EJC paediatric oncology最新文献

{"title":"New insights into hematopoietic cell transplantation in ALL: Who should be transplanted, when, and how","authors":"","doi":"10.1016/j.ejcped.2024.100200","DOIUrl":"10.1016/j.ejcped.2024.100200","url":null,"abstract":"<div><div>Advancements in multi-agent chemotherapy through clinical trials conducted by multi-institutional collaborative groups worldwide, along with risk stratification using molecular genetic features and treatment responses, including minimal residual disease, have significantly improved outcomes for children and adolescents with acute lymphoblastic leukemia (ALL) over the past half-century. However, for a certain number of high-risk patients, including those with relapsed or refractory disease, for whom existing chemotherapy alone is insufficient for cure, allogeneic hematopoietic cell transplantation (HCT) has provided a potential opportunity for leukemia cure. For these patients, the appropriate selection of donor and stem cell source, conditioning regimen, timing of transplantation, and comprehensive supportive care, including effective graft-versus-host disease prophylaxis, are prerequisites for successful HCT. While HCT from a human leukocyte antigen (HLA)-matched sibling has traditionally been the preferred option, less than 25 % of patients currently have such a donor in developed countries. Consequently, alternative donor HCT options, such as those from matched unrelated donors identified through high-resolution HLA typing, unrelated cord blood donors, and more recently, haploidentical donors using post-transplant cyclophosphamide or TCRαβ+/CD19+ cell-depleted grafts, are providing broader access to HCT for patients lacking matched sibling donors. Nonetheless, HCT carries the risk of various acute and late toxicities. In particular, the use of myeloablative conditioning with total body irradiation, a standard in pediatric ALL, is associated with significant long-term sequelae. As our understanding of the pathophysiology of the disease improves and novel molecular targeted therapies and immunotherapies are developed, the indication for HCT in pediatric ALL is becoming more selective, leading to a gradual decrease in the number of transplants performed. However, further optimization and evolution of allogeneic HCT are needed to both maximize its anti-leukemia effects and minimize transplant-related complications, as there remain cases that undoubtedly require HCT for the cure of leukemia.</div></div>","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2024-10-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142571256","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}

{"title":"Patients’, parents’, and survivors’ perspective about AI applications in pediatric oncology","authors":"","doi":"10.1016/j.ejcped.2024.100201","DOIUrl":"10.1016/j.ejcped.2024.100201","url":null,"abstract":"<div><div>Patients, parents and survivors of childhood cancer are those who represent the critical link between the clinical and technical research spheres. Thus, UNICA4EU worked towards a patient- centric approach to integrate AI in the care pathways for childhood cancer, with evidence-based patient advocacy at its core to build trust while protecting and guaranteeing patients’ fundamental rights <span><span>[1]</span></span>.</div><div>The task “Increase Knowledge and Transparency about AI among patients, parents and survivors” was led by CCI Europe as the biggest pan-European childhood cancer parents’ and survivors’ organization which represents childhood cancer parents´ and survivors´ groups and other childhood cancer organizations and reunites 63 member-organizations in 34 countries <span><span>[2]</span></span>.</div><div>To investigate the knowledge base of AI application in pediatric oncology among those who are affected, a survey was conducted. The survey translated into nine European languages, gathered responses from 332 individuals. To delve deeper into the survey findings, discussions were held with a diverse focus group, including four parents of childhood cancer former patients (survivors), three childhood cancer survivors, and one bereaved parent, each representing different backgrounds, age groups, and countries.</div><div>Insights and outcomes of this study produced a report for guiding the multi-stakeholder board of the project when defining the governance structures reg. data sharing, ownership, protection, access and usage.</div><div>Perspective of parents, patients and survivors of pediatric cancer regarding AI applications in Pediatric Oncology focused in six areas of interest including: data anonymization and data protection, data ownership, data withdrawal, ethical concerns of use of data, data types and, additionally, informed consents. This paper summarizes the respective results, along concluding policy recommendations.</div></div>","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2024-10-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142586831","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}

{"title":"Enhancing pediatric oncology clinical trials through patient reported outcomes (PROs)","authors":"","doi":"10.1016/j.ejcped.2024.100199","DOIUrl":"10.1016/j.ejcped.2024.100199","url":null,"abstract":"<div><div>Survival rates of children with cancer have significantly increased over the last decades in high income countries. However, cancer survivors often face significant long-term adverse effects on physical, psychosocial, and neurocognitive health. The use of Patient-Reported Outcomes (PROs) in clinical trials may help to further optimize treatment regimens to minimize acute and late adverse events. Despite clear recommendations of regulatory agencies, the use of PROs in pediatric clinical trials remains limited. In this article, we discuss the rationale to assess PROs in pediatric clinical trials with a specific highlight on electronic PRO (ePRO) assessment. In addition, we underscore the importance of recent international guideline developments such as the <em>Setting International Standards in Analyzing Patient-Reported Outcomes and Quality of Life Endpoints Data</em> (SISAQOL-IMI) and the <em>CONsolidated Standards of Reporting Trials – Patient-Reported Outcomes</em> (CONSORT-PRO) to improve the robustness of PRO data assessment in pediatric oncology, thus facilitating better patient care and more accurate assessments of treatment effects. Best practice examples for the use of modern technologies in ePRO assessments are presented and potential barriers and challenges are discussed. We conclude that – despite some challenges and barriers – the routine assessment of (e)PROs in pediatric clinical trials has the potential to substantially improve the quality of the trials and facilitate the usability of outcome data. We call for a joint effort to take proactive steps to incorporate PROs into clinical trials in pediatric oncology, thus giving children a voice.</div></div>","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2024-10-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142533856","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}

{"title":"A practical guide to apply AI in childhood cancer: Data collection and AI model implementation","authors":"","doi":"10.1016/j.ejcped.2024.100197","DOIUrl":"10.1016/j.ejcped.2024.100197","url":null,"abstract":"<div><div>Childhood cancer is a leading cause of death in children, and the increasing availability of digital healthcare data, coupled with rapid progress in artificial intelligence (AI), brings a transformative opportunity to revolutionise its diagnosis, treatment and ultimately improve patient outcomes by leveraging diverse data resources. However, the effective application of AI in childhood cancer requires strict adherence to regulatory and best practice guidelines for patient data preparation and AI model development. Currently, there is a lack of such regulatory and methodological guidance specifically tailored for the paediatric community. This review seeks to address this gap. Beginning with an overview of existing regulatory frameworks, it examines the types of data currently in use or with potential use in developing AI applications for childhood cancer. This encompasses data from traditional sources, such as patient data and electronic health records (EHRs), as well as emerging sources like social media data and social determinants of health. This review also outlines the rules and criteria for collecting, processing, and sharing these data. Informed consent and re-consent are required for data collection and re-use, and data quality, privacy, and security as well as data standardisation, harmonisation and interoperability are important for data processing. Additionally, this review clarifies the essential requirements and methodologies for developing AI models in childhood cancer and healthcare. It also emphasises the importance of AI being trustworthy, protecting privacy, and being accountable and validated in clinical settings. By systematically addressing these key components, this review aims to provide comprehensive knowledge and practical tools for the reliable application and implementation of AI in paediatric cancer to enhance AI acceptance and promote its widespread integration within the childhood cancer community. This, in turn, will lead to improved diagnosis, treatment and outcomes for children with cancer.</div></div>","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2024-10-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142533857","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}

{"title":"The impact of methylome analysis on the diagnosis and treatment of CNS tumours in children and adolescents: A population-based study in Greece","authors":"","doi":"10.1016/j.ejcped.2024.100198","DOIUrl":"10.1016/j.ejcped.2024.100198","url":null,"abstract":"<div><h3>Background</h3><div>The recently published WHO classification of central nervous system (CNS) tumours recognizes DNA methylation profiling as a desirable and, for some diagnoses, essential diagnostic tool adjunctive to conventional histopathology. DNA methylation profiling is not routinely available in many countries, including Greece.</div></div><div><h3>Methods</h3><div>In this collaborative study, we report the DNA methylation results in a series of children and adolescents with CNS tumours in Greece (2018–2023). In total, 130 tumour samples were analyzed using the latest applicable version of the Heidelberg brain tumour classifier.</div></div><div><h3>Results</h3><div>Upon initial analysis, 80 % (104/130) achieved calibrated scores (Cs) ≥ 0.9 and matched an established methylation class family/subclass. Among them, methylation results confirmed (90/104, 86.5 %), refined (50/104, 48 %) or changed (10/104, 9.6 %) the histological diagnosis. Only four results were regarded as non-contributing (4/104, 3.9 %). Twenty-six tumour samples received Cs &lt; 0.9. Despite low scores, methylation results supported the initial diagnosis with lower confidence in 38.5 % (10/26) and established the diagnosis in two tumours with non-conclusive histopathology. Additional t-distributed stochastic neighbour embedding (t-SNE) analysis allowed the possible classification of twelve tumours. Nine more samples reached high Cs using the newer brain tumour classifiers, since available. Samples co-tested in Greece demonstrated excellent test reproducibility, supporting the analysis' local implementation. Methylome profiling impacted the clinical management of 40 % of patients, modifying stratification, prognosis, or treatment approach.</div></div><div><h3>Conclusions</h3><div>This study supports the need to integrate methylome analysis into routine diagnostics in our country and highlights the importance of collaboration between European pediatric oncology centres.</div></div>","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2024-10-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142417479","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}

{"title":"Health-related quality of life of toddlers during and after cancer treatment","authors":"","doi":"10.1016/j.ejcped.2024.100194","DOIUrl":"10.1016/j.ejcped.2024.100194","url":null,"abstract":"<div><h3>Background</h3><div>There is a knowledge gap regarding health-related quality of life (HRQOL) in childhood cancer patients below 2 years of age. The aim of this study was therefore to compare HRQOL of young children during and after treatment for cancer, to healthy controls, and to investigate effects of biopsychosocial factors.</div></div><div><h3>Procedure</h3><div>The study is based on data from an online monitoring program. Parent-proxy reports of HRQOL in 205 children aged 12–24 months were compared to 108 healthy children. The parents filled out the TNO-AZL Preschool Quality of Life questionnaire for young children, which consists of 12 subscales, in addition to a parental distress thermometer.</div></div><div><h3>Results</h3><div>Participants undergoing treatment had less favorable HRQOL than children after treatment for eight of the subscales. They also had less favorable HRQOL than healthy children for seven scales, while the only significant difference between children off treatment and healthy controls was for motor functioning. For ten subscales, there were significant relationships between biopsychosocial variables and HRQOL outcomes. Parental distress and treatment with immunotherapy were the variables most frequently associated with lower HRQOL.</div></div><div><h3>Conclusions</h3><div>Parental distress should be considered when monitoring young childhood cancer patients. Treatment with immunotherapy is likely to be a marker of disease severity and might represent other underlying factors affecting HRQOL. The association between immunotherapy and HRQOL therefore needs more research. While there was a significant difference depending on treatment status, it cannot be concluded that this represents an improvement in HRQOL after end of treatment.</div></div>","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2024-10-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142417481","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}

{"title":"General context and relevant public datasets available for improving pathways in Paediatric Cancer applying Artificial Intelligence. A review","authors":"","doi":"10.1016/j.ejcped.2024.100196","DOIUrl":"10.1016/j.ejcped.2024.100196","url":null,"abstract":"<div><div>Due to the promise of transforming healthcare and medicine that Artificial Intelligence (AI) has posed, the number of applications has increased exponentially. These applications range from screening and disease diagnosis to prognosis, treatment planning, and follow-up. In complex topics such as childhood cancer, these techniques are being expanded with the ambition of improving the quality of care by allowing healthcare professionals to make more informed decisions. However, the adequate application of such techniques heavily depends on the data, which creates a set of challenges including collection, bias, and scarcity among others. Furthermore, ethical, legal, and regulatory frameworks increase even more the difficulties to develop AI-powered solutions. In this paper, we present an exhaustive literature review to identify and analyse public datasets targeting two common childhood cancer types, such as neuroblastoma and nephroblastoma. Moreover, the complex context for the development of AI- based software solutions is outlined. It includes the description of the most relevant techniques to address problems associated with data sharing and training. Finally, a set of code snippets is provided to perform exploratory analysis for the available data.</div></div>","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2024-10-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142417482","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}

{"title":"Risk of severe esophageal stricture among childhood cancer survivors – A population-based case-cohort study within the Adult Life after Childhood Cancer in Scandinavia (ALiCCS)","authors":"","doi":"10.1016/j.ejcped.2024.100195","DOIUrl":"10.1016/j.ejcped.2024.100195","url":null,"abstract":"<div><h3>Purpose</h3><div>Due to limited data on treatment-related risk factors associated with esophageal stricture in childhood cancer survivors, this study aimed to assess such factors in long-term survivors.</div></div><div><h3>Methods</h3><div>A case-cohort study was conducted involving 36 cases of five-year childhood cancer survivors with esophageal stricture and a sub-cohort of 540 survivors diagnosed with cancer in 1970–2007 as identified within the Nordic ‘Adult Life after Childhood Cancer in Scandinavia’ program. Individualized treatment details were retrieved from medical records. Radiation doses to each body region and average dose to the esophagus were reconstructed for patients that received radiotherapy. We used a modified Cox proportional hazard model to evaluate associations between esophageal stricture and risk factors by calculating incidence rate ratio (IRR), with 95 % confidence intervals (CIs).</div></div><div><h3>Results</h3><div>An increased rate of esophageal stricture was found in survivors who received total body irradiation (IRR=13.7, 95 %CI 4.6–41.1), chest- and neck-directed radiotherapy (IRR=23.5, 95 %CI 8.5−64.7) and doses of ≥12 Gy to the esophagus (IRR=26.8, 95 % CI=9.0–80.3) compared to non-irradiated survivors. Treatment with chemotherapy was also associated with esophageal stricture (IRR=8.4, 95 % CI=2.9–24.4). Notably, leukemia survivors faced an elevated rate (IRR=3.8, 95 % CI 1.8–8.1) compared with survivors of CNS and other solid tumors.</div></div><div><h3>Conclusions</h3><div>Our findings indicate an increased risk of esophageal stricture among childhood cancer survivors, with both neck- and chest-directed radiotherapy and chemotherapy as important risk factors.</div></div>","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2024-10-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142417480","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}

{"title":"Young paediatric oncologists and haematologists in Europe – Emerging needs and future perspectives","authors":"","doi":"10.1016/j.ejcped.2024.100192","DOIUrl":"10.1016/j.ejcped.2024.100192","url":null,"abstract":"<div><h3>Background</h3><div>The “Young NaPHOS” project was launched in 2021, by Young SIOPE, the group for junior members of the European Society for Paediatric Oncology (SIOPE). The Young NaPHOS project aims to build a network of national representatives of young paediatric haematologists and oncologists to discuss organisational and educational aspects at national and European level, differences in the training paths, and the needs to be addressed in the future.</div></div><div><h3>Methods</h3><div>From June 2021 until September 2022, three online meetings took place and one online survey was conducted. The meetings focused on presenting the structure and organisational aspects of existing national junior organisations in paediatric haematology and oncology, including their national activities. The survey aimed to investigate the European landscape of national organisations and training paths by inviting 39 young national representatives.</div></div><div><h3>Results</h3><div>Ten out of 34 responders confirmed the existence of a junior organisation in their country. Diversity was noted among the countries for the existence of such organisations, the organisations’ activities, or the membership criteria. Further, the training path to become a paediatric oncologist/haematologist differs a lot. Young paediatric oncologists also shared common concerns, especially regarding networking, international collaboration, and educational opportunities.</div></div><div><h3>Conclusion</h3><div>This project resulted in the creation of a European network among young paediatric oncologists and haematologists, in spreading ideas and offering support, and identified differences and areas for future action of Young SIOPE.</div></div>","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2024-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142417483","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}

{"title":"Response to induction chemotherapy modifies the effect of conventional prognostic factors in high-risk neuroblastoma: A report from the Children’s Oncology Group","authors":"","doi":"10.1016/j.ejcped.2024.100193","DOIUrl":"10.1016/j.ejcped.2024.100193","url":null,"abstract":"<div><h3>Background</h3><div>Response to induction chemotherapy has been shown to predict outcome in patients with high-risk neuroblastoma (HR-NB), with those achieving a complete response (CR) having superior outcomes.</div></div><div><h3>Methods</h3><div>We evaluated whether conventional prognostic factors remain prognostic in subsets of patients defined by response to induction. 1244 Patients from four COG high-risk trials were included. End-induction response was coded as CR, partial response (PR) or better, less than PR without progressive disease (PD), and PD. Cox regression models were performed to calculate event-free and overall survival (EFS, OS) hazard ratios, including interaction terms between induction response and prognostic factors including sex, age, stage, primary tumor location, LDH, ferritin, ploidy, <em>MYCN</em> status, <em>ALK</em> status, histology, MKI, grade, and study era.</div></div><div><h3>Results</h3><div>Among patients who achieved a CR after induction, INSS stage 4 disease and trial era were the only factors that remained significantly associated with inferior OS. For those who achieved less than a PR, adrenal primary site, <em>MYCN</em> amplification, and 1p LOH were associated with inferior outcomes. Multivariable models showed that end-induction response remained prognostic of EFS and OS even after controlling for other factors. Multiple significant statistical interactions were observed between end-induction response and other prognostic factors.</div></div><div><h3>Conclusion</h3><div>The impact of conventional prognostic factors is not static in patients with HR-NB. Instead, response to induction chemotherapy modifies the effect of conventional prognostic factors. These data can help to further refine prognosis for patients with variable responses to induction and help to identify candidates who might benefit from treatment other than standard post-induction therapy.</div></div>","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2024-09-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142417478","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}