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Experiences of receiving explanations about medical conditions among adolescent children with cancer: A qualitative study 青少年癌症患者接受医疗条件解释的经验:一项定性研究
Pub Date : 2026-01-20 DOI: 10.1016/j.ejcped.2026.100486
Yuta Kogumazaka , Takako Miyamura , Tae Kawahara , Tomoko Abe , Saaya Niino , Akemi Yamazaki

Background

Although international consensus has been reached on the implementation of open communication for children with cancer, few studies have captured the reality of their experiences of receiving explanations of their medical conditions. This study aimed to investigate the experiences of patients diagnosed with cancer during adolescence in terms of receiving explanations of their medical conditions from health-care professionals and parents, and to identify the trajectory of how adolescent children with cancer approach their treatment after receiving explanations.

Methods

Semi-structured interviews were conducted on 18 childhood cancer survivors who completed treatment between May 2024 and June 2025. The inclusion criteria included having been diagnosed with cancer between the ages of 11 and 18 years and having received an explanation of their condition. The grounded theory approach was used for the data analysis, which included a verbatim record and theoretical notes.

Results

Five categories were identified as Experiences of receiving explanations about medical conditions among adolescent children with cancer: “Receiving explanations of my medical condition”, “Mixed feelings”, “Concerns during hospitalization”, “I’ve got cancer”, and “The power to overcome treatment”.

Conclusions

The present findings suggest the need to provide psychological support during the process of explaining medical conditions to children. Furthermore, the concerns of children need to be captured in daily interactions, and practices that help them become aware of their disease and complete their treatment need to be developed.
虽然国际上已经就对癌症儿童实施公开沟通达成了共识,但很少有研究能够捕捉到他们接受医疗条件解释的现实经历。本研究旨在探讨青少年癌症患者在接受医疗保健专业人员和父母对其医疗状况的解释方面的经历,并确定青少年癌症患者在接受解释后如何接受治疗的轨迹。方法对2024年5月至2025年6月期间完成治疗的18例儿童癌症幸存者进行半结构化访谈。纳入标准包括在11岁至18岁之间被诊断患有癌症,并得到了对其病情的解释。数据分析采用扎根理论方法,包括逐字记录和理论注释。结果青少年癌症患者接受医疗状况解释的经历分为5类:“接受医疗状况解释”、“复杂的感受”、“住院期间的担忧”、“我得了癌症”和“战胜治疗的力量”。结论在向儿童解释医疗状况的过程中,需要提供心理支持。此外,需要在日常互动中关注儿童的关切,需要发展有助于儿童了解自己的疾病并完成治疗的做法。
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引用次数: 0
Making shared care work: A national qualitative case study on collaborative practice in pediatric oncology 共同护理工作:儿童肿瘤学合作实践的国家定性案例研究
Pub Date : 2026-01-14 DOI: 10.1016/j.ejcped.2026.100485
Natasja Dors , Martine van Grotel , Eline Zijtregtop , Nathalie van der Salm , Laura Keukens , Selma Lavrijssen , Charlotte Drykoningen , Anne Jonge Poerink , Rod Thompson , Jenneke Homan , Menco Weismuller , Wendy Nix , Hester Blufpand , Annemieke van der Wey , Daniëlle Martens , Marion Hekkelaan , Lotte Helder , Wouter Kollen , Max van Noesel , Marije P. Hennus

Background

In 2018, the Netherlands centralized all pediatric oncology care at the Prinses Máxima Center (Máxima), supported by a national network of 14 Shared Care centers. These centers deliver non-intensive treatment and supportive care closer to home, enabling a hybrid model of centralized expertise and regional accessibility. While structurally in place, little is known about how regional pediatricians experience this collaboration or which factors they consider essential to its success.

Methods

This national qualitative multiple case study explored interprofessional collaboration between the Máxima and its Shared Care partners, from the perspective of pediatricians in all 14 regional centers. Semi-structured interviews were conducted, transcribed, and analyzed inductively using ATLAS.ti. Analysis was informed by Kaats & Opheij’s collaboration framework, encompassing shared ambition, mutual interests, interpersonal relationships, structured collaboration, and meaningful processes. Peer debriefing was used to enhance trustworthiness and ensure clinical relevance.

Results

Participants emphasized that close collaboration is indispensable for delivering safe, high-quality pediatric oncology care. Enabling factors included mutual trust, personal familiarity, timely and clear communication, and professional recognition. Challenges were identified in areas such as inconsistent policy implementation, opaque decision-making, and insufficient acknowledgment of local expertise. Many called for differentiated, context-sensitive care agreements rather than one-size-fits-all approaches.

Conclusion

Effective shared care requires more than structural alignment: it demands investment in professional relationships, shared goals, and adaptive processes. These findings offer actionable insights to improve collaboration within the Dutch model and provide transferable lessons for other countries navigating centralized care in highcomplex, low-volume pediatric specialties.
2018年,荷兰将所有儿科肿瘤护理集中在普林斯Máxima中心(Máxima),由14个共享护理中心组成的国家网络提供支持。这些中心在离家更近的地方提供非重症治疗和支持性护理,实现了集中专业知识和区域可及性的混合模式。虽然在结构上已经到位,但对于区域儿科医生如何体验这种合作,或者他们认为哪些因素对其成功至关重要,人们知之甚少。方法本全国性定性多案例研究从所有14个区域中心的儿科医生的角度探讨了Máxima及其共享护理合作伙伴之间的跨专业合作。使用ATLAS.ti进行半结构化访谈,并进行转录和归纳分析。分析由Kaats & Opheij的协作框架提供,包括共同的目标、共同的利益、人际关系、结构化的协作和有意义的过程。采用同行评议提高可信度,确保临床相关性。结果与会者强调,密切合作对于提供安全、高质量的儿科肿瘤护理是不可或缺的。促成因素包括相互信任、个人熟悉、及时清晰的沟通和专业认可。在政策执行不一致、决策不透明以及对当地专业知识认识不足等领域确定了挑战。许多人呼吁制定有区别的、环境敏感的护理协议,而不是一刀切的方法。结论有效的共享医疗需要的不仅仅是结构上的一致:它需要在专业关系、共同目标和适应过程上的投资。这些发现为改善荷兰模式内的合作提供了可行的见解,并为其他国家在高复杂性、小容量儿科专科进行集中护理提供了可转移的经验教训。
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引用次数: 0
Multi-tyrosine kinase inhibitors in paediatric soft tissue sarcoma – A review 多种酪氨酸激酶抑制剂在小儿软组织肉瘤中的应用综述
Pub Date : 2026-01-13 DOI: 10.1016/j.ejcped.2026.100484
Kim P.J. Schellekens , Andrea Ferrari , Michael T. Meister , C. Michel Zwaan , Susanne A. Gatz , Max M. van Noesel , Michela Casanova , Reineke A. Schoot
Neoadjuvant systemic treatment for paediatric patients with soft tissue sarcomas (STS) consists of conventional chemotherapy, predominantly ifosfamide and doxorubicin, with a limited role for innovative therapies. Many multi-tyrosine kinase inhibitors (mTKIs) have been developed in the past decades for the treatment of various types of malignancies. Over a decade ago, the mTKI pazopanib was authorized by the EMA and FDA for the treatment of adults with STS. Paediatric development of innovative treatments often follows years after the first marketing authorisations in adults has been obtained. To date, a series of clinical trials have been conducted investigating the value of mTKIs in paediatric patients with STS. Nevertheless, the positioning of mTKIs in the treatment of paediatric STS remains unclear and selection of mTKIs for clinical trials is more often made on grounds of availability rather than on mechanism of action, efficacy data, or toxicity profile. In this manuscript, we provide an overview of studies evaluating mTKIs in paediatric, adolescent, and adult STS patients, aiming to assist in prioritization of future clinical trials in paediatric STS. We performed a systematic literature review with the purpose of collecting data on activity and safety. The results of 107 relevant clinical trials, including 23 trials conducted in the paediatric population and 84 trials in the adult population, were critically assessed and summarized (Appendix 1). Accelerating the development of innovative treatments in patients with such ultra-rare diseases will require innovative clinical trial designs, and international collaboration is necessary. Considering the age distribution of STS, collaboration between paediatric oncologists and medical oncologists should be considered.
小儿软组织肉瘤(STS)患者的新辅助全身治疗包括传统化疗,主要是异环磷酰胺和阿霉素,创新疗法的作用有限。在过去的几十年里,多种酪氨酸激酶抑制剂(mTKIs)被开发出来用于治疗各种类型的恶性肿瘤。十多年前,mTKI pazopanib被EMA和FDA批准用于治疗成人STS。儿科创新疗法的开发通常是在获得成人首次上市许可数年后进行的。迄今为止,已经进行了一系列临床试验,研究mTKIs在儿科STS患者中的价值。然而,mTKIs在儿科STS治疗中的定位仍不清楚,临床试验中mTKIs的选择更多是基于可获得性,而不是作用机制、疗效数据或毒性特征。在这篇文章中,我们概述了在儿科、青少年和成人STS患者中评估mTKIs的研究,旨在帮助确定儿科STS未来临床试验的优先级。我们进行了系统的文献综述,目的是收集有关活动和安全性的数据。对107项相关临床试验的结果进行了严格评估和总结(附录1),其中包括23项在儿科人群中进行的试验和84项在成人人群中进行的试验。要加快开发针对此类极罕见疾病患者的创新疗法,就需要创新的临床试验设计,国际合作是必要的。考虑到STS患者的年龄分布,应考虑儿科肿瘤学家和内科肿瘤学家之间的合作。
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引用次数: 0
Evaluation of the impact of adherence to infection prevention measures on the quality of life in children with hematologic malignancies 评估坚持感染预防措施对血液系统恶性肿瘤儿童生活质量的影响
Pub Date : 2026-01-09 DOI: 10.1016/j.ejcped.2026.100482
Dorine Poelmans , Jaques van Heerden

Purpose

The survival rate for paediatric hematological malignancies has improved significantly, shifting focus to Health Related Quality of Life (HRQOL). The objectives of this study was to investigate the impact of infection prevention lifestyle adjustments on HRQOL and to determine whether these adjustments were feasible and applicable in practice.

Methodology

A prospective quantitative cross-sectional study was done, in which 21 children between 6 and 18 years old, treated for an hematological malignancy at the University Hospital Antwerp were interviewed. The questionnaire was developed in accordance with PedsQL™, consisting of 27, five-point Likert scale questions.

Results

The impact of lifestyle modifications to avoid infections on the HRQOL was assessed using a physical, social, emotional and school domain, of which the median was 3.2 [IQR 2.6; 3.7], 3.5 [IQR 3; 4], 3.5 [IQR 2.9; 4.6] and 3.7 [IQR 3.3; 4.3] respectively. These corresponded to 'rarely to sometimes' having difficulty complying with these rules. The feasibility and applicability of complying in practice was tested in the physical, social and school domain, of which the median was 1.2 [IQR 1; 1.5], 1 [IQR 1; 1.5] and 1 [IQR 1; 1] respectively. These corresponded to 'always' being feasible and applicable.

Conclusion

The first study showed that HRQOL was impacted by lifestyle modifications to avoid infections and influenced in every domain, especially the physical domain. Although children and/or their proxy indicated that the guidelines were feasible and applicable in practice the evidence of these recommendations must be evaluated.
目的儿童恶性血液病的生存率显著提高,关注焦点转向健康相关生活质量(HRQOL)。本研究的目的是调查感染预防生活方式调整对HRQOL的影响,并确定这些调整在实践中是否可行和适用。方法采用前瞻性定量横断面研究,对在安特卫普大学医院接受血液恶性肿瘤治疗的21名6 - 18岁儿童进行访谈。调查问卷是根据PedsQL™开发的,由27个5分李克特量表问题组成。结果通过身体、社会、情感和学校四个方面评估生活方式改变对HRQOL的影响,其中中位数为3.2 [IQR 2.6;3.7], 3.5 [iqr 3;[4], 3.5 [iqr 2.9;4.6]和3.7 [IQR 3.3;分别为4.3)。这些对应于“很少到有时”难以遵守这些规则。在物理、社会和学校领域测试了实践中遵守的可行性和适用性,其中中位数为1.2 [IQR 1;1.5], 1 [iqr 1;1.5]和1 [IQR 1;分别1]。这些与“始终”是可行和适用的相对应。结论第一项研究表明,为避免感染而改变生活方式对患者的HRQOL有影响,并在各个领域受到影响,尤其是身体领域。虽然儿童和/或其代理人表示,这些准则在实践中是可行和适用的,但必须对这些建议的证据进行评价。
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引用次数: 0
Pain management during interventions in paediatric oncology in Europe – Results from a pan-European survey 欧洲儿科肿瘤学干预期间的疼痛管理-一项泛欧调查的结果
Pub Date : 2025-12-16 DOI: 10.1016/j.ejcped.2025.100481
Eva-Maria Wild , Gianluca Piccolo , Eleni Syrimi , Rahel Kasteler , Bob Philips , Gilles Vassal , Ciara Sheehan , Lejla Kameric , Theodora Armenkova , Katrin Scheinemann , Maria Otth

Background

Childhood cancer survival rates have improved over the last decades. Nevertheless, childhood cancer treatment is associated with painful interventions needed for disease monitoring or treatment. It is therefore crucial to ensure appropriate pain control for painful interventions. This European survey aims to map the approaches to manage such interventions in children and adolescents with cancer.

Methods

An online survey was sent to members of the European Society of Paediatric Oncology, asking the way potentially painful interventions are being managed. Interventions included lumbar puncture, bone marrow aspiration and biopsy, removal of drainages, insertion and removal of central venous lines, puncture of porth-a-cath or Ommaya reservoir, tumour biopsy, and insertion of nasogastric tube. Proposed approaches to pain management included local analgesia, systemic analgesia alone, systemic analgesia with sedation administrated by paediatric oncologists or anaesthesiologists, distraction techniques, and other methods. We further asked about drugs used and types of distraction.

Results

A total of 326 health care professionals responded, representing 37 countries. The proportion of participants reporting that an intervention is performed without any pain management ranged from one participant each for bone marrow aspiration/biopsy and tumor biopsy (0,3 %), to 101 participants (31 %) for nasogastric tube insertion. The intensity of analgesia or depth of sedation per intervention is heterogeneous, so are the drugs used.

Conclusion

The heterogenous results in use of analgesia or sedation during procedures indicate an unmet need. These results highlight the need for guidelines and their implementation in clinical practice. No child or adolescent undergoing cancer treatment should suffer from procedure-related pain.
儿童癌症存活率在过去几十年中有所提高。然而,儿童癌症治疗与疾病监测或治疗所需的痛苦干预有关。因此,确保对疼痛干预进行适当的疼痛控制至关重要。这项欧洲调查的目的是绘制出对患有癌症的儿童和青少年进行此类干预的方法。方法向欧洲儿科肿瘤学会的成员发送了一份在线调查,询问如何管理可能带来痛苦的干预措施。干预措施包括腰椎穿刺、骨髓穿刺和活检、清除引流管、插入和移除中心静脉线、穿刺导尿管或Ommaya储液器、肿瘤活检和插入鼻胃管。提出的疼痛管理方法包括局部镇痛,单独全身镇痛,全身镇痛与镇静由儿科肿瘤学家或麻醉学家管理,分散技术,和其他方法。我们进一步询问了使用的药物和分心的类型。结果共有326名卫生保健专业人员回应,代表37个国家。报告在没有任何疼痛管理的情况下进行干预的参与者比例从骨髓穿刺/活检和肿瘤活检各1名参与者(0.3 %)到鼻胃管插入101名参与者(31 %)不等。每次干预的镇痛强度或镇静深度是不同的,所使用的药物也是不同的。结论手术过程中使用镇痛或镇静的结果参差不齐,表明其需求尚未得到满足。这些结果突出了指南及其在临床实践中实施的必要性。接受癌症治疗的儿童或青少年不应遭受手术相关的疼痛。
{"title":"Pain management during interventions in paediatric oncology in Europe – Results from a pan-European survey","authors":"Eva-Maria Wild ,&nbsp;Gianluca Piccolo ,&nbsp;Eleni Syrimi ,&nbsp;Rahel Kasteler ,&nbsp;Bob Philips ,&nbsp;Gilles Vassal ,&nbsp;Ciara Sheehan ,&nbsp;Lejla Kameric ,&nbsp;Theodora Armenkova ,&nbsp;Katrin Scheinemann ,&nbsp;Maria Otth","doi":"10.1016/j.ejcped.2025.100481","DOIUrl":"10.1016/j.ejcped.2025.100481","url":null,"abstract":"<div><h3>Background</h3><div>Childhood cancer survival rates have improved over the last decades. Nevertheless, childhood cancer treatment is associated with painful interventions needed for disease monitoring or treatment. It is therefore crucial to ensure appropriate pain control for painful interventions. This European survey aims to map the approaches to manage such interventions in children and adolescents with cancer.</div></div><div><h3>Methods</h3><div>An online survey was sent to members of the European Society of Paediatric Oncology, asking the way potentially painful interventions are being managed. Interventions included lumbar puncture, bone marrow aspiration and biopsy, removal of drainages, insertion and removal of central venous lines, puncture of porth-a-cath or Ommaya reservoir, tumour biopsy, and insertion of nasogastric tube. Proposed approaches to pain management included local analgesia, systemic analgesia alone, systemic analgesia with sedation administrated by paediatric oncologists or anaesthesiologists, distraction techniques, and other methods. We further asked about drugs used and types of distraction.</div></div><div><h3>Results</h3><div>A total of 326 health care professionals responded, representing 37 countries. The proportion of participants reporting that an intervention is performed without any pain management ranged from one participant each for bone marrow aspiration/biopsy and tumor biopsy (0,3 %), to 101 participants (31 %) for nasogastric tube insertion. The intensity of analgesia or depth of sedation per intervention is heterogeneous, so are the drugs used.</div></div><div><h3>Conclusion</h3><div>The heterogenous results in use of analgesia or sedation during procedures indicate an unmet need. These results highlight the need for guidelines and their implementation in clinical practice. No child or adolescent undergoing cancer treatment should suffer from procedure-related pain.</div></div>","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":"7 ","pages":"Article 100481"},"PeriodicalIF":0.0,"publicationDate":"2025-12-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145765800","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Transition needs and preferences identified during peer visits to three European best-practice CAYA cancer Long-Term Follow-Up care centres: An EU-CAYAS-NET project 在对三个欧洲最佳CAYA癌症长期随访护理中心的同行访问中确定的过渡需求和偏好:欧盟-CAYA - net项目
Pub Date : 2025-12-01 DOI: 10.1016/j.ejcped.2025.100479
Jikke Wams , Jaap den Hartogh , Cherine Mathot , Jeroen te Dorsthorst , Marie-Therese Gubi , Kerstin Krottendorfer , Ulrike Leiss , Esther Lasheras Soria , Begonya Nafria Escalera , Urška Košir , Katie Rizvi , Carina Schneider , Barbara Brunmair , Ismay de Beijer , Renée L. Mulder , Leontien C.M. Kremer , Helena J.H. van der Pal , on behalf of the EU-CAYAS-NET consortium

Background

A successful transition from child-centred to adult-oriented healthcare for survivors of childhood and adolescent cancer is essential to help them engage in lifelong surveillance in survivorship care. Information on their needs and preferences for the transition process is needed to identify key components for a successful transition.

Objective

To describe the transition needs and preferences of young cancer survivors, their parents, and healthcare providers.

Methods

Three in-person peer visits to best-practice long-term follow-up care centres in Europe were conducted. In these visits, the needs and preferences of young cancer survivors, their parents, and healthcare providers were collected through interactive sessions and structured documentation. The outcomes were analysed inductively using thematic analysis, resulting in a list of unique needs and preferences.

Results

Sixty-five participants, including 32 survivors, six parents, 21 healthcare providers, and six researchers, shared 188 needs and preferences, leading to the identification of 58 distinct transition needs and preferences. Participants stressed the need for structured, patient-centred transition processes with clear coordination and planning, following a holistic approach. Key support needs include lifestyle, psychosocial, and employment assistance. Improved access to reliable information, tailored education, and readiness-based transfers were emphasised.

Conclusion

Our findings highlight the need for personalised and ongoing coordination and support for survivors and their families during transition. Strengthening informational and educational transition resources are crucial to address gaps experienced by patients and caregivers. These insights will be implemented into the evidence-based European transition guideline from the EU-CAYAS-NET project.
儿童和青少年癌症幸存者的医疗保健成功地从以儿童为中心过渡到以成人为导向,这对于帮助他们参与幸存者护理的终身监测至关重要。需要关于他们对过渡过程的需求和偏好的信息,以确定成功过渡的关键组成部分。目的描述年轻癌症幸存者、其父母和医疗保健提供者的过渡需求和偏好。方法对欧洲最佳实践的长期随访护理中心进行了三次面对面的同伴访问。在这些访问中,通过互动会议和结构化文档收集了年轻癌症幸存者、他们的父母和医疗保健提供者的需求和偏好。使用主题分析对结果进行归纳分析,得出独特需求和偏好列表。结果65名参与者,包括32名幸存者、6名父母、21名医疗服务提供者和6名研究人员,共有188种需求和偏好,从而确定了58种不同的过渡需求和偏好。与会者强调,有必要按照整体办法进行有组织的、以病人为中心的过渡进程,并进行明确的协调和规划。主要的支持需求包括生活方式、社会心理和就业援助。强调了改善获得可靠信息的机会、量身定制的教育和基于准备的转移。结论:我们的研究结果强调了在过渡期间对幸存者及其家人进行个性化和持续的协调和支持的必要性。加强信息和教育过渡资源对于解决患者和护理人员所经历的差距至关重要。这些见解将被应用到EU-CAYAS-NET项目的以证据为基础的欧洲过渡指南中。
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引用次数: 0
Characterising childhood cancer survivors in Germany: A retrospective observational study on demographic, clinical, and healthcare utilisation patterns 表征德国儿童癌症幸存者:一项关于人口统计学、临床和医疗保健利用模式的回顾性观察研究
Pub Date : 2025-12-01 DOI: 10.1016/j.ejcped.2025.100480
Ekaterina Aleshchenko , Pietro Trocchi , Christian Apfelbacher , Katja Baust , Gabriele Calaminus , Patrik Droege , Dirk Horenkamp-Sonntag , Peter Ihle , Melanie Klein , Jutta Kuepper-Nybelen , Thorsten Langer , Christian Luepkes , Ursula Marschall , Hiltrud Merzenich , Thomas Ruhnke , Claudia Spix , Enno Swart , Cecile Ronckers

Background

As survival rates for childhood cancer improve, understanding long-term outcomes and healthcare needs of childhood cancer survivors (CCS) is essential. In Germany, such data have been limited.

Objective

This study aimed to describe demographic and clinical characteristics of a long-term CCS cohort and assess healthcare utilisation patterns based on cancer registry and statutory health insurance (SHI) claims data.

Methods

The VersKiK study linked GCCR data with claims from 13 SHI providers, covering 69 % of the German statutorily insured population. The analysis of healthcare utilisation included 11,863 CCS diagnosed since 1991–2011, known to be alive in 01/2017 and maintained uninterrupted statutory health insurance coverage during 2017–2021.Healthcare utilisation was compared to a 1:3-matched comparison group (matching factors insurance duration, sex, birth year, and region).

Results

Long-term CCS had significantly higher healthcare utilisation across all metrics. They experienced 1.1–5.1 times more outpatient visits, hospital stays, medication prescriptions, and episodes of work incapacity than the control group. Utilisation varied considerably by cancer type, age at diagnosis, sex, and region. Survivors of CNS tumours, bone tumours, and soft tissue sarcomas showed the highest service use. Differences in utilisation between survivors and controls were greater among younger than older survivors.

Conclusions

This large, population-based record linkage study highlights significant differences in healthcare use between CCS and the general population, emphasising the need for evidence-based policies and tailored clinical care models. Further detailed analyses of claims data in this context can guide improvements in long-term care access, coordination, and outcomes for survivors.
随着儿童癌症存活率的提高,了解儿童癌症幸存者(CCS)的长期预后和医疗保健需求至关重要。在德国,这类数据一直很有限。目的:本研究旨在描述长期CCS队列的人口统计学和临床特征,并基于癌症登记和法定健康保险(SHI)索赔数据评估医疗保健利用模式。方法VersKiK研究将GCCR数据与13家SHI供应商的索赔联系起来,覆盖了69% %的德国法定保险人口。医疗保健利用分析包括自1991-2011年以来诊断的11,863例CCS,已知在2017年1月存活,并在2017-2021年期间保持不间断的法定医疗保险覆盖。将医疗保健利用情况与1:3匹配的对照组进行比较(匹配因素包括保险期限、性别、出生年份和地区)。结果长期CCS在所有指标上都有显著更高的医疗保健利用率。他们的门诊次数、住院时间、药物处方和丧失工作能力的次数是对照组的1.1-5.1 倍。使用情况因癌症类型、诊断年龄、性别和地区而有很大差异。中枢神经系统肿瘤、骨肿瘤和软组织肉瘤的幸存者显示出最高的服务使用率。幸存者和对照组之间的使用差异在年轻的幸存者中大于年长的幸存者。这项基于人群的大型记录关联研究强调了CCS与普通人群在医疗保健使用方面的显著差异,强调了基于证据的政策和量身定制的临床护理模式的必要性。在这种情况下,进一步详细分析索赔数据可以指导改善幸存者的长期护理获取、协调和结果。
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引用次数: 0
P-glycoprotein as a chemotherapy resistance mechanism and biomarker of poor response in high-risk neuroblastoma p -糖蛋白作为高危神经母细胞瘤化疗耐药机制和不良反应的生物标志物
Pub Date : 2025-11-08 DOI: 10.1016/j.ejcped.2025.100478
Caroline Atkinson , Alvin Kamili , Carole M. Tactacan , Nisitha Jayatilleke , Sebastian P.A. Rosser , Federica Saletta , Christine C. Gana , Philipp Graber , Amanda Wanninayaka , Rosa Mistica C. Ignacio , Maria Kavallaris , Andrew J. Gifford , Richard B. Lock , Murray D. Norris , Michelle Haber , Chelsea Mayoh , Christa E. Nath , Toby N. Trahair , Jamie I. Fletcher

Background

Half of all children with high-risk neuroblastoma progress or relapse. We identified ABCB1, encoding for the ATP binding cassette transporter P-glycoprotein (P-gp), as a candidate drug resistance mechanism based on differential expression in survivors and non-survivors and investigated whether P-gp expression limits the effectiveness of chemotherapy.

Methods

P-gp/ABCB1 expression and regulation were assessed in tumours, PDX models in NSG mice, and cell lines by RNA-sequencing, immunohistochemistry, western blotting and through publicly available ChIP-Seq and RNA-Seq data. Response to standard-of-care induction therapies and targeted agents was assessed in vitro and in vivo using cell line and PDX models with genetic and pharmacological P-gp inhibition.

Results

P-gp expression is common in high-risk neuroblastoma and elevated compared to other cancers. High relative expression at diagnosis is associated with poorer outcome, consistent with drug efflux. Pharmacological or genetic targeting of P-gp partially restores sensitivity of neuroblastoma cells to vincristine, doxorubicin, etoposide, and some targeted agents, but not to the ALK inhibitors crizotinib, ceritinib, alectinib and lorlatinib and sensitizes neuroblastoma xenografts to vincristine, extending survival.

Conclusions

P-gp is a clinically relevant drug resistance mechanism for high-risk neuroblastoma. Tumour P-gp levels could inform treatment options for individual patients to avoid ineffective treatments and unnecessary toxicities.
背景:半数高危神经母细胞瘤患儿进展或复发。我们确定了编码ATP结合盒转运蛋白p -糖蛋白(P-gp)的ABCB1,作为基于幸存者和非幸存者差异表达的候选耐药机制,并研究了P-gp的表达是否限制了化疗的有效性。方法通过rna测序、免疫组织化学、western blotting和公开的ChIP-Seq和RNA-Seq数据,评估sp -gp/ABCB1在NSG小鼠肿瘤、PDX模型和细胞系中的表达和调控。采用遗传和药理学P-gp抑制的细胞系和PDX模型,在体外和体内评估对标准治疗诱导疗法和靶向药物的反应。结果sp -gp在高危神经母细胞瘤中表达普遍,与其他肿瘤相比表达升高。诊断时的高相对表达与较差的预后相关,与药物外溢一致。P-gp的药理学或遗传学靶向部分恢复神经母细胞瘤细胞对长春新碱、阿霉素、依托泊苷和一些靶向药物的敏感性,但不恢复对ALK抑制剂克里唑替尼、塞瑞替尼、阿勒替尼和氯拉替尼的敏感性,并使神经母细胞瘤异种移植物对长春新碱敏感,延长生存期。结论sp -gp是高危神经母细胞瘤临床相关的耐药机制。肿瘤P-gp水平可以为个体患者提供治疗选择,以避免无效治疗和不必要的毒性。
{"title":"P-glycoprotein as a chemotherapy resistance mechanism and biomarker of poor response in high-risk neuroblastoma","authors":"Caroline Atkinson ,&nbsp;Alvin Kamili ,&nbsp;Carole M. Tactacan ,&nbsp;Nisitha Jayatilleke ,&nbsp;Sebastian P.A. Rosser ,&nbsp;Federica Saletta ,&nbsp;Christine C. Gana ,&nbsp;Philipp Graber ,&nbsp;Amanda Wanninayaka ,&nbsp;Rosa Mistica C. Ignacio ,&nbsp;Maria Kavallaris ,&nbsp;Andrew J. Gifford ,&nbsp;Richard B. Lock ,&nbsp;Murray D. Norris ,&nbsp;Michelle Haber ,&nbsp;Chelsea Mayoh ,&nbsp;Christa E. Nath ,&nbsp;Toby N. Trahair ,&nbsp;Jamie I. Fletcher","doi":"10.1016/j.ejcped.2025.100478","DOIUrl":"10.1016/j.ejcped.2025.100478","url":null,"abstract":"<div><h3>Background</h3><div>Half of all children with high-risk neuroblastoma progress or relapse. We identified <em>ABCB1,</em> encoding for the ATP binding cassette transporter P-glycoprotein (P-gp), as a candidate drug resistance mechanism based on differential expression in survivors and non-survivors and investigated whether P-gp expression limits the effectiveness of chemotherapy.</div></div><div><h3>Methods</h3><div>P-gp/<em>ABCB1</em> expression and regulation were assessed in tumours, PDX models in NSG mice, and cell lines by RNA-sequencing, immunohistochemistry, western blotting and through publicly available ChIP-Seq and RNA-Seq data. Response to standard-of-care induction therapies and targeted agents was assessed <em>in vitro</em> and <em>in vivo</em> using cell line and PDX models with genetic and pharmacological P-gp inhibition.</div></div><div><h3>Results</h3><div>P-gp expression is common in high-risk neuroblastoma and elevated compared to other cancers. High relative expression at diagnosis is associated with poorer outcome, consistent with drug efflux. Pharmacological or genetic targeting of P-gp partially restores sensitivity of neuroblastoma cells to vincristine, doxorubicin, etoposide, and some targeted agents, but not to the ALK inhibitors crizotinib, ceritinib, alectinib and lorlatinib and sensitizes neuroblastoma xenografts to vincristine, extending survival.</div></div><div><h3>Conclusions</h3><div>P-gp is a clinically relevant drug resistance mechanism for high-risk neuroblastoma. Tumour P-gp levels could inform treatment options for individual patients to avoid ineffective treatments and unnecessary toxicities.</div></div>","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":"6 ","pages":"Article 100478"},"PeriodicalIF":0.0,"publicationDate":"2025-11-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145527989","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
REFRACTORY CYTOPENIA OF CHILDHOOD WITH NORMAL KARYOTYPE: LONG-TERM RESULTS OF AN OBSERVATIONAL APPROACH 核型正常的儿童难治性细胞减少症:观察方法的长期结果
Pub Date : 2025-11-04 DOI: 10.1016/j.ejcped.2025.100330
Beatrice Drexler , Stephan Schwarz-Furlan , Irith Baumann , Martina Rudelius , Peter Noellke , Dirk Lebrecht , Senthilkumar Ramamoorthy , Natalia Rotari , Axel Karow , Shinsuke Hirabayashi , Fabian Beier , Yvonne Lisa Behrens , Gudrun Gohring , Reinhard Kalb , Marcin W. Wlodarski , Brigitte Strahm , Miriam Erlacher , Charlotte M. Niemeyer , Ayami Yoshimi
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引用次数: 0
SINGLE-CELL AND SPATIAL TRANSCRIPTOMICS OF PEDIATRIC MYELODYSPLASTIC SYNDROME WITH REFRACTORY CYTOPENIA OF CHILDHOOD REVEAL A DISRUPTED BONE MARROW NICHE DRIVING IMPAIRED HEMATOPOIESIS 儿童骨髓增生异常综合征伴儿童期难治性细胞减少症的单细胞和空间转录组学揭示了骨髓生态位的破坏导致造血功能受损
Pub Date : 2025-11-04 DOI: 10.1016/j.ejcped.2025.100331
Patrycja Fryzik, Li-Ting Chen, Evelyn S. Hanemaaijer, Ireen J. Kal, Tito Candelli, Brigit M. te Pas, Nina Epskamp, Valerie de Haas, Marijn Scheijde-Vermeulen, Wim J. de Jonge, Thanasis Margaritis, Mirjam Belderbos
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引用次数: 0
期刊
EJC paediatric oncology
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