Role of procalcitonin, C reactive protein and ferritin in cytokine release syndrome after CAR T-cell therapy in children and young adults.

PurposeChimeric antigen receptor (CAR) T-cell CD19 therapy has changed the treatment paradigm for patients with relapsed/refractory B-cell acute lymphoblastic leukemia. It is frequently associated with potentially severe toxicities: cytokine release syndrome (CRS) and immune effector cell-associated neurotoxicity syndrome (ICANS), and admission to PICU is often required. Some biomarkers seem to correlate with CRS severity.

Achieving Language Justice in Pediatric Hematology-Oncology: A Multinational Perspective for Language-Concordant Equitable Patient- and Family-Centered Care and Research Inclusion.

Language-discordant healthcare encounters-when the patient/caregiver and clinician are not able to communicate directly in the patient's/caregiver's preferred language-are associated with worse quality of care, increased adverse events, and research exclusion. Here, we describe the current state of language justice in clinical practice and research in the United States, Canada, and Spain, discuss the role of social determinants of health and language, in patient safety and health outcomes and review an example of culturally and linguistically concordant interventions to increase research participation. We close with practical and global strategies to increase multilingual research participation and to provide equitable patient- and family-centered care in pediatric hematology-oncology.

Risk Factors for Extraocular Relapse in Retinoblastoma.

Background: Metastatic retinoblastoma remains a significant challenge in pediatric oncology, with stark disparities in survival outcomes between high-income countries (HICs) and low-income countries (LICs). Delayed diagnosis and treatment, driven by socioeconomic factors and limitations in healthcare systems, contribute to poorer outcomes in LICs. Histopathological characteristics, including high-risk pathology factors (HRPFs) and the extent of ocular tumor invasion, are critical for predicting metastatic risk and guiding treatment strategies.

Multi-omics profiling reveals key factors involved in Ewing sarcoma metastasis.

Ewing sarcoma (EWS) is the second most common bone tumor affecting children and young adults, with dismal outcomes for patients with metastasis at diagnosis. Mechanisms leading to metastasis remain poorly understood. To deepen our knowledge on EWS progression, we have profiled tumors and metastases from a spontaneous metastasis mouse model using a multi-omics approach.

Mimicking Retinoblastoma Treatment With Repeated Topotecan or Melphalan Develops Cross-Resistance to Classic Agents But Not to Repurposed Drugs.

Purpose: Refractory or recurrent retinoblastoma results from acquired chemoresistance and the management of these eyes often requires surgical removal. Our objective was to develop retinoblastoma models resistant to chemotherapy by exposing cancer cells to repeated chemotherapy mimicking the clinical scenario. These newly resistant cells were used to evaluate potential novel therapies.

Diffuse pediatric high-grade glioma of methylation-based RTK2A and RTK2B subclasses present distinct radiological and histomolecular features including Gliomatosis cerebri phenotype.

Diffuse pediatric-type high-grade gliomas (pedHGG), H3- and IDH-wildtype, encompass three main DNA-methylation-based subtypes: pedHGG-MYCN, pedHGG-RTK1A/B/C, and pedHGG-RTK2A/B. Since their first description in 2017 tumors of pedHGG-RTK2A/B have not been comprehensively characterized and clinical correlates remain elusive. In a recent series of pedHGG with a Gliomatosis cerebri (GC) growth pattern, an increased incidence of pedHGG-RTK2A/B (n = 18) was observed.

Secondary haematological dysplasia after CAR-T-cell therapy for acute lymphoblastic leukaemia in children.

The use of CAR-T is becoming more widespread in the treatment of haematological malignancies. In adults, secondary myelodysplastic syndromes (MDS) after CAR-T have been described. However, there are currently no data on the risk of MDS following CAR-T in children treated for acute lymphoblastic leukaemia (ALL).

DNA methylation-array interlaboratory comparison trial demonstrates highly reproducible paediatric CNS tumour classification across 13 international centres.

Aims: DNA methylation profiling, recently endorsed by the World Health Organisation (WHO) as a pivotal diagnostic tool for brain tumours, most commonly relies on bead arrays. Despite its widespread use, limited data exist on the technical reproducibility and potential cross-institutional differences. The LOGGIC Core BioClinical Data Bank registry conducted a prospective laboratory comparison trial with 12 international laboratories to enhance diagnostic accuracy for paediatric low-grade gliomas, focusing on technical aspects of DNA methylation data generation and profile interpretation under clinical real-time conditions.

Use of Radioguided Surgery for Small and Difficult-to-Locate Relapsed MIBG (+) High-Risk Neuroblastoma Lesions.

Introduction: High-risk neuroblastoma, particularly in the relapse/refractory (R/R) setting, poses unique challenges to obtaining the representative-quality tissue that is mostly required for molecular analysis. This study explores the use of 123I-MIBG radioguided surgery to access complex locations of MIBG-positive neuroblastoma as a tool to overcome the difficulties associated with repeated surgeries in these patients.

Methods: This study is a retrospective review of all patients with R/R neuroblastoma and MIBG-uptaking lesions who underwent radioguided surgery between February 2020 and 2023 at SJD Barcelona Children's Hospital.

Efficacy and Safety of Acupuncture and Related Techniques in the Management of Oncological Children and Adolescent Patients: A Systematic Review.

/: Oncology acupuncture is emerging as a complementary treatment in pediatric cancer care centers. It is valued for its potential to manage symptoms associated with cancer and treatment toxicities without increasing polypharmacy. The aim of this review is to evaluate the efficacy and safety of acupuncture and related techniques in alleviating symptoms of cancer treatment in pediatric oncology patients.

Safety and outcome of children, adolescents and young adults participating in phase I/II clinical oncology trials: a 9-year center experience.

Introduction: Enrolling children with cancer in early phase trials is crucial to access innovative treatments, contributing to advancing pediatric oncology research and providing tailored therapeutic options. Our objective is to analyze the impact of these trials on patient outcomes and safety, and to examine the evolution and feasibility of trials in pediatric cancer over the past decade.

Methods: All patients recruited in pediatric anticancer phase I/II clinical trials from January 2014 to December 2022 were included.

The TeLeo Program: Tele-education in pediatric oncology as a tool to support training programs in Latin America.

The TeLeo Program offers a free-access 2-year online learning program to support fellowship programs in pediatric oncology, enhance networking opportunities, and facilitate the exchange of context-specific, educational content within the pediatric oncology community in training in Latin America. In its first edition beginning in 2021, 185 fellows from 40 centers in 12 Latin American countries were enrolled. Additional courses for other healthcare professionals related to oncology in the region were produced to further support the program.

Effectiveness of a Wilms tumour treatment guideline adapted to local circumstances in sub-Saharan Africa: A report from Wilms Africa Phase II-CANCaRe Africa.

Background: Wilms tumour (WT) is one of the cancer types targeted by the Global Initiative for Childhood Cancer (GICC). The objective of this study was to describe the outcomes of Wilms Africa Phase II in sub-Saharan Africa.

Methods: Wilms Africa Phase II used a comprehensive WT treatment protocol in a multi-centre, prospective study conducted in eight hospitals in Ethiopia (2), Ghana (2), Malawi, Cameroon, Zimbabwe and Uganda.

International Society of Paediatric Oncology (SIOP) Global Mapping Program: Analysis of healthcare centers in countries of the Latin American Society of Pediatric Oncology (SLAOP).

Background: The International Society of Paediatric Oncology Society Global Mapping Program aims to describe the local pediatric oncology capacities. Here, we report the data from Latin America.

Methods: A 10-question survey was distributed among chairs of pediatric oncology services.

Human EWS-FLI protein levels and neomorphic functions show a complex, function-specific dose-response relationship in .

Ewing sarcoma (EwS) is a cancer that arises in the bones and soft tissues, typically driven by the Ewing's sarcoma breakpoint region 1-Friend leukemia virus integration 1 (EWS-FLI) oncogene. Implementation of genetically modified animal models of EwS has proved difficult largely owing to EWS-FLI's high toxicity. The EWS-FLI frameshift variant that circumvents toxicity but is still able to perform key oncogenic functions provided the first study model in .

Beyond Needles: Pioneering Pediatric Care with Virtual Reality (VR) for TIVAD Access in Oncology.

Introduction: Pediatric oncology patients use totally implantable venous access devices (TIVADs) to enable central venous access. Anxiety, pain and/or discomfort are common despite anesthesia. Virtual reality (VR) is a non-pharmacological approach that may reduce pain and anxiety in these patients.

Targeting GPC2 on Intraocular and CNS Metastatic Retinoblastomas with Local and Systemic Delivery of CAR T Cells.

Purpose: Retinoblastoma is the most common intraocular malignancy in children. Although new chemotherapeutic approaches have improved ocular salvage rates, novel therapies are required for patients with refractory intraocular and metastatic disease. Chimeric antigen receptor (CAR) T cells targeting glypican-2 (GPC2) are a potential new therapeutic strategy.

Hamartoma of mature cardiac myocytes presenting as a polypoid epicardial tumor in the interatrial groove and with gene fusions by copy number anomalies of chromosome 7.

Hamartoma of mature cardiac myocytes (HMCM) is an extremely rare cardiac tumor characterized by benign growth of differentiated mature striated cardiac myocytes, and usually involves the ventricular myocardium. We describe the case of a 15-year-old female who presented with a short history of atrial fibrillation and a polypoid epicardial tumor that was attached to the interatrial groove by a short pedicle. The resected specimen showed features consistent with HMCM.

Desensitizing the autonomic nervous system to mitigate anti-GD2 monoclonal antibody side effects.

Background: Anti-GD2 monoclonal antibodies (mAbs) have shown to improve the overall survival of patients with high-risk neuroblastoma (HR-NB). Serious adverse events (AEs), including pain, within hours of antibody infusion, have limited the development of these therapies. In this study, we provide evidence of Autonomic Nervous System (ANS) activation as the mechanism to explain the main side effects of anti-GD2 mAbs.

Current Status of Fertility Preservation in Pediatric Oncology Patients.

Cancer poses significant emotional challenges for children and adolescents, despite improvements in survival rates due to new therapies. However, there is growing concern about the long-term effects, including fertility issues. This review examines recent advancements and future directions in fertility preservation within a pediatric population subjected to oncological therapies.