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Repeatability and sensitivity to change of non-invasive end points in PAH: the RESPIRE study.

ABSTRACT: End points that are repeatable and sensitive to change are important in pulmonary arterial hypertension (PAH) for clinical practice and trials of new therapies. In 42 patients with PAH, test-retest repeatability was assessed using the intraclass correlation coefficient and treatment effect size using Cohen's d statistic. Intraclass correlation coefficients demonstrated excellent repeatability for MRI, 6 min walk test and log to base 10 N-terminal pro-brain natriuretic peptide (log₁₀NT-proBNP). The treatment effect size for MRI-derived right ventricular ejection fraction was large (Cohen's d 0.81), whereas the effect size for the 6 min walk test (Cohen's d 0.22) and log₁₀NT-proBNP (Cohen's d 0.20) were fair. This study supports further evaluation of MRI as a non-invasive end point for clinical assessment and PAH therapy trials.Trial registration number NCT03841344.

SUBMITTER: Swift AJ

PROVIDER: S-EPMC8461450 | biostudies-literature | 2021 Oct

REPOSITORIES: biostudies-literature

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Repeatability and sensitivity to change of non-invasive end points in PAH: the RESPIRE study.

Swift Andrew J AJ Wilson Frederick F Cogliano Marcella M Kendall Lindsay L Alandejani Faisal F Alabed Samer S Hughes Paul P Shahin Yousef Y Saunders Laura L Oram Charlotte C Capener David D Rothman Alex A Garg Pankaj P Johns Christopher C Austin Matthew M Macdonald Alistair A Pickworth Jo J Hickey Peter P Condliffe Robin R Cahn Anthony A Lawrie Allan A Wild Jim M JM Kiely David G DG

Thorax 20210225 10

End points that are repeatable and sensitive to change are important in pulmonary arterial hypertension (PAH) for clinical practice and trials of new therapies. In 42 patients with PAH, test-retest repeatability was assessed using the intraclass correlation coefficient and treatment effect size using Cohen's d statistic. Intraclass correlation coefficients demonstrated excellent repeatability for MRI, 6 min walk test and log to base 10 N-terminal pro-brain natriuretic peptide (log<sub>10</sub>NT ...[more]

PMID: 33632769

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Project description:BackgroundThere is little published data investigating non-invasive cardiac output monitoring in the emergency department (ED). We assessed six non-invasive fluid responsiveness monitoring methods which measure cardiac output directly or indirectly for their feasibility and repeatability of measurements in the ED: (1) left ventricular outflow tract echocardiography derived velocity time integral, (2) common carotid artery blood flow, (3) suprasternal aortic Doppler, (4) bioreactance, (5) plethysmography with digital vascular unloading method, and (6) inferior vena cava collapsibility index.MethodsThis is a prospective observational study of non-invasive methods of assessing fluid responsiveness in the ED. Participants were non-ventilated ED adult patients requiring intravenous fluid resuscitation. Feasibility of each method was determined by the proportion of clinically interpretable measurements from the number of measurement attempts. Repeatability was determined by comparing the mean difference of two paired measurements in a fluid steady state (after participants received an intravenous fluid bolus).Results76 patients were recruited in the study. A total of 207 fluid responsiveness measurement sets were analysed. Feasibility rates were 97.6% for bioreactance, 91.3% for vascular unloading method with plethysmography, 87.4% for common carotid artery blood flow, 84.1% for inferior vena cava collapsibility index, 78.7% for LVOT VTI, and 76.8% for suprasternal aortic Doppler. The feasibility rates difference between bioreactance and all other methods was statistically significant.ConclusionOur study shows that non-invasive fluid responsiveness monitoring in the emergency department may be feasible with selected methods. Higher repeatability of measurements were observed in non-ultrasound methods. These findings have implications for further studies specifically assessing the accuracy of such non-invasive cardiac output methods and their effect on patient outcome in the ED in fluid depleted states such as sepsis.

Project description:ObjectivesEvaluate test-retest repeatability, ability to discriminate between osteoarthritic and healthy participants, and sensitivity to change over 6 months, of dynamic contrast-enhanced magnetic resonance imaging (DCE-MRI) biomarkers in knee OA.MethodsFourteen individuals aged 40-60 with mild-moderate knee OA and 6 age-matched healthy volunteers (HV) underwent DCE-MRI at 3 T at baseline, 1 month and 6 months. Voxelwise pharmacokinetic modelling of dynamic data was used to calculate DCE-MRI biomarkers including Ktrans and IAUC60. Median DCE-MRI biomarker values were extracted for each participant at each study visit. Synovial segmentation was performed using both manual and semiautomatic methods with calculation of an additional biomarker, the volume of enhancing pannus (VEP). Test-retest repeatability was assessed using intraclass correlation coefficients (ICC). Smallest detectable differences (SDDs) were calculated from test-retest data. Discrimination between OA and HV was assessed via calculation of between-group standardised mean differences (SMD). Responsiveness was assessed via the number of OA participants with changes greater than the SDD at 6 months.ResultsKtrans demonstrated the best test-retest repeatability (Ktrans/IAUC60/VEP ICCs 0.90/0.84/0.40, SDDs as % of OA mean 33/71/76%), discrimination between OA and HV (SMDs 0.94/0.54/0.50) and responsiveness (5/1/1 out of 12 OA participants with 6-month change > SDD) when compared to IAUC60 and VEP. Biomarkers derived from semiautomatic segmentation outperformed those derived from manual segmentation across all domains.ConclusionsKtrans demonstrated the best repeatability, discrimination and sensitivity to change suggesting that it is the optimal DCE-MRI biomarker for use in experimental medicine studies.Key points• Dynamic contrast-enhanced MRI (DCE-MRI) provides quantitative measures of synovitis in knee osteoarthritis which may permit early assessment of efficacy in experimental medicine studies. • This prospective observational study compared DCE-MRI biomarkers across domains relevant to experimental medicine: test-retest repeatability, discriminative validity and sensitivity to change. • The DCE-MRI biomarker Ktrans demonstrated the best performance across all three domains, suggesting that it is the optimal biomarker for use in future interventional studies.

Project description:BackgroundDiarrhea is the second leading cause of death in children < 5 years globally and the parasite genus Cryptosporidium is a leading cause of that diarrhea. The global disease burden attributable to cryptosporidiosis is substantial and the only approved chemotherapeutic, nitazoxanide, has poor efficacy in HIV positive children. Chemotherapeutic development is dependent on the calf model of cryptosporidiosis, which is the best approximation of human disease. However, the model is not consistently applied across research studies. Data collection commonly occurs using two different methods: Complete Fecal Collection (CFC), which requires use of confinement housing, and Interval Collection (IC), which permits use of box stalls. CFC mimics human challenge model methodology but it is unknown if confinement housing impacts study end-points and if data gathered via this method is suitable for generalization to human populations.MethodsUsing a modified crossover study design we compared CFC and IC and evaluated the impact of housing on study end-points. At birth, calves were randomly assigned to confinement (n = 14) or box stall housing (n = 9), or were challenged with 5 x 107 C. parvum oocysts, and followed for 10 days. Study end-points included fecal oocyst shedding, severity of diarrhea, degree of dehydration, and plasma cortisol.FindingsCalves in confinement had no significant differences in mean log oocysts enumerated per gram of fecal dry matter between CFC and IC samples (P = 0.6), nor were there diurnal variations in oocyst shedding (P = 0.1). Confinement housed calves shed significantly more oocysts (P = 0.05), had higher plasma cortisol (P = 0.001), and required more supportive care (P = 0.0009) than calves in box stalls.ConclusionHousing method confounds study end-points in the calf model of cryptosporidiosis. Due to increased stress data collected from calves in confinement housing may not accurately estimate the efficacy of chemotherapeutics targeting C. parvum.

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Repeatability and sensitivity to change of non-invasive end points in PAH: the RESPIRE study.

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Repeatability and sensitivity to change of non-invasive end points in PAH: the RESPIRE study.

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