Pediatric Blood & Cancer最新文献

{"title":"Malignant Rhabdoid Tumors of the Liver Are Associated With Inferior Outcomes Compared to Other Extracranial Rhabdoid Tumors.","authors":"Sebastian Bühner, Katharina Gastberger, Stefanie Tüchert-Knoll, Victoria E Fincke, Pascal D Johann, Patrick Melchior, Margarita Teleshova, Denis Kachanov, Alexey Shcherbakov, Irene Schmid, Kleoniki Roka, Reiner Siebert, Christian Vokuhl, Joachim Gerss, Jörg Fuchs, Rhoikos Furtwängler, Michael C Frühwald","doi":"10.1002/pbc.32062","DOIUrl":"https://doi.org/10.1002/pbc.32062","url":null,"abstract":"<p><strong>Background: </strong>Extracranial malignant rhabdoid tumors (eMRT) are rare, highly aggressive pediatric neoplasms. While the liver is a relatively common anatomic site of presentation, the clinical course of patients with hepatic eMRT (eMRT-L) is not well described.</p><p><strong>Methods: </strong>We retrospectively analyzed 30 children affected by eMRT-L treated on a consensus regimen provided by the European Rhabdoid Registry (EU-RHAB). Clinical characteristics, radiology features according to the Pre-Treatment Extent of Tumor (PRETEXT) system, treatment details, and outcome were assessed. We employed patients with rhabdoid tumors of the kidney (RTK; n = 30) and other eMRT (n = 60) as controls.</p><p><strong>Results: </strong>Median age at diagnosis was 8 months (range: 0-53 months), 16 of 30 patients (55%) presented with metastatic disease. R0 resection was achieved in seven patients (23%). Most tumors showed PRETEXT Stage ≥3 (66%) and frequently exhibited PRETEXT annotation factors. One-year overall and event-free survivals were both 17% (95% confidence interval: 7.5-37). Compared to RTK and other eMRT, patients with eMRT-L had significantly inferior outcomes (hazard ratios 2.47 and 4.39, respectively). Complete resection and absence of metastases were associated with improved survival. Consolidation therapies (i.e., radiotherapy or high-dose chemotherapy) were only rarely used.</p><p><strong>Conclusions: </strong>EMRT-L represents a distinct high-risk subgroup within the eMRT spectrum, characterized by inferior survival despite standardized multimodal therapy. Current treatment approaches demonstrate limited efficacy. Our results highlight the urgent need for prospective, collaborative studies to refine risk stratification and to evaluate novel treatment options.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e32062"},"PeriodicalIF":2.3,"publicationDate":"2025-09-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145092245","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":"Caregivers' Perspectives on Changes in Family Life During B-ALL Therapy: A Qualitative Study From the Children's Oncology Group.","authors":"Alexandra Dreyzin, Megan Ware, Katrina Stumbras, John Kairalla, Emily Hibbitts, Brenna Mossman, Lyn Balsamo, Rozalyn Rodwin, Kirsten K Ness, Claire C Conley, Elizabeth Raetz, Meenakshi Devidas, Lillian Sung, Mignon Loh, Stephen P Hunger, Reuven J Schore, Anne Angiolillo, Nina Kadan-Lottick","doi":"10.1002/pbc.32057","DOIUrl":"https://doi.org/10.1002/pbc.32057","url":null,"abstract":"<p><strong>Background: </strong>Treatment of pediatric B-acute lymphoblastic leukemia (B-ALL) impacts both patients and their caregivers. An understanding of family functioning during therapy can inform family-centered care. We aimed to prospectively identify negative and positive changes in family life as perceived by caregivers throughout ALL therapy.</p><p><strong>Methods: </strong>Caregivers of children aged ≥4 years with average-risk B-ALL enrolled on the Children's Oncology Group trial AALL0932 who consented to an ancillary study were asked: \"How has family life changed since your child's diagnosis of leukemia for the better or for the worse?\" Written free responses were collected at approximately 2, 8, 17, 26 (end of therapy for females), and 38 (end of therapy for males) months post-diagnosis. Inductive content analysis was used to create codes, subcategories, and categories. Descriptive statistics were used to characterize the sample and frequencies of reported codes.</p><p><strong>Results: </strong>Overall, 994 responses were collected from caregivers of 468 children across all timepoints. Twenty-seven individual codes were identified, categorized by negative changes (reported by 89% of caregivers) and positive changes (reported by 58% of caregivers). Subcategories of negative changes, including changes in daily routines, work and finance, patient health and care needs, effects on other family members, and emotional changes, were identified across all timepoints, but were most prevalent early in therapy. Importantly, positive changes were also identified, including family support, community support, and changes in outlook.</p><p><strong>Conclusion: </strong>This study identifies negative and positive family changes perceived by caregivers of children undergoing B-ALL therapy that can inform future interventions to better support families.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e32057"},"PeriodicalIF":2.3,"publicationDate":"2025-09-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145092262","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":"Exploring the Role of Bioprosthesis for Chest Wall Reconstruction in Pediatric Oncology.","authors":"Chiara Oreglio, Chiara Grimaldi, Alessandro Gonfiotti, Giulia Fusi, Elisa Severi, Roberto Lo Piccolo, Giovanni Beltrami, Angela Tamburini, Anna Maria Buccoliero, Maria Chiara Cianci, Antonino Morabito, Flavio Facchini","doi":"10.1002/pbc.32058","DOIUrl":"https://doi.org/10.1002/pbc.32058","url":null,"abstract":"<p><strong>Aim of the study: </strong>Investigating the possible role of bioprosthesis in the treatment of primary chest wall Ewing sarcoma (pCWES) after major chest wall resection in the pediatric oncologic population and its role in addressing the significant controversies related to the ongoing growth process in this population. This study presents the insights from a pediatric referral center, aiming at evaluating the oncological and functional outcomes of children treated with bioprosthesis.</p><p><strong>Methods: </strong>Data were collected retrospectively for all cases of pCWES managed at our facility over 5 years. All of the patients underwent the same surgical procedure for chest wall reconstruction, with positioning of a porcine biologic prosthesis covered by a latissimus dorsi muscle pedicled flap. A multidisciplinary evaluation was offered in all cases. Evaluated outcomes included morbidity, mortality, and subsequent functional and aesthetic results.</p><p><strong>Results: </strong>Three patients were included: two males and one female. Median age at diagnosis was 13 years (range: 18 months to 19 years). One patient presented with lung metastases at diagnosis. All patients underwent a neoadjuvant chemotherapy regimen. Postoperative chemotherapy was restarted after a median of 47 postoperative days (range: 40-59). All of the patients are alive at the latest follow-up (mean follow-up time = 29 months), and the rate of local recurrence was 0.</p><p><strong>Conclusion: </strong>Our series includes the youngest patient documented in the literature to be treated with a biologic prosthesis without the use of rigid materials. This approach appears to be both safe and effective for pediatric patients with pCWES. A multidisciplinary approach remains essential.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e32058"},"PeriodicalIF":2.3,"publicationDate":"2025-09-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145092277","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":"Exploring the Association of Time-After-Death on Psychological Distress in Parents Who Lost a Child to Cancer.","authors":"Peter Francis Raguindin, Jerina Deda, Anna Katharina Vokinger, Eva De Clercq, Katrin Scheinemann, Andre Oscar von Bueren, Eva Maria Tinner, Eva Bergstraesser, Eddy Carolina Pedraza, Gisela Michel","doi":"10.1002/pbc.32060","DOIUrl":"https://doi.org/10.1002/pbc.32060","url":null,"abstract":"<p><p>We conducted a cross-sectional survey of 101 bereaved parents in Switzerland to determine the association between time-after-death and psychological distress. Eligible deceased children were identified via the national registry and forwarded to former treating pediatric oncology centers, which then contacted the parents. Psychological distress was measured using the Brief Symptom Inventory (BSI-18). Time-after-death ranged from 2 to 24 years (mean = 11.3, SD = 5.6). Linear regression and spline models showed no significant association between time-after-death and psychological distress. Our findings suggest that time-after-death alone does not predict parental psychological distress.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e32060"},"PeriodicalIF":2.3,"publicationDate":"2025-09-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145075976","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":"Changes in Blood Test Indices of Infants With Down Syndrome-Associated Transient Abnormal Myelopoiesis.","authors":"Hideyuki Hawaka, Tomoyuki Shimokaze, Tomoko Yokosuka, Katsuaki Toyoshima, Tomoko Saito, Hiroaki Goto","doi":"10.1002/pbc.32064","DOIUrl":"https://doi.org/10.1002/pbc.32064","url":null,"abstract":"<p><strong>Background: </strong>Approximately 20% of infants with Down syndrome-associated transient abnormal myelopoiesis (TAM) die at an early age, mainly because of liver failure.</p><p><strong>Objective: </strong>To identify post-diagnosis changes in blood test indices that could predict TAM-related deaths as early as the postnatal period.</p><p><strong>Methods/results: </strong>This was a single-center retrospective study. Of the 505 children with Down syndrome admitted to our hospital from 1992 to 2024, we studied 12 infants with TAM-related death and 39 survivors. In the death and survival groups, the median gestational ages were 34.9 and 37.1 weeks, respectively (p = 0.146). At birth, the white blood cell (WBC) counts were 99.2 and 41.7 × 10⁹/L (p = 0.027), and the serum direct bilirubin (D-Bil) concentrations were 20 and 17 µmol/L (p = 0.45), respectively. After the first week of age, the WBC counts, blast percentages, platelet counts, and serum alanine aminotransferase and aspartate aminotransferase concentrations were similar between the groups. At 1 week of age, the serum D-Bil concentrations were 32 and 21 µmol/L (p = 0.007), respectively. At 2 weeks, they were 94 and 27 µmol/L (p < 0.001), respectively. The area under the receiver operating characteristic curve for the ability of serum D-Bil at 2 weeks to predict death was 0.94, with a threshold of ≥51 µmol/L (approximately 3.0 mg/dL) yielding a sensitivity of 1.00 and a specificity of 0.78.</p><p><strong>Conclusion: </strong>A serum D-Bil concentration of ≥51 µmol/L at 2 weeks of age may serve as a useful predictor of TAM-related death.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e32064"},"PeriodicalIF":2.3,"publicationDate":"2025-09-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145075953","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":"How We Approach the Integration of Psychological Services in the Care of Children With Cancer Predisposition Syndromes.","authors":"Joan W Hanania, Rowan Forbes Shepherd, Lori Wiener, Katianne M Howard Sharp, Morgan N Similuk, Avram E Denburg, Claire E Wakefield","doi":"10.1002/pbc.32065","DOIUrl":"https://doi.org/10.1002/pbc.32065","url":null,"abstract":"<p><p>The psychosocial aspects of pediatric hereditary cancer range beyond initial coping with the genetic diagnosis and are situated across the lifespan and continuum of care. Over the past 20 years, a growing body of evidence has demonstrated the need for tailored support to identify and manage psychosocial concerns of pediatric patients with, or at risk of, a cancer predisposition syndrome (CPS). As the unmet needs of young people with a CPS continue to grow, the importance of psychology in the genomic era has expanded. This paper discusses how psychologists can be integrated with inter- or multi-disciplinary teams to address complex psychosocial needs.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e32065"},"PeriodicalIF":2.3,"publicationDate":"2025-09-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145075974","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":"Prolonged Therapeutic Serum Asparaginase Activity Levels After Administration of Calaspargase Pegol.","authors":"Catherine E Martin, Madeleine B O'Keefe, Taylor Gullickson, Mira A Kohorst, Anand Srinivasan, Paul J Galardy, Linda McAllister, Jessica L Kalmes, Alexis K Kuhn","doi":"10.1002/pbc.32030","DOIUrl":"https://doi.org/10.1002/pbc.32030","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e32030"},"PeriodicalIF":2.3,"publicationDate":"2025-09-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145075530","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":"Emapalumab for Maladaptive Interferon-γ-Mediated Inflammation in Drug Reaction With Eosinophilia and Systemic Symptoms Complicating First-Line Antituberculosis Therapy.","authors":"Yahya Almodallal, Daniel Whitehurst, Sing Sing Way, Jane Koo, Ashish R Kumar","doi":"10.1002/pbc.32052","DOIUrl":"https://doi.org/10.1002/pbc.32052","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e32052"},"PeriodicalIF":2.3,"publicationDate":"2025-09-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145069912","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":"Renal Parenchyma Preservation in a Child With Giant Bilateral Wilms Tumors Following SIOP-Based Multistage Therapy.","authors":"Ramin Malikov, Gambar Ismayilov, Iqbal Babazade","doi":"10.1002/pbc.32047","DOIUrl":"https://doi.org/10.1002/pbc.32047","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e32047"},"PeriodicalIF":2.3,"publicationDate":"2025-09-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145069928","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":"Social Determinants of Health and Pediatric Brain Tumor Neuropsychological Morbidities.","authors":"Christina M Sharkey, Johanna Nielsen, Karin S Walsh, Hannah Weisman, Kristina K Hardy","doi":"10.1002/pbc.32050","DOIUrl":"https://doi.org/10.1002/pbc.32050","url":null,"abstract":"<p><strong>Introduction: </strong>Neurocognitive and psychological morbidity risk is well-documented among pediatric brain tumor (PBT) survivors, yet most known predictors are nonmodifiable, limiting the implementation of a prevention approach. The social and environmental context has been increasingly considered, yet extant research primarily studies individual-level proxy measures (e.g., insurance). This cross-sectional study aimed to examine community-level factors that may contribute to neurocognitive and psychological outcomes in PBT survivors.</p><p><strong>Methods: </strong>Participants were clinically referred PBT survivors who completed a neuropsychological evaluation (N = 160, M age = 11.28, SD = 4.69, 57.5% male, 75.0% White), including an age-appropriate Wechsler scale and parent-report questionnaires (Behavior Rating Inventory of Executive Function, Child Behavior Checklist). Nationally normed and locally normed Child Opportunity Index (COI) scores were assigned using census tract geocoding. Higher scores reflect more resources and higher opportunity.</p><p><strong>Results: </strong>Multivariate models including covariates found that National COI rank was associated with intellectual functioning (β = 0.37, 95% CI [0.24, 0.49]), and parent-reported executive functions (β = -0.20, 95% CI [-0.365, -0.06]), and psychosocial functioning (β = -0.18, 95% CI [-0.35, -0.05]). Local COI rank had similar effects (p < 0.01). Specific National and Local COI domains had differing effects across outcomes. Radiation treatment was significantly associated with parent-reported outcomes (p < 0.05) but not intellectual functioning.</p><p><strong>Conclusions: </strong>Findings suggest community-level social determinants of health are associated with neurocognitive and psychological outcomes in PBT survivors. The COI may be useful to inform individual-level interventions and community-level policy to mitigate neuropsychological problems following PBT treatment in children from lower-opportunity communities. Exploration of specific educational, health, or social/economic indicators may highlight targets for a problem-prevention approach to reducing PBT survivors' risk for morbidities.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e32050"},"PeriodicalIF":2.3,"publicationDate":"2025-09-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145069931","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}