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BACKGROUND: The Activities-specific Balance Confidence Scale was developed for testing the balance confidence of elderly individuals, and it has been used extensively for evaluating various patients. No such scale has been adapted for the Hungarian population. OBJECTIVE: To translate and culturally adapt the Activities-specific Balance Confidence Scale and test the reliability and validity of the Hungarian version. METHODS: The study included 167 independently mobile subjects, of whom 39 filled in the questionnaire twice, 1 week apart. Beaton's six-step principle was applied for cross-cultural adaptation. Reliability was assessed by internal consistency measured by Cronbach's alpha and through test-retest analysis. Types of validity evaluated were concurrent validity using the Berg Balance Scale and cross-cultural validity. RESULTS: Excellent internal consistency was shown by Cronbach's alpha = 0.977. Test-retest analysis resulted in an Intra-Class Correlation Coefficient of 0.962 (0.865-0.961, 95% CI, p < .001) for the whole test; no floor or ceiling effects were found. The convergent validity of the scale was tested by Spearman's rank correlation analysis using the Berg Balance scale for external validation and showed a strong positive correlation (Rho = 0.755, p < .001). Receiver Operating Characteristic curve analysis showed an Area Under the Curve of 0.821 (CI 95% 0.75, 0.892). Mean detectable change based on the 95% confidence interval was 10.49% on the scale ranging from 0 to 100%. CONCLUSIONS: The Hungarian version of the Activities-Specific Balance Confidence Scale provides a valid and reliable picture of the patients' self-assessed balance. It is recommended both for clinicians and for clinical studies.

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The Scarf osteotomy involves inherent risks and technical challenges. The short scarf osteotomy with single screw fixation has emerged as an alternative approach. This study aims to evaluate the patient-reported outcome measures (PROMs), radiological outcomes, and complications associated with the short scarf osteotomy fixed with a single screw for hallux valgus correction. A prospective cohort study was conducted between October 2018 and October 2023 at a single center, encompassing 271 osteotomies. PROMs were collected prospectively preoperatively and at various postoperative intervals. Radiographic data, including hallux valgus angle (HVA) and intermetatarsal angle (IMA), were measured preoperatively and six weeks postoperatively. Complications were also documented. Statistically significant improvements were observed in all PROMs and were sustained up to 24 months postoperatively. The Visual Analog Scale (VAS) pain score demonstrated a significant reduction from 46.97 at baseline to 12.52 at 24 months (p < 0.001). Likewise, the EQ-5D score improved notably from 0.73 preoperatively to 0.81 at 24 months (p = 0.002). Analysis of the Manchester-Oxford Foot Questionnaire (MOXFQ) revealed substantial improvements across its pain, walking/standing, and social interaction domains over the same period, with all changes showing statistical significance (all p < 0.001). Radiographically, the mean HVA improved significantly from 31.33° preoperatively to 13.33° postoperatively (p < 0.001). Similarly, the mean IMA improved significantly from 14.67° to 7.66° (p < 0.001). The most common complication was superficial wound infection, effectively managed with oral antibiotics. The were no cases of avascular necrosis or fracture. In conclusion, the short Scarf osteotomy fixed with a single screw demonstrates favorable outcomes. These findings support its efficacy and safety as a treatment option, with potential advantages over the conventional technique. Further prospective studies are warranted to validate these findings.

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BACKGROUND: Since its creation, the Mental Health Literacy Scale (MHLS) has been used worldwide in mental health literacy studies. OBJECTIVE: This study aimed to systematically evaluate, summarize, and compare the measurement properties of MHLS validation studies. METHODS: PsycINFO, CINAHL, ERIC, Scopus, Embase, MEDLINE, and PubMed databases were searched from May 30, 2015, to December 31, 2023. Peer-reviewed studies validating the MHLS and its measurement properties were included, irrespective of language, study population, and setting. Studies using the MHLS as an outcome measure, as a comparative instrument to validate another instrument, or using other MHL measures and grey literature was excluded. RESULTS: Of the 685 search results, 16 studies were deemed eligible. The COnsensus-based Standards for the selection of health status Measurement INstruments (COSMIN) RoB criteria showed 15/15 studies exhibited 'Very Good' or 'Adequate' internal consistency, 3/6 reliability, 1/8 content validity, 14/14 structural validity, 6/7 hypothesis testing for convergent validity, 2/7 hypothesis testing for known-group validity, and 0/1 error measurement. The Cronbach's alpha ranged from 0.720 to 0.890, and the Intra-class Correlation Coefficient ranged from 0.741 to 0.99, while content validity was limited regarding the quality of evidence rating. The four-factor and unidimensional structures were 35.7 % and 28.6 %, respectively, the most common models. CONCLUSION: The MHLS exhibited strong evidence of construct validity and reliability, ensuring consistent and accurate evaluation of MHL and improving research credibility and generalizability. However, the low number of identical language versions of MHLS studies prohibited statistical pooling and quantitative summaries.

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Our survey of 38 surgeons shows that Ecological Momentary Computerized Adaptive Testing (EMCAT), a platform to remotely collect patient-reported outcome measures (PROMs) is acceptable to surgeons, and 36 of 38 are willing to participate in future randomized control trials comparing EMCAT versus routine follow-up.Level of evidence: IV.

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Hereditary angioedema (HAE) symptoms can vary greatly. Disease burden evaluation is essential for providing adequate treatments for patients. Patient-reported outcome measures (PROMs), including the 12-Item Short Form Health Survey (SF-12), the Angioedema Quality of Life (AE-QoL), the Hospital Anxiety and Depression Scale (HADS), and the Work Productivity and Activity Impairment: Specific Health Problem (WPAI:SHP) questionnaires, were collected in 2021, before modern medications for long-term prophylaxis (LTP) of HAE were licensed in Japan. Patients also reported their HAE attack frequency as "annual" (several attacks annually), "monthly" (several attacks monthly) or "weekly" (several attacks weekly). Multiple linear regression analyses were conducted on the relationship between independent parameters (sex, age, attack frequency, HAE type, and HADS scores) and dependent parameters (AE-QoL and SF-12 scores). Fifty-four patients reported PROMs. All PROMs showed substantial health-related quality of life (HRQoL) impairment. Overall, the higher the attack frequencies, the greater the reported impairment in the PROMs tended to be. In multiple linear regression analyses, higher AE-QoL Fatigue/Mood and Fears/Shame domain scores (greater impairment) were associated with higher HADS anxiety subscale scores; higher AE-QoL total scores (greater HRQoL impairment) and lower SF-12 Physical and Mental Health Composite scores (greater general health impairment) were associated with higher HADS depression subscale scores. Patients with monthly or weekly HAE attacks reported numerically low absenteeism and numerically high presenteeism and work productivity loss as measured by the WPAI:SHP questionnaire. In this study, conducted before modern LTP options were available in Japan, patients with HAE reported notable impairment in HRQoL and work productivity. Weekly or monthly HAE attack frequencies were associated with a high disease burden. Furthermore, a substantial number of patients reported notable fatigue/mood impairment as measured by the AE-QoL and depression as measured by the HADS regardless of attack frequency. These results provide a basis for future studies evaluating the effect of LTP on the clinical manifestations and HRQoL in patients with HAE.

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BACKGROUND: N-methyl-D-aspartate-receptor (NMDAR) encephalitis is a rare neurological autoimmune disease with severe neuropsychiatric symptoms during the acute phase. Despite good functional neurological recovery, most patients continue to experience cognitive, psychiatric, psychological, and social impairments years after the acute phase. However, the precise nature and evolving patterns over time of these long-term consequences remain unclear, and their implications for the well-being and quality of life of predominantly young patients have yet to be thoroughly examined. METHODS: SAPIENCE is a European multi-center (n = 3) prospective observational cohort study studying the long-term cognitive, psychiatric, psychological, and social outcome in patients with NMDAR encephalitis. The study consists of three interconnected levels. Level 1 comprises a qualitative interview and focus groups with patients and their caregivers. Level 2 consists of a condensed form of the interview, standardized questionnaires, and a detailed neuropsychological examination of patients. Level 3 involves an online survey that will be open to patients world-wide and explores patient-reported outcomes (PROMs), and patient-reported experiences (PREMs) in association with clinical and cognitive outcomes. Levels 1 to 3 will progressively contribute developing of structured interviews, survey questions, and treatment guidelines by informing one another. DISCUSSION: SAPIENCE is an in-depth study of the long-term effects of NMDAR encephalitis and bridges the gap between standardized assessments and individual patient experiences, intending to improve patient care and to increase awareness of the psychosocial long-term consequences of the disease. Through collaboration of experts in clinical neurology and social and health psychology across Europe, SAPIENCE aims to create online assessment tools and formulate guidelines for patient-centered post-acute care that will help enhance the quality of life for patients and caregivers.

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PURPOSE: Patient-reported outcome measures (PROMs) such as the Neurological Disorders Depression Inventory in Epilepsy (NDDI-E), a 6-item epilepsy-specific PROM, is used to screen for major depressive disorder symptoms for patients with epilepsy (PWE). The validity and interpretation of PROMs can be affected by differential item functioning (DIF), which occurs when subgroups of patients with the same underlying health status respond to and interpret questions about their health status differently. This study aims to determine whether NDDI-E items exhibit DIF and to identify subgroups of PWE that exhibit DIF in NDDI-E items. METHODS: Data were from the Calgary Comprehensive Epilepsy Program database, a clinical registry of adult PWE in Calgary, Canada. A tree-based partial credit model based on recursive partitioning (PCTree) was used to identify subgroups that exhibit DIF on NDDI-E items using patients' characteristics as covariates. Differences in the identified subgroups were characterized using multinomial logistic regression. RESULTS: Of the 1,576 patients in this cohort, 806 (51.1%) were female, and the median age was 38.0 years. PCTree identified four patient subgroups defined by employment status, age, and sex. Subgroup 1 were unemployed patients ≤ 26 years old, subgroup 2 were unemployed patients > 26 years, subgroup 3 were employed females, while subgroup 4 were employed male patients. The subgroups exhibited significant differences on education level, comorbidity index scores, marital status, type of epilepsy, and driving status. CONCLUSION: PWE differed in their interpretation and responses to questions about their depression symptoms, and these differences were a function of sociodemographic and clinical characteristics.

Patient-reported depression screening measures are prone to differential item functioning (DIF), which occurs when patients with the same levels of depression respond to and interpret the questions on the measures differently, as a result of different socio-demographic characteristics. Heterogeneity in how epilepsy patients interpret and respond to depression measures, if not identified, could lead to measurement biases that threaten the validity of inferences from patient-reported outcome measure scores to inform clinical and healthcare decisions for epilepsy management. Using data from a clinical registry of patients with epilepsy, the tree-based item response theory model was used to examine the presence of DIF in the items comprising the patient-reported Neurological Disorders Depression Inventory in Epilepsy (NDDI-E) scale. Four subgroups of epilepsy patients were noted to exhibit DIF on NDDI-E items. These groups were defined by interactions among employment status, age and sex. These characteristics affect the interpretation of NDDI-E item scores. It is recommended that clinicians carefully scrutinize responses to individual items alongside the overall NDDI-E score in different patient groups to inform clinical decisions for epilepsy management.

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STUDY DESIGN: Retrospective chart review. OBJECTIVES: Transforaminal lumbar interbody fusion (TLIF) via open or minimally invasive (MI) techniques is commonly performed. Mobile applications for home-based therapy programs have grown in popularity. The purpose of this study was to (1) compare patient-reported outcome measures (PROMs) between postoperative patients who were the most and least compliant in using the mobile-based rehabilitation programs, (2) compare PROMs between open vs MI-TLIF cohorts, and (3) quantify overall compliance rates of home-based rehabilitation programs. METHODS: A retrospective chart review was performed. Patients were automatically enrolled in the rehabilitation program. Patient-Reported Outcomes Measurement Information System (PROMIS) and Oswestry Disability Index (ODI) scores were collected. Patients were separated into two study groups. Compliance rate was calculated as the difference between the number of active participants at the preoperative phase and final follow-up. RESULTS: 220 patients were included. Average follow-up time was 23.2 months. No difference was found in the change in (∆) PROMIS scores (P = 0.261) or ∆ODI scores (P = 0.690) regardless of patient compliance. No difference was found in outcome scores between open vs MI-TLIF techniques stratified by download compliance (downloaded, DL+; did not download, DL-) and phone reminder compliance (set reminder, R+; did not set reminder, R-) postoperatively. Both cohorts demonstrated clinical improvement exceeding minimal clinically important difference at final follow-up. Overall patient compliance was 71% at final postoperative follow up. CONCLUSION: Despite high long-term compliance and rising popularity, mobile applications for home-based postoperative rehabilitation programs have low clinical utility in patients undergoing TLIF.

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BACKGROUND: Medication Treatment Satisfaction (M-TS) from the patients' perspective is important for comprehensively evaluating the effect of medicines. The extent to which current patient-reported outcome measures (PROMs) for M-TS are valid, reliable, responsive, and interpretable remains unclear. To assess the measurement properties of existing PROMs for M-TS and to highlight research gaps. METHODS: Using PubMed, Embase (Ovid), Cochrane library (Ovid), IPA (Ovid), PsycINFO, Patient-Reported Outcome and Quality of Life Questionnaires biomedical databases, and four Chinese databases, we performed a systematic search for studies addressing the development and validation of PROMs for M-TS. Based on the Consensus-based Standards for the selection of health Measurement Instruments (COSMIN) guideline, pairs of reviewers independently assessed the measurement properties of the PROMs and rated the quality of evidence on the measurement properties of each PROM. (The Open Science Framework registration: https://doi.org/10.17605/OSF.IO/8S5ZM ). RESULTS: This review identified 69 PROMs for M-TS in 114 studies (four generic, 32 disease-specific, and 33 drug-specific) of which 60 were intended for adults. All provided limited or no information regarding interpretability. Most demonstrated appropriate construct validity including convergent validity (39/69) and discriminative or known-groups validity (40/69) (high to moderate quality of evidence). Only a few provided evidence of sufficient content validity (8/69), structural validity (13/69), and internal consistency (11/69). Of 38 PROMs reporting test-retest reliability, results in 24 provided evidence of satisfactory test-retest reliability (18 with high to moderate, 6 with low to very low quality of evidence). Few PROMs reported responsiveness (16/69). Two generic PROMs (Treatment Satisfaction Questionnaire for Medication initial Version 1.4, TSQM-1.4; Treatment Satisfaction with Medicines Questionnaire, SATMED-Q) and one drug-specific PROM (Insulin Treatment Satisfaction Questionnaire, ITSQ) demonstrated both satisfactory validity and reliability. CONCLUSIONS: Most existing PROMs for M-TS require further exploration of measurement properties. Reporting guidelines are needed to enhance the reporting quality of the development and validation of PROMs for M-TS.

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Introduction: Robotic total knee arthroplasty (rTKA), with its purported advantages of more accurate alignment, greater functional outcomes and patient satisfaction, is gaining popularity in patients undergoing TKA. The purpose of our study was to compare these parameters along with gait pattern and kneeling ability in a cohort of patients who underwent simultaneous TKA with manual instrumentation (mTKA) and rTKA in contralateral knees at a 1-year follow-up. Methods: This was a retrospective review of 135 consecutive patients who underwent simultaneous bilateral TKA using robotic assistance on one side and manual instrumentation on the contralateral side between January 2022 and June 2022. The target alignment in both cohorts was adjusted mechanical. Patients were followed up at 3, 6 and 12 months to assess and compare alignment, range of motion (ROM) and patient-reported outcome measures (PROM) data. Gait parameters and kneeling ability were assessed at 1-year follow-up. Results: While adjusted mechanical alignment was achieved in all rTKA patients, we recorded five outliers (≥3° with relation to 180° HKA axis) in the mTKA cohort (three varus and two valgus). There were no significant differences between both cohorts with regards to ROM, PROM scores, gait analysis parameters and kneeling ability at 1-year follow-up. Conclusion: rTKA helps in achieving the adjusted mechanical alignment more consistently than mTKA. This, however, does not contribute to better functional outcomes and patient satisfaction at 1-year follow-up. Level of Evidence: Level III.

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BACKGROUND: Patients with multiple sclerosis (MS) have to deal with a variable disease trajectory often associated with disability and productivity loss. OBJECTIVE: This study aimed to assess illness-related uncertainty and associated correlates in patients with relapsing-remitting multiple sclerosis (RRMS) beyond the near diagnosis phase. METHODS: We conducted a multicenter, non-interventional study including patients diagnosed with RRMS (2017 revised McDonald criteria) and a disease duration of 3 to 8 years. Perceived uncertainty was measured using the Mishel Uncertainty of Illness Scale (MUIS). Associations between the MUIS and different patient-based outcome measures were analyzed using Spearman's rank correlation. RESULTS: A total of 201 patients were studied (mean age (standard deviation): 38.7 (8.4) years, 71.4 % female). The median disease duration (interquartile range) was 6.0 (4.0-7.0) years and the median EDSS score was 1.0 (0.0-2.0). The mean MUIS score was 38.2 (10.8). Perceived uncertainty was positively correlated with fatigue (p < 0.001), symptom severity (p < 0.001), anxiety (p < 0.001), depressive symptoms (p < 0.001), and a threatening illness perception (p < 0.001), and negatively correlated with self-management (p < 0.001), self-efficacy (p < 0.001), processing speed (p < 0.001), knowledge of MS (p = 0.006), and quality of life (p < 0.001). CONCLUSION: Illness-related uncertainty was common in a population of mid-stage RRMS. Identifying uncertainty and its associated factors may be useful for implementing preventive strategies to help patients cope with the disease throughout life.

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INTRODUCTION: Patient-reported outcome measures (PROMs) are validated and standardised questionnaires that capture patients' own reports of their symptoms, functioning and well-being. PROMs can facilitate communication between patients and clinicians, reduce symptom burden, enhance quality of life and inform health service re-design. We aim to determine the acceptability of PROMs and the preferred timing of PROM completion in New South Wales (NSW) at the point of care, facilitated by the Health Outcomes and Patient Experiences (HOPE) platform. METHODS AND ANALYSIS: Semi-structured interviews with patients (~50-75, sampling across seven language groups and seven clinical cohorts), carers (~10-20) and clinicians (~18) enrolled in HOPE will be conducted via videoconference, telephone or in person. Participants will be asked questions about (1) what makes PROMs acceptable for use in chronic disease management (2) when patients would prefer to complete PROMs and when clinicians would like to use PROMs for clinical decision-making and (3) factors that impede the acceptability of PROMs for culturally and linguistically diverse patients. Interviews will be analysed using a reflexive thematic approach, guided by Normalisation Process Theory. ETHICS AND DISSEMINATION: Ethics approval has been obtained from the Sydney Local Health District Human Research Ethics Committee (SLHD HREC, Study Protocol #X24-0138). Results will be published in appropriate peer-reviewed journals, presented at conferences, disseminated to participants in the form of a plain language summary, and widely disseminated to consumer groups and professional stakeholders.

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Background: Grit, defined as perseverance and passion for long-term goals, and self-control, defined as the capacity to regulate impulses in the presence of momentarily gratifying temptations or diversion, have shown to be predictors of professional achievement. Their role in health care outcomes is less well understood. Purpose: We sought to determine whether grit and self-control are associated with patient-reported outcome measures (PROMs) following spine surgery. Methods: We conducted a retrospective review of adult patients who underwent cervical or lumbar procedures by a single fellowship-trained spine surgeon between March 2017 and October 2020. We included patients who underwent anterior cervical discectomy and fusion, cervical disk replacement, minimally invasive laminectomy/laminoplasty (MI-D), or minimally invasive transforaminal lumbar interbody fusion (MI-TLIF) with minimum 1-year follow-up. Grit and self-control scores were collected 1 year after the surgery. PROMs were collected preoperatively and at 6 postoperative timepoints. Grit and self-control were compared between patients who achieved substantial clinical benefit (SCB) in either physical or mental health versus those who did not. The association between grit/self-control and change in PROMs was also assessed. Results: In the 129 patients included in the analysis, we found that patients who achieved SCB in mental health had significantly higher grit scores than those who did not. In bivariate analysis, self-control was associated with greater improvement in leg pain scores at 1-year and 2-year follow-up following an MI-D. For the MI-TLIF cohort, grit was associated with a smaller change in Short Form 12 mental component score at 6 weeks, and self-control was associated with a smaller change in Patient-Reported Outcome Measurement Information System Physical Function at the 6-month timepoint. Grit and self-control were not associated with PROMs at other timepoints. Conclusion: This retrospective review found that grit and self-control were not significantly associated with PROMs at most postoperative timepoints in patients who underwent spine surgery.

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BACKGROUND: Acute rhinosinusitis (ARS) is a self-limiting inflammation of the nose and sinuses caused by viral or bacterial infections that requires primarily symptomatic treatment. Patient-reported outcome measures (PROMs) are suitable tools for the assessment of the effectiveness of remedies for ARS from the patient's perspective in clinical trials and real-world studies. Data regarding the quality of existing PROMs for ARS are limited. PURPOSE: To conduct a systematic review of the quality of existing disease-specific PROMs for use in adults and children with ARS according to the COnsensus-based Standards for the selection of health Measurement INstruments (COSMIN) methodology, and to derive recommendations for use of the identified instruments in future clinical studies. METHODS: We systematically searched PubMed, Web of Science and Embase for studies reporting on the development and/or validation of PROMs for ARS. We assessed the methodological quality of each included study, evaluated the quality of measurement properties per PROM and study, and graded the evidence. Based on the overall evidence, we derived recommendations for use of the instruments. RESULTS: We identified four studies on three PROMs measuring symptoms of ARS and quality of life in adults (Sinonasal Outcome Test-16, SNOT-16; Measurement of Acute Rhinosinusitis, MARS; Rhinosinusitis Quality-of-Life Assessment, RhinoQoL). For ARS in children, we identified two studies on two PROMs measuring symptoms of ARS (Pediatric Rhinosinusitis Symptom Scale, PRSS; Sinus Symptom Questionnaire, S5). Our assessment of measurement properties indicates that all instruments require further validation before they can be unrestrictedly recommended for use in future research (COSMIN category B). In particular, there were no content validity studies for any of the identified instruments, but also data on other important measurement properties, e.g., structural validity, are lacking. CONCLUSION: Currently, no PROM for ARS in adults and children can be unrestrictedly recommended based on the evaluation of their quality. Further validation is required for all identified PROMs. Content validation involving patients and experts should be given priority. SYSTEMATIC REVIEW REGISTRATION: OSF ( https://doi.org/10.17605/OSF.IO/VAP8U ).

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INTRODUCTION: Sentinel lymph node biopsy (SLNB) is a standard procedure for patients with clinically assessed negative axillary lymph nodes (cN0) during early-stage breast cancer (EBC). However, the majority of EBC patients have a negative pathological confirmation of the sentinel lymph node (SLN), and axillary surgery is inevitably associated with postoperative complications. Considering that SLNB has no therapeutic benefit, this trial aims to determine the safety of omitting SLNB in patients with cN0 early invasive breast cancer. METHODS AND ANALYSIS: The OMSLNB trial is a prospective, single-arm, non-inferiority, phase II, open-label study design involving female breast cancer patients with a tumor of ≤3 cm in diameter, who are considered axillary lymph-node-negative based on two or more radiological examinations, including axillary lymph node ultrasonography. Eligible patients will avoid axillary surgery but will undergo breast surgery, which is not limited to breast-conserving surgery. The trial begins in 2023 and is scheduled to end in 2027. The primary endpoint is 3 year invasive disease-free survival (iDFS). The secondary endpoints include the incidence of breast cancer-related lymphoedema, patient-reported outcomes, locoregional recurrence, local recurrence and regional recurrence. It is expected that the 3 year iDFS in patients undergoing SLNB is about 90%, combined with a non-inferiority cut-off of 5%, 80% power, 95% CIs, 0.05 test level, and 10% loss to follow-up rate, the planned enrollment is 311 patients. All enrolled patients will be included in the intention-to-treat analysis. ETHICS AND DISSEMINATION: This trial was approved by the Ethics Committee of the First Affiliated Hospital of Nanjing Medical University (No.2023-SR-193). All participants must provide written informed consent to be eligible. The protocol will be described in a peer-reviewed manuscript, and the results will be published in scientific journals and/or at academic conferences. TRIAL REGISTRATION NUMBER: NCT05935150.

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BACKGROUND: There are different modes and ways to assess patient-reported outcomes (PROs) in clinical trials. However, there is little systematic information on how often different modes of assessment (MOA) are used in cancer clinical trials and how exactly assessments are conducted. The goal of this scoping review is to gain an understanding of the MOA and data management of PROs in cancer randomised controlled trials (RCTs) and the reporting quality thereof. METHODS AND ANALYSIS: This scoping review protocol follows the Preferred Reporting Items for Systematic Reviews and Meta-Analyses guidelines. Relevant trials will be identified via their indexed publications. We will search PubMed for RCTs conducted in cancer populations that evaluate a biomedical treatment with a PRO endpoint. Trials with publications published between January 2019 and November 2023 will be included. Two independent reviewers will review the references for both the abstract and full-text screening. We will extract data from the publications from a trial and the trial protocol if a protocol can be traced. Data will be summarised at the trial level. We will focus on a descriptive analysis of the MOA of PROs and on the relative frequencies of the different MOA. We will also evaluate the quality of reporting for the relevant SPIRIT and CONSORT guidelines that refer to the assessment of PROs in trials. Due to the scoping nature of our review, we will not perform a dedicated quality assessment of all trials. ETHICS AND DISSEMINATION: The proposed review is based on secondary, published data. Hence, no ethics review is necessary. The review is part of an ongoing project on the use of electronic data capture methods in cancer clinical trials. The findings from the review will support the project and contribute to synthesising guidance to ultimately improve the (electronic) measurement of patient-reported outcome measures in clinical trials.

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OBJECTIVE: To systematically review the literature of existing evidence on the measurement properties of the Quality of Life in Neurological Disorders (Neuro-QoL) measurement system among neurorehabilitation populations. DATA SOURCES: The Consensus-based Standards for the selection of health Measurement Instruments (COSMIN) guided this systematic review in which we searched nine electronic databases and registries, and hand-searched reference lists of included articles. STUDY SELECTION: Two independent reviewers screened selected articles and extracted data from 28 included studies. DATA EXTRACTION: COSMIN's approach guided extraction and synthesizing measurement properties evidence (insufficient, sufficient), and the modified GRADE approach guided synthesizing evidence quality (very-low, low, moderate, high) by diagnosis. DATA SYNTHESIS: Neuro-QoL has sufficient measurement properties when used by individuals with Huntington's disease, Multiple Sclerosis, Parkinson's disease, stroke, lupus, cognitive decline, and amyotrophic lateral sclerosis. The strongest evidence is for the first four conditions, where test-retest reliability, construct validity, and responsiveness are nearly always sufficient (GRADE: moderate-high). Structural validity is assessed only in multiple sclerosis and stroke but is often insufficient (GRADE: moderate-high). Criterion validity is sufficient in some stroke and Huntington's disease domains (GRADE: high). Item response theory analyses were reported for some stroke domains only. There is limited, mixed evidence for responsiveness and measurement error (GRADE: moderate-high), and no cross-cultural validity evidence CONCLUSIONS: Neuro-QoL domains can describe and evaluate patients with Huntington's disease, multiple sclerosis, Parkinson's disease, and stroke, but predictive validity evidence would be beneficial. In the other conditions captured in this review, a limited number of Neuro-QoL domains have evidence for descriptive use only. For these conditions, further evidence of structural validity, measurement error, cross-cultural validity and predictive validity would enhance the use and interpretation of Neuro-QoL.

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BACKGROUND: Despite growing applications of backward walking (BW) in assessing and rehabilitating neuromuscular conditions, its effects on gait in chronic ankle instability (CAI) remain unclear. Moreover, linking patient-reported and clinically generated measures is imperative for understanding CAI. HYPOTHESES: Patients with CAI will exhibit worse and compensatory spatio-temporal and kinetic gait parameters during BW, and patient-reported outcomes (PROs) will be correlated significantly with gait parameters. STUDY DESIGN: Case-control study. LEVEL OF EVIDENCE: Level 4. METHODS: A total of 46 volunteers participated (23 per group). Patients filled out scales for pain, functions, and fear-avoidance beliefs before testing. All participants walked 6 times each in both forward and backward conditions, and gait was recorded using the Win-Track system. A 2-way mixed analysis of variance was performed to compare gait parameters. The relationship between PRO and gait outcomes was assessed through the Pearson product correlation coefficient. RESULTS: The CAI group demonstrated prolonged support and swing phases, increased walk-off angle, and plantar pressure area, but decreased step length and plantar pressure versus controls (P < 0.05). The CAI group had a smaller right walk-off angle during BW than FW; the control group showed the opposite (P < 0.05). The left single stance duration was greater in the CAI group, while the right was not (P < 0.05). PRO correlated significantly with gait parameters, particularly spatial parameters (P < 0.05). CONCLUSION: The CAI group exhibited worse gait parameters during BW. The CAI group exhibited a characteristic compensatory gait pattern. Linking the self-reported scores provides a better representation of gait changes in CAI. CLINICAL RELEVANCE: These results suggest that BW may be an effective strategy for identifying and evaluating CAI. It may be feasible to apply BW to the rehabilitation of CAI.

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INTRODUCTION: Currently, no quality-of-life instrument exists that captures the full experience of the mental health outlook (MHO), a coreHEM core outcome, in people with haemophilia, including the potential transformational experience of receiving gene therapy. AIM: To describe the methods used to develop a content validated patient-reported outcome measure (PROM) that measures MHO for people with haemophilia. METHODS: A conceptual framework, developed from a literature/evidence review, was used to create an interview guide and draft a questionnaire. Males aged 15 or older with severe/moderate haemophilia were eligible to participate in concept elicitation or cognitive debriefing interviews. The conceptual framework was refined based on a thematic analysis of concept elicitation interviews and PROM items were developed from the conceptual framework. Cognitive debriefing sessions that prioritised relevance and understanding of the PROM were held in two rounds; items were updated iteratively. RESULTS: A conceptual framework with five domains (stigma, anxiety, depression, life interference and identity) was constructed from over 300 identified MHO concepts. Fifty-three participants took part in interviews. After 32 concept elicitation interviews, the framework was updated by including eight new sub-concepts and eliminating two. Updates to the questionnaire included items added or removed and improved wording. The final coreHEM MHO PROM has 26 questions in two sections (general mental health associated with haemophilia, and a gene therapy section). CONCLUSIONS: The instrument is content-validated and can be used as an exploratory outcome. MHO scores can be measured and compared to give more insight into patient quality of life.

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The increasing burden of chronic kidney disease (CKD) on the health care system highlights the need to prioritize services and manage the use of resources efficiently. Amid these financial constraints, key decision makers must weigh the impact of an intervention or program on health care expenditure when determining the allocation of limited resources. Patient-reported outcome measures (PROMs) are relevant in health economic decision-making within nephrology. Health-related quality of life, a patient-reported outcome, can provide data that inform economic evaluations of treatments for patients with CKD. PROMs help determine the value of different therapies by assessing their impact on patients' daily lives beyond clinical outcomes and can help policymakers make decisions about funding and reimbursement that consider the priorities and preferences of patients. Economic evaluations often employ cost-utility analyses, which use quality-adjusted life years as a key metric. Quality-adjusted life years combine both the quality and quantity of life lived, allowing for comparison of the effectiveness of different interventions in a standardized manner. By integrating utilities derived from PROMs, these analyses quantify the benefits of CKD treatments in terms of how patients feel and function. Furthermore, PROMs contribute to quality improvement initiatives by identifying areas where patient care can be enhanced, guiding the implementation of programs that improve health-related quality of life while maintaining cost-effectiveness. In value-based financing environments, the integration of PROMs ensures that patient-centered outcomes are prioritized, leading to more effective and equitable health care delivery. In this article, we discuss the role of PROMs in economic evaluations in CKD and provide an overview of approaches for using PROMs in economic evaluations to inform decision-making in nephrology.