Category: Statistics

Int J Biostat. 2025 May 23. doi: 10.1515/ijb-2024-0021. Online ahead of print.

ABSTRACT

With the increasing complexity of data, researchers in various fields have become increasingly interested in estimating the causal effect of a matrix exposure, which involves complex multivariate treatments, on an outcome. Balancing covariates for the matrix exposure is essential to achieve this goal. While exact balancing and approximate balancing methods have been proposed for multiple balancing constraints, dealing with a matrix treatment introduces a large number of constraints, making it challenging to achieve exact balance or select suitable threshold parameters for approximate balancing methods. To address this challenge, the weighted Euclidean balancing method is proposed, which offers an approximate balance of covariates from an overall perspective. In this study, both parametric and nonparametric methods for estimating the causal effect of a matrix treatment is proposed, along with providing theoretical properties of the two estimations. To validate the effectiveness of our approach, extensive simulation results demonstrate that the proposed method outperforms alternative approaches across various scenarios. Finally, we apply the method to analyze the causal impact of the omics variables on the drug sensitivity of Vandetanib. The results indicate that EGFR CNV has a significant positive causal effect on Vandetanib efficacy, whereas EGFR methylation exerts a significant negative causal effect.

PMID:40421507 | DOI:10.1515/ijb-2024-0021

Nanoscale. 2025 May 27. doi: 10.1039/d5nr00016e. Online ahead of print.

ABSTRACT

Liposomal drug delivery systems have shown promising potential to improve drug delivery in several aspects. Precise in vitro characterization of formulated liposomes is vital to achieve proper in vivo function. In particular, in vitro release testing of liposomes offers crucial insights for predicting in vivo drug release behavior, guiding the development of more effective liposomal formulations. Herein, random forest (RF)- and XGboost-genetic algorithms were implemented to establish a model to predict release profiles of liposomes based on critical characteristics of carriers, cargoes, and release media. The models were trained with a dataset consisting of release parameters of 203 different liposomal formulations extracted from the literature, divided into three categories: all cargoes, therapeutics with log P < 1, and log P > 1. The SHapley additive exPlanations (SHAP) approach was used to determine how data contributes to the model prediction. A mean squared error of 1.648 highlighted the capability of the genetic algorithm to optimize the hyperparameters of the tree-based models. Furthermore, the developed models demonstrated high accuracy as evidenced by loss functions and statistical metrics such as root mean squared error, mean absolute error, and R-squared. Our results shed light on the distinct influence of phase transition temperature, drug concentration, log P, water solubility, molecular weight, vesicle size, cholesterol to phospholipid molar ratio, and surfactant concentration in the release medium on drug release at different phases of the release profiles. The predictive models developed in this study can facilitate the design of an ideal liposomal formulation for any desired release profile.

PMID:40421505 | DOI:10.1039/d5nr00016e

Minerva Cardiol Angiol. 2025 May 27. doi: 10.23736/S2724-5683.25.06719-5. Online ahead of print.

ABSTRACT

BACKGROUND: The aging population in the USA has led to a concomitant rise in the prevalence of vascular dementia (VaD), yet there remains a paucity of investigation into mortality trends associated with VaD among adults.

METHODS: This cross-sectional analysis utilized death certificate data from the Centers for Disease Control and Prevention’s WONDER database. VaD-associated mortality was identified using the International Statistical Classification of Diseases and Related Health Problems-10th revision (ICD-10) code F01. Crude, and age-adjusted VaD-associated mortality rates per 100,000 and their corresponding 95% confidence intervals (CI) were computed. Age-adjusted mortality rates (AAMRs) were standardized to the 2000 US census population.

RESULTS: From 2005 to 2020, there were 375,575 deaths attributed to VaD among older adults. We observed a gradual increase in AAMR (APC: 3.70, 95% CI [-4.14, 5.21]) from 2005 to 2015, succeeded by a pronounced escalation (APC: 9.07, 95% CI [6.09, 17.62]) until 2020. The highest AAMR was noted in the West (17.65, 95% CI [17.55, 17.76]), followed by the Midwest (AAMR: 12.66, 95% CI [12.58, 12.75]), the South (AAMR: 12.60, 95% CI [12.54, 12.67]), and the Northeast (AAMR: 8.60, 95% CI [8.53, 8.68]). Metropolitan areas exhibited higher AAMRs (10.9, 95% CI [10.8, 11.0]) compared to non-metropolitan areas (8.1, 95% CI [8.00, 8.3]). Among age groups, individuals aged 75-85 and older showed the highest overall AAMR (99.80, 95% CI [99.47, 100.14]). In addition, non-Hispanic Black or African-American subset of the population showed the highest overall AAMR (8.12, [95% CI: 8.03, 8.20]).

CONCLUSIONS: Our findings underscore the imperative for targeted public health interventions aimed at addressing regional disparities and age-specific vulnerabilities to mitigate the mounting burden of VaD-related mortality.

PMID:40421495 | DOI:10.23736/S2724-5683.25.06719-5

Int J Nurs Educ Scholarsh. 2025 May 19;22(1). doi: 10.1515/ijnes-2024-0077. eCollection 2025 Jan 1.

ABSTRACT

OBJECTIVES: Nursing education commonly includes aspects of cognitive, psychomotor, and affective learning. Subsequently, the evaluation of student affective learning has been recognized as an important aspect within educational processes. Affective learning is crucial to professional nursing role development. However, a gap in the literature exists in measuring and evaluating affective learning in nursing students. Using the taxonomy developed by Krathwohl, Bloom, and Masia (1964. Taxonomy of educational objectives, handbook II: affective domain. Philadelphia: David McKay Company, Inc.), faculty developed a tool for students to rate their perceived level of affective learning.

METHODS: Faculty launched a quality improvement project to evaluate the accuracy of student self-assessments of affective learning at the end of their first nursing course.

RESULTS: Student and expert identification of the level of affective learning were not statistically different, which demonstrated that students accurately identified their affective level. The sample demonstrated competency through the first level of affective learning (i.e., receiving).

CONCLUSIONS: Cultivating professional values can significantly impact the lives of patients and communities. Use of the tool may lead to a deeper understanding of student nurse development of affective competencies.

PMID:40421479 | DOI:10.1515/ijnes-2024-0077

J Hand Surg Eur Vol. 2025 May 27:17531934251342419. doi: 10.1177/17531934251342419. Online ahead of print.

ABSTRACT

The purpose of this study was to investigate the epidemiology of flexor tendon injuries and operative complications post-repair on a national scale. Using Hospital Episode Statistics data we identified a total of 91,239 patients in England who underwent a flexor tendon repair in the 20-year period between 1 April 1998 and 31 March 2018. Individuals were more likely to be male (74.6%) and of lower socioeconomic status. Median age was 31 years and patients had few medical comorbidities. After primary repair, 3.1% of patients required a revision repair within 90 days and 2.8% tenolysis within two years. Complications other than rupture, adhesions and wound site infection were rare. This study has shown that flexor tendon repair is primarily a procedure of working-age men, with rupture and adhesions the most common complication. The complication rate is less than previously reported in the literature.Level of evidence: III.

PMID:40421478 | DOI:10.1177/17531934251342419

Neurodegener Dis Manag. 2025 May 27:1-8. doi: 10.1080/17582024.2025.2510145. Online ahead of print.

ABSTRACT

BACKGROUND: While neuropsychiatric symptoms are common in Huntington’s Disease (HD), there is a dearth of evidence about the effectiveness of psychotropic medication for treating behavioral and cognitive symptoms. This article systematically reviews and aggregates the evidence of the effects of antidepressants on individuals with early HD.

METHODS: A systematic review and random-effects meta-analysis of RCTs comparing antidepressants to placebo in individuals with HD was performed, with a focus on outcomes of executive functioning, functional capacity, mood, motor function, and adverse events.

RESULTS: A total of 4 studies with 123 of patients with early HD, of whom a total of 63 (51.2%) received an antidepressant, were identified in our search. In our pooled analysis, a modest but statistically significant improvement in mood resulted from antidepressant treatment in HD (OR -1.22; 95% CI -2.16,-0.27; p = 0.02). No significant differences in the other outcomes of interest were found following antidepressant exposure.

CONCLUSIONS: Our investigation suggests that antidepressant use may modestly improve mood, but not ameliorate functional, cognitive, or movement-related symptoms in mild HD. Further studies involving novel agents, larger samples, and longer follow-up times are needed to better characterize the effect of antidepressants on neuropsychiatric symptoms in HD.

PMID:40421472 | DOI:10.1080/17582024.2025.2510145

BMC Cancer. 2025 May 26;25(1):943. doi: 10.1186/s12885-025-14323-5.

ABSTRACT

BACKGROUND: Numerous studies suggest that young adult cancer survivors (YA-CS) experience unmet needs regarding informational exchange about their disease and emotional support from peers. Currently, there is a lack of evaluated peer support interventions in Germany. This study aimed to evaluate the effectiveness of “Peer2Me”, a three-month one-to-one peer mentoring program, designed to improve self-efficacy among YA-CS.

METHODS: This study conducted a bi-center comprehensive cohort design and enrolled YA-CS (18-39 years old) undergoing acute treatment in Leipzig and Hamburg (Germany) across all tumour entities diagnosed within the last six months. YA-CS were allocated by preference to the intervention (IG, receiving peer mentoring) and comparison group (CG, care as usual). Following mentor training, tandems were matched by diagnosis, age, and gender. YA-CS completed questionnaires at baseline (t1), post-intervention (3 months later, t2) and three months post-intervention (t3). The primary outcome was self-efficacy measured with the Generalized Self-Efficacy Scale (GSES) and the Cancer Behaviour Inventory (CBI-B). Statistical analyses included mixed-design ANOVA and ANCOVA, controlling for baseline scores.

RESULTS: Out of 274 eligible YA-CS, a total of 106 YA-CS (IG: n = 77, CG: n = 29) completed the study. Two-thirds of YA-CS (66.7%) expressed a clear preference to be allocated to the IG. Baseline differences in coping behaviors were noted, with the IG demonstrating lower scores, suggesting a higher need for support. The ANCOVA revealed no group effects for the change of mean GSES scores from t1 to t2 (p =.897) or from t1 to t3 (p =.779). Also, no significant differences in the improvement of mean CBI-B scores between groups could be found from t1 to t2 (p =.903) or from t1 to t3 (p =.995).

CONCLUSIONS: The “Peer2Me” program did not demonstrate a significant effect on improving self-efficacy among YA-CS during acute treatment. Although interest in peer mentoring was high, the intervention showed no measurable benefit in comparison with standard care. While the need for support was evident, further research is required to optimize peer interventions for this group.

TRIAL REGISTRATION: The study was retrospectively registered on February 4, 2022 at clinicaltrials.gov (NCT05336318).

PMID:40420294 | DOI:10.1186/s12885-025-14323-5

BMC Psychol. 2025 May 27;13(1):564. doi: 10.1186/s40359-025-02897-5.

ABSTRACT

BACKGROUND: While belief in a just world has been extensively studied, the object of this belief still requires further specification. This study distinguishes two sources of uncertainty in future returns-nature and other people-and investigates whether belief in a just world is specifically directed toward human-sourced uncertainty but not nature-sourced uncertainty.

METHODS: To test this hypothesis, an experiment was conducted in which participants decided whether to make an investment based on their beliefs about the return they would receive from another player. The identity of the other player was varied (computer or human, representing nature-sourced uncertainty and human-sourced uncertainty, respectively), and participants’ level of belief in a just world was manipulated using a priming method. Multiple statistical analyses were conducted to examine the differences in investment behavior and expected returns between computer and human conditions.

RESULTS: When interacting with human players, participants in the just-world priming condition showed significantly higher investment rates and expected returns compared to those in the unjust-world priming condition. In contrast, when interacting with computer players, the differences between just-world and unjust-world conditions were non-significant, both for investment rates and expected returns. Mediation analysis further revealed that expected return mediated the relationship between priming condition and investment behavior in the human player condition.

CONCLUSION: These findings demonstrate that belief in a just world influences decision-making when interacting with human players but not with computer players, supporting our hypothesis that belief in a just world mainly targets human-sourced uncertainty but not nature-sourced uncertainty. This research advances our theoretical understanding of belief in a just world and contributes to our understanding of its functions for both individuals and society.

PMID:40420290 | DOI:10.1186/s40359-025-02897-5

Crit Care. 2025 May 26;29(1):214. doi: 10.1186/s13054-025-05452-1.

ABSTRACT

BACKGROUND: The optimal time for initiating kidney replacement therapy (KRT) in acute kidney injury (AKI) has been extensively studied in recent years. In contrast, there are currently insufficient data on the best time to discontinue KRT. One diagnostic option to unmask tubular reserve and indirectly estimate the glomerular filtration rate is the furosemide stress test (FST).

METHODS: We conducted a prospective, observational single-center trial. A FST was carried out in patients who developed spontaneous diuresis (SD) during ongoing KRT with a urine output of at least 400 ml in 24 h without any diuretic therapy. A positive FST was defined with urine output > 200 ml within 2 h following intravenous furosemide application. Follow-up was performed for 7 days and the need to restart KRT was assessed daily.

RESULTS: After 100 patients were enrolled in the trial, 98 patients were eligible for further evaluation. 76 patients were FST-positive, while 22 patients were FST-negative. Resumption of KRT within the 7-day follow-up was required in only 14.5% of the FST-positive, but 72.7% of the FST-negative patients (p < 0.001). The urine output after FST was also significantly associated with successful release from KRT (AUC 0.87; p < 0.001).

CONCLUSIONS: In critically ill patients with recovery of SD > 400ml/d during ongoing KRT, the FST helps to identify patients who can be successfully liberated from KRT. By detecting the tubular reserve using FST, the possibility of short-term kidney recovery after AKI can be estimated.

TRIAL REGISTRATION: German Clinical Trials Registry (DRKS00030560); date of registration 18/11/2022. https://drks.de/search/de/trial/DRKS00030560 .

PMID:40420285 | DOI:10.1186/s13054-025-05452-1

BMC Med Res Methodol. 2025 May 26;25(1):142. doi: 10.1186/s12874-025-02596-0.

ABSTRACT

BACKGROUND: Evaluating recurrent events within a time-to-event analysis framework effectively utilizes all relevant information to address the clinical question of interest fully and has certain advantages in randomized controlled trials (RCTs). However, the Prentice, Williams, and Peterson (PWP) model disrupts the randomness of the risk set for subsequent recurrent events other than the first and consequently introduces bias in estimating effects. This study aimed to propose a weighted PWP model, evaluate its statistical performance, and assess the potential consequences of using common practices when each recurrence has different baseline hazard functions.

METHODS: We proposed adjusting the estimate of treatment effect through a weighting strategy that constructed a virtual population balanced between groups in each risk set. A simulation study was carried out. The characteristic of the simulation data was the baseline hazard changed with the number of events. The proposed weighted PWP model was compared with current methods, including Cox for time-to-first-event, Poisson, negative binomial (NB), Andersen-Gill (AG), Lin-Wei-Yang-Ying (LWYY), and PWP models. Model performance was evaluated by bias, type I error rates, and statistical power. All models were applied to a real case from a randomization trial of Chemoprophylaxis treatment for Recurrent Stage I Bladder Tumors.

RESULTS: The results showed that the proposed weighted PWP model performed best with the lowest bias and highest statistical power. However, other models, including the Cox for time-to-first-event, Poisson, NB, AG, LWYY, and PWP models, all showed different degrees of bias and inflated type I error rates or low statistical power in the case of the baseline hazard changed with the number of events. Covariate adjustment via outcome regression can lead to inflated type I error rates. When the number of recurrent events was restricted, all weighting strategies yielded stable and nearly consistent results.

CONCLUSIONS: Recurrent event data should be analyzed with caution. The proposed methods may be generalized to model recurrent events. Our findings serve as an important clarification of how to deal with collider bias in the PWP model in RCTs.

PMID:40420279 | DOI:10.1186/s12874-025-02596-0