Tag: statistics

Zhonghua Liu Xing Bing Xue Za Zhi. 2026 Aug 10;47(8):1395-1400. doi: 10.3760/cma.j.cn112338-20260123-00067.

ABSTRACT

With the rapid growth of precision medicine and the increasing demand for novel drug development, traditional development strategies and trial designs are no longer adequate for contemporary clinical research, prompting regulatory agencies, industry, and academia to explore more flexible and efficient approaches. Bayesian methods are emerging as a vital statistical tool for innovative clinical trial design due to their flexibility and strong interpretability. In critical fields of clinical trials such as dose finding and optimization, adaptive design, external information borrowing, and master protocol design, Bayesian methods have demonstrated unique value distinct from traditional frequentist approaches. This paper systematically reviews common Bayesian methods in innovative clinical trial design and discusses crucial considerations for their application, aiming to guide the standardized, rational use of Bayesian methods.

PMID:42618473 | DOI:10.3760/cma.j.cn112338-20260123-00067

Zhonghua Liu Xing Bing Xue Za Zhi. 2026 Aug 10;47(8):1389-1394. doi: 10.3760/cma.j.cn112338-20260123-00066.

ABSTRACT

The prior distribution refers to a probabilistic description of the uncertainty of unknown parameters by researchers before observing sample data. It is a prerequisite and key step in Bayesian statistical methods, as well as a major challenge encountered in practical applications. Based on four dimensions-information content, probabilistic characteristics, relationship with the likelihood function, and structural hierarchy-this paper comprehensively sorts out the types of prior distributions, illustrates the impact of prior distributions on results through a combination of theory and case studies, and discusses in depth the key issues that need to be prioritized when selecting prior distributions. In addition, this paper elaborates on the importance and implementation methods of sensitivity analysis for prior distributions and introduces key points for reporting Bayesian statistical inference results. Only by rationally selecting prior distributions and standardizing Bayesian statistical inference can the scientific value of Bayesian statistics be fully realized.

PMID:42618472 | DOI:10.3760/cma.j.cn112338-20260123-00066

Zhonghua Liu Xing Bing Xue Za Zhi. 2026 Aug 10;47(8):1383-1388. doi: 10.3760/cma.j.cn112338-20260123-00064.

ABSTRACT

Bayesian statistics dates back to the 18th century. Its development was once hindered by computational complexity. However, as computing methods and technologies have advanced, Bayesian statistics has demonstrated its advantages across many research scenarios, including small-population studies, information borrowing, complex and innovative designs, and dynamic decision-making. Recently, regulatory authorities in China, the United States, and Europe have issued guidelines, acknowledging the role of Bayesian statistics in new drug research and development. This paper introduced the basic concepts, theoretical foundations, and inference frameworks of Bayesian statistics. Bayesian statistics uses Bayes’ theorem to combine prior information with sample data to derive the posterior distribution, which serves as the basis for Bayesian inference and decision-making. Unlike the traditional frequentist approach, which focuses on “analyzing only the data from the current study”, the Bayesian approach aims to “integrate and use previous information together with current data,” thereby forming a statistical decision-making approach that continuously accumulates and makes dynamic decision-making. The paper also discussed the strengths and weaknesses of the frequentist and the Bayesian, as well as their respective strengths and weaknesses. The authors note that the two inference frameworks will be used in a complementary manner in the future, and Bayesian methods play an invaluable role in advancing innovative medical research.

PMID:42618471 | DOI:10.3760/cma.j.cn112338-20260123-00064

J Visc Surg. 2026 Aug 19:S1878-7886(26)00139-6. doi: 10.1016/j.jviscsurg.2026.07.008. Online ahead of print.

ABSTRACT

INTRODUCTION: Anoperineal fistula (APF) treated by fistulectomy provides the best anatomical results but carries a high risk of anal incontinence. Sphincter reconstruction might improve these outcomes, but limited comparative data are available in the literature. The purpose of this study was to compare the outcomes of fistulotomy plus sphincter reconstruction (FSR) and sphincter-sparing techniques for APF repair.

METHOD: This two-center retrospective study included patients undergoing FSR or ligation of the intersphincteric fistula tract (LIFT) or rectal advancement flap (control group) techniques.

RESULTS: Of the 119 patients included, 95 (79.8%) underwent FSR. There were no statistically significant differences in demographic characteristics between groups. More inter- or trans-sphincteric fistulas were included in the control group, while there were more complex fistulas in the FSR group (P<0.001). The healing rate was 76.2% in the control group compared to 91.6% in the FSR group (P=0.056). No statistically significant difference was found in the overall complication (8.4%) or overall anal incontinence rates (14.7%) (P>0.9). In contrast, the recurrence rate was higher in the control group than in the FSR group (P<0.001). In multivariable analysis, FSR was associated with a lower risk of recurrence compared to the control group (OR=0.16; 95% CI: 0.03-0.77).

CONCLUSION: This study suggests that FSR may be superior to the other techniques in terms of cure without increasing morbidity or the risk of anal incontinence.

PMID:42618469 | DOI:10.1016/j.jviscsurg.2026.07.008

Cardiovasc Revasc Med. 2026 Aug 14:S1553-8389(26)00366-0. doi: 10.1016/j.carrev.2026.08.007. Online ahead of print.

ABSTRACT

BACKGROUND: Aortic root replacement with a prosthetic valve is often required during type A aortic dissection repair (TAAD). With the advent of transcatheter aortic valve replacement (TAVR), the use of bioprosthetic valves has increased.

METHODS: Patients undergoing acute TAAD repair from January 2007 through January 2025 were identified. Those who underwent aortic root replacement with a prosthetic valve were included. Multivariable Cox proportional hazards modeling was performed to evaluate the association between valve type and long-term mortality.

RESULTS: Of the 587 patients who underwent type A aortic dissection repair, 158 patients received a prosthetic valve during root replacement. Mechanical valves (MV) were used in 74 (46.8%) and bioprosthetic valves (BV) in 84 (53.2%). The MV group was younger (51.6 vs. 65.3 years, P < 0.001), with similar rates of malperfusion syndromes between groups. 30-day mortality was 8.2% and comparable between the groups. Reoperation for bleeding was more frequent in the MV group but not statistically different (8.5% vs. 4.8%, P = 0.51). Cox Hazard analysis showed that bioprosthetic valve was independently associated a higher risk for long-term mortality (HR 3.23, 95% CI 1.39-7.69, P < 0.001).

CONCLUSION: While reoperation for bleeding was more observed in patients receiving a mechanical valve, a bioprosthetic valve was associated with a higher risk for long-term mortality. While valve choice may not be available to patients in emergent high acuity situations, it is relevant when surgeons make decisions regarding appropriate prosthesis in patients undergoing surgery for acute TAAD.

PMID:42618428 | DOI:10.1016/j.carrev.2026.08.007

Transplant Proc. 2026 Aug 19:S0041-1345(26)00373-8. doi: 10.1016/j.transproceed.2026.07.028. Online ahead of print.

ABSTRACT

INTRODUCTION: Delayed graft function (DGF) is a common early complication after renal transplantation. We aimed to investigate the clinical efficacy and safety of prolonged intermittent renal replacement therapy (PIRRT) in the treatment of patients with DGF after renal transplantation.

METHODS: Clinical information on 80 patients who underwent renal transplantation in The 924th Hospital of the PLA Joint Logistics Support Force, from August 2021 to April 2024 was collected. According to the different hemodialysis methods, they were divided into: prolonged intermittent renal replacement therapy (PIRRT) and intermittent hemodialysis group (IHD). We investigate the effects of 2 dialysis methods on DGF after renal transplantation by comparing the recovery time of transplanted kidney function, the incidence of dialysis complications, and final blood creatinine level between the 2 groups.

RESULTS: Forty patients of PIRRT group and 40 patients of IHD group conformed to our inclusion criteria. Mean DGF time in the PIRRT group was shorter than in the HDF group(10.45 day2 VS 16.43 days, P < .05, urea clearance (Kt/V) in the PIRRT group was better than in the IHD group (1.58VS1.3, P < .05), the incidence of hemodialysis complications was also low in the PIRRT group and there was a statistical difference in total dialysis time between the 2 groups. After therapy, both groups had considerably decreased mean levels of PCT, CRP, and IL-6 (P < .05). However, the IHD group had significantly higher NGAL levels than the PIRRT group (444.39 ± 182.62∶ 274.45 ± 96.34, P < .05). There was no significant difference in KIM-1 levels between the IHD and PIRRT groups. The recovery of renal function in both groups was assessed, and there was no significant difference in serum creatinine levels before therapy (P = .682 > .05). However, there were significant changes in serum creatinine levels between the 2 groups 1 month and 3 months after therapy (P = .021 and P = .016, respectively), showing that the medication was effective in improving renal function recovery. However, there was no statistically significant difference in serum creatinine levels between the 2 groups 6 months later (P = .125), showing that the recovery of renal function in both groups approached similarity after a given amount of time.

CONCLUSION: PIRRT was used for hemodialysis treatment of patients with DGF after renal transplantation. It can shorten the duration of DGF, reduce renal damage during DGF, and promote the recovery of transplanted kidney function.

PMID:42618415 | DOI:10.1016/j.transproceed.2026.07.028

Inquiry. 2026 Jan-Dec;63:469580261448474. doi: 10.1177/00469580261448474. Epub 2026 Aug 19.

ABSTRACT

IntroductionMenopause is a natural life stage often accompanied by persistent symptoms, such as hot flushes, night sweats, and mood changes that can significantly affect quality of life. While symptom experiences vary across cultures, little is known about menopause-related quality of life among Qatari women. The objective of this study was to measure the association between menopause symptoms and the quality of life of Qatari women.MethodsAn online, self-administered cross-sectional survey was distributed to all Qatari women aged 30-64 attending Primary Health Care Corporation Centers in Qatar for any reason.ResultsA total of 512 women completed the Menopause-Specific Quality of Life questionnaire. The mean (SD) age of respondents was 48.8 (7.28) years, with a median of 50 years. Mean (SD) age at menopause was 48.83 (4.93) years. The most frequently experienced symptoms were feeling tired (88.0%), aching muscles/joints (87.0%), lacking energy (86.0%), decreased physical strength (84.0%), low backache (82.0%) and flatulence (80.9%). Menopause status was significantly related to hot flushes (p< 0.001), night sweats (p< 0.001), accomplishing less (p=0.042), aching muscles/joints (p=0.041), aching back/neck/head (p=0.011), decreased physical strength (p=0.028), vaginal dryness during intimacy (p=0.001), and avoiding intimacy (p=0.002). Psychosocial symptoms were reported by 63.2% of respondents, followed by vasomotor symptoms (66.3%), sexual symptoms (66.4%), and physical symptoms (75.9%). There were statistically significant differences in mean scores of vasomotor, physical, and sexual domains across the menopause groups.ConclusionQatari women experience the onset of menopause at a younger age compared to women from other Arab and high income countries and are mostly bothered by symptoms related to physical discomfort and fatigue. Qatari women experience a high quality of life in the psychosocial domain. To improve care, health systems should prioritize menopause awareness and education, develop culturally sensitive strategies, and integrate these into primary care to enhance the wellbeing of aging women.

PMID:42617154 | DOI:10.1177/00469580261448474

Brief Bioinform. 2026 Jul 3;27(4):bbag446. doi: 10.1093/bib/bbag446.

ABSTRACT

Spatial transcriptomics (ST) has enabled direct interrogation of cell-cell communication (CCC) within intact tissues, providing critical spatial context that is lost in single-cell RNA-sequencing-based inference and allowing more accurate identification of physically plausible and spatially organized interactions. A rapidly expanding community of computational tools has emerged to decode CCC from ST data. Here, we provide a comprehensive review of the conceptual evolution and methodological landscape of spatial CCC inference, classifying existing approaches into two major trajectories. One trajectory, spatial pattern-based methods, assumes CCC events manifest as identifiable spatial patterns, such as colocalization, coordinated spatial signals, or higher-order spatial organization captured by deep learning models. The other trajectory, expression modulation-based approaches, assumes that CCC events influence the transcriptomic state of receiver cells. We systematically dissect their biological assumptions, statistical and deep learning frameworks, strengths, and limitations, and highlight emerging challenges in validation, benchmarking, multimodal integration, and tissue-specific modeling. Finally, we outline future directions toward achieving dynamic, multilayered reconstruction of inter- and intracellular communication, de novo signaling, and integrative multi-omics modeling.

PMID:42617152 | DOI:10.1093/bib/bbag446

Brief Bioinform. 2026 Jul 3;27(4):bbag439. doi: 10.1093/bib/bbag439.

ABSTRACT

The rapid expansion of high-throughput omics has created molecular datasets of unprecedented scale and complexity. These data are rich in biological information yet inherently sparse and high-dimensional, often limiting the effectiveness of conventional machine learning techniques. Foundation models (FMs), built on large-scale self-supervised pretraining, offer a robust alternative by learning generalizable representations directly from raw biological data. This review systematically analyzes the emerging landscape of FMs in omics research, spanning sequence modeling, cell state characterization, and multimodal integration. We organize the current literature into three distinct paradigms-sequence-centric, cell-centric, and multi-omics-to clarify a field currently fragmented by diverse tokenization strategies and architectural choices. Beyond methodology, we evaluate the practical utility of these models in tasks ranging from biomarker discovery to perturbation response prediction. We also identify critical barriers to adoption, including high computational costs, interpretability challenges, and the lack of standardized benchmarks. To support reproducible research, we provide a curated catalog of essential datasets and evaluation frameworks. Finally, we propose a roadmap for the next generation of FMs, advocating for architectures that move beyond statistical correlation to incorporate causal reasoning, temporal dynamics, and autonomous experimental validation.

PMID:42617149 | DOI:10.1093/bib/bbag439

Neurology. 2026 Sep 22;107(6):e218375. doi: 10.1212/WNL.0000000000218375. Epub 2026 Aug 19.

ABSTRACT

BACKGROUND AND OBJECTIVES: Evidence remains inconclusive on whether paternal valproate exposure during spermatogenesis is associated with adverse offspring outcomes, with findings lacking beyond Nordic countries. This study aimed to assess the risks of neurodevelopmental disorders (NDDs) and congenital malformations in offspring attributable to paternal exposure to valproate and other antiseizure medications (ASMs) during sperm development.

METHODS: This nationwide birth cohort study used data from the Taiwan National Health Insurance Research Database, including individuals born 2001-2016 who were followed up through 2021. Paternal exposure to valproate or other ASMs was defined during the time of spermatogenesis (3 months before conception). Exposure discordant sibling sets were identified for sibling-comparison analysis to control for shared genetic and lifestyle factors. NDDs-including autism spectrum disorder (ASD), attention-deficit/hyperactivity disorder (ADHD), intellectual disability (ID), and tic disorder-and congenital malformations were defined by outpatient and inpatient medical record(s). Relative risks were estimated through Cox proportional hazards models for NDDs (hazard ratios [HRs]) and logistic regression for congenital malformations (odds ratios [ORs]).

RESULTS: In the population cohort of 2,583,503 individuals, 1,701 were exposed to paternal use of valproate and 548 exposure-discordant sibling sets were available for sibling-comparison analysis. In population-based analyses, paternal valproate exposure was not associated with the risk of NDDs (HRs = 0.86 [95% CI 0.61-1.22] for ASD, 1.02 [0.88-1.18] for ADHD, 0.80 [0.53-1.20] for tic disorders, and 0.81 [0.55-1.19] for ID) or congenital malformations (OR = 1.07 [0.83-1.37]) in offspring. These null effects persisted when restricting analyses to children of fathers with epilepsy to control for confounding by indication, and when the sibling-comparison analysis was conducted. For other ASMs, a few associations appeared at nominal significance, but none remained in sibling-comparison analyses.

DISCUSSION: In this large population-based study in Asia, we found no increased risk of NDDs in offspring following paternal exposure to valproate or other ASMs. These findings may contribute to ongoing debates about the management of valproate in men of reproductive age, although additional evidence is needed before drawing conclusions about changes to current practice.

PMID:42617146 | DOI:10.1212/WNL.0000000000218375