EJC paediatric oncology最新文献

{"title":"ASSESSMENT OF GENETIC TESTING FOLLOWING CURRENT COMPREHENSIVE CLINICAL DIAGNOSTIC EVALUATION FOR PEDIATRIC SEVERE APLASTIC ANEMIA","authors":"Helen D. Reed , Jill L. O. de Jong , Melissa Gaviria , Ashwin Koppayi , Youssef Ahmed , Ian Atkinson , Margret Joos , Maggie Malsch , Matthew McClung , Peter Nicholas , Mancy Shah , Erin E. Sullivan , Hongbo Xie , Silvia Zavarella , Yu Zhou , Barry Zorman , Lucy A. Godley , Taizo A. Nakano , Timothy S. Olson , Alison A. Bertuch , Akiko Shimamura","doi":"10.1016/j.ejcped.2025.100328","DOIUrl":"10.1016/j.ejcped.2025.100328","url":null,"abstract":"","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":"6 ","pages":"Article 100328"},"PeriodicalIF":0.0,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145428504","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":"IN-VIVO MOUSE MODEL OF ACUTE MYELOID LEUKEMIA DEVELOPMENT IN CONGENITAL NEUTROPENIA","authors":"Jérémy Haaf, Malte Ritter, Patricia Arreba-Tutusaus, Siarhei Kandabarau, Maksim Klimiankou, Julia Skokowa","doi":"10.1016/j.ejcped.2025.100333","DOIUrl":"10.1016/j.ejcped.2025.100333","url":null,"abstract":"","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":"6 ","pages":"Article 100333"},"PeriodicalIF":0.0,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145428509","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":"MITOTIC DEFECTS IMPAIRED CD34+ PROLIFERATION IN GATA2 DEFICIENCY","authors":"Maria Magallon-Mosella , Damia Romero-Moya , Cristina Calvo , Joan Pera , Eric Torralba-Sales , Miriam Erlacher , Òscar Molina , Alessandra Giorgetti","doi":"10.1016/j.ejcped.2025.100335","DOIUrl":"10.1016/j.ejcped.2025.100335","url":null,"abstract":"","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":"6 ","pages":"Article 100335"},"PeriodicalIF":0.0,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145428525","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":"BH3-MIMETIC TREATMENT REVEALS SUBTYPE-SPECIFIC BCL-2 PROTEIN DEPENDENCY IN PEDIATRIC MDS WITH EXCESS BLASTS","authors":"Sheila Bohler , Ayami Yoshimi Nöllke , Nastassja Scheidegger , Ben Haladik , Beat Bornhauser , Michael N. Dworzak , Kaan Boztug , Markus Schmugge , Charlotte M. Niemeyer , Miriam Erlacher","doi":"10.1016/j.ejcped.2025.100355","DOIUrl":"10.1016/j.ejcped.2025.100355","url":null,"abstract":"","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":"6 ","pages":"Article 100355"},"PeriodicalIF":0.0,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145428528","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":"STRATUS PRIMETM A GMP COMPLIANT PROCESS TO CREATE HIGH NUMBERS OF HLA COMPATIBLE DEFINITIVE HSPCS FROM IPSCS USING PIEZO1 AGONISTS","authors":"Michael P. Cooke , Aayudh Das , Gurinder Singh , Zhi-Jian Liu , Qiao Lin , Sita Karan Patel , Jack Li , Vijaya G. Tirunagaru","doi":"10.1016/j.ejcped.2025.100356","DOIUrl":"10.1016/j.ejcped.2025.100356","url":null,"abstract":"","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":"6 ","pages":"Article 100356"},"PeriodicalIF":0.0,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145428529","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":"MACROPHAGE POLARIZATION AND CD47 BLOCKADE IN PTPN11-MUTATED JUVENILE MYELOMONOCYTIC LEUKEMIA","authors":"Jun Wang , Jovana Rajak , Hui Xiao , Naile Koleci , Anna Lena Stippel , Ke Meng , Niels Anton Wehner , Sheila Bohler , Charlotte Niemeyer , Bertram Bengsch , Miriam Erlacher","doi":"10.1016/j.ejcped.2025.100409","DOIUrl":"10.1016/j.ejcped.2025.100409","url":null,"abstract":"","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":"6 ","pages":"Article 100409"},"PeriodicalIF":0.0,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145428627","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 PROFILING OF JUVENILE MYELOMONOCYTIC LEUKEMIA: CHARACTERIZATION OF PATIENTS WITH MULTIPLE ONCOGENIC VARIANTS USING A TARGETED NGS PANEL","authors":"Sâmia Frahia Bento da Silva , Juliana Costa Gaspar , Neysimelia Costa Villela , Rafael Balceiro , Rui Manuel Reis , Luiz Fernando Lopes , Anita Frisanco de Oliveira","doi":"10.1016/j.ejcped.2025.100416","DOIUrl":"10.1016/j.ejcped.2025.100416","url":null,"abstract":"","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":"6 ","pages":"Article 100416"},"PeriodicalIF":0.0,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145428634","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":"CSF3R MUTATIONS IN CONGENITAL NEUTROPENIA, A LONG STORY: DATA FROM THE ITALIAN REGISTRY","authors":"Francesco Pegoraro , Agnese Roveta , Laura Giunti , Maria Fay Cortella , Elena Mastrodicasa , Giorgio Costagliola , Baldassarre Martire , Francesco Saettini , Maria Carla Giarratana , Antonio Marzollo , Angelica Barone , Giovanna Giagnuolo , Marta Pillon , Carlo Dufour , Marinella Veltroni , Francesca Fioredda","doi":"10.1016/j.ejcped.2025.100399","DOIUrl":"10.1016/j.ejcped.2025.100399","url":null,"abstract":"","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":"6 ","pages":"Article 100399"},"PeriodicalIF":0.0,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145428616","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":"2025-12-01","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":"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}