Journal of Pediatric Hematology/Oncology最新文献

{"title":"Acute Encephalopathy in 3 Adolescent Patients With Acute Lymphoblastic Leukemia During Induction Chemotherapy: A Case Series.","authors":"Vivien W Y Li, Annie T G Chiu, Grace K S Lam, Sheila S N Wong, Wai L Yeung, Alex W K Leung","doi":"10.1097/MPH.0000000000003074","DOIUrl":"https://doi.org/10.1097/MPH.0000000000003074","url":null,"abstract":"<p><p>Three adolescent patients with B-cell acute lymphoblastic leukemia (B-ALL) presented with psychotic symptoms, mutism, movement disorders, and day-night disturbances during induction chemotherapy, which clinically resembled anti-NMDAR encephalitis. However, all patients were negative for anti-NMDAR antibodies. They also experienced chemotherapy-induced liver dysfunction, weight loss, and malnutrition, which led to biochemical changes (elevated blood ammonia, elevated blood glutamate, and copper deficiency) known to affect NMDA receptor activity. This suggests that there may be some common, undefined pathways in these 2 different pathologies. Early nutritional intervention should be considered for patients who may be at risk for this significant neurotoxicity.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":""},"PeriodicalIF":0.9,"publicationDate":"2025-06-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144497380","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":"Early Versus Late Discontinuation of Empirical Antibiotics in Pediatric Oncology Patients With Fever and Neutropenia.","authors":"Mahdi Asleh, Carmel Levi, Yusif Abu Alhasan, Hagit Miskin, Dana Danino","doi":"10.1097/MPH.0000000000003076","DOIUrl":"https://doi.org/10.1097/MPH.0000000000003076","url":null,"abstract":"<p><p>The optimal timing for discontinuing antibiotics in pediatric oncology patients with unexplained fever and neutropenia remains uncertain. We analyzed 367 episodes of fever and neutropenia in hospitalized patients 0 to 19 years of age (2015 to 2022). Patients were grouped, based on antibiotic discontinuation timing: early (afebrile ≥24 h and negative cultures ≥48 h), late (ANC ≥500 cells/mm³), and intermediate (ANC <500 but afebrile ≥24 h). Among 298 culture-negative episodes, early (24.2%), late (36.2%), and intermediate (39.6%) groups showed no differences in new infections, intensive care admissions, or mortality. Outcomes were comparable for high- and low-risk patients. Early discontinuation appears safe and effective, even in high-risk patients.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":""},"PeriodicalIF":0.9,"publicationDate":"2025-06-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144497381","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":"Cobalamin E Disease: An Ultrarare Treatable Cause of Hemolytic Anemia in Infancy.","authors":"Pankaj Prasun, Jennifer R Suarez, Katelyn J Watkin","doi":"10.1097/MPH.0000000000003072","DOIUrl":"https://doi.org/10.1097/MPH.0000000000003072","url":null,"abstract":"","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":""},"PeriodicalIF":0.9,"publicationDate":"2025-06-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144475742","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 Features of Pediatric Primary Hemophagocytic Lymphohistiocytosis With CNS Involvement.","authors":"Chenzi Zhao, Qing Zhang, Haoyue Jia, Sitong Chen, Liping Zhang, Rui Zhang, Zhigang Li","doi":"10.1097/MPH.0000000000003071","DOIUrl":"https://doi.org/10.1097/MPH.0000000000003071","url":null,"abstract":"<p><p>In this study, we analyzed genetic variation and prognostic factors in pediatric primary hemophagocytic lymphohistiocytosis (pHLH) with central nervous system (CNS) involvement, based on data from patients treated at Beijing Children's Hospital between September 2017 and September 2022. A total of 67 pHLH patients were included, with a median age of 4.3 years. Our findings revealed distinct genetic mutation distributions between CNS-pHLH and pHLH without CNS involvement; specifically, CNS-pHLH patients had significantly fewer XIAP mutations but more PRF1 mutations (P=0.006 and 0.002, respectively). In addition, the 3-year overall survival (OS) rate for CNS-pHLH patients was markedly lower compared with those without CNS involvement (50.2%±18.42% vs. 93.3%±9.02%, P<0.001). Hematopoietic stem cell transplantation (HSCT) was strongly associated with better prognosis in CNS-pHLH patients, yielding a 3-year OS of 82.0%±23.91% in transplanted patients, significantly higher than the 28.6%±19.40% in nontransplanted patients (P<0.001). Furthermore, a Cox regression analysis identified PRF1 mutation, cerebrospinal fluid (CSF) soluble CD25 levels above 280 pg/mL, and elevated ferritin levels as independent risk factors for poor 3-year event-free survival (EFS) in CNS-pHLH (P<0.05). These findings suggest that pHLH patients with PRF1 mutations should undergo regular CNS monitoring to prevent disease progression, and that HSCT may improve long-term prognosis in CNS-pHLH cases.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":""},"PeriodicalIF":0.9,"publicationDate":"2025-06-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144475744","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":"Wilms Tumor and Paraneoplastic Syndromes: Unusual Presentation With Acquired von Willebrand Disease, Polycythemia and Hyperviscosity-A Comprehensive Case Report and Literature Reviews.","authors":"Marina Parisi Dutra, Lilian Maria Cristofani, Maria Teresa Assis de Almeida, Vicente Odone Filho, Roberto Augusto Plaza Teixeira","doi":"10.1097/MPH.0000000000003055","DOIUrl":"https://doi.org/10.1097/MPH.0000000000003055","url":null,"abstract":"<p><p>A 10-month-old child presenting with renal tumor with polycythemia, acquired von Willebrand disease, and elevated serum hyaluronic acid, leading to a diagnosis of Wilms tumor. Laboratory profile showed elevated levels of hemoglobin and hematocrit, prolonged aPTT, and high levels of hyaluronic acid and erythropoietin, which normalized after chemotherapy and tumor resection. She had a complete response and has remained in remission. Paraneoplastic syndromes are rare manifestations of Wilms tumor (WT). The simultaneous occurrence of distinct types, as demonstrated in this report (polycythemia, hyperviscosity, and coagulopathy), has not been previously reported. This unique presentation poses a challenge for both diagnostic and clinical management.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":""},"PeriodicalIF":0.9,"publicationDate":"2025-06-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144475747","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":"Iohexol Clearance and Biomarker Analysis to Predict Toxicity in Patients With Acute Lymphoblastic Leukemia and Lymphoma Receiving High-Dose Methotrexate.","authors":"Amy L Walz, Masha Kocherginsky, Monica Newmark, Ellen Brooks, David Walterhouse","doi":"10.1097/MPH.0000000000003075","DOIUrl":"https://doi.org/10.1097/MPH.0000000000003075","url":null,"abstract":"<p><strong>Background: </strong>High-dose methotrexate (HDMTX) remains integral to acute lymphoblastic leukemia/lymphoma (ALL) treatment. However, high MTX concentrations can lead to acute kidney injury (KI) and other toxicities. We investigated whether measured GFR (mGFR) by iohexol clearance better predicts delayed MTX excretion and/or toxicity compared with standard of care using an estimated GFR (eGFR). We also examined if KI biomarkers (urine KIM-1 and clusterin, serum cystatin C, and plasma FGF23) identify KI more frequently than serum creatinine (sCr) alone.</p><p><strong>Procedure: </strong>ALL patients receive 4 doses of HDMTX with alkalinized IV fluids, leucovorin rescue, and MTX clearance per the standard of care. We obtained mGFRs before HDMTX doses 1 and 4. eGFR was calculated using the Schwartz formula and biomarkers of KI were collected around each HDMTX dose.</p><p><strong>Results: </strong>Overall, there were some associations between the mGFR/biomarkers with KI and other toxicities, but mGFR was not found to be a better predictor of delayed MTX clearance or toxicity than eGFR. The biomarkers did not predict KI development more frequently than sCr alone.</p><p><strong>Conclusions: </strong>On the basis of this study, there is no evidence that the current standard of care for determining the GFR in advance of HDMTX administration, nor postadministration management, should be adjusted.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":""},"PeriodicalIF":0.9,"publicationDate":"2025-06-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144475743","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":"Adolescent and Young Adult (AYA) Oncology in the United States in 2025: Finding Its Place in the Oncology World-Part 1 of 2.","authors":"Peter H Shaw, Sharon M Castellino, Jonathan Metts, Ryan Guerrettaz, Brandon Hayes-Lattin, Hong De Sa, Archie Bleyer","doi":"10.1097/MPH.0000000000003067","DOIUrl":"https://doi.org/10.1097/MPH.0000000000003067","url":null,"abstract":"<p><p>In 2015 this core of authors wrote a \"state of the union\" overview of AYA oncology care at the time titled \"Adolescent and Young Adult (AYA) Oncology in the United States: A Specialty in Its Late Adolescence.\" Since then, the landscape of cancer care in this unique population has changed, with encouraging improvement in some areas and persistent challenges in others. Nine years later, we have decided to update our review to demonstrate how far we have come in caring for 15 to 39-year olds with cancer in the United States and how much further we need to go to truly improve both their short-term and long-term outcomes. What started as a call to arms after the AYA-specific Progress Review Group (PRG) in 2006 became a national initiative, which has had successes and failures nationally, regionally, and locally in trying to move the needle for a group of patients that continues to straddle both the pediatric and adult oncology worlds. Back in 2015, we described the field as in its late adolescence, still trying to define itself. With this 2-part review, we hope to demonstrate that as a subspecialty it has grown up but is still trying to firmly establish its place in the larger world of oncology, much like a young adult that has moved away from home and is establishing its own identity in a changing world. In part 1 we focus on epidemiology of AYA cancer as well as acute lymphoblastic leukemia, Hodgkin lymphoma, non-Hodgkin lymphoma, and sarcoma in this unique population of patients.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":""},"PeriodicalIF":0.9,"publicationDate":"2025-06-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144475738","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":"Antifungal Use in Immunocompromised Children in Europe: A 12-Week Multicenter Weekly Point Prevalence Survey (CALYPSO).","authors":"Elisavet Chorafa, Elias Iosifidis, Andrea Oletto, Adilia Warris, Elio Castagnola, Roger Bruggemann, Andreas H Groll, Thomas Lehrnbecher, Laura F Antolin, Alessio Mesini, Aisha A Alkhaaldi, Fernando Baquero-Artigao, Benhur S Cetin, Daniel Ebrahimi-Fakhari, Marieke Emonts, Susanna Esposito, Valentina Fainardi, Elisabetta Ghimenton-Walters, Manuel Gijón, Alba G Guerrero, Carlos D Grasa, Igne Kairiene, Kornelija Kildonaviciute, Maria Kourti, Angela Manzanares, Natalia Mendoza-Palomar, Maria Noni, Eugenia Papakonstantinou, Stéphane Paulus, Thomas Perwein, Jelena Rascon, Elena Rincón-López, Pere Soler-Palacin, Galina Solopova, Vassiliki Spoulou, Volker Strenger, Kara Tedford, Christina Tzika, Borbala Zsigmond, Emmanuel Roilides","doi":"10.1097/MPH.0000000000003070","DOIUrl":"https://doi.org/10.1097/MPH.0000000000003070","url":null,"abstract":"<p><p>We prospectively analyzed antifungal use in immunocompromised children through a multicenter 12-week weekly point-prevalence survey in 31 hematology-oncology (HO) and hematopoietic stem cell/solid organ transplant (HSCT/SOT) units of 18 hospitals in 11 European countries. All patients hospitalized and receiving systemic antifungals were included. Ward policies, and weekly ward/patient data were collected. All 21 HO and 10 HSCT/SOT units had prophylaxis policies for high-risk patients (27/31 used azoles, 14/31 echinocandins and 15/31 liposomal amphotericin B [LAMB]). Among 572 courses recorded, prophylaxis was indicated in 439/572 (77%) and treatment in 133/572 (62/133 empirical, 43/133 pre-emptive, 28/133 targeted). Among patients receiving prophylaxis, 56% belonged to the non-high-risk group. Most common reasons for empirical, pre-emptive and targeted treatment were antibiotic-resistant febrile neutropenia (52%), abnormalities on chest-CT with/without positive galactomannan (77%) and candidiasis (82%), respectively. Fluconazole and LAMB were the most frequently prescribed agents both for prophylaxis (31%, 21%) and treatment (32%, 23%). Underdosing of micafungin for treatment in 50% of prescriptions and of fluconazole for treatment and prophylaxis in 70% of cases was noticed. In conclusion, most antifungal prescribing was for prophylaxis, with fluconazole being the main antifungal prescribed. Inadequate doses of antifungal prescribing and prophylaxis of non-high-risk patients could be targets for improvement.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":""},"PeriodicalIF":0.9,"publicationDate":"2025-06-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144475739","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":"Calcineurin-Inhibitor-Induced Pain Syndrome.","authors":"Yu Furui, Shoji Saito","doi":"10.1097/MPH.0000000000003068","DOIUrl":"https://doi.org/10.1097/MPH.0000000000003068","url":null,"abstract":"","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":""},"PeriodicalIF":0.9,"publicationDate":"2025-06-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144475740","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":"Clinical Implications of Solitary Subependymal Giant Cell Astrocytoma in the Absence of Tuberous Sclerosis Complex: Case Series and Comprehensive Literature Review.","authors":"Shea Gallus, Alexa Markl, Ben Posorske, Nishant Tiwari, Michael Kuwabara, Lisa Keller, Lindsey M Hoffman, Ross Mangum","doi":"10.1097/MPH.0000000000003063","DOIUrl":"https://doi.org/10.1097/MPH.0000000000003063","url":null,"abstract":"<p><p>Subependymal giant cell astrocytoma (SEGA) is a slow-growing glial or glioneuronal tumor that almost exclusively occurs in patients with Tuberous Sclerosis Complex (TSC), a rare autosomal dominant condition that causes growth of benign tumors throughout the body. Herein, we present 4 cases of isolated SEGA in patients with negative germline testing for TSC alterations and present a comprehensive literature review of other cases of sporadic SEGA. This case series emphasizes the importance of considering SEGA on the differential diagnosis for periventricular tumors even in the absence of other sequelae of TSC and illustrates the importance of long-term monitoring for tuberous sclerosis-related complications.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":""},"PeriodicalIF":0.9,"publicationDate":"2025-06-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144475741","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}