Pediatric Hematology and Oncology最新文献

{"title":"Effect of Conditioning Regimens on Glucose Metabolism in Children Undergoing Hematopoietic Stem Cell Transplantation.","authors":"Eymen Yilmaz, Baris Malbora, Gonul Buyukyilmaz, Dilek Kacar, Dilek Gürlek Gökçebay, Namık Yasar Ozbek","doi":"10.1080/08880018.2026.2656674","DOIUrl":"10.1080/08880018.2026.2656674","url":null,"abstract":"<p><p>Hematopoietic stem cell transplantation (HSCT) has greatly improved the survival of children with leukemia; however, long-term adverse effects remain a concern. This study aims to compare the effects of total body irradiation (TBI)-based and chemotherapy-based conditioning regimens on insulin resistance in children undergoing HSCT. Patients under 18 who underwent HSCT between April 2010 and October 2023 were evaluated. Seventy-six patients (M/F:48/28) with leukemia were included in this prospective descriptive cross-sectional study. Clinical and laboratory data, including oral glucose tolerance test (OGTT), were collected. The TBI group consisted of 45 patients with ALL (66.1%), and the non-TBI group consisted of 31 patients, including 23 with ALL and 8 with AML. The median time from HSCT to OGTT was 2.7 years (IQR, 1.8-3.8) and did not differ between the two groups. Body mass index and serum lipids, except HDL cholesterol, did not differ between the TBI and non-TBI groups. Twelve patients (26.6%) in the TBI group and 6 patients (19.3%) in the non-TBI group had impaired OGTT results (<i>p</i> > 0.05). All four obese patients had normal glucose tolerance. The TBI group had significantly higher peak and total insulin levels during the OGTT than the non-TBI group (<i>p</i> = 0.02, <i>p</i> = 0.02, respectively). Although HbA1c and HOMA-IR did not differ between the two groups, the Insulin Sensitivity Index (ISI) was significantly lower in the TBI group, indicating reduced peripheral insulin sensitivity (<i>p</i> = 0.04). TBI is a risk factor for reduced insulin sensitivity in pediatric HSCT survivors, which is a risk for type 2 diabetes and cardiovascular disease without leading to obesity.</p>","PeriodicalId":19746,"journal":{"name":"Pediatric Hematology and Oncology","volume":" ","pages":"201-212"},"PeriodicalIF":1.4,"publicationDate":"2026-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147777964","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 influence of elevated body mass index on protein expression in pediatric b-cell acute lymphoblastic leukemia.","authors":"Rachel S Werk, Jeremy M Chacón, Lucie M Turcotte, Justin R Ryder","doi":"10.1080/08880018.2026.2661067","DOIUrl":"10.1080/08880018.2026.2661067","url":null,"abstract":"<p><p>This study explored protein expression in B-cell acute lymphoblastic leukemia (B-ALL) patients with and without elevated weight or obesity and controls to understand global proteomic differences between newly diagnosed B-ALL patients and controls as well as the influences of elevated body mass index (BMI) on pretreatment inflammatory and immune-related protein expression in B-ALL patients.   Protein expression was measured in serum samples of pediatric patients (aged 1-21 years) with newly diagnosed B-ALL (<i>n</i> = 39), and age and sex-matched controls (<i>n</i> = 41) using OLink panels. We examined normalized protein expression data clustered by patient information in -unsupervised hierarchical clustering and principal component analysis. Of 239 assays, 128 assays differed significantly based on B-ALL diagnosis and 4 assays (APLP1, CDHR5, GHRL, SEZ6L) varied significantly as a function of BMI. In healthy individuals, oncology marker furin (p.adjust = 0.016) was more highly expressed in the high-BMI category; this trend was reversed for B-ALL individuals. Furin expression is often upregulated in malignancies and obesity; however this suggests its expression may follow unique patterns in pediatric B- ALL patients with elevated BMI.</p>","PeriodicalId":19746,"journal":{"name":"Pediatric Hematology and Oncology","volume":" ","pages":"213-226"},"PeriodicalIF":1.4,"publicationDate":"2026-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147778031","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":"Association of Tight Blood Pressure Control with PRES Prevention in Pediatric SCD Undergoing HSCT.","authors":"Anandini Rao, Holly Miller, Dana Salzberg, Kristen Beebe, Roberta H Adams, Stephanie Hsieh, Alexander Ngwube","doi":"10.1080/08880018.2026.2659153","DOIUrl":"10.1080/08880018.2026.2659153","url":null,"abstract":"","PeriodicalId":19746,"journal":{"name":"Pediatric Hematology and Oncology","volume":" ","pages":"193-197"},"PeriodicalIF":1.2,"publicationDate":"2026-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147691293","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":"Nationwide survey of ruxolitinib administration for Japanese children with hemophagocytic lymphohistiocytosis.","authors":"Katsuyuki Tanaka, Rintaro Ono, Takehiko Doi, Satoshi Miyamoto, Shiho Yasue, Shinsuke Kataoka, Eri Okura, Toshihiro Fujiki, Masahiro Ueki, Yoshihiro Gocho, Takayuki Takachi, Akira Morimoto, Hirokazu Kanegane, Yoko Shioda, Kenichi Sakamoto","doi":"10.1080/08880018.2026.2653020","DOIUrl":"10.1080/08880018.2026.2653020","url":null,"abstract":"<p><p>In the treatment of hemophagocytic lymphohistiocytosis (HLH), the effectiveness of ruxolitinib has been reported internationally. However, in Japan, ruxolitinib use has been limited due to its off-label status. We conducted a nationwide, multicenter, retrospective survey in Japan on the use of ruxolitinib in pediatric HLH. Seventeen patients from nine institutions were included: primary HLH (<i>n</i> = 5), Epstein-Barr virus-associated HLH (EBV-HLH; <i>n</i> = 3), and post-transplant HLH (PT-HLH; <i>n</i> = 9). Ruxolitinib was used for refractory disease (<i>n</i> = 14) as well as for maintenance therapy in primary HLH/EBV-HLH (<i>n</i> = 3). In most patients, ruxolitinib was employed in combination with conventional therapies. Among refractory cases, the overall response rate was 80% for primary HLH/EBV-HLH and 89% for PT-HLH. The three patients who received ruxolitinib as maintenance therapy achieved sustained remission: two remained in remission until subsequent transplantation, and one did not require transplantation. Considering the results, we propose that ruxolitinib is a promising therapeutic option for pediatric HLH.</p>","PeriodicalId":19746,"journal":{"name":"Pediatric Hematology and Oncology","volume":" ","pages":"170-184"},"PeriodicalIF":1.4,"publicationDate":"2026-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147618892","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":"Prognostic impact of onset timing on response to methylprednisolone pulse therapy for idiopathic pneumonia syndrome after allogeneic hematopoietic cell transplantation.","authors":"Hirozumi Sano, Masato Yanagi, Daiki Hori, Satoru Matsushima, Ryusuke Ishigaki, Junjiro Ohshima, Daisuke Suzuki, Ryoji Kobayashi","doi":"10.1080/08880018.2026.2636608","DOIUrl":"10.1080/08880018.2026.2636608","url":null,"abstract":"<p><p>Idiopathic pneumonia syndrome (IPS) is a serious complication following allogeneic hematopoietic cell transplantation (HCT), often treated with methylprednisolone (mPSL) pulse therapy. However, treatment responses vary. This study aimed to identify predictors of poor response to mPSL monotherapy. Among 289 patients who underwent allogeneic HCT, 25 developed IPS and received mPSL pulse therapy. Clinical responses were categorized as complete (CCR), partial (PCR), or no response (NR), based on oxygen requirements within 28 days. We compared baseline characteristics of responders (CCR: <i>n</i> = 5; PCR: <i>n</i> = 6) and non-responders (NR: <i>n</i> = 14). Univariate analysis revealed that IPS onset on day +73 or later (<i>p</i> = 0.033), reduced intensity conditioning (<i>p</i> = 0.033), use of total body irradiation (<i>p</i> = 0.049) or fludarabine (<i>p</i> = 0.042), and nonuse of busulfan (<i>p</i> = 0.049) in preparative regimens were associated with poor response. Multivariate analysis identified a longer time from transplantation to IPS onset as a significant predictor of poor response (Odds Ratio 1.017 per 1-day increase; 95% CI 1.007-1.036; <i>p</i> = 0.045). The present study may provide valuable insights into how the responsiveness to mPSL varies depending on the time of IPS onset. Alternative therapeutic strategies may be needed for patients with late-onset IPS.</p>","PeriodicalId":19746,"journal":{"name":"Pediatric Hematology and Oncology","volume":" ","pages":"146-157"},"PeriodicalIF":1.4,"publicationDate":"2026-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147378338","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":"Central auditory processing in children with sickle cell disease.","authors":"Heba G A Ali, Mohamed A ElLaboudy, Fatma Soliman Elsayed Ebeid, Abeer Mohamed Elgendy, Nouran Ahmed Almallah, Marwa Waheed Tolba","doi":"10.1080/08880018.2026.2628528","DOIUrl":"10.1080/08880018.2026.2628528","url":null,"abstract":"<p><p>The complexity of sickle cell disease (SCD) goes beyond hematological manifestations, affecting different organs and systems, including auditory system. We aimed to assess Central Auditory Processing (CAP) abilities in children with SCD as well as to detect risk factors for Auditory Processing Disorders (APD) in children with SCD. A diagnostic observational cross sectional study that included thirty-three patients aged 6-16 years with a confirmed diagnosis of SCD. Demographic, clinical and laboratory characteristics were collected. Audiological testing included tympanometry, pure tone audiometry, IQ testing, specific history for CAP abilities and APD screening test battery. Among the 33 screened patients, all had normal pure tone audiometry, and 2 patients had middle ear affection. Auditory Perception was assessed in 25 eligible participants. The majority of the studied patients (<i>n</i> = 21, 84%) APD. The most affected tests were patterning (<i>n</i> = 21, 84%) and auditory memory (<i>n</i> = 16, 64%). Results showed positive correlation between age of transfusion and dichotic listening, APD was commonly encountered in children with SCD, thus screening for APD is recommended in all patients with SCD for early detection and intervention of any abnormalities.</p>","PeriodicalId":19746,"journal":{"name":"Pediatric Hematology and Oncology","volume":" ","pages":"129-145"},"PeriodicalIF":1.4,"publicationDate":"2026-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147574402","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":"Leveraging artificial intelligence for decision-making in pediatric progressive and refractory solid tumors.","authors":"Oz Mordechai, Inbar Meir, Einav Blanche, Myriam Ben-Arush","doi":"10.1080/08880018.2026.2618260","DOIUrl":"10.1080/08880018.2026.2618260","url":null,"abstract":"<p><p>Pediatric patients with progressive and refractory solid tumors face a challenging prognosis. Despite advancements in treatments like immunotherapy and targeted therapy, survival rates remain low for certain tumor types. Decision-making in these complex cases often necessitates a multidisciplinary approach, integrating risk-based management, precision medicine, and access to clinical trials. Artificial intelligence (AI) technologies, particularly large language models (LLMs), hold promise for improving clinical reasoning and decision support in pediatric oncology. This study evaluated the decision-making capabilities of five AI tools-ChatGPT, Gemini, Claude, Perplexity, and OpenEvidence-in six hypothetical cases of refractory or progressive pediatric solid tumors. Each AI tool was presented with two sequential queries: a request to generate potential treatment options and then a request to identify and justify the most appropriate option from its initial list. The AI tools generated a total of 124 treatment recommendations, with an average of 24.8 per tool. Clinical trial enrollment was the most frequently selected \"best option,\" accounting for 55.2% of cases. Other notable recommendations included targeted therapy (17.2%), surgery (10.3%), chemotherapy (10.3%), best supportive care (10.3%), and immunotherapy (3.4%). Notably, the AI tools exhibited distinct tendencies in their decision-making approaches, with some favoring aggressive interventions and others emphasizing supportive or palliative care. AI tools demonstrate potential for assisting with complex treatment decisions in pediatric oncology, particularly by identifying clinical trial options. However, the observed variability in recommendations underscores the need for careful human oversight to ensure that AI-generated suggestions align with clinical evidence, patient and family preferences, and the overall goals of care. Future research should explore how AI tools can be further refined to incorporate nuanced patient-specific information and address the emotional and psychological impact of AI-assisted decision-making.</p>","PeriodicalId":19746,"journal":{"name":"Pediatric Hematology and Oncology","volume":" ","pages":"185-192"},"PeriodicalIF":1.4,"publicationDate":"2026-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146053318","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":"Ophthalmological manifestations of neuroblastoma - A retrospective multicenter study.","authors":"Katarzyna Kuchalska, Katarzyna Derwich, Walentyna Balwierz, Bartosz Urbański, Marek Ussowicz, Anna Tomoń, Anna Pytlik, Małgorzata Sawicka-Żukowska, Julia Geisler-Paliwoda, Danuta Januszkiewicz-Lewandowska, Szymon Janczar, Monika Pogorzała, Aleksandra Kiermasz, Szymon Skoczeń, Krzysztof Kałwak, Ninela Irga-Jaworska, Agnieszka Mizia-Malarz, Jan Styczyński, Katarzyna Drabko, Katarzyna Muszyńska-Rosłan, Katarzyna Machnik, Radosław Chaber, Tomasz Szczepański, Wanda Badowska, Bożenna Dembowska-Bagińska, Anna Gotz-Więckowska","doi":"10.1080/08880018.2026.2650745","DOIUrl":"10.1080/08880018.2026.2650745","url":null,"abstract":"<p><p>Neuroblastoma (NBL) constitutes the most common extracranial solid tumor in children that arises from the sympathetic ganglia. Its manifestations depend on the localization of the tumor and metastases. This research aimed to assess the incidence of ophthalmological symptoms and their correlations with different prognostic factors. We collected and analyzed retrospectively the data of 776 children with a clinical diagnosis of peripheral neuroblastic tumors reported between 2004 and 2022 from 14 Polish pediatric oncology centers. Ophthalmological symptoms occurred in 17.10% of the patients, and in 12.70% they were diagnosed at the time of the first presentation. The reported manifestations comprised opsoclonus-myoclonus syndrome (OMS), Horner syndrome (HS), exophthalmos, periorbital hemorrhages, with the highest incidence of OMS (4.74% of all the patients). Moreover, we described the cases with neurological manifestations of NBL like paraparesis and facial nerve paresis. The age correlated with the form of symptoms, and HS was observed in the youngest patients. Similarly, the time from the symptoms onset to NBL diagnosis differed significantly among the patients, therefore the diagnosis of HS was associated with the longest period. In conclusion, the ophthalmological manifestations of NBL represent an essential group of symptoms. Therefore, clinicians should consider them as possible signs of tumors, particularly HS, which may be most challenging in the diagnostic process.</p>","PeriodicalId":19746,"journal":{"name":"Pediatric Hematology and Oncology","volume":" ","pages":"158-169"},"PeriodicalIF":1.4,"publicationDate":"2026-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147675750","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":"Correction.","authors":"","doi":"10.1080/08880018.2026.2661318","DOIUrl":"10.1080/08880018.2026.2661318","url":null,"abstract":"","PeriodicalId":19746,"journal":{"name":"Pediatric Hematology and Oncology","volume":" ","pages":"198-199"},"PeriodicalIF":1.2,"publicationDate":"2026-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147778001","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":"Vitamin D receptor gene polymorphisms and 25-hydroxyvitamin D levels: association with cerebrovascular disease in sickle cell anemia.","authors":"Gabriela S Arcanjo, Jéssica V G F Batista, Jéssica M F Oliveira, Igor F Domingos, Thais H C Batista, Diego A Pereira-Martins, Betânia L D Hatzlhofer, Amanda B S Araújo, Talita S S França, Ana C M Anjos, Aderson S Araujo, Fernando F Costa, Antonio R Lucena-Araujo, Marcos A C Bezerra","doi":"10.1080/08880018.2025.2601121","DOIUrl":"10.1080/08880018.2025.2601121","url":null,"abstract":"<p><p>Sickle cell anemia (SCA) is associated with a high prevalence of cerebrovascular complications, such as ischemic stroke, particularly at young ages. Among various molecules, vitamin D has been implicated in stroke occurrence. By binding to the vitamin D receptor (VDR), vitamin D regulates several mechanisms, potentially influencing pathways involved in the pathophysiology of cerebral vasculopathy. Here, we evaluated the association between <i>VDR</i> gene polymorphisms and 25-hydroxyvitamin D (25(OH)D) levels with CVD in a cohort of patients with SCA. The frequency of CVD and laboratory data were retrospectively obtained from the medical records. Genotyping for the functional <i>FokI</i> (rs2228570) and <i>Cdx-2</i> (rs11568820) <i>VDR</i> polymorphisms was performed in 459 unrelated SCA patients. 25(OH)D levels were measured in a subset of 45 patients. The TT genotype of the <i>FokI VDR</i> polymorphism was associated with a higher frequency (OR: 2.82; 95% CI: 1.38-5.76, <i>p</i> = 0.006) and higher cumulative incidence (34% vs 16%, <i>p</i> = 0.001) of CVD compared to the other genotypes. Among patients under hydroxyurea (HU) therapy, individuals with the TT-<i>FokI</i> genotype (median: 13.9 ng/mL) had lower 25(OH)D levels compared to those with CC/CT genotypes (median: 20.2 ng/mL; <i>p</i> = 0.033). Similarly, patients with CVD and the TT-<i>FokI</i> genotype (median: 12.3 ng/mL) had reduced 25(OH)D levels compared to CC/CT carriers (median: 20.8 ng/mL; <i>p</i> = 0.034). These findings suggest that the <i>FokI VDR</i> polymorphism may serve as a potential genetic modulator of cerebrovascular complications in individuals with SCA.</p>","PeriodicalId":19746,"journal":{"name":"Pediatric Hematology and Oncology","volume":" ","pages":"63-75"},"PeriodicalIF":1.2,"publicationDate":"2026-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145844061","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}