TY - JOUR A1 - Biegstraaten, Marieke A1 - Arngrímsson, Reynir A1 - Barbey, Frederic A1 - Boks, Lut A1 - Cecchi, Franco A1 - Deegan, Patrick B A1 - Feldt-Rasmussen, Ulla A1 - Geberhiwot, Tarekegn A1 - Germain, Dominique P A1 - Hendriksz, Chris A1 - Hughes, Derralynn A A1 - Kantola, Ilkka A1 - Karabul, Nesrin A1 - Lavery, Christine A1 - Linthorst, Gabor E A1 - Mehta, Atul A1 - van de Mheen, Erica A1 - Oliveira, João P A1 - Parini, Rossella A1 - Ramaswami, Uma A1 - Rudnicki, Michael A1 - Serra, Andreas A1 - Sommer, Claudia A1 - Sunder-Plassmann, Gere A1 - Svarstad, Einar A1 - Sweeb, Annelies A1 - Terryn, Wim A1 - Tylki-Szymanska, Anna A1 - Tøndel, Camilla A1 - Vujkovac, Bojan A1 - Weidemann, Frank A1 - Wijburg, Frits A A1 - Woolfson, Peter A1 - Hollak, Carla EM T1 - Recommendations for initiation and cessation of enzyme replacement therapy in patients with Fabry disease: the European Fabry Working Group consensus document JF - Orphanet Journal of Rare Diseases N2 - Introduction: Fabry disease (FD) is a lysosomal storage disorder resulting in progressive nervous system, kidney and heart disease. Enzyme replacement therapy (ERT) may halt or attenuate disease progression. Since administration is burdensome and expensive, appropriate use is mandatory. We aimed to define European consensus recommendations for the initiation and cessation of ERT in patients with FD. Methods: A Delphi procedure was conducted with an online survey (n = 28) and a meeting (n = 15). Patient organization representatives were present at the meeting to give their views. Recommendations were accepted with ≥75% agreement and no disagreement. Results: For classically affected males, consensus was achieved that ERT is recommended as soon as there are early clinical signs of kidney, heart or brain involvement, but may be considered in patients of ≥16 years in the absence of clinical signs or symptoms of organ involvement. Classically affected females and males with non-classical FD should be treated as soon as there are early clinical signs of kidney, heart or brain involvement, while treatment may be considered in females with non-classical FD with early clinical signs that are considered to be due to FD. Consensus was achieved that treatment should not be withheld from patients with severe renal insufficiency (GFR < 45 ml/min/1.73 m\(^{2}\)) and from those on dialysis or with cognitive decline, but carefully considered on an individual basis. Stopping ERT may be considered in patients with end stage FD or other co-morbidities, leading to a life expectancy of <1 year. In those with cognitive decline of any cause, or lack of response for 1 year when the sole indication for ERT is neuropathic pain, stopping ERT may be considered. Also, in patients with end stage renal disease, without an option for renal transplantation, in combination with advanced heart failure (NYHA class IV), cessation of ERT should be considered. ERT in patients who are non-compliant or fail to attend regularly at visits should be stopped. Conclusion: The recommendations can be used as a benchmark for initiation and cessation of ERT, although final decisions should be made on an individual basis. Future collaborative efforts are needed for optimization of these recommendations. KW - Fabry disease KW - enzyme replacement therapy KW - recommendations KW - Delphi procedure Y1 - 2015 U6 - http://nbn-resolving.de/urn/resolver.pl?urn:nbn:de:bvb:20-opus-175374 VL - 10 IS - 36 ER - TY - JOUR A1 - Sherif, Mohammad A. A1 - Herold, Joerg A1 - Voelker, Wolfram A1 - Maniuc, Octavian A1 - Ertl, Georg A1 - Praast, Christian A1 - Braun-Dullaeus, Ruediger Christian T1 - Feasibility of a new method using two-dimensional transesophageal echocardiography for aortic annular sizing in patients undergoing transcatheter aortic valve implantation; a case-control study JF - BMC Cardiovascular Disorders N2 - Background: Accurate preoperative assessment of the aortic annulus dimension is crucial for successful transcatheter aortic valve implantation (TAVI). In this study we validated a new method using two-dimensional transesophageal echocardiography (2D-TEE) for measurement of the aortic annulus prior to TAVI. Methods: We analysed 124 patients who underwent successful TAVI using a self-expandable prosthesis, divided equally into two groups; in the study group we used the cross sectional short axis 2D-TEE for measurement of the aortic annulus and in the control group we used the long axis 2D-TEE. Results: Both groups were comparable regarding the clinical parameters. On the other hand, patients in the study group had less left ventricular ejection fraction (38.9 % versus 45.6 %, p = 0.01). The aortic valve annulus was, although not statistically significant, smaller in the study group (21.58 versus 23.28 mm, p = 0.25). Post procedural quantification of the aortic regurgitation revealed that only one patient in both groups had severe aortic regurgitation (AR), in this patient the valve was implanted deep. The incidence of significant AR was higher in the control group (29.0 % versus 12.9 %, p = 0.027). Conclusions: Sizing of the aortic valve annulus using cross-sectional 2D-TEE offers a safe and plausible method for patients undergoing TAVI using the self-expandable prosthesis and is significantly superior to using long axis 2D-TEE. KW - paravalvular regurgitation KW - multicenter KW - management KW - sizing KW - echocardiography KW - replacement KW - tomography KW - guidelines KW - outcomes KW - impact KW - recommendations KW - stenosis KW - TAVI Y1 - 2015 U6 - http://nbn-resolving.de/urn/resolver.pl?urn:nbn:de:bvb:20-opus-148328 VL - 15 IS - 78 ER - TY - JOUR A1 - Rose, Markus A. A1 - Damm, Oliver A1 - Greiner, Wolfgang A1 - Knuf, Markus A1 - Wutzler, Peter A1 - Liese, Johannes G. A1 - Krüger, Hagen A1 - Wahn, Ulrich A1 - Schaberg, Tom A1 - Schwehm, Markus A1 - Kochmann, Thomas F. A1 - Eichner, Martin T1 - The epidemiological impact of childhood influenza vaccination using live-attenuated influenza vaccine (LAIV) in Germany: predictions of a simulation study JF - BMC Infectious Diseases N2 - Background: Routine annual influenza vaccination is primarily recommended for all persons aged 60 and above and for people with underlying chronic conditions in Germany. Other countries have already adopted additional childhood influenza immunisation programmes. The objective of this study is to determine the potential epidemiological impact of implementing paediatric influenza vaccination using intranasally administered live-attenuated influenza vaccine (LAIV) in Germany. Methods: A deterministic age-structured model is used to simulate the population-level impact of different vaccination strategies on the transmission dynamics of seasonal influenza in Germany. In our base-case analysis, we estimate the effects of adding a LAIV-based immunisation programme targeting children 2 to 17 years of age to the existing influenza vaccination policy. The data used in the model is based on published evidence complemented by expert opinion. Results: In our model, additional vaccination of children 2 to 17 years of age with LAIV leads to the prevention of 23.9 million influenza infections and nearly 16 million symptomatic influenza cases within 10 years. This reduction in burden of disease is not restricted to children. About one third of all adult cases can indirectly be prevented by LAIV immunisation of children. Conclusions: Our results demonstrate that vaccinating children 2-17 years of age is likely associated with a significant reduction in the burden of paediatric influenza. Furthermore, annual routine childhood vaccination against seasonal influenza is expected to decrease the incidence of influenza among adults and older people due to indirect effects of herd protection. In summary, our model provides data supporting the introduction of a paediatric influenza immunisation programme in Germany. KW - vaccination KW - live-attenuated influenza vaccine KW - children KW - transmission model KW - metanalysis KW - recommendations KW - hospitalizations KW - burden KW - infection KW - young children KW - seasonal influenza KW - United States KW - disease KW - efficacy KW - Germany KW - influenza Y1 - 2014 U6 - http://nbn-resolving.de/urn/resolver.pl?urn:nbn:de:bvb:20-opus-117563 SN - 1471-2334 VL - 14 IS - 40 ER -