EJC paediatric oncology最新文献

{"title":"Evaluation of the impact of adherence to infection prevention measures on the quality of life in children with hematologic malignancies","authors":"Dorine Poelmans ,&nbsp;Jaques van Heerden","doi":"10.1016/j.ejcped.2026.100482","DOIUrl":"10.1016/j.ejcped.2026.100482","url":null,"abstract":"<div><h3>Purpose</h3><div>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.</div></div><div><h3>Methodology</h3><div>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.</div></div><div><h3>Results</h3><div>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.</div></div><div><h3>Conclusion</h3><div>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.</div></div>","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":"7 ","pages":"Article 100482"},"PeriodicalIF":0.0,"publicationDate":"2026-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145978275","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}

{"title":"Pharmacokinetic variability of mitotane in pediatric adrenocortical carcinoma and the role of CYP2B6 genotypes: a pediatric case series","authors":"Ivy A.E. Ankone ,&nbsp;Tirsa de Kluis ,&nbsp;Lidwien M. Hanff ,&nbsp;Antoinette Jaspers-Bakker ,&nbsp;Ronald R. de Krijger ,&nbsp;Jesse J. Swen ,&nbsp;Kathalijne A. Oudhoff ,&nbsp;Marieke J.M. Meijs ,&nbsp;Max M. van Noesel ,&nbsp;Alwin D.R. Huitema ,&nbsp;Meta H.M. Diekstra","doi":"10.1016/j.ejcped.2026.100499","DOIUrl":"10.1016/j.ejcped.2026.100499","url":null,"abstract":"<div><div>Despite therapeutic drug monitoring of mitotane, achieving target plasma concentrations remains challenging. Commonly reported enzymes involved in mitotane metabolism are cytochrome P450 (CYP)3A4 and 2B6, with CYP2B6 being highly polymorphic. We evaluated the impact of <em>CYP2B6</em> genotypes on achieving therapeutic concentrations in six patients. We observed intermediate, normal, and rapid metabolizer genotypes. Intermediate or normal metabolizers all reached therapeutic concentrations, receiving between 3.5–5.5 g/m²/day. The rapid metabolizer did not reach therapeutic concentrations despite receiving up to 8.0 g/m²/day. These results indicate <em>CYP2B6</em> genotypes may affect the dose needed to achieve and maintain therapeutic concentrations of mitotane in pediatric adrenocortical carcinoma.</div></div>","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":"7 ","pages":"Article 100499"},"PeriodicalIF":0.0,"publicationDate":"2026-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147384840","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}

{"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":"2026-06-01","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}

{"title":"Making shared care work: A national qualitative case study on collaborative practice in pediatric oncology","authors":"Natasja Dors ,&nbsp;Martine van Grotel ,&nbsp;Eline Zijtregtop ,&nbsp;Nathalie van der Salm ,&nbsp;Laura Keukens ,&nbsp;Selma Lavrijssen ,&nbsp;Charlotte Drykoningen ,&nbsp;Anne Jonge Poerink ,&nbsp;Rod Thompson ,&nbsp;Jenneke Homan ,&nbsp;Menco Weismuller ,&nbsp;Wendy Nix ,&nbsp;Hester Blufpand ,&nbsp;Annemieke van der Wey ,&nbsp;Daniëlle Martens ,&nbsp;Marion Hekkelaan ,&nbsp;Lotte Helder ,&nbsp;Wouter Kollen ,&nbsp;Max van Noesel ,&nbsp;Marije P. Hennus","doi":"10.1016/j.ejcped.2026.100485","DOIUrl":"10.1016/j.ejcped.2026.100485","url":null,"abstract":"<div><h3>Background</h3><div>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.</div></div><div><h3>Methods</h3><div>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 &amp; 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.</div></div><div><h3>Results</h3><div>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.</div></div><div><h3>Conclusion</h3><div>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.</div></div>","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":"7 ","pages":"Article 100485"},"PeriodicalIF":0.0,"publicationDate":"2026-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146023598","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}

{"title":"Congenital histiocytosis with central nervous system involvement and a novel PTPRJ::RASGRF1 fusion","authors":"Paul G. Kemps ,&nbsp;Marianna Bugiani ,&nbsp;Marijn A. Scheijde-Vermeulen ,&nbsp;Marco J. Koudijs ,&nbsp;Jean-François Emile ,&nbsp;Olvert A. Berkhemer ,&nbsp;Wes Onland ,&nbsp;Cor van den Bos","doi":"10.1016/j.ejcped.2026.100492","DOIUrl":"10.1016/j.ejcped.2026.100492","url":null,"abstract":"<div><div>Histiocytic neoplasms are rare diseases characterized by clonal expansions of cells with a macrophage or dendritic cell phenotype. They are driven by mutations activating the MAPK pathway and may involve diverse organs, including the central nervous system (CNS). We describe a newborn with congenital histiocytosis affecting the CNS, skin, lungs, lymph nodes, thyroid, fingernail, and soft tissues – including a tumor originating from the tongue obstructing the upper airway. Histopathology revealed an atypical histiocytosis with strong CD1a and variable Langerin expression; post-mortem transcriptome sequencing identified a novel <em>PTPRJ::RASGRF1</em> fusion. This case expands the molecular landscape of histiocytic neoplasms, highlighting the value of comprehensive genomic profiling.</div></div>","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":"7 ","pages":"Article 100492"},"PeriodicalIF":0.0,"publicationDate":"2026-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147384836","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}

{"title":"Experiences of receiving explanations about medical conditions among adolescent children with cancer: A qualitative study","authors":"Yuta Kogumazaka ,&nbsp;Takako Miyamura ,&nbsp;Tae Kawahara ,&nbsp;Tomoko Abe ,&nbsp;Saaya Niino ,&nbsp;Akemi Yamazaki","doi":"10.1016/j.ejcped.2026.100486","DOIUrl":"10.1016/j.ejcped.2026.100486","url":null,"abstract":"<div><h3>Background</h3><div>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.</div></div><div><h3>Methods</h3><div>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.</div></div><div><h3>Results</h3><div>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”.</div></div><div><h3>Conclusions</h3><div>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.</div></div>","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":"7 ","pages":"Article 100486"},"PeriodicalIF":0.0,"publicationDate":"2026-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146023597","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}

{"title":"High-grade/large B-cell lymphoma with 11q aberration: A distinct molecularly defined entity in contemporary pathobiological classifications","authors":"Matej Jelić ,&nbsp;Madalina Bota ,&nbsp;Ingrid Simonitsch-Klupp ,&nbsp;G.A. Amos Burke ,&nbsp;Andishe Attarbaschi","doi":"10.1016/j.ejcped.2026.100494","DOIUrl":"10.1016/j.ejcped.2026.100494","url":null,"abstract":"<div><h3>Purpose</h3><div>High-grade/large B-cell lymphoma with 11q aberration (HGBCL-11q) is an aggressive mature B-cell lymphoma that mimics Burkitt lymphoma (BL) but lacks <em>MYC</em>-rearrangements. Correct diagnosis is essential to avoid misclassification and to support (future) risk-adapted management.</div></div><div><h3>Methods</h3><div>We reviewed recent classification updates, pivotal clinicopathologic reports, and genomic studies addressing HGBCL-11q. Accordingly, we summarized diagnostic criteria including histomorphology, immunophenotype and genomics, and collate reported clinical characteristics and treatment outcomes.</div></div><div><h3>Results</h3><div>HGBCL-11q is characterized by a chromosome 11q copy-number profile with a proximal gain/amplification at 11q23.2–q23.3 and a distal loss at 11q24.1–qter. Tumours typically show a germinal centre B-cell immunophenotype and BL-like histomorphology, yet genome-wide profiling supports assignment within the large B-cell lymphoma (LBCL) spectrum rather than true BL. HGBCL-11q predominantly affects children and adolescents and often presents with localized nodal disease in the head-and-neck region. In the largest multinational cohort of 72 pediatric patients, 3-year event-free and overall survival were 94 % and 96 %, respectively, with inferior outcomes mainly confined to patients with inborn and acquired errors of immunity or cancer predisposition syndromes.</div></div><div><h3>Conclusion</h3><div>HGBCL-11q is a genetically defined non-Hodgkin lymphoma with an excellent lymphoma-related prognosis under pediatric mature B-cell lymphoma–type regimens. Standardized testing for 11q aberration in cases of diffuse LBCL, HGBCL or BL morphology lacking <em>MYC</em>-rearrangements improves classification consistency and may enable chemoimmunotherapy de-escalation trials. Systematic evaluation for immunodeficiency and cancer predisposition syndromes is recommended to identify patients at risk and guide adapted therapy, supportive care and follow-up.</div></div>","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":"7 ","pages":"Article 100494"},"PeriodicalIF":0.0,"publicationDate":"2026-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147384842","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}

{"title":"Responses can be achieved with a combination of the MEK inhibitor selumetinib and dexamethasone in patients with relapsed/refractory RAS-pathway mutated acute lymphoblastic leukemia: Results of a parallel cohort, dose-finding and expansion phase I/II trial","authors":"Britta Vormoor ,&nbsp;Joshua Savage ,&nbsp;Caroline Kristunas ,&nbsp;Sarah Johnson ,&nbsp;Mauricio Nicolás Ferrao Blanco ,&nbsp;Geoff Shenton ,&nbsp;Donna Lancaster ,&nbsp;Anna Castleton ,&nbsp;Adele K. Fielding ,&nbsp;Anne-Louise Latif ,&nbsp;Nick Morley ,&nbsp;Marion Strullu ,&nbsp;Ruta Tuckuviene ,&nbsp;Pamela Kearns ,&nbsp;Claire Jennings ,&nbsp;Shelby Barnett ,&nbsp;Gareth J. Veal ,&nbsp;Julie Irving ,&nbsp;Lucinda Billingham ,&nbsp;Tobias Menne ,&nbsp;Josef Vormoor","doi":"10.1016/j.ejcped.2026.100489","DOIUrl":"10.1016/j.ejcped.2026.100489","url":null,"abstract":"<div><h3>Background</h3><div>Relapsed/refractory acute lymphoblastic leukemia (ALL) remains a major clinical challenge. We have previously shown that relapsed leukemias frequently carry RAS-pathway activating</div><div>mutations that could be targeted by MEK-inhibitors in combination with glucocorticosteroids.</div></div><div><h3>Methods</h3><div>Based on these pre-clinical results, we designed a phase I/II trial to evaluate the safety and preliminary efficacy of dexamethasone in combination with the MEK-inhibitor selumetinib for the treatment of relapsed/refractory RAS-pathway mutated ALL. The trial recruited both children and adults. Treatment consisted of oral selumetinib and dexamethasone in 28-day cycles.</div></div><div><h3>Results</h3><div>Initial study participants experienced serious adverse events due to infections, with three deaths from sepsis and pneumonia. Urgent safety measures were therefore introduced. This included reduction of the dexamethasone dose and frequency and the introduction of mandatory infectious prophylaxis. Nevertheless, twelve patients were recruited (four children, all B-immunophenotype; eight adults, 6/8 T-ALL). Nine patients were evaluable for response of whom four achieved a morphological complete remission after four weeks of treatment. Two of the responding patients (T- immunophenotype) remained stable on the combination for three and five cycles, respectively. The leukemic blasts of one responding patient were further characterized at time of progression, revealing persistence of the original NRAS mutation and upregulation of cell cycle/division genes as a potential targetable resistance mechanism. The study was stopped due to poor recruitment highlighting the challenges of academic multi-national early phase clinical trials in rare patient populations.</div></div><div><h3>Conclusion</h3><div>The combination of MEK-inhibitors with corticosteroids merits further investigation in RAS-pathway activated acute lymphoblastic leukemia, particularly T-ALL.</div></div>","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":"7 ","pages":"Article 100489"},"PeriodicalIF":0.0,"publicationDate":"2026-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146173451","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}

{"title":"[18F]FDG-avid nodal gliomatosis associated with ovarian immature teratoma – The importance of histological confirmation","authors":"Luisa Queiró ,&nbsp;Caroline C.C. Hulsker ,&nbsp;Leendert H.J. Looijenga ,&nbsp;Ronald R. de Krijger ,&nbsp;Marijn A. Scheijde-Vermeulen ,&nbsp;Pascal J.H. Kusters ,&nbsp;Annemieke S. Littooij ,&nbsp;Jeanette van Leeuwen ,&nbsp;Arthur J.A.T. Braat ,&nbsp;Annelies M.C. Mavinkurve-Groothuis","doi":"10.1016/j.ejcped.2026.100493","DOIUrl":"10.1016/j.ejcped.2026.100493","url":null,"abstract":"<div><div>Gliomatosis is a rare condition mainly associated with ovarian teratomas. It is characterized by the presence of mature glial tissue in extraovarian sites and most frequently found in the peritoneum (gliomatosis peritonei). Gliomatosis in the lymph nodes (nodal gliomatosis) has rarely been reported. We discuss a case of a 4-year-old girl with immature ovarian teratoma with concomitant gliomatosis peritonei and nodal gliomatosis, detected with magnetic resonance imaging (MRI) and Fluorine-18 fluorodeoxyglucose positron-emission tomography/computed tomography ([¹⁸F]FDG PET/CT) as [¹⁸F]FDG-avid lesions. Histologic examination showed mature well differentiated glial tissue. We aim to highlight the importance of histopathologic assessment of [¹⁸F]FDG positive lesions that may initially appear metastatic.</div></div>","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":"7 ","pages":"Article 100493"},"PeriodicalIF":0.0,"publicationDate":"2026-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147384839","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}

{"title":"Multi-tyrosine kinase inhibitors in paediatric soft tissue sarcoma – A review","authors":"Kim P.J. Schellekens ,&nbsp;Andrea Ferrari ,&nbsp;Michael T. Meister ,&nbsp;C. Michel Zwaan ,&nbsp;Susanne A. Gatz ,&nbsp;Max M. van Noesel ,&nbsp;Michela Casanova ,&nbsp;Reineke A. Schoot","doi":"10.1016/j.ejcped.2026.100484","DOIUrl":"10.1016/j.ejcped.2026.100484","url":null,"abstract":"<div><div>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.</div></div>","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":"7 ","pages":"Article 100484"},"PeriodicalIF":0.0,"publicationDate":"2026-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145978274","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}