title: Recombinant factor IX (BAX326) in previously treated paediatric patients with haemophilia B: a prospective clinical trial.
abstract: A newly developed recombinant factor IX (BAX326(1) ) was investigated for prophylactic use in paediatric patients aged <12 years with severe (FIX level <1%) or moderately severe (FIX level 1-2%) haemophilia B. The aim of this prospective clinical trial was to assess the safety, haemostatic efficacy and pharmacokinetic profile of BAX326 in previously treated paediatric patients. BAX326 was administered as prophylaxis twice a week for a period of 6 months, and on demand for treatment of bleeds. Safety was assessed by the occurrence of related AEs, thrombotic events and immunologic assessments. Efficacy was evaluated by annualized bleeding rate (ABR), and by treatment response rating (excellent, good, fair, none). PK was assessed over 72 h. None of the 23 treated paediatric subjects had treatment-related SAEs or AEs. There were no thrombotic events, inhibitory or specific binding antibodies against FIX, rFurin or CHO protein. Twenty-six bleeds (19 non-joint vs. 7 joint bleeds) occurred (mean ABR 2.7 ± 3.14, median 2.0), of which 23 were injury-related. Twenty subjects (87%) did not experience any bleeds of spontaneous aetiology. Haemostatic efficacy of BAX326 was excellent or good for >96% of bleeds (100% of minor, 88.9% of moderate and 100% of major bleeds); the majority (88.5%) resolved after 1-2 infusions. Longer T1/2 and lower IR were observed in younger children (<6 years) compared to those aged 6 to 12 years. BAX326 administered as prophylactic treatment as well as for controlling bleeds is efficacious and safe in paediatric patients aged <12 years with haemophilia B.
authors.affiliation: Department of Paediatrics, Paediatric Haematology and Oncology, Pomeranian Medical University in Szczecin, Szczecin, Poland.
authors.forename: T
authors.initials: T
authors.lastname: Urasinski
authors.affiliation: Institute of Blood Pathology and Transfusion Medicine of the Academy of Medical Sciences of Ukraine, Lviv, Ukraine.
authors.forename: O
authors.initials: O
authors.lastname: Stasyshyn
authors.affiliation: Republican Center for Haemophilia Treatment, St. Petersburg, Russia.
authors.forename: T
authors.initials: T
authors.lastname: Andreeva
authors.affiliation: Prof. Dr. C. T. Nicolau National Institute for Transfusional Hematology, Bucharest, Romania.
authors.forename: L
authors.initials: L
authors.lastname: Rusen
authors.affiliation: Regional Clinical Hospital, Ekaterinburg, Russia.
authors.forename: F G
authors.initials: FG
authors.lastname: Perina
authors.affiliation: Baxter BioScience, Westlake Village, CA, USA.
authors.forename: M S
authors.initials: MS
authors.lastname: Oh
authors.affiliation: Baxter BioScience, Vienna, Austria.
authors.forename: M
authors.initials: M
authors.lastname: Chapman
authors.affiliation: Baxter BioScience, Vienna, Austria.
authors.forename: B G
authors.initials: BG
authors.lastname: Pavlova
authors.affiliation: Baxter BioScience, Vienna, Austria.
authors.forename: B
authors.initials: B
authors.lastname: Valenta-Singer
authors.affiliation: Baxter BioScience, Vienna, Austria.
authors.forename: B E
authors.initials: BE
authors.lastname: Abbuehl
mesh_terms: D000293:Adolescent; D000367:Age Factors; D002648:Child; D002675:Child, Preschool; D005164:Factor IX / Q000494:pharmacology* / Q000627:therapeutic use*; D005260:Female; D002836:Hemophilia B / Q000097:blood / Q000150:complications / Q000188:drug therapy*; D006470:Hemorrhage / Q000188:drug therapy / Q000209:etiology / Q000517:prevention & control; D006801:Humans; D007223:Infant; D008297:Male; D011292:Premedication; D011994:Recombinant Proteins / Q000494:pharmacology* / Q000627:therapeutic use*; D019233:Retreatment; D013997:Time Factors; D016896:Treatment Outcome
chemical_list: D011994:Recombinant Proteins; D005164:Factor IX
keywords: haemophilia B; paediatric; previously treated patients; recombinant factor IX