However, based on current knowledge, it cannot be excluded that antibody responses against proteins such as FVIII could also occur in a T-cell independent way. Such antibodies should be of low affinity due to the lack of affinity maturation. “
“Summary.  Effective treatment with factor IX (FIX) requires a thorough consideration of the properties of the concentrate to be used as replacement therapy, to date, the only available treatment for haemophilia B. The aim of the study was to determine the pharmacokinetics, clinical efficacy and safety in routine clinical use of AlphaNine®, a high-purity human FIX concentrate. This open,

single-arm, multicentre, non-randomized trial included 25 subjects (age ≥ 12) with moderate/severe haemophilia B. Pharmacokinetics was assessed at baseline and after a 6-month follow-up. The degree of haemostasis control DAPT clinical trial achieved was evaluated during a 12-month follow-up. Safety was evaluated in terms of tolerance, thrombogenicity, immunogenicity and viral safety. Mean recovery was 1.01 ± 0.19 IU dL−1 per IU kg−1 at baseline and 1.23 ± 0.34 IU dL−1 per IU kg−1

6 months later. Terminal half-life was 34.5 ± 6.2 h and 33.7 ± 5.4 h, selleck inhibitor respectively. Ratios of each parameter between the two pharmacokinetic studies were all close to 1. A total of 1,576,890 IU AlphaNine® were administered in 889 infusions (mean dose per infusion: 1774 IU; 3.2 infusions per month per patient). The main reasons for infusion were mild/moderate bleeding (62.3%) and prophylaxis (20.5% continuous, 15.6% intermittent). Overall, 93.0% of the efficacy assessments were rated

as excellent/good and 88.8% of bleedings resolved after the first infusion. Twenty-one adverse events were reported in eight patients, none of which was considered related to the study medication. AlphaNine® showed a pharmacokinetic profile Roflumilast in agreement with that of other plasma-derived FIX concentrates and provides safe and clinically effective substitution therapy for patients with haemophilia B. “
“The temporary correction of the coagulation defect is the mainstay of treatment in hemophilia. However, the “ideal” dose of factor VIII (FVIII) or factor IX (FIX) that needs to be administered to invariably achieve hemostasis without “overtreating” is unknown. Dosing for hemophilia has been studied since the 1940s when initial data was based on the use of plasma in hemophilic dogs. These observations were fundamental for the understanding of dosing in prophylaxis. Years later, the use of plasma in hemophilia patients was instated followed by cryoprecipitate and a fraction of human plasma in the 1960s.