Tag: statistics

Pharmacoepidemiol Drug Saf. 2025 Sep;34(9):e70217. doi: 10.1002/pds.70217.

ABSTRACT

BACKGROUND: Assessing medication safety in the pediatric population can take many forms, but given the shortcomings of traditional methods, there has been a shift toward leveraging real-world data to bolster these efforts.

OBJECTIVES: To characterize demographics, enrollment, and health characteristics among pediatric members in the Sentinel Distributed Database (SDD).

METHODS: Using administrative healthcare data from the SDD between January 1, 2000, and May 8, 2023, we used descriptive statistics to characterize the demographics, enrollment, and select health characteristics of pediatric members in the following age groups: 29 days-< 24 months (infants), 2-< 6 years (young children), 6 -< 12 years (older children), 12-< 18 years (early adolescents), and 18-21 years (late adolescents).

RESULTS: Older children (6-< 12 years of age) represented the largest pediatric age group in the SDD, with over 46 million members, though there were between 27.5 and 45.4 million members in each of the other age groups as well. Estimates of common health conditions and medication use were in line with current national estimates.

CONCLUSIONS: The FDA’s Sentinel Distributed Database accurately captures key aspects of pediatric health and can be used as an adjunct to current methods to assess and monitor the safety of approved medical products in the pediatric United States population.

PMID:40947306 | DOI:10.1002/pds.70217

Pediatr Obes. 2025 Sep 14:e70055. doi: 10.1111/ijpo.70055. Online ahead of print.

ABSTRACT

BACKGROUND: Overweight/obesity among children under five is a growing global concern, especially in low- and middle-income countries (LMICs), yet consolidated evidence of this burden remains limited, which is crucial to target interventions. This review synthesises data from community-based studies to estimate the overall and individual pooled prevalences of overweight/obesity in this population and explores the factors associated.

METHODOLOGY: PubMed, Scopus and EMBASE were searched and screened for eligible studies published between April 2014 and 2024 individually by two researchers. Quality assessment of the eligible studies was conducted using the Joanna Briggs Institute (JBI) tool. Data regarding the prevalence of overweight, obesity and their associated factors, including maternal education, area of residence, maternal body weight, household incomes and breastfeeding practices, as reported in these included studies, were extracted. A random-effect model was used for the meta-analysis, and heterogeneity was evaluated using I² statistics. Sources of heterogeneity were explored through subgroup analyses comparing effect sizes across categories of geographic regions, overweight/obesity measurement guidelines, sample sizes and publication years. Meta-regression assessed the relationship between these variables and the pooled estimates.

RESULTS: Of the 13 734 studies screened, 21 were eligible for inclusion. The pooled prevalence of overweight/obesity among the under-five children in LMICs was 8.57% (95% confidence interval (CI) 5.24-12.63, I² 100% at p value < 0.01) with significant heterogeneity. Overnutrition prevalence increased from 7.43% during the 5 years of 2014-2018 to 9.12% during 2019-2023. The pooled prevalence of overweight/obesity varied significantly across geography, with the highest prevalence in the African region (17%) and the lowest in the Southeast Asian region (3.41%). Across the four studies that reported maternal education, children of mothers with secondary school education or higher had increased odds of having overweight/obesity [Odds Ratio (OR) (95% CI): 1.51 (1.21-1.88)]. Prevalence estimates also differed significantly by gender, with 6.89% for under-five boys and 4.75% for girls [OR (95% CI): 1.48 (1.08-2.04)]. Although individual studies reported significant associations with other factors such as area of residence, maternal body weight, income status and breastfeeding practices, a meta-analysis could not be performed due to heterogeneity.

CONCLUSION: Trends in childhood overnutrition in LMICs are concerning, with overall prevalence approaching estimates of those reported in a few high-income nations. Geographic variability, gender disparity and association with maternal education emphasise the need for region-specific policies, focusing on community engagement and awareness towards rising overnutrition.

PMID:40947305 | DOI:10.1111/ijpo.70055

Carbohydr Polym. 2025 Nov 15;368(Pt 2):124082. doi: 10.1016/j.carbpol.2025.124082. Epub 2025 Jul 27.

ABSTRACT

Arabinoxylan (AX) with varying arabinose to xylose (A/X) ratios of 0.85, 0.57 and 0.39 was extracted from wheat bran, and the conformations of the AX polysaccharides dispersed in water were investigated using small-angle X-ray scattering. The persistence length (L_p) and the conformation statistics (expressed by the Flory exponent v or the mass fractal) of the AX varied with their A/X ratios. The L_p decreased with decreasing A/X ratio, from 4.5 nm to 1.5 nm, where the AX with high and intermediate A/X ratios can be considered semi-flexible chains, while AX with the lowest A/X ratio behaves as a flexible chain. The mass fractal increased from 1.7 to 2.5 between the highest and lowest A/X ratios, indicating increasingly compact polymer conformations. AX with the highest A/X ratio behaved as chains in a good solvent and was well dispersed even in the semi-dilute regime. AX with intermediate and low A/X ratios showed stronger tendencies to aggregate and were not well dispersed at higher concentrations. The results presented show that the macroscopic properties of AX dispersions can be understood based on the chemical composition and fine structure of the AX polysaccharide.

PMID:40947242 | DOI:10.1016/j.carbpol.2025.124082

J Allergy Clin Immunol Pract. 2025 Sep;13(9):2535. doi: 10.1016/j.jaip.2025.06.017.

NO ABSTRACT

PMID:40947180 | DOI:10.1016/j.jaip.2025.06.017

J Allergy Clin Immunol Pract. 2025 Sep;13(9):2460-2474. doi: 10.1016/j.jaip.2025.06.015.

ABSTRACT

BACKGROUND: Primary immunodeficiency diseases (PIDDs) are rare genetic disorders impairing immunity. Studies evaluating diagnostic rates of PIDDs in historically marginalized US populations are limited.

OBJECTIVE: To conduct a scoping review that identifies the extent of race and ethnicity reporting in US-based observational studies of people with PIDDs, and the demographic composition of study populations compared with the broader US population.

METHODS: We conducted pragmatic searches of MEDLINE in April 2024 and ultimately included studies dating back 10 years. Results were screened and extracted against prespecified eligibility criteria by a single reviewer. Included data were compared with US census data using χ² tests.

RESULTS: We identified 126 publications publishing observational PIDD studies that report patient characteristics, 62 of which (49%) reported race or ethnicity data. After grouping for data source and PIDD type to avoid overlapping studies, 25 publications were prioritized for extraction. Of these, seven were fully compliant with current Food and Drug Administration-recommended reporting guidelines. The populations of the extracted studies were not statistically representative of the broader US population, with overrepresentation of non-Hispanic White patients.

CONCLUSIONS: Primary immunodeficiency disease cohort and other studies inconsistently report demographic data on patient race and ethnicity according to current Food and Drug Administration recommendations. Efforts to improve understanding of the prevalence, characteristics, and diagnostic rates of PIDD in different US populations (as well as differences among study populations and overall US demographics) would likely be facilitated by a greater effort toward comprehensive demographic reporting.

PMID:40947178 | DOI:10.1016/j.jaip.2025.06.015

J Neurosurg. 2025 Sep 12:1-10. doi: 10.3171/2025.5.JNS243092. Online ahead of print.

ABSTRACT

OBJECTIVE: Prolactinomas are the most common type of pituitary adenoma. Historically, surgery was the primary treatment, but the introduction of dopaminergic agonists in the 1970s changed therapeutic practices. Recent guidelines (2023) from the Pituitary Society now recommend surgery as the first-line option for select prolactinomas, particularly those with certain grades as defined by the Knosp classification system. This systematic review and meta-analysis was performed to evaluate the safety of and the biochemical remission rates following resection of prolactinomas classified preoperatively by Knosp grade.

METHODS: A comprehensive literature search was conducted across the MEDLINE (via PubMed), Scopus, Web of Science, LILACS (Latin American and Caribbean Literature on Health Sciences), and Cochrane CENTRAL (Central Register of Controlled Trials) databases from inception to February 2024. Eligible studies reported individual participant data on the biochemical remission and surgical outcomes of patients with prolactinomas stratified by Knosp grade. A random effects meta-analysis was performed to synthesize biochemical remission rates and relative risks, with results presented in forest plots. Subgroup analyses were conducted according to Knosp grade, heterogeneity was assessed using the I2 statistic, prediction intervals were reported, and publication bias was evaluated through funnel plots and Egger’s test. This review was registered in the International Prospective Register of Systematic Reviews (registration no.: CRD42024602904) and followed the PRISMA guidelines.

RESULTS: Twelve studies involving 1010 patients with prolactinomas undergoing transsphenoidal surgery were included. Prolactinomas classified as Knosp grades 0-2 demonstrated significantly higher biochemical remission rates of 75% (95% CI 67%-82%, prediction interval 42%-96%, I2 = 81.5%, p < 0.0001) compared to 22% (95% CI 16%-31%, prediction interval 11%-38%, I2 = 17.2%, p = 0.57) for Knosp grades 3-4. Transsphenoidal surgery was associated with favorable outcomes characterized by low complication rates and no reported deaths.

CONCLUSIONS: Resection for Knosp grade 0-2 prolactinomas appears to be an effective first-line treatment option, resulting in favorable biochemical remission rates and low complication risks. These findings support considering surgery in appropriately selected patients, particularly at experienced medical centers. However, the high heterogeneity among and the observational design of most included studies limit the strength of the conclusions. Thus, further studies are needed to compare surgical and medical management strategies across Knosp grades and to refine patient selection.

PMID:40939216 | DOI:10.3171/2025.5.JNS243092

J Neurosurg. 2025 Sep 12:1-10. doi: 10.3171/2025.5.JNS243092. Online ahead of print.

ABSTRACT

OBJECTIVE: Prolactinomas are the most common type of pituitary adenoma. Historically, surgery was the primary treatment, but the introduction of dopaminergic agonists in the 1970s changed therapeutic practices. Recent guidelines (2023) from the Pituitary Society now recommend surgery as the first-line option for select prolactinomas, particularly those with certain grades as defined by the Knosp classification system. This systematic review and meta-analysis was performed to evaluate the safety of and the biochemical remission rates following resection of prolactinomas classified preoperatively by Knosp grade.

METHODS: A comprehensive literature search was conducted across the MEDLINE (via PubMed), Scopus, Web of Science, LILACS (Latin American and Caribbean Literature on Health Sciences), and Cochrane CENTRAL (Central Register of Controlled Trials) databases from inception to February 2024. Eligible studies reported individual participant data on the biochemical remission and surgical outcomes of patients with prolactinomas stratified by Knosp grade. A random effects meta-analysis was performed to synthesize biochemical remission rates and relative risks, with results presented in forest plots. Subgroup analyses were conducted according to Knosp grade, heterogeneity was assessed using the I2 statistic, prediction intervals were reported, and publication bias was evaluated through funnel plots and Egger’s test. This review was registered in the International Prospective Register of Systematic Reviews (registration no.: CRD42024602904) and followed the PRISMA guidelines.

RESULTS: Twelve studies involving 1010 patients with prolactinomas undergoing transsphenoidal surgery were included. Prolactinomas classified as Knosp grades 0-2 demonstrated significantly higher biochemical remission rates of 75% (95% CI 67%-82%, prediction interval 42%-96%, I2 = 81.5%, p < 0.0001) compared to 22% (95% CI 16%-31%, prediction interval 11%-38%, I2 = 17.2%, p = 0.57) for Knosp grades 3-4. Transsphenoidal surgery was associated with favorable outcomes characterized by low complication rates and no reported deaths.

CONCLUSIONS: Resection for Knosp grade 0-2 prolactinomas appears to be an effective first-line treatment option, resulting in favorable biochemical remission rates and low complication risks. These findings support considering surgery in appropriately selected patients, particularly at experienced medical centers. However, the high heterogeneity among and the observational design of most included studies limit the strength of the conclusions. Thus, further studies are needed to compare surgical and medical management strategies across Knosp grades and to refine patient selection.

PMID:40939216 | DOI:10.3171/2025.5.JNS243092

J Neurosurg Spine. 2025 Sep 12:1-10. doi: 10.3171/2025.5.SPINE25215. Online ahead of print.

ABSTRACT

OBJECTIVE: Mid- to long-term data on the natural history of degenerative lumbar spinal stenosis (LSS) remain limited as surgery is increasingly favored. The aim of this study was to characterize the prevalence of clinical deterioration over long-term follow-up and to identify risk and protective factors.

METHODS: In this retrospective cohort study, adult patients with symptomatic LSS and a follow-up period ≥ 5 years were analyzed. Clinical deterioration was defined by at least one of the following factors: myotomal lower limb weakness, sphincter disturbance, or a decrease in walking tolerance to ≤ 10 minutes due to neurogenic claudication. Radiological assessment included standing lumbar radiographs and lumbosacral MR images obtained after symptom onset. A univariate analysis was performed, with variables demonstrating significance levels of p < 0.1 included in the subsequent multivariable logistic regression analysis. Receiver operating characteristic (ROC) curves and Kaplan-Meier survival curves were plotted for statistically significant risk factors.

RESULTS: A total of 202 patients with symptomatic LSS and adequate follow-up were included. The mean age was 65.2 ± 4.2 years at the onset of neurological symptoms and the mean follow-up duration was 121 ± 40 months. Clinical deterioration occurred in 39 patients (19.3%). Among those with deterioration, 36 (92.3%) reported reduced walking tolerance due to neurogenic claudication, 8 (20.5%) had myotomal weakness, and 2 (5.1%) experienced sphincter disturbance. Upon multivariate analysis, the presence of lumbar developmental spinal stenosis was a risk factor for deterioration (p = 0.031), while an increased dural sac area was protective (p = 0.045); adjusted hazard ratios were 10.11 and 0.98, respectively. A dural sac area < 55 mm2 had an area under the ROC curve of 0.781 for predicting clinical deterioration within 5 years of symptom onset.

CONCLUSIONS: Patients with lumbar stenosis and neurogenic claudication mostly remained ambulatory without developing motor deficits or sphincter dysfunction. Conservative management is an option for patients with tolerable symptomatology and low functional expectations, especially in the absence of the identified risk factors of developmental narrowing of lumbar canal dimensions and critically reduced dural sac area over the most stenotic level.

PMID:40939213 | DOI:10.3171/2025.5.SPINE25215

Blood. 2025 Sep 12:blood.2025028823. doi: 10.1182/blood.2025028823. Online ahead of print.

ABSTRACT

The IELSG37 trial enrolled 545 patients with primary mediastinal B-cell lymphoma (PMBCL) and demonstrated that consolidation radiotherapy (RT) can be omitted in patients with complete metabolic response (CMR), defined by the Lugano classification as Deauville score (DS) 1-3. This report evaluates outcomes following different frontline rituximab- and doxorubicin-based immunochemotherapy regimens chosen according to local practice. Patients treated with R-CHOP21 showed a significantly higher percentage of DS 5 than those on other regimens (23.8% vs. 8.2% average, P< 0.001) as well as a trend toward additional unplanned treatments (53.2% vs. 46.9%, P=0.30). The increased risk of poor response was confirmed in a multinomial logistic regression analysis adjusted for age, sex, IPI score, and performance status. R-CHOP21 was also associated with smaller reductions in MTV and less pronounced decreases in SUVmax. Patients with DS 5 more often received additional treatment (RT and/or salvage chemotherapy with or without autologous consolidation) after induction immunochemotherapy (96% vs. 41%, P< 0.001) and experienced significantly poorer outcomes. Although differences in progression-free and overall survival between R-CHOP21 and more aggressive regimens were not statistically significant, R-CHOP21 may increase the risk of additional treatments and may be inadvisable as frontline therapy for PMBCL. NCT01599559.

PMID:40939190 | DOI:10.1182/blood.2025028823

JCO Glob Oncol. 2025 Sep;11:e2500180. doi: 10.1200/GO-25-00180. Epub 2025 Sep 12.

ABSTRACT

PURPOSE: Malignant apocrine and eccrine tumors (MAETs) are extremely rare cutaneous adnexal malignancies, accounting for only 0.005%-0.01% of all skin tumors. These tumors are highly metastatic, and evidence regarding optimal chemotherapy and prognostic outcomes remains limited. This study aimed to evaluate the efficacy of systemic chemotherapy and overall prognosis in Japanese patients with unresectable MAETs.

PATIENTS AND METHODS: We conducted a retrospective, multicenter study involving 81 patients with unresectable MAETs treated at 27 institutions across Japan. Patients received one of three primary chemotherapy regimens: platinum-based (cisplatin or carboplatin), taxane-based (docetaxel or paclitaxel), or TS-1 (tegafur/gimeracil/oteracil). Patient demographics, objective response rates (ORRs), overall survival (OS), and progression-free survival were assessed. Survival curves were estimated using the Kaplan-Meier method.

RESULTS: The estimated ORRs for the platinum-based, taxane-based, and TS-1 groups were 37.0%, 25.0%, and 22.2%, respectively, with no statistically significant differences among them (P = .527). The median OS and 5-year OS rate for the entire cohort were 29.0 months and 34.0%, respectively. The median OS and 5-year OS rates by regimen were as follows: platinum-based, 29.0 months and 36.2%; taxane-based, 22.0 months and 39.7%; and TS-1, 30.0 months and 13.9%, with no significant differences observed (P = .907). In addition, there were no significant differences in ORR or OS between patients receiving combined chemoradiotherapy and those receiving chemotherapy alone.

CONCLUSION: No single chemotherapeutic regimen demonstrated superior efficacy in patients with unresectable MAETs. These findings highlight the need for further investigations using larger, prospective cohorts and multidisciplinary approaches to establish optimal therapeutic strategies for this rare malignancy.

PMID:40939132 | DOI:10.1200/GO-25-00180