EJC paediatric oncology最新文献

{"title":"An assessment of circulating biomarkers and germline genetic variants in predicting chemotherapy associated mucositis in Ewing sarcoma","authors":"Pawan Gulati ,&nbsp;Vickyanne Carruthers ,&nbsp;Claire Hutton ,&nbsp;Chloe Cassidy ,&nbsp;Edward B. Amankwatia ,&nbsp;Séréna Pascual ,&nbsp;Electra Florence ,&nbsp;Tsegay G. Gebru ,&nbsp;Andrea L. Jorgensen ,&nbsp;Alastair Greystoke ,&nbsp;Guy Makin ,&nbsp;Martin G. McCabe ,&nbsp;Daniel B. Hawcutt ,&nbsp;Gareth J. Veal ,&nbsp;David Jamieson","doi":"10.1016/j.ejcped.2025.100216","DOIUrl":"10.1016/j.ejcped.2025.100216","url":null,"abstract":"<div><h3>Background</h3><div>Treatment of Ewing sarcoma is associated with severe toxicities including chemotherapy-induced mucositis. Here we investigated the role of circulating biomarkers and genetic factors in predicting mucositis severity in Ewing sarcoma.</div></div><div><h3>Methods</h3><div>Blood samples were collected from 111 Ewing sarcoma patients during treatment. Circulating total CK18 and FLT3 ligand concentrations were measured in plasma. Germline DNA was used to investigate associations between genetic variants and mucositis severity.</div></div><div><h3>Results</h3><div>An increase in median tCK18 levels was observed during cycle 1, from 189 (86−736) U/L at cycle 1 day 1 pre-chemotherapy to 311 (141–1138) U/L on cycle 1 day 2–5 (p = 0.0001). Patients experiencing a ≥ 1.3-fold increase in tCK18 at 48–120 hours after administration of the first cycle had a higher likelihood of developing grade 3 mucositis in any cycle (p = 0.044). Genetic analysis revealed an association between a gene set associated with chemotherapy induced severe mucositis and differentially expressed genes set for minor salivary gland (p = 4.12 ×10⁻⁴) and esophageal mucosa (p = 1.55 ×10⁻⁵).</div></div><div><h3>Discussion</h3><div>Early increases in circulating CK18 levels correlated with grade 3 mucositis. Genome-wide association analysis highlighted genes that may be associated with mucositis pathology, offering insights into biological mechanisms underlying susceptibility.</div></div>","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":"5 ","pages":"Article 100216"},"PeriodicalIF":0.0,"publicationDate":"2025-01-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143104304","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"How to use monoclonal antibody-based therapy in ALL","authors":"Erica Brivio ,&nbsp;Sujith Samarasinghe","doi":"10.1016/j.ejcped.2025.100214","DOIUrl":"10.1016/j.ejcped.2025.100214","url":null,"abstract":"<div><div>Acute Lymphoblastic Leukemia (ALL) represents a significant challenge in haematology, particularly due to its aggressive nature, high relapse rates in adults and toxicity of treatment. Over the past decade, monoclonal antibody (MoAb)-based therapies have emerged as a promising approach to target specific antigens on leukemic cells, offering higher specificity, reduced toxicity, and improved response rates. This article provides an in-depth examination of the journey from laboratory research to clinical application, discussing mechanisms of action, key MoAbs used in ALL, their clinical applications, current challenges, and potential future directions in antibody-based therapy.</div></div>","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":"5 ","pages":"Article 100214"},"PeriodicalIF":0.0,"publicationDate":"2025-01-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143171720","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"The safety and efficacy of immune checkpoint blockade in children, adolescents, and young adults: A systematic review and meta-analysis","authors":"Pedro C.A. Reis ,&nbsp;João Evangelista Ponte Conrado ,&nbsp;Mariana Macambira Noronha ,&nbsp;Luís Felipe Leite da Silva ,&nbsp;Erick Figueiredo Saldanha ,&nbsp;Jonathan Metts","doi":"10.1016/j.ejcped.2025.100215","DOIUrl":"10.1016/j.ejcped.2025.100215","url":null,"abstract":"<div><h3>Background</h3><div>Immune checkpoint blockade (ICB) has changed the treatment landscape for many types of adult cancer. However, for children, adolescents, and young adults (CAYAs) clinical experience lags behind that of adults. Therefore, we performed a systematic review and meta-analysis to evaluate the safety and efficacy of ICB in the CAYA population.</div></div><div><h3>Methods</h3><div>We searched PubMed, Embase, and Cochrane Library databases for clinical trials evaluating ICB therapies for cancer in CAYA patients. We pooled the incidences of treatment-related adverse events (TRAEs), objective response rates (ORRs), stable disease (SD), and their corresponding confidence intervals (CIs). For the ORR and TRAE endpoints, we performed a subgroup analysis of each drug (PD-1, PD-L1, and CTLA-4) and tumor type.</div></div><div><h3>Results</h3><div>15 trials were included, comprising 797 patients (median age ranging from 6.5 to 16.0 years). All-grade TRAE rate of 66 % was found (95 % CI 60–71), while the proportion of grade 3/4 TRAEs was 19 % (95 % CI 14–27). For tumor type subgroup analysis of all-grade TRAEs and grade 3/4 TRAEs, solid tumors had the highest rates, 92 % (95 % CI 41–99) and 32 % (95 % CI 11–63), respectively. Fatigue, anemia, and nausea were the most frequently reported TRAEs. The ORR was 13 % (95 % CI 5–27). In subgroup analyses, PD-1 inhibitors and Hodgkin Lymphoma had the highest ORR, with 25 % (95 % CI 8–56) and 59 % (95 % CI 23–87), respectively. SD was noted in 21 % (95 % CI 14–30) of patients.</div></div><div><h3>Conclusions</h3><div>Overall, ICB is well tolerated in CAYA patients with different cancer types, and certain subsets of CAYA cancer are ICB-responsive.</div></div>","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":"5 ","pages":"Article 100215"},"PeriodicalIF":0.0,"publicationDate":"2025-01-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143104358","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"UNCAN.eu, the European platform to understand cancer, and childhood cancers","authors":"Gilles Vassal","doi":"10.1016/j.ejcped.2024.100212","DOIUrl":"10.1016/j.ejcped.2024.100212","url":null,"abstract":"<div><div>The EU will create the UNCAN.eu platform, a European Federated Data Hub to provide access to high -quality cancer research data in order to better understand cancer and drive innovation. A Coordinated and Supported Action has delivered to the EU in November 2023 a blueprint of the UNCAN.eu including a strategic roadmap, the needed data and infrastructures, the interactions with all EU infrastructures and initiatives, a governance, a financial plan and a budget. SIOP Europe and CCI Europe were partners of the consortium to address the needs of children, adolescents and young adult with cancer as well as childhood cancer survivors. This article summarizes the blueprint proposal and how the UNCAN.eu could facilitate the implementation of a European Childhood Cancer Data Strategy through facilitated access to comprehensive childhood cancer data (molecular data, imaging, pathology, clinical trials and real world data) to accelerate innovation for children, adolescents and young adults with cancer.</div></div>","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":"5 ","pages":"Article 100212"},"PeriodicalIF":0.0,"publicationDate":"2025-01-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143104303","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Preparedness of European pediatric oncologists to integrate AI in the clinical routine","authors":"Alberto E. Tozzi ,&nbsp;Diana Ferro ,&nbsp;Ileana Croci ,&nbsp;Francesco Fabozzi ,&nbsp;Angela Mastronuzzi","doi":"10.1016/j.ejcped.2024.100213","DOIUrl":"10.1016/j.ejcped.2024.100213","url":null,"abstract":"<div><h3>Background</h3><div>Artificial intelligence (AI) holds promise in pediatric oncology, yet its full potential faces challenges. We undertook a survey aimed at assessing the viewpoints of European pediatric oncologists delving into their perceptions and expectations regarding the potential influence of AI in their clinical workflows.</div></div><div><h3>Method</h3><div>We conducted a survey by means of four hypothetical scenarios using AI and the Shinners Artificial Intelligence Perception (SHAIP) tool to assess healthcare professionals' perceptions of AI in pediatric oncology. We performed multinomial logistic regression to explore associations of responses to clinical scenarios with age and SHAIP scores.</div></div><div><h3>Results</h3><div>We obtained 140 responses and the analysis was performed on 108. The SHAIP questionnaire mean total score was 3.29 (SD 0.93) for the professional impact, and 2.37 (SD 0.61) for preparedness. Regarding the clinical scenarios, 34.9 % of respondents would ask for a procedure for confirming their diagnosis in case of discrepancy between AI decision support and human diagnosis; 55.8 % would be concerned about the generalizability an AI decision support system in case of lack of data from certain geographic areas during algorithm training; 47.6 % would feel uncomfortable in the informed consent process for an AI intervention; 10.2 % would no longer trust AI in case of a cyberattack affecting AI support for diagnosis.</div></div><div><h3>Discussion</h3><div>This survey underscores the importance of AI tools in pediatric oncology that incorporate human oversight in clinical decision-making and training AI algorithms with diverse and representative data. Our findings suggest that pediatric oncologists may not be adequately prepared for the seamless integration of AI in clinical practice.</div></div>","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":"5 ","pages":"Article 100213"},"PeriodicalIF":0.0,"publicationDate":"2025-01-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143171719","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Local application of sodium thiosulfate as an otoprotectant for cisplatin-exposed patients – A narrative literature review to explore the potential benefit for children with cancer","authors":"Nienke Streefkerk ,&nbsp;Amirhossein Masroor ,&nbsp;James I. Geller ,&nbsp;Martine van Grotel ,&nbsp;Marc Ansari ,&nbsp;Eric Bouffet ,&nbsp;Archie Bleyer ,&nbsp;Brice Fresnau ,&nbsp;Michael Sullivan ,&nbsp;Alwin D.R. Huitema ,&nbsp;Alexander E. Hoetink ,&nbsp;Per Kogner ,&nbsp;Rudolf Maibach ,&nbsp;Allison F. O’Neill ,&nbsp;Vassilios Papadakis ,&nbsp;Kaukab M. Rajput ,&nbsp;Gareth J. Veal ,&nbsp;Penelope R. Brock ,&nbsp;Annelot J.M. Meijer ,&nbsp;Marry M. van den Heuvel-Eibrink","doi":"10.1016/j.ejcped.2024.100211","DOIUrl":"10.1016/j.ejcped.2024.100211","url":null,"abstract":"<div><h3>Background</h3><div>Ototoxicity is a highly prevalent, serious, and irreversible side effect in cisplatin-treated childhood cancer patients, which can significantly impact speech-language development, psychosocial development, and quality of life. In this respect, the development and implementation of reliable and safe otoprotectants is urgently needed. Sodium thiosulfate (STS*) is an otoprotective drug, recently approved for intravenous administration in cisplatin-treated children with non-disseminated cancer. Intratympanic STS application has been developed as a potential strategy to reduce systemic exposure. To explore potential opportunities for this approach, we have reviewed available literature addressing efficacy, safety, and pharmacokinetics of local STS administration.</div></div><div><h3>Methods</h3><div>A PubMed search, focused on clinical and pre-clinical efficacy, safety, and pharmacokinetics of local STS application in cisplatin-exposed subjects of all ages was performed.</div></div><div><h3>Findings</h3><div>From 256 studies, ten studies met the inclusion criteria, including seven preclinical studies, and three clinical studies. Four studies (two preclinical, two clinical) which included pharmacokinetic data, showed that locally administered STS was associated with low systemic serum STS levels. Preclinical studies in guinea pigs showed a significant protective effect on outer hair cell loss or hearing function. However, two clinical trials in adults did not show convincing evidence of otoprotection, by locally administered STS.</div></div><div><h3>Interpretation</h3><div>Preclinical studies suggest a potential benefit of locally administered STS, however clinical evidence for a significant otoprotective effect is not yet available. The burden and potential sequalae of repeated intratympanic procedures in children, together with the low level of evidence of efficacy, currently limited to pre-clinical data, suggests that further study and potentially improved technology to apply local STS is required for childhood cancer patients receiving cisplatin.</div></div>","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":"5 ","pages":"Article 100211"},"PeriodicalIF":0.0,"publicationDate":"2024-12-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143171718","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"European standard clinical practice recommendations for paediatric high-grade gliomas","authors":"Elwira Szychot ,&nbsp;Géraldine Giraud ,&nbsp;Darren Hargrave ,&nbsp;Dannis van Vuurden ,&nbsp;Jacques Grill ,&nbsp;Veronica Biassoni ,&nbsp;Maura Massimino ,&nbsp;André O. von Bueren ,&nbsp;Rejin Kebudi ,&nbsp;Maria João Gil-da-Costa ,&nbsp;Sophie Veldhuijzen van Zanten ,&nbsp;Simon Bailey ,&nbsp;Michael Karremann ,&nbsp;Stephanie Bolle ,&nbsp;Thankamma Ajithkumar ,&nbsp;Mechthild Krause ,&nbsp;Yasmin Lassen-Ramshad ,&nbsp;Geert Janssens ,&nbsp;Giovanni Morana ,&nbsp;Ulrike Löbel ,&nbsp;Christof M. Kramm","doi":"10.1016/j.ejcped.2024.100210","DOIUrl":"10.1016/j.ejcped.2024.100210","url":null,"abstract":"<div><div>Paediatric high-grade gliomas (pedHGGs) are highly invasive brain tumours accounting for approximately 15 % of all central nervous system (CNS) tumours in children and adolescents. The outcome for these tumours is generally poor with 5-year survival rates of less than 20 %. Despite improved biological insights into pedHGGs and the promise of more effective therapies, little progress has been made in the effective treatment and the outcome of these tumours over the last four decades. Much of the evidence for the use of chemotherapy in pedHGGs is extrapolated from adult data, and the evidence for its use in the paediatric population is still weak. This guideline was written by members of the SIOPE HGG Working Group as part of the European Standard Clinical Practice (ESCP) Project of the European Reference Network for Paediatric Oncology, ERN PaedCan. The guideline aims to integrate available evidence-based and expert opinion-based information to assist healthcare professionals in the management of pedHGGs and in an attempt to provide equity in healthcare reflecting the varying resources of each European country.</div></div>","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":"5 ","pages":"Article 100210"},"PeriodicalIF":0.0,"publicationDate":"2024-12-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143104305","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Artificial intelligence in paediatric cancer: Insights from innovation experts in the UNICA4EU project","authors":"Pilar Gangas ,&nbsp;Norbert Graf ,&nbsp;Shuping Wen ,&nbsp;Carlotta Cattaneo ,&nbsp;Marilena Bicchieri ,&nbsp;Niamh Lennox-Chhugani","doi":"10.1016/j.ejcped.2024.100208","DOIUrl":"10.1016/j.ejcped.2024.100208","url":null,"abstract":"<div><div>This article presents the results of interviews with AI development and innovation experts, focusing on the potential contribution of AI to paediatric cancer treatment, its barriers and facilitators. AI-based technologies are expanding in health care provision and potentially to paediatric oncology, particular imageology. However, no AI based technology specifically developed for paediatric cancer has been identified. Interviews identified key barriers, including legal, regulatory and ethical challenges; validation and evaluation standards; integration with public healthcare systems; acceptance, explainability and trust of AI technologies; digital literacy and skills development; data management and privacy Protection; and promoting multidisciplinary collaboration. Facilitators largely coincide, including legal, regulatory and ethical considerations; data management and storage; building trust and ensuring privacy; engaging key stakeholders; promoting multidisciplinary collaboration through AI technologies; and education, training and innovation programmes. Barriers can be turned into facilitators if properly managed. These interviews were conducted under the EU funded project UNICA4EU (Towards a UNIque approach for artificial intelligence data-driven solutions to fight Childhood cAncer FOR EUrope)(1), which was implemented between 2022 and 2024. The goal of the overall project was to analyse the current landscape of Artificial Intelligence (AI) and to map applications to Childhood Cancer. This work further develops and confirms the results of a previous EU-funded pilot project, A crowdsourced ecosystem to fight childhood cancer (EU4CHILD)(1).</div></div>","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":"5 ","pages":"Article 100208"},"PeriodicalIF":0.0,"publicationDate":"2024-12-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143104302","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Superior outcome but high incidence of pseudoprogression and unfavorable neurocognitive outcome in children with embryonal tumor with multylayered rosettes treated with radiation and high-dose chemotherapy with tandem autologous stem cell rescue","authors":"Elena Carceller ,&nbsp;Felisa Vázquez-Gómez ,&nbsp;Sara Sirvent ,&nbsp;José Luis Moreno ,&nbsp;Marta González-Vicent ,&nbsp;Borja Esteso ,&nbsp;Luis Madero ,&nbsp;Alvaro Lassaletta","doi":"10.1016/j.ejcped.2024.100207","DOIUrl":"10.1016/j.ejcped.2024.100207","url":null,"abstract":"<div><h3>Objective</h3><div>Embryonal tumor with multilayered rosettes (ETMR) is a rare and highly aggressive embryonal brain tumor in young children. Treatment of this tumor often includes maximal safe resection, radiotherapy, and high-dose chemotherapy (HDCT) with autologous stem cell transplantation (ASCT). Early radiation has been recommended according to recent outcome results. The combination of this multimodal treatment may provoke radiographic changes that can mimic tumor progression. This phenomenon is known as pseudoprogression.</div></div><div><h3>Methods</h3><div>We report four consecutive patients under 5 years old with ETMR, treated according to CCG 99703 protocol (which includes conventional chemotherapy and HDCT with ASCT). Off protocol, radiation was given before (three patients) or after (one patient) high-dose chemotherapy.</div></div><div><h3>Results</h3><div>All patients developed pseudoprogression. All patients but one presented symptoms such as irritability, hypotonia, headache and vomiting. Severe neurocognitive impairment was evident in all patients. At a median follow-up of 50.5 months (range, 37–64), three of the patients are alive. Patients who are alive, continue to require significant multidisciplinary support to address treatment sequelae.</div></div><div><h3>Conclusion</h3><div>This case series demonstrates that, while survival rates appear encouraging, combining radiation therapy with high-dose chemotherapy and autologous stem cell transplantation (ASCT) in the treatment of embryonal tumors with multilayered rosettes may carry a substantial risk of pseudoprogression and notable neurological damage.</div></div>","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":"5 ","pages":"Article 100207"},"PeriodicalIF":0.0,"publicationDate":"2024-12-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143104360","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"KMT2A-rearranged acute lymphoblastic leukaemia","authors":"Rishi S. Kotecha ,&nbsp;Rob Pieters ,&nbsp;Janine Stutterheim","doi":"10.1016/j.ejcped.2024.100204","DOIUrl":"10.1016/j.ejcped.2024.100204","url":null,"abstract":"<div><div><em>KMT2A</em>-rearranged acute lymphoblastic leukaemia (ALL) represents a high risk subtype of childhood ALL. Historical treatment strategies have comprised of intensification with conventional chemotherapy. However, outcomes have remained consistently poor compared to the advances that have been seen for other ALL subtypes, particularly for infants diagnosed before their first birthday. The advent of novel immunotherapeutic approaches has led to a change in the treatment paradigm, with the integration of blinatumomab to the current suite of clinical trials for <em>KMT2A</em>-rearranged ALL expected to result in marked improvements. Furthermore, significant progress has been made to understand the unique biology of <em>KMT2A</em>-rearranged ALL, which has led to the development of novel agents that directly target the <em>KMT2A</em> complex or dysregulated proteins/pathways. Clinical trials are currently poised to evaluate therapies such as venetoclax and menin inhibitors, offering further hope for achieving a cure. In this review, we discuss the remarkable progress that has been made for <em>KMT2A</em>-rearranged ALL, leading to much optimism for improved outcomes in the future.</div></div>","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":"4 ","pages":"Article 100204"},"PeriodicalIF":0.0,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143181358","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}