clinical research

临床研究
  • 文章类型: Journal Article
    生活质量(QOL)受损在炎症性肠病(IBD)患者中很常见。更快速地识别生活质量受损高风险的IBD患者的工具改善了早期干预的机会并改善了长期预后。这项研究的目的是使用机器学习(ML)方法来开发风险分层模型,以评估IBD相关的QOL损害。
    使用在线问卷收集了2021年9月至2022年5月分布在中国22个省的42家医院的2478例IBD患者的临床数据。开发并验证了八个用于预测IBD相关QOL损害风险的ML模型。使用一组索引评估模型性能,并使用本地可解释模型-不可知模型解释(LIME)算法解释最佳ML模型。
    基于支持向量机(SVM)分类器算法的模型优于其他ML模型,其接收器工作特性曲线(AUC)下的面积和精度分别为0.80和0.71。SVM分类器算法计算的特征重要性揭示了糖皮质激素的使用,焦虑,腹痛,睡眠障碍,更严重的疾病导致生活质量受损的风险更高,而病程较长以及使用生物制剂和免疫抑制剂与较低的风险相关。
    用于评估IBD相关QOL损害的ML方法是可行且有效的。这种机制是胃肠病学家鉴定生活质量受损高风险IBD患者的有希望的工具。
    UNASSIGNED: Impaired quality of life (QOL) is common in patients with inflammatory bowel disease (IBD). A tool to more quickly identify IBD patients at high risk of impaired QOL improves opportunities for earlier intervention and improves long-term prognosis. The purpose of this study was to use a machine learning (ML) approach to develop risk stratification models for evaluating IBD-related QOL impairments.
    UNASSIGNED: An online questionnaire was used to collect clinical data on 2478 IBD patients from 42 hospitals distributed across 22 provinces in China from September 2021 to May 2022. Eight ML models used to predict the risk of IBD-related QOL impairments were developed and validated. Model performance was evaluated using a set of indexes and the best ML model was explained using a Local Interpretable Model-Agnostic Explanations (LIME) algorithm.
    UNASSIGNED: The support vector machine (SVM) classifier algorithm-based model outperformed other ML models with an area under the receiver operating characteristic curve (AUC) and an accuracy of 0.80 and 0.71, respectively. The feature importance calculated by the SVM classifier algorithm revealed that glucocorticoid use, anxiety, abdominal pain, sleep disorders, and more severe disease contributed to a higher risk of impaired QOL, while longer disease course and the use of biological agents and immunosuppressants were associated with a lower risk.
    UNASSIGNED: An ML approach for assessing IBD-related QOL impairments is feasible and effective. This mechanism is a promising tool for gastroenterologists to identify IBD patients at high risk of impaired QOL.
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  • 文章类型: Journal Article
    背景:复方斑茅胶囊(FFBM),中药,多年来一直被用于治疗原发性肝癌(PLC)。然而,生物活性成分,FFBM治疗PLC的机制尚不清楚。我们的目标是利用网络药理学来研究这些方面,并随后通过临床数据验证其有效性。
    方法:从HERB数据库获得FFBM成分,并使用SwissTargetPrediction数据库筛选生物活性成分。PharmMapper和GEO数据库用于获取FFBM和PLC的靶标和差异表达基因(DEGs),分别。使用维恩图确定了常见目标,然后进行富集和蛋白质-蛋白质相互作用(PPI)分析。此外,利用Cytoscape软件鉴定Hub基因并构建成分靶途径网络。随后,回顾性收集2008年1月至2019年12月在我院接受肝动脉化疗栓塞(TACE)治疗的确诊为不可切除PLC的患者.最后,进行Cox分析以揭示FFBM在不可切除的PLC的治疗中的作用。
    结果:FFBM有232个目标,PLC有1582个DEG。HSP90AA1和SRC被确定为关键靶标。阿尔法-桑他洛尔,甘草酸,和莫罗尼甙被确定为前三种生物活性成分。富集分析揭示了FFBM用于治疗PLC与多种途径之间的显着联系,如化学致癌作用,PI3K-AKT,Rap1,FoxO,MAPK,和VEGF途径。临床数据显示,食用FFBM可显着改善不可切除的PLC的预后,风险比为0.69。
    结论:我们的研究确定了FFBM的生物活性成分及其治疗PLC的潜在机制。此外,我们通过临床数据验证了有效性.
    BACKGROUND: Fufang Banmao capsules (FFBM), a traditional Chinese medicine, has been used to treat primary liver cancer (PLC) for several years. However, the bioactive ingredients, and mechanism of FFBM for treating PLC remains unclear. Our objective is to utilize network pharmacology to investigate these aspects and subsequently validate their effectiveness through clinical data.
    METHODS: The FFBM ingredients were obtained from the HERB database and screened for bioactive ingredients using the SwissTargetPrediction database. The PharmMapper and GEO database were used to acquire targets and differentially expressed genes (DEGs) for FFBM and PLC, respectively. Common targets were identified using Venn diagrams, followed by enrichment and protein-protein interaction (PPI) analysis. Furthermore, the Cytoscape software was utilized to identify Hub genes and construct the ingredienttarget- pathway network. Subsequently, patients diagnosed with unresectable PLC who underwent transcatheter arterial chemoembolization (TACE) at our hospital between January 2008 and December 2019 were retrospectively collected. Finally, Cox analysis was conducted to reveal the role of FFBM in the treatment of unresectable PLC.
    RESULTS: FFBM had 232 targets, and PLC had 1582 DEGs. HSP90AA1 and SRC were identified as crucial targets. Alpha-santalol, glycyrrhizin, and morroniside were identified as the top three bioactive ingredients. Enrichment analysis revealed a significant connection between FFBM utilization for treating PLC and multiple pathways, such as chemical carcinogenesis, PI3K-AKT, Rap1, FoxO, MAPK, and VEGF pathway. Clinic data revealed that consuming FFBM significantly improved the prognosis of unresectable PLC with a hazard ratio of 0.69.
    CONCLUSIONS: Our study identified the bioactive ingredients of FFBM and its potential mechanisms for treating PLC. Additionally, we validated the effectiveness through clinical data.
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  • 文章类型: Journal Article
    基因编辑是一种不断发展的基因工程技术,它可以对广谱的基因调节疾病进行精确编辑以实现治愈性治疗,也有可能作为疾病常规治疗的辅助手段。基因编辑技术,主要基于成簇的规则间隔回文重复(CRISPR)-CRISPR相关蛋白系统,能够在体细胞中产生遗传修饰,为广泛的人类疾病的基因治疗提供了有希望的新策略。目前,基因编辑技术在人类多种疾病中显示出巨大的应用前景,不仅在治疗潜力方面,而且在人类疾病动物模型的构建方面。本文阐述了基因编辑技术在血液病中的应用,实体瘤,免疫疾病,眼科疾病,和代谢性疾病;重点介绍了基因编辑技术在镰状细胞病中的治疗策略;概述了基因编辑技术在人类疾病动物模型构建中的作用;并讨论了基因编辑技术在疾病治疗中的局限性,旨在为基因编辑技术在人类疾病中的应用提供重要参考。
    Gene editing is a growing gene engineering technique that allows accurate editing of a broad spectrum of gene-regulated diseases to achieve curative treatment and also has the potential to be used as an adjunct to the conventional treatment of diseases. Gene editing technology, mainly based on clustered regularly interspaced palindromic repeats (CRISPR)-CRISPR-associated protein systems, which is capable of generating genetic modifications in somatic cells, provides a promising new strategy for gene therapy for a wide range of human diseases. Currently, gene editing technology shows great application prospects in a variety of human diseases, not only in therapeutic potential but also in the construction of animal models of human diseases. This paper describes the application of gene editing technology in hematological diseases, solid tumors, immune disorders, ophthalmological diseases, and metabolic diseases; focuses on the therapeutic strategies of gene editing technology in sickle cell disease; provides an overview of the role of gene editing technology in the construction of animal models of human diseases; and discusses the limitations of gene editing technology in the treatment of diseases, which is intended to provide an important reference for the applications of gene editing technology in the human disease.
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  • 文章类型: Journal Article
    心脏骤停(CA)是重症监护病房(ICU)中的常见事件,严重威胁患者的预后。因此,确定一个简单有效的临床指标来判断患者在接受CA治疗后的预后至关重要。目的探讨乳酸脱氢酶与白蛋白比值(LAR)与CA术后患者预后的关系。
    参与者的临床数据是从重症监护医学信息集市(MIMIC-IV,v2.0;2008年至2019年)。根据30天的预后,患者分为存活组(n=216)和非存活组(n=304).使用受限三次样条(RCS)确定最佳LAR阈值,将患者分为高LAR组(≥15.50,n=257)和低LAR组(<15.50,n=263)。在Kaplan-Meier生存分析后,绘制了两组的ICU住院时间和30天累积生存曲线。采用多因素Cox回归分析LAR与CA患者预后的关系。绘制受试者工作特征(ROC)曲线以评估LAR对30天全因死亡率的预测功效,并使用敏感性分析来检查结果的可靠性。
    共纳入520例CA患者,30天死亡率为58.46%。非幸存者组的LAR高于幸存者组。RCS显示LAR与患者在ICU住院期间和30天的死亡风险呈线性趋势关系;随着LAR的增加,死亡的风险也是如此。Kaplan-Meier生存曲线显示,与低LAR组相比,在CA患者中,高LAR组ICU住院和30天的累积生存率较低(p<0.001).多因素Cox回归分析显示,LAR升高(≥15.50)是ICU住院和30天死亡的独立危险因素(p<0.005)。ROC分析表明,LAR在预测CA患者30天全因死亡率方面优于序贯器官衰竭评估(SOFA)评分(曲线下面积(AUC)=0.676,95%置信区间[CI]:0.629-0.723)。为了验证我们发现的可靠性,我们进行了敏感性分析,发现结果是可靠的.
    LAR升高可能是ICU住院期间和30天CA后患者死亡率的预测因子,从而为这些患者的临床管理提供参考。
    UNASSIGNED: Cardiac arrest (CA) is a common event in the intensive care unit (ICU), which seriously threatens the prognosis of patients. Therefore, it is crucial to determine a simple and effective clinical indicator to judge the prognosis of patients after a CA for later treatments. The purpose of this study was to investigate the relationship between the lactate dehydrogenase to albumin ratio (LAR) and the prognosis of patients after a CA.
    UNASSIGNED: The clinical data of participants was obtained from the Medical Information Mart for Intensive Care IV (MIMIC-IV, v2.0; 2008 to 2019). According to the 30-day prognosis, patients were divided into a survivors group (n = 216) and a non-survivors group (n = 304). The optimal LAR threshold was determined using restricted cubic spline (RCS), which divided patients into a high LAR group ( ≥ 15.50, n = 257) and a low LAR group ( < 15.50, n = 263). The ICU hospitalization and 30-day accumulative survival curves of the two groups were plotted following the Kaplan-Meier survival analysis. Multivariate Cox regression was used to analyze the relationship between the LAR and the prognosis of CA patients. Receiver operating characteristic (ROC) curves were drawn to evaluate the predictive efficacy of the LAR on 30-day all-cause mortality, and sensitivity analysis was used to check the reliability of the findings.
    UNASSIGNED: A total of 520 patients with CA were enrolled and the 30-day mortality was 58.46%. The LAR in the non-survivors group was higher than in the survivors group. The RCS showed a linear trend relationship between the LAR and the mortality risk in patients during their ICU stay and 30 days; moreover, as the LAR increased, so did the risk of mortality. The Kaplan-Meier survival curve showed that compared with the low LAR group, the cumulative survival rates of ICU hospitalization and 30 days were lower in the high LAR group among CA patients (p < 0.001). Multivariate Cox regression analysis showed that an elevated LAR ( ≥ 15.50) was an independent risk factor for mortality during ICU stay and 30 days (p < 0.005). ROC analysis suggested that the LAR was superior to the sequential organ failure assessment (SOFA) score in predicting the 30-day all-cause mortality in CA patients (area under the curve (AUC) = 0.676, 95% confidence interval [CI]: 0.629-0.723). To verify the reliability of our findings, we performed sensitivity analyses and found that the findings were reliable.
    UNASSIGNED: An elevated LAR might be a predictor of mortality in patients following a CA during ICU hospitalization and 30 days, thereby it can be used to provide a reference for the clinical management of these patients.
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  • 文章类型: Journal Article
    本研究旨在深入了解在中国儿童医院开展的儿科临床试验,为儿童医院的发展和儿科药物的研发提供有价值的参考。
    对中国临床试验注册中心(ChiCTR)和ChinaDrugTrials.org进行了全面搜索。cn收集所有涉及18岁以下受试者的临床试验信息,包括在儿童医院进行的那些。检索期延长至2022年12月31日。
    共收集了459项儿科临床试验,包括来自ChiCTR的299个和来自药物临床试验注册和信息公开平台(信息平台)的160个。上市后药物研究和III期临床试验占研究阶段的大部分。这些试验涵盖了广泛的疾病/系统,特别关注呼吸系统疾病,肿瘤,内分泌失调,营养或代谢疾病。化学药物构成了研究最广泛的类别,而中药/天然药物受到的关注相对较少。临床试验活动主要集中在中国东部沿海地区,多中心试验是最主要的。427项研究获得了伦理委员会的批准。
    中国儿童医院进行的儿科临床试验总体呈上升趋势,专注于中医的研究有限,以及严重的区域和体制失衡。此外,道德审查程序仍有改进的空间。建议儿童医院在提高科研能力的同时,优化资源配置,有效满足医疗服务需求。此外,培育更多专注于研究的儿童医院将有助于中国儿童健康的高质量发展。
    UNASSIGNED: This study aimed to gain insights into pediatric clinical trials conducted in children\'s hospitals in China and provide valuable references for the development of children\'s hospitals and the research and development of pediatric drugs.
    UNASSIGNED: A comprehensive search was performed on the Chinese Clinical Trial Registry (Chi CTR) and ChinaDrugTrials.org.cn to collect information on all clinical trials involving subjects under 18 years, including those conducted in children\'s hospitals. The retrieval period was extended until 31 December 2022.
    UNASSIGNED: A total of 459 pediatric clinical trials were collected, comprising 299 from Chi CTR and 160 from the Drug Clinical Trial Registration and Information Publicity Platform (Information Platform). Post-marketing drug studies and phase III clinical trials accounted for the majority of research stages. These trials covered a wide range of diseases/systems, with a particular focus on respiratory system disorders, tumors, endocrine disorders, and nutritional or metabolic diseases. Chemical drugs constituted the most extensively studied category, while traditional Chinese medicine/natural drugs received comparatively less attention. Clinical trial activities were primarily geographically focused on the eastern coastal regions of China, with multicenter trials being the most predominant. Ethics committee approval was obtained for 427 studies.
    UNASSIGNED: The pediatric clinical trials conducted by children\'s hospitals in China have shown an overall upward trend; however, there is limited research focusing on traditional Chinese medicine, along with significant regional and institutional imbalances. Furthermore, there is still room for improvement regarding ethical review processes. It is recommended that children\'s hospitals enhance their scientific research capabilities while optimizing resource allocation to meet medical service demands effectively. Additionally, fostering more research-focused children\'s hospitals will contribute to the high-quality development of children\'s health in China.
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  • 文章类型: Journal Article
    胶质瘤是中枢神经系统最常见的恶性肿瘤,给患者带来巨大的健康负担。由于神经胶质瘤的内在特征和治疗方式缺乏突破,大多数患者的预后仍然较差。这导致全球范围内沉重的心理和财务负担。近年来,大麻二酚(CBD)由于其抗肿瘤作用而引起了广泛的关注和研究,抗炎,和神经保护特性。本文综述了CBD在脑胶质瘤治疗中的临床前和临床研究。以及CBD纳米药物配方的现状,并讨论了CBD在未来胶质瘤治疗中的潜力和挑战。
    Glioma is the most common malignant tumor in central nervous system, with significant health burdens to patients. Due to the intrinsic characteristics of glioma and the lack of breakthroughs in treatment modalities, the prognosis for most patients remains poor. This results in a heavy psychological and financial load worldwide. In recent years, cannabidiol (CBD) has garnered widespread attention and research due to its anti-tumoral, anti-inflammatory, and neuroprotective properties. This review comprehensively summarizes the preclinical and clinical research on the use of CBD in glioma therapy, as well as the current status of nanomedicine formulations of CBD, and discusses the potential and challenges of CBD in glioma therapy in the future.
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  • 文章类型: Journal Article
    白藜芦醇(RES)是天然存在的多酚化合物。最近的研究已经确定了RES的多种潜在健康益处,包括抗氧化剂,抗炎,抗肥胖,抗癌,抗糖尿病,心血管,和神经保护特性。本文的目的是总结和分析RES在疾病预防和治疗中的生物学活性的研究,以及它的新陈代谢和生物利用度。并讨论了其临床应用面临的挑战和未来的研究方向。RES在许多疾病的预防和治疗中显示出显著的潜力。RES研究的未来方向应集中在提高其生物利用度,进行更多的临床试验以确定其在人体中的有效性,并研究其作用机制。一旦克服了这些挑战,RES有望成为一种有效的健康干预措施。
    Resveratrol (RES) is a naturally occurring polyphenolic compound. Recent studies have identified multiple potential health benefits of RES, including antioxidant, anti-inflammatory, anti-obesity, anticancer, anti-diabetic, cardiovascular, and neuroprotective properties. The objective of this review is to summarize and analyze the studies on the biological activities of RES in disease prevention and treatment, as well as its metabolism and bioavailability. It also discusses the challenges in its clinical application and future research directions. RES exhibits significant potential in the prevention and treatment of many diseases. The future direction of RES research should focus on improving its bioavailability, conducting more clinical trials to determine its effectiveness in humans, and investigating its mechanism of action. Once these challenges have been overcome, RES is expected to become an effective health intervention.
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  • 文章类型: Journal Article
    宫颈癌是全球影响女性健康的第二大恶性肿瘤,宫颈癌的发病率和死亡率在全球范围内持续上升。复发或转移性宫颈癌患者的5年生存率明显降低,现有的治疗方式疗效低,不良反应大,所以非常需要新的,有效,和耐受性良好的疗法。抗体-药物缀合物(ADC)是一种新的靶向治疗方式,可以有效地杀死肿瘤细胞。这篇综述旨在总结文章的组成,研究,以及ADC的发展历史和作用机制,综述ADC在宫颈癌治疗中的研究进展,并对ADC的应用进行了总结和展望。
    Cervical cancer is the second most prevalent malignancy affecting women\'s health globally, and the number of morbidity and mortality from cervical cancer continues to rise worldwide. The 5-year survival rate of patients with recurrent or metastatic cervical cancer is significantly reduced, and existing treatment modalities have low efficacy and high adverse effects, so there is a strong need for new, effective, and well-tolerated therapies. Antibody-drug conjugates (ADCs) are a new targeted therapeutic modality that can efficiently kill tumor cells. This review aims to summarize the composition, research, and development history and mechanism of action of ADCs, to review the research progress of ADCs in the treatment of cervical cancer, and to summarize and prospect the application of ADCs.
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  • 文章类型: Journal Article
    Clerodendranthusspicatus(Thunb.)C.Y.Wu(CS)是一种被广泛研究的植物,显示出治疗泌尿系疾病的潜力。以前的研究集中在它的化学成分上,药理作用,和临床应用。这篇综述旨在对现有的CS文献进行全面的总结和评价。它还提出了未来的研究方向,以增加我们对其药用价值的理解。从几个数据库中选择了129篇文献,包括PubMed,WebofScience,中国国家知识基础设施(CNKI),万方数据库,和谷歌学者,并进行了分析。CS的45种活性化合物具有降低尿酸等药理作用,抗炎,抗氧化,和肾脏保护。这些作用的潜在机制可能与抑制转化生长因子β1(TGF-β1)的激活有关。减少炎症因子,如IL-8,IL-1β,TNF-α,PGE2,IFN-γ,和IL-6水平,抑制NF-κB的激活,JAK/STAT通路,增强ROS的清除,MDADPPH·,和O2--,调节凋亡相关通路和蛋白的表达。本文还讨论了CS的质量控制及其治疗泌尿系疾病的有效性和安全性。该研究得出结论,CS具有很高的治疗泌尿系疾病的潜力。未来的研究应集中在观察CS活性化合物在体内的代谢变化以及研究CS对关键信号通路的影响。此外,应进行更加规范合理的临床研究和安全性评价实验,以获得更多的临床数据。
    Clerodendranthus spicatus (Thunb.) C.Y.Wu (CS) is a widely studied plant that shows potential in treating urinary diseases. Previous studies have focused on its chemical composition, pharmacological effects, and clinical applications. This review aims to provide a comprehensive summary and evaluation of the existing literature on CS. It also suggests future research directions to increase our understanding of its medicinal value. 129 pieces of literature were selected from several databases, including PubMed, Web of Science, China National Knowledge Infrastructure (CNKI), Wan-fang Database, and Google Scholar, and were analyzed. Forty-five active compounds of CS have pharmacological effects such as lowering uric acid, anti-inflammation, anti-oxidation, and kidney protection. The potential mechanisms of these effects may be related to inhibiting transforming growth factor β1 (TGF-β1) activation, reducing inflammatory factors such as IL-8, IL-1β, TNF-α, PGE2, IFN-γ, and IL-6 levels, suppressing the activation of NF-κB, JAK/STAT pathway, enhancing the clearance of ROS, MDA DPPH·, and O2 ̇ -, and regulating the expression of apoptosis-related pathways and proteins. This paper also discusses the quality control of CS and its efficacy and safety in treating urinary diseases. The study concludes that CS has a high potential for treating urinary diseases. Future studies should focus on observing the metabolic changes of CS active compounds in vivo and investigating the effects of CS on key signaling pathways. Additionally, more standardized and reasonable clinical studies and safety evaluation experiments should be conducted to obtain more clinical data.
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  • 文章类型: Journal Article
    脑机接口(BCI)技术在医学研究和应用中迅速发展。作为一种新兴的生物医学工程技术,它在脑疾病诊断和治疗的临床研究中引起了极大的关注,神经康复,和心理健康。然而,BCI在临床研究中也提出了一些挑战和伦理问题。在这篇文章中,作者调查和讨论了BCI在医药保健中的三个方面:伦理治理状况,临床研究中与BCI相关的多维伦理挑战,以及对伦理审查的暗示关注。尽管BCI在医疗保健领域的前沿研究和发展潜力巨大,自身引发的伦理挑战,临床研究和脑功能的复杂性为BCI的伦理学提出了新的特殊领域。确保BCI在医疗保健和医学领域的“负责任的创新”研究,建立道德全球治理框架和体系,以及医学前沿BCI研究的特殊指南。
    Brain-computer interface (BCI) technology is rapidly advancing in medical research and application. As an emerging biomedical engineering technology, it has garnered significant attention in the clinical research of brain disease diagnosis and treatment, neurological rehabilitation, and mental health. However, BCI also raises several challenges and ethical concerns in clinical research. In this article, the authors investigate and discuss three aspects of BCI in medicine and healthcare: the state of international ethical governance, multidimensional ethical challenges pertaining to BCI in clinical research, and suggestive concerns for ethical review. Despite the great potential of frontier BCI research and development in the field of medical care, the ethical challenges induced by itself and the complexities of clinical research and brain function have put forward new special fields for ethics in BCI. To ensure \"responsible innovation\" in BCI research in healthcare and medicine, the creation of an ethical global governance framework and system, along with special guidelines for cutting-edge BCI research in medicine, is suggested.
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