ESMO Real World Data and Digital Oncology最新文献

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Prevalence, treatment response, and survival in a real-world NTRK gene fusion-positive microsatellite instability-high metastatic colorectal cancer cohort NTRK基因融合阳性微卫星不稳定性-高转移性结直肠癌队列的患病率、治疗反应和生存率
ESMO Real World Data and Digital Oncology Pub Date : 2025-12-01 Epub Date: 2025-09-12 DOI: 10.1016/j.esmorw.2025.100180
S.J. Schraa , M.M. Laclé , K. Zwart , E.H. Gort , W.W. de Leng , M. Koopman , G.R. Vink , G.M. Bol , PLCRC Working Group
{"title":"Prevalence, treatment response, and survival in a real-world NTRK gene fusion-positive microsatellite instability-high metastatic colorectal cancer cohort","authors":"S.J. Schraa ,&nbsp;M.M. Laclé ,&nbsp;K. Zwart ,&nbsp;E.H. Gort ,&nbsp;W.W. de Leng ,&nbsp;M. Koopman ,&nbsp;G.R. Vink ,&nbsp;G.M. Bol ,&nbsp;PLCRC Working Group","doi":"10.1016/j.esmorw.2025.100180","DOIUrl":"10.1016/j.esmorw.2025.100180","url":null,"abstract":"<div><h3>Background</h3><div>Tropomyosin receptor kinase (TRK) inhibitors are approved for patients with neurotrophic tyrosine receptor kinase (<em>NTRK</em>) fusion-positive solid tumors, but clinical benefit in metastatic colorectal cancer (mCRC) is uncertain. We aimed to determine the prevalence, treatment response, and overall survival of <em>NTRK</em> fusion-positive, microsatellite instability-high (MSI-H), or mismatch repair-deficient (dMMR) mCRC patients in a real-world cohort.</div></div><div><h3>Materials and methods</h3><div>Tumor tissue was collected for all patients diagnosed with MSI-H/dMMR mCRC between 2015 and 2021 in the Netherlands. Pan-TRK immunohistochemistry was carried out, and positive cases were assessed for <em>NTRK</em> fusions by RNA-based next-generation sequencing. Clinical data on treatment duration and overall survival were obtained from the Netherlands Cancer Registry.</div></div><div><h3>Results</h3><div>Nine out of 268 MSI-H/dMMR colorectal tumors (3.4%) harbored an <em>NTRK</em> fusion. All <em>NTRK</em> fusion-positive tumors were <em>RAS</em>/<em>BRAF</em><sup><em>V600E</em></sup> wild-type. In the subgroup of <em>RAS</em>/<em>BRAF</em><sup><em>V600E</em></sup> wild-type MSI-H/dMMR patients, the prevalence of <em>NTRK</em> fusions was 22%. The benefit of chemotherapy (2/7) and anti-epidermal growth factor receptor (EGFR) therapy (0/2) in <em>NTRK</em> fusion-positive patients was limited. All four patients treated with programmed death-(ligand) 1 [PD-(L)1] inhibitors had sustained responses and a minimum overall survival of 36 months.</div></div><div><h3>Conclusions</h3><div>In this real-world cohort study, <em>NTRK</em> fusions are most prevalent in <em>RAS</em>/<em>BRAF</em><sup><em>V600E</em></sup> wild-type MSI-H/dMMR mCRC patients. This is the first study to suggest resistance to chemotherapy and anti-EGFR therapy in <em>NTRK</em> fusion-positive tumors, leading to impaired overall survival. In contrast, PD-(L)1 inhibitors seem effective in this population. Therapeutic strategies regarding TRK inhibitors versus PD-(L)1 inhibitors need investigation. We recommend early testing for <em>NTRK</em> fusions in <em>RAS</em> and <em>BRAF</em><sup><em>V600E</em></sup> wild-type MSI-H/dMMR mCRC patients.</div></div>","PeriodicalId":100491,"journal":{"name":"ESMO Real World Data and Digital Oncology","volume":"10 ","pages":"Article 100180"},"PeriodicalIF":0.0,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145049867","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Transportability of overall survival estimates from the USA to France in patients with metastatic triple-negative breast cancer 转移性三阴性乳腺癌患者的总生存估计从美国到法国的可移植性
ESMO Real World Data and Digital Oncology Pub Date : 2025-12-01 Epub Date: 2025-11-21 DOI: 10.1016/j.esmorw.2025.100648
C. Esnault , L. Simomia , G. Machuron , M. Robain , B. Adamson , V. Machuron
{"title":"Transportability of overall survival estimates from the USA to France in patients with metastatic triple-negative breast cancer","authors":"C. Esnault ,&nbsp;L. Simomia ,&nbsp;G. Machuron ,&nbsp;M. Robain ,&nbsp;B. Adamson ,&nbsp;V. Machuron","doi":"10.1016/j.esmorw.2025.100648","DOIUrl":"10.1016/j.esmorw.2025.100648","url":null,"abstract":"<div><h3>Background</h3><div>Metastatic triple-negative breast cancer (mTNBC) represents an aggressive breast cancer subtype with limited treatment options. The external validity of transportability for real-world evidence (RWE) applied across health care systems remains uncertain. This study evaluated the transportability of overall survival (OS) from the USA to France.</div></div><div><h3>Patients and methods</h3><div>We conducted a retrospective cohort study using de-identified or aggregated electronic health record-derived data from the USA and France. The study included adult female patients diagnosed with mTNBC initiating first-line systemic therapy before the widespread use of poly(ADP-ribose) polymerase (PARP) and programmed cell death (ligand) protein 1 (PD-(L)1) checkpoint inhibitors. To account for population differences, we reweighted the United States cohort to match the French population characteristics. OS was assessed from the start of first-line therapy using Kaplan–Meier curves.</div></div><div><h3>Results</h3><div>After adjustment, the United States (<em>n</em> = 812) and French (<em>n</em> = 2797) cohorts were well-balanced (standardized mean differences close to 0). Median OS was comparable between the unadjusted United States cohort [13.8 months, 95% confidence interval (CI) 12.6-15.0 months], the adjusted United States cohort (13.2 months, 95% CI 11.6-14.6 months) and the French cohort (13.8 months, 95% CI 13.2-14.6 months). OS rates at 1, 2, and 3 years in the French cohort were 55%, 30%, and 18%, respectively, and did not differ by &gt;3% from those in the USA, regardless of adjustment.</div></div><div><h3>Conclusions</h3><div>This study shows evidence that OS estimates for mTNBC derived from United States RWE are transportable to the French setting. Findings support the use of international RWE in health technology assessment contexts, potentially facilitating more efficient and evidence-based decision making for novel therapies.</div></div>","PeriodicalId":100491,"journal":{"name":"ESMO Real World Data and Digital Oncology","volume":"10 ","pages":"Article 100648"},"PeriodicalIF":0.0,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145571604","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Trends in smoking and obesity prevalence at breast cancer diagnosis: results from a 35-year real-world observational study 吸烟和肥胖在乳腺癌诊断中的流行趋势:一项35年真实世界观察性研究的结果
ESMO Real World Data and Digital Oncology Pub Date : 2025-12-01 Epub Date: 2025-11-17 DOI: 10.1016/j.esmorw.2025.100632
R.D.S. Gbessemehlan , F. Nguyen Van Long , N.K.D. Adenyo , O. Haroun , A. Turgeon , A. Boubaker , C. Laflamme , J. Lemieux , H. Nabi
{"title":"Trends in smoking and obesity prevalence at breast cancer diagnosis: results from a 35-year real-world observational study","authors":"R.D.S. Gbessemehlan ,&nbsp;F. Nguyen Van Long ,&nbsp;N.K.D. Adenyo ,&nbsp;O. Haroun ,&nbsp;A. Turgeon ,&nbsp;A. Boubaker ,&nbsp;C. Laflamme ,&nbsp;J. Lemieux ,&nbsp;H. Nabi","doi":"10.1016/j.esmorw.2025.100632","DOIUrl":"10.1016/j.esmorw.2025.100632","url":null,"abstract":"<div><h3>Background</h3><div>Breast cancer (BC) is the most commonly diagnosed cancer among women. Understanding long-term trends in modifiable risk factors at the time of diagnosis is essential to inform and evaluate the effectiveness of BC prevention strategies. This study assessed 35-year trends in the prevalence of two modifiable risk factors—obesity and smoking—among BC women.</div></div><div><h3>Methods</h3><div>We conducted a retrospective cohort study including 14 595 women diagnosed with BC at the Centre des maladies du sein in Quebec City, Canada, between 1987 and 2021. Prevalence ratios (PRs) for obesity and smoking were estimated using multivariable log-binomial regression models adjusted for demographic and clinical characteristics.</div></div><div><h3>Results</h3><div>Over the 35-year period, obesity prevalence at diagnosis significantly increased [PR 1.19; 95% confidence interval (CI) 1.15-1.24; <em>P</em> &lt; 0.0001], while smoking prevalence declined (PR 0.92; 95% CI 0.89-0.95; <em>P</em> &lt; 0.0001). Stratified analyses showed more pronounced increase in obesity (PR 1.40; 95% CI 1.25-1.57; <em>P</em> &lt; 0.0001) and decline in smoking (PR 0.82; 95% CI 0.78-0.86; <em>P</em> &lt; 0.0001) among premenopausal women, compared with postmenopausal women (obesity: PR 1.16; 95% CI 1.11-1.21; <em>P</em> &lt; 0.0001; smoking: PR 0.97; 95% CI 0.93-1.01; <em>P</em> = 0.1555).</div></div><div><h3>Conclusions</h3><div>The rising prevalence of obesity among BC patients at diagnosis underscores the need to strengthen public health efforts targeting obesity prevention. While anti-smoking measures appear to have been effective, additional strategies are warranted to address the growing burden of obesity and its potential impact on BC incidence in Canada and elsewhere.</div></div>","PeriodicalId":100491,"journal":{"name":"ESMO Real World Data and Digital Oncology","volume":"10 ","pages":"Article 100632"},"PeriodicalIF":0.0,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145571522","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
The digital shift in oncology EMRs in LMICs 中低收入国家肿瘤电子病历的数字化转变
ESMO Real World Data and Digital Oncology Pub Date : 2025-12-01 Epub Date: 2025-09-09 DOI: 10.1016/j.esmorw.2025.100183
N.H. Rajput
{"title":"The digital shift in oncology EMRs in LMICs","authors":"N.H. Rajput","doi":"10.1016/j.esmorw.2025.100183","DOIUrl":"10.1016/j.esmorw.2025.100183","url":null,"abstract":"","PeriodicalId":100491,"journal":{"name":"ESMO Real World Data and Digital Oncology","volume":"10 ","pages":"Article 100183"},"PeriodicalIF":0.0,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145020670","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Comparison of machine learning and deep learning models for survival prediction in early-stage hormone receptor-positive/HER2-negative breast cancer receiving neoadjuvant chemotherapy 机器学习与深度学习模型对早期激素受体阳性/ her2阴性乳腺癌新辅助化疗患者生存预测的比较
ESMO Real World Data and Digital Oncology Pub Date : 2025-12-01 Epub Date: 2025-09-16 DOI: 10.1016/j.esmorw.2025.100184
L. Mastrantoni , G. Garufi , G. Giordano , N. Maliziola , E. Di Monte , G. Arcuri , V. Frescura , A. Rotondi , A. Orlandi , L. Carbognin , A. Palazzo , L. Pontolillo , A. Fabi , S. Pannunzio , I. Paris , F. Marazzi , A. Franco , G. Franceschini , G. Scambia , D. Giannarelli , E. Bria
{"title":"Comparison of machine learning and deep learning models for survival prediction in early-stage hormone receptor-positive/HER2-negative breast cancer receiving neoadjuvant chemotherapy","authors":"L. Mastrantoni ,&nbsp;G. Garufi ,&nbsp;G. Giordano ,&nbsp;N. Maliziola ,&nbsp;E. Di Monte ,&nbsp;G. Arcuri ,&nbsp;V. Frescura ,&nbsp;A. Rotondi ,&nbsp;A. Orlandi ,&nbsp;L. Carbognin ,&nbsp;A. Palazzo ,&nbsp;L. Pontolillo ,&nbsp;A. Fabi ,&nbsp;S. Pannunzio ,&nbsp;I. Paris ,&nbsp;F. Marazzi ,&nbsp;A. Franco ,&nbsp;G. Franceschini ,&nbsp;G. Scambia ,&nbsp;D. Giannarelli ,&nbsp;E. Bria","doi":"10.1016/j.esmorw.2025.100184","DOIUrl":"10.1016/j.esmorw.2025.100184","url":null,"abstract":"<div><h3>Background</h3><div>We compared machine learning (ML) and deep learning (DL) models to predict disease-free survival (DFS) and overall survival (OS) in patients with hormone receptor (HR)-positive/human epidermal growth factor receptor 2 (HER2)-negative breast cancer (BC) receiving neoadjuvant chemotherapy (NACT), using routine clinicopathological features before and after surgery.</div></div><div><h3>Materials and methods</h3><div>In this retrospective cohort, 572 patients with stage I-III HR-positive/HER2-negative BC treated with anthracycline/taxane-based NACT and surgery were analyzed. Data were split into training (<em>n</em> = 463) and validation (<em>n</em> = 109) sets. Five ML models (random survival forest, extra survival tree, gradient boosting machine, support vector machine, regularized Cox) and four neural networks (DeepSurv, DeepHit, logistic hazard, multi-task logistic regression) were trained via five-fold cross-validation. Performance was assessed on the validation cohort by the C-index and integrated Brier score (iBS).</div></div><div><h3>Results</h3><div>Median age was 49 years and pathological complete response (pCR) rate was 15%. Median DFS was 103 months [95% confidence interval (CI) 84.4 months-not estimable (NE)], and 5-year OS was 78.6% (95% CI 74.8% to 82.5%). DeepSurv yielded the best overall performance, with a C-index of 0.70 (95% CI 0.60-0.78, iBS 0.22) for DFS and 0.68 (95% CI 0.56-0.79, iBS 0.17) for OS. The top ML model achieved C-indices of 0.64 (DFS) and 0.68 (OS). Key predictors were nodal status, estrogen receptor/progesterone receptor expression, tumor size, Ki-67 and pCR.</div></div><div><h3>Conclusions</h3><div>Both ML and DL models predicted survival post-NACT in HR-positive/HER2-negative BC, suggesting that simple models can perform as well as DL architectures in small datasets. The marginally higher discrimination of DL models should be weighed against computational demands and lower interpretability compared with ML methods.</div></div>","PeriodicalId":100491,"journal":{"name":"ESMO Real World Data and Digital Oncology","volume":"10 ","pages":"Article 100184"},"PeriodicalIF":0.0,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145097935","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Clinical evaluation of an automated pan-organ combined PD-L1 scoring using artificial intelligence on immunostained whole-slide images 人工智能在免疫染色全片图像上自动泛器官联合PD-L1评分的临床评价
ESMO Real World Data and Digital Oncology Pub Date : 2025-12-01 Epub Date: 2025-09-12 DOI: 10.1016/j.esmorw.2025.100181
C. Bossard , C. Magois , H. Roussel , F. Thomas , B. Cormier , A. Collin , V. Lemerle , I. Chokri , L. Lambros , F. Jossic , J.-F. Jazeron , C. Eymerit-Morin , A. Dhouibi , N. Labaied , A. Mensah , B. Gourdin , F. Leclair , D. Pommeret , Y. Salhi , M. Cecchini , J. Chetritt
{"title":"Clinical evaluation of an automated pan-organ combined PD-L1 scoring using artificial intelligence on immunostained whole-slide images","authors":"C. Bossard ,&nbsp;C. Magois ,&nbsp;H. Roussel ,&nbsp;F. Thomas ,&nbsp;B. Cormier ,&nbsp;A. Collin ,&nbsp;V. Lemerle ,&nbsp;I. Chokri ,&nbsp;L. Lambros ,&nbsp;F. Jossic ,&nbsp;J.-F. Jazeron ,&nbsp;C. Eymerit-Morin ,&nbsp;A. Dhouibi ,&nbsp;N. Labaied ,&nbsp;A. Mensah ,&nbsp;B. Gourdin ,&nbsp;F. Leclair ,&nbsp;D. Pommeret ,&nbsp;Y. Salhi ,&nbsp;M. Cecchini ,&nbsp;J. Chetritt","doi":"10.1016/j.esmorw.2025.100181","DOIUrl":"10.1016/j.esmorw.2025.100181","url":null,"abstract":"<div><h3>Background</h3><div>Programmed death-ligand 1 (PD-L1) inhibitors have shown remarkable results in oncology; however, many patients fail to respond, highlighting the need for reliable assessment of PD-L1 expression for patient selection. PD-L1 scoring, especially the combined positive score (CPS), is hindered by inter- and intraobserver variability, complex staining patterns, and technical discrepancies, all of which can impact therapeutic decisions. Artificial intelligence (AI) offers a solution by standardizing PD-L1 evaluation. This study evaluates a PD-L1 CPS AI, designed for reproducible and robust PD-L1 scoring across various tumor types and conditions.</div></div><div><h3>Materials and methods</h3><div>AI performance was validated on 142 samples spanning multiple tumor types (gastrointestinal, head and neck, breast, and uterine cervix) and sourced from four centers, reflecting diverse staining protocols. Routine scores were available. A gold standard was established through independent retrospective scoring by three senior pathologists enabling the assessment of variability. The scoring process was followed by collegial discussions to resolve discordant cases and ensure medical consensus. After a washout period, cases were reassessed with AI assistance. AI and routine manual scores were compared with the gold standard using organ-specific cut-offs.</div></div><div><h3>Results</h3><div>AI assistance improved interobserver agreement among pathologists, increasing intraclass correlation coefficient (ICC) from 62% to 74%, with a particularly pronounced effect in challenging cases with CPS &lt; 20 (<em>n</em> = 91), where ICC improved from 19% to 62%, underscoring the value of AI in reducing variability near clinical decision thresholds. Based on clinical cut-offs, AI-based scoring outperformed routine manual scoring in accuracy (88% versus 75%) and sensitivity (96% versus 78%), while maintaining a comparable positive predictive value (88% versus 87%), indicating an improved ability to detect true-positive cases.</div></div><div><h3>Conclusions</h3><div>This study highlights the potential of an AI-driven tool—DiaKwant PD-L1 algorithm—to improve PD-L1 scoring accuracy and reduce observer variability, particularly near clinical thresholds, across various solid carcinomas, independently of pre-analytical and digitization platforms. Its integration into clinical workflows could enhance efficiency and optimize patient eligibility for immunotherapy.</div></div>","PeriodicalId":100491,"journal":{"name":"ESMO Real World Data and Digital Oncology","volume":"10 ","pages":"Article 100181"},"PeriodicalIF":0.0,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145049851","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Synthetic data for clinical research and innovation: opportunities, challenges and future directions 临床研究与创新的合成数据:机遇、挑战和未来方向
ESMO Real World Data and Digital Oncology Pub Date : 2025-12-01 Epub Date: 2025-11-20 DOI: 10.1016/j.esmorw.2025.100651
Mattia Delleani
{"title":"Synthetic data for clinical research and innovation: opportunities, challenges and future directions","authors":"Mattia Delleani","doi":"10.1016/j.esmorw.2025.100651","DOIUrl":"10.1016/j.esmorw.2025.100651","url":null,"abstract":"<div><div>Data form the backbone of modern health care, driving improvements in patient care, enabling clinical research and supporting public health initiatives. The demand for high-quality individual-level data is growing, with electronic health records and clinical trial data offering critical opportunities for secondary analyses, hypothesis testing and methodological innovation. However, accessing and sharing this high-quality real-world data to enable personalized care and to respond to rapidly changing conditions is often limited by strict privacy requirements and complex regulations. This perspective highlights the promise of synthetic data in enabling research access, accelerating artificial intelligence development and supporting clinical trials while also discussing key barriers, including legal and ethical uncertainty, re-identification risk and the need for standardized validation frameworks. In this context, synthetic data present a promising path forward by enabling research while addressing privacy concerns, streamlining administrative processes and decreasing costs.</div></div>","PeriodicalId":100491,"journal":{"name":"ESMO Real World Data and Digital Oncology","volume":"10 ","pages":"Article 100651"},"PeriodicalIF":0.0,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145571602","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Cost-effectiveness of first-line osimertinib informed by electronic medical records via text-mining: a real-world Italian case study of EGFR-mutated advanced NSCLC patients 通过文本挖掘的电子医疗记录告知一线奥西替尼的成本效益:一个真实的意大利egfr突变晚期NSCLC患者的案例研究
ESMO Real World Data and Digital Oncology Pub Date : 2025-12-01 Epub Date: 2025-11-03 DOI: 10.1016/j.esmorw.2025.100198
F. Corso , P. Baili , F. Scotti , L. Mazzeo , V. Torri , G. Lo Russo , C. Proto , C. Giani , M. Ganzinelli , R.M. di Mauro , A. Cappozzo , A.M. Paganoni , A. Prelaj , F. Ieva
{"title":"Cost-effectiveness of first-line osimertinib informed by electronic medical records via text-mining: a real-world Italian case study of EGFR-mutated advanced NSCLC patients","authors":"F. Corso ,&nbsp;P. Baili ,&nbsp;F. Scotti ,&nbsp;L. Mazzeo ,&nbsp;V. Torri ,&nbsp;G. Lo Russo ,&nbsp;C. Proto ,&nbsp;C. Giani ,&nbsp;M. Ganzinelli ,&nbsp;R.M. di Mauro ,&nbsp;A. Cappozzo ,&nbsp;A.M. Paganoni ,&nbsp;A. Prelaj ,&nbsp;F. Ieva","doi":"10.1016/j.esmorw.2025.100198","DOIUrl":"10.1016/j.esmorw.2025.100198","url":null,"abstract":"<div><h3>Background</h3><div>Tyrosine kinase inhibitors (TKIs) have significantly changed the therapeutic landscape of non-small-cell lung cancer (NSCLC) with epidermal growth factor receptor (<em>EGFR</em>) gene mutation. The primary objective is to assess the use of real-world data (RWD) extracted from electronic health records (EHRs) via a text-mining technique. Then, RWD are used to inform a cost-effectiveness model of first-line TKI third-generation osimertinib compared with standard <em>EGFR</em> inhibitors.</div></div><div><h3>Materials and methods</h3><div>A cohort of 190 NSCLC <em>EGFR</em>-mutated patients has been enrolled from an Italian cancer institute. A rule-based named-entity recognition algorithm is carried out to extract RWD from medical records stored in the data warehouse. A Bayesian cost-effectiveness analysis is developed from the Italian health care system perspective. Survival curves are extrapolated over an observed follow-up horizon of a real-world population to estimate the time to treatment discontinuation (TTD) and overall survival (OS) distributions.</div></div><div><h3>Results</h3><div>An incremental cost-effectiveness ratio (ICER) of 48 398€/quality-adjusted life year (QALY) is found for the base-case scenario, while 51 779€/QALY is found for a second scenario. While higher total annual costs are observed, effectiveness analysis confirms greater QALYs associated with osimertinib versus non-osimertinib. This result is also supported by significantly longer TTD (median 15 months) and OS (median 27 months) for osimertinib. Sensitivity and scenario analyses are used to validate the results.</div></div><div><h3>Conclusions</h3><div>This study represents a pilot assessment for encouraging the adoption of text-mining approaches for RWD generation and informing health economic evaluations in oncology. RWD analysis confirms that osimertinib is cost-effective as a first-line treatment compared with previous generations of TKIs for advanced <em>EGFR</em>-mutated NSCLC patients.</div></div>","PeriodicalId":100491,"journal":{"name":"ESMO Real World Data and Digital Oncology","volume":"10 ","pages":"Article 100198"},"PeriodicalIF":0.0,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145465436","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Outcomes in relapsed/refractory diffuse large B-cell lymphoma in an Asian tertiary cancer center: real-world interventions as benchmark for novel therapy 亚洲三级癌症中心复发/难治性弥漫性大b细胞淋巴瘤的结局:现实世界干预作为新疗法的基准
ESMO Real World Data and Digital Oncology Pub Date : 2025-12-01 Epub Date: 2025-10-06 DOI: 10.1016/j.esmorw.2025.100195
N.-A. Lim , Y. Sun , J.Y. Tan , R.M.H. Lim , Y.H. Tan , L.C.K. Ng , F.L.W.I. Lim , Y.T. Goh , J.T.M. Hoe , J. Chiang , E.W.Y. Chang , E.Y.L. Poon , N. Somasundaram , M. Tao , S.T. Lim , E.S. Mulvihill , A. Hanna-Elias , J.Y. Chan
{"title":"Outcomes in relapsed/refractory diffuse large B-cell lymphoma in an Asian tertiary cancer center: real-world interventions as benchmark for novel therapy","authors":"N.-A. Lim ,&nbsp;Y. Sun ,&nbsp;J.Y. Tan ,&nbsp;R.M.H. Lim ,&nbsp;Y.H. Tan ,&nbsp;L.C.K. Ng ,&nbsp;F.L.W.I. Lim ,&nbsp;Y.T. Goh ,&nbsp;J.T.M. Hoe ,&nbsp;J. Chiang ,&nbsp;E.W.Y. Chang ,&nbsp;E.Y.L. Poon ,&nbsp;N. Somasundaram ,&nbsp;M. Tao ,&nbsp;S.T. Lim ,&nbsp;E.S. Mulvihill ,&nbsp;A. Hanna-Elias ,&nbsp;J.Y. Chan","doi":"10.1016/j.esmorw.2025.100195","DOIUrl":"10.1016/j.esmorw.2025.100195","url":null,"abstract":"<div><h3>Background</h3><div>Recent advances have led to the approval of several new therapies for relapsed/refractory diffuse large B-cell lymphoma (R/R DLBCL). However, real-world data for treatment patterns and outcomes in R/R DLBCL in Asian countries are lacking, limiting the benchmarking of novel treatments.</div></div><div><h3>Patients and methods</h3><div>We conducted a retrospective single-center cohort study including 227 patients with R/R DLBCL diagnosed from 2010 to 2022. Outcomes including progression-free survival (PFS) and overall survival (OS) were assessed.</div></div><div><h3>Results</h3><div>In the overall cohort, median OS was 13.5 months and 5-year OS was 29.8% from the time of first relapse. Median time to first relapse from completion of first-line immunochemotherapy was 7.03 months (range 0-148.4 months). Time to first relapse was significantly associated with OS outcomes. Patients who had earlier relapse, defined as 12 months or less from first-line treatment, had worse OS at 8.6 months, as compared with patients who relapsed &gt;12 months after first-line treatment, who had OS of 34.2 months (hazard ratio 1.73, 95% confidence interval 1.26-2.37, <em>P</em> = 0.007). Median PFS was 3.2 months as compared with 15.3 months, respectively. The majority (<em>n</em> = 121, 53.3%) were transplant-ineligible and only 44 (19.4%) were transplanted. The median PFS for non-transplant recipients was 3.5 months versus 34.4 months for transplant recipients. The median OS for non-transplant recipients was 8.5 months versus 114.1 months for transplant recipients.</div></div><div><h3>Conclusion</h3><div>Poor outcomes remain for patients with R/R DLBCL. Novel therapeutics including bispecifics and CAR-T may improve outcomes of these patients.</div></div>","PeriodicalId":100491,"journal":{"name":"ESMO Real World Data and Digital Oncology","volume":"10 ","pages":"Article 100195"},"PeriodicalIF":0.0,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145268340","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Investigation into the clinical features, treatment patterns, and survival of older men with prostate cancer in Sweden and Belgium 瑞典和比利时老年男性前列腺癌的临床特征、治疗模式和生存率的调查
ESMO Real World Data and Digital Oncology Pub Date : 2025-12-01 Epub Date: 2025-11-17 DOI: 10.1016/j.esmorw.2025.100631
G. George , A.-B. Axelson , K. Decaestecker , H. Garmo , N. Lumen , M. Rousseau , P. Stattin , C. Van Praet , W. Everaerts , A. Clinckaert , T.M. de Reijke , M. Van Hemelrijck
{"title":"Investigation into the clinical features, treatment patterns, and survival of older men with prostate cancer in Sweden and Belgium","authors":"G. George ,&nbsp;A.-B. Axelson ,&nbsp;K. Decaestecker ,&nbsp;H. Garmo ,&nbsp;N. Lumen ,&nbsp;M. Rousseau ,&nbsp;P. Stattin ,&nbsp;C. Van Praet ,&nbsp;W. Everaerts ,&nbsp;A. Clinckaert ,&nbsp;T.M. de Reijke ,&nbsp;M. Van Hemelrijck","doi":"10.1016/j.esmorw.2025.100631","DOIUrl":"10.1016/j.esmorw.2025.100631","url":null,"abstract":"<div><h3>Background</h3><div>A Dutch population-based study showed that older men with intermediate- or high-risk prostate cancer (PCa) were treated with curative intent less often than younger patients. To assess generalisability, we investigated treatment patterns in men with all PCa stages aged &lt;70 years versus ≥70 years in Sweden and Belgium.</div></div><div><h3>Patients and methods</h3><div>Men diagnosed with PCa between 2005 and 2015 were identified from the Prostate Cancer data Base Sweden and from two centres in Ghent and Leuven, Belgium. Multivariable logistic regression, adjusting for PCa stage, prostate-specific antigen, Gleason score, and Charlson comorbidity index, assessed the impact of age (&lt;/≥70 years) on curative treatment in intermediate- and high-risk PCa. Ten-year relative survival was calculated for four age groups per country to assess excess mortality versus the general population.</div></div><div><h3>Results</h3><div>In total, 58 092 men &lt;70 years and 50 886 men ≥70 years were diagnosed in Sweden, Leuven, and Ghent. In both countries, patients aged ≥70 years were less likely to receive curative treatments for intermediate- and high-risk PCa versus those &lt;70 years. In Sweden, 10-year relative survival was roughly 90-95% across all four age groups. In Leuven, survival was 100% for all except those ≥80 years, where it was 90%.</div></div><div><h3>Conclusion</h3><div>Men aged ≥70 years were treated with curative intent less frequently for intermediate- and high-risk PCa compared with younger men. The differing relative survival rates reflect heterogeneity in PCa stage distribution between countries. Age should be considered when planning curative treatments, but it should not be the sole determinant of therapeutic decisions.</div></div>","PeriodicalId":100491,"journal":{"name":"ESMO Real World Data and Digital Oncology","volume":"10 ","pages":"Article 100631"},"PeriodicalIF":0.0,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145571521","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
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