Assessment Of Liver and Spleen Fibrosis in Patients With Gaucher Disease Using Fibroscan
FibrosisThe investigated cohort will examine liver and spleen fibrosis in patients with Gaucher disease divided into two groups, naive GD patients and GD patients treated with ERT. As liver biopsy in these patients not recommended because the risk of bleeding using Fibroscan is a safe with diagnostic accuracy regarding the liver (& Spleen) fibrosis. Estimating spleen fibrosis is an innovative approach in liver disease and Gaucher. The evaluation of fibrosis with this new and safe method could avoid complications antiinvasive procedure in GD patients. The addition of fibrosis biomarkers will help for patients score evaluation. The finding of liver and spleen stiffness will be evaluated in native and ERT treated Gaucher patients in order to assess ERT effect on fibrosis. The Aims are: 1) To assess liver and spleen stiffness measurement using fibroscan and evaluate liver and spleen fibrosis in patients with GD. 2) To compare the elastography in two cohorts of GD patients: ERT treated and naïve GD patients and two control groups of patients: healthy and Non Alcoholic Steatohepatitis (NASH) patients. 3) To correlate the elastography findings with clinical and laboratory data in the four patient groups focusing on Gaucher disease manifestations and GD severity. To compare the elastography in GD naïve and ERT treated patients.
The Effect of Pirfenidone on Cough in Patients With Idiopathic Pulmonary Fibrosis
Idiopathic Pulmonary FibrosisIn this study we evaluate the effect of Pirfenidone on cough and quality of life in patients with idiopathic pulmonary fibrosis (IPF) that are treated with Pirfenidone in daily practice. The hypothesis is that Pirfenidone will decrease cough and increase quality of life.
Ivacaftor in French Patients With Cystic Fibrosis and a G551D Mutation
Cystic FibrosisThe purpose of this study is to determine whether the treatment with Ivacaftor remains effective and safe in the patients with cystic fibrosis (and at least one G551D CFTR mutation) in the real life setting, after the drug has been approved by the Health authorities.
Using Remote Telemonitoring to Detect Early Decline in Lung Function & Streamline Clinics in Adults...
Cystic FibrosisLung Health research study (Development of a predictive model) - To determine whether the I-neb breathing parameters (flow data) can act as a surrogate marker for lung function (Forced Expiratory Volume in 1 second FEV1) hence allow early detection of decline in lung function in cystic fibrosis patients.
Relationship Between HbA1c and Chronic Glycemia in Patients With Cirrhosis
CirrhosisDiabetes Mellitus1 moreCirrhosis and advanced liver disease have been associated with an increased risk for hyperglycemia and type 2 diabetes mellitus (T2DM). The diagnostic yield of common tests used to define diabetes and insulin resistance in the general population differs significantly from the one observed in patients with liver disease. Glycosylated hemoglobin A1c (HbA1c), a reliable test to assess chronic glycemia and recommended both for the diagnosis and monitoring of T2DM, is neither accurate nor reliable in patients with cirrhosis. A validation study has not been performed to define its true usefulness in the setting of cirrhosis. The study aims to determine the level of HbA1c that better corresponds to the diagnosis of T2DM - as determined by an oral glucose tolerance test (OGTT) - and to correlate the levels of HbA1c with the average glucose over a 12-week period in patients with cirrhosis and known T2DM, in cirrhotic patients with different degrees of liver impairment as compared to patients with T2DM and no liver disease.
Safety and Efficacy of Bemiparin in the Prevention of Thrombotic Events in Hospitalized Cirrhotic...
Cirrhosis and CoagulationProspectively evaluation of the incidence of thromboembolic events in hospitalized cirrhotic patients. 2. Efficacy and safety of use of bemiparin in preventing peripheral and portal thrombosis. 3. Monitoring antiXa levels.
Lung MRI and Allergic Broncho-pulmonary Aspergillosis in Cystic Fibrosis
Pulmonary Cystic FibrosisABPAIn this diagnostic study, the aim is at evaluating the diagnostic accuracy of MRI (Magnetic Resonance Imaging) to detect allergic broncho-pulmonary aspergillosis in patients with cystic fibrosis.
Icodextrin Effects on Glucose Transporter Activation and Mediators of Fibrosis
End Stage Renal DiseaseThe time on peritoneal dialysis may be limited for a significant number of patients that use this modality of renal replacement therapy due to the inability of the peritoneal membrane to clear solutes or achieve adequate ultrafiltration, termed peritoneal membrane failure (PMF). This can be devastating for patients who have become accustomed to the quality of life provided by peritoneal dialysis and who otherwise have done well on this therapy. There is clinical evidence suggesting that icodextrin preserves the peritoneal membrane transport characteristics which may be linked to reduced cumulative glucose exposure of the peritoneal mesothelial cells. Theories to explain the role of dextrose in PMF have focused for the most part on the high intracellular concentrations of glucose without consideration to the potential pathogenic role of the glucose transporters which allow glucose entry into the cell. Experimental evidence in non-mesothelial cell lines indicate that some cellular processes that occur under high glucose conditions may not be related to intracellular glucose metabolism but to the type of glucose transporter allowing glucose entry. 3,4 However, little is known about these glucose transporters in peritoneal mesothelial cells and their potential role in the development of PMF. We hypothesize the following The presence of Sodium Glucose Co-transporter (SGLT1) on peritoneal mesothelial cells plays a role in PMF under hyperglycemic conditions. Regulation of pro-fibrotic mediators such as reactive oxidative species,transforming growth factor β, and vascular endothelial growth factor are modulated by SGLT1 activation by glucose rather than glucose metabolism or concentration. Icodextrin does not activate the SGLT1 transporter establishing a mechanism that may explain the beneficial effects of icodextrin on peritoneal membrane transport.
A Follow-up Evaluation Study of PRI-724-1101
Hepatitis C Virus-infected CirrhosisThe purpose of this study To evaluate the safety and efficacy of PRI-724 administration in patients with cirrhosis due to hepatitis C by 12-month follow-up.
Role of Nox2 in CNI-induced Renal Fibrosis
Kidney DiseaseCalcineurin Inhibitors (CNI) are drugs used to suppress the immune system when a person has a solid organ transplant. Although these drugs keep the transplanted organ from being rejected they are toxic to kidneys, or nephrotoxic. CNIs cause damage, called fibrosis, to kidneys. Fibrosis is a type of scarring that occurs in kidney tissue. Fibrosis can eventually lead to kidney failure. One of the pathways that cause fibrosis is a chronic lack of oxygen to the kidney tissue called "hypoxia". There is a protein called Nox2 that may be involved in how this hypoxia happens in the kidney. The Department of Medicine-Nephrology at the University of Wisconsin is conducting a research study to see how much of the Nox2 protein is present in kidneys that may have fibrosis caused by CNIs and whether a certain type of Magnetic Resonance Imaging (MRI) can be used to tell in advance if the disease caused by CNIs is getting worse. Study hypothesis: MRI, a non-invasive technique, can be used to determine whether CNI induced kidney disease is getting worse. Additionally, the study aims to determine the role of Nox2 in CNI nephrotoxicity.