Search results for "Replacement"
showing 10 items of 561 documents
Albumin replacement in patients with severe sepsis or septic shock.
2014
BACKGROUND: Although previous studies have suggested the potential advantages of albumin administration in patients with severe sepsis, its efficacy has not been fully established. METHODS: In this multicenter, open-label trial, we randomly assigned 1818 patients with severe sepsis, in 100 intensive care units (ICUs), to receive either 20% albumin and crystalloid solution or crystalloid solution alone. In the albumin group, the target serum albumin concentration was 30 g per liter or more until discharge from the ICU or 28 days after randomization. The primary outcome was death from any cause at 28 days. Secondary outcomes were death from any cause at 90 days, the number of patients with or…
Clinical manifestations of Fabry disease in children: data from the Fabry Outcome Survey.
2006
Background Fabry disease is a rare X-linked disorder caused by deficient activity of the enzyme alpha-galactosidase A. This produces progressive lysosomal accumulation of globotriaosylceramide throughout the body, leading to organ failure and premature death. Aim Here, we present the clinical manifestations of Fabry disease in children enrolled in FOS--the Fabry Outcome Survey--a European database of the natural history of Fabry disease and the effects of enzyme replacement therapy with agalsidase alfa (Replagal). Methods Currently, there are 545 patients in FOS, from 11 European countries. We analysed the baseline demographic and clinical characteristics of 82 of these patients (40 boys, 4…
Inner ear function in children with Fabry disease
2007
Aim: The prevalence of hearing loss in patients with Fabry disease is still uncertain. This paper examines hearing loss in a group of young patients with Fabry disease. Methods: A clinical ear nose and throat examination,pure-tone air and bone conduction audiometry, speech audiometry and middle ear testing (tympanometry and acoustic reflex testing) were carried out in four girls and two boys with Fabry disease (age, 7-17 years), receiving enzyme replacement therapy (ERT). Results: None of the patients complained of a hearing disorder or suffered from hearing loss. Three female patients reported tinnitus; however, this was not reported as being a problem. One boy reported tinnitus for the fi…
High prevalence of hypovitaminosis D in Sicilian children affected by growth hormone deficiency and its improvement after 12 months of replacement tr…
2014
PURPOSE: Although the correlation between vitamin D and growth hormone (GH)-insulin-like growth factor 1 (IGF1) axis is documented, as of date, few and conflicting studies have prospectively analyzed vitamin D before and after GH treatment. Our aim was to evaluate as to how the condition of GH deficiency (GHD) or GH treatment influences vitamin D in children. METHODS: Eighty Sicilian GHD children (M/F 58/22; mean age 10.3 years), grouped according to the season of evaluation in group A (June-September; 41 children) and group B (November-February; 39 children), were evaluated at baseline and after 12 months of GH treatment. RESULTS: Twenty-eight children (35 %) were vitamin D insufficient an…
Measuring patient experiences in Fabry disease: validation of the Fabry-specific Pediatric Health and Pain Questionnaire (FPHPQ)
2012
Abstract Introduction Common symptoms for children with Anderson-Fabry Disease (FD) such as acroparaesthesia and gastrointestinal manifestations can only be objectively assessed in patients using a valid instrument. To date, no such instrument exists. Methods A preliminary 40-item measure of symptoms and experience with FD, the Fabry-specific Paediatric Health and Pain Questionnaire (FPHPQ) was developed, but lacked a formal assessment of its measurement properties. The FPHPQ was used in the Fabry Outcome Survey (FOS), a registry for all patients with a confirmed diagnosis of FD who are receiving agalsidase alfa, or are treatment naïve and who are managed by physicians participating in FOS.…
Hunter disease before and during enzyme replacement therapy.
2011
Mucopolysaccharidosis type II (Hunter disease) is a lysosomal storage disease attributable to X-linked deficiency of the enzyme α-L-iduronate-sulfatase. Because of this deficiency, glycosaminoglycanes accumulate in various tissues and body fluids. We describe three patients representing the broad spectrum of Hunter disease and their response to enzyme replacement therapy. Patient 1 did not manifest central nervous system involvement, patient 2 manifested moderate neurologic disease, and patient 3 had already manifested a severe neurologic course during early infancy. In all patients, improvements in visceral organ size, physical capacity, and gastrointestinal functioning were reported. More…
Growth hormone replacement therapy prevents sarcopenia by a dual mechanism: improvement of protein balance and of antioxidant defenses.
2013
The aim of our study was to elucidate the role of growth hormone (GH) replacement therapy in three of the main mechanisms involved in sarcopenia: alterations in mitochondrial biogenesis, increase in oxidative stress, and alterations in protein balance. We used young and old Wistar rats that received either placebo or low doses of GH to reach normal insulin-like growth factor-1 values observed in the young group. We found an increase in lean body mass and plasma and hepatic insulin-like growth factor-1 levels in the old animals treated with GH. We also found a lowering of age-associated oxidative damage and an induction of antioxidant enzymes in the skeletal muscle of the treated animals. GH…
Nocturnal blood pressure and progression to end-stage renal disease or death in nondiabetic chronic kidney disease stages 3 and 4
2010
Objective The objective was to assess the role of office and ambulatory blood pressure (BP) on the development of end-stage renal disease (ESRD) in nondiabetic chronic renal failure. Design and method Seventy-nine patients [mean age 57 (standard deviation 11) years, 47 men, BMI 28 (4), office BP 151 (25)/92 (14) mmHg, estimated glomerular filtration rate 28 (14) ml/min per 1.73 m3] were included. The causes of renal disease were nephrosclerosis (n = 33), glomerulonephritis (n = 19), interstitial (n = 12) and others (n = 15). The average follow-up was 44 months (range 9-72 months). The primary outcome was a composite of death, from any cause, or the development of ESRD that require initiatio…
ECG-GATED MULTIDETECTOR COMPUTED TOMOGRAPHY FOR THE ASSESSMENT OF THE POSTOPERATIVE ASCENDING AORTA
2009
This study was undertaken to define the role of electrocardiographically (ECG)-gated multidetector computed tomography (MDCT) in the assessment of the postoperative ascending aorta. From November 2006 to June 2007, 21 patients, [11 men, ten women; age +/- standard deviation (SD): 62.7 +/- 10.8 years] with a history of ascending aorta replacement underwent ECG-gated MDCT and were prospectively included in our study. Ascending aorta replacement had been performed with different surgical techniques: Bentall-De Bono (four patients, 19%), Tirone-David (five patients, 23%), and modified Tirone-David with creation of aortic neosinuses (12 patients, 57%). Two patients were excluded from MDCT evalua…
The role of muscular trauma in the development of heterotopic ossification after hip surgery: An animal-model study in rats.
2015
Abstract Background Heterotopic ossification (HO), the formation of bone in soft tissues, is a frequent complication after surgery of the hip and the pelvis. Although the pathophysiological entities responsible for the formation of HO remain largely unclear, muscle trauma is alleged to play a central role in the pathogenic mechanisms underlying HO. However, for this observation, made by many surgeons for decades, no objective evidence has been provided yet. Methods Fifty male Wistar rats were subjected to surgery of the right hip. The femoral canal was reamed in three steps up to 2 mm. Animals formed 2 groups: in group 1 (25 animals) every effort was taken not to injure the muscles. In cont…