Pediatric Blood & Cancer最新文献

{"title":"Generalized Eruptive Histiocytosis With a GAB2::BRAF Fusion in a Child: A Targetable Driver of Undefined Prognostic Weight.","authors":"Martina Minkov, Zofia Hélias-Rodzewicz, Jean-François Emile, Milen Minkov","doi":"10.1002/1545-5017.70759","DOIUrl":"https://doi.org/10.1002/1545-5017.70759","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e70759"},"PeriodicalIF":2.4,"publicationDate":"2026-10-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149165062","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":"Abatacept for Prophylaxis of Acute and Chronic Graft-Versus-Host-Disease in Children Undergoing Allogeneic Hematopoietic Stem Cell Transplantation-A Systematic Review and Meta-Analysis of Cohort Studies.","authors":"Matthijs D Kruizinga, Anne B Verbeek, Caroline A Lindemans, Emilie P Buddingh","doi":"10.1002/1545-5017.70746","DOIUrl":"https://doi.org/10.1002/1545-5017.70746","url":null,"abstract":"<p><strong>Background: </strong>Graft-versus-host disease (GvHD) remains one of the most significant complications of hematopoietic stem cell transplantation (HSCT). Abatacept, a selective T-cell costimulation modulator, is a relatively novel prophylactic agent, though pediatric data remain limited.</p><p><strong>Procedure: </strong>A systematic review and meta-analysis was conducted to evaluate the efficacy and safety of abatacept for GvHD prophylaxis in pediatric HSCT.</p><p><strong>Results: </strong>Fifteen cohort studies were identified (eight comparative and seven single-arm). Studies included abatacept as adjunctive agent to traditional regimens. Pooled analyses demonstrated that abatacept-inclusive regimens significantly reduced the risk of Grade II-IV acute GvHD (aGvHD) (risk ratio [RR] 0.31, 95% confidence interval [CI] 0.14-0.65) and severe Grade III-IV aGvHD (RR 0.14, 95% CI 0.05-0.37). Abatacept administration was not associated with decreased moderate-to-severe chronic GvHD incidence, although extended regimens were associated with reduced risk (RR 0.25, 95% CI 0.08-0.78). Abatacept-inclusive GvHD prophylaxis was not associated with compromised immune reconstitution or increased viral reactivations, whereas it was associated with a reduced incidence of bacterial infections (RR 0.39, 95% CI 0.21-0.74) and improved overall survival (RR 1.18, 95% CI 1.03-1.34).</p><p><strong>Conclusions: </strong>Despite limitations inherent to retrospective study designs and cohort heterogeneity, these findings indicate that abatacept is an efficacious and safe addition to GvHD prophylaxis in pediatric HSCT.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e70746"},"PeriodicalIF":2.4,"publicationDate":"2026-10-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149168736","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":"Mizoribine in Place of 6-Mercaptopurine for Maintenance in a Child With Relapsed NT5C2-Mutant B-Cell Acute Lymphoblastic Leukemia: A Case Report.","authors":"Jiaqi Qu, Jinfeng Yang","doi":"10.1002/1545-5017.70763","DOIUrl":"https://doi.org/10.1002/1545-5017.70763","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e70763"},"PeriodicalIF":2.4,"publicationDate":"2026-10-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149168516","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":"HOOK3::FGFR1 Fusion in a Background of FGFR1 Amplification in Childhood B-Cell Acute Lymphoblastic Leukemia.","authors":"Aishwarya Karthikeyan, Venus Thakur, Himangi Tak, Shailja Rathore, Anshu Anshu, Praveen Sharma, Richa Jain, Sreejesh Sreedharanunni","doi":"10.1002/1545-5017.70750","DOIUrl":"https://doi.org/10.1002/1545-5017.70750","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e70750"},"PeriodicalIF":2.4,"publicationDate":"2026-10-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149165903","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":"Identifying Patterns of Hospitalizations Among Childhood Cancer Survivors: A Population-Based Study.","authors":"Hai Pham, Teyl Engstrom, Peter Baade, Natalie Bradford, Sarah Pont, Louise Marquart-Wilson, Natasha Nassar, Jason D Pole","doi":"10.1002/1545-5017.70740","DOIUrl":"https://doi.org/10.1002/1545-5017.70740","url":null,"abstract":"<p><strong>Background: </strong>Childhood cancer survivors (CCSs) experience more hospitalizations than the general population. We aimed to identify distinct hospitalization patterns among Queensland CCSs and the associated characteristics.</p><p><strong>Methods: </strong>Cases (aged ≤14 years) diagnosed between 1995 and 2019 while residents of Queensland were identified through the Australian Childhood Cancer Registry and linked with statewide hospital admissions through December 31, 2021 or death. Survivorship was divided into four periods: 0-<3, 3-<5, 5-<10, and ≥10 years post-diagnosis. Latent class analysis identified subgroups on the basis of hospitalization frequency and reasons; demographic and clinical characteristics were compared across subgroups.</p><p><strong>Results: </strong>Among 3252 cases (median follow-up 12 years), distinct hospitalization patterns emerged. High hospitalization rates (adjusted for person-years at risk) in the first 5 years were most common among males, younger age at diagnosis, leukemia diagnoses, and those receiving intensive treatments, such as chemotherapy and stem cell transplants. Between 5 and <10 years after diagnosis, survivors with high hospitalization rates were more likely to have experienced relapses or received stem cell transplants. Beyond 10 years, most survivors had low hospitalization rates, although a very small group (N = 17, 1%) continued to experience elevated rates of hospitalization, relapse, or death.</p><p><strong>Conclusion: </strong>These findings highlight the need for long-term, risk-stratified follow-up care.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e70740"},"PeriodicalIF":2.4,"publicationDate":"2026-10-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149137932","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":"Dose-Capping Glucarpidase in Pediatric Patients for the Management of Delayed Clearance of High-Dose Methotrexate.","authors":"Christopher Phillips, Colleen Koehler, Gianna Galioto","doi":"10.1002/1545-5017.70737","DOIUrl":"https://doi.org/10.1002/1545-5017.70737","url":null,"abstract":"<p><strong>Background: </strong>High-dose methotrexate (HDMTX) can lead to toxicities including nephrotoxicity, myelosuppression, and mucositis. Glucarpidase is a recombinant bacterial enzyme that inactivates extracellular methotrexate (MTX). Prior studies suggest that glucarpidase doses <50 units/kg may effectively decrease serum MTX concentrations. The purpose of this study was to evaluate dose-capping glucarpidase at 1000 units in pediatric and young adults patients.</p><p><strong>Procedure: </strong>This retrospective, single-center study (2013-2024) included glucarpidase administrations of 1000 units after HDMTX administration (doses greater than 500 mg/m²). Glucarpidase doses ≥50 units/kg were excluded. The primary endpoint was the incidence of administrations achieving a clinically significant reduction (>95% reduction) in serum MTX concentrations following dose-capped glucarpidase, at first measurement by high-performance liquid chromatography (HPLC). MTX-related toxicities were reported.</p><p><strong>Results: </strong>Fourteen glucarpidase administrations were included. The median age was 14.3 years and the median glucarpidase dose was 19.2 units/kg. Twelve administrations (86%) had Grade 2-4 acute kidney injury (AKI) prior to glucarpidase. All 12 administrations with available HPLC data (100%) achieved a clinically significant reduction in serum MTX concentrations, with a median reduction of 99.5%. Among administrations with Grade 2-4 AKI, the median time to renal recovery was 6.9 days.</p><p><strong>Conclusions: </strong>Glucarpidase doses capped at 1000 units (<50 units/kg) were associated with clinically significant reductions in serum MTX concentrations. Reduced-dose glucarpidase was generally well tolerated, with no unexpected safety concerns. While dose-capping may be considered in select patients, further prospective, dose-finding studies in humans are needed before employing this approach outside clinical trials.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e70737"},"PeriodicalIF":2.4,"publicationDate":"2026-10-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149137946","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":"Skipping of Exon 38 in Neurofibromatosis Type 1 Gene Is Not a Pathogenic Variant.","authors":"Marzia Ognibene, Maria Cristina Diana, Marco Di Duca, Valeria Capra, Federico Zara, Patrizia De Marco","doi":"10.1002/1545-5017.70745","DOIUrl":"https://doi.org/10.1002/1545-5017.70745","url":null,"abstract":"<p><p>Neurofibromatosis type 1 (NF1) is an autosomal dominant disorder caused by mutations in the NF1 tumor suppressor gene, encoding neurofibromin. Pathogenic variants include point mutations, exon deletions, and splicing alterations, leading to diverse clinical manifestations, including central nervous system tumors. We analyzed cDNA from 32 pediatric and young adult patients, unaffected parents, and controls and observed the constant skipping of exon 38 in NF1 gene. This event, previously considered pathogenic, was detected in both affected and unaffected individuals, suggesting it is a normal alternative transcript rather than a disease-causing variant.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e70745"},"PeriodicalIF":2.4,"publicationDate":"2026-10-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149128129","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":"Routine Serologic Duffy Testing in Pediatric Oncology: Clinical Utility and Feasibility in a Single-Center Cohort.","authors":"Ipsa Mishra, Joshua Glass","doi":"10.1002/1545-5017.70752","DOIUrl":"https://doi.org/10.1002/1545-5017.70752","url":null,"abstract":"<p><strong>Background: </strong>Absolute neutrophil count (ANC) guides chemotherapy dosing, treatment timing, and clinical trial eligibility throughout pediatric oncology. Duffy-null-associated neutrophil count (DANC) is a physiologic variant characterized by lower ANC that may be misinterpreted as treatment-related neutropenia. We evaluated the implementation and clinical impact of routine serologic Duffy antigen testing in a pediatric oncology program.</p><p><strong>Methods: </strong>We conducted a retrospective observational study of pediatric oncology patients who underwent serologic Duffy antigen testing (Fya/Fyb phenotyping) at one institution between January 2023 and June 2026. Implementation outcomes included testing volume, laboratory workflow, turnaround time, and insurance reimbursement. Clinical utility was assessed by changes in interpretation of neutropenia or management following Duffy-null identification. Duffy-null patients were analyzed as an embedded case series.</p><p><strong>Results: </strong>Sixty-three patients underwent testing without additional laboratory infrastructure. Testing was integrated into the institutional blood bank workflow, with a 24-48 h turnaround and insurance coverage. Six patients with high-risk B-cell acute lymphoblastic leukemia (ALL), Down syndrome ALL, acute promyelocytic leukemia, Wilms tumor, pilocytic astrocytoma, and low-grade glioma were identified as Duffy-null. Before testing, these patients experienced chemotherapy delays, antimetabolite dose reductions or holds, prolonged diagnostic evaluation, or uncertainty regarding persistent neutropenia. Recognition of Duffy-null status clarified interpretation of neutropenia and informed management. No infectious complications were observed following Duffy-null identification.</p><p><strong>Conclusions: </strong>Routine serologic Duffy testing was feasible within an existing pediatric oncology program and identified clinically meaningful Duffy-null status across hematologic malignancies, central nervous system (CNS) tumors, and solid tumors. These findings support prospective multicenter evaluation of Duffy-informed ANC interpretation, chemotherapy delivery, and clinical outcomes.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e70752"},"PeriodicalIF":2.4,"publicationDate":"2026-10-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149128073","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":"Obesity's Impact on Prognosis in Ph-Like Acute Lymphoblastic Leukemia.","authors":"Maiah Zarrabi, Erica Ma, Yueh-Yun Chi, Gordana Raca, Steven D Mittelman, Yong-Mi Kim, Etan Orgel","doi":"10.1002/1545-5017.70748","DOIUrl":"https://doi.org/10.1002/1545-5017.70748","url":null,"abstract":"<p><p>Obesity is associated with chemotherapy resistance and poorer survival in pediatric high-risk B-lymphoblastic leukemia (B-ALL). Philadelphia-like (Ph-like) B-ALL is a high-risk subset associated with poor outcomes. Our prior work linked CRLF2-overexpressed Ph-like B-ALL with obesity. However, the prognostic impact of obesity in Ph-like B-ALL has not been defined. Patients with end-of-induction measurable residual disease (EOI MRD)-positivity did poorly regardless of obesity status; however, among patients with EOI MRD-negativity, obesity was associated with inferior event-free survival (EFS) (3-year EFS 59% vs. 100%, p = 0.014). These data support obesity as a poor prognostic factor in Ph-like B-ALL, even among patients achieving early remission.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e70748"},"PeriodicalIF":2.4,"publicationDate":"2026-10-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149128107","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":"Textbook Outcome in Pediatric Nephroblastoma Surgery After Neoadjuvant Chemotherapy: Defining Standards, Introducing Near-Miss, and Predictors of Failure.","authors":"Khalil Ghandour, Hadeel Halalsheh, Tamador Al-Shamaileh, Sidra Tul Montaha, Azza Gharaibeh, Rula Al-Qawabah, Mohammad Salim Omari, Sakher Alshwayyat, Iyad Sultan","doi":"10.1002/1545-5017.70730","DOIUrl":"https://doi.org/10.1002/1545-5017.70730","url":null,"abstract":"<p><strong>Background: </strong>Textbook outcome (TO)-simultaneous achievement of all predefined surgical quality criteria-is established in adult oncological surgery but has never been defined for nephroblastoma surgery after Société Internationale d'Oncologie Pédiatrique (SIOP) neoadjuvant chemotherapy. We defined a nine-criterion TO, reported the TO rate, introduced a near-miss TO (NM-TO) category, and identified predictors of failure.</p><p><strong>Procedure: </strong>Single-center retrospective cohort of pediatric patients below the age of 18 years with nephroblastoma treated at King Hussein Cancer Center between 2015 and 2023. Treatment followed SIOP protocols. The nine TO criteria were as follows: no intraoperative tumor spill, lymph node (LN) harvest ≥7, R0 margin, no major vessel injury, no major organ injury, no Clavien-Dindo ≥3 complication, no unplanned return to the theater within 30 days, adjuvant chemotherapy within 14 days, and estimated blood loss (EBL) <10 mL/kg. NM-TO required exactly 8/9 criteria.</p><p><strong>Results: </strong>Seventy-one patients were analyzed. Full TO was achieved in 35/71 (49.3%); NM-TO in 21 (29.6%), giving a combined rate of 78.9%. The most common NM failures were LN harvest <7 (11 patients) and EBL ≥10 mL/kg (9 patients). Tumor dimension was negatively associated with achieving TO in this univariable, uncorrected analysis (Spearman ρ = -0.405; p < 0.001); stage was not (p = 0.853). Systemic relapse occurred in 12 patients (16.9%); there were no postsurgical local recurrences.</p><p><strong>Conclusions: </strong>This is the first TO analysis in SIOP nephroblastoma surgery. LN harvest is the main modifiable gap; optimizing this single parameter could raise the institutional TO rate to 64.8%. Weight-adjusted EBL adds a pediatric-specific criterion absent from adult TO frameworks. The NM category identifies which single criterion was missed and where to focus quality improvement.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e70730"},"PeriodicalIF":2.4,"publicationDate":"2026-10-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149128070","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}