Pub Date : 2024-12-01DOI: 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.
{"title":"The impact of targeted therapies on molecular alterations identified by an institutional molecular tumor board: an approach based on ESCAT classification","authors":"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","doi":"10.1016/j.esmorw.2024.100092","DOIUrl":"10.1016/j.esmorw.2024.100092","url":null,"abstract":"<div><h3>Background</h3><div>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.</div></div><div><h3>Patients and methods</h3><div>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.</div></div><div><h3>Results</h3><div>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 (<em>P</em> = 0.009 and <em>P</em> = 0.014, respectively).</div></div><div><h3>Conclusions</h3><div>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.</div></div>","PeriodicalId":100491,"journal":{"name":"ESMO Real World Data and Digital Oncology","volume":"6 ","pages":"Article 100092"},"PeriodicalIF":0.0,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143173393","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}
Pub Date : 2024-12-01DOI: 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
{"title":"Bridging research and practice: enhancing regulatory decisions with pragmatic clinical trials in oncology","authors":"M.D. Stewart , A.E. McKee , R.S. Herbst , H.S. Andrews , B.A. McKelvey , E.V. Sigal , J.D. Allen","doi":"10.1016/j.esmorw.2024.100065","DOIUrl":"10.1016/j.esmorw.2024.100065","url":null,"abstract":"","PeriodicalId":100491,"journal":{"name":"ESMO Real World Data and Digital Oncology","volume":"6 ","pages":"Article 100065"},"PeriodicalIF":0.0,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143173395","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}
Pub Date : 2024-12-01DOI: 10.1016/S2949-8201(24)00079-1
{"title":"Editoiral Board","authors":"","doi":"10.1016/S2949-8201(24)00079-1","DOIUrl":"10.1016/S2949-8201(24)00079-1","url":null,"abstract":"","PeriodicalId":100491,"journal":{"name":"ESMO Real World Data and Digital Oncology","volume":"6 ","pages":"Article 100101"},"PeriodicalIF":0.0,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143174431","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}
Pub Date : 2024-12-01DOI: 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.
{"title":"Characteristics and impact of real-world evidence studies in oncology: comprehensive mapping review of publications evaluating targeted therapies in solid tumours","authors":"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","doi":"10.1016/j.esmorw.2024.100091","DOIUrl":"10.1016/j.esmorw.2024.100091","url":null,"abstract":"<div><h3>Background</h3><div>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.</div></div><div><h3>Methods</h3><div>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.</div></div><div><h3>Results</h3><div>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.</div></div><div><h3>Conclusions</h3><div>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.</div></div>","PeriodicalId":100491,"journal":{"name":"ESMO Real World Data and Digital Oncology","volume":"6 ","pages":"Article 100091"},"PeriodicalIF":0.0,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143173394","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}
Pub Date : 2024-11-19DOI: 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。
{"title":"Neoadjuvant compared to adjuvant chemotherapy combined with trastuzumab in patients with HER2-positive breast cancer: a register-based cohort study","authors":"S. Hosseini-Mellner , Å. Wickberg , E. Olsson , A. Karakatsanis , A. Valachis","doi":"10.1016/j.esmorw.2024.100093","DOIUrl":"10.1016/j.esmorw.2024.100093","url":null,"abstract":"<div><h3>Background</h3><div>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.</div></div><div><h3>Patients and methods</h3><div>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.</div></div><div><h3>Results</h3><div>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.</div></div><div><h3>Conclusion</h3><div>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.</div></div>","PeriodicalId":100491,"journal":{"name":"ESMO Real World Data and Digital Oncology","volume":"6 ","pages":"Article 100093"},"PeriodicalIF":0.0,"publicationDate":"2024-11-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142698586","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}
Pub Date : 2024-11-19DOI: 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.
{"title":"Real-world evaluation of ImmuCare-PRO patient-reported outcomes in melanoma patients treated with immune checkpoint inhibitors","authors":"S. Belkaïd , S. Milley , R. Saux , M. Bonjour , A. Augros , P.-J. Souquet , D. Maillet , D. Maucort-Boulch , C. Dolla , L. Thomas , S. Dalle","doi":"10.1016/j.esmorw.2024.100090","DOIUrl":"10.1016/j.esmorw.2024.100090","url":null,"abstract":"<div><h3>Background</h3><div>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.</div></div><div><h3>Patients and methods</h3><div>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.</div></div><div><h3>Results</h3><div>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, <em>n</em> = 31) were not detected. False alerts often resulted from functional or non-specific symptoms (32.3%), such as fatigue or general pain.</div></div><div><h3>Conclusion</h3><div>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.</div></div>","PeriodicalId":100491,"journal":{"name":"ESMO Real World Data and Digital Oncology","volume":"6 ","pages":"Article 100090"},"PeriodicalIF":0.0,"publicationDate":"2024-11-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142698585","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}
Pub Date : 2024-11-18DOI: 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.
{"title":"Challenges and opportunities for real-world evidence in clinical oncology—a view from the UK: proceedings of a national workshop","authors":"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","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}
Pub Date : 2024-10-23DOI: 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.
{"title":"Implementing digital patient-reported outcomes in routine cancer care: barriers and facilitators","authors":"S.G. Aanes , S. Wiig , C. Nieder , 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}
Pub Date : 2024-10-18DOI: 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。
{"title":"Efficacy of cabozantinib and sunitinib for the treatment of intermediate/poor risk renal cell carcinoma based upon UK real-world data","authors":"D. Lee , G.J. Melendez-Torres , A. Challapalli , R. Frazer , J. McGrane , A. Bahl","doi":"10.1016/j.esmorw.2024.100087","DOIUrl":"10.1016/j.esmorw.2024.100087","url":null,"abstract":"<div><h3>Background</h3><div>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.</div></div><div><h3>Materials and methods</h3><div>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.</div></div><div><h3>Results</h3><div>Cabozantinib patients (<em>n</em> = 106) had poorer risk status, less prior nephrectomy, shorter time to therapy, and more clear cell histology than sunitinib patients (<em>n</em> = 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; <em>P</em> = 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.</div></div><div><h3>Conclusions</h3><div>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.</div></div>","PeriodicalId":100491,"journal":{"name":"ESMO Real World Data and Digital Oncology","volume":"6 ","pages":"Article 100087"},"PeriodicalIF":0.0,"publicationDate":"2024-10-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142529798","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}
Pub Date : 2024-10-07DOI: 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.
{"title":"Human-centered AI as a framework guiding the development of image-based diagnostic tools in oncology: a systematic review","authors":"K. Allen , A.K. Yawson , S. Haggenmüller , J.N. Kather , 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}