However, there is a lack of evidence-based recommendations for the use of prophylaxis in adults. “
“von Willebrand disease
(VWD) is the most common inherited bleeding disorder and is due to a deficiency and/or abnormality of von Willebrand factor (VWF), the high-molecular-weight glycoprotein that plays a major role in the early phases of hemostasis. VWD is inherited by autosomal dominant or recessive pattern, but women with milder VWD forms are apparently more selleck chemicals symptomatic. VWD is also very heterogeneous disorder and therefore patients with mild VWD forms are sometimes under- and misdiagnosed, due to physiologic changes of VWF within the same individual and to the relative high variability of diagnostic tests. Three main criteria are required for correct diagnoses of VWD: (1) positive bleeding history since childhood; (2) reduced
Crizotinib in vitro VWF activity in plasma; and (3) history of bleeding in the family. The bleeding score (BS) calculated following a detailed questionnaire devised to quantify symptoms was useful to confirm the diagnosis of VWD1. BS together with baseline VWF levels and family history have been proposed as more evidence-based criteria for VWD1. More recently, the use of BS and threshold levels of VWF activity have been investigated in a prospective study to predict clinical outcome and the need of therapy with desmopressin and/or VWF concentrates in a large cohort of patients with different VWD types. “
“Summary. MCE MC710, a combined product of plasma-derived activated factor VII (FVIIa) and factor X (FX) at a
protein weight ratio of 1:10, is a novel bypassing agent for haemostasis in haemophilia patients with inhibitors. In this study, pharmacokinetic (PK), pharmacodynamic (PD) parameters and safety of single doses of MC710 were investigated in 11 male haemophilia patients with inhibitors in a non-bleeding state. This was a multi-centre, open-labelled, non-randomized, active controlled crossover, dose-escalation study of five doses (20–120 μg kg−1 of FVIIa) with re-administration of different MC710 dosages to the same subjects. The active controls were NovoSeven (120 μg kg−1) and/or FEIBA (50 and 75 U kg−1) which were used to compare PD parameters. The area under the curve (AUC) and maximum plasma concentration (Cmax) of MC710 active ingredients increased dose-dependently within the range of 20 and 120 μg kg−1. After administration of MC710, activated partial thromboplastin time (APTT) was dose-dependently improved and prothrombin time (PT) was shortened to approximately 6 s at 10 min, and APTT improvement and PT shortening effects were maintained until 12 h after administration of MC710 at all doses.