Pediatric Blood & Cancer最新文献

{"title":"What Is the Expected Clearance of Methotrexate? A Therapeutic Drug Monitoring Reference Guide for High-Dose Methotrexate Use in Pediatric Malignancies.","authors":"Zachary L Taylor, Tamara P Miller, Sarah G Board, Ethan A Poweleit, Ashley Chavana, Allison Weisnicht, Austin L Brown, Melanie B Bernhardt, Eric S Schafer, Maureen M O'Brien, Sharon M Castellino, Laura B Ramsey","doi":"10.1002/pbc.31744","DOIUrl":"https://doi.org/10.1002/pbc.31744","url":null,"abstract":"<p><p>High-dose methotrexate dosage and infusion durations differ across treatment protocols for pediatric leukemia, lymphoma, and osteosarcoma. Supportive care interventions are dependent on a patient's elimination of methotrexate (MTX). Therefore, it is important to establish the expected MTX elimination across protocols. Using modeling and simulation of real-world data, we determined the expected MTX concentrations at common time points from the start of infusion (24, 36, 42, 48, 60, and 72 hours) and the time that each patient would reach a typical discharge threshold (0.4, 0.2, 0.15, and 0.10 µM). These data provide a reference for MTX concentrations for common pediatric protocols.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e31744"},"PeriodicalIF":2.4,"publicationDate":"2025-04-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144005216","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Reply to: Comment on Anemia in Young Children and the Association With Socioeconomic Deprivation Indices.","authors":"Lindsay Haacker, Lisa Littner, Mathew Martin, Cole Brokamp, Andrew F Beck, Lori Luchtman-Jones","doi":"10.1002/pbc.31743","DOIUrl":"https://doi.org/10.1002/pbc.31743","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e31743"},"PeriodicalIF":2.4,"publicationDate":"2025-04-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143973440","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Identification of Anticancer Drugs Associated With Cancer Therapy-Related Cardiac Dysfunction in Pediatrics-Analysis of the WHO Pharmacovigilance Database.","authors":"Fabien Labombarda, Jérémie Rouger, Damien Legallois, Charles Dolladille, Joachim Alexandre, Basile Chrétien","doi":"10.1002/pbc.31727","DOIUrl":"https://doi.org/10.1002/pbc.31727","url":null,"abstract":"<p><strong>Aims: </strong>Cardiovascular toxicities associated with anticancer drugs constitute a significant concern for pediatric patients undergoing cancer treatment. Comprehensive data on the burden of cancer therapy-related cardiac dysfunction (CTRCD) are lacking, particularly for this high-risk population susceptible to develop myocardial toxicity. By analyzing VigiBase, the World Health Organization's individual case safety report database, we sought to determine anticancer drugs associated with CTRCD in pediatric patients.</p><p><strong>Methods and results: </strong>To evaluate the association between 249 anticancer drugs labeled by the FDA or EMA and CTRCD reporting, we performed a disproportionality analysis, calculating multivariable adjusted reporting odds ratios (aROR) with their 95% confidence intervals (CI) across four pediatric age classes (0-27 days, 28 days to 23 months, 2-11 years, 12-17 years); ClinicalTrial registration number: NCT05602103. We identified 796 cases of CTRCD associated with at least one anticancer drug in VigiBase. Multivariate analysis across the pediatric age spectrum revealed 16 anticancer drugs significantly associated with CTRCD, of which 10 (63%) are primarily used for hematologic malignancies. Two drugs, a topoisomerase 1 inhibitor (topotecan) and cytotoxic antibiotics (dactinomycin), represented novel associations with CTRCD not previously documented in the literature.</p><p><strong>Conclusion: </strong>Within VigiBase, we pinpointed 16 anticancer drugs significantly associated with CTRCD reporting in pediatrics. Our research validated several associations already thoroughly reported in children (such as with anthracyclines), and unveiled novel signals for systemic exposure to topotecan and dactinomycin. The relevance of these findings, especially considering the frequency of co-administration of agents and the lack of information regarding radiation exposure and chemotherapy dosage, would need to be evaluated in the context of clinical trials that use or have used these agents.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e31727"},"PeriodicalIF":2.4,"publicationDate":"2025-04-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143992998","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Comment on: Anemia in Young Children and the Association With Socioeconomic Deprivation Indices.","authors":"Rongbin Shen, Jingjing Xiang","doi":"10.1002/pbc.31711","DOIUrl":"https://doi.org/10.1002/pbc.31711","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e31711"},"PeriodicalIF":2.4,"publicationDate":"2025-04-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144010284","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Long-Term Outcomes and Quality of Life of Children With Intracranial Ependymoma Treated With Pencil Beam Scanning Proton Therapy.","authors":"Eymeric Le Reun, Ilya Kotov, Dominic Leiser, Alessia Pica, Miriam Vazquez, Gabriele Calaminus, Damien Charles Weber","doi":"10.1002/pbc.31728","DOIUrl":"https://doi.org/10.1002/pbc.31728","url":null,"abstract":"<p><strong>Background: </strong>Ependymoma is a common brain tumor in children and adolescents. Adjuvant radiation therapy improves prognosis but carries potential toxicity risks, particularly for young patients. Proton therapy (PT) offers better conformal treatments and reduces dose exposure compared to traditional photon radiotherapy.</p><p><strong>Procedure: </strong>This study retrospectively analyzed long-term outcomes of children treated with pencil beam scanning (PBS) PT for intracranial ependymomas (EPs) at the Paul Scherrer Institute (PSI) between 2004 and 2022.</p><p><strong>Results: </strong>We identified 119 children, with most having infra-tentorial tumors (70.6%) and anaplastic ependymomas (82.4%). The median PT dose was 59.4 Gy_RBE delivered in 1.8 Gy_RBE/fraction. Follow-up at 5 years showed 70.4% local control, 63.5% progression-free survival (PFS), and 82.2% overall survival (OS). OS was better with upfront than relapse treatment (83% vs. 69.8%; p = 0.024), and complete resection improved both LC (74% vs. 65.1%; p = 0.033) and PFS (67.5% vs. 57.1%; p = 0.049) compared to subtotal resection. No hearing loss was observed with cochlea D_max not exceeding 48 Gy_RBE (10.5% vs. 0%; p = 0.0097), whereas the risk of hormone deficiency was significantly increased with pituitary D_mean above 38 Gy_RBE (33.3% vs. 6.0%; p = 0.00007). Most patients (72.3%) had no late toxicity. Four secondary brain malignancies (3.4%) occurred within a median of 9.3 years after PT (range: 3.7-15). Quality of life 5 years after PT was good in older (>4 years) patients, though proxy-rated social functioning was poorer than the norm group.</p><p><strong>Conclusion: </strong>Intracranial PBS-PT offers excellent tumor control and low late toxicity, and revealed good overall quality of life in children with ependymoma, both by proxy- and self-assessment.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e31728"},"PeriodicalIF":2.4,"publicationDate":"2025-04-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144047631","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Comparison of Letermovir Versus Ganciclovir for Cytomegalovirus Prophylaxis in Pediatric Hematopoietic Stem Cell Transplant Recipients.","authors":"Catherine E Martin, Mira A Kohorst, Asmaa Ferdjallah, Theresa Madigan, Laura M Dinnes, Alexis K Kuhn","doi":"10.1002/pbc.31732","DOIUrl":"https://doi.org/10.1002/pbc.31732","url":null,"abstract":"<p><strong>Background: </strong>Patients at high risk for cytomegalovirus (CMV) infection after allogeneic hematopoietic stem cell transplant (HCT) should receive CMV prophylaxis. Historically, ganciclovir has been the preferred agent. However, its side effect profile, particularly myelosuppression, is undesirable. Letermovir is a newer agent that is not myelosuppressive and has replaced ganciclovir as the preferred agent for CMV prophylaxis in high-risk adult patients.</p><p><strong>Methods: </strong>The primary objective was to compare the incidence of dose modifications or discontinuation of CMV prophylaxis between pediatric patients who received ganciclovir versus letermovir. Secondary objectives included comparing the use of granulocyte colony-stimulating factor (G-CSF) support and the incidence of CMV DNAemia. We reviewed records of all pediatric patients who received an HCT at our institution between 2000 and 2024 and received either ganciclovir/valganciclovir or letermovir for CMV prophylaxis.</p><p><strong>Results: </strong>Between 2000 and 2024, 65 pediatric patients receiving prophylactic ganciclovir, valganciclovir, or letermovir were evaluated. The median age at transplant was 12.2 years. No patients receiving letermovir required dose modification or discontinuation as opposed to 14 (29.2%) patients receiving ganciclovir (p = 0.014). After engraftment, 12 (25%) patients receiving ganciclovir required G-CSF support compared to seven (41.2%) patients receiving letermovir (p = 0.23). In the ganciclovir group, 10 (20.8%) patients experienced CMV infection versus one (5.9%) patient receiving letermovir (p = 0.26).</p><p><strong>Conclusion: </strong>Compared with those receiving letermovir, a significantly higher proportion of patients in the ganciclovir/valganciclovir group experienced dose modifications or discontinuation of CMV prophylaxis due to adverse effects. Letermovir shows great promise as a CMV prophylactic agent in pediatric HCT patients due to its favorable side effect profile.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e31732"},"PeriodicalIF":2.4,"publicationDate":"2025-04-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144037657","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Congenital Langerhans Cell Histiocytosis With Novel KCL1::RAF1 Gene Fusion Identified Through Routine Whole-Genome Sequencing.","authors":"Fiona E Wright, Gemma Barnard, Shivani Bailey, C Elizabeth Hook, Nicholas Coleman, Natasha Stembridge, Rowena Guermech, James Watkins, Jamie Trotman, Patrick Tarpey, Vasanta Nanduri, Matthew J Murray","doi":"10.1002/pbc.31723","DOIUrl":"https://doi.org/10.1002/pbc.31723","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e31723"},"PeriodicalIF":2.4,"publicationDate":"2025-04-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144019699","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"A Rare Case of Recurrent Infant Mediastinal Malignant Rhabdoid Tumor With MSH2 Germline Mutation.","authors":"Qizi Wu, Jian Li, Xuefen Zhao, Lulu He, Jianfeng Zhou, Tao Li, Li Zhou","doi":"10.1002/pbc.31737","DOIUrl":"https://doi.org/10.1002/pbc.31737","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e31737"},"PeriodicalIF":2.4,"publicationDate":"2025-04-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144031471","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Correction to \"Salvage Therapy Efficacy is Modified by Risk Group at Diagnosis in Patients With Relapsed Rhabdomyosarcoma.\"","authors":"","doi":"10.1002/pbc.31690","DOIUrl":"https://doi.org/10.1002/pbc.31690","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e31690"},"PeriodicalIF":2.4,"publicationDate":"2025-04-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144036528","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Correction to \"Fertility Preservation in Pediatric Solid Tumors: A Report From the Children's Oncology Group\".","authors":"","doi":"10.1002/pbc.31685","DOIUrl":"https://doi.org/10.1002/pbc.31685","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e31685"},"PeriodicalIF":2.4,"publicationDate":"2025-04-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143999787","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}