Pediatric Blood & Cancer最新文献

{"title":"Anything to Help.","authors":"Harmony Farner","doi":"10.1002/pbc.32130","DOIUrl":"https://doi.org/10.1002/pbc.32130","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e32130"},"PeriodicalIF":2.3,"publicationDate":"2025-10-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145355705","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":"Building Pediatric Hematology/Oncology Workforce Capacity in Latin America.","authors":"Daniel C Moreira, Federico Antillón-Klussmann, Roy Rosado, Claudia Garrido, Oscar González-Ramella, Violeta Salceda-Rivera, Luiz Fernando Lopes, Monica Cypriano, Fabiana Morosini, Guillermo Chantada, Shirley Montufar, Allyson Andujar, Leeanna Fox Irwin, Jordan Horner, Paola Friedrich, Saman K Hashmi, Carlos Rodríguez-Galindo","doi":"10.1002/pbc.32135","DOIUrl":"https://doi.org/10.1002/pbc.32135","url":null,"abstract":"<p><strong>Background: </strong>This study aimed to assess five pediatric hematology/oncology fellowship programs in Latin America by analyzing the profile of trainees and graduates, and their integration into the workforce.</p><p><strong>Methods: </strong>To train pediatric hematologists/oncologists, the St. Jude Global Academy collaborates with five institutions in Latin America: Unidad Nacional de Oncología Pediátrica (Guatemala), Hospital Civil de Guadalajara (Mexico), Hospital Pereira Rossell (Uruguay), Hospital Oncológico de Barretos (Brazil), and GRAACC (Brazil). Data on the origin of graduates, current job roles, and responsibilities were assessed.</p><p><strong>Results: </strong>Between 2003 and 2024, 110 physicians from 14 countries in Latin America started training in the five programs. Sixty-eight (61.8%) physicians completed training, while 36 (32.7%) are still currently in training. Six trainees (5.5%) withdrew from training prior to completion. Of 59 survey respondents, 47 (79.7%) currently work at a pediatric cancer unit. Thirty-seven (62.7%) graduates started working in pediatric oncology immediately after finishing training. The mean time for the 47 graduates working in pediatric cancer units to find a position was 0.18 years. After entering the pediatric oncology workforce, the mean time dedicated to pediatric oncology was 52.0%, with 15.5% spent on benign hematology, 12.8% on other clinical areas, 9.2% on administrative tasks, 6.9% on teaching, and 4.2% on research. Twenty-six (44%) graduates currently have a leadership role at their institutions.</p><p><strong>Conclusion: </strong>The five programs have trained Latin American specialists to care for children with cancer, with high integration into the pediatric oncology workforce. Graduates now contribute across the region, though further analyses are needed to define a comprehensive regional workforce strategy.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e32135"},"PeriodicalIF":2.3,"publicationDate":"2025-10-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145346390","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":"Infectious Complications During Early Treatment of Childhood Acute Lymphoblastic Leukemia-A Comparison Between the ALLTogether and NOPHO ALL-2008 Protocols.","authors":"Hannah van Bunningen, Johannes Fermér, Arja Harila, Susanna Ranta, Isabella Donnér, Anne Wretman, Hartmut Vogt, Anna Sällfors Holmqvist, Anders Valind, Ludvig Henriksson, Jonas Abrahamsson, Magnus Borssén, Anna Nilsson, Mats Heyman","doi":"10.1002/pbc.32120","DOIUrl":"https://doi.org/10.1002/pbc.32120","url":null,"abstract":"<p><strong>Introduction: </strong>Infection remains the most common treatment-related toxicity of childhood ALL, emphasizing the need to identify patients at risk and to tailor treatment strategies accordingly.</p><p><strong>Aims: </strong>The primary aim was to compare infectious toxicity during early treatment for childhood ALL following the ALLTogether and NOPHO ALL-2008 (ALL-2008) protocols, and second, to identify risk factors for infectious toxicity.</p><p><strong>Methods: </strong>A national retrospective matched cohort study was conducted, including 345 patients aged 1-17 years diagnosed with ALL and treated in Sweden according to the ALLTogether or ALL-2008 protocols. Nonparametric tests were used to compare infectious outcomes between protocols, and regression modeling was used to identify risk factors of the infectious outcomes.</p><p><strong>Results: </strong>Treatment following ALL-2008 showed higher infectious toxicity during induction, whereas treatment following ALLTogether showed increased infectious toxicity during consolidation 1. Overall, treatment according to ALL-2008 was associated with a higher incidence of infections. Anthracycline use and young age (1-9 years) were associated with both higher infectious incidence and more severe infectious complications. Dexamethasone was associated with both lower incidence and lower severity of infectious complications as compared to prednisone.</p><p><strong>Conclusions: </strong>A notable shift in the timing of infectious toxicity was observed between the two treatment protocols. Risk factors for infectious toxicity during early treatment include anthracycline use and young age. Dexamethasone as an induction steroid was associated with lower infectious burden, although its effect is difficult to isolate from the simultaneous anthracycline effect. The findings suggest that treatment composition plays a central role in determining both the extent and timing of infectious complications.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e32120"},"PeriodicalIF":2.3,"publicationDate":"2025-10-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145346404","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":"Emicizumab in Previously Untreated Patients and Minimally Treated Patients With Hemophilia A: A Comparative Study Between Two International Cohorts.","authors":"Sarina Levy-Mendelovich, Tlalit Barhod, Ivan Budnik, Jonathan Lancashire, Jesal Patel, Assaf Arie Barg, Einat Avishai, Rima Dardik, Tami Brutman Barazani, Tami Livnat, Jayashree Motwani, Gili Kenet","doi":"10.1002/pbc.32118","DOIUrl":"https://doi.org/10.1002/pbc.32118","url":null,"abstract":"<p><strong>Background: </strong>Hemophilia A (HA) is a rare bleeding disorder caused by coagulation factor VIII (FVIII) deficiency. Prophylactic FVIII replacement therapy is essential for preventing bleeds, but it carries a risk of inhibitor development, especially in previously untreated and minimally treated patients (PUPs and MTPs, respectively). Emicizumab, a bispecific monoclonal antibody that mimics FVIII function, offers a promising alternative for HA prophylaxis due to its subcutaneous administration and favorable safety profile.</p><p><strong>Methods: </strong>This study evaluated the real-world safety and efficacy of emicizumab prophylaxis in two international hemophilia treatment centers (HTC). The first HTC included a cohort of 22 MTPs with severe HA (FVIII <1%) and fewer than five prior FVIII exposure days, while the second HTC enrolled 19 PUPs with severe HA.</p><p><strong>Results: </strong>The median age at emicizumab prophylaxis initiation was 5 months for the MTPs and 8 months for the PUPs. Patients were followed for a median of 27 and 29 months, respectively. The median time to first bleed was 13 months for MTPs, with a significantly longer time to first bleed noted in the PUPs cohort. Safety outcomes were favorable, with neither intracranial hemorrhage nor anti-emicizumab antibodies reported. Four patients, 18% of all MTPs, developed FVIII inhibitors, all associated with high-risk genetic mutations and prior bleeding events.</p><p><strong>Conclusions: </strong>Our findings support the early use of emicizumab as a safe and effective prophylactic strategy in infants with severe HA. However, the observed inhibitor rate underscores the need for ongoing monitoring and research to optimize care, particularly in vulnerable populations.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e32118"},"PeriodicalIF":2.3,"publicationDate":"2025-10-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145346388","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":"Reflections on Sperm Banking Decisions and Support Needs Among Adolescent Males and Their Caregivers 1 Year After Cancer Diagnosis: A Qualitative Study.","authors":"Tanvi Karkare, Charleen I Roche, Megan M Griffith, Gwendolyn P Quinn, Sarah H O'Brien, Charis J Stanek, James L Klosky, Zachary Colton, Anthony Audino, Nicholas Yeager, Stacy Whiteside, Jennifer English, Cynthia A Gerhardt, Leena Nahata","doi":"10.1002/pbc.32128","DOIUrl":"https://doi.org/10.1002/pbc.32128","url":null,"abstract":"<p><strong>Background/objectives: </strong>Approximately half of male cancer survivors experience infertility following cancer treatment, which can lead to psychosocial distress. The aim of this study was to identify support needs and reflections on the decision-making process related to sperm banking among adolescent male cancer survivors and their caregivers at 1 year post-diagnosis.</p><p><strong>Methods: </strong>As part of a randomized controlled trial testing a family-centered sperm banking decision-making intervention, males diagnosed with cancer (12-25 years old) and their caregivers completed semi-structured interviews 1 year post-diagnosis. Thematic analysis was conducted by three independent coders (κ = 0.80) and focused on two interview questions: (1) Is there anything you wish you would have known or done before making the [sperm banking] decision? and (2) What information or support do you think is needed regarding your/your son's future fertility goals?</p><p><strong>Results: </strong>Qualitative interviews with adolescents (n = 20) and caregivers (n = 18) revealed three primary themes: (1) satisfaction with information received at diagnosis, but retrospective desire for more decision-making time; (2) current desire for additional fertility-related support; (3) potential need for future fertility-related support.</p><p><strong>Conclusion: </strong>Despite satisfaction with the oncofertility consultation at diagnosis, clinical teams should prioritize fertility education moving forward and allow additional time for sperm banking decision-making (when possible) at diagnosis. Counseling gaps can lead to uncertainty, unplanned pregnancies, and adverse mental health outcomes. Thus, it is important to revisit issues surrounding fertility and family planning after treatment, particularly among adolescents transitioning to adulthood.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e32128"},"PeriodicalIF":2.3,"publicationDate":"2025-10-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145346489","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":"Leukoencephalopathy, a Frequent Complication After High-Dose Methotrexate in Treatment of Osteosarcoma.","authors":"Sandra Raimbault, Jean Louis Amegnizin, Audrey Longaud, Brice Fresneau, Nathalie Gaspar, Pablo Berlanga, Virginie Kieffer, Anne Maraval, Lucy Metayer, Christelle Dufour, Dominique Valteau-Couanet, Pierre Brugieres, Blanche Bapst, Laurence Brugieres","doi":"10.1002/pbc.32104","DOIUrl":"https://doi.org/10.1002/pbc.32104","url":null,"abstract":"<p><strong>Background: </strong>Methotrexate-associated leukoencephalopathy is poorly documented in osteosarcoma. Since 2007, our institution has monitored osteosarcoma patients with sequential magnetic resonance imaging (MRI) and neuropsychological evaluations during treatment and follow-up.</p><p><strong>Methods: </strong>We analyzed data from consecutive osteosarcoma patients younger than 25 years enrolled at Gustave Roussy in the OS2006 study (2007-2015) and treated with high-dose methotrexate (MTX) and etoposide-ifosfamide. Eligible patients received four or more MTX courses and at least one brain MRI. MTX-related neurotoxicity was defined as neurological symptoms attributed to MTX after excluding other causes.</p><p><strong>Results: </strong>MTX-related neurotoxicity occurred in seven (13%) of 53 eligible patients. Additionally, 12 had severe depression, nine reported attention/memory deficits, and 25 had headaches during MTX infusion. Acute symptoms resolved in all but one. Forty-nine patients had an MRI during treatment, and 47 after. Leukoencephalopathy was found in 44 (83%): Grade 1 in six, Grade 2 in 15, and Grade 3 in 23. Grade 2-3 leukoencephalopathy was more frequent in patients with severe depression (p = 0.02) and those receiving more than 12 MTX courses (p = 0.03); no association was found with age, sex, MTX-related neurotoxicity, or cognitive complaints. Among 24 patients with MRI ≥3 years post-treatment, 20 still showed leukoencephalopathy (12 Grade 1, eight Grade 2). Neurocognitive evaluations showed IQ scores consistent with norms, except for lower processing speed, which improved post-treatment. At last follow-up (median 8.5 years), most patients had integrated into school or work.</p><p><strong>Conclusion: </strong>MTX-associated leukoencephalopathy is frequent in osteosarcoma, even in asymptomatic patients, and often persists after treatment. Serious neurocognitive sequelae appear uncommon, but long-term cognitive monitoring is essential to detect subtle deficits.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e32104"},"PeriodicalIF":2.3,"publicationDate":"2025-10-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145346496","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":"Aggressive Systemic Mastocytosis Related to Germline p.D816V KIT Mutation.","authors":"Antonia Kiwit, Julian Trah, Amelie T van der Ven, Ilske Oschlies, Nikolas von Bubnoff, Carsten Müller, Ingo Müller, Martin Blohm, Philipp Deindl, Sofia Apostolidou, Dominique Singer, Kai Lehmberg","doi":"10.1002/pbc.32129","DOIUrl":"https://doi.org/10.1002/pbc.32129","url":null,"abstract":"<p><p>Systemic mastocytosis (SM) in adults is, in most cases, caused by a somatic mutation leading to p.D816V in KIT. We report the first proof of this variant as a heterozygous de novo germline mutation in a female infant with severe neonatal SM. The girl presented prenatally with hepatosplenomegaly and postnatally with diffuse cutaneous lesions and organ dysfunction. Histopathologic specimens displayed infiltration with atypical mastocytes. Molecular analyses confirmed the KIT mutation in multiple non-infiltrated tissues, demonstrating the germline nature of the mutation. Despite targeted treatment with pharmacokinetically monitored midostaurin and adjunct therapies, the disease progressed rapidly, resulting in fatal multiorgan failure.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e32129"},"PeriodicalIF":2.3,"publicationDate":"2025-10-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145346357","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":"Efanesoctocog Alfa Prophylaxis in a Patient With Combined Mild Hemophilia A and Type 1 von Willebrand Disease.","authors":"Destiny Uwaezuoke, An Pham, Jessica Garcia","doi":"10.1002/pbc.32125","DOIUrl":"https://doi.org/10.1002/pbc.32125","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e32125"},"PeriodicalIF":2.3,"publicationDate":"2025-10-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145346411","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: Perceptions of the Stressful Job Search for Pediatric Hematology/Oncology Fellows.","authors":"Meera Rayar, Marta Wilejto, Adam Fleming, Lisa Jacques, Sylvia Cheng, Kathy Brodeur-Robb","doi":"10.1002/pbc.32099","DOIUrl":"https://doi.org/10.1002/pbc.32099","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e32099"},"PeriodicalIF":2.3,"publicationDate":"2025-10-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145337440","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":"Efficacy and Safety of Trametinib Therapy for Complex Lymphatic Malformations.","authors":"Yulang Xu, Jiangyuan Zhou, Yuru Lan, Yi Ji","doi":"10.1002/pbc.32119","DOIUrl":"https://doi.org/10.1002/pbc.32119","url":null,"abstract":"<p><p>This study evaluated trametinib in five pediatric patients (median age 9 years) with complex lymphatic malformations, such as kaposiform lymphangiomatosis (KLA) and generalized lymphatic anomaly (GLA). All showed significant clinical improvement: coagulopathy resolved (platelet increase 54%-362%, D-dimer decrease > 91%), 80% achieved partial response radiographically, and toxicity was minimal (one Grade 2 rash). NRAS proto-oncogene mutations were detected in 80% of cases, but clinical benefit occurred without mutation. Trametinib is an effective, safe, first-line therapy for KLA/GLA.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e32119"},"PeriodicalIF":2.3,"publicationDate":"2025-10-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145337417","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}