Diagn Progn Res. 2026 Apr 20;10(1):12. doi: 10.1186/s41512-026-00229-8.
NO ABSTRACT
PMID:42010734 | DOI:10.1186/s41512-026-00229-8
Tag: statistics
Trials. 2026 Apr 20;27(1):312. doi: 10.1186/s13063-026-09713-4.
ABSTRACT
BACKGROUND: The estimand framework was introduced into guidance on good clinical practice to address a variety of shortcomings and ambiguities in the reporting of trials, including the use of terms such as “intention to treat” and the handling of non-adherence to treatment. The framework was primarily grounded in individually randomised trials, and some thorny issues still cloud understanding of its application to cluster randomised trials.
ESTIMANDS IN CLUSTER RANDOMISED TRIALS: This commentary addresses some of the challenges in thinking about the estimands behind cluster randomised trials. These challenges include informative cluster size-the possibility that a treatment effect may be modified by the size of the cluster. In particular, we consider the perspectives of different actors-a population-level decision maker or politician, a cluster manager, and a patient-and examine possible estimands for each, and how they differ.
CONCLUSIONS: In the cluster randomised trial context, the estimand framework can be complex to navigate. Different perspectives lead to different estimands. We caution against abandoning careful statistical modelling. This is particularly true in the presence of informative cluster size, where modelling any interaction between cluster size and treatment effect could be useful from a number of perspectives.
PMID:42010658 | DOI:10.1186/s13063-026-09713-4
BMC Anesthesiol. 2026 Apr 20;26(1):248. doi: 10.1186/s12871-026-03827-7.
NO ABSTRACT
PMID:42010465 | DOI:10.1186/s12871-026-03827-7
Eur J Hum Genet. 2026 Apr 20. doi: 10.1038/s41431-026-02110-0. Online ahead of print.
ABSTRACT
This paper continues our development of methods for discovery of genetic modifiers of the Duchenne muscular dystrophy (DMD) phenotype. DMD is an X-linked recessive disorder involving progressive muscle tissue loss with replacement by fat and fibrotic tissue, leading in most cases to loss of ambulation (LOA) by early to mid-adolescence. The standard pharmacologic treatment is corticosteroid administration, which increases average LOA by 2-3 years. There is variation in LOA due to specific DMD mutations, some of which permit the production of residual or partial dystrophin protein and lead to milder phenotypes. But there is also believed to be variation due to genetic modifiers acting even in patients whose DMD mutations preclude dystrophin production altogether, based in part on animal models, and several genes have been implicated as potential modifiers of LOA in DMD patients. Here we consider whether the mechanism of action of any of these genes might be to influence LOA by modifying the effects of corticosteroid exposure. We develop and evaluate a novel statistic, the PPIGxE; we consider the issue of potential “phenocopies,” or individuals whose late LOA might be due to residual dystrophin production; and we apply our approach to 12 candidate SNPs using our DMD dataset. We find evidence of genotype x steroid interaction effects for 4 out of the 12 SNPs we tested, which can be linked to the TGF-β pathway. These results corroborate the hypothesis that modifiers in the TGF-β pathway affect LOA by modulating the efficacy of corticosteroid administration.
PMID:42010352 | DOI:10.1038/s41431-026-02110-0
Sci Rep. 2026 Apr 20;16(1):12875. doi: 10.1038/s41598-026-49144-4.
ABSTRACT
Two simple and selective spectrophotometric methods, ratio difference and derivative ratio, were developed and validated for the simultaneous determination of etoricoxib and tramadol in binary mixtures and pharmaceutical formulations. The methods effectively resolved the sever spectral overlap of both drugs without prior separation. In the ratio difference method, amplitude differences between 294 and 239 nm for etoricoxib and 218 and 272 nm for tramadol showed excellent linearity. In the derivative ratio method, derivative amplitudes measured at 287 nm etoricoxib, and 229 nm tramadol provided high selectivity and sensitivity. The linear ranges were 2-23 µg/mL for etoricoxib and 3-40 µg/mL for tramadol, with correlation coefficients above 0.999. Both methods achieved recoveries between 98.78 and 101.09% and precision values (%RSD) below 2%. Statistical comparison with a reported HPLC method showed no significant difference. The proposed methods are accurate, cost-effective, and supported by AGREE-based greenness assessment, making them suitable for routine quality control of etoricoxib and tramadol formulations.
PMID:42010288 | DOI:10.1038/s41598-026-49144-4
Mol Imaging Biol. 2026 Apr 20. doi: 10.1007/s11307-026-02102-y. Online ahead of print.
ABSTRACT
Quantification of the dopamine D2/3 receptors tracer [18F]fallypride is usually performed by defining the cerebellum (CB) as the reference region for its use in kinetic modeling. In mouse studies, [18F]fallypride is defluorinated causing gradual uptake in the skull which, in addition to extra-striatal binding, can contaminate the CB activity, therefore introducing errors in reference region kinetic modeling. Here we propose a method using non-negative matrix factorization (NMF) to accurately extract the reference region time activity curve (TAC) unaffected by spill-over from surrounding regions. We compared the NMF method with template-based CB reference region (erodedCB), where the region was eroded to avoid spill-over. The different methods were applied in a drug challenge study using RX821002, and compared with results obtained using [11C]raclopride. Striatal and brain parametric maps of nondisplaceable binding potential (BPND) were calculated with the different methods, and differences between baseline and challenge were investigated. The NMF reference region TACs showed higher peak and lower tail activity compared with erodedCB. Striatal BPND values calculated with NMF were about 20% higher compared to those calculated with erodedCB, and difference between baseline and challenge increased using NMF (NMF: 11.5%, erodedCB: 7.0%). Parametric t-statistic maps show clusters with significant differences using NMF but not with erodedCB. [11C]raclopride BPND differences between baseline and challenge were lower (6.1%) than with [18F]fallypride, but BPND variability was lower. In summary, NMF allowed us to extract reference region TACs without contamination from skull or extra-striatal uptake, which improved voxel-wise detection of differences in a drug challenge study.
PMID:42010231 | DOI:10.1007/s11307-026-02102-y
Indian J Gastroenterol. 2026 Apr 20. doi: 10.1007/s12664-025-01963-5. Online ahead of print.
ABSTRACT
BACKGROUND AND OBJECTIVES: Transient elastography of liver and spleen, along with platelet count, identify cirrhosis patients with esophageal varices needing treatment (VNT). Inflammation can drive rapid rise in portal pressure and development and progression of varices in acute-on-chronic liver failure (ACLF) patients. We aimed at developing a non-invasive model to predict VNT in ACLF patients.
METHODS: Baseline liver stiffness measurement (LSM) and spleen stiffness measurement (SSM) were done using Fibroscan 630 Expert and esophagogastroduodenoscopy was performed to detect VNT. Varices with red color signs or large size were considered VNT. A model was developed and validated. Decision curve analysis (DCA) was used to assess net benefit (NB) of model at different threshold probabilities.
RESULTS: Of the 216 patients analyzed (males 95.4%, mean age 42.2 yr, model for end-stage liver disease [MELD] score 25.5, alcohol etiology 83.4%), 104 (48.14%) had VNT. Patients with VNT had higher SSM (kPa) (76.71 ± 17.54 vs. 60.56 ± 20.21, p = 0.006), LSM (kPa) (71.96 ± 6.87 vs. 61.93 ± 17.76, p = 0.001) and lower platelet count (103 /µl) (91.73 ± 17.71 vs. 126.94 ± 42.97, p < 0.001). A model incorporating these parameters had an area under the receiver operating characteristic (AUROC) of 0.842 and could spare esophagogastroduodenoscopy in 34.3% of patients with missing VNT rate of 4.8%. The results were internally validated by bootstrap analysis. On DCA, NB of model was higher for threshold probabilities range 0.07 to 0.8. The model had good calibration with Brier score of 0.15. It performed well in alcohol and non-alcohol related causes with AUROCs of 0.830 and 0.884, respectively.
CONCLUSION: A model comprising SSM, LSM and platelet count identifies VNT non-invasively in ACLF patients.
PMID:42010208 | DOI:10.1007/s12664-025-01963-5
J Robot Surg. 2026 Apr 21;20(1):456. doi: 10.1007/s11701-026-03319-3.
ABSTRACT
The aim of the study is to evaluate the effectiveness of Individualised Counselling Session (ICS) on Robotic Assisted Surgery to reduce stress and anxiety among parents of children undergoing Robotic Assisted Surgery. The objective was to assess the level of pre-operative stress and anxiety among the parents of children undergoing Robotic Assisted Surgery in selected surgical wards in experimental and control groups. A Quasi experimental study design and 50 samples were taken. The samples were selected by purposive sampling techniques, who fulfilled the sample criteria. The demographic variable and structured questionnaire were used for data collection. The descriptive and inferential statistics were used for data analysis with the help of SPSS software version 27. Out of 50 samples (25 each in experimental and control group) the experimental group RAS type pyeloplasty counts for 10(41.7%), Ureteric Reimplantation 4(16.7%), Bladder reconstruction surgery 5(20.8%), Mitrofanoff surgery 6(20.8%). Length of the stay for patient before surgery were 4-8 days in 12 patients (50%), 8-10 days in 11 patient (45.8%), 10-12 days in 2 patient (4.2%). Level of education of father who were included in the study were secondary education counts for 2(4.2%), Senior secondary education 7(29.2%), Graduation 16(66.7%) whereas level of education of mother who were included in the study were primary education counts for 3(12.5%), secondary education 4(12.5%), senior secondary 11(45.8%), graduation 7(29.2%) under the control group male were included in the study were 22(88%) and female were 3(12%).The RAS type pyeloplasty counts for 12(48%), Ureteric Reimplantation 4(16%), Bladder reconstruction surgery 3(12%), Mitrofanoff surgery 6(24%). Length of the stay for patient before surgery were 4-8 days in 10 patients (40%), 8-10 days in 13 patient (52%), 10-12 days in 2 patient (8%). Level of education of father who were included in the study were primary education counts for 2(8%), Senior secondary education 9(36%), Graduation 14(56%) whereas level of education of mother who were included in the study were primary education counts for 2(8%), secondary education 6(24%), senior secondary 10(40%), graduation 7(28%). Effectiveness of Individualised counselling Session (ICS) among parents were assessed with the help of structured tool APAIS and B-MEPS for anxiety and stress respectively in experimental and control group. In the experimental group in pre-test their Median was 1 and 26 in stress and anxiety respectively and after giving the Individualised Counselling Session (ICS) their post Test Score was Median 0.78 and 26 in stress and anxiety respectively. After apply the Wilcoxon Signed rank Test the p-value was significant i.e. < 0.001 for both stress and anxiety. The Z-value was -4.374 and -4.391in stress and anxiety respectively. In the control group in pre-test median was 1 and 25 in stress and anxiety respectively, and the post- test score was 0.78 and 26 in stress and anxiety respectively. After applying the Wilcoxon signed rank test the p-value was < 0.001 for stress and 0.412 for anxiety. It shows significantly increase in anxiety but not stress. The Z-value was -4.05 and -0.821 in stress and anxiety respectively. Therefore, it is concluded that there is effectiveness in experimental group after giving the counselling their stress and anxiety got reduced whereas in control group their stress got reduced but anxiety was persistent pre-operatively.
PMID:42010175 | DOI:10.1007/s11701-026-03319-3
Pediatr Res. 2026 Apr 20. doi: 10.1038/s41390-026-05001-8. Online ahead of print.
ABSTRACT
BACKGROUND: Respiratory syncytial virus (RSV) is a leading cause of infant mortality and morbidity, yet national trends in RSV outcomes, particularly during the COVID-19 era, are not well defined. This study evaluated temporal patterns in RSV-related infant mortality and hospitalizations across the United States.
METHODS: We analyzed RSV-associated infant mortality data from 2007-2022 using the CDC WONDER database and hospitalization rates from 2018-2025 using the hospitalization surveillance network (RSV-NET). Infant mortality and hospitalization patterns were assessed by season, race/ethnicity, and birth weight.
RESULTS: Over 16 years, RSV-related infant mortality remained relatively stable, with no statistically significant year-to-year variation. Mortality rates were disproportionately higher among Black or African American infants and those with low birth weight. Seasonal hospitalization patterns varied substantially: the 2020-2021 RSV season recorded the lowest hospitalization rate, likely reflecting widespread COVID-19 mitigation measures, whereas the 2022-2023 season recorded the highest rate since 2018.
CONCLUSION: Although infant RSV mortality has remained stable, persistent disparities exist, and hospitalization rates show substantial seasonal fluctuations. These findings provide critical baseline data for assessing the impact of emerging RSV immunization strategies and highlight the need for targeted strategies to reduce persistent inequities in infant outcomes in the post-COVID-19 era.
IMPACT: RSV-related infant mortality in the United States has shown both stability and a sharp post-pandemic surge, with persisting disparities by sex, birthweight and race. This study integrates temporal, demographic, and geographic patterns to provide the national-level evidence of RSV mortality and hospitalization patterns among infants during COVID-19 era. These findings establish essential baseline estimates to evaluate effectiveness of preventive measures, while underscoring the urgency of targeted strategies to reduce disproportionate risks among vulnerable infants.
PMID:42010159 | DOI:10.1038/s41390-026-05001-8
Clin Neuroradiol. 2026 Apr 20. doi: 10.1007/s00062-026-01654-4. Online ahead of print.
ABSTRACT
PURPOSE: Despite advances in mechanical thrombectomy for acute ischemic stroke (AIS), the relationship between successful angiographic reperfusion and functional outcome remains imperfect. We investigated the value of an optimal immediate postthrombectomy National Institutes of Health Stroke Scale (NIHSS) cutoff, termed the “NIHSS drop,” as a predictor of favorable 90-day outcome and compared its prognostic performance with first-pass effect (FPE).
METHODS: We conducted a single-center retrospective analysis of prospectively collected data from 554 patients with AIS who underwent mechanical thrombectomy between 2018 and 2024. Eligible patients presented with an NIHSS score > 8, an Alberta Stroke Program Early CT Score (ASPECTS) of 6-10, and M1 occlusion. Immediate postprocedural NIHSS assessment allowed calculation of the NIHSS drop. Receiver operating characteristic (ROC) analysis was used to determine the optimal cutoff, and logistic regression was used to assess its predictive value after adjustment for complete reperfusion (Thrombolysis in Cerebral Infarction [TICI] grade 3 vs 2b), FPE, and other major confounders.
RESULTS: An NIHSS drop ≥ 6 was strongly associated with favorable outcome (area under the curve [AUC] = 0.762, sensitivity 63%, specificity 82%). Patients with an NIHSS drop ≥ 6 (n = 235) had better outcomes (modified Rankin Scale [mRS] 0-2: 83% vs 40%, p < 0.001), fewer complications, and lower median NIHSS scores at 24 h (3 vs 12) and at discharge (1 vs 5), as well as lower in-hospital mortality (1.7% vs 15%, p < 0.001) and 90-day mortality (3.0% vs 19%, p < 0.001). In multivariable analysis, an NIHSS drop ≥ 6 remained the strongest predictor of favorable outcome (odds ratio [OR] 7.21, 95% confidence interval [CI] 4.62-11.5; p < 0.001), showing a stronger association than complete reperfusion or FPE individually.
CONCLUSION: Immediate neurological improvement after thrombectomy was strongly associated with favorable functional outcome. An NIHSS drop ≥ 6 may serve as a useful early prognostic marker and may complement traditional procedural metrics such as TICI grade and FPE, although prospective validation is needed.
PMID:42010130 | DOI:10.1007/s00062-026-01654-4