Category: Statistics

JAMA Intern Med. 2024 Nov 25. doi: 10.1001/jamainternmed.2024.6212. Online ahead of print.

ABSTRACT

IMPORTANCE: Clinicians often approach urate-lowering therapy (ULT) cautiously in patients with gout and impaired kidney function because they are concerned about the risk of progression to severe or end-stage kidney disease. However, evidence from randomized clinical trials of this association remains inconclusive.

OBJECTIVE: To evaluate the association between achieving target serum urate level with ULT and progression of chronic kidney disease (CKD) to severe or end-stage in patients with gout and impaired kidney function.

DESIGN, SETTING, AND PARTICIPANTS: This was a cohort study using the target trial emulation approach using data from a general practice database (IQVIA Medical Research Database) for 2000 to 2023. Eligible patients were 40 to 89 years old and had gout and CKD stage 3. Data analyses were performed from November 2023 to September 2024.

EXPOSURES: Lowering serum urate level to target level (<6 mg/dL) using ULT.

MAIN OUTCOMES AND MEASURES: Severe or end-stage kidney disease, determined by an estimated glomerular filtration rate of less than 30 mL/min/1.73 m2 on at least 2 occasions more than 90 days apart within 1 year, or at least 1 Read code (per the Refined Etiology, Anatomical Site, and Diagnosis classification) for CKD stages 4 or 5, hemodialysis, peritoneal dialysis, or kidney transplant. The prespecified noninferiority margin of the hazard ratio (HR) was set at 1.2, comparing those who achieved the target serum urate level with those who did not.

RESULTS: Among the 14 792 participants (mean [SD] age, 73.1 [9.5] years; 9215 men [62.3%] and 5577 women [37.7%]) with gout and with CKD stage 3, the 5-year risk of severe or end-stage kidney disease was 10.32% for those who achieved the target serum urate level and 12.73% for those who did not. Compared with those not achieving the target level, the adjusted 5-year risk difference and HR of severe or end-stage kidney disease for patients achieving the target serum urate level was -2.41% (95% CI, -4.61% to -0.21%) and 0.89 (95% CI, 0.80 to 0.98), respectively.

CONCLUSIONS AND RELEVANCE: The findings of this cohort study indicate that in patients with gout and CKD stage 3, lowering serum urate level to less than 6 mg/dL vs 6 mg/dL or greater using ULT was not associated with an increased risk of severe or end-stage kidney disease. These findings support optimizing ULT to achieve target serum urate levels when treating patients with gout and impaired kidney function.

PMID:39585678 | DOI:10.1001/jamainternmed.2024.6212

JAMA Intern Med. 2024 Nov 25. doi: 10.1001/jamainternmed.2024.5451. Online ahead of print.

ABSTRACT

IMPORTANCE: There is increased interest in public reporting of, and linking financial incentives to, the performance of organizations on health equity metrics, but variation across organizations could reflect differences in performance or selection bias.

OBJECTIVE: To assess whether differences across health plans in sex- and age-adjusted racial disparities are associated with performance or selection bias.

DESIGN, SETTING, AND PARTICIPANTS: This cross-sectional study leveraged a natural experiment, wherein a southern US state randomly assigned much of its Medicaid population to 1 of 5 plans after shifting to managed care in 2012. Enrollee-level administrative claims and enrollment data from 2011 to 2015 were obtained for self-identified Black and White enrollees. The analyses were limited to Black and White Medicaid enrollees because they accounted for the largest percentages of the population and could be compared with greater statistical power than other groups. Data were analyzed from June 2021 to September 2024.

EXPOSURES: Plan enrollment via self-selection (observational population) vs random assignment (randomized population).

MAIN OUTCOMES AND MEASURES: Annual counts of primary care visits, low-acuity emergency department visits, prescription drug fills, and total spending. For observational and randomized populations, models of each outcome were fit as a function of plan indicators, indicators for race, interactions between plan indicators and race, and age and sex. Models estimated the magnitude of racial differences within each plan and tested whether this magnitude varied across plans.

RESULTS: Of 118 101 enrollees (mean [SD] age, 9.3 [7.5] years; 53.0% female; 61.4% non-Hispanic Black; and 38.6% non-Hispanic White), 70.2% were included in the randomized population, and 29.8% were included in the observational population. Within-plan differences in primary care visits, low-acuity emergency department visits, prescription drug use, and total spending between Black and White enrollees were large but did not vary substantially and were not statistically significantly different across plans in the randomized population, suggesting minimal effects of plans on racial differences in these measures. In contrast, in the observational population, racial differences varied substantially across plans (standard deviations 2-3 times greater than in the randomized population); this variation was statistically significant after adjustment for multiple testing, except for emergency department visits. Greater between-plan variation in racial differences in the observational population was only partially explained by sampling error. Stratifying by race did not bring observational estimates of plan effects meaningfully closer to randomized estimates.

CONCLUSIONS AND RELEVANCE: This cross-sectional study showed that selection bias may mischaracterize plans’ relative performance on measures of health care disparities. It is critical to address disparities in Medicaid, but adjusting plan payments based on disparity measures may have unintended consequences.

PMID:39585673 | DOI:10.1001/jamainternmed.2024.5451

JAMA Neurol. 2024 Nov 25. doi: 10.1001/jamaneurol.2024.3976. Online ahead of print.

ABSTRACT

IMPORTANCE: Several psychiatric disorders have been found to occur more frequently in persons with epilepsy (PWE) than in persons without epilepsy.

OBJECTIVE: To summarize the prevalence of 20 psychiatric disorders in PWE compared with persons without epilepsy.

DATA SOURCES: The search included records from inception to February 2024 in Ovid, MEDLINE, Embase, and PsycINFO.

STUDY SELECTION: Published epidemiological studies examining the prevalence of psychiatric disorders among PWE compared with persons without epilepsy were systematically reviewed. There were no restrictions on language or publication date.

DATA EXTRACTION AND SYNTHESIS: Abstracts were reviewed in duplicate, and data were extracted using a standardized electronic form. Descriptive statistics and meta-analyses are presented.

MAIN OUTCOMES AND MEASURES: Data were recorded on the prevalence of 20 psychiatric disorders among PWE compared with persons without epilepsy. Meta-analyses were performed along with descriptive analyses.

RESULTS: The systematic search identified 10 392 studies, 27 of which met eligibility criteria. The meta-analyses included 565 443 PWE and 13 434 208 persons without epilepsy. The odds of most psychiatric disorders studied were significantly increased in PWE compared with those without epilepsy, including anxiety (odds ratio [OR], 2.11; 95% CI, 1.73-2.58); depression (OR, 2.45; 95% CI, 1.94-3.09); bipolar disorder (OR, 3.12; 95% CI, 2.23-4.36); suicidal ideation (OR, 2.25; 95% CI, 1.75-2.88) but not suicide attempt (OR, 3.17; 95% CI, 0.49-20.46); psychotic disorder (OR, 3.98; 95% CI, 2.57-6.15); schizophrenia (OR, 3.72; 95% CI, 2.44-5.67); obsessive-compulsive disorder (OR, 2.71; 95% CI, 1.76-4.15); posttraumatic stress disorder (OR, 1.76; 95% CI, 1.14-2.73); eating disorders (OR, 1.87; 95% CI, 1.73-2.01); alcohol misuse (OR, 3.64; 95% CI, 2.27-5.83) and alcohol dependence (OR, 4.94; 95% CI, 3.50-6.96) but not alcohol abuse (OR, 2.10; 95% CI, 0.60-7.37); substance use disorder (OR, 2.75; 95% CI, 1.61-4.72); autism spectrum disorder (OR, 10.67; 95% CI, 6.35-17.91); and attention-deficit/hyperactivity disorder (OR, 3.93; 95% CI, 3.80-4.08).

CONCLUSIONS AND RELEVANCE: In this comprehensive study, most psychiatric comorbidities examined were significantly more prevalent in PWE than in those without epilepsy. These findings show the high burden of psychiatric comorbidities in PWE. This, in turn, underscores the need for appropriately identifying and treating psychiatric comorbidity in epilepsy to manage patients effectively and improve quality of life.

PMID:39585664 | DOI:10.1001/jamaneurol.2024.3976

JAMA Pediatr. 2024 Nov 25. doi: 10.1001/jamapediatrics.2024.4782. Online ahead of print.

ABSTRACT

IMPORTANCE: Taxation of sweetened beverages is a proposed strategy to reduce excess sugar consumption. The association of such taxes with health outcomes is not well studied. Philadelphia, Pennsylvania, is the largest US city with a beverage tax.

OBJECTIVE: To assess whether the 2017 Philadelphia beverage tax was associated with changes in pediatric weight outcomes.

DESIGN, SETTING, AND PARTICIPANTS: This study used difference-in-differences models weighted by inverse probability of treatment weights to adjust for differences between youth in Philadelphia (tax exposed) and in the surrounding counties (control) on age, sex, race, ethnicity, Medicaid insurance status, health care use, and census-tract socioeconomic index. Mixed-effects linear and logistic regression models estimated differences in posttax changes in standardized body mass index (zBMI) and prevalence of obesity (a BMI 95th percentile or higher for age and sex) between Philadelphia and control. Stratified analyses assessed differences by age, sex, race, Medicaid insurance status, and baseline weight. Data came from electronic health records of a primary care network operating in the Philadelphia region. A panel analysis included youth 2 to 18 years old with 1 or more BMI measurement pretax (2014 to 2016) and 1 or more BMI measurement posttax (2018 to 2019). A cross-sectional analysis included youth 2 to 18 years old with 1 or more BMI measurement at any time from 2014 to 2019. These data were analyzed from December 2020 through July 2024.

EXPOSURE: Living in Philadelphia after implementation of the beverage tax.

MAIN OUTCOMES AND MEASURES: zBMI and obesity prevalence.

RESULTS: In panel analysis of 136 078 youth, the tax was associated with a difference in zBMI change of -0.004 (95% CI, -0.009 to 0.001) between Philadelphia and the control and a 1.02 odds ratio (95% CI, 0.97-1.08) of BMIs in the 95th percentile or higher. In cross-sectional analysis of 258 584 youth, the difference in zBMI change was -0.004 (95% CI, -0.009 to 0.001) and the odds ratio of a BMI in the 95th percentile or higher was 1.01 (95% CI, 0.95-1.07). In subgroup analyses, some differences in zBMI change were evident by race, age, Medicaid insurance status, and baseline weight but these differences were small and inconsistent across samples.

CONCLUSIONS AND RELEVANCE: These results show that 2 years after implementation, the Philadelphia beverage tax was not associated with changes in youth zBMI or obesity prevalence. Though certain subgroups demonstrated small statistically significant changes in zBMI, they are of low clinical significance.

PMID:39585659 | DOI:10.1001/jamapediatrics.2024.4782

JNCI Cancer Spectr. 2024 Nov 25:pkae117. doi: 10.1093/jncics/pkae117. Online ahead of print.

ABSTRACT

BACKGROUND: In non-small cell lung cancer (NSCLC), social determinants of health (SDOHs) influence treatment, but SDOHs with geographic precision are infrequently used in real-world research due to privacy considerations. This research aims to characterize the influence of census-tract level SDOHs on treatment for stage I and IIa NSCLC.

METHODS: Patients diagnosed between 1/1/17 and 9/30/22 with stages I and IIa NSCLC in the Syapse Learning Health Network had their addresses geocoded and linked to five census tract-level indicators of SDOH (social vulnerability index (SVI), percent (%) housing burden, % broadband internet access, primary care shortage area, and rurality). Clinical and demographic characteristics were ascertained from medical records. Nested multinomial logistic regression models estimated associations between SDOHs and initial treatment using two-sided Wald tests. The collective statistical significance of SDOHs was assessed with a likelihood ratio test (LRT) comparing nested models. Descriptive statistics described time-to-treatment-initiation.

RESULTS: Among 3595 patients, 58% were initially treated with surgery, 29% with radiation, and 12% with “other.” Two SDOH variables were associated with increased relative risk ratios (RRR) for radiation therapy compared to surgery: living in primary care shortage areas (RRR 1.61, 95% CI: (1.23-2.10)) and living in non-metropolitan areas (RRR 1.45 (1.02-2.07)). The LRT suggested that the five SDOH variables collectively improved the treatment model. Further, patients in areas with high SVI, low internet access, and high housing-burden initiated treatment later.

CONCLUSION: When using precise estimates of geospatial SDOHs, these measures were associated with treatment, and should be considered in analyses of cancer outcomes.

PMID:39585653 | DOI:10.1093/jncics/pkae117

Qual Life Res. 2024 Nov 25. doi: 10.1007/s11136-024-03855-1. Online ahead of print.

ABSTRACT

PURPOSE: The Myelofibrosis Symptom Assessment Form version 4.0 (MFSAF v4.0) comprises 7 common MF symptom items (fatigue, night sweats, pruritus, abdominal discomfort, pain under the left ribs, early satiety, bone pain) and is the first patient-reported outcome (PRO) instrument designed to assess MF symptom burden. Given that information on the psychometric properties of this instrument has been limited, we sought to evaluate its measurement properties and validate its use in the phase 3 MOMENTUM trial.

METHODS: Data were pooled to assess MFSAF item distribution, structural validity, reliability (test-retest and internal consistency), construct validity (convergent, divergent, and known-groups), and sensitivity to change. Other PRO measures included Patient Global Impression of Severity/Change (PGIS/PGIC), EORTC QLQ-C30, PROMIS Physical Function Short Form 10b, and ECOG performance status.

RESULTS: Participants (N = 195) showed high completion rates (> 93%) across 24 weeks. Moderate to strong Spearman correlation coefficients among items were mostly observed at baseline (range, 0.289-0.772) and week 24 (range, 0.391-0.829), which supported combining items into a multi-item scale and total score. Internal consistency (Cronbach’s α, 0.877 at baseline and 0.903 at week 24) and test-retest reliability (intraclass correlation coefficient, > 0.829) were satisfactory across selected time intervals. Reliability was also supported by McDonald’s omega (ω) coefficient (> 0.875). MFSAF moderately correlated with PRO measures of similar content, differentiated between PGIS and ECOG groups (P < .001), and was able to detect change over time.

CONCLUSIONS: The MFSAF v4.0 is a valid tool to assess MF symptom burden, supporting its use in future trials in similar populations.

PMID:39585603 | DOI:10.1007/s11136-024-03855-1

Bull Exp Biol Med. 2024 Nov 25. doi: 10.1007/s10517-024-06301-x. Online ahead of print.

ABSTRACT

In adult male mice with high (HR) and low (LR) resistance to hypoxia, on days 21 and 28 after subcutaneous injection of Lewis lung carcinoma cells, a morphological and morphometric study of the primary tumor nodes and metastases in the lungs was carried out. Peripheral blood parameters and subpopulation composition of blood cells, the expression of genes responsible for the development of inflammation (Nfkb, Il1b, Il6, Tnfa, Il10, and Tgfb) and the response to hypoxia (Hif1a) in the liver were also assessed. The tumors were detected in 84.6% HR and 91.7% LR mice. The mitotic index of tumor cells in the subcutaneous nodes of HR animals was statistically significantly higher. The metastases area on days 21 and 28 did not differ. In animals of both groups, an increase in the absolute number of leukocytes, monocytes, and granulocytes, a decrease in the hemoglobin content and the absolute number of erythrocytes in the peripheral blood were detected on day 28 of the experiment. Only in LR animals, an increase in the absolute number of CD11b⁺ monocytes was found on day 28 of the experiment in comparison with the control group. The expression of Hif1a, Nfkb, Tnfa, and Tgfb genes in the liver of LR animals was higher than in HR mice, which attested to more pronounced systemic inflammatory response. These data should be taken into account when developing new approaches for the treatment of neoplastic disorders.

PMID:39585596 | DOI:10.1007/s10517-024-06301-x

Bull Exp Biol Med. 2024 Nov 25. doi: 10.1007/s10517-024-06288-5. Online ahead of print.

ABSTRACT

We studied the effect of bone marrow cells (BMC) and nanosecond repetitively pulsed microwaves (RPMs, 10 GHz, pulse duration 100 nsec, pulse repetition rate 8 Hz, peak power flux density (pPFD) 140 W/cm²) on the regeneration of thermally damaged skin in rats. The combined use of BMC and RPMs accelerates separation of an eschar with its complete rejection on day 14 of the experiment. Histological analysis demonstrated a statistically significant increase in the relative area of the granulation tissue and thickness of newly formed epidermis in the group with combined exposure. Combined therapy ensured completion of wound epithelialization in 100% of rats by day 30 of the study.

PMID:39585592 | DOI:10.1007/s10517-024-06288-5

Clin Rheumatol. 2024 Nov 25. doi: 10.1007/s10067-024-07244-4. Online ahead of print.

ABSTRACT

OBJECTIVE: To develop a simplified predictive model for identifying psoriatic arthritis (PsA) in psoriasis patients.

METHODS: Data from the National Health and Nutrition Examination Survey (NHANES) database were analyzed, including patients with psoriasis without arthritis (PsC) or PsA. The least absolute shrinkage and selection operator, Boruta algorithm, random forest, and stepwise regression were employed to select key variables from 38 potential predictors. Logistic regression models were constructed for each combination of selected variables and evaluated using receiver operating characteristic (ROC) curves, precision-recall (PR) curves, calibration plots, Brier scores, and decision curve analysis (DCA).

RESULTS: The study included 587 patients with psoriasis, 238 of whom had PsA. The variable combinations proposed by the Boruta algorithm exhibited the best overall performance. Key predictors in the Borutamodel included age, fasting glucose, education level, thyroid disease, hypertension, and chronic bronchitis. This model achieved area under the curve (AUC) of 0.781 (95% CI, 0.737-0.826) for the training set and 0.780 (95% CI, 0.712-0.848) for the testing set in the ROC curve analyses. The AUC values in the PR curves were 0.687 (95% CI, 0.611-0.757) and 0.653 (95% CI, 0.535-0.770), respectively. The Brier scores of 0.186 and 0.191 for the testing and training sets indicated a good fit, further supported by the calibration curves. DCA showed a net clinical benefit for decision thresholds ranging from 0.2 to 0.8 in both datasets.

CONCLUSION: The Borutamodel represents a promising tool for early risk assessment of PsA. Key Points • National Database Utilization: This study leverages the NHANES database to predict psoriatic arthritis risk, addressing previous limitations tied to regional or ethnic constraints. • Comprehensive Variable Analyses: The research examines 38 variables, including demographics, health conditions, laboratory results, and lifestyle factors, using four distinct screening methods and thorough evaluations of model performance. • Innovative Risk Model: The study introduces a novel risk assessment model that integrates age, fasting glucose, education, and comorbidities including hypertension, thyroid disease, and chronic bronchitis, thus moving beyond traditional focus on skin lesions and joint symptoms.

PMID:39585569 | DOI:10.1007/s10067-024-07244-4

Environ Monit Assess. 2024 Nov 25;196(12):1248. doi: 10.1007/s10661-024-13444-x.

ABSTRACT

Temperature accelerates the deterioration processes affecting grotto temples. As such, studies of the temperature distribution characteristics of grotto temples can provide an important basis for their protection. In this paper, the hourly surface temperatures of 123 grotto temples in China were studied using ERA5-Land hourly data from 1981 to 2020, obtained through the AI Earth platform. Using the local Python development environment, the daily surface temperature difference and highest and lowest temperatures of grotto temples were linearly fitted for each year, after which the monthly average temperature difference distribution was statistically analyzed to determine trends in temperature change. Then, the GIS Spatially Constrained Multivariate Clustering method was used to cluster the surface temperature characteristics. The results showed that the grotto temples in China can be mainly divided into seven regions, namely Xinjiang, Qinghai-Xizang Plateau, Hexi, Longdong, Shaanxi and North China, Southwest, and East and Southeast. The highest average surface temperature, greater than 15 °C, occurred in South China, and the lowest, close to 0 °C, occurred in the Qinghai-Xizang Plateau. The average surface temperature of the seven regions identified showed an increasing trend. The Qinghai-Xizang Plateau was affected by severe temperature differences throughout the year, with annual average daily temperature differences approaching 30 °C, followed by Xinjiang and Hexi region, with a perennial temperature difference of approximately 25 °C. The Longdong, Shaanxi, and North China regions had annual average daily temperature differences of 15-20 °C, whereas values for the South China region were less than 15 °C. The daily surface temperature differences of grotto temples reached their maximum values in April to May and their minimum values in December to January. All studied regions are subject to temperature-induced challenges: Xinjiang region faces particularly severe high-temperature influences, with a mean daily surface temperature of almost 45 °C in summer, followed by Hexi region with 35 °C or above, and the other regions with approximately 30 °C. The Qinghai-Xizang Plateau exhibits perennially low temperatures, with a mean daily minimum temperature below – 25 °C in winter; less than – 10 °C in the Xinjiang, Hexi, Longdong, Shaanxi, and North China regions; and approximately 0 °C in southern China. The relative impacts of temperature on grotto temples in each region are as follows: Xinjiang and Hexi > Qinghai-Xizang Plateau > Longdong, Shaanxi, and North China > Southwest China > East and Southeast China. This study has revealed the characteristics of surface temperature distribution in grotto temples in China and proposes appropriate protection measures, which will help improve national-scale practical mitigation of the threats facing these important cultural heritage sites.

PMID:39585564 | DOI:10.1007/s10661-024-13444-x