Tag: statistics

JAMA Netw Open. 2025 Jan 2;8(1):e2452168. doi: 10.1001/jamanetworkopen.2024.52168.

ABSTRACT

IMPORTANCE: Spousal involvement in diabetes care is recommended theoretically, but effectiveness in clinical settings and among diverse populations is unclear.

OBJECTIVE: To test the effect of a couple-based intervention among Chinese older patients with type 2 diabetes and their spouses.

DESIGN, SETTING, AND PARTICIPANTS: This multicenter randomized clinical trial comprised 2 arms: a couple-based intervention arm and an individual-based control. The trial was conducted across 14 community health care centers in Guangzhou, China, between September 1, 2020, and June 30, 2022, and included patients with confirmed type 2 diabetes aged 55 years or older. Eligible partners were married to or cohabitated with the patients. Patients who previously participated in type 2 diabetes education courses were excluded, as were couples who both had diabetes, to make a clear distinction between patients and spouses. The data were analyzed between January 2023 and April 2024.

INTERVENTIONS: The interventions consisted of 4 weekly group education sessions followed by behavior change booster telephone calls over 2 months that targeted either patients and spouses (ie, intervention arm) or patients alone (ie, control arm). Follow-up assessments were conducted at 6 and 12 months post intervention.

MAIN OUTCOMES AND MEASURES: The primary outcome was hemoglobin A1c (HbA1c) levels for patients and quality of life for their spouses. Collective efficacy and behaviors were secondary outcomes for both patients and spouses. Group comparisons were conducted using multilevel models based on an intention-to-treat approach, with outcome measures assessed for both patients and their spouses.

RESULTS: A total of 207 couples were included in the study, with 106 randomized to the intervention arm and 101 to the control arm. The mean (SD) age of patients was 66.0 (6.5) years, with 105 (50.7%) being men; spouses had similar demographics. Patients’ HbA1c levels decreased in both arms over the 12-month follow-up, with no significant between-arm differences (β = -0.08; 95% CI, -0.57 to 0.42). Collective efficacy and collective behavior for patients increased after intervention but with a similar magnitude between arms. None of these measures showed between-arm differences among spouses in either arm. In subgroup analysis, decreases in HbA1c levels were constant and lasting in patients with high baseline HbA1c levels (≥8.0%) with a statistically significant difference.

CONCLUSIONS AND RELEVANCE: These findings show that the overall treatment effect of the couple-based intervention was weak. However, the couple-based intervention benefited patients with poor glucose control. Patients’ glucose levels, spouses’ availability to provide support, and couples’ collaborative preferences for mutual or individual diabetes management should be considered in tailoring treatment strategies among older adults with type 2 diabetes.

TRIAL REGISTRATION: Chinese Clinical Trial Registry Identifier: ChiCTR1900027137.

PMID:39745703 | DOI:10.1001/jamanetworkopen.2024.52168

JAMA Netw Open. 2025 Jan 2;8(1):e2452677. doi: 10.1001/jamanetworkopen.2024.52677.

ABSTRACT

IMPORTANCE: Influenza vaccination remains the most important intervention to prevent influenza morbidity and mortality among nursing home residents. The additional effectiveness of recombinant influenza vaccine vs standard dose vaccines was demonstrated in outpatient older adults but has not been evaluated in nursing home populations.

OBJECTIVE: To compare hospitalization rates among residents in nursing homes immunized with a recombinant vs a standard dose egg-based influenza vaccine.

DESIGN, SETTING, AND PARTICIPANTS: This pragmatic cluster randomized trial assessed nursing home residents 65 years or older residing in a US facility for 100 or more days before the start of influenza season (October 1). The study was conducted across the 2019 to 2020 and 2020 to 2021 influenza seasons and randomly assigned nursing homes 1:1 within blocks categorized by proportion of Black residents and prior resident hospitalization rates. Medicare claims data were used to evaluate resident-level hospitalization outcomes. Enrollment and allocation to treatment groups began on July 20, 2019. Data analysis began on January 1, 2021, with primary end points finalized June 30, 2024.

INTERVENTION: Nursing homes were cluster randomized to vaccinate all residents with recombinant quadrivalent influenza vaccine (RIV4) or standard egg-based quadrivalent inactivated influenza vaccine (IIV4).

MAIN OUTCOME AND MEASURES: The primary outcome was respiratory-related hospitalization. Secondary outcomes included death and hospitalization due to any cause.

RESULTS: A total of 144 565 person observations (mean [SD] age, 77.4 [13.1] years; 63.0% female) at 1078 nursing homes were included, with 72 005 residents in nursing homes randomized to provide RIV4 and 72 560 residents in nursing home randomized to provide IIV4. In total, 85.6% of the residents received influenza vaccination. Baseline resident characteristics were comparable across treatment groups. For the primary end point of respiratory-related hospitalizations, there were 1387 hospitalizations (1.9%) in the RIV4 group vs 1424 (2.0%) in the IIV4 group (hazard ratio, 1.01; 95% CI, 0.62-2.17). Hospitalization rates by vaccine were similar for other hospitalization outcomes and death, overall, and by season and subgroups (gender, race, and comorbidities).

CONCLUSIONS AND RELEVANCE: In this cluster randomized trial of nursing homes, there was no significant difference between recombinant or standard dose vaccine for reducing hospitalizations associated with influenza illness. However, the COVID-19 pandemic restricting influenza activity along with poor vaccine match to circulating strains substantially limits the conclusions.

TRIAL REGISTRATION: Clinicaltrials.gov Identifier: NCT03965195.

PMID:39745702 | DOI:10.1001/jamanetworkopen.2024.52677

JAMA Netw Open. 2025 Jan 2;8(1):e2452821. doi: 10.1001/jamanetworkopen.2024.52821.

ABSTRACT

IMPORTANCE: Evolving breast cancer treatments have led to improved outcomes but carry a substantial financial burden. The association of treatment costs with the cost-effectiveness of screening mammography is unknown.

OBJECTIVE: To determine the cost-effectiveness of population-based breast cancer screening in the context of current treatment standards.

DESIGN, SETTING, AND PARTICIPANTS: In this economic evaluation, the Canadian Partnership Against Cancer/Statistics Canada OncoSim-Breast microsimulation model was used to estimate the impact of various screening schedules in terms of clinical outcomes and treatment costs. Breast cancer treatment costs were derived from activity-based costing published in 2023 specific to a publicly funded health system in Ontario, Canada. A single birth cohort of individuals assigned female at birth in 1975 was modeled until death or age 99 years (whichever came first).

EXPOSURES: Five screening scenarios were modeled: no screening, biennial (ages 50-74 years and 40-74 years), hybrid (biennial ages 40-49 years and annual ages 50-74 years), and annual screening (ages 40-74 years).

MAIN OUTCOMES AND MEASURES: Incremental cost-effectiveness ratios for deaths averted, life-years (LYs) gained, and incremental cost-utility ratios for quality-adjusted life-years (QALYs) gained were determined for screening scenarios. Sensitivity analyses were conducted by varying screening participation rates and reducing recall rates to 5% and the estimated mortality benefits of screening.

RESULTS: Earlier initiation of breast cancer screening at age 40 years (vs age 50 years) was associated with improved clinical outcomes (deaths averted, LYs saved, and QALYs gained) and reduced health care spending on breast cancer treatment. From a health system perspective, incremental cost-effectiveness ratios for biennial screening at ages 40 to 74 years compared with biennial screening at ages 50 to 74 years were cost saving, with CAD$49 759 saved per death averted, $1558 per LY saved, and $2007 saved per QALY gained. Annual screening at ages 40 to 74 years was cost-effective while achieving the best breast cancer outcomes, with costs of $25 501 per death averted, $1100 per LY saved, and $1447 per QALY gained compared with the current Canadian standard of biennial screening at ages 50 to 74 years.

CONCLUSIONS AND RELEVANCE: In this economic analysis, although screening costs increased according to the number of lifetime screens, they were completely or largely offset by reduced breast cancer therapy costs. Digital mammography was a highly cost-effective tool to reduce breast cancer mortality. These results have important policy implications for all single-payer health systems and call for greater investment in screening programs.

PMID:39745700 | DOI:10.1001/jamanetworkopen.2024.52821

JAMA Netw Open. 2025 Jan 2;8(1):e2452890. doi: 10.1001/jamanetworkopen.2024.52890.

ABSTRACT

IMPORTANCE: Cardiovascular disease (CVD) and cancer are the leading causes of mortality in the US. Large-scale population-based and mechanistic studies support a direct effect of CVD on accelerated tumor growth and spread, specifically in breast cancer.

OBJECTIVE: To assess whether individuals presenting with advanced breast cancers are more likely to have prevalent CVD compared with those with early-stage breast cancers at the time of diagnosis.

DESIGN, SETTING, AND PARTICIPANTS: This population-based case-control study used data from the Surveillance, Epidemiology, and End Results-Medicare linked databases from 2009 to 2020. The analysis was completed from May 2023 to August 2024. Participants were female patients aged at least 66 years diagnosed with invasive breast cancer. Cases were matched with controls by breast cancer stage at diagnosis and propensity scores using factors known to be associated with delayed cancer diagnosis.

EXPOSURE: Prevalent CVD prior to breast cancer diagnosis.

MAIN OUTCOMES AND MEASURES: The outcome of interest was the odds of locally advanced (T3-4 or N+) or metastatic (M+) breast cancer status at diagnosis.

RESULTS: The full analytic cohort included 19 292 matched individuals, with median (IQR) age 73 (70-79) years, of whom 1676 (8.7%) were Black and 16 681 (86.5%) were White; 9478 individuals (49.1%) had prevalent CVD. Propensity score-matched, multivariable-adjusted models found that individuals with locally advanced or metastatic breast cancer at diagnosis had statistically significantly increased odds of prevalent CVD (odds ratio [OR], 1.10; 95% CI, 1.03-1.17; P = .007). This association was observed among hormone receptor-positive (OR, 1.11; 95% CI, 1.03-1.19; P = .006) but not hormone receptor-negative (OR, 1.02; 95% CI, 0.86-1.21; P = .83) breast cancer. ORs were directionally consistent when separately examining locally advanced (OR, 1.09; 95% CI, 1.02-1.17; P = .02) and metastatic (OR, 1.20; 95% CI, 0.94-1.54; P = .15) disease, among all receptor subtypes.

CONCLUSIONS AND RELEVANCE: This case-control study found that individuals with more advanced breast cancer at diagnosis were more likely to have prevalent CVD. This finding may be specific to hormone receptor-positive and ERBB2-negative (formerly HER2) disease. Future studies are needed to confirm these findings and investigate interventions to improve patient outcomes, including personalized cancer screening.

PMID:39745699 | DOI:10.1001/jamanetworkopen.2024.52890

JAMA Surg. 2025 Jan 2. doi: 10.1001/jamasurg.2024.5920. Online ahead of print.

ABSTRACT

IMPORTANCE: Growing trends in private equity acquisition of acute care hospitals in the US have motivated investigations into quality of care delivered at these health centers. While some studies have explored comparative outcomes for high-acuity medical conditions, care trends and outcomes of complex surgical procedures, such as esophagectomy, at private equity-acquired hospitals is unknown.

OBJECTIVE: To compare structural characteristics and postoperative outcomes following esophagectomy between private equity-acquired and nonacquired health centers.

DESIGN, SETTING, AND PARTICIPANTS: This retrospective cohort study included Medicare beneficiaries aged 65 to 99 years who underwent elective esophagectomy at US health centers between January 1, 2016, and December 31, 2020. Health centers were designated as private equity acquired using the Agency for Healthcare Research and Quality Compendium of US Health Systems. Data were analyzed between October 15, 2023, and March 30, 2024.

EXPOSURE: Patient cohorts were created based on whether they received care at private equity-acquired or nonacquired health centers.

MAIN OUTCOMES AND MEASURES: The main outcome was 30-day postoperative complications, mortality, failure to rescue, and readmission using summary statistics and multivariable logistic regression.

RESULTS: A total of 9462 patients (mean [SD] age, 72.9 [5.6] years; 6970 male [73.7%]) underwent esophagectomy during the study period, with 517 (5.5%) receiving care at private equity-acquired institutions. Annual procedure volume was lower at private equity-acquired hospitals vs nonacquired hospitals (median, 2 [IQR, 1-4] vs 7 [IQR, 3-15] procedures per year). Compared with patients treated at nonacquired hospitals, patients treated at private equity-acquired hospitals had significantly higher 30-day mortality (8.1% [95% CI, 5.8%-10.3%] vs 4.9% [95% CI, 4.5%-5.3%]; odds ratio [OR], 1.82 [95% CI, 1.25-2.64]; P = .002), any complications (36.6% [95% CI, 32.9%-40.3%] vs 30.1% [95% CI, 29.2%-30.9%]; OR, 1.46 [95% CI, 1.18-1.80]), serious complications (17.5% [95% CI, 14.5%-20.6%] vs 14.3% [95% CI, 13.7%-15.0%]; OR, 1.34 [95% CI, 1.03-1.77]; P = .03), and failure to rescue (5.9% [95% CI, 3.9%-7.9%] vs 3.4% [95% CI, 3.1%-3.8%]; OR, 1.86 [95% CI, 1.22-2.84]; P = .004).

CONCLUSIONS AND RELEVANCE: These findings suggest that patients who undergo esophagectomy at private equity-acquired hospitals may be at risk for worse outcomes. Further understanding of the drivers of these outcomes is needed to improve performance and inform policy pertaining to care allocation for select surgical conditions.

PMID:39745696 | DOI:10.1001/jamasurg.2024.5920

JAMA Oncol. 2025 Jan 2. doi: 10.1001/jamaoncol.2024.5636. Online ahead of print.

ABSTRACT

IMPORTANCE: The current standard-of-care salvage therapy in relapsed/refractory classic Hodgkin lymphoma (cHL) includes consolidation high-dose chemotherapy (HDCT)/autologous stem cell transplant (aSCT).

OBJECTIVE: To investigate whether presalvage risk factors and fludeoxyglucose-18 (FDG) positron emission tomography (PET) response to reinduction chemotherapy can guide escalation or de-escalation between HDCT/aSCT or transplant-free consolidation with radiotherapy to minimize toxic effects while maintaining high cure rates.

DESIGN, SETTING, AND PARTICIPANTS: EuroNet-PHL-R1 was a nonrandomized clinical trial that enrolled patients younger than 18 years with first relapsed/refractory cHL across 68 sites in 13 countries in Europe between January 2007 and January 2013. Data were analyzed between September 2022 and July 2024.

INTERVENTION: Reinduction chemotherapy consisted of alternating IEP (ifosfamide, etoposide, prednisolone) and ABVD (adriamycin, bleomycin, vinblastine, dacarbazine). Patients with low-risk disease (late relapse after 2 cycles of first-line chemotherapy and any relapse with an adequate response after 1 IEP/ABVD defined as complete metabolic response on FDG-PET and at least 50% volume reduction) received a second IEP/ABVD cycle and radiotherapy (RT) to all sites involved at relapse. Patients with high-risk disease (all primary progressions and relapses with inadequate response after 1 IEP/ABVD cycle) received a second IEP/ABVD cycle plus HDCT/aSCT with or without RT.

MAIN OUTCOMES AND MEASURES: The primary end point was 5-year event-free survival. Secondary end points were overall survival (OS) and progression-free survival (PFS). PFS was identical to event-free survival because no secondary cancers were observed. PFS data alone are presented for simplicity.

RESULTS: Of 118 patients analyzed, 58 (49.2%) were female, and the median (IQR) age was 16.3 () years. The median (IQR) follow-up was 67.5 (58.5-77.0) months. The overall 5-year PFS was 71.3% (95% CI, 63.5%-80.1%), and OS was 82.7% (95% CI, 75.8%-90.1%). For patients in the low-risk group (n = 59), 41 received nontransplant salvage with a 5-year PFS of 89.7% (95% CI, 80.7%-99.8%) and OS of 97.4% (95% CI, 92.6%-100%). In contrast, 18 received HDCT/aSCT off protocol, with a 5-year PFS of 88.9% (95% CI, 75.5%-100%) and OS of 100%. All 59 patients with high-risk disease received HDCT/aSCT (and 23 received post-HDCT/aSCT RT) with a 5-year PFS of 53.3% (95% CI, 41.8%-67.9%) and OS of 66.5% (95% CI, 54.9%-80.5%).

CONCLUSION AND RELEVANCE: In this nonrandomized clinical trial, FDG-PET response-guided salvage in relapsed cHL may identify patients in whom transplant-free salvage achieves excellent outcomes. HDCT/aSCT may be reserved for primary progression and relapsed cHL with inadequate response.

TRIAL REGISTRATION: ClinicalTrials.gov Identifier: NCT00433459.

PMID:39745682 | DOI:10.1001/jamaoncol.2024.5636

Invest Ophthalmol Vis Sci. 2025 Jan 2;66(1):2. doi: 10.1167/iovs.66.1.2.

ABSTRACT

PURPOSE: To investigate the 10-year changes in visual function and incidence of visual impairment (VI) in highly myopic eyes.

METHODS: This longitudinal study enrolled highly myopic individuals who were followed up for 10 years. All participants underwent detailed ophthalmic examinations at baseline and follow-up visits. Best-corrected visual acuity (BCVA) was measured using Early Treatment of Diabetic Retinopathy Study protocol, and visual field (VF) was assessed with standard automatic perimetry.

RESULTS: A total of 568 highly myopic eyes (284 participants) were included, with mean baseline age of 22.49 ± 13.07 years, spherical equivalent refraction (SER) of -9.72 ± 3.02 D, and axial length of 27.39 ± 1.53 mm. Over 10 years, the mean BCVA loss was -0.06 logMAR (95% confidence internal [CI], 0.05-0.07). The mean change rates in mean deviation (MD) and pattern standard deviation over time were -0.07 dB/y (95% CI, -0.08 to -0.06) and 0.036 dB/y (95% CI, 0.028 to 0.044), respectively. The 10-year incidence of monocular moderate and severe VI (MSVI), per World Health Organization (WHO) and US criteria, was 3.52% (95% CI, 2.16%-5.39%) and 6.35% (95% CI, 4.46%-8.72%). Higher MSVI incidence, defined by WHO and US criteria, was associated with more myopic SER and lower baseline MD. Additionally, higher US-defined MSVI incidence was correlated with worse baseline BCVA.

CONCLUSIONS: In a highly myopic population, both BCVA and VF deteriorated over time, with increasing MSVI incidence. Raising public awareness of vision risks linked to high myopia and implementing strategies to reduce the burden in high-risk individuals are essential.

PMID:39745680 | DOI:10.1167/iovs.66.1.2

Health Psychol. 2025 Jan 2. doi: 10.1037/hea0001410. Online ahead of print.

ABSTRACT

OBJECTIVE: Although sexual minority men experience substantial discrimination, in addition to increased risk for several serious mental and somatic health problems, the biological mechanisms underlying these effects are unclear. To address this issue, we examined how experiences of social safety (i.e., community connection) and social threat (i.e., discrimination, in the forms of homophobia and racism) were related to conserved transcriptional response to adversity (CTRA) gene expression profiles across time, and whether these associations differed across HIV status, in a well-characterized, racially diverse sample of sexual minority men (M_age = 22.61, SD = 1.90).

METHOD: Experiences of community connection, homophobia, and racism were assessed via self-report, and blood samples were obtained at three timepoints over approximately 2 years. We then used these blood samples to characterize participants’ CTRA gene expression, which we quantified using an a priori 53-transcript composite score derived from RNA sequencing data from peripheral blood leukocytes.

RESULTS: As hypothesized, greater community connection was significantly related to decreased CTRA gene expression across time. These effects were similar regardless of HIV status and were robust to statistical adjustment for several potential confounding factors. In contrast, neither homophobia nor racism were related to CTRA gene expression.

CONCLUSION: These results suggest that community connection may be a protective factor that reduces biological processes known to negatively impact health. Consequently, interventions and policies aimed at reducing health disparities in marginalized populations may benefit from increasing community connection and inclusion. (PsycInfo Database Record (c) 2025 APA, all rights reserved).

PMID:39745665 | DOI:10.1037/hea0001410

Methods Mol Biol. 2025;2886:177-199. doi: 10.1007/978-1-0716-4310-5_9.

ABSTRACT

Measurements of cell phylogeny based on natural or induced mutations, known as lineage barcodes, in conjunction with molecular phenotype have become increasingly feasible for a large number of single cells. In this chapter, we delve into Quantitative Fate Mapping (QFM) and its computational pipeline, which enables the interrogation of the dynamics of progenitor cells and their fate restriction during development. The methods described here include inferring cell phylogeny with the Phylotime model, and reconstructing progenitor state hierarchy, commitment time, population size, and commitment bias with the ICE-FASE algorithm. Evaluation of adequate sampling based on progenitor state coverage statistics is emphasized for interpreting the QFM results. Overall, this chapter describes a general framework for characterizing the dynamics of cell fate changes using lineage barcoding data.

PMID:39745641 | DOI:10.1007/978-1-0716-4310-5_9

Eur J Pediatr. 2025 Jan 2;184(1):114. doi: 10.1007/s00431-024-05923-7.

ABSTRACT

Knowledge about trends and epidemiology of pediatric burns is useful to identify patterns, to advance medical research, and to design prevention programs and resource allocation. The aim of this study is to describe the epidemiology and trends of pediatric burns between 2009 and 2022 in the three Dutch burn centers. A secondary objective of this study is to evaluate the influence of the COVID-19 pandemic on the pattern of pediatric burns. A register-based cohort study was conducted based on data from the Dutch Burn Repository R3. Patients between 0 and 17 years at the time of the burn injury admitted between 2009 and 2022 to one of the three burn centers were included. Descriptive statistics were used to investigate the incidence and patient, burn, and treatment characteristics. The COVID-19 pandemic years (2020-2021) were compared with the pooled results from pre-COVID-19 years (2017-2019). A total of 4017 patients were included in this study, of which 3085 (77%) were overnight admissions. The incidence and absolute number of pediatric burn admissions gradually increased over the years, with a small temporary decrease in 2020-2021. Patient and burn characteristics remained relatively consistent over the years. Three quarters of all patients were between 0 and 3 years old, and the majority were boys (59%). A decreasing ratio of length of stay per % total burned surface area and an increase in day admissions was observed since 2016 onwards, which intensified during the COVID-19 pandemic.

CONCLUSIONS: There was a slight increase in the number of pediatric admissions to the burn centers between 2009 and 2022. Young children (0-3 years) remain the most frequently affected group. A shorter relative length of stay was observed, as well as an increase in day admissions, which was reinforced by the COVID-19 pandemic.

WHAT IS KNOWN: • Burns are a leading cause of death and disability among children globally. The largest group affected are young children (0-3 years) and most are scald burns. • In the Netherlands, since the late nineties there has been a trend towards more admissions to specialized burn centers, especially for young children with less severe burns.

WHAT IS NEW: • There was a slight increase in the number of pediatric admissions to the burn centers between 2009 and 2022, with a temporary decrease during the COVID-19 pandemic (2020-2021). • Admissions now tend to be shorter per percentage total burned surface area (TBSA), with an increase in day admissions, which intensified during the COVID-19 years.

PMID:39745598 | DOI:10.1007/s00431-024-05923-7