AIM:This study aims to examine weight-loss history and pre-treatment concerns, and their associations with current functioning and well-being in adults undergoing incretin-based pharmacotherapy for obesity. MATERIALS AND METHODS:This cross-sectional study analysed 411 adults treated with three different incretin-based medications. Demographics, previous weight-loss interventions, barriers to earlier attempts, reasons for initiating incretin therapy, treatment characteristics and Treatment-Related Impact Measure for Weight (TRIM-Weight) total scores were assessed. Associations between specific pre-treatment concerns and functioning and well-being were analysed using univariate models and a multivariable regression model that adjusted for treatment related variables. RESULTS:Participants had a median age of 39 years (IQR 33-47), 79.1% were women, and the median BMI was 36.3 kg/m2 (IQR 32.2-42.3). The median TRIM-Weight total score was 68.2 (IQR 64.0-73.4). Most respondents (95.1%) had attempted weight loss, typically through combined diet and physical activity (71.8%), intermittent fasting (69.6%) and digital tracking tools (65.7%). In fully adjusted models, fear of side effects (β = -3.53, 95% CI -4.05 to -3.01), concerns about cost (β = -0.92, 95% CI -1.37 to -0.47) and injection anxiety (β = -0.67, 95% CI -1.16 to -0.18) were associated with lower functioning and well-being. Doubts regarding effectiveness showed a small positive association (β = 0.61, 95% CI 0.03-1.19). CONCLUSION:Among retrospectively reported pre-treatment concerns, fear of side effects, treatment costs and injection anxiety were most strongly associated with lower functioning and well-being, whereas moral or psychosocial concerns showed no independent associations after multivariable adjustment.
Objectives Post-exertional malaise (PEM) is increasingly understood not as a singular symptom but as a multidimensional exertion-response pattern involving diverse triggers, variable onset, and prolonged recovery. This study aimed to develop and content-validate the Vienna Post-Exertional Malaise (VPEM) Questionnaire, a multidimensional patient-reported outcome measure for PEM in myalgic encephalomyelitis/chronic fatigue syndrome (ME/CFS), designed to assess the consequences of PEM, including functional decline, prolonged recovery, new crash-associated symptoms, and pacing-related adaptations. Methods An iterative mixed-methods instrument-development study was conducted, combining quantitative ratings and qualitative feedback across sequential evaluation cycles involving individuals with lived experience and an interdisciplinary expert panel. An initial 13-item draft, derived from PEM, fatigue, and functional-capacity instruments, as well as consensus literature, was evaluated by individuals with lived experience of ME/CFS or Long COVID using Likert ratings, open-ended feedback, and semi-structured interviews. Quantitative data were summarised descriptively; qualitative data were analysed using deductive–inductive structuring content analysis. Revisions were agreed upon by a core development team and documented in revision matrices. The revised instrument version was evaluated by an interdisciplinary expert panel using domain-level ratings and item-level ratings of relevance and clarity. Results Twelve individuals with ME/CFS and one individual with Long COVID who also fulfilled ME/CFS criteria evaluated the questionnaire. Ratings showed high approval for questionnaire length (92.3%) and terminology (76.9%), while ratings for completeness and clarity were more heterogeneous. Qualitative analysis revealed conceptual ambiguities concerning the distinction between PEM and non-specific fatigue, the absence of trigger domains such as sensory, orthostatic and positively valenced emotional triggers, and inadequate representation of intra-individual variability. Following revision, the expert panel (n = 11) rated PEM coverage as either complete or largely complete (100%), and the multidimensional structure as either suitable or very suitable (90.9%). Item-level ratings revealed high content validity in terms of relevance (S-CVI/Ave = 0.96) and acceptable clarity (S-CVI/Ave = 0.82). The complete revised questionnaire was subsequently pretested online with five participants. Conclusions The VPEM Questionnaire operationalises PEM as a multidimensional pattern of post-exertional deterioration. Findings support initial content and face validity and provide the basis for psychometric validation.
Background:The cumulative probability of patients with Crohn's disease (CD) requiring a stoma at a tertiary center is unknown. We sought to evaluate the time to stoma formation after diagnosis as well as risk factors for stoma formation in CD patients. Methods:This is a retrospective, longitudinal cohort study on consecutive patients with CD at an Austrian tertiary referral university center. In brief, patients with CD were identified and disease-specific data were captured prior to June 2023. The probability of stoma-free survival by subgroups of various potential risk factors was assessed by means of Kaplan-Meier estimates (Log-rank test). Results:Of 1267 CD patients, 142 (11.2%) underwent a stoma formation (80 ileostomies, 62 colostomies), of which 51 (35.9%) were permanent stomas. The probability of stoma-free survival at 10 and 20 years after diagnosis was 92% and 86%, respectively. Ileocolonic and colonic location, penetrating behavior, and perianal disease were associated with a higher risk for stoma formation (each P < .001). If these risk factors coincided, the probability of stoma-free survival at 10 and 20 years after diagnosis was only 76.4% and 67.2%, respectively. Patients diagnosed from 2000 onward had a trend for a lower stoma risk than patients diagnosed earlier (P = .084). Conclusions:The coincidence of colonic or ileocolonic involvement, penetrating behavior, and perianal fistulas significantly reduced the probability of stoma-free survival to 68% 20 years after diagnosis. Identification of these risk factors for stoma formation may help identify patients at risk and thus raise awareness for this group.
Background:Spinal muscular atrophy (SMA) is a genetic motor neuron disease marked by the progressive decline of motor function. Risdiplam, an orally administered SMN2 splicing modifier, was approved for the treatment of 5q-associated SMA (5q-SMA) across all age groups. However, clinical trial data have primarily focused on paediatric populations, with limited evidence available for adult patients. This study aimed to evaluate the efficacy and safety of risdiplam in treatment-naïve adults with 5q-SMA in a real-world, multicentre setting. Methods:We conducted a nationwide, observational cohort study across eight neuromuscular centres in Austria. Patients aged ≥16 years at treatment initiation with genetically confirmed 5q-SMA, who were previously untreated and initiated risdiplam between December 2020 and September 2024 were eligible for inclusion if they had received risdiplam for ≥3 months and had functional motor assessments available at baseline (T0) and at least one follow-up. Functional outcomes were assessed at four predefined intervals after baseline: 3-<6 months (T1), 6-<12 months (T2), 12-<18 months (T3), and ≥18 months (T4). The primary outcome was the change from baseline in the Hammersmith Functional Motor Scale Expanded (HFMSE). Secondary outcomes included changes in the Revised Upper Limb Module (RULM), Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R), and 6-min walk test (6MWT). Adverse events were extracted from medical records. Findings:A total of 87 patients had received risdiplam, of whom 57 fulfilled the inclusion criteria and were included in this study. The median age at treatment initiation was 35.7 years (IQR 28.8-43.4), with a median disease duration of 29.6 years (IQR 24.2-36.3). Most individuals had SMA type II (40.4%) or III (47.4%). Mean HFMSE changes from baseline were +1.00 (95% CI 0.05-1.95, p = 0.0100) at T1, +0.97 (95% CI 0.22-1.72, p = 0.0132) at T2, +1.78 (0.66-2.89, p = 0.0008) at T3, and +1.73 (0.49-2.97, p = 0.0049) at T4. Clinically meaningful improvements in motor function (≥3 points in HFMSE and/or ≥2 in RULM) were observed in 63.9% of patients at T4. Improvements were more pronounced in patients with higher baseline function, ambulatory status, or without a history of spinal surgery. Risdiplam was generally well tolerated, with predominantly mild and non-specific adverse events reported in 14.0% of patients. Interpretation:In this nationwide observational study in a real-world setting, adult patients with 5q-SMA demonstrated consistent and clinically meaningful functional improvements with risdiplam over time, particularly by 18 months and beyond. These findings support the long-term use of risdiplam in adults with SMA and help close a critical evidence gap in this underrepresented population. Funding:This study was financially supported by F. Hoffmann-La Roche Ltd.
In the context of evaluating physical function in individuals with stroke, the 3-Meter Backward Walk Test (3MBWT) emerges as a potential tool of interest. The purpose of this study was to assess the test–retest reliability and concurrent validity of the 3MBWT and its correlation with falling incidents. Conducted in a neurological rehabilitation center, 35 ambulatory individuals with stroke were enrolled within a month post-stroke onset. These participants, with a Functional Ambulation Category score of ≥4, underwent the 3MBWT, Functional Gait Assessment (FGA), 10-Meter Walk Test (10MWT), and 6-Minute Walk Test (6MWT) under the supervision of different physiotherapists. The results indicate that the 3MBWT demonstrated high reliability, with an Intraclass Correlation Coefficient of 0.97 (95% CI: 0.95, 0.98). It also showed significant concurrent validity with other established walking tests like the 6MWT (r = −0.78) and 10MWT (r = 0.71), with a moderate correlation with the FGA (r = −0.54). No marked differences in test outcomes were observed between participants based on their fall history. Conclusively, the 3MBWT proves to be highly reliable and agrees well with existing walking function assessments for stroke patients, suggesting its potential as a time-efficient alternative.
In the context of evaluating physical function in individuals with stroke, the 3-meter backward walk test (3MBWT) emerges as a potential tool of interest. The purpose of this study was to assess the test-retest reliability and construct validity of the 3MBWT and its correlation with falling incidents. Conducted in a neurological rehabilitation center, 35 ambulatory individuals with stroke were enrolled within a month post-stroke onset. These participants, with a Functional Ambulation Category score of ≥4, underwent the 3MBWT, Functional Gait Assessment (FGA), 10-Meter Walk Test (10MWT), and 6-Minute Walk Test (6MWT) three times by different physiotherapists. Results indicated the 3MBWT displayed high reliability (r=0.97-0.98) and showed significant construct validity with other established walking tests like the 6MWT (r=-0.78) and 10MWT (r=0.71), with a moderate correlation to the FGA (r=-0.54). No marked differences in test outcomes were observed between participants based on their fall history. Conclusively, the 3MBWT proves to be highly reliable and validates well with existing walking function assessments for stroke patients, suggesting its potential as a time-efficient alternative.
BACKGROUND:We evaluated the associations of handgrip strength and cognitive function in cancer survivors ≥ 60 years old using data from the National Health and Nutrition Examination Survey (NHANES). METHODS:Data in two waves of NHANES (2011-2014) were aggregated. Handgrip strength in kilogram (kg) was defined as the maximum value achieved using either hand. Two cognitive function tests were conducted among adults 60 years and older. The Animal Fluency Test (AFT) examines categorical verbal fluency (a component of executive function), and the Digital Symbol Substitution test (DSST) assesses processing speed, sustained attention, and working memory. Survey analysis procedures were used to account for the complex sampling design of the NHANES. Multiple linear regression models were used to estimate associations of handgrip strength with cognitive test scores, adjusting for confounders (age, gender, race/ethnicity, education, marital status, smoking status, depressive symptoms and leisure time physical activity). RESULTS:Among 383 cancer survivors (58.5% women, mean age = 70.9 years, mean BMI = 29.3 kg/m2), prevalent cancer types were breast (22.9%), prostate (16.4%), colon (6.9%) and cervix (6.2%). In women, each increase in kg of handgrip strength was associated with 0.20 (95% CI: 0.08 to 0.33) higher score on AFT and 0.83 (95% CI: 0.30 to 1.35) higher score on DSST. In men, we observed an inverted U-shape association where cognitive function peaked at handgrip strength of 40-42 kg. CONCLUSIONS:Handgrip strength, a modifiable factor, appears to be associated with aspects of cognitive functions in cancer survivors. Prospective studies are needed to address their causal relationship.