Abstract:
AIMS:This study estimated the resource implications and budget impact of managing adults with Fabry disease in Italy, from the perspective of the Servizio Sanitario Nazionale (SSN). METHODS:A decision model was constructed using published clinical outcomes and clinician-derived resource utilisation estimates depicting the management of adults with Fabry disease in Italy. RESULTS:The expected annual cost of managing 220 existing and 20 new Fabry patients in Italy was estimated to be €28·3 million. In an average year, patients receiving enzyme replacement therapy (ERT) with 0·2 mg kg(-1) agalsidase alfa (Replagal; Shire Human Genetic Therapies, Basingstoke, Hampshire, UK) or 1·0 mg kg(-1) agalsidase beta (Fabrazyme; Genzyme Europe BV, Naarden, The Netherlands) are collectively expected to make 4500 hospital attendances to a day ward for infusions, which equates to 2000 eight-h days on the day ward associated with ERT. If all ERT-treated patients received their infusions at home, there would be a marginal reduction in the annual health care cost to manage these patients, and the total annual number of days on the day ward associated with ERT in the second year could potentially be reduced from a mean 2000 to zero, thereby releasing substantial hospital resources for use by non-Fabry patients. Currently, only agalsidase alfa is licensed for home treatment in Italy; hence, only patients receiving this enzyme could be offered home treatment. CONCLUSION:Use of agalsidase alfa (0·2 mg kg(-1) ) instead of agalsidase beta (1·0 mg kg(-1)) has the potential to reduce health care costs and release hospital resources in different specialities for alternative use by non-Fabry patients, thereby improving the efficiency of the public health care system in Italy.
journal_name
Eur J Clin Investjournal_title
European journal of clinical investigationauthors
Guest JF,Concolino D,Di Vito R,Feliciani C,Parini R,Zampetti Adoi
10.1111/j.1365-2362.2010.02458.xsubject
Has Abstractpub_date
2011-07-01 00:00:00pages
710-8issue
7eissn
0014-2972issn
1365-2362journal_volume
41pub_type
杂志文章abstract::The pregnancies of two patients with mild intrahepatic cholestasis of pregnancy (RCP) were followed with detailed analyses of bile acids in urine. About twenty-five different bile acids were determined by GC/MS following separation according to mode of conjugation. The results were collated with the clinical course of...
journal_title:European journal of clinical investigation
pub_type: 杂志文章
doi:10.1111/j.1365-2362.1979.tb00906.x
更新日期:1979-12-01 00:00:00
abstract:BACKGROUND:Chronic hyponatremia is common and associated with increased morbidity and mortality. However, whether treatment improves outcome in patients without significant symptoms is unclear. We here assessed the therapeutic outcome on clinical symptoms, neurocognitive and neuromuscular function in patients with chro...
journal_title:European journal of clinical investigation
pub_type: 杂志文章
doi:10.1111/eci.13022
更新日期:2018-11-01 00:00:00
abstract:BACKGROUND:Buerger's disease (BD) is a segmental occlusive vascular disease. The aim of this study was to detect functional changes in brachial artery and asymptomatic morphological changes in extra-cranial carotid arteries not affected by the disease process and to assess markers of inflammation and endothelial damage...
journal_title:European journal of clinical investigation
pub_type: 杂志文章
doi:10.1111/j.1365-2362.2006.01646.x
更新日期:2006-06-01 00:00:00
abstract::The electrical parameters and the unidirectional fluxes of 45Ca and 3H-mannitol were measured in preparations of rat colon descendens freed from the muscularis externa and mounted in a modified Ussing-chamber. Two criteria were used to differentiate between changes in the trans- and the paracellular calcium transport ...
journal_title:European journal of clinical investigation
pub_type: 杂志文章
doi:10.1111/j.1365-2362.1986.tb01007.x
更新日期:1986-10-01 00:00:00
abstract:BACKGROUND:Nowadays there is an increased interest in the role of aortic stiffness in the pathophysiology of heart failure (HF), as it is a major determinant of left ventricular (LV) performance. We aimed at assessing the predictive value of the aortic stiffness parameters, measured by echocardiography, in patients aff...
journal_title:European journal of clinical investigation
pub_type: 杂志文章
doi:10.1111/eci.12950
更新日期:2018-07-01 00:00:00
abstract::The impact of running and menstrual disturbances on regional and total body fat distribution and serum lipids was investigated in 205 women. Body composition was measured by dual-energy X-ray absorptiometry. The total fat mass in the elite runners was approximately half of the normally active's (7.3 [0.48] kg vs. 14.3...
journal_title:European journal of clinical investigation
pub_type: 杂志文章
doi:10.1111/j.1365-2362.1995.tb01745.x
更新日期:1995-08-01 00:00:00
abstract::The relationship between changes in ketone concentrations and leucine metabolism (seven obese subjects), glucose and alanine metabolism (seven obese subjects) was investigated using radioisotopic techniques after 12 h, 60 h and 2 weeks starvation. Leucine metabolism was also measured in five lean subjects after 12 h a...
journal_title:European journal of clinical investigation
pub_type: 杂志文章
doi:10.1111/j.1365-2362.1995.tb01755.x
更新日期:1995-08-01 00:00:00
abstract::The effect of renal function on the cyclic AMP (cAMP) response to exogenous parathyroid hormone (PTH) was examined in patients with chronic renal failure (n = 22) and primary hyperparathyroidism (n = 19). In the patients with chronic renal failure there was marked resistance to the effect of exogenous PTH. In primary ...
journal_title:European journal of clinical investigation
pub_type: 杂志文章
doi:10.1111/j.1365-2362.1985.tb00141.x
更新日期:1985-02-01 00:00:00
abstract::Urine was collected in four healthy infants, born after 35-39 weeks of gestation. The collections were made for 24 h starting immediately after birth. Bile acids were extracted, separated into conjugate groups, solvolysed, hydrolysed and quantified by gas-liquid chromatography as methyl ester trimethylsilyl ether deri...
journal_title:European journal of clinical investigation
pub_type: 杂志文章
doi:10.1111/j.1365-2362.1982.tb02236.x
更新日期:1982-08-01 00:00:00
abstract:BACKGROUND:Humming greatly increases nasal nitric oxide (NO) in healthy people by causing a rapid washout of NO from the sinuses. This increase is abolished in patients with complete sinus ostial obstruction. OBJECTIVE:Allergic rhinitis is a risk factor for development of sinusitis and we wanted to study whether nasal...
journal_title:European journal of clinical investigation
pub_type: 杂志文章
doi:10.1111/j.1365-2362.2004.01384.x
更新日期:2004-08-01 00:00:00
abstract:BACKGROUND:Cardiovascular patients suffer from reduced blood flow leading to ischaemia and impaired tissue metabolism. Unfortunately, an increasing group of elderly patients cannot be treated with current revascularization methods. Thus, new treatment strategies are urgently needed. Hypoxia-inducible factors (HIFs) upr...
journal_title:European journal of clinical investigation
pub_type: 杂志文章
doi:10.1111/eci.12333
更新日期:2014-10-01 00:00:00
abstract::Renal extraction and renal plasma clearance of atrial natriuretic peptide from pigs with complete unilateral ureteral obstruction (UUO) and from intact anaesthetized pigs were determined from arteriovenous differences in plasma atrial natriuretic peptide and measured renal plasma flow. The effect of administration of ...
journal_title:European journal of clinical investigation
pub_type: 杂志文章
doi:10.1111/j.1365-2362.1995.tb01556.x
更新日期:1995-04-01 00:00:00
abstract::We measured plasma sulphur amino acids in twenty-two patients with chronic renal failure and compared the findings with those obtained in twenty-two normal subjects. In fasting blood (08.00 hours) cysteine-homocysteine mixed disulphide was significantly increased in the renal patients, mean values (+/- SD) being 8.2 +...
journal_title:European journal of clinical investigation
pub_type: 杂志文章
doi:10.1111/j.1365-2362.1979.tb00888.x
更新日期:1979-08-01 00:00:00
abstract:BACKGROUND:Plasma concentrations of vitamins A and E are positively correlated with those of concurrent lipids and, on the other hand, lipid levels are influenced by apolipoprotein E polymorphism. Therefore, the effect of this polymorphism on both vitamins was analysed in an adult population. MATERIALS AND METHODS:Sub...
journal_title:European journal of clinical investigation
pub_type: 杂志文章
doi:10.1046/j.1365-2362.2002.00983.x
更新日期:2002-04-01 00:00:00
abstract::Plasma glucose concentration is the best predictor for the development of non-insulin-dependent diabetes mellitus (NIDDM). However, obesity is also a recognized risk factor for development of the disease, and is easier to track over time. Thus obesity could be of considerable clinical importance as a predictor of diab...
journal_title:European journal of clinical investigation
pub_type: 杂志文章
doi:10.1046/j.1365-2362.1998.0280s2003.x
更新日期:1998-09-01 00:00:00
abstract::Disseminated intravascular coagulation (DIC) is a frequently occurring complication of sepsis and may contribute to multiple organ failure. More insight into the pathogenesis of this derangement of the coagulation system is necessary to develop more effective therapeutic strategies for this condition. Recently, more d...
journal_title:European journal of clinical investigation
pub_type: 杂志文章,评审
doi:10.1046/j.1365-2362.1997.570614.x
更新日期:1997-01-01 00:00:00
abstract:BACKGROUND:We hypothesized the existence of different hormonal response patterns to repeated lower body negative pressure (LBNP) and head-up tilt (HUT) in healthy males. We compared hormonal, cardiovascular and plasma volume changes from rest to stress within- and between-LBNP and HUT applications. Hormones investigate...
journal_title:European journal of clinical investigation
pub_type: 杂志文章
doi:10.1111/j.1365-2362.2011.02476.x
更新日期:2011-08-01 00:00:00
abstract:BACKGROUND:Little is known about gallbladder motility in patients with black pigment stones when compared to cholesterol gallstone patients, or about their relationship to biliary composition, crystallization and stone characteristics. DESIGN:Fasting and postprandial gallbladder volumes were studied by ultrasonography...
journal_title:European journal of clinical investigation
pub_type: 杂志文章
doi:10.1046/j.1365-2362.2000.00639.x
更新日期:2000-04-01 00:00:00
abstract::The ATP-binding cassette transporter P-glycoprotein is now recognized as an important determinant for disposition of multiple drugs. The use of P-glycoprotein-expressing cell lines, the generation of P-glycoprotein knockout mice as well as studies in animals and humans contributed to a better understanding on the role...
journal_title:European journal of clinical investigation
pub_type: 杂志文章,评审
doi:10.1046/j.1365-2362.33.s2.4.x
更新日期:2003-11-01 00:00:00
abstract::The venous system plays a pivotal role in volume and blood pressure homeostasis. We tested the hypothesis that the visco-elastic properties of the peripheral venous system are reduced in patients with (incipient) diabetic nephropathy. Twenty-two normotensive patients with long-term insulin-dependent diabetes mellitus ...
journal_title:European journal of clinical investigation
pub_type: 临床试验,杂志文章
doi:10.1111/j.1365-2362.1994.tb02180.x
更新日期:1994-06-01 00:00:00
abstract:BACKGROUND:Soluble ST2 (sST2), a member of the IL-1 receptor family, has been proposed as a novel biomarker with predictive value for heart failure and mortality in patients suffering from cardiovascular diseases. The influence of clinical characteristics on variability of sST2 levels is relatively unexplored. Here, we...
journal_title:European journal of clinical investigation
pub_type: 杂志文章
doi:10.1111/eci.12022
更新日期:2013-02-01 00:00:00
abstract:BACKGROUND:Angiotensin converting enzyme 2 (ACE2), a monocarboxylase that degrades angiotensin II to angiotensin 1-7, is also the functional receptor for severe acute respiratory syndrome (SARS) coronavirus (SARS-CoV) and is highly expressed in the lungs and heart. Patients with SARS also suffered from cardiac disease ...
journal_title:European journal of clinical investigation
pub_type: 杂志文章
doi:10.1111/j.1365-2362.2009.02153.x
更新日期:2009-07-01 00:00:00
abstract:BACKGROUND:Graves' disease (GD) is an autoimmune disorder characterized by hyperthyroidism, which can relapse in many patients after antithyroid drug treatment withdrawal. Several studies have been performed to predict the clinical course of GD in patients treated with antithyroid drugs, without conclusive results. The...
journal_title:European journal of clinical investigation
pub_type: 杂志文章
doi:10.1111/j.1365-2362.2004.01318.x
更新日期:2004-03-01 00:00:00
abstract::The aim of the present study was to investigate possible relationships in piglets between myocardial energy-related metabolites and intracellular electrolytes during open-chest cardiopulmonary resuscitation (OCCPR) supplemented by the administration of alkaline buffers with varying sodium content. Our hypothesis was t...
journal_title:European journal of clinical investigation
pub_type: 杂志文章
doi:10.1046/j.1365-2362.1997.1280676.x
更新日期:1997-05-01 00:00:00
abstract:BACKGROUND:After the Q fever outbreak in the Netherlands between 2007 and 2010, more than 300 patients with chronic Q fever have been identified. Some patients were also diagnosed with systemic sclerosis, a rare immune-mediated disease. We aimed to increase awareness of concomitant chronic Q fever infection and systemi...
journal_title:European journal of clinical investigation
pub_type: 杂志文章
doi:10.1111/eci.13123
更新日期:2019-07-01 00:00:00
abstract::Prostacyclin, an unstable metabolite of arachidonic acid metabolism, has been shown to have potent platelet antiaggregatory and vasodilator activity. Available evidence suggests that arterial prostacyclin production is diminished in the diabetic state. In this study we have examined the sensitivity of platelets from d...
journal_title:European journal of clinical investigation
pub_type: 杂志文章
doi:10.1111/j.1365-2362.1982.tb00686.x
更新日期:1982-10-01 00:00:00
abstract::Adequate pregnancy care of women with systemic lupus erythematosus (SLE) rests on three pillars: a coordinated medical-obstetrical care, an agreed and well-defined management protocol and a good neonatal unit. Pregnancy should be planned following a preconceptional visit for counselling. Women with severe active disea...
journal_title:European journal of clinical investigation
pub_type: 杂志文章,评审
doi:10.1111/j.1365-2362.2010.02443.x
更新日期:2011-06-01 00:00:00
abstract:BACKGROUND:Variation of percentageCD4+ T cells may accurately reflects the kinetics of a comprehensive immune recovery independently of the antiretroviral (ART) regimen. To test this we have investigated the relationship among peripheral blood T-cell subsets with the variation of percentageCD4+ T cells during follow up...
journal_title:European journal of clinical investigation
pub_type: 杂志文章
doi:10.1046/j.1365-2362.2003.01168.x
更新日期:2003-07-01 00:00:00
abstract:BACKGROUND:Renal impairment and type 2 diabetes mellitus (DM) are well-known independent risk factors for mortality. The evidence of their combined effects on mortality is unclear, but of importance because it may determine aggressiveness of treatment. This study sought to assess and quantify the effect modification of...
journal_title:European journal of clinical investigation
pub_type: 杂志文章
doi:10.1111/eci.12035
更新日期:2013-02-01 00:00:00
abstract:BACKGROUND:Tumour necrosis factor alpha (TNF) mRNA expression has been reported to be up-regulated in adipose tissue from several rodent models of obesity and diabetes and from obese humans. This elevated expression has been assumed to be associated with the development of insulin resistance. However, the biological si...
journal_title:European journal of clinical investigation
pub_type: 杂志文章
doi:10.1046/j.1365-2362.1999.00520.x
更新日期:1999-08-01 00:00:00