Machine learning models for abstract screening task - A systematic literature review application for health economics and outcome research.

OBJECTIVE: Systematic literature reviews (SLRs) are critical for life-science research. However, the manual selection and retrieval of relevant publications can be a time-consuming process. This study aims to (1) develop two disease-specific annotated corpora, one for human papillomavirus (HPV) associated diseases and the other for pneumococcal-associated pediatric diseases (PAPD), and (2) optimize machine- and deep-learning models to facilitate automation of the SLR abstract screening. METHODS: This study constructed two disease-specific SLR screening corpora for HPV and PAPD, which contained citation metadata and corresponding abstracts. Performance was evaluated using precision, recall, accuracy, and F1-score of multiple combinations of machine- and deep-learning algorithms and features such as keywords and MeSH terms. RESULTS AND CONCLUSIONS: The HPV corpus contained 1697 entries, with 538 relevant and 1159 irrelevant articles. The PAPD corpus included 2865 entries, with 711 relevant and 2154 irrelevant articles. Adding additional features beyond title and abstract improved the performance (measured in Accuracy) of machine learning models by 3% for HPV corpus and 2% for PAPD corpus. Transformer-based deep learning models that consistently outperformed conventional machine learning algorithms, highlighting the strength of domain-specific pre-trained language models for SLR abstract screening. This study provides a foundation for the development of more intelligent SLR systems.

Network meta-analysis for an ordinal outcome when outcome categorization varies across trials.

BACKGROUND: Binary outcomes are likely the most common in randomized controlled trials, but ordinal outcomes can also be of interest. For example, rather than simply collecting data on diseased versus healthy study subjects, investigators may collect information on the severity of disease, with no disease, mild, moderate, and severe disease as possible levels of the outcome. While some investigators may be interested in all levels of the ordinal variable, others may combine levels that are not of particular interest. Therefore, when research synthesizers subsequently conduct a network meta-analysis on a network of trials for which an ordinal outcome was measured, they may encounter a network in which outcome categorization varies across trials. METHODS: The standard method for network meta-analysis for an ordinal outcome based on a multinomial generalized linear model is not designed to accommodate the multiple outcome categorizations that might occur across trials. In this paper, we propose a network meta-analysis model for an ordinal outcome that allows for multiple categorizations. The proposed model incorporates the partial information provided by trials that combine levels through modification of the multinomial likelihoods of the affected arms, allowing for all available data to be considered in estimation of the comparative effect parameters. A Bayesian fixed effect model is used throughout, where the ordinality of the outcome is accounted for through the use of the adjacent-categories logit link. RESULTS: We illustrate the method by analyzing a real network of trials on the use of antibiotics aimed at preventing liver abscesses in beef cattle and explore properties of the estimates of the comparative effect parameters through simulation. We find that even with the categorization of the levels varying across trials, the magnitudes of the biases are relatively small and that under a large sample size, the root mean square errors become small as well. CONCLUSIONS: Our proposed method to conduct a network meta-analysis for an ordinal outcome when the categorization of the outcome varies across trials, which utilizes the adjacent-categories logit link, performs well in estimation. Because the method considers all available data in a single estimation, it will be particularly useful to research synthesizers when the network of interest has only a limited number of trials for each categorization of the outcome.

COS-PPA: protocol to develop a core outcome set for primary progressive aphasia.

INTRODUCTION: The term primary progressive aphasia (PPA) describes a group of language-led dementias. Disease-modifying treatments that delay, slow or reverse progression of PPA are currently lacking, though a number of interventions to manage the symptoms of PPA have been developed in recent years. Unfortunately, studies exploring the effectiveness of these interventions have used a variety of different outcome measures, limiting comparability. There are more constructs, apart from word retrieval, that are important for people with PPA that have not received much attention in the research literature. Existing core outcome sets (COS) for dementia and non-progressive aphasia do not meet the needs of people with PPA, highlighting a need to develop a specific COS for PPA. METHODS AND ANALYSIS: This protocol describes a three-stage study to identify a COS for PPA interventions in research and clinical practice. The stage 1 systematic review will identify existing speech, language and communication measures used to examine the effectiveness of interventions for PPA in the research literature. Employing a nominal group technique, stage 2 will identify the most important outcomes for people with PPA and their families. The data collected in stages 1 and 2 will be jointly analysed with the project PPI group and will inform the stage 2 modified Delphi consensus study to identify a core outcome measurement set for PPA among a range of research disciplines undertaking intervention studies for people with PPA. ETHICS AND DISSEMINATION: Ethical approval for stage 2 of the study has been sought individually in each country at collaborating institutions and is stated in detail in the manuscript. Stage 3 has been granted ethical approval by the Chairs of UCL Language and Cognition Department Ethics, Project ID LCD-2023-06. Work undertaken at stages 1, 2 and 3 will be published in open-access peer-reviewed journal articles and presented at international scientific conferences. PROSPERO REGISTRATION NUMBER: CRD42022367565.

Factors Associated With Early Motor Function Trajectories in DMD After Glucocorticoid Initiation: Post Hoc Analysis of the FOR-DMD Trial.

BACKGROUND AND OBJECTIVES: Clinical trials in Duchenne muscular dystrophy (DMD) require 3-6 months of stable glucocorticoids, and the primary outcome is explored at 48-52 weeks. The factors that influence the clinical outcome assessment (COA) trajectories soon after glucocorticoid initiation are relevant for the design and analysis of clinical trials of novel drugs. We describe early COA trajectories, associated factors, and the time from glucocorticoid initiation to COA peak. METHODS: This was a prospective 18-month analysis of the Finding the Optimum Corticosteroid Regimen for Duchenne Muscular Dystrophy study. Four COAs were investigated: rise from supine velocity (RFV), 10-meter walk/run velocity (10MWRV), North Star Ambulatory Assessment (NSAA) total score, and 6-minute walk test distance (6MWT). The relationships of baseline age (4-5 vs 6-7 years), COA baseline performance, genotype, and glucocorticoid regimen (daily vs intermittent) with the COA trajectories were evaluated using linear mixed-effects models. RESULTS: One hundred ninety-six glucocorticoid-naïve boys with DMD aged 4-7 years were enrolled. The mean age at baseline was 5.9 ± 1.0 years, 66% (n = 130) were on daily regimens, 55% (n = 107) showed a 6MWT distance >330 metres; 41% (n = 78) showed RFV >0.2 rise/s; 76% (n = 149) showed 10MWRV >0.142 10m/s, and 41.0% (n = 79) showed NSAA total score >22 points. Mean COA trajectories differed by age at glucocorticoid initiation (p < 0.01 for RFV, 10MWRV, and NSAA; p < 0.05 for 6MWT) and regimen (p < 0.01 for RFV, 10MWRV, and NSAA). Boys younger than 6 years reached their peak performance 12-18 months after glucocorticoid initiation. Boys aged 6 years or older on a daily regimen peaked between months 9 and 12 and those on an intermittent regimen by 9 months. The baseline COA performance was associated with the NSAA (p < 0.01) and the 6MWT trajectory in boys younger than 6 years on a daily regimen (p < 0.01). Differences in the mean trajectories by genotype were not significant. DISCUSSION: Glucocorticoid regimen, age, duration of glucocorticoid exposure, and baseline COA performance need to be considered in the design and analysis of clinical trials in young boys with DMD.

Changes in cognitive function after a 12-week POWER rehabilitation in older adults with schizophrenia and frailty.

BACKGROUND: The effectiveness of isolated resistance training (RT) on cognitive function among older adults with schizophrenia is insufficiently investigated. This study investigated the effectiveness of 12-weeks POWER rehabilitation, a novel RT regimen, on cognitive function among older patients with schizophrenia and frailty. METHODS: Thirty-two older adults with schizophrenia and frailty were enrolled and randomized to receive either a 12-week, twice weekly POWER rehabilitation, or without add-on training. Cognitive functioning was assessed using mini-mental state examination (MMSE), digit symbol substitution test, color trail task (CTT), and digit span task (DST). Physical performance was assessed by walking speed and hand grip strength. The generalized estimating equations was used to compare pre- and post-training outcome measure between groups. RESULTS: Between-group analysis revealed significant improvement in CTT1 and hand grip strength in the intervention group compared to the controls. Subgroup analyses showed CTT1 performance significantly improved after 12 weeks of POWER rehabilitation in the intervention group (time, p < .001), independent of age, educational level, global cognition, depressive symptoms, and psychotropic medication use. Increased hand grip strength was significantly associated with improved performance in MMSE, CTT1, and DST forward at study endpoint. CONCLUSION: A 12-week POWER rehabilitation for older patients with schizophrenia and frailty is safe and feasible, and may benefit physical and some domains of cognitive functioning.

Is the QCI framework suited for monitoring outcomes and costs in a teaching hospital using value-based healthcare principles? A retrospective cohort study.

OBJECTIVES: The objective is to develop a pragmatic framework, based on value-based healthcare principles, to monitor health outcomes per unit costs on an institutional level. Subsequently, we investigated the association between health outcomes and healthcare utilisation costs. DESIGN: This is a retrospective cohort study. SETTING: A teaching hospital in Rotterdam, The Netherlands. PARTICIPANTS: The study was performed in two use cases. The bariatric population contained 856 patients of which 639 were diagnosed with morbid obesity body mass index (BMI) <45 and 217 were diagnosed with morbid obesity BMI ≥45. The breast cancer population contained 663 patients of which 455 received a lumpectomy and 208 a mastectomy. PRIMARY AND SECONDARY OUTCOME MEASURES: The quality cost indicator (QCI) was the primary measures and was defined asQCI = (resulting outcome * 100)/average total costs (per thousand Euros)where average total costs entail all healthcare utilisation costs with regard to the treatment of the primary diagnosis and follow-up care. Resulting outcome is the number of patients achieving textbook outcome (passing all health outcome indicators) divided by the total number of patients included in the care path. RESULTS: The breast cancer and bariatric population had the highest resulting outcome values in 2020 Q4, 0.93 and 0.73, respectively. The average total costs of the bariatric population remained stable (avg, €8833.55, min €8494.32, max €9164.26). The breast cancer population showed higher variance in costs (avg, €12 735.31 min €12 188.83, max €13 695.58). QCI values of both populations showed similar variance (0.3 and 0.8). Failing health outcome indicators was significantly related to higher hospital-based costs of care in both populations (p <0.01). CONCLUSIONS: The QCI framework is effective for monitoring changes in average total costs and relevant health outcomes on an institutional level. Health outcomes are associated with hospital-based costs of care.

Divergent perspectives: exploring the relationships between St. George's Respiratory Questionnaire and outcome measures in systemic sclerosis-associated interstitial lung disease.

INTRODUCTION/OBJECTIVES: Controversy exists regarding the concordance of patient-reported outcome measures (PROMs) with other assessment parameters in systemic sclerosis-associated interstitial lung disease (SSc-ILD). This study aims to explore the association between the St. George's Respiratory Questionnaire (SGRQ) and various outcome measures in patients with SSc-ILD within a real-world cross-sectional setting. METHOD: Patients with SSc-ILD were consecutively recruited from our SSc cohort. Simultaneous administration of SGRQ, scleroderma Health Assessment Questionnaire (sHAQ), respiratory visual analog scale (R-VAS), pulmonary function tests (PFTs), and the 6-min walking test (6-MWT) was conducted. The total extent of lung fibrosis was quantified using high-resolution computed tomography (HRCT) images. Relationships between SGRQ and functional, radiographic, and other patient-reported outcome measures were analyzed. RESULTS: The total SGRQ score demonstrated correlations with forced vital capacity (FVC) and R-VAS (r = - 0.397, p = 0.016 and r = 0.418, p = 0.027, respectively). Symptom score correlated with ILD-extension (r = 0.430, p = 0.005); activity score correlated with FVC and R-VAS (r = - 0.502, p = 0.002 and r = 0.395, p = 0.038, respectively); impact score correlated with R-VAS (r = 0.386, p = 0.043). In patients with fibrosis extent exceeding 20%, total SGRQ score was associated with sHAQ and R-VAS (r = 0.398, p = 0.049; r = 0.524, p = 0.021, respectively), activity score with R-VAS (r = 0.478, p = 0.038), and impact score with 6-MWT-D and R-VAS (r = - 0.489, p = 0.034; r = 0.545, p = 0.016, respectively). The symptom score and activity score demonstrated optimal performance in identifying patients with interstitial lung disease (ILD) extent exceeding 20% and forced vital capacity (FVC) less than 70% (area under the curve [AUC] 0.799, p = 0.002, and AUC 0.792, p = 0.03, respectively). CONCLUSIONS: Our study reveals varying degrees of correlation between SGRQ and distinct outcome measures. Given the incomplete alignment of SGRQ with other outcome measures, an integrative approach utilizing existing criteria as complementary tools is recommended. Key Points • Patient-reported outcome measures (PROMs) derive from patients' subjective evaluations of the impact of the disease on their daily activities, social interactions, and psychological well-being. • PROMs frequently serve as outcome measures in randomized controlled trials, yet conflicting findings have emerged in relation to primary outcomes. • This study aims to assess the appropriateness and interrelation of PROMs with both radiological and functional outcome measures, providing insight into the current state of our patients in a real-life context. The investigation delves into the compatibility of these measures with each other.

Will Generative AI Tools Improve Access to Reliable Health Information?

This Medical News article is an interview with JAMA Editor in Chief Kirsten Bibbins-Domingo and Virologist Davey Smith, head of the Division of Infectious Diseases and Global Public Health at the University of California, San Diego.

Outcome measures for children with speech sound disorder: an umbrella review.

OBJECTIVE: Speech sound disorder (SSD) describes a 'persistent difficulty with speech sound production that interferes with speech intelligibility or prevents verbal communication'. There is a need to establish which care pathways are most effective and efficient for children with SSD. Comparison of care pathways requires clearly defined, evidence-based, interventions and agreement on how to measure the outcomes. At present, no definitive list of assessments, interventions or outcomes exists. The objective of this umbrella review paper is to provide a rigorous and detailed list of assessments, interventions and outcomes which target SSD in children. DESIGN: In December 2022, a systematic search of Ovid Medline, OVID Embase, CINAHL, PsycInfo and Cochrane and a number of grey literature platforms were undertaken. 18 reviews were included, and subsequently 415 primary research articles were assessed for data related to assessments, interventions or outcomes. The AMSTAR (Assessing the Methodological Quality of Systematic Reviews) framework was used to assess the quality of the retained reviews. SETTING: Reviews were retained which took place in any setting. PARTICIPANTS: The population is children of any age with a diagnosis of SSD of unknown origin. PRIMARY AND SECONDARY OUTCOME MEASURES: Reviews reporting outcomes, assessment and interventions for children with SSD. RESULTS: Extraction and analysis identified 37 assessments, 46 interventions and 30 outcome measures used in research reporting of SSD. Not all of the listed outcomes were linked to specific outcome measurement tools, but these were measurable through the use of one or more of the assessments extracted from the retained reviews. CONCLUSIONS: The findings of this review will be used to develop a Core Outcome Set for children with SSD. The findings are part of a rigorous process essential for advancing healthcare research and practice in the specific area of speech and language therapy for children with SSD. PROSPERO REGISTRATION NUMBER: CRD42022316284.

Trends in outcomes used to measure the effectiveness of UK-based support interventions and services targeted at adults with experience of domestic and sexual violence and abuse: a scoping review.

OBJECTIVES: In the UK, a range of support services and interventions are available to people who have experienced or perpetrated domestic and sexual violence and abuse (DSVA). However, it is currently not clear which outcomes and outcome measures are used to assess their effectiveness. The objective of this review is to summarise, map and identify trends in outcome measures in evaluations of DSVA services and interventions in the UK. DESIGN: Scoping review. DATA SOURCES: MEDLINE, EMBASE, PsycINFO, Social Policy and Practice, ASSIA, IBSS, Sociological abstracts and SSCI electronic databases were searched from inception until 21 June 2022. Grey literature sources were identified and searched. ELIGIBILITY: We included randomised controlled trials, non-randomised comparative studies, pre-post studies and service evaluations, with at least one outcome relating to the effectiveness of the support intervention or service for people who have experienced and/or perpetrated DSVA. Outcomes had to be assessed at baseline and at least one more time point, or compared with a comparison group. CHARTING METHODS: Outcome measures were extracted, iteratively thematically grouped into categories, domains and subdomains, and trends were explored. RESULTS: 80 studies reporting 87 DSVA interventions or services were included. A total of 426 outcome measures were extracted, of which 200 were used more than once. The most commonly reported outcome subdomain was DSVA perpetration. Cessation of abuse according to the Severity of Abuse Grid was the most common individual outcome. Analysis of temporal trends showed that the number of studies and outcomes used has increased since the 1990s. CONCLUSIONS: Our findings highlight inconsistencies between studies in outcome measurement. The increase in the number of studies and variety of measures suggests that as evaluation of DSVA services and interventions matures, there is an increased need for a core of common, reliable metrics to aid comparability. PROTOCOL REGISTRATION: https://osf.io/frh2e.

The Promise and Challenges of Practice-Oriented Research: A Commentary on the Special Issue.

At the centre of POR is the concept of collaboration between patients, therapists, agencies, and third-party payers. For this commentary, I review the articles of the special issue with attention to both the opportunities and challenges offered by practice-oriented research (POR). I also reviewed some previous research on practice-research networks and how that research might inform POR. The use of routine outcome monitoring (ROM), artificial intelligence (AI), and program evaluation (PE) models show promise for advancing POR. However, each comes with its challenges. The use of ROM to improve patient outcomes has research support. However, researchers have identified problems with implementing ROM because of low uptake by clinicians and because clinicians may see ROM as a potential intrusion. AI shows promise to improve patient outcomes by potentially providing therapists with immediate and nuanced data to inform interventions and interpersonal stances specific to each patient. However, the scaling up of AI runs the risk of dehumanizing psychological interventions. PE may provide a context for allowing therapists to engage in POR to address real-world processes and outcomes of mental health interventions. However, like ROM PE faces the challenge of trust among clinicians and patients who may be reluctant to participate. Despite these challenges, and because of efforts to nurture and maintain respectful collaborations, articles in this special issue highlight how POR can play a pivotal role in bridging the gap between theory and practice.

Evaluating mFARS in pediatric Friedreich's ataxia: Insights from the FACHILD study.

OBJECTIVES: Friedreich ataxia (FRDA) is a rare genetic disorder caused by mutations in the FXN gene, leading to progressive coordination loss and other symptoms. The recently approved omaveloxolone targets this condition but is limited to patients over 16 years of age, highlighting the need for pediatric treatments due to the disorder's early onset and more rapid progression in children. This population also experiences increased non-neurological complications; the FACHILD study aimed to augment and expand the knowledge about the natural history of the disease and clinical outcome assessments for trials in children in FRDA. METHODS: The study enrolled 108 individuals aged 7-18 years with a confirmed FRDA diagnosis, with visits occurring from October 2017 to November 2022 across three institutions. Several measures were introduced to minimize the impact of the COVID-19 pandemic, including virtual visits. Outcome measures centered on the mFARS score and its subscores, and data were analyzed using mixed models for repeated measures. For context and to avoid misinterpretation, the analysis was augmented with data from patients enrolled in the Friedreich's Ataxia Clinical Outcome Measures Study. RESULTS: Results confirmed the general usefulness of the mFARS score in children, but also highlighted issues, particularly with the upper limb subscore (FARS B). Increased variability, limited homogeneity across study subgroups, and potential training effects might limit mFARS application in clinical trials in pediatric populations. INTERPRETATION: The FARS E (Upright Stability) score might be a preferred outcome measure in this patient population.

Exploring preconception health in adolescents and young adults: Identifying risk factors and interventions to prevent adverse maternal, perinatal, and child health outcomes-A scoping review.

BACKGROUND: Preconception health provides an opportunity to examine a woman's health status and address modifiable risk factors that can impact both a woman's and her child's health once pregnant. In this review, we aimed to investigate the preconception risk factors and interventions of early pregnancy and its impact on adverse maternal, perinatal and child health outcomes. METHODS: We conducted a scoping review following the PRISMA-ScR guidelines to include relevant literature identified from electronic databases. We included reviews that studied preconception risk factors and interventions among adolescents and young adults, and their impact on maternal, perinatal, and child health outcomes. All identified studies were screened for eligibility, followed by data extraction, and descriptive and thematic analysis. FINDINGS: We identified a total of 10 reviews. The findings suggest an increase in odds of maternal anaemia and maternal deaths among young mothers (up to 17 years) and low birth weight (LBW), preterm birth, stillbirths, and neonatal and perinatal mortality among babies born to mothers up to 17 years compared to those aged 19-25 years in high-income countries. It also suggested an increase in the odds of congenital anomalies among children born to mothers aged 20-24 years. Furthermore, cancer treatment during childhood or young adulthood was associated with an increased risk of preterm birth, LBW, and stillbirths. Interventions such as youth-friendly family planning services showed a significant decrease in abortion rates. Micronutrient supplementation contributed to reducing anaemia among adolescent mothers; however, human papillomavirus (HPV) and herpes simplex virus (HSV) vaccination had little to no impact on stillbirths, ectopic pregnancies, and congenital anomalies. However, one review reported an increased risk of miscarriages among young adults associated with these vaccinations. CONCLUSION: The scoping review identified a scarcity of evidence on preconception risk factors and interventions among adolescents and young adults. This underscores the crucial need for additional research on the subject.

Acute spinal cord injury serum biomarkers in human and rat: a scoping systematic review.

STUDY DESIGN: Scoping systematic review. OBJECTIVES: To summarize the available experimental clinical and animal studies for the identification of all CSF and serum-derived biochemical markers in human and rat SCI models. SETTING: Tehran, Iran. METHODS: In this scoping article, we systematically reviewed the electronic databases of PubMed, Scopus, WOS, and CENTRAL to retrieve current literature assessing the levels of different biomarkers in human and rat SCI models. RESULTS: A total of 19,589 articles were retrieved and 6897 duplicated titles were removed. The remaining 12,692 studies were screened by their title/abstract and 12,636 were removed. The remaining 56 were considered for full-text assessment, and 11 papers did not meet the criteria, and finally, 45 studies were included. 26 studies were human observational studies comprising 1630 patients, and 19 articles studied SCI models in rats, including 832 rats. Upon reviewing the literature, we encountered a remarkable heterogeneity in terms of selected biomarkers, timing, and method of measurement, studied models, extent, and mechanism of injury as well as outcome assessment measures. CONCLUSIONS: The specific expression and distribution patterns of biomarkers in relation to spinal cord injury (SCI) phases, and their varied concentrations over time, suggest that cerebrospinal fluid (CSF) and blood biomarkers are effective measures for assessing the severity of SCI.

Effectiveness of Motor Imagery in the Rehabilitation of People With Parkinson's Disease: A Systematic Review and Meta-Analysis.

BACKGROUND AND OBJECTIVE: With more research completed using Motor imagery (MI) in people with Parkinson's disease, this study gathered and synthesized evidence on the use of MI for Parkinson's disease in improving rehabilitation outcomes. METHODS: Medical Literature Analysis and Retrieval System Online, Embase, Web of Science, The Cochrane Library, PsycINFO, Cumulative Index to Nursing and Allied Health Literature, and Scopus were searched from inception to May 2023. We included randomized controlled trials that examine the effects of MI on individuals with Parkinson's disease. Two reviewers selected articles and extracted study characteristics and results independently. The Physiotherapy Evidence Database scale was used to assess the methodological quality. Mean differences and 95% confidence intervals were calculated. Heterogeneity was assessed using the I2 statistic. RESULTS: Thirteen articles with 12 studies were included, involving 320 individuals with Parkinson's disease, with moderate to high methodological quality (mean = 6.62/10). Compared with the control group, 3 articles reported significant greater improvements in cognitive function, 7 reported significant greater improvement in motor function, 1 article reported significant greater improvement in quality of life, and 1 reported significant greater confidence in daily task performance. No statistically significant effects were found in the meta-analyses. Conclusion. Results of individual articles were in favor of the MI intervention. No statistically significant results were found in the meta-analyses. This might be due to the small number of studies and the heterogeneity of interventions and outcome measures used. MI may be effective in improving some rehabilitation outcomes, but meta-analytic evidence is lacking. More research with larger sample size and less heterogeneous samples, interventions, and outcome measures, is warranted. SYSTEMATIC REVIEW REGISTRATION: PROSPERO registration number CRD42021230556.

Differences in Daily Activity Performance Between Inpatient Rehabilitation Facility and Home Among Stroke Survivors.

BACKGROUND: Stroke survivors are one of the largest consumer groups of rehabilitation services. Despite improvement in daily activities while in inpatient rehabilitation, many have difficulty performing daily activities at home after discharge. The difference in performance between a standard clinical context and at home is poorly understood. OBJECTIVE: To better understand differences in activity performance during transition from inpatient rehabilitation facility (IRF) to home, we examined daily activity performance scores from 2 different environments (IRF and home) at the same time point (discharge). METHODS: This was a cross-sectional analysis using baseline data from a randomized controlled trial. Participants were stroke survivors aged ≥50 who planned to discharge home from the IRF. The Functional Independence Measure and Section GG codes (both converted to International Classification of Functioning, Disability, and Health scores) were conducted per protocol first at home and then in the IRF at discharge (≤3 days apart, order not randomized). RESULTS: Among 57 participants, activity scores at home were significantly worse than scores at IRF discharge. Over 40% of participants had discharge scores indicating no-to-mild impairment for shower/tub transfer, walking, and going up/down stairs, while home visit scores indicated moderate-to-complete impairment for those activities. The greatest differences in scores were for shower/tub transfer (median difference 1.5, 95% CI 1.00-2.00) and going up/down stairs (median difference 1.50, 95% CI 1.00-2.00). CONCLUSION: The environment plays an important role in stroke survivors' functioning at home. Future studies should further examine how the environment impacts activity performance upon returning home following stroke.

Stride Velocity 95th Centile Detects Decline in Ambulatory Function Over Shorter Intervals than the 6-Minute Walk Test or North Star Ambulatory Assessment in Duchenne Muscular Dystrophy.

Background: Stride Velocity 95th Centile (SV95C) is the first wearable device-derived clinical outcome assessment (COA) to receive European Medicines Agency (EMA) qualification as a primary endpoint in ambulant patients with Duchenne muscular dystrophy (DMD) aged ≥4 years. Objective: To compare SV95C-in its first-ever clinical trial application as a secondary endpoint-with established motor function COAs used in the trial (Four-Stair Climb [4SC] velocity, North Star Ambulatory Assessment [NSAA], and Six-Minute Walk Distance [6MWD]). Methods: SV95C was a secondary endpoint in a subset (n = 47) of participants in the SPITFIRE/WN40227 trial of taldefgrobep alfa, which was discontinued due to lack of clinical benefit. Participants in the ≤48-week SV95C sub-study were 6-11 years old and received corticosteroids for ≥6 months pre-treatment. Pearson correlations were used to compare SV95C with the other COAs. Responsiveness and changes over time were respectively assessed via standardized response means (SRMs) based on absolute changes and mixed models for repeated measures. Results: SV95C change at Week 24 was -0.07 m/s, with limited variability (standard deviation: 0.16, n = 27). The SRM for SV95C indicated moderate responsiveness to clinical change at the earliest timepoint (Week 12, n = 46), while those of the other COAs did not indicate moderate responsiveness until Week 36 (6MWD, n = 33) or Week 48 (4SC velocity, n = 20; NSAA total score, n = 20). Baseline correlations between SV95C and other COAs were strong (r = 0.611-0.695). Correlations between SV95C change from baseline to Week 48 and changes in other COAs were moderate to strong (r = 0.443-0.678).∥. Conclusions: Overall, SV95C demonstrated sensitivity to ambulatory decline over short intervals, low variability, and correlation with established COAs. Although the negative trial precluded demonstration of SV95C's sensitivity to drug effect, these findings support the continued use of SV95C in DMD clinical trials.

Efficacy of Walking Adaptability Training on Walking Capacity in Ambulatory People With Motor Incomplete Spinal Cord Injury: A Multicenter Pragmatic Randomized Controlled Trial.

BACKGROUND AND OBJECTIVE: Balance and walking capacity are often impaired in people with motor incomplete spinal cord injury (iSCI), frequently resulting in reduced functional ambulation and participation. This study aimed to assess the efficacy of walking adaptability training compared to similarly dosed conventional locomotor and strength training for improving walking capacity, functional ambulation, balance confidence, and participation in ambulatory people with iSCI. METHODS: We conducted a 2-center, parallel-group, pragmatic randomized controlled trial. Forty-one people with iSCI were randomized to 6 weeks of (i) walking adaptability training (11 hours of Gait Real-time Analysis Interactive Lab (GRAIL) training-a treadmill in a virtual reality environment) or (ii) conventional locomotor and strength training (11 hours of treadmill training and lower-body strength exercises). The primary measure of walking capacity was maximal walking speed, measured with an overground 2-minute walk test. Secondary outcome measures included the Spinal Cord Injury Functional Ambulation Profile (SCI-FAP), the Activities-specific Balance Confidence (ABC) scale, and the Utrecht Scale for Evaluation of Rehabilitation-Participation (USER-P). RESULTS: No significant difference in maximal walking speed between the walking adaptability (n = 17) and conventional locomotor and strength (n = 18) training groups was found 6 weeks after training at follow-up (-0.05 m/s; 95% CI = -0.12-0.03). In addition, no significant group differences in secondary outcomes were found. However, independent of intervention, significant improvements over time were found for maximal walking speed, SCI-FAP, ABC, and USER-P restrictions scores. Conclusions. Our findings suggest that walking adaptability training may not be superior to conventional locomotor and strength training for improving walking capacity, functional ambulation, balance confidence, or participation in ambulatory people with iSCI. TRIAL REGISTRATION: Dutch Trial Register; Effect of GRAIL training in iSCI.

Malnutrition-Related Health Outcomes in Older Adults with Hip Fractures: A Systematic Review and Meta-Analysis.

Hip fracture is a common condition in older adults, leading to disability and mortality. Several studies have demonstrated the association between nutritional status and the risk of a negative health outcome after fractures. In this systematic review, we evaluated the association between malnutrition and mortality, changes in mobility/living arrangements, and postoperative complications, such as delirium, in older patients with hip fractures. A literature search on the PubMed, Web of Science, and Scopus databases, up to September 2023, was conducted to identify all studies involving older subjects that reported an association between MNA/GNRI/PNI/CONUT and health outcome after hip fracture. Meta-analysis was performed by a random-effects model using risk values (RR, OR, and HR) extracted from the 14 eligible selected studies. Malnutrition significantly increased the risk of any analyzed adverse outcome by 70% at 1 month, and up to 250% at 1 year. Malnutrition significantly increased delirium risk by 275% (OR = 2.75; 95% CI 1.80-4.18; p ≤ 0.05), mortality risk by 342% (OR = 3.42; 95% CI 2.14-5.48; p ≤ 0.05), mortality hazard risk by 351% (HR = 3.51; 95% CI 1.63-7.55; p ≤ 0.05) at 1 month, and transfer-to-more-supported-living-arrangements risk by 218% (OR = 2.18; 95% CI 1.58-3.01; p ≤ 0.05), and declined mobility risk by 41% (OR = 1.41; 95% CI 1.14-1.75; p ≤ 0.05), mortality risk by 368% (OR = 3.68; 95% CI 3.00-4.52; p ≤ 0.05), and mortality hazard risk by 234% (HR = 2.34; 95% CI 1.91-2.87; p ≤ 0.05) at 1 year. Malnutrition of older patients increases the risk of death and worsens mobility and independence after hip fractures. The results of the present study highlight the importance of nutritional status evaluation of older subjects with hip fractures in order to prevent potential adverse outcomes (Registration No: CRD42023468751).