Great Ormond Street Hospital for Children NHS Foundation Trust

Paediatric haematopoietic stem cell transplantation research priorities: Results from an international multi‐stakeholder priority setting partnership

Summary Co‐designed research in paediatric HSCT is limited. We sought to determine research priorities which represent the shared priorities of patients, parents, carers and healthcare professionals (HCP) within Australia, New Zealand, the Netherlands and the United Kingdom. An i...

Where in the Prenatal Screening Pathway do Ethnic and Socioeconomic Inequalities Occur? A UK Retrospective Cohort Study

ABSTRACT Objective To examine if and where ethnic and socioeconomic inequalities arise within the English NHS prenatal screening pathway by investigating associations between ethnicity, socioeconomic deprivation, screening test type, further testing uptake, and pregnancy outcomes...

MRI Characteristics of Myelin Oligodendrocyte Glycoprotein Antibody-Associated Disease

Importance Myelin oligodendrocyte glycoprotein antibody-associated disease (MOGAD) is a demyelinating disease distinct from multiple sclerosis (MS) and aquaporin-4 antibody-positive neuromyelitis optica spectrum disorder (AQP4+NMOSD). Magnetic resonance imaging (MRI) is central t...

Diagnostic Yield of Sequencing in Prenatal Agenesis of the Corpus Callosum in a Well‐Phenotyped International Cohort

ABSTRACT Objective To evaluate the incremental diagnostic yield of sequencing in a large, well‐phenotyped international cohort of fetuses with prenatally diagnosed agenesis of the corpus callosum (ACC) and to identify associated genes and variants. Methods Retrospective multicent...

Clinical efficacy/effectiveness, safety, and health-related quality of life associated with relapsed/refractory pediatric low-grade glioma treatments: systematic literature review

Abstract Background Patients with relapsed/refractory pediatric low-grade glioma (R/R pLGG) face considerable long-term morbidity. Most cases are driven by mitogen-activated protein kinase (MAPK) pathway alterations, for which several targeted agents have been evaluated. Systemat...

Strategic roadmap for delivery of clinical trials in rare childhood central nervous system (CNS) tumours: a multi-stakeholder consensus

Abstract Rare paediatric central nervous system (CNS) tumours comprise a biologically-heterogeneous group of low-incidence diseases that present significant challenges to both therapeutic development and clinical research, limiting the availability of high-quality evidence to gui...

Radiological Late Dental Adverse Effects Following Treatment for Paediatric Head and Neck Rhabdomyosarcoma: Results of a Transatlantic Multicentre Cohort Study

ABSTRACT Objectives Rhabdomyosarcoma represents the most common paediatric soft tissue sarcoma, with 40% occurring in the head and neck region. Given that survival rates now reach 80%, minimising late adverse effects, particularly dental developmental complications, has become a...