Pediatric Blood & Cancer最新文献

{"title":"Synchronous Mediastinal Germ Cell Tumor with Sarcomatous Transformation and Acute Myeloid Leukemia.","authors":"Elysia Cohen, Olayinka Okeleji, David McCall, Miriam Garcia, Cesar Nunez, Branko Cuglievan, Cynthia Herzog, Shehla Razvi, Priti Tewari, Harrison Douglas, Amber Gibson","doi":"10.1002/pbc.32092","DOIUrl":"https://doi.org/10.1002/pbc.32092","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e32092"},"PeriodicalIF":2.3,"publicationDate":"2025-10-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145213303","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":"Bridging the Gap in Pediatric Relapsed Acute Lymphoblastic Leukemia Treatment: Insights and Outcomes From the ALL-IC REL 2016 Guidelines.","authors":"Marko Kavcic, Dániel J Erdélyi, Volkan Hazar, Mirella Ampatzidou, Boryana Avramova, Anca Colita, Monica Makiya, Sophia Polychronopoulou, Bence Hőbör, Tomaz Prelog, Andrea Reyes, Juan Tordecilla, Koray Yalçin, Arend von Stackelberg, Janez Jazbec","doi":"10.1002/pbc.32063","DOIUrl":"https://doi.org/10.1002/pbc.32063","url":null,"abstract":"<p><strong>Background: </strong>The Acute Lymphoblastic Leukemia InterContinental (ALL-IC) Study Group exemplifies the potential of broad international collaboration. Patient outcomes have improved by standardizing therapeutic options and employing flow cytometry-based minimal residual disease (MRD) for treatment stratification. Nevertheless, relapse occurs in 10%-20% of cases, with survival rates falling short of benchmarks set by top-tier published studies.</p><p><strong>Objectives: </strong>We aimed to unify treatment guidelines for children with first relapse of ALL across the ALL-IC network, analyze post-relapse outcomes, and report findings from an observational registry.</p><p><strong>Methods: </strong>Patients were stratified as standard-risk (SR) or high-risk (HR) based on relapse features and genetics. HR criteria included T-cell immunophenotype, very early or early isolated bone marrow relapse, and relapse post-stem cell transplant (SCT). SR was assigned to all others. SCT was indicated in the whole HR group and in SR patients with poor responses (MRD ≥ 0.1% on Day 29).</p><p><strong>Results: </strong>Among 370 patients (mean age 9 years; 33.2% female) diagnosed with first relapse between 2017 and 2021, 90.5% had received ALL-IC-Berlin-Frankfurt-Münster (BFM) 2009 treatment initially. Upon relapse, 46.8% were classified as SR and 53.2% as HR. Complete remission rates post-induction were 84% (SR) and 56% (HR). MRD < 0.1% was achieved by 53% (SR) and 29% (HR). Five-year overall survival was 50.5% (74% SR, 32% HR). HR outcomes were hindered by disease progression, treatment toxicity, and posttransplant complications.</p><p><strong>Conclusions: </strong>This inaugural ALL-IC REL Consortium report demonstrates promising SR outcomes, akin to the International Study for the Treatment of Childhood Relapsed ALL (IntReALL) findings, but highlights poor HR outcomes with standard chemotherapy. Novel therapeutic strategies are urgently needed in upcoming ALL-IC-BFM REL protocols.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e32063"},"PeriodicalIF":2.3,"publicationDate":"2025-10-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145213289","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":"Seroreversion of Human Papilloma Virus Antibodies in a Childhood Solid Tumor Survivor Following Three-Dose Vaccination.","authors":"Yali Han, Qing Cao, Melissa M Hudson, Jiaoyang Cai","doi":"10.1002/pbc.32095","DOIUrl":"https://doi.org/10.1002/pbc.32095","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e32095"},"PeriodicalIF":2.3,"publicationDate":"2025-10-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145213368","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":"Beyond the Diagnosis: A Child, a Mother, and Cancer in Eastern Congo.","authors":"Criss Koba Mjumbe, Christian Tague, Freddy Ahadi Ndiwemo, John Isiko","doi":"10.1002/pbc.32091","DOIUrl":"https://doi.org/10.1002/pbc.32091","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e32091"},"PeriodicalIF":2.3,"publicationDate":"2025-10-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145213309","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":"Pediatric Intravenous Iron Therapy: Evaluating the Safety and Efficacy of Iron Isomaltoside and Low-Molecular-Weight Dextran.","authors":"Ernesta Dragūnė, Agnė Morkūnaitė, Gaudas Benediktas Trakymas, Ramunė Vaišnorė, Sonata Šaulytė Trakymienė","doi":"10.1002/pbc.32093","DOIUrl":"https://doi.org/10.1002/pbc.32093","url":null,"abstract":"<p><strong>Background: </strong>Iron deficiency (ID) and iron deficiency anemia (IDA) are prevalent among children and adolescents, affecting growth and development. Although oral iron is the first-line treatment, intravenous (IV) iron is required in cases of poor tolerance or inadequate response. While some IV formulations are used in pediatric clinical practice, no published data on the safety and efficacy of iron isomaltoside (IIM) in the pediatric population exist, while data on low-molecular-weight dextran (LMWD) remain limited.</p><p><strong>Procedure: </strong>Our retrospective cohort study at Vilnius University Hospital Santaros Klinikos (2021-2023) included pediatric patients (<18 years) with ID/IDA receiving IIM or LMWD. Hematological response assessment was based on reticulocyte hemoglobin equivalent (Ret-He) and reticulocyte percentage at 3-7 days (early response), and red blood cell count, hemoglobin (HGB), mean corpuscular volume, serum ferritin, and transferrin saturation at 4-6 weeks (late response).</p><p><strong>Results: </strong>Seventy-two patients (IIM: 50, LMWD: 22; 135 infusions), 68% female, with a median age of 13.5 years, were included in the study. Mean HGB increase was 32.4 g/L (IIM) versus 33.1 g/L (LMWD) (p = 0.9). Median ferritin increase was 71.4 µg/L (IIM) versus 117.8 µg/L (LMWD) (p = 0.3). Ret-He improved significantly (p < 0.05). Mild AEs occurred in 5.6%, with similar AE rates in both groups (IIM: 6.0%, LMWD: 4.5%).</p><p><strong>Conclusion: </strong>Our findings suggest that IIM and LMWD are effective and safe IV iron therapies for pediatric ID/IDA. Given the lack of data on the safety and efficacy of IIM in the pediatric population, our findings provide valuable preliminary evidence.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e32093"},"PeriodicalIF":2.3,"publicationDate":"2025-09-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145200482","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":"Retinoblastoma Research in Africa: A Scoping Review.","authors":"Sung Park, Yaw A Essuman, Sadik T Sherief, Sameh E Soliman, Vera A Essuman, Helen Dimaras","doi":"10.1002/pbc.32074","DOIUrl":"https://doi.org/10.1002/pbc.32074","url":null,"abstract":"<p><p>Achieving equity in childhood cancer represents a global challenge. In Africa, where retinoblastoma has high mortality and morbidity, strengthening research capacity is crucial in developing clinical guidelines from local evidence. This review identified the scope of retinoblastoma research conducted in Africa. A systematic search identified English-language studies on retinoblastoma in Africa, published between January 1, 2003 and May 15, 2025. Studies were excluded if retinoblastoma was not the primary focus or if Africa was not the main study location. Data collected included journal information, author affiliations, study type, country, purpose, and results. Quantitative findings were summarized with descriptive statistics, while qualitative findings were narratively synthesized. Of the 1546 citations retrieved, 89 met the inclusion criteria. Single-country studies (n = 85) represented 20 of 54 (37%) African countries, while four multi-country studies increased representation to 43 countries (80%). Most studies were clinical observational (55/89, 62%). Of the 89 studies, 49 (55%) were authored solely by researchers in Africa, but studies which included foreign authors tended to be published in journals with higher journal impact factors (p<0.001). The growth of retinoblastoma research in Africa reflects both expanding local research capacity and increased international collaboration. However, limited experimental research and basic science studies point to opportunities to strengthen the local evidence base needed to inform clinical guidelines for improving retinoblastoma outcomes in Africa.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e32074"},"PeriodicalIF":2.3,"publicationDate":"2025-09-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145186427","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":"Neurological, Neuropsychological, and Social Outcomes in Pediatric Patients Diagnosed With Cerebellar Mutism Syndrome: A Systematic Review.","authors":"Margaret H Hughes, Joseph Giuseppe R Paturzo, Callie M Fernandez, Kareena S Garg, Jincy Rajan, Suditi Rahematpura, John R DiBello, Umayr R Shaikh, Jordan E Wrigley, Nicholas S Philips, Darcy Raches, Melissa M Hudson, Supriya Sarvode","doi":"10.1002/pbc.32072","DOIUrl":"https://doi.org/10.1002/pbc.32072","url":null,"abstract":"<p><strong>Background: </strong>Cerebellar mutism syndrome (CMS) is a postsurgical complication following posterior fossa tumor resection. This review explores long-term neurological, neuropsychological, and social outcomes of children with CMS.</p><p><strong>Methods: </strong>A search was performed across multiple databases, focusing on terms related to neoplasms, postoperative complications, and mutism. Selected studies included individuals less than 18 years of age who developed CMS following posterior fossa tumor removal, and developed at least one neurological, neuropsychological, or social outcome.</p><p><strong>Results: </strong>Among 2514 publications, 34 studies were included, describing 631 patients aged 1-25.7 years (median = 12 patients; range: 5-107 patients). The prevalence of CMS ranged from 3.7% to 63%, with a median follow-up of 26.5 months (ranging from 2 months to 14.2 years). Ataxia was the most common neurologic outcome reported in 17 studies. Five of nine studies assessing cognitive outcomes noted significantly lower full-scale intelligence quotient/general intellectual ability scores among CMS patients. Emotional changes were observed in 40%-100% of participants across 23 studies.</p><p><strong>Conclusions: </strong>CMS survivors face ongoing neurological, neuropsychological, and behavioral challenges, impacting their quality of life and highlighting the need for advancements in surgical techniques and rehabilitation interventions.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e32072"},"PeriodicalIF":2.3,"publicationDate":"2025-09-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145186442","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":"Pravastatin for Radiation-Induced Fibrosis of the Extremity: A Single-Center Experience.","authors":"Rachel Offenbacher, Lara Fabish, Alice Lee, Rui Yang, Bang Hoang, David S Geller, Jana L Fox, David M Loeb, Alissa Baker","doi":"10.1002/pbc.32084","DOIUrl":"https://doi.org/10.1002/pbc.32084","url":null,"abstract":"<p><p>Radiation therapy (RT) is a mainstay of treatment for several sarcomas. Radiation-induced fibrosis (RIF) is a common side effect of RT with an incidence of approximately 80%. RIF is caused by excessive proliferation of myofibroblasts, which produce collagen and extracellular matrix components, coupled with a reduction of remodeling enzymes. Targeting this pathway with pravastatin to prevent and ameliorate RIF has been shown to improve outcomes in patients with head and neck cancer. We share our single-center experience using pravastatin to treat RIF involving the joints in children with sarcomas who developed RIF, as well as in a population administered prophylactically.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e32084"},"PeriodicalIF":2.3,"publicationDate":"2025-09-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145192346","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":"Dual Use of Skeletal Muscle Index and Body Mass Index Measured at the Time of Diagnosis Predicts Prognosis in Children With Malignant Solid Tumors: A Single-Center Retrospective Study.","authors":"Wataru Kudo, Keita Terui, Katsuhiro Nishimura, Daisuke Katsumi, Ryoya Furugane, Ayako Takenouchi, Shugo Komatsu, Satoru Oita, Yunosuke Kawaguchi, Tomoro Hishiki","doi":"10.1002/pbc.32082","DOIUrl":"https://doi.org/10.1002/pbc.32082","url":null,"abstract":"<p><strong>Background: </strong>Sarcopenia, a progressive muscle-wasting disorder, is increasingly being recognized in childhood cancers. While skeletal muscle index (SMI) is commonly used to assess sarcopenia, we investigated the combined utility of SMI and body mass index (BMI) in predicting the prognosis of children with malignant solid tumors.</p><p><strong>Methods: </strong>The BMI and SMI of 119 children with different types of malignant solid tumors were analyzed. Computed tomography and height at the time of diagnosis were used to determine the SMI. SMI and BMI were standardized using reference values. Cluster and survival time analyses were conducted to identify subpopulations.</p><p><strong>Results: </strong>Cluster analysis performed using z-scores for SMI and BMI yielded four clusters: Cluster 1 had high z-scores for both indices; Cluster 2 had average z-scores for both indices; Cluster 3 had low z-scores for SMI and average z-scores for BMI; and Cluster 4 had low z-scores for both indices. Cluster classification was an independent prognostic factor for both event-free survival (hazard ratio 1.56; 95% confidence interval: 1.09-2.25) and overall survival (hazard ratio 1.70; 95% confidence interval: 1.10-2.64) after combining age and metastasis. Cluster 1 had low metastasis prevalence and a favorable prognosis, whereas Cluster 4 exhibited a poorer prognosis.</p><p><strong>Conclusion: </strong>The potential prognoses of the four distinct patient clusters based on the z-scores for SMI and BMI at the time of diagnosis were determined. The dual use of SMI and BMI may facilitate the identification of children with malignant solid tumors who are at a high risk of poor outcomes.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e32082"},"PeriodicalIF":2.3,"publicationDate":"2025-09-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145192369","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":"Psychosocial Health and Chronic Health Conditions Among Bereaved Siblings: A Report From the Childhood Cancer Survivorship Study (CCSS).","authors":"Prachi Sharma, Nivya George, Deokumar Srivastava, Eric J Chow, Melissa A Alderfer, Wendy Leisenring, Kristin A Long, Anne E Lown, Kevin C Oeffinger, Lonnie K Zeltzer, Gregory T Armstrong, Kevin R Krull, Tara M Brinkman, David Buchbinder","doi":"10.1002/pbc.32076","DOIUrl":"https://doi.org/10.1002/pbc.32076","url":null,"abstract":"<p><strong>Objective: </strong>To compare psychosocial health and chronic health conditions (CHCs) in bereaved and non-bereaved adult siblings impacted by childhood cancer and to identify predictors of emotional distress and health-related quality of life among bereaved siblings.</p><p><strong>Methods: </strong>A total of 4558 adult siblings (733 bereaved; 3825 non-bereaved) of 5-year survivors of childhood cancer completed measures of emotional distress (Brief Symptom Inventory [BSI]-18) and health-related quality of life (Medical Outcomes Survey Short Form [SF]-36) and reported their social attainment milestones (i.e., educational attainment, employment, and marital status). CHCs' burden was classified as none/low versus medium/high/very severe. Cancer-associated complications prior to the patient's death, sibling age at bereavement, and social attainment variables were examined as predictors of emotional distress and health-related quality of life among bereaved siblings using multivariable modified Poisson regression.</p><p><strong>Results: </strong>Bereaved siblings in this sample reported excellent psychosocial health long term (e.g., depressive symptoms 6.5%, somatization 4.4%, anxiety 3.5%). Bereaved siblings had an elevated risk of depression (relative risk [RR] 1.53; 1.10-2.13, p = 0.01), reduced social quality of life (RR 1.35; 1.00-1.82, p = 0.05), diminished educational attainment, and greater CHC burden than non-bereaved siblings. No differences were observed for other subscales or social attainment outcomes. Among bereaved siblings, risk factors for depression included male sex (RR 0.42; 0.19-0.93, p = 0.05), never being married (RR 3.02; 1.45-6.28, p = 0.05), and greater CHC burden (RR 2.42; 1.18-4.99, p = 0.05). Risk factors for poor social functioning included unemployment (RR 2.24; 1.12-4.45, p = 0.05) and never being married (RR 2.16; 1.22-3.82, p = 0.05).</p><p><strong>Conclusion: </strong>Bereaved siblings report excellent psychosocial health long-term and demonstrate only a marginally elevated risk of experiencing symptoms of depression and poor social quality of life compared to non-bereaved siblings.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e32076"},"PeriodicalIF":2.3,"publicationDate":"2025-09-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145186507","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}