Category: Statistics

Int J Clin Oncol. 2024 Aug 17. doi: 10.1007/s10147-024-02597-x. Online ahead of print.

ABSTRACT

BACKGROUND: This retrospective observational study explored the therapeutic potential of combined androgen blockade (CAB) with bicalutamide (Bic-CAB) as an initial treatment for metastatic hormone-sensitive prostate cancer (mHSPC) in Japan.

METHODS: The electronic health records of 159 patients with mHSPC from three Japanese institutions who received initial treatment with Bic-CAB between 2007 and 2017 were analyzed. The time to prostate-specific antigen (PSA) progression, duration of Bic-CAB treatment, and overall survival (OS), with various definitions for PSA progression, were assessed. A multivariate Cox proportional hazards model was constructed using clinical parameters to predict time to the end of Bic-CAB treatment and OS.

RESULTS: The median observation period was 46.4 months, and the median age of patients at diagnosis was 71 years. A total of 46.5% patients experienced PSA progression with a median survival duration of 29 months (according to Prostate Cancer Clinical Trials Working Group 3 criteria), and 49.1% patients achieved a PSA nadir < 0.2 ng/mL in a median time of 4.7 months. When stratified by PSA nadir and PSA change, patients at low risk for disease progression with a small PSA change due to low initial PSA had a 5-year OS of 100% and a 10-year OS of 75%. The OS during the observation period was 72.9 months.

CONCLUSION: These findings highlight the potential effect of Bic-CAB in patients with mHSPC who were at low risk for disease progression. Initial treatment with Bic-CAB and adjusting treatment early based on PSA dynamics may be a reasonable treatment plan for these patients.

PMID:39153094 | DOI:10.1007/s10147-024-02597-x

Neuroradiology. 2024 Aug 17. doi: 10.1007/s00234-024-03450-8. Online ahead of print.

ABSTRACT

BACKGROUND: It is difficult to distinguish between tumor progression (TP) and treatment-related abnormalities (TRA) in treated glioblastoma patients via conventional MRI, but this distinction is crucial for treatment decision making. Glioblastoma is known to exhibit an invasive growth pattern along white matter architecture and vasculature. This study quantified lesion development patterns in treated glioblastoma lesions and their relation to white matter microstructure to distinguish TP from TRA.

MATERIALS AND METHODS: Glioblastoma patients with confirmed TP or TRA with T1-weighted contrast-enhanced and DTI MR scans from two posttreatment follow-up timepoints were reviewed. The contrast-enhancing regions were segmented, and the regions were coregistered to the DTI data. Lesion increase vectors were categorized into two groups: parallel (0-20 degrees) and perpendicular (70-90 degrees) to white matter. FA-values were also extracted. To test for a statistically significant difference between the TP and TRA groups, a Mann‒Whitney U test was performed.

RESULTS: Of 73 glioblastoma patients, fifteen were diagnosed with TRA, whereas 58 patients suffered TP. TP had a 25.8% (95% CI 24.1%-27.6%) increase in parallel lesions, and TRA had a 25.4% (95% CI 20.9%-29.9%) increase in parallel lesions. The perpendicular increase was 14.7% for TP (95% CI 13.0%-16.4%) and 18.0% (95% CI 13.5%-22.5%) for TRA. These results were not significantly different (p = 0.978). FA value for TP showed to be 0.248 (SD = 0.054) and for TRA it was 0.231 (SD = 0.075), showing no statistically significant difference (p = 0.121).

CONCLUSIONS: Based on our results, quantifying posttreatment contrast-enhancing lesion development directionality with DTI in glioblastoma patients does not appear to effectively distinguish between TP and TRA.

PMID:39153088 | DOI:10.1007/s00234-024-03450-8

Arch Dermatol Res. 2024 Aug 17;316(8):530. doi: 10.1007/s00403-024-03249-w.

ABSTRACT

Patients’ experience accessing dermatologic care is understudied. The purpose of this cross-sectional study was to examine current wait times for new patients to receive dermatological care in NYC. Websites at 58 accredited private and public hospitals in the five boroughs of NYC were reviewed to identify dermatology practices. Office telephone numbers listed on each website were called to collect information pertaining to whether the physician was accepting new patients, type of insurance accepted (public, private, both, or none), and the number of days until a new patient could be seen for an appointment. Data pertaining to the time kept on hold and availability of web-based booking were also collected. Mean waiting time for an appointment was 50 days [standard deviation, SD 66] – nearly 2 months, but the distribution was considerably skewed. The median waiting time was 19.5 days [Interquartile range, IQR 4-60]. The time kept on hold to make the appointment was negligible at about 1 min (63 s, SD = 77) but could take up to ~ 7 min. Two-thirds of dermatologists accepted private, Medicare, and Medicaid insurance (n = 228, 66%); a small number accepted only private insurance (n = 12, 4%) or no insurance at all (n = 16, 5%). The median waiting time for an appointment for the 228 providers that accepted Medicaid was 30.5 days (IQR = 5.0-73.25) while for providers who did not accept Medicaid (n = 116) the median wait time for an appointment was 13.0 days (IQR = 3.0-38.0). Just over half (56%) of the dermatologists allowed for appointments to be booked on their website (n = 193). This research highlights the necessity of incorporating new strategies into routine dermatology appointments in order to increase treatment availability and decrease healthcare inequality.

PMID:39153084 | DOI:10.1007/s00403-024-03249-w

Rheumatol Int. 2024 Aug 17. doi: 10.1007/s00296-024-05690-6. Online ahead of print.

ABSTRACT

Systemic lupus erythematosus (SLE) is a chronic autoimmune disease that affects women of childbearing age and has been reported to cause sexual dysfunction in women. Although there are articles on sexual function in women with SLE, the number of articles is small, and the factors affecting sexual function in women with SLE are controversial. Based on this, this study aimed to investigate the prevalence of sexual dysfunction in Chinese female SLE patients and to explore the factors that influence it. The study design was a cross-sectional study conducted from December 2023 to April 2024 in the Department of Rheumatology and Immunology of a tertiary hospital in Hefei, Anhui Province. A total of 293 female patients diagnosed with SLE were enrolled using face-to-face questionnaires and online questionnaires. The questionnaire consisted of four parts: general information questionnaire, fatigue severity scale (FSS), depression-anxiety-stress scale (DASS-21), and female sexual functioning index (FSFI) scale. A total of 173 (59.04%) patients had sexual dysfunction, including 251 (85.67%) with decreased libido and 186 (63.46%) with difficulty in sexual arousal. There was a correlation between the patients’ total FSFI scores and age (p = 0.028), marital satisfaction (p < 0.001), own education level (p = 0.008), partner’s education level (p = 0.003), place of residence (p = 0.039), monthly household income (p < 0.001), family financial satisfaction(p < 0.001), menstrual status (p = 0.003), hormone use (p = 0.021),immunosuppressant use (p = 0.042), disease activity (p = 0.016), FSS score (p < 0.001), stress score (p < 0.001), anxiety score (p < 0.001) and depression score (p < 0.001)were correlated. The results of stepwise regression analysis showed that marital satisfaction (b = 2.011, t = 3.797, p < 0.001), monthly household income (b = 0.854, t = 2.316, p = 0.021), menstrual status (b = 1.218, t = 2.350, p = 0.019), fatigue scale score (b = – 0.069, t = – 2.302, p = 0.022), and depression score (b = – 0.117, t = – 2.910, p = 0.004) were the influencing factors of FSFI total score, and the difference was statistically significant. The incidence of sexual dysfunction in Chinese female SLE patients is high, and medical personnel should pay more attention to patients’ sexual problems, to provide theoretical and practical bases for further prevention, treatment, and care of sexual dysfunction in female SLE patients.

PMID:39153077 | DOI:10.1007/s00296-024-05690-6

Int Urogynecol J. 2024 Aug 17. doi: 10.1007/s00192-024-05889-y. Online ahead of print.

ABSTRACT

INTRODUCTION AND HYPOTHESIS: Condition-specific sexual questionnaires are important patient-reported outcome measures. The aim of this study was to translate and validate the Pelvic Organ Prolapse/Incontinence Sexual Questionnaire-International Urogynecology Association Revised (PISQ-IR) into Brazilian Portuguese and to clinically validate it in a Brazilian Portuguese-speaking population.

METHODS: Translation and validation of the PISQ-IR was performed according to the International Urogynecological Association-recommended process and guidelines. For external validity, PISQ-IR subscales were compared with the clinical measures, Pelvic Organ Prolapse Quantification system (POP-Q) stage, pelvic floor muscle tone, and Oxford Grading Scale (Pearson correlations). Descriptive statistics, internal consistency (Cronbach’s alpha coefficient), and test-retest reliability (interclass correlation coefficient) were calculated for all PISQ-IR subscales.

RESULTS: A total of 120 sexually active and 106 not sexually active women were enrolled in the study between March 2015 and July 2019. Internal consistency was acceptable, with Cronbach’s alpha values 0.60-0.80, except for the sexual arousal and orgasm, sexual arousal and partner-related issues, sexual arousal and condition-specific issues, global rating of sexual quality and condition impact subscales. PISQ-IR demonstrated good reliability (α > 0.6, CIC = 0.996). The agreement for each individual questionnaire item also individually presented substantial agreement between the assessments (κ 0.61-0.8). There was a correlation between PISQ-IR and POP, mixed, stress and fecal incontinence diagnosis and a positive correlation with pelvic floor muscle function according to the Oxford Scale in sexually active women. For sexually inactive women there was a correlation between PISQ-IR and mixed urinary symptoms.

CONCLUSIONS: The Brazilian Portuguese version of PISQ-IR is a reliable and valid tool that can be easily used for the identification and assessment of sexual function in Brazilian Portuguese-speaking women with pelvic floor disorders.

PMID:39153070 | DOI:10.1007/s00192-024-05889-y

Environ Sci Pollut Res Int. 2024 Aug 17. doi: 10.1007/s11356-024-34713-9. Online ahead of print.

ABSTRACT

Twenty-two eco-friendly, novel Schiff bases were synthesized from 2,4,5-trichloro aniline and characterized by using FT-IR, ¹H NMR, and ¹³C NMR techniques. Fungicidal activity against pathogenic fungi Sclerotium rolfsii and Rhizoctonia bataticola and insecticidal activity against the stored grain insect pest Callosobruchus maculatus of the test compounds were evaluated under control condition. All of the investigated compounds, according to the study, exhibited moderate to good antifungal and insecticidal activities. The best antifungal activity against both pathogenic fungi was demonstrated by C15 and C16 whose ED₅₀ values were recorded 11.4 and 10.4 μg/mL against R. bataticola and 10.6 and 11.9 μg/mL against S. rolfsii, respectively. They were further screened in for disease suppression against both pathogenic fungi under pot condition through different methods of applications in green gram (Vigna radiata L.) crop. The compounds C10 and C18 had the highest insecticidal activity, with LD₅₀ values of 0.024 and 0.042 percentages, respectively. Stepwise regression analysis using root mean square error (RMSE) and correlation coefficient (R) method used to validate the quantitative structure activity relationship (QSAR) of synthesized compounds in addition to their fungicidal and insecticidal actions. To the best of our knowledge, this investigation on the 22 new Schiff bases as possible agrochemicals is the first one that has been fully reported.

PMID:39153063 | DOI:10.1007/s11356-024-34713-9

Dysphagia. 2024 Aug 17. doi: 10.1007/s00455-024-10738-7. Online ahead of print.

ABSTRACT

Multiple bolus trials are administered during clinical and research swallowing assessments to comprehensively capture an individual’s swallowing function. Despite valuable information obtained from these boluses, it remains common practice to use a single bolus (e.g., the worst score) to describe the degree of dysfunction. Researchers also often collapse continuous or ordinal swallowing measures into categories, potentially exacerbating information loss. These practices may adversely affect statistical power to detect and estimate smaller, yet potentially meaningful, treatment effects. This study sought to examine the impact of aggregating and categorizing penetration-aspiration scale (PAS) scores on statistical power and effect size estimates. We used a Monte Carlo approach to simulate three hypothetical within-subject treatment studies in Parkinson’s disease and head and neck cancer across a range of data characteristics (e.g., sample size, number of bolus trials, variability). Different statistical models (aggregated or multilevel) as well as various PAS reduction approaches (i.e., types of categorizations) were performed to examine their impact on power and the accuracy of effect size estimates. Across all scenarios, multilevel models demonstrated higher statistical power to detect group-level longitudinal change and more accurate estimates compared to aggregated (worst score) models. Categorizing PAS scores also reduced power and biased effect size estimates compared to an ordinal approach, though this depended on the type of categorization and baseline PAS distribution. Multilevel models should be considered as a more robust approach for the statistical analysis of multiple boluses administered in standardized swallowing protocols due to its high sensitivity and accuracy to compare group-level changes in swallowing function. Importantly, this finding appears to be consistent across patient populations with distinct pathophysiology (i.e., PD and HNC) and patterns of airway invasion. The decision to categorize a continuous or ordinal outcome should be grounded in the clinical or research question with recognition that scale reduction may negatively affect the quality of statistical inferences in certain scenarios.

PMID:39153045 | DOI:10.1007/s00455-024-10738-7

J Neurooncol. 2024 Aug 17. doi: 10.1007/s11060-024-04738-8. Online ahead of print.

ABSTRACT

PURPOSE: Stereotactic Radiosurgery (SRS) is the primary treatment for patients with limited numbers of small brain metastases. Head fixation is usually performed with framed-based (FB) fixation; however, mask-based (MB) fixation has emerged as a less invasive alternative. A comparative meta-analysis between both approaches has not been performed.

METHODS: Databases were searched until August 28th, 2023, to identify studies comparing MB and FB SRS in the treatment of brain metastases. Our outcomes of interest included local tumor control (LTC), radiation necrosis (RN), mortality, and treatment time (TT). Mean difference (MD), risk ratio (RR), and hazard ratio (HR) were used for statistical comparisons.

RESULTS: From 295 articles initially identified, six studies (1 clinical trial) involving 509 patients were included. LTC revealed comparable RR at 6-months (RR = 0.95[95%CI = 0.89-1.01], p = 0.12) and a marginal benefit in FB SRS at 1-year (RR = 0.87[95%CI = 0.78-0.96], p = 0.005). However, in oligometastases exclusively treated with single-fraction SRS, LTC was similar among groups (RR = 0.92 [95%CI = 0.89-1.0], p = 0.30). Similarly, in patients with oligometastases treated with single-fraction SRS, RN (HR = 1.69; 95%CI = 0.72-3.97, p = 0.22), TT (MD = -29.64; 95%CI = -80.38-21.10, p = 0.25), and mortality were similar among groups (RR = 0.62; 95%CI = 0.22-1.76, p = 0.37).

CONCLUSION: Our findings suggest that FB and MB SRS, particularly oligometastases treated with single-fraction, are comparable in terms of LTC, RN, TT, and mortality. Further research is essential to draw definitive conclusions.

PMID:39153030 | DOI:10.1007/s11060-024-04738-8

Clin Oral Investig. 2024 Aug 17;28(9):489. doi: 10.1007/s00784-024-05883-0.

ABSTRACT

OBJECTIVE: The present study aimed to determine the salivary flow and metabolomic profile of stimulated and unstimulated saliva in children.

MATERIALS AND METHODS: Children who attended the Pediatric Dentistry Clinic of the State University of Rio de Janeiro -UERJ between 3 and 12 years of age were selected. Unstimulated and stimulated whole saliva, using mechanical stimulus, were collected. The samples were centrifuged at 12,000 g, 4^oC for 1 h. The ¹H- NMR spectra were acquired in 500 MHz equipment. The data were extracted into 0.03 ppm buckets in AMIX, and multivariate analysis (PLS-DA and O-PLS-DA) was performed in Metaboanalyst 2.0. For other analyses, such as salivary flow, the data was tabulated in the SPSS 20.0 statistical package, analyzed descriptively, and after applying the Wilcoxon test. The interval of confidence was set at 95%.

RESULTS: The mean age was 7.5 (± 1.94), and 47.0% (n = 31) were female, 63.6% (n = 42). The median flow rate for stimulated saliva was 0.74 (IC 0.10-2.40) and was statistically higher (p < 0.001; Wilcoxon test) than unstimulated was 0.39 (IC 0.00-1.80). Children older than seven years old also presented a higher difference between unstimulated and stimulated saliva (p = 0.003; Mann-Whitney test). The PLS-DA and O-PLS-DA demonstrated a different profile in stimulated and unstimulated saliva. Acetate, glucose, propionate, and lysine were higher in the unstimulated whole saliva than in stimulated saliva. Isoleucine, N-acetyl sugar, hydroxybutyrate, glutamate, leucine, propionate, butyrate, valine, isoleucine, succinate, saturated fatty acid, and histidine were found in greater amounts in the saliva of patients with stimulated saliva.

CONCLUSION: The stimulated saliva presented a higher flow rate, and older children exhibited a higher flow rate resulting from it’s the stimulus. The mechanical stimulus increased the levels of the major metabolites.

PMID:39153029 | DOI:10.1007/s00784-024-05883-0

Psychometrika. 2024 Aug 17. doi: 10.1007/s11336-024-09997-y. Online ahead of print.

ABSTRACT

A well-known person fit statistic in the item response theory (IRT) literature is the $l_z$ statistic (Drasgow et al. in Br J Math Stat Psychol 38(1):67-86, 1985). Snijders (Psychometrika 66(3):331-342, 2001) derived $l_z^{\ast }$ , which is the asymptotically correct version of $l_z$ when the ability parameter is estimated. However, both statistics and other extensions later developed concern either only the unidimensional IRT models or multidimensional models that require a joint estimate of latent traits across all the dimensions. Considering a marginalized maximum likelihood ability estimator, this paper proposes $l_{\mathrm{zt}}$ and $l_{\mathrm{zt}}^{\ast }$ , which are extensions of $l_z$ and $l_z^{\ast }$ , respectively, for the Rasch testlet model. The computation of $l_{\mathrm{zt}}^{\ast }$ relies on several extensions of the Lord-Wingersky algorithm (1984) that are additional contributions of this paper. Simulation results show that $l_{\mathrm{zt}}^{\ast }$ has close-to-nominal Type I error rates and satisfactory power for detecting aberrant responses. For unidimensional models, $l_{\mathrm{zt}}$ and $l_{\mathrm{zt}}^{\ast }$ reduce to $l_z$ and $l_z^{\ast }$ , respectively, and therefore allows for the evaluation of person fit with a wider range of IRT models. A real data application is presented to show the utility of the proposed statistics for a test with an underlying structure that consists of both the traditional unidimensional component and the Rasch testlet component.

PMID:39153026 | DOI:10.1007/s11336-024-09997-y