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Background. Wilson disease (WD) is an autosomal-recessive disorder of copper metabolism, caused by mutations in the ATP7B gene, which codes for a membrane-bound copper-binding ATPase. This leads to progressive copper accumulation in the liver, with hepatic injury and subsequent copper release into the blood. Aim. To analyze the efficacy and side-effects of the current copper chelating agents used in treatment of Wilson disease. Material and methods. Retrospective study of 37 adult patients diagnosed with Wilson disease at the Gastroenterology and Neurology Departments of Fundeni Clinical Institute between 2012 and 2017. Patients were grouped into three categories: (a) those with isolated liver disease, (b) those with isolated neurologic (or psychiatric) involvement and (c) those with both liver and neurologic involvement. Results. There were 54% females (n=20). Mean age at diagnosis was 23 ± 10 years-old. Liver cirrhosis was diagnosed in 54% of cases. Neurologic involvement was described in 65% of patients. Dysarthria was the most common neurologic feature (43%), followed by Parkinsonism (41%). D-Penicillamine was used as initial treatment in 89% of patients, but was interrupted in 27% of them (n=9) due to its adverse reactions. Keyser-Fleischer rings were absent in a significant proportion (40%) of patients with neurologic involvement. Both Trientine and D-Penicillamine were associated with improved or stationary liver fibrosis, however results were slightly better for Trientine. D-Penicillamine appeared to be a better option than Trientine in patients with neurologic involvement. Conclusions. Based on our study, we recommend treatment with D-Penicillamine as first-line therapy in patients with neurologic involvement. However, due to the frequent side-effects of D-Penicillamine, Trientine could be considered firstline treatment in patients with isolated hepatic involvement. [ABSTRACT FROM AUTHOR]

Background & Aims: Ombitasvir/Paritaprevir/ritonavir/Dasabuvir (OBV/PTV/r+DSV) is one of the elective direct-acting antivirals (DAAs) recommended by international guidelines and the only one covered by the National Insurance System in Romania until November 2016. Our aim was to present the first prospective Romanian cohort evaluating the effectiveness and safety in clinical practice of this 3DAA combination in patients with HCV genotype-1b Child A liver cirrhosis. Methods: 681 patients received OBV/PTV/r+DSV+RBV for 12 weeks and were assessed clinically and biologically at baseline, week 4, 8, 12 (end of treatment, EOT), and 12 weeks after therapy (sustained viral response, SVR). Results: Per protocol, EOT virological response was 99.8% and SVR12 rate was 99.4%. Adverse events were present in 36.4% of patients. Permanent discontinuation of 3DAA regimen due to side effects was reported in 11 patients (1.6%). In 47.6% (185/389) of patients, Transient Elastography values were >20kPa (defined as clinically significant portal hypertension, CSPH) at baseline. Independent variables associated with CSPH were: baseline cholesterol level (p=0.003), platelet count <120,000/mm? (p=0.02), MELD score (p=0.01). Liver stiffness measurement has significantly improved between baseline (26.6±12.7kPa) and SVR12 (21.6±11.8kPa) (p<0.0001). The same was true for APRI score (2.66±0.15 at baseline vs 0.85±0.02 at SVR12, p<0.0001) and FIB4 score (5.53±0.28 vs 3.24±0.08, p<0.0001), but not for Lok score (0.57±0.01 vs 0.63±0.01, p<0.0001). Conclusions: We report a high efficacy of the 3DAA regimen in a homogeneous compensated HCV genotype- 1b liver cirrhosis population, in a real-life setting. Noninvasive fibrosis scores significantly improved at SVR12. [ABSTRACT FROM AUTHOR]