commonly administered when the patient is in the office and are infrequently administered through other means. Lack of time to administer or review PRO surveys (45.1%), and lack of reimbursement (38.9%) and EMR integration (38.9%) were the most common reported barriers to using PROMs in the respondents’ practices. Conclusions: Despite the majority of respondents finding PROs useful in practice, a minority use formal measures to record PROs. Further, usage is infrequent across patient visits. Lack of time, lack of reimbursement, and logistic barriers are the most common reasons for not using PROMs in routine care. These barriers may be overcome by efforts or guideline changes from regulatory agencies, payors, or clinical guideline organizations. Future work will assess how physicians are using PROMs in treatment selection for their patients with cancer.

A meta-ethnography of how children and young people with chronic non-cancer pain and their families experience and understand their condition, pain services, and treatments.

A meta-ethnography of how children and young people with chronic non-cancer pain and their families experience and understand their condition, pain services, and treatments.

Does Patient-Reported Outcome Measures Use at New Foot and Ankle Patient Clinic Visits Improve Patient Activation, Experience, and Satisfaction?

Category: Other; Ankle Introduction/Purpose: Patient-reported outcome measures (PROMs) can help predict clinical outcomes and improve shared clinical decision-making discussions. There remains a paucity of research assessing how the use of PROMs may drive improved patient experience and patient activation. This randomized, controlled trial study objectives: 1) Determine if the active use and discussion of PROMs during new patient visits are associated with patient satisfaction and experience 2) Determine if the active use and discussion of PROMs during new patient clinic visits are associated with patient activation; and 3) To determine if the objectives (1) and (2) differ based on surgeon or sociodemographic factors. Methods: This Institutional Review Board (IRB) approved singled-blinded RCT was registered prior to data collection on ClinicalTrials.gov (NCT04654910). As a standard of care, new foot and ankle patients completed PROMIS Physical Function (PF), Pain Interference (PI), and Depression assessments. Patients were then randomized to viewing and discussing their PROMIS scores with their surgeon or not. Following the clinic visit, patients completed a series of Clinician & Group Survey-Consumer Assessment of Healthcare Providers and Systems (CG-CAHPS) questions and the Patient Activation Measure (PAM). CG-CAHPS findings and PAM responses were compared between the two groups and after clustering on surgeon. Potential interaction effects by social deprivation were also explored. Results: After enrolling patients but removing those lost to follow-up or with missing data, 97 and 116 patients remained in the intervention control cohorts, respectively. No difference was found in CG-CAHPS responses, nor PAM scores between the two groups (p>0.05). All surgeons were highly rated by all patients. When clustered by surgeon, intervention subjects were less likely to indicate “top box” scores for the understanding domain of the CG-CAHPS question (OR:0.51;p < 0.001) and had decreased odds of high patient activation compared to control subjects (OR:0.67;p=0.005). Among the most socially disadvantaged patients, there was no difference in control and intervention subjects in their likelihood of having high patient activation (p=0.09). Conclusion: Well perceived foot and ankle surgeons who show and discuss PROMs results may not improve the patient experience or activation and may, in fact, decrease understanding or patient activation in select populations. Future work is needed to determine when PROMs discussions are most beneficial and how best to present PROMs data. Health literacy tools and/or communication training may better engage different patient groups regarding PROMs.

Use of patient-reported outcome measures for oncology drugs receiving accelerated approval.

Patient-reported outcomes (PROs) represent an important evaluation of health-related quality of life that has become more commonly incorporated into oncology drug clinical trials. The frequency of PRO inclusion as an endpoint in oncology drug clinical trials leading to the initial accelerated approval of a new therapy is not yet known. We conducted a cross-sectional study evaluating all new drug applications submitted to the FDA over the past 10 years (2013-2022) that led to the initial approval of an oncology drug through the accelerated approval process. The objective was to assess whether the trials leading to such an approval included PROs. Between 2013 and 2022, the FDA approved 59 unique drugs for an oncology indication via the accelerated approval pathway, and 35 (59%) included a PRO assessment in the clinical trial. A median of 1 PRO measurement was used in each trial, with 23 different types of PRO assessment tools were used across the 59 new drug applications. In summary, we found that PRO measurements are inconsistently utilized in trials leading to initial accelerated approval of oncology drugs, and there seems to be a lack of harmonization of different PRO measurement tools used across trials.

Quantifying the Subjective Experience of Nasal Obstruction: A Review.

Nasal obstruction is an exceedingly common problem and challenging to treat due to its multifactorial etiology. Therefore, measuring treatment outcomes of nasal obstruction can be equally complex yet vital to appropriately assessing symptom improvement or resolution. Both physiologic and anatomic assessments of the nasal airway exist in addition to validated patient-reported outcome measures (PROMs), which objectify subjective nasal obstruction and sinonasal symptoms. Correlation between objective and subjective treatment outcome measures is controversial with clinical guidelines favoring the use of PROMs for surgical treatment of nasal obstruction. In this review, the anatomic and physiologic measurements of the nasal airway and validated PROMs will be discussed, as well as the rationale for implementing PROMs into the rhinoplasty surgeon's practice.

Evaluating the Use of PROMs in Paediatric Orthopaedic Registries.

Patient-reported outcome measures (PROMs) provide structured information on the patient's health experience and facilitate shared clinical decision-making. Registries that collect PROMs generate essential information about the clinical course and efficacy of interventions. Whilst PROMs are increasingly being used in adult orthopaedic registries, their use in paediatric orthopaedic registries is not well known. The purpose of this systematic review was to identify the frequency and scope of registries that collect PROMs in paediatric orthopaedic patient groups. In July 2023, six databases were systematically searched to identify studies that collected PROMs using a registry amongst patients aged under 18 years with orthopaedic diagnoses. Of 3190 identified articles, 128 unique registries were identified. Three were exclusively paediatric, 27 were majority paediatric, and the remainder included a minority of paediatric patients. One hundred and twenty-eight registries collected 72 different PROMs, and 58% of these PROMs were not validated for a paediatric population. The largest group of orthopaedic registries collected PROMs on knee ligament injuries (21%). There are few reported dedicated orthopaedic registries collecting PROMs in paediatric populations. The majority of PROMs collected amongst paediatric populations by orthopaedic registries are not validated for patients under the age of 18 years. The use of non-validated PROMs by registries greatly impedes their utility and impact. Dedicated orthopaedic registries collecting paediatric-validated PROMs are needed to increase health knowledge, improve decision-making between patients and healthcare providers, and optimise orthopaedic management.

Defining and Promoting Pediatric Pulmonary Health: Leveraging Patient Reported Outcomes.

Patient-reported outcomes are based on patient (or caregiver) descriptions without direct measurement by a health care provider. To capture patient-reported outcomes, various patient-reported outcome measures (PROMs) have been created. Using PROMs has been linked to improved patient satisfaction, patient-provider communication, and clinical outcomes in many pediatric fields. Despite a long-standing history of utilizing PROMs for the evaluation and management of childhood asthma, pediatric pulmonologists lag behind other pediatric subspecialists in the use of PROMs. During the National Heart, Lung, and Blood Institute's "Defining and Promoting Pediatric Pulmonary Health" workshop, critical knowledge gaps and research opportunities in the use of PROMs for childhood respiratory health were reviewed. In particular, PROMs can be employed as screening tools in the general population for the primary or secondary prevention of pediatric lung diseases. Incorporating these PROMs into the pediatric primary care setting would be especially impactful. In addition, the use of PROMs for the evaluation and management of asthma suggests that they can be applied to other childhood respiratory diseases. Ongoing multicenter studies or national consortia that study pediatric lung diseases could be leveraged to conduct research designed to develop, validate, and assess the utility of PROMs to assess childhood respiratory health. Harnessing the electronic health record will be critical for the successful adoption of PROMs in children with lung diseases. Ultimately, an integrative approach to systematically address numerous barriers at the level of the provider, patient, and health care system will be needed to attain this goal and achieve sustainability.

Reliability, Measurement Error, Responsiveness, and Minimal Important Change of the Patient-Specific Functional Scale 2.0 for Patients With Nonspecific Neck Pain.

The Patient-Specific Functional Scale (PSFS) is a patient-reported outcome measure used to assess functional limitations. Recently, the PSFS 2.0 was proposed; this instrument includes an inverse numeric rating scale and an additional list of activities that patients can choose. The aim of this study was to assess the test-retest reliability, measurement error, responsiveness, and minimal important change of the PSFS 2.0 when used by patients with nonspecific neck pain. Patients with nonspecific neck pain completed a numeric rating scale, the PSFS 2.0, and the Neck Disability Index at baseline and again after 12weeks. The Global Perceived Effect (GPE) was also collected at 12weeks and used as an anchor. Test-retest measurement was assessed by completion of a second PSFS 2.0 after 1week. Measurement error was calculated using a Bland-Altman plot. The receiver operating characteristic method with the anchor (GPE) functions as the reference standard was used for calculating the minimal important change. One hundred patients were included, with 5 lost at follow-up. No floor and ceiling effects were reported. In the test-retest analysis, the mean difference was 0.15 (4.70 at first test and 4.50 at second test). The ICC (mixed models) was 0.95, indicating high agreement (95% CI = 0.92-0.97). For measurement error, the upper and lower limits of agreement were 0.95 and -1.25 points, respectively, with a smallest detectable change of 1.10. The minimal important change was determined to be 2.67 points. The PSFS 2.0 showed satisfactory responsiveness, with an area under the curve of 0.82 (95% CI = 0.70-0.93). There were substantial to high correlations between the change scores of the PSFS 2.0 and the Neck Disability Index and GPE (0.60 and 0.52, respectively; P < .001). The PSFS 2.0 is a reliable and responsive patient-reported outcome measure for use by patients with neck pain.

Cross-cultural adaptation of the Genetic Counseling Outcome Scale (GCOS-24) for use in Canada: A qualitative study.

The Genetic Counselling Outcome Scale (GCOS)-24 is a patient-reported outcome measure (PROM) developed and validated in the United Kingdom (UK). The aim of this study was to cross-culturally adapt GCOS-24 to Canadian Clinical Genetic Services (CGS). This was achieved through a qualitative study exploring whether the existing GCOS-24 maintains its intended meaning in a Canadian population and assessing whether GCOS-24 items could be better worded to meet the needs of members of families affected by genetic conditions in Canada. Thirteen participants were recruited from Canadian Patient Organizations supporting people and families affected by genetic conditions. Data were collected through semi-structured cognitive interviews, as these allow exploration of participants' comprehension, opinions, thoughts, and feelings regarding GCOS-24's instructions, response options, and the meaning/relevance of each item. Thematic analysis was utilized for data analysis, and an inductive approach to coding was followed to allow for themes to emerge from the data. Themes were organized in respect to their questionnaire item and further classified into their respective Empowerment dimension. The GCOS-24 instructions were found easy to understand by all thirteen participants. Although the response options were also found to be straightforward, the data suggest the questionnaire would benefit from the addition of a "non-applicable" option. Semantic validation of the GCOS-24 showed that items within the Cognitive Control and Emotional Regulation dimensions were found easy to understand by participants. However, items within the Decisional Control, Behavioural Control and Hope dimensions presented semantic difficulties. Participants provided feedback on syntactic changes to support understanding, and this feedback was used to develop a final Canadian-adapted version of GCOS-24, GCOS-Canada. This study provides the first step towards a valid, culturally adapted PROM for use in Canadian CGS service evaluation and research. GCOS-Canada would benefit from psychometric validation to ensure validity, reliability, responsiveness, minimal clinically important difference and internal consistency.

Patient experience of spontaneous intracranial hypotension (SIH): qualitative interviews for concept elicitation

Background & objectivesSpontaneous intracranial hypotension (SIH) is an underdiagnosed and debilitating condition caused by a spinal cerebrospinal fluid (CSF) leak. Although SIH can lead to substantial morbidity and disability, little data exists about patients’ perspectives. Without hearing directly from patients, our understanding of the full experience of having SIH is limited, as is our ability to identify and use appropriate patient-reported outcome measures (PROMs) within clinical care and research. The purpose of this study was to conduct qualitative interviews with confirmed SIH patients to fully describe their experiences and identify relevant concepts to measure.MethodsPatients were recruited from an SIH specialty clinic at a large, U.S.-based healthcare center. Patients undergoing an initial consultation who were ≥ 18 years old, English-speaking, met the International Classification of Headache Disorders-3 criteria for SIH, and had a brain MRI with contrast that was positive for SIH were eligible to participate. During semi-structured qualitative interviews with a trained facilitator, participants were asked to describe their current SIH symptoms, how their experiences with SIH had changed over time, and the aspects of SIH that they found most bothersome. Analysts reviewed the data, created text summaries, and wrote analytic reports.ResultsFifteen participants completed interviews. Common symptoms reported by patients included headache, tinnitus, ear fullness/pressure/pain, and neck or interscapular pain. Patients reported that their symptoms worsened over the course of their day and with activity. The most bothersome aspect of SIH was disruption to daily activities and limits to physical activities/exercise, which were severe. With regard to symptoms, the most bothersome and impactful included physical pain and discomfort (including headache), as well as fatigue.ConclusionsPatients reported a diverse set of symptoms that were attributed to SIH, with devastating impacts on functioning and high levels of disability. Researchers considering use of PROMs for SIH should consider inclusion of both symptom scales and aspects of functioning, and future work should focus on evaluating the validity of existing measures for this patient population using rigorous qualitative and quantitative methods in diverse samples. Additionally, these data can be used to assist clinicians in understanding the impacts of SIH on patients.

Patient-Reported Outcome Measure Use by Acute Care Pediatric Physical Therapists

Purpose: To describe the current use of and perceived benefits and barriers to using patient-reported outcome measures (PROMs) by pediatric physical therapists (PTs) in the acute care setting and to explore demographic factors that may explain the use of PROMs. Methods: An electronic survey was distributed to PTs who work in a pediatric acute care setting. Descriptive statistics were used to assess the frequency of PROM use, identify differences in PROM use based on demographic factors, and evaluate the likelihood of various benefits and barriers. Open-ended survey responses were analyzed inductively to identify common themes. Results: The survey was completed by a convenience sample of 92 pediatric acute care PTs (30% response rate). Half of the respondents (n = 46; 50%) reported “rarely” or “never” using PROMs, while only 4.3% (n = 4) reported “always” using PROMs. No significant differences were found in the frequency of PROM between years of experience, entry-level degree, clinical specialization, or other demographic factors. Participants were “somewhat” or “extremely likely” to use the rate of perceived exertion (71%), the Ages and Stages Questionnaire (24%), and the Pediatric Quality of Life Inventory (17%). The primary facilitator was improving communication with patients (68%), while the main barrier was lack of availability (57%). Thematic analysis of open-ended responses indicated that the benefits of using PROMs include improving communication, providing objective data, and measuring progress. Barriers identified include inadequate resources, lack of time, and irrelevance to clinical practice. Conclusion: Many pediatric acute care PTs underutilize PROMs, with reported barriers of limited access and lack of time hindering implementation within the pediatric acute care setting. Survey respondents reported the desire to measure health-related quality of life and participation, which may be feasible through future quality improvement or knowledge translation initiatives.

Perceptions of Patient-Reported Outcome Measures in Acute Care Pediatric Physical Therapy

Purpose: To explore the perceptions and experiences of acute care pediatric physical therapists (PTs) regarding the use of patient-reported outcome measures (PROMs) and to understand the influence of PROMs on clinical decision-making and the plan of care. Methods: Thirteen acute care pediatric PTs participated in individual semistructured interviews using a qualitative phenomenological approach. Interview questions focused on how PROMs influence clinical practice and decision-making. Interviews also explored scenarios where using PROMs positively or negatively impacted patient and caregiver interactions. Researchers analyzed the interview transcripts using an inductive process to generate codes and resultant themes. Results: Participants described their experiences using PROMs in pediatric acute care, and 4 themes emerged: (1) PROMs can be difficult to implement in pediatric acute care, (2) PROMs can benefit pediatric acute care practice, (3) PROMs can improve service delivery and family-centered care, and (4) ideal PROM use in future practice. The Theoretical Domains Framework (TDF) was used to categorize positive and negative factors related to implementing evidence-based guidelines in the pediatric acute care setting. Conclusions: Acute care pediatric PTs report benefits and barriers to using PROMs with children and caregivers in the hospital setting. Findings support the need for future knowledge translation initiatives to facilitate the use of PROMs in pediatric acute care.

Patient-Reported Outcome Measures in Patients with Thrombotic Thrombocytopenic Purpura: A Systematic Review of the Literature.

Health-related quality of life (HRQoL) impacts of thrombotic thrombocytopenic purpura (TTP) have been captured in clinical studies using patient-reported outcome (PRO) measures (PROMs) that are validated for other diseases. However, the validity evidence to support the use of existing PROMs in patients with TTP is unknown. In a systematic review of the literature, including studies of adults and children with TTP, we assessed the validity evidence for use of PROMs in clinical research and clinical practice, characterized HRQoL, described the integration of PROMs in clinical practice and evaluated PRO scores for patients with TTP compared with reference populations. From an initial 4518 studies, we identified 14 studies using 16 PROMs to assess general HRQoL domains in patients in remission. No identified studies assessed the validity of PROMs for the context of use of TTP and no studies described PROM integration into TTP clinical practice or evaluated PROMs that were specific for patients with TTP. Moreover, PRO scores were worse in patients with TTP compared with reference populations and other chronic conditions. We conclude that, in patients with TTP, PROMs pick up on important patient experiences not captured by clinical outcomes at present. There is, therefore, a need for studies that assess the validity of existing PROMs in patients with TTP to determine if TTP-specific PROMs specific to patients with TTP should be developed.

ANALYZING TRENDS IN PROM AND MCID REPORTING IN TOTAL JOINT REPLACEMENT OVER THE PAST DECADE

AbstractIntroductionMinimum clinically important differences (MCIDs) are critical to understanding changes in patient-reported outcome measure (PROM) scores after total joint arthroplasty (TJA). The usage and adoption of MCIDs not been well-studied. This study was performed to IDENTIFY trends in PROM and MCID use after TJA over the past decade.MethodsAll articles published in the calendar years of 2010 and 2020 in CORR, JBJS, and the Journal of Arthroplasty were reviewed. Articles relating to clinical outcomes in primary total hip arthroplasty (THA) or total knee arthroplasty (TKA) were included. For each article, all reported PROMs and (if present) accompanying MCIDs were recorded. The use of PROMs and MCIDs were compared between articles published in 2010 and 2020.ResultsOverall, 263 articles from 2010 and 546 articles from 2020 were included. The total number of articles reporting any PROM after THA and TKA increased from 131 in 2010 to 194 in 2020, but the proportion of articles reporting PROMs decreased from 49.8% (131/263) to 35.5% (194/546). Both the total number and proportion of articles reporting MCIDs increased from 2.3% (3/131) in 2010 to 16.5% (32/194) in 2020. These trends persisted when analyzing THA and TKA articles individually.ConclusionsBoth the absolute number and proportion of articles reporting MCIDs in conjunction with PROMs after TJA has increased in the past decade but remains low. We recommend that journal editors and meeting organizers encourage the inclusion of MCID information in all reports on clinical outcomes after joint replacement.

Agreement between video-based clinician-rated tools and patient-reported outcomes on gait assessment in individuals with multiple sclerosis.

To our knowledge, no studies compared the video-clinician-based tools and patient-reported questionnaires in assessing gait and balance in people with MS (pwMS). The present study investigated the correlation and agreement between video-clinician-based objective measurement tools and patient-reported outcome measures (PROMs) in gait and balance evaluation. A prospective cross-sectional study was conducted with 55 pwMS. Video analysis-based gait was evaluated by the Tinetti Gait Assessment (TGA), Gait Assessment and Intervention Tool (GAIT), and Functional Ambulation Classification Scale (FACS) by the clinician. Participants' self-reported gait and balance were assessed with the Multiple Sclerosis Walking Scale-12 (MSWS-12) and Activity-Specific Balance Confidence Scale (ABC). There was a moderate positive correlation between ABC with TGA and FACS (r1: 0.552, r2: 0.510, p < 0.001). ABC was strongly correlated with GAIT (r: - 0.652, p < 0.001). A moderate positive correlation was observed between MSWS-12 with TGA and FACS (r1: - 0.575, r2: - 0.524, p < 0.001). In addition, there was a strong positive correlation between MSWS-12 and GAIT (r: - 0.652, p < 0.001). Clinician-rated tools and PROMs were within the agreement limits regarding the unstandardized beta values p < 0.001). Clinician-based gait and balance tools demonstrate consistent results with PROMs in pwMS. Considering the low cost and practical use of PROMs, in cases where video-based clinician-based measurements cannot be provided (time, space, and technical inadequacies), questionnaires can provide concordant results at moderate and severe levels compared with objective tools.

Do clinical trials prepare to fail by failing to prepare? An examination of MS trials and recommendations for patient-reported outcome measure selection.

Many clinical trials use patient-reported outcome (PRO) measures, which can influence treatment decision-making, drug approval and label claims. Given that many PRO measure options exist, and there are conceptual and contextual complexities with PRO measurement, we aimed to evaluate how and why specific PRO measures have been selected for pivotal multiple sclerosis (MS) clinical trials. Specifically, we aimed to identify the reasons documented for PRO measure selection in contemporary phase III MS disease-modifying treatment (DMT) clinical trials. We searched for phase III clinical trials of MS DMTs published between 2015 and 2021 and evaluated trial protocols, or primary publications where available, for PRO measure selection information. Specifically, we examined study documents for their clarification of clinical concepts measured, definitions of concepts measured, explanations of which PRO measures were considered, why specific PRO measures were chosen, and trade-offs in PRO measure selection. We identified 1705 abstracts containing 61 unique phase III MS DMT clinical trials. We obtained and examined 27/61 trial protocols. Six protocols were excluded: four contained no mention of PRO measures and two contained redacted sections preventing adequate assessment, leaving 21 protocols for assessment. For the remaining 34 trials (61-27), we retrieved 31 primary publications; 15 primary publications mentioned the use of a PRO measure. None of the 36 clinical trials that mentioned the use of PRO measures (21 protocols and 15 primary publications) documented clear PRO or clinical outcome assessment (COA) measurement strategies, provided clear justifications for PRO selection, or reasons why specific PRO measures were selected when alternatives existed. PRO measure selection for clinical trials is not evidence-based or underpinned by structured systematic approaches. This represents a critical area for study design improvement as PRO measure results directly affect patient care, PRO measurement has conceptual and contextual complexities, and there is a wide range of options when selecting a PRO measure. We recommend trial designers use formal approaches for PRO measure selection to ensure PRO measurement-based decisions are optimised. We provide a simple, logical, five-stage approach for PRO measure selection in clinical trials.

Psychometrics of the patient-reported outcomes measurement information system measures in hemophilia: the applicability of the pediatric item banks

BackgroundThe use of patient-reported outcomes measures (PROMs) is important in hemophilia care, as it facilitates communication between patients and clinicians and promotes patient-centered care. Currently, a variety of PROMs with insufficient psychometric properties are used. Patient-reported outcomes measurement information system (PROMIS) measures, including Computer Adaptive Tests, were designed to measure generically and more efficiently and, therefore, are an alternative for the existing PROMs. ObjectivesTo assess the feasibility, measurement properties, and outcomes of 8 PROMIS pediatric measures for boys with hemophilia. MethodsIn this multicenter study, boys with hemophilia completed 8 PROMIS measures and 2 legacy instruments. Feasibility was determined by the number of completed items and floor or ceiling effects (percentage of participants that achieved the lowest or highest possible score). Reliability was assessed as the percentage of scores with a SE ≤ 4.5. Construct validity was evaluated by comparing the PROMIS measures with the legacy instruments. Mean PROMIS T-scores were calculated and compared with the Dutch general population. ResultsIn total, 77 boys with hemophilia participated. Reliability was good for almost all PROMIS measures and legacy instruments. The total number of completed items varied from 49 to 90 for the PROMIS pediatric measures, while the legacy instruments contained 117 to 130 items. Floor and ceiling effects were observed in both the PROMIS measures (0-39.5%) and legacy instruments (0-66.7%), but were higher for the legacy instruments. ConclusionsThe PROMIS pediatric measures are feasible to use for boys with hemophilia. With the use of the PROMIS measures in clinical care and research, a step toward worldwide standardization of PROM administration can be taken.

Properties of Patient-Reported Outcome Measures for Post-Traumatic Stress Disorder in North Korean Defectors: A Scoping Review.

Posttraumatic stress disorder (PTSD) is highly prevalent among North Korean defectors. This scoping review aimed to appraise the measurement properties of patient-reported outcome measures (PROMs) that assess PTSD among North Korean defectors. A systematic search was conducted using Research Information Sharing Service, ScienceON, PubMed, and Embase from their inception up to September 15, 2022. Each measurement properties of the included PROMs were evaluated using COnsensus-based Standards for the selection of health status Measurement INstruments (COSMIN) methodology. First, the methodological quality of each study on measurement properties was evaluated using the COSMIN Risk of Bias checklist. Second, the result of each single study on a measurement property was rated against the updated criteria for good measurement properties. After screening the articles, nine instruments from 40 studies were included. Among these instruments, Impact of Event ScaleRevised was the most frequently used (13 studies [38.1%]), followed by the Trauma Scale for North Korean Refugees, PTSD Checklist, and Posttraumatic Diagnostic Scale. The theoretical basis of all PROMs was secure in terms of content validity. The most frequently tested characteristics in measurement properties for internal structure were internal consistency, criterion validity, and convergent validity. No study reported sufficient structural validity as confirmatory factor analysis results with a model fit. There is insufficient evidence to support the recommendation of a specific PROM for use among North Korean defectors with PTSD.