EJC paediatric oncology最新文献

{"title":"Current insights and future directions in systemic therapies for gastroenteropancreatic neuroendocrine neoplasms (GEP-NENs) in children and adolescents: a critical review of advancements and challenges","authors":"Michaela Kuhlen ,&nbsp;Katharina Karges ,&nbsp;Marina Kunstreich ,&nbsp;Maximilian Schmutz ,&nbsp;Antje Redlich ,&nbsp;Rainer Claus ,&nbsp;Constantin Lapa","doi":"10.1016/j.ejcped.2025.100318","DOIUrl":"10.1016/j.ejcped.2025.100318","url":null,"abstract":"<div><div>Gastroenteropancreatic neuroendocrine neoplasms (GEP-NENs) in children and adolescents are rare and biologically heterogeneous. Due to their low incidence, therapeutic strategies are largely adapted from adult protocols, underscoring a critical need for paediatric-specific evidence.</div><div>Surgical resection remains the mainstay of curative treatment for localized disease and should be prioritized before the initiation of systemic therapy whenever feasible. This review synthesizes current knowledge on systemic therapies in paediatric GEP-NENs,</div><div>including somatostatin analogues (SSAs), peptide receptor radionuclide therapy (PRRT), chemotherapy, small molecules (e.g., everolimus, sunitinib), and immune checkpoint inhibitors (ICIs). While SSAs remain the mainstay for well-differentiated, somatostatin receptor (SSTR)-positive tumours, emerging data support the safety and potential efficacy of PRRT in paediatric populations, despite limited prospective evidence. Chemotherapy continues to play a role in high-grade or progressive disease, although responses are variable.</div><div>Supportive therapies, including high-dose proton pump inhibitors (PPIs), are also important in managing functional tumours and can significantly alleviate clinical symptoms in advanced disease.</div><div>Novel approaches, including SSTR antagonists, α- and β-emitting radiopharmaceuticals, and oncolytic virotherapy (e.g., SVV-001), are under active investigation in adults and may inform future paediatric protocols. Resistance mechanisms—particularly to SSAs—highlight the dynamic nature of tumour evolution and the need for individualized strategies.</div><div>These insights underscore the importance of molecular profiling and imaging-based SSTR assessment to guide therapeutic selection, particularly in refractory or complex paediatric cases. Future efforts should prioritize international collaboration, the design of rational combination regimens, and the integration of radiomics, genomics, and biomarker-driven approaches to advance precision medicine in paediatric GEP-NENs.</div></div>","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":"6 ","pages":"Article 100318"},"PeriodicalIF":0.0,"publicationDate":"2025-07-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144748756","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":"Developing patient reported outcome measures for children with cancer: A current perspective","authors":"Madeleine Adams ,&nbsp;Rachel M. Taylor","doi":"10.1016/j.ejcped.2025.100312","DOIUrl":"10.1016/j.ejcped.2025.100312","url":null,"abstract":"<div><div>Patient reported outcomes measures (PROM) are the gold standard for evaluating patient experience and quality of life (QOL), both in research studies and clinical practice. In paediatric oncology, the wide spectrum of disease and treatment strategies means that correct choice of PROM and robust methodology when developing new PROMs is vital to ensuring that the desired outcomes (e.g. pain, functional ability or quality of life) are accurately assessed. Children differ from adults both medically and developmentally meaning that specific factors should be considered including age and developmental ability, use of proxy/observer reports and mode of administration. Best practice guidelines outline the methodology for developing the content of a new PROM as well as assessing psychometric properties. This paper provides a review of the background, current guidelines, and methodology for developing and choosing PROMs for children with cancer.</div></div>","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":"6 ","pages":"Article 100312"},"PeriodicalIF":0.0,"publicationDate":"2025-07-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144748605","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":"Local therapy for rhabdomyosarcoma of the bladder and/or prostate without nodal or metastatic spread during the European paediatric Soft tissue sarcoma Study Group (EpSSG) RMS2005 study","authors":"Naima Smeulders ,&nbsp;Florent Guerin ,&nbsp;Mark N. Gaze ,&nbsp;Timothy Rogers ,&nbsp;Sheila Terwisscha van Scheltinga ,&nbsp;Federica De Corti ,&nbsp;Julia Chisholm ,&nbsp;Olga Slater ,&nbsp;Veronique Minard-Colin ,&nbsp;Beatrice Coppadoro ,&nbsp;Ilaria Zanetti ,&nbsp;Ross Craigie ,&nbsp;Gabriela Guillen Burrieza ,&nbsp;Patrizia Dall'Igna ,&nbsp;Raquel Davila Fajardo ,&nbsp;Pei S. Lim ,&nbsp;Cyrus Chargari ,&nbsp;Sophie Espenel ,&nbsp;Ana L. Luis Huertas ,&nbsp;Alexander Cho ,&nbsp;Helene Martelli","doi":"10.1016/j.ejcped.2025.100313","DOIUrl":"10.1016/j.ejcped.2025.100313","url":null,"abstract":"<div><h3>Background</h3><div>During the EpSSG RMS2005 trial, organ-sparing surgery (OSS) with brachytherapy (BT) became the local therapy (LT) of choice for selected patients with bladder-prostate rhabdomyosarcoma (BP-RMS). We compare this LT technique with surgical resection and/or external-beam radiotherapy.</div></div><div><h3>Methods</h3><div>Patients with BP-RMS without nodal or metastatic spread enrolled in RMS2005 were categorized by their LT, differentiating OSS from organ-depleting surgery (ODS) and BT from external-beam radiotherapy (EBRT). Progressive disease, relapse or death were considered events for progression-free survival (PFS) and all deaths for overall survival (OS).</div></div><div><h3>Results</h3><div>The cohort comprised 176 patients, aged 10days-21.8years (median 2.5years). Median follow-up was 6.5years (22months-12.5years): 5year-PFS was 80.3 % (95 %CI:73.6–85.5 %); 5year-OS was 90.7 % (95 %CI:85.3–94.2 %).</div><div>Patients selected for surgery alone or BT with/without OSS (BT+/-OSS) differed significantly in age, tumour size and location from those offered EBRT alone or any other surgery and radiotherapy. Nevertheless, 5year-PFS was similar for the LT groups. However, 5year-OS differed significantly, being highest in patients suitable for surgery alone (100 %; by ODS in 55 %) or BT+ /-OSS (98.1 %; 95 %CI:87.4–99.7 %). Patients with local tumour progression/relapse after EBRT failed salvage: 5year-OS was 81.8 % (95 %CI:58.5–92.8 %) for EBRT alone and 85.3 % (95 %CI:71.6–92.7 %) for surgery and radiotherapy.</div><div>Postponing LT until after chemotherapy cycle 7 did not significantly impact 5year-PFS or OS.</div></div><div><h3>Conclusions</h3><div>The risk of events was similar for different LT modalities; poor salvage after EBRT significantly reduced 5year-OS. Although not feasible for all, BT+ /-OSS offers an excellent prospect of cure, the best chance of organ retention while avoiding EBRT, and may be delayed for chemotherapy responsive tumours.</div></div>","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":"6 ","pages":"Article 100313"},"PeriodicalIF":0.0,"publicationDate":"2025-07-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144711246","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":"Preoperative short-term fasting in paediatric cancer care is safe, feasible and activates protective responses","authors":"D.S.J. Komninos ,&nbsp;C.A.J. Oudmaijer ,&nbsp;R.A. Ozinga ,&nbsp;K. Smit ,&nbsp;J.N.M. Ijzermans ,&nbsp;J.H.J. Hoeijmakers ,&nbsp;R.C. Minnee ,&nbsp;M.M. van den Heuvel-Eibrink ,&nbsp;W.P. Vermeij","doi":"10.1016/j.ejcped.2025.100309","DOIUrl":"10.1016/j.ejcped.2025.100309","url":null,"abstract":"<div><h3>Background</h3><div>Paediatric cancer patients often face treatment-induced complications significantly affecting Quality of Life. Short-term fasting (STF) could mitigate therapy-related adverse toxicity by triggering a protective survival response. While STF and other nutritional interventions have shown clinical benefits in adults, application in paediatric cancer remains unexplored. This study aims to explore the safety and feasibility of incorporating STF into the treatment regimen for children with renal cancer, while simultaneously showing a protective transcriptomic signature.</div></div><div><h3>Methods</h3><div>Here we selected children with a localized renal tumour at the Princess Máxima Center. In a randomized controlled setting preceding surgery (FIURTT-study), these children fasted for 10–18 h, depending on age, during which patient/parent experiences and blood measurements were collected. To investigate the STF-induced effects on enhancing postoperative recovery, kidney tissue was used for transcriptome analysis, and compared to a well-defined 2.5-day fasting response in adults.</div></div><div><h3>Results</h3><div>Blood measurements of 13 patients revealed significantly decreased glucose levels after STF, without occurrence of hypoglycaemia. Questionnaires demonstrated that patients and parents experienced no obvious burden and described the intervention as highly feasible. RNA-expression patterns in normal kidney tissue unveiled distinctions between cases with STF and those without, displaying significant difference in relevant pathways linked to activated protective-responses, resembling those in adults.</div></div><div><h3>Conclusion</h3><div>Our results indicate pre-operative STF to be safe and feasible in children with cancer. Also, we observed induction of the survival response at the transcriptional level, suggesting that STF induced a protective gene expression profile already after relatively short fasting periods.</div></div>","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":"6 ","pages":"Article 100309"},"PeriodicalIF":0.0,"publicationDate":"2025-07-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144631888","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":"Familial risks and incidence rates for childhood nervous system tumors in Sweden","authors":"Kari Hemminki ,&nbsp;Xinjun Li ,&nbsp;Kristina Sundquist ,&nbsp;Jan Sundquist ,&nbsp;Steffen Hirsch ,&nbsp;Jianguang Ji ,&nbsp;Akseli Hemminki ,&nbsp;Asta Försti","doi":"10.1016/j.ejcped.2025.100310","DOIUrl":"10.1016/j.ejcped.2025.100310","url":null,"abstract":"<div><h3>Background</h3><div>Childhood (&lt; 20 years) nervous system tumors manifest in some rare cancer syndromes but how commonly they present with familial clustering between various histological types is not well known at a nation-wide level. Our aim is to enhance understanding of familial risks in childhood nervous system cancers.</div></div><div><h3>Methods</h3><div>We used the Swedish population and cancer data from years 1958–2021 to address familial risks among nervous system cancers when the case was diagnosed before age 20 years but the proband could be diagnosed at any age. Adjusted familial (relative) risks were expressed as standardized incidence ratios (SIRs).</div></div><div><h3>Results</h3><div>Familial childhood cancer cases amounted to 123 in the brain, 15 in the spinal cord and 9 in peripheral nerves. Familial risk for concordant brain cancer was about 2.0 irrespective of proband. Concordant risk of spinal cord cancer was high when mothers (17.92) or siblings were probands (24.91). High familial risk of 34.53 was recorded for hemangioblastoma, and moderate high risks were observed also for schwannoma (4.07), ependymoblastoma (3.48) and female ganglioneuroma (7.72) whereas astrocytoma risks were at the level of common cancers at other sites (1.7–2.0). Hemangioblastoma families appeared not to be related to von Hippel-Lindau syndrome because of lack of pathognomonic signs other than hemangioblastoma in the families.</div></div><div><h3>Conclusion</h3><div>In spite of the low number of childhood nervous system tumors, we observed high familial risks for, probably novel, syndromic hemangioblastoma, and schwannomas for which concordant clusters were found particularly in the spinal cord. Inquiring about a detailed family history at diagnosis of a nervous system cancer in childhood may reveal a syndromic disease due to a constitutional variant amenable to treatment.</div></div>","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":"6 ","pages":"Article 100310"},"PeriodicalIF":0.0,"publicationDate":"2025-07-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144605535","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":"Corrigendum to “European standard clinical practice recommendations for newly diagnosed ependymoma of childhood and adolescence” [EJC Paediatr. Oncol. 5 (2025) 100227]","authors":"Alba Rubio-San-Simón ,&nbsp;Timothy A. Ritzmann ,&nbsp;Denise Obrecht-Sturm ,&nbsp;Martin Benesch ,&nbsp;Beate Timmermann ,&nbsp;Pierre Leblond ,&nbsp;John-Paul Kilday ,&nbsp;Geraldina Poggi ,&nbsp;Nicola Thorp ,&nbsp;Maura Massimino ,&nbsp;Marie-Lise van Veelen ,&nbsp;Martin Schuhmann ,&nbsp;Ulrich-Wilhelm Thomale ,&nbsp;Stephan Tippelt ,&nbsp;Ulrich Schüller ,&nbsp;Stefan Rutkowski ,&nbsp;Richard G. Grundy ,&nbsp;Stephanie Bolle ,&nbsp;Ana Fernández-Teijeiro ,&nbsp;Kristian W. Pajtler","doi":"10.1016/j.ejcped.2025.100305","DOIUrl":"10.1016/j.ejcped.2025.100305","url":null,"abstract":"","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":"6 ","pages":"Article 100305"},"PeriodicalIF":0.0,"publicationDate":"2025-07-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144580183","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":"Temozolomide-based radio-chemotherapy for newly diagnosed pediatric high-grade gliomas (HIT-HGG-2007): A prospective, multicenter, single-arm, phase II trial","authors":"Michael Karremann ,&nbsp;Thomas Perwein ,&nbsp;André O. von Bueren ,&nbsp;Gerrit H. Gielen ,&nbsp;Martin Benesch ,&nbsp;Gunther Nussbaumer ,&nbsp;Lea L. Friker ,&nbsp;Andreas Waha ,&nbsp;Dominik Sturm ,&nbsp;David T.W. Jones ,&nbsp;Olaf Witt ,&nbsp;Stefan M. Pfister ,&nbsp;Matthias Eyrich ,&nbsp;Ulrich W. Thomale ,&nbsp;Chiara Valentini ,&nbsp;Mechthild Krause ,&nbsp;Stefaan W. Van Gool ,&nbsp;Michael C. Frühwald ,&nbsp;Pablo Hernaiz-Driever ,&nbsp;Martin Ebinger ,&nbsp;Christof M. Kramm","doi":"10.1016/j.ejcped.2025.100308","DOIUrl":"10.1016/j.ejcped.2025.100308","url":null,"abstract":"<div><h3>Background</h3><div>The HIT-HGG-2007 trial investigated temozolomide (TMZ) radio-chemotherapy for pediatric patients with high-grade gliomas (pHGG) to demonstrate therapeutic non-inferiority compared to previous intensive radio-chemotherapy regimens (HIT-GBM-C/-D).</div></div><div><h3>Methods</h3><div>Between June 2009 and December 2016, 456 patients were enrolled into this international, prospective, single-arm, multicenter phase II trial in Germany, Austria, and Switzerland of whom 438 patients were evaluable for confirmatory analysis. Patients from the HIT-GBM-C/-D trials served as historic control (n = 439). Tumors of both cohorts with available tissue were re-classified according to the 2021 WHO classification of CNS tumors (n = 140).</div></div><div><h3>Results</h3><div>Regarding event-free-survival (EFS) rate at 6 months, non-inferiority of the HIT-HGG-2007 regimen was confirmed (p = 0.0125). In terms of exploratory analyses, median EFS and overall survival (OS) was 9.5 months (95 % confidence interval [CI95], 8.9–10.4) and 14.7 months (CI95, 13.5–16.3), superior to intensive chemotherapy regimens (p &lt; 0.0001 and p = 0.0328). EFS and OS remained superior after re-classification of tumors according to WHO2021 criteria. TMZ radio-chemotherapy had lower rates of severe hematological, gastrointestinal, and hepatic toxicity compared to HIT-GBM-C/-D. Younger age, WHO grade IV histology, tumor location in the brainstem or basal ganglia, and lower extent of resection were independent adverse risk factors for OS and EFS. <em>MGMT</em> gene promoter methylation status had no impact on EFS and OS.</div></div><div><h3>Conclusions</h3><div>The HIT-HGG-2007 trial demonstrated non-inferiority compared with intensive chemotherapy regimens. <em>MGMT</em> promotor methylation status had no impact on survival. Exploratory analysis supports treatment of newly diagnosed non-pontine pHGG according to the HIT-HGG-2007 regimen due to improved EFS and OS rates together with a favorable toxicity profile.</div></div>","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":"6 ","pages":"Article 100308"},"PeriodicalIF":0.0,"publicationDate":"2025-07-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144587417","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":"Hearing your child is diagnosed with leukemia: A qualitative study into parents’ perspectives","authors":"Petra Buursma ,&nbsp;Daniël Zwerus ,&nbsp;Esther M.M. van den Bergh ,&nbsp;Natasja Dors ,&nbsp;Peter M. Hoogerbrugge ,&nbsp;Martha A. Grootenhuis ,&nbsp;Marijke C. Kars ,&nbsp;Sasja A. Schepers","doi":"10.1016/j.ejcped.2025.100307","DOIUrl":"10.1016/j.ejcped.2025.100307","url":null,"abstract":"<div><h3>Introduction</h3><div>Hearing a pediatric leukemia diagnosis is overwhelming. Parent satisfaction with the diagnostic conversation contributes to building trust in the oncologist and adaptation to the disease. Limited research has explored parental communication needs during this critical moment. This study aimed to explore parental experiences with communication during diagnostic conversations in pediatric leukemia.</div></div><div><h3>Methods</h3><div>In this qualitative study, we conducted a thematic analysis on semi-structured interviews with parents of children diagnosed with leukemia between June 2022 and February 2023.</div></div><div><h3>Results</h3><div>In total, 25 interviews were conducted with 30 parents (47 % male) of 19 children. Parents described entering the diagnostic conversation in emotional shock, while simultaneously trying to regain control to fulfill their new parental role. We identified three themes facilitating this process. First, parents felt supported by (1) the freedom to ask questions and (2) appropriate responses to their emotions when they were expressed. Nevertheless, if they did not express their emotions, they preferred not being asked about them explicitly. Second, parents appreciated practical information to organize and maximize chances of survival, but felt additional burden when the child was overlooked during the conversation. Third, parents established trust in the oncologist based on their expertise, calm attitude and appearance of mutual respect. Parents valued decisions being made for them, if oncologists were transparent about their reasoning.</div></div><div><h3>Conclusion</h3><div>Results show the importance of validating parents’ preferences during the diagnostic conversation (e.g. about including the child and decision-making). Communication training may support oncologists in understanding the importance of exploring parental communication preferences during diagnostic conversations.</div></div>","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":"6 ","pages":"Article 100307"},"PeriodicalIF":0.0,"publicationDate":"2025-07-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144587418","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":"HOW ARE YOU FEELING TODAY? – Gaps and opportunities in capturing patient reported outcomes (PROs) in Pediatric Cancer Care","authors":"Anna Zettl ,&nbsp;Hannah Gsell ,&nbsp;Karolina Maślak ,&nbsp;Antti Karjalainen ,&nbsp;Ulrike Leiss ,&nbsp;Anita Kienesberger ,&nbsp;Carina Schneider","doi":"10.1016/j.ejcped.2025.100306","DOIUrl":"10.1016/j.ejcped.2025.100306","url":null,"abstract":"<div><div>Including children's perspectives in healthcare and clinical trials, particularly in pediatric oncology, is critical to promoting child-centered care. Research has shown that even very young children can understand complex medical concepts, highlighting the psychological and practical benefits of involving children in their care. Patient-Reported Outcome Measures (PROMs) play a critical role in capturing young patients' insights about their symptoms and well-being. This enables individualized care and an improvement in quality of life. Despite their potential, the use of PROMs in pediatric oncology faces significant challenges, including age-related barriers to understanding and the exclusion of cognitively impaired children. PROMs, when used effectively, can empower patients, improve communication and influence clinical decisions in favor of young patients. However, their relevance depends on the active participation of patients and caregivers in their design and use. PROMs also need to be further developed in the area of survivorship to close gaps in long-term care where many physical and psychological problems otherwise go unrecognized. Tailoring PROMs to patients' needs ensures that every patient, regardless of age or neurocognitive ability, contributes to a comprehensive understanding of the impact of treatment. Incorporating PROMs into routine care and clinical trials, supported by patient advocacy and systematic implementation, can address critical gaps. This approach ensures that pediatric oncology adapts to the diverse needs of children and survivors to improve long-term outcomes and promote a truly patient-centered model.</div></div>","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":"6 ","pages":"Article 100306"},"PeriodicalIF":0.0,"publicationDate":"2025-07-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144653147","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":"From the bench of molecular understanding to the bedside of optimal therapy for BCR::ABL1 and ABL-class acute lymphoblastic leukemia in children and adolescents","authors":"Sarah K. Tasian ,&nbsp;Judith M. Boer ,&nbsp;Monique L. den Boer","doi":"10.1016/j.ejcped.2025.100304","DOIUrl":"10.1016/j.ejcped.2025.100304","url":null,"abstract":"<div><div>This review aims to discuss similarities and dissimilarities between <em>BCR</em>::<em>ABL1</em>-rearranged (Philadelphia chromosome-positive [Ph+]) and ABL-class fusion-driven <em>BCR::ABL1</em>-like (Ph-like) acute lymphoblastic leukemia (ALL) in children and adolescents. Recent insights into the biology of these historically high-risk leukemias, modern laboratory diagnostics, current treatment approaches, potential causes of treatment failure, emerging new targeted therapies and immunotherapeutic approaches for patients will be discussed. While the primary focus of this review is upon children and adolescents with <em>BCR</em>::<em>ABL1</em>-positive and ABL-class ALL, extended knowledge from recent adult clinical trials will also be addressed.</div></div>","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":"6 ","pages":"Article 100304"},"PeriodicalIF":0.0,"publicationDate":"2025-06-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144523438","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}