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. Nonacog alfa, a recombinant factor IX (FIX) used for the treatment of haemophilia B, was approved in Europe in 1998. In accordance with European Medicines Agency requirements, a registry study was conducted from 2002 to 2009. A reformulated iso-osmotic version was approved for European use in 2007. This study was conducted to evaluate the safety of nonacog alfa in a usual care setting, and provide clinical trial and postmarketing surveillance data support. This open-label, non-interventional, prospective observational cohort study (registry) comprised 52 sites in nine European countries. Patients with haemophilia B receiving nonacog alfa in either formulation for prevention or treatment were followed on a usual care schedule. A total of 218 patients were enrolled, of whom 66 (30.3%) were <18 years of age. Haemophilia severity was evenly distributed, with baseline FIX activity of <1%, 1-5% and >5% in 33.3%, 36.6% and 30.1% of patients, respectively. One hundred thirty-eight patients received the original formulation alone; 80 switched to or received only the new formulation. There was a low incidence of events of special interest (ESIs), with less-than-expected therapeutic effect in five patients (2.2%), inhibitor development in two (0.9%), thrombosis in one (0.5%) and allergic events in eight (3.7%). These accounted for the majority of the 15 serious AEs reported in six patients. Six patients discontinued because of AEs, primarily related to hypersensitivity. Nonacog alfa was shown to be safe for the treatment of haemophilia B, with a low incidence of serious AEs and ESIs. [ABSTRACT FROM AUTHOR]

KOGENATE® Bayer (rFVIII-FS) with Bio-Set® is designed to prevent patient contact with exposed needles during recombinant factor VIII reconstitution. This postmarketing surveillance study evaluated patient satisfaction before and after switching to the new Bio-Set reconstitution method. Male children and adults with haemophilia A were enrolled from nine European countries. A preference questionnaire was administered to patients after Bio-Set training and at the end of the observation period (≥20 exposure days or 3 months). Physician assessments of patient compliance and satisfaction were conducted at the end of the observation period. Patients ( N = 306) received a mean ± SD of 28 ± 23 infusions of rFVIII-FS with Bio-Set. A majority of patients (82%) preferred the Bio-Set method, with domain scores for ease of use, safety from needlesticks, and speed of reconstitution being highest after training and at the end of the observation period. The Bio-Set method received higher mean scores than previous reconstitution methods for worry/safety and ease/confidence domains at both time points. Physician-reported patient compliance with the Bio-Set method was similar or greater compared with the previous method for 94% of the patients, with physicians reporting that 92% of the patients were satisfied or very satisfied with Bio-Set. Thirteen adverse events (AEs) occurred in nine patients, and five serious AEs occurred in five patients; none was related to rFVIII-FS. No de novo or recurrent inhibitor development was observed during the observation period. rFVIII-FS with Bio-Set was well tolerated and well accepted by haemophilia A patients, which may improve treatment compliance. [ABSTRACT FROM AUTHOR]

Using a patient chart review process, we conducted a retrospective study to describe the frequency of allergic reactions in individuals with haemophilia B receiving factor IX (FIX) replacement therapy. The number of allergic reactions in individuals receiving a recombinant FIX (rFIX) product (BeneFix(®)) was then compared with the number of reactions in patients receiving plasma-derived FIX (pdFIX) products. Of the 180 subjects in the study, 163 received rFIX, 88 received pdFIX; 71 received both product types. A total of seven (3.89%) subjects had a moderate or severe allergic reaction to a FIX product (95% confidence interval [CI], 1.06-6.71%). Among those receiving rFIX, four subjects (2.45%) had an allergic reaction (95% CI, 0.08-4.83%). Of individuals taking pdFIX products, three (3.41%) developed an allergic reaction (95% CI, 0-7.20%). It was noted that three (1.84%) of those taking rFIX developed an inhibitor to FIX (95% CI, 0-3.90%), while four (4.55%) of those receiving a pdFIX product developed an inhibitor (95% CI, 0.19-8.90%). Inhibitor development was frequently associated with allergic reaction. These results provide evidence that there is no difference in the frequency of allergic reactions or inhibitor development in individuals receiving rFIX compared with those receiving pdFIX concentrates. The current study and a previous study of similar design have now compared the rate of allergic reactions associated with rFIX and pdFIX concentrates has now been compared in a total of 414 subjects; this represents the largest collection of data to date on this rare complication of haemophilia B therapy., (© 2011 Blackwell Publishing Ltd.)