Journal of Pediatric Hematology/Oncology最新文献

{"title":"The Contribution of Bleeding Assessment Score and Pictorial Blood Loss Assessment Chart to the Diagnosis of Von Willebrand Disease in Adolescent Girls With Iron Deficiency Anemia.","authors":"Asli Turgutoglu Yilmaz, Berkin Berk Akbeyaz, Merve Sahin Yilmaz, Harun Kasapoglu, Nilsu Turkmen, Neslihan Caliskan, Yilmaz Ay","doi":"10.1097/MPH.0000000000003303","DOIUrl":"https://doi.org/10.1097/MPH.0000000000003303","url":null,"abstract":"<p><strong>Background: </strong>Heavy menstrual bleeding (HMB) is common among adolescents and is an important driver of iron deficiency anemia. However, clinicians under-recognize inherited bleeding disorders. Iron deficiency transiently elevates von Willebrand factor (VWF) and factor VIII levels, both of which are acute-phase reactants, thereby masking quantitative VWF deficiency.</p><p><strong>Methods: </strong>This prospective cross-sectional study enrolled iron-deficient adolescent girls at a single center. We used a structured bleeding history (ISTH-BAT), menstrual blood loss quantification (PBAC), and coagulation testing after iron repletion to detect VWD-spectrum abnormalities.</p><p><strong>Results: </strong>A total of 235 girls aged 11 to 18 years with iron deficiency anemia completed the International Society on Thrombosis and Hemostasis Bleeding Assessment Tool and the Pictorial Blood Loss Assessment Chart and were stratified into 3 prespecified groups: high ISTH-BAT score with PBAC-defined HMB (n=23), low ISTH-BAT score with PBAC-defined HMB (n=131), and low ISTH-BAT score without PBAC-defined HMB (n=78). VWF: Ag, VWF: RCo, and FVIII:C levels were measured after iron repletion. Among 232 participants with complete data, VWD-screen positivity was defined as VWF: Ag and/or VWF: RCo <50 IU/dL. Overall, 40.9% (95/232) were VWD-screen-positive, with a significant group gradient (65.2%, 40.5%, 34.6%; P=0.033), preserved under stricter 2021-aligned thresholds (<30 IU/dL; 34.8%, 6.1%, 3.8%; P<0.001). Lower VWF:Ag and VWF:RCo levels drove these differences (both P<0.001), suggesting quantitative VWF abnormalities. FVIII:C levels were lower in patients who screened positive (P=0.005). Routine coagulation tests and PFA-100 closure times did not differ between the groups.</p><p><strong>Conclusion: </strong>Combined ISTH-BAT and PBAC stratification enriches for postrepletion VWD-screen positivity; a 2-step triage strategy following iron repletion warrants prospective validation in this population.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":""},"PeriodicalIF":0.9,"publicationDate":"2026-10-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149137313","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":"Complications of Central Venous Catheters in Egyptian Children and Adolescents With Malignancy: A Prospective Single-Center Observational Study.","authors":"Safinaz A Elhabashy, Mohamed El-Laboudy, Eman M Elsayed, Noha A E Fahim, Heba Hussain, Heba G A Ali","doi":"10.1097/MPH.0000000000003290","DOIUrl":"https://doi.org/10.1097/MPH.0000000000003290","url":null,"abstract":"<p><p>Central venous catheters (CVCs) are essential in pediatric oncology but are associated with a considerable risk of complications, particularly in immunocompromised patients. Evidence from low- and middle-income settings remains limited. This prospective observational study was conducted from 2020 to 2023 at a tertiary pediatric oncology center. A total of 141 patients (younger than 18 y), including 382 CVCs, were followed for 5869 catheter-days. Clinical, laboratory, and catheter-related data were collected. Complication rates were expressed per 1000 catheter-days, and risk factors were analyzed using univariate and multivariate models. CVC-related complications occurred in 97/382 devices (25.4%), and mortality was 0.3%. Infectious complications were most frequent (53.1%), followed by vascular (32.8%) and mechanical events (14.1%). Gram-positive organisms predominated in peripheral cultures (26.5%), while Gram-negative pathogens were more common in central cultures (22.4%). Patients with complications had significantly lower leukocyte counts (P=0.001), neutrophils (P<0.001), lymphocytes (P=0.006), and platelets (P=0.002), with higher D-dimer levels (P<0.001). Hematological malignancy independently increased complication risk (P=0.002), while alcohol disinfection of catheter hubs was protective (P=0.007). Thrombosis represented 85.7% of vascular events and was managed conservatively in most cases. CVC-related complications remain common in pediatric oncology and are influenced by disease-related and modifiable factors. Strengthening infection control practices may significantly reduce complications.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":""},"PeriodicalIF":0.9,"publicationDate":"2026-10-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149111013","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":"From a Single Clock to a Segmented Pathway: Measuring Time to Antibiotics in Pediatric Febrile Neutropenia.","authors":"Muzna Sarfraz","doi":"10.1097/MPH.0000000000003298","DOIUrl":"https://doi.org/10.1097/MPH.0000000000003298","url":null,"abstract":"","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":""},"PeriodicalIF":0.9,"publicationDate":"2026-10-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149111113","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":"Primary Surgical Resection of Localized Cervical Kaposiform Hemangioendothelioma in a Neonate: A Potentially Curative Strategy in Selected Patients.","authors":"Mustafa Bilici, Muzaffer Sağlam, Didem Cemile Yeşilırmak, Yakup Sancar Barış, Ferit Bernay","doi":"10.1097/MPH.0000000000003297","DOIUrl":"https://doi.org/10.1097/MPH.0000000000003297","url":null,"abstract":"<p><p>Kaposiform hemangioendothelioma (KHE) is a rare, locally aggressive vascular tumor of infancy that may be associated with Kasabach-Merritt phenomenon (KMP). We report a term female neonate presenting with a 4×4 cm cervical mass at birth. Imaging demonstrated a well-circumscribed, highly vascular lesion without infiltration or compression of vital structures, and no coagulopathy was detected. Although these imaging features were atypical for KHE, the lesion was anatomically resectable, and malignancy could not be excluded from the differential diagnosis. Complete surgical excision was therefore performed on day 10. Histopathology confirmed KHE. The postoperative course was uneventful, with no recurrence at 1-year follow-up. This case highlights an uncommon radiologic presentation of KHE and suggests that complete surgical excision may represent a potentially curative option in carefully selected patients with anatomically resectable disease.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":""},"PeriodicalIF":0.9,"publicationDate":"2026-10-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149111124","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":"Splenomegaly With Thrombocytopenia: A Case of the Rare and Obvious.","authors":"Ajmeera Azeez, Prateek Bhatia, Manu Jamwal, Swetha Palla, Reena Das, Richa Jain","doi":"10.1097/MPH.0000000000003294","DOIUrl":"https://doi.org/10.1097/MPH.0000000000003294","url":null,"abstract":"<p><strong>Background: </strong>A 12-year-old boy presented with incidental splenomegaly, mild anemia, and thrombocytopenia, prompting evaluation, with Gaucher disease considered in the differential diagnosis.</p><p><strong>Observation: </strong>Enzyme testing revealed low β-glucosidase activity twice; however, Sanger sequencing was negative for mutations in the glucocerebrosidase 1 (GBA1) gene. A peripheral smear review was the turning point; it showed stomatocytes and giant platelets, suggestive of sitosterolemia. Next-generation sequencing confirmed a homozygous, likely pathogenic variant in ATP-binding cassette subfamily G member 5 (ABCG5). The child was started on Ezetimibe and a low plant-sterol diet.</p><p><strong>Conclusions: </strong>This case reiterates the importance of peripheral smear examination in leading to a very specific yet uncommon diagnosis of sitosterolemia.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":""},"PeriodicalIF":0.9,"publicationDate":"2026-10-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149111149","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":"Identifying Risk Factors for PICU Mortality in Pediatric Cancer Patients: A 10-Year Retrospective Study.","authors":"Selman Kesici, Ozlem Saritas Nakip, Merve Erdem, Sule Unal, Bulent B Kuskonmaz, Sevkiye S Aytac Eyupoglu, Guzide B Aydin, Bilgehan Yalcin, Mustafa T Kutluk, Benan Bayrakci","doi":"10.1097/MPH.0000000000003296","DOIUrl":"https://doi.org/10.1097/MPH.0000000000003296","url":null,"abstract":"<p><p>This 10-year retrospective study of pediatric cancer patients requiring intensive care evaluates mortality risk factors. The data included patients' demographics, cancer types, admission reasons, treatment modalities, and laboratory results. Standardized mortality ratio (SMR), mortality at different intervals, length of stay, and risk factors for death were analyzed. The most common malignancy was leukemia (n=116, 37.0%), followed by noncentral nervous system solid tumors (n=71, 22.6%). The acute kidney injury (AKI) rate was 23.7% (n=74), with 55.4% requiring renal replacement therapy (RRT). RRT indications included tumor lysis syndrome, AKI, fluid overload, and treatment toxicity. 66.7% of leukemia patients and 64.7% of lymphoma patients needed RRT (P=0.012). Three patients underwent extracorporeal membrane oxygenation, one of them was successfully weaned; however, died later due to sepsis. The crude mortality rate was 17.6% (n=47). SMR was 0.51. The highest PICU mortality rate occurred among leukemia patients (19.1%). Multivariate analysis showed that age and invasive mechanical ventilation (IMV) were significantly associated with PICU mortality. NIV was used in 25 patients, and only one patient required intubation. IMV was independently associated with increased mortality (OR: 9.029, 95% CI: 2.921-27.914, P<0.001).</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":""},"PeriodicalIF":0.9,"publicationDate":"2026-10-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149111182","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":"Surgical Cytoreduction and Intraoperative Microwave Ablation in Pediatric Desmoplastic Small Round Cell Tumor: A 24-Year Single-Center Experience.","authors":"Burak Ardıçlı, İdil Rana User, Berna Oğuz, Emre Ünal, Melis Gültekin, Ferah Yıldız, Nilgün Kurucu, Mustafa Tezer Kutluk, Diclehan Orhan, Saniye Ekinci, İbrahim Karnak","doi":"10.1097/MPH.0000000000003247","DOIUrl":"10.1097/MPH.0000000000003247","url":null,"abstract":"<p><p>Desmoplastic small round cell tumor (DSRCT) is a rare, aggressive pediatric malignancy, and optimal management remains challenging because most patients present with metastatic disease, and evidence guiding multimodal treatment is limited. We retrospectively reviewed 11 children with DSRCT treated at our institution between 2000 and 2023. Demographics, tumor characteristics, treatment modalities-including neoadjuvant chemotherapy, surgical resection, and intraoperative microwave ablation (MWA)-and outcomes were analyzed. Tumor burden was assessed using the Peritoneal Cancer Index (PCI), and survival was estimated using the Kaplan-Meier method. The median age at diagnosis was 11.6 years (male-to-female ratio, 2.6:1). Primary tumors were abdominopelvic (45.5%), pelvic (36.4%), or abdominal (18.2%). Lymphadenopathy occurred in 91% of patients, and 55% had extranodal metastases, predominantly hepatic. After chemotherapy, all patients underwent surgery: complete resection (R0) in 3, partial excision in 8, and intraoperative MWA for liver metastases in 2. The median follow-up was 26 months. The 5-year overall and event-free survival rates were 47% and 30%, respectively, and R0 resection was strongly associated with disease-free survival. Aggressive surgical cytoreduction remains central to improving outcomes in pediatric DSRCT. Intraoperative MWA may be a safe adjunct for selected patients with hepatic metastases. PCI appears useful for quantifying tumor burden, although validation in larger cohorts is warranted.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"331-338"},"PeriodicalIF":0.9,"publicationDate":"2026-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148604241","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":"Emergent Reversal of Intracranial Hemorrhage Using 4-Factor Prothrombin Complex Concentrate in a Pediatric Patient.","authors":"Ruchi P Jain, Keri Bicking, Amanda Clouser","doi":"10.1097/MPH.0000000000003261","DOIUrl":"10.1097/MPH.0000000000003261","url":null,"abstract":"<p><strong>Background: </strong>Direct oral anticoagulant (DOACs) use for pediatric venous thromboembolism has increased, yet data on reversal strategies using 4-factor prothrombin complex concentrate (4F-PCC) remains limited.</p><p><strong>Observation: </strong>We report the first case of intracranial hemorrhage (ICH) in a patient receiving rivaroxaban who underwent reversal with 4F-PCC. An 11-year-old male with sinusitis complicated by multifocal intracranial abscesses, and superior sagittal sinus thrombus treated with rivaroxaban, was found to have ICH. For urgent craniotomy, rivaroxaban was reversed with 2223 units (∼45 units/kg) 4F-PCC. No thromboembolism occurred within 3 months.</p><p><strong>Conclusion: </strong>This case adds to the limited literature supporting 4F-PCC for DOAC reversal in pediatrics.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"353-357"},"PeriodicalIF":0.9,"publicationDate":"2026-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148794659","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":"Beyond Tumor Size: Hilar-to-Central Vessel Distance as a Complementary Safety Metric for Laparoscopic Nephrectomy in Wilms Tumor.","authors":"Bianca R Rosa, Rodrigo C Ribeiro, Mauricio G Ieiri Yamanari, Fernanda M Pereira Souza, Carlos E Bezerra Cavalcante, Raniela F Faria, Leticia Semissato Dos Santos, Wilson Elias de Oliveira Junior","doi":"10.1097/MPH.0000000000003264","DOIUrl":"10.1097/MPH.0000000000003264","url":null,"abstract":"<p><strong>Background: </strong>Laparoscopic nephrectomy for Wilms tumor remains controversial despite growing evidence supporting its feasibility. This study analyzed surgical and oncological outcomes according to the SIOP criteria and evaluated the hilar-to-central vessel distance as a potential selection parameter.</p><p><strong>Methods: </strong>Retrospective analysis of 50 children with unilateral Wilms tumor treated between 2014 and 2020. Patients underwent open (ON=38) or laparoscopic (LN=12) nephrectomy following SIOP protocols. Laparoscopic selection required: tumor confined to the kidney, not crossing vertebral border, no venous thrombus, volume ≤300 mL, and adequate chemotherapy response. CT/MRI measurements included tumor volume and the distance between the hilar vessels and the aorta/vena cava. Outcomes included operative time, complications (Clavien-Dindo classification), conversion rate, lymph node yield, recurrence, and survival.</p><p><strong>Results: </strong>Groups differed significantly in staging (stage I: LN 83.3% vs. ON 26.3%, P =0.003). Mean tumor volume after chemotherapy was lower in the LN group (87.81 vs. 394.72 cm 3 , P <0.001). Hilar-to-central vessel distance after chemotherapy was greater in the LN group (10.83 vs. 5.42 mm, P =0.005). Conversion rate was 16.7% (2/12) due to bleeding. No intraoperative tumor spillage occurred in the LN group, compared with 3 in the ON group. Postoperative complications occurred only in the ON group (4/38, 10.5%). Average LOS was 2.42 days (LN) versus 4.53 days (ON). Five-year event-free survival was 91.7% (LN) and 84.2% (ON).</p><p><strong>Conclusion: </strong>Laparoscopic nephrectomy for carefully selected Wilms tumor patients treated according to SIOP protocols demonstrated favorable perioperative outcomes consistent with appropriate patient selection. The hilar-to-central vessel distance showed statistical association with surgical approach at the group level, but did not discriminate conversion risk at the individual level. Integration of this metric should be studied as a potential adjunct to comprehensive assessment by experienced surgical teams, with prospective multicenter validation required before clinical implementation.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"323-330"},"PeriodicalIF":0.9,"publicationDate":"2026-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148761493","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":"A Case Report of Bilateral Pulmonary Emboli in a Patient With Fibroadipose Venous Anomaly Treated With Sirolimus.","authors":"Meredith Farrell, Janice McDaniel, Nicole E Kendel","doi":"10.1097/MPH.0000000000003249","DOIUrl":"10.1097/MPH.0000000000003249","url":null,"abstract":"<p><strong>Background: </strong>Sirolimus is a first-line treatment for venous malformations in pediatric patients. Current studies describing adverse events have not shown an increased incidence of venous thromboembolism (VTE).</p><p><strong>Observation: </strong>We present a pediatric patient with a vascular anomaly who developed bilateral pulmonary emboli while on sirolimus with minimal other risk factors for clot development.</p><p><strong>Conclusions: </strong>While it is not possible to conclusively prove that the development of pulmonary emboli was related to sirolimus, given the possible association, clinicians who treat patients with vascular anomalies may need to consider patients treated with sirolimus at a higher risk for VTE.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"358-360"},"PeriodicalIF":0.9,"publicationDate":"2026-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148673908","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}