0000000000273693
AUTHOR
Ulrike Nowak-göttl
Final Evaluation Of a Prospective, Non-Interventional Study On Efficacy and Tolerability Of a New Generation VWF/FVIII Concentrate In The Treatment Of Von Willebrand Disease
Background With marketing authorization in 2005, a non-interventional study (SET = Surveillance of Efficacy and Tolerability) with a double virus inactivated VWF/FVIII concentrate (Wilate®) was initiated in Germany. In 2012, the inclusion of patient documentation was terminated for final evaluation of the study data, representing the treatment of 170 patients suffering from von Willebrand’s disease (VWD). Aim The presented study was performed to assess the haemostatic efficacy and safety of a newly introduced VWF/FVIII product in the treatment of all types of VWD patients in every day clinical setting and to validate the results from pivotal clinical trials. Methods Patients of any age suf…
Age-dependent control of collagen-dependent platelet responses by thrombospondin-1 : Comparative analysis of platelets from neonates, children, adolescents, and adults
Platelet function is developmentally regulated. Healthy neonates do not spontaneously bleed, but their platelets are hypo-reactive to several agonists. The mechanisms underlying immature platelet function in neonates are incompletely understood. This critical issue remains challenging for the establishment of age-specific reference ranges. In this study, we evaluated platelet reactivity of five pediatric age categories, ranging from healthy full-term neonates up to adolescents (11–18 years) in comparison to healthy adults (>
Pharmacokinetics of Direct Oral Anticoagulants in Emergency Situations: Results of the Prospective Observational RADOA-Registry
Thrombosis and haemostasis 122(4), 552-559 (2022). doi:10.1055/a-1549-6556
Results of a Prospective, Open-Label Trial to Assess the Efficacy, Safety and Immunogenicity of Wilate® in Children Under 6 Years of Age with Inherited Von Willebrand Disease,
Abstract Abstract 3330 Introduction: Wilate® is a new-generation plasma-derived concentrate of von Willebrand factor (VWF) and factor VIII (FVIII) developed for treatment of patients with von Willebrand disease (VWD) and haemophilia A. The objective of this study was to assess efficacy, safety and immunogenicity of Wilate in children below 6 years of age with inherited VWD. Methods: The prospective, multi-centre trial included patients with inherited VWD of any type below 6 years of age with insufficient response to DDAVP. Any clinical event requiring VWF/FVIII concentrate treatment within 1 year was treated with Wilate. Efficacy was recorded by using a 4-point VRS and in vivo recovery of F…