{"title":"Myeloproliferative Neoplasm With Eosinophilia Exhibiting a Rare GOLGA4-PDGFRB Rearrangement in an Infant: Navigating a Rare Malignancy Amidst Limited Pediatric Guidelines.","authors":"Yamini Krishnan, Smitha Bhaskaran, Gazel Sainulabdin, Krishnan Vettakorumakankavu Parameswaran, Abhishek Mohanty","doi":"10.1097/MPH.0000000000003189","DOIUrl":"10.1097/MPH.0000000000003189","url":null,"abstract":"<p><p>Myeloid/lymphoid neoplasms with eosinophilia (MLN-eos) are rare hematological malignancies in childhood and limited to isolated case reports in the literature. We discuss the case of an infant who was evaluated for persistent hypereosinophilia and diagnosed with a rare genetic rearrangement; GOLGA4::PDGFRB was previously reported only once in a 13-month-old boy. A literature review of all children with PDGFRB rearrangement was collated in our analysis. The challenges of diagnosis, treatment, and follow-up posed by such a rare genetic disorder with no pediatric guidelines are being discussed.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"173-177"},"PeriodicalIF":0.8,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147529683","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 Characteristics and Outcome of Leukemia and Malignant Solid Tumors Among Infant Age Group: A 15-Year Study in a Tertiary Care Hospital in Egypt.","authors":"Esraa M A Hasan, Nayera H K Elsherif","doi":"10.1097/MPH.0000000000003190","DOIUrl":"10.1097/MPH.0000000000003190","url":null,"abstract":"<p><strong>Background and objectives: </strong>Cancer in infancy represents a unique and challenging entity. They differ clinically and biologically from childhood cancer, resulting in different management and outcomes. Cancer epidemiological studies and population-based registries among this age group are limited, herby we aimed to reflect our hospital-based data on the incidence and survival of cancer among infants over a 15-year period.</p><p><strong>Patients and methods: </strong>We retrospectively analyzed the clinical data of infants aged 0 to 12 months diagnosed with hematological malignancies and malignant solid tumors registered in the Pediatric Oncology Department of our university hospital from January 2006 to December 2021.</p><p><strong>Results: </strong>Infant malignancies represented 4.6% of all pediatric malignancies diagnosed at our institute over the same study period. Hematological malignancies accounted for 18.3%, while solid malignant tumors accounted for 81.7%. Retinoblastoma was the most common malignant solid tumor in this study (39.7%), followed by neuroblastoma and germ cell tumor. The 5- and 10-year overall survival rates for hematological and solid tumors were 30.8% and 75.9%, respectively. The overall survival rates were highly significant among solid tumors compared with hematological malignancies ( P ≤0.001).</p><p><strong>Conclusions: </strong>The prevalence of infant malignant tumors varies geographically. This study provides estimates on the incidence and survival rates of cancer among the infant age group in our country. Our overall survival rate was comparable to that reported in the literature. Collaborative research is required to achieve a more comprehensive understanding of infant cancer epidemiology and characteristics, with the aim of improving their prognosis.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"153-159"},"PeriodicalIF":0.8,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147530214","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":"Complete Urinalysis and Urine Culture Results in Children With Febrile Neutropenic Leukemia.","authors":"Veysiye Hülya Üzel","doi":"10.1097/MPH.0000000000003188","DOIUrl":"10.1097/MPH.0000000000003188","url":null,"abstract":"<p><strong>Introduction: </strong>The frequency of urinary tract infection (UTI) in children with leukemia and febrile neutropenia (FN) is still not known exactly. In this study, we aimed to determine the frequency of UTIs in children with leukemia presenting with FN.</p><p><strong>Materials and methods: </strong>Urine culture and complete urinalysis analysis of 87 febrile neutropenia episodes in children with acute leukemia aged 0 to 18 years between January 2015 and December 2020 were retrospectively analyzed.</p><p><strong>Results: </strong>Of the 87 patients, 11 (12.6%) had acute myeloblastic leukemia (AML) and 76 (87.3%) had acute lymphoblastic leukemia (ALL). Complete urinalysis showed negative results in 78 (89.6%) samples and positive results in 9 (10.4%) samples (leukocyte and/or nitrite positivity). Positive urine cultures were found in 15 patients, indicating a UTI prevalence of 17.2%. Of the 15 patients with growth in culture, 6 (40%) had no abnormalities in urinalysis. Most detected microorganisms were gram-negative (n=12, 80%), with Escherichia coli being the most common isolate (n=6, 40%).</p><p><strong>Conclusion: </strong>UTI is as common in children with FEN leukemia as in healthy children. In the neutropenic period, no abnormality may be detected in a complete urinalysis. TMP-SMZ may also provide protection from UTI when used prophylactically in these patients. However, resistant bacteria are becoming increasingly common, and complete urinalysis and urine culture should be routinely studied in the diagnostic evaluation of patients with leukemia presenting with fever and neutropenia. Since this study is retrospective, studies with larger sample sizes are needed.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"197-200"},"PeriodicalIF":0.8,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147530231","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}
Alexander I Karachunskiy, Sergey G Kovalenko, Vladimir V Lebedev, Olga I Plaksina, Natalia I Ponomareva, Vanessa Seif, Qian Meng, Sergey Grankov, Lorene Simonot, Larisa G Fechina, Yulia V Dinikina
{"title":"Phase 2, Multicenter Studies of Pegaspargase in Pediatric Patients With Previously Untreated Acute Lymphoblastic Leukemia in Russia: Comparative Study of Liquid and Lyophilized Pegaspargase and Roll-Over Study of Lyophilized Formulation.","authors":"Alexander I Karachunskiy, Sergey G Kovalenko, Vladimir V Lebedev, Olga I Plaksina, Natalia I Ponomareva, Vanessa Seif, Qian Meng, Sergey Grankov, Lorene Simonot, Larisa G Fechina, Yulia V Dinikina","doi":"10.1097/MPH.0000000000003176","DOIUrl":"10.1097/MPH.0000000000003176","url":null,"abstract":"<p><p>Pegaspargase, a well-established pediatric acute lymphoblastic leukemia (ALL) therapy, is available as a liquid formulation, and a lyophilized formulation with an improved shelf life. The pharmacokinetics and safety of lyophilized and liquid pegaspargase were assessed in pediatric patients with ALL in Russia. In Study 1, patients were randomly assigned (1:1) to receive 1 intravenous injection of lyophilized or liquid pegaspargase; patients who benefited from pegaspargase continued into Study 2 and received lyophilized pegaspargase every 2 weeks for 9 infusions. Of the 89 patients in Study 1, 74 continued into Study 2. In Study 1, maximum observed plasma asparaginase activity and area under the PAA-time curve were similar between groups. Geometric mean ratios were 93.5% (90% confidence interval [CI]: 82.9, 105.5) and 102.8% (90% CI: 91.4, 115.6), respectively; therefore, the 90% CIs were completely contained within the predefined acceptance interval of 80% to 125%. Pegaspargase-related adverse events occurred in 88.4% and 91.1% in the lyophilized and liquid groups, respectively, in Study 1. The safety profile of lyophilized pegaspargase after repeated infusion in Study 2 was consistent with the known safety profile of pegaspargase. Pharmacokinetic exposure does not depend on pegaspargase formulation. These studies support the use of lyophilized pegaspargase.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"160-167"},"PeriodicalIF":0.8,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13095057/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147609186","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":"Infantile Ganglioneuroblastoma Causing Growth Failure and Hypertensive Cardiomyopathy From Excessive Catecholamine Production.","authors":"Haruka Shimizu, Yuki Shimizu, Tomohei Nakao, Atsuhiko Ohta, Ryuhei Tanaka, Keisuke Suzuki, Kouichi Toda, Yujiro Tanaka, Takashi Fukushima","doi":"10.1097/MPH.0000000000003192","DOIUrl":"10.1097/MPH.0000000000003192","url":null,"abstract":"<p><p>Unlike pheochromocytomas, neuroblastomas-which are the most common pediatric extracranial solid tumors-are rarely associated with cardiomyopathy and heart failure due to hypertension. We report the case of a 3-year-old girl with growth failure who had hypertensive cardiomyopathy caused by excessive epinephrine and norepinephrine production from a ganglioneuroblastoma. Circulation improved with alpha-blockers and other medications, which enabled partial tumor resection. Histopathologic analysis confirmed ganglioneuroblastoma. Four courses of James' therapy did not lead to tumor shrinkage; subtotal resection was required, which reduced catecholamine levels. This case underscores the importance of multidisciplinary treatment for hypertensive cardiomyopathy due to excess neuroblastoma-related catecholamines.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"181-184"},"PeriodicalIF":0.8,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147530197","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":"Invasive Fungal Infection in Patients With Initial Treatment of Pediatric Acute Lymphoblastic Leukemia: A Single-Center Study in Japan.","authors":"Ryoji Kobayashi, Daiki Hori, Hirozumi Sano, Satoru Matsushima, Junjiro Ohshima, Daisuke Suzuki","doi":"10.1097/MPH.0000000000003191","DOIUrl":"10.1097/MPH.0000000000003191","url":null,"abstract":"<p><p>The prognosis for pediatric hematological malignancies has improved greatly, but infectious diseases, especially invasive fungal infection (IFI), are an important complication that may make it difficult to continue treatment. In this study, IFIs in the treatment of the initial diagnosis of acute lymphoblastic leukemia (ALL), excluding relapse and transplantation, were investigated retrospectively. The patients were 152 first-diagnosed ALL cases (85 males and 67 females) admitted to our hospital between April 2007 and December 2024. The median age of these patients was 6.8 years. The median leukocyte count was 10.2×10 9 /L and the cell surface markers of the blasts were B precursor in 124 cases, T in 20 cases, mature B in 3 cases, and mixed lineage in 5 cases. The diagnosis of IFI was based on EORTC/MSG diagnostic criteria. Sixteen of the 152 patients (10.5%) were found to have IFIs; one was \"proven,\" 7 were \"probable,\" and 8 were \"possible.\" The median time of IFI onset from the start of ALL treatment was 41 days (-7 to 258 d), with 56.3% of cases occurring at first onset of ALL or during induction therapy. Although 4 of 16 patients with IFIs died, IFI was the direct cause of death in 2 cases. Age 7.5 years or older was the only risk factor for IFI.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"191-196"},"PeriodicalIF":0.8,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147530187","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}
Irvin Yi, Michelle Foley, Aron Flagg, Stephanie Massaro, Daniel Prior
{"title":"Two Cases of Severe Cytomegalovirus Disease in Nontransplant Pediatric Acute Lymphoblastic Leukemia Patients Treated With Tyrosine Kinase Inhibitors.","authors":"Irvin Yi, Michelle Foley, Aron Flagg, Stephanie Massaro, Daniel Prior","doi":"10.1097/MPH.0000000000003193","DOIUrl":"10.1097/MPH.0000000000003193","url":null,"abstract":"<p><strong>Background: </strong>Cytomegalovirus (CMV) disease is a rare but significant complication among nontransplant pediatric leukemia patients. CMV infection has been observed in patients receiving tyrosine kinase inhibitors (TKIs) indicated for the treatment of chronic myeloid leukemia (CML), Philadelphia chromosome-positive (Ph+), and Philadelphia chromosome-like (Ph-like) acute lymphoblastic leukemia (ALL). However, there is limited published data regarding CMV disease in nontransplant leukemia patients treated with TKIs.</p><p><strong>Observations: </strong>We present 2 cases of severe CMV disease in Ph+ and Ph-like ALL patients treated with TKI plus chemotherapy without transplant.</p><p><strong>Conclusions: </strong>We highlight this uncommon but serious adverse event in the pediatric population.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"201-206"},"PeriodicalIF":0.8,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147529669","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":"Follicular Dendritic Cell Sarcoma in an Adolescent Patient.","authors":"Hannah Glanz, Allison Weisnicht, Kristin Bradley, Jessica Gulliver, Darya Buehler, Tabassum Kennedy, Cathy Lee-Miller","doi":"10.1097/MPH.0000000000003194","DOIUrl":"10.1097/MPH.0000000000003194","url":null,"abstract":"<p><strong>Background: </strong>Follicular dendritic cell sarcoma (FDCS) is a rare neoplasm that usually occurs in the adult population. Since its first description, only 9 cases have been reported in children. Due to its rarity, there is no standardized treatment.</p><p><strong>Observations: </strong>We present a case of multiply-relapsed FDCS in an adolescent patient that responded favorably to chemotherapy.</p><p><strong>Conclusion: </strong>For many, wide local excision of FDCS may be curative. However, more research is required to determine the best course of treatment for patients requiring adjuvant chemotherapy or radiation. In addition, expanding genetic research has the potential to provide targeted therapy in the future.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"178-180"},"PeriodicalIF":0.8,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147623206","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}
Bridgette McCarty, Heather Gochnauer, Xochitl Longstaff, Angelina Labib, John Naheedy, Art Kulatti, Jun Q Mo, Natalie Ellington, Hilda Ding, Karen Lee, Benjamin Keller, Dawn Eichenfield, Deborah Schiff
{"title":"Intraosseous Venous Malformation of the Rib With EWSR1-NFATC1 Fusion Mimicking Malignancy: A Pediatric Case Report.","authors":"Bridgette McCarty, Heather Gochnauer, Xochitl Longstaff, Angelina Labib, John Naheedy, Art Kulatti, Jun Q Mo, Natalie Ellington, Hilda Ding, Karen Lee, Benjamin Keller, Dawn Eichenfield, Deborah Schiff","doi":"10.1097/MPH.0000000000003196","DOIUrl":"10.1097/MPH.0000000000003196","url":null,"abstract":"<p><strong>Background: </strong>Intraosseous venous malformations are rare, benign vascular bone lesions that may mimic malignant sarcomas on imaging.</p><p><strong>Observations: </strong>A 14-year-old male presented with fever and cough. Imaging revealed an incidental ossified rib mass with aggressive features. CT-guided biopsy was nondiagnostic, whereas open biopsy demonstrated a vascular lesion positive for CD31, FLI-1, and ERG. Molecular testing identified an EWSR1-NFATC1 fusion. Gross total resection was achieved, followed by prolonged disease-free survival, and the patient resumed full athletic activity 7 months after surgery.</p><p><strong>Conclusions: </strong>This case highlights the value of open biopsy and molecular testing to prevent misdiagnosis and overtreatment of intraosseous venous malformations.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"168-172"},"PeriodicalIF":0.8,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13095055/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147645735","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}
Olivia A Do, Laura C Huang, Lydia T Tam, Spencer Raub, Ralph P Ermoian, Sarah E S Leary, David M Werny, Richard G Ellenbogen, Samuel Emerson, Manuel Ferreira, Amy Lee, Jacob J Ruzevick, Rebecca J Ronsley
{"title":"Postoperative Visual Outcomes in Pediatric Craniopharyngioma: A Comparison of Endoscopic Endonasal and Transcranial Approaches.","authors":"Olivia A Do, Laura C Huang, Lydia T Tam, Spencer Raub, Ralph P Ermoian, Sarah E S Leary, David M Werny, Richard G Ellenbogen, Samuel Emerson, Manuel Ferreira, Amy Lee, Jacob J Ruzevick, Rebecca J Ronsley","doi":"10.1097/MPH.0000000000003195","DOIUrl":"10.1097/MPH.0000000000003195","url":null,"abstract":"<p><strong>Objective: </strong>To compare postoperative visual outcomes in pediatric patients undergoing resection of craniopharyngioma via endoscopic endonasal versus transcranial craniotomy approaches.</p><p><strong>Methods: </strong>We retrospectively reviewed 37 pediatric patients (74 eyes) who underwent resection of histologically confirmed craniopharyngioma at a tertiary center between 1995 and 2023. Ophthalmologic evaluations included best-corrected visual acuity (VA), optic nerve findings, and visual fields. Outcomes were assessed at early, intermediate, and long-term follow-up and compared by surgical approach.</p><p><strong>Results: </strong>Twenty-five patients (68%) underwent craniotomy and 12 (32%) endoscopic resection. Preoperatively, 51% had normal vision in both eyes, while 6 were legally blind. Postoperatively, no significant differences in VA or visual fields were observed between approaches at any interval. Optic nerve pallor was somewhat more frequent in the endoscopic group, though not statistically significant. Gross total resection was achieved in 66% of endoscopic versus 34% of craniotomy cases. Most craniotomies (88%) occurred before 2015, compared with 16% of endoscopic cases.</p><p><strong>Conclusions: </strong>Both endoscopic and transcranial approaches yielded comparable visual outcomes in pediatric craniopharyngioma surgery. Rates of gross total resection were higher in the endoscopic cohort, suggesting that surgical approach should remain anatomy-driven, with either technique capable of preserving visual function in appropriately selected patients.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"185-190"},"PeriodicalIF":0.8,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147592742","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}