A national French noninterventional study to assess the long-term safety and efficacy of reformulated nonacog alfa.
Nonacog alfa, the recombinant Factor IX (F IX) used for the treatment of hemophilia B, was approved in Europe in 1998. A reformulated version was approved for European use in 2007.



This postmarketing study, as recommended by the risk management plan, was conducted to confirm the safety of reformulated nonacog alfa in a usual care setting in France. This open-label, noninterventional, prospective, longitudinal postmarketing study comprised 19 French hemophilia centers. Patients with hemophilia B receiving reformulated nonacog alfa for prophylaxis or on-demand treatment were followed up on usual care schedule.



A total of 58 subjects were enrolled, of whom 29 (50%) were less than 18 years of age. Hemophilia was severe (baseline F IX activity < 1%) in 47 (81%) patients. All subjects except one were already treated with reformulated nonacog alfa before enrollment. One subject was receiving reformulated nonacog alfa as immune tolerance induction at time of enrollment. At enrollment, treatment regimen was mainly prophylactic in subjects less than 18 years and on-demand in subjects 18 years or older. Median duration of follow-up in the survey was 3.3 (2.3-3.8) years. The median annualized bleeding rate was 3.9 (1.5-5.2) for prophylaxis regimen and 12.2 (3.9-22.1) for on-demand regimen. One subject, a previously untreated patient, developed F IX inhibitors during follow-up. No allergic reaction, no blood cell agglutination, no lack of efficacy or recovery, and no thrombotic events were reported.



Reformulated nonacog alfa was shown to be safe in a usual care setting.
Haemophilia Care Centre, Bicêtre APHP University Hospital, Le Kremlin, Bicêtre, France.
Thierry
T
Lambert
Haemophilia Care Centre, Department of Haematology, Necker APHP University Hospital, Paris, France.
Chantal
C
Rothschild
Haemophilia Care Centre, Bocage University Hospital, Dijon, France.
Fabienne
F
Volot
Haemophilia Care Centre, Côte de Nacre University Hospital, Caen, France.
Annie
A
Borel-Derlon
Haemophilia Care Centre, Nantes University Hospital, Nantes, France.
Marc
M
Trossaërt
Haemophilia Care Centre, Purpan University Hospital, Toulouse, France.
Ségolène
S
Claeyssens-Donadel
Pfizer France SAS, Paris, France.
Sepideh
S
Attal
D000293:Adolescent; D000328:Adult; D000367:Age Factors; D019774:Blood Coagulation Factor Inhibitors / Q000097:blood*; D002648:Child; D002675:Child, Preschool; D005164:Factor IX / Q000008:administration & dosage* / Q000009:adverse effects; D005260:Female; D005500:Follow-Up Studies; D005602:France; D002836:Hemophilia B / Q000097:blood / Q000188:drug therapy; D006801:Humans; D008137:Longitudinal Studies; D008297:Male; D008875:Middle Aged; D011358:Product Surveillance, Postmarketing; D011446:Prospective Studies
D019774:Blood Coagulation Factor Inhibitors; D005164:Factor IX