JAMA Netw Open. 2023 Oct 2;6(10):e2340307. doi: 10.1001/jamanetworkopen.2023.40307.
NO ABSTRACT
PMID:37902758 | DOI:10.1001/jamanetworkopen.2023.40307
Tag: pubmed
JAMA Netw Open. 2023 Oct 2;6(10):e2340256. doi: 10.1001/jamanetworkopen.2023.40256.
ABSTRACT
IMPORTANCE: Oncologic outcomes among patients with rectal cancer after developing local recurrence and/or distant metastases remain poorly studied.
OBJECTIVE: To analyze the trend of overall survival after treatment failure for patients with rectal cancer within three consecutive phase 2 or 3 trials of the German Rectal Cancer Study Group.
DESIGN, SETTING, AND PARTICIPANTS: This cohort study is a post hoc analysis of 3 randomized phase 2 or 3 trials (CAO/ARO/AIO-94, -04, and -12 trials, conducted in Germany) that included 1948 patients with locally advanced rectal adenocarcinoma. The CAO/ARO/AIO-94 trial recruited patients between February 1995 and September 2002, the CAO/ARO/AIO-04 trial recruited patients between July 2006 and February 2010, and the CAO/ARO/AIO-12 trial recruited patients between June 2015 and January 2018. Statistical analysis was conducted between September 2022 and March 2023.
EXPOSURES: A total of 119 of 391 patients in the CAO/ARO/AIO-94 trial group A, 295 of 1236 patients in the CAO/ARO/AIO-04 trial, and 69 of 306 in the CAO/ARO/AIO-12 trial experienced treatment failure (R2 resection or local recurrence or distant metastases) and were included in further analyses.
MAIN OUTCOMES AND MEASURES: Characteristics of treatment failure and overall survival were assessed in all 3 trial cohorts.
RESULTS: Of the 1948 patients treated in the 3 trials, 15 were excluded because of missing data. Of the remaining 1933 patients (median age, 62.5 years [range, 19-84 years]; 1363 men [71%] and 570 women [29%]) with locally advanced rectal adenocarcinoma (cT3 or 4 or cN+) treated within 3 consecutive clinical trials, 483 experienced treatment failure and were analyzed. After a median follow-up of 36 months (IQR, 24-51 months) for all patients, overall survival after treatment failure was significantly improved in the CAO/ARO/AIO-04 trial (at 3 years, 44% [IQR, 37%-51%]; hazard ratio [HR], 0.61 [95% CI, 0.47-0.79]) and further improved in the CAO/ARO/AIO-12 trial (at 3 years, 73% [IQR, 60%-87%]; HR, 0.32 [95% CI, 0.18-0.54]) compared with the CAO/ARO/AIO-94 trial (at 3 years, 30% [IQR, 22%-39%]) (both P < .001). Distant metastasis was the main reason for treatment failure throughout a 5-year follow-up (range, 67%-87%), and the relative risk for treatment failure was highest in the first 18 months in all 3 trials. ypTNM stage was significantly associated with the risk and time interval to treatment failure. Improvement in overall survival after treatment failure was independent of sex.
CONCLUSIONS AND RELEVANCE: This cohort study suggests that advancements in salvage strategies during the past decades have likely improved overall survival among patients with rectal cancer who experienced treatment failure.
PMID:37902752 | DOI:10.1001/jamanetworkopen.2023.40256
Invest Ophthalmol Vis Sci. 2023 Oct 3;64(13):46. doi: 10.1167/iovs.64.13.46.
ABSTRACT
PURPOSE: The purpose of this study was to explore the cortical deficits of patients with acquired concomitant esotropia (AACE) using the resting-state functional magnetic resonance imaging (rs-fMRI) technique.
METHODS: Rs-fMRI signals from 25 patients with AACE and 25 matched controls were collected. The repeated-measures analysis of variance (RM-ANOVA) test and two-sample t-test were used to investigate statistical differences of the amplitudes of low-frequency fluctuation (ALFF) signals and correlation analysis was performed to validate the relationship of signal change and clinical features.
RESULTS: The AACE group showed decreased ALFF in both hemispheres symmetrically (t = 0.38, P = 0.71), with peak t in both middle occipital gyrus. The ALFF signal from the upper left inferior frontal gyrus was negatively correlated with the age of onset (r = 0.62, P = 0.0008), and the ALFF signal from the right superior temporal gyrus was negatively correlated with the near work hours (r = 0.63, P = 0.0008). The ALFF signal in the left fusiform gyrus was positively correlated with both near (r = 0.48, P = 0.01) and far (r = 0.44, P = 0.03) deviation, whereas it was only positively correlated with far deviation (r = 0.44, P = 0.03) in the right. Besides, the age of onset and the near work hour were independent factors of signal changes.
CONCLUSIONS: Using the ALFF signal of rs-fMRI, we found functional deficits in the primary visual cortex and dorsal pathway in patients with AACE. There were functional changes in the fusiform gyrus, and the greater the deviation angle, the higher the changing level. These findings reveal the association of AACE and the visual center, giving us more clues about the treatment of AACE.
PMID:37902746 | DOI:10.1167/iovs.64.13.46
JAMA Intern Med. 2023 Oct 30. doi: 10.1001/jamainternmed.2023.5656. Online ahead of print.
ABSTRACT
IMPORTANCE: Despite guideline recommendations, clinicians do not systematically use prior screening or health history to guide colorectal cancer (CRC) screening decisions in older adults.
OBJECTIVE: To evaluate the effect of a personalized multilevel intervention on screening orders in older adults due for average-risk CRC screening.
DESIGN, SETTING, AND PARTICIPANTS: Interventional 2-group parallel unmasked cluster randomized clinical trial conducted from November 2015 to February 2019 at 2 US Department of Veterans Affairs (VA) facilities: 1 academic VA medical center and 1 of its connected outpatient clinics. Randomization at the primary care physician/clinician (PCP) level, stratified by study site and clinical full-time equivalency. Participants were 431 average-risk, screen-due US veterans aged 70 to 75 years attending a primary care visit. Data analysis was performed from August 2018 to August 2023.
INTERVENTION: The intervention group received a multilevel intervention including a decision-aid booklet with detailed information on screening benefits and harms, personalized for each participant based on age, sex, prior screening, and comorbidity. The control group received a multilevel intervention including a screening informational booklet. All participants received PCP education and system-level modifications to support personalized screening.
MAIN OUTCOMES AND MEASURES: The primary outcome was whether screening was ordered within 2 weeks of clinic visit. Secondary outcomes were concordance between screening orders and screening benefit and screening utilization within 6 months.
RESULTS: A total of 436 patients were consented, and 431 were analyzed across 67 PCPs. Patients had a mean (SD) age of 71.5 (1.7) years; 424 were male (98.4%); 374 were White (86.8%); 89 were college graduates (21.5%); and 351 (81.4%) had undergone prior screening. A total of 258 (59.9%) were randomized to intervention, and 173 (40.1%) to control. Screening orders were placed for 162 of 258 intervention patients (62.8%) vs 114 of 173 control patients (65.9%) (adjusted difference, -4.0 percentage points [pp]; 95% CI, -15.4 to 7.4 pp). In a prespecified interaction analysis, the proportion receiving orders was lower in the intervention group than in the control group for those in the lowest benefit quartile (59.4% vs 71.1%). In contrast, the proportion receiving orders was higher in the intervention group than in the control group for those in the highest benefit quartile (67.6% vs 52.2%) (interaction P = .049). Fewer intervention patients (106 of 256 [41.4%]) utilized screening overall at 6 months than controls (96 of 173 [55.9%]) (adjusted difference, -13.4 pp; 95% CI, -25.3 to -1.6 pp).
CONCLUSIONS AND RELEVANCE: In this cluster randomized clinical trial, patients who were presented with personalized information about screening benefits and harms in the context of a multilevel intervention were more likely to receive screening orders concordant with benefit and were less likely to utilize screening.
TRIAL REGISTRATION: ClinicalTrials.gov Identifier: NCT02027545.
PMID:37902744 | DOI:10.1001/jamainternmed.2023.5656
Diabetes Technol Ther. 2023 Oct 30. doi: 10.1089/dia.2023.0373. Online ahead of print.
ABSTRACT
INTRODUCTION: To evaluate time in tight range (TITR) 70-140 mg/dL (3.9- 7.8 mmol/L), its correlation with standard continuous glucose monitoring (CGM) metrics and the clinical variables that possibly have a substantial impact on its value, in a large cohort of pediatric subjects using different treatment strategies.
MATERIAL AND METHODS: A total of 854 children and adolescents with type 1 diabetes were consecutively recruited in this real-world, dual-center, cross-sectional study. Participants were categorized into four treatment groups (multiple daily injections + real-time CGM, multiple daily injections + intermittently scanned CGM, sensor augmented pump, and hybrid closed loop (HCL)). Demographical and clinical data, including CGM data, were collected and analyzed.
RESULTS: The overall study population exhibited an average TITR of 36.4±12.8%. HCL users showed higher TITR levels compared to the other treatment groups (p<0.001). A time in range (TIR) cut-off value of 71.9% identified subjects achieving a TITR≥50% (AUC 0.98; 95%CI 0.97-0.99, p<0.001), and a strong positive correlation between these two metrics was observed (r=0.95, p<0.001). An increase in TIR of 1% was associated with 1.84 (R2 Nagelkerke=0.35, p<0.001) increased likelihood of achieving TITR≥50%. Use of HCL systems (B=7.78; p<0.001), disease duration (B=-0.26, p=0.006), coefficient of variation (B=-0.30, p=0.004), and glycated haemoglobin (B=-8.82; p<0.001) emerged as significant predictors of TITR levels.
CONCLUSIONS: Our study highlights that most children and adolescents with type 1 diabetes present TITR levels below 50%, except those using HCL. Tailored interventions and strategies should be implemented to increase TITR.
PMID:37902743 | DOI:10.1089/dia.2023.0373
Haemophilia. 2023 Oct 30. doi: 10.1111/hae.14882. Online ahead of print.
ABSTRACT
INTRODUCTION: Etranacogene dezaparvovec gene therapy for haemophilia B demonstrated superior efficacy at 24 months in reducing bleeds versus a ≥6-month lead-in period of prophylaxis with FIX products in the phase 3 trial, HOPE-B. In the absence of head-to-head comparisons of etranacogene dezaparvovec versus FIX products, indirect treatment comparisons (ITC) can be used.
AIM: To compare the efficacy of etranacogene dezaparvovec versus rIX-FP, rFIXFc and N9-GP using ITC, and support HOPE-B results.
METHODS: Data were leveraged from Phase 3 pivotal trials: HOPE-B, PROLONG-9FP, B-LONG and Paradigm 2. Annualised bleeding rates (ABR), spontaneous (AsBR) and joint (AjBR) bleeding rates, percentage of patients with no bleeds, and FIX consumption were assessed using inverse probability of treatment weighting and matching adjusted indirect comparisons.
RESULTS: Etranacogene dezaparvovec demonstrated statistically significantly lower bleeding rates versus all comparators. Rate ratios for ABR, AsBR and AjBR versus rIX-FP were 0.19 (p < .0001), 0.08 (p < .0001) and 0.09 (p < .0001), respectively. Rate ratios for ABR, AsBR and AjBR versus rFIXFc were 0.14 (p < .0001), 0.13 (p = .0083) and 0.15 (p = .0111), respectively. Rate ratios for ABR and AsBR, versus N9-GP were 0.24 (p = .0231) and 0.13 (p = .0071), respectively. Etranacogene dezaparvovec demonstrated significantly higher percentage of patients with no bleeds versus rIX-FP and rFIXFc; odds ratios: 17.60 (p < .0001) and 5.65 (p = .0037), respectively. Etranacogene dezaparvovec resulted in significantly lower FIX consumption than all comparators.
CONCLUSIONS: ITC suggests that etranacogene dezaparvovec offers patients with haemophilia B (≤2% of normal FIX expression) a single dose treatment that can significantly reduce bleeding rates and eliminate routine infusions associated with FIX therapies.
PMID:37902714 | DOI:10.1111/hae.14882
Urol Pract. 2023 Oct 30:101097UPJ0000000000000468. doi: 10.1097/UPJ.0000000000000468. Online ahead of print.
ABSTRACT
PURPOSE: This study aimed to investigate the association between social vulnerability, as measured by the Centers for Disease Control and Prevention’s Social Vulnerability Index (SVI), and the quality of life (QoL) of kidney stone patients using the validated Wisconsin Stone Quality of Life Questionnaire (WISQOL).
MATERIALS AND METHODS: A retrospective analysis was conducted on medical records of new urolithiasis patients who completed the WISQOL at the University of Rochester Medical Center kidney stone clinic. The primary outcome was WISQOL score, which was measured across multiple domains. SVI was used to assess social vulnerability. Neighborhoods with high SVI were defined by a threshold greater than or equal to the 75th percentile nationally. Demographic and clinical data were collected. Statistical analyses, including univariate tests and multivariate linear regression, were performed to evaluate the relationships between social vulnerability and disease specific QoL.
RESULTS: A total of 1718 patients were included in the study. 105 subjects (6.1%) were from neighborhoods of high social vulnerability. Patients residing in neighborhoods with high social vulnerability (SVI quartile) reported significantly lower QoL scores (69.1 vs 77.2; P = .001) and this persisted across all domains, including social impact (32.6 vs 35.1; P = .002), emotional impact (25.2 vs 27.5; P = .006), disease impact (28.5 vs 31.4; P = .001), and vitality (10.3 vs 11.2; P = .015). Younger age, female sex, and higher number of comorbidities were identified as independent predictors of lower QoL scores. However, non-white race and Latinx ethnicity did not exhibit a significant association with QoL scores.
CONCLUSIONS: These findings highlight the negative impact of high social vulnerability on QoL, emphasizing the importance of considering socioeconomic factors in patient care. These results emphasize the need for targeted interventions to support vulnerable populations. While this study offers initial insights, further research is essential to corroborate these outcomes across larger and more diverse populations.
PMID:37902693 | DOI:10.1097/UPJ.0000000000000468
Psychol Methods. 2023 Oct 30. doi: 10.1037/met0000600. Online ahead of print.
ABSTRACT
How to model cross-lagged relations in panel data continues to be a source of disagreement in psychological research. While the cross-lagged panel model (CLPM) was the modeling approach of choice for many years, it has also been criticized repeatedly for its inability to separate within-person dynamics from stable between-person differences. Hence, various alternative models that disentangle these forms of variability have been proposed, and these are now rapidly gaining popularity. But not everyone agrees this is the right way forward. CLPM advocates point out that many psychological theories are concerned with longer-lasting differences between individuals, while these differences are not allowed to contribute to the estimation of cross-lagged effects in the novel within-between approaches. Reasoning this way, it is argued that the CLPM is superior when studying such processes, precisely because it includes the chronic between-person differences when estimating prospective effects. The goal of the current paper is to consider this within-between dispute in its broader context and to examine various directions in which this discussion needs expansion. To this end, three different perspectives are adopted: that of the study design, patterns in empirical data, and the nature of our research questions. It will be argued that to move forward, we need to look beyond the narrow focus on how to model our correlational panel data. Progress will involve theorizing more deliberately about the timescale that a process operates on, being more explicit about our research questions, considering alternative designs and models, and familiarizing ourselves with relevant discussions in other disciplines. (PsycInfo Database Record (c) 2023 APA, all rights reserved).
PMID:37902677 | DOI:10.1037/met0000600
Psychol Assess. 2023 Nov;35(11):1054-1067. doi: 10.1037/pas0001211.
ABSTRACT
To assess the public health impact of the COVID-19 pandemic on mental health, investigators from the National Institutes of Health Environmental influences on Child Health Outcomes (ECHO) research program developed the Pandemic-Related Traumatic Stress Scale (PTSS). Based on the Diagnostic and Statistical Manual of Mental Disorders, 5th Edition (DSM-5) acute stress disorder symptom criteria, the PTSS is designed for adolescent (13-21 years) and adult self-report and caregiver-report on 3-12-year-olds. To evaluate psychometric properties, we used PTSS data collected between April 2020 and August 2021 from non-pregnant adult caregivers (n = 11,483), pregnant/postpartum individuals (n = 1,656), adolescents (n = 1,795), and caregivers reporting on 3-12-year-olds (n = 2,896). We used Mokken scale analysis to examine unidimensionality and reliability, Pearson correlations to evaluate relationships with other relevant variables, and analyses of variance to identify regional, age, and sex differences. Mokken analysis resulted in a moderately strong, unidimensional scale that retained nine of the original 10 items. We detected small to moderate positive associations with depression, anxiety, and general stress, and negative associations with life satisfaction. Adult caregivers had the highest PTSS scores, followed by adolescents, pregnant/postpartum individuals, and children. Caregivers of younger children, females, and older youth had higher PTSS scores compared to caregivers of older children, males, and younger youth, respectively. (PsycInfo Database Record (c) 2023 APA, all rights reserved).
PMID:37902671 | DOI:10.1037/pas0001211
Psychol Assess. 2023 Nov;35(11):925-937. doi: 10.1037/pas0001258.
ABSTRACT
Psychological assessment underwent substantive challenges and changes when the COVID-19 pandemic began, and these changes are likely to endure given the rapid growth of telehealth clinical practice and assessment research using virtual procedures. COVID-19-related changes to assessment practices have impacted accordingly how we study overreporting scale functioning, including the modality through which we administer measures. No available research provides direct comparisons of overreporting scale effectiveness within simulation research across in-person and telehealth modalities, despite early support for novel instruments relying on remote procedures within the historic context of the pandemic. We used simulated feigning conditions collected using best telehealth practices to examine if, and how, overreporting scales differed in effectiveness by evaluating mean scores, elevation rates, and classification accuracy statistics, relative to parallel in-person conditions. Results indicate no meaningful differences in scale effectiveness, particularly when exclusion procedures included a posttest questionnaire. Our findings support telehealth assessment practice and the integration of research collected virtually into the traditional, in-person feigning literature. Limitations and future directions are discussed. (PsycInfo Database Record (c) 2023 APA, all rights reserved).
PMID:37902662 | DOI:10.1037/pas0001258