Tag: statistics

J Nutr Educ Behav. 2025 Mar 28:S1499-4046(25)00051-X. doi: 10.1016/j.jneb.2025.02.012. Online ahead of print.

ABSTRACT

OBJECTIVE: To examine the associations between parental perception, concern, and dissatisfaction with child weight and their feeding practices among Chinese families.

DESIGN: A cross-sectional study.

SETTING: Four public kindergartens in Yangzhou, China.

PARTICIPANTS: Chinese parents of preschool children (n = 1,779).

MAIN OUTCOME MEASURES: Three responsive feeding practices (i.e., encouragement of healthy eating, monitoring, and modeling) and 3 nonresponsive feeding practices (i.e., pressure to eat, restriction, and use of food as a reward).

ANALYSIS: Hierarchical multiple regression analysis was performed to examine their associations. The agreement was evaluated with kappa statistics.

RESULTS: Parents who perceived children as overweight or obese reported less pressure to eat (P = 0.04); parents who were concerned about children with underweight reported more pressure to eat (P = 0.01); parents who rated children’s body weight size as underweight were less likely to encourage children to eat healthy food (P = 0.04) and restrict food intake (P = 0.02); parents who desired a slimmer child’s body size reported less modeling (P < 0.001) and more restriction (P = 0.04). The disagreements between parental self-reported and visual perception of child weight and actual child weight were statistically significant, respectively (P < 0.01).

CONCLUSIONS AND IMPLICATIONS: The results suggested the significant influence of parental perception, concern, and dissatisfaction with child weight on feeding practices. Our findings may inform public health practitioners and primary care providers in designing interventions to enhance parental accurate weight perception and optimize feeding practices.

PMID:40152838 | DOI:10.1016/j.jneb.2025.02.012

Clin Transplant. 2026 Apr;39(4):e70132. doi: 10.1111/ctr.70132.

ABSTRACT

INTRODUCTION: Predicting graft failure risk in pediatric liver transplantation (LT) recipients could identify areas for improving management. Persistent cognitive, motor, academic, and functional deficits are common in recipients and their impact on graft survival following LT helps inform risk prediction.

METHODS: Using SRTR data 2008-2023, we evaluated the cognitive, motor, academic, and functional deficits of LT recipients at time of transplant to 14 years post-LT. We compared all cause graft failure (ACGF) among patients with versus without pre-LT and 1-year post-LT deficits using Cox regression, adjusting for recipient characteristics. We calculated an individual risk score for ACGF.

RESULTS: In 8062 pediatric LT recipients median age 3 (IQR: 1, 10), 28.0%, 29.5%, 35.0%, and 79.8% of recipients had pre-LT deficits in cognition, motor, academic activity, and functional status respectively. This decreased to 23.0%, 18.1%, 14.2%, and 38.7% 1-year post-LT. Increased hazard of ACGF was noted in recipients with pre-LT decreased functional status (aHR = 1.13 (per 10% decrease), 95% CI: 1.10-1.15, p < 0.001), definite motor delay (aHR = 1.60, 95% CI: 1.21-2.10, p < 0.001), and inability to participate in academics (aHR = 1.49, 95% CI: 1.08-1.89, p = 0.01), but not delays in cognition (aHR = 0.91, 95% CI: 0.69-1.21, p = 0.19). Our risk score predicting ACGF demonstrated improved predictive performance compared to clinical parameters alone (C-statistic = 0.70 (0.67, 0.72) vs. 0.66 (0.64, 0.69), p < 0.001).

CONCLUSIONS: Pediatric LT recipients with pre- or post-LT motor, academic, and functional deficits are at higher risk for ACGF. Care should be taken to assess deficits to identify patients who may benefit from functional intervention to potentially reduce ACGF risk.

PMID:40152814 | DOI:10.1111/ctr.70132

J Manag Care Spec Pharm. 2025 Apr;31(4):366-376. doi: 10.18553/jmcp.2025.31.4.366.

ABSTRACT

BACKGROUND: Direct oral anticoagulants (DOACs) are used to prevent thrombosis in patients with nonvalvular atrial fibrillation (NVAF) and venous thromboembolism (VTE). Despite their clinical benefits, some patients abandon their DOAC prescription.

OBJECTIVE: To retrospectively evaluate the association between patient out-of-pocket (OOP) costs and abandonment of the first DOAC prescription among patients with NVAF or VTE in the United States.

METHODS: Data from Symphony Health, an ICON plc Company, PatientSource (April 1, 2017, to October 31, 2020) were used to select patients with NVAF or VTE with an approved or abandoned claim for a DOAC (apixaban, dabigatran, rivaroxaban). OOP costs (2021 US dollars) of the index claim were described by abandonment status, and multivariable logistic regression models were used to evaluate the association between OOP costs of the index DOAC claim and abandonment. Analyses were performed in patients with NVAF and VTE separately.

RESULTS: Among 753,755 patients with NVAF, 88.5% had an approved index DOAC claim and 11.5% had an abandoned index DOAC claim. Among 308,429 patients with VTE, 91.5% had an approved index DOAC claim and 8.5% had an abandoned index DOAC claim. Mean OOP costs of the index DOAC claim were lower in those with an approved than abandoned claim (NVAF approved vs abandoned: $79 vs $175; VTE approved vs abandoned: $65 vs $133). Among patients with NVAF, 21.4% of those with an approved claim and 9.1% of those with an abandoned claim had no OOP costs, 58.7% (approved) and 49.0% (abandoned) had OOP costs greater than $0 to less than $100, and 19.9% (approved) and 41.9% (abandoned) had OOP costs greater than or equal to $100; among patients with VTE, 27.8% (approved) and 15.6% (abandoned) had no OOP costs, 58.4% (approved) and 54.8% (abandoned) had OOP costs greater than $0 to less than $100, and 13.8% (approved) and 29.6% (abandoned) had OOP costs greater than or equal to $100. In multivariable models, the risk of abandonment increased by 21% (NVAF) and 17% (VTE) for each $100 in OOP costs (both P < 0.001). Relative to patients with no OOP costs, patients with OOP costs greater than $0 to less than $50 were 86% (NVAF) and 55% (VTE) more likely to abandon their index DOAC, patients with OOP costs greater than $50 to less than $100 were 80% (NVAF) and 111% (VTE) more likely to abandon their index DOAC, and patients with OOP costs greater than or equal to $100 were 332% (NVAF) and 244% (VTE) more likely to abandon their index DOAC (all P < 0.001).

CONCLUSIONS: Among patients with NVAF or VTE, OOP costs of the first DOAC claim greater than or equal to $100 were associated with the highest risk of abandoning the first DOAC prescription.

PMID:40152803 | DOI:10.18553/jmcp.2025.31.4.366

J Manag Care Spec Pharm. 2025 Apr;31(4):377-385. doi: 10.18553/jmcp.2025.31.4.377.

ABSTRACT

BACKGROUND: Clinical guidelines recommend the use of proprotein convertase subtilisin/kexin type 9 inhibitors (PCSK9is) in patients with atherosclerotic cardiovascular disease (ASCVD) and nonoptimal low-density lipoprotein.

OBJECTIVE: To evaluate the association between discontinuation of statin use after PCSK9i initiation and subsequent ASCVD events.

METHODS: This pre-post retrospective comparative study used national administrative data of adult statin medication users (age ≥18 years) with an index PCSK9i claim (January 1, 2019, to April 30, 2021), prior ASCVD diagnosis, and a 2-year follow-up period. Proportions and probability of ASCVD events post-index (PCSK9i) vs pre-index (PCSK9i) for patients who discontinued statins (discontinued cohort) and those who continued statins (continued cohort) were compared. Propensity score weighting was used to balance patient baseline characteristics. Multivariate Poisson regression and time-to-event Cox regression models were used to assess the association between statin discontinuation and ASCVD events.

RESULTS: There were 294 and 46 patients in the continued and discontinued cohorts, respectively. Unweighted results showed that patients in the continued cohort were more likely to receive high-intensity statins (32% vs 22%; P = 0.4) and have a Charlson Comorbidity Index score of 3 or more (62% vs 54%; P = 0.5) at baseline. Baseline statin adherence was lower in the discontinued cohort (6.7% vs 59%; P < 0.001) but 30% each in the propensity 1:1 matched cohort. The 2 cohorts (after matching) had similar ASCVD event prevalence (discontinued cohort: 24% vs continued cohort: 26%) in the baseline and the same lower prevalence (6.5% each; P > 0.9) in the 24-month follow-up period. The odds of any ASCVD event post-index was comparable between the 2 cohorts (reference: continued cohort; odds ratio = 1.88; 95% CI = 0.28-14.6; P = 0.51). There were no statistically significant differences between the 2 groups in the Cox regression (P = 0.47).

CONCLUSIONS: Post-ASCVD event rates were significantly lower in both cohorts, but discontinuation of statins was not associated with unfavorable ASCVD outcomes.

PMID:40152801 | DOI:10.18553/jmcp.2025.31.4.377

J Manag Care Spec Pharm. 2025 Apr;31(4):386-395. doi: 10.18553/jmcp.2025.31.4.386.

ABSTRACT

BACKGROUND: Heart failure (HF) is a leading cause of mortality in the United States, often complicated by comorbidities like diabetes mellitus. These patients face high hospitalization risks, impacting clinical outcomes and health care resources. The Effect of Sotagliflozin on Cardiovascular Events in Patients with Type 2 Diabetes Post Worsening Heart Failure (SOLOIST-WHF) trial showed that sotagliflozin, a sodium-glucose cotransporter inhibitor, reduced rehospitalizations in patients with HF and diabetes mellitus. Although clinically beneficial, the economic impact of sotagliflozin from a payer perspective remains unclear, warranting further pharmacoeconomic analysis to guide managed care decisions.

OBJECTIVE: To quantify the budget impact of sotagliflozin for US payers over a 5-year time horizon.

METHODS: A payer-perspective budget impact model was developed to assess the financial impact of incorporating sotagliflozin for the treatment of patients recently hospitalized for HF with comorbid type 2 diabetes (T2D) over 5 years to US payer health plans. The study used a population reflecting the SOLOIST-WHF clinical trial, with economic parameters adjusted by payer mix (all payer, commercial, Medicare, and Medicaid). Health care resource utilization included hospitalization, emergency department (ED) visit, and adverse events’ care. Economic outcomes examined the medical and pharmacy budget impact for payers at the per-user, per member per month (PMPM), and total plan costs levels.

RESULTS: For a hypothetical 1-million-member all-payer plan, 1,516 patients hospitalized for HF with comorbid T2D would be eligible for sotagliflozin. For all-payer plans, annual per-user costs increased by $4,996 because of higher pharmacy costs ($8,260) but were partially offset by lower medical costs (-$2,608) because of reduced rehospitalization and ED visits from sotagliflozin. PMPM total budget impact of sotagliflozin would be $0.08 PMPM in year 1 and $0.38 in year 5, corresponding with total plan cost of $75,736 in year 1 and $378,681 by year 5. Commercial payer PMPM costs were lower ($0.02 in year 1; $0.11 in year 5), and higher for Medicare ($0.23 PMPM in year 1, increasing to 1.13 PMPM in year 5). Breakeven rebate rates ranged between 31.5% and 79.4%.

CONCLUSIONS: Although sotagliflozin increases pharmacy costs for recently hospitalized HF patients with T2D, approximately 21%-68% of pharmacy costs were offset from reduced rehospitalization and ED visits.

PMID:40152799 | DOI:10.18553/jmcp.2025.31.4.386

J Manag Care Spec Pharm. 2025 Apr;31(4):406-420. doi: 10.18553/jmcp.2025.31.4.406.

ABSTRACT

BACKGROUND: The cost of health care for patients with Hodgkin lymphoma (HL) is projected to rise, making it essential to understand expenditure drivers across different demographics, including the older adult population. Although older HL patients constitute a significant number of HL patients, the literature on health care expenditures in older HL patients is lacking. Predictive capabilities of machine learning (ML) methods enhance our ability to leverage a data-driven approach, which helps identify key predictors of expenditures and strategically plan future expenditures.

OBJECTIVE: To determine the leading predictors of health care expenditures among older HL survivors across prediagnosis, treatment, and posttreatment phases of care.

METHODS: The study uses a retrospective research design to identify the incident cases of HL diagnosed between 2009 and 2017 using Surveillance, Epidemiology, and End Results-Medicare data. Three phases of cancer care (prediagnosis, treatment, and posttreatment) were indexed around the diagnosis date, with each phase divided into 12 months of baseline and 12 months of follow-up. ML methods, including XGBoost, Random Forest, and Cross-Validated linear regressions, were used to identify the best regression model for predicting Medicare and out-of-pocket (OOP) health care expenditures. Interpretable ML SHapley Additive exPlanations method was used to identify the leading predictors of Medicare and OOP health care expenditures in each phase.

RESULTS: The study analyzed 1,242 patients in the prediagnosis phase, 902 in the treatment phase, and 873 in the posttreatment phase. XGBoost regression outperformed Random Forest and Cross-Validated linear regression models with overall performance in predicting Medicare expenditures, with R-squared (root mean square error) values of 0.42 (1.39), 0.43 (0.56), and 0.46 (0.90) across the 3 phases of care, respectively. Interpretable ML methods highlighted baseline expenditures, number of prescription medications, and cardiac dysrhythmia as the leading predictors for Medicare and OOP expenditures in the prediagnosis phase. Chemotherapy and immunotherapy and surgical treatment and immunotherapy were the leading predictors of expenditures in the treatment and posttreatment phases, respectively.

CONCLUSIONS: As ML applications increase in predicting health care expenditure, researchers should consider implementing models in different phases of care to identify the changes in the predictors. Leading predictors of health care expenditures can be targeted for informed policy development to address financial hardship in HL survivors.

PMID:40152796 | DOI:10.18553/jmcp.2025.31.4.406

J Manag Care Spec Pharm. 2025 Apr;31(4):351-365. doi: 10.18553/jmcp.2025.31.4.351.

ABSTRACT

BACKGROUND: Calcitonin gene-related peptide monoclonal antibodies (CGRP mAbs) are approved for migraine prevention. Limited information is available comparing the health care resource utilization (HCRU) and direct costs associated with initiating different CGRP mAbs.

OBJECTIVE: To compare all-cause and migraine-related HCRU and direct costs in US patients with migraine initiating the self-injectable CGRP mAbs, galcanezumab, fremanezumab, or erenumab.

METHODS: This retrospective cohort study used data from Merative Marketscan Commercial and Medicare Databases. Adults with at least 1 claim (first claim=index) for the above CGRP mAbs between May 2018 and September 2020 (index period), with continuous enrollment for 12 months pre-index (baseline [BL]) and post-index (follow-up [FU]) were included. Patients with a claim for index drug during BL were excluded. Mean HCRU and mean total costs (inpatient, outpatient, and outpatient pharmacy costs) were evaluated over 12 months post-index. Propensity score matching was used to balance the galcanezumab vs fremanezumab (2:1) and galcanezumab vs erenumab (1:1) cohorts. P values of <0.05 were considered statistically significant.

RESULTS: After matching, patient demographics and clinical characteristics were similar between galcanezumab vs fremanezumab (n=2,674 sets) and galcanezumab vs erenumab (n=3,503 sets) cohorts. Relative to BL, numerically lower all-cause and migraine-related HCRU (inpatient and outpatient visits) were observed in all cohorts over the 12-month post-index period, whereas outpatient pharmacy HCRU was numerically higher. All-cause and migraine-related total costs (mean) were higher over the FU period in all cohorts (all P < 0.001). Mean all-cause and migraine-related cost increases were numerically similar for galcanezumab vs fremanezumab ($503 vs $518 [P=0.825] and $467 vs $468 [P=0.990]), and for galcanezumab vs erenumab ($504 vs $499 [P=0.934] and $462 vs $443 [P=0.375]). Outpatient pharmacy costs contributed greatly to migraine-related costs, whereas all-cause costs were greatly driven by outpatient costs.

CONCLUSIONS: HCRU and direct cost differences observed at 12 months following initiation of self-injectable CGRP mAbs for migraine prevention were numerically similar across cohorts for patients treated with galcanezumab, fremanezumab, and erenumab. More work should be done to learn if these drugs perform differently with respect to other important factors not examined here.

PMID:40152794 | DOI:10.18553/jmcp.2025.31.4.351

Crit Rev Toxicol. 2025 Mar 28:1-19. doi: 10.1080/10408444.2025.2451885. Online ahead of print.

ABSTRACT

Assessments of a chemical agent’s carcinogenicity based on rodent bioassays rely on their appropriate interpretation. This involves attention to study details, including reliable histopathologic diagnoses, and proper statistical analyses, including consideration of multiple comparisons, concurrent and historical controls. A major factor is evaluation of their likely mode of action and the human relevance of any identified tumors. We present a critical evaluation of the assessment of the 2-year inhalation bioassays of n-butyl methacrylate (n-BMA) in rats and mice performed by the Japan Bioassay Research Center (JBRC) and an assessment of the International Agency for Research on Cancer (IARC) review and classification as Group 2B, possible human carcinogen. The tumors of concern for assessment of its carcinogenicity included mononuclear cell leukemia (MCL) in male rats, thyroid C-cell tumors in female rats, liver tumors and histiocytic sarcomas in male mice, and hemangiosarcomas in female mice. Our review of these studies raises concerns regarding the accuracy of histopathology diagnoses and human relevance of MCL. Most critically, the statistical evaluation/interpretation of all tumor types indicates no carcinogenic effects, since the frequency of increases (at p < 0.05) in tumor incidences in the study is totally consistent with chance expectation (i.e. not treatment related). Furthermore, the plausibility of n-BMA being carcinogenic is questionable since it is non-genotoxic, and the weight of evidence including read-across to the close structural analog methyl methacrylate indicates no concern for cancer. After thorough review of these bioassays, we conclude that there is no convincing evidence of carcinogenicity for n-BMA, contrary to the conclusion of the JBRC and the decision by the IARC.

PMID:40152788 | DOI:10.1080/10408444.2025.2451885

Cancer Prev Res (Phila). 2025 Mar 28. doi: 10.1158/1940-6207.CAPR-24-0501. Online ahead of print.

ABSTRACT

Multiple myeloma (MM) is preceded by monoclonal gammopathy of undetermined significance (MGUS). Only a minority of MGUS patients will develop MM, but precise prediction of progression is impossible using routine clinical biomarkers. Changes in the levels of blood immune markers can help predict disease progression. Data remain inconsistent for some markers of interest such as monocyte chemotactic protein-3 (MCP-3), macrophage inflammatory protein-1 alpha (MIP-1α), fibroblast growth factor-2 (FGF-2), vascular endothelial growth factor (VEGF), fractalkine, and transforming growth factor-alpha (TGF-α). We aimed to investigate the associations between the pre-diagnostic serum levels of these candidate biomarkers and future MM risk, as well as to assess marker changes over time. We performed a nested case-control study using prospective samples from the Janus Serum Bank in Norway to investigate associations between MM risk and pre-diagnostic serum levels of MCP-3, MIP-1α, FGF-2, VEGF, fractalkine, and TGF-α. The study included 293 future MM cases with serum samples collected 20 years (median) before MM diagnosis and 293 matched cancer-free controls. MM patients had an additional pre-diagnostic sample collected up to 42 years before diagnosis to identify marker changes over time. Markers with >60% detection rate (MIP-1α, VEGF, and TGF-α) were included in the statistical analysis. We observed no statistically significant associations between MM risk and serum levels of MIP-1α, VEGF, or TGF-α in samples collected 20 years before diagnosis. However, TGF-α levels decreased significantly closer to the diagnosis in MM patients (p<0.001). The decrease in TGF-α levels may reflect subtle microenvironmental changes related to MM progression.

PMID:40152768 | DOI:10.1158/1940-6207.CAPR-24-0501

Dtsch Arztebl Int. 2025 Apr 18;(Forthcoming):arztebl.m2025.0015. doi: 10.3238/arztebl.m2025.0015. Online ahead of print.

ABSTRACT

BACKGROUND: It is important for patients with back pain to be well-informed. The well-informed patient is better prepared for self-management and for joint decision-making with the physician. This is why we developed tala-med, a web portal providing up-to-date, evidence-based, independent information on back pain. Primary care physicians can use it in their practices and make it accessible to their patients.

METHODS: A cluster randomized controlled trial (registration number DRKS00014279) was carried out in which the primary care physicians (PCPs) in the intervention group (IG: 33 physicians) were directed to use the tala-med web portal in their consultations with patients who had back pain (180 patients). The PCPs in the control group (CG: 12 physicians) were not given access to tala-med and treated their patients with back pain (136 patients) as they had done before. The patients’ informedness about back pain was the primary endpoint: it was assessed by the patients themselves in terms of their subjective degree of knowledge and subjective state of informedness about back pain. Pain intensity, the second endpoint, was assessed with a visual analog scale.

RESULTS: Intention-to-treat analyses revealed that the patients’ subjective degree of knowledge about back pain increased to a greater extent in the intervention group than in the control group (B = 0.25, 95% confidence interval [0.07; 0.43]), as did their subjective state of informedness about back pain (B = 0.51 [0.13; 0.89]). Patients in the intervention group also reported a larger reduction of pain intensity compared to patients in the control group (B = -10.46 [-18.52; -2.38]).

CONCLUSION: Use of the tala-med web portal by primary care physicians and their patients made patients better informed about back pain and lessened the intensity of their pain. These improvements, although statistically significant, were not large enough to be clinically important.

PMID:40152763 | DOI:10.3238/arztebl.m2025.0015