JAMA Netw Open. 2025 Feb 3;8(2):e2460460. doi: 10.1001/jamanetworkopen.2024.60460.
NO ABSTRACT
PMID:39964687 | DOI:10.1001/jamanetworkopen.2024.60460
Tag: statistics
JAMA Netw Open. 2025 Feb 3;8(2):e2460087. doi: 10.1001/jamanetworkopen.2024.60087.
ABSTRACT
IMPORTANCE: Foot ulcers are a common and feared complication for people with diabetes because 20% of foot ulcers become infected and lead to a lower extremity amputation.
OBJECTIVE: To evaluate the effect of daily foot care using chlorhexidine wipes vs soap-and-water wipes for 1 year on the risk of developing new foot complications in veterans with diabetes.
DESIGN, SETTING, AND PARTICIPANTS: This double-blind, placebo-controlled, phase 2b randomized clinical trial was conducted at the Baltimore Veterans Affairs (VA) Medical Center between January 2019 to January 2023. Veterans were eligible if they had a diabetes diagnosis, were at high risk for diabetic foot complications, were ambulatory, had both feet, and did not have a current foot infection. Participants were randomly assigned 1:1 to receive either soap-and-water wipes (control group) or 2% chlorhexidine wipes (chlorhexidine group). Intention-to-treat data analysis was conducted from October 5, 2023, to April 24, 2024.
INTERVENTION: Daily use of a 2% chlorhexidine wipe or a soap-and-water wipe on the feet for 1 year. Wipes were nearly identical in color, size, shape, thickness, feel, and scent. Both chlorhexidine and control groups received the same lotion for application on the feet after wipe use and education on foot self-care.
MAIN OUTCOMES AND MEASURES: The primary outcome was time in days from randomization to new foot complication, including chronic foot ulcer, foot infection, or foot amputation.
RESULTS: A total of 175 participants (170 males [97%]; mean [SD] age at enrollment, 68 [9] years; 1 Asian [1%], 117 Black or African American [67%], 53 White [30%] individuals) were randomly assigned to the chlorhexidine group (n = 88) or the control group (n = 87). Twelve participants (14%) in the chlorhexidine group and 14 participants (16%) in the control group developed a new foot complication within 1 year. Median (IQR) time from randomization to development of a new foot complication was 232 (115-315) days. The reduction in hazard of new foot complications in the chlorhexidine group compared with the control group was not significant (hazard ratio, 0.83; 95% CI, 0.39-1.80). The intervention was well tolerated, with 145 participants (83%) continuing it over the study period. Sixty adverse events occurred, but none was related to the study products or procedures.
CONCLUSIONS AND RELEVANCE: This randomized clinical trial found that daily use of chlorhexidine wipes for foot washing for 1 year did not lead to a significant reduction in the risk of new foot complications compared with daily use of soap-and-water wipes. The intervention was well tolerated, and the trial provides important lessons for future studies on diabetic foot ulcer prevention.
TRIAL REGISTRATION: ClinicalTrials.gov Identifier: NCT03503370.
PMID:39964684 | DOI:10.1001/jamanetworkopen.2024.60087
JAMA Netw Open. 2025 Feb 3;8(2):e2460312. doi: 10.1001/jamanetworkopen.2024.60312.
ABSTRACT
IMPORTANCE: Metaplastic breast cancer (MpBC) is a rare, heterogeneous disease often associated with inferior outcomes. A growing body of literature describes the clinical and molecular features of MpBC, yet limited data describe the pathogenic germline variants (PGVs) in breast cancer susceptibility genes among affected individuals.
OBJECTIVE: To examine the frequency and types of PGVs in breast cancer genes among patients with MpBC.
DESIGN, SETTING, AND PARTICIPANTS: This is a descriptive retrospective cohort study of patients who received a diagnosis of MpBC at the University of Pennsylvania between January 2010 and May 2023. Electronic medical records were reviewed for demographic, clinicopathologic, and germline genetic testing information. Germline variant status was independently confirmed by a licensed genetic counselor and a physician with expertise in genetics. MpBC diagnosis and subtype were confirmed by a breast pathologist. Participants were identified via query of an institutional pathology database for reports signed between January 2010 and May 2023 including the term metaplastic. Among 320 initially obtained reports, 272 individuals had confirmed MpBC and were included in the study.
EXPOSURE: Germline genetic testing to investigate the presence of PGVs in breast cancer susceptibility genes.
MAIN OUTCOMES AND MEASURES: The primary outcome measurement was the prevalence of PGVs in breast cancer susceptibility genes among participants. The hypothesis that individuals with MpBC have an enrichment of PGVs in genes associated with inherited breast cancer risk was formulated before data collection.
RESULTS: The total sample size was 272 women, and the median age at diagnosis was 58 years (range, 20-102 years); all were biological female patients; 143 of 272 (52.6%) had documentation of germline genetic testing; and participants with testing were significantly younger than those without (median age, 53 years [range, 20-79 years] vs 63 years [range, 29-102 years]; P < .001). Of the 143 patients, 24 (16.8%) had a PGV in a breast cancer susceptibility gene (BRCA1, n = 17; BRCA2, n = 5; PALB2, n = 1; CHEK2, n = 1). Patients with PGV-associated MpBC received a diagnosis at a younger age than those with sporadic disease, but there were no significant differences in hormone receptor positivity, ERBB2 status, or metaplastic subtype.
CONCLUSIONS AND RELEVANCE: In this cohort study of patients with MpBC, a substantial proportion of clinically tested patients had a PGV in a breast cancer susceptibility gene, most commonly BRCA1. Germline testing was high yield in patients with MpBC, many of whom would be included in current germline testing eligibility criteria.
PMID:39964682 | DOI:10.1001/jamanetworkopen.2024.60312
JAMA Netw Open. 2025 Feb 3;8(2):e2460315. doi: 10.1001/jamanetworkopen.2024.60315.
ABSTRACT
IMPORTANCE: Acute pulmonary embolism (PE) is a major cause of morbidity and mortality in patients with cancer in the US and worldwide.
OBJECTIVES: To assess the trends in PE-related mortality from 2011 to 2020 among US patients with cancer across age, sex, ethnic and racial groups, urbanicity, and regionality.
DESIGN, SETTING, AND PARTICIPANTS: This cohort study used the Centers for Disease Control and Prevention Wide-Ranging Online Data for Epidemiologic Research data system to determine national trends in age-adjusted mortality rates (AAMRs) due to acute PE among US patients with cancer aged 15 years or older from January 2011 to December 2020. Concomitant trends in cancer mortality and incidence that may have contributed to PE-related mortality were obtained from US Cancer Statistics. Data were analyzed from September to November 2024.
EXPOSURE: PE-related mortality.
MAIN OUTCOMES AND MEASURES: The primary outcome was PE-related deaths among individuals with cancer. AAMRs and cancer incidence were assessed using joinpoint regression modeling, expressed as an average annual percentage change (AAPC) with relative 95% CIs.
RESULTS: From 2011 to 2020, a total of 27 280 194 individuals aged 15 years or older (13 897 519 male [50.9%]; 13 382 675 female [49.1%]) died in the US. The AAMR for PE-related mortality in patients with cancer increased during this time period (AAPC, 2.5%; 95% CI, 1.4% to 3.6%; P = .001), without differences between sexes (P for parallelism = .38). The AAMR increased among those aged 15 to 64 years (AAPC, 3.2%; 95% CI, 1.9% to 4.6%; P = .001), non-Hispanic and non-Latinx White individuals (AAPC, 2.7%; 95% CI, 1.52% to 3.94%; P = .001), non-Hispanic and non-Latinx Black or African American individuals (AAPC, 2.2%; 95% CI, 0.7% to 3.7%; P = .001), Hispanic and Latinx individuals (AAPC, 2.6%; 95% CI, 0.7% to 4.5%; P = .006), and among individuals residing in the Southern US (AAPC, 3.7%; 95% CI, 1.3% to 6.2%; P = .003). During the same period, age-adjusted cancer incidence and cancer-related mortality decreased while the absolute number of new cancer diagnoses and cancer-related deaths increased.
CONCLUSIONS AND RELEVANCE: This cohort study found that despite decreases in cancer-related mortality rates, age-adjusted PE-related mortality in US patients with cancer increased over the last decade; concerning trends included rising PE-related mortality in younger individuals aged 15 to 64 years, particular ethnic and racial groups, and the Southern region of the US. Recognition of such patterns may inform further research into thromboprophylaxis and treatment of PE as a complication of cancer and cancer-directed therapy.
PMID:39964681 | DOI:10.1001/jamanetworkopen.2024.60315
JAMA Netw Open. 2025 Feb 3;8(2):e2460465. doi: 10.1001/jamanetworkopen.2024.60465.
ABSTRACT
IMPORTANCE: Despite the importance of patient trust in health care, there are no patient-reported outcome measures (PROMs) for trust in their clinician that have been developed empirically in Spanish, which is the second most common language in the US.
OBJECTIVE: To develop and validate a Spanish-language PROM for trust in pregnancy care clinician.
DESIGN, SETTING, AND PARTICIPANTS: This cross-sectional study used a national online panel of patients who reported a Spanish language preference and had limited English proficiency and were currently pregnant or had given birth within the 12 months before the survey. Participants resided in the United States, and data were collected from January to May 2024.
EXPOSURES: Participants had clinical interactions during pregnancy and/or postpartum care. Data collected included demographics, Confianza (Trust) Scale candidate items, and 4 measures for concurrent validity evidence: the Trust in Physician Scale (TPS), the Mothers on Respect Index, the Edinburgh Postpartum Depression Scale (EPDS), and the Patient-Reported Outcomes Measurement Information System Global 10.
MAIN OUTCOMES AND MEASURES: The main outcomes were psychometric properties of the Confianza scale and its association with validated scales (validity coefficients). Item response theory (IRT) analyses were conducted to evaluate the psychometric properties of the candidate items, select the best item subset for the Confianza scale, examine its correlation with other measures, and compare scores according to demographic characteristics.
RESULTS: Of the included 204 participants (mean [SD] age, 26 [7] years; 62 participants from South America [30%]; 32 participants from Mexico [16%]), 117 participants were pregnant (57%), and 87 were within 1-year post partum (43%) at the time of survey completion. Four items were removed based on exploratory factor analysis. Using results from IRT analysis on the remaining 12 items, 5 items were selected to represent communication, caring, competency, accompaniment, and overall trust for the final measure. The 5-item Confianza scale had high measurement precision, with reliability above 0.90 across a wide range of the trust continuum. The Confianza scale (mean [SD] score, 21.5 [4.6] out of 25) was positively correlated with the TPS (r = 0.47; 95% CI, 0.36 to 0.57; P < .001) and negatively correlated with the EPDS (r = -0.41; 95% CI, -0.52 to -0.29; P <.001). Higher trust scores were obtained when there was language concordance with clinicians (mean [SD], 23.6 [2.3] vs 20.0 [5.3]; P < .001) and care continuity (mean [SD], 22.3 [3.8] vs 20.9 [5.3]; P = .001).
CONCLUSIONS AND RELEVANCE: In this cross-sectional study of pregnant and postpartum Spanish-speaking individuals, a Spanish-language PROM for trust in pregnancy care clinician had initial validity.
PMID:39964680 | DOI:10.1001/jamanetworkopen.2024.60465
JAMA Netw Open. 2025 Feb 3;8(2):e2460471. doi: 10.1001/jamanetworkopen.2024.60471.
ABSTRACT
IMPORTANCE: Research on cumulative disabilities and tobacco use in the current tobacco landscape is lacking.
OBJECTIVE: To examine prevalence of tobacco use across cumulative disabilities within and across years among US adults.
DESIGN, SETTING, AND PARTICIPANTS: Representative cross-sectional surveys of the US adult (aged ≥18 years) civilian, noninstitutionalized population from the Population Assessment of Tobacco and Health (PATH) Study were compared across 2013 to 2014 (wave 1), 2016 to 2018 (wave 4), and 2022 to 2023 (wave 7). Age-adjusted estimates and change scores were compared using design-based significance tests. Data were analyzed from November 2023 and April 2024.
MAIN OUTCOMES AND MEASURES: Past 30-day use of tobacco (cigarette, electronic nicotine [e-product], traditional cigar, filtered cigar, cigarillo, pipe, hookah, and smokeless products); disability types (mobility, self-care, independent living, cognitive, hearing, and vision) summed to generate a 4-level cumulative disability measure (0, 1, 2, or ≥3).
RESULTS: In 2013 to 2014, among 32 314 participants at wave 1, 3110 participants were 65 years or older (18.2%), 15 993 (51.9%) were female, 5536 (15.2%) were Hispanic, 4496 (11.2%) were non-Hispanic Black, 19 295 (66.0%) were non-Hispanic White, and 2428 (7.5%) were another non-Hispanic race. A total of 32 314 adults were included in 2013 to 2014, 33 638 in 2016 to 2018, and 30 681 in 2022 to 2023. In 2013 to 2014, any tobacco use prevalence for those with no disabilities was 25.4% (95% CI, 24.7% to 26.1%), while prevalence was 40.2% (95% CI, 38.6% to 41.8%), 48.9% (95% CI, 45.4% to 52.5%), and 51.8% (95% CI, 48.6% to 55.0%) for those with 1, 2, and 3 or more disabilities, respectively. Any tobacco, cigarette, e-product, cigarillo, filtered cigar, and pipe use were consistently higher among those with multiple disabilities compared with those with 0 or 1 disability. Prevalence was highest among those with 3 or more disabilities for all products. Tobacco use declined over time, except e-product use increased. However, not all declines in prevalence between 2013 to 2014 and 2022 to 2023 were significant among those with 3 or more disabilities. The declines for those who used any tobacco (-4.6%; 95% CI, -8.7% to -0.6%) and cigarettes (-8.9%; 95% CI, -12.7% to -5.1%) were small, and increases in e-product use were large (4.6%; 95% CI, 1.0% to 8.3%) for those with 3 or more disabilities compared with other groups.
CONCLUSIONS AND RELEVANCE: In this survey study, disparities in tobacco use existed across cumulative disabilities. Despite declines in tobacco use over time, differences in use across cumulative disabilities persisted or worsened, especially among those with 3 or more disabilities. Targeted prevention, screening, and cessation efforts inclusive of multiple products are needed.
PMID:39964679 | DOI:10.1001/jamanetworkopen.2024.60471
Stat Med. 2025 Feb 28;44(5):e10304. doi: 10.1002/sim.10304.
ABSTRACT
Stepped wedge cluster randomized trials (SWCRTs) often face challenges related to potential confounding by time. Traditional frequentist methods may not provide adequate coverage of an intervention’s true effect using confidence intervals, whereas Bayesian approaches show potential for better coverage of intervention effects. However, Bayesian methods remain underexplored in the context of SWCRTs. To bridge this gap, we propose two innovative Bayesian hierarchical penalized spline models. Our first model accommodates large numbers of clusters and time periods, focusing on immediate intervention effects. To evaluate this approach, we compared this model to traditional frequentist methods. We then extend our approach to account for time-varying intervention effects, conducting a comprehensive comparison with an existing Bayesian monotone effect curve model and alternative frequentist methods. The proposed models were applied in the Primary Palliative Care for Emergency Medicine stepped wedge trial to evaluate the effectiveness of the intervention. Through extensive simulations and real-world application, we demonstrate the robustness of our proposed Bayesian models. Notably, the Bayesian immediate effect model consistently achieves the nominal coverage probability, providing more reliable interval estimations while maintaining high estimation accuracy. Furthermore, our proposed Bayesian time-varying effect model represents a significant advancement over the existing Bayesian monotone effect curve model, offering improved accuracy and reliability in estimation while also achieving higher coverage probability than alternative frequentist methods. To the best of our knowledge, this marks the first development of Bayesian hierarchical spline modeling for SWCRTs. Our proposed models offer promising tools for researchers and practitioners, enabling more precise evaluation of intervention impacts.
PMID:39964677 | DOI:10.1002/sim.10304
Stat Med. 2025 Feb 28;44(5):e70004. doi: 10.1002/sim.70004.
ABSTRACT
For clinical prediction applications, we are often faced with small sample size data compared to the number of covariates. Such data pose problems for variable selection and prediction, especially when the covariate-response relationship is complicated. To address these challenges, we propose to incorporate external information on the covariates into Bayesian additive regression trees (BART), a sum-of-trees prediction model that utilizes priors on the tree parameters to prevent overfitting. To incorporate external information, an empirical Bayes (EB) framework is developed that estimates, assisted by a model, prior covariate weights in the BART model. The proposed EB framework enables the estimation of the other prior parameters of BART as well, rendering an appealing and computationally efficient alternative to cross-validation. We show that the method finds relevant covariates and that it improves prediction compared to default BART in simulations. If the covariate-response relationship is non-linear, the method benefits from the flexibility of BART to outperform regression-based learners. Finally, the benefit of incorporating external information is shown in an application to diffuse large B-cell lymphoma prognosis based on clinical covariates, gene mutations, DNA translocations, and DNA copy number data.
PMID:39964672 | DOI:10.1002/sim.70004
Obes Surg. 2025 Feb 18. doi: 10.1007/s11695-025-07743-6. Online ahead of print.
ABSTRACT
BACKGROUND: Laparoscopic Roux-En-Y Gastric Bypass (LRYGB) contributes significantly to higher weight loss at 6 to 12 months when compared to Laparoscopic Sleeve Gastrectomy (LSG) in patients with severe obesity (SO-body mass index (BMI) ≥ 50 kg/m2). However, there is still no consensus regarding the best procedure in terms of mortality and complication rates. We performed a systematic review and meta-analysis to compare the complication rates between these two surgical procedures.
METHODS: PubMed, EMBASE, and Cochrane Central were searched for studies that compared LRYGB and LSG in SO patients. We pooled outcomes for mortality and complications, defined as bleeding, cardiovascular events, conversion to open procedure, and a composite endpoint of leak, abscess, fistulas, and reoperation. Length of stay and operative time were also pooled. A random-effects model was used, and statistical analyses were performed using R version 4.4.0.
RESULTS: A total of 156,767 patients from 28 observational studies were included, of whom 79,324 (50.6%) underwent LRYGB and 77,443 (49.4%) LSG. Length of stay (MD 0.45; 95% CI 0.42-0.48; P < 0.01) and operative time (MD 58.88; 95% CI 37.88-79.87; P < 0.01) were lower in the LSG group. Overall, there was no difference in mortality (OR 1.28; 95% CI 0.80-2.04; P = 0.311) and in complication rates (OR 1.22; 95% CI 0.85-1.76; P = 0.287). A subgroup analysis showed lower conversion to open procedure for patients who underwent LSG (OR 2.75; 95% CI 1.90-3.98; P < 0.001), and no difference was noted in bleeding (OR 0.98; 95% CI 0.47-2.07; P = 0.965), cardiovascular events (OR 0.99; 95% CI 0.43-2.29; P = 0.983), and a composite endpoint of leak, abscess, and fistulas (OR 0.82; 95% CI 0.67-1.01; P = 0.066).
CONCLUSION: Our meta-analysis suggests that there is no difference in mortality and complication rates between the two groups. However, length of stay and operative time were lower in SO patients who underwent LSG.
PMID:39964665 | DOI:10.1007/s11695-025-07743-6
Jpn J Ophthalmol. 2025 Feb 18. doi: 10.1007/s10384-025-01166-7. Online ahead of print.
ABSTRACT
PURPOSE: This study investigated the long-term efficacy and safety of Autologous Platelet Concentrate (APC) injection as an adjunct to pars plana vitrectomy (PPV) in the treatment of macular holes (MHs) with a high risk of surgical failure, including large, highly myopic, or recurrent MHs.
STUDY DESIGN: Retrospective study.
METHODS: This study included patients who underwent PPV with or without APC injection for MHs at Seoul St. Mary’s Hospital between March 2017 and February 2020, with a minimum postoperative follow-up period of 3 years. Anatomical success rates, alterations in foveal microstructure, and improvements in best-corrected visual acuity (BCVA) were evaluated.
RESULTS: Fifty-four patients were included, exhibiting a trend toward enhanced anatomical success in the PPV with APC group (95.0%) compared to the PPV with flap techniques (85.7%) and PPV without the adjunct of flap techniques or APC injection (84.6%), though not statistically significant. Significant findings included increased glial proliferation within the PPV with APC group, sustained for up to one year (P < 0.01), and a significant correlation between glial proliferation and MH closure (P = 0.032). Improvements in BCVA were substantial and found to be significantly associated with preoperative BCVA, the reconstruction of the external limiting membrane (ELM) and ellipsoid zone (EZ) (P = 0.008, 0.010, and 0.047, respectively).
CONCLUSION: The use of APC as adjuvant to PPV can be advantageous in cases of challenging MHs, reflecting comparable anatomical success rates, potential for glial proliferation critical for hole closure, and established safety profile.
PMID:39964664 | DOI:10.1007/s10384-025-01166-7