Pub Date : 2025-12-01Epub Date: 2025-11-04DOI: 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
{"title":"SINGLE-CELL AND SPATIAL TRANSCRIPTOMICS OF PEDIATRIC MYELODYSPLASTIC SYNDROME WITH REFRACTORY CYTOPENIA OF CHILDHOOD REVEAL A DISRUPTED BONE MARROW NICHE DRIVING IMPAIRED HEMATOPOIESIS","authors":"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","doi":"10.1016/j.ejcped.2025.100331","DOIUrl":"10.1016/j.ejcped.2025.100331","url":null,"abstract":"","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":"6 ","pages":"Article 100331"},"PeriodicalIF":0.0,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145428507","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}
Pub Date : 2025-12-01Epub Date: 2025-11-04DOI: 10.1016/j.ejcped.2025.100343
Jolien De Waele , Mattias Hofmans , Alexandra Fischer , Albert Catala , Michael Dworzak , Miriam Erlacher , Henrik Hasle , Riccardo Masetti , Markus Schmugge , Marek Ussowicz , Steven Keogh , Ozlem Tufekci , Mehmet Akif Yesilipek , Ayami Yoshimi , Dirk Lebrecht , Maximilian Schönung , Peter Nöllke , Barbara De Moerloose , Charlotte M. Niemeyer
{"title":"LONG-TERM OUTCOME OF AN OBSERVATIONAL APPROACH IN PATIENTS WITH NRAS-MUTATED JUVENILE MYELOMONOCYTIC LEUKEMIA AND ABSENCE OF HIGH RISK FEATURES","authors":"Jolien De Waele , Mattias Hofmans , Alexandra Fischer , Albert Catala , Michael Dworzak , Miriam Erlacher , Henrik Hasle , Riccardo Masetti , Markus Schmugge , Marek Ussowicz , Steven Keogh , Ozlem Tufekci , Mehmet Akif Yesilipek , Ayami Yoshimi , Dirk Lebrecht , Maximilian Schönung , Peter Nöllke , Barbara De Moerloose , Charlotte M. Niemeyer","doi":"10.1016/j.ejcped.2025.100343","DOIUrl":"10.1016/j.ejcped.2025.100343","url":null,"abstract":"","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":"6 ","pages":"Article 100343"},"PeriodicalIF":0.0,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145428516","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}
Pub Date : 2025-12-01Epub Date: 2025-11-04DOI: 10.1016/j.ejcped.2025.100350
Davide Leardini , Miriam Erlacher , Luca Vinci , Barbara De Moerloose , Henrik Hasle , Katja Hetnik , Marko Kavcic , Kirsten Thus , Dominik Turkiewicz , Riccardo Masetti , Michael Dworzak , Yvonne L. Behrens , Martina Pigazzi , Peter Nöllke , Senthilkumar Ramamoorthy , Dirk Lebrecht , for the MDS-EB Working Group of EWOG-MDS and EWOG-MDS National Coordinators
{"title":"GENETIC LANDSCAPE OF PRIMARY MYELODYSPLASTIC SYNDROMES WITH EXCESS OF BLASTS (MDS-EB) IN CHILDREN AND ADOLESCENTS","authors":"Davide Leardini , Miriam Erlacher , Luca Vinci , Barbara De Moerloose , Henrik Hasle , Katja Hetnik , Marko Kavcic , Kirsten Thus , Dominik Turkiewicz , Riccardo Masetti , Michael Dworzak , Yvonne L. Behrens , Martina Pigazzi , Peter Nöllke , Senthilkumar Ramamoorthy , Dirk Lebrecht , for the MDS-EB Working Group of EWOG-MDS and EWOG-MDS National Coordinators","doi":"10.1016/j.ejcped.2025.100350","DOIUrl":"10.1016/j.ejcped.2025.100350","url":null,"abstract":"","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":"6 ","pages":"Article 100350"},"PeriodicalIF":0.0,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145428523","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}
Pub Date : 2025-12-01Epub Date: 2025-07-05DOI: 10.1016/j.ejcped.2025.100307
Petra Buursma , Daniël Zwerus , Esther M.M. van den Bergh , Natasja Dors , Peter M. Hoogerbrugge , Martha A. Grootenhuis , Marijke C. Kars , Sasja A. Schepers
Introduction
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.
Methods
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.
Results
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.
Conclusion
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.
{"title":"Hearing your child is diagnosed with leukemia: A qualitative study into parents’ perspectives","authors":"Petra Buursma , Daniël Zwerus , Esther M.M. van den Bergh , Natasja Dors , Peter M. Hoogerbrugge , Martha A. Grootenhuis , Marijke C. Kars , 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":"2025-12-01","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}
Febrile neutropenia (FN) is a life-threatening complication for children with cancer. Early administration of broad–spectrum antibiotics has significantly improved outcome. In many countries, children with culture negative FN remain hospitalized on intravenous antibiotic treatment until neutrophil count recovery. The aim of this study was to explore the safety of short course antibiotic regimens in children with culture negative FN.
Methods
A prospective cohort study with 1:1 matched historical controls was conducted in a single center. Children with cancer admitted with a low-risk episode of FN from 2017 until 2020 and met the inclusion criteria, were included in the study. The study was initiated after institutional adoption of an early antibiotic discontinuation protocol for low-risk FN. All children received empiric treatment with cefepime or piperacillin/tazobactam combined with an aminoglycoside. Antibiotics were discontinued after 48 h of defervescence, when blood cultures were negative regardless of neutrophil count.
Results
Thirty-six out of 456 FN episodes met inclusion criteria. There were no readmissions due to fever or infection during neutropenia. Median neutrophil count at discontinuation was 0.16 × 109/L. Median length of hospitalization was 2 days, compared with 6 days until neutrophil recovery (p < 0.0001) and 7 days for the control group (p < 0.0001). There was significant reduction of hospital charges compared to controls.
Conclusion
This study provides evidence that early discontinuation of antibiotics in oncology patients with a low-risk episode of FN is safe, regardless of neutrophil count. Reduced exposure to antibiotics, shorter hospitalization and lower costs are beneficial secondary outcomes observed in this study.
{"title":"Early discontinuation of empiric antibiotic therapy in children treated for cancer who develop febrile neutropenia: A prospective cohort study","authors":"Smaragda Papachristidou , Dimitrios Doganis , Georgia Kourlaba , George Pantalos , Sophia Pasparaki , Margarita Baka , Apostolos Pourtsidis , Lydia Kossiva , Vasiliki Papaevangelou , Nikolaos Spyridis , Maria Tsolia","doi":"10.1016/j.ejcped.2025.100321","DOIUrl":"10.1016/j.ejcped.2025.100321","url":null,"abstract":"<div><h3>Introduction</h3><div>Febrile neutropenia (FN) is a life-threatening complication for children with cancer. Early administration of broad–spectrum antibiotics has significantly improved outcome. In many countries, children with culture negative FN remain hospitalized on intravenous antibiotic treatment until neutrophil count recovery. The aim of this study was to explore the safety of short course antibiotic regimens in children with culture negative FN.</div></div><div><h3>Methods</h3><div>A prospective cohort study with 1:1 matched historical controls was conducted in a single center. Children with cancer admitted with a low-risk episode of FN from 2017 until 2020 and met the inclusion criteria, were included in the study. The study was initiated after institutional adoption of an early antibiotic discontinuation protocol for low-risk FN. All children received empiric treatment with cefepime or piperacillin/tazobactam combined with an aminoglycoside. Antibiotics were discontinued after 48 h of defervescence, when blood cultures were negative regardless of neutrophil count.</div></div><div><h3>Results</h3><div>Thirty-six out of 456 FN episodes met inclusion criteria. There were no readmissions due to fever or infection during neutropenia. Median neutrophil count at discontinuation was 0.16 × 10<sup>9</sup>/L. Median length of hospitalization was 2 days, compared with 6 days until neutrophil recovery (p < 0.0001) and 7 days for the control group (p < 0.0001). There was significant reduction of hospital charges compared to controls.</div></div><div><h3>Conclusion</h3><div>This study provides evidence that early discontinuation of antibiotics in oncology patients with a low-risk episode of FN is safe, regardless of neutrophil count. Reduced exposure to antibiotics, shorter hospitalization and lower costs are beneficial secondary outcomes observed in this study.</div></div>","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":"6 ","pages":"Article 100321"},"PeriodicalIF":0.0,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145098895","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}
Pub Date : 2025-12-01Epub Date: 2025-09-30DOI: 10.1016/j.ejcped.2025.100420
Brenda Mallon , Chérif Dial , Fatou Binetou Akonde , Marie Jo Diémé Ahouidi , Ndella Diouf , Malick Anne , Awa Toure , Francis Diedhiou , Adama Faye , Aïssatou Ndiaye , Laila Hessissen , Eva Steliarova-Foucher , Jacqueline Clavel
Introduction and Background
The Global Initiative for Childhood Cancer of the World Health Organisation emphasise the importance of registration, especially in low-and middle-income countries, where data are sparse. The Hospital-Based Cancer Registry (HBCR) established in the Paediatric Oncology Unit (POU) at Le Dantec Hospital in Dakar, Senegal, collects data about patients from the whole of Senegal. This study investigates the potential for establishing a Population-Based Childhood Cancer Registry (PBCR) in the region of Dakar in Senegal.
Methodology
In an attempt to cover the entire Dakar region in addition to those included in the HBCR, cases were also sought in other hospitals and departments susceptible to diagnose or treat cancer in children. Completeness was assessed and cancer patterns described.
Results
From 2017–2019, 568 cancers in children under 18 were recorded in the HBCR, including 133 residents of the Dakar region. An additional 283 cases were identified from other sources, 149 of them without information on their place of residence. Discrepancies in cancer type distribution were observed between the data sources. The overall incidence rate estimate was 33 per million person-years.
Discussion
The additional cases found outside the HBCR, the observed variability of cancer types, and the low incidence rates highlight the need for a PBCR to standardize the registration process and improve completeness of ascertainment. Given the few identified data sources, a PBCR in the Dakar region would provide accurate information about childhood cancer burden in the covered population. However, political and financial support is necessary to sustain the registry.
{"title":"The path towards a population-based childhood cancer registry in the region of Dakar in Senegal: A feasibility study of the Franco-African Paediatric Oncology Group (GFAOP)","authors":"Brenda Mallon , Chérif Dial , Fatou Binetou Akonde , Marie Jo Diémé Ahouidi , Ndella Diouf , Malick Anne , Awa Toure , Francis Diedhiou , Adama Faye , Aïssatou Ndiaye , Laila Hessissen , Eva Steliarova-Foucher , Jacqueline Clavel","doi":"10.1016/j.ejcped.2025.100420","DOIUrl":"10.1016/j.ejcped.2025.100420","url":null,"abstract":"<div><h3>Introduction and Background</h3><div>The Global Initiative for Childhood Cancer of the World Health Organisation emphasise the importance of registration, especially in low-and middle-income countries, where data are sparse. The Hospital-Based Cancer Registry (HBCR) established in the Paediatric Oncology Unit (POU) at Le Dantec Hospital in Dakar, Senegal, collects data about patients from the whole of Senegal. This study investigates the potential for establishing a Population-Based Childhood Cancer Registry (PBCR) in the region of Dakar in Senegal.</div></div><div><h3>Methodology</h3><div>In an attempt to cover the entire Dakar region in addition to those included in the HBCR, cases were also sought in other hospitals and departments susceptible to diagnose or treat cancer in children. Completeness was assessed and cancer patterns described.</div></div><div><h3>Results</h3><div>From 2017–2019, 568 cancers in children under 18 were recorded in the HBCR, including 133 residents of the Dakar region. An additional 283 cases were identified from other sources, 149 of them without information on their place of residence. Discrepancies in cancer type distribution were observed between the data sources. The overall incidence rate estimate was 33 per million person-years.</div></div><div><h3>Discussion</h3><div>The additional cases found outside the HBCR, the observed variability of cancer types, and the low incidence rates highlight the need for a PBCR to standardize the registration process and improve completeness of ascertainment. Given the few identified data sources, a PBCR in the Dakar region would provide accurate information about childhood cancer burden in the covered population. However, political and financial support is necessary to sustain the registry.</div></div>","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":"6 ","pages":"Article 100420"},"PeriodicalIF":0.0,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145332443","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}
Pub Date : 2025-12-01Epub Date: 2025-07-28DOI: 10.1016/j.ejcped.2025.100318
Michaela Kuhlen , Katharina Karges , Marina Kunstreich , Maximilian Schmutz , Antje Redlich , Rainer Claus , Constantin Lapa
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.
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,
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.
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.
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.
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.
{"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 , Katharina Karges , Marina Kunstreich , Maximilian Schmutz , Antje Redlich , Rainer Claus , 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":"2025-12-01","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}
Pub Date : 2025-12-01Epub Date: 2025-06-26DOI: 10.1016/j.ejcped.2025.100304
Sarah K. Tasian , Judith M. Boer , Monique L. den Boer
This review aims to discuss similarities and dissimilarities between BCR::ABL1-rearranged (Philadelphia chromosome-positive [Ph+]) and ABL-class fusion-driven BCR::ABL1-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 BCR::ABL1-positive and ABL-class ALL, extended knowledge from recent adult clinical trials will also be addressed.
{"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 , Judith M. Boer , 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":"2025-12-01","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}
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