Journal of Pediatric Hematology/Oncology最新文献

{"title":"Clinical Heterogeneity of Class III MEK1 Mutations in Histiocytic Neoplasms: Insights From Adult and Pediatric Cases.","authors":"Jing-Jing Sun, Zhi Wan, Xue Tang","doi":"10.1097/MPH.0000000000003311","DOIUrl":"https://doi.org/10.1097/MPH.0000000000003311","url":null,"abstract":"","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":""},"PeriodicalIF":0.9,"publicationDate":"2026-10-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149224338","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Beyond Reirradiation: Third-Course Radiation Therapy in Recurrent Diffuse Intrinsic Pontine Glioma-Case Series and Literature Review.","authors":"Haruna Sakagami, Koichi Morokawa, Yoshitaka Mizushima, Masamitsu Mikami, Yosuke Miyairi, Takehisa Takagi, Keiichiro Koiwai, Masao Kobayashi, Katsuyoshi Koh, Mitsutaka Shiota, Kazuo Sakashita, Kohei Fukuoka","doi":"10.1097/MPH.0000000000003304","DOIUrl":"https://doi.org/10.1097/MPH.0000000000003304","url":null,"abstract":"<p><p>Diffuse intrinsic pontine glioma (DIPG) is a highly aggressive brainstem tumor with a poor prognosis, for which radiotherapy remains the only established treatment. Although reirradiation at first progression has been reported to provide symptomatic relief and survival benefit, the role of a third course of irradiation remains unclear. In this study, we report 3 cases of DIPG treated with a third course of radiotherapy at second recurrence and conduct a literature review to evaluate its clinical efficacy and safety. All 3 patients demonstrated radiologic disease stabilization following the third course of irradiation, with 1 patient showing partial neurological improvement. No permanent severe toxicity attributable to the third course of radiotherapy was observed; 2 patients transiently experienced consciousness disturbance, cerebral edema, and/or respiratory dysfunction. To further assess the clinical significance of this approach, we analyzed previously reported cases of DIPG treated with 3 courses of radiotherapy. Of the 21 identified rare cases, 13 with sufficient clinical data were included in the analysis. The median radiation doses for the first, second, and third courses were 54.0 (39.0 to 54.0), 20.0 (19.8 to 36.0), and 20.0 (12.0 to 24.0) Gy, respectively. Total median radiation doses of 3-course irradiation were 94.0 Gy (79.0 to 111.6). The median survival after the third irradiation was 5.5 (2 to 13) months, and the median overall survival was 20.5 (15 to 102) months, measured from the start of the first radiation. A radiologic response greater than stable disease was observed in 69.2% of cases, whereas clinical improvement or stabilization was noted in 93.7% of cases. Although further studies with larger cohorts are needed to clarify the efficacy and particularly the safety of this treatment due to the limited data on adverse events, 3 courses of irradiation may be a viable option for children with DIPG, with careful consideration of the risks and benefits.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":""},"PeriodicalIF":0.9,"publicationDate":"2026-10-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149194743","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Real-World Use of Dinutuximab Beta for High-Risk Neuroblastoma in Clinical Settings in France: The QUATUOR Study.","authors":"Anne-Sophie Defachelles, Gudrun Schleiermacher, Dominique Plantaz, Stéphanie Proust, Sarah Benezech, Marianna Deparis, Ludovic Mansuy, Joy Benadiba, Julie Tandonnet, Estelle Thebaud, Sarah Jannier, Chrystelle Dupraz, Véronique Laithier, Carole Coze, Dominique Valteau-Couanet","doi":"10.1097/MPH.0000000000003302","DOIUrl":"https://doi.org/10.1097/MPH.0000000000003302","url":null,"abstract":"<p><p>Dinutuximab beta (DB) is recommended for maintenance therapy in high-risk neuroblastoma (HR-NB) in Europe. In France, early access to DB was provided in the Temporary Use Authorization (ATU) program. The QUATUOR (Qarziba Use ATU Outcomes Research) study is an observational, multicenter, retrospective study that evaluated the effectiveness and safety of DB in HR-NB patients who received DB in maintenance between February 2017 and August 2018 in real-world clinical settings in France as part of the ATU program. Fourteen patients received DB in the frontline and 17 in the refractory/relapsed setting. In the frontline group, 2-year EFS and OS rates were 64.3% (95% CI: 34.3-83.3%) and 78.6% (95% CI: 47.3-92.5%), respectively, and in the relapsed/refractory group, 57.5% (95% CI: 30.7-77.2%) and 80.9% (95% CI: 51.3-93.5%), respectively. Complete or partial response of the metastatic sites was maintained by 23/31 evaluable patients at the end of treatment. Overall, DB was effective, with acceptable tolerability, in patients with HR-NB treated as part of the ATU program in France. This study demonstrates the importance of successful collaboration between national health authorities, pharmaceutical companies, physicians, and the neuroblastoma community to provide early access to innovative treatments.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":""},"PeriodicalIF":0.9,"publicationDate":"2026-10-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149194784","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Howell-Jolly Body-Like Inclusions in Neutrophils and Monocytes of a Preterm Neonate During Sepsis.","authors":"Nesrine Gongi, Hager Zarrouk, Hamida Jouini, Khaoula Meftah, Nada Missaoui","doi":"10.1097/MPH.0000000000003308","DOIUrl":"https://doi.org/10.1097/MPH.0000000000003308","url":null,"abstract":"","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":""},"PeriodicalIF":0.9,"publicationDate":"2026-10-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149182287","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Incidence and Predictors of Symptomatic Thrombosis During Asparaginase-containing Therapy in Children With Acute Lymphoblastic Leukemia: A Meta-analysis and Narrative Synthesis.","authors":"Chong Cheng, Chanjuan Tong, Lili Jiang, Yanqing Wang","doi":"10.1097/MPH.0000000000003307","DOIUrl":"https://doi.org/10.1097/MPH.0000000000003307","url":null,"abstract":"<p><strong>Objectives: </strong>Symptomatic thrombosis is a clinically important complication of asparaginase-containing therapy in children with acute lymphoblastic leukemia (ALL), but reported incidence and predictors vary across studies. We conducted a systematic review and meta-analysis to estimate its incidence and synthesize reported predictors.</p><p><strong>Methods: </strong>PubMed, Embase, Web of Science Core Collection, Scopus, and the Cochrane Library were searched from inception through April 15, 2026. Eligible studies enrolled patients aged ≤21 years with ALL receiving asparaginase-containing therapy and reported extractable incidence or predictor data for overall symptomatic thrombosis or symptomatic venous thromboembolism. Proportions were pooled after logit transformation using a random-effects model. Predictor data were synthesized narratively when quantitative pooling was inappropriate.</p><p><strong>Results: </strong>Twenty-three studies met the eligibility criteria; 15 studies involving 17,809 children and 655 events contributed to the incidence meta-analysis. The pooled incidence of symptomatic thrombosis was 5.6% (95% CI: 3.5%-9.0%), with substantial heterogeneity (I²=96.9%). Study-specific estimates ranged from ∼1% to 22.7%. Older age was the most consistently reported predictor; associations with inherited thrombophilia, non-O blood group, high-risk treatment status, and T-ALL phenotype were inconsistent across cohorts.</p><p><strong>Conclusions: </strong>Symptomatic thrombosis affects ∼6% of children with ALL receiving asparaginase-containing therapy, although incidence varies considerably across studies. Older age was the most reproducible risk factor, whereas evidence for other predictors remains heterogeneous.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":""},"PeriodicalIF":0.9,"publicationDate":"2026-10-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149182233","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Body Composition Assessment and Correlation to Metabolic Profile in Survivors of Childhood Brain Tumors.","authors":"Asha Kalyan, Miranda C Bradford, Anna Dubnicka, Isabella Terrones, Kristin Yao, Daniel V Runco, Catherine Pihoker, K Scott Baker, Rebecca Ronsley","doi":"10.1097/MPH.0000000000003300","DOIUrl":"https://doi.org/10.1097/MPH.0000000000003300","url":null,"abstract":"<p><strong>Introduction: </strong>Survivors of childhood brain tumors (CBT) are at increased risk for metabolic syndrome, contributing to long-term cardiometabolic morbidity. BMI may underestimate risk by failing to capture altered body composition in this population. More precise tools such as air displacement plethysmography (ADP) may improve early risk detection. This study aimed to identify ADP-derived markers of adiposity and characterize early metabolic abnormalities at the end of cancer therapy.</p><p><strong>Methods: </strong>This was a prospective, single-visit study enrolling pediatric survivors of high-grade CBT aged 2 to 21 years, off therapy for <5 years. Data included anthropometric measurements, metabolic markers (glucose, insulin, lipids, adiponectin, leptin, IL-6), and body composition (fat mass, fat-free mass, percent fat, measured by ADP). Descriptive statistics were used to summarize demographics and clinical characteristics, and categorical variables were presented as counts and percentages. Pearson correlation coefficients were calculated for log adiponectin versus percent body fat and BMI z-score.</p><p><strong>Results: </strong>Thirty survivors of CBT (n=30; age: 6 to 23 y) were evaluated a median of 0.3 years after treatment. Over half were normal weight (53%), and 47% were overweight or obese; 20% had hypertension and 23% had elevated waist circumference. Body composition (n=29) showed high adiposity, with a mean fat mass of 33% and 59% having elevated fat percentage. Metabolic assessment demonstrated early cardiometabolic dysfunction: insulin resistance and significant insulin resistance by HOMA-IR were seen in 50% and 33%, respectively, and lipid abnormalities and the metabolic syndrome were seen in 27% and 15%, respectively. Higher adiposity was associated with worse metabolic health, with greater fat mass percentage in insulin-resistant patients (P<0.01) and higher BMI z-score (P=0.02). Adiponectin was inversely correlated with fat mass% (r=-0.72), but not with BMI z-score.</p><p><strong>Conclusion: </strong>Childhood brain tumor survivors demonstrated a high prevalence of metabolic abnormalities and excess adiposity shortly after therapy completion. BMI underestimated abnormal adiposity compared with ADP-derived body composition assessment, supporting the need for comprehensive metabolic screening and early intervention during survivorship.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":""},"PeriodicalIF":0.9,"publicationDate":"2026-10-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149182033","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Predictive Factors for Intensive Care Unit Admission in Children and Young Adults Undergoing Hematopoietic Cell Transplantation.","authors":"Kayleigh M Schwartz, Samantha B Newman, Tzu-Hsuan Lin, Ruby Siada, Jessica L Spruit, Susan Murray, Gregory A Yanik","doi":"10.1097/MPH.0000000000003279","DOIUrl":"https://doi.org/10.1097/MPH.0000000000003279","url":null,"abstract":"<p><p>Hematopoietic cell transplantation (HCT) carries significant morbidity and mortality. Defining factors that impact intensive care unit (ICU) admission rates in children and young adults undergoing HCT is required. Retrospective review of allogeneic (n=221) or autologous HCT (n=115) patients from 2013 to 2023 was completed. Subjects were 30 years old or younger, undergoing transplant for malignant or nonmalignant disorders. Clinical and laboratory variables were evaluated. The primary endpoint was ICU admission or death within 180 days following initial HCT. Overall, 22% of allogeneic and 8.9% of autologous HCT recipients required ICU hospitalization within 180 days post-HCT. In allogeneic HCT recipients, age <12 months, inherited metabolic disorders, systemic steroids ≥0.5 mg/kg/day, GVHD grade 3-4, secondary therapy for GVHD, and relapse were associated with increased risk of an event. Relapses (HR: 11.10; 95% CI: 4.23-28.90, P<0.001) and secondary therapy for GVHD (HR: 4.65; 95% CI: 1.39-15.60, P=0.013) remained significant on multivariate analysis. Autologous HCT risk factors included CD34 dose (>10 million CD34/kg) and absolute lymphocyte count on day 28 post-HCT. No other parameter impacted ICU risk. In the allogeneic HCT setting, refractory GVHD or disease relapse were associated with increased ICU admission. In the autologous setting, higher infused cell doses were associated with increased risk for ICU admission.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":""},"PeriodicalIF":0.9,"publicationDate":"2026-10-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149148162","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Urolithiasis in Pediatric Acute Lymphoblastic Leukemia and Lymphoblastic Lymphoma (ALL/LLy): A Single-Center Case Series of Clinical, Metabolic, and Imaging Features.","authors":"Burak Yuzuguldu, Marissa Krieger, Julie Comella-Higgins, Ahmad Mohamed, Ezekiel Young, Meghan Higman, Matthew Barth, Kara M Kelly","doi":"10.1097/MPH.0000000000003301","DOIUrl":"https://doi.org/10.1097/MPH.0000000000003301","url":null,"abstract":"<p><strong>Background: </strong>Urolithiasis is an underrecognized complication in pediatric acute lymphoblastic leukemia/lymphoma (ALL/LLy), often associated with glucocorticoid therapy.</p><p><strong>Observations: </strong>We report 4 cases of urolithiasis occurring at different diagnostic and treatment phases, 3 requiring intervention. Presenting symptoms included flank pain, hematuria, and urinary discomfort. In 1 case, renal abnormalities were incidentally identified on PET/CT performed for remission assessment.</p><p><strong>Conclusions: </strong>Urolithiasis may occur across ALL/LLy diagnosis and treatment, particularly after glucocorticoid exposure and in patients with additional risk factors. Early recognition in patients with unexplained abdominal or urinary symptoms is essential to prevent complications.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":""},"PeriodicalIF":0.9,"publicationDate":"2026-10-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149148487","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Clarifications and Concerns Regarding the Manuscript \"Comparing Iron Prophylaxis Strategies in Infants: Is Sucrosomial Iron a Better Alternative?\"","authors":"Germano Tarantino, Maria Sole Rossato, Elisa Brilli","doi":"10.1097/MPH.0000000000003299","DOIUrl":"https://doi.org/10.1097/MPH.0000000000003299","url":null,"abstract":"","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":""},"PeriodicalIF":0.9,"publicationDate":"2026-10-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149148204","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Revisiting Pediatric ITP Prognosis: A Systematic Review and Meta-analysis of Simple Clinical and Laboratory Predictors of Chronic Disease.","authors":"Ahmad Sameer Ashi, Omar Ahmed Alshaikhi, Elaf Yahia Alnasser","doi":"10.1097/MPH.0000000000003305","DOIUrl":"https://doi.org/10.1097/MPH.0000000000003305","url":null,"abstract":"<p><strong>Background: </strong>Pediatric immune thrombocytopenia (ITP) usually has a favorable prognosis, although some children progress to chronic disease (>12 mo). A 2014 meta-analysis by Heitink-Pollé and colleagues identified important prognostic markers that informed tools such as the Nordic prediction score. Given advances in diagnosis over the past decade, updated evidence focusing on simple bedside predictors is needed.</p><p><strong>Objective: </strong>To evaluate post-2014 evidence on baseline clinical and laboratory predictors of chronic pediatric ITP.</p><p><strong>Methods: </strong>Following PRISMA 2020 guidelines, PubMed and Embase were searched for observational studies. Of 876 identified records, 26 studies were included in the systematic review and 24 in the meta-analysis (N>4000). Pooled odds ratios (ORs) and 95% confidence intervals (CIs) were calculated using a random-effects model.</p><p><strong>Results: </strong>Older age at diagnosis (OR 2.25) and insidious onset (OR 6.20) remained the strongest predictors of chronicity. Absence of preceding infection (OR 2.66) and female sex (OR 1.43) were also associated with chronic disease. Higher baseline platelet count (OR 2.14) demonstrated a similar trend, though this finding is limited by extreme heterogeneity (I2=78%) across studies. In a single study, mild bleeding predicted chronicity compared with moderate/severe bleeding (OR 2.86).</p><p><strong>Conclusion: </strong>These findings validate the Nordic Score framework and support risk stratification using simple, reproducible clinical parameters.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":""},"PeriodicalIF":0.9,"publicationDate":"2026-10-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149137258","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}