Outcome prediction

结果预测
  • 文章类型: Journal Article
    小儿霍奇金和非霍奇金淋巴瘤在生物学和治疗上与成人病例不同,然而,缺乏针对小儿淋巴瘤的生存分析。我们分析了1975年至2018年的淋巴瘤数据,比较了7,871名儿童患者和226,211名成人患者的生存趋势。确定了儿童淋巴瘤生存的关键危险因素,开发了一个预测列线图,并利用机器学习来预测长期淋巴瘤特异性死亡风险。在1975年至2018年期间,我们观察到1年内大幅增长(19.3%),5年期(41.9%),儿科淋巴瘤患者的10年总生存率(48.8%)。预后因素,如年龄,性别,种族,安阿伯舞台,淋巴瘤亚型,和放疗被纳入列线图。列线图表现出出色的预测性能,一年的曲线下面积(AUC)值为0.766、0.724和0.703,五年,十年的生存,分别,在训练组中,验证队列中的AUC值为0.776、0.712和0.696。重要的是,列线图在生存预测方面优于AnnArbor分期系统。机器学习模型在预测淋巴瘤特异性死亡风险方面实现了约0.75的AUC值,超过了常规方法(AUC=〜0.70)。我们还观察到儿科淋巴瘤幸存者在10年后患淋巴瘤的风险大大降低。UT面临非淋巴瘤疾病的风险越来越大。该研究强调了小儿淋巴瘤生存率的实质性改善,提供可靠的预测工具,并强调了长期监测儿科患者非淋巴瘤健康问题的重要性.
    Pediatric Hodgkin and non-Hodgkin lymphomas differ from adult cases in biology and management, yet there is a lack of survival analysis tailored to pediatric lymphoma. We analyzed lymphoma data from 1975 to 2018, comparing survival trends between 7,871 pediatric and 226,211 adult patients, identified key risk factors for pediatric lymphoma survival, developed a predictive nomogram, and utilized machine learning to predict long-term lymphoma-specific mortality risk. Between 1975 and 2018, we observed substantial increases in 1-year (19.3%), 5-year (41.9%), and 10-year (48.8%) overall survival rates in pediatric patients with lymphoma. Prognostic factors such as age, sex, race, Ann Arbor stage, lymphoma subtypes, and radiotherapy were incorporated into the nomogram. The nomogram exhibited excellent predictive performance with area under the curve (AUC) values of 0.766, 0.724, and 0.703 for one-year, five-year, and ten-year survival, respectively, in the training cohort, and AUC values of 0.776, 0.712, and 0.696 in the validation cohort. Importantly, the nomogram outperformed the Ann Arbor staging system in survival prediction. Machine learning models achieved AUC values of approximately 0.75, surpassing the conventional method (AUC =  ~ 0.70) in predicting the risk of lymphoma-specific death. We also observed that pediatric lymphoma survivors had a substantially reduced risk of lymphoma after ten years b,ut faced an increasing risk of non-lymphoma diseases. The study highlights substantial improvements in pediatric lymphoma survival, offers reliable predictive tools, and underscores the importance of long-term monitoring for non-lymphoma health issues in pediatric patients.
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  • 文章类型: Journal Article
    这项研究提出了一种结局预测方法,以提高基于全脑特征多样性的缺血性卒中结局预测的准确性和有效性,不使用患者基本信息和病变图像特征。
    在这项研究中,我们直接从动态磁化率对比灌注加权成像(DSC-PWI)中提取动态影像组学特征(DRF),并进一步从最小强度投影(MinIP)图中提取静态影像组学特征(SRF)和静态编码特征(SEF),这是从DSC-PWI图像的时间维度生成的。在从DRF的组合中选择整个大脑特征之后,SRF,和SEF通过Lasso算法,各种机器和深度学习模型被用于评估Ffuse在预测卒中结局中的作用.
    实验结果表明,从DRF产生的特征Ffuse,SRF,SEF(Resnet18)优于其他单一和组合特征,在机器学习模型和深度学习模型上都取得了0.971的最佳平均得分,95%CI分别为(0.703,0.877)和(0.92,0.983)。分别。此外,深度学习模型通常比机器学习模型表现更好。
    我们研究中使用的方法可以在不分割缺血性病变的情况下实现对卒中结果的准确评估,这对快速,高效,和准确的临床中风治疗。
    UNASSIGNED: This study proposed an outcome prediction method to improve the accuracy and efficacy of ischemic stroke outcome prediction based on the diversity of whole brain features, without using basic information about patients and image features in lesions.
    UNASSIGNED: In this study, we directly extracted dynamic radiomics features (DRFs) from dynamic susceptibility contrast perfusion-weighted imaging (DSC-PWI) and further extracted static radiomics features (SRFs) and static encoding features (SEFs) from the minimum intensity projection (MinIP) map, which was generated from the time dimension of DSC-PWI images. After selecting whole brain features Ffuse from the combinations of DRFs, SRFs, and SEFs by the Lasso algorithm, various machine and deep learning models were used to evaluate the role of Ffuse in predicting stroke outcomes.
    UNASSIGNED: The experimental results show that the feature Ffuse generated from DRFs, SRFs, and SEFs (Resnet 18) outperformed other single and combination features and achieved the best mean score of 0.971 both on machine learning models and deep learning models and the 95% CI were (0.703, 0.877) and (0.92, 0.983), respectively. Besides, the deep learning models generally performed better than the machine learning models.
    UNASSIGNED: The method used in our study can achieve an accurate assessment of stroke outcomes without segmentation of ischemic lesions, which is of great significance for rapid, efficient, and accurate clinical stroke treatment.
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  • 文章类型: Journal Article
    散发性脑海绵状畸形(CCM)患者的(再)出血是CCM管理的主要目的。然而,提前准确识别散发性CCM患者的潜在(再)出血仍然是一个挑战。这项研究旨在开发机器学习模型,以检测散发性CCM患者的潜在(再)出血。本研究基于开放数据平台Dryad中731名零星CCM患者的数据集。2003年1月至2018年12月对散发性CCM患者进行5年随访。支持向量机(SVM)堆叠概括,和极端梯度提升(XGBoost)用于构建模型。通过受试者工作特征曲线下面积(AUROC)评估模型的性能,精确率-召回率曲线下面积(PR-AUC)和其他指标。共纳入517例散发性CCM患者(330例女性[63.8%],诊断时的平均[SD]年龄,42.1[15.5]年)。随访期间发生76例(再)出血(14.7%)。在3种机器学习模型中,XGBoost模型在交叉验证中产生最高平均值(SD)AUROC(0.87[0.06])。XGBoost模型的前4个特征以SHAP(Shapley添加剂扩张)排名。All-ElementsXGBoost模型在测试集中实现了0.84的AUROC和0.49的PR-AUC,灵敏度为0.86,特异性为0.76。重要的是,使用前4个特征开发的4元素XGBoost模型在测试集中获得0.83的AUROC和0.40的PR-AUC,0.79的灵敏度和0.72的特异性。两个基于机器学习的模型在识别散发性CCM患者5年内的潜在(再)出血方面实现了准确的性能。这些模型可以为临床决策提供见解。
    The (re)hemorrhage in patients with sporadic cerebral cavernous malformations (CCM) was the primary aim for CCM management. However, accurately identifying the potential (re)hemorrhage among sporadic CCM patients in advance remains a challenge. This study aims to develop machine learning models to detect potential (re)hemorrhage in sporadic CCM patients. This study was based on a dataset of 731 sporadic CCM patients in open data platform Dryad. Sporadic CCM patients were followed up 5 years from January 2003 to December 2018. Support vector machine (SVM), stacked generalization, and extreme gradient boosting (XGBoost) were used to construct models. The performance of models was evaluated by area under receiver operating characteristic curves (AUROC), area under the precision-recall curve (PR-AUC) and other metrics. A total of 517 patients with sporadic CCM were included (330 female [63.8%], mean [SD] age at diagnosis, 42.1 [15.5] years). 76 (re)hemorrhage (14.7%) occurred during follow-up. Among 3 machine learning models, XGBoost model yielded the highest mean (SD) AUROC (0.87 [0.06]) in cross-validation. The top 4 features of XGBoost model were ranked with SHAP (SHapley Additive exPlanations). All-Elements XGBoost model achieved an AUROCs of 0.84 and PR-AUC of 0.49 in testing set, with a sensitivity of 0.86 and a specificity of 0.76. Importantly, 4-Elements XGBoost model developed using top 4 features got a AUROCs of 0.83 and PR-AUC of 0.40, a sensitivity of 0.79, and a specificity of 0.72 in testing set. Two machine learning-based models achieved accurate performance in identifying potential (re)hemorrhages within 5 years in sporadic CCM patients. These models may provide insights for clinical decision-making.
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  • 文章类型: Journal Article
    嵌合抗原受体(CAR)T细胞疗法在治疗淋巴瘤方面取得了很大进展,然而,患者的结果仍然有很大差异。淋巴瘤微环境可能是影响CAR-T治疗效果的重要因素。在这项研究中,我们设计了一个高度多重成像质量细胞计数(IMC)组,以同时量化13例接受CAR19/22T细胞治疗的复发/难治性弥漫性大B细胞淋巴瘤(DLBCL)患者的31种生物标志物.在CART细胞输注之前或在输注后复发时取样总共20个切片。共鉴定出35个细胞簇,注释,随后重新定义为10个元显示器。CD4+T细胞分数与缓解时间呈正相关。T细胞中Ki67、CD57和TIM3水平显著升高,CD69水平显著降低,特别是CD8+/CD4+Tem和Te细胞亚群,在预后不良的患者中观察到。含有更多免疫细胞的细胞邻域与更长的缓解相关。在CAR-T治疗后反应差、缓解时间短的患者中,成纤维细胞和血管内皮细胞更接近肿瘤细胞。我们的工作全面系统地解剖了细胞组成之间的关系,state,DLBCL微环境的空间排列和CAR-T细胞治疗的结果,这有利于预测CAR-T疗法的疗效。
    Chimeric antigen receptor (CAR) T cell therapy has made great progress in treating lymphoma, yet patient outcomes still vary greatly. The lymphoma microenvironment may be an important factor in the efficacy of CAR T therapy. In this study, we designed a highly multiplexed imaging mass cytometry (IMC) panel to simultaneously quantify 31 biomarkers from 13 patients with relapsed/refractory diffuse large B cell lymphoma (DLBCL) who received CAR19/22 T cell therapy. A total of 20 sections were sampled before CAR T cell infusion or after infusion when relapse occurred. A total of 35 cell clusters were identified, annotated, and subsequently redefined into 10 metaclusters. The CD4+ T cell fraction was positively associated with remission duration. Significantly higher Ki67, CD57, and TIM3 levels and lower CD69 levels in T cells, especially the CD8+/CD4+ Tem and Te cell subsets, were seen in patients with poor outcomes. Cellular neighborhood containing more immune cells was associated with longer remission. Fibroblasts and vascular endothelial cells resided much closer to tumor cells in patients with poor response and short remission after CAR T therapy. Our work comprehensively and systematically dissects the relationship between cell composition, state, and spatial arrangement in the DLBCL microenvironment and the outcomes of CAR T cell therapy, which is beneficial to predict CAR T therapy efficacy.
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  • 文章类型: Journal Article
    背景:叉头盒蛋白P1(FOXP1)被认为具有致癌和肿瘤抑制特性,取决于肿瘤的异质性。然而,FOXP1在肝内胆管癌(ICC)中的作用尚未见报道.
    方法:免疫组化法检测FOXP1在ICC和正常肝组织中的表达。评价FOXP1水平与ICC患者临床病理特征的关系。最后,进行了体外和体内实验以检查FOXP1在ICC细胞中的调节作用。
    结果:与肿瘤周围组织相比,FOXP1在ICC中显著下调(p<0.01)。分化差的患者FOXP1阳性率明显降低,淋巴结转移,侵入周围器官,和晚期(p<0.05)。值得注意的是,FOXP1阳性患者的预后(总生存期)优于FOXP1阴性患者(p<0.05),正如Kaplan-Meier生存分析所揭示的。此外,Cox多因素分析显示,FOXP1表达阴性,先进的TNM阶段,入侵,淋巴结转移是ICC患者预后的独立危险因素。最后,过表达FOXP1抑制细胞增殖,迁移,和ICC细胞的侵袭和促进凋亡,而FOXP1的敲除具有相反的作用。
    结论:我们的研究结果表明,FOXP1可能是ICC的一种新的预后预测因子,也可能是一种有助于癌症治疗的肿瘤抑制因子。
    BACKGROUND: Forkhead-box protein P1 (FOXP1) has been proposed to have both oncogenic and tumor-suppressive properties, depending on tumor heterogeneity. However, the role of FOXP1 in intrahepatic cholangiocarcinoma (ICC) has not been previously reported.
    METHODS: Immunohistochemistry was performed to detect FOXP1 expression in ICC and normal liver tissues. The relationship between FOXP1 levels and the clinicopathological characteristics of patients with ICC was evaluated. Finally, in vitro and in vivo experiments were conducted to examine the regulatory role of FOXP1 in ICC cells.
    RESULTS: FOXP1 was significantly downregulated in the ICC compared to their peritumoral tissues (p < 0.01). The positive rates of FOXP1 were significantly lower in patients with poor differentiation, lymph node metastasis, invasion into surrounding organs, and advanced stages (p < 0.05). Notably, patients with FOXP1 positivity had better outcomes (overall survival) than those with FOXP1 negativity (p < 0.05), as revealed by Kaplan-Meier survival analysis. Moreover, Cox multivariate analysis showed that negative FOXP1 expression, advanced TNM stages, invasion, and lymph node metastasis were independent prognostic risk factors in patients with ICC. Lastly, overexpression of FOXP1 inhibited the proliferation, migration, and invasion of ICC cells and promoted apoptosis, whereas knockdown of FOXP1 had the opposite role.
    CONCLUSIONS: Our findings suggest that FOXP1 may serve as a novel outcome predictor for ICC as well as a tumor suppressor that may contribute to cancer treatment.
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  • 文章类型: Journal Article
    结果预测对于危重患者的管理和治疗至关重要。对那些病人来说,持续监测临床测量结果,且随时间变化的数据包含用于评估患者状态的丰富信息.然而,目前尚不清楚如何有效地捕获动态信息。在这项工作中,多种特征提取方法,即统计特征分类方法和时态建模方法,如递归神经网络(RNN),在18415例重症疾病数据集上进行了分析。实验结果表明,当维数从10增加到50时,RNN算法逐渐优于逻辑简单的统计特征分类方法。RNN模型实现了最大的AUC值0.8463。因此,时间建模方法有望捕获预测患者预后的时间特征,并且可以在更多的临床应用中扩展。
    Outcome prediction is essential for the administration and treatment of critically ill patients. For those patients, clinical measurements are continuously monitored and the time-varying data contains rich information for assessing the patients\' status. However, it is unclear how to capture the dynamic information effectively. In this work, multiple feature extraction methods, i.e. statistical feature classification methods and temporal modeling methods, such as recurrent neural network (RNN), were analyzed on a critical illness dataset with 18415 cases. The experimental results show when the dimension increases from 10 to 50, the RNN algorithm is gradually superior to the statistical feature classification methods with simple logic. The RNN model achieves the largest AUC value of 0.8463. Therefore, the temporal modeling methods are promising to capture temporal features which are predictive of the patients\' outcome and can be extended in more clinical applications.
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  • 文章类型: Journal Article
    背景:关于基于高级机器学习(ML)算法的预后工具在动脉瘤性蛛网膜下腔出血(aSAH)患者中的优越性存在争议。不清楚,ML预后模型是否会使患者受益,由于缺乏全面的评估。我们旨在开发和评估预测aSAH患者不良功能结局的ML模型,并确定具有最大性能的模型。
    方法:在这项回顾性研究中,在3~6个月的随访期间,我们利用955例aSAH患者的数据集构建并验证通过改良Rankin量表评估的功能结局的预后模型.临床评分和入院时的临床和放射学特征以及继发性并发症被用于基于五种ML算法构建模型(逻辑回归[LR],k-最近邻[KNN],极端梯度增强[XGB],随机森林[RF]和人工神经网络[ANN])。对于模型之间的评估,接受者工作特征曲线下面积(AUROC),精度-召回曲线(AUPR)下的面积,校正曲线,采用决策曲线分析(DCA)。
    结果:在预测不利的功能结局方面,复合模型的AUROC明显高于简单模型。与其他具有良好校准的复合模型(RF和XGB)相比,LR具有最高的AUPR值,并且在DCA中显示出最大的益处。
    结论:从所有研究的ML模型来看,常规LR在预测预后方面优于高级算法,可以成为医疗保健专业人员的有用工具。
    Controversy exists regarding the superiority of the performance of prognostic tools based on advanced machine learning (ML) algorithms for patients with aneurysmal subarachnoid hemorrhage (aSAH). However, it is unclear whether ML prognostic models will benefit patients due to the lack of a comprehensive assessment. We aimed to develop and evaluate ML models for predicting unfavorable functional outcomes for aSAH patients and identify the model with the greatest performance.
    In this retrospective study, a dataset of 955 patients with aSAH was used to construct and validate prognostic models for functional outcomes assessed using the modified Rankin scale during a follow-up period of 3-6 months. Clinical scores and clinical and radiological features on admission and secondary complications were used to construct models based on 5 ML algorithms (i.e., logistic regression [LR], k-nearest neighbor, extreme gradient boosting, random forest, and artificial neural network). For evaluation among the models, the area under the receiver operating characteristic curve, area under the precision-recall curve, calibration curve, and decision curve analysis were used.
    Composite models had significantly higher area under the receiver operating characteristic curves than did simple models in predicting unfavorable functional outcomes. Compared with other composite models (random forest and extreme gradient boosting) with good calibration, LR had the highest area under the precision-recall score and showed the greatest benefit in decision curve analysis.
    Of the 5 studied ML models, the conventional LR model outperformed the advanced algorithms in predicting the prognosis and could be a useful tool for health care professionals.
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  • 文章类型: Journal Article
    背景:动脉瘤性蛛网膜下腔出血(aSAH)导致长期的功能依赖和死亡。通过适当的干预策略,早期预测aSAH患者的功能结局可以降低不良预后的风险。因此,我们旨在建立术前和术后动态可视化列线图,以预测接受弹簧圈栓塞的aSAH患者的1年功能结局.
    方法:数据来自中国湖南省人民医院(2015-2019)收治的400例接受血管内盘绕的aSAH患者。关键指标是修改后的兰金评分(mRS),3-6代表较差的功能结果。建立了基于多变量逻辑回归(MLR)的视觉列线图,以分析基线特征和术后并发症。列线图性能的评估包括辨别(用C统计量衡量),校准(通过Hosmer-Lemeshow测试和校准曲线测量),和临床有用性(通过决策曲线分析衡量)。
    结果:59例aSAH患者(14.8%)的预后较差。两个列线图都显示出良好的区分度,术后列线图显示出优于术前列线图的区别,C统计量为0.895(95%CI:0.844-0.945)与0.801(95%CI:0.733-0.870)。每个都很好地校准了Hosmer-Lemeshowp值0.498与0.276.此外,决策曲线分析表明,这两个列线图在临床上都是有用的,术后列线图比术前列线图产生更多的净效益。已经开发了基于Web的在线计算器,以大大提高临床应用的效率。
    结论:术前和术后动态列线图可以支持aSAH患者的术前治疗决策和术后管理,分别。此外,这项研究表明,将术后变量纳入列线图可提高aSAH患者不良结局的预测准确性.
    BACKGROUND: Aneurysmal subarachnoid hemorrhage (aSAH) causes long-term functional dependence and death. Early prediction of functional outcomes in aSAH patients with appropriate intervention strategies could lower the risk of poor prognosis. Therefore, we aimed to develop pre- and post-operative dynamic visualization nomograms to predict the 1-year functional outcomes of aSAH patients undergoing coil embolization.
    METHODS: Data were obtained from 400 aSAH patients undergoing endovascular coiling admitted to the People\'s Hospital of Hunan Province in China (2015-2019). The key indicator was the modified Rankin Score (mRS), with 3-6 representing poor functional outcomes. Multivariate logistic regression (MLR)-based visual nomograms were developed to analyze baseline characteristics and post-operative complications. The evaluation of nomogram performance included discrimination (measured by C statistic), calibration (measured by the Hosmer-Lemeshow test and calibration curves), and clinical usefulness (measured by decision curve analysis).
    RESULTS: Fifty-nine aSAH patients (14.8%) had poor outcomes. Both nomograms showed good discrimination, and the post-operative nomogram demonstrated superior discrimination to the pre-operative nomogram with a C statistic of 0.895 (95% CI: 0.844-0.945) vs. 0.801 (95% CI: 0.733-0.870). Each was well calibrated with a Hosmer-Lemeshow p-value of 0.498 vs. 0.276. Moreover, decision curve analysis showed that both nomograms were clinically useful, and the post-operative nomogram generated more net benefit than the pre-operative nomogram. Web-based online calculators have been developed to greatly improve the efficiency of clinical applications.
    CONCLUSIONS: Pre- and post-operative dynamic nomograms could support pre-operative treatment decisions and post-operative management in aSAH patients, respectively. Moreover, this study indicates that integrating post-operative variables into the nomogram enhanced prediction accuracy for the poor outcome of aSAH patients.
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  • 文章类型: Journal Article
    目的:使用端到端深度学习模型将具有时不变和时变特征的患者记录表示为单个向量,并进一步预测心力衰竭(HF)患者的肾衰竭(KF)状态和死亡率。
    方法:时不变的EMR数据包括人口统计信息和合并症,随时间变化的EMR数据是实验室测试。我们使用Transformer编码器模块来表示时不变数据,并改进了一个长短期记忆(LSTM),顶部附有一个Transformer编码器来表示时变数据,取原始测量值及其相应的嵌入向量,掩蔽向量,和两种类型的时间间隔作为输入。具有时间不变和时变数据的患者的建议表示用于预测KF状态(5268例诊断为KF的HF患者中有949例)和HF患者的死亡率(463例住院死亡)。在所提出的模型和一些代表性的机器学习模型之间进行了比较实验。还围绕时变数据表示进行了消融实验,包括用标准的LSTM代替精制的LSTM,GRU-D和T-LSTM,分别,并删除Transformer编码器和时变数据表示模块,分别。时间不变和时变特征的注意力权重的可视化用于临床解释预测性能。我们使用接受者工作特征曲线下面积(AUROC),精度-召回曲线下的面积(AUPRC),和F1分数来评估模型的预测性能。
    结果:所提出的模型取得了优越的性能,平均AUROC,KF预测的AUPRC和F1评分分别为0.960、0.610和0.759,死亡率预测的AUPRC和F1评分分别为0.937、0.353和0.537,分别。通过添加来自更长时间段的时变数据,预测性能得到了改善。所提出的模型在两个预测任务中都优于比较和消融参考。
    结论:所提出的统一深度学习模型可以有效地表示患者的时变和时变EMR数据。这在临床预测任务中显示出更高的性能。在当前研究中使用时变数据的方法有望用于其他类型的时变数据和其他临床任务。
    To represent a patient record with both time-invariant and time-varying features as a single vector using an end-to-end deep learning model, and further to predict the kidney failure (KF) status and mortality of heart failure (HF) patients.
    The time-invariant EMR data included demographic information and comorbidities, and the time-varying EMR data were lab tests. We used a Transformer encoder module to represent the time-invariant data, and refined a long short-term memory (LSTM) with a Transformer encoder attached to the top to represent the time-varying data, taking the original measured values and their corresponding embedding vectors, masking vectors, and two types of time intervals as inputs. The proposed representations of patients with time-invariant and time-varying data were used to predict KF status (949 out of 5268 HF patients diagnosed with KF) and mortality (463 in-hospital deaths) for HF patients. Comparative experiments were conducted between the proposed model and some representative machine learning models. Ablation experiments were also performed around the time-varying data representation, including replacing the refined LSTM with the standard LSTM, GRU-D and T-LSTM, respectively, and removing the Transformer encoder and the time-varying data representation module, respectively. The visualization of the attention weights of the time-invariant and time-varying features was used to clinically interpret the predictive performance. We used the area under the receiver operating characteristic curve (AUROC), the area under the precision-recall curve (AUPRC), and the F1-score to evaluate the predictive performance of the models.
    The proposed model achieved superior performance, with average AUROCs, AUPRCs and F1-scores of 0.960, 0.610 and 0.759 for KF prediction and 0.937, 0.353 and 0.537 for mortality prediction, respectively. Predictive performance improved with the addition of time-varying data from longer time periods. The proposed model outperformed the comparison and ablation references in both prediction tasks.
    Both time-invariant and time-varying EMR data of patients could be efficiently represented by the proposed unified deep learning model, which shows higher performance in clinical prediction tasks. The way to use time-varying data in the current study is hopeful to be used in other kinds of time-varying data and other clinical tasks.
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  • 文章类型: Journal Article
    背景:由于新出现的癌症亚型和治疗选择,口咽鳞状细胞癌(OPSCC)患者越来越需要个性化治疗。结果预测模型可以帮助识别可能适合接受降级或强化治疗方法的低风险或高风险患者。
    目的:开发一种基于深度学习(DL)的模型,用于基于计算机断层扫描(CT)预测OPSCC患者的多个和相关疗效终点。
    方法:本研究使用了两个患者队列:一个由524名OPSCC患者组成的发展队列(70%用于训练,30%用于独立测试)和一个396名患者的外部测试队列。治疗前CT扫描与大体原发肿瘤体积轮廓(GTVt)和临床参数可用于预测终点,包括2年本地控制(LC),区域控制(RC),局部控制(LRC),无远处转移生存期(DMFS),疾病特异性生存率(DSS),总生存期(OS),无病生存率(DFS)。我们提出了具有多标签学习(MLL)策略的DL结果预测模型,该策略基于临床因素和CT扫描整合了不同终点的关联。
    结果:对于所有端点,多标签学习模型的性能优于基于单个端点开发的模型,尤其是对于2年RC而言,AUC高≥0.80,DMFS,DSS,操作系统,和内部独立测试集中的DFS,以及外部测试集中除2年LRC外的所有端点。此外,随着所开发的模型,可以将患者分为高危组和低危组,这两组在内部测试集中的所有终点和外部测试集中的所有终点(DMFS除外)均存在显著差异.
    结论:MLL模型在内部测试中对所有2年疗效终点的判别能力优于单一结果模型,在外部集合中除LRC以外的所有终点。
    BACKGROUND: Personalized treatment is increasingly required for oropharyngeal squamous cell carcinoma (OPSCC) patients due to emerging new cancer subtypes and treatment options. Outcome prediction model can help identify low or high-risk patients who may be suitable to receive de-escalation or intensified treatment approaches.
    OBJECTIVE: To develop a deep learning (DL)-based model for predicting multiple and associated efficacy endpoints in OPSCC patients based on computed tomography (CT).
    METHODS: Two patient cohorts were used in this study: a development cohort consisting of 524 OPSCC patients (70% for training and 30% for independent testing) and an external test cohort of 396 patients. Pre-treatment CT-scans with the gross primary tumor volume contours (GTVt) and clinical parameters were available to predict endpoints, including 2-year local control (LC), regional control (RC), locoregional control (LRC), distant metastasis-free survival (DMFS), disease-specific survival (DSS), overall survival (OS), and disease-free survival (DFS). We proposed DL outcome prediction models with the multi-label learning (MLL) strategy that integrates the associations of different endpoints based on clinical factors and CT-scans.
    RESULTS: The multi-label learning models outperformed the models that were developed based on a single endpoint for all endpoints especially with high AUCs ≥ 0.80 for 2-year RC, DMFS, DSS, OS, and DFS in the internal independent test set and for all endpoints except 2-year LRC in the external test set. Furthermore, with the models developed, patients could be stratified into high and low-risk groups that were significantly different for all endpoints in the internal test set and for all endpoints except DMFS in the external test set.
    CONCLUSIONS: MLL models demonstrated better discriminative ability for all 2-year efficacy endpoints than single outcome models in the internal test and for all endpoints except LRC in the external set.
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