Pediatric Hematology and Oncology最新文献

{"title":"Current Perspective: The Role of Liquid Biopsy in Diagnosis and Management of Pediatric Rhabdomyosarcomas (RMS).","authors":"Katherine Lehovsky, Christina Putnam, Nigel Hall, Juliet Gray, Zoë S Walters","doi":"10.1080/08880018.2026.2696405","DOIUrl":"10.1080/08880018.2026.2696405","url":null,"abstract":"<p><p>Rhabdomyosarcoma is the third most common extracranial solid tumor of childhood. Despite extensive research over the preceding decades, prognosis in children with metastatic or recurrent disease remains very poor with overall 5-year survival rates under 20%. Liquid biopsy is emerging as an effective, clinically applicable, noninvasive adjunct to conventional oncological management. To date, liquid biopsy has predominantly been applied in the field of adult oncology. Translation into pediatric malignancies, including rhabdomyosarcoma, remains at an early stage with small cohort studies addressing feasibility and technique optimization. This narrative review addresses the current literature in the field of liquid biopsies in rhabdomyosarcoma considering a variety of circulating biomarkers including ctDNA, mRNA, miRNA, proteins, and tumor cells. Liquid biopsies have the potential to revolutionize real-time tumor monitoring and personalized oncological treatment. The results of further large-scale prospective clinical trials in rhabdomyosarcoma are necessary to enable successful integration into standard clinical care.</p>","PeriodicalId":19746,"journal":{"name":"Pediatric Hematology and Oncology","volume":" ","pages":"344-354"},"PeriodicalIF":1.4,"publicationDate":"2026-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148437304","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":"Infection profile and risk factors for adverse outcomes in children with febrile neutropenia: a single-center study from Northwestern India.","authors":"Sagar S Kamat, Kuldeep Singh, Siyaram Didel, Varuna Vyas, Aliza Mittal, Bharat Choudhary, Daisy Khera, Vidhi Jain, Abhishek Purohit, Mithu Banerjee, Henuka Verma, Debasish Barman, Shrawan Choudhary, Sapna Yadav, Kanakam Sai Saranya, Shanto Shila Guha","doi":"10.1080/08880018.2026.2731104","DOIUrl":"10.1080/08880018.2026.2731104","url":null,"abstract":"<p><p>Febrile neutropenia (FN) is a critical medical emergency in pediatric oncology. Our study addressed the lack of research on Indian children with acute leukemia, aiming to improve FN management using region-specific data. This cross-sectional study was conducted in Northwestern India from January 2022 to October 2023. FN episodes that met the inclusion criteria were enrolled in the study. The microbiological spectrum, antibiotic susceptibility patterns, risk factors, prognostic markers, and clinical outcomes were investigated. Logistic regression analysis was used to identify significant predictors of adverse outcomes. In total,207 FN episodes were documented in 90 children. Microbiological confirmation was observed in 17.9% (<i>n</i> = 37) of the FN episodes, and bloodstream infection was present in 10.6% (<i>n</i> = 22). Gram-negative bacteria accounted for 85.2% (<i>n</i> = 23) of the bacterial isolates, the most common being <i>Pseudomonas aeruginosa</i> (<i>n</i> = 7). Invasive fungal disease was observed in 3.4% (<i>n</i> = 7) of episodes. Intensive care was required for management in 14.5% (<i>n</i> = 30) episodes, the commonest indication being septic shock. Mortality during FN episodes was found to be 6.3% (<i>n</i> = 13). Profound neutropenia at presentation was a predictor of prolonged hospital stay. CRP was a predictor of mortality and the need for intensive care. Procalcitonin was a predictor of all adverse outcomes explored in the study-bloodstream infection, mortality, need for intensive care, and prolonged hospital stay. According to the available data, gram-negative organisms dominate bloodstream infections in this part of the world. Procalcitonin is a valuable predictor of adverse outcomes in FN, guiding early antibiotic escalation to improve outcomes.</p>","PeriodicalId":19746,"journal":{"name":"Pediatric Hematology and Oncology","volume":" ","pages":"455-465"},"PeriodicalIF":1.4,"publicationDate":"2026-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148977642","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":"The burden of childhood cancer in Nigeria - a focus on leukemia and lymphoma: epidemiology, biology, and health system challenges.","authors":"Ogochukwu O Izuegbuna","doi":"10.1080/08880018.2026.2701081","DOIUrl":"10.1080/08880018.2026.2701081","url":null,"abstract":"<p><p>Childhood cancer represents an increasingly recognized priority in global health, with the World Health Organization's Global Initiative for Childhood Cancer aiming to achieve 60% survival for six index cancers by 2030. In Nigeria, leukemia and lymphoma constitute a substantial proportion of pediatric malignancies, yet outcomes remain dramatically poorer than in high-income countries. This review examines the current state of childhood leukemia and lymphoma in Nigeria, synthesizing available epidemiological data, discussing unique biological features, and analyzing the multifactorial challenges that perpetuate poor survival. Treatment abandonment exceeds 37%, mortality approaches 32%, and critical infrastructure gaps-including limited radiotherapy access, absence of bone marrow transplantation, and inadequate diagnostic capacity-compound the problem. The WHO's interventions through the Global Initiative and technical support for registry development offer frameworks for progress. Future perspectives emphasize the necessity of population-based cancer registries, expanded health insurance coverage, investment in regional diagnostic and treatment centers, and sustainable financing mechanisms to bridge the survival gap.</p>","PeriodicalId":19746,"journal":{"name":"Pediatric Hematology and Oncology","volume":" ","pages":"355-365"},"PeriodicalIF":1.4,"publicationDate":"2026-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148437336","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":"Telemedicine in pediatric oncology during the COVID-19 pandemic: a cross-sectional questionnaire-based study evaluating the patient perspective.","authors":"Fabian-Simon Frielitz, Annika Verena Rademacher, Britta Exner, Jana Dördelmann, Denis Martin Schewe","doi":"10.1080/08880018.2026.2725653","DOIUrl":"10.1080/08880018.2026.2725653","url":null,"abstract":"<p><p>The COVID-19 pandemic was a major driver for the integration of telemedicine in many different medical fields, but applications in pediatric oncology in Germany remain scarce. This study evaluated the perceptions of benefits and limitations of telemedicine among pediatric oncology patients and their families during intensive cancer treatment prior to its implementation. A cross-sectional questionnaire survey was conducted at the University Hospital Schleswig-Holstein, Campus Kiel, between February and April 2021 during the COVID-19 lockdown, with responses from 119 participants. Factors such as travel time, time at home, COVID-19 exposure, and potential impacts on diagnostics and psychosocial support were assessed. Overall, telemedicine was viewed favorably, with advantages rated higher than disadvantages by both caregivers and patients (mean score 3.79 vs. 3.36, <i>p</i> < 0.01). Key benefits included reduced travel and waiting times, more time at home, and lower COVID-19 exposure, each with mean scores above 4. Reported disadvantages were the lack of on-site physical examinations, absence of blood testing, and reduced social interaction with hospital staff, other patients, and their families. These findings suggest generally positive perceptions of telemedicine in pediatric oncology care, and indicate that a hybrid model combining remote and in-person visits could potentially address diagnostic and social needs in this vulnerable patient group.</p>","PeriodicalId":19746,"journal":{"name":"Pediatric Hematology and Oncology","volume":" ","pages":"383-394"},"PeriodicalIF":1.4,"publicationDate":"2026-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148888119","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":"Prevalence of High Tumor Mutational Burden Among Pediatric Patients With Solid Tumors.","authors":"Wenjun Zhong, E J Dettman, Lei Zhang, Alexander Gozman, Roman Groisberg, Razvan Cristescu, Matthew J Marton, Saumya D Sisoudiya, Meagan Montesion, Karthikeyan Murugesan, Irene M Shui","doi":"10.1080/08880018.2026.2714775","DOIUrl":"10.1080/08880018.2026.2714775","url":null,"abstract":"<p><p>High tumor mutational burden (TMB-H), defined as ≥10 non-driver somatic mutations per mega base of genome sequenced (mut/Mb), and high microsatellite instability (MSI-H), which can lead to TMB-H, are both associated with response to the immunotherapy pembrolizumab. Data from the KEYNOTE-158 study demonstrated that the association between TMB-H and response to pembrolizumab is independent of MSI-H status. Because data in children are limited, the prevalence of TMB-H in pediatric solid tumors is unclear. This study sought to examine TMB-H prevalence across solid tumor types that are most common among pediatric tumors using real-world data. This retrospective, cross-sectional study included data from FoundationCORE^® solid tumor biopsies that were sequenced by comprehensive genomic profiling between 2013 and 2023. All patients were <18 years of age at the time of specimen collection. TMB-H prevalence was assessed among patients with non-MSI-H tumors (including those with MSI-low and microsatellite-stable tumors) and by key patient demographics and disease characteristics. Among 3533 patients with non-MSI-H solid tumors, TMB-H prevalence was 1.42% and was significantly higher in children aged 12-17 years than in children aged <12 years (2.17% vs 0.98%; <i>p</i> = 0.006). There were no significant differences based on sex, genomic ancestry, tissue of origin, disease stage at testing, or whether the tissue sample was from a metastatic versus nonmetastatic site. Prevalence varied by tumor type but was <3% across subgroups, including glioma, central nervous system non-glioma, and peripheral nervous system tumors. Overall, TMB-H prevalence was low across solid tumor types that are frequently observed in children.</p>","PeriodicalId":19746,"journal":{"name":"Pediatric Hematology and Oncology","volume":" ","pages":"445-454"},"PeriodicalIF":1.4,"publicationDate":"2026-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148865859","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":"Parental and Patients' Awareness, Knowledge, and Willingness for Human Papillomavirus Vaccination Among Survivors of Childhood Hematologic Malignancies.","authors":"Damla Seyhanlı, Eda Karadag-Oncel, Ayşe Cakıl-Guzın, Şilem Ozdem-Alatas, EsmaTuğba Kasıkcı-Mermer, Osman Tolga İnce, Şebnem Yılmaz, Özlem Tufekcı-Gurocak, Hale Oren, Nurşen Belet","doi":"10.1080/08880018.2026.2685015","DOIUrl":"10.1080/08880018.2026.2685015","url":null,"abstract":"<p><p>In childhood, cancer survivors are at a higher risk of developing secondary cancers, including human papillomavirus (HPV)-related malignancies, compared with the general population. In Turkey, HPV vaccines are not included in the National Immunization Program (NIP). This study assessed the knowledge, awareness of HPV, and the willingness for HPV vaccines among survivors of childhood hematologic malignancies and their parents. This cross-sectional study was conducted with 156 parents and their children (age 9-18 years), in accordance with the sample size calculation. A structured questionnaire prepared for both parents (19 questions) and children (15 questions) was employed for data collection, and the data were gathered through face-to-face interviews or by phone. Regarding the questions evaluating knowledge of HPV, 34% of the parents and 29.5% of the children participating in the study had heard of HPV and the HPV vaccine. The most important sources of information for parents were physicians (43.4%) and television (26.4%), while for children it was social media (52.7%) and physicians (21.7%). HPV vaccination rates were very low in both parents and children (3.2% and 2.6%, respectively). The most common reasons for not receiving the vaccine were the lack of a physician's recommendation and insufficient information in both groups. Logistic regression analysis evaluating factors associated with HPV awareness revealed that high educational level (OR: 2.85 [95% CI: 1.22-6.61]; <i>p</i> = 0.011), receipt of privately administered vaccines (not including in NIP) (OR: 2.45 [95% CI: 1.35-4.45]; <i>p</i> = 0.004, and female sex (OR: 1.97 [95% CI: 1.19-3.26], <i>p</i> = 0.007) were significant predictors. Raising awareness about HPV vaccines is essential to improve immunization coverage especially in survivors of hematologic cancers. Efforts are required to strengthen the training of different departments and encourage their collaboration and increase vaccination rates.</p>","PeriodicalId":19746,"journal":{"name":"Pediatric Hematology and Oncology","volume":" ","pages":"254-267"},"PeriodicalIF":1.4,"publicationDate":"2026-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148295443","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":"Chimerism kinetics in pediatric hematopoietic stem cell transplantation: from biological basis to advanced monitoring technologies.","authors":"Prasad Iyer, Satyendra Katewa","doi":"10.1080/08880018.2026.2634279","DOIUrl":"10.1080/08880018.2026.2634279","url":null,"abstract":"<p><p>Monitoring donor-recipient chimerism following hematopoietic stem cell transplantation (HSCT) is essential for assessing engraftment success, early identification of graft failure and relapse, and guiding immunomodulating interventions. Recent advancements in molecular technologies, particularly digital droplet PCR (ddPCR) and next-generation sequencing (NGS), have markedly improved the sensitivity and precision of chimerism detection, allowing the identification of recipient cells at levels below 0.1%. These advancements enable more accurate and dynamic monitoring compared to traditional methods, which are limited in terms of sensitivity and specificity. In pediatric patients, the interpretation of chimerism results is complicated by unique factors, including thymic recovery, exposure to serotherapy agents such as ATG or alemtuzumab, and constraints related to small blood volume. These factors affect the kinetics of donor cell engraftment and the dynamics of mixed chimerism in different hematopoietic lineages. This review presents the current understanding of the biological basis of chimerism, compares traditional and advanced detection methodologies, and details lineage-specific chimerism kinetics in children. It also provides practical guidance for interpreting serial chimerism data to inform timely and preemptive clinical interventions. Owing to the limited pediatric-specific data, adult findings were integrated where appropriate, underscoring the urgent need for pediatric validation, assay harmonization, and standardized implementation protocols to optimize transplant outcomes in children.</p>","PeriodicalId":19746,"journal":{"name":"Pediatric Hematology and Oncology","volume":" ","pages":"291-305"},"PeriodicalIF":1.4,"publicationDate":"2026-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147777946","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":"How we Investigate Bone Marrow Failure Syndromes in Pediatric Patients.","authors":"Marilia Bazzo Catto, David J Young, Luiz Fernando Bazzo Catto","doi":"10.1080/08880018.2026.2676558","DOIUrl":"10.1080/08880018.2026.2676558","url":null,"abstract":"<p><p>Bone marrow failure syndromes (BMFS) encompass a spectrum of acquired and constitutional disorders characterized by impaired hematopoiesis and cytopenias. In pediatric patients, constitutional BMFS represent up to 50% of cases, yet their diagnosis remains challenging due to subtle or absent extra-hematologic manifestations that mimic immune aplastic anemia. Accurate differentiation is essential, as it directly influences prognosis, therapeutic strategy, surveillance for extra-hematopoietic complications, and donor selection for hematopoietic cell transplantation. This review presents a structured diagnostic framework for pediatric BMFS, integrating clinical history, physical examination, bone marrow morphology, flow cytometry, cytogenetic analysis, disease-specific functional assays, and next-generation sequencing (NGS). Chromosomal breakage testing remains the gold-standard assay for Fanconi anemia, while telomere length measurement is the strongest predictor for differentiating constitutional from immune BMF. NGS enables comprehensive germline variant detection, resolves diagnostically ambiguous presentations, and informs genotype-guided familial screening. Recognition of disease-specific patterns of clonal hematopoiesis further refines diagnostic precision and risk stratification. A stepwise, evidence-based approach is essential to optimize early diagnosis and long-term outcomes in pediatric patients with BMFS.</p>","PeriodicalId":19746,"journal":{"name":"Pediatric Hematology and Oncology","volume":" ","pages":"268-290"},"PeriodicalIF":1.4,"publicationDate":"2026-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148138652","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":"Feasibility and Validity of the 6-Minute Cycling Test in Childhood Cancer Patients.","authors":"Lena Wypyrsczyk, Mareike Kühn, Elias Dreismickenbecker, Marie A Neu, Heidi Diel, Joerg Faber","doi":"10.1080/08880018.2026.2661758","DOIUrl":"10.1080/08880018.2026.2661758","url":null,"abstract":"<p><p>The 6-min cycling test (6MCT), a submaximal endurance test, has not yet been applied in pediatric oncology. This study evaluates its feasibility and validity in 71 childhood cancer patients (9.6 ± 4.0 years). For validation, 46 patients additionally underwent cardiopulmonary exercise testing (CPET). Performance in the 6MCT (total revolutions) was correlated with peak oxygen uptake (VO₂_peak) and peak work rate (WR_peak) using Spearman's correlation. Linear regressions assessed the predictive value of VO₂_peak and WR_peak on 6MCT performance. Sixty-six participants (93%) successfully completed the 6MCT, averaging 550 ± 129 revolutions. Revolutions correlated moderately with VO₂_peak (<i>ρ</i> = 0.46, <i>p</i> = 0.001) and strongly with WR_peak (<i>ρ</i> = 0.64, <i>p</i> < 0.001). VO₂_peak significantly predicted 6MCT performance (<i>p</i> = 0.001, <i>R</i>² = 0.214), whereas WR_peak explained more variance (<i>p</i> < 0.001, <i>R</i>² = 0.488). The results demonstrate that the 6MCT is a feasible, valid endurance assessment in this population, offering a promising alternative when gold standard testing is not available.</p>","PeriodicalId":19746,"journal":{"name":"Pediatric Hematology and Oncology","volume":" ","pages":"227-240"},"PeriodicalIF":1.4,"publicationDate":"2026-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147777953","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":"Prepubertal Initiation of Imatinib is Associated with Reduced Final Height in Children with Chronic Myeloid Leukemia.","authors":"Rohit Sadanand, Rama Walia, Swetha Palla, Naresh Sachdeva, Harvinder Kaur, Shano Naseem, Richa Jain, Amita Trehan, Deepak Bansal","doi":"10.1080/08880018.2026.2680922","DOIUrl":"10.1080/08880018.2026.2680922","url":null,"abstract":"<p><p>Imatinib in children with chronic myeloid leukemia (CML) is associated with growth deceleration. However, the long-term impact on final height remains less clear. The aim was to evaluate the effect of prolonged imatinib on linear growth at skeletal maturity. A cross-sectional study was conducted at a single center (2020-2021). Patients with chronic-phase CML on imatinib, diagnosed before 13 years and who had attained skeletal maturity at enrollment, were included. Anthropometry, sexual maturity rating, bone age, and evaluation for causes of short stature were performed. Longitudinal height data were retrieved from clinic records and compared with population-specific growth charts. Of 46 screened patients, 13 fulfilled the inclusion criteria. Mean age at diagnosis and enrollment was 9.3 ± 2.3 and 23.7 ± 2.6 years. The median duration of imatinib therapy was 14.3 years (IQR: 14.2-16), with 184.4 patient-years of follow-up. Mean height z-score declined from -0.6 ± 1.2 at diagnosis to -1.1 ± 0.9 at maturity (<i>p</i> = 0.04). In those administered imatinib before the onset of pubertal growth spurt, the decline was significant (-0.5 ± 0.6 to -1.3 ± 0.6, <i>p</i> = 0.03), compared to those administered after the pubertal growth spurt (<i>p</i> = 0.60). In one of the longest follow-up cohorts of children with CML reported to date, imatinib resulted in growth deceleration, particularly when initiated before the onset of pubertal growth spurt. Despite catch-up growth during adolescence, final height z-scores remained reduced at skeletal maturity.</p>","PeriodicalId":19746,"journal":{"name":"Pediatric Hematology and Oncology","volume":" ","pages":"241-253"},"PeriodicalIF":1.4,"publicationDate":"2026-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148138681","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}