Pediatric Blood & Cancer最新文献

{"title":"Integration of Metabolic Profiling and Functional Genomics Suggests IGJ as a Driver of Chemoresistance in B-ALL","authors":"Karen Lizeth Pachón-Meza,&nbsp;Camilo Ernesto Moreno-Cristancho,&nbsp;José Luis Padilla-Agudelo,&nbsp;Ana Isabel Ramos-Murillo,&nbsp;Diana Londoño-Barbosa,&nbsp;Gustavo Salguero,&nbsp;Sandra Milena Sanabria-Barrera,&nbsp;Claudia Chica,&nbsp;Nataly Cruz-Rodriguez,&nbsp;Rubén Darío Godoy-Silva,&nbsp;José Arturo Gutiérrez-Triana","doi":"10.1002/pbc.32068","DOIUrl":"10.1002/pbc.32068","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background</h3>\u0000 \u0000 <p>B-cell precursor acute lymphoblastic leukemia (B-ALL) is a hematologic malignancy characterized by the uncontrolled proliferation of immature B lymphoblasts. Despite significant advancements in treatment, chemoresistance remains a major challenge. Previous studies, including those involving Colombian patient cohorts, have identified a gene signature involving <i>ID1, ID3</i>, and <i>IGJ</i>, which correlates with poor prognosis; however, the underlying mechanisms remain unclear. This study integrates metabolic profiling with gene expression analysis of <i>ID1, ID3</i>, and <i>IGJ</i> in patient-derived lymphoblasts. It explores the role of <i>IGJ</i> overexpression in NALM-6 cells to assess its potential contribution to chemoresistance.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>Bone marrow samples from 33 newly diagnosed B-ALL patients were analyzed for <i>ID1, ID3</i>, and <i>IGJ</i> gene expression. Seahorse XF metabolic assays were conducted on 13 patient samples to assess mitochondrial respiration and glycolytic function. Functional studies were performed in the NALM-6 B-ALL cell line using CRISPRa-mediated <i>IGJ</i> overexpression, followed by metabolic and chemoresistance assays using resazurin-based viability testing with key chemotherapeutic agents.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Patient-derived lymphoblasts exhibited a quiescent metabolic phenotype, with two distinct metabolic subgroups based on oxygen consumption and glycolysis rates. Higher <i>IGJ</i> expression was significantly associated with the glycolytic subgroup and correlated with worse event-free survival (EFS, <i>p</i> = 0.0179) and overall survival (OS, <i>p</i> = 0.0205). In NALM-6 cells, <i>IGJ</i> overexpression led to increased metabolic activity and conferred resistance to dexamethasone, cytarabine, doxorubicin, and methotrexate, but not cyclophosphamide.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p><i>IGJ</i> upregulation promotes metabolic reprogramming and chemoresistance in B-ALL, suggesting a potential role for <i>IGJ</i> as a biomarker and therapeutic target in overcoming treatment resistance. These findings provide new insights into the metabolic mechanisms underlying B-ALL progression and highlight <i>IGJ</i> as a candidate for future targeted interventions.</p>\u0000 </section>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 12","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-10-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145233268","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":"Clinical and Economic Impact of the Mexico in Alliance With St. Jude Golden Hour Collaborative in a Mexican Hospital","authors":"Norma Araceli López Facundo,&nbsp;Ana Alcántara Garduño B,&nbsp;Ivonne Esteban,&nbsp;Martha Cervantes Jimenez,&nbsp;Edith De La Torre Sánchez,&nbsp;Elena Gavia Encastin,&nbsp;Nanci Reyes Felipe,&nbsp;Concepción Hernández Cruz,&nbsp;Karla Ivonne González Hernández,&nbsp;Erika Patricia Buendía Tinoco,&nbsp;Yolanda Tejeda,&nbsp;Karen Tomasini Padilla,&nbsp;Isidoro Tejocote,&nbsp;Cecilia Rodríguez Castillejos,&nbsp;Roberto Sánchez Suárez,&nbsp;Jocelyn Becerril Becerril,&nbsp;Jafet Arrieta,&nbsp;Naomi Echandia-Abud,&nbsp;Paola Friedrich,&nbsp;Julia Esther Colunga Pedraza","doi":"10.1002/pbc.32075","DOIUrl":"10.1002/pbc.32075","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background</h3>\u0000 \u0000 <p>In low- and middle-income countries, infections remain the leading cause of treatment-related mortality in pediatric patients with cancer. To address this, the Golden Hour Collaborative—developed in Mexico in alliance with St. Jude—was designed to promote timely antibiotic administration for febrile pediatric hemato-oncology patients through a multidisciplinary approach, continuous caregiver and staff training, standardized protocols, and supply availability. This study evaluates the clinical and economic impact of implementing the MAS Collaborative at the ISSEMyM Maternal–Infant Hospital.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Procedure</h3>\u0000 \u0000 <p>A prospective cohort study with historical controls was conducted. Outcomes were analyzed with nonparametric tests. Costs per febrile neutropenia (FN) episode were estimated using official state cost tables (adjusted to 2025 USD).</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>A total of 160 episodes were analyzed: 80 in the pre-intervention period (2015–2018) and 80 in the post-intervention period (2019–2022). After implementation, the proportion of patients receiving antibiotics within 60 min increased from 3.8% to 98% (median time-to-antibiotic (TTA) 198 vs. 42 min, <i>p</i> &lt; 0.001). Outcomes improved with shorter hospital stays (9 vs. 8 days, <i>p</i> = 0.03), reduced pediatric intensive care unit (PICU) admissions (20% vs. 7.5%, OR 0.32, <i>p</i> = 0.02), decreased septic shock (25% vs. 8.7%), and no infection-related deaths post-intervention (vs. 7.5% pre-intervention). Discharge without complications rose from 50% to 83%. Mean healthcare cost decreased from USD 8,580 to USD 2,835.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>In this resource-limited setting, the MAS Collaborative shows that multidisciplinary, context-adapted interventions can improve timeliness of care, reduce complications and mortality, and lower short-term costs, highlighting their potential for scalability in LMICs.</p>\u0000 </section>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 12","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-10-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145233308","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":"Synchronous Mediastinal Germ Cell Tumor with Sarcomatous Transformation and Acute Myeloid Leukemia","authors":"Elysia Cohen,&nbsp;Olayinka Okeleji,&nbsp;David McCall,&nbsp;Miriam Garcia,&nbsp;Cesar Nunez,&nbsp;Branko Cuglievan,&nbsp;Cynthia Herzog,&nbsp;Shehla Razvi,&nbsp;Priti Tewari,&nbsp;Harrison Douglas,&nbsp;Amber Gibson","doi":"10.1002/pbc.32092","DOIUrl":"10.1002/pbc.32092","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 12","pages":""},"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":"Two Surveys, 16 Years Apart: Comparing Traditional and Complementary Medicine Use by Children With Cancer in Jordan","authors":"Murad Sawalha,&nbsp;Anwar Alnaasan,&nbsp;Anas Alsharawneh,&nbsp;Ahmed Farrag,&nbsp;Mohammad R. Alqudimat","doi":"10.1002/pbc.32096","DOIUrl":"10.1002/pbc.32096","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background</h3>\u0000 \u0000 <p>Traditional and complementary medicine (T&amp;CM) is commonly used by children with cancer alongside conventional treatments. However, data on its use in Jordan remain limited. This study aimed to assess the current prevalence, types, motivations, and disclosure rates of T&amp;CM use in Jordan, and to compare these findings with those from the original 2008 study conducted in the same setting.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>A cross-sectional study was conducted in the pediatric oncology department at King Hussein Cancer Center between November 2023 and May 2024. A brief proxy-report T&amp;CM questionnaire with evidence of validity and reliability was used.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>A total of 85 patients participated (response rate = 85%). Overall, 29.4% reported T&amp;CM use, primarily for perceived safety (52%), managing cancer-related symptoms (44%), and potential therapeutic benefit (40%). Nutritional (68%) and psychological (24%) approaches were the most used modalities. Disclosure of T&amp;CM use to healthcare providers was reported by 75% of users. Reasons for non-use included a lack of recommendation by healthcare providers and limited awareness. A statistically significant association was found between T&amp;CM use and current therapy stage, with higher use among patients in active treatment. No other demographic or clinical variables were significantly associated with T&amp;CM use.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>Compared to earlier findings, this study shows a lower prevalence of T&amp;CM use and a higher disclosure rate, suggesting improved communication and awareness. Parents used T&amp;CM to complement conventional care rather than as an alternative. These findings underscore the importance of integrating discussions about T&amp;CM into pediatric oncology care to support safe and informed use.</p>\u0000 </section>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 12","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-10-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145225709","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":"Chemo-Immunotherapy Rescue for High-Risk Neuroblastoma Patients With Progressive Disease Before High-Dose Chemotherapy: Real-World Data From the SACHA-France Study","authors":"Claudia Pasqualini,&nbsp;Stéphanie Proust,&nbsp;Gudrun Schleiermacher,&nbsp;Marion Gambart,&nbsp;Sarah Jannier,&nbsp;Arnaud Petit,&nbsp;Chrystelle Dupraz,&nbsp;Estelle Thebaud,&nbsp;Yves Reguerre,&nbsp;Lee Aymar Ndounga-Diakou,&nbsp;Salim Laghouat,&nbsp;Anne Sophie Defachelles,&nbsp;Pablo Berlanga","doi":"10.1002/pbc.32080","DOIUrl":"10.1002/pbc.32080","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background</h3>\u0000 \u0000 <p>Patients with high-risk neuroblastoma (HR-NBL) who experience disease progression (PD) during first-line treatment prior to high-dose chemotherapy (HDC) represent a rare and understudied subgroup, for whom treatment strategies are poorly defined and prognosis appears to be extremely poor.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Aims</h3>\u0000 \u0000 <p>We report real-world data on the off-label use of chemo-immunotherapy for HR-NBL patients with PD before HDC. The primary endpoint of our analysis is the best response during the chemo-immunotherapy treatment.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>The SACHA-France registry prospectively documents safety and efficacy data on compassionate/off-label treatments for patients of ≤25 years old (NCT04477681).</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Between January 2020 and September 2024, 13 patients with HR-NBL received chemo-immunotherapy due to PD before HDC, and were included in SACHA-France. They had a median age of 3.4 years (1.3–8.0). Six patients had NMYC-amplified disease. Five PD occurred during/end-of-induction chemotherapy, and eight during temozolomide-based chemotherapy after initial insufficient metastatic response or toxicity. All but one had metastatic progression. The chemo-immunotherapy consisted of topotecan–cyclophosphamide (<i>n</i> = 9) or temozolomide–irinotecan (<i>n</i> = 4), combined with dinutuximab beta (dB) for a maximum of six cycles. Objective responses (ORs) were seen in five of 13 patients (38%)—four partial responses (PR) and one complete response (CR). Three out of the five patients with PD during/end of induction had PR, including two with NMYC-amplified tumors. Overall, six patients underwent tandem HDC, with two remaining progression-free after 1.7 and 2.1 years, and one remaining disease-free at 3.4 years.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>Chemo-immunotherapy can benefit HR-NBL patients with PD before HDC, including those with progression during/at the end of the induction chemotherapy and NMYC amplification. These findings support its early inclusion in HR-NBL trials.</p>\u0000 </section>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 12","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-10-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/pbc.32080","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145225764","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":"Association of Food Insecurity With Disease-Related Complications and Healthcare Utilization for Patients With Sickle Cell Disease.","authors":"Sharjeel Syed, Andrew Palmer, Austin Wesevich, Kristen Wroblewski, Gabrielle Lapping-Carr, Radhika Peddinti, Wendy S Darlington","doi":"10.1002/pbc.32100","DOIUrl":"https://doi.org/10.1002/pbc.32100","url":null,"abstract":"<p><strong>Background: </strong>Food insecurity is one of several household material hardships (HMH) recognized for their impact on disease severity and healthcare utilization in patients with chronic disease. Sickle cell disease (SCD) is a chronic disease, disproportionately affecting patients of lower socioeconomic status, that results in high rates of disease-related complications and high healthcare utilization. This study examines the relationship between food insecurity and SCD-related complications and healthcare utilization.</p><p><strong>Methods: </strong>Patients (ages 2-24 years) and/or their parents were surveyed to assess food insecurity status during routine SCD clinic visits from July 2015 to July 2019. Food insecurity status was assessed using the United States Department of Agriculture (USDA) Food Security Short Form (six-item), with a lookback period of 12 months. Sociodemographic characteristics, disease-related complications, and healthcare utilization were abstracted from electronic health records.</p><p><strong>Results: </strong>Overall, 22% (n = 25) of participant households were food insecure. Food insecurity was associated with significantly higher annual rates of acute chest syndrome (aOR = 3.12, 95% CI: 1.27-7.67), prevalence of cholecystectomy (aOR = 6.29, 95% CI: 1.66-23.80), and a higher number of hospitalizations (aOR = 2.40, 95% CI: 1.04-5.52).</p><p><strong>Conclusions: </strong>The rates of food insecurity among sampled households of pediatric and young adult patients with SCD were much higher than national (13.4%) and local county rates (13%). Food insecurity was independently associated with more disease-related complications and higher healthcare utilization even after adjusting for age, sex, and sickle cell type. These results suggest food insecurity may be a modifiable contributor impacting morbidity in patients with SCD and should prompt further study into these relationships.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e32100"},"PeriodicalIF":2.3,"publicationDate":"2025-10-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145225702","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,&nbsp;Dániel J. Erdélyi,&nbsp;Volkan Hazar,&nbsp;Mirella Ampatzidou,&nbsp;Boryana Avramova,&nbsp;Anca Colita,&nbsp;Monica Makiya,&nbsp;Sophia Polychronopoulou,&nbsp;Bence Hőbör,&nbsp;Tomaz Prelog,&nbsp;Andrea Reyes,&nbsp;Juan Tordecilla,&nbsp;Koray Yalçin,&nbsp;Arend von Stackelberg,&nbsp;Janez Jazbec","doi":"10.1002/pbc.32063","DOIUrl":"10.1002/pbc.32063","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background</h3>\u0000 \u0000 <p>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>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Objectives</h3>\u0000 \u0000 <p>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>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>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>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>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 &lt; 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>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusions</h3>\u0000 \u0000 <p>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>\u0000 </section>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 12","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-10-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/pbc.32063","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":"OA","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,&nbsp;Qing Cao,&nbsp;Melissa M. Hudson,&nbsp;Jiaoyang Cai","doi":"10.1002/pbc.32095","DOIUrl":"10.1002/pbc.32095","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 12","pages":""},"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":"Pravastatin for Radiation-Induced Fibrosis of the Extremity: A Single-Center Experience","authors":"Rachel Offenbacher,&nbsp;Lara Fabish,&nbsp;Alice Lee,&nbsp;Rui Yang,&nbsp;Bang Hoang,&nbsp;David S. Geller,&nbsp;Jana L. Fox,&nbsp;David M. Loeb,&nbsp;Alissa Baker","doi":"10.1002/pbc.32084","DOIUrl":"10.1002/pbc.32084","url":null,"abstract":"<div>\u0000 \u0000 <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>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 12","pages":""},"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,&nbsp;Keita Terui,&nbsp;Katsuhiro Nishimura,&nbsp;Daisuke Katsumi,&nbsp;Ryoya Furugane,&nbsp;Ayako Takenouchi,&nbsp;Shugo Komatsu,&nbsp;Satoru Oita,&nbsp;Yunosuke Kawaguchi,&nbsp;Tomoro Hishiki","doi":"10.1002/pbc.32082","DOIUrl":"10.1002/pbc.32082","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background</h3>\u0000 \u0000 <p>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>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>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>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Cluster analysis performed using <i>z</i>-scores for SMI and BMI yielded four clusters: Cluster 1 had high <i>z</i>-scores for both indices; Cluster 2 had average <i>z</i>-scores for both indices; Cluster 3 had low <i>z</i>-scores for SMI and average <i>z</i>-scores for BMI; and Cluster 4 had low <i>z</i>-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>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>The potential prognoses of the four distinct patient clusters based on the <i>z</i>-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>\u0000 </section>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 12","pages":""},"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}