Efficacy evaluation

疗效评价
  • 文章类型: Journal Article
    严重急性呼吸道综合征冠状病毒-2(SARS-CoV-2)的出现和冠状病毒病-19(COVID-19)大流行的发作导致了对治疗性治疗方案的迫切需要。开发治疗性抗体以填补传统抗病毒药物不可用的空白。在2020年末,美国政府致力于在病毒中和测定和SARS-CoV-2感染的仓鼠模型中比较候选治疗性抗体。随着SARS-CoV-2变种的出现,这项工作扩大到评估近50种产品对主要变体的功效。进一步评价产物的子集在仓鼠中的治疗功效。这里我们报告仓鼠研究的结果,包括多种变异的致病性,产品的中和能力,以及针对Delta和Omicron变体的产品功效测试。这些研究证明了具有变体出现的早期产品的功效丧失,并且支持使用仓鼠模型来评估治疗剂。
    The emergence of severe acute respiratory syndrome coronavirus-2 (SARS-CoV-2) and onset of the coronavirus disease-19 (COVID-19) pandemic led to an immediate need for therapeutic treatment options. Therapeutic antibodies were developed to fill a gap when traditional antivirals were not available. In late 2020, the United States Government undertook an effort to compare candidate therapeutic antibodies in virus neutralization assays and in the hamster model of SARS-CoV-2 infection. With the emergence of SARS-CoV-2 variants, the effort expanded to evaluate the efficacy of nearly 50 products against major variants. A subset of products was further evaluated for therapeutic efficacy in hamsters. Here we report results of the hamster studies, including pathogenicity with multiple variants, neutralization capacity of products, and efficacy testing of products against Delta and Omicron variants. These studies demonstrate the loss of efficacy of early products with variant emergence and support the use of the hamster model for evaluating therapeutics.
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  • 文章类型: Journal Article
    背景:认知行为疗法(CBT)和习惯逆转训练(HRT)已显示出在解决抽动症状和共病精神病方面的应用潜力。尽管他们的理论潜力,关于其综合疗效的经验证据仍然有限。
    目的:评价CBT联合HRT治疗Tourette综合征(TS)患儿焦虑障碍的疗效。
    方法:收集我院2022年1月至2023年6月收治的TS患儿的临床资料,将患者分为常规治疗(对照组)组和CBT联合HRT组。基线特征,焦虑分数,tic严重程度评分,治疗依从性,并对家长满意度进行了评估。使用t检验进行统计分析,卡方检验,和相关分析。
    结果:共有136名患者,其中对照组65例,CBT联合HRT组71例,包括在内。CBT联合HRT组较对照组有显著改善。干预后评估显示焦虑评分从63.52±1.81下降到40.53±1.64(t=2.022,P=0.045),耶鲁全球抽动严重程度量表总分由22.14±5.67降至16.28±4.91(t=2.288,P=0.024)。CBT联合HRT组治疗依从性(85.47±7.62%)明显高于对照组(82.32±6.54%;t=2.596,P=0.010)。CBT联合HRT组家长满意度得分(8.69±1.77)高于对照组(7.87±1.92;t=2.592,P=0.011)。
    结论:这项研究表明,CBT联合HRT可显著降低TS患儿的焦虑症状和抽动严重程度,具有更高的治疗依从性和父母满意度。这些发现支持这种综合治疗方法在TS治疗中的潜在应用。
    BACKGROUND: Cognitive-behavioral therapy (CBT) and habit reversal training (HRT) have shown application potential in addressing tic symptoms and comorbid psychiatric conditions. Despite their theoretical potential, empirical evidence on their combined efficacy remains limited.
    OBJECTIVE: To evaluate the efficacy of CBT combined with HRT on anxiety disorders in children with Tourette\'s syndrome (TS).
    METHODS: Clinical data of children with TS admitted to our hospital from January 2022 to June 2023 were collected, and the patients were grouped into the conventional therapy (control) group and the CBT combined with HRT group. Baseline characteristics, anxiety scores, tic severity scores, treatment adherence, and parental satisfaction were assessed. Statistical analysis was performed using t-tests, chi-square tests, and correlation analysis.
    RESULTS: A total of 136 patients, including 65 patients in the control group and 71 patients in the CBT combined with HRT group, were included. The CBT combined with HRT group showed remarkable improvements compared with the control group. Post-intervention assessment revealed a decrease in anxiety scores from 63.52 ± 1.81 to 40.53 ± 1.64 (t = 2.022, P = 0.045), and the Yale Global Tic Severity Scale total score decreased from 22.14 ± 5.67 to 16.28 ± 4.91 (t = 2.288, P = 0.024). Treatment adherence was significantly higher in the CBT combined with HRT group (85.47 ± 7.62%) compared with the control group (82.32 ± 6.54%; t = 2.596, P = 0.010). Parental satisfaction scores were also higher in the CBT combined with HRT group (8.69 ± 1.77) compared with the control group (7.87 ± 1.92; t = 2.592, P = 0.011).
    CONCLUSIONS: This study demonstrates that CBT combined with HRT significantly reduces anxiety symptoms and tic severity in children with TS, with higher treatment adherence and parental satisfaction. These findings support the potential application of this comprehensive therapeutic approach for TS treatment.
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  • 文章类型: Journal Article
    癌症免疫疗法用于通过调节免疫系统来治疗肿瘤。尽管在临床试验之前已经评估了癌症免疫疗法的抗癌功效,传统的体内动物和终点模型不能充分复制肿瘤消除的复杂过程,并反映人类特异性免疫系统。因此,必须采用更复杂的模拟肿瘤-免疫微环境的模型来评估免疫治疗的有效性.此外,使用实时成像技术,可以应用逐步评估,可以更精确地评估治疗效果。这里,我们概述了最近为癌症免疫治疗应用开发的各种基于成像的评估平台.具体来说,我们讨论了使用直接成像稳定观察基于免疫细胞的肿瘤细胞杀伤的基本技术,一个再现空间观察的密闭空间的微孔,促进细胞-细胞相互作用的液滴测定,和重建血管环境的三维微生理系统。此外,我们建议未来的评估平台追求更多类似人类的免疫系统。本文受版权保护。保留所有权利。
    Cancer immunotherapy is used to treat tumors by modulating the immune system. Although the anticancer efficacy of cancer immunotherapy has been evaluated prior to clinical trials, conventional in vivo animal and endpoint models inadequately replicate the intricate process of tumor elimination and reflect human-specific immune systems. Therefore, more sophisticated models that mimic the complex tumor-immune microenvironment must be employed to assess the effectiveness of immunotherapy. Additionally, using real-time imaging technology, a step-by-step evaluation can be applied, allowing for a more precise assessment of treatment efficacy. Here, an overview of the various imaging-based evaluation platforms recently developed for cancer immunotherapeutic applications is presented. Specifically, a fundamental technique is discussed for stably observing immune cell-based tumor cell killing using direct imaging, a microwell that reproduces a confined space for spatial observation, a droplet assay that facilitates cell-cell interactions, and a 3D microphysiological system that reconstructs the vascular environment. Furthermore, it is suggested that future evaluation platforms pursue more human-like immune systems.
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  • 文章类型: Journal Article
    缺乏有效的治疗肺动脉高压的药物。先前的研究已经证明了新肽ACTY116的积极心血管系统保护作用。然而,它在普通水溶液注射中的稳定性差,在体内的半衰期短,这阻碍了筹备工作的发展。本研究旨在制备肽ACTY116的原位形成植入物(ISFI),并研究其对肺动脉高压的影响。我们使用NMP/TA作为溶剂和PLGA作为聚合物制备ISFI。这些ISFI表现出低粘度,低毒性和持续释放特性。在SU5416/低氧诱导的小鼠肺动脉高压模型中,ISFI和ACTY116肽均有效降低肺动脉高压,心肌肥厚和肺血管壁厚度。总之,这项研究强调了ACTY116作为肺动脉高压治疗的潜力,并提示将其纳入原位凝胶植入物可能是一个有前景的选择.
    There is a lack of effective therapeutic drugs for pulmonary arterial hypertension. Previous studies have demonstrated the positive cardiovascular system protective effects of the new peptide ACTY116. However, its stability in ordinary aqueous solution injections is poor and its half-life in the body is short, which has hindered the development of preparations. This study aimed to prepare in situ forming implants (ISFIs) of the peptide ACTY116 and investigate its impact on pulmonary arterial hypertension. We prepared ISFIs using NMP/TA as a solvent and PLGA as a polymer. These ISFIs exhibited low viscosity, low toxicity and sustained release properties. In a mouse model of pulmonary hypertension induced by SU5416/hypoxia, both ISFIs and ACTY116 peptides effectively reduced pulmonary hypertension, cardiac hypertrophy and pulmonary blood vessel wall thickness. In conclusion, this study highlights the potential of ACTY116 as a treatment for pulmonary arterial hypertension and suggests that incorporating it into an in-situ gel implant could be a promising option.
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  • 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: Journal Article
    目的:本研究评估利妥昔单抗治疗视神经脊髓炎谱系障碍(NMOSD)的疗效。方法:该研究最初包括40例诊断为NMOSD的患者,排除不符合完全纳入标准的患者后.常规组患者接受常规临床治疗,而研究组患者在常规治疗的基础上加用利妥昔单抗治疗。收集所有患者的基线数据和临床相关指标,比较两组疗效。结果:两组基线资料具有可比性(P>0.05)。研究组临床治疗后EDSS评分低于常规组,治疗前后EDSS评分差异高于常规组(P<0.05)。两组治疗前后视力矫正率差异无统计学意义(P>0.05)。研究组临床治疗后各项实验室指标均优于常规组(均P<0.05)。研究组临床治疗后复发率明显低于常规组(P<0.05)。研究组临床治疗后的不良反应少于常规组(P<0.05)。结论:本研究发现利妥昔单抗在预防NMOSD急性发作和复发方面具有显著疗效。强调其相对较好的安全性和耐受性。它强调了利妥昔单抗治疗NMOSD的潜力,并为未来的疾病管理提供了有价值的见解。
    UNASSIGNED: This study assesses the efficacy of rituximab in the treatment of neuromyelitis optica spectrum disorders (NMOSD).
    UNASSIGNED: The study initially included 40 patients with NMOSD diagnosed, after excluding patients who did not meet the complete inclusion criteria. Patients in the conventional group received routine clinical treatment, while patients in the study group received additional treatment with rituximab on the basis of the conventional treatment. Baseline data and clinically relevant indicators were collected for all patients, and the efficacy was compared between the two groups.
    UNASSIGNED: Baseline data were comparable between the two groups (p > 0.05). The EDSS scores after clinical treatment in the study group were lower than those in the conventional group, and the difference in EDSS scores before and after treatment was higher than that in the conventional group (p < 0.05). The difference in visual acuity correction before and after treatment was not significant between the two groups (p > 0.05). Laboratory indicators in the study group after clinical treatment were superior to those in the conventional group (all p < 0.05). The recurrence rate after clinical treatment in the study group was significantly lower than that in the conventional group (p < 0.05). Adverse reactions after clinical treatment in the study group were less than those in the conventional group (p < 0.05).
    UNASSIGNED: This study found that rituximab demonstrated significant efficacy in the acute attacks and recurrence prevention of NMOSD, emphasizing its relatively good safety and tolerability. It highlights the potential of rituximab in treating NMOSD and provides valuable insights for future disease management.
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  • 文章类型: Journal Article
    单光子发射计算机断层扫描(SPECT)通气灌注成像是诊断肺栓塞的主要成像方法,在慢性血栓栓塞性肺动脉高压(CTEPH)的诊断和疗效评价中的应用越来越受到重视。近年来,随着计算机软件技术的发展,通气/灌注(V/Q)成像定量分析技术日趋成熟。这项研究的目的是研究肺V/Q闪烁显像定量分析在评估CTEPH患者球囊肺血管成形术(BPA)疗效中的实用性。
    在此回顾性分析中,我们收集了2018年4月至2020年9月在中日友好医院接受BPA治疗的CTEPH患者的数据.样本由23名男性和28名女性组成,平均年龄55.1±12.7岁。所有患者在手术前一周内进行了V/Q闪烁显像,我们回顾了最后一次BPA手术后1-3个月内的肺动脉造影。我们在肺动脉造影前或后1周内重复进行V/Q闪烁显像,在收集这些患者的临床和血流动力学参数时。我们根据术后残余肺动脉高压的存在将患者分为两组,并使用t检验比较术前和术后定量肺灌注缺陷百分比评分(PPDs%)。
    总之,对51例患者进行了102V/Q闪烁扫描。定量PPDs%与血流动力学指标平均肺动脉压(mPAP)呈正相关,肺血管阻力(PVR),和平均右心室压(RVP)(r分别为0.605、0.391和0.464,所有P<0.001),并且与6分钟步行距离(6MWD)呈负相关(r=-0.254,P=0.010)。术前平均定量PPD%为(49.0±15.6)%,术后为(33.5±13.9)%,差异有统计学意义(t=11.249,P<0.001)。术前定量PPDs%在残余肺动脉高压组和非残余肺动脉高压组分别为(54.7±15.7)%和(44.0±13.8)%,分别为(t=2.599,P=0.012)。术后定量PPDs%分别为(41.5±12.5)%和(26.3±11.0)%,在残余肺动脉高压组和非残余肺动脉高压组中,(t=4.647,P<0.001)。
    在这项研究中,我们发现SPECT肺V/Q闪烁显像的定量分析充分反映了CTEPH患者的肺动脉压和临床状况.我们的结果表明,它在预测CTEPH患者的残留肺动脉高压和评估BPA术后疗效方面具有明确的实用性。
    UNASSIGNED: Single-photon emission computed tomography (SPECT) ventilation perfusion imaging is the main imaging method for the diagnosis of pulmonary embolism, and its application in the diagnosis and efficacy evaluation of chronic thromboembolic pulmonary hypertension (CTEPH) has been paid more and more attention. In recent years, with the development of computer software technology, ventilation/perfusion (V/Q) imaging quantitative analysis technology has become more and more mature. The objective of this study was to investigate the utility of quantitative analysis of pulmonary V/Q scintigraphy in evaluating the efficacy of balloon pulmonary angioplasty (BPA) in patients with CTEPH.
    UNASSIGNED: In this retrospective analysis, we collected data of patients diagnosed with CTEPH who underwent BPA at the China-Japan Friendship Hospital from April 2018 to September 2020. The sample consisted of 23 males and 28 females, with an average age of 55.1±12.7 years. All patients underwent V/Q scintigraphy within one week before surgery, and we reviewed the pulmonary angiography within 1-3 months following the last BPA procedure. We repeated V/Q scintigraphy within 1 week before or after the pulmonary angiography, at the time of collecting clinical and hemodynamic parameters of these patients. We divided the patients into two groups based on the presence of residual pulmonary hypertension post-surgery and compared the pre- and post-operative quantitative pulmonary perfusion defect percentage scores (PPDs%) using the t-test.
    UNASSIGNED: In all, 102 V/Q scintigraphy scans were performed in 51 patients. The quantitative PPDs% were positively correlated with the hemodynamic indexes mean pulmonary arterial pressure (mPAP), pulmonary vascular resistance (PVR), and mean right ventricular pressure (RVP) (r=0.605, 0.391, and 0.464, respectively, all P<0.001) and negatively correlated with the 6-minute walking distance (6MWD) (r=-0.254, P=0.010). The average preoperative quantitative PPDs% were (49.0±15.6)% which significantly decreased to (33.5±13.9)% after surgery (t=11.249, P<0.001). The preoperative quantitative PPDs% were (54.7±15.7)% and (44.0±13.8)% in the residual pulmonary hypertension group and the non-residual pulmonary hypertension group, respectively (t=2.599, P=0.012). The postoperative quantitative PPDs% were (41.5±12.5)% and (26.3±11.0)%, in the residual pulmonary hypertension group and the non-residual pulmonary hypertension group, respectively (t=4.647, P<0.001).
    UNASSIGNED: In this study, we found that quantitative analysis of SPECT pulmonary V/Q scintigraphy adequately reflected the pulmonary artery pressure and clinical status in patients with CTEPH. Our results demonstrate its definite utility in predicting residual pulmonary hypertension and in evaluating the postoperative efficacy of BPA in patients with CTEPH.
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  • 文章类型: Journal Article
    这项研究探讨了达鲁那韦(DRV)的治疗效果,利匹韦林(RPV),和ETV(ETV)对UM-UC-5膀胱癌细胞,解决膀胱癌研究中创新治疗的关键需求。通过全面评估他们在不同时间间隔内的个体和组合效应,ETV成为最有效的药物,最低IC50为5.9µM,紧随其后的是RPV(最低IC50为9.6µM),而DRV表现出最小的有效性(最低IC50为25.6µM)。值得注意的是,ETV和RPV组合具有明显的协同作用,特别是在48和72小时的低浓度。ETV和DRV也观察到了协同作用,尽管程度较小,主要是在48小时。相反,DRV和RPV组合产生的效果最小,在自然界中主要是添加剂。总之,这项临床前调查强调了ETV和RPV的有希望的治疗潜力,作为独立治疗和组合治疗,暗示膀胱癌治疗的再利用机会,这可以为这种疾病提供一种新的治疗方法,该方法更快,并且没有抗癌药物那样严重的副作用。这些发现代表了在癌症研究中推进个性化医学的实质性进展,并将在即将进行的研究中进一步审查。
    This research explores the therapeutic efficacy of Darunavir (DRV), Rilpivirine (RPV), and Etravirine (ETV) against UM-UC-5 bladder cancer cells, addressing the critical need for innovative treatments in bladder cancer research. Through a comprehensive assessment of their individual and combined effects across diverse time intervals, ETV emerges as the most potent drug, with a lowest IC50 of 5.9 µM, closely followed by RPV (lowest IC50 of 9.6 µM), while DRV exhibits the least effectiveness (lowest IC50 of 25.6 µM). Notably, a significant synergistic effect is evident in the ETV and RPV combination, especially at 48 and 72 h for low concentrations. Synergies are also observed with ETV and DRV, albeit to a lesser extent and primarily at 48 h. Conversely, the DRV and RPV combination yields minimal effects, predominantly additive in nature. In summary, this pre-clinical investigation underscores the promising therapeutic potential of ETV and RPV, both as standalone treatments and in combination, hinting at repurposing opportunities in bladder cancer therapy, which could give a new treatment method for this disease that is faster and without as severe side effects as anticancer drugs. These findings represent a substantial stride in advancing personalized medicine within cancer research and will be further scrutinized in forthcoming studies.
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  • 文章类型: Journal Article
    目的:本研究旨在使用18F-FDGPET/CT多参数定量分析来确定新辅助化疗对局部进展性胃癌患者的疗效。
    方法:我们对34例经病理证实接受新辅助化疗和手术的胃癌患者进行了回顾性分析。遵循化疗方案并进行18F-FDGPET/CT。我们确定了治疗前后靶病变的多参数,并确定了生物标志物变化百分比的理想临界值。采用二元logistic回归对独立因素进行评价。反应分类系统用于探索代谢和解剖反应与病理缓解程度之间的关联。
    结果:二元logistic回归分析显示,Lauren肠型和总病变糖酵解改变>45.2%是新辅助化疗疗效的风险预测因子;总病变糖酵解显示最佳预测效果。两模块反应(代谢和解剖反应)组的分类变量系统比单模块反应(代谢或解剖反应)组具有更高的预测准确性。
    结论:使用18F-FDGPET/CT多参数定量分析,Lauren肠型和总病变糖酵解改变>45.2%是胃腺癌患者新辅助化疗疗效的独立预测因子。此外,双模块评估显示出较高的预测效能.
    OBJECTIVE: This study aimed to use an 18F-FDG PET/CT multiparametric quantitative analysis to determine the efficacy of neoadjuvant chemotherapy in patients with locally progressive gastric cancer.
    METHODS: We conducted a retrospective analysis of 34 patients with pathologically identified gastric cancer who received neoadjuvant chemotherapy and surgery. Chemotherapy regimens were followed and 18F-FDG PET/CT was conducted. We ascertained multiparamaters of the target lesions pre- and post-treatment and determined the ideal cutoff values for the percentage change in biomarkers. Independent factors were evaluated using binary logistic regression. A response classification system was used to explore the association between metabolic and anatomical responses and the degree of pathological remission.
    RESULTS: Binary logistic regression analysis showed that Lauren bowel type and change in total lesion glycolysis >45.2% were risk predictors for the efficacy of neoadjuvant chemotherapy; total lesion glycolysis demonstrated the best predictive efficacy. The categorical variable system of the two-module response (metabolic and anatomical response) group had a higher predictive accuracy than that of the single-module response (metabolic or anatomical response) group.
    CONCLUSIONS: Using 18F-FDG PET/CT multiparametric quantitative analysis, Lauren bowel type and change in total lesion glycolysis >45.2% were independent predictors of the efficacy of neoadjuvant chemotherapy in patients with gastric adenocarcinoma. Additionally, the dual-module assessment demonstrated high predictive efficacy.
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  • 文章类型: Journal Article
    高尿酸血症(HUA),由嘌呤代谢紊乱引起的严重代谢疾病,会导致体内血清尿酸(SUA)水平异常升高。研究表明,HUA与痛风高度相关,高血压,糖尿病,冠心病,慢性肾脏疾病,等等。中药因其独特的多代谢产物、多靶点优势,在治疗HUA方面表现出优异的效果。本文报道了在建立HUA模型的基础上,使用中药成分进行降尿酸(UA)活性,具有优异的疗效和低副作用。这项工作总结了各种HUA疾病模型用于疗效评估的优点和局限性。对中医药在HUA治疗中的应用也进行了详细的讨论。本文揭示了HUA在构建评价模型和系统的中医干预方面的最新研究进展。这将为HUA模型的建立提供科学依据,并为未来中医药相关HUA研究提供建议。
    Hyperuricemia (HUA), a severe metabolic disease derived from purine metabolism disorder, will lead to abnormally increased serum uric acid (SUA) levels in the body. Studies have shown that HUA is highly related to gout, hypertension, diabetes, coronary heart disease, chronic kidney diseases, and so on. Traditional Chinese medicine (TCM) shows excellent results in treating HUA because of its unique advantages of multi-metabolites and multi-targets. This article reports on the use of TCM components for uric acid (UA)-lowering activity with excellent efficacy and low side effects based on established HUA models. This work summarizes the advantages and limitations of various HUA disease models for efficacy evaluation. Applications of TCM in HUA treatment have also been discussed in detail. This paper reveals recent research progress on HUA in constructing evaluation models and systematic TCM interventions. It will provide a scientific reference for establishing the HUA model and suggest future TCM-related HUA studies.
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