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The impact of targeted therapies on molecular alterations identified by an institutional molecular tumor board: an approach based on ESCAT classification
Pub Date : 2024-12-01 DOI: 10.1016/j.esmorw.2024.100092
K. Rahmani Narj Abadi , C. Dupain , I. Guillou , R. Sanchez , K. Nedara , G. Marret , S. Hescot , M-P. Sablin , Z. Castel-Ajgal , C. Neuzillet , E. Borcoman , D. Bello Roufai , M. Rodrigues , A. Asnacios Lecerf , C. Callens , O. Trabelsi-Grati , S. Melaabi , K. Driouch , S. Antonio , E. Lemaitre , C. Le Tourneau

Background

The European Society of Medical Oncology Scale for Clinical Actionability of Molecular Targets (ESCAT) classification system provides a standardized framework for categorizing genomic alterations (GAs) of patients with recurrent, metastatic, or rare cancer. This study aimed to present outcomes of patients discussed at the molecular tumor board (MTB) in general and according to ESCAT.

Patients and methods

We included 1226 patients with recurrent and/or metastatic cancer presented at the MTB from 2018 to 2022. Clinical and demographic data collected included age, gender, type of specimen, tumor type, number of prior treatments received, techniques used for molecular analyses, GAs identified, MTB recommendations, and inclusion or not into a clinical trial. The clinical endpoints collected were overall response rate (ORR), progression-free survival (PFS), and overall survival (OS), and were correlated with ESCAT.

Results

Successful molecular profiling was carried out in 895 of 1226 (73%) patients. Actionable GAs were found in 595 (49%) patients, and 206 (17%) patients were oriented to matched therapies. Eventually, 101 (8%) patients received a matched therapy. For these patients, PFS and OS were significantly longer for GAs classified as ESCAT tiers I/II, compared with tiers III/IV (P = 0.009 and P = 0.014, respectively).

Conclusions

Detection of actionable GAs through MTB molecular screening enabled to treat 8% of patients with matched therapy. Patients treated with matched therapy based on ESCAT tiers I/II had statistically longer PFS and OS, compared with ESCAT tiers III/IV.
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引用次数: 0
Bridging research and practice: enhancing regulatory decisions with pragmatic clinical trials in oncology
Pub Date : 2024-12-01 DOI: 10.1016/j.esmorw.2024.100065
M.D. Stewart , A.E. McKee , R.S. Herbst , H.S. Andrews , B.A. McKelvey , E.V. Sigal , J.D. Allen
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引用次数: 0
Editoiral Board
Pub Date : 2024-12-01 DOI: 10.1016/S2949-8201(24)00079-1
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引用次数: 0
Characteristics and impact of real-world evidence studies in oncology: comprehensive mapping review of publications evaluating targeted therapies in solid tumours
Pub Date : 2024-12-01 DOI: 10.1016/j.esmorw.2024.100091
A. Pellat , T. Grinda , P. Cresta Morgado , A. Prelaj , V. Miskovic , A. Valachis , I. Zerdes , D. Martins-Branco , L. Provenzano , A. Spagnoletti , G. Nader-Marta , B.E. Wilson , Y.-H. Yang , G. Pentheroudakis , S. Delaloge , L. Castelo-Branco , M. Koopman

Background

A mapping review of real-world evidence (RWE) publications on targeted therapy (TT) for solid tumours was carried out to describe their characteristics, strengths, and limitations.

Methods

RWE publications were identified that: (i) focused on TTs in patients with solid tumours; (ii) included study objectives of effectiveness, predictive or prognostic factors, safety or quality of life; (iii) were published between 1 January 2020 and 22 December 2022. Associations between study variables and journal impact factor (IF) were explored through regression and cluster network analyses.

Results

Of 7775 publications identified, 1251 were eligible for analysis. The number of publications per year increased over time. Most studies were conducted in Asia (50%), Europe (25%), and North America (17%), with only 8% in more than one country. Data sources were mostly health records (55%) and registries (11%). Most studies were retrospective (85%) and only 16% were population based. Gastrointestinal tumours were the most frequently studied (30%), followed by lung (22%) and breast (21%). The median journal IF was 4.4. Involvement of >10 centres and studies originating from Europe were significantly associated with a higher IF (≥7) in multivariable analysis. Network analysis demonstrated strong associations between countries and the number of publications in specific tumour types.

Conclusions

The number of RWE publications on TT for solid tumours is increasing, but studies are heterogeneous, mostly retrospective, and published in low IF journals. International collaboration and promotion of standardised data sources is imperative to enhance the relevance of RWE research to complement clinical guidelines and impact clinical practice.
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引用次数: 0
Neoadjuvant compared to adjuvant chemotherapy combined with trastuzumab in patients with HER2-positive breast cancer: a register-based cohort study HER2阳性乳腺癌患者接受新辅助化疗与曲妥珠单抗联合辅助化疗的比较:一项基于登记的队列研究
Pub Date : 2024-11-19 DOI: 10.1016/j.esmorw.2024.100093
S. Hosseini-Mellner , Å. Wickberg , E. Olsson , A. Karakatsanis , A. Valachis

Background

The aim of the study was to compare trastuzumab-based neoadjuvant therapy (NAT) with adjuvant therapy (AT) in a register-based cohort of Swedish patients with primary operable human epidermal growth factor receptor 2 (HER2)-positive breast cancer.

Patients and methods

The Swedish nationwide research database BCBaSe 3.0 was used to identify eligible patients with primary operable HER2-positive breast cancer treated with either NAT or AT between 2008 and 2019. To mitigate confounding by indication bias, propensity score matching (PSM) and inverse probability of treatment weighting (IPTW) were applied.

Results

In total, 7258 patients with primary operable HER2-positive breast cancer were identified; 1789 (24.6%) received NAT and 5469 (75.4%), AT. After 1 : 1 PSM, 1258 patients in each therapeutic strategy were available for comparisons. No statistically significant differences between NAT and AT were observed [hazard ratio (HR) for distant disease-free survival 0.97, 95% confidence (CI) 0.72-1.30; HR for breast cancer-specific survival (BCSS) 0.69, 95% CI 0.45-1.07; HR for overall (OS) 0.72, 95% CI 0.50-1.05]. In subgroup analysis, NAT resulted in better BCSS (HR 0.44, 95% CI 0.22-0.89) and OS (HR 0.49, 95% CI 0.29-0.90) in patients with clinical node positivity (cN+) at diagnosis.

Conclusion

The study shows an equivalence of NAT and AT in terms of prognosis for patients with operable HER2-positive disease whereas a potential benefit of NAT in patients with cN+ is implied. Considering the emerging treatment strategies in the neoadjuvant setting for HER2-positive breast cancer that are not reflected in the study cohort, NAT should be considered as the strategy with a higher possibility of improving long-term prognosis for patients with HER2-positive disease.
背景该研究旨在比较基于曲妥珠单抗的新辅助治疗(NAT)与辅助治疗(AT)在基于登记的瑞典原发性可手术人表皮生长因子受体2(HER2)阳性乳腺癌患者队列中的疗效。患者与方法瑞典全国性研究数据库BCBaSe 3.0用于识别2008年至2019年间接受NAT或AT治疗的符合条件的原发性可手术HER2阳性乳腺癌患者。为减少适应症偏倚带来的混杂,采用了倾向评分匹配(PSM)和逆治疗概率加权(IPTW)。结果共确定了7258例可手术的原发性HER2阳性乳腺癌患者,其中1789例(24.6%)接受了NAT治疗,5469例(75.4%)接受了AT治疗。经过1:1 PSM后,每种治疗策略各有1258名患者可供比较。NAT和AT在统计学上无明显差异[无远处疾病生存期的危险比(HR)为0.97,95%置信度(CI)为0.72-1.30;乳腺癌特异性生存期(BCSS)的危险比(HR)为0.69,95%置信度(CI)为0.45-1.07;总生存期(OS)的危险比(HR)为0.72,95%置信度(CI)为0.50-1.05]。在亚组分析中,NAT使诊断时临床结节阳性(cN+)患者的BCSS(HR 0.44,95% CI 0.22-0.89)和OS(HR 0.49,95% CI 0.29-0.90)更好。考虑到新辅助治疗 HER2 阳性乳腺癌的新兴治疗策略并未反映在研究队列中,NAT 应被视为更有可能改善 HER2 阳性患者长期预后的策略。
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引用次数: 0
Real-world evaluation of ImmuCare-PRO patient-reported outcomes in melanoma patients treated with immune checkpoint inhibitors 对接受免疫检查点抑制剂治疗的黑色素瘤患者进行ImmuCare-PRO患者报告结果的真实世界评估
Pub Date : 2024-11-19 DOI: 10.1016/j.esmorw.2024.100090
S. Belkaïd , S. Milley , R. Saux , M. Bonjour , A. Augros , P.-J. Souquet , D. Maillet , D. Maucort-Boulch , C. Dolla , L. Thomas , S. Dalle

Background

Toxicity profile of immune checkpoint inhibitors (ICI) poses challenges for early detection of immune-related adverse events (IrAEs). In oncology, patient-reported outcomes (PROs) are reported to have a beneficial effect; however, their efficacy for IrAE detection in melanoma patients remains unclear. A remote patient-monitoring system was created in our department; we investigated its real-world impact in detecting grade 2 or above IrAEs occurring during ICI treatment in melanoma patients.

Patients and methods

Patients receiving ICI for a melanoma were followed using a weekly online questionnaire containing 11 symptoms suggestive of IrAE. Moderate/severe symptoms generated an alert score and an intervention by an oncology nurse or physician. The system’s performance in detecting grade 2 or above IrAEs, as well as reasons for missed detections, were retrospectively assessed.

Results

A total of 5202 questionnaires completed by 136 patients led to 783 (15.0%) alert scores; 64 of them were associated with 69 grade 2 or above IrAEs, with 22 (34.4%) questionnaires correctly detecting 27 grade 2 or above IrAEs, saving a mean 4.1 days on the next scheduled visit and leading to only one emergency room visit. Forty-two grade 2 or above IrAEs (mainly blood disorders, n = 31) were not detected. False alerts often resulted from functional or non-specific symptoms (32.3%), such as fatigue or general pain.

Conclusion

The ImmuCare-PRO system correctly detected a third of moderate-to-severe IrAEs, and most of those had clinical impact such as skin toxicities, colitis, and rheumatological IrAEs. This enables earlier management and could avoid unnecessary emergency room visits.
背景免疫检查点抑制剂(ICI)的毒性特征为早期检测免疫相关不良事件(IrAEs)带来了挑战。据报道,在肿瘤学领域,患者报告结果(PROs)具有有益的作用;然而,它们对黑色素瘤患者IrAE检测的功效仍不明确。我们研究了该系统在检测黑色素瘤患者接受 ICI 治疗期间出现的 2 级或 2 级以上 IrAE 方面的实际效果。中度/重度症状会产生警报评分,并由肿瘤科护士或医生进行干预。结果 136 名患者共填写了 5202 份问卷,获得了 783 个(15.0%)警报评分;其中 64 个警报评分与 69 个 2 级或 2 级以上 IrAE 相关联,22 个(34.4%)问卷正确检测出了 27 个 2 级或 2 级以上 IrAE,平均节省了 4.1 天的下次预定就诊时间,仅导致一次急诊就诊。有 42 例 2 级或以上非器质性损伤(主要是血液疾病,n = 31)未被检测到。结论ImmuCare-PRO系统正确检测出了三分之一的中度至重度IrAE,其中大部分具有临床影响,如皮肤毒性、结肠炎和风湿性IrAE。这样就能更早地进行处理,避免不必要的急诊就诊。
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引用次数: 0
Challenges and opportunities for real-world evidence in clinical oncology—a view from the UK: proceedings of a national workshop 临床肿瘤学真实世界证据的挑战与机遇--来自英国的观点:全国研讨会记录
Pub Date : 2024-11-18 DOI: 10.1016/j.esmorw.2024.100089
M. Craddock , C. Dempsey , D. Abdulwahid , J.P.C. Baldwin , K. Banfill , A. Carver , A. Chaturvedi , S. Cheeseman , G.W. Cowell , M. Daly , A. Dekker , S.R. Dubash , S. Duffield , I. Fornacon-Wood , M.A.C. Garcia , P. Goodley , H. Green , R.J. Holley , S. Ingram , S. Jones , C. Faivre-Finn
Real-world data (RWD) are defined as information collected about patients as a routine part of treatment. To understand the status of RWD initiatives in oncology in the UK, an online survey and in-person workshop were conducted which aimed to characterise current perceptions of RWD, establish where real-world evidence (RWE) could support unmet clinical need, and to identify the barriers and solutions to obtaining this evidence. Self-selecting health care professionals including oncologists, physicists, radiographers, and health data researchers, as well as patient representatives, participated in an anonymous survey (N = 55) and/or a 1-day workshop (N = 46). The workshop consisted of introductory presentations followed by three 1 hour grouped breakout sessions. An inductive thematic analysis synthesizing the outcomes of the survey and workshop was carried out post hoc. Despite issues of perceived poor data quality and the prevalence of unstructured data, 92% of survey respondents recognised the potential of RWD to provide novel evidence. Suggested applications of RWE were validation of trial results in the general population, continuous evaluation of new technologies, decision-making in rare disease groups, and resource allocation. Barriers to progression of RWD initiatives identified were data accessibility, data quality, and prioritisation. Potential solutions include streamlining information governance processes, training staff in data science skills, and demonstrating clinical benefit. The potential of RWD to provide novel evidence is strongly recognised in the UK radiotherapy community. While barriers to progress were identified, none of them are insurmountable. To move forwards, the profile of RWE needs to be elevated to attract higher prioritisation and resourcing.
真实世界数据 (RWD) 的定义是在常规治疗过程中收集的患者信息。为了解英国肿瘤学中真实世界数据(RWD)计划的现状,我们开展了一项在线调查和现场研讨会,旨在了解当前对真实世界数据的看法,确定真实世界证据(RWE)在哪些方面可以支持未满足的临床需求,并找出获得这些证据的障碍和解决方案。自我选择的医护专业人员(包括肿瘤学家、物理学家、放射技师和健康数据研究人员)以及患者代表参加了匿名调查(55 人)和/或为期一天的研讨会(46 人)。研讨会由介绍性发言和三个 1 小时的分组讨论组成。事后对调查和研讨会的结果进行了归纳专题分析。尽管存在数据质量差和普遍存在非结构化数据的问题,但 92% 的调查对象认识到 RWD 在提供新证据方面的潜力。建议将 RWE 应用于普通人群试验结果的验证、新技术的持续评估、罕见疾病群体的决策以及资源分配。已确定的推进 RWD 计划的障碍包括数据可获取性、数据质量和优先次序。潜在的解决方案包括简化信息管理流程、对员工进行数据科学技能培训以及展示临床效益。英国放射治疗界强烈认可 RWD 提供新证据的潜力。虽然发现了一些阻碍进展的因素,但这些因素都不是不可克服的。要想取得进展,就必须提升 RWE 的形象,以吸引更多的优先关注和资源。
{"title":"Challenges and opportunities for real-world evidence in clinical oncology—a view from the UK: proceedings of a national workshop","authors":"M. Craddock ,&nbsp;C. Dempsey ,&nbsp;D. Abdulwahid ,&nbsp;J.P.C. Baldwin ,&nbsp;K. Banfill ,&nbsp;A. Carver ,&nbsp;A. Chaturvedi ,&nbsp;S. Cheeseman ,&nbsp;G.W. Cowell ,&nbsp;M. Daly ,&nbsp;A. Dekker ,&nbsp;S.R. Dubash ,&nbsp;S. Duffield ,&nbsp;I. Fornacon-Wood ,&nbsp;M.A.C. Garcia ,&nbsp;P. Goodley ,&nbsp;H. Green ,&nbsp;R.J. Holley ,&nbsp;S. Ingram ,&nbsp;S. Jones ,&nbsp;C. Faivre-Finn","doi":"10.1016/j.esmorw.2024.100089","DOIUrl":"10.1016/j.esmorw.2024.100089","url":null,"abstract":"<div><div>Real-world data (RWD) are defined as information collected about patients as a routine part of treatment. To understand the status of RWD initiatives in oncology in the UK, an online survey and in-person workshop were conducted which aimed to characterise current perceptions of RWD, establish where real-world evidence (RWE) could support unmet clinical need, and to identify the barriers and solutions to obtaining this evidence. Self-selecting health care professionals including oncologists, physicists, radiographers, and health data researchers, as well as patient representatives, participated in an anonymous survey (<em>N</em> = 55) and/or a 1-day workshop (<em>N</em> = 46). The workshop consisted of introductory presentations followed by three 1 hour grouped breakout sessions. An inductive thematic analysis synthesizing the outcomes of the survey and workshop was carried out <em>post hoc</em>. Despite issues of perceived poor data quality and the prevalence of unstructured data, 92% of survey respondents recognised the potential of RWD to provide novel evidence. Suggested applications of RWE were validation of trial results in the general population, continuous evaluation of new technologies, decision-making in rare disease groups, and resource allocation. Barriers to progression of RWD initiatives identified were data accessibility, data quality, and prioritisation. Potential solutions include streamlining information governance processes, training staff in data science skills, and demonstrating clinical benefit. The potential of RWD to provide novel evidence is strongly recognised in the UK radiotherapy community. While barriers to progress were identified, none of them are insurmountable. To move forwards, the profile of RWE needs to be elevated to attract higher prioritisation and resourcing.</div></div>","PeriodicalId":100491,"journal":{"name":"ESMO Real World Data and Digital Oncology","volume":"6 ","pages":"Article 100089"},"PeriodicalIF":0.0,"publicationDate":"2024-11-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142698584","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
Implementing digital patient-reported outcomes in routine cancer care: barriers and facilitators 在常规癌症护理中实施数字患者报告结果:障碍与促进因素
Pub Date : 2024-10-23 DOI: 10.1016/j.esmorw.2024.100088
S.G. Aanes , S. Wiig , C. Nieder , E.C. Haukland

Background

In cancer care research, there exists a gap between patient-reported outcomes (PROs) and health care personnel-reported outcomes. PROs have shown significant benefits in terms of symptoms, quality of life, reduced hospital admissions and increased overall survival. However, throughout the past decade, PROS have been used mainly in clinical trials and slowly implemented in routine cancer care. We wanted to review specific published experiences identified as barriers to and facilitators of the implementation of digital PROs in routine cancer care.

Results

A literature search was conducted in PubMed and Ovid Medline Evidence-Based Medicine Reviews (EBMRs) from 1 January 2017 to 29 August 2023. A total of 313 records were screened, of which 15 records were included. Facilitators identified were a user-friendly electronic PRO (ePRO) solution routinely used by care providers in a revised workflow that engaged key stakeholders and provided sufficient support and infrastructure. Common barriers were lack of information about benefits, time constraints, literacy or lack of access to text or information technology (IT) together with a negative impact on workflow, inadequate IT infrastructure, not engaging staff and costs.

Conclusions

Successful implementation of ePRO systems needs to address identified barriers and leverage facilitators. Using implementation frameworks and guidelines with quality improvement methods can enhance successful implementation as they address both local barriers and facilitators in a system thinking perspective. There is still an unmet need for real-world evidence on how sustainable PROs can be implemented most efficiently over time, thus highlighting the need for a bridge between medical and implementation science.
背景在癌症护理研究中,患者报告的结果(PROs)与医护人员报告的结果之间存在差距。PROs在症状、生活质量、减少入院次数和提高总生存率等方面显示出明显的优势。然而,在过去的十年中,PROS 主要用于临床试验,在常规癌症护理中的应用进展缓慢。我们希望回顾已发表的具体经验,这些经验被认为是在常规癌症护理中实施数字 PRO 的障碍和促进因素。结果 在 PubMed 和 Ovid Medline 循证医学综述(EBMR)中进行了文献检索,时间为 2017 年 1 月 1 日至 2023 年 8 月 29 日。共筛选出313条记录,其中15条记录被纳入其中。已确定的促进因素包括护理提供者在修订的工作流程中常规使用的用户友好型电子PRO(ePRO)解决方案,该解决方案吸引了主要利益相关者的参与,并提供了足够的支持和基础设施。常见的障碍包括:缺乏有关益处的信息、时间限制、不识字或无法获取文本或信息技术(IT),以及对工作流程的负面影响、IT 基础设施不足、员工参与度不高和成本问题。使用具有质量改进方法的实施框架和指南可以提高实施的成功率,因为它们可以从系统思维的角度解决当地的障碍和促进因素。关于如何在一段时间内最有效地实施可持续的 PROs,仍然需要实际的证据来证明,因此需要在医学和实施科学之间架起一座桥梁。
{"title":"Implementing digital patient-reported outcomes in routine cancer care: barriers and facilitators","authors":"S.G. Aanes ,&nbsp;S. Wiig ,&nbsp;C. Nieder ,&nbsp;E.C. Haukland","doi":"10.1016/j.esmorw.2024.100088","DOIUrl":"10.1016/j.esmorw.2024.100088","url":null,"abstract":"<div><h3>Background</h3><div>In cancer care research, there exists a gap between patient-reported outcomes (PROs) and health care personnel-reported outcomes. PROs have shown significant benefits in terms of symptoms, quality of life, reduced hospital admissions and increased overall survival. However, throughout the past decade, PROS have been used mainly in clinical trials and slowly implemented in routine cancer care. We wanted to review specific published experiences identified as barriers to and facilitators of the implementation of digital PROs in routine cancer care.</div></div><div><h3>Results</h3><div>A literature search was conducted in PubMed and Ovid Medline Evidence-Based Medicine Reviews (EBMRs) from 1 January 2017 to 29 August 2023. A total of 313 records were screened, of which 15 records were included. Facilitators identified were a user-friendly electronic PRO (ePRO) solution routinely used by care providers in a revised workflow that engaged key stakeholders and provided sufficient support and infrastructure. Common barriers were lack of information about benefits, time constraints, literacy or lack of access to text or information technology (IT) together with a negative impact on workflow, inadequate IT infrastructure, not engaging staff and costs.</div></div><div><h3>Conclusions</h3><div>Successful implementation of ePRO systems needs to address identified barriers and leverage facilitators. Using implementation frameworks and guidelines with quality improvement methods can enhance successful implementation as they address both local barriers and facilitators in a system thinking perspective. There is still an unmet need for real-world evidence on how sustainable PROs can be implemented most efficiently over time, thus highlighting the need for a bridge between medical and implementation science.</div></div>","PeriodicalId":100491,"journal":{"name":"ESMO Real World Data and Digital Oncology","volume":"6 ","pages":"Article 100088"},"PeriodicalIF":0.0,"publicationDate":"2024-10-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142529799","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
Efficacy of cabozantinib and sunitinib for the treatment of intermediate/poor risk renal cell carcinoma based upon UK real-world data 基于英国真实世界数据的卡博替尼和舒尼替尼治疗中危/低危肾细胞癌的疗效
Pub Date : 2024-10-18 DOI: 10.1016/j.esmorw.2024.100087
D. Lee , G.J. Melendez-Torres , A. Challapalli , R. Frazer , J. McGrane , A. Bahl

Background

The purpose of this study was to explore the effectiveness of cabozantinib versus sunitinib for the treatment of first-line metastatic renal cell carcinoma in intermediate/poor risk patients.

Materials and methods

Retrospective review of cases between 1 January 2018 and 30 June 2021 across 17 UK centres. Univariable and multivariable Cox proportional hazards modelling to identify prognostic factors. Inverse probability of treatment weighting, to estimate the causal effect of first-line treatment type.

Results

Cabozantinib patients (n = 106) had poorer risk status, less prior nephrectomy, shorter time to therapy, and more clear cell histology than sunitinib patients (n = 218). More sunitinib patients received a second or third line of subsequent treatment (56% and 23% versus 43% and 13%). Though there was no significant difference between treatments in overall survival (OS) or progression-free survival (PFS) across models, the difference in PFS bordered on significant in a multipredictor analysis (benefit in favour of cabozantinib; P = 0.06). When the Kaplan–Meier curves were stratified by risk status (intermediate versus poor), patients had similar OS within the risk groups. PFS appeared to differ with poor risk patients performing better on cabozantinib. Inverse probability of treatment weighting analysis showed little difference from the unadjusted results: OS hazard ratio = 1.119 (95% confidence interval (CI) 0.823-1.521); PFS hazard ratio 0.825 (95% CI 0.636-1.070) for cabozantinib versus sunitinib.

Conclusions

Our results showed no significant difference in either OS or PFS between treatments. Cabozantinib trended towards improved PFS and reduced OS. Decision-making for tyrosine kinase inhibitor monotherapy should consider later-line treatment options. This analysis is of particular relevance as sunitinib is now off-patent meaning that the cost of a course of treatment has considerably reduced.
背景本研究旨在探讨卡博替尼对舒尼替尼治疗中危/低危患者一线转移性肾细胞癌的有效性。采用单变量和多变量 Cox 比例危险度模型确定预后因素。结果与舒尼替尼患者(n = 218)相比,卡博赞替尼患者(n = 106)的风险状况更差、既往肾切除术更少、治疗时间更短、透明细胞组织学更多。接受二线或三线后续治疗的舒尼替尼患者更多(56% 和 23% 对 43% 和 13%)。虽然不同治疗方法在总生存期(OS)或无进展生存期(PFS)方面没有显著差异,但在多预测因子分析中,PFS的差异接近显著(卡博赞替尼获益;P = 0.06)。当 Kaplan-Meier 曲线按风险状态(中度风险与重度风险)分层时,各风险组患者的 OS 相似。PFS似乎存在差异,风险较低的患者使用卡博替尼后表现更好。治疗的逆概率加权分析显示,与未调整的结果差别不大:卡博替尼对舒尼替尼的OS危险比=1.119(95%置信区间(CI)0.823-1.521);PFS危险比0.825(95% CI 0.636-1.070)。卡博替尼有改善 PFS 和降低 OS 的趋势。酪氨酸激酶抑制剂单药治疗的决策应考虑晚期治疗方案。由于舒尼替尼目前已过专利期,这意味着一个疗程的费用已大大降低,因此这项分析具有特别重要的意义。
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引用次数: 0
Human-centered AI as a framework guiding the development of image-based diagnostic tools in oncology: a systematic review 以人为本的人工智能作为肿瘤学图像诊断工具开发的指导框架:系统综述
Pub Date : 2024-10-07 DOI: 10.1016/j.esmorw.2024.100077
K. Allen , A.K. Yawson , S. Haggenmüller , J.N. Kather , T.J. Brinker

Background

Artificial intelligence diagnostic tools (AIDTs) in oncology show high image classification accuracy but limited clinical adoption. Their adoption could be enhanced by (i) using user feedback during the software design, (ii) demonstrating that AIDTs improve the user’s decisions, and (iii) providing explanations of AI decisions tailored to the user, three aspects central to human-centered AI (HCAI). This review assesses these three aspects in AIDTs for oncology in general, exemplifying its concepts in the established field of skin cancer diagnostics as a specific use case.

Materials and methods

We carried out three Preferred Reporting Items for Systematic reviews and Meta-Analyses (PRISMA) searches using PubMed and ScienceDirect, limiting the results to articles published from 2019 to 2024. The first search focused on articles that used user feedback to develop AIDTs. The second search addressed whether AIDT improves dermatologists’ decisions. The third search targeted explainable AI in skin cancer.

Results

Five studies incorporated user feedback in AIDT design for cancer. Zooming in on AIDT for skin cancer, nine studies (3/37 in 2019, 3/93 in 2023) indicated that AIDTs improve dermatologists’ decisions in experimental (n = 5) and clinical settings (n = 1). Explainable AI was common in skin cancer diagnostics (n = 26), with papers assessing the user’s preference for explainable AI (XAI) methods or the impact of XAI on the user’s trust in AI diagnosis.

Conclusions

User feedback has been used to develop AIDTs tailored to clinicians’ needs. Evidence shows that AIDTs can improve clinicians’ decisions. This, combined with XAI, increases clinicians’ trust in AIDTs, potentially favoring their widespread usage.
背景肿瘤学领域的人工智能诊断工具(AIDT)显示出很高的图像分类准确性,但临床应用却很有限。可以通过以下方式提高其采用率:(i) 在软件设计过程中使用用户反馈;(ii) 证明人工智能诊断工具能改善用户的决策;(iii) 提供针对用户的人工智能决策解释,这三个方面是以人为本的人工智能(HCAI)的核心。本综述从总体上评估了肿瘤学 AIDTs 的这三个方面,并以皮肤癌诊断这一成熟领域的具体使用案例为例说明了其概念。材料与方法我们使用 PubMed 和 ScienceDirect 进行了三次系统综述和 Meta 分析首选报告项目(PRISMA)检索,检索结果仅限于 2019 年至 2024 年发表的文章。第一项检索侧重于使用用户反馈开发 AIDT 的文章。第二项搜索涉及 AIDT 是否改善了皮肤科医生的决策。第三次搜索针对皮肤癌中的可解释人工智能。结果五项研究将用户反馈纳入了癌症AIDT的设计中。在皮肤癌 AIDT 方面,9 项研究(2019 年 3/37 项,2023 年 3/93 项)表明,AIDT 在实验(5 项)和临床(1 项)环境中改善了皮肤科医生的决策。可解释人工智能在皮肤癌诊断中很常见(n = 26),有论文评估了用户对可解释人工智能(XAI)方法的偏好或 XAI 对用户对人工智能诊断信任度的影响。有证据表明,人工智能诊断可改善临床医生的决策。这一点与 XAI 相结合,增加了临床医生对 AIDTs 的信任,从而有可能促进 AIDTs 的广泛使用。
{"title":"Human-centered AI as a framework guiding the development of image-based diagnostic tools in oncology: a systematic review","authors":"K. Allen ,&nbsp;A.K. Yawson ,&nbsp;S. Haggenmüller ,&nbsp;J.N. Kather ,&nbsp;T.J. Brinker","doi":"10.1016/j.esmorw.2024.100077","DOIUrl":"10.1016/j.esmorw.2024.100077","url":null,"abstract":"<div><h3>Background</h3><div>Artificial intelligence diagnostic tools (AIDTs) in oncology show high image classification accuracy but limited clinical adoption. Their adoption could be enhanced by (i) using user feedback during the software design, (ii) demonstrating that AIDTs improve the user’s decisions, and (iii) providing explanations of AI decisions tailored to the user, three aspects central to human-centered AI (HCAI). This review assesses these three aspects in AIDTs for oncology in general, exemplifying its concepts in the established field of skin cancer diagnostics as a specific use case.</div></div><div><h3>Materials and methods</h3><div>We carried out three Preferred Reporting Items for Systematic reviews and Meta-Analyses (PRISMA) searches using PubMed and ScienceDirect, limiting the results to articles published from 2019 to 2024. The first search focused on articles that used user feedback to develop AIDTs. The second search addressed whether AIDT improves dermatologists’ decisions. The third search targeted explainable AI in skin cancer.</div></div><div><h3>Results</h3><div>Five studies incorporated user feedback in AIDT design for cancer. Zooming in on AIDT for skin cancer, nine studies (3/37 in 2019, 3/93 in 2023) indicated that AIDTs improve dermatologists’ decisions in experimental (<em>n</em> = 5) and clinical settings (<em>n</em> = 1). Explainable AI was common in skin cancer diagnostics (<em>n</em> = 26), with papers assessing the user’s preference for explainable AI (XAI) methods or the impact of XAI on the user’s trust in AI diagnosis.</div></div><div><h3>Conclusions</h3><div>User feedback has been used to develop AIDTs tailored to clinicians’ needs. Evidence shows that AIDTs can improve clinicians’ decisions. This, combined with XAI, increases clinicians’ trust in AIDTs, potentially favoring their widespread usage.</div></div>","PeriodicalId":100491,"journal":{"name":"ESMO Real World Data and Digital Oncology","volume":"6 ","pages":"Article 100077"},"PeriodicalIF":0.0,"publicationDate":"2024-10-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142420554","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
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ESMO Real World Data and Digital Oncology
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