Pub Date : 2025-11-04DOI: 10.1016/j.ejcped.2025.100398
Michelle Boals, Passant Shaker, Lili Kotmayer, Nathan Gray, Kelsey Ray, Marcin Wlodarski, Alyssa L. Kennedy
{"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":"2025-11-04","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}
Pub Date : 2025-11-04DOI: 10.1016/j.ejcped.2025.100400
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
{"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":"2025-11-04","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}
Pub Date : 2025-11-04DOI: 10.1016/j.ejcped.2025.100477
Antonio Juan Ribelles , Lucas Moreno , Chitose Ogawa , Anne Thorwarth , Antonio Ruggiero , Celine Pitou , Yanhong Zhou , Molly C. Hardebeck , Holly Knoderer , Alvaro Lassaletta
Background
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.
Procedure
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.
Results
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.
Conclusions
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.
{"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 , Lucas Moreno , Chitose Ogawa , Anne Thorwarth , Antonio Ruggiero , Celine Pitou , Yanhong Zhou , Molly C. Hardebeck , Holly Knoderer , 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/m<sup>2</sup> 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/m<sup>2</sup> 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/m<sup>2</sup> 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":"2025-11-04","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}
Pub Date : 2025-11-04DOI: 10.1016/j.ejcped.2025.100327
Brigitte Strahm , Ayami Yoshimi , Ivana Bodova , Jochen Buechner , Albert Catala , Valérie de Haas , Barbara De Moerloose , Michael Dworzak , Henrik Hasle , Krisztian Kallay , Franco Locatelli , Riccardo Masetti , Peter Noellke , Markus Schmugge , Owen Smith , Jan Stary , Dominik Turkiewicz , Marek Ussovicz , Luca Vinci , Miriam Erlacher , Charlotte Niemeyer
{"title":"IMPROVED ENGRAFTMENT FOLLOWING A TREOSULFAN-FLUDARABINE CONDITIONING REGIMEN COMPARED TO THIOTEPA-FLUDARABINE IN PATIENTS WITH REFRACTORY CYTOPENIA OF CHILDHOOD","authors":"Brigitte Strahm , Ayami Yoshimi , Ivana Bodova , Jochen Buechner , Albert Catala , Valérie de Haas , Barbara De Moerloose , Michael Dworzak , Henrik Hasle , Krisztian Kallay , Franco Locatelli , Riccardo Masetti , Peter Noellke , Markus Schmugge , Owen Smith , Jan Stary , Dominik Turkiewicz , Marek Ussovicz , Luca Vinci , Miriam Erlacher , Charlotte Niemeyer","doi":"10.1016/j.ejcped.2025.100327","DOIUrl":"10.1016/j.ejcped.2025.100327","url":null,"abstract":"","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":"6 ","pages":"Article 100327"},"PeriodicalIF":0.0,"publicationDate":"2025-11-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145428308","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 : 2025-11-04DOI: 10.1016/j.ejcped.2025.100362
Daniel Dulla , Michaela Reiterova , Michael Svaton , Martina Vaskova , Andrea Rennerova , Veronika Kanderova , Vít Campr , Tomáš Votava , Pavel Timr , Dagmar Pospisilova , Ondřej Zapletal , Tomáš Kuhn , Iveta Janotova , Ondřej Hrusak , Jan Trka , Lucie Sramkova , Jan Stary , Eva Fronkova
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