Tag: pubmed

Stat Med. 2026 Mar;45(6-7):e70488. doi: 10.1002/sim.70488.

ABSTRACT

In the design of clinical trials, it is essential to assess the design operating characteristics (e.g., power and the type I error rate). Common practice for the evaluation of operating characteristics in clinical trials that employ Bayesian analysis and decision procedures relies on estimating the sampling distribution of posterior summaries via Monte Carlo simulation. It is computationally intensive to repeat this estimation process for each design configuration considered, particularly for clustered data that are analyzed using complex, high-dimensional models. In this paper, we propose an efficient method to assess operating characteristics and determine sample sizes for Bayesian trials with clustered data. We prove theoretical results that enable posterior probabilities to be modeled as a function of the number of clusters. Using these functions, we assess operating characteristics at a range of sample sizes given simulations conducted at only two values for the number of clusters. These theoretical results are also leveraged to quantify the impact of simulation variability on our sample size recommendations. The applicability of our methodology is illustrated using an example Bayesian cluster-randomized clinical trial.

PMID:41820232 | DOI:10.1002/sim.70488

Stat Med. 2026 Mar;45(6-7):e70473. doi: 10.1002/sim.70473.

ABSTRACT

Although randomized controlled trials (RCTs) are the gold standard for evaluating the efficacy and safety of treatments, they are challenged by cost, duration, enrollment, or ethical concerns. A possible solution is to incorporate external control data as a hybrid control group, for which various statistical methods are available. However, only a few of them account for confounding bias due to unknown/unmeasured covariates between the internal and external control data. Moreover, the amount of this potential bias cannot be measured using most existing methods without extensive simulations. Here, we propose a novel method for estimating the confounding effects of unmeasured covariates based on model-based regression standardization, inverse probability weighting, and augmented inverse probability weighting for continuous or binary outcomes. We also propose an estimator that dynamically borrows external data and uses a weighted mean, adjusting weights according to the estimated confounding effect of unmeasured covariates. In the proposed method, the expected amount of bias can be controlled within a prespecified “bias-tolerance cap,” which may facilitate a better discussion among stakeholders about whether an effect estimate has unacceptable bias by utilizing external control data in a planning phase. Simulations showed that the proposed method regulates bias within the tolerance cap, regardless of the magnitude of confounding by unmeasured covariates, while greatly improving power and efficiency when confounding is absent. Finally, we illustrate an applicational example of our proposed method to an actual RCT and the external control datasets for patients with advanced pancreatic cancer.

PMID:41820222 | DOI:10.1002/sim.70473

Eur Ann Otorhinolaryngol Head Neck Dis. 2026 Mar 11:S1879-7296(26)00048-7. doi: 10.1016/j.anorl.2026.02.008. Online ahead of print.

ABSTRACT

OBJECTIVES: Temporal bone drilling simulators are valuable tools for developing anatomical knowledge and drill handling motor skills. However, there are currently no simulators focusing on learning fine microsurgical gestures using micro-instruments. The aim of this study was to validate a training kit and simulation program dedicated to otologic microsurgery.

MATERIALS AND METHODS: A prospective single-center comparative study was conducted. The Micro-TRAIN kit comprised a mannequin and six interchangeable exercise modules. Two simulation sessions were conducted two months apart, with debriefing for two selected subgroups after the first session. Progression in performance, assessed by task completion time, execution time and technical skills, was evaluated in three groups: 10 novices, 10 intermediate level and 5 experts. Content validity, face validity and participant satisfaction were also assessed.

RESULTS: In session 1, there were significant differences in performance between the three groups (P<0.005). Both the novice and intermediate groups showed improvement between sessions. Improvement in total score in the intermediate group was statistically significant (P=0.0019) and suggestive in the novice group (P=0.0059). Participants who received debriefing tended to improve more (novices: P=0.012; intermediate: P=0.036). Experts rated the simulator’s realism and relevance as above 8/10.

CONCLUSION: This study confirmed the content, face and construct validity of the Micro-TRAIN simulator. It is an effective tool for acquiring microsurgical skills in otologic surgery.

PMID:41820176 | DOI:10.1016/j.anorl.2026.02.008

J Cyst Fibros. 2026 Mar 11:S1569-1993(26)00038-X. doi: 10.1016/j.jcf.2026.02.009. Online ahead of print.

ABSTRACT

BACKGROUND: Cystic fibrosis (CF) transplant recipients infected with SARS-CoV-2 are at high risk for hospitalization or death. We aimed to (1) assess whether time since solid organ transplantation impacts severity of SARS-CoV-2 infection and (2) to evaluate the impact of SARS-CoV-2 infection on the slope of lung function trajectory.

METHODS: This is a retrospective international cohort study of individuals with CF post-solid organ transplant with a confirmed SARS-CoV-2 infection between January 2020 and December 2021. The primary outcome was death or hospitalization. The secondary outcome was change in lung function trajectory following infection. To assess the impact of time from transplant on the primary outcome, logistic regression was performed while lung function trajectory was assessed using a linear mixed-effects model.

RESULTS: A total of 526 SARS-CoV-2 infections from 19 countries were recorded. The median age at time of infection was 36 years (IQR 29-44). Median time since transplant was 5.8 years (IQR 3.3-10.8). The timing of transplant relative to infection was not significantly associated with hospitalization or death (OR 0.975 CI 0.928-1.025). A higher baseline ppFEV₁ was associated with a decreased odds of death or hospitalization (OR 0.989, 95% CI 0.983, 0.995). In a subgroup of participants, lung function trajectory did not change significantly in the year following SARS-CoV-2 infection.

CONCLUSIONS: In a diverse global post-transplant CF population, the timing of transplantation was not significantly associated with severe outcomes following SARS-CoV-2 infection. Those with more severe lung disease were at increased risk for worse outcomes and should be monitored closely.

PMID:41820127 | DOI:10.1016/j.jcf.2026.02.009

J Voice. 2026 Mar 11:S0892-1997(26)00049-4. doi: 10.1016/j.jvoice.2026.01.045. Online ahead of print.

ABSTRACT

OBJECTIVES: Adductor laryngeal dystonia (AdLD) is a neurological voice disorder characterized by involuntary spasms of the adductor laryngeal muscles during phonation. An automated creak detector has shown promise in differentiating English speakers with AdLD from controls. However, no study has yet investigated creak in AdLD in Spanish speakers. In fact, there is a paucity of research validating tools to identify laryngeal dystonia (LD) in languages other than English. The purpose of this study was to determine whether creak differentiates Spanish-speaking individuals with and without AdLD.

METHODS: Twenty speakers with AdLD, twenty speakers without voice disorders (controls), and twenty speakers with glottic insufficiency were recorded in a clinical environment. Each participant read a set of recently developed Spanish stimuli designed for LD screening, containing voiced and voiceless loaded sentences. An open-source creak detector was used to calculate the percentage of creak in each speaker’s recording. Mean smoothed cepstral peak prominence (CPPS) per speaker was calculated in Praat.

RESULTS: A Pearson’s correlation revealed a moderate relationship between creak and CPPS across groups (r = -0.56). An analysis of covariance revealed a statistically significant effect of creak between groups (F(3, 56) = 13.52, P > 0.05, R² = 0.39). Three receiver operating characteristic curve analyses indicated that creak differentiated AdLD and Controls (area under the curve [AUC] = 0.88), as well as AdLD and Glottic Insufficiency with acceptable diagnostic accuracy (AUC = 0.73), but not between control or glottic insufficiency groups.

CONCLUSIONS: Creak differentiated Spanish speakers with and without AdLD with moderate discrimination. Although creak and CPPS were moderately correlated, when controlling for CPPS, creak was statistically different between speakers with AdLD and speakers with glottic insufficiency, and between speakers with AdLD and controls. Further work is needed to determine the clinical utility of creak in aiding a differential diagnosis of AdLD and muscle tension dysphonia in Spanish-speaking individuals.

PMID:41820120 | DOI:10.1016/j.jvoice.2026.01.045

Zhonghua Yan Ke Za Zhi. 2026 Mar 11;62(3):214-222. doi: 10.3760/cma.j.cn112142-20250425-00203.

ABSTRACT

Objective: To investigate the clinical, pathological and molecular genetic characteristics of conjunctival melanoma (CoM). Methods: A retrospective case series study was conducted. The clinical, imaging and pathological data of patients diagnosed with CoM from January 2004 to June 2025 at Shaanxi Eye Hospital of Xi’an People’s Hospital (Xi’an Fourth Hospital) and Xi’an First Hospital were analyzed. Some patients were detected for the BRAF V600E mutation. The χ² test or the χ² correction test was used for statistical analysis. Results: A total of 70 patients (70 eyes) with CoM were enrolled, aged (60.8±10.6) years, including 40 males (57.1%) and 30 females (42.9%). There were 33 cases (47.1%) in the left eye and 37 cases (52.9%) in the right eye. The tumor was located at the bulbar conjunctiva in 37 cases (52.9%), at the vault conjunctiva in 24 cases (34.2%), and at the palpebral conjunctiva in 9 cases (12.9%), involving the orbit in 13 cases (18.6%), the eyeball in 3 cases (4.3%), and the lacrimal sac in 6 cases (8.6%). Most patients presented with black nodules or cauliflower-like masses in the conjunctival area. Some tumors were accompanied by superficial vascular hyperplasia and pigmentation of adjacent tissues, while some invaded the cornea or orbit, resulting in clinical manifestations such as visual field defects, exophthalmos and limited movement. Imaging showed irregular soft tissue density shadows at the conjunctival site. According to the criteria of the American Joint Committee on Cancer, the tumor was at the T1 stage in 10 cases (14.3%), T2 stage in 41 cases (58.6%), and T3 stage in 19 cases (27.1%), with superficial ulcers in 6 cases (8.6%). Histopathology results disclosed that 67 cases (95.7%) were of the nodular type, 3 cases (4.3%) were of the superficially diffuse type, 47 cases (67.1%) were of the epithelial cell type, and 23 cases (32.9%) were of the mixed cell type. Sixty-three cases (90.0%) were accompanied by melanin, 32 cases (45.7%) were accompanied by primary acquired melanosis, including 23 cases (32.9%) with atypical primary acquired melanosis and 2 cases (2.9%) with conjunctival nevus, and 48 cases (68.6%) had tumor infiltrating lymphocytes. Immunohistochemistry demonstrated positive melanin markers such as human melanoma-associated antigen 45, melanocyte antigen A, S-100 protein and sex-determining region Y-frame protein 10. Thirty cases (42.9%) completed the detection of BRAF V600E mutations, of which 11 had BRAF V600E point mutations, with a positive rate of 36.7%. Sixty-two patients (88.6%) were followed up, with a recurrence rate of 27.4% (17/62), a metastasis rate of 19.4% (12/62), and a case fatality rate of 54.8% (34/62). The recurrence rate was 4/5 among patients with ulcers, versus 22.8% (13/57) among patients without ulcers (χ²=4.96, P=0.026); it was 8/13 among patients with orbital invasion, versus 18.3% (9/49) among patients without orbital invasion (χ²=9.62, P=0.002). Thirty-four patients (48.5%) underwent complete resection of the ocular mass, 16 (22.9%) underwent partial resection, and 20 (28.6%) underwent ocular or intraorbital enucleation. Ten patients (14.3%) received BRAF inhibitor-targeted therapy. Seven patients had a significant reduction of the residual mass and no tumor progression, and 3 patients did not respond to the treatment and died from the disease progression. Conclusions: CoM mostly occurs in the unilateral eye of middle-aged and elderly patients, more common at the bulbar conjunctiva and fornix conjunctiva, and histopathological epithelial cell types are the main types, with a high recurrence and metastasis rate.

PMID:41820066 | DOI:10.3760/cma.j.cn112142-20250425-00203

Zhonghua Yan Ke Za Zhi. 2026 Mar 11;62(3):202-208. doi: 10.3760/cma.j.cn112142-20250425-00198.

ABSTRACT

Objective: To report the recurrence of peripheral angle synechiae (PAS) in primary angle-closure glaucoma (PACG) after phacoemulsification combined with intraocular lens implantation and goniosynechialysis (PEI-GSL) during an intermediate-and long-term follow-up. Methods: This retrospective case series included patients with acute or chronic PACG who underwent PEI-GSL performed by the same surgeon at the Eye Hospital of Wenzhou Medical University between January 2017 and December 2019, with complete postoperative gonioscopic records. The primary outcome measures were the incidence and extent (in clock hours) of recurrent PAS at the last follow-up. The secondary outcomes included the surgical success rate, intraocular pressure, and the number of intraocular pressure-lowering medications. The intergroup comparisons were performed using the generalized estimating equation. The intragroup comparisons before and after surgery were analyzed using the Wilcoxon signed-rank test. The categorical data were compared using the Chi-square test or Fisher’s exact test. Results: A total of 57 patients (69 eyes) were included, with a mean follow-up of 23.3±9.0 months. Among them, 37 patients (41 eyes) had acute PACG [9 males (24.3%), 28 females (75.7%); mean age, (68.6±8.6) years], and 20 patients (28 eyes) had chronic PACG [8 males (40.0%), 12 females (60.0%); mean age, (65.1±8.8) years]. There were no statistically significant differences in gender or age between the two groups (both P>0.05). At the last follow-up, 46.4% (32/69) of eyes maintained a full-circle anterior chamber angle opening or had only several spot adhesions, 82.6% (57/69) had PAS limited to one quadrant, and 89.9% (62/69) had PAS involving fewer than 6 clock hours. The recurrence rate of PAS was 31.9% (22/69) in all eyes, while it was 26.8% (11/41) in eyes with acute PACG, and 39.3% (11/28) in eyes with chronic PACG, with no statistically significant difference (χ²=1.92, P=0.165). However, the extent of recurrent PAS was greater in chronic PACG patients [4.50 (2.50, 8.50) clock hours] than in acute PACG patients [3.00 (1.50, 4.50) clock hours], and this difference was statistically significant (Wald χ²=5.39, P=0.020). Conclusions: Approximately 30% of eyes with PACG develop recurrent PAS after PEI-GSL, but 90% maintain an angle opening over 180°.

PMID:41820064 | DOI:10.3760/cma.j.cn112142-20250425-00198

Zhonghua Yan Ke Za Zhi. 2026 Mar 11;62(3):187-192. doi: 10.3760/cma.j.cn112142-20250619-00279.

ABSTRACT

Objective: To investigate the correlation between macular edema and the anterior chamber angle status in patients with retinal vein occlusion (RVO). Methods: A retrospective case series analysis was adopted. Consecutive RVO patients treated at the Ophthalmology Department of Peking University Third Hospital from October 2011 to May 2013 were included. All patients underwent fluorescein fundus angiography for RVO classification. They were divided into the macular edema and non-macular edema groups based on the angiography results. Static and dynamic gonioscopy were performed to determine the anterior chamber angle status. The statistical analysis was performed using the independent samples t-test, Welch’s t-test, chi-square test, or Fisher’s exact test. The multivariate logistic regression analysis was used to identify factors associated with the occurrence of macular edema by calculating odds ratios and their 95% confidence intervals. Results: A total of 308 RVO patients (308 eyes) who completed all required examinations were included in the study, consisting of 155 males and 153 females, with a mean age of (58.78±14.45) years. Among them, 59 cases (19.16%) had angle closure, while 249 cases (80.84%) had open angles; 221 patients had macular edema (71.75%). The multivariate regression analysis revealed that the main risk factor for the development of macular edema in eyes with RVO was angle closure (odds ratio=2.59, 95% confidence interval: 1.23-6.05, P=0.018), while other factors such as age, gender, eye laterality, intraocular pressure, type of RVO, and cup-to-disc ratio showed no statistically significant association (all P>0.05). Conclusion: The risk of macular edema is significantly higher in RVO patients with angle closure compared to those with open angles.

PMID:41820063 | DOI:10.3760/cma.j.cn112142-20250619-00279

Zhonghua Yi Xue Za Zhi. 2026 Mar 17;106(10):934-940. doi: 10.3760/cma.j.cn112137-20251226-03425-1.

ABSTRACT

Objective: To evaluate the vaccine effectiveness (VE) of the Lanzhou lamb rotavirus vaccine (LLR) against rotavirus gastroenteritis (RVGE) in children using a propensity score-matched (PSM) test-negative case-control design. Methods: Based on electronic health records from medical institutions and the National Immunization Program Information System, demographic information, disease diagnoses, rotavirus pathogen test results, and rotavirus vaccination details (vaccine type, number of doses, dates) were collected for acute gastroenteritis cases aged 2 months to 4 years in Chaoyang District (Beijing), Guangdong Province, and Wuhan City (Hubei Province) from 2020 to 2024. Patients were divided into a case group (rotavirus-positive) and a control group (rotavirus-negative) based on etiological test results, and vaccination proportions were calculated for each group. To reduce effects of confounding factors, a PSM test-negative case-control design was employed. The VE of LLR against different clinical outcomes was calculated using a conditional logistic regression model, and the VE of LLR against healthcare visits for rotavirus infection across different age groups was calculated using a multivariate logistic regression model. Results: A total of 16 460 children with acute gastroenteritis aged 2 months to 4 years were collected, with an age [M (Q₁, Q₃)] of 1.4 (0.7, 2.7) years, and 9 713 (59.01%) were male. The number of children who received 1, 2, and 3 doses of LLR was 2 391 (14.53%), 894 (5.43%), and 180 (1.09%) cases, respectively. There were 1 799 cases (10.93%) in the case group. Among them, the majority were from Hubei Province (953 cases, 52.97%), with a visiting age of 2-4 years (947 cases, 52.64%), males (1 082 cases, 60.14%), in the outpatient department (1 430 cases, 79.49%), with a visiting month of April (574 cases, 18.73%), and with 0 doses of vaccination (1 523 cases, 84.66%). There were statistically significant differences in province, age, case type, month of visit and dose of vaccination between the case and the control groups (P<0.001). After PSM matching, the VE (95%CI) of 1-dose and 2-dose LLR against the RVGE visits were 55.66% (45.83%-63.71%) and 59.37% (45.48%-69.72%), respectively, and for RVGE hospitalizations were 63.87% (38.96%-78.62%) and 82.95% (58.37%-93.01%), respectively. Multivariate logistic regression models analysis showed that among the RVGE visits, the VE of children aged 2 months to 1 year and 1 to 2 years after one dose of LLR was greater than that of children aged 2-4 years [54.96% (16.42%-78.62%) vs 59.29% (45.69%-69.98%) vs 49.53% (37.22%-59.75%), all P<0.001]. Conclusions: LLR has a good protective effect on children aged 2 months to 4 years. Two doses provide better protection against rotavirus infection, especially for hospitalized patients with RVGE and children under 2 years old.

PMID:41820057 | DOI:10.3760/cma.j.cn112137-20251226-03425-1

Zhonghua Yi Xue Za Zhi. 2026 Mar 17;106(10):912-918. doi: 10.3760/cma.j.cn112137-20251208-03229.

ABSTRACT

Objective: To explore the effect of sodium bicarbonate Ringer’s solution on postoperative acute kidney injury (AKI) in elderly patients undergoing off-pump coronary artery bypass grafting (OPCABG). Methods: The medical records of patients undergoing elective OPCABG at Tianjin Chest Hospital, Tianjin University, between September 2021 and October 2023 were retrospectively reviewed. The inclusion criteria were as follows: age ≥65 years, body mass index (BMI) of 18 to 30 kg/m², and American Society of Anesthesiologists (ASA) physical status class Ⅲ-Ⅳ. Patients were allocated to either the sodium bicarbonate Ringer’s solution group or the compound electrolyte solution group (control group) based on the type of intraoperative crystalloid administered. Potential confounders were adjusted using propensity score matching (PSM) and multivariable logistic regression models. The primary outcome was the incidence of AKI within 7 days postoperatively. Results: A total of 868 patients aged (69±5) years were included in the final analysis, with 591 males and 277 females. Postoperative AKI occurred in 105 patients (12.1%). After PSM, there were no statistically significant differences in baseline characteristics and intraoperative variables between the two groups (all P>0.05). Multivariable logistic regression analyses demonstrated that, intraoperative use of sodium bicarbonate Ringer’s solution was a protective factor against postoperative AKI following OPCABG (OR=0.52, 95%CI: 0.28-0.97, P=0.040). Conclusion: Compared with compound electrolyte solution, use of sodium bicarbonate Ringer’s solution during surgery is associated with a lower risk of AKI after OPCABG.

PMID:41820054 | DOI:10.3760/cma.j.cn112137-20251208-03229