Efficacy evaluation

疗效评价
  • 中医证候疗效是中医特有的、能够体现中医优势的评价指标。中医证候疗效评价方法和测量工具的发展,可以为中医临床疗效评价和新的中药制剂的研制提供客观和定量的依据,是中医药疗效评价创新方法和技术的探索方向。常规的评价方法是主观的,限于症状的缓解和体征的改善,这使得难以形成统一的评估标准。此外,评价方法缺乏统一性,客观性,和定量研究。科学内涵,评价思路和方法,证候疗效评价的关键技术尚不清楚,这导致了不同的评估模式,方法,和索引。中医证候疗效量表为中医证候的客观量化和规范化提供了新思路。本文系统总结了研究方法和存在的问题,介绍了评价量表的研究进展,并对中医证候疗效评价的特点提出了一些思考,旨在为该领域的研究提供见解。
    Traditional Chinese medicine(TCM) syndrome-based efficacy is an evaluation index which is unique to TCM and can reflect the advantages of TCM. The development of the methods and measurement tools for evaluating TCM syndrome-based efficacy can provide objective and quantitative evidence for the clinical efficacy evaluation of TCM and the development of new Chinese medicine preparations, being the exploration direction of innovative methods and technologies for evaluating TCM efficacy. The conventional evaluation methods are subjective and limited to the mitigation of symptoms and the improvement of physical signs, which make it difficult to form a unified evaluation standard. In addition, the evaluation methods lack unity, objectivity, and quantitative research. The scientific connotation, evaluation ideas and methods, and key technologies of the evaluation for the therapeutic effect on syndromes remain unclear, which leads to diverse evaluation modes, methods, and indexes. The syndrome-based efficacy scale provides a new idea for the objective quantification and standardization of TCM syndromes. This review systematically summarizes the methods and problems, introduces the research progress in the evaluation scales, and puts forward some thoughts on the characteristics of TCM syndrome-based efficacy evaluation, aiming to provide insights for the research in this field.
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  • 文章类型: Systematic Review
    草药(HM)的临床经验已用于治疗各种人类难治性疾病。由于使用HM治疗疾病具有多成分、多靶点的特点,很难确定生物活性成分,探索分子靶标并揭示作用机制。代谢组学由于其在检测内源性小分子代谢物变化方面的独特优势,经常用于表征外部干扰对生物体的影响。它的系统性和完整性符合HM的有效特征。HM干预后,代谢组学能够准确捕获和描述内源性代谢产物在功能化合物干扰下的行为,这将用于解码HM的生物活性成分并阐明分子靶标。代谢组学可以提供一种解释HM的方法,解决不明确的临床疗效和作用机制。在这次审查中,系统介绍了代谢组学策略及其在HM中的应用,这为代谢组学方法表征HM的药理作用和分子靶标提供了有价值的见解。
    Clinical experiences of herbal medicine (HM) have been used to treat a variety of human intractable diseases. As the treatment of diseases using HM is characterized by multi-components and multi-targets, it is difficult to determine the bio-active components, explore the molecular targets and reveal the mechanisms of action. Metabolomics is frequently used to characterize the effect of external disturbances on organisms because of its unique advantages on detecting changes in endogenous small-molecule metabolites. Its systematicity and integrity are consistent with the effective characteristics of HM. After HM intervention, metabolomics can accurately capture and describe the behavior of endogenous metabolites under the disturbance of functional compounds, which will be used to decode the bioactive ingredients of HM and expound the molecular targets. Metabolomics can provide an approach for explaining HM, addressing unclear clinical efficacy and undefined mechanisms of action. In this review, the metabolomics strategy and its applications in HM are systematically introduced, which offers valuable insights for metabolomics methods to characterizing the pharmacological effects and molecular targets of HM.
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  • 文章类型: Journal Article
    实体瘤的反应评估标准(RECIST1.1)是经典的,并且在公众中很受欢迎。然而,最近有一些问题。例如,部分响应范围从30%到99%,客观反应是二分法的,所以可能有一些异质性。评估疗效的新指标一直在研究,如早期肿瘤缩小,响应时间和响应深度(DpR)。DpR已用于恶性血液病,并被认为是效率的预测指标。在实体瘤中,DpR最早由Mansmann等人提出。在转移性结直肠癌(mCRC)中,定义为肿瘤缩小的百分比,这是一个连续的度量,并且可以避免由于反应二分法而导致的信息丢失,该反应已广泛应用于几种实体瘤。一些作者发现DpR和OS之间存在关联,DpR是mCRC的有价值的替代端点,转移性乳腺癌,转移性黑色素瘤和晚期胰腺癌。然而,DpR对肺癌和胃癌的预测价值尚不明确。这表明DpR尚未形成成熟统一的应用标准。本文总结了DpR作为实体瘤长期预后预测因子的研究。并讨论了DpR应用中的挑战和局限性。
    Response Evaluation Criteria in Solid Tumors (RECIST 1.1) is classical and popular for public. However, there are some problems recently. For example, partial response ranges from 30% to 99%, objective response is dichotomous, so there may be some heterogeneity. New metrics for evaluating the efficacy have been investigating, such as early tumor shrinkage, time to response and depth of response (DpR). DpR has been used in hematologic malignancies and is considered as a predictor of efficiency. In solid tumors, DpR was firstly proposed by Mansmann et al. in metastatic colorectal cancer (mCRC) and defined as the percentage of tumor shrinkage, which is a continuous metric, and could avoid the information loss due to dichotomization of responses that has been widely applied to several kinds of solid tumors. Some authors have found associations between DpR and OS, DpR is a valuable surrogate endpoint for mCRC, metastatic breast cancer, metastatic melanoma and advanced pancreatic cancer. However, the predictive value of DpR is still uncertain in the research of lung cancer and gastric cancer. Which indicating that a mature and unified application standard has not yet been formed for DpR. This article summarizes researches on the DpR as a predictor of the long-term outcomes for solid tumors, it also discusses the challenges and limitations in the applications of DpR.
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  • 文章类型: Journal Article
    Photodynamic therapy (PDT) is a new option for oral lichen planus (OLP) management; however, there are different opinions on the efficacy of PDT for OLP. The aim of this study was to comprehensively assess the efficacy of PDT in the treatment of OLP and compare PDT with steroid therapy.
    A systematic review and meta-analysis were conducted to assess the curative effect of PDT. Five electronic databases were searched, PubMed, Web of Science, the Cochrane Library, Embase, and EBSCO up to 1 December, 2019. Random and fixed effects models for pooled estimates calculation were used and the Meta package of R was applied.
    Pooled estimates revealed that, after PDT, the lesion size decreased by 1.53 cm2 (95% confidence interval (CI): 0.71-2.35) after PDT and the partial response (PR) was 0.77 (95% CI: 0.65-0.85). The visual analogue scale (VAS) score decreased by 3.82 (95% CI: 2.80-4.85) and the Thongprasom sign score decreased by 1.33 (95% CI: 0.56-2.10) after PDT. Subgroup analyses revealed that the 5-aminolevulinic acid (5-ALA) was more effective than methylene blue (MB), with a PR of 0.87 (95% CI: 0.80-0.91). The topical use of 5-ALA yielded a better response than gargling methylene blue. In terms of VAS, the diode laser showed a better clinical PR in the treatment of OLP. In terms of changes in lesion size, the efficacy of the semiconductor laser was higher than that of the diode laser. PDT had a similar efficacy to topical corticosteroids, as shown by pooled estimates of five randomised controlled trials with 139 lesions.
    This systematic review indicates that PDT is an effective treatment modality for the management of OLP. PDT is as effective as topical corticosteroid in the treatment of OLP and could be used for cases resistant to steroids or when steroids are contraindicated.
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  • 文章类型: Journal Article
    抗生素耐药性的出现和传播是一个日益增长的全球威胁,并引起了全球对限制抗生素在畜牧业中使用的兴趣。当我们进入后抗生素时代时,越来越需要确定用于预防和治疗的抗生素替代品。虽然植物治疗的补救措施是可用的,并在欧洲使用,其疗效的证据目前非常有限,但仍然是必要的。已使用PICOS方法对用于治疗和预防农场动物传染病的植物疗法进行了系统评价。根据协议对研究中的信息进行分析和提取,包括:出版年份和来源,研究机构,物种,耕作制度,应用的目的,疾病的焦点,诊断方法,使用的补救措施(补救措施的来源和成分),补救措施的生产者,管理方式,研究设计,和对照组,效果度量,和研究结果。共筛选出1705篇论文,但只有44项(包括53项独立研究)符合纳入标准.确定的大多数科学研究表明在研究设计以及所研究的植物疗法的呈现和标准化方面存在局限性,这限制了得出坚定结论的可能性。在89%的研究中调查了预防效果。与对照组相比,一半的研究报告了植物疗法对亚临床措施的影响不确定。审查强调了尚未解决的问题,例如需要标准化的对照试验和通过分析方法改善植物产品的标准化,如高效液相色谱法。为了实现可复制的结果并在农场实践中建立植物疗法的功效和安全性,有必要考虑这一点。农场动物替代药物的可管理性也需要进一步调查,以确定是否为其使用提供了所有必要的先决条件。
    The emergence and spread of antibiotic resistance is a growing global threat and has aroused a global interest in limiting antibiotic use in animal agriculture. As we are entering the post-antibiotic era, there is a growing need for identifying alternatives to antibiotics for prevention and treatment. Although phytotherapeutic remedies are available and are used in Europe, evidence of their efficacy is currently very limited but is nevertheless still necessary. A systematic review of phytotherapy used for the treatment and prevention of infectious diseases in farm animals has been conducted using the PICOS approach. Analysis and extraction of information from studies was performed according to a protocol, and included: publication year and source, research body, species, farming system, purpose of application, disease in focus, diagnostic method, remedy used (origin and ingredients of the remedy), producer of remedy, way of administration, study design, and control groups, measure of effect, and outcome of the study. A total of 1,705 papers were screened, but only 44 (comprising 53 independent studies) met the inclusion criteria. The majority of the scientific studies identified show limitations in the study design as well as in presentation and standardization of the botanical remedies studied, which limits the possibilities of drawing firm conclusions. Preventive effect was investigated in 89% of the studies. Half of the studies reported uncertain effects of the botanical remedies on subclinical measures in comparison to a control group. The review highlights unresolved questions such as the need for standardized controlled trials and for improved standardization of the botanical products by analytical methods, such as high-performance liquid chromatography. It is necessary to consider this in order to achieve replicable outcomes and establish efficacy and safety of phytotherapy in farm practice. The manageability of alternative medicine in farm animals also warrants further investigation so as to ascertain whether all necessary preconditions for their use are being granted.
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