Journal of Evidence‐Based Medicine最新文献

Aim: To survey the physician's attention to the workload of combining clinical practice with Traditional Chinese Medicine (TCM) data collection.

Background: With the development of artificial intelligence technology in the medical field, the task of collecting diverse clinical data in TCM has increased. Based on the TCM's diagnostic and treatment principles, the collection of research data accompanying clinical practice is inevitable, which may have an impact on TCM clinical practice.

Method: A previous research was conducted to collect diverse instant TCM diagnostic and treatment data, and physicians and research designers proposed many suggestions focusing on the workload of combining clinical practice with TCM data collection. In this study, A 54-item questionnaire was developed based on the suggestions. Forty-eight participants with data-collection experience participated in a questionnaire survey, and they needed to grade each item, which reflected their attention to the workload of combining clinical practice with TCM data collection.

Results: The survey received 40 valid questionnaires, with 49 items scoring 4 or above. Three items in the content dimension (Q9, Q10, Q11) and two items in the spatial dimension (Q31, Q48) are scored lower. Additionally, 25 supplementary suggestions were collected during the study.

Conclusion: The workload of combining clinical practice with TCM data collection needs to be considered. The items in this survey could be regarded as a basis for developing a tool to consider the relationship between clinical practice and data collection.

Background: Percutaneous transhepatic cholangioscopy (PTCS) is a minimally invasive treatment for biliary diseases; however, postoperative biliary drainage can impair quality of life and cause complications. We developed a biodegradable blockage (BB) for tract embolization to replace drainage; this is the first study investigating this approach after PTCS.

Methods: In this prospective study, 10 patients with bile duct stones underwent PTCS with BB embolization (June-August 2024). Outcomes and complications were recorded over 3 months. A 1:1 propensity-matched control group from historical PTCS patients with standard drainage was established for comparison of hemoglobin levels and complications.

Results: BB placement was successful in all patients with no procedure-related deaths. No significant differences were found between the embolization and drainage groups in operative time, hemoglobin changes, or complication rates, though the small sample size warrants caution. One patient in the embolization group had a Grade II complication, versus three complications (two Grade I, one Grade II) in the drainage group. The embolization group had no serious adverse events during follow-up.

Conclusion: Tract embolization with BB appears to be a safe and feasible alternative to conventional drainage after PTCS. Larger randomized trials are needed for validation.

Objective: Integration of traditional Chinese and modern medicine (TCM-MM) aids rehabilitation of muscle strength among ischemic stroke (IS) survivors. However, it faces statistical challenges (e.g., multicollinearity, small sample) in the real-world setting. This study tried to provide an analytical framework for investigating linear causality with a retrospective small-sample case series.

Methods: Original data was sourced from hospital information system and processed by many means. Wilcoxon signed-rank test was utilized to execute a self-controlled before-and-after comparison, before multiple linear regression (MLR) models were established for exploring prognostic factors of muscle strength improvement. Afterward, Bayesian networks (BN), mediation analysis and between-subjects effects tests were undertaken the detection of underlying multicollinearity sources progressively. Both clinical interpretability and model performance, containing R² and mean squared error (MSE), served as the indices for modelling comparison.

Results: Muscle strength was significantly improved among 112 post-IS patients after accepting TCM-MM therapies (p < 0.01). Initially, MLR analysis with 11 explanatory variables (EVs) (MLR_1) revealed a probable multicollinearity-driven bias, resulting in reduced interpretability. Consequently, we traced collinearity among EVs using a BN structure that provided clues to mediating and mutual effects for establishing MLR with interactions embracing 11 EVs (MLR_2). Eventually, MLR_2 demonstrated superior model performance (ΔR² = 0.097, ΔMSE = -0.004), and better clinical interpretability. Whereas, we cannot deny a 1/3 probability of diminished statistical efficacy due to the small sample size.

Conclusion: Our study proposed a practically hybrid approach for exploring linear causality under multicollinearity using real-world small-sample data, which suggested that balancing model performance with clinical interpretability can resolve statistical trade-offs in modelling optimization.

Objective: To systematically identify immune cell phenotypes causally associated with oral lichen planus (OLP) susceptibility using Mendelian randomization (MR).

Methods: This two-sample MR study evaluated causal relationships between 731 immune cell phenotypes and OLP risk. Single nucleotide polymorphisms (SNPs) were linkage disequilibrium-clumped (r² < 0.001, 10,000 kb), filtered by F-statistic (>10), and harmonized across datasets (palindromic SNPs with intermediate allele frequencies removed). Inverse variance weighting was the primary method, complemented by MR-Egger, weighted median, and mode-based estimations. Heterogeneity (Cochran's Q), horizontal pleiotropy (MR-Egger intercept, MR pleiotropy residual sum, and outlier), and leave-one-out analyses were used for sensitivity checks. Associations passing multiple-testing correction were interpreted.

Results: Twenty-eight immune phenotypes demonstrated significant causal associations: 19 protective and 9 risk-increasing. Five of six regulatory T-cell (Treg) phenotypes showed protective effects, with odds ratios (ORs) ranging from 0.916 to 0.958, and CD3 on CD4 Tregs showing the strongest effect (OR = 0.916). CD8-bright leukocytes showed the strongest risk association (OR = 1.487). Eight B cell phenotypes conferred protection, particularly human leukocyte antigen DR (HLA DR) on B cells (OR = 0.889). Monocyte phenotypes exhibited divergent effects: Myeloid-derived suppressor cells were protective (OR = 0.840), whereas HLA DR-expressing monocytes increased risk (OR range: 1.276-1.281).

Conclusions: This study provides genetic evidence that OLP pathogenesis involves immunoregulatory imbalance between protective regulatory mechanisms and pathogenic effector responses. Findings support precision therapeutic strategies targeting specific immune pathways and offer insights for other oral autoimmune diseases.

Objective: Upper tract urothelial carcinoma (UTUC) accounts for about 31% of urothelial malignancies in China, a markedly higher proportion than in Western countries. Limited molecular understanding hampers diagnosis and therapy. This study aimed to explore molecular mechanisms and identify potential biomarkers through a cohort-based integrative multi-omics analysis.

Methods: We analyzed 48 paired UTUC tumor and adjacent normal tissues. RNA and whole-exome sequencing were performed to explore difference. Weighted gene co-expression network analysis (WGCNA) was used to identify modules and hub genes associated with pathological traits. Validation included survival analysis within the discovery cohort, cross-cancer evaluation using The Cancer Genome Atlas Bladder Urothelial Carcinoma dataset (TCGA-BLCA) via gene expression profiling interactive analysis, and independent confirmation with a UTUC cohort from cBioPortal (n = 32). Protein expression was examined using immunohistochemistry (IHC) data from the Human Protein Atlas.

Results: We identified 3968 differentially expressed genes enriched in genitourinary development and calcium signaling pathways. WGCNA revealed four co-expression modules associated with infiltration, with 64 genes linked to pathological traits. Integrative analysis prioritized methionine sulfoxide reductase B3 (MSRB3) and Synaptopodin 2 (SYNPO2) as hub genes. Within the UTUC cohort, both genes showed trends toward poorer progression-free survival; in TCGA-BLCA, their expression and survival patterns provided supportive cross-cancer evidence; and in the independent UTUC dataset, clinicopathological correlations were directionally consistent with the discovery findings. IHC suggested lower protein expression in UTUC compared with normal urothelium.

Conclusions: This integrative multi-omics cohort study suggests MSRB3 and SYNPO2 as candidate biomarkers for UTUC progression, offering insights for risk stratification and potential therapeutic development.

Fanconi anemia (FA) is an inherited bone marrow failure syndrome characterized by pancytopenia, cancer predisposition, and physical abnormalities, due to its variable disease manifestations, diagnostic delays are common, making the management of FA is challenging. The core pathophysiology of FA lies in defects in the FA DNA repair pathway, which is crucial for resolving interstrand crosslinks (ICLs) and maintaining genomic stability. Hematopoietic stem cell transplantation (HSCT) remains the only curative treatment for FA-associated hematological abnormalities, but its success is influenced by donor type, patient age, conditioning regimens, and graft-versus-host disease (GVHD) management. This review synthesizes current knowledge on FA genetics and pathophysiology, comprehensively analyzes HSCT outcomes in pediatric patients, discusses factors influencing transplant success, and explores emerging therapeutic strategies including gene therapy. By integrating data from large multicenter studies and recent mechanistic insights, this review provides a comprehensive update for clinicians and researchers involved in the care of FA patients.

The exponential growth of secondary literature has created an imperative for researchers to identify credible and methodologically sound studies within an increasingly complex information landscape. In light of the growing emphasis on evidence-based medicine in both domestic and international contexts, umbrella reviews (URs) have emerged as a critical methodological approach in biomedical research. As an advanced form of tertiary evidence synthesis, URs systematically integrate findings from multiple systematic reviews and meta-analyses, thereby providing a comprehensive evidence base for specific research questions or related fields. This methodological framework enhances the quality of evidence through critically evaluating the validity and reliability of conclusions drawn from secondary or primary studies. The present overview systematically examines the conceptual framework, distinctive characteristics, development process, and methodological quality assessment of URs, with the objective of establishing a theoretical foundation and practical reference for future research in this domain.

Background: Data from Hospital Information Systems are crucial components of real-world data, but concerns arise regarding the transparency of measurement time-points when directly utilizing for efficacy studies. The objective of the study was to analyze the distribution characteristics of time-points for laboratory test records from HIS.

Method: Medical records before December 31, 2019 from Affiliated Hospital of Shandong University of Traditional Chinese Medicine, for patients primarily diagnosed with coronary heart disease (CHD), or heart failure combined with secondary diagnosis of CHD were retrieved from HIS. Fifteen test groups were extracted. The number of records, average of test times, and distribution characteristics of time points were analyzed.

Results: The renal function tests have the most records, and the blood glucose have the most times. Tests time-points distribution showed concentration in the early stage of hospitalization, with the majority occurring within the first 0-10%. For those measured ≥2 times, their first tests are also centrally distributed in the early stage, while the lasts in all period of hospitalization. Besides, substantial difference is showed in the time span of the first and last test. Abnormal value may be a trigger that promotes more intensive examination, and normal or abnormal status of the first examination is significantly weak to moderate correlated with the number of examinations and time span.

Discussion: The disclosure of time-points of clinical studies based on HIS should be encouraged and the pre-survey of data measuring time point is essential to the design of trial.

Objective: To develop a core outcome set (COS) for clinical trials on post COVID-19 condition (PCC), that is, what, when, and how to measure PCC.

Method: A comprehensive collection of outcomes (including their measurement methods and phases) was launched via literature review and clinician and patient surveys. Two rounds of Delphi surveys were conducted under the predefined criteria for rating, followed by a consensus meeting to finalize the COS for PCC (COS-PCC).

Results: Fifty-two outcomes within 7 categories and 206 measurement methods were identified. Sixty participants from five stakeholder groups completed the first round of the Delphi survey and 41 the second. Consensus was reached among 36 representatives on four domains of respiratory, physical, neuropsychological, and health conditions, including nine core outcomes and their respective measurement methods of priority: dyspnea (modified Medical Research Council scale), cough (Leicester Cough Questionnaire), exercise capacity (6-min walk test), fatigue (Fatigue Severity Scale), pain (Numerical Rating Scale), sleeping disturbance (Pittsburgh Sleep Quality Index), anxiety (Generalized Anxiety Disorder Scale-7), depression (Patient Health Questionnaire-9), and health status (36-item Short Form Health Survey); 16 optional measurement methods achieved consensus for supplement. Measuring phases of each core outcome were prioritized by importance through short and long terms of PCC.

Conclusions: The COS-PCC highlights the key PCC concerns and provides an essential outcome set for PCC assessment in clinical trials and evidence synthesis. With improving the understanding of PCC and accumulating research evidence, the COS-PCC needs to be continuously updated and improved in practice.