EJC paediatric oncology最新文献

{"title":"HAPLOIDENTICAL HEMATOPOIETIC STEM CELL TRANSPLANTATION WITH POST-TRANSPLANT CYCLOPHOSPHAMIDE FOR CHILDREN WITH JUVENILE MYELOMONOCYTIC LEUKEMIA","authors":"Anita Frisanco Oliveira , Rafaela Ferreira , Luiz Fernando Mantovani , Angela Venancio , Rafael Balceiro , Luiz Fernando Lopes , Neysimelia Villela","doi":"10.1016/j.ejcped.2025.100384","DOIUrl":"10.1016/j.ejcped.2025.100384","url":null,"abstract":"","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":"6 ","pages":"Article 100384"},"PeriodicalIF":0.0,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145428570","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":"RISK-ADAPTED THERAPY FOR JUVENILE MYELOMONOCYTIC LEUKEMIA: A MULTICENTER, PROSPECTIVE STUDY IN CHINA","authors":"Xia Qin , Maoquan Qin , Shaoyan Hu , Jie Yu , Xiaojun Xu , Lijun Qu , Yiping Zhu , Sen Chen , Ruijuan Sun , Ningling Wang , Hao Xiong , Rong Liu , Ansheng Liu , Huihong Dou , Yanna Mao , Benshan Zhang , Xiangling He , Dao Wang , Yunyan He , Yongjun Fang , Chen Jing","doi":"10.1016/j.ejcped.2025.100390","DOIUrl":"10.1016/j.ejcped.2025.100390","url":null,"abstract":"","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":"6 ","pages":"Article 100390"},"PeriodicalIF":0.0,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145428576","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":"MOLECULAR SURVEILLANCE IN BONE MARROW FAILURE: PREDICTIVE VALUE OF CLONAL HEMATOPOIESIS","authors":"Nathan Gray, Enrico Attardi, Michelle Boals, Sara Lewis, Passant Shaker, Kelsey Ray, Jessica Uhrich, Melvanique Hale, Alyssa Kennedy, Marcin W. Wlodarski","doi":"10.1016/j.ejcped.2025.100329","DOIUrl":"10.1016/j.ejcped.2025.100329","url":null,"abstract":"","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":"6 ","pages":"Article 100329"},"PeriodicalIF":0.0,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145428505","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":"FUNCTIONAL ANALYSIS OF NON-CANONICAL FANCJ VARIANTS IN FANCONI ANEMIA","authors":"Anja Droste , Anna Repczynska , Isabell Popp , Britta Fiebig , Julia Vodopiutz , Andreas-Robert Janecke , Detlev Schindler , Jost Knaup , Regina Hark , Renata Kulka , Alexandra Sobeck , Reinhard Kalb","doi":"10.1016/j.ejcped.2025.100337","DOIUrl":"10.1016/j.ejcped.2025.100337","url":null,"abstract":"","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":"6 ","pages":"Article 100337"},"PeriodicalIF":0.0,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145428511","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":"GATA2 GENE HAPLOINSUFFICIENCY: A CLINICAL AND LABORATORY CHARACTERIZATION OF BRAZILIAN PATIENTS REGISTERED IN GCB-SMD-PED","authors":"Anita Frisanco Oliveira , Guilherme Rodrigues , Neysimelia Villela , Samia Fahria , Glaucia Regina Murra , Rafael Balceiro , Eduardo Caetano Silva , Luiz Fernando Lopes , Juliana Gaspar","doi":"10.1016/j.ejcped.2025.100371","DOIUrl":"10.1016/j.ejcped.2025.100371","url":null,"abstract":"","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":"6 ","pages":"Article 100371"},"PeriodicalIF":0.0,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145428622","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":"CHARACTERIZATION OF MURINE STEM AND PROGENITOR CELLS HARBORING ONCOGENIC PTPTN11 OR KRAS MUTATIONS","authors":"Hui Xiao , Shuhan Jin , Jun Wang , Jovana Rajak , Naile Koleci , Sheila Bohler , Miriam Erlacher","doi":"10.1016/j.ejcped.2025.100413","DOIUrl":"10.1016/j.ejcped.2025.100413","url":null,"abstract":"","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":"6 ","pages":"Article 100413"},"PeriodicalIF":0.0,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145428631","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":"A CASE OF SEVERE CONGENITAL THROMBOCYTOPENIA WITH HETEROZYGOUS PATHOGENIC NBEAL2 MUTATION","authors":"Michelle Boals, Passant Shaker, Lili Kotmayer, Nathan Gray, Kelsey Ray, Marcin Wlodarski, Alyssa L. Kennedy","doi":"10.1016/j.ejcped.2025.100398","DOIUrl":"10.1016/j.ejcped.2025.100398","url":null,"abstract":"","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":"6 ","pages":"Article 100398"},"PeriodicalIF":0.0,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145428615","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":"GENETIC RE-EVALUATION OF SDS-LIKE AND NEUTROPENIA CONDITIONS","authors":"Helen D. Reed , Sarah Brnich , Jonas Gudera , Stephanie DiTroia , Anne O’Donnell-Luria , Meng-Ju Li , Hermann Yang , Karyn Brundige , Sara Loveless , Leah Cheng , Felicia Andresen , Mark Fleming , Kasiani Myers , Akiko Shimamura","doi":"10.1016/j.ejcped.2025.100400","DOIUrl":"10.1016/j.ejcped.2025.100400","url":null,"abstract":"","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":"6 ","pages":"Article 100400"},"PeriodicalIF":0.0,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145428617","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":"Recommended phase 2 dose for abemaciclib in combination with irinotecan and temozolomide in pediatric and young adult patients with relapsed/refractory solid tumors: Results from JPCS study (Part A)","authors":"Antonio Juan Ribelles ,&nbsp;Lucas Moreno ,&nbsp;Chitose Ogawa ,&nbsp;Anne Thorwarth ,&nbsp;Antonio Ruggiero ,&nbsp;Celine Pitou ,&nbsp;Yanhong Zhou ,&nbsp;Molly C. Hardebeck ,&nbsp;Holly Knoderer ,&nbsp;Alvaro Lassaletta","doi":"10.1016/j.ejcped.2025.100477","DOIUrl":"10.1016/j.ejcped.2025.100477","url":null,"abstract":"<div><h3>Background</h3><div>Abemaciclib is a cyclin-dependent kinase (CDK) 4/6 inhibitor approved for breast cancer. The CDK4/6 pathway is essential for cell cycle progression. Pediatric cancers often harbor aberrations in this pathway, potentially rendering sensitivity to CDK4/6 inhibition.</div></div><div><h3>Procedure</h3><div>In this multicenter, open-label, phase 1 trial (JPCS; NCT04238819), children and young adults with relapsed/refractory solid tumors were nonrandomly assigned to standard-dose irinotecan and temozolomide with abemaciclib in a 3 + 3 escalation design with 4 planned dose levels (55, 70 [starting dose], 90, and 115 mg/m² twice daily [BID]). An expansion phase followed. The primary objective was recommended phase 2 dose (RP2D) determination for abemaciclib based on safety and pharmacokinetics. A secondary endpoint was antitumor activity.</div></div><div><h3>Results</h3><div>Twenty patients enrolled (median age: 12 [7−17] years) with central nervous system tumors as the most frequent diagnosis. The abemaciclib RP2D was 55 mg/m² BID. One of 12 dose-limiting toxicity (DLT)-evaluable patients experienced a DLT (thrombocytopenia) at the RP2D. At the RP2D, the most frequent treatment-related adverse events (TRAEs) were diarrhea (92 %), neutropenia (77 %), thrombocytopenia (62 %), and anemia (62 %), and the most common grade 3/4 TRAEs were neutropenia (62 %), thrombocytopenia, leukopenia, and anemia (each 31 %). At the RP2D, patients received a median of 4 (2−35) cycles of abemaciclib, and best overall response included 1 (5 %) complete response and 6 (46 %) stable disease. Abemaciclib plasma concentrations were within the range associated with efficacy in adults.</div></div><div><h3>Conclusions</h3><div>Abemaciclib 55 mg/m² BID with irinotecan and temozolomide was tolerable and showed potential antitumor activity in children and young adults with relapsed/refractory malignant solid tumors.</div></div>","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":"6 ","pages":"Article 100477"},"PeriodicalIF":0.0,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145473775","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":"Transition needs and preferences identified during peer visits to three European best-practice CAYA cancer Long-Term Follow-Up care centres: An EU-CAYAS-NET project","authors":"Jikke Wams ,&nbsp;Jaap den Hartogh ,&nbsp;Cherine Mathot ,&nbsp;Jeroen te Dorsthorst ,&nbsp;Marie-Therese Gubi ,&nbsp;Kerstin Krottendorfer ,&nbsp;Ulrike Leiss ,&nbsp;Esther Lasheras Soria ,&nbsp;Begonya Nafria Escalera ,&nbsp;Urška Košir ,&nbsp;Katie Rizvi ,&nbsp;Carina Schneider ,&nbsp;Barbara Brunmair ,&nbsp;Ismay de Beijer ,&nbsp;Renée L. Mulder ,&nbsp;Leontien C.M. Kremer ,&nbsp;Helena J.H. van der Pal ,&nbsp;on behalf of the EU-CAYAS-NET consortium","doi":"10.1016/j.ejcped.2025.100479","DOIUrl":"10.1016/j.ejcped.2025.100479","url":null,"abstract":"<div><h3>Background</h3><div>A successful transition from child-centred to adult-oriented healthcare for survivors of childhood and adolescent cancer is essential to help them engage in lifelong surveillance in survivorship care. Information on their needs and preferences for the transition process is needed to identify key components for a successful transition.</div></div><div><h3>Objective</h3><div>To describe the transition needs and preferences of young cancer survivors, their parents, and healthcare providers.</div></div><div><h3>Methods</h3><div>Three in-person peer visits to best-practice long-term follow-up care centres in Europe were conducted. In these visits, the needs and preferences of young cancer survivors, their parents, and healthcare providers were collected through interactive sessions and structured documentation. The outcomes were analysed inductively using thematic analysis, resulting in a list of unique needs and preferences.</div></div><div><h3>Results</h3><div>Sixty-five participants, including 32 survivors, six parents, 21 healthcare providers, and six researchers, shared 188 needs and preferences, leading to the identification of 58 distinct transition needs and preferences. Participants stressed the need for structured, patient-centred transition processes with clear coordination and planning, following a holistic approach. Key support needs include lifestyle, psychosocial, and employment assistance. Improved access to reliable information, tailored education, and readiness-based transfers were emphasised.</div></div><div><h3>Conclusion</h3><div>Our findings highlight the need for personalised and ongoing coordination and support for survivors and their families during transition. Strengthening informational and educational transition resources are crucial to address gaps experienced by patients and caregivers. These insights will be implemented into the evidence-based European transition guideline from the EU-CAYAS-NET project.</div></div>","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":"6 ","pages":"Article 100479"},"PeriodicalIF":0.0,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145623419","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}