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Insights Into the Patient Experience of Hormone Therapy for Early Breast Cancer Treatment Using Patient Forum Discussions and Natural Language Processing. 利用患者论坛讨论和自然语言处理深入了解早期乳腺癌治疗中激素疗法的患者体验。
IF 3.3 Q2 ONCOLOGY Pub Date : 2024-08-01 DOI: 10.1200/CCI.24.00038
Sameet Sreenivasan, Chao Fang, Emuella M Flood, Natasha Markuzon, Jasmine Y Y Sze

Purpose: Understanding the real-world experience of patients with early breast cancer (eBC) is imperative for optimizing outcomes and evolving patient care. However, there is a lack of patient-level data, hindering clinical development. This social listening study was performed to understand patient insights into symptoms and impacts of hormone therapy (HT) for eBC using posts from patient forums on breastcancer.org to inform future clinical research.

Methods: Natural language processing (NLP) and machine learning techniques were used to identify themes related to eBC from a sample of 500,000 posts. After relevant data selection, 362,074 eBC posts were retained for further analysis of symptoms and impacts related to HT, as well as insights into symptom severity, pain locations, and symptom management using exercise and yoga.

Results: Overall, 32 symptoms and nine impacts had significant associations with ≥one HT. Hot flush (relative risk [RR], 6.70 [95% CI, 3.36 to 13.36]), arthralgia (RR, 6.67 [95% CI, 3.53 to 12.59]), weight increased (RR, 4.83 [95% CI, 3.20 to 7.28]), mood swings (RR, 7.36 [95% CI, 5.75 to 9.42]), insomnia (RR, 4.76 [95% CI, 3.14 to 7.22]), and depression (RR, 3.05 [95% CI, 1.71 to 5.44]) demonstrated the strongest associations. Severe headache, dizziness, back pain, and muscle spasms showed significant associations with ≥one HT despite their low overall prevalence in eBC posts.

Conclusion: The social listening approach allowed the identification of real-world insights from posts specific to eBC HT from a large-scale online breast cancer forum that captured experiences from a uniquely diverse group of patients. Using NLP has a potential to scale analysis of patient feedback and reveal actionable insights into patient experiences of treatment that can inform the development of future therapies and improve the care of patients with eBC.

目的:了解早期乳腺癌(eBC)患者的真实经历对于优化治疗效果和发展患者护理至关重要。然而,由于缺乏患者层面的数据,阻碍了临床开发。本社交聆听研究利用乳腺癌网站(breastcancer.org)患者论坛上的帖子了解患者对激素疗法(HT)治疗早期乳腺癌的症状和影响的见解,为未来的临床研究提供信息:方法:使用自然语言处理 (NLP) 和机器学习技术从 500,000 个帖子样本中识别与 eBC 相关的主题。在对相关数据进行筛选后,保留了 362,074 篇 eBC 帖子,用于进一步分析与 HT 相关的症状和影响,以及对症状严重程度、疼痛部位和使用运动和瑜伽进行症状管理的见解:总的来说,32 种症状和 9 种影响与≥一种高血压有显著关联。潮热(相对风险 [RR],6.70 [95% CI,3.36 至 13.36])、关节痛(RR,6.67 [95% CI,3.53 至 12.59])、体重增加(RR,4.83 [95% CI,3.20 至 7.28])、情绪波动(RR,7.36 [95% CI, 5.75 to 9.42])、失眠(RR, 4.76 [95% CI, 3.14 to 7.22])和抑郁(RR, 3.05 [95% CI, 1.71 to 5.44])显示出最强的关联性。尽管严重头痛、头晕、背痛和肌肉痉挛在 eBC 帖子中的总体发生率较低,但这些症状与≥一种 HT 有显著关联:通过社会聆听方法,可以从一个大型在线乳腺癌论坛的eBC HT帖子中发现真实世界的见解,该论坛收集了来自独特的不同患者群体的经验。使用 NLP 有可能扩大对患者反馈的分析范围,并揭示出患者治疗经历中的可行见解,从而为未来疗法的开发提供依据,并改善对 eBC 患者的护理。
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引用次数: 0
Machine Learning-Based Prediction of 1-Year Survival Using Subjective and Objective Parameters in Patients With Cancer. 基于机器学习的癌症患者 1 年生存期主客观参数预测法
IF 3.3 Q2 ONCOLOGY Pub Date : 2024-08-01 DOI: 10.1200/CCI.24.00041
Maria Rosa Salvador Comino, Paul Youssef, Anna Heinzelmann, Florian Bernhardt, Christin Seifert, Mitra Tewes

Purpose: Palliative care is recommended for patients with cancer with a life expectancy of <12 months. Machine learning (ML) techniques can help in predicting survival outcomes among patients with cancer and may help distinguish who benefits the most from palliative care support. We aim to explore the importance of several objective and subjective self-reported variables. Subjective variables were collected through electronic psycho-oncologic and palliative care self-assessment screenings. We used these variables to predict 1-year mortality.

Materials and methods: Between April 1, 2020, and March 31, 2021, a total of 265 patients with advanced cancer completed a patient-reported outcome tool. We documented objective and subjective variables collected from electronic health records, self-reported subjective variables, and all clinical variables combined. We used logistic regression (LR), 20-fold cross-validation, decision trees, and random forests to predict 1-year mortality. We analyzed the receiver operating characteristic (ROC) curve-AUC, the precision-recall curve-AUC (PR-AUC)-and the feature importance of the ML models.

Results: The performance of clinical nonpatient variables in predictions (LR reaches 0.81 [ROC-AUC] and 0.72 [F1 score]) are much more predictive than that of subjective patient-reported variables (LR reaches 0.55 [ROC-AUC] and 0.52 [F1 score]).

Conclusion: The results show that objective variables used in this study are much more predictive than subjective patient-reported variables, which measure subjective burden. These findings indicate that subjective burden cannot be reliably used to predict survival. Further research is needed to clarify the role of self-reported patient burden and mortality prediction using ML.

目的:建议对预期寿命不长的癌症患者进行姑息治疗 材料与方法:在 2020 年 4 月 1 日至 2021 年 3 月 31 日期间,共有 265 名晚期癌症患者填写了患者报告结果工具。我们记录了从电子健康记录中收集的客观和主观变量、自我报告的主观变量以及所有临床变量。我们使用逻辑回归(LR)、20 倍交叉验证、决策树和随机森林预测 1 年死亡率。我们分析了接收者操作特征曲线(ROC)-AUC、精确度-召回曲线-AUC(PR-AUC)以及ML模型的特征重要性:结果:临床非患者变量的预测性能(LR 达到 0.81 [ROC-AUC] 和 0.72 [F1 分数])远高于患者主观报告变量的预测性能(LR 达到 0.55 [ROC-AUC] 和 0.52 [F1 分数]):结果表明,本研究中使用的客观变量比患者报告的主观变量(衡量主观负担的变量)更具预测性。这些结果表明,主观负担不能可靠地用于预测生存率。还需要进一步研究,以明确患者自我报告的负担和使用 ML 预测死亡率的作用。
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引用次数: 0
Automated Extraction of Patient-Centered Outcomes After Breast Cancer Treatment: An Open-Source Large Language Model-Based Toolkit. 自动提取乳腺癌治疗后以患者为中心的结果:基于大型语言模型的开源工具包。
IF 3.3 Q2 ONCOLOGY Pub Date : 2024-08-01 DOI: 10.1200/CCI.23.00258
Man Luo, Shubham Trivedi, Allison W Kurian, Kevin Ward, Theresa H M Keegan, Daniel Rubin, Imon Banerjee

Purpose: Patient-centered outcomes (PCOs) are pivotal in cancer treatment, as they directly reflect patients' quality of life. Although multiple studies suggest that factors affecting breast cancer-related morbidity and survival are influenced by treatment side effects and adherence to long-term treatment, such data are generally only available on a smaller scale or from a single center. The primary challenge with collecting these data is that the outcomes are captured as free text in clinical narratives written by clinicians.

Materials and methods: Given the complexity of PCO documentation in these narratives, computerized methods are necessary to unlock the wealth of information buried in unstructured text notes that often document PCOs. Inspired by the success of large language models (LLMs), we examined the adaptability of three LLMs, GPT-2, BioGPT, and PMC-LLaMA, on PCO tasks across three institutions, Mayo Clinic, Emory University Hospital, and Stanford University. We developed an open-source framework for fine-tuning LLM that can directly extract the five different categories of PCO from the clinic notes.

Results: We found that these LLMs without fine-tuning (zero-shot) struggle with challenging PCO extraction tasks, displaying almost random performance, even with some task-specific examples (few-shot learning). The performance of our fine-tuned, task-specific models is notably superior compared with their non-fine-tuned LLM models. Moreover, the fine-tuned GPT-2 model has demonstrated a significantly better performance than the other two larger LLMs.

Conclusion: Our discovery indicates that although LLMs serve as effective general-purpose models for tasks across various domains, they require fine-tuning when applied to the clinician domain. Our proposed approach has the potential to lead more efficient, adaptable models for PCO information extraction, reducing reliance on extensive computational resources while still delivering superior performance for specific tasks.

目的:以患者为中心的治疗结果(PCOs)直接反映了患者的生活质量,因此在癌症治疗中至关重要。尽管多项研究表明,影响乳腺癌相关发病率和生存率的因素受到治疗副作用和坚持长期治疗的影响,但这些数据通常只能在较小范围内或从单一中心获得。收集这些数据的主要挑战在于,这些结果是以自由文本的形式记录在临床医生撰写的临床叙述中的:鉴于这些叙述中 PCO 记录的复杂性,有必要采用计算机化的方法来挖掘通常记录 PCO 的非结构化文本笔记中埋藏的大量信息。受大型语言模型(LLM)成功的启发,我们研究了三种 LLM(GPT-2、BioGPT 和 PMC-LaMA)在梅奥诊所、埃默里大学医院和斯坦福大学这三个机构的 PCO 任务中的适应性。我们开发了一个用于微调 LLM 的开源框架,可直接从门诊笔记中提取 PCO 的五个不同类别:我们发现,这些未进行微调(零点学习)的 LLM 在完成具有挑战性的 PCO 提取任务时非常吃力,即使使用一些特定任务的示例(少量学习),其表现也几乎是随机的。与未进行微调的 LLM 模型相比,我们针对特定任务进行微调的模型性能明显更优。此外,经过微调的 GPT-2 模型的性能明显优于其他两个较大的 LLM:我们的发现表明,虽然 LLM 是适用于不同领域任务的有效通用模型,但在应用于临床医生领域时需要对其进行微调。我们提出的方法有可能为 PCO 信息提取提供更高效、适应性更强的模型,减少对大量计算资源的依赖,同时还能为特定任务提供卓越的性能。
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引用次数: 0
Cureit: An End-to-End Pipeline for Implementing Mixture Cure Models With an Application to Liposarcoma Data. Cureit:应用于脂肪肉瘤数据的端到端混合治愈模型实施流程
IF 3.3 Q2 ONCOLOGY Pub Date : 2024-08-01 DOI: 10.1200/CCI.23.00234
Karissa Whiting, Teng Fei, Samuel Singer, Li-Xuan Qin

Purpose: Cure models are a useful alternative to Cox proportional hazards models in oncology studies when there is a subpopulation of patients who will not experience the event of interest. Although software is available to fit cure models, there are limited tools to evaluate, report, and visualize model results. This article introduces the cureit R package, an end-to-end pipeline for building mixture cure models, and demonstrates its use in a data set of patients with primary extremity and truncal liposarcoma.

Methods: To assess associations between liposarcoma histologic subtypes and disease-specific death (DSD) in patients treated at Memorial Sloan Kettering Cancer Center between July 1982 and September 2017, mixture cure models were fit and evaluated using the cureit package. Liposarcoma histologic subtypes were defined as well-differentiated, dedifferentiated, myxoid, round cell, and pleomorphic.

Results: All other analyzed liposarcoma histologic subtypes were significantly associated with higher DSD in cure models compared with well-differentiated. In multivariable models, myxoid (odds ratio [OR], 6.25 [95% CI, 1.32 to 29.6]) and round cell (OR, 16.2 [95% CI, 2.80 to 93.2]) liposarcoma had higher incidences of DSD compared with well-differentiated patients. By contrast, dedifferentiated liposarcoma was associated with the latency of DSD (hazard ratio, 10.6 [95% CI, 1.48 to 75.9]). Pleomorphic liposarcomas had significantly higher risk in both incidence and the latency of DSD (P < .0001). Brier scores indicated comparable predictive accuracy between cure and Cox models.

Conclusion: We developed the cureit pipeline to fit and evaluate mixture cure models and demonstrated its clinical utility in the liposarcoma disease setting, shedding insights on the subtype-specific associations with incidence and/or latency.

目的:在肿瘤学研究中,当有一部分患者不会发生相关事件时,治愈模型是 Cox 比例危险度模型的一种有效替代方法。虽然有软件可用于拟合治愈模型,但评估、报告和可视化模型结果的工具却很有限。本文介绍了 cureit R 软件包--一种用于构建混合治愈模型的端到端管道,并展示了其在原发性四肢和躯干脂肪肉瘤患者数据集中的应用:为了评估1982年7月至2017年9月期间在纪念斯隆-凯特琳癌症中心接受治疗的脂肪肉瘤组织学亚型与疾病特异性死亡(DSD)之间的关联,使用cureit软件包拟合并评估了混合治愈模型。脂肪肉瘤组织学亚型被定义为分化良好型、去分化型、肌样型、圆形细胞型和多形性:结果:在治愈模型中,与分化良好的脂肪肉瘤相比,所有其他分析的脂肪肉瘤组织学亚型都与较高的DSD显著相关。在多变量模型中,与分化良好的患者相比,类肌瘤(几率比[OR],6.25[95% CI,1.32至29.6])和圆形细胞(OR,16.2[95% CI,2.80至93.2])脂肪肉瘤的DSD发生率较高。相比之下,低分化脂肪肉瘤与DSD的潜伏期有关(危险比为10.6 [95% CI, 1.48 to 75.9])。多形性脂肪肉瘤在发病率和DSD潜伏期方面的风险都明显更高(P < .0001)。Brier评分表明,治愈模型和Cox模型的预测准确性相当:我们开发了 cureit 管道来拟合和评估混合治愈模型,并证明了其在脂肪肉瘤疾病环境中的临床实用性,揭示了亚型与发病率和/或潜伏期的特异性关联。
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引用次数: 0
Evaluation of Real-World Tumor Response Derived From Electronic Health Record Data Sources: A Feasibility Analysis in Patients With Metastatic Non-Small Cell Lung Cancer Treated With Chemotherapy. 评估从电子健康记录数据源得出的真实世界肿瘤反应:对接受化疗的转移性非小细胞肺癌患者进行可行性分析。
IF 3.3 Q2 ONCOLOGY Pub Date : 2024-08-01 DOI: 10.1200/CCI.24.00091
Brittany A McKelvey, Elizabeth Garrett-Mayer, Donna R Rivera, Amy Alabaster, Hillary S Andrews, Elizabeth G Bond, Thomas D Brown, Amanda Bruno, Lauren Damato, Janet L Espirito, Laura L Fernandes, Eric Hansen, Paul Kluetz, Xinran Ma, Andrea McCracken, Pallavi S Mishra-Kalyani, Yanina Natanzon, Danielle Potter, Nicholas J Robert, Lawrence Schwartz, Regina Schwind, Connor Sweetnam, Joseph Wagner, Mark D Stewart, Jeff D Allen

Purpose: Real-world data (RWD) holds promise for ascribing a real-world (rw) outcome to a drug intervention; however, ascertaining rw-response to treatment from RWD can be challenging. Friends of Cancer Research formed a collaboration to assess available data attributes related to rw-response across RWD sources to inform methods for capturing, defining, and evaluating rw-response.

Materials and methods: This retrospective noninterventional (observational) study included seven electronic health record data companies (data providers) providing summary-level deidentified data from 200 patients diagnosed with metastatic non-small cell lung cancer (mNSCLC) and treated with first-line platinum doublet chemotherapy following a common protocol. Data providers reviewed the availability and frequency of data components to assess rw-response (ie, images, radiology imaging reports, and clinician response assessments). A common protocol was used to assess and report rw-response end points, including rw-response rate (rwRR), rw-duration of response (rwDOR), and the association of rw-response with rw-overall survival (rwOS), rw-time to treatment discontinuation (rwTTD), and rw-time to next treatment (rwTTNT).

Results: The availability and timing of clinician assessments was relatively consistent across data sets in contrast to images and image reports. Real-world response was analyzed using clinician response assessments (median proportion of patients evaluable, 77.5%), which had the highest consistency in the timing of assessments. Relative consistency was observed across data sets for rwRR (median 46.5%), as well as the median and directionality of rwOS, rwTTD, and rwTTNT. There was variability in rwDOR across data sets.

Conclusion: This collaborative effort demonstrated the feasibility of aligning disparate data sources to evaluate rw-response end points using clinician-documented responses in patients with mNSCLC. Heterogeneity exists in the availability of data components to assess response and related rw-end points, and further work is needed to inform drug effectiveness evaluation within RWD sources.

目的:真实世界数据(RWD)为将真实世界(Rw)结果归因于药物干预带来了希望;然而,从 RWD 中确定治疗的 Rw 反应可能具有挑战性。癌症研究之友 "组织了一次合作,以评估与真实世界数据源中的治疗反应相关的可用数据属性,从而为捕获、定义和评估治疗反应的方法提供信息:这项回顾性非干预性(观察性)研究包括七家电子健康记录数据公司(数据提供者),它们提供了 200 名确诊为转移性非小细胞肺癌(mNSCLC)并按照共同方案接受一线铂双t化疗的患者的摘要级去标识化数据。数据提供者审查了用于评估 rw 反应(即图像、放射成像报告和临床医生反应评估)的数据组件的可用性和频率。采用通用方案评估和报告rw反应终点,包括rw反应率(rwRR)、rw反应持续时间(rwDOR),以及rw反应与rw总生存期(rwOS)、rw终止治疗时间(rwTTD)和rw下次治疗时间(rwTTNT)的关系:结果:与图像和图像报告相比,各数据集的临床医生评估的可用性和时间相对一致。使用临床医生的反应评估(可评估患者的中位比例为 77.5%)分析真实世界的反应,评估时间的一致性最高。各数据集的 rwRR(中位数为 46.5%)以及 rwOS、rwTTD 和 rwTTNT 的中位数和方向性也相对一致。不同数据集的 rwDOR 存在差异:这项合作证明了利用临床医生记录的 mNSCLC 患者的反应,整合不同数据源以评估 rw 反应终点的可行性。在评估反应和相关rw终点的数据组件的可用性方面存在异质性,需要进一步开展工作,以便在RWD来源中为药物疗效评估提供信息。
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引用次数: 0
Deep-Transfer-Learning-Based Natural Language Processing of Serial Free-Text Computed Tomography Reports for Predicting Survival of Patients With Pancreatic Cancer. 基于深度传输学习的自然语言处理连续自由文本计算机断层扫描报告,用于预测胰腺癌患者的生存率。
IF 3.3 Q2 ONCOLOGY Pub Date : 2024-08-01 DOI: 10.1200/CCI.24.00021
Sunkyu Kim, Seung-Seob Kim, Eejung Kim, Michael Cecchini, Mi-Suk Park, Ji A Choi, Sung Hyun Kim, Ho Kyoung Hwang, Chang Moo Kang, Hye Jin Choi, Sang Joon Shin, Jaewoo Kang, Choong-Kun Lee

Purpose: To explore the predictive potential of serial computed tomography (CT) radiology reports for pancreatic cancer survival using natural language processing (NLP).

Methods: Deep-transfer-learning-based NLP models were retrospectively trained and tested with serial, free-text CT reports, and survival information of consecutive patients diagnosed with pancreatic cancer in a Korean tertiary hospital was extracted. Randomly selected patients with pancreatic cancer and their serial CT reports from an independent tertiary hospital in the United States were included in the external testing data set. The concordance index (c-index) of predicted survival and actual survival, and area under the receiver operating characteristic curve (AUROC) for predicting 1-year survival were calculated.

Results: Between January 2004 and June 2021, 2,677 patients with 12,255 CT reports and 670 patients with 3,058 CT reports were allocated to training and internal testing data sets, respectively. ClinicalBERT (Bidirectional Encoder Representations from Transformers) model trained on the single, first CT reports showed a c-index of 0.653 and AUROC of 0.722 in predicting the overall survival of patients with pancreatic cancer. ClinicalBERT trained on up to 15 consecutive reports from the initial report showed an improved c-index of 0.811 and AUROC of 0.911. On the external testing set with 273 patients with 1,947 CT reports, the AUROC was 0.888, indicating the generalizability of our model. Further analyses showed our model's contextual interpretation beyond specific phrases.

Conclusion: Deep-transfer-learning-based NLP model of serial CT reports can predict the survival of patients with pancreatic cancer. Clinical decisions can be supported by the developed model, with survival information extracted solely from serial radiology reports.

目的:利用自然语言处理(NLP)技术探索序列计算机断层扫描(CT)放射学报告对胰腺癌生存率的预测潜力:用连续的自由文本 CT 报告对基于深度传输学习的 NLP 模型进行了回顾性训练和测试,并提取了韩国一家三级医院连续确诊的胰腺癌患者的生存信息。外部测试数据集包括从美国一家独立三甲医院随机挑选的胰腺癌患者及其序列 CT 报告。计算了预测生存率和实际生存率的一致性指数(c-index)以及预测1年生存率的接收者操作特征曲线下面积(AUROC):2004年1月至2021年6月期间,2677名患者的12255份CT报告和670名患者的3058份CT报告分别被分配到训练数据集和内部测试数据集。在预测胰腺癌患者的总生存率方面,根据单次、首次 CT 报告训练的 ClinicalBERT(来自变换器的双向编码器表征)模型的 c 指数为 0.653,AUROC 为 0.722。从最初的报告开始,ClinicalBERT 对多达 15 份连续报告进行了训练,结果显示 c 指数提高到 0.811,AUROC 提高到 0.911。在包含 273 名患者和 1,947 份 CT 报告的外部测试集上,AUROC 为 0.888,这表明我们的模型具有普适性。进一步的分析表明,我们的模型对特定短语之外的上下文进行了解释:结论:基于深度传输学习的序列 CT 报告 NLP 模型可以预测胰腺癌患者的生存率。开发出的模型可为临床决策提供支持,其生存信息仅从序列放射学报告中提取。
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引用次数: 0
The More, the Better? Modalities of Metastatic Status Extraction on Free Medical Reports Based on Natural Language Processing. 越多越好?基于自然语言处理的免费医学报告转移状态提取模式
IF 3.3 Q2 ONCOLOGY Pub Date : 2024-08-01 DOI: 10.1200/CCI.24.00026
Emmanuelle Kempf, Sonia Priou, Ariel Cohen, Akram Redjdal, Etienne Guével, Xavier Tannier
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引用次数: 0
Prostate-Specific Antigen Screening and Prostate Cancer Mortality: An Emulation of Target Trials in US Medicare. 前列腺特异性抗原筛查与前列腺癌死亡率:美国医疗保险中目标试验的模拟。
IF 3.3 Q2 ONCOLOGY Pub Date : 2024-08-01 DOI: 10.1200/CCI.24.00094
Xabier García-Albéniz, John Hsu, Ruth Etzioni, June M Chan, Joy Shi, Barbra Dickerman, Miguel A Hernán

Purpose: No consensus about the effectiveness of prostate-specific antigen (PSA) screening exists among clinical guidelines, especially for the elderly. Randomized trials of PSA screening have yielded different results, partly because of variations in adherence, and it is unlikely that new trials will be conducted. Our objective was to estimate the effect of annual PSA screening on prostate cancer (PC) mortality in Medicare beneficiaries age 67-84 years.

Methods: This is a large-scale, population-based, observational study of two screening strategies: annual PSA screening and no screening. We used data from 537,599 US Medicare (2001-2008) beneficiaries age 67-84 years who had a good life expectancy, no previous PC, and no PSA test in the 2 years before baseline. We estimated the 8-year PC mortality and incidence, treatments for PC, and treatment complications of PSA screening.

Results: In men age 67-74 years, the estimated difference in 8-year risk of PC death between PSA screening and no screening was -2.3 (95% CI, -4.1 to -1.1) deaths per 1,000 men (a negative risk difference favors screening). Treatment complications were more frequent under PSA screening than under no screening. In men age 75-84 years, risk difference estimates were closer to zero.

Conclusion: Our estimates suggest that under conventional statistical criteria, annual PSA screening for 8 years is highly compatible with reductions of PC mortality from four to one fewer PC deaths per 1,000 screened men age 67-74 years. As with any study using real-world data, the estimates could be affected by residual confounding.

目的:临床指南对前列腺特异性抗原(PSA)筛查的有效性尚未达成共识,尤其是针对老年人。PSA 筛查的随机试验产生了不同的结果,部分原因是坚持率存在差异,而且不太可能进行新的试验。我们的目标是估算每年进行 PSA 筛查对 67-84 岁医疗保险受益人前列腺癌(PC)死亡率的影响:这是一项大规模、基于人群的观察性研究,涉及两种筛查策略:每年进行 PSA 筛查和不进行筛查。我们使用了 537,599 名美国医疗保险(2001-2008 年)受益人的数据,这些受益人年龄在 67-84 岁之间,预期寿命良好,既往未患 PC,基线前 2 年未进行 PSA 检测。我们估算了 8 年的 PC 死亡率和发病率、PC 治疗方法以及 PSA 筛查的治疗并发症:在 67-74 岁的男性中,PSA 筛查与不做筛查的 8 年 PC 死亡风险估计差异为-2.3(95% CI,-4.1 至-1.1)/1,000(负风险差异有利于筛查)。筛查 PSA 比不筛查更容易出现治疗并发症。在 75-84 岁的男性中,风险差异估计值更接近于零:我们的估算结果表明,根据传统的统计标准,每年进行一次为期 8 年的 PSA 筛查可将 PC 死亡率降低到每 1,000 名接受筛查的 67-74 岁男性中 PC 死亡人数减少 4 到 1 人的水平。与任何使用真实世界数据的研究一样,估计值可能会受到残余混杂因素的影响。
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引用次数: 0
Classification and Regression Trees to Predict for Survival for Patients With Hepatocellular Carcinoma Treated With Atezolizumab and Bevacizumab. 用分类树和回归树预测接受阿特珠单抗和贝伐珠单抗治疗的肝细胞癌患者的生存率
IF 3.3 Q2 ONCOLOGY Pub Date : 2024-08-01 DOI: 10.1200/CCI.23.00220
Timothy J Brown, Phyllis A Gimotty, Ronac Mamtani, Thomas B Karasic, Yu-Xiao Yang

Purpose: Systemic therapy with atezolizumab and bevacizumab can extend life for patients with advanced hepatocellular carcinoma (HCC). However, there is substantial variability in response to therapy and overall survival. Although current prognostic models have been validated in HCC, they primarily consider covariates that may be reflective of the severity of the underlying liver disease of patients with HCC. We developed and internally validated a classification and regression tree (CART) to identify patient characteristics associated with risks of early mortality, at or before 6 months from treatment initiation.

Methods: This retrospective cohort study used the nationwide Flatiron Health electronic health record-derived deidentified database and included patients with a diagnosis of HCC after January 1, 2020, who received initial systemic therapy with atezolizumab and bevacizumab. CART was developed from available baseline clinical and demographic information to predict mortality within 6 months from treatment initiation. Model characteristics were compared to the albumin-bilirubin (ALBI) model and was further validated against a contemporary validation cohort of patients after a data update.

Results: A total of 293 patients were analyzed. The CART identified seven cohorts of patients from baseline demographic and laboratory characteristics. The model had an area under the receiver operating curve (AUROC) of 0.739 (95% CI, 0.683 to 0.794) for predicting 6-month mortality. This model was internally valid and performed more favorably than the ALBI model, which had an AUROC of 0.608 (95% CI, 0.557 to 0.660). The model applied to the contemporary validation cohort (n = 111) had an AUROC of 0.666 (95% CI, 0.506 to 0.826).

Conclusion: Using CART, we identified unique cohorts of patients with HCC treated with atezolizumab and bevacizumab with distinct risks of early mortality. This approach outperformed the ALBI model and used clinical and laboratory characteristics that are readily available to oncologists caring for these patients.

目的:使用阿特珠单抗和贝伐单抗进行全身治疗可延长晚期肝细胞癌(HCC)患者的生存期。然而,患者对治疗的反应和总生存期存在很大差异。虽然目前的预后模型已在 HCC 中得到验证,但它们主要考虑的是可能反映 HCC 患者基础肝病严重程度的协变量。我们开发并在内部验证了一种分类和回归树(CART),以确定与治疗开始后 6 个月或 6 个月之前早期死亡风险相关的患者特征:这项回顾性队列研究使用了全国性的 Flatiron Health 电子健康记录衍生去标识数据库,纳入了 2020 年 1 月 1 日之后诊断为 HCC 并接受了阿特珠单抗和贝伐珠单抗初始系统治疗的患者。根据现有的基线临床和人口统计学信息开发了 CART,用于预测治疗开始后 6 个月内的死亡率。模型特征与白蛋白-胆红素(ALBI)模型进行了比较,并在数据更新后与当代验证患者队列进行了进一步验证:结果:共分析了 293 名患者。结果:共分析了 293 名患者,CART 从基线人口学和实验室特征中识别出七个患者队列。该模型预测 6 个月死亡率的接收者操作曲线下面积 (AUROC) 为 0.739(95% CI,0.683 至 0.794)。该模型具有内部有效性,其表现优于ALBI模型,后者的AUROC为0.608(95% CI,0.557至0.660)。应用于当代验证队列(n = 111)的模型的AUROC为0.666(95% CI,0.506至0.826):利用 CART,我们确定了接受阿特珠单抗和贝伐珠单抗治疗的 HCC 患者中具有不同早期死亡风险的独特队列。这种方法的效果优于 ALBI 模型,而且使用的临床和实验室特征对于治疗这些患者的肿瘤学家来说是唾手可得的。
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引用次数: 0
Clinical Calculator for Predicting Freedom From Recurrence After Resection of Stage I-III Colon Cancer in Patients With Microsatellite Instability. 用于预测微卫星不稳定性 I-III 期结肠癌患者切除术后复发自由度的临床计算器。
IF 3.3 Q2 ONCOLOGY Pub Date : 2024-08-01 DOI: 10.1200/CCI.23.00233
Ayyuce Begum Bektas, Lynn Hakki, Asama Khan, Maria Widmar, Iris H Wei, Emmanouil Pappou, J Joshua Smith, Garrett M Nash, Philip B Paty, Julio Garcia-Aguilar, Andrea Cercek, Zsofia Stadler, Neil H Segal, Jinru Shia, Mithat Gonen, Martin R Weiser

Purpose: Outcome for patients with nonmetastatic, microsatellite instability (MSI) colon cancer is favorable: however, high-risk cohorts exist. This study was aimed at developing and validating a nomogram model to predict freedom from recurrence (FFR) for patients with resected MSI colon cancer.

Patients and methods: Data from patients who underwent curative resection of stage I, II, or III MSI colon cancer in 2014-2021 (model training cohort, 384 patients, 33 events; median follow-up, 38.8 months) were retrospectively collected from institutional databases. Variables associated with recurrence in multivariable analysis were selected for inclusion in the clinical calculator. The calculator's predictive accuracy was measured with the concordance index and validated using data from patients who underwent treatment for MSI colon cancer in 2007-2013 (validation cohort, 164 patients, eight events; median follow-up, 84.8 months).

Results: T category and number of positive lymph nodes were significantly associated with recurrence in multivariable analysis and were selected for inclusion in the clinical calculator. The calculator's concordance index for FFR in the model training cohort was 0.812 (95% CI, 0.742 to 0.873), compared with 0.759 (95% CI, 0.683 to 0.840) for the staging schema of the eighth edition of the American Joint Committee on Cancer Staging Manual. The concordance index for the validation cohort was 0.744 (95% CI, 0.666 to 0.822), confirming robust predictive accuracy.

Conclusion: Although in general patients with nonmetastatic MSI colon cancer had favorable outcome, patients with advanced T category and multiple metastatic lymph nodes had higher risk of recurrence. The clinical calculator identified patients with MSI colon cancer at high risk for recurrence, and this could inform surveillance strategies. In addition, the model could be used in trial design to identify patients suitable for novel adjuvant therapy.

目的:非转移性微卫星不稳定性(MSI)结肠癌患者的预后良好:但也存在高风险人群。本研究旨在开发和验证一个提名图模型,用于预测切除的 MSI 结肠癌患者的复发率(FFR):从机构数据库中回顾性收集了2014-2021年接受根治性切除的I、II或III期MSI结肠癌患者的数据(模型训练队列,384名患者,33个事件;中位随访时间,38.8个月)。多变量分析中与复发相关的变量被选入临床计算器。计算器的预测准确性通过一致性指数进行测量,并利用2007-2013年接受MSI结肠癌治疗的患者数据进行了验证(验证队列,164名患者,8起事件;中位随访时间,84.8个月):结果:在多变量分析中,T类别和阳性淋巴结数量与复发显著相关,并被选入临床计算器。在模型训练队列中,计算器的FFR一致性指数为0.812(95% CI,0.742至0.873),而美国癌症分期联合委员会手册第八版分期方案的一致性指数为0.759(95% CI,0.683至0.840)。验证队列的一致性指数为 0.744(95% CI,0.666 至 0.822),证实了预测的准确性:结论:虽然非转移性MSI结肠癌患者的预后一般较好,但T类晚期和多处转移淋巴结的患者复发风险较高。临床计算器识别出了复发风险较高的 MSI 结肠癌患者,这可以为监测策略提供参考。此外,该模型还可用于试验设计,以确定适合新型辅助疗法的患者。
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引用次数: 0
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JCO Clinical Cancer Informatics
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