Ibrahim Halil Karahan, Tezer Kutluk, Bilgehan Yalcin, Burca Aydin, Nilgun Kurucu, Melis Gultekin, Ferah Yildiz, Diclehan Orhan, Gokhan Gedikoglu, Ali Varan
{"title":"Clinical Features and Treatment Results in Children With Head and Neck Rhabdomyosarcoma.","authors":"Ibrahim Halil Karahan, Tezer Kutluk, Bilgehan Yalcin, Burca Aydin, Nilgun Kurucu, Melis Gultekin, Ferah Yildiz, Diclehan Orhan, Gokhan Gedikoglu, Ali Varan","doi":"10.1097/MPH.0000000000003078","DOIUrl":"10.1097/MPH.0000000000003078","url":null,"abstract":"<p><p>Rhabdomyosarcoma constitutes 3% to 4% of childhood cancers, with nearly half seen in the head and neck location. We aimed to investigate the clinical features and treatment outcomes of 65 children diagnosed and treated for head and neck rhabdomyosarcoma (RMS) between 2004 and 2018. The median age was 5.8 years with a 37:28 M/F ratio. The primary location was parameningeal in 49.2%, orbital in 35.4%, and other nonparameningeal in 15.4% patients. The most common histopathologic subtype was the embryonal subtype (73.8%). The chemotherapy regimens of CDCV (cisplatin, doxorubicin, cyclophosphamide, vincristine); VAC/VAdrC (vincristine, actinomycin-D, cyclophosphamide/vincristine, doxorubicin, cyclophosphamide); PIAV (ifosfamide, cisplatin, vincristine, doxorubicin); and VDC/IE (vincristine, doxorubicin, cyclophosphamide, ifosfamide, etoposide) were used depending on the years of diagnosis. The tumor location, risk grouping, and stage were found as the significant prognostic factors. The 5-year event-free survival (EFS) rate for all patients 41.2% and the overall survival (OS) rate was 59.3%. The 5-year OS rates were 85.2% and 80% in the orbital and other nonparameningeal RMS, respectively, it was 34.2% in the parameningeal RMS patients ( P =0.01). The patients with advanced stage, parameningeal disease have poor prognosis. New treatment approaches should be investigated to improve the outcomes in these groups.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"e241-e246"},"PeriodicalIF":0.9,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144540610","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}
Amy L Walz, Masha Kocherginsky, Monica Newmark, Ellen Brooks, David Walterhouse
{"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":"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":"e235-e240"},"PeriodicalIF":0.9,"publicationDate":"2025-08-01","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}
Mahdi Asleh, Carmel Levi, Yusif Abu Alhasan, Hagit Miskin, Dana Danino
{"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":"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":"e249-e253"},"PeriodicalIF":0.9,"publicationDate":"2025-08-01","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}
Thao Minh Nguyen, Sara Sadiq, Joshua M Peterson, Leonard K Wang, Gulrukh Botiralieva, Yaroslav Chernov, Akila Muthukumar, Kirill A Lyapichev
{"title":"Concurrent Case of Glucose-6-Phosphate Deficiency and Dehydrated Hereditary Stomatocytosis in a 4-Month-old Boy.","authors":"Thao Minh Nguyen, Sara Sadiq, Joshua M Peterson, Leonard K Wang, Gulrukh Botiralieva, Yaroslav Chernov, Akila Muthukumar, Kirill A Lyapichev","doi":"10.1097/MPH.0000000000003057","DOIUrl":"10.1097/MPH.0000000000003057","url":null,"abstract":"<p><p>Glucose-6-phosphate (G6PD) deficiency is the most prevalent enzyme deficiency and is estimated to affect 400 million people. The patients are usually asymptomatic and diagnosed following hemolytic episodes triggered by oxidative stress. Another type of hemolytic anemia known as dehydrated hereditary stomatocytosis (DHSt) is estimated to affect less than 1 per 1,000,000 people. DHSt is caused by increased cation efflux and dehydration in red blood cells, which leads to decreased flexibility making them more vulnerable to lysis. Compared with G6PD, DHSt has a mild presentation, where most patients (84%) with isolated DHSt exhibit chronic hemolysis. Both diseases, G6PD deficiency and DHSt, are inherited hemolytic anemias and to the best of our knowledge have never been reported to coexist in the same patient. Herein, we present the first case of concurrent G6PD deficiency and DHS in a 4-month-old male. We discuss the clinical presentation and hematopathology findings from this patient as well as provide a comparison literature review. We believe this presentation will add to the current body of knowledge for these conditions and help to guide future investigation and management.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"e278-e281"},"PeriodicalIF":0.9,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144248389","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}
Jacob Silverman, Melissa Mégalli, Emilie Giguère, Sarah Elbaz, Melanie Letourneau, Catherine Achim, Cynthia Hawkins, William Legallou, Panagiota Giannakouros, Sébastien Perreault, Geneviève Legault, Eric Bouffet, Nada Jabado, Hallie Coltin, Louis Crevier, Samuele Renzi
{"title":"Adjuvant Etoposide for Very High-risk PFA Ependymoma: A Case Report.","authors":"Jacob Silverman, Melissa Mégalli, Emilie Giguère, Sarah Elbaz, Melanie Letourneau, Catherine Achim, Cynthia Hawkins, William Legallou, Panagiota Giannakouros, Sébastien Perreault, Geneviève Legault, Eric Bouffet, Nada Jabado, Hallie Coltin, Louis Crevier, Samuele Renzi","doi":"10.1097/MPH.0000000000003080","DOIUrl":"10.1097/MPH.0000000000003080","url":null,"abstract":"<p><strong>Background: </strong>Ependymomas of the posterior fossa type A (PF-A) with a combined chromosome 1q gain and 6q loss are associated with an extremely high risk of recurrence and a very poor outcome.</p><p><strong>Observations: </strong>We report the case of a 4-year-old girl who received adjuvant oral etoposide for 1 year after conventional treatment (surgery and focal radiation). The patient remains in clinical and radiologic remission 2.5 years post-diagnosis.</p><p><strong>Conclusions: </strong>This approach of using oral etoposide could be considered in ultra-high-risk 6q loss PF-A ependymoma to try and decrease the risk of relapse, awaiting further evaluation in a clinical trial.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"e269-e272"},"PeriodicalIF":0.9,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144540609","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":"Hypercalcemia of Malignancy in Pediatric Population: Clinical Challenges Explored Through Cases-A Case Series.","authors":"Alpas Anand, Neha Goel, Amitabh Singh, Prashant Prabhakar, Meghna Kumari, Hashmat Singh, Suruchi Viz, Nidhi Chopra, Sumit Mehndiratta","doi":"10.1097/MPH.0000000000003079","DOIUrl":"10.1097/MPH.0000000000003079","url":null,"abstract":"<p><strong>Background: </strong>Hypercalcemia is an uncommon but clinically significant complication of pediatric malignancies that often presents with symptoms. However, in this case series, we report 4 children with nonspecific symptoms of hypercalcemia detected incidentally on routine biochemical evaluation at the time of leukemia diagnosis.</p><p><strong>Case presentation: </strong>Patients, 5 to 9 years of age, had underlying malignancies, including Hodgkin lymphoma (stage IIA), pre-B-cell acute lymphoblastic leukemia, and acute promyelocytic leukemia. All the patients presented with severe hypercalcemia, necessitating prompt intervention. Treatment consisted of hyperhydration, loop diuretics, bisphosphonates, and calcitonin, along with the management of the underlying malignancy. Successful resolution of hypercalcemia was achieved in all cases, with no future recurrence.</p><p><strong>Conclusion: </strong>This case series highlights the importance of the routine biochemical screening for newly diagnosed pediatric malignancies, particularly for nonspecific symptoms of hypercalcemia, which may otherwise go unrecognized. Although hypercalcemia in hematological malignancies is well-documented, our findings stress the silent presentation and reinforce the clinical need for early identification to avoid metabolic complications.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":"47 6","pages":"e254-e259"},"PeriodicalIF":0.9,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144698890","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}
Iris Fried, Michael Weintraub, Dalia Waldman, Shoshana Revel-Vilk
{"title":"Real-life Use of IV Naxitamab for Children With Relapsed/Refractory Neuroblastoma.","authors":"Iris Fried, Michael Weintraub, Dalia Waldman, Shoshana Revel-Vilk","doi":"10.1097/MPH.0000000000003081","DOIUrl":"https://doi.org/10.1097/MPH.0000000000003081","url":null,"abstract":"<p><p>The anti-GD2 antibody Naxitamab requires complicated pain management due to significant adverse effects (AEs). Herein, we report the safety and feasibility of a simplified administration protocol. Between November 2021 and July 2023, 102 Naxitamab infusions were administered at Shaare Zedek Hospital with ketamine as the only analgesic drug during Naxitamab infusion. Pain was controlled in all the cases. Other grade 3-4 AEs, occurred in 10/102 infusions (10%), and none of the patients stopped Naxitamab due to infusion-related AEs. Single-agent ketamine is safe and effective for Naxitamab-infusion-related pain. Thus, its use is feasible in multiple clinical settings. Significant infusion-related AEs after the completion of the 3rd treatment cycle are rare.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":"47 6","pages":"e282-e284"},"PeriodicalIF":0.9,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144698892","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":"Ovarian Mitotically Active Cellular Fibroma of Childhood: Review of Literature and Report of a Case.","authors":"Aileen Azari-Yam, Sahar Montazeri, Bahar Ashjaei, Soheila Sarmadi","doi":"10.1097/MPH.0000000000003059","DOIUrl":"10.1097/MPH.0000000000003059","url":null,"abstract":"<p><p>Ovarian fibromas, especially those of the mitotically active cellular subtype, are very rare in children; however, children with nevoid basal cell carcinoma syndrome (NBCCS) are prone to these neoplasms. We present a 12-year-old girl with an ovarian mitotically active cellular fibroma (MACF) with no personal or family history of NBCCS who underwent right salpingo-oophorectomy. We searched the English literature on pediatric ovarian MACF. To date, only 3 cases of MACF in children have been reported. We present a pediatric case that represents the youngest reported case of MACF thus far. Special attention is required to differentiate this tumor from its malignant mimics to avoid overtreatment, such as radical surgeries. Long-term follow-up and thorough investigation of the manifestations of hereditary cancer syndromes should be considered.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"279-283"},"PeriodicalIF":0.9,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144248392","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}
Peter H Shaw, Sharon M Castellino, Jonathan Metts, Ryan Guerrettaz, Brandon Hayes-Lattin, Hong De Sa, Archie Bleyer
{"title":"Adolescent and Young Adult 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":"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":"265-278"},"PeriodicalIF":0.9,"publicationDate":"2025-08-01","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}