Assessing the Trajectory of Schizophrenia Effectively in Order to Treat Effectively.

INTRODUCTION: Schizophrenia is a complex psychiatric disorder characterized by heterogeneous symptom trajectories that significantly impact patient outcomes. We believe that the study of the trajectories of Schizophrenia is useful in assessing treatment options and outcomes. While the Positive and Negative Syndrome scale is usually used on one occasion to measure symptoms at a single time, if measured repeatedly , the PANSS is also useful in measuring trajectories. In order to illustrate and promote this serial use, we have reviewed papers which describe the delineation of Trajectories of Symptoms in Schizophrenia based on PANSS scores. This review integrates findings from longitudinal studies focusing on the trajectories of positive symptoms, negative symptoms, the relation between positive and negative symptoms and cognition, soft neurological signs, and treatment response in schizophrenia. METHODS: Studies were identified from the PUBMED database .Studies included in this review employed diverse methodologies such as trajectory analyses, longitudinal assessments, and clinical trials. Data were extracted from a range of patient cohorts, including those with first-episode psychosis and chronic schizophrenia. RESULTS: Longitudinal studies consistently demonstrate variability in the trajectories of positive symptoms, with most patients experiencing early stable remission, though a subgroup exhibits persistent or fluctuating symptomatology. Negative symptoms, on the other hand, often show poor improvement over time, correlating with impaired social and neurocognitive functioning. Cognitive deficits also vary, with some domains showing improvement while others, such as logical memory, deteriorate in certain patient subgroups. The relationship between positive and negative symptom trajectories highlights their complex relationship, influencing overall functioning and treatment outcomes. Antipsychotic medications demonstrate varied responses across patient cohorts, with distinct trajectory patterns observed based on medication type and patient-specific factors such as co-morbid substance abuse and duration of untreated psychosis. CONCLUSION: Understanding the longitudinal trajectories of symptoms in schizophrenia is crucial for optimizing therapeutic strategies and improving patient outcomes. Personalised interventions tailored to individual symptom profiles and early clinical responses are recommended to enhance treatment efficacy and promote recovery. The PANSS scale can be used to delineate Trajectories of various symptom Groups in Schizophrenia.

Selecting informative patients for phase 2 progressive trials in MS: Design considerations for phase 2 clinical trials in progressive MS.

While relapsing-remitting multiple sclerosis (MS) has many therapeutic options, progressive forms of MS remain largely untreatable. Phase 2 clinical trials are our main tool to advance new treatments for progressive MS. Given the complexities of progressive MS, it will likely require many phase 2 trials to improve its treatment. To conduct informative and efficient phase 2 trials, it is important that such trials are designed in a way that they can identify a successful treatment as quickly and with as few participants as possible. In this topical review, we discuss cohort selection, outcome selection, cohort enrichment, and dosing selection as strategies to optimize the efficiency of phase 2 clinical trials in progressive MS.

Perceptions of adults with type 1 diabetes toward diabetes-specific quality of life measures: a survey-based qualitative exploration.

BACKGROUND: Diabetes-specific quality of life (QoL) questionnaires are commonly used to assess the impact of diabetes and its management on an individual's quality of life. While several valid and reliable measures of diabetes-specific QoL exist, there is no consensus on which to use and in what setting. Furthermore, there is limited evidence of their acceptability to people with diabetes. Our aim was to explore perceptions of adults with type 1 diabetes (T1D) toward five diabetes-specific QoL measures. METHODS: Adults (aged 18 + years) with T1D living in Australia or the United Kingdom (UK) were eligible to take part in 'YourSAY: QoL', an online cross-sectional survey. Recruitment involved study promotion on diabetes-related websites and social media, as well as direct invitation of people with T1D via a hospital client list (UK only). In random order, participants completed five diabetes-specific QoL measures: Audit of Diabetes-Dependent Quality of Life (ADDQoL-19); Diabetes Care Profile: Social and Personal Factors subscale (DCP); DAWN Impact of Diabetes Profile (DIDP); Diabetes-Specific Quality of Life Scale: Burden Subscale (DSQoLS); Diabetes Quality of Life Questionnaire (Diabetes QOL-Q). They were invited to provide feedback on each questionnaire in the form of a brief free-text response. Responses were analysed using inductive, thematic template analysis. RESULTS: Of the N = 1,946 adults with T1D who completed the survey, 20% (UK: n = 216, Australia: n = 168) provided qualitative responses about ≥ 1 measure. All measures received both positive and negative feedback, across four themes: (1) clarity and ease of completion, e.g., difficulty isolating impact of diabetes, dislike of hypothetical questions, and preference for 'not applicable' response options; (2) relevance and comprehensiveness, e.g., inclusion of a wide range of aspects of life to improve personal relevance; (3) length and repetition, e.g., length to be balanced against respondent burden; (4) framing and tone, e.g., preference for respectful language and avoidance of extremes. CONCLUSIONS: These findings suggest opportunities to improve the relevance and acceptability of existing diabetes-specific QoL measures, and offer considerations for developing new measures, which need to be better informed by the preferences of people living with diabetes.

Intergenerational transmission of adverse and positive childhood experiences and associations with child well-being.

BACKGROUND: Parental adverse childhood experiences (ACEs) contribute to offspring adversity and poor health outcomes, but little is known about whether and to what extent parental positive childhood experiences (PCEs) influence offspring positive experiences and well-being. OBJECTIVE: To investigate the association between parent and child ACEs and PCEs and their impact on child well-being and psychopathology. PARTICIPANTS AND SETTING: A national sample of n = 1016 US parents of 1-5-year-olds completed online surveys in September 2019. METHODS: Mediation analysis in a path modeling framework was used with stratified probability weights for generalizability to the US population. RESULTS: Each additional parent PCE equated to 0.32 (95 % CI: 0.20, 0.45) increase in child PCEs and each additional parent ACE equated to 0.18 (95 % CI: 0.06, 0.31) increase in child PCEs and 0.13 (95 % CI: 0.08, 0.18) increase in child ACEs. In turn, an increase in 1 child PCE was associated with 0.10-0.16 SD increase in well-being and 0.06-0.10 decrease in psychopathology, and each additional child ACE equated to 0.10-0.18 SD increase in psychopathology. CONCLUSIONS: Results support the intergenerational transmission of PCEs and ACEs, advancing understanding of the role that parent PCEs play in promoting child PCEs and fostering child well-being. Findings underscore the importance of extending clinical surveillance of ACEs to include PCEs in pediatric and adult healthcare settings. Dual-generation programs that address the negative consequences of parental ACEs may be able to increase their impact by adding a parallel emphasis on PCEs and providing parents with tools to foster PCEs in their children.

Cognitive interviewing validation of the Chinese version of the neurogenic bladder symptom score.

Background: The neurogenic bladder symptom score (NBSS) has been widely used to specifically measure symptoms and consequences of neurogenic bladder (NB). The cognitive interviewing (CI) is effective in assessing item clarity and identifying key issues related to the comprehension of the instrument. We aim to translate the NBSS into Chinese and use the CI approach to explore the thought processes of patients with NB in responding the Chinese Version of the NBSS, identify and modify the factors hinder the thought processes to enhance the face validity of the NBSS. Methods: The translation of the NBSS into Chinese was conducted with the guidance of the recommended frameworks. Patients with NB were recruited by purpose sampling. CI with the combination of thinking aloud and verbal probing techniques were used to explore thought processes. The interviews were transcribed and analyzed based on Tourangeau four-stage response model. Results: Two rounds of CI were carried out. The problems of comprehension, judgement and response mapping were identified in 8 items. Four items were revised based on the results of the interview. The revised items were verified and eventually integrated into the final version. Conclusion: The Chinese Version of the NBSS was easy to comprehend and use. The use of CI methodologies can increase the comprehensibility and cultural applicability of the NBSS, providing the evidence for the development of a clearer and more appropriate questionnaire.

Outcome measurement of cognitive impairment and dementia in serious digital games: a scoping review.

PURPOSE: Dementia prevalence is increasing worldwide. With the emergence of digital rehabilitation, serious digital games are a potential tool to maintain and monitor function in people living with dementia. It is unclear however whether games can measure changes in cognition. We conducted a scoping review to identify the types of outcomes measured in studies of serious digital games for people with dementia and cognitive impairment. METHODS: We included primary research of any design including adults with cognitive impairment arising from dementia or another health condition; reported data about use of serious digital games; and included any cognitive outcome. We searched Medline (via EBSCO), PsycInfo, CINAHL, Web of Science, from inception to 4th March 2024 and extracted study characteristics. RESULTS: We reviewed 5899 titles, including 25 full text studies. We found heterogeneity in domains and measures used: global cognition (n = 15), specific cognitive processes (n = 13), motor function (n = 5), mood (n = 6), activities of daily living (n = 5), physiological processes (n = 4) and quality of life (n = 2). Use of outcome measurement tools was inconsistent; the most frequently used measures were the Montreal Cognitive Assessment (n = 8), the Mini-Mental State Examination (n = 7), and the Trail Making Test (n = 7). Nine studies used in-game measures, most of which were related to game performance. CONCLUSION: We found very few studies with assessment of cognition within the game. Studies of serious games for people with dementia and cognitive impairment should develop digital outcome tools based on recommendations in Core Outcome Sets, to increase consistency between studies.

Fewer than half of the studies we identified used in-game measures, most of which were related to game performance, indicating that digital measures of cognition within serious digital games is a largely unexplored research area.Only eight out of 25 included studies used measurements tools recommended in Core Outcome Sets for studies of people living with dementia.Given the heterogeneity of measures identified in this review, critical appraisal of relevant outcome measures would be the next step in determining suitable measures for use in future research.Following critical appraisal, exploration of digital measures of cognition is needed to determine their integration into serious games, and whether existing tests can be digitised and administered remotely whilst retaining psychometric properties.

Changes in hand function and health state utility after cubital tunnel release using the United Kingdom Hand Registry.

This study aimed to analyse and contrast changes in health-related quality of life (HR-QoL) and hand symptoms in the first 6 months after surgical treatment for primary cubital tunnel syndrome. Data originated from the United Kingdom Hand Registry. HR-QoL was assessed using the generic EuroQol five-dimensional assessment tool (EQ-5D-5L) and hand symptoms using the Patient Evaluation Measure (PEM). In total, 281 patients were included in the statistical analysis. Cubital tunnel release resulted in clinically relevant relief of hand symptoms. However, no improvement in HR-QoL was detected by the EQ-5D-5L. As a result, current health economic models, such as those used by the National Institute for Health Care Excellence (NICE) in the UK, might conclude that cubital tunnel release is not cost-effective. This discrepancy requires exploration, and hand-specific preference-based measures might be needed for value-based healthcare in hand surgery.Level of evidence: III.

Current use of patient-reported outcome measures by musculoskeletal physiotherapists in Portugal.

BACKGROUND: Musculoskeletal conditions are the leading cause of disability, requiring continuous outcome measurement, including the utilization of patient-reported outcome measures (PROMs). OBJECTIVE: The aims of the study were a) to characterize the use PROMs by Portuguese physiotherapists in patients with musculoskeletal conditions; b) to analyze the associated factors of their use and; c) to identify the barriers and facilitators to their implementation. METHODS: A cross-sectional online survey was carried out with musculoskeletal physiotherapists working in Portugal. Closed questions were used to describe the use of PROMs. The factors associated with the non-use of PROMs were analysed through logistic regression models (p= 0.05). Barriers and facilitators were assessed through open questions and analyzed using thematic analysis. RESULTS: One hundred and fifty-six physiotherapists answered the online survey. Most physiotherapists do not use outcome measures consistently and physical tests are often chosen (70.5%) over PROMs (17.3%). The work sector (p= 0.01; p= 0.015; p= 0.009). the workplace stimulus (p= 0.001) and the physiotherapists' knowledge about PROMs (p= 0.017) were identified as factors associated with the non-use of PROMs. Barriers and facilitators to the use of PROMs are related to physiotherapists' skills, the patients' characteristics, the workplace conditions, and the instruments' characteristics. CONCLUSION: This study showed the poor use of PROMs by musculoskeletal physiotherapists and identified the associated factors, barriers, and facilitators that should be considered to increase its future use by musculoskeletal physiotherapists in Portugal.

Construct validity of PROMIS pain interference, fatigue, and physical function as patient-reported outcomes in adults with idiopathic inflammatory myopathies: An international study from the OMERACT myositis working group.

BACKGROUND: Validated patient-reported outcome measures to assess disease impact in patients with adult idiopathic inflammatory myopathies (IIMs) are needed. The objective of this study was to assess the construct validity of PROMIS Pain Interference, Fatigue, and Physical Function measures in comparison with core disease activity measures. METHODS: Adults with IIM, excluding inclusion body myositis, from OMERACT Myositis Working Group (MWG) clinic sites completed PROMIS Short Form v1.0-Pain Interference 6a, PROMIS Short Form v1.0-Fatigue 7a, and PROMIS Short Form v2.0-Physical Function 8b measures. Core disease activity measures including patient and physician global disease activity assessments, manual muscle testing, serum creatine kinase activity, and Health Assessment Questionnaire Disability Index (HAQ-DI) were simultaneously assessed. To evaluate construct validity, a priori hypotheses for the expected correlations between PROMIS measures, age, and core disease measures were determined by >70 % agreement among MWG members and were compared against observed Pearson's correlations. Internal consistency of items and floor or ceiling effects for the PROMIS measures were also assessed. Subgroup analysis according to IIM subtype (dermatomyositis vs. non-dermatomyositis IIM) was performed. RESULTS: 135 adults with IIM from 5 countries across North America, Europe, Asia, and Australia were included. For construct validity, a priori hypotheses were confirmed for 5 of 6 (83 %) PROMIS Pain Interference, 4 of 5 (80 %) PROMIS Fatigue, and 3 of 4 (75 %) PROMIS Physical Function correlations. Internal consistency was high for each PROMIS measure (Cronbach's alpha >0.9). Ceiling effects were observed only for PROMIS Pain Interference, with low/no pain in 29 % of patients. Subgroup analysis between dermatomyositis (n = 65) and non-dermatomyositis (n = 70) subtypes demonstrated similar correlations between PROMIS measures and disease activity measures. CONCLUSIONS: PROMIS Short Form v1.0-Pain Interference 6a, PROMIS Short Form v1.0-Fatigue 7a, and PROMIS Short Form v2.0-Physical Function 8b measures demonstrate strong construct validity when compared to core disease activity measures in IIM, with consistent results across IIM subtypes. These findings support the use of these selected PROMIS measures to assess core domains of interest for measuring life impact in IIMs.

Supporting follow-up care in colorectal cancer patients: first experiences with the Assessment of Burden of ColoRectal Cancer (ABCRC)-tool.

PURPOSE: The Assessment of Burden of ColoRectal Cancer (ABCRC)-tool is a unique tool that includes a PROM focused on health-related quality of life (HRQoL), functional outcomes and lifestyle assessment. Furthermore, it provides visualization of results and treatment advice. The tool aims to support follow-up consultations of colorectal cancer (CRC) patients. The purpose of this study was to evaluate the first experiences of patients and healthcare professionals (HCPs) with the ABCRC-tool. METHODS: The ABCRC-tool was implemented in two Dutch hospitals and used by 25 patients and 5 HCPs during follow-up care. Consultations were audio-recorded and their content was analyzed independently by two researchers. Semi-structured interviews with patients and HCPs were conducted after the consultation. Interviews focused on the overall experience with the tool, ease of use, interpretation of the PROM and the visualized results and on the added value of the tool. RESULTS: Audio recording revealed that multiple topics, relevant to patients, were discussed during consultations with the ABCRC-tool. Patients and HCPs appreciated the ABCRC-tool as it added structure to the consultation, was helpful in the preparation of consultations and provided useful and convenient treatment options. The tool was easy to use, and the visualization was clear and informative. HCPs suggested that the tool is likely to be most relevant for patients in the first year of follow-up. CONCLUSIONS: This study implies that the ABCRC-tool is of added value for patients and their HCPs. Future research should focus on the evaluation of broad implementation, across a wide range of CRC patients. IMPLICATIONS FOR CANCER SURVIVORS: The ABCRC-tool is a valid tool to support CRC survivors and HCPs to monitor and visualize experienced burden of disease and lifestyle parameters in order to optimize personalized care.

Measuring successful conversations in couples with and without aphasia: A scoping review.

BACKGROUND: Aphasia impacts communication and relationships. While counselling is increasingly recognised as a component of the speech-language therapy role, the success of conversation partner training is typically measured in terms of communication alone. This scoping review aimed to describe how successful conversation is currently measured with couples-with and without aphasia, to inform the development of an ecologically valid measure for couples with aphasia. METHODS AND PROCEDURES: The scoping review was reported in alignment with the Preferred Reporting Items for Systematic Reviews and Meta-Analysis Extensions for Scoping Reviews (PRISMA-SCR). MEDLINE, EMBASE, CINAHL and PsycINFO databases were searched for conversation treatment studies for couples with and without aphasia. Patient-reported outcome measures (PROMs) were extracted from included studies and screened against the three-tier model of situated language to shortlist those that measure everyday communication. Items within shortlisted PROMs were further described using the refined International Classification of Functioning, Disability and Health linking rules. RESULTS: Following screening and full-text review, 46 studies were included, consisting of 24 studies conducted with couples with aphasia and 22 studies conducted with couples without aphasia. For couples with aphasia, 13 PROMs were identified that measured everyday communication. Of these, 23% were dyadic (i.e., measured from the perspectives of both members of the couple); however, they usually only appraised the communication behaviours of the person with aphasia. For couples without aphasia, eight PROMs were identified that measured everyday communication; all were dyadic and measured both attitudes and communication behaviours of both partners. CONCLUSION: Conversation relies on the interaction of two people, and success in conversation is best rated by those having the conversation. The use of PROMs is recommended as part of person and relationship-centred practice; however, there is currently no validated PROM for conversation in aphasia that considers the perspectives and behaviours of both the person with aphasia and their communication partner. The PROM items identified in this study will form the basis of future research to develop a PROM for couples' conversations in aphasia. WHAT THIS PAPER ADDS: What is already known on the subject Communication partner training (CPT) for couples, where one person has aphasia, can improve conversation skills and enhance relationships. Clinician-rated measures, such as conversation analysis, have been used to measure the outcomes of CPT. However, there is a lack of person-centred or self-rated outcome measurement instruments. that is, patient-reported outcome measures (PROMs) for couples' conversations in aphasia. What this paper adds to existing knowledge We have identified outcome measurement instruments used in conversation treatment studies for couples with and without aphasia. We found that most PROMs used in aphasia treatment studies were not dyadic, that is, they did not include the self-report of both communication partners. In contrast, the PROMs used with couples without aphasia were dyadic and contained items that measured a more complex range of both partners' emotions, behaviours and attitudes. What are the potential or actual clinical implications of this work? This study provides insights into the content and characteristics of PROMs for couples' conversation therapy and may assist clinicians in selecting outcome measurement instruments in their practice.

Definitions of and Instruments for Disease Activity, Remission, and Relapse in Polymyalgia Rheumatica: A Systematic Literature Review.

OBJECTIVE: To perform a systematic literature review on definitions and instruments used to measure remission, relapse, and disease activity in polymyalgia rheumatica (PMR), to inform an OMERACT project to endorse instruments for these outcomes. METHODS: A search of Pubmed/MEDLINE, EMBASE, CINAHL, Cochrane, and Epistemonikos was performed May 2021 and updated August 2023. Qualitative and quantitative studies published in English were included if they recruited people with isolated PMR regardless of treatment. Study selection and data extraction was performed independently by two investigators and disagreement was resolved through discussion. Data extracted encompassed definitions of disease activity, remission and relapse, and details regarding the instruments used to measure these outcomes. RESULTS: From the 5,718 records, we included 26 articles on disease activity, 36 on remission, and 53 on relapse; 64 studies were observational and 15interventional, and none used qualitative methods. Some heterogeneity was found regarding definitions and instruments encompassing the domains pain, stiffness, fatigue, laboratory markers (mainly acute phase reactants), and patient and physician global assessment of disease activity. However, instruments for clinical signs were often poorly described. Whilst measurement properties of the polymyalgia rheumatica activity score (PMR-AS) have been assessed, data to support its use for measurement of remission and relapse is limited. CONCLUSION: Remission, relapse, and disease activity have been defined heterogeneously in clinical studies. Instruments to measure these disease states still need to be validated. Qualitative research is needed to better understand the concepts of remission and relapse in PMR. REVIEW REGISTRATION: PROSPERO identification: CRD42021255925.

The Use of a Comprehensive Concept of Capability for Wellbeing Assessment: A Best-Fit Framework Synthesis.

Developing an instrument with the capability approach can be challenging, since the capability concept of Sen is ambiguous concerning the burdens that people experience whilst achieving their capabilities. A solution is to develop instruments with a comprehensive concept of capability, such as the concept of 'option-freedom'. This study aims to develop a theoretical framework for instrument development with the concept of option-freedom. A best-fit framework synthesis was conducted with seven qualitative papers by one researcher. Two researchers supported the synthesis by discussing interim results during the synthesis. A priori concepts of option-freedom were used to deductively code against. Themes and subthemes were developed inductively when data did not match a priori themes. Seven paper were identified that fulfilled the eligibility criteria. Four themes emerged from the synthesis. (1) Option Wellbeing represents a range of options that need to be satisfied for individuals to experience wellbeing. (2) Self-Realization represents that there are experiences in an individual's life that have value beyond realizing options. (3) Perceived Access to Options represents the perceived ability of individuals to realize freedoms. (4) Perceived Control represents the experience of having control. Developing an instrument with the proposed framework has two benefits. First, it acknowledges the importance of assessing impediments in realizing capabilities for wellbeing assessment. Secondly, the themes form a broad informational base by including themes related to subjective wellbeing. Future research should study the feasibility of implementing the framework for instrument development.

COSMIN guideline for systematic reviews of patient-reported outcome measures version 2.0.

PURPOSE: Systematic reviews of patient-reported outcome measures (PROMs) are important tools to select the most suitable PROM for a study or clinical application. Conducting these reviews is challenging, and the quality of these reviews needs to be improved. We updated the COSMIN guideline for systematic reviews of PROMs, including the COSMIN Risk of Bias checklist, and the COSMIN criteria for good measurement properties. METHODS: Adaptations to the methodology were based on our experience with applying the COSMIN guideline, through discussions among the authors, and results from two related Delphi studies. RESULTS: The updated guideline places more emphasis on key aspects that are often missing or sub optimally conducted in published systematic reviews of PROMs, such as formulating a well-defined research question and developing a comprehensive search strategy, assessing risk of bias, applying criteria for good measurement properties, summarizing results, and grading the quality of the evidence. We also stress the importance of evaluating the measurement properties of each subscale of a PROM separately and evaluating content validity of all included PROMs. CONCLUSION: The quality of systematic reviews of PROMs can be improved by using this updated version of the COSMIN guideline for systematic reviews of PROMs. Improved quality will lead to better PROM selection and increased standardization of PROM use.

Patient-reported outcome measures (PROMs) are questionnaires that measure aspects of health from the patient perspective. To measure a specific health aspect, often dozens of PROMs are available. To choose the best PROM, a systematic review of PROMs can be conducted, in which all information on the quality and feasibility of each available PROM is collected, rated, and compared. Based on such a review a choice for the most suitable PROM for a certain study or clinical application can be made. However, conducting a systematic review of PROMs is very challenging, because nine quality aspects of PROMs need to be taken into account.In this article, we present an updated step-by-step guideline for conducting systematic reviews of PROMs. Each of these steps is described in detail in an accompanying manual. This updated guideline helps researchers to conduct systematic reviews of PROMs in a systematic and transparent way. It also helps readers of systematic reviews to understand how the review was conducted and to check the conclusions about which PROMs are recommended based on their quality.

Methodological quality of 100 recent systematic reviews of health-related outcome measurement instruments: an overview of reviews.

PURPOSE: Systematic reviews evaluating and comparing the measurement properties of outcome measurement instruments (OMIs) play an important role in OMI selection. Earlier overviews of review quality (2007, 2014) evidenced substantial concerns with regards to alignment to scientific standards. This overview aimed to investigate whether the quality of recent systematic reviews of OMIs lives up to the current scientific standards. METHODS: One hundred systematic reviews of OMIs published from June 1, 2021 onwards were randomly selected through a systematic literature search performed on March 17, 2022 in MEDLINE and EMBASE. The quality of systematic reviews was appraised by two independent reviewers. An updated data extraction form was informed by the earlier studies, and results were compared to these earlier studies' findings. RESULTS: A quarter of the reviews had an unclear research question or aim, and in 22% of the reviews the search strategy did not match the aim. Half of the reviews had an incomprehensive search strategy, because relevant search terms were not included. In 63% of the reviews (compared to 41% in 2014 and 30% in 2007) a risk of bias assessment was conducted. In 73% of the reviews (some) measurement properties were evaluated (58% in 2014 and 55% in 2007). In 60% of the reviews the data were (partly) synthesized (42% in 2014 and 7% in 2007); evaluation of measurement properties and data syntheses was not conducted separately for subscales in the majority. Certainty assessments of the quality of the total body of evidence were conducted in only 33% of reviews (not assessed in 2014 and 2007). The majority (58%) did not make any recommendations on which OMI (not) to use. CONCLUSION: Despite clear improvements in risk of bias assessments, measurement property evaluation and data synthesis, specifying the research question, conducting the search strategy and performing a certainty assessment remain poor. To ensure that systematic reviews of OMIs meet current scientific standards, more consistent conduct and reporting of systematic reviews of OMIs is needed.

Understanding factors impacting patient-reported outcome measures integration in routine clinical practice: an umbrella review.

PURPOSE: Patient-report outcome measures (PROMs) have gained widespread support as a mechanism to improve healthcare quality. We aimed to map out key enablers and barriers influencing PROMs implementation strategies in routine clinical practice. METHODS: An umbrella review was conducted to identify reviews exploring enablers and barriers related to the integration of PROMs in routine clinical practice from January 2000 to June 2023. Information on key enablers and barriers was extracted and summarised thematically according to the Theoretical Domains Framework. RESULTS: 34 reviews met our criteria for inclusion. Identified reviews highlighted barriers such as limited PROMs awareness among clinicians and patients, perceived low value by clinicians and patients, PROMs that were too complex or difficult for patients to complete, poor usability of PROMs systems, delayed feedback of PROMs data, clinician concerns related to use of PROMs as a performance management tool, patient concerns regarding privacy and security, and resource constraints. Enablers encompassed phased implementation, professional training, stakeholder engagement prior to implementation, clear strategies and goals, 'change champions' to support PROMs implementation, systems to respond to issues raised by PROMs, and integration into patient pathways. No consensus favoured paper or electronic PROMs, yet offering both options to mitigate digital literacy bias and integrating PROMs into electronic health records emerged as important facilitators. CONCLUSIONS: The sustainable implementation of PROMs is a complex process that requires multicomponent organisational strategies covering training and guidance, necessary time and resources, roles and responsibilities, and consultation with patients and clinicians.

Real-world application of the pediatric Glucocorticoid Toxicity Index in childhood-onset lupus.

OBJECTIVES: The pediatric Glucocorticoid Toxicity Index (pGTI) is a new, pediatric-specific tool to quantify glucocorticoid (GC)-related morbidity in children. We evaluated the feasibility and construct validity of retrospective pGTI scoring in patients with pediatric-onset systemic lupus erythematosus (pSLE) and identified risk factors for cumulative toxicity. METHODS: We conducted a retrospective cohort study of patients with pSLE treated with GCs at two pediatric centers (1999-2023). GC exposure was estimated using interval-averaged oral prednisone-equivalent dose and cumulative prednisone-equivalent dose. We scored change in GC toxicity every 6 months (±2) using a modified pGTI including 7 of 10 domains. We calculated the Cumulative Worsening Score (CWS), a continuous summation of toxicity accrued. Mixed effects linear regression was used to identify factors associated with CWS. RESULTS: There were 126 patients with pSLE, including 88 with nephritis, with a median of 6 visits/patient. Nearly half (47 %) experienced toxicity in the Blood Pressure domain. Other common toxicities were mood disturbance (25 %), followed by increased body mass index (BMI), striae, and sleep disturbance (21 % each). Decreased growth velocity was observed in 18 %. There was modest correlation between cumulative GC dose and CWS (rho 0.3; p < 0.01). Greater cumulative toxicity was associated with younger age, elevated BMI, and rituximab use at the time of GC initiation, albeit indications for the latter were not captured. CONCLUSIONS: Patients with pSLE experience a high burden of GC toxicity, particularly related to blood pressure, BMI, sleep, and growth. Standardized, pediatric-specific GC toxicity assessment is feasible in real-world settings and can facilitate evaluation of strategies to reduce morbidity in children requiring chronic GC treatment.

Utilization of orthoses and assistive devices among a national sample of lower limb orthosis users.

Introduction: Lower limb orthoses (LLOs) and assistive devices (ADs) can be used together or separately to improve mobility when performing daily activities. The goal of this study was to examine utilization of LLOs and ADs in a national sample of adult LLO users. Methods: A survey was designed to ask participants whether they typically use their LLOs and/or ADs to perform 20 daily activities. LLO users from orthotic clinics across the United States were invited to complete the survey. Descriptive statistics were used to examine utilization trends. Results: Survey responses from 1036 LLO users were analyzed. Community-based activities were performed with LLOs by at least 80% of participants. Activities that involved walking short distances in the home were more often performed without LLOs or ADs. Among participants with the four most prevalent health conditions, LLO use in the community was greatest among participants with Charcot-Marie-Tooth disease. Conclusions: LLOs were frequently used for a wide range of community-based activities. Simultaneous use of ADs and LLOs may be most beneficial for LLO users when performing activities outside of the home. Clinicians can discuss LLO and AD use with patients to optimize their functional outcomes at home and in the community.

Development of a core domain set for ankle osteoarthritis: An international consensus study of patients and health professionals.

OBJECTIVES: To develop an internationally agreed-upon core domain set for ankle osteoarthritis (OA). METHODS: In a three-part Delphi process, a group of multidisciplinary health professionals with expertise in ankle OA and people with ankle OA responded to online questionnaires. The questionnaires proposed a list of 29 candidate domains derived from a systematic review of ankle OA research, and interviews with people with ankle OA and health professionals. Consensus was defined a priori as ≥70% agreement in people with ankle OA and health professionals whether a domain should or should not be included in a core domain set. An online consensus meeting was held to discuss and resolve undecided candidate domains. RESULTS: A total of 100 people (75 health professionals and 25 people with ankle OA) from 18 countries (4 continents) participated in this study. Five domains reached consensus for inclusion in a core domain set for ankle OA - pain severity, health-related quality of life, function, disability and ankle range of motion. Twenty-one candidate domains reached agreement not to be included in the core domain set, and three domains remained undecided (ankle instability, physical capacity, and mental health). CONCLUSION: This international consensus study, which included people with ankle OA and health professionals, has established a core domain set for ankle OA with five domains that should be measured and reported in all ankle OA trials - pain severity, health-related quality of life, function, disability and ankle range of motion. This core domain set will guide the reporting of outcomes in clinical trials on ankle OA. Future research should determine which outcome measurement instruments should be used to measure each of the core domains.

Guideline for reporting systematic reviews of outcome measurement instruments (OMIs): PRISMA-COSMIN for OMIs 2024.

PURPOSE: Although comprehensive and widespread guidelines on how to conduct systematic reviews of outcome measurement instruments (OMIs) exist, for example from the COSMIN (COnsensus-based Standards for the selection of health Measurement INstruments) initiative, key information is often missing in published reports. This article describes the development of an extension of the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) 2020 guideline: PRISMA-COSMIN for OMIs 2024. METHODS: The development process followed the Enhancing the QUAlity and Transparency Of health Research (EQUATOR) guidelines and included a literature search, expert consultations, a Delphi study, a hybrid workgroup meeting, pilot testing, and an end-of-project meeting, with integrated patient/public involvement. RESULTS: From the literature and expert consultation, 49 potentially relevant reporting items were identified. Round 1 of the Delphi study was completed by 103 panelists, whereas round 2 and 3 were completed by 78 panelists. After 3 rounds, agreement (≥ 67%) on inclusion and wording was reached for 44 items. Eleven items without consensus for inclusion and/or wording were discussed at a workgroup meeting attended by 24 participants. Agreement was reached for the inclusion and wording of 10 items, and the deletion of 1 item. Pilot testing with 65 authors of OMI systematic reviews further improved the guideline through minor changes in wording and structure, finalized during the end-of-project meeting. The final checklist to facilitate the reporting of full systematic review reports contains 54 (sub)items addressing the review's title, abstract, plain language summary, open science, introduction, methods, results, and discussion. Thirteen items pertaining to the title and abstract are also included in a separate abstract checklist, guiding authors in reporting for example conference abstracts. CONCLUSION: PRISMA-COSMIN for OMIs 2024 consists of two checklists (full reports; abstracts), their corresponding explanation and elaboration documents detailing the rationale and examples for each item, and a data flow diagram. PRISMA-COSMIN for OMIs 2024 can improve the reporting of systematic reviews of OMIs, fostering their reproducibility and allowing end-users to appraise the quality of OMIs and select the most appropriate OMI for a specific application. NOTE: In order to encourage its wide dissemination this article is freely accessible on the web sites of the journals: Health and Quality of Life Outcomes; Journal of Clinical Epidemiology; Journal of Patient-Reported Outcomes; Quality of Life Research.