HB patients GENOMIC STUDY TRANSCRIPTOMIC STUDY METHYLATION STUDY CytoScan HD ®-array RNA-sequencing/ ddPCR HTA ®-array/ RT-qPCR 850K (EPIC)-array/ QUAlu Dysregulation of global RNA & BLCAP editing Overexpression of 14q32 DLK1-DIO3 genes 16 + VIM-gene signature (C1/C2/C2B) 2 epigenomic HB subtypes (Epi-CA & Epi-CB) CLINICAL PARAMETERS: prognostic marker identification Poor prognostic factors:-4q,-18, 17q11.2 AI (NF1) CHKA new therapeutic target Molecular risk stratification MRS1 MRS2 MRS3 Strong 14q32 Epi-CB Time Survival Highlights Hepatoblastoma (HB) involves global dysregulation of RNA editing, including in the tumor suppressor BLCAP. Overexpression of a 300 kb region within the 14q32 DLK1/DIO3 locus is a new hallmark of HB. We identified 2 epigenomic HB subtypes-Epi-CA and Epi-CB-with distinct degrees of DNA hypomethylation and CpG island hypermethylation. The molecular risk stratification of HB, based on the 14q32-signature and epigenomic subtypes, is associated with patient outcomes. The enzyme CHKA could be a novel therapeutic target for patients with HB.

BACKGROUND AND OBJECTIVE: Parapneumonic empyema (PPE) is a frequent complication of acute bacterial pneumonia in children. There is limited evidence regarding the optimal treatment of this condition. The aim of this study was to compare the efficacy of drainage plus urokinase versus video-assisted thoracoscopic surgery in the treatment of PPE in childhood. METHODS: This prospective, randomized, multicenter clinical trial enrolled patients aged <15 years and hospitalized with septated PPE. Study patients were randomized to receive urokinase or thoracoscopy. The main outcome variable was the length of hospital stay after treatment. The secondary outcomes were total length of hospital stay, number of days with the chest drain, number of days with fever, and treatment failures. The trial was approved by the ethics committees of all the participating hospitals. RESULTS: A total of 103 patients were randomized to treatment and analyzed; 53 were treated with thoracoscopy and 50 with urokinase. There were no differences in demographic characteristics or in the main baseline characteristics between the 2 groups. No statistically significant differences were found between thoracoscopy and urokinase in the median postoperative stay (10 vs 9 days), median hospital stay (14 vs 13 days), or days febrile after treatment (4 vs 6 days). A second intervention was required in 15% of children in the thoracoscopy group versus 10% in the urokinase group (P = .47). CONCLUSIONS: Drainage plus urokinase instillation is as effective as video-assisted thoracoscopic surgery as first-line treatment of septated PPE in children.

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The endoscopic approach of late-presenting Morgagni and Bochdalek CDH is a safe technique. It offers all the advantages of minimally invasive surgery (MIS), and laparoscopy also permits the diagnosis and treatment of other associated conditions. There are a short number of cases reported and no prospective study comparing open with the MIS approach for the treatment of diaphragmatic malformations.

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