title: An ultrapure plasma-derived monoclonal antibody-purified factor IX concentrate (Nonafact®), results of phase III and IV clinical studies.
abstract: Nonafact(®), an ultrapure, monoclonal antibody-purified factor IX concentrate (FIX) was developed to minimize risk of thrombotic complications and viral transmission. To investigate the pharmacokinetics, efficacy and safety, phase III/IV studies were performed in the Netherlands and Poland from 1996 to 2007. The mean half-life, in vivo response and recovery of Nonafact(®) were 18.7 (SD 2.0) h, 1.1 (SD 0.2) IU dL(-1) per IU kg(-1) b.w. of FIX infused and 49% (SD 10%), respectively. Eleven surgical procedures were performed in eight patients. During two surgeries, both high-risk, blood loss was observed. No postoperative bleeding occurred. The in vivo recovery of FIX was higher than expected. In the phase III follow-up study, 26 previously treated patients (PTP) were included with a median follow-up of 1130 days. From the 1617 minor bleedings, 80.5% was stopped after a single infusion. In the phase IV study thirteen patients were treated for a median study period of 737 days. In the two follow-up studies the investigators rated the effect of Nonafact(®) as excellent/good in 95% of major bleedings. Surgeries for which Nonafact(®) was given prophylactically were without bleeding problems. In total more than 10 million units of Nonafact(®) were used during almost 120 person-years. Only one minor adverse event was reported. No inhibitors, viral transmissions and thrombogenic events occurred. In conclusion, Nonafact(®) is safe and provides excellent haemostasis in haemophilia B patients treated for spontaneous bleeding or undergoing surgical procedures. Due to the excellent in vivo recovery characteristic, treatment with Nonafact(®) is cost saving compared to other FIX products.
authors.affiliation: Van Creveldkliniek and Hematology, University Medical Centre Utrecht, Utrecht, The Netherlands.
authors.forename: E P
authors.initials: EP
authors.lastname: Mauser-Bunschoten
authors.forename: I
authors.initials: I
authors.lastname: Kleine Budde
authors.forename: S
authors.initials: S
authors.lastname: Lopaciuk
authors.forename: M M W
authors.initials: MM
authors.lastname: Koopman
authors.forename: F J M
authors.initials: FJ
authors.lastname: Van Der Meer
authors.forename: I R O
authors.initials: IR
authors.lastname: Novàkovà
authors.forename: P
authors.initials: P
authors.lastname: Ypma
authors.forename: P W G
authors.initials: PW
authors.lastname: Van Der Linden
authors.forename: J
authors.initials: J
authors.lastname: Windyga
authors.forename: P F W
authors.initials: PF
authors.lastname: Strengers
mesh_terms: D000293:Adolescent; D000328:Adult; D000911:Antibodies, Monoclonal / Q000493:pharmacokinetics / Q000627:therapeutic use*; D016063:Blood Loss, Surgical / Q000517:prevention & control; D005164:Factor IX / Q000493:pharmacokinetics / Q000627:therapeutic use*; D005500:Follow-Up Studies; D002836:Hemophilia B / Q000188:drug therapy* / Q000601:surgery; D006488:Hemostasis, Surgical / Q000379:methods; D006801:Humans; D008875:Middle Aged; D009426:Netherlands; D011044:Poland; D019106:Postoperative Hemorrhage / Q000517:prevention & control; D055815:Young Adult
chemical_list: D000911:Antibodies, Monoclonal; D005164:Factor IX