Repeated measures designs are widely used in practice to increase power, reduce sample size, and increase efficiency in data collection. Correlation between repeated measurements is one of the first ...research questions that needs to be addressed in a repeated-measure study. In addition to an estimate for correlation, confidence interval should be computed and reported for statistical inference. The asymptotic interval based on the delta method is traditionally calculated due to its simplicity. However, this interval is often criticized for its unsatisfactory performance with regards to coverage and interval width. Bootstrap could be utilized to reduce the interval width, and the widely used bootstrap intervals include the percentile interval, the bias-corrected interval, and the bias-corrected with acceleration interval. Wilcox (Comput Stat Data Anal 22:89–98,1996) suggested a modified percentile interval with the interval levels adjusted by sample size to have the coverage probability close to the nominal level. For a study with repeated measures, more parameters in addition to sample size would affect the coverage probability. For these reasons, we propose modifying the percentiles in the percentile interval to guarantee the coverage probability based on simulation studies. We analyze the correlation between imaging volumes and memory scores from the Alzheimer’s Disease Neuroimaging Initiative (ADNI) study to illustrate the application of the considered intervals. The proposed interval is exact with the coverage probability guaranteed, and is recommended for use in practice.
•Parkinson’s disease with freezing of gait is associated with sleep and mood problems.•Non-motor Parkinson’s disease symptoms can predict freezing of gait development.•Processing speed, learning ...decline, and increased sleepiness predict future freezing.
The etiology of freezing of gait in Parkinson’s disease (PD) is yet to be clarified. Non-motor risk factors including cognitive impairment, sleep disturbance and mood disorders have been shown in freezing of gait.
We aimed to determine the predictive value of non-motor features in freezing of gait development.
Data were obtained from the Parkinson’s Progression Markers Initiative. Fifty PD patients with self-reported freezing of gait, and 50 PD patients without freezing of gait at the fourth year visit were included. Groups were matched for Movement Disorders Society-Unified Parkinson’s Disease Rating Scale Part III scores. Several cognitive and non-cognitive tests were used for non-motor features at baseline and over time. Executive function, visuospatial function, processing speed, learning and memory tests were used for cognition. Non-cognitive tests included sleepiness, REM sleep behavior disorder, depression and anxiety scales.
Patients with freezing of gait had higher scores on sleepiness, REM sleep behavior disorder, depression and anxiety scales. However, predictor model analysis revealed that baseline processing speed, learning and sleepiness scores were predictive of self-reported freezing of gait development over time.
Our findings suggest that specific cognitive deficits and sleep disorders are predictive of future freezing of gait. These features may be helpful in identifying underlying networks in freezing of gait and should be further investigated with neuroimaging studies.
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GEOZS, IJS, IMTLJ, KILJ, KISLJ, NLZOH, NUK, OILJ, PNG, SAZU, SBCE, SBJE, UILJ, UL, UM, UPCLJ, UPUK, ZAGLJ, ZRSKP
Meta-analysis provides a useful statistical tool to effectively estimate treatment effect from multiple studies. When the outcome is binary and it is rare (e.g., safety data in clinical trials), the ...traditionally used methods may have unsatisfactory performance.
We propose using importance sampling to compute confidence intervals for risk difference in meta-analysis with rare events. The proposed intervals are not exact, but they often have the coverage probabilities close to the nominal level. We compare the proposed accurate intervals with the existing intervals from the fixed- or random-effects models and the interval by Tian et al. (2009).
We conduct extensive simulation studies to compare them with regards to coverage probability and average length, when data are simulated under the homogeneity or heterogeneity assumption of study effects.
The proposed accurate interval based on the random-effects model for sample space ordering generally has satisfactory performance under the heterogeneity assumption, while the traditionally used interval based on the fixed-effects model works well when the studies are homogeneous.
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DOBA, IZUM, KILJ, NUK, PILJ, PNG, SAZU, SIK, UILJ, UKNU, UL, UM, UPUK
In ophthalmologic studies, measurements obtained from both eyes of an individual are often highly correlated. Ignoring the correlation could lead to incorrect inferences. An asymptotic method was ...proposed by Tang and others (2008) for testing equality of proportions between two groups under Rosner's model. In this article, we investigate three testing procedures for general g ≥ 2 groups. Our simulation results show the score testing procedure usually produces satisfactory type I error control and has reasonable power. The three test procedures get closer when sample size becomes larger. Examples from ophthalmologic studies are used to illustrate our proposed methods.
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DOBA, IZUM, KILJ, NUK, PILJ, PNG, SAZU, SIK, UILJ, UKNU, UL, UM, UPUK
Ensemble methods, such as the traditional bagging algorithm, can usually improve the performance of a single classifier. However, they usually require large storage space as well as relatively ...time-consuming predictions. Many approaches were developed to reduce the ensemble size and improve the classification performance by pruning the traditional bagging algorithms. In this article, we proposed a two-stage strategy to prune the traditional bagging algorithm by combining two simple approaches: accuracy-based pruning (AP) and distance-based pruning (DP). These two methods, as well as their two combinations, “AP+DP” and “DP+AP” as the two-stage pruning strategy, were all examined. Comparing with the single pruning methods, we found that the two-stage pruning methods can furthermore reduce the ensemble size and improve the classification. “AP+DP” method generally performs better than the “DP+AP” method when using four base classifiers: decision tree, Gaussian naive Bayes, K-nearest neighbor, and logistic regression. Moreover, as compared to the traditional bagging, the two-stage method “AP+DP” improved the classification accuracy by 0.88%, 4.06%, 1.26%, and 0.96%, respectively, averaged over 28 datasets under the four base classifiers. It was also observed that “AP+DP” outperformed other three existing algorithms Brag, Nice, and TB assessed on 8 common datasets. In summary, the proposed two-stage pruning methods are simple and promising approaches, which can both reduce the ensemble size and improve the classification accuracy.
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FZAB, GIS, IJS, IZUM, KILJ, NLZOH, NUK, OILJ, PILJ, PNG, SAZU, SBCE, SBMB, UL, UM, UPUK
Acute care physical therapists recommend discharge locations and services in part to help prevent falls during post-discharge recovery. Therapists may use standardized tests to inform their ...recommendation decisions, but evidence linking test scores with fall risk after discharge is lacking. The primary purpose of this study was to explore the associations between Tinetti Performance-Oriented Mobility Assessment (POMA) and Activity Measure for Post-Acute Care Inpatient Mobility Short Form (AM-PAC IMSF) scores and falls in the first 30 days after hospital discharge. Anticipating that agreement between therapist recommendations and discharge locations and services (discharge agreement), age, and sex could impact those associations, these factors were included in this investigation.
In this observational cohort study, 258 hospitalized patients consented to medical record data extraction and answered a phone survey 30 days after discharge to report whether they had experienced a fall since leaving the hospital. POMA and AM-PAC IMSF tests were administered for every patient. Participants' age, sex, diagnosis, last POMA score, last AM-PAC IMSF score, physical therapist discharge recommendations, actual discharge location and services, discharge date, and phone number were collected from their medical records.
When analyzed alone, higher POMA scores were associated with lower odds of falling, but the association was not significant after adjustment for other factors. Neither AM-PAC IMSF scores, age, nor sex were associated with falls. Discharge agreement, however, was associated with 59% lower odds of falling after adjustment for other factors.
Participants, who were discharged to the location with the services recommended by their physical therapist, were less likely to fall. Tinetti POMA and AM-PAC IMSF scores did not discriminate well participants who would fall.
Findings in this study inform those involved in discharge planning on the value of implementing physical therapist recommendations in reducing fall risk after hospital discharge.
If physical therapist discharge recommendations are implemented, patients are less likely to fall during the month after hospital discharge. Balance and mobility test scores may provide therapists valuable information, but they are limited in their ability to identify who will fall after discharge.
Objectives
Gallbladder cancer (GBC) is a rare, poor‐prognosis cancer with unique demographics, comorbidities and a paucity of research. This study investigated inpatient palliative care and its ...associations with demographics, comorbidities (e.g., obesity), length of stay and hospital charges in GBC in US hospitals (2007–2016).
Methods
Data were extracted from the National Inpatient Sample (NIS) database that contains deidentified clinical and nonclinical information for each hospitalisation. Inpatient palliative care utilisation was identified using the International Classification of Diseases (ICD‐9 and ICD‐10) codes (V66.7 and Z51.5). Generalised regression analysis was conducted with adjustment for variations in predictors.
Results
Of the 4921 reported GBC hospitalizations, only 10.3% encountered palliative care. Palliative care was associated with reduced hospital charges by $12,405 per hospitalisation (P < 0.0001) with no change in length of stay. Palliative care utilisation increased over time (P = 0.004). It was associated with age >80 years, with more severe disease, and in‐hospital death (P < 0.0001). Obesity had a negative association with palliative care utilisation (P = 0.0029).
Discussion
Our novel study found that obese people were less likely to use palliative care services in GBC. Interventions are needed to increase palliative care consultation in GBC patients, particularly in obese patients.
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BFBNIB, FZAB, GIS, IJS, KILJ, NLZOH, NUK, OILJ, SBCE, SBMB, UL, UM, UPUK
Biomedical studies, such as clinical trials, often require the comparison of measurements from two correlated tests in which each unit of observation is associated with a binary outcome of interest ...via relative risk. The associated confidence interval is crucial because it provides an appreciation of the spectrum of possible values, allowing for a more robust interpretation of relative risk. Of the available confidence interval methods for relative risk, the asymptotic score interval is the most widely recommended for practical use. We propose a modified score interval for relative risk and we also extend an existing nonparametric U-statistic-based confidence interval to relative risk. In addition, we theoretically prove that the original asymptotic score interval is equivalent to the constrained maximum likelihood-based interval proposed by Nam and Blackwelder. Two clinically relevant oncology trials are used to demonstrate the real-world performance of our methods. The finite sample properties of the new approaches, the current standard of practice, and other alternatives are studied via extensive simulation studies. We show that, as the strength of correlation increases, when the sample size is not too large the new score-based intervals outperform the existing intervals in terms of coverage probability. Moreover, our results indicate that the new nonparametric interval provides the coverage that most consistently meets or exceeds the nominal coverage probability.
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EMUNI, FIS, FZAB, GEOZS, GIS, IJS, IMTLJ, KILJ, KISLJ, MFDPS, NLZOH, NUK, OILJ, PNG, SAZU, SBCE, SBJE, SBMB, SBNM, UKNU, UL, UM, UPUK, VKSCE, ZAGLJ
When multiple influential covariates need to be balanced during a clinical trial, stratified blocked randomization and covariate-adaptive randomization procedures are frequently used in trials to ...prevent bias and enhance the validity of data analysis results. The latter approach is increasingly used in practice for a study with multiple covariates and limited sample sizes. Among a group of these approaches, the covariate-adaptive procedures proposed by Pocock and Simon are straightforward to be utilized in practice. We aim to investigate the optimal design parameters for the patient treatment assignment probability of their developed three methods. In addition, we seek to answer the question related to the randomization performance when additional covariates are added to the existing randomization procedure. We conducted extensive simulation studies to address these practically important questions.
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DOBA, IZUM, KILJ, NUK, PILJ, PNG, SAZU, SIK, UILJ, UKNU, UL, UM, UPUK
This research was motivated by a clinical trial design for a cognitive study. The pilot study was a matched‐pairs design where some data are missing, specifically the missing data coming at the end ...of the study. Existing approaches to determine sample size are all based on asymptotic approaches (e.g., the generalized estimating equation (GEE) approach). When the sample size in a clinical trial is small to medium, these asymptotic approaches may not be appropriate for use due to the unsatisfactory Type I and II error rates. For this reason, we consider the exact unconditional approach to compute the sample size for a matched‐pairs study with incomplete data. Recommendations are made for each possible missingness pattern by comparing the exact sample sizes based on three commonly used test statistics, with the existing sample size calculation based on the GEE approach. An example from a real surgeon‐reviewers study is used to illustrate the application of the exact sample size calculation in study designs.
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BFBNIB, DOBA, FZAB, GIS, IJS, IZUM, KILJ, NLZOH, NUK, OILJ, PILJ, PNG, SAZU, SBCE, SBMB, SIK, UILJ, UKNU, UL, UM, UPUK