Peter P Moschovis, Anupama Dinesh, Anna-Sophia Boguraev, Brett D Nelson
Remote online global health education among U.S. medical students during COVID-19 and beyond
BMC medical education
paediatric hepatocellular carcinomas hcc traditionally arise context normal structural functional liver carry dismal prognosis chemotherapy frontline standard emerging interest study immunotherapies paediatric patients relapsedrefractory disease limited data support whether immunotherapies utility patient population six paediatric patients median age16 years range 1217 time treatment advanced hepatocellular neosplams either conventional hepatocellular fibrolamellar carcinoma treated immunotherapy patients consented institutional genomic profiling biobanking protocols baseline samples serial tissue samples available evaluated somatic mutation rate actionable gene mutations panimmune bulk rna expression profiling results correlated clinical course three patients responded checkpoint inhibition one achieved complete durable response two prolonged stable disease three additional patients progressed diagnostic tissue complete responder demonstrated higher relative mutational burden robust immune infiltrate pretreatment samples three responders demonstrated decreased expression genes associated tcell dysfunction subset patients primary paediatric hepatocellular tumours respond immunotherapy immunotherapies currently prospective study relapsedrefractory liver tumours paediatric patients results report support prospective collection serial serum tissue samples may identify genomic immunophenotypic patterns predictive response work supported philanthropic funds pan mass challenge team angus team perspective