BAX326 (recombinant coagulation factor IX) for the treatment and prophylaxis of hemophilia B

References

• National Hemophilia Foundation. Hemophilia B (factor IX). Available from: www.hemophilia.org/NHFWeb/MainPgs/MainNHF.aspx?menuid=181&contentid=46 [Last accessed 6 November 2013]
   Google Scholar
• World Federation of Hemophilia report on t he annual global survey 2010
   Google Scholar
• Franchini M, Frattini F, Crestani S, et al. Treatment of hemophilia B: focus on recombinant factor IX. Biologics 2013;7:33-8
   PubMedGoogle Scholar
• Bolton-Maggs PH, Pasi KJ, Haemophilias A and B. Lancet. 2003;361(9371):1801-9
   Google Scholar
• Tagariello G, Iorio A, Santagostino E, et al. Comparison of the rates of joint arthroplasty in patients with severe factor VIII and IX deficiency: an index of different clinical severity of the 2 coagulation disorders. Blood 2009;114(4):779-84
   PubMed Web of Science ®Google Scholar
• Lowe GD, Ludlam CA. Less severe bleeding in hemophilia B than in hemophilia A. J Thromb Haemost 2008;6(11):1982-3
   PubMed Web of Science ®Google Scholar
• Nagel K, Walker I, Decker K, et al. Comparing bleed frequency and factor concentrate use between haemophilia A and B patients. Haemophilia 2011;17(6):872-4
   PubMed Web of Science ®Google Scholar
• Mannucci PM, Franchini M. Is haemophilia B less severe than haemophilia A? Haemophilia 2013;19(4):499-502
   PubMed Web of Science ®Google Scholar
• Den Uijl IE, Roosendaal G, Fischer K. Insufficient evidence to suggest less stringent therapy in hemophilia B? Blood 2009;114(23):4907; author reply 4907-4908
   PubMed Web of Science ®Google Scholar
• Centers for Disease Control and Prevention. Summary Report of UDC Activity. National. Treatment/Clinical Characteristics (Hemophilia). https://www2a.cdc.gov/ncbddd/htcweb/UDC_Report/UDC_view1.asp?para1=NATION&para2=TREATH&para3=&ScreenWidth=1364&ScreenHeight=768 [Last] [accessed 25 October 2013]
   Google Scholar
• Dimichele D. The North American immune tolerance registry: contributions to the thirty-year experience with immune tolerance therapy. Haemophilia 2009;15(1):320-8
   PubMed Web of Science ®Google Scholar
• Franchini M, Frattini F, Crestani S, Bonfanti C. Haemophilia B: current pharmacotherapy and future directions. Expert Opin Pharmacother 2012;13(14):2053-63
   PubMed Web of Science ®Google Scholar
• Srivastava A, Brewer AK, Mauser-Bunschoten EP, et al. Guidelines for the management of hemophilia. Haemophilia 2013;19(1):e1-47
   PubMed Web of Science ®Google Scholar
• National Hemophilia Foundation. MASAC Recommendation Concerning Prophylaxis. MASAC Document #179. www.hemophilia.org/NHFWeb/Resource/StaticPages/menu0/menu5/menu57/masac179.pdf [Last accessed 11 December 2013]
   Google Scholar
• Panicker J, Warrier I, Thomas R, Lusher JM. The overall effectiveness of prophylaxis in severe haemophilia. Haemophilia 2003;9(3):272-8
   PubMed Web of Science ®Google Scholar
• Nilsson IM, Berntorp E, Lofqvist T, Pettersson H. Twenty-five years’ experience of prophylactic treatment in severe haemophilia A and B. J Intern Med 1992;232(1):25-32
   PubMed Web of Science ®Google Scholar
• Berntorp E, Shapiro AD, Waters J, Astermark J. The International Factor IX Treatment Network survey. Haemophilia 2012;18(3):e60-2
   Web of Science ®Google Scholar
• Khair K, Lawrence K, Butler R, et al. Assessment of treatment practice patterns for severe hemophilia A: a global nurse perspective. Acta Haematol 2008;119(2):115-23
   PubMed Web of Science ®Google Scholar
• Geraghty S, Dunkley T, Harrington C, et al. Practice patterns in haemophilia A therapy -- global progress towards optimal care. Haemophilia 2006;12(1):75-81
   PubMed Web of Science ®Google Scholar
• Manco-Johnson MJ, Abshire TC, Shapiro AD, et al. Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia. N Engl J Med 2007;357(6):535-44
   PubMed Web of Science ®Google Scholar
• Gringeri A, Lundin B, Von Mackensen S, et al. A randomized clinical trial of prophylaxis in children with hemophilia A (the ESPRIT Study). J Thromb Haemost 2011;9(4):700-10
   PubMed Web of Science ®Google Scholar
• Klukowska A, Laguna P, Svirin P, et al. Efficacy and safety of OCTANINE F in children with haemophilia B. Haemophilia. 2008;14(3):531-8
   Google Scholar
• Monahan PE, Liesner R, Sullivan ST, et al. Safety and efficacy of investigator-prescribed BeneFIX prophylaxis in children less than 6 years of age with severe haemophilia B. Haemophilia 2010;16(3):460-8
   PubMed Web of Science ®Google Scholar
• Puetz J, Soucie JM, Kempton CL, Monahan PE. Prevalent inhibitors in haemophilia B subjects enrolled in the Universal Data Collection database. Haemophilia 2014;20(1):25-31
   PubMed Web of Science ®Google Scholar
• Fischer K, Lewandowski D, Marijke Van Den Berg H, Janssen MP. Validity of assessing inhibitor development in haemophilia PUPs using registry data: the EUHASS project. Haemophilia 2012;18(3):e241-6
   PubMed Web of Science ®Google Scholar
• Wight J, Paisley S. The epidemiology of inhibitors in haemophilia A: a systematic review. Haemophilia 2003;9(4):418-35
   PubMed Web of Science ®Google Scholar
• Peden A, Mccardle L, Head MW, et al. Variant CJD infection in the spleen of a neurologically asymptomatic UK adult patient with haemophilia. Haemophilia 2010;16(2):296-304
   PubMed Web of Science ®Google Scholar
• Sharp CP, Lail A, Donfield S, et al. Virologic and clinical features of primary infection with human parvovirus 4 in subjects with hemophilia: frequent transmission by virally inactivated clotting factor concentrates. Transfusion 2012;52(7):1482-9
   PubMed Web of Science ®Google Scholar
• Azzi A, Ciappi S, Zakvrzewska K, et al. Human parvovirus B19 infection in hemophiliacs first infused with two high-purity, virally attenuated factor VIII concentrates. Am J Hematol 1992;39(3):228-30
   PubMed Web of Science ®Google Scholar
• Ludlam CA, Turner ML. Managing the risk of transmission of variant Creutzfeldt Jakob disease by blood products. Br J Haematol 2006;132(1):13-24
   PubMed Web of Science ®Google Scholar
• US FDA. News and Events. FDA News Release. FDA approves first recombinant coagulation factor IX that is specifically indicated for routine use in preventing bleeding episodes (prophylaxis). www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm358918.htm [Last accessed 6 November 2013]
   Google Scholar
• BeneFIX [package insert]. New York, NY: Pfizer; 2011. http://labeling.pfizer.com/showlabeling.aspx?id=492 [Last accessed 22 November 2013]
   Google Scholar
• RIXUBIS [package insert]. Westlake Village, CA: Baxter Healthcare Corp. www.baxter.com/downloads/healthcare_professionals/products/RIXUBIS_PI.pdf [Last accessed 22 November 2013]
   Google Scholar
• Mannucci PM. Back to the future: a recent history of haemophilia treatment. Haemophilia 2008;14(suppl 3):10-18
   PubMed Web of Science ®Google Scholar
• Choo KH, Gould KG, Rees DJ, Brownlee GG. Molecular cloning of the gene for human anti-haemophilic factor IX. Nature 1982;299(5879):178-80
   PubMed Web of Science ®Google Scholar
• Pipe SW. Recombinant clotting factors. Thromb Haemost 2008;99(5):840-50
   PubMed Web of Science ®Google Scholar
• Roth DA, Kessler CM, Pasi KJ, et al. Human recombinant factor IX: safety and efficacy studies in hemophilia B patients previously treated with plasma-derived factor IX concentrates. Blood 2001;98(13):3600-6
   PubMed Web of Science ®Google Scholar
• Poon MC, Lillicrap D, Hensman C, et al. Recombinant factor IX recovery and inhibitor safety: a Canadian post-licensure surveillance study. Thromb Haemost 2002;87(3):431-5
   PubMed Web of Science ®Google Scholar
• Rocca A, Pizzinelli S, Oliovecchio E, et al. Replacement therapy with recombinant factor IX. A multicentre evaluation of current dosing practices in Italy. Blood Transfus 2011;9(1):60-9
   PubMed Web of Science ®Google Scholar
• Ewenstein BM, Joist JH, Shapiro AD, et al. Pharmacokinetic analysis of plasma-derived and recombinant F IX concentrates in previously treated patients with moderate or severe hemophilia B. Transfusion 2002;42(2):190-7
   PubMed Web of Science ®Google Scholar
• Kisker CT, Eisberg A, Schwartz B. Prophylaxis in factor IX deficiency product and patient variation. Haemophilia 2003;9(3):279-84
   PubMed Web of Science ®Google Scholar
• Dietrich B, Schiviz A, Hoellriegl W, et al. Preclinical safety and efficacy of a new recombinant FIX drug product for treatment of hemophilia B. Int J Hematol 2013;98(5):525-32
   PubMed Web of Science ®Google Scholar
• Martinowitz U, Shapiro A, Quon DV, et al. Pharmacokinetic properties of IB1001, an investigational recombinant factor IX, in patients with haemophilia B: repeat pharmacokinetic evaluation and sialylation analysis. Haemophilia 2012;18(6):881-7
   PubMed Web of Science ®Google Scholar
• Martinowitz U, Lubetsky A. Phase I/II, open-label, multicenter, safety, efficacy and PK study of a recombinant coagulation factor IX albumin fusion protein (rIX-FP) in subjects with hemophilia B. Thromb Res 2013;131(suppl 2):S11-14
   PubMed Web of Science ®Google Scholar
• Santagostino E. PROLONG-9FP clinical development program--phase I results of recombinant fusion protein linking coagulation factor IX with recombinant albumin (rIX-FP). Thromb Res 2013;131(suppl 2):S7-S10
   PubMedGoogle Scholar
• Shapiro AD, Ragni MV, Valentino LA, et al. Recombinant factor IX-Fc fusion protein (rFIXFc) demonstrates safety and prolonged activity in a phase 1/2a study in hemophilia B patients. Blood 2012;119(3):666-72
   PubMed Web of Science ®Google Scholar
• Ivens IA, Baumann A, Mcdonald TA, et al.Gylated therapeutic proteins for haemophilia treatment: a review for haemophilia caregivers. Haemophilia 2013;19(1):11-20
   PubMed Web of Science ®Google Scholar
• Négrier C, Knobe K, Tiede A, et al. Enhanced pharmacokinetic properties of a glycoPEGylated recombinant factor IX: a first human dose trial in patients with hemophilia B. Blood 2011;118(10):2695-701
   PubMed Web of Science ®Google Scholar
• Windyga J, Lissitchkov T, Stasyshyn O, et al. Pharmacokinetics, efficacy and safety of BAX326, a novel recombinant factor IX: a prospective, controlled, multicentre phase I/III study in previously treated patients with severe (FIX level <1%) or moderately severe (FIX level ≤2%) haemophilia B. Haemophilia 2014;20(1):15-24
   PubMed Web of Science ®Google Scholar
• Gray E, Tubbs J, Thomas S, et al. Measurement of activated factor IX in factor IX concentrates: correlation with in vivo thrombogenicity. Thromb Haemost 1995;73(4):675-9
   PubMed Web of Science ®Google Scholar
• Pivotal study (Pharmacokinetics, Efficacy, Safety) of BAX 326 (rFIX) in hemophilia B patients. Available from: http://clinicaltrials.gov/ct2/show/NCT01174446
   Google Scholar
• Windyga J, Lin VW, Epstein JD, et al. Improvement in health-related quality of life with recombinant factor IX prophylaxis in severe or moderately-severe haemophilia B: results from the BAX326 pivotal study. Haemophilia 2014;20(3):362-8
   PubMed Web of Science ®Google Scholar
• Windyga J, Lissitchkov T, Mamonov V, et al. Efficacy and safety of a recombinant factor IX (Bax326)* in previously treated patients with severe or moderately severe hemophilia B undergoing surgical or other invasive procedures: a prospective, open-label, uncontrolled, multicenter, phase III study. Haemophilia 2014. [Epub ahead of print]
   Web of Science ®Google Scholar
• Urasinski T, Stasyshyn O, Tatiana A, et al. Pharmacokinetics, efficacy and safety of a recombinant factor IX (BAX326) in previously-treated pediatric patients <12 years of age with severe or moderately severe hemophilia B. Blood 2013;122(21):1118
   Web of Science ®Google Scholar
• Collins PW, Bjorkman S, Fischer K, et al. Factor VIII requirement to maintain a target plasma level in the prophylactic treatment of severe hemophilia A: influences of variance in pharmacokinetics and treatment regimens. J Thromb Haemost 2010;8(2):269-75
   PubMed Web of Science ®Google Scholar
• Broderick CR, Herbert RD, Latimer J, et al. Association between physical activity and risk of bleeding in children with hemophilia. JAMA 2012;308(14):1452-9
   PubMed Web of Science ®Google Scholar
• US Department of Health and Human Services. Study of recombinant coagulation factor IX Fc fusion protein, BIIB 029, in pediatric ptp subjects with hemophilia B. NCT01440946. Available from: http://clinicaltrials.gov/show/NCT01440946 [Last accessed 24 October 2013]
   Google Scholar
• US Department of Health and Human Services. Safety, efficacy and pharmacokinetics of NNC-0156-0000-0009 in previously treated children with haemophilia B (paradigm™5). NCT01467427. Available from: http://clinicaltrials.gov/ct2/show/NCT01467427?term=n9-gp&rank=1 [Last accessed 24 October 2013]
   Google Scholar
• US Department of Health and Human Services. Study of recombinant factor IX product, IB1001, in previously treated pediatric subjects With Hemophilia B. NCT01271868. Available from: http://clinicaltrials.gov/ct2/show/NCT01271868?term=NCT01271868&rank=1 [Last accessed 24 October 2013]
   Google Scholar