Category: Statistics

JAMA Netw Open. 2025 Apr 1;8(4):e256193. doi: 10.1001/jamanetworkopen.2025.6193.

ABSTRACT

IMPORTANCE: Excess cryopreserved embryos present financial, logistical, and ethical constraints on patients and fertility clinics that are further complicated by the recently evolving US judicial landscape that may alter the legal status of frozen embryos and limit disposition options. It is therefore important to consider limiting the number of supernumerary embryos created from fresh donor oocytes.

OBJECTIVE: To determine the optimal number of fresh donor oocytes to fertilize to achieve 1 live birth while minimizing supernumerary blastocysts.

DESIGN, SETTING, AND PARTICIPANTS: This cohort study used data from recipients of fresh donor oocytes at member clinics of the Society for Assisted Reproductive Technology Clinic Outcomes Report System (SART CORS) between January 1, 2016, and December 31, 2020. Data were analyzed between August 2023 and June 2024.

MAIN OUTCOMES AND MEASURES: The number of supernumerary blastocysts for a given number of fresh donor oocytes retrieved.

RESULTS: There were 9395 fresh donor oocyte recipients included. The median (IQR) oocyte donor age at oocyte retrieval was 26 years (24-28 years), and the median (IQR) patient age at cycle start was 42 years (38-45 years). The median (IQR) number of oocytes retrieved was 20 (14-28), fertilized oocytes (2 pronuclei [2PN]) was 11 (7-17), usable embryos was 6 (3-10), and supernumerary blastocysts was 5 (2-8). When patients were divided evenly into quartiles based on the number of fresh donor oocytes retrieved (14 or fewer, 15 to 20, 21 to 28, 29 or more), it was associated with a significant increase in the number of cryopreserved supernumerary blastocysts (median [IQR] blastocysts by quartile: 2 [1-4] vs 4 [2-7] vs 6 [3-9] vs 9 [4-14], respectively; P < .001). However, the live birth rate was lower only for those in the first quartile receiving 14 or fewer oocytes (50.8% [1207 of 2376] vs 64.1% [1497 of 2336] vs 67.9% [1631 of 2401] vs 67.9% [1550 of 2282]; P < .001).

CONCLUSIONS AND RELEVANCE: In this retrospective cohort study of fresh donor oocyte cycles, the number of supernumerary blastocysts to achieve 1 live birth for a given number of fresh donor oocytes exposed to sperm was approximated, which may be valuable for counseling patients.

PMID:40257793 | DOI:10.1001/jamanetworkopen.2025.6193

JAMA Pediatr. 2025 Apr 21. doi: 10.1001/jamapediatrics.2025.0560. Online ahead of print.

ABSTRACT

IMPORTANCE: Toddler’s fractures (TF) of the tibia are commonly treated with casts and fracture clinic follow-up. However, a prefabricated removable boot might be sufficient and reduce unnecessary follow-up.

OBJECTIVES: To determine whether pain in children with TF treated with a removable boot is not worse than those managed with a circumferential cast at 4 weeks postinjury.

DESIGN, SETTING, AND PARTICIPANTS: This pragmatic, multicenter, assessor-blinded, noninferiority randomized clinical trial was conducted between October 2019 and February 2024 at 4 urban, tertiary care, pediatric Canadian emergency departments. Children aged between 9 months and 4 years with a radiograph-visible TF were eligible for inclusion.

INTERVENTIONS: Prefabricated walking boot for up to 3 weeks (removable at caregivers’ discretion) vs circumferential cast immobilization (site standard of care) for 3 weeks.

MAIN OUTCOMES AND MEASURE: The primary outcome was pain score, measured with the Evaluation Enfant Douleur (EVENDOL) scale (maximum score: 15). Additional outcomes included return to baseline activities, complications, parental satisfaction and care burden.

RESULTS: In 129 enrolled children, the mean (SD) age was 2.2 (0.8) years, and 56 children (43%) were female. Of these, 118 children (92%) completed the 4-week follow-up, and the boot (n = 64) vs cast (n = 54) groups demonstrated mean (SD) EVENDOL pain scores of 1.21 (1.54) and 1.76 (2.13), respectively (difference, -0.55; 95% CI, -1.23 to 0.13). The percentage of participants who returned to baseline activities “almost all of the time” was 49 of 64 children (77%) in the boot group and 22 of 54 (41%) in the cast group (difference, 36%; 95% CI, 9%-63%). Skin complications occurred in both groups (boot: 46 total complications [72%], with 5 stage 1 pressure sores; cast: 27 total complications (50%), with 1 pressure sore]; difference, 22%; 95% CI, -6% to 50%). There was no statistically significant difference in the percentage of caregivers who were satisfied or very satisfied with the treatment (difference, 9%; 95% CI, -24% to 43%). Fewer caregivers reported bathing care burden (difference, -32%; 95% CI, -47% to -18%) and challenges with carrying the child (difference, -21%; 95% CI, -27% to -15%) in the boot vs cast group.

CONCLUSIONS AND RELEVANCE: In this multicenter randomized clinical trial examining the management of children with TF, a removable boot without physician follow-up was noninferior to circumferential casting with respect to pain recovery. While there was a clinically relevant but not statistically significant trend toward more skin complications in the boot group, there was no difference in caregiver satisfaction, and the boot strategy demonstrated reduced childcare-related challenges.

TRIAL REGISTRATION: ClinicalTrials.gov Identifier: NCT03971448.

PMID:40257790 | DOI:10.1001/jamapediatrics.2025.0560

Invest Ophthalmol Vis Sci. 2025 Apr 1;66(4):54. doi: 10.1167/iovs.66.4.54.

ABSTRACT

PURPOSE: We determined the association between Life’s Essential 8 (LE8) scores and AMD incidence and ascertained whether genetic susceptibility modifies it.

METHODS: This prospective cohort study included 268,634 UK Biobank study participants with high, moderate, or low cardiovascular health based on LE8 scores. High and low cardiovascular health corresponded to the lowest and highest cardiovascular disease risks, respectively. The hazard ratios (HRs) and 95% confidence intervals (CIs) for AMD were estimated using Cox proportional hazards models. The dose-response relationships were evaluated using restricted cubic spline analysis. Stratified analyses using the AMD-polygenic risk score categories were used to assess potential modification. A composite variable combined LE8 and AMD-polygenic risk to examine their joint effects.

RESULTS: Over an average of 13.76 years, 5253 participants developed AMD. Compared with the lowest cardiovascular health, moderate and high cardiovascular health had adjusted HRs of 0.86 (95% CI, 0.76, 0.97) and 0.79 (0.69, 0.91), respectively. Each standard deviation increase in the LE8 score corresponded to an HR of 0.93 (0.91, 0.96). The LE8 behavior (HR = 0.94 [0.91, 0.96]) and biological (HR = 0.97 [0.95, 1.00]) subscale scores were inversely associated with AMD, whereas the blood lipids component was positively associated (HR = 1.07 [1.04, 1.10]). The AMD-polygenic risk and LE8 scores showed no significant interaction. The HR for having low AMD-polygenic risk and high cardiovascular health relative to having high AMD-polygenic risk and low cardiovascular health was 0.47 (0.37, 0.58).

CONCLUSIONS: Maintaining good cardiovascular health can reduce AMD incidence regardless of genetic background.

PMID:40257787 | DOI:10.1167/iovs.66.4.54

Invest Ophthalmol Vis Sci. 2025 Apr 1;66(4):58. doi: 10.1167/iovs.66.4.58.

ABSTRACT

PURPOSE: To compare the impact of sodium-glucose cotransporter 2 (SGLT2) and dipeptidyl peptidase 4 (DPP-4) inhibitors on age-related macular degeneration (AMD) risk among patients with type 2 diabetes mellitus (T2DM).

METHODS: This multinational, retrospective cohort study used electronic medical records from healthcare institutions across 21 countries. Adults 50 years or older with T2DM who had a prior prescription of metformin and initiated SGLT2 or DPP-4 inhibitors from 2013 to 2023 were included. The SGLT2 and DPP-4 inhibitor groups were propensity score matched in a 1:1 ratio to balance baseline characteristics and were followed for up to 5 years to observe the occurrence of AMD. Statistical analysis was performed using the Cox proportional hazards model and Kaplan-Meier analysis.

RESULTS: Our final analysis included 20,966 T2DM patients prescribed SGLT2 inhibitors and 20,966 prescribed DPP-4 inhibitors. Compared to the DPP-4 inhibitor group, the SGLT2 inhibitor group was associated with significantly lower risks of AMD (hazard ratio [HR], 0.71; 95% confidence interval [CI], 0.58-0.85) and dry AMD (HR, 0.61; 95% CI, 0.46-0.80) but not wet AMD (HR, 0.74; 95% CI, 0.48-1.16). SGLT2 inhibitors compared with DPP-4 inhibitors were linked to a reduced risk of AMD in the White population, patients prescribed empagliflozin or dapagliflozin, and individuals with glycated hemoglobin < 8.5%, estimated glomerular filtration rate ≥ 60 mL/min/1.73 m2, hypertension, or dyslipidemia, regardless of body mass index level.

CONCLUSIONS: In patients with T2DM, those prescribed SGLT2 inhibitors may experience lower risks of AMD and dry AMD compared to those prescribed DPP-4 inhibitors.

PMID:40257783 | DOI:10.1167/iovs.66.4.58

Invest Ophthalmol Vis Sci. 2025 Apr 1;66(4):60. doi: 10.1167/iovs.66.4.60.

ABSTRACT

PURPOSE: To analyze the clinical spectrum and natural history of MYO7A-associated Usher syndrome type I (USH1).

METHODS: Patients with molecularly confirmed MYO7A-associated USH1 in a single tertiary referral center. Data was extracted from physical and electronic case notes, including imaging and electrophysiology. Genetic results were reviewed, and the detected variants were assessed. Main outcome measures were clinical findings, qualitative and quantitative analysis of retinal imaging, and electrophysiology.

RESULTS: Eighty patients were identified and evaluated longitudinally. The mean age (±SD) of onset of symptoms was 12.0 ± 5.8 years of age, and a mean follow-up time of 16.2 years. BCVA was 0.4 ± 0.5 LogMAR at baseline, and 0.7 ± 0.8 LogMAR at the last visit for both eyes. The change in BCVA over time was 0.02 LogMAR per year. A hyperautofluorescent (hyperAF) ring was present in 51% of the patients. The mean ellipsoid zone width (EZW) at baseline was 2568.2 ± 1528.9 µm OD and 2527.9 ± 1609.3 µm OS, which decreased to 2012.3 ± 1705.1 µm OD and 1806.3 ± 1647.1 µm OS at last visit. Electrophysiology revealed rod and cone dysfunction with relative or complete sparing of macular function. There were statistically significant changes in BCVA, EZW, and hyperAF ring between baseline and follow-up. Genetic analysis identified 83 variants in MYO7A, including 18 novel variants.

CONCLUSIONS: Longitudinal analysis shows that the majority of patients retain central visual function and structure until the fifth decade of life, which informs advice on prognosis and the window for therapeutic intervention.

PMID:40257781 | DOI:10.1167/iovs.66.4.60

Int J Palliat Nurs. 2025 Apr 2;31(4):173-180. doi: 10.12968/ijpn.2023.0044.

ABSTRACT

BACKGROUND: Patients with cancer can have problems sleeping. One measure that can help patients with cancer to improve the quality of their sleep is the 5A Self Management Model method.

AIM: This study aimed to determine the effect of the 5A Self Management Model on the quality of sleep of patients with cancer.

METHODS: In this clinical trial, 106 patients with cancer were recruited by convenient sampling, and randomly allocated to experimental and control groups. Data collection tools were a demographic checklist and Pittsburgh Sleep Quality Questionnaire. Patients in the experimental group received a self-management programme with the 5A method for 3 months and the control group obtained the routine intervention. Data were analysed via SPSS 25 software using Wilcoxon, Mann-Whitney U, independent t-test, paired t-test and descriptive statistics.

RESULTS: Before the intervention, the mean scores of sleep quality were 13.41 (1.78) in the experimental group and 9.64 (2.45) in the control group. However, after the intervention, the mean score of sleep quality decreased in the experimental group to 5.07 (1.19) and increased by 12.81 (2.26) in the control group, significantly.

CONCLUSION: The 5A Self Management Model programme improved the quality of sleep of patients with cancer in this trial. It is recommended nurses consider this method as a part of caring for patients with cancer.

PMID:40257767 | DOI:10.12968/ijpn.2023.0044

Int J Palliat Nurs. 2025 Apr 2;31(4):195-204. doi: 10.12968/ijpn.2024.0031.

ABSTRACT

BACKGROUND: Few studies evaluate the satisfaction of patients with cancer receiving palliative care with the nursing care received.

OBJECTIVE: To evaluate satisfaction among patients with cancer in hospitalised palliative care with respect to the nursing care they received.

METHOD: This was a quantitative, descriptive-correlational and cross-sectional study that was reported in accordance with Strengthening the Reporting of Observational Studies in Epidemiology (STROBE) guidelines. It was carried out in a university hospital in the city of Belém, Brazil. The participants were adults with cancer who were receiving palliative care and who had been hospitalised for more than 72 hours. Data collection took place between December 2022 and September 2023, and instruments were used for bedside interviews.

RESULTS: Patients receiving minimal care reported being satisfied with their nursing care in the technical-professional dimension (p=0.04).

CONCLUSION: The association between patient satisfaction and the degree of dependence is unprecedented in the scientific scenario and has the potential to motivate more in-depth studies in this area.

PMID:40257762 | DOI:10.12968/ijpn.2024.0031

JAMA Pediatr. 2025 Apr 21. doi: 10.1001/jamapediatrics.2025.0528. Online ahead of print.

NO ABSTRACT

PMID:40257760 | DOI:10.1001/jamapediatrics.2025.0528

JMIR Form Res. 2025 Apr 21;9:e68173. doi: 10.2196/68173.

ABSTRACT

BACKGROUND: Previous research has demonstrated a correlation between nursing students’ self-efficacy and their clinical performance, competence, and behavior during clinical practice placements. Assessing students’ self-efficacy in clinical performance could be a valuable method for identifying areas that need reinforcement and for recognizing students who may require additional support during clinical practice placements.

OBJECTIVE: This study aimed to translate the Self-Efficacy in Clinical Performance Scale (SECP) from English into Norwegian and to evaluate the psychometric properties of the Norwegian version.

METHODS: A cross-sectional study design was used. The SECP was translated into Norwegian following a 6-step process: forward translation, forward translation synthesis, backward translation, backward translation synthesis, cognitive debriefing, and psychometric testing. The validity and reliability of the translated version were assessed using confirmatory factor analysis (CFA), Cronbach α, McDonald ω, and composite reliability.

RESULTS: A total of 399 nursing students completed the Norwegian version of the SECP. The CFA goodness-of-fit indices (χ2/df ratio=1.578, comparative fit index=0.98, Tucker-Lewis index=0.98, standardized root mean square residual=0.056, root mean square error of approximation=0.038) indicated an acceptable model fit. Reliability measures, including Cronbach ⍺, McDonald ω, and composite reliability, were high, with factor-level values ranging from 0.94 to 0.98.

CONCLUSIONS: The Norwegian version of the SECP demonstrated strong potential as an instrument for assessing self-efficacy in both current and required competencies among nursing students in clinical practice within nursing education. Future research should aim to confirm the factor structure of the SECP and evaluate its test-retest reliability.

PMID:40257751 | DOI:10.2196/68173

Bioinformatics. 2025 Apr 21:btaf182. doi: 10.1093/bioinformatics/btaf182. Online ahead of print.

ABSTRACT

MOTIVATION: The intra-tumor heterogeneity of protein expression is well recognized and may provide important information for cancer prognosis and predicting treatment responses. Analytic methods that account for spatial heterogeneity remain methodologically complex and computationally demanding for single-cell protein expression. For many functional proteins, single-cell expressions vary independently of spatial localization in substantial proportion of the tumor tissues, and incorporation of spatial information may not affect the prognostic value of such protein biomarkers.

RESULTS: We developed a new framework for using the distributions of functional single-cell protein expression levels as cancer biomarkers. The quantile functions of single-cell expressions are used to fully capture the heterogeneity of protein expression across all cancer cells. The quantile index (QI) biomarker is defined as an integral of an unspecified function which may depend linearly or nonlinearly on a tissue-specific quantile function. Linear and nonlinear versions of QI biomarkers based on single-cell expressions of ER, Ki67, TS, and CyclinD3 were derived and evaluated as predictors of progression-free survival or high mitotic index in a large breast cancer dataset. We evaluated performance and demonstrated advantages of non-linear QI biomarkers through simulation studies.

AVAILABILITY: The associated R package Qindex is available at https://CRAN.R-project.org/package=Qindex and R package hyper.gam is available at https://github.com/tingtingzhan/hyper.gam. Examples of R code and detailed instructions could be found in vignette quantile-index-predictor (https://CRAN.R-project.org/package=hyper.gam/vignettes/applications.html#quantile-index-predictor).

SUPPLEMENTARY INFORMATION: Supplementary Material is available at Bioinformatics online.

PMID:40257750 | DOI:10.1093/bioinformatics/btaf182