Tag: pubmed

Stat Methods Med Res. 2025 Sep 30:9622802251374838. doi: 10.1177/09622802251374838. Online ahead of print.

ABSTRACT

Randomized controlled trials in oncology often allow control group participants to switch to experimental treatments, a practice that, while often ethically necessary, complicates the accurate estimation of long-term treatment effects. When switching rates are high or sample sizes are limited, commonly used methods for treatment switching adjustment (such as the rank-preserving structural failure time model, inverse probability of censoring weights, and two-stage estimation) may produce imprecise estimates. Real-world data can be used to develop an external control arm for the randomized controlled trial, although this approach ignores evidence from trial subjects who did not switch and ignores evidence from the data obtained prior to switching for those subjects who did. This article introduces “augmented two-stage estimation” (ATSE), a method that combines data from non-switching participants in a randomized controlled trial with an external dataset, forming a “hybrid non-switching arm”. While aiming for more precise estimation, the augmented two-stage estimation requires strong assumptions. Namely, conditional on all the observed covariates: (1) a participant’s decision to switch treatments must be independent of their post-progression survival, and (2) individuals from the randomized controlled trial and the external cohort must be exchangeable. With a simulation study, we evaluate the augmented two-stage estimation method’s performance compared to two-stage estimation adjustment and an external control arm approach. Results indicate that performance is dependent on scenario characteristics, but when unconfounded external data are available, augmented two-stage estimation may result in less bias and improved precision compared to two-stage estimation and external control arm approaches. When external data are affected by unmeasured confounding, augmented two-stage estimation becomes prone to bias, but to a lesser extent compared to an external control arm approach.

PMID:41026983 | DOI:10.1177/09622802251374838

J Med Internet Res. 2025 Sep 30;27:e74177. doi: 10.2196/74177.

ABSTRACT

BACKGROUND: In the era of data and intelligence, artificial intelligence has been widely applied in the medical field. As the most cutting-edge technology, the large language model (LLM) has gained popularity due to its extraordinary ability to handle complex tasks and interactive features.

OBJECTIVE: This study aimed to systematically review current applications of LLMs in lung cancer (LC) care and evaluate their potential across the full-cycle management spectrum.

METHODS: Following PRISMA (Preferred Reporting Items for Systematic Reviews and Meta-Analyses) guidelines, we conducted a comprehensive literature search across 6 databases up to January 1, 2025. Studies were included if they satisfied the following criteria: (1) journal articles, conference papers, and preprints; (2) studies that reported the content of LLMs in LC; (3) including original data and LC-related data presented separately; and (4) studies published in English. The exclusion criteria were as follows: (1) books and book chapters, letters, reviews, conference proceedings; (2) studies that did not report the content of LLMs in LC; and (3) no original data, and LC-related data that are not presented separately. Studies were screened independently by 2 authors (SC and ZL) and assessed for quality using Quality Assessment of Diagnostic Accuracy Studies-2, Prediction Model Risk of Bias Assessment Tool, and Risk Of Bias in Non-randomized Studies – of Interventions tools, selected based on study type. Key data items extracted included model type, application scenario, prompt method, input and output format, outcome measures, and safety considerations. Data analysis was conducted using descriptive statistics.

RESULTS: Out of 706 studies screened, 28 were included (published between 2023 and 2024). The ability of LLMs to automatically extract medical records, popularize general knowledge about LC, and assist clinical diagnosis and treatment has been demonstrated through the systematic review, emerging visual ability, and multimodal potential. Prompt engineering was a critical component, with varying degrees of sophistication from zero-shot to fine-tuned approaches. Quality assessments revealed overall acceptable methodological rigor but noted limitations in bias control and data security reporting.

CONCLUSIONS: LLMs show considerable potential in improving LC diagnosis, communication, and decision-making. However, their responsible use requires attention to privacy, interpretability, and human oversight.

PMID:41026980 | DOI:10.2196/74177

Blood Adv. 2025 Sep 30:bloodadvances.2025017254. doi: 10.1182/bloodadvances.2025017254. Online ahead of print.

ABSTRACT

Hydroxyurea provides effective disease-modifying treatment for people with sickle cell anemia (SCA), especially when escalated to maximum tolerated dose (MTD), but has wide interpatient dosing variability due to pharmacokinetic (PK) differences. Whether hydroxyurea PK parameters differ among children with SCA in different global regions is unknown. We compared hydroxyurea PK parameters among children with SCA from five clinical trials: HUSTLE (USA, NCT00305175), TREAT (USA, NCT02286154), NOHARM (Uganda, NCT01976416), REACH (Uganda and Kenya, NCT01966731), and EXTEND (Jamaica, NCT02556099). Key hydroxyurea PK parameters were determined using HdxSim™, a validated hydroxyurea PK-software program. The results were compared across regions by one way analysis of variance. The influence of laboratory and clinical variables on PK-guided doses were evaluated by linear regression. PK profiles from 451 children with SCA were included: 146 from the USA, 265 from Africa, and 40 from the Caribbean. Children from Africa had slightly lower volumes of distribution (p<0.001), but absorption rate (p=0.07) and clearance (p=0.2) were similar across regions. The PK-recommended doses to achieve MTD were statistically different but clinically similar: 26.6 ± 5.9, 27.6 ± 6.5, and 25.2 ± 4.7 mg/kg/day, respectively (p=0.04). In multivariable regression, younger age and increased reticulocyte counts were associated with higher PK-recommended doses. Hydroxyurea PK parameters in children with SCA differ minimally across global populations, predicting clinically similar doses to achieve MTD. Individualized hydroxyurea dosing based on a PK-population model derived from American children with SCA can be used broadly to maximize the benefits of this critical medication in other global populations.

PMID:41026975 | DOI:10.1182/bloodadvances.2025017254

JCO Oncol Pract. 2025 Sep 30:OP2500266. doi: 10.1200/OP-25-00266. Online ahead of print.

ABSTRACT

PURPOSE: Germline testing is recommended for patients with high-risk localized, locally advanced, and metastatic prostate cancer; however, implementation remains suboptimal. Novel strategies are needed to engage oncology clinicians and patients in germline testing.

METHODS: A single-arm investigator-initiated study, conducted from October 1, 2022, to December 31, 2023, used video education as pretest counseling for patients meeting National Cancer Comprehensive Network germline testing criteria. Patients attended a one-on-one in-person session with an educational video, followed by prevideo and postvideo questionnaires assessing knowledge and satisfaction. The primary end point was the proportion who underwent germline testing after intervention, compared with a contemporaneous standard-of-care (SOC) group. Secondary end points included changes in knowledge and perceptions.

RESULTS: All 50 enrolled patients completed the intervention. Patients were predominantly White (78.0%), non-Hispanic (92.0%), English-speaking (98.0%), and college-educated (70%). Most had high-risk localized (46.0%) or metastatic hormone-sensitive prostate cancer (26.0%). Germline testing uptake was significantly higher in the intervention group (82.0%, 41/50) compared with the SOC group (37.94%, 107/282, P < .001). In the intervention group, urologists ordered 22.5% of tests and medical oncologists ordered 77.5%. Pathogenic/likely pathogenic alterations were identified in 10.0% of patients. Most participants (64%) indicated that the most important factor in their decision was whether results could guide treatment. Those who declined testing cited lack of clinical value. Most scored high on the prevideo genetic knowledge test (mean 9.1/11.0) with no statistical difference postvideo (P = .88). Most found the video satisfactory and useful (94%).

CONCLUSION: Germline testing uptake was high after video education. Most patients had high baseline genetic knowledge but were more likely to pursue testing after intervention if it influenced their treatment. Virtual educational aids should be integrated into clinical practice to increase testing rates.

PMID:41026959 | DOI:10.1200/OP-25-00266

J Clin Oncol. 2025 Sep 30:JCO2500307. doi: 10.1200/JCO-25-00307. Online ahead of print.

ABSTRACT

PURPOSE: Older patients with ALL receiving conventional chemotherapy have poor survival due to toxic death and relapse. We hypothesized that a chemotherapy-free, targeted regimen using the anti-CD22 antibody-calicheamicin conjugate inotuzumab ozogamicin followed by the bispecific anti-CD19/CD3 T-cell engager blinatumomab would reduce toxic death and yield high rates of prolonged remission and survival.

METHODS: Eligible patients were age 60 years and older with untreated, Philadelphia chromosome (Ph)-negative, CD22-positive, B-cell ALL. Patients received up to two cycles of inotuzumab ozogamicin followed by four or five cycles of blinatumomab with intrathecal methotrexate CNS prophylaxis. The primary end point was 1-year event-free survival (EFS).

RESULTS: The 33 eligible patients had a median age of 71 years (range, 60-84) and a median CD22 expression of 92% (range, 21%-100%). Eight (24%) had previous chemotherapy or radiation for other cancers, six for multiple myeloma. The composite complete remission rate was 85% after two cycles of inotuzumab ozogamicin and 97% by the end of two cycles of blinatumomab. At a median follow-up of 30 months, the 1-year EFS and overall survival were 75% (95% CI, 61 to 92) and 85% (95% CI, 73 to 98), respectively. EFS was shorter with lower CD22 expression or detectable measurable residual disease at any time point.

CONCLUSION: Inotuzumab ozogamicin then blinatumomab without maintenance chemotherapy in older patients with untreated, Ph-negative, CD22-positive, B-cell ALL yields a high remission rate and excellent EFS. Given the lack of standard, safe, and effective therapies in this population, the regimen should be considered a standard treatment option.

PMID:41026957 | DOI:10.1200/JCO-25-00307

J Appl Biomed. 2025 Sep;23(3):138-143. doi: 10.32725/jab.2025.010. Epub 2025 Aug 21.

ABSTRACT

INTRODUCTION: ORBEYE^TM exoscope offers superior visualization with clearer imaging compared to standard methods and supports narrow band imaging (NBI). ORBEYE^TM provides better visualization of tissue structures, thus increasing the accuracy of the surgical procedure. The systematic use of ORBEYE^TM in transoral exoscopic laryngeal surgery (TOLES) is rarely documented. This study evaluates the sensitivity and specificity of TOLES using ORBEYE^TM with white light and NBI modalities.

METHODS: Between 8/2021 and 8/2024, 84 patients underwent TOLES using ORBEYE^TM with white light and NBI modes. All surgeries were performed in a specialized setting with consistent preoperative and intraoperative imaging protocols.

RESULTS: TOLES using ORBEYE^TM was successfully performed in all 84 patients. A statistically significant dependence (p < 0.001) was observed between preoperative/perioperative (TOLES) findings and definitive histology for both white light and NBI modalities. ORBEYE^TM facilitated detailed imaging of tissue structures and allowed targeted biopsies.

CONCLUSION: TOLES with perioperative NBI/white light endoscopy achieved a high correlation between pre-histopathological diagnoses and final histopathological results. ORBEYE^TM exoscope provides three-dimensional 4K resolution, superior imaging, and improved ergonomics for the surgeon, reducing workload and increasing efficiency. It delivered more efficient surgical team collaboration and experience sharing. The integration of NBI into the TOLES system facilitated accurate navigation and targeting of the biopsy, helping to establish correct definitive diagnosis. TOLES showed more accurate scoring of perioperative NBI findings.

PMID:41026948 | DOI:10.32725/jab.2025.010

J Eval Clin Pract. 2025 Oct;31(7):e70290. doi: 10.1111/jep.70290.

ABSTRACT

BACKGROUND: The implementation of Diagnosis-Related Group (DRG) payment systems globally has intensified pressure on healthcare institutions to control costs while maintaining quality. Quality Control Circles (QCCs) have emerged as a promising continuous quality improvement method in nursing management. This study investigated the effectiveness of QCC-based nursing quality improvement on DRG cost control in general surgery departments.

METHODS: A cross-sectional survey was conducted among 50 healthcare professionals (18 physicians, 21 nurses, 8 nursing quality control staff, and 3 others) in a general surgery department. The survey assessed QCC implementation intensity, DRG knowledge, nursing improvement effects, and cost control obstacles. Descriptive statistics, Spearman correlation analysis, and Mann-Whitney U tests were performed to analyze relationships between variables.

RESULTS: QCC implementation showed high intensity scores (M = 4.16, SD = 0.93) with strong correlations to standardized operational procedures (ρ = 0.798). Nursing quality improvements were substantial (M = 4.38, SD = 0.73), and higher scores in key dimensions such as standardized preoperative education and multimodal analgesia were associated with fewer postoperative complications and shorter length of stay, thereby directly supporting DRG cost control.

CONCLUSION: QCC-based nursing quality improvement demonstrates effectiveness in supporting DRG cost control in general surgery through standardized processes and enhanced nursing practices. Among these achievements, the rationalization management score of high‑value consumables was the highest (M = 4.40), directly reducing the proportion of consumables cost within the DRG group, which makes the cost‑control effect more specific and clinically meaningful. The findings suggest that systematic implementation of QCC methodologies can facilitate both quality improvement and cost containment objectives within DRG payment frameworks.

CLINICAL TRIAL NUMBER: Not applicable.

PMID:41025865 | DOI:10.1111/jep.70290

J Eval Clin Pract. 2025 Oct;31(7):e70288. doi: 10.1111/jep.70288.

ABSTRACT

BACKGROUND: Individuals with visual impairment encounter considerable challenges in managing their treatments, which stem from difficulties in identifying medications, as well as reading and recalling information. Despite the implementation of various management strategies, errors remain prevalent and have the potential to compromise health. Consequently, pragmatic recommendations have been formulated in French for community pharmacists to enhance the quality of care for this vulnerable population.

AIM AND OBJECTIVE: To build an interdisciplinary consensus on guidelines for the care of individuals with visual impairment, with the aim of promoting their inclusion in healthcare.

METHOD: A consensus-building Delphi survey was conducted between May and October 2024 by a European panel of experts. The interdisciplinary panel consisted of ophthalmologists with a specialisation in low vision, orthoptists, ergotherapists, psychologists, and community pharmacists. The recommendations were encoded in the form of an online questionnaire on the LimeSurvey platform. The experts were invited to indicate their degree of agreement on a nine-point Likert scale. Descriptive statistics were produced using IBM® SPSS 29 Advanced software. This process was repeated until a consensus was reached between all the experts.

RESULTS: A total of four rounds of the Delphi method were required by the panel of ten experts to evaluate the initial set of 32 recommendations. In conclusion, an introduction to the guidelines and 30 recommendations, which have been grouped into six main categories, were validated.

CONCLUSION: The consensus process has facilitated the consolidation of recommendations and ensured their relevance, thereby enabling the dissemination of high-quality content to French-speaking community pharmacists to support their practice and promote health equity.

PMID:41025863 | DOI:10.1111/jep.70288

J Eval Clin Pract. 2025 Oct;31(7):e70289. doi: 10.1111/jep.70289.

ABSTRACT

BACKGROUND: Readmission following acute myocardial infarction (AMI) poses significant challenges to health systems and patient outcomes. The LACE index, a composite of Length of stay, Acuity of admission, Comorbidities, and Emergency department visits, is widely used for readmission prediction. However, its performance in large-scale, real-world Korean cohorts remains understudied.

OBJECTIVE: This study aimed to validate the predictive performance of the LACE index for 30-day readmissions in AMI patients using a nationally representative Korean cohort.

METHODS: This retrospective cohort study analyzed data from the Korean National Health Insurance Service Sample (NHISS) database from 2011 to 2020. A total of 609,640 adult patients hospitalized for AMI were included. The LACE index was calculated for each patient, and 30-day readmissions were identified. Logistic regression was used to estimate odds ratios (ORs) for readmission. Model discrimination was assessed using ROC curve analysis and C-statistics. Subgroup and survival analyses were performed by age, LACE score, and comorbidity burden.

RESULTS: Among 609,640 AMI patients, 205 (0.034%) experienced 30-day readmission. Patients with a LACE score of ≥ 10 had significantly higher odds of readmission (OR = 2.65; 95% CI: 1.68-4.19, p < 0.001) compared to those with scores 0-4. Middle-aged adults (35-64 years) also showed elevated readmission risk (OR = 3.42; 95% CI: 1.74-6.73, p < 0.001), while older adults (≥ 65 years) did not have significantly different risk. The LACE index showed moderate discriminatory performance (C-statistics = 0.71). Kaplan-Meier survival curves demonstrated significantly lower 30-day survival among patients with LACE ≥ 10.

CONCLUSIONS: Study findings suggest the LACE index is a useful tool for predicting 30-day readmissions among AMI patients in Korea. Its simplicity and moderate accuracy support its application in clinical and policy-level risk stratification strategies. Future prospective studies should refine prediction models by incorporating additional clinical variables.

PMID:41025862 | DOI:10.1111/jep.70289

J Head Trauma Rehabil. 2025 Sep 22. doi: 10.1097/HTR.0000000000001114. Online ahead of print.

ABSTRACT

OBJECTIVE: This study aimed to provide evidence on the long-term risk and comparative outcomes of hydrocephalus, a serious complication of traumatic brain injury (TBI) and hemorrhagic stroke (HS).

SETTING: The study analyzed data from the Korean National Health Insurance Service-National Sample Cohort (2002-2013). Data from 2002 to 2005 were used as a washout period, and cases identified from 2005 to 2013 were included in the analysis. New-onset hydrocephalus was defined as at least 2 medical claims with code G91 of the International Classification of Diseases, 10th revision.

PARTICIPANTS: A total of 17 331 patients diagnosed with TBI or HS were matched with 86 655 controls using propensity score matching.

DESIGN: A retrospective cohort study comparing patients with TBI or HS to matched controls.

MAIN MEASURES: Crude incidence rates (IRs), incidence rate ratios (IRRs), and adjusted hazard ratios were estimated using time-stratified Cox models over a 9-year follow-up period.

RESULTS: The incidence of hydrocephalus was significantly higher in the case group (IR, 1.88 per 1000 person-years) than in the control group (IR, 0.10). The overall IRR was 19.64 (95% confidence interval [CI], 13.30-29.00). Stratified analyses showed an IRR of 8.21 (95% CI, 5.32-12.68) for TBI and 35.49 (95% CI, 20.53-61.36) for HS. The adjusted hazard ratios declined over time but remained elevated for up to 6 years post-diagnosis. Risk was high in younger individuals, smokers, and alcohol users.

CONCLUSION: TBI and HS are associated with a substantially increased long-term risk of hydrocephalus, especially in the early years following diagnosis. HS confers a greater risk than TBI. These findings underscore the need for prolonged surveillance in high-risk individuals and appropriate management of hydrocephalus.

PMID:41025853 | DOI:10.1097/HTR.0000000000001114