Early radiologic tumour volume response in non-metastatic rhabdomyosarcoma is not predictive for survival.

Background: There is an urgent need for early radiological markers predicting survival in paediatric rhabdomyosarcoma patients.

Objective: To analyse the predictive value of early radiological tumour volume response to chemotherapy in non-metastatic rhabdomyosarcoma patients.

Methods: The European paediatric Soft tissue sarcoma Study Group (EpSSG) RMS 2005 study prospectively included non-metastatic rhabdomyosarcoma patients.

The Clinical Utility of Next-Generation Sequencing in Childhood and Adolescent/Young Adult Solid Tumors: A Systematic Review and Meta-Analysis.

Background: Next-generation sequencing (NGS) has emerged as a transformative tool in precision medicine, offering insights into actionable genomic alterations and informing clinical decision-making in childhood and adolescent/young adult (AYA) solid tumors.

Methods: We conducted a systematic review and meta-analysis to assess the utility of NGS in identifying actionable genomic alterations and its impact on clinical decision-making. Studies involving patients aged 0-40 years with solid tumors were included.

Maintenance Chemotherapy in Patients With High-Risk Rhabdomyosarcoma: Long-Term Survival Analysis of the European Soft Tissue Sarcoma Study Group RMS 2005 Trial.

The European Paediatric Soft Tissue Sarcoma Study Group (EpSSG) RMS 2005 trial evaluated maintenance chemotherapy in high-risk rhabdomyosarcoma (RMS). Patients were randomly assigned to either discontinue treatment (standard arm) or receive six 28-day cycles of vinorelbine (25 mg/m) once per day on days 1, 8, and 15, plus once daily low-dose cyclophosphamide (25 mg/m; experimental arm). Initial results showed improved overall survival (OS), but disease-free survival (DFS) improvement was not statistically significant.

Molecular Characteristics of a Fusion Gliosarcoma: A Case Report.

The combination of ependymoma and gliosarcoma elements in the same tumor is extremely rare, and the molecular characteristics of these entities are not clear. Here, we present a rare aggressive brain tumor in a 12-year-old boy harboring a gene fusion, a characteristic feature of supratentorial ependymomas. On the other hand, the histopathological, molecular, and methylation profiles were compatible with a diagnosis of a mesenchymal type, IDH wild-type glioblastoma multiforme (GBM).

Prognostic role of bone erosion in orbital RMS: a report from the European Pediatric Soft Tissue Sarcoma Study Group (EpSSG).

Background: Orbital rhabdomyosarcoma (RMS) is often limited to the orbital cavity and has a favorable prognosis. In some cases, the tumor can erode the orbital bone and behave as a parameningeal RMS (PM-RMS); thus, it is treated more intensively. However, the current protocols do not provide any guidance on how to consider different grades of bone erosion (BE) that can vary widely, hampering a uniform classification and the subsequent treatment assignment.

Parameningeal Rhabdomyosarcoma: Results of the European Pediatric Soft Tissue Sarcoma Study Group RMS 2005 Study.

Background: Parameningeal (PM) site is an unfavorable characteristic in rhabdomyosarcoma (RMS). We described the treatment and outcome for patients with PM RMS and investigated the prognostic value of risk factors. We scored PM site by originating site and by highest risk extension.

An Integrative Pediatric Oncology Program Addressing Parents' Quality of Life-Related Concerns.

Background: Parents of children with cancer face bio-psycho-social-spiritual concerns which can significantly reduce quality of life (QoL). We examined the impact of an integrative oncology (IO) intervention on QoL-related concerns among parents of children in a pediatric hematology-oncology department.

Measures: The study was prospective, controlled, nonrandomized and patient-preferenced.

Localized incompletely resected standard risk rhabdomyosarcoma in children and adolescents: Results from the European Paediatric Soft Tissue Sarcoma Study Group RMS 2005 trial.

Background: The authors report the prospective evaluation of reduced dose alkylator chemotherapy combined with radiotherapy for European Pediatric Soft Tissue Sarcoma Study Group (EpSSG) standard risk nonalveolar rhabdomyosarcoma (NA-RMS).

Patients And Methods: Localized node negative Intergroup Rhabdomyosarcoma Study (IRS) II/III NA-RMS at favorable sites (subgroup C), <25 years old, received five cycles of ifosfamide, vincristine, and dactinomycin (IVA) chemotherapy (30 g/m ifosfamide) and four cycles of vincristine and dactinomycin (if receiving radiotherapy), or nine cycles of IVA (54 g/m ifosfamide) ± radiotherapy. Delayed primary tumor excision was considered for IRS III tumors.

Clinical outcome of pediatric medulloblastoma patients with Li-Fraumeni syndrome.

Background: The prognosis for Li-Fraumeni syndrome (LFS) patients with medulloblastoma (MB) is poor. Comprehensive clinical data for this patient group is lacking, challenging the development of novel therapeutic strategies. Here, we present clinical and molecular data on a retrospective cohort of pediatric LFS MB patients.

Nonmetastatic Rhabdomyosarcoma in Children and Adolescents: Overall Results of the European Pediatric Soft Tissue Sarcoma Study Group RMS2005 Study.

JCO The RMS2005 study included two phase III randomized trials for high-risk (HR) and observational trials for low (LR), standard (SR), and very high-risk (VHR) patients who have been partially reported. Herein, we present a comprehensive report of results achieved for the complete unselected nonmetastatic cohort and analyze the evolution of treatment in comparison with previous European protocols. After a median follow-up of 73.

[SURVIVAL OF PEDIATRIC PATIENTS WITH NON-MALIGNANT DISEASES REQUIRING HOSPITALIZATION IN THE INTENSIVE CARE UNIT FOLLOWING HEMATOPOIETIC STEM CELL TRANSPLANTATION AND RISK FACTORS FOR MORTALITY].

Introduction: Although Hematopoietic Stem Cell Transplantation (HSCT) is the only curative option for children with certain non-malignant disorders, a proportion of these children are admitted to the Pediatric Intensive Care Unit (PICU) due to treatment related life-threatening complications.

Aims: To analyze risk factors for ICU hospitalizations, morbidity and mortality in children with genetic diseases who have undergone HSCT and were admitted to intensive care units.

Methods: This retrospective study is based on the collection and analysis of clinical and laboratory data from the medical records of patients from the departments of Bone Marrow Transplantations and Intensive Care, from 2 hospitals, Hadassah and Rambam Medical Centers.

Epithelioid hemangioendothelioma in children: The European Pediatric Soft Tissue Sarcoma Study Group experience.

The European pediatric Soft tissue sarcoma Study Group analyzed all children with epithelioid hemangioendothelioma prospectively registered in the NRSTS-05 (EUDRACT 2005-001139-31) and in MTS-2008 (NCT00379457) studies: 10 patients with localized and one with metastatic disease. Median age was 14.3 years (range, 9.

Therapy and prognostic significance of regional lymph node involvement in embryonal rhabdomyosarcoma: a report from the European paediatric Soft tissue sarcoma Study Group.

Purpose: Regional lymph node disease (N1) is a component of the risk-based treatment stratification in rhabdomyosarcoma (RMS). The purpose of this study was to determine the contribution of nodal disease to the prognosis of patients with non-metastatic embryonal RMS (ERMS) and analyse their outcome by treatment received.

Patients And Methods: Between 2005 and 2016, 1294 children with ERMS were enrolled in the European paediatric Soft tissue sarcoma Study Group (EpSSG) RMS 2005 protocol, 143 patients with N1.

Metastatic Rhabdomyosarcoma: Results of the European Soft Tissue Sarcoma Study Group MTS 2008 Study and Pooled Analysis With the Concurrent BERNIE Study.

Purpose: Outcome for patients with metastatic rhabdomyosarcoma (RMS) is poor. This study presents the results of the MTS 2008 study with a pooled analysis including patients from the concurrent BERNIE study.

Patients And Methods: In MTS 2008, patients with metastatic RMS received four cycles of ifosfamide, vincristine, and actinomycin D (IVA) plus doxorubicin, five cycles of IVA, and 12 cycles of maintenance chemotherapy (low-dose cyclophosphamide and vinorelbine).

Adolescents and young adults with rhabdomyosarcoma treated in the European paediatric Soft tissue sarcoma Study Group (EpSSG) protocols: a cohort study.

Background: Adolescent and young adult patients with rhabdomyosarcoma often have poorer outcomes than do children. We aimed to compare the findings of adolescent and young adult patients with children enrolled in two prospective clinical protocols.

Methods: This retrospective observational analysis was based on data from the European paediatric Soft tissue sarcoma Study Group (EpSSG) rhabdomyosarcoma 2005 trial (phase 3 randomised trial for localised rhabdomyosarcoma, open from April, 2006, to December, 2016) and the EpSSG MTS 2008 protocol (prospective, observational, single-arm study for metastatic rhabdomyosarcoma, open from June, 2010, to December, 2016), which involved 108 centres from 14 different countries in total.

Brachytherapy for Pediatric Patients at Gustave Roussy Cancer Campus: A Model of International Cooperation for Highly Specialized Treatments.

Purpose: Childhood cancer is rare, and treatment is frequently associated with long-term morbidity. Disparities in survival and long-term side effects encourage the establishment of networks to increase access to complex organ-conservative strategies, such as brachytherapy. We report our experience of an international cooperation model in childhood cancers.

Survival of pediatric patients requiring admission in the intensive care unit post hematopoietic stem cell transplantation: Prognostic factors associated with mortality.

Background: Although hematopoietic stem cell transplantation (HSCT) is the only curative option for some children with malignant and nonmalignant disorders, the procedure itself carries a high risk of complications. A proportion of children undergoing HSCT develop severe transplant-related complications requiring hospitalization in the pediatric intensive care unit (PICU).

Methods: A retrospective cohort study included 793 children with malignant and nonmalignant diseases that underwent 963 HSCTs in two large pediatric hospitals over 15 years.

Congenital rhabdomyosarcoma: A report from the European paediatric Soft tissue sarcoma Study Group.

Procedure: Congenital rhabdomyosarcoma (RMS) represents a challenging disease due to its characteristics and the difficulties in delivering treatment in this immature population.

Methods: We analyzed treatment and outcome of patients with congenital RMS, defined as tumor diagnosed in the first 2 months of life, enrolled in the European paediatric Soft tissue sarcoma Study Group protocols.

Results: Twenty-four patients with congenital RMS were registered.

Ultrasound-Guided Core Biopsy for Tissue Diagnosis in Pediatric Oncology: 16-Year Experience With 597 Biopsies.

Percutaneous imaging-guided core needle biopsies (CNBs) for cancer diagnosis in pediatric patients are gaining interest because of their availability, lower rate of complications, and high diagnostic power compared with traditional surgical biopsies. Nevertheless, their precise role in the diagnostic algorithm of pediatric oncology is still unknown. The purpose of this study was to report our accumulated 16-year experience with CNB; discuss the availability, safety, and diagnostic accuracy of the procedure and the adequacy of ancillary testing; and compare our findings with the available literature.

Mesothelioma in children and adolescents: the European Cooperative Study Group for Pediatric Rare Tumors (EXPeRT) contribution.

Introduction: Very little is known about the characteristics of mesothelial tumours in the paediatric population. In adults with malignant mesothelioma, the pemetrexed-based regimen with cytoreductive surgery (CRS) is a standard of care in limited tumours, but long-term survival is uncommon.

Material And Methods: The European Cooperative Study Group on Pediatric Rare Tumors (EXPeRT) retrospectively reviewed children, adolescents and young adults (≤21 year) diagnosed with mesothelial tumours treated between 1987 and 2018.

Nasopharyngeal carcinoma in children and young adults-Beyond 5-year survival.

Nasopharyngeal carcinoma (NPC) is a rare and locally aggressive form of childhood cancer. Treatment of NPC includes chemotherapy and radiotherapy. With current treatment protocols, survival rates for patients with nonmetastatic disease is over 80%.