Category: Statistics

JMIR Mhealth Uhealth. 2025 May 1;13:e67151. doi: 10.2196/67151.

ABSTRACT

BACKGROUND: The digital education platform Doctorvice (iKooB Inc.) offers face-to-face physician-patient education during outpatient clinic visits, remote glucose monitoring, and the delivery of educational messages, and is expected to be effective for personalized diabetes care.

OBJECTIVE: This study aims to evaluate the effectiveness of the digital education platform for diabetes care by comparing cases that included both face-to-face education and remote monitoring with those that included only face-to-face education.

METHODS: This was a randomized clinical study conducted at the Diabetes Center of Seoul St. Mary’s Hospital. Participants were aged ≥19 years and had glycated hemoglobin (HbA_1c) levels between 7.5% and 9.5%. In the intervention group, physicians used the digital education platform to provide face-to-face education at enrollment and at the 3- and 6-month visits, along with remote monitoring during the first 3 months of the 6-month study period. The control group received conventional outpatient education. Both groups completed questionnaires-assessing satisfaction with diabetes treatment, diabetes-related stress, and adherence to diabetes medication-at the beginning and end of the study. The primary endpoint was the change in HbA_1c levels.

RESULTS: A total of 66 participants were enrolled between August 1, 2022, and August 31, 2023. Of these, 26 in the intervention group and 30 in the control group were analyzed, excluding 10 participants who dropped out of the study. The mean baseline HbA_1c levels were 8.3% (SD 0.6%) in the intervention group and 8.0% (SD 0.5%) in the control group. At the 3-month follow-up, mean HbA_1c decreased by 0.5%-7.8% (SD 0.9%; P=.01) in the intervention group and by 0.2%-7.8% (SD 0.7%) in the control group. HbA_1c levels substantially improved during the first 3 months with both face-to-face education and remote glucose monitoring. However, HbA_1c tended to increase during the 3- to 6-month follow-up in the intervention group without the remote monitoring service. Satisfaction with diabetes treatment significantly improved at the end of the study compared with baseline in the intervention group (mean change +3.6 points; P=.006). Medication adherence improved in both groups, with no significant difference at 6 months (P=.59), although the intervention group showed a greater increase from baseline. Subgroup analysis indicated that the reduction in HbA_1c was greater for patients with baseline HbA_1c levels ≥8.0%, those aged ≥65 years, smokers, drinkers, and those with obesity in the intervention group.

CONCLUSIONS: The digital education platform for personalized diabetes management may be beneficial for glycemic control in type 2 diabetes mellitus. Its effectiveness appears to be enhanced when physicians provide personalized face-to-face education combined with remote feedback.

TRIAL REGISTRATION: Clinical Research Information Service (CRiS) of Republic of Korea KCT0007953; https://cris.nih.go.kr/cris/search/detailSearch.do?seq=23507&search_page=L.

PMID:40310669 | DOI:10.2196/67151

JAMA Netw Open. 2025 May 1;8(5):e258072. doi: 10.1001/jamanetworkopen.2025.8072.

ABSTRACT

IMPORTANCE: Medicare covers gender-affirming surgical procedures on a case-by-case basis. The proportion of Medicare beneficiaries who receive gender-affirming surgical procedures is unknown.

OBJECTIVE: To examine the frequency and trends of gender-affirming surgical procedures for Medicare beneficiaries.

DESIGN, SETTING, AND PARTICIPANTS: This cross-sectional study used national Medicare data to analyze transgender and gender diverse beneficiaries’ use of gender-affirming surgical procedures from January 1, 2016, to February 29, 2020, as well as use of the same surgical procedures by beneficiaries not identified as transgender. Transgender Medicare beneficiaries were identified using a claims-based algorithm; nontransgender individuals were selected using propensity score matching. Analyses were conducted from November 2022 through October 2024.

EXPOSURES: Race and ethnicity, Medicare-Medicaid dual status, age, original reason for Medicare entitlement, geographic area, and chronic conditions.

MAIN OUTCOMES AND MEASURES: Descriptive analyses and generalized estimating equations were used to assess gender-affirming surgical procedures and beneficiary characteristics associated with surgical procedures.

RESULTS: This study included transgender individuals (mean [SD] age, 60.0 [18.6] years; 43.2% transfeminine individuals, 33.0% transmasculine individuals, and 23.9% individuals with unclassified gender) and individuals (mean [SD] age, 62.7 [21.0] years; 55.5% women and 44.5% men) not identified as transgender. Of 142 703 total person-years, there were 2156 instances of transgender individuals having at least 1 gender-affirming surgical procedure in the calendar year. The rate of individuals receiving at least 1 surgical procedure decreased from between 2.1% and 2.2% in 2016 and 2017 to 1.4% in 2018 and 2019. There was substantial variability in surgical procedures across beneficiary characteristics. Transgender beneficiaries who underwent surgery were younger (31-40 years vs 61-65 years: adjusted odds ratio [AOR], 1.91 [95% CI, 1.55-2.34]) and had higher enrollment in Medicaid (AOR, 1.14 [95% CI, 1.02-1.26]), higher rates of chronic conditions (≥10 conditions vs 0: AOR, 2.10 [95% CI, 1.79-2.46]), and higher proportions of residents on the West Coast (Alaska, Idaho, Oregon, and Washington) vs the Northeast (Connecticut, Maine, Massachusetts, New Hampshire, Rhode Island, and Vermont) (AOR, 1.30 [95% CI, 1.05-1.61]) compared with those who did not undergo surgery. Geographically, the largest difference was observed when comparing the Northeast with the Southeast (Alabama, Florida, Georgia, Kentucky, Mississippi, North Carolina, South Carolina, and Tennessee) (AOR, 0.70 [95% CI, 0.58-0.86]; P < .001) and other parts of the South (Arkansas, Louisiana, New Mexico, Oklahoma, and Texas) (AOR, 0.56 [95% CI, 0.44-0.71]; P < .001). There was no significant difference in receipt of surgical procedures across regions for those not identified as transgender.

CONCLUSIONS AND RELEVANCE: This cross-sectional study of the Medicare program found that gender-affirming surgical procedures were very rare, particularly for those who were older, were not dually enrolled in Medicare and Medicaid, and who resided in the South. Although there were substantive regional differences in receipt of gender-affirming surgical procedures among transgender Medicare beneficiaries, beneficiaries not identified as transgender did not experience regional differences. Further study is warranted to examine barriers to accessing gender-affirming surgery for transgender and gender diverse beneficiaries.

PMID:40310645 | DOI:10.1001/jamanetworkopen.2025.8072

JAMA Netw Open. 2025 May 1;8(5):e258086. doi: 10.1001/jamanetworkopen.2025.8086.

ABSTRACT

IMPORTANCE: Among older women (aged ≥50 years) with ERBB2 (formerly HER2 or HER2/neu)-positive breast cancer, research has shown racial and ethnic disparities in access to ERBB2-targeted therapies, with Black women receiving treatment at lower rates than their White counterparts.

OBJECTIVE: To examine racial and ethnic disparities in receipt of ERBB2-targeted therapies and changes in receipt over time.

DESIGN, SETTING, AND PARTICIPANTS: This retrospective cohort study used Surveillance, Epidemiology, and End Results-Medicare linked data from January 1, 2010, to December 31, 2020. Beneficiaries who were diagnosed with ERBB2-positive breast cancer between 2010 and 2019, were aged 66 years or older at diagnosis, were continuously enrolled in Medicare Parts A and B in the 12 months before and after diagnosis, and had localized or regional stage disease at diagnosis were included. Data were analyzed from February through September 2024.

EXPOSURE: Race and ethnicity defined as non-Hispanic Black or African American, Hispanic, or non-Hispanic White.

MAIN OUTCOME AND MEASURES: The primary outcome was receipt of ERBB2-targeted therapies in the 12 months after diagnosis of ERBB2-positive breast cancer. Modified Poisson regression was used to evaluate the probability of receiving ERBB2-targeted therapy by race and ethnicity.

RESULTS: Among 12 765 beneficiaries with ERBB2-positive breast cancer (median [IQR] age, 74 [69-80] years; 99.2% female), 8.1% were of Black, 6.9% Hispanic, and 85.0% White race and ethnicity, and 54.2% received ERBB2-targeted therapy. The overall proportion who received ERBB2-targeted therapies increased from 41.3% in 2010-2011 to 64.3% in 2018-2019. Compared with White patients, Black patients had a lower likelihood of receiving ERBB2-targeted therapies in 2010-2011 (adjusted risk ratio [ARR], 0.81; 95% confidence limit [CL], 0.68-0.97), as did Hispanic patients (ARR, 0.75; 95% CL, 0.62-0.92). Racial and ethnic disparities in receipt of ERBB2-targeted therapies narrowed over time, with no significant differences observed across racial and ethnic groups in 2018-2019 for Black patients (ARR, 0.97; 95% CL, 0.87-1.08) and Hispanic patients (ARR, 1.05; 95% CL, 0.95-1.16).

CONCLUSIONS AND RELEVANCE: These findings suggest a narrowing of racial and ethnic disparities in receipt of ERBB2-targeted therapies over time among older Medicare beneficiaries with ERBB2-positive breast cancer. Future research is needed to understand the practices that contributed to the narrowing of racial and ethnic disparities and to develop implementation strategies to effectively improve the quality and equity of breast cancer care.

PMID:40310643 | DOI:10.1001/jamanetworkopen.2025.8086

Transl Vis Sci Technol. 2025 May 1;14(5):2. doi: 10.1167/tvst.14.5.2.

ABSTRACT

PURPOSE: To investigate whether cataract surgical skill performance metrics automatically generated by artificial intelligence (AI) models can differentiate between trainee and faculty surgeons and the correlation between AI metrics and expert-rated skills.

METHODS: Routine cataract surgical videos from residents (N = 28) and attendings (N = 29) were collected. Three video-level metrics were generated by deep learning models: phacoemulsification probe decentration, eye decentration, and zoom level change. Three types of instrument- and landmark- specific metrics were generated for the limbus, pupil, and various surgical instruments: total path length, maximum velocity, and area. Expert human judges assessed the surgical videos using the Objective Structured Assessment of Cataract Surgical Skill (OSACSS). Statistical differences between AI and human-rated scores between attending surgeons and trainees were assessed using t-tests, and the correlations between them were examined by Pearson correlation coefficients.

RESULTS: The phacoemulsification probe had significantly lower total path lengths, maximum velocities, and area metrics in attending videos. Attending surgeons demonstrated better phacoemulsification centration and eye centration. Most AI metrics negatively correlated with OSACSS scores, including phacoemulsification decentration (r = -0.369) and eye decentration (r = -0.394). OSACSS subitems related to eye centration and different steps of surgery also exhibited significant negative correlations with corresponding AI metrics (r ranging from -0.77 to -0.49).

CONCLUSIONS: Automatically generated AI metrics can be used to differentiate between attending and trainee surgeries and correlate with the human expert evaluation on surgical performance.

TRANSLATIONAL RELEVANCE: AI-generated useful metrics that correlate with surgeon skill may be useful for improving cataract surgical education.

PMID:40310637 | DOI:10.1167/tvst.14.5.2

Clin Auton Res. 2025 May 1. doi: 10.1007/s10286-025-01130-y. Online ahead of print.

ABSTRACT

PURPOSE: The aim of this work is to determine the relationship between the cross-sectional area (CSA) of the vagus nerve and cardiovagal function in people with multiple sclerosis (pwMS) and healthy controls (HC).

METHODS: We enrolled 50 pwMS and 50 HC. All participants underwent an ultrasound of the vagus nerve and autonomic nervous system testing. The Croatian version of the COMPASS-31 questionnaire was used as a measure of autonomic symptom burden. Cardiovagal function was evaluated with the respiratory sinus arrhythmia (RSA), Valsalva ratio (VR), and heart rate variability.

RESULTS: The mean vagus CSA in pwMS was 2.03 ± 0.49 mm² on the right side and 1.72 ± 0.38 mm² on the left side. The mean vagus CSA in HC was 2.08 ± 0.50 mm² on the right side and 1.74 ± 0.37 mm² on the left side. There was no statistically significant difference between the two groups in right (p = 0.615) or left (p = 0.866) vagus CSA. In the HC, there was a statistically significant positive correlation between the mean right CSA and both RSA (r_p = 0.331, p = 0.019) and VR (r_p = 0.327, p = 0.020). On univariable linear regression analysis in the HC group, the mean right CSA was a predictor of both RSA (B = 5.599, 95% CI 0.974-10.224, p = 0.019) and VR (B = 0.253, 95% CI 0.041-0.466, p = 0.020). These findings were not present in pwMS.

CONCLUSIONS: The loss of correlation between vagus nerve CSA and parameters of parasympathetic nervous system function in pwMS corroborates the presence of cardiovagal dysfunction in multiple sclerosis.

PMID:40310594 | DOI:10.1007/s10286-025-01130-y

Genet Med. 2025 Apr 25:101449. doi: 10.1016/j.gim.2025.101449. Online ahead of print.

ABSTRACT

BACKGROUND: Cerebrotendinous xanthomatosis (CTX) is a rare autosomal recessive disorder caused by pathogenic variants in CYP27A1 resulting in sterol 27-hydroxylase deficiency and accumulation of cholestanol and bile alcohols. Clinical features include cholestasis, diarrhea, cataracts, tendon xanthomas, and neurological deterioration. Chenodeoxycholic acid (CDCA) is the standard treatment for CTX. The effects of CDCA withdrawal on CTX biomarkers and safety in adult patients were evaluated.

MATERIALS AND METHODS: Patients (≥16 years) received CDCA 750 mg/day for two 8-week open-label periods followed by double-blinded (DB) CDCA or placebo for two 4-week periods. Key endpoints included changes from baseline in CTX biomarkers (23S-pentol, cholestanol, 7αC4, 7α12αC4) and the proportion of patients requiring CDCA rescue during DB periods.

RESULTS: CDCA withdrawal resulted in a 20-fold increase in 23S-pentol, and increases in cholestanol (2.8-fold), 7αC4 (50-fold) and 7α12αC4 (14-fold). During the DB withdrawal periods, 61% of participants on placebo required rescue medication. CDCA treatment was well tolerated; the most common treatment-emergent adverse events were diarrhea and headache, most mild/moderate in severity and not considered treatment-related.

CONCLUSIONS: CDCA withdrawal caused statistically significant increases in CTX biomarkers and necessitated rescue therapy in most participants. CDCA treatment is critical for control of biochemical abnormalities and helps avoid disease progression.

PMID:40297984 | DOI:10.1016/j.gim.2025.101449

Crisis. 2025 Apr 29. doi: 10.1027/0227-5910/a000998. Online ahead of print.

ABSTRACT

Background: Youth suicidality presents challenges for healthcare workers, particularly in Nova Scotia, Canada, where rates exceed national averages. Professional confidence, comfort, and anxiety in managing suicidal youth may be associated with training and education. Aims: This study examined associations between healthcare workers’ educational backgrounds, their mental health training, and their perceptions, attitudes, and emotional responses when working with suicidal youth. Method: A cross-sectional survey of 123 healthcare workers in Nova Scotia assessed demographics, training, and clinical responses. Descriptive statistics, Pearson’s correlations, t-tests, and regression analyses (adjusting for experience) examined associations between training, confidence, comfort, and anxiety. Results: Participants reported moderate confidence (M = 3.7, SD = 0.9) and comfort (M = 3.7, SD = 0.9), with higher anxiety (M = 3.0, SD = 1.0), particularly in family-related contexts (M = 3.5, SD = 1.1). General training was associated with greater confidence and comfort but not significantly with anxiety. Specialized training (e.g., mental health degrees) was associated with lower anxiety and reduced avoidance of suicidal youth and families. Limitations: The cross-sectional design, self-report measures, and purposive sampling limit causal inference and generalizability. Conclusion: Findings suggest training type relates to professional responses, highlighting the need for further research on tailored educational interventions.

PMID:40297952 | DOI:10.1027/0227-5910/a000998

J Neonatal Perinatal Med. 2025 Apr 29:19345798251339650. doi: 10.1177/19345798251339650. Online ahead of print.

ABSTRACT

IntroductionInfants born less than 32-week gestation are at increased risk of respiratory distress syndrome due to underdeveloped pulmonary alveoli. Despite known respiratory benefits associated with positive end expiratory pressure (PEEP), our staff expressed concern that prolonged exposure to PEEP would contribute to increased risk of pneumothorax and delayed initiation of oral feeding.​ This quality improvement project aimed to standardize PEEP use with a guideline for early initiation, continuation, and subsequent discontinuation in infants born at less than 32-week gestation.MethodsWe developed and implemented a “Keep the PEEP” guideline, which included recommendations for initiation of PEEP at delivery, continuation through 32-week post-menstrual age (PMA), and discontinuation. Data collection occurred between July 2019-July 2021 (pre-intervention) and July 2021-August 2023 (post-intervention). Data analysis included run charts for monthly compliance, and bivariate analysis of pre- and post-intervention data. Interventions included team member education and team engagement through auditing.ResultsWe achieved a median compliance of 100% with PEEP use through 32-week PMA. We found no statistically significant differences in a pre- versus post-intervention comparison of pneumothorax incidence [14 versus 13, p = 0.86], bronchopulmonary dysplasia (BPD) incidence [82 versus 68, p = 0.65], median post-menstrual age at first oral feed [34.1 versus 34.1, p = 0.56], or median length of stay [55 versus 43, p = 0.2].ConclusionsWe successfully implemented a new respiratory guideline to standardize PEEP use in our unit during the study period. Despite initial concerns, the incidence of pneumothorax and the median gestational age at initiation of oral feedings remained unchanged.

PMID:40297950 | DOI:10.1177/19345798251339650

Facts Views Vis Obgyn. 2025 Mar 28;17(1):61-67. doi: 10.52054/FVVO.2024.13453.

ABSTRACT

BACKGROUND: Various surgical techniques for the treatment of colorectal endometriosis have been described, and the benefit of a preventive stoma remains unclear.

OBJECTIVES: The aim of our study is to evaluate the risk of complications in patients who underwent surgery for colorectal endometriosis without a policy of preventive stoma.

METHODS: Retrospective cohort study of 97 consecutive patients treated for colorectal endometriosis in an expert centre from January 2022 to January 2024.

MAIN OUTCOME MEASURES: Complications after colorectal endometriosis surgery in patients without preventive stoma.

RESULTS: Forty-three patients were managed by segmental resection, 20 patients by single-disc excision, 5 patients by double-disc excision and 29 patients by rectal shaving. 48 patients required vaginal suturing. We found complications in 14% of patients. Severe complications (Clavien-Dindo ≥3) were encountered in 8.24% of patients. 3.09% developed a rectovaginal fistula. Patients with a colorectal endometriosis nodule larger than 3 cm had more complications than patients with smaller nodules (57.1% vs. 42.9% of total complications), with a P-value close to the statistical significance.

CONCLUSIONS: Surgery for colorectal endometriosis performed in high-volume centres by expert surgeons leads to a reduction in the risk of postoperative complications. In our study, we did not perform routine preventive stoma formation, and we did not find an increase in postoperative complications compared to the literature.

WHAT IS NEW?: This study provides data on the risk of postoperative complications in patients undergoing surgery for colorectal endometriosis without a preventive stoma policy.

PMID:40297948 | DOI:10.52054/FVVO.2024.13453

Biometrics. 2025 Apr 2;81(2):ujaf045. doi: 10.1093/biomtc/ujaf045.

ABSTRACT

Confounding and exposure measurement error can introduce bias when drawing inference about the marginal effect of an exposure on an outcome of interest. While there are broad methodologies for addressing each source of bias individually, confounding and exposure measurement error frequently co-occur, and there is a need for methods that address them simultaneously. In this paper, corrected score methods are derived under classical additive measurement error to draw inference about marginal exposure effects using only measured variables. Three estimators are proposed based on g-formula, inverse probability weighting, and doubly-robust estimation techniques. The estimators are shown to be consistent and asymptotically normal, and the doubly-robust estimator is shown to exhibit its namesake property. The methods, which are implemented in the R package mismex, perform well in finite samples under both confounding and measurement error as demonstrated by simulation studies. The proposed doubly-robust estimator is applied to study the effects of two biomarkers on HIV-1 infection using data from the HVTN 505 preventative vaccine trial.

PMID:40297938 | DOI:10.1093/biomtc/ujaf045