EJC paediatric oncology最新文献

New insights into hematopoietic cell transplantation in ALL: Who should be transplanted, when, and how 对 ALL 造血细胞移植的新认识：谁应该移植、何时移植、如何移植

EJC paediatric oncology

Pub Date : 2024-10-29 DOI: 10.1016/j.ejcped.2024.100200

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.

在过去的半个世纪里，通过全球多机构合作小组开展的临床试验，以及利用分子遗传特征和治疗反应（包括最小残留病）进行的风险分层，多药化疗取得了进展，大大改善了急性淋巴细胞白血病（ALL）儿童和青少年患者的治疗效果。然而，对于一定数量的高危患者，包括复发或难治性患者，仅靠现有的化疗不足以治愈，异基因造血细胞移植（HCT）为治愈白血病提供了潜在的机会。对这些患者来说，选择合适的供体和干细胞来源、调理方案、移植时机和全面的支持治疗，包括有效的移植物抗宿主病预防措施，是成功进行 HCT 的先决条件。虽然人类白细胞抗原（HLA）匹配的同胞进行造血干细胞移植历来是首选，但在发达国家，目前只有不到 25% 的患者有这样的供体。因此，通过高分辨率 HLA 分型确定的匹配非亲属捐献者、非亲属脐带血捐献者以及最近使用移植后环磷酰胺或 TCRαβ+/CD19+ 细胞贫化移植物的单倍体捐献者等替代捐献者的 HCT 选择，为缺乏匹配兄弟姐妹捐献者的患者提供了更广泛的 HCT 途径。尽管如此，造血干细胞移植仍存在各种急性和晚期毒性反应的风险。特别是，使用全身照射的髓脱落调理（小儿 ALL 的标准治疗方法）会带来严重的长期后遗症。随着我们对该病病理生理学认识的提高以及新型分子靶向疗法和免疫疗法的开发，小儿 ALL 的造血干细胞移植适应症变得更具选择性，从而导致移植数量逐渐减少。然而，异基因造血干细胞移植仍需进一步优化和发展，以最大限度地发挥其抗白血病作用，并减少移植相关并发症，因为仍有一些病例无疑需要通过造血干细胞移植来治愈白血病。

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引用次数: 0

Patients’, parents’, and survivors’ perspective about AI applications in pediatric oncology 患者、家长和幸存者对人工智能在儿科肿瘤学中应用的看法

EJC paediatric oncology

Pub Date : 2024-10-28 DOI: 10.1016/j.ejcped.2024.100201

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 [1].

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 [2].

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.

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.

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.

儿童癌症患者、家长和幸存者是临床和技术研究领域之间的重要纽带。因此，UNICA4EU 致力于采取以患者为中心的方法，将人工智能纳入儿童癌症的治疗路径，其核心是以证据为基础的患者宣传，以建立信任，同时保护和保障患者的基本权利[1]。增加患者、家长和幸存者对人工智能的了解和透明度 "的任务由欧洲儿童癌症协会（CCI Europe）牵头，该协会是泛欧最大的儿童癌症家长和幸存者组织，代表儿童癌症家长和幸存者团体及其他儿童癌症组织，并联合了 34 个国家的 63 个成员组织[2]。该调查被翻译成九种欧洲语言，收集了 332 人的回复。为了深入了解调查结果，我们与不同的焦点小组进行了讨论，其中包括四位儿童癌症前患者（幸存者）的父母、三位儿童癌症幸存者和一位失去亲人的父母，他们分别代表了不同的背景、年龄组和国家。小儿癌症家长、患者和幸存者对人工智能在小儿肿瘤学中应用的看法主要集中在六个方面，包括：数据匿名化和数据保护、数据所有权、数据撤回、数据使用的伦理问题、数据类型以及知情同意。本文总结了相关结果，并提出了政策建议。

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引用次数: 0

Enhancing pediatric oncology clinical trials through patient reported outcomes (PROs) 通过患者报告结果（PROs）加强儿科肿瘤临床试验

EJC paediatric oncology

Pub Date : 2024-10-16 DOI: 10.1016/j.ejcped.2024.100199

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 Setting International Standards in Analyzing Patient-Reported Outcomes and Quality of Life Endpoints Data (SISAQOL-IMI) and the CONsolidated Standards of Reporting Trials – Patient-Reported Outcomes (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.

过去几十年来，高收入国家儿童癌症患者的存活率显著提高。然而，癌症幸存者往往在身体、社会心理和神经认知健康方面面临严重的长期不良影响。在临床试验中使用 "患者报告结果"（PROs）有助于进一步优化治疗方案，最大限度地减少急性和晚期不良反应。尽管监管机构提出了明确的建议，但在儿科临床试验中使用患者报告结果仍然有限。在本文中，我们讨论了在儿科临床试验中评估PRO的理由，并特别强调了电子PRO（ePRO）评估。此外，我们还强调了近期国际指南发展的重要性，如《分析患者报告结果和生活质量终点数据的国际标准设定》（SISAQOL-IMI）和《试验报告综合标准--患者报告结果》（CONSORT-PRO），以提高儿科肿瘤学中PRO数据评估的稳健性，从而促进更好的患者护理和更准确的治疗效果评估。我们介绍了在 ePRO 评估中使用现代技术的最佳实践范例，并讨论了潜在的障碍和挑战。我们的结论是，尽管存在一些挑战和障碍，但在儿科临床试验中常规评估（e）PROs 有可能大大提高试验质量，促进结果数据的可用性。我们呼吁大家共同努力，采取积极措施，将 PROs 纳入儿科肿瘤临床试验，从而让儿童有发言权。

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引用次数: 0

A practical guide to apply AI in childhood cancer: Data collection and AI model implementation 将人工智能应用于儿童癌症的实用指南：数据收集和人工智能模型实施

EJC paediatric oncology

Pub Date : 2024-10-09 DOI: 10.1016/j.ejcped.2024.100197

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.

儿童癌症是儿童死亡的主要原因之一，数字医疗数据的可用性不断提高，再加上人工智能（AI）的快速发展，为利用各种数据资源彻底改变儿童癌症的诊断和治疗并最终改善患者预后带来了变革性机遇。然而，要在儿童癌症中有效应用人工智能，就必须严格遵守有关患者数据准备和人工智能模型开发的法规和最佳实践指南。目前，还缺乏专门针对儿科群体的监管和方法指导。本综述旨在填补这一空白。它首先概述了现有的监管框架，然后研究了目前正在使用或可能用于开发儿童癌症人工智能应用的数据类型。这包括传统来源的数据，如患者数据和电子健康记录 (EHR)，以及新兴来源的数据，如社交媒体数据和健康的社会决定因素。本综述还概述了收集、处理和共享这些数据的规则和标准。数据收集和再利用需要知情同意和再同意，数据质量、隐私和安全以及数据标准化、协调性和互操作性对数据处理非常重要。此外，本综述还阐明了在儿童癌症和医疗保健领域开发人工智能模型的基本要求和方法。它还强调了人工智能在临床环境中值得信赖、保护隐私、负责和验证的重要性。通过系统地讨论这些关键要素，本综述旨在为人工智能在儿童癌症领域的可靠应用和实施提供全面的知识和实用工具，以提高人工智能的接受度，促进其在儿童癌症领域的广泛应用。这反过来又会改善癌症儿童的诊断、治疗和预后。

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引用次数: 0

The impact of methylome analysis on the diagnosis and treatment of CNS tumours in children and adolescents: A population-based study in Greece 甲基组分析对儿童和青少年中枢神经系统肿瘤诊断和治疗的影响：希腊人口研究

EJC paediatric oncology

Pub Date : 2024-10-06 DOI: 10.1016/j.ejcped.2024.100198

Background

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.

Methods

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.

Results

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 < 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.

Conclusions

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.

背景世界卫生组织（WHO）最近公布的中枢神经系统（CNS）肿瘤分类认为，DNA甲基化分析是一种理想的诊断工具，对于某些诊断而言，是辅助传统组织病理学的必要诊断工具。方法在这项合作研究中，我们报告了希腊一系列中枢神经系统肿瘤儿童和青少年的 DNA 甲基化结果（2018-2023 年）。共使用海德堡脑肿瘤分类器的最新适用版本分析了 130 个肿瘤样本。结果经初步分析，80%（104/130）的样本达到校准分数（Cs）≥ 0.9，并与已建立的甲基化类族/亚类相匹配。其中，甲基化结果证实（90/104，86.5%）、完善（50/104，48%）或改变（10/104，9.6%）了组织学诊断。只有四项结果被视为无影响（4/104，3.9%）。26 个肿瘤样本的 Cs 值为 0.9。尽管得分较低，但甲基化结果支持了 38.5%（10/26）置信度较低的初步诊断，并确定了两个组织病理学结果不确定的肿瘤的诊断。附加的 t 分布随机邻域嵌入（t-SNE）分析可对 12 个肿瘤进行分类。此外，还有九个样本在使用最新的脑肿瘤分类器后达到了较高的 Cs 值。在希腊共同测试的样本显示出极佳的测试重现性，支持了分析在当地的实施。这项研究支持了将甲基组分析纳入我国常规诊断的必要性，并强调了欧洲儿科肿瘤中心之间合作的重要性。

{"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 < 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}

引用次数: 0

Health-related quality of life of toddlers during and after cancer treatment 癌症治疗期间和之后幼儿的健康相关生活质量

EJC paediatric oncology

Pub Date : 2024-10-05 DOI: 10.1016/j.ejcped.2024.100194

Background

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.

Procedure

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.

Results

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.

Conclusions

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.

背景关于两岁以下儿童癌症患者的健康相关生活质量（HRQOL），目前还存在知识空白。因此，本研究旨在将癌症治疗期间和治疗后的幼儿与健康对照组的 HRQOL 进行比较，并调查生物心理社会因素的影响。将 205 名年龄在 12-24 个月的儿童的家长代理 HRQOL 报告与 108 名健康儿童进行了比较。家长们填写了TNO-AZL学龄前儿童生活质量调查问卷，该问卷由12个分量表组成，此外还有一个家长痛苦温度计。与健康儿童相比，接受治疗的儿童在七个分量表中的 HRQOL 也较差，而未接受治疗的儿童与健康对照组之间唯一的显著差异是运动功能。在十个分量表中，生物心理社会变量与 HRQOL 结果之间存在显著关系。结论：在监测幼儿癌症患者时，应考虑家长的苦恼。免疫治疗可能是疾病严重程度的标志，也可能是影响 HRQOL 的其他潜在因素。因此，需要对免疫疗法与 HRQOL 之间的关系进行更多研究。虽然不同的治疗状态有显著差异，但不能就此断定治疗结束后患者的 HRQOL 有所改善。

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引用次数: 0

General context and relevant public datasets available for improving pathways in Paediatric Cancer applying Artificial Intelligence. A review 应用人工智能改善儿科癌症治疗路径的一般背景和相关公共数据集。综述

EJC paediatric oncology

Pub Date : 2024-10-05 DOI: 10.1016/j.ejcped.2024.100196

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.

由于人工智能（AI）有望改变医疗保健和医学，因此其应用数量呈指数级增长。这些应用范围从筛查和疾病诊断到预后、治疗计划和随访。在儿童癌症等复杂问题上，这些技术正在不断扩展，目的是通过让医疗专业人员做出更明智的决定来提高医疗质量。然而，这些技术的充分应用在很大程度上取决于数据，而数据的收集、偏差和稀缺性等问题带来了一系列挑战。此外，伦理、法律和监管框架也增加了开发人工智能解决方案的难度。在本文中，我们通过详尽的文献综述，确定并分析了针对神经母细胞瘤和肾母细胞瘤这两种常见儿童癌症类型的公共数据集。此外，我们还概述了开发基于人工智能的软件解决方案的复杂背景。其中包括描述解决数据共享和训练相关问题的最相关技术。最后，还提供了一组代码片段，用于对现有数据进行探索性分析。

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引用次数: 0

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) 儿童癌症幸存者患严重食管狭窄的风险--斯堪的纳维亚半岛儿童癌症后成人生活（ALiCCS）中的一项基于人群的病例队列研究

EJC paediatric oncology

Pub Date : 2024-10-03 DOI: 10.1016/j.ejcped.2024.100195

Purpose

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.

Methods

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).

Results

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.

Conclusions

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.

目的由于儿童癌症幸存者中与食管狭窄相关的治疗相关风险因素的数据有限，本研究旨在评估长期幸存者中的此类因素。方法进行了一项病例队列研究，涉及 36 例患有食管狭窄的五年期儿童癌症幸存者，以及北欧 "斯堪的纳维亚儿童癌症后的成人生活 "计划中确定的 1970-2007 年期间诊断为癌症的 540 名幸存者的子队列。从医疗记录中获取了个性化的治疗细节。我们重建了接受放射治疗的患者身体各部位的辐射剂量和食道的平均剂量。通过计算发病率比（IRR）和 95 % 置信区间（CIs），我们使用改良的 Cox 比例危险模型来评估食管狭窄与风险因素之间的关联。7，95 %CI 4.6-41.1）、胸部和颈部定向放疗（IRR=23.5，95 %CI 8.5-64.7）和食管剂量≥12 Gy（IRR=26.8，95 % CI=9.0-80.3）的幸存者的食管狭窄率高于未接受放疗的幸存者。化疗也与食管狭窄有关（IRR=8.4，95 % CI=2.9-24.4）。值得注意的是，与中枢神经系统和其他实体瘤的幸存者相比，白血病幸存者的食管狭窄发生率更高（IRR=3.8，95 % CI=1.8-8.1）。

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引用次数: 0

Young paediatric oncologists and haematologists in Europe – Emerging needs and future perspectives 欧洲年轻儿科肿瘤学家和血液学家--新需求和未来展望

EJC paediatric oncology

Pub Date : 2024-10-01 DOI: 10.1016/j.ejcped.2024.100192

Background

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.

Methods

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.

Results

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.

Conclusion

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.

背景 "Young NaPHOS "项目于 2021 年由 Young SIOPE（欧洲儿科肿瘤学会 (SIOPE) 初级会员团体）发起。Young NaPHOS 项目旨在建立一个由各国年轻儿科血液学和肿瘤学专家代表组成的网络，讨论国家和欧洲层面的组织和教育问题、培训路径的差异以及未来需要解决的问题。方法从 2021 年 6 月到 2022 年 9 月，共举行了三次在线会议，并进行了一次在线调查。会议重点介绍了儿科血液学和肿瘤学现有国家初级组织的结构和组织方面，包括其国家活动。调查旨在通过邀请 39 位年轻的国家代表调查欧洲国家组织和培训途径的情况。各国在此类组织的存在、组织活动或成员标准方面存在差异。此外，成为儿科肿瘤学家/血液学家的培训途径也大不相同。年轻的儿科肿瘤学家也有共同的担忧，尤其是在网络、国际合作和教育机会方面。结论：该项目在年轻的儿科肿瘤学家和血液学家中建立了一个欧洲网络，传播了理念并提供了支持，同时也发现了不同之处以及青年 SIOPE 未来的行动领域。

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引用次数: 0

Response to induction chemotherapy modifies the effect of conventional prognostic factors in high-risk neuroblastoma: A report from the Children’s Oncology Group 对诱导化疗的反应可改变传统预后因素对高危神经母细胞瘤的影响：儿童肿瘤学小组的报告

EJC paediatric oncology

Pub Date : 2024-09-27 DOI: 10.1016/j.ejcped.2024.100193

Background

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.

Methods

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, MYCN status, ALK status, histology, MKI, grade, and study era.

Results

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, MYCN 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.

Conclusion

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.

背景诱导化疗反应已被证明可预测高危神经母细胞瘤（HR-NB）患者的预后，其中获得完全反应（CR）的患者预后更佳。我们纳入了来自四项COG高风险试验的1244名患者。诱导末期反应分为CR、部分反应（PR）或更好、小于PR但无进展性疾病（PD）和PD。采用Cox回归模型计算无事件生存率和总生存率（EFS、OS）危险比，包括诱导反应与性别、年龄、分期、原发肿瘤位置、LDH、铁蛋白、倍体、MYCN状态、ALK状态、组织学、MKI、分级和研究年代等预后因素之间的交互项。在获得PR以下的患者中，肾上腺原发部位、MYCN扩增和1p LOH与不良预后相关。多变量模型显示，即使在控制了其他因素后，诱导末期反应仍是EFS和OS的预后因素。结论常规预后因素对HR-NB患者的影响并非一成不变。相反，诱导化疗反应会改变传统预后因素的影响。这些数据有助于进一步完善对诱导化疗反应不一的患者的预后，并帮助确定可能从诱导化疗后标准治疗以外的治疗中获益的患者。

{"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}

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