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Nevin Manimala Statistics

Evaluation of Large-Scale Proteomics for Prediction of Cardiovascular Events

JAMA. 2023 Aug 22;330(8):725-735. doi: 10.1001/jama.2023.13258.

ABSTRACT

IMPORTANCE: Whether protein risk scores derived from a single plasma sample could be useful for risk assessment for atherosclerotic cardiovascular disease (ASCVD), in conjunction with clinical risk factors and polygenic risk scores, is uncertain.

OBJECTIVE: To develop protein risk scores for ASCVD risk prediction and compare them to clinical risk factors and polygenic risk scores in primary and secondary event populations.

DESIGN, SETTING, AND PARTICIPANTS: The primary analysis was a retrospective study of primary events among 13 540 individuals in Iceland (aged 40-75 years) with proteomics data and no history of major ASCVD events at recruitment (study duration, August 23, 2000 until October 26, 2006; follow-up through 2018). We also analyzed a secondary event population from a randomized, double-blind lipid-lowering clinical trial (2013-2016), consisting of individuals with stable ASCVD receiving statin therapy and for whom proteomic data were available for 6791 individuals.

EXPOSURES: Protein risk scores (based on 4963 plasma protein levels and developed in a training set in the primary event population); polygenic risk scores for coronary artery disease and stroke; and clinical risk factors that included age, sex, statin use, hypertension treatment, type 2 diabetes, body mass index, and smoking status at the time of plasma sampling.

MAIN OUTCOMES AND MEASURES: Outcomes were composites of myocardial infarction, stroke, and coronary heart disease death or cardiovascular death. Performance was evaluated using Cox survival models and measures of discrimination and reclassification that accounted for the competing risk of non-ASCVD death.

RESULTS: In the primary event population test set (4018 individuals [59.0% women]; 465 events; median follow-up, 15.8 years), the protein risk score had a hazard ratio (HR) of 1.93 per SD (95% CI, 1.75 to 2.13). Addition of protein risk score and polygenic risk scores significantly increased the C index when added to a clinical risk factor model (C index change, 0.022 [95% CI, 0.007 to 0.038]). Addition of the protein risk score alone to a clinical risk factor model also led to a significantly increased C index (difference, 0.014 [95% CI, 0.002 to 0.028]). Among White individuals in the secondary event population (6307 participants; 432 events; median follow-up, 2.2 years), the protein risk score had an HR of 1.62 per SD (95% CI, 1.48 to 1.79) and significantly increased C index when added to a clinical risk factor model (C index change, 0.026 [95% CI, 0.011 to 0.042]). The protein risk score was significantly associated with major adverse cardiovascular events among individuals of African and Asian ancestries in the secondary event population.

CONCLUSIONS AND RELEVANCE: A protein risk score was significantly associated with ASCVD events in primary and secondary event populations. When added to clinical risk factors, the protein risk score and polygenic risk score both provided statistically significant but modest improvement in discrimination.

PMID:37606673 | DOI:10.1001/jama.2023.13258

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Nevin Manimala Statistics

Accelerated 3D MR neurography of the brachial plexus using deep learning-constrained compressed sensing

Eur Radiol. 2023 Aug 22. doi: 10.1007/s00330-023-09996-0. Online ahead of print.

ABSTRACT

OBJECTIVES: To explore the use of deep learning-constrained compressed sensing (DLCS) in improving image quality and acquisition time for 3D MRI of the brachial plexus.

METHODS: Fifty-four participants who underwent contrast-enhanced imaging and forty-one participants who underwent unenhanced imaging were included. Sensitivity encoding with an acceleration of 2 × 2 (SENSE4x), CS with an acceleration of 4 (CS4x), and DLCS with acceleration of 4 (DLCS4x) and 8 (DLCS8x) were used for MRI of the brachial plexus. Apparent signal-to-noise ratios (aSNRs), apparent contrast-to-noise ratios (aCNRs), and qualitative scores on a 4-point scale were evaluated and compared by ANOVA and the Friedman test. Interobserver agreement was evaluated by calculating the intraclass correlation coefficients.

RESULTS: DLCS4x achieved higher aSNR and aCNR than SENSE4x, CS4x, and DLCS8x (all p < 0.05). For the root segment of the brachial plexus, no statistically significant differences in the qualitative scores were found among the four sequences. For the trunk segment, DLCS4x had higher scores than SENSE4x (p = 0.04) in the contrast-enhanced group and had higher scores than SENSE4x and DLCS8x in the unenhanced group (all p < 0.05). For the divisions, cords, and branches, DLCS4x had higher scores than SENSE4x, CS4x, and DLCS8x (all p ≤ 0.01). No overt difference was found among SENSE4x, CS4x, and DLCS8x in any segment of the brachial plexus (all p > 0.05).

CONCLUSIONS: In three-dimensional MRI for the brachial plexus, DLCS4x can improve image quality compared with SENSE4x and CS4x, and DLCS8x can maintain the image quality compared to SENSE4x and CS4x.

CLINICAL RELEVANCE STATEMENT: Deep learning-constrained compressed sensing can improve the image quality or accelerate acquisition of 3D MRI of the brachial plexus, which should be benefit in evaluating the brachial plexus and its branches in clinical practice.

KEY POINTS: •Deep learning-constrained compressed sensing showed higher aSNR, aCNR, and qualitative scores for the brachial plexus than SENSE and CS at the same acceleration factor with similar scanning time. •Deep learning-constrained compressed sensing at acceleration factor of 8 had comparable aSNR, aCNR, and qualitative scores to SENSE4x and CS4x with approximately half the examination time. •Deep learning-constrained compressed sensing may be helpful in clinical practice for improving image quality and acquisition time in three-dimensional MRI of the brachial plexus.

PMID:37606664 | DOI:10.1007/s00330-023-09996-0

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Nevin Manimala Statistics

Efficacy and Safety of Anxiolytics in Mohs Micrographic Surgery: A Randomized, Double-Blinded, Placebo-Controlled Trial

Dermatol Surg. 2023 Aug 18. doi: 10.1097/DSS.0000000000003905. Online ahead of print.

ABSTRACT

BACKGROUND: Patient anxiety can complicate surgical outcomes by elevating blood pressure, increasing the need for postoperative pain management, and reducing overall patient satisfaction. Despite the use of anxiolytic medications in outpatient procedures, there is limited comparative evidence on the efficacy and safety of these agents in Mohs micrographic surgery.

OBJECTIVE: To compare the effectiveness and safety of different preprocedural anxiolytic agents in Mohs surgery on perioperative patient anxiety and patient satisfaction.

MATERIALS AND METHODS: A double-blinded, randomized, placebo-controlled trial was conducted of 6 different preprocedural anxiolytic agents (lorazepam, diazepam, alprazolam, gabapentin, pregabalin, and melatonin) in 350 patients undergoing Mohs surgery. Anxiety and vital signs were recorded.

RESULTS: Diazepam demonstrated a statistically significant, sustained reduction in anxiety levels compared with placebo (p = .03). Gabapentin significantly reduced early anxiety (p = .02). Alprazolam showed a trend to early anxiety reduction (p = .08). Lorazepam (p = .73), pregabalin (p = .53), and melatonin (p = .24) failed to reduce patient anxiety compared with placebo at any time point. No anxiolytic significantly impacted any patient vital sign or cognition.

CONCLUSION: Although short-acting benzodiazepines and gamma-aminobutyric acid medications may have transient anxiolytic effects, a single oral dose of 5 mg of diazepam can provide a sustained anxiolytic effect in Mohs surgery, with excellent patient safety.

PMID:37606659 | DOI:10.1097/DSS.0000000000003905

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Nevin Manimala Statistics

Cannabidiol as an Adjunct to Botulinum Toxin in Blepharospasm – A Randomized Pilot Study

Transl Vis Sci Technol. 2023 Aug 1;12(8):17. doi: 10.1167/tvst.12.8.17.

ABSTRACT

PURPOSE: The purpose of this study was to evaluate the safety and efficacy of low dose cannabidiol (CBD; Epidiolex) as adjunctive therapy for idiopathic adult-onset blepharospasm (BPS), as well as develop a novel objective assessment methodology to gauge response.

METHODS: Prospective, randomized, double-masked, placebo-controlled crossover design of 6 months duration of 12 patients with BPS undergoing routine maximal botulinum toxin (BTX) therapy and experiencing breakthrough symptoms. Participants received their standard BTX every 3 months and were randomized to group A = CBD daily in cycle 1, followed by placebo in cycle 2 or group B = placebo followed by CBD. Videos recorded at days 0, 45, and 90 of each cycle were analyzed to quantify eyelid kinematics. The Jankovic Rating Scale (JRS) was used to provide a clinical rating.

RESULTS: All 12 patients completed the study without adverse events. CBD decreased median eyelid closure amplitude by 19.1% (-1.66 mm, confidence interval [CI] = -3.19 to -0.14 mm, P = 0.03), decreased median eyelid closure duration by 15.8% (-18.35 ms, CI = -29.37 to -7.32 ms, P = 0.001), and increased the maximum eyelid closure velocity by 34.8% (-13.26 mm/ms, CI = -20.93 to -5.58 mm/ms, P = 0.001). The JRS showed a 0.5 reduction in severity and frequency, which was not statistically significant.

CONCLUSIONS: Low dose CBD was safely tolerated and improved several BPS kinematic parameters. The clinical scale suggested a direction of effect but may have been underpowered. Further studies are needed to better quantify the clinical relevance.

TRANSLATIONAL RELEVANCE: This work describes a novel assessment methodology and therapeutic approach to bBPS.

PMID:37606606 | DOI:10.1167/tvst.12.8.17

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Nevin Manimala Statistics

Why it is so challenging to perform economic evaluations of interventions in autism and what to do about it

Autism Res. 2023 Aug 22. doi: 10.1002/aur.3014. Online ahead of print.

ABSTRACT

Economic evaluation is used to determine the optimal provision of services and programs under budget constraints and to inform public and private payer funding decisions. To maximize value-for-money in the design and delivery of programs and services for persons with autism spectrum disorder (ASD), it’s essential to generate high-quality economic evidence to inform budget allocation. There is a paucity however, of economic evaluations of interventions for ASD. This is due in part to challenges in conducting economic evaluations in this population and the lack of guidance on suitable approaches. These challenges are related to the inherent heterogeneity of the autistic population; establishing short- and long-term effectiveness; measurement of costs and the availability of valid instruments for collecting economic data; the appropriateness of outcomes for use in economic evaluation; and achieving statistical power. This commentary addresses a lack of awareness and needed guidance on these issues by discussing the challenges and providing recommendations for how economic evaluations in ASD could be improved to generate high-quality evidence for program funding decision-making.

PMID:37606004 | DOI:10.1002/aur.3014

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Nevin Manimala Statistics

Predictive factors of surgery in metastatic colorectal cancer: a retrospective cohort study

Acta Chir Belg. 2023 Aug 22:1-8. doi: 10.1080/00015458.2023.2231211. Online ahead of print.

ABSTRACT

INTRODUCTION: Current management of metastatic colorectal cancer is based on neoadjuvant chemotherapy. Few studies have reported on surgery procedures in patients with metastatic colorectal cancer. The objective of this study was to describe our institutional experience with emergency surgery performed in patients with metastatic colorectal cancer during chemotherapy.

PATIENTS AND METHODS: This was a retrospective cohort study including adult patients of ≤80 years with a metastatic colorectal cancer between 2017 and 2020 and undergoing surgery during chemotherapy. Statistical analyses were based on Kaplan-Meier’s curve and Cox proportional hazard model. The surgery statistical risk during chemotherapy was studied through all tumor and patient’s characteristics. Multivariable logistic regression models were used to identify predictive factors of emergency surgery in these patients.

RESULTS: Seventy-two cases were identified and 60% patients undergone an emergency surgery. By Kaplan-Meier’s analyses, intestinal surgery was much more frequent and early in patients who have severe stenosis (either blocking or only permeable using a gastroscope) at the time of diagnosis. Patients with severe malignant stenosis presented a 6.28 time higher surgery risk (p < .0001). The median time between admission and surgery was 54 days in patients with severe stenosis who were operated.

CONCLUSION: The degree of colorectal tumor stenosis measured by endoscopy was a risk factor for emergency surgery in patients with metastatic colorectal cancer during neoadjuvant chemotherapy. In this group of patients presenting low survival outcomes, further studies are needed to define the place of preventive surgery, avoiding emergency surgery and morbidity in such fragile patients.

PMID:37605980 | DOI:10.1080/00015458.2023.2231211

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Nevin Manimala Statistics

Echocardiographic image collection and evaluation in infants with CHD: lessons learned from the imaging core lab for the Residual Lesion Score study

Cardiol Young. 2023 Aug 22:1-6. doi: 10.1017/S1047951123003037. Online ahead of print.

ABSTRACT

Many factors affect patient outcome after congenital heart surgery, including the complexity of the heart disease, pre-operative status, patient specific factors (prematurity, nutritional status and/or presence of comorbid conditions or genetic syndromes), and post-operative residual lesions. The Residual Lesion Score is a novel tool for assessing whether specific residual cardiac lesions after surgery have a measurable impact on outcome. The goal is to understand which residual lesions can be tolerated and which should be addressed prior to leaving the operating room. The Residual Lesion Score study is a large multicentre prospective study designed to evaluate the association of Residual Lesion Score to outcomes in infants undergoing surgery for CHD. This Pediatric Heart Network and National Heart, Lung, and Blood Institute-funded study prospectively enrolled 1,149 infants undergoing 5 different congenital cardiac surgical repairs at 17 surgical centres. Given the contribution of echocardiographic measurements in assigning the Residual Lesion Score, the Residual Lesion Score study made use of a centralised core lab in addition to site review of all data. The data collection plan was designed with the added goal of collecting image quality information in a way that would permit us to improve our understanding of the reproducibility, variability, and feasibility of the echocardiographic measurements being made. There were significant challenges along the way, including the coordination, de-identification, storage, and interpretation of very large quantities of imaging data. This necessitated the development of new infrastructure and technology, as well as use of novel statistical methods. The study was successfully completed, but the size and complexity of the population being studied and the data being extracted required more technologic and human resources than expected which impacted the length and cost of conducting the study. This paper outlines the process of designing and executing this complex protocol, some of the barriers to implementation and lessons to be considered in the design of future studies.

PMID:37605979 | DOI:10.1017/S1047951123003037

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Nevin Manimala Statistics

Effectiveness of mobile health interventions for pregnant women with gestational diabetes mellitus: a systematic review and meta-analysis

J Obstet Gynaecol. 2023 Dec;43(2):2245906. doi: 10.1080/01443615.2023.2245906.

ABSTRACT

Gestational diabetes mellitus is a growing global health problem. Inadequate management during pregnancy can lead to maternal and foetal complications. Currently, mobile health (mHealth) delivers healthcare services, playing an increasingly important role in the management of blood glucose in GDM. This study aimed to systematically evaluate the effectiveness of mHealth intervention in pregnant women with GDM. Based on randomised controlled trials of mHealth application in GDM patients searched from the database, literature screening, data extraction, and quality evaluation were conducted independently by two researchers. Statistical analysis was performed using Review Manager 5.4 software. The review included 27 studies with a total of 3483 patients. The results showed a significant improvement in glycemic control. In addition, mHealth interventions could reduce the occurrence of adverse pregnancy outcomes and improve self-management ability. In a subgroup analysis, recording of delivery mode and WeChat combined phone call indicated significant differences with mHealth interventions. It was suggested that mHealth interventions imposed a positive effect on glycemic control and reduction of adverse pregnancy outcomes in GDM patients. Our results demonstrated that the application of mHealth interventions can act as an effective and feasible approach to self-management to promote the self-management level and awareness of GDM patients.

PMID:37605977 | DOI:10.1080/01443615.2023.2245906

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Health-Care and Supportive Services in General Population Disaster Shelters

Disaster Med Public Health Prep. 2023 Aug 22;17:e457. doi: 10.1017/dmp.2023.114.

ABSTRACT

OBJECTIVES: The Communication (C), Maintaining Health (M), Independence (I), Services, Support and Self-Determination (S), and Transportation (T) is a framework (C-MIST) for identifying functional needs in an emergency response. A C-MIST documentation tool provides shelter staff with a list of potential client needs and actions to address them. This retrospective review describes the needs and actions indicated on completed C-MIST documentation tools (ie, records) within domestic general population shelters following Hurricane Florence in 2018.

METHODS: A convenience sample of 1209 records completed by shelter disaster health services personnel was provided by the American Red Cross. The records correspond to client stays in 19 shelters between September and October 2018. Data abstracted from hardcopy forms were de-identified and recorded in a database. Summary statistics were computed.

RESULTS: High incidence needs included medical supplies for everyday care (including medications) not related to mobility (15.4%), medically or culturally needed diets (12.2%), durable medical equipment (9.7%), mental health care (8.8%), and transportation (8.4%). High incidence actions included replacement medication (9.3%), refer to Disaster Mental Health Services (6.4%), provide assistive mobility equipment (5.1%), provide diabetes management supplies (5.0%), provide alternative food and beverages (4.1%), and provide transportation (3.9%).

CONCLUSIONS: The process for identifying health and functional support needs in shelters should be standardized through the use of the C-MIST framework.

PMID:37605973 | DOI:10.1017/dmp.2023.114

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Nevin Manimala Statistics

Some differences between social work, spiritual care, and psychology: Content variance in end-of-life conversations

Palliat Support Care. 2023 Aug 22:1-8. doi: 10.1017/S1478951523000652. Online ahead of print.

ABSTRACT

OBJECTIVES: Within the multidisciplinary team, there can sometimes be lack of clarity as to the specific different contributions of each of the psycho-social-spiritual professionals: social workers, psychologist, and spiritual caregivers. This study examined the content of their end-of-life conversations with patients.

METHODS: A total of 180 patients with terminal cancer received standard multidisciplinary care, including conversations with a social worker, psychologist, and spiritual caregiver. After each patient’s death, these professionals reported using a structured tool which content areas had arisen in their conversations with that patient.

RESULTS: Across all content areas, there were significant differences between social work and spiritual care. The difference between social work and psychology was slightly smaller but still quite large. Psychology and spiritual care were the most similar, though they still significantly differed in half the content areas. The differences persisted even among patients who spoke with more than 1 kind of professional. The 6 content areas examined proved to subdivide into 2 linked groups, where patients speaking about 1 were more likely to speak about the others. One group, “reflective” topics (inner and transpersonal resources, interpersonal relationships, one’s past, and end of life), included all those topics which arose more often with spiritual caregivers or psychologists. The second group, “decision-making” topics (medical coping and life changes), was comprised of those topics which arose most commonly with social workers, bridging between the medical and personal aspects of care and helping patients navigate their new physical, psychological, and social worlds.

SIGNIFICANCE OF RESULTS: These findings help shed light on the differences, in practice, between patients’ conversations with social workers, psychologists, and spiritual caregivers and the roles these professionals are playing; can aid in formulating individualized care plans; and strengthen the working assumption that all 3 professions contribute in unique, complementary ways to improving patients’ and families’ well-being.

PMID:37605972 | DOI:10.1017/S1478951523000652