Search results for "Replacement therapy"
showing 10 items of 289 documents
Optimizing ceftolozane-tazobactam dosage during continuous renal replacement therapy: additional insights
2019
Influence of long-term postmenopausal hormone-replacement therapy on estimated structural bone strength: A study in discordant monozygotic twins
2010
Although postmenopausal hormone-replacement therapy (HRT) is known to prevent fractures, knowledge on the influence of long-term HRT on bone strength and its determinants other than areal bone mineral density is scarce. This study used a genetically controlled design with 24 monozygotic female twin pairs aged 54 to 72 years in which one cotwin was using HRT (mean duration 8 years) and the other had never used HRT. Estimated bone strength, cross-sectional area, volumetric bone mineral density, bone mineral mass, and cross-sectional density and mass distributions were assessed in the tibial shaft, distal tibia, and distal radius with peripheral computed tomography (pQCT). In the tibial shaft,…
Diagnostic efficacy of the fluorometric determination of enzyme activity for Pompe disease from dried blood specimens compared with lymphocytes-possi…
2009
Pompe disease is a rare, autosomal-recessive disorder which results from a defect in the lysosomal enzyme acid alpha-glucosidase (GAA). The onset of this disease is highly variable, with infantile types being the most severe. Traditionally, lymphocytes, fibroblasts or muscle biopsies were necessary for enzyme activity measurement, because these materials do not express maltase-glucoamylase (MGA) that interferes with the assay. Recently, acarbose was found to inhibit MGA activity selectively, so that dried blood became accessible for GAA assessment.To evaluate the diagnostic efficacy of GAA measurement in dried blood specimens (DBSs) in comparison with lymphocytes. If DBSs provided reliable …
Safety of agalsidase alfa in patients with Fabry disease under 7 years
2011
Aim: To evaluate the safety and explore the efficacy of enzyme replacement therapy (ERT) for Fabry disease with agalsidase alfa in young children enrolled in the Fabry Outcome Survey (FOS). Methods: This retrospective chart review identified eight children (mean age = 5.0 ± 1.6 [mean ± SD]) in FOS who began treatment with agalsidase alfa (0.2 mg/kg, i.v., every other week) when <7 years old. Vital signs and adverse events were monitored throughout the study period. Glomerular filtration rate (GFR) was estimated, and left ventricular mass indexed to height2.7 (LVMi) was assessed with echocardiography. Patients received 1.2–6.7 years of treatment (mean = 4.2 years). Results: Infusion react…
Long-term, open-labeled extension study of idursulfase in the treatment of Hunter syndrome.
2011
Purpose: This study evaluated the safety and effectiveness of long-term enzyme replacement therapy with idursulfase (recombinant human iduronate-2-sulfatase) in patients with Hunter syndrome. Methods: All 94 patients who completed a 53-week double-blinded study of idursulfase enrolled in this open-labeled extension study and received intravenous idursulfase at a dose of 0.5 mg/kg weekly for 2 years, and clinical outcomes and safety were assessed. Results: No change in percent predicted forced vital capacity was seen, but absolute forced vital capacity demonstrated sustained improvement and was increased 25.1% at the end of the study. Statistically significant increases in 6-minute walking t…
Enzyme replacement therapy in Fabry disease: Comparison of agalsidase alfa and agalsidase beta
2008
The Heart in Fabry Disease – from Pathogenesis to Enzyme Replacement Therapy
2010
The cardiovascular involvement in Fabry disease is progressive, and accounts for one of the major reasons for abbreviated life expectancy and increased morbidity. The majority of patients develop signs and symptoms, related to heart failure and arrhythmias. Hypertrophic cardiomyopathy, in later stages combined with myocardial fibrosis, is one of the leading features. Both genders are affected, and females develop severe cardiac Fabry disease approximately 10–15 years later in life than male patients. Diastolic dysfunction and reduced longitudinal mid-wall related systolic function are the mechanical consequences of the myocardial and epithelial changes related to accumulation of the storage…
Long-term enzyme-replacement therapy (ERT) with alglucosidase alfa: Evolution of two siblings with juvenile late-onset Pompe disease
2015
Symptomatic and Ancillary Therapy
2010
Although enzyme replacement therapy has had a considerable impact on the management of patients with Fabry disease, it is essential that attention is also given to supportive therapy. In this chapter a general overview about ancillary therapy is given. The great variability of clinical symptoms faced by patients with Fabry disease need the involvement from many different specialists, as well as a wide range of concomitant treatments. Coordinating a good multidisciplinary approach for each patient and ensuring that treatment is made as convenient as possible may offer a more positive impact on the quality of life of those patients affected by Fabry disease.