BACKGROUND: The SIOP-Renal Tumor Study Group (RTSG) does not advocate invasive procedures to determine histology before the start of therapy. This may induce misdiagnosis-based treatment initiation, but only for a relatively small percentage of approximately 10% of non-Wilms tumors (non-WTs). MRI could be useful for reducing misdiagnosis, but there is no global consensus on differentiating characteristics. PURPOSE: To identify MRI characteristics that may be used for discrimination of newly diagnosed pediatric renal tumors. STUDY TYPE: Consensus process using a Delphi method. POPULATION: Not applicable. FIELD STRENGTH/SEQUENCE: Abdominal MRI including T1- and T2-weighted imaging, contrast-enhanced MRI, and diffusion-weighted imaging at 1.5 or 3 T. ASSESSMENT: Twenty-three radiologists from the SIOP-RTSG radiology panel with ≥5 years of experience in MRI of pediatric renal tumors and/or who had assessed ≥50 MRI scans of pediatric renal tumors in the past 5 years identified potentially discriminatory characteristics in the first questionnaire. These characteristics were scored in the subsequent second round, consisting of 5-point Likert scales, ranking- and multiple choice questions. STATISTICAL TESTS: The cut-off value for consensus and agreement among the majority was ≥75% and ≥60%, respectively, with a median of ≥4 on the Likert scale. RESULTS: Consensus on specific characteristics mainly concerned the discrimination between WTs and non-WTs, and WTs and nephrogenic rest(s) (NR)/nephroblastomatosis. The presence of bilateral lesions (75.0%) and NR/nephroblastomatosis (65.0%) were MRI characteristics indicated as specific for the diagnosis of a WT, and 91.3% of the participants agreed that MRI is useful to distinguish NR/nephroblastomatosis from WT. Furthermore, all participants agreed that age influenced their prediction in the discrimination of pediatric renal tumors. DATA CONCLUSION: Although the discrimination of pediatric renal tumors based on MRI remains challenging, this study identified some specific characteristics for tumor subtypes, based on the shared opinion of experts. These results may guide future validation studies and innovative efforts. LEVEL OF EVIDENCE: 3 Technical Efficacy Stage: 3.
MyPal is a European initiative focusing on the use of the electronic patient reported outcome (ePRO) measures to enhance patient engagement in palliative cancer care via digital self-reporting palliative care for patients with cancer. As a part of its approach, MyPal also focuses on pediatric patients, implementing a specific digital health platform including a serious game to facilitate the reporting of the symptoms and overall status regarding their quality of life (QoL). To this end, the reduction of psychological burden related to frequent reporting, a.k.a. as "reporting fatigue" has been identified as a priority. In this study, we present the MyPal-CHILD platform, emphasizing on the serious game named AquaScouts and its key design decisions, while also emphasizing on the respective challenges. More specifically, we provide insights on the participatory design approach applied during the design of the platform and the high-level goals defined based on end-user input. In addition, the validation process applied before the use of the platform under real-world conditions is also presented. Finally, we discuss a number of challenges and the prospects of deploying eHealth interventions to support palliative care.
- Publikační typ
- časopisecké články MeSH
INTRODUCTION: Electronic patient-reported outcomes (ePROs) have tremendous potential to optimise palliative and supportive care for children with cancer, their families and healthcare providers. Particularly, these children and their families are subjected to multiple strains caused by the disease and its treatment. The MyPal digital health platform is designed to address these complex demands by offering pursuant ePRO-based functionalities via two mobile applications, one developed for children and the other for their parents. METHODS AND ANALYSIS: In this observational prospective feasibility study, 100 paediatric oncology patients aged between 6 and 17 years and at least one of their parents/legal guardians will be recruited at three clinical sites in two European countries (Germany and Czech Republic). They will use the mobile applications which are part of the novel digital health platform. During a 6-month study period, participants will complete various ePROs via the applications addressing quality of life, satisfaction with care and impact of the disease on the family at monthly intervals. Additionally, priority-based symptom reporting is integrated into a serious game for children. Outcomes that will be assessed concern the feasibility and the evaluation of the newly designed digital health platform to contribute to the evidence base of clinical ePRO use in paediatric oncology and palliative care process. ETHICS AND DISSEMINATION: The MyPal-Child study obtained ethical approval from the Ethics Committee responsible for the University of Saarland, that is, the Ärztekammer des Saarlandes, the Ethics Committee of the Medical School Hannover and the Ethics Committee of the University of Brno. Study results will be disseminated through scientific publications, presentations at international conferences, congresses and a final report to the European Commission. General publicly accessible information can be found on the project website (www.mypal-project.eu) and social media. TRIAL REGISTRATION NUMBERS: U1111-1251-0043, DRKS00021458, NCT04381221.
- MeSH
- dítě MeSH
- kvalita života MeSH
- lidé MeSH
- mladiství MeSH
- nádory * terapie MeSH
- paliativní péče * MeSH
- prospektivní studie MeSH
- studie proveditelnosti MeSH
- Check Tag
- dítě MeSH
- lidé MeSH
- mladiství MeSH
- Publikační typ
- časopisecké články MeSH
- pozorovací studie MeSH
- práce podpořená grantem MeSH
- Geografické názvy
- Česká republika MeSH
- Evropa MeSH
- Německo MeSH
BACKGROUND: Controversy exists as to what may be defined as standard of care (including markers for stratification) for patients with atypical teratoid/rhabdoid tumors (ATRTs). The European Rhabdoid Registry (EU-RHAB) recruits uniformly treated patients and offers standardized genetic and DNA methylation analyses. METHODS: Clinical, genetic, and treatment data of 143 patients from 13 European countries were analyzed (2009-2017). Therapy consisted of surgery, anthracycline-based induction, and either radiotherapy or high dose chemotherapy following a consensus among European experts. Fluorescence in situ hybridization, multiplex ligation-dependent probe amplification, and sequencing were employed for assessment of somatic and germline mutations in SWItch/sucrose nonfermentable related, matrix associated, actin dependent regulator of chromatin, subfamily B (SMARCB1). Molecular subgroups (ATRT-SHH, ATRT-TYR, and ATRT-MYC) were determined using DNA methylation arrays, resulting in profiles of 84 tumors. RESULTS: Median age at diagnosis of 67 girls and 76 boys was 29.5 months. Five-year overall survival (OS) and event-free survival (EFS) were 34.7 ± 4.5% and 30.5 ± 4.2%, respectively. Tumors displayed allelic partial/whole gene deletions (66%; 122/186 alleles) or single nucleotide variants (34%; 64/186 alleles) of SMARCB1. Germline mutations were detected in 26% of ATRTs (30/117). The patient cohort consisted of 47% ATRT-SHH (39/84), 33% ATRT-TYR (28/84), and 20% ATRT-MYC (17/84). Age <1 year, non-TYR signature (ATRT-SHH or -MYC), metastatic or synchronous tumors, germline mutation, incomplete remission, and omission of radiotherapy were negative prognostic factors in univariate analyses (P < 0.05). An adjusted multivariate model identified age <1 year and a non-TYR signature as independent negative predictors of OS: high risk (<1 y + non-TYR; 5-y OS = 0%), intermediate risk (<1 y + ATRT-TYR or ≥1 y + non-TYR; 5-y OS = 32.5 ± 8.7%), and standard risk (≥1 y + ATRT-TYR, 5-y OS = 71.5 ± 12.2%). CONCLUSIONS: Age and molecular subgroup status are independent risk factors for survival in children with ATRT. Our model warrants validation within future clinical trials.
- MeSH
- dítě MeSH
- dospělí MeSH
- hybridizace in situ fluorescenční MeSH
- lidé MeSH
- metylace DNA MeSH
- mladiství MeSH
- mladý dospělý MeSH
- rhabdoidní nádor * genetika terapie MeSH
- rizikové faktory MeSH
- teratom * genetika terapie MeSH
- věkové rozložení MeSH
- Check Tag
- dítě MeSH
- dospělí MeSH
- lidé MeSH
- mladiství MeSH
- mladý dospělý MeSH
- mužské pohlaví MeSH
- ženské pohlaví MeSH
- Publikační typ
- časopisecké články MeSH
- práce podpořená grantem MeSH
- Geografické názvy
- Evropa MeSH
