Pediatric Hematology Oncology Journal最新文献

{"title":"CAR-T cell therapy for pediatric high grade gliomas: Opportunities, challenges, and future directions","authors":"Mahdi Mehmandoost ,&nbsp;Sara Ahmadi ,&nbsp;Farzan Fahim","doi":"10.1016/j.phoj.2026.100810","DOIUrl":"10.1016/j.phoj.2026.100810","url":null,"abstract":"<div><div>Pediatric gliomas account for approximately 30% of primary CNS tumors in children and range from low-grade pilocytic astrocytoma to aggressive diffuse midline gliomas. High-grade gliomas carry a 5-year survival below 20% despite maximal multimodal therapy, underscoring the need for innovative options. Chimeric antigen receptor T-cell (CAR-T) therapy enables tumor-specific targeting with potential for reduced systemic toxicity and increased survival. Preclinical studies targeting B7-H3, GD2, and HER2 have shown potent cytotoxicity and survival benefits in pediatric glioma models. Early-phase clinical trials of HER2-and GD2-CAR-T in children and adolescents demonstrate feasibility, manageable safety, and occasional radiographic responses, though durable remissions remain rare. Key barriers include antigen heterogeneity, immunosuppressive tumor microenvironment, blood brain barrier, therapy-related toxicities, and limited CAR-T persistence. Emerging strategies such as multi-antigen CAR designs, resistance engineering, locoregional or BBB-penetrant delivery, cytokine armoring, and rapid toxicity mitigation offer promise for overcoming these obstacles. Integrating these innovations into biomarker-driven, multicenter pediatric trials with robust correlative studies will be essential to validate efficacy and ensure safety in this vulnerable population.</div></div>","PeriodicalId":101004,"journal":{"name":"Pediatric Hematology Oncology Journal","volume":"11 1","pages":"Article 100810"},"PeriodicalIF":0.0,"publicationDate":"2026-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147397789","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"High-grade surface Osteosarcoma – Data from the Cooperative Osteosarcoma Study Group (COSS)","authors":"Patricia J. Schäfenacker ,&nbsp;Stefanie Hecker-Nolting ,&nbsp;Stefan Bielack ,&nbsp;Matthias Kevric ,&nbsp;Benjamin Sorg ,&nbsp;Arndt Borkhardt ,&nbsp;Bernd Hertenstein ,&nbsp;Peter Reichardt ,&nbsp;Daniel A. Müller ,&nbsp;Leo Kager ,&nbsp;Mathias Werner ,&nbsp;Claudia Blattmann","doi":"10.1016/j.phoj.2026.100809","DOIUrl":"10.1016/j.phoj.2026.100809","url":null,"abstract":"<div><h3>Background</h3><div>High-grade surface osteosarcoma (HGSOS) is a rare subtype of osteosarcoma, accounting for approximately 1% of all cases. Due to its rarity, data on clinical characteristics, treatment, and outcomes remain limited.</div></div><div><h3>Methods</h3><div>Patients with a histologically confirmed diagnosis of HGSOS registered in the Cooperative Osteosarcoma Study Group (COSS) between 1978 and 2021 were retrospectively analyzed. Event-free survival (EFS) and overall survival rate (OAS) were estimated using Kaplan–Meier analysis, and potential prognostic factors were explored.</div></div><div><h3>Results</h3><div>Thirty-one patients met the inclusion criteria. Median age at diagnosis was 16.8 years, and tumors were predominantly located in the extremities (83.9%). Primary metastatic disease at diagnosis was rare (n = 1). At a median follow-up of 5.33 years, 3- and 5-year EFS were 79.5% and 75.3%, while 3- and 5-year OAS were 82.7% and 78.1%, respectively. Larger tumor volume, poor histological response to neoadjuvant chemotherapy, and early recurrence were associated with inferior outcomes in univariate analyses. In multivariate analysis, tumor localization in an extremity was identified as the only independent predictor of overall survival.</div></div><div><h3>Conclusions</h3><div>Patients with HGSOS treated within multimodal osteosarcoma protocols demonstrated favorable survival outcomes. Prognostic interpretation is limited by the small cohort size; however, tumor localization, tumor volume, histological response, and timing of recurrence appear to influence outcome. Larger collaborative studies are needed to further define prognostic factors in this rare tumor entity.</div></div>","PeriodicalId":101004,"journal":{"name":"Pediatric Hematology Oncology Journal","volume":"11 1","pages":"Article 100809"},"PeriodicalIF":0.0,"publicationDate":"2026-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147397790","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Hemophagocytic lymphohistiocytosis secondary to Varicella Zoster in paediatric Acute Lymphoblastic Leukemia","authors":"Zaibaish Khan,&nbsp;Nishant Verma","doi":"10.1016/j.phoj.2026.100804","DOIUrl":"10.1016/j.phoj.2026.100804","url":null,"abstract":"<div><div>Hemophagocytic lymphohistiocytosis (HLH) is a rare life-threatening syndrome of excessive immune activation. HLH can be inherited or secondary to other diseases. An association between primary varicella-zoster virus infection and HLH has already been reported in adults. Extreme hyperferritinemia is seen in HLH in the pediatric population. Rapid progression and significant mortality require a high clinical index of suspicion and prompt diagnosis for optimal outcome. We report 3 children with acute lymphoblastic leukemia in remission who presented with varicella infection-induced HLH. Initially, laboratory data showed neutropenia in two patients and, hepatitis in one patient. Antiviral therapy, and antibiotics along with supportive treatment were initiated. Patients had persistent symptoms with raised serum ferritin, high clinical index of suspicion for HLH was kept. Treatment was directed towards suppressing the overt immune response. Prednisolone was started and tapered over eight weeks. All three patients responded well to treatment and were discharged.</div></div>","PeriodicalId":101004,"journal":{"name":"Pediatric Hematology Oncology Journal","volume":"11 1","pages":"Article 100804"},"PeriodicalIF":0.0,"publicationDate":"2026-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147397793","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"An update in the diagnosis and therapy of hemophagocytic lymphohistiocytosis","authors":"Elpis Mantadakis,&nbsp;Sonia Alexiadou","doi":"10.1016/j.phoj.2025.100802","DOIUrl":"10.1016/j.phoj.2025.100802","url":null,"abstract":"<div><div>Primary hemophagocytic lymphohistiocytosis (pHLH) is a fatal, autosomal recessive hyperinflammatory syndrome. Infections, neoplasia, and underlying autoimmune and autoinflammatory conditions trigger secondary HLH. In pHLH, immunosuppression with dexamethasone and etoposide has been used as the standard of care before hematopoietic transplantation, the only curative option. However, approximately one-third of the patients do not achieve remission and die before transplantation. As a result, the focus has shifted to novel biologic agents and we discuss these here. Emapalumab (Gamifant®, a monoclonal antibody that neutralizes free and receptor-bound interferon-γ), has been approved in the US for HLH since 2020 based on its efficacy in 34 patients. The European Medicines Agency has refused marketing authorization, expressing concerns regarding its efficacy, since most responding patients required additional therapy. As the JAK/STAT pathway is critical for cytokine signaling and has a significant role in HLH, its suppression by ruxolitinib (Jakafi®, a potent anti-inflammatory inhibitor) has been successful in animal models of HLH. Over the last 6 years, several clinical studies, mainly from China, have studied ruxolitinib's use in HLH. All have shown ruxolitinib to be safe and effective, minimizing or avoiding exposure to chemotherapy. Beyond emapalumab and ruxolitinib, other agents have shown promise in managing aspects of HLH, including interleukin-1 receptor antagonists, such as anakinra, tocilizumab (Actemra®, a recombinant humanized monoclonal antibody against the interleukin-6 receptor), alemtuzumab (Campath® and Lemtrada®, a monoclonal antibody against CD52), and finally, tadekinig alpha, a recombinant interleukin-18 binding protein with a high affinity for IL-18, a major inflammatory cytokine. In summary, a growing number of biological agents are expected to play a key role in the treatment of HLH in the immediate future.</div></div>","PeriodicalId":101004,"journal":{"name":"Pediatric Hematology Oncology Journal","volume":"11 1","pages":"Article 100802"},"PeriodicalIF":0.0,"publicationDate":"2026-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145908861","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Parental experience of hemophilia diagnosis disclosure in Morocco: a mixed-methods study on communication quality and psychosocial impact","authors":"H. Eddukar,&nbsp;M. El Khorassani","doi":"10.1016/j.phoj.2026.100807","DOIUrl":"10.1016/j.phoj.2026.100807","url":null,"abstract":"<div><h3>Background</h3><div>Disclosure of a haemophilia diagnosis in paediatric settings is a critical moment that shapes parental understanding, emotional adjustment, and long-term engagement in care. In Morocco, where healthcare resources are constrained and cultural factors strongly influence family dynamics, little is known about how this diagnosis is communicated to parents. Exploring parental experiences is essential to improve family-centred care and disease management.</div></div><div><h3>Materials and methods</h3><div>A cross-sectional mixed-methods study was conducted at the Paediatric Hematology-Oncology Department of IBN SINA University Hospital, Rabat, in September and October 2024. Thirty-six parents of children with haemophilia A or B were enrolled after informed consent. Data were collected using a structured 34-item questionnaire assessing sociodemographic characteristics, diagnostic disclosure quality based on the SPIKES (Setting, Perception, Invitation, Knowledge, Empathy, Summary) protocol, emotional impact, satisfaction, and parental preferences. Quantitative and qualitative analyses were performed using Jamovi software.</div></div><div><h3>Results</h3><div>Diagnosis disclosure occurred in specialized centres in 66.7 % of cases and was associated with longer consultations (median 30 vs. 10 min, p = 0.011), higher parental satisfaction (83.3 % vs. 16.7 %, p &lt; 0.001), and greater perceived empathy (79.2 % vs. 16.7 %, p &lt; 0.001). Anxiety or depressive symptoms were reported by 83.3 % of parents following disclosure. Higher parental education level was inversely associated with satisfaction, reflecting unmet expectations for detailed information and shared decision-making. Identified barriers included insufficient psychosocial support, unclear explanations, and linguistic discordance. Parents emphasized the importance of empathy, adequate consultation time, follow-up sessions, and gradual disclosure to the child.</div></div><div><h3>Conclusion</h3><div>Significant disparities persist in haemophilia diagnosis disclosure in Morocco. Adapting the SPIKES protocol to the local context, strengthening psychosocial support, and enhancing intercultural communication training may improve parental experience and long-term engagement in care.</div></div>","PeriodicalId":101004,"journal":{"name":"Pediatric Hematology Oncology Journal","volume":"11 1","pages":"Article 100807"},"PeriodicalIF":0.0,"publicationDate":"2026-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146188561","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Induction chemotherapy followed by arsenic and all-trans retinoic acid for acute promyelocytic leukemia: Outcomes from a single-center protocol in Singapore","authors":"Debbra Jayne Yi Xin Chong ,&nbsp;Xue Na Goh ,&nbsp;Prasad Iyer","doi":"10.1016/j.phoj.2026.100808","DOIUrl":"10.1016/j.phoj.2026.100808","url":null,"abstract":"<div><div>KKH-APML 17 is a single-center protocol for the treatment of pediatric acute promyelocytic leukemia (APML) in Singapore, which commences with standard induction chemotherapy followed by arsenic trioxide (ATO) and all-trans retinoic acid (ATRA) for consolidation. It was designed to reduce anthracycline exposure while maintaining therapeutic efficacy. All three patients treated with this protocol achieved and maintained morphological and molecular remission. Pseudotumor cerebri was an adverse effect of ATRA treatment but no other major adverse effects were observed. In resource-limited settings where ATO is not readily available, this protocol may serve as an interim measure while awaiting the import of ATO.</div></div>","PeriodicalId":101004,"journal":{"name":"Pediatric Hematology Oncology Journal","volume":"11 1","pages":"Article 100808"},"PeriodicalIF":0.0,"publicationDate":"2026-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146189321","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Cryptococcosis in B-cell acute lymphoblastic leukemia with suspected dissemination: A case report","authors":"S. Suganya ,&nbsp;M. Mohammed Shakeel ,&nbsp;Jaswanthini ,&nbsp;Suganth ,&nbsp;Karthik Kumar ,&nbsp;S. Kalpana ,&nbsp;Hemachitra","doi":"10.1016/j.phoj.2026.100803","DOIUrl":"10.1016/j.phoj.2026.100803","url":null,"abstract":"<div><h3>Background</h3><div>Acute lymphoblastic leukemia (ALL) is the most common childhood cancer. With improved risk stratification and MRD-guided therapy, the overall prognosis of ALL is more than 90 %. Nevertheless, Induction mortality remains a significant clinical concern in low- and middle-income countries (LMIC), with invasive fungal infection (IFI) of increasing concern.</div></div><div><h3>Case report</h3><div>We present a case of a 9-year-old boy with B-cell acute lymphoblastic leukemia (B-ALL) who developed persistent fever due to IFI during induction chemotherapy. Despite hematologic remission, he developed IFI of the kidneys with subacute thyroiditis suspicious for disseminated infection, necessitating modification of chemotherapy and prolonged antifungal therapy.</div></div><div><h3>Conclusion</h3><div>This case highlights the diagnostic challenges in IFI management in pediatric leukemia in LMIC.</div></div>","PeriodicalId":101004,"journal":{"name":"Pediatric Hematology Oncology Journal","volume":"11 1","pages":"Article 100803"},"PeriodicalIF":0.0,"publicationDate":"2026-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145928648","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Editorial Board Information","authors":"","doi":"10.1016/S2468-1245(26)00019-7","DOIUrl":"10.1016/S2468-1245(26)00019-7","url":null,"abstract":"","PeriodicalId":101004,"journal":{"name":"Pediatric Hematology Oncology Journal","volume":"11 1","pages":"Article 100821"},"PeriodicalIF":0.0,"publicationDate":"2026-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147538898","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Hemoglobin SC disease and its clinical manifestations in pediatric patients: where is the data?","authors":"Carlos Tourinho Lapa Filho ,&nbsp;Mônica Pinheiro de Almeida Verissimo ,&nbsp;Thais Novaes Ferreira","doi":"10.1016/j.phoj.2025.100801","DOIUrl":"10.1016/j.phoj.2025.100801","url":null,"abstract":"<div><div>Hemoglobin SC Disease is among the main hemoglobinopathies related to sickle cell anemia (only surpassed in prevalence by Hemoglobin SS Disease). Although considered a pathology of lesser severity and complications, information on its clinical manifestations seem to lack support in consistent studies. In the pediatric population, data is even more scarce. The present study carried out an integrative review of the clinical manifestations reported in pediatric patients with hemoglobin SC disease, with their age of occurrence, the most prevalent manifestations, evolution of pathology and associated morbidity. The articles analyzed were able to provide guidance on the issues examined, but the data lacked consistency. New and greater number of prospective studies are necessary to increase the level of evidence for the clinical findings presented in this entity.</div></div>","PeriodicalId":101004,"journal":{"name":"Pediatric Hematology Oncology Journal","volume":"11 1","pages":"Article 100801"},"PeriodicalIF":0.0,"publicationDate":"2026-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146079063","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Central venous access in Paediatric Oncology at a tertiary care cancer care centre in North India: device selection, CLABSI burden and multidrug-resistant gram-negative antibiogram","authors":"Varsha Mishra Sopori,&nbsp;Bhagwan Bardwal,&nbsp;Vijeta Sharma,&nbsp;Abhishek Sharma,&nbsp;Sanjukta Mukhopadhyay,&nbsp;Jasmeet Sidhu","doi":"10.1016/j.phoj.2026.100959","DOIUrl":"10.1016/j.phoj.2026.100959","url":null,"abstract":"","PeriodicalId":101004,"journal":{"name":"Pediatric Hematology Oncology Journal","volume":"11 3","pages":"Article 100959"},"PeriodicalIF":0.0,"publicationDate":"2026-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149094096","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}