Undifferentiated embryonal sarcoma might be suggested in a non-secreting unifocal tumour with well-defined borders, fluid-filled spaces on US, hypoattenuation and serpiginous vessels on CT, and if there are signs of internal bleeding or rupture on CT or MRI.

The aim of this Phase 1/2, 2-part, multicenter trial was to report clinical safety and efficacy of long-term golodirsen treatment among ambulatory patients with exon 53 skip-amenable Duchenne muscular dystrophy (DMD). Part 1 was a 12-week, randomized, double-blind, placebo-controlled, dose-titration study followed by 9-week safety review. Part 2 was a 168-week, open-label evaluation of golodirsen 30 mg/kg. Part 1 primary endpoint was safety. Part 2 primary endpoints were dystrophin protein expression and 6-minute walk test (6MWT); secondary endpoints were percent predicted forced vital capacity (FVC%p) and safety. Post hoc ambulation analyses used mutation-matched external natural history controls. All patients from Part 1 (golodirsen, n = 8; placebo, n = 4) plus 13 additional patients entered Part 2; 23 completed the study. Adverse events were generally mild, nonserious, and unrelated to golodirsen, with no safety-related discontinuations or deaths. Golodirsen increased dystrophin protein (16.0-fold; P < 0.001) and exon skipping (28.9-fold; P < 0.001). At 3 years, 6MWT change from baseline was −99.0 m for golodirsen-treated patients versus −181.4 m for external controls ( P = 0.067), and loss of ambulation occurred in 9% versus 26% ( P = 0.21). FVC%p declined 8.4% over 3 years in golodirsen-treated patients, comparing favorably with literature-reported rates. This study provides evidence for golodirsen biologic activity and long-term safety in a declining DMD population and suggests functional benefit versus external controls. Clinical Trial Registration number: NCT02310906.

The aim of this study is to compare the outcomes and the complications between the 2 most adopted procedures for gastrostomy placement: percutaneous endoscopic gastrostomy (PEG) and laparoscopic gastrostomy (LG) in children. We present our study on 69 patients (male: 46/female: 23): group 1 (37 patients, 54%) undergoing PEG, group 2 (32 patients, 46%) undergoing LG. A total of 5 major complications were observed all in the PEG group (13.5%), no major complication was observed in the LG group (P-value<0.05). A total of 12 minor complications were observed: 4 occurred in the PEG group (10.8%) and 8 (25%) in the laparoscopic gastrostmoy group, not statistically relevant. We suggest that the LG should be considered the preferred technique for gastrostomy placement in pediatric patients, particularly in newborns, children with significant skeletal malformations, and patients who underwent previous abdominal surgery.

USL presents with painful mixed cystic and solid liver mass. If misdiagnosed and mistreated (enucleation or unroofing), the usual good outcome of this malignancy could be impaired. Preoperative chemotherapy is recommended.