clinical value

临床价值
  • 文章类型: Journal Article
    在临床实践中,很少有有效的生物标志物用于识别非酒精性脂肪性肝病(NAFLD).本研究旨在探讨γ-谷氨酰转肽酶与血小板比值(GPR)联合甘油三酯(TG)对NAFLD的诊断价值。
    共有14,415人参加了年度体检。采用多因素logistic回归分析探讨与NAFLD相关的暴露因素。进行Spearman分析以评估NAFLD暴露因素之间的相关性。此外,采用受试者工作特征曲线(ROC)分析GPR与TG联合对NAFLD的诊断效能.
    多因素logistic回归分析结果显示,BMI(OR=1.619),收缩压(SBP)(OR=1.014),舒张压(DBP)(OR=1.028),探地雷达(OR=12.809),TG(OR=2.936)均为NAFLD的危险因素,HDL-C(OR=0.215)是保护因素。Spearman相关分析显示探地雷达与SBP呈显著正相关,DBP,BMI,TG(p<0.01),GPR与HDL-C呈负相关(p<0.01)。TG仅与GPR呈正相关(p<0.001)。ROC曲线分析表明,GPR联合TG诊断NAFLD的曲线下面积(AUC)为0.855(95%CI:0.819-0.891),敏感性为83.45%,特异性为73.56%。
    这项研究表明,高水平的GPR和TG是NAFLD的危险因素,并在诊断NAFLD中显示出良好的临床价值。
    UNASSIGNED: In clinical practice, there are few effective biomarkers for identifying non-alcoholic fatty liver disease (NAFLD). The aim of this study is to investigate the diagnostic value of γ-glutamyl transpeptidase to platelet ratio (GPR) combined with triglyceride (TG) in NAFLD.
    UNASSIGNED: A total of 14,415 individuals participated in the annual physical examination. Multivariate logistic regression analysis was conducted to investigate the exposure factors associated with NAFLD. Spearman\'s analysis was performed to assess the correlation among the exposure factors of NAFLD. Furthermore, the diagnostic efficacy of the combination of GPR and TG in NAFLD was analyzed using the receiver operating characteristic curve (ROC).
    UNASSIGNED: The results of the multivariate logistic regression analysis showed that BMI (OR = 1.619), Systolic Blood Pressure (SBP) (OR = 1.014), Diastolic Blood Pressure (DBP) (OR = 1.028), GPR (OR = 12.809), and TG (OR = 2.936) were all risk factors for NAFLD, while HDL-C (OR = 0.215) was a protective factor. Spearman correlation analysis revealed significant positive correlations between GPR and SBP, DBP, BMI, TG (p < 0.01), but a negative correlation between GPR and HDL-C (p < 0.01). TG was only positively correlated with GPR (p < 0.001). ROC curve analysis demonstrated that the area under the curve (AUC) of GPR combined with TG for diagnosis of NAFLD was 0.855 (95 % CI: 0.819-0.891), sensitivity was 83.45 % and specificity was 73.56 %.
    UNASSIGNED: This study indicated that high levels of GPR and TG were risk factors for NAFLD and demonstrated good clinical value in diagnosing NAFLD.
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  • 文章类型: Journal Article
    肝细胞癌(HCC)是一种常见的恶性消化道肿瘤。许多基因突变已被记录在肝癌,然而,这些突变的临床意义在很大程度上仍未被研究.这项研究的目的是确定在HCC中含有xin肌动蛋白结合重复2(XIRP2)突变的临床价值和生物学效应。使用来自癌症基因组图谱和国际癌症基因组联盟数据库的数据检查HCC的基因突变景观。通过KM图分析评估XIRP2突变的预后意义。使用TIDE算法和CCK-8实验研究了药物敏感性与XIRP2突变之间的关联。通过qRT-PCR评估XIRP2突变的生物学效应,蛋白质稳定性实验,和相关的生物实验。XIRP2突变是HCC中的高频突变之一。并与不良预后相关。与XIRP2野生型相比,在具有XIRP2突变的HCC组织中观察到总共72个差异表达基因(DEGs)。这些DEGs与离子代谢过程密切相关。XIRP2突变与氟达拉滨敏感性的改变有关,奥沙利铂,WEHI-539和LCL-161。CCK-8试验表明,携带XIRP2突变的HCC细胞对氟达拉滨和奥沙利铂的耐药性增加,但与XIRP2野生型的HCC细胞相比,对WEHI-539和LCL-161的敏感性增强。发现XIRP2突变对组织和细胞中XIRP2的mRNA水平没有影响,但它确实增强了XIRP2蛋白的稳定性。机械上,XIRP2的抑制通过锌离子升高和钙离子过载导致对奥沙利铂的敏感性显着增加。总之,XIRP2突变有可能作为预测HCC预后和药物敏感性的生物标志物,并可作为提高奥沙利铂疗效的治疗靶点.
    Hepatocellular carcinoma (HCC) is a prevalent malignant digestive tumor. Numerous genetic mutations have been documented in HCC, yet the clinical significance of these mutations remains largely unexplored. The objective of this study is to ascertain the clinical value and biological effects of xin actin binding repeat containing 2 (XIRP2) mutation in HCC. The gene mutation landscape of HCC was examined using data from the Cancer Genome Atlas and the International Cancer Genome Consortium databases. The prognostic significance of the XIRP2 mutation was assessed through KM plot analysis. The association between drug sensitivity and the XIRP2 mutation was investigated using the TIDE algorithm and CCK-8 experiments. The biological effects of the XIRP2 mutation were evaluated through qRT-PCR, protein stability experiments, and relevant biological experiments. The XIRP2 mutation is one of the high-frequency mutations in HCC, and is associated with poor prognosis. A total of 72 differentially expressed genes (DEGs) were observed in HCC tissues with the XIRP2 mutation as compared to those with the XIRP2 wildtype, and these DEGs were closely related to ion metabolic processes. The XIRP2 mutation was linked to alterations in the sensitivity of fludarabine, oxaliplatin, WEHI-539, and LCL-161. CCK-8 assays demonstrated that HCC cells carrying the XIRP2 mutation exhibited increased resistance to fludarabine and oxaliplatin, but enhanced sensitivity to WEHI-539 and LCL-161 as compared with those HCC cells with the XIRP2 wildtype. The XIRP2 mutation was found to have no impact on the mRNA levels of XIRP2 in tissues and cells, but it did enhance the stability of the XIRP2 protein. Mechanically, the inhibition of XIRP2 resulted in a significant increase in sensitivity to oxaliplatin through an elevation in zinc ions and a calcium ion overload. In conclusion, the XIRP2 mutation holds potential as a biomarker for predicting the prognosis and drug sensitivity of HCC and serves as a therapeutic target to enhance the efficacy of oxaliplatin.
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  • 文章类型: Journal Article
    背景:本研究的目的是探讨计算机断层扫描血管造影(CTA)在诊断主动脉瘤中的临床价值。
    方法:回顾性分析2017年4月至2020年4月南京医科大学第一附属医院放射科60例疑似主动脉瘤患者的影像学资料。CTA和数字减影血管造影(DSA)检查患者,收集CTA影像结果并与DSA结果进行比较。
    结果:DSA[98.33%(59/60)]和CTA[95.00%(57/60)]诊断主动脉瘤的准确性差异无统计学意义(P>0.05)。在图像特征的诊断上没有显着差异(例如,打字,site,形式)主动脉瘤(P>0.05)。
    结论:CTA可用于成功确认患者是否患有主动脉瘤;它产生具有高特异性的高质量图像,灵敏度,和准确性,可以在临床实践中推广。
    BACKGROUND: The aim of this study is to explore the clinical value of computed tomography angiography (CTA) in the diagnosis of aortic aneurysm.
    METHODS: The imaging data of 60 patients suspected of having aortic aneurysms who were examined in the Radiology Department of the First Affiliated Hospital of Nanjing Medical University from April 2017 to April 2020 were analyzed retrospectively. CTA and digital subtraction angiography (DSA) were used to examine the patients, and CTA image findings were collected and compared with DSA findings.
    RESULTS: There was no significant difference in the accuracy of diagnosing aortic aneurysms (P > 0.05) between DSA [98.33% (59/60)] and CTA [95.00% (57/60)]. There was no significant difference in the diagnosis of image features (e.g., typing, site, form) of aortic aneurysms (P > 0.05).
    CONCLUSIONS: CTA can be used to successfully confirm if patients suffer from an aortic aneurysm; it produces quality images with high specificity, sensitivity, and accuracy and can be promoted in clinical practice.
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  • 文章类型: Journal Article
    The Dragon Gate Stone Inscription prescriptions are the earliest surviving stone inscriptions of medical formulas in China, covering various departments such as internal medicine, surgery, gynecology, pediatrics and ophthalmology. By reviewing 28 moxibustion prescriptions recorded in the Dragon Gate Stone Inscriptions, the following application characteristics are summarized: moxibustion mainly treats acute diseases such as mania, deficiency-cold syndrome, and jaundice; in terms of point selection, specific points such as the thirteen ghost points, eight influential points, and front-mu points are used, emphasizing the extraordinary meridians such as governor vessel and conception vessel, as well as specific single acupoints with unique therapeutic effects; in clinical application, it follows the principle of treating according to syndrome differentiation, uses multiple acupoints simultaneously, employs food-medicine homology, and adjusts the moxibustion dosage according to individual conditions. The Dragon Gate Stone Inscription prescriptions reflect that the application of moxibustion therapy during the Northern Wei to Tang Dynasty period had already reached a relatively mature level, indicating a high level of proficiency in moxibustion techniques during that time.
    龙门石刻药方是我国目前现存最早的石刻药方,其所治病证涉及内、外、妇、儿、五官等科,通过梳理龙门石刻药方所载的28首灸方,总结具有以下应用特点:癫狂病证、虚寒病证以及黄疸等急性病证是其主治病证;选穴方面,针灸并用十三鬼穴、八会穴、募穴等特定穴位,重视督脉、任脉等奇经以及具有独特主治作用的单穴;临床应用遵循辨证施治原则,多穴并用、使用药食同源之品、灸量上因人制宜施灸。龙门石刻药方反映了北魏至唐这一时期对灸法的应用已经相对成熟,灸法已经有了较高的应用水平。.
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  • 文章类型: Journal Article
    通过术前定位,外科医生可以轻松定位磨玻璃结节(GGNs)并有效控制切除范围。因此,有必要选择合适的穿刺定位方法。目的评价医用胶和定位钩在GGNs术前定位中的有效性和安全性,为临床选择提供参考。
    从2020年3月30日至2022年6月13日,共有859例CT诊断为需要手术切除的GGNs的患者被纳入我们医院的研究。其中,排除了21例因各种原因选择退出或无法进行术前定位的患者。此外,还排除了475例使用医用胶进行术前定位的患者和363例通过定位钩进行术前定位的患者。我们对基线数据进行了统计分析,成功率,并发症,其余患者的病理结果。成功率,并发症发生率,比较两组的病理结果-接受医用胶定位的患者和接受定位钩定位的患者。
    两组患者在年龄方面无统计学差异,身体质量指数,吸烟史,结节的位置,结节与胸膜的距离,或术后病理结果(P>0.05)。医用胶和定位钩的成功率为100%。单结节定位过程中医用胶和定位钩的并发症发生率分别为39.18%和23.18%,分别,差异有统计学意义(p<0.001);多结节定位的并发症发生率分别为49.15%和49.18%,分别,差异无统计学意义(p>0.05)。此外,定位方法和患者的临床特征未发现是发生并发症的独立危险因素。在COVID-19流行的2020-2022年期间,肺结节的检出率也与COVID-19的传播呈正相关。
    定位单个节点时,定位钩的安全性大于定位多个节点时,医用胶和定位钩的安全性相当,应根据患者的个体情况选择合适的定位方法。
    UNASSIGNED: Through preoperative localization, surgeons can easily locate ground glass nodules (GGNs) and effectively control the extent of resection. Therefore, it is necessary to choose an appropriate puncture positioning method. The purpose of this study was to evaluate the effectiveness and safety of medical glue and positioning hooks in the preoperative positioning of GGNs and to provide a reference for clinical selection.
    UNASSIGNED: From March 30, 2020 to June 13, 2022, a total of 859 patients with a CT diagnosis of GGNs requiring surgical resection were included in our study at the hospital. Among them, 21 patients who either opted out or could not undergo preoperative localization for various reasons were excluded. Additionally, 475 patients who underwent preoperative localization using medical glue and 363 patients who underwent preoperative localization through positioning hooks were also excluded. We conducted statistical analyses on the baseline data, success rates, complications, and pathological results of the remaining patients. The success rates, complication rates, and pathological results were compared between the two groups-those who received medical glue localization and those who received positioning hook localization.
    UNASSIGNED: There was no statistically significant difference between the two groups of patients in terms of age, body mass index, smoking history, location of the nodule, distance of the nodule from the pleura, or postoperative pathological results (P > 0.05). The success rate of medical glue and positioning hooks was 100%. The complication rates of medical glue and positioning hooks during single nodule positioning were 39.18% and 23.18%, respectively, which were significantly different (p < 0.001); the complication rates during multiple nodule positioning were 49.15% and 49.18%, respectively, with no statistically significant differences (p > 0.05). In addition, the method of positioning and the clinical characteristics of the patients were not found to be independent risk factors for the occurrence of complications. The detection rate of pulmonary nodules also showed some positive correlation with the spread of COVID-19 during the 2020-2022 period when COVID-19 was prevalent.
    UNASSIGNED: When positioning a single node, the safety of positioning hooks is greater than when positioning multiple nodes, the safety of medical glue and positioning hooks is comparable, and the appropriate positioning method should be chosen according to the individual situation of the patient.
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  • 文章类型: Journal Article
    背景:区分不同类型的胸腔积液(PE)对于临床诊断和治疗至关重要。这项研究评估了癌胚抗原(CEA)和干扰素-γ(IFN-γ)水平在PE和血清中的诊断价值。以及这些标志物的PE/血清比率,在对PE进行分类时。
    方法:我们回顾性分析了99例PE患者,将它们归类为恶性胸腔积液(MPE),结核性胸腔积液(TPE),良性PE组。定量PE和血清中的CEA和IFN-γ水平并计算它们的比率。使用接收器工作特性分析评估诊断性能,关注曲线下面积(AUC)以确定这些生物标志物的功效。
    结果:与良性和TPE组相比,MPE组PE和血清中的CEA水平明显升高,PE/血清CEA比率提供了实质性的诊断价值(AUC:PE=0.843,血清=0.744)。相反,IFN-γ在PE和血清中的水平明显高于TPE组,表现出显著的诊断准确性(AUCs:PE=0.970,血清=0.917)。
    结论:CEA和IFN-γ在区分MPE和TPE方面均显示出很高的临床实用性。这些生物标志物的PE/血清比率提高了诊断的准确性,可能有助于更早和更准确的治疗干预。
    BACKGROUND: Distinguishing between different types of pleural effusions (PEs) is crucial for clinical diagnosis and treatment. This study evaluates the diagnostic value of carcinoembryonic antigen (CEA) and interferon-gamma (IFN-γ) levels in PE and serum, as well as the PE/serum ratios of these markers, in classifying PE.
    METHODS: We retrospectively analyzed 99 patients with PE, categorizing them into malignant pleural effusion (MPE), tuberculous pleural effusion (TPE), and benign PE groups. Levels of CEA and IFN-γ in PE and serum were quantified and their ratios were calculated. Diagnostic performance was assessed using receiver operating characteristic analysis, focusing on the area under the curve (AUC) to determine the efficacy of these biomarkers.
    RESULTS: Significantly elevated levels of CEA in PE and serum were observed in the MPE group compared to the benign and TPE groups, with the PE/serum CEA ratio offering substantial diagnostic value (AUCs: PE = 0.843, serum = 0.744). Conversely, IFN-γ levels in PE and serum were markedly higher in the TPE group, demonstrating notable diagnostic accuracy (AUCs: PE = 0.970, serum = 0.917).
    CONCLUSIONS: Both CEA and IFN-γ demonstrate high clinical utility in differentiating between MPE and TPE. The PE/serum ratio of these biomarkers enhances diagnostic accuracy, potentially facilitating earlier and more accurate therapeutic interventions.
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  • 文章类型: Journal Article
    循环肿瘤DNA(ctDNA)和无细胞DNA(cfDNA)在胰腺恶性肿瘤中的诊断和预后临床价值尚不清楚。在这里,我们旨在进行meta分析,以评估ctDNA和cfDNA作为潜在的诊断和预后生物标志物.
    遵循PRISMA报告指南进行当前的荟萃分析。ThePubMed/Medline,Scopus,和WebofScience(WoS)数据库进行了详细扫描,以确定该研究的合格论文。根据REMARK标准进行质量评估。在所有疾病阶段中,ctDNA与碳水化合物抗原19.9(CA19.9)的诊断准确性的风险比(RR)以及ctDNA在总体生存(OS)中的预后作用的风险比(HRs)用95%置信区间(CI)计算。
    总共评估了18篇论文,以评估与胰腺恶性肿瘤相关的生物标志物的诊断准确性和预后价值。汇总分析表明,CA19.9在所有疾病阶段的诊断准确性均高于ctDNA或cfDNA(RR=0.64,95%CI:0.50-0.82,p<0.001)。此外,在关注预后的次要分析中,ctDNA阳性患者的OS显著降低(HR=2.00,95%CI:1.51-2.66,p<0.001).
    这项荟萃分析的结果表明,在所有疾病阶段,CA19-9仍然比ctDNA或cfDNA中的KRAS突变具有更高的诊断准确性。尽管如此,ctDNA检测水平的存在与OS方面较差的患者结局相关.越来越需要对这一主题进行进一步的研究。
    https://doi.org/10.37766/inplasy2023.12.0092,标识符INPLASY2023120092。
    UNASSIGNED: The diagnostic and prognostic clinical value of circulating tumor DNA (ctDNA) and cell-free DNA (cfDNA) in pancreatic malignancies are unclear. Herein, we aimed to perform a meta-analysis to evaluate ctDNA and cfDNA as potential diagnostic and prognostic biomarkers.
    UNASSIGNED: PRISMA reporting guidelines were followed closely for conducting the current meta-analysis. The PubMed/Medline, Scopus, and Web of Science (WoS) databases were scanned in detail to identify eligible papers for the study. A quality assessment was performed in accordance with the REMARK criteria. The risk ratios (RRs) of the diagnostic accuracy of ctDNA compared to that of carbohydrate antigen 19.9 (CA 19.9) in all disease stages and the hazard ratios (HRs) of the prognostic role of ctDNA in overall survival (OS) were calculated with 95% confidence intervals (CIs).
    UNASSIGNED: A total of 18 papers were evaluated to assess the diagnostic accuracy and prognostic value of biomarkers related to pancreatic malignancies. The pooled analysis indicated that CA19.9 provides greater diagnostic accuracy across all disease stages than ctDNA or cfDNA (RR = 0.64, 95% CI: 0.50-0.82, p < 0.001). Additionally, in a secondary analysis focusing on prognosis, patients who were ctDNA-positive were found to have significantly worse OS (HR = 2.00, 95% CI: 1.51-2.66, p < 0.001).
    UNASSIGNED: The findings of this meta-analysis demonstrated that CA19-9 still has greater diagnostic accuracy across all disease stages than KRAS mutations in ctDNA or cfDNA. Nonetheless, the presence of detectable levels of ctDNA was associated with worse patient outcomes regarding OS. There is a growing need for further research on this topic.
    UNASSIGNED: https://doi.org/10.37766/inplasy2023.12.0092, identifier INPLASY2023120092.
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  • 文章类型: Journal Article
    抗癌药物的高价格引起了人们的关注,因为它们对患者和医疗保健系统的财务影响。本研究旨在评估在中国报销的抗癌药的初始和最新标价和临床价值,Japan,和韩国。
    我们确定了2012年1月至2022年6月由中国国家药品监督管理局新批准的抗癌药物,并由日本药品和医疗器械局和韩国食品和药品安全部批准,直至2022年6月。我们比较了各国的初始和最新治疗价格,并使用患者的生存率评估了临床价值,生活质量(QoL),和欧洲医学肿瘤学会临床获益量表(ESMO-MCBS)。我们计算了各个国家的治疗价格与临床价值的Spearman等级相关系数,并采用回归分析来调查价格与临床价值之间的关系是否因国家环境而改变。
    我们的队列包括91种抗癌药物适应症,在中国有60人被列入报销名单,91在日本,87在韩国。日本的治疗价格中位数最高,其次是韩国,在中国最低,两者均为初始价格(64082美元对US$45529vs.19144美元,p<0.0001)和最新价格(50859美元与US$31611vs.18666美元,p<0.0001)。随着时间的推移,与日本(β=-0.013,p=0.011)相比,中国(β=-0.047,p<0.0001)和韩国(β=-0.049,p<0.0001)的价格下降幅度更大。与日本相比,中国和韩国的初始和最新治疗价格与临床价值(QoL和ESMO-MCBS)之间的相关性更为显着和更强,尽管与中国和韩国相比,日本在生存率方面表现出更强的相关性。临床价值和治疗价格之间的关系可能不会因国家设定而改变。
    相比之下,韩国的标价及其与临床价值的相关性似乎是合理的。日本的政策制定者可以通过控制价格并使价格与临床价值保持一致来提高效率,而中国将需要采取实质性措施来扩大抗癌药物的覆盖范围。
    国家自然科学基金(72374149和72074163),和中国南亚研究中心,四川大学。
    UNASSIGNED: High prices of anticancer drugs have raised concerns due to their financial impact on patients and healthcare systems. This study aimed to assess the initial and latest list prices and clinical value of reimbursed anticancer drugs in China, Japan, and South Korea.
    UNASSIGNED: We identified anticancer drugs newly approved by the National Medical Products Administration of China from January 2012 to June 2022, and by the Pharmaceuticals and Medical Devices Agency of Japan and the Ministry of Food and Drug Safety of South Korea up until June 2022. We compared initial and latest treatment prices between countries and assessed clinical value using patients\' survival, quality of life (QoL), and European Society for Medical Oncology Magnitude of Clinical Benefit Scale (ESMO-MCBS). We calculated Spearman rank correlation coefficients of treatment prices with clinical value for individual countries and employed regression analyses to investigate whether the relationship between prices and clinical value was modified by the country setting.
    UNASSIGNED: Our cohort included 91 anticancer drug indications, with 60 listed for reimbursement in China, 91 in Japan, and 87 in South Korea. Median treatment prices were highest in Japan, followed by South Korea, and lowest in China, both for initial prices (US$64082 vs. US$45529 vs. US$19144, p < 0.0001) and latest prices (US$50859 vs. US$31611 vs. US$18666, p < 0.0001). Over time, China (β = -0.047, p < 0.0001) and South Korea (β = -0.049, p < 0.0001) witnessed more significant price reductions compared to Japan (β = -0.013, p = 0.011). The correlations between both initial and latest treatment prices and clinical value (QoL and ESMO-MCBS) were more significant and stronger in China and South Korea than in Japan, although Japan exhibited slightly stronger correlations in terms of survival compared to China and South Korea. The relationship between clinical value and treatment prices may not be modified by the country setting.
    UNASSIGNED: In comparison, South Korea\'s list prices and their correlations with clinical value appear reasonable. Policymakers in Japan could enhance efficiency by controlling prices and aligning them with clinical value, while China would need to take substantial steps to expand anticancer drug coverage.
    UNASSIGNED: National Natural Science Foundation of China (72374149 and 72074163), and China Center for South Asian Studies, Sichuan University.
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  • 文章类型: Journal Article
    背景:在中国实施与报销挂钩的价格谈判后,研究抗癌药物的上市价格和临床价值与报销决策的关系。
    方法:2017年1月至2022年6月中国国家药监局批准的抗癌药物符合纳入条件。所包括的药物适应症的批准和报销日期是从公开可用的资源中检索的。我们收集了临床价值的测量,包括生存,生活质量,以及关键临床试验的总体反应率和启动时计算的治疗价格。单变量和多变量Cox比例风险模型被用来估计发射价格之间的关联,临床价值,和中国抗癌药物的报销决定。
    结果:对于随机对照试验支持的93种适应症,中位补偿滞后为579天(IQR:402-936),对于单组临床试验支持的42种适应症,中位补偿滞后为637天(IQR373-858)。60(65%)和23(55%)的适应症得到了随机对照和单臂临床试验的支持,分别。在多元回归分析中,抗癌药物的上市价格与报销决定无关。临床价值较高的随机对照试验支持的适应症更有可能获得报销(生存率HR=1.07,95CI:1.00-1.15,p=0.037),而单组临床试验支持的适应症的总体缓解率与获得报销的可能性无关(HR=2.09,95CI:0.14~32.28,p=0.595).
    结论:抗癌药的上市价格可能不会对报销决定产生重大影响,虽然在中国实施与报销挂钩的价格谈判,但优先考虑了具有较高临床价值的抗癌药物,但仅限于随机对照试验支持的适应症。需要努力优先考虑在价格谈判过程中具有更高价值的单臂临床试验支持的适应症。
    BACKGROUND: The potential role played by launch price and clinical value in reimbursement decisions has not been sufficiently established in China. This study aimed to investigate the association of launch price and clinical value with reimbursement decisions for anticancer drugs after the implementation of reimbursement-linked price negotiation in China.
    METHODS: Anticancer drugs approved by the National Medical Products Administration (NMPA) of China from January 2017 to June 2022 were eligible for inclusion. Approval and reimbursement dates of included drug indications were retrieved from publicly available resources. We collected measures of clinical value, including survival, quality of life (QoL), and overall response rate from pivotal clinical trials and calculated treatment price at launch. Univariate and multivariate Cox proportional hazards models were employed to estimate the association between launch price, clinical value, and reimbursement decisions of anticancer drugs in China.
    RESULTS: The median reimbursement lag was 579 days (interquartile range [IQR]: 402-936) for 93 indications supported by randomized controlled trials and 637 days (IQR: 373-858) for 42 indications supported by single-arm clinical trials. Reimbursement was granted to 60 (65%) and 23 (55%) indications supported by randomized controlled and single-arm clinical trials, respectively. The launch price of anticancer drugs was not associated with reimbursement decisions in multivariate regression analyses. Indications supported by randomized controlled trials with higher clinical value were more likely to be reimbursed (hazard ratio [HR] for survival=1.07, 95% CI: 1.00-1.15, P=.037), while the overall response rate of indications supported by single-arm clinical trials was not associated with the likelihood of being reimbursed (HR=2.09, 95% CI: 0.14-32.28, P=.595).
    CONCLUSIONS: The launch price of anticancer drugs may not have a significant impact on reimbursement decisions, while the implementation of reimbursement-linked price negotiation in China has prioritized anticancer drugs with higher clinical value, but only for indications supported by randomized controlled trials. Efforts are needed to prioritize indications supported by single-arm clinical trials that have higher value during the process of price negotiation.
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  • 文章类型: Journal Article
    背景:开发了BRENDA评分,并将其用于预测乳腺癌(BC)患者的预后。进行这项研究是为了验证该工具在中国原发性侵袭性BC患者中的使用。
    方法:2009年1月至2016年12月接受BC手术的患者。通过受试者工作特征(ROC)曲线(AUC)下面积评估鉴别。通过比较总体队列和患者亚组的预测和观察到的5年和10年无转移生存期(MFS)来评估校准。
    结果:共纳入2029例BC患者。Kaplan-Meier分析显示MFS风险组存在显著差异(对数秩检验P<0.01)。ROC分析显示5年MFS(AUC0.779)具有良好的准确性,10年MFS(AUC0.728)具有相当的准确性。BRENDA-Score准确预测了整个队列和所有其他预定义子组中的5年和10年MFS,除了年龄<40岁的亚组的5年MFS,这是高估的(预测和观察到的MFS之间的差异为6.7%,P<0.05)。ER阳性和ER阴性患者的5年MFS率分别为90.9%和80.6%,分别为(P<0.05)。ER阳性和ER阴性患者的10年MFS率分别为78.0%和73.7%,分别(P=.25)。
    结论:BRENDA评分准确预测了5年和10年的MFS。结果具有良好的有效性,可运输性,和潜在的临床价值。然而,对于年龄<40岁的患者,应仔细解读5年MFS的结果.10年后,ER作为预后因素的价值不那么重要。
    BACKGROUND: The BRENDA-Score was developed and used to predict the prognosis of patients with breast cancer (BC). This study was performed to validate the use of this tool in Chinese patients with primary invasive BC patients.
    METHODS: Patients underwent surgery for BC from January 2009 to December 2016. Discrimination was assessed by the area under the receiver operating characteristic (ROC) curve (AUC). Calibrations were assessed by comparing predicted and observed 5-year and 10-year metastasis-free survival (MFS) in the overall cohort and patient subgroups.
    RESULTS: A total of 2029 BC patients were enrolled. Kaplan-Meier analysis revealed significant differences in MFS risk groups (log-rank test P < .01). ROC analysis showed good accuracy for 5-year MFS (AUC 0.779) and fair accuracy for 10-year MFS (AUC 0.728). The BRENDA-Score accurately predicted 5-year and 10-year MFS in the entire cohort and in all other predefined subgroups, except for the 5-year MFS in the subgroup aged<40 years, which was overestimated (differences between the predicted and observed MFS were 6.7%, P < .05). The 5-year MFS rates of ER- positive and ER-negative patients were 90.9% and 80.6%, respectively (P < .05). The 10-year MFS rates of ER-positive and ER-negative patients were 78.0% and 73.7%, respectively (P = .25).
    CONCLUSIONS: The BRENDA-Score accurately predicted 5-year and 10-year MFS. The results showed good validity, transportability, and potential clinical value. However, the results for 5-years MFS should be interpreted carefully in patients aged <40 years. After 10 years the value of the ER as a prognostic factor was less important.
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