Journal of Pediatric Hematology/Oncology最新文献

{"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":"https://doi.org/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":""},"PeriodicalIF":0.9,"publicationDate":"2025-07-02","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}

{"title":"Universal Premedication Does Not Impact Transition to Short-acting Asparaginase or Incidence of Hypersensitivity to Pegylated Asparaginase: A Single-institution Experience.","authors":"Lindsey A Murphy, Zanette K Bradley, Allie N Elozory, Dexiang Gao, Kelly E Faulk","doi":"10.1097/MPH.0000000000003034","DOIUrl":"10.1097/MPH.0000000000003034","url":null,"abstract":"<p><p>Asparaginase is a critical component of therapy for acute lymphoblastic leukemia/lymphoma but is associated with hypersensitivity reactions. Severe reactions necessitate a transition to an Erwinia asparaginase product, which requires more frequent dosing and higher costs. Children's Hospital Colorado implemented universal pegaspargase premedication in May 2020 and a retrospective analysis was conducted to compare the rates of transition to Erwinia products and hypersensitivity reactions between the preimplementation and postimplementation cohorts. In this large single-institution experience, universal premedication did not impact rates of Erwinia transition or hypersensitivity reactions.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"e181-e186"},"PeriodicalIF":0.9,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144022926","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":"Use of Animal-assisted Therapy in Children With Cancer.","authors":"Hüseyin Çaksen","doi":"10.1097/MPH.0000000000003041","DOIUrl":"10.1097/MPH.0000000000003041","url":null,"abstract":"","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"e209"},"PeriodicalIF":0.9,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144031411","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":"Tracing a Rare Genetic Disease: Familial Congenital CD59 Deficiency and Carrier Cases Identified through Village Screening: Erratum.","authors":"Şefika Lknur Kökcü Karadağ, Medine Karadağ Alparslan, Hüseyin Karadağ, Eda Turgut Uğurtay, Cansu Can, Alisan Yildiran","doi":"10.1097/MPH.0000000000003054","DOIUrl":"https://doi.org/10.1097/MPH.0000000000003054","url":null,"abstract":"","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":"47 5","pages":"e214"},"PeriodicalIF":0.9,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144475748","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":"Comparison of Consolidation Strategies for Pediatric Patients With Acute Myeloid Leukemia: Results of the Randomized GATLA 8-LMA-P'07 Trial.","authors":"Alejandra Deana, Sergio M Gomez, Alcira Beatriz Fynn, Daniel Freigeiro, Maria Cecilia Riccheri, Lorena Elizabeth Moran, Monica Leonor Makiya, Lilian Sung","doi":"10.1097/MPH.0000000000003028","DOIUrl":"10.1097/MPH.0000000000003028","url":null,"abstract":"<p><strong>Objective: </strong>The primary objective was to determine whether consolidation (CONS) with 2 short chemotherapy cycles using cytarabine plus idarubicin and high dose cytarabine plus mitoxantrone (2-cycle) reduced the cumulative incidence of relapse compared with the standard regimen of a 6-week CONS phase among newly diagnosed pediatric patients with acute myeloid leukemia (AML).</p><p><strong>Patients and methods: </strong>GATLA 8-LMA-P'07 was a phase 3 trial conducted in 26 centers in Argentina. We included newly diagnosed pediatric patients with AML 0 to 18 years of age. Patients with M3 AML were excluded. After 2 cycles of induction, patients in remission were randomized to either CONS or 2-cycle CONS chemotherapy. High-risk patients received matched family stem cell transplantation or maintenance therapy for 12 months.</p><p><strong>Results: </strong>One hundred seven patients younger than 18 years with de novo AML were randomized to CONS (n = 52) or 2-cycle (n = 57). Cumulative incidence (SE) of relapse was not significantly different between CONS (31% [0.1]) and 2-cycle (39% [0.1]) CONS ( P = 0.25). There was no significant difference in 5-year event-free survival (53.6% [0.8] vs 44.3 [0.7], P = 0.31) or 5-year overall survival (55.0% [0.8] vs 53.7% [0.7], P = 0.91) for CONS and 2-cycle CONS respectively.</p><p><strong>Conclusions: </strong>CONS with 2 cycles of chemotherapy was not significantly better than the standard CONS in reducing the cumulative risk of relapse among newly diagnosed children with AML from Argentina. Future research should evaluate new approaches to improve outcomes for pediatric patients with AML.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"250-256"},"PeriodicalIF":0.9,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144078677","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":"Managing and Preventing Hypofibrinogenemia in Pediatric and AYA Leukemia Patients: Insights From MD Anderson Cancer Center.","authors":"Jiasen He, Faryal Munir, Camila Ayerbe, Samantha Dickson, Samanta Catueno, Branko Cuglievan, Amber Gibson, David McCall, Cesar Nunez, Michael Roth, Priti Tewari, Miriam B Garcia, Nidra Rodriguez, Jose Cortes","doi":"10.1097/MPH.0000000000003042","DOIUrl":"10.1097/MPH.0000000000003042","url":null,"abstract":"<p><strong>Background: </strong>Cryoprecipitate is often used to prevent and treat complications associated with low fibrinogen levels in pediatric leukemia patients. Cryoprecipitate, rich in fibrinogen, is administered to augment fibrinogen levels and mitigate the risk of bleeding in these patients. The use of cryoprecipitate is often strategic, involving both prophylactic measures and interventions in response to bleeding events. This approach plays a crucial role in the comprehensive care of pediatric leukemia patients, particularly during periods of heightened vulnerability to bleeding complications. However, data regarding the use of cryoprecipitate in children with leukemia are lacking.</p><p><strong>Methods: </strong>We conducted a retrospective chart review of children, adolescents, and young adults with leukemia who received cryoprecipitate at the University of Texas MD Anderson Cancer Center from 2020 to 2022. We gathered baseline clinical and demographic data, cryoprecipitate usage details, and fibrinogen levels. Paired-samples t tests were used to compare fibrinogen levels before and after cryoprecipitate infusion.</p><p><strong>Results: </strong>We identified 36 patients who received cryoprecipitate, 1 to 25 years of age, 67% of whom (24/36) were male. In this cohort, 27/36 (75%) were recently exposed to asparaginase, 2/36 (6%) had a history of venous thromboembolism, and 6/36 (17%) had a history of major bleeding. Cryoprecipitate was used to treat active bleeding (11/36, 31%), bleeding prevention (22/36, 61%), and preoperatively (3/36, 8%). Patients frequently required transfusions of other blood products. Common comorbidities in patients receiving cryoprecipitate included disseminated intravascular coagulation (10/36, 28%) and sepsis (10/36, 28%). The median baseline fibrinogen level across the entire study population was 85.5 mg/dL (IQR: 59.8 to 113). The median dose of cryoprecipitate infused was 6.6 mL/kg (IQR: 3.09 to 14.6), and the median postinfusion peak fibrinogen level was 185 mg/dL (IQR: 155 to 292) ( P <0.001). No major direct treatment-related adverse events were reported.</p><p><strong>Conclusion: </strong>Cryoprecipitate is commonly administered to children with leukemia, effectively raising fibrinogen levels with minimal associated side effects. However, further research, ideally through randomized trials, is needed to assess its true clinical benefits in this population. Currently, there is a notable lack of pediatric-focused randomized transfusion medicine trials. Integrating these studies into ongoing oncologic trials could be a practical and valuable approach that warrants careful consideration and planning.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"235-241"},"PeriodicalIF":0.9,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143970857","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":"Successful Unmanipulated Haploidentical Hematopoietic Stem Cell Transplant With Post-transplant Cyclophosphamide in a Child With Down Syndrome and Myelodysplastic Syndrome.","authors":"Chane Choed-Amphai, Yigal Dror, Michaela Cada, Tal Schechter, Joerg Krueger, Muhammad Ali, Yogi Chopra","doi":"10.1097/MPH.0000000000003051","DOIUrl":"10.1097/MPH.0000000000003051","url":null,"abstract":"<p><p>Allogeneic hematopoietic stem cell transplant (HSCT) in children with Down syndrome and hematologic malignancies is challenging and is reserved for those who develop relapsed/refractory disease due to concerns regarding transplant-related mortality. Haploidentical HSCT, although performed in limited cases using graft manipulation methods to prevent graft-versus-host disease (GVHD), often results in dismal outcomes. Herein, we report a case of a 12-year-old boy with Down syndrome and myelodysplastic syndrome who underwent unmanipulated haploidentical HSCT using a reduced toxicity treosulfan-based conditioning and in vivo T-cell depletion with post-transplant cyclophosphamide. At the 1-year follow-up, he is alive with complete donor chimerism and no chronic GVHD.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"e199-e202"},"PeriodicalIF":0.9,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144078682","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":"Exploring Maintenance Therapy in Pediatric Embryonal Tumor With Multilayered Rosettes.","authors":"Mohamed M Ahmed, Fernando Carceller, Leslie R Bridges, Conor Mallucci, Navneet Singh, Sucheta Vaidya","doi":"10.1097/MPH.0000000000003033","DOIUrl":"10.1097/MPH.0000000000003033","url":null,"abstract":"<p><p>Embryonal tumors with multilayered rosettes (ETMR) represent a distinct entity characterized by aggressive behavior. Historical retrospective analyses have documented dire overall survival rates ranging from 0% to 14% at 1 year. However, a contemporary report by Khan and colleagues shows overall survival rates reaching 29% at 2 years and 27% at 4 years. We present the case of an 18-month-old girl diagnosed with ETMR, confirmed by chromosome 19 microRNA cluster amplification following initial presentation with focal seizures. The patient underwent a combination of surgical interventions, high-dose chemotherapy with stem cell rescue, and proton therapy, achieving a disease-free status after completing standard treatment. Subsequently, a 12-month maintenance regimen comprising intrathecal topotecan, oral sodium valproate, and oral cis-retinoic acid was administered. The maintenance therapy was well tolerated, with manageable adverse effects. The patient remains progression-free for 32 months postmaintenance therapy (50 months from initial presentation). This study explores the feasibility and safety profile of maintenance therapy in ETMR. Future studies may explore this approach to determine its efficacy in children with ETMR.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"242-245"},"PeriodicalIF":0.9,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143803458","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":"Ex Vivo Drug Screening: An Emerging Paradigm in the Treatment of Childhood Cancer.","authors":"Anees Ahmed, Ellen Cox, Louis Lane, Ola Rominiyi, Sarah Danson, Helen E Bryant, Greg Wells, David King","doi":"10.1097/MPH.0000000000003017","DOIUrl":"10.1097/MPH.0000000000003017","url":null,"abstract":"<p><p>Developing and providing the right therapy for the right patient (or personalized targeted treatments) is key to reducing side-effects and improving survival in childhood cancers. Most efforts aiming to personalize childhood cancer treatment use genomic analysis of malignancies to identify potentially targetable genetic events. But it is becoming clear that not all patients will have an actionable change, and in those that do there is no additional way to determine if treatments will be effective. Ex vivo drug screening is a laboratory technique used to test the effects of various drugs or compounds, on biological tissues or cells that have been removed from an organism. This information is then used to predict which cancer treatments will be most effective based on the therapeutic response in the tissue or cells removed from that individual. Its utility in personalizing treatments in childhood cancer is increasingly recognized. In this review we describe the different methods for ex vivo drug screening and the advantages and disadvantages of each technique. We also present recent evidence that ex vivo screening may have utility in a variety of childhood malignancies including an overview of current clinical trials appraising its use. Finally, we discuss the research questions and hurdles that must be overcome before ex vivo screening can be widely used in pediatric oncology.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"e144-e154"},"PeriodicalIF":0.9,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143630576","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":"Pyrites: A Thigh Mass.","authors":"Sam Lyvannak, Has Sotherak, Thy Bunpaov, Bun Sereyleak, Jason Jarzembowski, Bruce Camitta","doi":"10.1097/MPH.0000000000003036","DOIUrl":"10.1097/MPH.0000000000003036","url":null,"abstract":"","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"263-264"},"PeriodicalIF":0.9,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143972494","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}