Minimal clinically important differences in six-minute walking distance in late-onset Pompe disease
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ABSTRACT:
SUBMITTER: Claeys K
PROVIDER: S-EPMC11008008 | biostudies-literature | 2024 Jan
REPOSITORIES: biostudies-literature
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Project description:OBJECTIVE: To investigate the prevalence and severity of fatigue in adult patients with Pompe disease. METHODS: The Fatigue Severity Scale (FSS) was assessed in an international population of 225 adults with Pompe disease, a metabolic disorder presenting as a slowly progressive proximal myopathy. The FSS scores were compared to those of healthy controls and the relationship between the level of fatigue and other patient characteristics was investigated. RESULTS: The mean age of the participants was 47 (SD 13) years and the mean disease duration 11 (SD 8) years. 43% used a wheelchair and 46% had respiratory support, 29% needed both. 67% of the participants had a FSS score > or =5, indicating severe fatigue. The mean FSS score was 5.2 (SD 1.5), which was significantly higher than that of healthy controls (p < 0.001). Fatigue was not related to age, sex or disease duration. Patients who used a wheelchair or respiratory support were on average more fatigued than those who did not (p = 0.01). However, of the patients who did not use these aids, 59% also had a FSS score > or =5. FSS scores were highest among patients who reported a high frequency of sleep disorders, but patients who never experienced sleep difficulties were also fatigued (mean FSS score = 4.8). CONCLUSION: Fatigue is highly prevalent among both mildly and severely affected adult patients with Pompe disease. The FSS appears a useful tool in assessing fatigue in Pompe disease.
| S-EPMC2779379 | biostudies-other
Project description:BACKGROUND:The six-minute walking test (6MWT) is a tool that plays a key role in evaluating the functional exercise capacity, prognosis and evaluation of treatment response of patients with various cardiopulmonary diseases. However, standard reference equations are currently unavailable for the six-minute walking distance (6MWD) for people aged 60-85?years in China. The purpose of this study was to 1) measure the 6MWD of healthy Chinese people aged 60-85?years, 2) establish reference equations for predicting the 6MWD, and 3) compare our reference equations with equations reported in previously published studies. METHOD:We obtained informed consent from each participant prior to the test, and the research design was approved by the Ethics Committee of Wenzhou People's Hospital. The demographic and anthropometric data and the 6MWD of healthy Chinese subjects aged 60-85?years old were measured using a standardized protocol. Every subject completed two 6MWTs, and the longest 6MWD further analyzed. RESULTS:Two hundred sixty-six subjects (128 males and 138 females) completed the 6MWT, and the mean walking distance was 518?±?72?m. Males achieved a longer walking distance than females (518?±?72?m vs. 487?±?70?m; p <?0.0001), and active subjects achieved a longer walking distance than nonactive subjects (512?±?76?m vs. 485?±?63?m; p <?0.0001). According to the univariate analysis, the 6MWD was significantly associated with age, height, body mass index (BMI), heart rate and blood pressure after exercise and changes in heart rate before and after exercise. The stepwise multivariate regression analysis identified age, height and BMI as independent predictors of the 6MWD. The reference equations for Caucasians and South Americans tended to overestimate the 6MWD of our subjects, while the equations for Asian and African populations tended to underestimate the 6MWD. CONCLUSIONS:This study is the first to describe the 6MWD of healthy Chinese people aged 60-85?years, and reference prediction equations were proposed. These findings will help to improve the evaluation of Chinese patients with diseases that affect exercise capacity.
| S-EPMC7310198 | biostudies-literature
Project description:ObjectivesThis study aimed to determine the association between six-minute walking distance (6MWD) and maximum phonation time (MPT) in healthy adults.Patients and methodsThe cross-sectional study was conducted with 50 sedentary nonsingers (32 females, 18 males; mean age: 33.5±8.3 years; range, 18 to 50 years) between February 2021 and April 2021. Subjects with a history of smoking, respiratory symptoms in the last two weeks, and heart, lung, musculoskeletal, and balance problems were excluded. The measurements of MPT and 6MWD were carried out by two different assessors blinded to each other.ResultsThe mean MPT was higher in male subjects (27.4±7.4 sec vs. 20.6±5.1 sec, p<0.001). In the bivariate analysis, there was a significant correlation between MPT and 6MWD (r=0.621, p<0.001), as well as body height (r=0.421, p=0.002) and the mean fundamental frequency (r=-0.429, p=0.002); however, no association was found with age, body weight, and the mean sound pressure level. After multiple regression, 6MWD was the only factor associated with MPT (p=0.002).ConclusionThere is a significant association between 6MWD and MPT in healthy adults, and the results suggest that aerobic capacity might have a role in improving the ability to sustain phonation.
| S-EPMC10186023 | biostudies-literature
Project description:ObjectiveThe 6-min walk distance (6MWD), a widely used test of functional capacity, has limited evidence of construct validity among patients surviving acute respiratory failure (ARF) and ARDS. The objective of this study was to examine construct validity and responsiveness and estimate minimal important difference (MID) for the 6MWD in patients surviving ARF/ARDS.MethodsFor this secondary data analysis of four international studies of adult patients surviving ARF/ARDS (N = 641), convergent and discriminant validity, known group validity, predictive validity, and responsiveness were assessed. MID was examined using anchor- and distribution-based approaches. Analyses were performed within studies and at various time points after hospital discharge to examine generalizability of findings.ResultsThe 6MWD demonstrated good convergent and discriminant validity, with moderate to strong correlations with physical health measures (|r| = 0.36-0.76) and weaker correlations with mental health measures (|r| = 0.03-0.45). Known-groups validity was demonstrated by differences in 6MWD between groups with differing muscle strength and pulmonary function (all P < .01). Patients reporting improved function walked farther, supporting responsiveness. 6MWD also predicted multiple outcomes, including future mortality, hospitalization, and health-related quality of life. The 6MWD MID, a small but consistent patient-perceivable effect, was 20 to 30 m. Findings were similar for 6MWD % predicted, with an MID of 3% to 5%.ConclusionsIn patients surviving ARF/ARDS, the 6MWD is a valid and responsive measure of functional capacity. The MID will facilitate planning and interpretation of future group comparison studies in this population.
| S-EPMC4420183 | biostudies-literature
Project description:BackgroundThe 6-min walk test (6MWT) is a commonly used clinical assessment of exercise capacity in patients with cardiopulmonary or neuromuscular disease, but normal values are lacking for young adults, who are frequent subjects of testing.MethodsIn a two-center study, 272 young adults, ages 18-50, underwent American Thoracic Society protocolized 6-min walk testing, and 56 underwent repeat testing. A linear regression model was developed based on anthropomorphic data. This model was compared to existing prediction equations.ResultsMedian 6MWD for the cohort was 637 m (IQR 584-686 m) and was not significantly impacted by age. This is in contrast to existing equations extrapolated from older subjects that predict increasing 6MWD in younger subjects. We found weak correlation of 6MWD with height, weight, BMI, and resting heart rate. Heart rate at completion correlated most strongly with 6MWD (rho 0.53 p < 0.0001). Repeat 6MWD was surprisingly variable, with a median difference between tests of 32.5 ± 31.9 m. Established reference equations performed poorly in this population, largely because age has much less effect on 6MWD in this group than in older adults.ConclusionsEstablished reference equations should be reconfigured to include data from young adults, as age has minimal effect on 6MWD in this population. Heart rate response may be a valuable measure of effort in normal subjects. Six-minute walk distance, as with pulmonary function and exercise testing, should have predictive equations across the spectrum of age to allow for accurate assessment of exercise limitation.
| S-EPMC7174378 | biostudies-literature
Project description:There is limited data on pregnancy outcomes in Pompe Disease (PD) resulting from deficiency of the lysosomal enzyme acid alpha-glucosidase. Late-onset PD is characterized by progressive proximal muscle weakness and decline of respiratory function secondary to the involvement of the respiratory muscles. In a cohort of twenty-five females, the effects of both PD on the course of pregnancy and the effects of pregnancy on PD were investigated. Reproductive history, course of pregnancy, use of Enzyme replacement therapy (ERT), PD symptoms, and outcomes of each pregnancy were obtained through a questionnaire. Among 20 subjects that reported one or more pregnancies, one subject conceived while on ERT and continued therapy through two normal pregnancies with worsening of weakness during pregnancy and improvement postpartum. While fertility was not affected, pregnancy may worsen symptoms, or cause initial symptoms to arise. Complications with pregnancy or birth were not higher, except for an increase in the rate of stillbirths (3.8% compared to the national average of 0.2-0.7%). Given small sample size and possible bias of respondents being only women who have been pregnant, further data may be needed to better analyze the effects of pregnancy on PD, and the effects of ERT on pregnancy outcomes.
| S-EPMC7556025 | biostudies-literature
Project description:BackgroundThe responsiveness of the endurance shuttle walk to functional changes following bronchodilation has recently been reported. The current literature suggests that the 6 min walking test (6MWT) is less responsive to bronchodilation than the endurance shuttle walk.AimTo compare bronchodilator-induced changes in exercise performance with the 6MWT and the endurance shuttle walk.MethodsIn a randomised, double-blind, placebo-controlled, crossover trial, 14 patients with chronic obstructive pulmonary disease (forced expiratory volume in 1 s (FEV(1)) 50 (8)% predicted) completed two 6MWTs and two endurance shuttle walks, each preceded by nebulised placebo or 500 microg ipratropium bromide. Cardiorespiratory parameters were monitored during each walking test with a portable telemetric gas analyser. Quadriceps twitch force was measured by magnetic stimulation of the femoral nerve before and after each walking test.ResultsThe 6 min walking distance did not change significantly after bronchodilation despite a significant increase in FEV(1) of 0.18 (0.09) litres (p<0.001). A similar change in FEV(1) (0.18 (0.12) litres, p<0.001) was associated with a significant improvement in the distance walked on the endurance shuttle walk (Deltadistance ipratropium bromide--placebo = 144 (219) m, p = 0.03). Quadriceps muscle fatigue was infrequent (<15% of patients) after both walking tests.ConclusionThe endurance shuttle walk is more responsive than the 6MWT for detecting changes in exercise performance following bronchodilation.
| S-EPMC2092479 | biostudies-literature
Project description: Not available
| S-EPMC10664940 | biostudies-literature
Project description:BackgroundMaximal inspiratory pressure (PImax) and 6-minutes walk distance test (6MWD) may be more available and feasible alternatives for prognostic assessment than cardiopulmonary testing. We hypothesized that the PImax and 6MWD combination could improve their individual accuracy as risk predictors. We aimed to evaluate PImax ability as a mortality predictor in HF and whether the combination to 6MWD could improve risk stratification.MethodsProspective cohort from HF Clinics of three University Hospitals. PImax, 6MWD and pVO2 were obtained at baseline. The end point was all cause mortality.ResultsConsecutive 256 individuals (50% woman, 57.4±10.4years) with low ejection fraction (LVEF) (31.8±8.6%) were followed up to 10years. During a median follow-up of 34.7 (IQR 37) months, 110 participants died. Mean±SD values were: pVO2 14.9±5.1mL/kg/min, PImax 5.5±1.3kPa and 6MWD 372±118m. In multivariate Cox regression, pVO2, PImax, 6MWD and LVEF were independent mortality predictors. The pVO2 showed gold standard accuracy, followed by PImax (AUC = 0.84) and 6MWD (AUC = 0.74). Kaplan-Meier mean survival time (MST±SE) for lower (≤5.0kPa) and higher (>6.0kPa) PImax tertiles, were 37.9±2.8months and 105.0±5.2months respectively, and addition of 6MWD did not restratified risk. For intermediate PImax tertile, MST was 81.5±5.5months, but adding 6MWD, MST was lower (53.3±7.6months) if distance was ≤350m and higher (103.1±5.7months) for longer distances.ConclusionPImax is an independent mortality predictor in HF, more accurate than 6MWD and LVEF. Addition of 6MWD empowers risk stratification only for intermediate PImax tertile. Although less accurate than pVO2, this simpler approach could be a feasible alternative as a prognostic assessment.
| S-EPMC6675323 | biostudies-literature
Project description:BackgroundThe 3-minute chair rise test (3-minute CRT) and the Disability Related to COPD Tool (DIRECT) are two reproducible and valid short tests that can assess the benefit of pulmonary rehabilitation (PR) in terms of functional capacity and dyspnea in everyday activities.MethodsWe determined the minimal clinically important difference (MCID) of the DIRECT questionnaire and 3-minute CRT using distribution methods and anchor encroaches with a panel of eight standard tests in a cohort of 116 COPD patients who completed a PR program in real-life settings.ResultsThe estimated MCID for the 3-minute CRT and DIRECT scores was five repetitions and two units, respectively, using separate and combined independent anchors. The all-patient (body mass index-obstruction-dyspnea-exercise [BODE] scores 0-7), BODE 0-2 (n=42), and BODE 3-4 (n=50) groups showed improvements greater than the MCID in most tests and questionnaires used. In contrast, the BODE 5-7 group (n=24) showed improvements greater than MCID in only the 3-minute CRT, 6-minute walk test, endurance exercise test, and DIRECT questionnaire.Discussion and conclusionThis study demonstrates that the short and simple DIRECT questionnaire and 3-minute CRT are responsive to capture the beneficial effects of a PR program in COPD patients, including those with severe disease.Trial registration numberNCT03286660.
| S-EPMC6349077 | biostudies-literature