Tag: statistics

Clin Transl Sci. 2025 Feb;18(2):e70117. doi: 10.1111/cts.70117.

ABSTRACT

There has been a growing trend that activities relating to clinical trials take place at locations other than traditional trial sites (hence decentralized clinical trials or DCTs), some of which are at settings of real-world clinical practice. Although there are numerous benefits of DCTs, this also brings some implications on a number of issues relating to the design, conduct, and analysis of DCTs. The Real-World Evidence Scientific Working Group of the American Statistical Association Biopharmaceutical Section has been reviewing the field of DCTs and provides in this paper considerations for decentralized trials from a statistical perspective. This paper first discusses selected critical decentralized elements that may have statistical implications on the trial and then summarizes regulatory guidance, framework, and initiatives on DCTs. More discussions are presented by focusing on the design (including construction of estimand), implementation, statistical analysis plan (including missing data handling), and reporting of safety events. Some additional considerations (e.g., ethical considerations, technology infrastructure, study oversight, data security and privacy, and regulatory compliance) are also briefly discussed. This paper is intended to provide statistical considerations for decentralized trials of medical products to support regulatory decision-making.

PMID:39972404 | DOI:10.1111/cts.70117

Diabetes Obes Metab. 2025 Feb 19. doi: 10.1111/dom.16272. Online ahead of print.

ABSTRACT

AIMS: To determine the impact of body roundness index (BRI) on the 10-year risk of cardiovascular disease (CVD) and all-cause mortality in Chinese adults.

MATERIALS AND METHODS: We utilized data from a nationwide prospective cohort of 165 785 Chinese adults (aged ≥40 years, free of CVD and cancer at baseline), which was extracted from the China Cardiometabolic Disease and Cancer Cohort (4C). Cox regression and restricted cubic splines quantified BRI-outcome associations and identified optimal BRI cutoffs.

RESULTS: During a median follow-up of 10.1 years, we documented 10 538 CVD events and 8679 deaths. A monotonic increasing dose-response association was observed between BRI and CVD risk, while BRI and all-cause mortality exhibited a U-shaped relationship (p for non-linear <0.001), with the nadir at a BRI of 3.9. Gender differences indicated that increased CVD risk was associated with higher BRI in men. As age increases, the excess risk of CVD and mortality associated with BRI diminishes, with a significant harmful effect of low BRI observed on mortality in individuals aged ≥75 years. Moreover, the inclusion of BRI in the model significantly enhanced the C-statistic for CVD prediction by 0.00412 (p <0.001) and for mortality prediction by 0.00045 (p = 0.0097).

CONCLUSIONS: Our findings indicate a positive association between BRI and CVD and a U-shaped association between BRI and mortality. These findings underscore the importance of the BRI in assessing the risk of CVD and all-cause mortality, particularly when considering different genders and age groups.

PMID:39972403 | DOI:10.1111/dom.16272

Acta Neuropathol Commun. 2025 Feb 19;13(1):34. doi: 10.1186/s40478-025-01939-8.

ABSTRACT

BACKGROUND: Tumor-infiltrating lymphocytes (TILs) and tumor-associated macrophages (TAMs) have increasingly been reported to impact the brain metastatic process of solid tumors. However, data on intra-individual differences between primary tumor and brain metastasis (BM), as well as their correlation with clinical outcome parameters, is scarce.

METHODS: We retrospectively identified patients who received resection of the primary tumor and BM between 01/1990 and 10/2022. Density quantification of TAMs (CD68⁺, CD163⁺) and TILs (CD3⁺, CD8⁺, CD45RO⁺, FOXP3⁺) was performed by immunohistochemical staining of matched tumor tissue samples. Images were processed with QuPath software and heterogeneity of generated heatmaps was measured by Shannon Entropy. Time-to-BM (TTBM) was defined as the time from diagnosis of the primary tumor until the first diagnosis of BM.

RESULTS: In total, 104 patients (46.2% female; median age 57.3 years at BM diagnosis) were included: 78/104 (75%) non-small cell lung cancer, 18/104 (17%) breast cancer, 8/104 (8%) renal cell carcinomas. Densities of CD3⁺ (p < 0.001) and CD8⁺-TILs (p < 0.001) were higher in primary tumor samples, while CD68⁺ (p = 0.035) and CD163⁺-TAM densities (p < 0.001) were higher in the matched BM. Higher CD3⁺, CD8⁺-TILs and CD163⁺-TAMs densities in primary tumors were associated with shorter TTBM (p = 0.005, p = 0.015 and p = 0.006, respectively). Higher entropies of CD3⁺ (p < 0.001) and FOXP3⁺ (p = 0.011) TILs were observed in primary tumors compared to BM. Longer TTBM was associated with higher entropy of FOXP3⁺ TILs (p = 0.024) and lower entropy in CD163⁺ TAMs (p = 0.039). No significant associations of immune cell densities or entropies with OS after BM diagnosis were found.

DISCUSSION: By utilizing a unique cohort of matched primary tumor and BM tissue samples, we could demonstrate higher TIL densities in primary tumors and higher TAM densities in BM, respectively. Higher cell densities of CD3⁺, CD8⁺-TILs and CD163⁺-TAMs in primary tumors were associated with shorter TTBM, while a larger difference between CD3⁺ and CD8⁺ densities between primary tumor and BM was associated with longer TTBM. These findings highlight the potential of targeting TAMs as a therapeutic strategy to mitigate the development of brain metastases.

PMID:39972401 | DOI:10.1186/s40478-025-01939-8

BMC Psychol. 2025 Feb 19;13(1):140. doi: 10.1186/s40359-025-02423-7.

ABSTRACT

BACKGROUND: Facial attractiveness, influenced by dental esthetics, impacts self-perception and social interactions. The rise of social media has further fueled the demand for cosmetic dental treatments. This study aimed to examine the influence of self-esteem on the inclination of dentists, dental students, and laypeople toward esthetic dental treatments.

METHODS: This cross-sectional study involved 410 participants, including dentists, dental students, and laypeople, selected through convenience sampling. Data were collected using a questionnaire that captured sociodemographic characteristics, oral hygiene status, previous experience with esthetic treatments, inclinations toward esthetic dental treatments, and self-esteem levels using the Rosenberg Self-Esteem Scale (RSES). The questionnaire, created on the Porsline© website, was distributed via social media platforms. Data were analyzed using SPSS version 21, employing the Chi-square test and logistic regression, with a p-value of less than 0.05 considered statistically significant.

RESULTS: The RSES showed that 16.3% of participants had low self-esteem, while 83.7% had high self-esteem. Among participants with previous cosmetic procedures, 84.3% expressed interest in pursuing additional treatments compared to 47.7% of those without such experience (P < 0.01). Among dental professionals, 38.3% of high and 37.5% of low self-esteem individuals expressed interest in cosmetic treatments (P = 0.42), while among laypeople, 66.6% of high and 82.2% of low self-esteem individuals indicated interest (P = 0.04). Regardless of profession, 66.7% of individuals with low self-esteem and 55.4% of those with high self-esteem expressed interest in esthetic treatments (P = 0.08). RSES scores varied significantly across age groups, with participants aged > 45 years showing the highest scores (P = 0.03). Regression analysis indicated that laypeople were more likely to seek esthetic dental treatments than dentists or dental students (OR = 3.02, 95% CI = 1.88-4.83). Participants with a history of esthetic dental treatments were also more likely to pursue further procedures (OR = 5.39, 95% CI = 2.97-9.77).

CONCLUSIONS: Self-esteem significantly correlates with laypeople’s inclination toward cosmetic dental treatments. Dentists and dental students tend to express less interest in such procedures, likely due to their understanding of potential long-term complications. Additionally, individuals with prior experience in esthetic dental treatments showed greater interest in pursuing further treatments.

PMID:39972400 | DOI:10.1186/s40359-025-02423-7

Health Res Policy Syst. 2025 Feb 19;23(1):20. doi: 10.1186/s12961-025-01289-w.

ABSTRACT

BACKGROUND: Learning healthcare communities (LHCCs) have been proposed as a next-generation evolution of learning health systems that can advance health equity; however, a practical mechanism for enabling the active and continuous community engagement required for one has not yet been described in the literature. Recognizing that community-based participatory research (CBPR) could potentially meet this need, a team at the Montefiore Medical Center/Albert Einstein College of Medicine designed a novel evidence-based CBPR model – Bridging Research, Accurate Information and Dialogue (BRAID) – that initiates meaningful, longitudinal dialogues to foster bidirectional trust between researchers, clinicians, scientists and communities.

METHODS: A mixed-methods cohort study of two BRAID cohorts was conducted between 2022 and 2023. Eligible participants were recruited from the Bronx, New York, United States and convened in a series of conversation circles. Multimodal data was collected from all participants, including quantitative pre- and post-series surveys and same-day conversation circle feedback forms. Surveys were administered using SurveyMonkey and descriptive statistics were completed in Excel and SPSS.

RESULTS: A total of 42 participants were enrolled, most of whom were people of colour who had not participated in research before. Among them, 40 participants provided at least one response to a same-day conversation circle feedback form, which reflected consistently positive experiences with BRAID. This was consistent with evidence from the post-series survey, in which every one of the 36 respondents stated that they would either definitely (83.3%, N = 30/36) or probably (16.7%, N = 6/36) recommend participation in BRAID to someone like them. Of note, 91.7% (N = 33/36) had already disseminated health information learned through BRAID downstream and 84.4% (N = 27/32) indicated that BRAID strengthened their trust in science and research, highlighting unique and distinguishing features of the model.

CONCLUSIONS: Our quantitative evidence suggests that BRAID is effective, efficient and scalable, with experiential evidence supporting that it is reproducible. These factors suggest that BRAID implementation can facilitate rapid, bidirectional information sharing that builds trust between healthcare organizations and communities. This has laid the groundwork for an LHCC in the Bronx, with the potential to be adopted by healthcare organizations elsewhere.

PMID:39972399 | DOI:10.1186/s12961-025-01289-w

Neurol Res Pract. 2025 Feb 20;7(1):8. doi: 10.1186/s42466-025-00365-4.

ABSTRACT

BACKGROUND: Several publications have raised concerns that female stroke patients may be at a disadvantage when accessing stroke treatment services. These publications have found significant regional differences in the provision of stroke treatment to male and female patients. In this study, we provide current nationwide data on stroke management differences between men and women in Germany.

METHODS: This large retrospective cohort study used national datasets from the German Federal Statistical Office for 2017-2022. We examined differences between female and male stroke patients in terms of case volume, intravenous thrombolysis (IVT), mechanical thrombectomy (MTE), stroke unit (SU) treatment, intrahospital mortality, and prevalence of atrial fibrillation (AF).

RESULTS: Data from more than 1.3 million hospitalised stroke patients were included. Forty-seven percent of the patients were female. Female patients were older and more frequently ≥ 80 years old (50.3% versus 29.4%). Rates of IVT (16.3% versus 16.3%) were similar for both sexes but higher in females when adjusted for age. MTE rates (8.2% versus 6.3%) were consistently higher in female patients across all age groups. Female patients had higher rates of intrahospital mortality (9.1% versus 6.2%), and admission to SUs (73.6% versus 76.0%) was less common. Treatment rates in intensive care units were similar (10.6% versus 10.5%). AF, a surrogate for embolic (and more severe) strokes, was more prevalent in females (32.6% versus 25.4%).

CONCLUSIONS: We found no evidence that female stroke patients in Germany face any disadvantage in accessing stroke treatment services. Acute stroke treatment rates were generally similar or higher when compared to males. Higher intrahospital mortality and lower SU rates were attributed to greater age, comorbidities, and stroke severity. However, the differences were not fully explained when adjusting for AF and age. Further research is needed on sex differences in stroke mechanisms and outcomes.

PMID:39972395 | DOI:10.1186/s42466-025-00365-4

Glob Health Res Policy. 2025 Feb 19;10(1):5. doi: 10.1186/s41256-024-00398-3.

ABSTRACT

BACKGROUND: Although the implementation of a hierarchical medical system (HMS) has been shown to improve the allocation of medical resources and patient health-seeking behaviour, its role in patient’s perceived quality of primary care remains unexplored. This study aimed to assess the impact of HMS implementation on rural and urban residents’ perceived quality of primary care.

METHODS: Data were obtained from the China Family Panel Study for 2012, 2014, 2016, and 2018. A total of 40,011 rural and 22,482 urban residents were included in the research participants for analysis. This study adopted a quasi-natural experimental design, and the multiple-period difference-in-differences method was used to capture changes in patient’s perceived quality of primary care before and after the introduction of HMS.

RESULTS: We found that HMS implementation declined the perceived quality of primary care by an average of 18% among rural residents (OR: 0.82, 95% CI 0.68-0.99), while there was no significant change among urban residents (OR: 1.13, 95% CI 0.87-1.46). There was a 24% reduction in the perceived quality of primary care (OR: 0.76, 95% CI 0.61-0.96) one year after HMS among rural residents, and there was no statistically significant difference two years after HMS. After HMS implementation, the level of perceived quality of primary care by rural patients with chronic diseases decreased by 72% (OR: 0.28, 95% CI 0.11-0.78).

CONCLUSIONS: HMS has a limited effect on improving residents’ perceived quality of primary care, especially for those living in rural areas. Policymakers are suggested to establish a quality monitoring system that incorporates patient experience as an essential standard to systematically evaluate the impacts of the HMS, with more efforts being put into helping vulnerable groups such as residents under 60 years old and patients with chronic diseases.

PMID:39972386 | DOI:10.1186/s41256-024-00398-3

J Intellect Disabil Res. 2025 Feb 19. doi: 10.1111/jir.13223. Online ahead of print.

ABSTRACT

BACKGROUND: Individuals with PWS need constant support and/or supervision, which creates a high caregiver burden on their parents and siblings. Previous research has identified adverse stress outcomes in relatively small and country-specific samples. This study’s aims were to examine stress outcomes in a large multi-country sample of parents and siblings and to expand upon previous research by incorporating data on psychosocial factors that may mitigate stress outcomes.

METHODS: The sample comprised 135 parents of a child with PWS, with additional data for 45 siblings as reported by parents. Participants were recruited from 31 countries, spanning Europe, North and South America, Africa, Asia and Australasia, who participated by completing an online questionnaire that included standardised psychometric measures of depression and anxiety (HADS), life stress (PSS), PTSD symptoms (CATS-C) and family cohesion (FACES II). Outcomes were compared to published population norms, and multiple regression was used to investigate the role of potential exacerbating and mitigating factors.

RESULTS: Findings revealed high rates of mental pathologies in both parents and siblings. Parents’ scores for depression and anxiety indicated high rates of caseness: 67.4% of parents exhibited ‘abnormal’ levels of anxiety, while 15.6% exhibited ‘borderline abnormal’ levels; 34.8% exhibited ‘abnormal’ levels of depression, with 22.2% exhibiting ‘borderline abnormal’ levels. Younger parents exhibited higher anxiety than older parents (p = 0.007); younger male parents reported higher depression than older male parents (p = 0.029). Parents whose child with PWS lived in the family home exhibited higher depression scores than parents whose child with PWS lived away from home (p = 0.035). Family cohesion was inversely associated with parental depression (p < 0.001) and parental anxiety (p = 0.012), even when statistically controlling for age of parent, age of child with PWS and parental education level. Scores for life stress were markedly higher than population norms, with 88.7% of parents exhibiting ‘high’ or ‘moderate’ life stress. Parental life stress was significantly correlated with temper outburst severity in their child with PWS (p < 0.001) and with food problem severity (p < 0.001). All siblings exhibited at least one symptom of PTSD, with 28.9% of siblings exhibiting ‘clinically relevant’ levels of PTSD symptoms. Sibling PTSD symptom levels were significantly associated with temper outburst severity in the child with PWS (p = 0.025) but not with ratings of food problem severity (p = 0.114). Family cohesion was inversely associated with PTSD symptoms in siblings (p = 0.022).

CONCLUSIONS: PWS impacts families negatively, and relatives suffer as a result. The findings of this study confirm that parents and siblings of persons with PWS exhibit clinically notable levels of mental pathology. Strategies to enhance family cohesion should be employed to help diminish adverse outcomes among PWS families.

PMID:39970479 | DOI:10.1111/jir.13223

J Back Musculoskelet Rehabil. 2025 Jan;38(1):71-82. doi: 10.1177/10538127241296340. Epub 2025 Jan 9.

ABSTRACT

BACKGROUND: In recent years, the femoral neck system (FNS) has become increasingly widely used for Pauwels type III femoral neck fractures.

OBJECTIVE: We performed a meta-analysis to investigate the safety and efficacy of FNS and the mainstream internal fixation method, cannulated screw (CS), in the treatment of Pauwels type III femoral neck fractures.

METHODS: We systematically searched all research studies from PubMed, Embase, Web of Science, Cochrane, WANFANG, and the Chinese Academic Journal Full-Text Database (CNKI) from 2018 to July 2024 and conducted data extraction. The study was conducted on July 26, 2024. We used the PRISMA guidelines to assess the quality of the results of all included studies. In addition, we searched for references to relevant articles. After completing the literature search, we performed a manual literature screening to obtain studies that met the inclusion criteria. The outcomes were Harris Hip Score, operation duration, intraoperative blood loss, hospital stay, healing time, and postoperative complications.

RESULTS: This meta-analysis ultimately included nine studies (n = 524) and conducted quality evaluations. All nine articles were retrospective cohort studies. The study exhibited statistically significant variations between the FNS and CS groups in relation to total complications (95% confidence interval (CI)= [0.11, 0.40], odds ratio (OR) = 0.21, P < 0.001), Harris Hip Score (95%CI = [2.14, 3.65], mean difference (MD) = 2.89, P < 0.001), Femoral head necrosis (95%CI = [0.17, 0.86], OR = 0.38, P = 0.02), Internal fixation failure (95%CI = [0.06, 0.50], OR = 0.17, P = 0.001), Medium femoral neck shortening (95%CI = [0.17, 0.53], OR = 0.30, P < 0.001), intraoperative blood loss (95%CI = [11.57, 26.80], MD = 19.19, P < 0.001), Operation duration (95%CI = [-15.20, -2.17], MD = -8.69, P = 0.009), and healing time (95%CI = [-1.30, -0.32], MD = -0.81, P = 0.001).

CONCLUSION: This meta-analysis showed that in Pauwels type III femoral neck fractures, the femoral neck system has a shorter operative time, a lower rate of postoperative complications, a shorter healing time, less shortening of the femoral neck, and a higher Harris Hip Score compared with cannulated screws, but increases intraoperative blood loss. However, more clinical studies are needed to confirm our conclusions due to our small sample size and lack of randomized controlled trials.

PMID:39970468 | DOI:10.1177/10538127241296340

J Back Musculoskelet Rehabil. 2025 Jan;38(1):83-92. doi: 10.1177/10538127241296342. Epub 2024 Dec 19.

ABSTRACT

BACKGROUND: Forward head posture (FHP) is a common malalignment affecting the cervicothoracic spine. This deviation is associated with neck disability and muscle imbalance.

OBJECTIVE: This study aimed to investigate the efficacy of FHP correction using regional versus comprehensive spinal programs on the craniovertebral (CV) angle, neck disability, and spinal muscle activity.

METHODS: Sixty participants with FHP were randomly assigned to receive either a cervicothoracic correction program (control group) or a cervicothoracic plus lumbopelvic program (experimental group). The CV angle, neck disability index (NDI), and normalized electromyography as a percentage of maximum voluntary isometric contraction (%MVIC) from spinal muscles were measured before and after the intervention.

RESULTS: Post-intervention, both groups showed significant improvement across time in CV angle and NDI (p < 0.001, p = 0.002). However, the between-group comparison was not statistically significant. The NDI showed significant improvement only in the experimental group (p = 0.005). The minimal clinical important difference (MCID) obtained was 6.44 for the NDI. A reduction in %MVIC over time was observed in both groups for cervical erector spinae (Right, p = 0.006, Left, p = 0.001). The between-group comparison of spinal muscle activation was not significantly different.

CONCLUSION: The study suggested that FHP management using a cervicothoracic or cervicothoracic plus lumbopelvic protocol could improve cervical posture and lower cervical muscle demand. Incorporating a lumbopelvic into the cervicothoracic protocol was more effective in reducing short-term neck pain and disability than a cervicothoracic protocol alone. A comprehensive spine program may be a clinically beneficial rehabilitation protocol for FHP to improve neck pain and disability.

PMID:39970467 | DOI:10.1177/10538127241296342