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Utilization and safety of trastuzumab emtansine (T-DM1): a nationwide population-based study using the French National Health Data System 曲妥珠单抗埃坦新(T-DM1)的使用情况和安全性:利用法国国家卫生数据系统开展的全国人口研究
Pub Date : 2024-06-01 DOI: 10.1016/j.esmorw.2024.100045
H. Jourdain , I. Mansouri , A. Di Meglio , M. Zureik , N. Haddy

Background

Trastuzumab emtansine (T-DM1), the first antibody–drug conjugate targeting human epidermal growth factor receptor 2 (HER2), has provided new alternatives for metastatic breast cancer (mBC) treatment, and for early-stage breast cancer (esBC) since 2020. We characterized T-DM1 users and assessed the risk of hospitalisation for certain adverse events.

Materials and methods

Using data from the French National Health Data System (SNDS), we identified patients receiving T-DM1 from 2014 to 2022. Sociodemographic data, medical history, and overall survival were described by indication. The risk of adverse event-related hospitalisation was quantified.

Results

We included 12 822 patients initiating T-DM1 treatment in the study: 9232 (72%) with mBC (median age: 59 years, 54.6% with at least one comorbid condition) and 3590 (28%) with esBC (median age: 54 years, 33.9% with at least one comorbid condition). In mBC, median overall survival was 32.6 months, showing a gradual improvement over the years. At 1 year, 75.8% of patients had discontinued treatment and 22.0% had died. Patients in the study faced higher hospitalisation risks than those with incident breast cancer, with increases of 5.7 and 10.5 times for thrombocytopenia, 3.0 and 4.5 times for interstitial lung disease, 3.3 and 5.9 times for acute liver injury, and 2.3 and 7.3 times for sepsis, for esBC and mBC, respectively.

Conclusions

Real-world T-DM1 users frequently present with comorbid conditions and prior treatments, with a higher risk of hospitalisation for severe toxicity events than the general population of incident breast cancers. This study highlights the importance of monitoring treated patients to manage the risk of toxicity and prevent treatment discontinuation.

背景Trastuzumab emtansine(T-DM1)是首个靶向人类表皮生长因子受体2(HER2)的抗体药物共轭物,它为转移性乳腺癌(mBC)的治疗提供了新的选择,自2020年以来也为早期乳腺癌(esBC)的治疗提供了新的选择。我们对T-DM1使用者进行了特征描述,并评估了因某些不良事件住院的风险。材料与方法利用法国国家卫生数据系统(SNDS)的数据,我们确定了2014年至2022年期间接受T-DM1治疗的患者。社会人口学数据、病史和总生存期按适应症进行了描述。我们对与不良事件相关的住院风险进行了量化:其中,9232 例(72%)为 mBC 患者(中位年龄:59 岁,54.6% 至少患有一种并发症),3590 例(28%)为 esBC 患者(中位年龄:54 岁,33.9% 至少患有一种并发症)。在间变性乳腺癌患者中,中位总生存期为 32.6 个月,在过去几年中显示出逐渐改善的趋势。1年后,75.8%的患者停止了治疗,22.0%的患者死亡。与乳腺癌患者相比,研究中的患者面临更高的住院风险,血小板减少症的住院风险分别增加了 5.7 倍和 10.5 倍,间质性肺病的住院风险分别增加了 3.0 倍和 4.5 倍,急性肝损伤的住院风险分别增加了 3.3 倍和 5.9 倍,败血症的住院风险分别增加了 2.3 倍和 7.3 倍。结论现实世界中的 T-DM1 使用者经常出现合并症和既往治疗,因严重毒性事件住院的风险高于一般乳腺癌患者。这项研究强调了对接受治疗的患者进行监测以控制毒性风险和防止治疗中断的重要性。
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引用次数: 0
Real-world effectiveness of post-trastuzumab emtansine treatment for human epidermal growth factor receptor 2-positive metastatic breast cancer: a multicenter, matched cohort analysis from the Epidemiology Strategy and Medical Economics database (2008-2018) 人表皮生长因子受体 2 阳性转移性乳腺癌曲妥珠单抗埃坦单抗后治疗的实际效果:来自流行病学战略和医疗经济学数据库(2008-2018 年)的多中心匹配队列分析
Pub Date : 2024-06-01 DOI: 10.1016/j.esmorw.2024.100043
C. Courtinard , V. Barbet , R. Schiappa , F. Pilleul , S. Michiels , S. Dabakuyo , S. Gourgou , A. Jaffre , B. Asselain , L. Bosquet , K. Dunton , M. Rosenlund , Z. Liang , J. Cathcart , S. Delaloge

Background

Real-world data (RWD) can contextualize clinical trial data. We present real-world evidence that supplemented the single-arm DESTINY-Breast01 trial, which assessed the efficacy and safety of trastuzumab deruxtecan (T-DXd) in patients with human epidermal growth factor receptor 2-positive (HER2+) metastatic breast cancer (mBC) previously treated with trastuzumab emtansine (T-DM1).

Patients and methods

Patients from the French Epidemiology Strategy and Medical Economics (ESME) mBC database who initiated treatment for HER2+ mBC between 1 January 2008 and 31 December 2016, and received one or more treatment lines following T-DM1 were propensity score matched 1 : 1 to patients from DESTINY-Breast01 to create an ESME DB-01 matched cohort. Treatment patterns, real-world best overall response, real-world objective response rate (rwORR), real-world disease control rate (rwDCR), real-world progression-free survival (rwPFS), and overall survival (OS) were estimated, including by prior pertuzumab exposure and de novo/relapsed mBC status.

Results

A total of 137 patients from the ESME mBC database (78 received prior pertuzumab, 59 did not) were matched to 137 patients from DESTINY-Breast01. In the ESME DB-01 matched cohort, 73.7% received an anti-HER2 drug after T-DM1 treatment. The rwORR was 12.2% (95% confidence interval [CI] 6.2% to 18.2%; only partial responses) and rwDCR was 73.9% (95% CI 65.9% to 81.9%). The median rwPFS was 4.7 months (95% CI 3.8-6.0 months) and similar regardless of prior pertuzumab exposure or de novo/relapsed mBC status. The median OS was 24.1 months (95% CI 18.5-26.4 months) and longer in patients naive to versus exposed to pertuzumab and in patients with de novo versus relapsed mBC.

Conclusion

These RWD contextualized results of DESTINY-Breast01 and demonstrated an unmet medical need in patients with HER2+ mBC after T-DM1 treatment.

背景真实世界数据(RWD)可以为临床试验数据提供背景信息。该试验评估了曲妥珠单抗德鲁司坦(T-DXd)在既往接受过曲妥珠单抗恩坦辛(T-DM1)治疗的人表皮生长因子受体 2 阳性(HER2+)转移性乳腺癌(mBC)患者中的疗效和安全性。患者和方法将法国流行病学战略和医疗经济学(ESME)mBC数据库中2008年1月1日至2016年12月31日期间开始接受HER2+ mBC治疗、在T-DM1治疗后接受过一次或多次治疗的患者与DESTINY-Breast01中的患者进行倾向评分1:1匹配,以创建ESME DB-01匹配队列。对治疗模式、真实世界最佳总反应、真实世界客观反应率(rwORR)、真实世界疾病控制率(rwDCR)、真实世界无进展生存期(rwPFS)和总生存期(OS)进行了估算,包括按既往接受过百妥珠单抗治疗和新发/复发 mBC 状态进行估算。在ESME DB-01匹配队列中,73.7%的患者在T-DM1治疗后接受了抗HER2药物治疗。rwORR为12.2%(95%置信区间[CI] 6.2%至18.2%;仅为部分反应),rwDCR为73.9%(95%置信区间[CI] 65.9%至81.9%)。中位rwPFS为4.7个月(95% CI为3.8-6.0个月),与之前服用过百妥珠单抗或新发/复发mBC的情况相似。中位OS为24.1个月(95% CI为18.5-26.4个月),在未使用过与使用过百妥珠单抗的患者中,以及在新发与复发的mBC患者中,OS时间更长。
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引用次数: 0
Real-world behavioral practices of cancer patients: misconceptions compromising daily life activities 癌症患者的真实世界行为实践:影响日常生活活动的错误观念
Pub Date : 2024-05-13 DOI: 10.1016/j.esmorw.2024.100041
E. Shachar , S. Peleg-Hasson , D. Vorobiof , N. Moisa , E. Waller , T. Safra , I. Wolf

Background

Health-related quality of life is commonly used as an endpoint in clinical trials. While it evaluates the presence of symptoms, it does not measure patients’ ability to maintain normal daily life activities (DLA). We designed a daily life assessment tool, validated among actively treated cancer patients at a single center before the coronavirus 2019 pandemic. We discovered that most patients reported compromised daily activities. In this study we aimed to examine DLA in an international cohort.

Methods

A locally validated questionnaire was distributed internationally using the Belong.life digital health platform. We examined real-world patient-reported practices. The survey consisted of demographic, clinical, behavioral parameters and sources guiding and supporting patient practices.

Results

The study comprised 1395 patient-reported outcomes. The majority of participants (1005, 73%) reported at least one adopted limitation in daily activities, and 305 (22%) maintained more than half of these constraints. Daily life restrictions included avoiding sun exposure (779, 58%), international travel (417, 33%), indoor public places (431, 33%), hair dyeing (271, 23%), domestic tourism (284, 22%), contact with friends and family (231, 18%), children and grandchildren (202, 16%), public spaces (190, 14.62%), and contact with pets (135, 10%). Multiple sources were implicated by patients guiding their behavior, including healthcare professionals (951, 66%), non-medical authorities [(internet, patient forums, partners, friends, and family (171, 12%)], and both non-medical authorities and the healthcare team (320, 22.19%). There was no association between country of origin (P = 0.12), and education level (P = 0.36) across patients who maintained strict (≥50% of the limitations) and less strict restrictions (<50% of the limitations). A significant association was noted between younger age (P = 0.001), female sex (P = 0.01) and primary cancer site (P < 0.0001), and the adoption of strict restrictions.

Conclusion

The majority of patients globally reported compromised daily activities, which are likely attributed to misconceptions about therapy and disease. These findings call for the assessment of an overlooked measure, DLA reflecting real-life quality of life, as an additional endpoint of clinical trials, aiming to achieve the ultimate benefit for our patients, a measure of a full and meaningful life.

背景健康相关的生活质量通常被用作临床试验的终点。虽然它能评估症状的存在,但不能衡量患者维持正常日常生活活动(DLA)的能力。我们设计了一种日常生活评估工具,并在 2019 年冠状病毒大流行之前在一个中心对积极接受治疗的癌症患者进行了验证。我们发现,大多数患者的日常生活活动受到了影响。在这项研究中,我们旨在对国际队列中的 DLA 进行检查。方法 我们使用 Belong.life 数字健康平台在国际范围内分发了经过当地验证的调查问卷。我们研究了真实世界中患者报告的实践。调查内容包括人口统计学、临床、行为参数以及指导和支持患者实践的来源。大多数参与者(1005 人,占 73%)报告在日常活动中至少采用了一种限制,其中 305 人(占 22%)保持了一半以上的限制。日常生活限制包括避免日晒(779 人,58%)、国际旅行(417 人,33%)、室内公共场所(431 人,33%)、染发(271 人,23%)、国内旅游(284 人,22%)、与朋友和家人接触(231 人,18%)、与子女和孙辈接触(202 人,16%)、公共场所(190 人,14.62%)以及与宠物接触(135 人,10%)。指导患者行为的来源有多种,包括医护人员(951 人,66%)、非医疗机构[(互联网、患者论坛、伴侣、朋友和家人(171 人,12%)],以及非医疗机构和医护团队(320 人,22.19%)。在保持严格限制(≥50% 的限制)和不太严格限制(50% 的限制)的患者中,原籍国(P = 0.12)和受教育程度(P = 0.36)之间没有关联。年轻(P = 0.001)、女性(P = 0.01)和原发癌症部位(P <0.0001)与采取严格限制之间存在明显关联。这些研究结果呼吁评估一项被忽视的指标,即反映现实生活质量的 DLA,作为临床试验的额外终点,以实现患者的最终获益,即衡量充实而有意义的生活。
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引用次数: 0
Evaluating GPT-4 as an academic support tool for clinicians: a comparative analysis of case records from the literature 评估作为临床医生学术支持工具的 GPT-4:文献中病例记录的比较分析
Pub Date : 2024-05-13 DOI: 10.1016/j.esmorw.2024.100042
B.L. Fabre , M.A.F. Magalhaes Filho , P.N. Aguiar Jr , F.M. da Costa , B. Gutierres , W.N. William Jr , A. Del Giglio

Background

Artificial intelligence (AI) and natural language processing (NLP) advancements have led to sophisticated tools like GPT-4.0, allowing clinicians to explore its utility as a health care management support tool. Our study aimed to assess the capability of GPT-4 in suggesting definitive diagnoses and appropriate work-ups to minimize unnecessary procedures.

Materials and methods

We conducted a retrospective comparative analysis, extracting clinical data from 10 cases published in the New England Journal of Medicine after 2022 and inputting this data into GPT-4 to generate diagnostic and work-up recommendations. Primary endpoint: the ability to correctly identify the final diagnosis. Secondary endpoints: its ability to list the definitive diagnosis as the first of the five most likely differential diagnoses and determine an adequate work-up.

Results

The AI could not identify the definitive diagnosis in 2 out of 10 cases (20% inaccuracy). Among the eight cases correctly identified by the AI, five (63%) listed the definitive diagnosis at the top of the differential diagnosis list. In terms of diagnostic tests and exams, the AI suggested unnecessary procedures in two cases, representing 40% of the cases where it failed to correctly identify the final diagnosis. Moreover, the AI could not suggest adequate treatment for seven cases (70%). Among them, the AI suggested inappropriate management for two cases, and the remaining five received incomplete or non-specific advice, such as chemotherapy, without specifying the best regimen.

Conclusions

Our study demonstrated GPT-4’s potential as an academic support tool, although it cannot correctly identify the final diagnosis in 20% of the cases and the AI requested unnecessary additional diagnostic tests for 40% of the patients. Future research should focus on evaluating the performance of GPT-4 using a more extensive and diverse sample, incorporating prospective assessments, and investigating its ability to improve diagnostic and therapeutic accuracy.

背景人工智能(AI)和自然语言处理(NLP)的进步催生了像 GPT-4.0 这样的先进工具,使临床医生能够探索其作为医疗保健管理支持工具的效用。我们的研究旨在评估GPT-4在建议明确诊断和适当检查以尽量减少不必要手术方面的能力。材料与方法我们进行了一项回顾性比较分析,从2022年以后发表在《新英格兰医学杂志》上的10个病例中提取临床数据,并将这些数据输入GPT-4,以生成诊断和检查建议。主要终点:正确识别最终诊断的能力。次要终点:将明确诊断列为五个最可能的鉴别诊断之首,并确定充分工作检查的能力。结果在 10 个病例中,人工智能有 2 个无法确定明确诊断(不准确率为 20%)。在人工智能正确识别的 8 个病例中,有 5 个病例(63%)将明确诊断列在了鉴别诊断列表的首位。在诊断化验和检查方面,人工智能在两个病例中建议了不必要的程序,占人工智能未能正确确定最终诊断的病例的 40%。此外,有 7 个病例(70%)的人工智能无法提出适当的治疗建议。结论我们的研究证明了 GPT-4 作为学术支持工具的潜力,尽管它不能正确识别 20% 病例的最终诊断,而且人工智能要求对 40% 的患者进行不必要的额外诊断检测。未来的研究应侧重于使用更广泛、更多样的样本来评估 GPT-4 的性能,纳入前瞻性评估,并研究其提高诊断和治疗准确性的能力。
{"title":"Evaluating GPT-4 as an academic support tool for clinicians: a comparative analysis of case records from the literature","authors":"B.L. Fabre ,&nbsp;M.A.F. Magalhaes Filho ,&nbsp;P.N. Aguiar Jr ,&nbsp;F.M. da Costa ,&nbsp;B. Gutierres ,&nbsp;W.N. William Jr ,&nbsp;A. Del Giglio","doi":"10.1016/j.esmorw.2024.100042","DOIUrl":"https://doi.org/10.1016/j.esmorw.2024.100042","url":null,"abstract":"<div><h3>Background</h3><p>Artificial intelligence (AI) and natural language processing (NLP) advancements have led to sophisticated tools like GPT-4.0, allowing clinicians to explore its utility as a health care management support tool. Our study aimed to assess the capability of GPT-4 in suggesting definitive diagnoses and appropriate work-ups to minimize unnecessary procedures.</p></div><div><h3>Materials and methods</h3><p>We conducted a retrospective comparative analysis, extracting clinical data from 10 cases published in the <em>New England Journal of Medicine</em> after 2022 and inputting this data into GPT-4 to generate diagnostic and work-up recommendations. Primary endpoint: the ability to correctly identify the final diagnosis. Secondary endpoints: its ability to list the definitive diagnosis as the first of the five most likely differential diagnoses and determine an adequate work-up.</p></div><div><h3>Results</h3><p>The AI could not identify the definitive diagnosis in 2 out of 10 cases (20% inaccuracy). Among the eight cases correctly identified by the AI, five (63%) listed the definitive diagnosis at the top of the differential diagnosis list. In terms of diagnostic tests and exams, the AI suggested unnecessary procedures in two cases, representing 40% of the cases where it failed to correctly identify the final diagnosis. Moreover, the AI could not suggest adequate treatment for seven cases (70%). Among them, the AI suggested inappropriate management for two cases, and the remaining five received incomplete or non-specific advice, such as chemotherapy, without specifying the best regimen.</p></div><div><h3>Conclusions</h3><p>Our study demonstrated GPT-4’s potential as an academic support tool, although it cannot correctly identify the final diagnosis in 20% of the cases and the AI requested unnecessary additional diagnostic tests for 40% of the patients. Future research should focus on evaluating the performance of GPT-4 using a more extensive and diverse sample, incorporating prospective assessments, and investigating its ability to improve diagnostic and therapeutic accuracy.</p></div>","PeriodicalId":100491,"journal":{"name":"ESMO Real World Data and Digital Oncology","volume":"4 ","pages":"Article 100042"},"PeriodicalIF":0.0,"publicationDate":"2024-05-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2949820124000201/pdfft?md5=f32f81c8fc771b7987718bc67be461a8&pid=1-s2.0-S2949820124000201-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140924464","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Changes in survival of >3800 patients with metastatic colorectal cancer in Germany: results from a 16-year prospective longitudinal real-world data analysis 德国 3800 多名转移性结直肠癌患者的生存率变化:16 年前瞻性纵向真实世界数据分析结果
Pub Date : 2024-05-07 DOI: 10.1016/j.esmorw.2024.100040
N. Marschner , T. Seufferlein , K. Potthoff , M.-O. Zahn , J. Uhlig , S. Dörfel , A. Karcher , A. Sauer , C. Maintz , S. Fruehauf , U. Hutzschenreuter , S. Tech , M. Grafetstätter , L. Kruggel , M. Jänicke

Background

Results from recent clinical trials, showing median survival times of >30 months for patients with metastatic colorectal cancer (mCRC), set a new benchmark for first-line treatment. As, however, most patients are treated outside of trials, evidence from population-based studies is warranted to evaluate whether survival times translate to real world.

Patients and methods

Data on treatment and outcome of 3816 patients with mCRC, observed between 2006 and 2022, were collected from 166 sites in Germany into the prospective Tumor Registry Colorectal Cancer. Data were analyzed according to start of first-line palliative treatment, divided into three time periods (2006-2010, 2011-2014 and 2015-2018). Overall survival (OS) was estimated using the Kaplan–Meier method.

Results

Most patients received doublet chemotherapy (CTx) across all time periods (about 82%). The use of triplet combination CTx increased over time from 0.9% (2006-2010), over 1.3% (2011-2014) to 5.6% (2015-2018). More patients received anti-epidermal growth factor receptor antibodies over time (2006-2010: 6.5%; 2011-2014: 18.7%; 2015-2018: 25.3%). Median OS for patients who started palliative treatment between 2006 and 2010, 2011 and 2014 and 2015 and 2018 was 21.4 months [95% confidence interval (CI) 20.3-23.1 months], 21.9 months (95% CI 20.8-23.4 months) and 22.9 months (95% CI 21.8-24.9 months), respectively.

Conclusion

Since the successful introduction of doublet chemotherapy with monoclonal antibodies, median OS of patients with mCRC in clinical practice remained unchanged at about 22 months over a period of 16 years. As such, our data highlight the need for new treatment options to further improve survival of patients with mCRC.

背景最近的临床试验结果显示,转移性结直肠癌(mCRC)患者的中位生存时间为30个月,为一线治疗树立了新的标杆。然而,由于大多数患者是在试验之外接受治疗的,因此有必要通过基于人群的研究来评估生存时间是否与实际情况相符。患者和方法在前瞻性肿瘤登记结直肠癌中收集了德国166个地点的数据,其中包括2006年至2022年间观察到的3816名mCRC患者的治疗情况和结果。数据根据一线姑息治疗的开始时间进行分析,分为三个时间段(2006-2010 年、2011-2014 年和 2015-2018 年)。结果在所有时间段内,大多数患者都接受了双联化疗(CTx)(约占82%)。随着时间的推移,三联联合化疗(CTx)的使用率从0.9%(2006-2010年)、1.3%(2011-2014年)增至5.6%(2015-2018年)。随着时间的推移,更多患者接受了抗表皮生长因子受体抗体治疗(2006-2010年:6.5%;2011-2014年:18.7%;2015-2018年:25.3%)。2006年至2010年、2011年至2014年、2015年至2018年期间开始接受姑息治疗的患者的中位OS分别为21.4个月[95%置信区间(CI)20.3-23.1个月]、21.9个月(95% CI 20.8-23.4个月)和22.9个月(95% CI 21.8-24.9个月)。结论自成功引入单克隆抗体双联化疗以来,16年间临床实践中mCRC患者的中位OS一直保持在22个月左右。因此,我们的数据突出表明,需要新的治疗方案来进一步提高mCRC患者的生存率。
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引用次数: 0
Evaluation of machine learning methods for the retrospective detection of ovarian cancer recurrences from chemotherapy data 评估从化疗数据中回顾性检测卵巢癌复发的机器学习方法
Pub Date : 2024-05-01 DOI: 10.1016/j.esmorw.2024.100038
A.D. Coles , C.D. McInerney , K. Zucker , S. Cheeseman , O.A. Johnson , G. Hall

Background

Cancer recurrences are poorly recorded within electronic health records around the world. This hinders research into the efficacy of cancer treatments. Currently, the retrospective identification of recurrence/progression diagnosis dates is achieved by staff who manually review patients’ health records. This is expensive, time-consuming, and inefficient. Machine Learning models may expedite the review of health records and facilitate the assessment of alternative cancer therapies.

Materials and methods

This paper evaluates the use of four machine learning models (random forests, conditional inference trees, decision trees, and logistic regression) in identifying proxy dates of epithelial ovarian cancer recurrence/progression from chemotherapy data, in 531 patients at Leeds Teaching Hospital Trust.

Results

The random forest achieved the highest F1 score of 0.941 (95% confidence interval 0.916-0.968) when identifying recurrence events. Both the random forest and decision tree models’ classifications closely conform to chart-reviewed time to next treatment, serving as a surrogate for recurrence-free survival. Additionally, all models reached an F1 score >0.940 when identifying patients whose cancer recurred/progressed.

Conclusions

Our models proficiently identify both proxy dates for recurrence/progression diagnoses and patients whose cancer recurred/progressed. Considering the similar performance of the random forest and decision tree, model preference should be determined by the interpretability required to assist chart review and the ease of implementation into existing architecture.

背景世界各地的电子健康记录中对癌症复发的记录很少。这阻碍了对癌症治疗效果的研究。目前,复发/进展诊断日期的回顾性识别是由工作人员手动查看患者的健康记录来实现的。这种方法成本高、耗时长、效率低。材料与方法本文评估了四种机器学习模型(随机森林、条件推理树、决策树和逻辑回归)在识别利兹教学医院信托基金 531 名患者化疗数据中上皮性卵巢癌复发/进展替代日期方面的应用。随机森林模型和决策树模型的分类结果与图表显示的下次治疗时间非常吻合,可作为无复发生存期的替代指标。此外,在识别癌症复发/进展患者时,所有模型的 F1 分数都达到了 0.940。考虑到随机森林和决策树的性能相似,应根据协助病历审查所需的可解释性以及在现有架构中实施的难易程度来决定是否选择模型。
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引用次数: 0
Health-related quality of life in patients with gastrointestinal stromal tumor: data from a real-world cohort compared with a normative population 胃肠道间质瘤患者的健康相关生活质量:来自真实世界队列的数据与常模人群的比较
Pub Date : 2024-04-24 DOI: 10.1016/j.esmorw.2024.100037
D. van de Wal , D. den Hollander , I.M.E. Desar , H. Gelderblom , A.W. Oosten , A.K.L. Reyners , N. Steeghs , W.T.A. van der Graaf , O. Husson

Background

Treatment and follow-up (FU) care procedures for gastrointestinal stromal tumors (GISTs) impose great challenges on patients and could potentially affect their health-related quality of life (HRQoL). The aims of our study were to (i) assess HRQoL among patients with GIST in different treatment phases and settings and to compare this with the HRQoL of an age- and sex-matched normative population, (ii) determine the occurrence of disease- and treatment-specific symptoms, and (iii) investigate which sociodemographic and clinical characteristics and symptoms were associated with HRQoL.

Methods

A total of 328 Dutch patients with GIST (response rate 63%) completed a one-time survey including the European Organization for Research and Treatment of Cancer Quality of Life Core Questionnaire (EORTC QLQ-C30), which was used to assess HRQoL. HRQoL scores are presented as means and standard deviations (mean ± SD), and were compared with those of an age- and sex-matched normative population.

Results

The global QoL of patients receiving imatinib in a curative setting (mean ± SD 81.2 ± 12.6) was comparable with the normative population (mean ± SD 77.1 ± 18.2), while patients who had completed their curative treatment [including those discharged from FU (mean ± SD 85.2 ± 14.0) and still in FU (mean ± SD 82.7 ± 15.0)] had a significant better global QoL with comparable functioning scores. Patients on tyrosine kinase inhibitors in a palliative setting scored significantly lower on global QoL (mean ± SD 71.6 ± 19.4) and all functioning scales compared with the normative population. HRQoL was most affected by fatigue, in addition to pain, dyspnea, and financial difficulties, which all occurred more often in patients treated in a palliative setting compared with patients in the curative setting.

Conclusion

With these results, medical oncologists can reassure patients with GIST treated in an adjuvant setting that their HRQoL will not be permanently affected by imatinib and provide appropriate support to patients in the palliative setting.

背景胃肠道间质瘤(GIST)的治疗和后续(FU)护理程序给患者带来了巨大挑战,并可能影响他们的健康相关生活质量(HRQoL)。我们的研究目的是:(i) 评估不同治疗阶段和环境下 GIST 患者的 HRQoL,并将其与年龄和性别匹配的标准人群的 HRQoL 进行比较;(ii) 确定疾病和治疗特异性症状的发生情况;(iii) 调查哪些社会人口学和临床特征及症状与 HRQoL 相关。方法 共有 328 名荷兰 GIST 患者(回复率为 63%)完成了一次性调查,包括欧洲癌症研究和治疗组织生活质量核心问卷(EORTC QLQ-C30),该问卷用于评估 HRQoL。HRQoL得分以平均值和标准差(平均值±标准差)表示,并与年龄和性别匹配的常模人群进行比较。6)与常模人群(平均± SD 77.1 ± 18.2)相当,而完成治愈性治疗的患者[包括出院(平均± SD 85.2 ± 14.0)和仍在治疗(平均± SD 82.7 ± 15.0)]的总体 QoL 显著提高,功能评分相当。与常模人群相比,服用酪氨酸激酶抑制剂的姑息治疗患者的总体QoL(平均值(± SD)71.6 ± 19.4)和所有功能量表得分明显较低。除疼痛、呼吸困难和经济困难外,对HRQoL影响最大的是疲劳,与治愈性治疗患者相比,姑息性治疗患者的疲劳发生率更高。结论根据这些结果,肿瘤内科医生可以向辅助治疗的GIST患者保证,伊马替尼不会永久性地影响他们的HRQoL,并为姑息性治疗患者提供适当的支持。
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引用次数: 0
Toward optimizing patient selection for EGFR antibody therapies in metastatic colorectal cancer: outcomes and resistance features in real-world data 优化转移性结直肠癌表皮生长因子受体(EGFR)抗体疗法的患者选择:真实世界数据中的疗效和耐药性特征
Pub Date : 2024-04-15 DOI: 10.1016/j.esmorw.2024.100036
M.J. Emmett , J.C.F. Quintanilha , R.P. Graf , G. Li , H. Tukachinsky , A.B. Schrock , S. Morley , V.A. Fisher , G.R. Oxnard , C.H. Lieu , P.A. Myer , S.J. Klempner

Background

Patients with metastatic colorectal cancer (mCRC) with RAS- or BRAF-mutant tumors do not benefit from epidermal growth factor receptor (EGFR) monoclonal antibody (mAb) therapy. Among patients with RAS/BRAF wild-type (WT) tumors, a substantial portion still do not benefit from EGFR mAb treatment. Using real-world clinicogenomic data, we investigated the impact of primary and acquired genomic resistance alterations upon treatment outcomes and determined the prevalence of alterations before and after EGFR mAb treatment.

Materials and methods

This study utilized a de-identified mCRC clinicogenomic database from ∼280 US cancer clinics between March 2014 and April 2023. We examined real-world progression-free survival (rwPFS) and overall survival (rwOS) between patients with and those without pre-specified genomic alterations (PSGAs) by Cox models and an adjusted risk score. Genomic alterations were also compared between samples collected before and after EGFR mAb therapy.

Results

Nearly, one-third of microsatellite stable (MSS) RAS/BRAF WT tumors harbor intrinsic resistance alterations before treatment. MSS mCRC patients with WT RAS/BRAF tumors having resistance alterations within the PSGA set [non-canonical RAS/RAF/MAPK and PI3K/PTEN/AKT pathway components; ERBB2 alterations; alternative receptor tyrosine kinases (RTKs) including FGFR1, FGFR2, EGFR, MET, RET, PDGFRA, and NRTK1 fusion] demonstrated decreased rwPFS and/or rwOS on first-line EGFR mAb treatment. The prevalence of RAS/RAF/MAPK and RTK alterations was higher in samples collected after EGFR mAb therapy. The risk of acquiring an RTK resistance alteration increased with the total duration of EGFR mAb treatment.

Conclusions

Detection of genomic resistance alterations in MSS RAS/BRAF WT patients confers less favorable EGFR mAb treatment outcomes. The duration of EGFR mAb treatment increased the risk of emergence of an acquired resistance alteration.

背景RAS或BRAF突变的转移性结直肠癌(mCRC)患者无法从表皮生长因子受体(EGFR)单克隆抗体(mAb)治疗中获益。在RAS/BRAF野生型(WT)肿瘤患者中,仍有相当一部分患者无法从表皮生长因子受体单克隆抗体(EGFR mAb)治疗中获益。利用真实世界的临床基因组学数据,我们调查了原发性和获得性基因组耐药改变对治疗结果的影响,并确定了EGFR mAb治疗前后耐药改变的发生率。我们通过 Cox 模型和调整后的风险评分,研究了有和没有预先指定的基因组改变(PSGAs)的患者之间的实际无进展生存期(rwPFS)和总生存期(rwOS)。结果近三分之一的微卫星稳定(MSS)RAS/BRAF WT肿瘤在治疗前存在内在耐药改变。具有WT RAS/BRAF肿瘤的MSS mCRC患者在接受PSGA组[非典型RAS/RAF/MAPK和PI3K/PTEN/AKT通路成分;ERBB2改变;替代受体酪氨酸激酶(RTKs),包括FGFR1、FGFR2、EGFR、MET、RET、PDGFRA和NRTK1融合]治疗后的rwPFS和/或rwOS下降。在表皮生长因子受体 mAb 治疗后采集的样本中,RAS/RAF/MAPK 和 RTK 基因改变的发生率较高。获得RTK耐药改变的风险随着表皮生长因子受体mAb治疗总持续时间的延长而增加。表皮生长因子受体 mAb 治疗的持续时间增加了出现获得性耐药改变的风险。
{"title":"Toward optimizing patient selection for EGFR antibody therapies in metastatic colorectal cancer: outcomes and resistance features in real-world data","authors":"M.J. Emmett ,&nbsp;J.C.F. Quintanilha ,&nbsp;R.P. Graf ,&nbsp;G. Li ,&nbsp;H. Tukachinsky ,&nbsp;A.B. Schrock ,&nbsp;S. Morley ,&nbsp;V.A. Fisher ,&nbsp;G.R. Oxnard ,&nbsp;C.H. Lieu ,&nbsp;P.A. Myer ,&nbsp;S.J. Klempner","doi":"10.1016/j.esmorw.2024.100036","DOIUrl":"https://doi.org/10.1016/j.esmorw.2024.100036","url":null,"abstract":"<div><h3>Background</h3><p>Patients with metastatic colorectal cancer (mCRC) with <em>RAS</em><em>-</em> or <em>BRAF</em>-mutant tumors do not benefit from epidermal growth factor receptor (EGFR) monoclonal antibody (mAb) therapy. Among patients with <em>RAS/BRAF</em> wild-type (WT) tumors, a substantial portion still do not benefit from EGFR mAb treatment. Using real-world clinicogenomic data, we investigated the impact of primary and acquired genomic resistance alterations upon treatment outcomes and determined the prevalence of alterations before and after EGFR mAb treatment.</p></div><div><h3>Materials and methods</h3><p>This study utilized a de-identified mCRC clinicogenomic database from ∼280 US cancer clinics between March 2014 and April 2023. We examined real-world progression-free survival (rwPFS) and overall survival (rwOS) between patients with and those without pre-specified genomic alterations (PSGAs) by Cox models and an adjusted risk score. Genomic alterations were also compared between samples collected before and after EGFR mAb therapy.</p></div><div><h3>Results</h3><p>Nearly, one-third of microsatellite stable (MSS) <em>RAS/BRAF</em> WT tumors harbor intrinsic resistance alterations before treatment. MSS mCRC patients with WT <em>RAS/BRAF</em> tumors having resistance alterations within the PSGA set [non-canonical RAS/RAF/MAPK and PI3K/PTEN/AKT pathway components; ERBB2 alterations; alternative receptor tyrosine kinases (RTKs) including <em>FGFR1</em>, <em>FGFR2</em>, <em>EGFR</em>, <em>MET</em>, <em>RET</em>, <em>PDGFRA</em>, <em>and NRTK1</em> fusion] demonstrated decreased rwPFS and/or rwOS on first-line EGFR mAb treatment. The prevalence of RAS/RAF/MAPK and RTK alterations was higher in samples collected after EGFR mAb therapy. The risk of acquiring an RTK resistance alteration increased with the total duration of EGFR mAb treatment.</p></div><div><h3>Conclusions</h3><p>Detection of genomic resistance alterations in MSS <em>RAS/BRAF</em> WT patients confers less favorable EGFR mAb treatment outcomes. The duration of EGFR mAb treatment increased the risk of emergence of an acquired resistance alteration.</p></div>","PeriodicalId":100491,"journal":{"name":"ESMO Real World Data and Digital Oncology","volume":"4 ","pages":"Article 100036"},"PeriodicalIF":0.0,"publicationDate":"2024-04-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2949820124000146/pdfft?md5=0dfbceb2f2a77c9b421cd2bce220ba31&pid=1-s2.0-S2949820124000146-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140554843","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Commentary to Corti et al.: Exploring the utility and limitations of ChatGPT in scientific literature searches 对 Corti 等人的评论:探索 ChatGPT 在科学文献检索中的实用性和局限性
Pub Date : 2024-03-01 DOI: 10.1016/j.esmorw.2024.100028
K.S. Olsen , M. Lukic
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引用次数: 0
Impact of artificial intelligence in transforming the doctor–cancer patient relationship 人工智能对改变医患关系的影响
Pub Date : 2024-03-01 DOI: 10.1016/j.esmorw.2024.100026
P.-E. Heudel , H. Crochet , J.-Y. Blay

Background

The integration of Artificial Intelligence (AI) in oncology is a developing field, impacting the doctor-patient relationship and the efficacy of cancer care. While AI’s role in improving clinical efficiency and personalized care through the analysis of vast medical datasets is acknowledged, its full scope and impact are not yet completely understood.

Materials and methods

This article synthesizes empirical studies and expert opinions to offer a comprehensive understanding of AI’s current role in oncology. The methodology focuses on exploring the balance between technological advancements and the essential elements of patient-centered care.

Results

The paper hypothesizes that AI can enhance the quality of cancer care, but notes challenges such as potential depersonalization, data privacy issues, and ethical dilemmas. It also highlights AI’s potential in facilitating Shared Decision-Making, empowering patients and assisting oncologists in making more informed decisions. However, the risk of AI-driven paternalism and the need for balancing AI recommendations with patient autonomy are discussed.

Conclusions

AI holds significant potential to transform cancer care. The paper concludes that for AI to be beneficial, its integration should be collaborative and patient-centered, ensuring that technological advancements support and enhance the quality of the doctor-patient relationship, rather than undermining it. The article emphasizes the importance of transparent communication, patient education about AI, and the need for oncologists to effectively understand and convey AI-generated data.

背景人工智能(AI)与肿瘤学的结合是一个不断发展的领域,它影响着医患关系和癌症治疗的效果。虽然人工智能通过分析庞大的医疗数据集在提高临床效率和个性化护理方面的作用已得到认可,但其全部范围和影响尚未被完全理解。结果本文假设人工智能可以提高癌症护理的质量,但也指出了一些挑战,如潜在的人格解体、数据隐私问题和伦理困境。本文还强调了人工智能在促进 "共同决策"、增强患者能力和协助肿瘤学家做出更明智决策方面的潜力。然而,本文也讨论了人工智能驱动的家长作风的风险以及平衡人工智能建议与患者自主权的必要性。本文总结道,要想让人工智能带来益处,就应该以合作和患者为中心进行整合,确保技术进步能够支持和提高医患关系的质量,而不是破坏医患关系。文章强调了透明沟通、对患者进行人工智能教育的重要性,以及肿瘤学家有效理解和传达人工智能生成数据的必要性。
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引用次数: 0
期刊
ESMO Real World Data and Digital Oncology
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