Category: Statistics

Circ Heart Fail. 2024 Oct 22:e012067. doi: 10.1161/CIRCHEARTFAILURE.124.012067. Online ahead of print.

ABSTRACT

BACKGROUND: Pulmonary arterial hypertension (PAH) is a disease of progressive right ventricular (RV) failure with high morbidity and mortality. Our goal is to investigate proteomic features and pathways associated with RV-focused outcomes including mortality, RV dilation, and NT-proBNP (N-terminal pro-B-type natriuretic peptide) in PAH.

METHODS: Participants in a single-institution cohort with 3 years of follow-up underwent proteomic profiling of their plasma using 7288 aptamers (targeting 6467 unique human proteins). Partial least squares discriminant analysis was performed to assess global protein variation associated with mortality, RV dilation, and NT-proBNP levels. Differentially abundant proteins and enriched pathways associated with outcomes were identified following baseline adjustments. RV vulnerability models estimated associations for individuals with similar afterload following adjustment for pulmonary vascular resistance.

RESULTS: A total of 117 participants with PAH were included. Partial least squares discriminant analysis of the proteome showed clear separation between survivors and nonsurvivors, participants with dilated versus nondilated RVs, and across NT-proBNP levels. Proteins and pathways involving the ECM (extracellular matrix) were upregulated in participants who died during follow-up, those with severe RV dilation, and those with higher levels of NT-proBNP. Pulmonary vascular resistance adjustment reinforced the importance of ECM proteins in the association with RV vulnerability, independent of afterload. These findings were confirmed in independent PAH cohorts with available plasma proteomics and RV tissue gene and protein expression.

CONCLUSIONS: Distinct plasma proteomic profiles are associated with mortality, RV dilation, and NT-proBNP in PAH. Proteins and pathways governing tissue remodeling are strongly associated with poor outcomes, may mediate RV vulnerability to right heart failure, and represent promising candidates as biomarkers and potential therapeutic targets.

PMID:39435559 | DOI:10.1161/CIRCHEARTFAILURE.124.012067

Stroke. 2024 Oct 22. doi: 10.1161/STROKEAHA.124.046125. Online ahead of print.

ABSTRACT

Designing a clinical trial to evaluate the efficacy of an intervention is often complicated by uncertainty over aspects of the study population, potential treatment effects, most relevant outcomes, dropouts, and other factors. However, once participants begin to be enrolled and partial trial data become available, this level of uncertainty is reduced. Adaptive clinical trials are designed to take advantage of the accumulating data during the conduct of a trial to make changes according to prespecified decision rules to increase the likelihood of success or statistical efficiency. Common adaptive rules address early stopping for benefit or futility, sample size reestimation, adding or dropping treatment arms or altering randomization ratios, and changing the eligibility criteria to focus on responder patient subgroups. Adaptive clinical trials are gaining popularity for clinical stroke research. We provide an overview of the methods, practical considerations, challenges and limitations, and potential future role of adaptive clinical trials in advancing knowledge and practice in stroke.

PMID:39435555 | DOI:10.1161/STROKEAHA.124.046125

J Int Med Res. 2024 Oct;52(10):3000605241287021. doi: 10.1177/03000605241287021.

ABSTRACT

OBJECTIVE: This study was performed to determine the dimensions of the sella turcica (ST) in relation to sex, age groups, and sphenoid sinus (SS) pneumatization patterns in a cohort of Iraqi individuals.

METHODS: This cross-sectional study analyzed computed tomography images of 99 individuals (53 men, 46 women) aged 24 to 59 years to measure ST dimensions, including length, width, height, depth, and surface area. SS pneumatization was classified by sex and age. We also examined the relationship between ST size and SS pneumatization patterns.

RESULTS: Women exhibited a significantly greater mean ST depth (9.37 mm) than men (8.19 mm). The ST diameter was significantly larger in the middle-aged group (13.1 mm) than in the young adult group (11.83 mm). The ST area showed a highly significant difference between sexes. The sellar pneumatization pattern was the most common, whereas the conchal pattern had the lowest prevalence. A significant correlation was observed between ST size and SS pneumatization.

CONCLUSION: The ST depth and diameter showed statistically significant differences across sex and age groups, respectively. The variability in SS pneumatization patterns underscores the importance of considering pneumatization types during surgical planning to minimize the risk of iatrogenic injury and improve treatment outcomes.

PMID:39435554 | DOI:10.1177/03000605241287021

Stroke. 2024 Oct 22. doi: 10.1161/STROKEAHA.123.044144. Online ahead of print.

ABSTRACT

While the majority of stroke researchers use frequentist statistics to analyze and present their data, Bayesian statistics are becoming more and more prevalent in stroke research. As opposed to frequentist approaches, which are based on the probability that data equal specific values given underlying unknown parameters, Bayesian approaches are based on the probability that parameters equal specific values given observed data and prior beliefs. The Bayesian paradigm allows researchers to update their beliefs with observed data to provide probabilistic interpretations of key parameters, for example, the probability that a treatment is effective. In this review, we outline the basic concepts of Bayesian statistics as they apply to stroke trials, compare them to the frequentist approach using exemplary data from a randomized trial, and explain how a Bayesian analysis is conducted and interpreted.

PMID:39435547 | DOI:10.1161/STROKEAHA.123.044144

J Comput Biol. 2024 Oct 22. doi: 10.1089/cmb.2024.0600. Online ahead of print.

ABSTRACT

Fleming-Viot diffusions are widely used stochastic models for population dynamics that extend the celebrated Wright-Fisher diffusions. They describe the temporal evolution of the relative frequencies of the allelic types in an ideally infinite panmictic population, whose individuals undergo random genetic drift and at birth can mutate to a new allelic type drawn from a possibly infinite potential pool, independently of their parent. Recently, Bayesian nonparametric inference has been considered for this model when a finite sample of individuals is drawn from the population at several discrete time points. Previous works have fully described the relevant estimators for this problem, but current software is available only for the Wright-Fisher finite-dimensional case. Here, we provide software for the general case, overcoming some nontrivial computational challenges posed by this setting. The R package FVDDPpkg efficiently approximates the filtering and smoothing distribution for Fleming-Viot diffusions, given finite samples of individuals collected at different times. A suitable Monte Carlo approximation is also introduced in order to reduce the computational cost.

PMID:39435537 | DOI:10.1089/cmb.2024.0600

Stroke. 2024 Oct 22. doi: 10.1161/STROKEAHA.124.046129. Online ahead of print.

ABSTRACT

Evidence generated from randomized clinical trials (RCTs) plays an indispensable role in advancing clinical stroke care. Although the number of stroke-related RCTs published every year has grown exponentially over the past 25 years, the execution and completion of RCTs, particularly those conducted in a hyperacute setting, have grown more complicated and challenging over the years. In addition to the practical challenges associated with conducting a clinical trial, like obtaining human subjects approval, identifying clinical sites, training trial personnel, and enrolling the target number of patients within the available funding and timeline, the complexity of contemporary RCT designs and analyses has become much more exacting. It is no longer sufficient to have a decent understanding of the 2-arm, placebo-controlled RCT, combined with a rudimentary grasp of the P value; things are now much more complicated. Innovations in trial design and analysis, including adaptive, Bayesian, platform, and noninferiority designs, have occurred to address the problems of poor trial efficiency. However, these advances require the end user to have a much greater level of understanding regarding the rationale, conduct, analysis, and interpretation of each design. While these newer designs seek greater efficiency, there are inevitably tradeoffs that need to be understood. In this month’s edition of Stroke, we introduce a new series designed to help fill in these knowledge gaps. Over the next few months, 4 papers will be published that address major design innovations (adaptive, Bayesian, platform, and noninferiority) with the aim of illustrating how these approaches can make trials more efficient (where efficiency is defined as getting to the right answer, sooner, with a potentially lower sample size). In addition to introducing this series, this current article also reviews traditional hypothesis testing and the common misinterpretations of the P value; fortunately, new philosophical schools of inference are beginning to vanquish the overreliance on the P value. We are excited about the opportunity to educate the Stroke readership about these new trial designs and the profound implications that they bring.

PMID:39435534 | DOI:10.1161/STROKEAHA.124.046129

J Sci Food Agric. 2024 Oct 22. doi: 10.1002/jsfa.13978. Online ahead of print.

ABSTRACT

BACKGROUND: Fortified wine is an important category in the wine world with very famous wines such as Porto or Jerez-wine type. The quality of fortified wines increased significantly with barrel aging not only because of a long oxidation process, but also because, in Porto wines such as Ruby or Vintage styles, the long period in bottle permits their fining. Reducing the time of oxidation can favor the development of this technique even for less known sweet wines, making them good quality and less expensive. In the present study, we have used Gamay red variety subjected to postharvest controlled dehydration at 20-22 °C and 70-75% relative humidity with an airflow of 1 m s^-1. Then the grapes were pressed, and alcohol was added to the must up to an alcohol content of 15.85% (mystelle-type wine). The mass was split into six glass jars, three were oxygenated (OX) and three not (Control), and the oxygenation lasted 62 days.

RESULTS: Wine that was oxygenated had a slightly higher volatile acidity, lower alcohol content (13.00%), and lower anthocyanins and polyphenols content. In term of volatile organic compounds (VOCs), the Control wine had a higher content of alcohols, whereas the OX sample had a higher content of lactones, furans and esters. Sensory evaluation confirmed the VOCs analysis; the two wines had a statistically different profile depending on the oxidation treatment. In general, OX wine was more appreciated in terms of visual attractiveness, taste and olfactory pleasantness.

CONCLUSION: In conclusion, the technique described in the present study could be a valid alternative to traditional aging of fortified sweet wines, reducing time and costs. © 2024 Society of Chemical Industry.

PMID:39435533 | DOI:10.1002/jsfa.13978

J Clin Periodontol. 2024 Oct 22. doi: 10.1111/jcpe.14081. Online ahead of print.

ABSTRACT

AIM: While the importance of gingival aesthetics is increasingly recognized, a questionnaire quantifying its psychosocial impact is still lacking. We have adapted the validated Psychosocial Impact of Dental Aesthetics Questionnaire (PIDAQ) for the gingival context. This study’s aim was to develop a new Psychosocial Impact of Gingival Aesthetics Questionnaire (PIGAQ) and test its validity for the general population in Spain, exploring the underlying dimensions of the construct assessed.

MATERIALS AND METHODS: The PIDAQ was adapted to address gingival aesthetics. PIGAQ (23 items) was completed by 200 patients aged from 18 to 85 years, and socio-demographic variables were recorded. The four-dimensional structure of the PIDAQ was respected, adapting the subscales to focus on the gingiva. Descriptive analysis, assessments of reliability and validity and an exploratory factor analysis were performed.

RESULTS: The revised PIGAQ (now containing 20 items) has a Cronbach’s alpha coefficient of 0.88 with four underlying factors explaining 61.5% of the total variance. Three items (SI-1, PI-2 and PI-5) were eliminated because of unacceptable fit indices. The Cronbach’s alpha coefficients for the subscales are as follows: Gingival Self-Confidence, 0.85; Social Impact, 0.84; Psychological Impact, 0.79; and Aesthetic Concern, 0.76.

CONCLUSION: PIGAQ is a reliable and potentially valid instrument for assessing psychosocial perceptions of gingival aesthetics. The scale was shown to have a satisfactory factor structure and level of internal consistency.

PMID:39435532 | DOI:10.1111/jcpe.14081

Thorac Cancer. 2024 Oct 22. doi: 10.1111/1759-7714.15471. Online ahead of print.

ABSTRACT

BACKGROUND: Patients with non-small-cell lung cancer (NSCLC) receiving immunotherapy face a potential risk of developing checkpoint inhibitor-related pneumonitis (CIP). However, there is no clear understanding of the specific link between interstitial lung abnormality (ILA) and CIP in patients with small-cell lung cancer (SCLC). In addition, the prognosis of SCLC patients with ILA who receive chemoimmunotherapy is uncertain. Our study aimed to investigate the effect of ILA on the occurrence of CIP in SCLC patients receiving first-line chemoimmunotherapy and to assess its relationship with prognosis.

METHODS: We conducted a retrospective analysis of SCLC patients who received chemoimmunotherapy as a first-line treatment between January 2018 and April 2024. The diagnosis of ILA was assessed by two experienced pulmonologists based on pretreatment chest computed tomography images. We investigated independent risk factors for CIP using logistic regression analysis and factors affecting PFS and OS using Cox regression analysis.

RESULTS: A total of 128 patients with SCLC were included in the study. ILA was present in 41 patients (32.03%), and CIP occurred in 16 patients (12.50%). In multivariate logistic regression analysis, previous ILA (OR, 5.419; 95% CI, 1.574-18.652; p = 0.007) and thoracic radiation therapy (TRT) (OR, 5.259; 95% CI, 1.506-18.365; p = 0.009) were independent risk factors for CIP. ILA (HR, 2.083; 95% CI, 1.179-3.681; p = 0.012) and LDH (HR, 1.002; 95% CI, 1.001-1.002; p < 0.001) were statistically significant for increased mortality risk in multivariate Cox regression analysis.

CONCLUSIONS: In SCLC patients receiving first-line chemoimmunotherapy, baseline ILA is a risk factor for CIP and is associated with poorer prognosis.

PMID:39435523 | DOI:10.1111/1759-7714.15471

J Korean Med Sci. 2024 Oct 21;39(40):e267. doi: 10.3346/jkms.2024.39.e267.

ABSTRACT

BACKGROUND: The persistent coronavirus disease 2019 (COVID-19) pandemic has had direct and indirect effects on mortality, making it essential to analyze excess mortality to fully understand the impact of the pandemic. In this study, we constructed a mathematical model using number of deaths from Statistics Korea and analyzed excess mortality between 2020 and 2022 according to age, sex, and dominant severe acute respiratory syndrome coronavirus 2 variant period.

METHODS: Number of all-cause deaths between 2010 and 2022 were obtained from the annual cause-of-death statistics provided by Statistics Korea. COVID-19 mortality data were acquired from the Korea Disease Control and Prevention Agency. A multivariate linear regression model with seasonal effect, stratified by sex and age, was used to estimate the number of deaths in the absence of COVID-19. The estimated excess mortality rate was calculated.

RESULTS: Excess mortality was not significant between January 2020 and October 2021. However, it started to increase monthly from November 2021 and reached its highest point during the omicron-dominant period. Specifically, in March and April 2022, during the omicron BA.1/BA.2-dominant period, the estimated median values for excess mortality were the highest at 17,634 and 11,379, respectively. Both COVID-19-related deaths and excess mortality increased with age. A notable increase in excess mortality was observed in individuals aged ≥ 65 years. In the context of excess mortality per 100,000 population based on the estimated median values in March 2022, the highest numbers were found among males and females aged ≥ 85 years at 1,048 and 910, respectively.

CONCLUSION: This study revealed that the prolonged COVID-19 pandemic coupled with its high transmissibility not only increased COVID-19-related deaths but also had a significant impact on overall mortality rates, especially in the elderly. Therefore, it is crucial to concentrate healthcare resources and services on the elderly and ensure continued access to healthcare services during pandemics. Establishing an excess mortality monitoring system in the early stages of a pandemic is necessary to understand the impact of infectious diseases on mortality and effectively evaluate pandemic response policies.

PMID:39435517 | DOI:10.3346/jkms.2024.39.e267