Southern European Prospective Investigation Into Childhood Cancer and Nutrition (EPICkids): Study design and protocol.

The survival rates for children with cancer have increased appreciably over the last few decades; however, childhood cancer survivors continue to suffer from long-lasting sequelae. Studies have demonstrated that the presence of malnutrition, over- and under-nutrition, at diagnosis or the duration of malnutrition during treatment is associated with increased toxicity, infection, and inferior survival. Dietary habits, along with behavioral and socioeconomic status, are known factors that lead to obesity or undernutrition and can affect the prognosis and quality of life of children with cancer.

Implementation of the precision oncology program in catalonia's public health system: results, lessons learned, and future prospects.

Purpose: The Precision Oncology Program (POP) in Catalonia aims to provide equitable access to molecular testing for individuals with cancer, integrating Next-Generation Sequencing (NGS) into clinical practice to inform diagnosis, prognosis, and treatment decisions for both adult and pediatric patients with solid and hematologic malignancies, including somatic and germline alterations. This study evaluates the program's outcomes and impact.

Methods: This evaluation covers the period from the program's implementation in July 2021 through December 2023, with a more detailed analysis focusing on 2022-2023.

International neuroblastoma risk group consortium: a model of a networking for rare cancers.

It is critical to share knowledge and harmonize approaches to optimize progress in rare cancers. The International Neuroblastoma Risk Group (INRG) Task Force was formed by the four major neuroblastoma cooperative groups in 2004 to achieve this goal. Strategies developed for neuroblastoma are an exemplar for other rare malignancies.

Prognostic factors of early mortality in children and adolescents with relapsed/refractory solid tumors participating in dose-finding trials in the targeted and immune therapies era: An ITCC study.

Introduction: Phase I/II trials are essential to introduce novel agents for children with cancer. Defining risk factors of early mortality could maximize the efficiency of such trials.

Methods: Patients < 18 years with relapsed/refractory solid tumors in their first phase I/II trial were eligible in retrospect.

An Evaluation of Quality of Life During Participation in Early-Phase Clinical Trials in Paediatric Oncology.

Background: Given the poor prognoses and potential treatment toxicities faced by paediatric patients in early-phase oncology trials, evaluating quality of life (QoL) is crucial for both families and physicians when considering patient recruitment. This prospective, longitudinal study evaluates the impact of trial participation on QoL in children, agreement between self-reports, proxy-reports and physician assessments, and associations with demographic and clinical factors.

Procedure: Patients aged 5-18 years enrolled in Phase I/II clinical trials at Hospital Niño Jesus from 2019 to 2023, and their caregiver proxies were eligible.

Accelerating Drug Development for Neuroblastoma: Consensus Statement From the Third Neuroblastoma Drug Development Strategy Forum.

High-risk neuroblastoma is a poor prognosis cancer of the sympathetic nervous system that accounts for a disproportionate number of childhood cancer deaths. Many viable biological targets have been identified, and the number of potential combinations is even larger. Several products have attained marketing authorization for treatment of patients with neuroblastoma.

A comprehensive clinico-pathological review of a series of pediatric, adolescents and young adults with high-grade osteosarcoma: from clinics to biomarker discovery.

Background: We analyzed clinical and immunohistochemical characteristics of pediatric, adolescents and young adults with high-grade osteosarcoma (HGOS) to validate prognostic factors, identify targetable and prognostic biomarkers and define management of multiple relapses.

Methods: Retrospective analysis of 67 patients with HGOS between 2001 and 2020 was studied. BTN3A2, HSP90 and GLYPICAN1 were further analyzed based on their high expression on in silico model.

A 2035 Clinical Research Vision and Roadmap for High-Risk Neuroblastoma.

Despite the introduction of anti-GD2 antibody therapy, outcomes for children with high-risk neuroblastoma remain poor, with low cure rates and a high proportion of survivors facing long-term sequelae. In this report, leaders from international cooperative groups and patient advocacy organizations review lessons learnt, identify current challenges, and provide a vision to bring new agents into frontline therapy to increase cure rates and reduce long-term toxicities over the next decade. The implementation of this vision requires improved global collaboration, incorporation of novel biomarkers, and a strengthened interaction with the regulatory landscape.

Management and outcome of children with high-risk neuroblastoma: insights from the Spanish Society of Pediatric Hematology and Oncology (SEHOP) neuroblastoma group on refractory and relapse/progressive disease.

Purpose: Outcome for children with refractory and relapse/progressive high-risk neuroblastoma (HR-NB) remains poor, without an internationally agreed standard second-line approach. Heterogeneity in patients' disease and treatment strategies challenges clinical management. The survival rate for patients with resistant disease does not exceed 20% at 5 years.

A phase 1 dose-escalation study of LY3295668 erbumine as monotherapy and in combination with topotecan and cyclophosphamide in children with relapsed/refractory neuroblastoma.

Background: This study evaluated the safety, pharmacokinetics, and antitumor activity of LY3295668 erbumine as monotherapy and combination therapy in children with relapsed/refractory neuroblastoma.

Methods: Patients aged 2-21 years who had relapsed/refractory neuroblastoma were enrolled. LY3295668 erbumine was evaluated at two dose levels (12 and 15 mg/m) and administered orally twice daily continuously as monotherapy and in combination with intravenous topotecan and cyclophosphamide in 28-day cycles.

Stakeholder Perspectives on Randomized Clinical Trials for Children With Poor-Prognosis Cancers.

Importance: In poor-prognosis children's cancers, new therapies may carry fresh hope for patients and parents. However, there is an absolute requirement for any new therapy to be properly evaluated to fulfill scientific, regulatory, and reimbursement requirements. Randomized clinical trials (RCTs) are considered the gold standard, but no consensus exists on how and when they should be deployed to best meet the needs of all stakeholders.

Risk of developing neuroblastoma influenced by maternal stressful life events during pregnancy and congenital pathologies.

Objective: A retrospective multicenter study to investigate the potential association between descriptive information related to pregnancy history and perinatal features and the risk of neuroblastoma (NB) in children.

Study Design: Data from 56 mothers during 105 pregnancies (56 cases of NB, 49 control siblings) were collected through face-to-face or telephone interviews with mothers of children diagnosed with NB, along with information extracted from Health System databases. Descriptive information related to (a) pregnancy history as maternal stressful life events with perceived distress during pregnancy, weight gain, alcohol and tobacco consumption, mode of delivery and gestational age; and (b) perinatal features as congenital pathologies, weight at birth and type of feeding were examined to identify potential risk factors for NB.

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.

Advances in the approaches used to repurpose drugs for neuroblastoma.

Introduction: Neuroblastoma (NB) remains a challenging pediatric malignancy with limited treatment options, particularly for high-risk cases. Drug repurposing offers a convenient and cost-effective strategy for treating rare diseases like NB. Using existing drugs with known safety profiles accelerates the availability of new treatments, reduces development costs, and mitigates risks, offering hope for improved patient outcomes in challenging conditions.

Incorporation of patient-reported outcomes in early-phase clinical trials for childhood and adolescent cancer.

Introduction: The assessment of quality of life (QoL) should be one of the main objectives in paediatric clinical trials. Even though researchers, regulators and advocates support the use of patient-reported outcomes (PROs), this has not been fully implemented. The aim of this study is to assess the measurement of QoL and the usage of PROs, palatability assessments and medication diaries in early-phase clinical trials for childhood and adolescent cancer.

ECLIM-SEHOP: how to develop a platform to conduct academic trials for childhood cancer.

Introduction: ECLIM-SEHOP platform was created in 2017. Its main objective is to establish the infrastructure to allow Spanish participation into international academic collaborative clinical trials, observational studies, and registries in pediatric oncology. The aim of this manuscript is to describe the activity conducted by ECLIM-SEHOP since its creation.

The first microcaddisflies Metrichia Ross, 1938 recorded in Tocantins State, northern Brazil (Trichoptera: Hydroptilidae): Metrichia elongata sp. nov. and Metrichia vulgaris Santos, Takiya & Nessimian 2016.

The microcaddisfly Neotropical genus Metrichia Ross, is recorded for the first time from the state of Tocantins, northern Brazil, through the discovery of two species: Metrichia elongata sp. nov. and Metrichia vulgaris Santos, Takiya & Nessimian 2016.

BPTF cooperates with MYCN and MYC to link neuroblastoma cell cycle control to epigenetic cellular states.

The nucleosome remodeling factor BPTF is required for the deployment of the MYC-driven transcriptional program. Deletion of one allele delays tumor progression in mouse models of pancreatic cancer and lymphoma. In neuroblastoma, MYCN cooperates with the transcriptional core regulatory circuitry (CRC).

Bevacizumab, Irinotecan, or Topotecan Added to Temozolomide for Children With Relapsed and Refractory Neuroblastoma: Results of the ITCC-SIOPEN BEACON-Neuroblastoma Trial.

Purpose: Outcomes for children with relapsed and refractory high-risk neuroblastoma (RR-HRNB) remain dismal. The BEACON Neuroblastoma trial (EudraCT 2012-000072-42) evaluated three backbone chemotherapy regimens and the addition of the antiangiogenic agent bevacizumab (B).

Materials And Methods: Patients age 1-21 years with RR-HRNB with adequate organ function and performance status were randomly assigned in a 3 × 2 factorial design to temozolomide (T), irinotecan-temozolomide (IT), or topotecan-temozolomide (TTo) with or without B.