Assessing an eHealth Intervention on Quality of Life in Patients With Chronic Lymphocytic Leukemia and Myelodysplastic Syndromes: The MyPal Randomized Controlled Trial.

Purpose: The MyPal study (ClinicalTrials.gov identifier: NCT04370457) is a randomized controlled clinical trial assessing an eHealth intervention on the quality of life (QoL) of patients with chronic lymphocytic leukemia (CLL) and myelodysplastic syndromes (MDS).

Methods: Patients who were receiving or had previously received treatment for CLL or MDS were randomly assigned (1:1) to access the MyPal digital health platform versus standard of care.

Anthracycline-Induced Cardiomyopathy After Nephro-/Neuroblastoma in Childhood: The Importance of Cardiological Reference Assessment.

Background: Long-term childhood cancer survivors (CCS) may develop anthracycline-induced cardiomyopathy. Our cross-sectional study focused on the question of whether a central echocardiographic reference assessment is associated with a higher detection rate of cardiac dysfunction in a population-based cohort of affected children with neuroblastoma or nephroblastoma. We also examined the prevalence of anthracycline-induced cardiomyopathy and its risk factors.

Outcome of patients with a first relapse after intermediate- or high-risk Wilms tumor, treated according to SIOP WT 2001/UK-IMPORT study; A report from the SIOP renal tumor study group.

Background: Patients with a Wilms tumor (WT) who relapse following initial therapy with more than only vincristine and actinomycin-D are considered high-risk (group BB) or very high-risk (group CC) relapse by the International Society of Pediatric Oncology - Renal Tumor Study Group (SIOP-RTSG). We aimed to retrospectively analyze the characteristics and outcome of BB and CC patients.

Methods: We included all patients with first relapsed WT that would currently be considered BB (n = 148) and CC (n = 72) relapse and registered in the SIOP 2001/UK-IMPORT study.

Late toxicity after upper abdominal radiotherapy in pediatric Wilms tumor and neuroblastoma survivors. A systematic review on behalf of SIOPEN and SIOP-RTSG.

Background: Radiotherapy plays a crucial role in the multimodal treatment of Wilms tumor and neuroblastoma subtypes with an increased risk of locoregional failure. Unfortunately, radiotherapy can be associated with late toxicities in survivors. This systematic review provides an overview of the quality of evidence related to late effects following upper abdominal radiotherapy in survivors of Wilms tumor and neuroblastoma.

Remaining Challenges in the Treatment of Relapsed Wilms Tumor: Children's Oncology Group and International Society of Paediatric Oncology Perspectives.

Wilms tumor (WT) is the most common pediatric renal tumor, and with multidisciplinary treatment overall outcomes are excellent. However, a small subset of patients with WT will relapse. The ideal treatment of relapsed WT is yet to be defined.

Distinct pathways for genetic and epigenetic predisposition in familial and bilateral Wilms tumor.

Background: Genetic predisposition is particularly common in children with the kidney cancer, Wilms tumor. In 10% of these children, this manifests as a family history of Wilms tumor or bilateral disease. The frequency and spectrum of underlying changes have not been systematically investigated.

Exploring the adaptation of the Nephroblastoma Oncosimulator to MRI scans, treatment data, and histological profiles of patients from different risk groups.

Introduction: Nephroblastoma or Wilms' tumor is the most prevalent type of renal tumor in pediatric oncology. Although the overall survival rate for this condition is excellent today (∼90%), there have been no significant improvements over the past two decades. In silico models aim to simulate tumor progression and treatment responses over time; they hold immense potential for enhancing the predictive accuracy and optimizing treatment protocols as they are inspired by the digital twin paradigm.

Testicular and ovarian Juvenile granulosa cell tumors in children and adolescents: Analysis of 113 patients registered to the German Registry for Rare Pediatric Tumors (STEP).

Background: In juvenile granulosa cell tumors (juvGCTs), impaired survival was reported after preoperative tumor rupture, peritoneal metastases, or high mitotic rate (≥20 mitoses per 10 high-power fields). Therefore, a risk stratification was developed to select patients for chemotherapy.

Methods: Between 2001 and 2019, 89 female patients and 24 male patients were prospectively enrolled.

Model-Based Prediction of Clinically Relevant Thrombocytopenia after Allogeneic Hematopoietic Stem Cell Transplantation.

Platelet reconstitution after allogeneic hematopoietic cell transplantation (allo-HCT) is heterogeneous and influenced by various patient- and transplantation-related factors, associated with poor prognoses for poor graft function (PGF) and isolated thrombocytopenia. Tailored interventions could improve the outcome of patients with PGF and post-HCT thrombocytopenia. To provide individual predictions of 180-day platelet counts from early phase data, we developed a model of long-term platelet reconstitution after allo-HCT.

The Clinical Impact of Somatic Copy Number Variations in Patients With Stage IV Wilms Tumor Enrolled in the SIOP 2001 Trial and Study.

Background: Recent research elucidated the prognostic significance of molecular biology in Wilms tumor (WT) by linking somatic genomic variants (such as gain of chromosome 1q) to unfavorable patient outcomes. This analysis describes the clinical impact of copy number variations (CNV) in tumor samples of WT patients with stage IV disease.

Methods: Tumor samples of 55 WT patients with stage IV disease from the United Kingdom, France, and Germany enrolled in the SIOP 2001 study and treated with preoperative chemotherapy (pCHT) were examined for their CNVs of chromosome 1q and other regions of interest using multiplex ligation-dependent probe amplification (MLPA).

Hallmark discoveries in the biology of non-Wilms tumour childhood kidney cancers.

Approximately 20% of paediatric and adolescent/young adult patients with renal tumours are diagnosed with non-Wilms tumour, a broad heterogeneous group of tumours that includes clear-cell sarcoma of the kidney, congenital mesoblastic nephroma, malignant rhabdoid tumour of the kidney, renal-cell carcinoma, renal medullary carcinoma and other rare histologies. The differential diagnosis of these tumours dates back many decades, when these pathologies were identified initially through clinicopathological observation of entities with outcomes that diverged from Wilms tumour, corroborated with immunohistochemistry and molecular cytogenetics and, subsequently, through next-generation sequencing. These advances enabled near-definitive recognition of different tumours and risk stratification of patients.

Wilms tumor primary cultures capture phenotypic heterogeneity and facilitate preclinical screening.

Wilms tumors (WT) are characterized by variable contributions of blastemal, epithelial and stromal elements, reflecting their diverse cellular origins and genetic drivers. In vitro models remain rare, despite a growing need to better characterize tumor biology and evaluate new treatments. Using three approaches, we have now established a large collection of long-term cultures that represent this diversity.

Predisposition Footprints in the Somatic Genome of Wilms Tumors.

Approximately 10% of children with cancer harbor a mutation in a predisposition gene. In children with the kidney cancer Wilms tumor, the prevalence is as high as 30%. Certain predispositions are associated with defined histological and clinical features, suggesting differences in tumorigenesis.

Towards verifiable cancer digital twins: tissue level modeling protocol for precision medicine.

Cancer exhibits substantial heterogeneity, manifesting as distinct morphological and molecular variations across tumors, which frequently undermines the efficacy of conventional oncological treatments. Developments in multiomics and sequencing technologies have paved the way for unraveling this heterogeneity. Nevertheless, the complexity of the data gathered from these methods cannot be fully interpreted through multimodal data analysis alone.

Outcome after treatment with axitinib in children, young adults, and adults with renal cell carcinoma: a narrative review.

Renal cell carcinoma (RCC) is a very rare type of renal cancer in children and young adults. When metastasized or recurrent, no standards of care are available, and outcome is still poor. The tyrosine kinase inhibitor axitinib is approved for treatment of RCC in adults, but its effects in children and young adults with RCC remains unclear.

Targeted therapies in children with renal cell carcinoma (RCC): An International Society of Pediatric Oncology-Renal Tumor Study Group (SIOP-RTSG)-related retrospective descriptive study.

Introduction: Introduction: Renal cell carcinoma (RCC) is a very rare pediatric renal tumor. Robust evidence to guide treatment is lacking and knowledge on targeted therapies and immunotherapy is mainly based on adult studies. Currently, the International Society of Pediatric Oncology-Renal Tumor Study Group (SIOP-RTSG) 2016 UMBRELLA protocol recommends sunitinib for metastatic or unresectable RCC.

Characteristics and outcome of synchronous bilateral Wilms tumour in the SIOP WT 2001 Study: Report from the SIOP Renal Tumour Study Group (SIOP-RTSG).

Background: Among patients with nephroblastoma, those with bilateral disease are a unique population where maximising tumour control must be balanced with preserving renal parenchyma.

Methods: The SIOP 2001 protocol recommended surgery after neoadjuvant cycle(s) of Dactinomycin and Vincristine (AV) with response-adapted intensification, if needed. Adjuvant treatment was given based on the lesion with the worst histology.

Reviewing the current state of virtual reality integration in medical education - a scoping review.

Background: In medical education, new technologies like Virtual Reality (VR) are increasingly integrated to enhance digital learning. Originally used to train surgical procedures, now use cases also cover emergency scenarios and non-technical skills like clinical decision-making. This scoping review aims to provide an overview of VR in medical education, including requirements, advantages, disadvantages, as well as evaluation methods and respective study results to establish a foundation for future VR integration into medical curricula.

Irinotecan and temozolomide in combination with dasatinib and rapamycin versus irinotecan and temozolomide for patients with relapsed or refractory neuroblastoma (RIST-rNB-2011): a multicentre, open-label, randomised, controlled, phase 2 trial.

Background: Neuroblastoma is the most common extracranial solid tumour in children. Relapsed or refractory neuroblastoma is associated with a poor outcome. We assessed the combination of irinotecan-temozolomide and dasatinib-rapamycin (RIST) in patients with relapsed or refractory neuroblastoma.

Medulloblastoma in children with Fanconi anemia: Association with FA-D1/FA-N, SHH type and poor survival independent of treatment strategies.

Background: The outcome of children with medulloblastoma (MB) and Fanconi Anemia (FA), an inherited DNA repair deficiency, has not been described systematically. Treatment is complicated by high vulnerability to treatment-associated side effects, yet structured data are lacking. This study aims to give a comprehensive overview of clinical and molecular characteristics of pediatric FA MB patients.

Can Lymph Node Involvement in Unilateral Wilms Tumor be Predicted by Preoperatively known Data in Combination with Intraoperative Findings?

Objective: This study aimed to identify parameters that allow the estimation of tumor-infiltrated lymph nodes (LN) after pretreatment for unilateral Wilms tumor (WT).

Summary Background Data: Complete tumor resection with removal of regional LN is always necessary. Positive LNs require local irradiation influencing benefits in case of NSS in long-term follow-up.

A Multidisciplinary Hyper-Modeling Scheme in Personalized In Silico Oncology: Coupling Cell Kinetics with Metabolism, Signaling Networks, and Biomechanics as Plug-In Component Models of a Cancer Digital Twin.

The massive amount of human biological, imaging, and clinical data produced by multiple and diverse sources necessitates integrative modeling approaches able to summarize all this information into answers to specific clinical questions. In this paper, we present a hypermodeling scheme able to combine models of diverse cancer aspects regardless of their underlying method or scale. Describing tissue-scale cancer cell proliferation, biomechanical tumor growth, nutrient transport, genomic-scale aberrant cancer cell metabolism, and cell-signaling pathways that regulate the cellular response to therapy, the hypermodel integrates mutation, miRNA expression, imaging, and clinical data.

The impact of the route to diagnosis in nephroblastoma.

Introduction: Wilms tumor (WT) is the most common childhood kidney cancer. It is a rapid growing embryonal tumor in young children and can be diagnosed with and without tumor related symptoms.

Methods: We retrospectively analyzed the route to diagnosis of WT treated prospectively according to the SIOP 93-01/GPOH and 2001/GPOH in Germany between 1993 and 2022.

Gliomatosis cerebri in children: A poor prognostic phenotype of diffuse gliomas with a distinct molecular profile.

Background: The term gliomatosis cerebri (GC), a radiology-defined highly infiltrating diffuse glioma, has been abandoned since molecular GC-associated features could not be established.

Methods: We conducted a multinational retrospective study of 104 children and adolescents with GC providing comprehensive clinical and (epi-)genetic characterization.

Results: Median overall survival (OS) was 15.