Pediatric Hematology and Oncology最新文献

{"title":"Hemorrhagic complications and outcomes of children with acute promyelocytic leukemia at initial hospital admission: a multicenter cohort study.","authors":"Balagangadhar Totapally, Ariella Barhen, Guillermo De Angulo","doi":"10.1080/08880018.2025.2506414","DOIUrl":"10.1080/08880018.2025.2506414","url":null,"abstract":"<p><p>Acute promyelocytic leukemia (APL) is an uncommon subtype of acute myeloid leukemia that is associated with hemorrhagic complications and early death. Our primary objective was to describe the frequency of hemorrhagic complications and outcomes associated with APL during the initial admission using the Pediatric Health Information System (PHIS) database. We performed a retrospective cross-sectional analysis of children with APL not in remission. Demographic characteristics, resource utilization, and outcomes were compared between those with and without hemorrhagic complications. Out of 173 patients with APL, hemorrhagic complications (intracranial, gastrointestinal, or pulmonary) occurred in 32%. Children with hemorrhagic complications were more likely to have acute respiratory failure, coagulopathy, stroke, and cerebral edema. Children with hemorrhagic complications experienced more ICU admissions and mechanical ventilation, and they also received transfusions, low molecular weight heparin, hydroxyurea, and rasburicase more frequently. The median length of stay and mortality rate was not different between the groups. The median length of stay of children who died was significantly lower (<i>p</i> = 0.003) as the majority (78%) who died death occurred within 7 days. Hemorrhagic complications occurred in a 3^rd of patients with the initial admission of APL, and most deaths occurred during the first week.</p>","PeriodicalId":19746,"journal":{"name":"Pediatric Hematology and Oncology","volume":" ","pages":"265-275"},"PeriodicalIF":1.2,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144094271","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":"Relapse patterns among children and adolescents with Kaposi sarcoma in Malawi.","authors":"Toni Chanroo, Allison Silverstein, Casey L McAtee, William Kamiyango, Jimmy Villiera, Parth S Mehta, Erin Peckham-Gregory, Mark Zobeck, Michael E Scheurer, Carl E Allen, Rizine Mzikamanda, Nmazuo W Ozuah, Nader Kim El-Mallawany","doi":"10.1080/08880018.2025.2518365","DOIUrl":"10.1080/08880018.2025.2518365","url":null,"abstract":"<p><p>Kaposi sarcoma (KS) is a common childhood cancer in Malawi, but few studies have explored clinical characteristics of relapsed disease. We aimed to characterize clinical patterns of relapse to improve treatment and, ultimately, long-term survival in patients with pediatric KS. A retrospective cohort study was conducted among patients ages <19 years of age at time of KS diagnosis in Lilongwe, Malawi between August 1, 2010 and March 15, 2020. Specifically, emphasis was placed on patients who had relapsed disease and excluded patients with refractory disease or those who died whilst receiving front-line treatment. Salvage therapy typically involved an intensified chemotherapy regimen compared to front-line therapy - namely nonliposomal doxorubicin plus bleomycin/vincristine or paclitaxel monotherapy. One-hundred and ninety patients with pediatric KS were included in this analysis, 50 of whom experienced relapse (26%). Older median age was associated with occurrence of relapse (10 vs. 6.7 years, <i>p</i>-value = 0.004). Median time from diagnosis to first relapse was 10.6 months (range 2.3-49 months). Three-year post-relapse overall survival (OS) for the entire cohort was 60% with a median follow-up time of 4.7 years after relapse. Survival was significantly higher for patients who relapsed with the woody edema clinical phenotype of pediatric KS versus those with visceral/disseminated disease - 3-year OS 79% (95% CI 62-100) vs. 29% (14-61). These data demonstrate potential for continued survival after KS relapse in the pediatric population and identify subsets of high-risk patients. The higher mortality observed in patients with visceral/disseminated KS highlights the need for improved therapeutic strategies.</p>","PeriodicalId":19746,"journal":{"name":"Pediatric Hematology and Oncology","volume":" ","pages":"276-286"},"PeriodicalIF":1.2,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12236444/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144369116","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"The novel <i>XIAP</i> Lys396Ter variant alters mitochondrial membrane potential and endoplasmic reticulum intensity in monocytes of two XIAP-deficient patients.","authors":"Benedetta Elena Di Majo, Fabiola Guerra, Mario Mauri, Maria Luisa Coniglio, Elena Sieni, Silvia Parolini, Giovanna Tabellini, Cristina Bugarin, Fabiola Dell'Acqua, Giovanna Lucchini, Sonia Bonanomi, Adriana Cristina Balduzzi, Jasmin Mann, Stephan Ehl, Andrea Biondi, Francesco Saettini","doi":"10.1080/08880018.2025.2521122","DOIUrl":"10.1080/08880018.2025.2521122","url":null,"abstract":"","PeriodicalId":19746,"journal":{"name":"Pediatric Hematology and Oncology","volume":" ","pages":"287-295"},"PeriodicalIF":1.2,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144497615","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":"Retrospective analysis of 26 children with severe autoimmune hemolytic anemia treated with transfusions of red blood cells.","authors":"Xue Tang, Xiaoqing Li, Yang Wang, Min Yang, Li-Li Luo","doi":"10.1080/08880018.2025.2497873","DOIUrl":"10.1080/08880018.2025.2497873","url":null,"abstract":"<p><p>Autoimmune hemolytic anemia (AIHA) is rare inchildren. Little is known about the efficiency of red blood cells (RBCs) transfusions and the risk of reactions among children with severe AIHA. This article describes the clinical features and outcomes in AIHA children after RBCs transfusions. A retrospective study was conducted among hospitalized AIHA children from July 2019 to October 2024 in a tertiarycare medical center in China. Twenty-six patients received 47 RBCs transfusions, with a median age at diagnosis of 59.5 months. Sixteen patients had secondary AIHA, with 8 systemic lupus erythematosus patients. Only 7 RBCs transfusions were compatible with both major and minor cross-matching. After transfusions, the median hemoglobin levels increased from 43 to 67 g/L. All patients were treated with steroids and 24 patients were treated with intravenous immunoglobulin before RBCs transfusions. No adverse reaction was observed. The median follow-up period was 39 months and the median steroid duration was 14 months. Nine patients experienced relapses after reducing steroid dosage. In conclusion, systemic lupus erythematosus is the main causes of secondary severe AIHA in children. Blood transfusions after steroids and intravenous immunoglobulin, even with incompatible RBCs, are safe and efficient for for children with severe AIHA.</p>","PeriodicalId":19746,"journal":{"name":"Pediatric Hematology and Oncology","volume":" ","pages":"257-264"},"PeriodicalIF":1.2,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144120565","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":"Intracardiac thrombi in children: A decade of experience at a tertiary care hospital.","authors":"Tzofnat Farbstein-Aljanati, Tal Tirosh-Wagner, Ivan Budnik, Uriel Katz, Sharon Borik, Assaf A Barg, Gili Kenet, Sarina Levy-Mendelovich","doi":"10.1080/08880018.2025.2531011","DOIUrl":"https://doi.org/10.1080/08880018.2025.2531011","url":null,"abstract":"<p><p>Intracardiac thrombus (ICT) in children is a serious and potentially life-threatening condition, particularly among hospitalized patients. Pediatric-specific data on ICT remain scarce, posing challenges for optimal, evidence-based management. This study aimed to analyze pediatric ICT cases over a decade to identify clinical characteristics, management strategies, and outcomes.</p><p><p>This retrospective study was conducted at a tertiary referral center and included all pediatric patients (ages 0-18) diagnosed with ICT between 2012 and 2023. Clinical and demographic data were extracted from electronic medical records, following IRB approval.</p><p><p>Twenty-three children were identified (median age: 39 months; 52% female). Major risk factors included central venous catheters (60.9%), congenital or acquired heart disease (52.2%), and malignancy (34.8%). Most thrombi (78.3%) were right-sided. ICT was incidentally diagnosed in 56.5% of cases; in others, symptoms led to imaging <i>via</i> echocardiography, CT, or cardiac catheterization.</p><p><p>Anticoagulation therapy was initiated in 87% of patients. Eight required indefinite treatment due to persistent thrombi or ongoing risk factors, while others received a median therapy duration of four months. Three patients did not receive anticoagulation due to clinical considerations. Surgical thrombectomy was performed in four cases (17.4%), and one patient underwent catheter-based thrombolysis. There were no major bleeding events. Complete thrombus resolution was achieved in 60.9% of cases, with partial improvement in 26.1%.</p><p><p>ICT in children is associated with identifiable risk factors and is commonly managed with anticoagulation. Surgical or interventional procedures may be needed in select cases. Early identification of high-risk patients is essential to improve outcomes.</p>","PeriodicalId":19746,"journal":{"name":"Pediatric Hematology and Oncology","volume":" ","pages":"1-10"},"PeriodicalIF":1.2,"publicationDate":"2025-07-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144760750","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":"Aberrant serum lipid levels predict clinical outcomes in patients with neuroblastoma.","authors":"Zhixia Yue, Wen Zhao, Chao Duan, Xisi Wang, Chao Gao, Xiaoli Ma, Yan Su","doi":"10.1080/08880018.2025.2522808","DOIUrl":"https://doi.org/10.1080/08880018.2025.2522808","url":null,"abstract":"<p><p>This study aimed to explore how serum lipid levels are related to clinical features and outcomes in neuroblastoma(NB). We analyzed data from 169 patients diagnosed with NB, focusing on their serum lipid levels and how these relate to disease characteristics and prognosis. We found that older age (>18 months), high-risk disease features (e.g. elevated LDH, NSE, serum ferritin), genetic abnormalities (such as MYCN amplification, 1p36/11q23 LOH, PHOX2B expression), tumor size ≥10 cm, bone marrow metastasis, and multiple organ involvement were linked to abnormal lipid profiles, including elevated total cholesterol (TC), triglycerides (TG), LDL-C, and lipid ratios (TC/HDL-C, TG/HDL-C), along with reduced HDL-C. Interestingly, high TG and TG/HDL-C levels correlated with increased regulatory T cells (Tregs), which play a role in immune response. Patients with unfavorable lipid profiles-particularly high TC, TG, LDL-C, and lipid ratios or low HDL-C-had worse 5-year event-free survival (EFS) rates. This was especially true for high-risk patients. In statistical models, the TC/HDL-C ratio emerged as an independent predictor of poorer EFS. To assess lipid dynamics during treatment, we monitored patients at multiple time points and observed decreasing TC, TG, LDL-C, TC/HDL-C, and TG/HDL-C levels, alongside increasing HDL-C, suggesting treatment-induced improvement in lipid metabolism. In patients with disease progression or relapse, lipid levels (TC, LDL-C, HDL-C) were significantly elevated at the time of the event compared to post-chemotherapy levels.In summary, dysregulation of serum lipids is common in NB and lipid profiles are closely linked to NB progression and may serve as potential biomarkers for prognosis and treatment monitoring.</p>","PeriodicalId":19746,"journal":{"name":"Pediatric Hematology and Oncology","volume":" ","pages":"1-15"},"PeriodicalIF":1.2,"publicationDate":"2025-07-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144760749","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":"Adherence to the febrile neutropenia protocol in pediatric leukemia patients at a Brazilian Cancer Center-A retrospective audit.","authors":"Jessica Talita Mariana Wicthoff Raniero, Anita Cassoli Cortez, Cecilia Maria Lima da Costa, Luciana Mariano Palanch Piotto, Ivan Leonardo Avelino França E Silva, Viviane Sonaglio, Marjorie Vieira Batista","doi":"10.1080/08880018.2025.2525267","DOIUrl":"https://doi.org/10.1080/08880018.2025.2525267","url":null,"abstract":"<p><p>Pediatric patients with hematologic cancers, especially those undergoing chemotherapy, are highly vulnerable to infections and febrile neutropenia (FN). Noncompliance with FN protocols often leads to excessive antibiotic use, raising the risk of multidrug-resistant bacteria and contributing to higher mortality rates. This retrospective study aimed to assess inappropriate empirical antibiotic treatment (IEAT) rates during FN episodes and identify risk factors for mortality among pediatric patients diagnosed with acute leukemia and FN at the A.C. Camargo Cancer Center between January 1, 2010, and December 31, 2020. A total of 329 FN episodes in 84 patients were analyzed, revealing an overall IEAT rate of 40%. The study identified key issues such as 2% of inadequate Gram-positive coverage, 78% of failure to de-escalate antibiotic treatment and 62% of maintenance antibiotic administration despite negative cultures Bloodstream infections were found in 77% of episodes, with age over 13 years (<i>p</i> 0.003), and bloodstream infection (<i>p</i> 0.002) being significant risk factors for mortality.</p>","PeriodicalId":19746,"journal":{"name":"Pediatric Hematology and Oncology","volume":" ","pages":"1-11"},"PeriodicalIF":1.2,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144541812","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":"Reducing empiric antibiotic administration in pediatric oncology patients with non-neutropenic fever: a single-center quality improvement initiative.","authors":"Alexandra Satty, Jessica Stiefel, Audrey Mauguen, Zahra Hudda, Madhavi Lakkaraja, Gabriela Llaurador, Mauricio Rendon Bernot, Susan K Seo, Julia Glade Bender, Maria Luisa Sulis, Farid Boulad, James S, Killinger","doi":"10.1080/08880018.2025.2498664","DOIUrl":"10.1080/08880018.2025.2498664","url":null,"abstract":"<p><p>The management of non-neutropenic fever in pediatric oncology varies widely, with many providers choosing to administer empiric antibiotics. We conducted a quality improvement intervention seeking to decrease empiric antibiotic administration in pediatric oncology patients with a central venous catheter presenting in the outpatient setting with non-neutropenic fever assessed to be low-risk for bacteremia. Over a 1-year period, empiric antibiotics were administered at the index visit in 9.6% of episodes, compared with 97% prior to the intervention. In patients not receiving empiric antibiotics, the bacteremia rate was 2.3% and composite event rate 5.1%, similar to pre-intervention baseline. These data suggest that empiric antibiotics can be withheld in low-risk patients with non-neutropenic fever.</p>","PeriodicalId":19746,"journal":{"name":"Pediatric Hematology and Oncology","volume":" ","pages":"242-249"},"PeriodicalIF":1.2,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144035100","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":"Evaluation of psychosocial outcomes of pediatric cancer survivors in a resource limited setting.","authors":"Divya Rajkumar, Venkatraman RadhaKrishnan, Gargi Das, Balaji Thiruvengadam Konthandan, Prasanth Srinivasan, Surendran Veeriah","doi":"10.1080/08880018.2025.2496365","DOIUrl":"10.1080/08880018.2025.2496365","url":null,"abstract":"<p><p>Pediatric cancer survivorship presents significant challenges globally, with varying survival rates and survivorship care models across different income settings. This present study focuses on the psycho-social outcomes of pediatric cancer survivors attending an After Completion Therapy (ACT) Clinic in a low-middle-income country, where survivorship care resources are limited compared to high-income countries. The study included 394 pediatric cancer survivors, aged 18 years and above, who had completed two years of disease-free survival post-treatment. Study data was collected from the ACT Clinic's survivorship proforma standardized by the experts in the field of Psycho-oncology and pediatric cancer survivorship, NCCN Distress Thermometer, and Visual Analog Pain Scale. Statistical analysis included descriptive statistics and chi-square tests to assess associations between demographic, clinical, and psycho-social variables. The majority of survivors were male (69.3%) hailing from rural areas (68.3%), with a mean age of 21 years. Haematological malignancies (61.3%) were predominant, treated mainly with chemotherapy (79%). Moderate distress was reported by 53% of female survivors. Cancer diagnosis (<i>χ</i>²(9) = 19.642, <i>p</i> < 0.020) and treatment modality (<i>χ</i>²(9) = 17.888^a, <i>p</i> < 0.036) are significantly influenced by distress levels. Academic and occupational status did not show a significant impact on distress but observed a notable percentage of challenges in post-treatment normalcy. Pediatric cancer survivors attending the ACT Clinic in resource-limited setting face substantial psycho-social challenges, influenced by gender, occupation, cancer type, and treatment history. The study emphasis the critical need for gender-sensitive and holistic survivorship care programs which should be tailored to address these challenges comprehensively. There is a need for more healthcare collaborations, mental health support, educating care givers on the importance of survivorship and improving advocacy through survivors support group activities. These programs can enhance the framework of survivorship care in low middle-income countries.</p>","PeriodicalId":19746,"journal":{"name":"Pediatric Hematology and Oncology","volume":" ","pages":"217-227"},"PeriodicalIF":1.2,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144026297","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":"Alpelisib in pediatric PIK3CA- and TIE-2-mutant vascular anomalies: a case series on safety, efficacy, and drug exposure.","authors":"Albert Etingin, Amandine Remy, Thomas Sonea, Francis Fortin, Josée Dubois, Sandrine Essouri, Sandra Ondrejchak, Chantal Lapointe, Yves Théôret, Audrey Denoncourt, Facundo Garcia-Bournissen, Thierry Ducruet, Jérôme Coulombe, Julie Powell, Thai Hoa Tran, Niina Kleiber","doi":"10.1080/08880018.2025.2498660","DOIUrl":"10.1080/08880018.2025.2498660","url":null,"abstract":"<p><p>Alpelisib was recently approved by the FDA for the management of pediatric patients with PIK3CA-related overgrowth spectrum. However, this medication was approved in the absence of pediatric pharmacokinetic data, as a fixed 50 mg dose, with no consideration of weight, the primary pharmacokinetically relevant covariate. This raises concerns regarding potential under and over-exposure. Given this gap in information, we aimed to assess the effect of alpelisib in relation to drug exposure (clinical response and drug safety). Alpelisib plasma concentrations were obtained from eight patients under treatment for vascular malformations. Drug exposure determined with area under the curve (AUC) was correlated to drug effect determined by a decrease in the size of lesions and grade of adverse events. Analysis was performed retrospectively. Eight patients received oral alpelisib through the compassionate use program of Novartis. AUC revealed substantial variability (3036 to 16620 ug*h/L) and inversely correlated to weight. Alpelisib resulted in marked clinical improvement, reducing pain, resolving coagulopathy, and improving mobility. Volumetric MRI indicated a 17.4% decrease in targeted vascular anomaly volume after 6 months of alpelisib therapy (<i>p</i> < 0.05), although volume decrease did not correlate with AUC. Adverse events including insulin resistance (<i>n</i> = 8/8) and growth restriction (<i>n</i> = 1/8) were documented, with severity directly correlating to drug exposure. We observed significant weight-related variability in alpelisib plasma concentrations, suggesting that the FDA-approved fixed-dose regimen of alpelisib is not optimal for pediatric patients. Weight-based dosing and therapeutic drug monitoring should be considered to enhance alpelisib safety.</p>","PeriodicalId":19746,"journal":{"name":"Pediatric Hematology and Oncology","volume":" ","pages":"228-241"},"PeriodicalIF":1.2,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143992352","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}