title: A prospective registry of European haemophilia B patients receiving nonacog alfa, recombinant human factor IX, for usual use.
abstract: 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.
authors: Malmö Centre for Thrombosis and Haemostasis, Lund University, Skåne University Hospital, SE-205 02, Malmö, Sweden. erik.berntorp@med.lu.se
authors: E
authors: E
authors: Berntorp
authors: D
authors: D
authors: Keeling
authors: M
authors: M
authors: Makris
authors: A
authors: A
authors: Tagliaferri
authors: C
authors: C
authors: Male
authors: E P
authors: EP
authors: Mauser-Bunschoten
authors: R
authors: R
authors: Musso
authors: C A
authors: CA
authors: Roca
authors: A
authors: A
authors: Hassoun
authors: C
authors: C
authors: Kollmer
authors: R
authors: R
authors: Charnigo
authors: J
authors: J
authors: Baumann
authors: P
authors: P
authors: Rendo
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D002648:Child; D002675:Child, Preschool; D003029:Coagulants / Q000009:adverse effects / Q000627:therapeutic use*; D005060:Europe; D005164:Factor IX / Q000009:adverse effects / Q000627:therapeutic use*; D005260:Female; D002836:Hemophilia B / Q000188:drug therapy*; D006801:Humans; D007223:Infant; D008297:Male; D008875:Middle Aged; D011446:Prospective Studies; D011994:Recombinant Proteins / Q000009:adverse effects / Q000627:therapeutic use; D012042:Registries; D055815:Young Adult
chemical_list: D003029:Coagulants; D011994:Recombinant Proteins; D005164:Factor IX