Pediatric Blood & Cancer最新文献

{"title":"Moyamoya Syndrome in Patients With Down Syndrome and Acute Lymphoblastic Leukemia.","authors":"Robert Lisac, Ryan J Summers, D John Bergsagel, Frank G Keller, Bryan Philbrook, David Wrubel, Gary Woods, Sunil S Raikar","doi":"10.1002/pbc.32045","DOIUrl":"https://doi.org/10.1002/pbc.32045","url":null,"abstract":"<p><p>Moyamoya syndrome (MMS) is a chronic vasculopathy characterized by progressive stenosis of intracerebral arteries, leading to an increased risk of stroke. Children with Down syndrome (DS) are at an increased risk of co-occurring medical conditions, including MMS and leukemia. We report four patients with the triad of DS, MMS, and acute lymphoblastic leukemia (ALL). Two patients had a diagnosis of MMS prior to developing ALL, whereas two were diagnosed with MMS during ALL therapy. This series highlights the need to remain cognizant of MMS in patients with DS and ALL, particularly in those with acute neurologic changes.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e32045"},"PeriodicalIF":2.3,"publicationDate":"2025-09-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145016020","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":"Multiple Hematopoietic Stem Cell Transplantations in Pediatric Acute Myeloid Leukemia: Prognostic Significance of Remission and Severe Sinusoidal Obstruction Syndrome.","authors":"Hideki Yoshida, Shotaro Iwamoto, Akira Shimada, Takako Miyamura, Kiminori Terui, Hirozumi Sano, Akihiro Tamura, Yoko Mizoguchi, Kyoko Moritani, Toshihiko Imamura, Yuko Osugi, Asahito Hama","doi":"10.1002/pbc.32036","DOIUrl":"https://doi.org/10.1002/pbc.32036","url":null,"abstract":"<p><strong>Background: </strong>Relapsed or refractory cases of pediatric acute myeloid leukemia (AML) have poor outcomes despite advancements in chemotherapy and hematopoietic stem cell transplantation (HSCT). While a second HSCT is often a salvage option, its outcomes vary widely, and prognostic factors remain unclear.</p><p><strong>Objectives: </strong>This study aimed to evaluate outcomes and identify prognostic factors in pediatric patients with AML who underwent multiple HSCTs.</p><p><strong>Methods: </strong>We conducted a retrospective, multicenter study of 49 pediatric patients with AML who underwent two or more HSCTs at 18 Japan Association of Childhood Leukemia Study institutions during 2000-2019. Clinical data on patient demographics, disease status, transplant characteristics, and complications were collected. The primary endpoint was 5-year overall survival (OS) after the second HSCT. Kaplan-Meier and multivariate Cox regression analyses were performed.</p><p><strong>Results: </strong>Of the 49 patients, three and 46 underwent three and two HSCTs, respectively. Among these 46 patients, 5-year OS after the second HSCT was 28.3%. Achieving hematological complete remission (CR) before the second HSCT was associated with significantly better outcomes (5-year OS: 45.0% vs. 0%, p < 0.01). Severe sinusoidal obstruction syndrome (SOS) after the second HSCT was a strong predictor of transplant-related mortality (5-year OS: 0% in severe SOS cases, p < 0.01). Patient age, donor type, conditioning regimen, and acute/chronic graft-versus-host disease were not significantly associated with survival in multivariate analysis.</p><p><strong>Conclusions: </strong>Achieving CR before the second HSCT is critical for long-term survival, while severe SOS significantly worsens prognosis. Optimized pre-transplant strategies to reduce SOS risk are essential to improve outcomes of pediatric patients with AML who undergo multiple HSCTs.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e32036"},"PeriodicalIF":2.3,"publicationDate":"2025-09-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145016074","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":"Do Surveillance Tests for Relapse Improve Survival After Chemotherapy for Paediatric Acute Leukaemia? A Systematic Review.","authors":"Connor Evans, Hollie Melton, Yiwen Liu, Rebecca E Ling, John Moppett, Bob Phillips, Brenda Gibson, Helen Fulbright, Jessica E Morgan","doi":"10.1002/pbc.31973","DOIUrl":"https://doi.org/10.1002/pbc.31973","url":null,"abstract":"<p><p>Acute leukaemias are the commonest cancers in children and young people (CYP). Off-treatment surveillance is assumed to improve relapse detection, but whether this affects subsequent survival and quality of life is unclear. This systematic review searched 13 databases and two trial registries in December 2022. Studies after 1990 investigating post-treatment surveillance in CYP ≤17 years old with acute leukaemias were included. Extracted outcomes included overall and event-free survivals, surveillance programme performance and cost-effectiveness. Screening, data extraction and quality assessment (using the Quality in Prognostic Studies tool) were conducted in duplicate. Data were narratively synthesised. Of 7899 records screened, 64 studies were included, of which 30 evaluated post-chemotherapy surveillance. 5672 CYP (5181 ALL, 491 AML) were included. 747 CYP experienced 800 relapses. Poor reporting hindered data extraction and quality assessment. Symptom-detected relapses were most common. Surveillance did not impact survival outcomes. Two studies demonstrated the high costs of surveillance programmes. No study reported on the experience or burdens of surveillance. The currently available evidence suggests that there is little support for the role of surveillance in detecting relapse in CYP with acute leukaemia. Prospective, longitudinal, randomised studies focused on key outcomes and high-quality reporting are needed. PROSPERO: CRD42023389281.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e31973"},"PeriodicalIF":2.3,"publicationDate":"2025-09-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145016050","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":"Tumor-Specific Critical Intraoperative Findings Influence Multidisciplinary Therapy.","authors":"Abdelhafeez Abdelhafeez, Amos Loh, Derek Harison, Alia Ahmad, Thelma Beatriz Velasquez Herrera, Bilal Mazhar Qureshi, Faizan Malik, Lorna Renner, Gordan Vujanic, Jeannette Parkes, Laura Galluzzo, Michael Nightingale, John T Lucas, Joyce Kambugu, Sharon Cox, Simone de Campos Vieira Abib, Kokila Lakhoo","doi":"10.1002/pbc.32031","DOIUrl":"https://doi.org/10.1002/pbc.32031","url":null,"abstract":"<p><strong>Background: </strong>Local control strategies in pediatric oncology are guided by disease-specific considerations. Effective communication of the goals of surgical procedure and associated intraoperative events plays a crucial role in shaping subsequent treatment decisions. However, accurately and comprehensively documenting these findings remains challenging, with considerable variability across different tumor types. This investigation aims to achieve a consensus on critical intraoperative oncologic observations pertaining to pediatric solid tumors, thereby facilitating the enhancement of surgical reporting and the optimization of subsequent treatment strategies.</p><p><strong>Methods: </strong>An expert panel comprising childhood cancer specialists from diverse disciplines and geographical regions participated in a Delphi consensus process. After reviewing relevant literature and engaging in multiple voting rounds, the panel identified essential tumor-specific intraoperative documentation elements.</p><p><strong>Results: </strong>A Delphi panel of 16 experts from diverse geographical locations completed two rounds of voting with a 94% participant retention rate and achieved consensus on 15 key statements. Essential documentation components included completeness of resection, evaluation of locoregional spread, and vascular involvement, with tumor-specific variations. For instance, neuroblastoma required documentation of resection percentage, while sarcoma emphasized biopsy tract resection and plane of resection. Ovarian germ cell tumors necessitated ascitic fluid sampling and contralateral ovary evaluation. Additionally, the presence of tumor thrombus was highlighted as particularly relevant in renal, liver, and adrenocortical carcinomas. Despite recognizing the significance of these findings, the panel noted deficiencies in operative reports, including omissions of documentation of tumor spillage, lymph node sampling, and residual disease, underscoring the need for improved documentation to support multidisciplinary decision-making.</p><p><strong>Conclusions: </strong>This study highlights the critical role of precise intraoperative documentation in guiding multidisciplinary care for pediatric solid tumors. The variability across tumor types underscores the need for tailored documentation guidelines. While a standardized synoptic operative report could improve consistency and communication, a hybrid model combining universal elements with tumor-specific details may offer an effective solution for comprehensive and adaptable reporting.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e32031"},"PeriodicalIF":2.3,"publicationDate":"2025-09-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145016093","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":"Rib Ewing Sarcomas in Children and Young Adults: A Large National Retrospective Series.","authors":"Audrey Claren, Alexandre Beige, Magali Morelle, Jérôme Doyen, Cyril Lervat, Luc Ollivier, Cécile Vérité, Perrine Marec-Bérard, Maria Jolnerovski, Anne Ducassou, Valentine Martin, Line Claude","doi":"10.1002/pbc.32012","DOIUrl":"https://doi.org/10.1002/pbc.32012","url":null,"abstract":"<p><strong>Background and purpose: </strong>Ewing sarcoma (ES) is the most prevalent malignant thoracic tumor in childhood and young adults. This study reports the outcome of a national cohort treated in an international prospective trial for a localized rib ES, with a long follow-up.</p><p><strong>Material and methods: </strong>All the patients treated in a prospective trial (December 1999-April 2013) were included. Data were collected from a prospective database and supplemented by a retrospective review of the patients' files. Local control alone rate, progression-free survival (PFS), and overall survival (OS) were assessed. Multivariate analyses were conducted to adjust for classical prognosis factors.</p><p><strong>Results: </strong>Eighty-two patients, median age 13.6 years, were included. All received induction chemotherapy. Surgery was completed (R0) in 88%. Overall, 66% had good histological response (GR). Radiotherapy (RT) was performed on the primary tumor in 28%, on the hemithorax in 13%, or on both in 32%. With a median follow-up of 12 years, 28 patients (34%) relapsed. Five-year local control rate was 95% (95%CI [89%-95%]), 5-year PFS was 73% (95%CI [62%-81%]), and 5-year OS was 79% (95%CI [69%-87%]). In multivariate analysis, pleural effusion was significantly associated with poor PFS (p = 0.049). In the case of R0 and GR (n = 41), no significant difference in PFS/OS (p = 0.16/p = 0.36) was reported according to adjuvant local RT. Overall, 38% of scoliosis and 7.3% of secondary cancer were reported.</p><p><strong>Conclusion: </strong>A multidisciplinary approach in high-risk patients can provide good local control in rib ES. In patients who have undergone R0 surgery and are GR, omitting adjuvant RT of the surgical bed may be discussed.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e32012"},"PeriodicalIF":2.3,"publicationDate":"2025-09-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145006496","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":"Describing Pediatric Oncology Patients Enrolled in Phase 1 Clinical Trials","authors":"Andrea Cuviello,&nbsp;Mariela Trejo,&nbsp;Emily Zeng,&nbsp;Deena Levine,&nbsp;Holly Spraker-Perlman","doi":"10.1002/pbc.32010","DOIUrl":"10.1002/pbc.32010","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background</h3>\u0000 \u0000 <p>Phase 1 trials may expose pediatric oncology patients to potential adverse effects beyond drug-related toxicity, including delays in advance care planning and suboptimal quality of end-of-life (EoL) care. Pediatric palliative care (PPC) can provide symptom management support and assist with EoL planning and care for patients and families enrolling in Phase 1 trials; however, little is known about children with cancer who enroll in Phase 1 studies.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>A retrospective medical record review of pediatric oncology patients enrolled on a Phase 1 clinical trial over a 9-year period was completed at an academic cancer hospital. Data collected included sociodemographic, clinical, and EoL-related variables. Descriptive analyses were performed.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>A total 539 patient charts met the inclusion criteria for review; 53% (<i>n</i> = 285) were male, 76% (<i>n</i> = 411) were Caucasian, and 82% (<i>n</i> = 442) were deceased. Central nervous system (CNS) tumors were the most common diagnosis (44%, <i>n</i> = 237), followed by leukemias (33%, <i>n</i> = 179), solid tumors (20%, <i>n</i> = 108), and lymphomas (3%, <i>n</i> = 15). All patients enrolled in at least one Phase 1 study, with 15% (<i>n</i> = 83) enrolling in two or more. The median length of time on study was 62 days, with time on study decreasing for subsequent Phase 1 trial participation. Two-thirds of patients (68%, <i>n</i> = 368) had PPC consultation; however, only 21% (<i>n</i> = 76) had PPC consulted prior to Phase 1 trial enrollment.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusions</h3>\u0000 \u0000 <p>Although most patients enrolled in Phase 1 studies have PPC involvement, referrals are typically made far beyond trial enrollment. Earlier and systematic PPC integration in this high-risk patient population may offer the potential benefit of maximal EoL support, care, and planning.</p>\u0000 </section>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 11","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-09-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145006432","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":"Characteristics of Transfusion and Association With Oncologic Outcomes in Hepatoblastoma Resection.","authors":"Stephanie F Polites, Jennifer H Aldrink, Timothy B Lautz, Robert A Vierkant, Mecklin V Ragan, Audra Reiter, Stephanie Y Chen, Eugene S Kim, Hannah N Rinehardt, Marcus M Malek, Andrew M Fleming, Andrew J Murphy, Jonathan P Roach, Sridharan Radhakrishnan, Zachary J Kastenberg, Nelson Piche, Yasmin Osman, Harold N Lovvorn Iii, Elisabeth T Tracy, Juan Favela, Hau D Le, John Marquart, Brian Craig, Dave R Lal, Natashia Seemann, Robin Petroze, Barrie S Rich, Richard D Glick, Leigh Selesner, Ashley Yoo, Elizabeth Fialkowski, Erin G Brown, Chloe Boehmer, Roshni Dasgupta, Max R Langham","doi":"10.1002/pbc.32029","DOIUrl":"10.1002/pbc.32029","url":null,"abstract":"<p><strong>Introduction: </strong>Children with hepatoblastoma (HB) and other solid tumors frequently undergo intraoperative blood transfusion (IBT) with unknown impact on oncologic outcomes and scant data to guide transfusion in this population. This study tested the hypothesis that IBT is associated with poorer survival in children with HB.</p><p><strong>Methods: </strong>A multicenter retrospective observational study of patients aged <18 years with HB who underwent primary tumor resection, including liver transplantation, from 2010 to 2019 was performed at 19 institutions. The association of IBT with risk of recurrence and death were determined using propensity score reweighted (PSR) Cox proportional hazards regression analyses.</p><p><strong>Results: </strong>There were 338 patients identified who met inclusion criteria and had sufficient data for inclusion. Of those, 257 (76%) received IBT, including 253 (98%) who received packed red blood cells (pRBC), 84 (33%) who received plasma, and 28 (11%) who received platelets. IBT was associated with higher pretreatment extent of disease (p < 0.001), presence of annotation factors (+VPEFR: 50%, n = 129 vs. 37%, n = 30, p = 0.035), and complex resections (extended, meso-, or total hepatectomy: 54%, n = 139 vs. 27%, n = 22, p < 0.001); these differences were mitigated after applying propensity score weighting. Patients who received IBT had greater postoperative hemoglobin (g/dL) (median 10 (I8-11) vs. 9 (8-10), p = 0.013) and required more postoperative plasma and platelet transfusions (p < 0.05). Median follow-up was 4.4 (2.0-8.3) years. Compared with non-IBT patients, those with IBT had higher incidence of death (PSR HR 2.35, 95% CI 1.10-5.02). Recurrence did not significantly differ across groups (PSR HR = 0.82, 95% CI 0.45-1.48).</p><p><strong>Conclusion: </strong>IBT was associated with greater hazard of death. Postoperative hemoglobin levels suggested that unnecessary transfusions occurred and a pRBC-focused approach to IBT led to coagulopathy. Development of optimal transfusion strategies for HB are needed to minimize unnecessary transfusions.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e32029"},"PeriodicalIF":2.3,"publicationDate":"2025-09-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144964429","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":"Atypical Neuroblastoma With Absent Urinary Catecholamine Excretion and 123ImIBG Avidity Are of Favorable Outcome: A Retrospective French Single-Center Study","authors":"Anna Borovkov,&nbsp;Juliette Assy,&nbsp;Isabelle Aerts,&nbsp;Franck Bourdeaut,&nbsp;Camille Cordero,&nbsp;Valérie Laurence,&nbsp;Amaury Leruste,&nbsp;Sarah Winter,&nbsp;Jean Michon,&nbsp;Daniel Orbach,&nbsp;Hélène Pacquement,&nbsp;Pascale Philippe-Chomette,&nbsp;Gaelle Pierron,&nbsp;Julien Masliah-Planchon,&nbsp;Sylvie Helfre,&nbsp;Isabelle Janoueix-Lerosey,&nbsp;Anne-Sophie Defachelles,&nbsp;Claudia Pasqualini,&nbsp;Nina Jehanno,&nbsp;Joanna Cyrta,&nbsp;Arnaud Gauthier,&nbsp;Véronique Mosseri,&nbsp;François Doz,&nbsp;Sabine Sarnacki,&nbsp;Liesbeth Cardoen,&nbsp;Hervé J. Brisse,&nbsp;Marie Luporsi,&nbsp;Gudrun Schleiermacher","doi":"10.1002/pbc.32025","DOIUrl":"10.1002/pbc.32025","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background</h3>\u0000 \u0000 <p>In neuroblastoma (NB), urinary catecholamine excretion and ¹²³ImIBG avidity—depending on tumor enzymatic activity and norepinephrine transporter expression, respectively—are diagnostic standards. The prognostic impact of atypical NB, without urinary catecholamine excretion and/or ¹²³ImIBG avidity, remains to be determined. We sought to determine the frequency and prognosis of atypical NB and investigate the significance of catecholamine profiles and ¹²³ImIBG avidity at diagnosis.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>From 2000 to 2020, 275 children with NB, aged 0–20 years at diagnosis, treated at Institut Curie, France, were retrospectively analyzed.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Overall, 24% of NB had atypical features (<i>n</i> = 67/275). Lower INRG stages L1/L2 were more frequent in atypical NB, 66% versus 28% (<i>n</i> = 44/67 vs. 59/208), with less INRG Stage M than in typical NB, 25% versus 61% (<i>n</i> = 17/67 vs. 126/208), <i>p</i> &lt; 0.001. Atypical tumors more frequently harbored favorable molecular features with less frequent <i>MYCN</i> amplification, 12% (<i>n</i> = 8/64) versus 29% (<i>n</i> = 58/201), <i>p</i> &lt; 0.01, and fewer cases with segmental chromosomal alterations, 30% (<i>n</i> = 13/44) versus 60% (<i>n</i> = 69/115), <i>p</i> &lt; 0.05. Event-free survival (EFS) and overall survival (OS) were better in atypical than typical NB (5-year EFS: 77% ± 5% vs. 50% ± 4% and OS 87% ± 4% vs. 65% ± 4%, <i>p</i> &lt; 0.001). However, in multivariate analysis, atypical features in NB were not significant independent markers of prognosis.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusions</h3>\u0000 \u0000 <p>Atypical NB constitute a subgroup of interest for biomolecular analyses, including transcriptomics, which might provide further insights into disease-associated molecular features and our understanding of NB development.</p>\u0000 </section>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 11","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-09-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/pbc.32025","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144992977","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Aliança AMARTE: A Collaborative Network to Improve Oncologic Pediatric Care in Brazil","authors":"Luiz Fernando Lopes,&nbsp;Patrícia Loggetto,&nbsp;André de Oliveira Junior,&nbsp;Bruna Farias,&nbsp;Carla Nunes,&nbsp;Aline Esmeraldo Andrade de Almeida,&nbsp;Anna Beatriz Costa Neves do Amaral,&nbsp;Anita Frisanco,&nbsp;Carmem Maria Costa Mendonça Fiori,&nbsp;Daniela Santana,&nbsp;Débora Garcia Gasperini,&nbsp;Eliane Francelino,&nbsp;Fabianne Carlesse,&nbsp;Fernanda Crizol Bazaglia,&nbsp;Flora Mitie Watanabe,&nbsp;Isis Quezado Magalhães,&nbsp;Joaquim Caetano de Aguirre Neto,&nbsp;Juliana Teixeira Costa,&nbsp;Karla Emilia de Sá Rodrigues,&nbsp;Mara A. D. Pianovski,&nbsp;Marcelo Milone,&nbsp;Maristella Bergamo Francisco dos Reis,&nbsp;Monnie Abraham,&nbsp;Rosemary Sanz,&nbsp;Seila Prado,&nbsp;Simone de Campos Viera Abib,&nbsp;Thelma Alves de Oliveira,&nbsp;Valdenize Tiziani,&nbsp;Viviany Oliveira Viana,&nbsp;Paola Friedrich,&nbsp;Monika L. Metzger,&nbsp;Carlos Rodriguez-Galindo","doi":"10.1002/pbc.31986","DOIUrl":"10.1002/pbc.31986","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Introduction</h3>\u0000 \u0000 <p>In Brazil, inequalities in access to treatment and survival of childhood cancer are observed across different geographic regions. In 2014, under the coordination of Hospital de Câncer Infantojuvenil de Barretos (Fundação Pio XII), six hospitals met weekly for case discussion. In 2019, the AMARTE (Apoio Maior Aumentando Recursos e Treinamentos Especializados) Alliance officially began with the aim to improve the quality of pediatric oncology care in Brazil and became a collaborative network of innovation, research, and learning with 31 member institutions in 2024.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>AMARTE follows four strategic pillars: (1) equalize diagnostics, (2) homogenize treatment, (3) epidemiological and survival studies, and (4) scientific development and innovation. Fundação Pio XII and St. Jude Children's Research Hospital provide infrastructure and human resources. Implementation resources are supported mainly by the member institution. The first strategic goal was to maintain sustainable member engagement. A semestral assessment (SCORE) monitors the progress and engagement level, based on defined indicators.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Three engagement reports have been published, with an increase in members’ scores: 17 hospitals increased their SCORE, 7 decreased, and 1 hospital did not change. Key achievements include a common data registry, diagnostic and treatment protocols, regular technical-scientific meetings, and the formation of a fundraising group to achieve financial sustainability.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>The first decade of the AMARTE Alliance highlights the relevance of collaboration in pediatric oncology. Challenges refer to the sustainable participation of specialists and heterogeneous governance structure across members, requiring flexibility in the implementation of shared initiatives. Outcome indicators are being developed to assess long-term impact.</p>\u0000 </section>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 11","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-09-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/pbc.31986","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144992969","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Significant Impact of Treatment Abandonment on Survival of Children With “Common and Curable” GICC Index Cancers in Sub-Saharan Africa—A Multicenter Prospective CANCaRe Africa Study","authors":"Mulugeta Yimer,&nbsp;Diriba Fufa,&nbsp;Festus Njuguna,&nbsp;Cecilia Mdoka,&nbsp;George Chagaluka,&nbsp;Elizabeth Molyneux,&nbsp;Francine Kouya,&nbsp;Lillian Sung,&nbsp;Vivian Paintsil,&nbsp;Barnabas Atwiine,&nbsp;Trijn Israels","doi":"10.1002/pbc.31997","DOIUrl":"10.1002/pbc.31997","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background</h3>\u0000 \u0000 <p>The World Health Organization Global Initiative for Childhood Cancer (GICC) targets a global survival rate of 60% for childhood cancer, focusing initially on six common, curable cancers. This study describes survival rates of five of these cancers in sub-Saharan Africa and assesses the impact of treatment abandonment (TxA).</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>This multicenter, prospective, observational cohort study included newly diagnosed children (&lt;16 years) with Burkitt lymphoma (BL), acute lymphoblastic leukemia (ALL), Wilms tumor (WT), retinoblastoma (RB), or Hodgkin lymphoma (HL), enrolled between January and December 2022 from seven hospitals in Malawi, Ethiopia (<i>n</i> = 2), Ghana, Kenya, Uganda, and Cameroon. Two-year event-free survival (EFS) and treatment abandonment rates were analyzed.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Of 366 patients enrolled (median age 7.0 years, 64% males), Burkitt lymphoma was most common (30%, 108/366). Median follow-up was 27.6 months (range: 8–37.4). Treatment abandonment occurred in 24% of patients (89/366), highest in RB patients (38%, 15/39). Overall 2-year EFS was 40.8% ± 2.7%, improving to 59.2% ± 3.0% when TxA cases were censored. EFS rates with TxA as an event were BL 39.1% ± 5.0%, ALL 27.4% ± 4.4%, WT 51.5% ± 5.4%, RB 39.9% ± 8.0%, and HL 68.5% ± 9.3%.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>Survival for common, curable childhood cancers in sub-Saharan Africa remains low, averaging approximately 41% at 2 years, ranging from 27% (ALL) to 69% (HL). Treatment abandonment significantly impacts outcomes, affecting nearly 25% of patients. Preventing abandonment could potentially increase average survival to approximately 60%, emphasizing the need for effective intervention strategies.</p>\u0000 </section>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 11","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-09-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144964376","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}