Measures Of Function Research Articles

Residual reversibility in COPD patients already on long-acting bronchodilator: The OscilloRevers Study

BackgroundDyspnea is a complex symptom of chronic obstructive pulmonary disease (COPD) which is not strongly correlated with lung function measures. Long-acting bronchodilators (LAB) may reduce this dyspnea, but some patients report persistent chronic dyspnea despite this treatment. This study aims to assess residual reversibility and clinical response after short-acting bronchodilator (SAB) in COPD patients already treated by LAB and reporting persistent dyspnea. MethodsCOPD patients with a persistent dyspnea (modified Medical Research Council scale (mMRC) ≥1) despite current stable treatment with at least one LAB were included. Spirometry, plethysmography and impulse oscillometry (IOS) were performed at peak effect of their LAB and repeat 45 min after the intake of two SAB (400 µg of salbutamol and 80 µg of ipratropium). Dyspnea improvement was assessed at 45 min after SAB through a comparative two-sided VAS (-100 mm for maximal improvement; +100 mm for maximal degradation). ResultsTwenty-two COPD patients were analyzed, mainly men (59.1 %) with a mean age of 60.6 years and a median FEV1 of 54 % of predicted values. Fifty percent of patients reported a severe basal dyspnea (mMRC ≥2). After SAB, spirometric and plethysmographic measurements were statistically improved. For IOS measurement, reactance at 5 Hz (X5) and area of reactance (AX) were also improved. Fifty percent of patients reported a clinically relevant improvement of their resting dyspnea. However, no correlation was found between dyspnea improvement and functional measures. ConclusionsFifty percent of COPD patients regularly treated with one or two LAB still report a relevant improvement of resting dyspnea after the adjunctive intake of double short-acting bronchodilators. Physiological mechanisms associated with this improvement remain to be determined. Clinical Trial RegistrationNCT02928744

Epidemiology and Comorbidities of Vestibular Disorders: Preliminary Findings of the AVOCADO Study.

Studies on incidence and prevalence of vestibular disorders tend to focus on small pockets of patients recruited from specialized clinics and often exclude measures of vestibular function. The objectives of the study were to characterize patients with common vestibular disorders, estimate the prevalence of common vestibular disorders, and ascertain whether patients with vestibular disorders experience increased risks of falls and morbidity. This retrospective cohort study includes both inpatient and outpatient routine clinical care data culled from a nationally representative, population-based sample. Patients were included if their record in the TriNetX Diamond Cohort comprised at least one vestibular function test or vestibular diagnosis. The main outcome measures were diagnosis with a vestibular disorder, a fall, or a common medical comorbidity (e.g., diabetes, cerebrovascular disease). The cohort includes n = 4,575,724 patients, of which 55% (n = 2,497,136) had a minimum of one vestibular diagnosis. Patients with vestibular diagnoses were 61.3 ± 16.6 years old (mean ± standard deviation), 67% women, 28% White race (69% unknown race), and 30% of non-Hispanic or Latino ethnicity (66% unknown ethnicity). The prevalence of vestibular disorders was estimated at 2.98% (95% confidence interval [CI]: 2.98-2.98%). Patients with vestibular diagnoses experienced a significantly greater odds of falls (odds ratio [OR] = 1.04; 95% CI: 1.02-1.05), cerebrovascular disease (OR = 1.42; 95% CI: 1.40-1.43), ischemic heart disease (OR = 1.17; 95% CI: 1.16-1.19), and diabetes (OR = 1.14; 95% CI: 1.13-1.15), among others. Vestibular disorders affect an estimated 3% of the U.S. population, after weighting. Patients with these disorders are at greater risk for many common, consequential medical conditions.

Is shockwave therapy effective in the management of greater trochanteric pain syndrome? A systematic review and meta-analysis.

Evidence is lacking for the efficacy of shockwave therapy (SWT) in the treatment of greater trochanteric pain syndrome (GTPS). To investigate the efficacy of SWT on pain and function in the management of GTPS. A systematic search of electronic databases and grey literature was conducted up to May 2023. Studies utilising SWT on adults for GTPS, providing measures of pain and/or function at baseline and at follow-up were considered for inclusion. Meta-analysis was undertaken using converted pain and functional outcomes. Studies were assessed for quality and risk of bias, and assigned a level of evidence as per the Grading of Recommendations, Assessment, Development and Evaluations criteria. Twelve articles (n=1121 subjects) were included, including five randomised controlled trials (RCTs) and seven non-RCTs. No statistical differences were observed for pain over time f(1,5)=1.349 (p=0.298) or between SWT and control f(1,5)=1.782 (p=0.238). No significant differences in functional outcomes in short- (H=2.591, p=0.181) and medium-term follow-up (H=0.189, p=0.664) were identified between SWT and control. Moderate magnitude treatment effects for pain (Hedges-G [HG] 0.71) favouring SWT groups over control was identified, decreasing to low for function (HG 0.20). Further pain and functional treatment effects were identified at higher magnitudes across follow-up time-points in SWT groups compared to control. Moderate-quality evidence demonstrated no statistically significant improvements in pain and function post-SWT compared to control. Low-quality evidence established clinical improvements throughout all included studies favouring SWT over control. Consequently, owing to relatively low incidence of side effects, SWT should be considered a viable option for the management of GTPS. Issues with both clinical and statistical heterogeneity of studies and during meta-analysis require consideration, and more robust RCTs are recommended if the efficacy of SWT for the management of GTPS is to be comprehensively determined.

A domain-oriented approach to characterizing movement-evoked pain.

Movement-evoked pain (MEP) impacts a substantial proportion of US adults living with chronic pain. Evidence suggests that MEP is influenced by numerous biopsychosocial factors and mediated by mechanisms differing from those of spontaneous pain. However, both characteristic and mechanistic knowledge of MEP remain limited, hindering effective diagnosis and treatment. We asked (1) can chronic pain, functional, psychosocial, and behavioral measures be grouped into descriptive domains that characterize MEP? and (2) what relationships exist between biopsychosocial factors across multiple domains of MEP? We formed 6 characteristic domains from 46 MEP-related variables in a secondary analysis of data from 178 individuals (aged 45-85 years) with knee pain. Ratings of pain during 3 functional activities (ie, Balance, Walking, Chair Stand) were used as primary MEP variables. Pearson correlations were calculated to show linear relationships between all individual domain variables. Relationships between variables were further investigated through weighted correlation network analysis. We observed a unique combination of pain characteristics associated with MEP apart from general pain. Notably, minutes doing physical activity were inversely associated with multiple variables within 4 of the 6 domains. Weighted correlation network analysis largely supported our classification of MEP domains. Additional interdomain relationships were observed, with the strongest existing between MEP, Mechanical Pain, and Multiple Pain Characteristics and Symptoms. Additional relationships were observed both within and between other domains of the network. Our analyses bolster fundamental understanding of MEP by identifying relevant mechanistic domains and elucidating biopsychosocial and interdomain relationships.

Brain Responses to Real and Imagined Interpretation of Tonal Versus Atonal Music

Professional musicians have been teaching/learning/interpreting Western classical tonal music for longer than atonal music. This may be reflected in their brain plasticity and playing efficiency. To test this idea, EEG connectivity networks (EEG-CNs) of expert cellists at rest and during real and imagined musical interpretation of tonal and atonal excerpts were analyzed. Graphs and connectomes were constructed as models of EEG-CNs, using functional connectivity measurements of EEG phase synchronization in different frequency bands. Tonal and atonal interpretation resulted in a global desynchronization/dysconnectivity versus resting—irrespective of frequency bands—particularly during imagined-interpretation. During the latter, the normalized local information-transfer efficiency (NLE) of graph-EEG-CN’s small-world structure at rest increased significantly during both tonal and atonal interpretation, and more significantly during atonal-interpretation. Regional results from the graphs/connectomes supported previous findings, but only certain EEG frequency bands. During imagined-interpretation, the number of disconnected regions and subnetworks, as well as regions with higher NLE, were greater in atonal-interpretation than in tonal-interpretation for delta/theta/gamma-EEG-CNs. The opposite was true during real-interpretation, specifically limited to alpha-EEG-CN. Our EEG-CN experimental paradigm revealed perceptual differences in musicians’ brains during tonal and atonal interpretations, particularly during imagined-interpretation, potentially due to differences in cognitive roots and brain plasticity for tonal and atonal music, which may affect the musicians’ interpretation.

Associations between neonicotinoids and liver function measures in US adults: National Health and Nutrition Examination Survey 2015–2016

Background: Toxicological studies indicate that neonicotinoids may be associated with disruptions in liver function due to an increase in oxidative stress. There are scant epidemiological studies investigating the chronic hepatotoxic effects of neonicotinoids. Objective: To examine the association between detectable concentrations of parent neonicotinoids and neonicotinoid metabolites with liver function markers among US adults, and whether sex modifies this association. Methods: National Health and Nutrition Examination Survey 2015–2016 data were used to estimate associations between detectable neonicotinoids and serum alkaline phosphatase (ALP), alanine aminotransferase, aspartate aminotransferase, gamma-glutamyl transaminase (GGT), albumin, total bilirubin, total protein, and Hepatic Steatosis Index (HSI) using multiple linear regression. Results: Detectable levels of N-desmethyl-acetamiprid were associated with a decrease in GGT (β = −3.54 unit/l; 95% confidence interval [CI] = −6.48, −0.61) and detectable levels of 5-hydroxy-imidacloprid were associated with a decrease in HSI (β = −1.11; 95% CI = −2.14, −0.07). Sex modified the association between any parent neonicotinoid and ALP (P int = 0.064) and the association between clothianidin and ALP (P int = 0.019), with a pattern of positive associations in males and inverse associations in females, though stratified associations did not reach statistical significance. Sex also modified the association between 5-hydroxy-imidacloprid and total protein (P int = 0.062), with a significant positive association in females (β = 0.14 g/dl; 95% CI = 0.03, 0.25) and a null association in males. Conclusion: Detectable concentrations of neonicotinoid metabolites were inversely associated with GGT and HSI in US adults. Evidence suggests neonicotinoids may influence liver function differently depending on sex. Future research is recommended to replicate the findings as the study was limited in its cross-sectional nature and inability to examine continuous neonicotinoid concentrations with liver function.

Retinotopic cortical mapping in objective functional monitoring of macular therapy

Background/AimsTo determine the suitability of functional MRI (fMRI) as an objective measure of macular function following therapeutic intervention; conventional psychophysical measures rely heavily on patient compliance.MethodsTwenty patients with neovascular age-related...

Highlighting Unseen Activity Through 48-Hour Continuous Measurement in Subacute Stroke Rehabilitation: Preliminary Cohort Study.

Motor impairments not only lead to a significant reduction in patient activity levels but also trigger a further deterioration in motor function due to deconditioning, which is an issue that is particularly pronounced during hospitalization. This deconditioning can be countered by sustaining appropriate activity levels. Activities that occur outside of scheduled programs, often overlooked, are critical in this context. Wearable technology, such as smart clothing, provides a means to monitor these activities. This study aimed to observe activity levels in patients who had strokes during the subacute phase, focusing on both scheduled training sessions and other nontraining times in an inpatient rehabilitation environment. A smart clothing system is used to simultaneously measure heart rate and acceleration, offering insights into both the amount and intensity of the physical activity. In this preliminary cohort study, 11 individuals undergoing subacute stroke rehabilitation were enrolled. The 48-hour continuous measurement system, deployed at admission and reassessed 4 weeks later, monitored accelerometry data for physical activity (quantified with a moving SD of acceleration [MSDA]) and heart rate for intensity (quantified with percent heart rate reserve). The measurements were performed using a wearable activity monitoring system, the hitoe (NTT Corporation and Toray Industries, Inc) system comprising a measuring garment (wear or strap) with integrated electrodes, a data transmitter, and a smartphone. The Functional Independence Measure was used to assess the patients' daily activity levels. This study explored factors such as differences in activity during training and nontraining periods, correlations with activities of daily living (ADLs) and age, and changes observed after 4 weeks. A significant increase was found in the daily total MSDA after the 4-week program, with the average percent heart rate reserve remaining consistent. Physical activity during training positively correlated with ADL levels both at admission (ρ=0.86, P<.001) and 4 weeks post admission (ρ=0.96, P<.001), whereas the correlation between age and MSDA was not significant during training periods at admission (ρ=-0.41, P=.21) or 4 weeks post admission (ρ=-0.25, P=.45). Conversely, nontraining activity showed a negative correlation with age, with significant negative correlations with age at admission (ρ=-0.82, P=.002) and 4 weeks post admission (ρ=-0.73, P=.01). Inpatient rehabilitation activity levels were positively correlated with ADL levels. Further analysis revealed a strong positive correlation between scheduled training activities and ADL levels, whereas nontraining activities showed no such correlation. Instead, a negative correlation between nontraining activities and age was observed. These observations suggest the importance of providing activity opportunities for older patients, while it may also suggest the need for adjusting the activity amount to accommodate the potentially limited fitness levels of this demographic. Future studies with larger patient groups are warranted to validate and further elucidate these findings.

Relationship between skeletal mitochondrial function and digital markers of free-living physical activity in older adults.

This study examined the association between in vivo skeletal mitochondrial function and digital free-living physical activity patterns-a measure that summarizes biological, phenotypic, functional, and environmental effects on mobility. Among 459 participants (mean age 68years; 55% women) in the Baltimore Longitudinal Study of Aging, mitochondrial function was quantified as skeletal muscle oxidative capacity via post-exercise phosphocreatine recovery rate (τPCr) in the vastus lateralis muscle of the left thigh, using 31P magnetic resonance spectroscopy. Accelerometry was collected using a 7-day, 24-h wrist-worn protocol and summarized into activity amount, intensity, endurance, and accumulation patterning metrics. Linear regression, two-part linear and logistic (bout analyses), and linear mixed effects models (time-of-day analyses) were used to estimate associations between τPCr and each physical activity metric. Interactions by age, sex, and gait speed were tested. After covariate adjustment, higher τPCr (or poorer mitochondrial function) was associated with lower activity counts/day (β = - 6593.7, SE = 2406.0; p = 0.006) and activity intensity (- 81.5 counts, SE = 12.9; p < 0.001). For activity intensity, the magnitude of association was greater for men and those with slower gait speed (interaction p < 0.02 for both). Conversely, τPCr was not associated with daily active minutes/day (p = 0.15), activity fragmentation (p = 0.13), or endurance at any bout length (p > 0.05 for all). Time-of-day analyses show participants with high τPCr were less active from 6:00 a.m. to 12:00 a.m. than those with low τPCr. Results indicate that poorer skeletal mitochondrial function is primarily associated with lower engagement in high intensity activities. Our findings help define the connection between laboratory-measured mitochondrial function and real-world physical activity behavior.

Exploratory study to evaluate the acceptability of a wearable accelerometer to assess motor progression in motor neuron disease.

Motor neuron disease (MND) is a rapidly progressive condition traditionally assessed using a questionnaire to evaluate physical function, the revised amyotrophic lateral sclerosis functional rating scale (ALSFRS-R). Its use can be associated with poor sensitivity in detecting subtle changes over time and there is an urgent need for more sensitive and specific outcome measures. The ActiGraph GT9X is a wearable device containing multiple sensors that can be used to provide metrics that represent physical activity. The primary aim of this study was to investigate the initial suitability and acceptability of limb-worn wearable devices to group of people with MND in Scotland. A secondary aim was to explore the preliminary associations between the accelerometer sensor data within the ActiGraph GT9X and established measures of physical function. 10 participants with MND completed a 12-week schedule of assessments including fortnightly study visits, both in-person and over videoconferencing software. Participants wore the device on their right wrist and right ankle for a series of movements, during a 6-min walking test and for a period of 24-h wear, including overnight. Participants also completed an ALSFRS-R and questionnaires on their experience with the devices. 80% of the participants found wearing these devices to be a positive experience and no one reported interference with daily living or added burden. However, 30% of the participants experienced technical issues with their devices. Data from the wearable devices correlated with established measures of physical function.

Afamelanotide for Treatment of the Protoporphyrias: Impact on Quality of Life and Laboratory Parameters in a US Cohort

Background: Erythropoietic protoporphyria (EPP) and X-linked protoporphyria (XLP) are rare disorders of heme biosynthesis characterized by severe cutaneous phototoxicity. Afamelanotide, an α-melanocyte-stimulating hormone analogue, is the only approved treatment for protoporphyria and leads to increased light tolerance and improved quality of life (QoL). However, published experience with afamelanotide in the US is limited. Methods: Here, we report on all adults who received at least one dose of afamelanotide at the Massachusetts General Hospital Porphyria Center from 2021 to 2022. Changes in the time to phototoxic symptom onset, QoL, and laboratory parameters were assessed before and during treatment with afamelanotide. Results: A total of 29 patients with protoporphyria were included, 26 of whom (72.2%) received ≥2 afamelanotide implants. Among the patients who received ≥2 implants, the median time to symptom onset following sunlight exposure was 12.5 min (IQR, 5–20) prior to the initiation of afamelanotide and 120 min (IQR, 60–240) after treatment (p &lt; 0.001). Improvements in QoL during afamelanotide treatment were measured using two QoL tools, with good correlation observed between these two instruments. Finally, we found no improvements in the median levels of metal-free erythrocyte protoporphyrin, plasma protoporphyrin, or liver biochemistries during versus prior to the initiation of afamelanotide treatment. Conclusions: This study highlights a dramatic clinical benefit of afamelanotide in relation to light tolerance and QoL in protoporphyria, albeit without improvement in protoporphyrin levels or measures of liver function.

The dark side of personality functioning: associations between antisocial cognitions, personality functioning (AMPD), empathy and mentalisation

IntroductionWith the introduction of the new psychiatric diagnostic manuals, personality functioning has gained new prominence. Several studies have reported consistent findings that individual showing high levels of antisocial features are associated with alterations in interpersonal functioning domains such as empathy and mentalisation. The focus of the current study (N = 198) is to examine antisocial cognitions, as measured by the Scrambled Sentences Task (SST), and to what extent this approach can help to better understand the relationship between antisocial traits and personality functioning/empathy.MethodWe implemented a hypothesis-driven approach using logistic regression and a data-driven approach using machine learning to examine distinct but related measures of personality functioning as predictors of antisocial cognitions.ResultsAntisocial cognitions were associated with low interpersonal functioning as expected, but only when not adjusting for antisocial traits, which accounted for almost all the association. The data-driven analysis revealed that individual items assessing empathic concern in personality functioning scales (as opposed to the whole scores) explained low antisocial cognitions even when adjusting for antisocial traits.DiscussionAntisocial cognitions appear to be associated to two distinct traits, the antisocial and a specific type of personality functioning. This finding is discussed in terms of the possible distinction between two motivational forces: to harm others/prioritize one’s advantage, and to help suffering others.

Non-invasive assessment of left ventricular contractility by myocardial work index in veno-arterial membrane oxygenation patients: rationale and design of the MIX-ECMO multicentre observational study

Introduction and aimsVeno-arterial extracorporeal membrane oxygenation (VA-ECMO) is an increasingly utilized therapeutic choice in patients with cardiogenic shock, however, high complication rate often counteracts with its beneficial cardiopulmonary effects. The assessment of left ventricular (LV) function in key in the management of this population, however, the most commonly used measures of LV performance are substantially load-dependent. Non-invasive myocardial work is a novel LV functional measure which may overcome this limitation and estimate LV function independent of the significantly altered loading conditions of VA-ECMO therapy. The Usefulness of Myocardial Work IndeX in ExtraCorporeal Membrane Oxygenation Patients (MIX-ECMO) study aims to examine the prognostic role of non-invasive myocardial work in VA-ECMO-supported patients.MethodsThe MIX-ECMO is a multicentric, prospective, observational study. We aim to enroll 110 patients 48–72 h after the initiation of VA-ECMO support. The patients will undergo a detailed echocardiographic examination and a central echocardiography core laboratory will quantify conventional LV functional measures and non-invasive myocardial work parameters. The primary endpoint will be failure to wean at 30 days as a composite of cardiovascular mortality, need for long-term mechanical circulatory support or heart transplantation at 30 days, and besides that other secondary objectives will also be investigated. Detailed clinical data will also be collected to compare LV functional measures to parameters with established prognostic role and also to the Survival After Veno-arterial-ECMO (SAVE) score.ConclusionsThe MIX-ECMO study will be the first to determine if non-invasive myocardial work has added prognostic value in patients receiving VA-ECMO support.

Cardiac myosin inhibitor, CK-586, minimally reduces systolic function and ameliorates obstruction in feline hypertrophic cardiomyopathy.

Hypertrophic cardiomyopathy (HCM) remains the most common cardiomyopathy in humans and cats with few preclinical pharmacologic interventional studies. Small-molecule sarcomere inhibitors are promising novel therapeutics for the management of obstructive HCM (oHCM) patients and have shown efficacy in left ventricular outflow tract obstruction (LVOTO) relief. The objective of this study was to explore the 6-, 24-, and 48-hour (h) pharmacodynamic effects of the cardiac myosin inhibitor, CK-586, in six purpose-bred cats with naturally occurring oHCM. A blinded, randomized, five-treatment group, crossover preclinical trial was conducted to assess the pharmacodynamic effects of CK-586 in this oHCM model. Dose assessments and select echocardiographic variables were assessed five times over a 48-h period. Treatment with oral CK-586 safely ameliorated LVOTO in oHCM cats. CK-586 treatment dose-dependently eliminated obstruction (reduced LVOTOmaxPG), increased measures of systolic chamber size (LVIDs Sx), and decreased select measures of heart function (LV FS% and LV EF%) in the absence of impact on heart rate. At all tested doses, a single oral CK-586 dose resulted in improved or resolved LVOTO with well-tolerated, dose-dependent, reductions in LV systolic function. The results from this study pave the way for the potential use of CK-586 in both the veterinary and human clinical setting.

Sacubitril/valsartan for the treatment of non-obstructive hypertrophic cardiomyopathy: An open label randomized controlled trial (SILICOFCM).

Sacubitril/valsartan treatment reduces mortality and hospitalizations in heart failure with reduced ejection fraction but has limited application in hypertrophic cardiomyopathy (HCM). The aim of this study was to evaluate the effect of sacubitril/valsartan on peak oxygen consumption (VO2) in patients with non-obstructive HCM. This is a phase II, randomized, open-label multicentre study that enrolled adult patients with symptomatic non-obstructive HCM (New York Heart Association class I-III) who were randomly assigned (2:1) to receive sacubitril/valsartan (target dose 97/103 mg) or control for 16 weeks. The primary endpoint was a change in peak VO2. Secondary endpoints included echocardiographic measures of cardiac structure and function, natriuretic peptides and other cardiac biomarkers, and Minnesota Living with Heart Failure quality of life. Between May 2018 and October 2021, 354 patients were screened for eligibility, 115 patients (mean age 58 years, 37% female) met the study inclusion criteria and were randomly assigned to sacubitril/valsartan (n = 79) or control (n = 36). At 16 weeks, there was no significant change in peak VO2 from baseline in the sacubitril/valsartan (15.3 [4.3] vs. 15.9 [4.3] ml/kg/min, p = 0.13) or control group (p = 0.47). No clinically significant changes were found in blood pressure, cardiac structure and function, plasma biomarkers, or quality of life. In patients with HCM, a 16-week treatment with sacubitril/valsartan was well tolerated but had no effect on exercise capacity, cardiac structure, or function.

Randomised controlled trial of an online cognitive training program in school-aged children with cerebral palsy

BackgroundChildren with cerebral palsy (CP) experience deficits in nonverbal reasoning. The SMART online cognitive intervention has been associated with gains in IQ and nonverbal IQ in previous studies in typically developing school-aged children and children experiencing learning difficulties. AimTo assess the efficacy of an online cognitive intervention in school-aged children with CP. Methods and Procedures21 children with CP (male n = 17; 76.2%), mean age 9 y 8 m, SD 1 y 1 month (range 8 y 3 m to 12 y 6 m) were randomised into the intervention group (n = 9) or a waitlist control group. A mixed-methods approach with an explanatory sequential design was used, with a randomised controlled trial followed by qualitative interviews. Participants were assessed on measures of intelligence, academic ability, attention and executive functioning, and social-emotional functioning at baseline, then after completing the training, or the waitlist period. Analyses included ANCOVAs and paired samples t tests. Semi-structured interviews explored participants’ experiences with the training. Results and OutcomesTraining completion was low with a mean of 16.9 modules completed out of 55 available. No significant effect of training was found for the primary outcome of intelligence, or for any secondary outcomes. Participants reported barriers and facilitators for accessing the program. ImplicationsCognitive training programs addressing relational framing ability may require significant modifications before they can be effectively tested with children with CP.

Quantitative muscle magnetic resonance imaging in limb-girdle muscular dystrophy type R1 (LGMDR1): A prospective longitudinal cohort study.

Limb-girdle muscular dystrophy (LGMD) type R1 (LGMDR1) is the most common subtype of LGMD in Europe. Prospective longitudinal data, including clinical assessments and new biomarkers such as quantitative magnetic resonance imaging (qMRI), are needed to evaluate the natural course of the disease and therapeutic options. We evaluated eight thigh and seven leg muscles of 13 LGMDR1 patients (seven females, mean age 36.7 years, body mass index 23.9 kg/m2) and 13 healthy age- and gender-matched controls in a prospective longitudinal design over 1 year. Clinical assessment included testing for muscle strength with quick motor function measure (QMFM), gait analysis and patient questionnaires (neuromuscular symptom score, activity limitation [ACTIVLIM]). MRI scans were performed on a 3-T MRI scanner, including a Dixon-based sequence, T2 mapping and diffusion tensor imaging. The qMRI values of fat fraction (FF), water T2 relaxation time (T2), fractional anisotropy, mean diffusivity, axial diffusivity and radial diffusivity were analysed. Within the clinical outcome measures, significant deterioration between baseline and follow-up was found for ACTIVLIM (p= 0.029), QMFM (p= 0.012). Analysis of qMRI parameters of the patient group revealed differences between time points for both FF and T2 when analysing all muscles (FF: p< 0.001; T2: p= 0.016). The highest increase of fat replacement was found in muscles with an FF of between 10% and 50% at baseline. T2 in muscles with low-fat replacement increased significantly. No significant differences were found for the diffusion metrics. Significant correlations between qMRI metrics and clinical assessments were found at baseline and follow-up, while only T2 changes in thigh muscles correlated with changes in ACTIVLIM over time (ρ = -0.621, p< 0.05). Clinical assessments can show deterioration of the general condition of LGMDR1 patients. qMRI measures can give additional information about underlying pathophysiology. Further research is needed to establish qMRI outcome measures for clinical trials.

P.060 Provider and patient perspectives on outcome measure use in clinical care for chronic inflammatory neuropathy

Background: The use of patient reported and functional outcome measures in routine practice enhances shared decision making and supports patient-centred care. This study compared the perspectives of Chronic Inflammatory Neuropathy (CIN) patients and providers regarding their experience using an outcome measure panel. Methods: A one year study was conducted to evaluate a nine measure outcome set in routine clinical practice for CIN. The panel included patient-reported outcome measures (e.g., I-RODS and EQ-5D-5L) and functional measures (e.g., grip strength). At the conclusion of the study, participants and providers completed an online questionnaire on their experience. Results: 25 patients and five providers completed the questionnaire. Both patients and providers reported benefit in tracking disease progression, supporting treatment-related decisions, and broadening views of health. Both groups agreed patient involvement in care was enhanced. Preference for specific measures, frequency, and data presentation differed. Providers emphasized integration into electronic medical records and streamlining processes. 100% of providers and 80% of patients wanted to continue completing outcome measures. Conclusions: CIN patients and providers recognize the value of integrating outcome measures into routine care. To effectively implement these measures in clinical settings, it is important to understand the patient and provider perspective and prevent unnecessary burdens to ensure sustainability of use.

Association between physical measures of spinopelvic alignment and physical functioning with patient reported outcome measures (PROMs) after total hip arthroplasty: Protocol for systematic review and meta-analysis.

Prevalence of total hip arthroplasty (THA) has trended upwards over past decades and is projected to increase further. Optimizing outcomes after surgery is essential to avoid surgical revision and maximize outcomes. Low back pain is reported as a problem post THA. Patient-reported outcome measures (PROMs) are commonly used to evaluate THA outcomes but have limitations (e.g., ceiling effects). It is therefore important to assess a comprehensive range of outcomes. Physical outcome measures of spinopelvic alignment and physical functioning demonstrate potential value, but no evidence synthesis has investigated their association with PROMs. The objectives of this systematic review are to evaluate the association between spinopelvic alignment and physical outcome measures of physical functioning with PROMs and characteristics of low back pain after THA. This protocol is aligned with the Preferred Reporting Items for Systematic Review and Meta-Analysis Protocols. Cross-sectional and longitudinal cohort studies evaluating the association between the physical outcome measures and PROMs (any outcome measures reported) following THA by any approach/implant will be included except surface replacement and revision THA. Studies investigating THA for developmental pathology and inflammatory conditions will be excluded. A systematic search in MEDLINE (Ovid), Embase (Ovid), Scopus, Web of Science, CINAHL, and the grey literature will be carried out from inception to July 31, 2023. Two independent reviewers will evaluate eligibility of retrieved articles, extract data and assess risk of bias (NIH quality assessment tool) of included studies. A third reviewer will mediate disagreements. Random-effects meta-analyses will be conducted if studies are sufficiently homogeneous in design, population, physical measures and PROMs; reporting odds ratios and 95% confidence intervals. Where meta-analyses are not possible, a narrative synthesis will be conducted. Confidence in cumulative evidence will be assessed using a modified GRADE (Grading of Recommendations Assessment, Development, and Evaluation). PROSPERO Registration number CRD42023412744.

Subjective Versus Psychophysical Measures of Chemosensory Alterations Following COVID-19.

Olfactory dysfunction is a common symptom of COVID-19. However, subjective perception of olfactory function does not always correlate well with more objective measures. This study seeks to clarify associations between subjective and psychophysical measures of olfaction and gustation in patients with subjective chemosensory dysfunction following COVID-19. Adults with persistent COVID-19-associated chemosensory disturbance were recruited for a prospective, longitudinal cohort study at a tertiary care institution. Participants provided subjective measures of olfactory and gustatory function and underwent psychophysical assessment using Sniffin' Sticks olfactory and Monell gustatory tests. Data analysis (n=65) showed a statistically significant association between subjective and psychophysical measures of olfaction (p&lt;0.001). For each one-point increase in subjectively-reported olfactory ability, there is, on average, a 0.11 (95% CI: 0.06, 0.16; p&lt;0.001) point increase in TDI score while adjusting for age at baseline assessment, sex, and follow-up time. For each one-point increase in subjectively-reported olfactory ability, there is, on average, a 0.04 (95% CI: 0.02, 0.06; p&lt;0.001) point and 0.05 (95% CI: 0.03, 0.07; p&lt;0.001) point increase in discrimination and identification scores, respectively, when adjusting for age at baseline assessment, sex, and follow-up time. Subjective olfaction shows a mild to moderate association with psychophysical measures, but it fails to comprehensively assess persistent COVID-19-associated chemosensory deficits. The lack of significant association between subjective olfaction and threshold limits the utility of subjective olfaction in tracking recovery. These findings support the push for more widespread psychophysical chemosensory testing.