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Background: Neuroblastoma is a heterogeneous disease with adrenergic (ADRN)- and therapy resistant mesenchymal (MES)-like cells driven by distinct transcription factor networks. Here, we investigate the expression of immunotherapeutic targets in each neuroblastoma subtype and propose pan-neuroblastoma and cell state specific targetable cell-surface proteins.
Methods: We characterized cell lines, patient-derived xenografts, and patient samples as ADRN-dominant or MES-dominant to define subtype-specific and pan-neuroblastoma gene sets.
New onset refractory status epilepticus: Long-term outcomes beyond seizures.
Epilepsia
January 2025
Department of Clinical Neurological Sciences, Schulich School of Medicine and Dentistry, Western University, London, Ontario, Canada.
We propose and prioritize important outcome domains that should be considered for future research investigating long-term outcomes (LTO) after new onset refractory status epilepticus (NORSE). The study was led by the international NORSE Institute LTO Working Group. First, literature describing the LTO of NORSE survivors was identified using a PubMed search and summarized to identify knowledge gaps.
View Article and Find Full Text PDFPrevalence of and risk factors for osteoporosis and fragility fracture in adults with cerebral palsy: A systematic review.
Dev Med Child Neurol
January 2025
Centre for Endocrinology and Metabolism, Hudson Institute of Medical Research, Clayton, VIC, Australia.
Aim: To systematically review the prevalence and incidence of osteoporosis, osteopenia, low bone mass, and fragility fracture in adults with cerebral palsy (CP), and identify the risk factors for osteoporosis and fracture.
Method: A systematic literature search was performed in the MEDLINE, PubMed, CINAHL, AMED, Cochrane Reviews, EMBASE, and EBM database reviews from inception until May 2024. Search terms covered a combination of keywords for CP, fracture, osteoporosis, incidence and prevalence, and risk factors.
Transition-to-home arrangements for very preterm infants and related parental needs at perinatal centres in Austria and Switzerland: a cross-sectional multicentred study.
BMJ Paediatr Open
January 2025
Department of Pediatrics II, Neonatology, Medical University of Innsbruck, Innsbruck, Austria
Introduction: The current study aims to give an overview of transition-to-home services provided by perinatal centres in Austria and Switzerland and to evaluate parental satisfaction with the care provided.
Methods: This cross-sectional multicentred study was conducted by performing two surveys between May 2022 and November 2023: one among all level III perinatal centres in Austria (n=7) and Switzerland (n=9) (institutional survey) and one among parents of very preterm infants treated at one selected perinatal centre in each of the two countries (parental survey). Both questionnaires consisted of matching questions focusing on current transition-to-home services.
Exploring the experience of stigma among children and adolescents with epilepsy in China: A qualitative study.
Epilepsy Behav
January 2025
Objective: Epilepsy-related stigma negatively affects the lives of children and adolescents. This study aims to explore the experience of stigma among children and adolescents with epilepsy in China.
Methods: This study used purposive sampling to select children and adolescents aged 10-18 diagnosed with epilepsy.
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