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
Summary BAX326 is a recombinant factor IX (rFIX; nonacog gamma) manufactured without the addition of any materials of human or animal origin, and with two viral inactivation steps (solvent/detergent treatment and 15 nm nanofiltration). The aim of this prospective trial was to investigate the pharmac...
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| Published in | Haemophilia : the official journal of the World Federation of Hemophilia Vol. 20; no. 1; pp. 15 - 24 |
|---|---|
| Main Authors | , , , , , , , , , , , |
| Format | Journal Article |
| Language | English |
| Published |
England
Blackwell Publishing Ltd
01.01.2014
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| Subjects | |
| Online Access | Get full text |
| ISSN | 1351-8216 1365-2516 1365-2516 |
| DOI | 10.1111/hae.12228 |
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| Abstract | Summary
BAX326 is a recombinant factor IX (rFIX; nonacog gamma) manufactured without the addition of any materials of human or animal origin, and with two viral inactivation steps (solvent/detergent treatment and 15 nm nanofiltration). The aim of this prospective trial was to investigate the pharmacokinetics, haemostatic efficacy and safety of BAX326 in previously treated patients aged 12–65 years with severe or moderately severe haemophilia B. BAX326 was safe and well tolerated in all 73 treated subjects; adverse events considered related to treatment (2.7% incidence, all non‐serious) were transient and mild, and no hypersensitivity reactions, inhibitor formation or thrombotic events were observed. Pharmacokinetic (PK) equivalence (n = 28) between BAX326 and a licensed rFIX was confirmed in terms of the ratio of geometric mean AUC0–72 h per dose. Twice‐weekly prophylaxis [mean duration 6.2 (±0.7) months; 1.8 (±0.1) infusions per week, 49.5 (±4.8) IU kg−1 per infusion] was effective in preventing bleeding episodes, with a significantly lower (79%, P < 0.001) annualized bleed rate (4.2) compared to an on‐demand treatment in a historical control group (20.0); 24 of 56 subjects on prophylaxis (43%) did not bleed throughout the study observation period. Of 249 total acute bleeds, 211 (84.7%) were controlled with one to two infusions of BAX326. Haemostatic efficacy at resolution of bleed was rated excellent or good in 96.0% of all treated bleeding episodes. The results of this study indicate that BAX326 is safe and efficacious in treating bleeds and routine prophylaxis in patients aged 12 years and older with haemophilia B. |
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| AbstractList | BAX326 is a recombinant factor IX (rFIX; nonacog gamma) manufactured without the addition of any materials of human or animal origin, and with two viral inactivation steps (solvent/detergent treatment and 15 nm nanofiltration). The aim of this prospective trial was to investigate the pharmacokinetics, haemostatic efficacy and safety of BAX326 in previously treated patients aged 12-65 years with severe or moderately severe haemophilia B. BAX326 was safe and well tolerated in all 73 treated subjects; adverse events considered related to treatment (2.7% incidence, all non-serious) were transient and mild, and no hypersensitivity reactions, inhibitor formation or thrombotic events were observed. Pharmacokinetic (PK) equivalence (n = 28) between BAX326 and a licensed rFIX was confirmed in terms of the ratio of geometric mean AUC0-72 h per dose. Twice-weekly prophylaxis [mean duration 6.2 ( plus or minus 0.7) months; 1.8 ( plus or minus 0.1) infusions per week, 49.5 ( plus or minus 4.8) IU kg-1 per infusion] was effective in preventing bleeding episodes, with a significantly lower (79%, P < 0.001) annualized bleed rate (4.2) compared to an on-demand treatment in a historical control group (20.0); 24 of 56 subjects on prophylaxis (43%) did not bleed throughout the study observation period. Of 249 total acute bleeds, 211 (84.7%) were controlled with one to two infusions of BAX326. Haemostatic efficacy at resolution of bleed was rated excellent or good in 96.0% of all treated bleeding episodes. The results of this study indicate that BAX326 is safe and efficacious in treating bleeds and routine prophylaxis in patients aged 12 years and older with haemophilia B. BAX326 is a recombinant factor IX (rFIX; nonacog gamma) manufactured without the addition of any materials of human or animal origin, and with two viral inactivation steps (solvent/detergent treatment and 15 nm nanofiltration). The aim of this prospective trial was to investigate the pharmacokinetics, haemostatic efficacy and safety of BAX326 in previously treated patients aged 12-65 years with severe or moderately severe haemophilia B. BAX326 was safe and well tolerated in all 73 treated subjects; adverse events considered related to treatment (2.7% incidence, all non-serious) were transient and mild, and no hypersensitivity reactions, inhibitor formation or thrombotic events were observed. Pharmacokinetic (PK) equivalence (n = 28) between BAX326 and a licensed rFIX was confirmed in terms of the ratio of geometric mean AUC(0-72) h per dose. Twice-weekly prophylaxis [mean duration 6.2 (±0.7) months; 1.8 (±0.1) infusions per week, 49.5 (±4.8) IU kg(-1) per infusion] was effective in preventing bleeding episodes, with a significantly lower (79%, P < 0.001) annualized bleed rate (4.2) compared to an on-demand treatment in a historical control group (20.0); 24 of 56 subjects on prophylaxis (43%) did not bleed throughout the study observation period. Of 249 total acute bleeds, 211 (84.7%) were controlled with one to two infusions of BAX326. Haemostatic efficacy at resolution of bleed was rated excellent or good in 96.0% of all treated bleeding episodes. The results of this study indicate that BAX326 is safe and efficacious in treating bleeds and routine prophylaxis in patients aged 12 years and older with haemophilia B. Summary BAX326 is a recombinant factor IX (rFIX; nonacog gamma) manufactured without the addition of any materials of human or animal origin, and with two viral inactivation steps (solvent/detergent treatment and 15 nm nanofiltration). The aim of this prospective trial was to investigate the pharmacokinetics, haemostatic efficacy and safety of BAX326 in previously treated patients aged 12–65 years with severe or moderately severe haemophilia B. BAX326 was safe and well tolerated in all 73 treated subjects; adverse events considered related to treatment (2.7% incidence, all non‐serious) were transient and mild, and no hypersensitivity reactions, inhibitor formation or thrombotic events were observed. Pharmacokinetic (PK) equivalence (n = 28) between BAX326 and a licensed rFIX was confirmed in terms of the ratio of geometric mean AUC0–72 h per dose. Twice‐weekly prophylaxis [mean duration 6.2 (±0.7) months; 1.8 (±0.1) infusions per week, 49.5 (±4.8) IU kg−1 per infusion] was effective in preventing bleeding episodes, with a significantly lower (79%, P < 0.001) annualized bleed rate (4.2) compared to an on‐demand treatment in a historical control group (20.0); 24 of 56 subjects on prophylaxis (43%) did not bleed throughout the study observation period. Of 249 total acute bleeds, 211 (84.7%) were controlled with one to two infusions of BAX326. Haemostatic efficacy at resolution of bleed was rated excellent or good in 96.0% of all treated bleeding episodes. The results of this study indicate that BAX326 is safe and efficacious in treating bleeds and routine prophylaxis in patients aged 12 years and older with haemophilia B. BAX326 is a recombinant factor IX (rFIX; nonacog gamma) manufactured without the addition of any materials of human or animal origin, and with two viral inactivation steps (solvent/detergent treatment and 15 nm nanofiltration). The aim of this prospective trial was to investigate the pharmacokinetics, haemostatic efficacy and safety of BAX326 in previously treated patients aged 12-65 years with severe or moderately severe haemophilia B. BAX326 was safe and well tolerated in all 73 treated subjects; adverse events considered related to treatment (2.7% incidence, all non-serious) were transient and mild, and no hypersensitivity reactions, inhibitor formation or thrombotic events were observed. Pharmacokinetic (PK) equivalence (n = 28) between BAX326 and a licensed rFIX was confirmed in terms of the ratio of geometric mean AUC(0-72) h per dose. Twice-weekly prophylaxis [mean duration 6.2 (±0.7) months; 1.8 (±0.1) infusions per week, 49.5 (±4.8) IU kg(-1) per infusion] was effective in preventing bleeding episodes, with a significantly lower (79%, P < 0.001) annualized bleed rate (4.2) compared to an on-demand treatment in a historical control group (20.0); 24 of 56 subjects on prophylaxis (43%) did not bleed throughout the study observation period. Of 249 total acute bleeds, 211 (84.7%) were controlled with one to two infusions of BAX326. Haemostatic efficacy at resolution of bleed was rated excellent or good in 96.0% of all treated bleeding episodes. The results of this study indicate that BAX326 is safe and efficacious in treating bleeds and routine prophylaxis in patients aged 12 years and older with haemophilia B.BAX326 is a recombinant factor IX (rFIX; nonacog gamma) manufactured without the addition of any materials of human or animal origin, and with two viral inactivation steps (solvent/detergent treatment and 15 nm nanofiltration). The aim of this prospective trial was to investigate the pharmacokinetics, haemostatic efficacy and safety of BAX326 in previously treated patients aged 12-65 years with severe or moderately severe haemophilia B. BAX326 was safe and well tolerated in all 73 treated subjects; adverse events considered related to treatment (2.7% incidence, all non-serious) were transient and mild, and no hypersensitivity reactions, inhibitor formation or thrombotic events were observed. Pharmacokinetic (PK) equivalence (n = 28) between BAX326 and a licensed rFIX was confirmed in terms of the ratio of geometric mean AUC(0-72) h per dose. Twice-weekly prophylaxis [mean duration 6.2 (±0.7) months; 1.8 (±0.1) infusions per week, 49.5 (±4.8) IU kg(-1) per infusion] was effective in preventing bleeding episodes, with a significantly lower (79%, P < 0.001) annualized bleed rate (4.2) compared to an on-demand treatment in a historical control group (20.0); 24 of 56 subjects on prophylaxis (43%) did not bleed throughout the study observation period. Of 249 total acute bleeds, 211 (84.7%) were controlled with one to two infusions of BAX326. Haemostatic efficacy at resolution of bleed was rated excellent or good in 96.0% of all treated bleeding episodes. The results of this study indicate that BAX326 is safe and efficacious in treating bleeds and routine prophylaxis in patients aged 12 years and older with haemophilia B. BAX326 is a recombinant factor IX (rFIX; nonacog gamma) manufactured without the addition of any materials of human or animal origin, and with two viral inactivation steps (solvent/detergent treatment and 15 nm nanofiltration). The aim of this prospective trial was to investigate the pharmacokinetics, haemostatic efficacy and safety of BAX326 in previously treated patients aged 12–65 years with severe or moderately severe haemophilia B. BAX326 was safe and well tolerated in all 73 treated subjects; adverse events considered related to treatment (2.7% incidence, all non‐serious) were transient and mild, and no hypersensitivity reactions, inhibitor formation or thrombotic events were observed. Pharmacokinetic (PK) equivalence ( n = 28) between BAX326 and a licensed rFIX was confirmed in terms of the ratio of geometric mean AUC 0–72 h per dose. Twice‐weekly prophylaxis [mean duration 6.2 (±0.7) months; 1.8 (±0.1) infusions per week, 49.5 (±4.8) IU kg −1 per infusion] was effective in preventing bleeding episodes, with a significantly lower (79%, P < 0.001) annualized bleed rate (4.2) compared to an on‐demand treatment in a historical control group (20.0); 24 of 56 subjects on prophylaxis (43%) did not bleed throughout the study observation period. Of 249 total acute bleeds, 211 (84.7%) were controlled with one to two infusions of BAX326. Haemostatic efficacy at resolution of bleed was rated excellent or good in 96.0% of all treated bleeding episodes. The results of this study indicate that BAX326 is safe and efficacious in treating bleeds and routine prophylaxis in patients aged 12 years and older with haemophilia B. |
| Author | Lissitchkov, T. Fritsch, S. Rusen, L. Oh, M.-S. Lamas, J. L. Mamonov, V. Windyga, J. Stasyshyn, O. Pavlova, B. G. Wong, W.-Y. Chapman, M. Abbuehl, B. E. |
| Author_xml | – sequence: 1 givenname: J. surname: Windyga fullname: Windyga, J. organization: Institute of Hematology and Transfusion Medicine, Warsaw, Poland – sequence: 2 givenname: T. surname: Lissitchkov fullname: Lissitchkov, T. organization: Specialized Hematological Hospital "Joan Pavel", Sofia, Bulgaria – sequence: 3 givenname: O. surname: Stasyshyn fullname: Stasyshyn, O. organization: State Institution "Institute of Blood Pathology and Transfusion Medicine of the Academy of Medical Sciences of Ukraine", Lviv, Ukraine – sequence: 4 givenname: V. surname: Mamonov fullname: Mamonov, V. organization: Hematological Research Center, Moscow, Russia – sequence: 5 givenname: L. surname: Rusen fullname: Rusen, L. organization: Prof. Dr. C. T. Nicolau National Institute for Transfusional Hematology, Bucharest, Romania – sequence: 6 givenname: J. L. surname: Lamas fullname: Lamas, J. L. organization: Hospital Dr. Sotero del Rio, Santiago, Chile – sequence: 7 givenname: M.-S. surname: Oh fullname: Oh, M.-S. organization: Global Clinical Research and Development, Baxter BioScience, CA, Westlake Village, USA – sequence: 8 givenname: M. surname: Chapman fullname: Chapman, M. organization: Global Clinical Research and Development, Baxter BioScience, Vienna, Austria – sequence: 9 givenname: S. surname: Fritsch fullname: Fritsch, S. organization: Global Clinical Research and Development, Baxter BioScience, Vienna, Austria – sequence: 10 givenname: B. G. surname: Pavlova fullname: Pavlova, B. G. organization: Global Clinical Research and Development, Baxter BioScience, Vienna, Austria – sequence: 11 givenname: W.-Y. surname: Wong fullname: Wong, W.-Y. organization: Global Clinical Research and Development, Baxter BioScience, CA, Westlake Village, USA – sequence: 12 givenname: B. E. surname: Abbuehl fullname: Abbuehl, B. E. email: brigitt_abbuehl@baxter.com organization: Global Clinical Research and Development, Baxter BioScience, Vienna, Austria |
| BackLink | https://www.ncbi.nlm.nih.gov/pubmed/23834666$$D View this record in MEDLINE/PubMed |
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| Keywords | previously treated patients BAX326 haemophilia B recombinant factor IX |
| Language | English |
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| Notes | ArticleID:HAE12228 istex:3022A0AF90E48359AE992850C5E502E80F198B23 Figure S1. Annual bleeding rate during on-demand treatment with factor IX. Data points scaled by meta-analytic weight. Error bars depict 95% confidence intervals. ark:/67375/WNG-RPBVPXTX-3 ObjectType-Article-1 SourceType-Scholarly Journals-1 ObjectType-Feature-2 content type line 23 ObjectType-Undefined-3 ObjectType-Article-2 ObjectType-Feature-1 |
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Dev Biol Stand 1992; 76: 201-7. Panicker J, Warrier I, Thomas R, Lusher JM. The overall effectiveness of prophylaxis in severe haemophilia. Haemophilia 2003; 9: 272-8. Hay CR, Brown S, Collins PW, Keeling DM, Liesner R. The diagnosis and management of factor VIII and IX inhibitors: a guideline from the United Kingdom Haemophilia Centre Doctors Organisation. Br J Haematol 2006; 133: 591-605. Berntorp E, Fischer K, Miners A. Models of prophylaxis. Haemophilia 2012; 18(Suppl. 4): 136-40. Castaldo G, Nardiello P, Bellitti F et al. Haemophilia B: from molecular diagnosis to gene therapy. Clin Chem Lab Med 2003; 41: 445-51. Ewing NP, Kasper CK. In vitro detection of mild inhibitors to factor VIII in hemophilia. Am J Clin Pathol 1982; 77: 749-52. 2001; 344 1995; 73 2010; 16 2011 1991; 54 2010; 106 1982; 77 2008; 36 2005; 437 2008; 14 1992; 79 2012; 18 2013; 121 2011; 17 1992; 76 2007; 13 2006; 133 2011; 9 2006; 46 2002; 42 2005; 105 2003; 9 1997; 78 2003; 1 1976; 16 2003; 41 2010; 30 2001; 98 1998; 35 e_1_2_9_31_1 e_1_2_9_11_1 e_1_2_9_10_1 e_1_2_9_35_1 e_1_2_9_13_1 e_1_2_9_32_1 e_1_2_9_12_1 e_1_2_9_33_1 Chambost H (e_1_2_9_20_1) 2011; 9 Fukui H (e_1_2_9_41_1) 1991; 54 e_1_2_9_15_1 e_1_2_9_14_1 e_1_2_9_39_1 e_1_2_9_17_1 Bond M (e_1_2_9_34_1) 1998; 35 e_1_2_9_36_1 e_1_2_9_16_1 e_1_2_9_37_1 e_1_2_9_18_1 Dinowitz M (e_1_2_9_7_1) 1992; 76 Maruish ME (e_1_2_9_28_1) 2011 White G (e_1_2_9_30_1) 1998; 35 Valluri S (e_1_2_9_38_1) 2012; 18 e_1_2_9_40_1 e_1_2_9_22_1 Valentino LA (e_1_2_9_23_1) 2011; 9 e_1_2_9_21_1 e_1_2_9_24_1 e_1_2_9_8_1 e_1_2_9_6_1 e_1_2_9_5_1 e_1_2_9_4_1 e_1_2_9_3_1 e_1_2_9_2_1 Siegmund B (e_1_2_9_19_1) 2010; 30 e_1_2_9_9_1 e_1_2_9_26_1 e_1_2_9_25_1 e_1_2_9_27_1 e_1_2_9_29_1 |
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patient variation publication-title: Haemophilia – volume: 1 start-page: 922 year: 2003 end-page: 30 article-title: The future of recombinant coagulation factors publication-title: J Thromb Haemost – volume: 14 start-page: 531 year: 2008 end-page: 8 article-title: Efficacy and safety of OCTANINE F in children with haemophilia B publication-title: Haemophilia – volume: 9 start-page: 24 year: 2003 end-page: 37 article-title: Nanofiltration of plasma‐derived biopharmaceutical products publication-title: Haemophilia – volume: 9 start-page: 371 issue: Suppl. 2 year: 2011 article-title: Five‐year post‐marketing surveillance of haemophilia B patient receiving a factor IX concentrate: final results [abstract] publication-title: J Thromb Haemost – volume: 77 start-page: 749 year: 1982 end-page: 52 article-title: In vitro detection of mild inhibitors to factor VIII in hemophilia publication-title: Am J Clin Pathol – volume: 133 start-page: 591 year: 2006 end-page: 605 article-title: The diagnosis and management of factor VIII and IX inhibitors: a guideline from the United Kingdom Haemophilia Centre Doctors Organisation publication-title: Br J Haematol – volume: 16 start-page: 460 year: 2010 end-page: 8 article-title: Safety and efficacy of investigator‐prescribed BeneFix prophylaxis in children less than 6 years of age with severe hemophilia B publication-title: Haemophilia – volume: 17 start-page: 439 year: 2011 end-page: 45 article-title: An ultrapure plasma‐derived monoclonal antibody‐purified factor IX concentrate (Nonafact ), results of phase III and IV clinical studies publication-title: Haemophilia – volume: 9 start-page: 272 year: 2003 end-page: 8 article-title: The overall effectiveness of prophylaxis in severe haemophilia publication-title: Haemophilia – volume: 105 start-page: 518 year: 2005 end-page: 25 article-title: The safety and efficacy of recombinant human blood coagulation factor IX in previously untreated patients with severe or moderately severe hemophilia B publication-title: Blood – volume: 41 start-page: 445 year: 2003 end-page: 51 article-title: Haemophilia B: from molecular diagnosis to gene therapy publication-title: Clin Chem Lab Med – volume: 18 start-page: 554 year: 2012 end-page: 60 article-title: Patient‐ and caregiver‐reported bleeding symptoms and reasons for starting and stopping treatment with recombinant factor VIIa: analysis of the Dosing Observational Study in Haemophilia (DOSE) publication-title: Haemophilia – volume: 17 start-page: 179 year: 2011 end-page: 84 article-title: A commentary on the differences in pharmacokinetics between recombinant and plasma‐ erived factor IX and their implications for dosing publication-title: Haemophilia – volume: 106 start-page: 598 year: 2010 end-page: 607 article-title: Virus susceptibility of Chinese hamster ovary (CHO) cells and detection of viral contaminations by adventitious agent testing publication-title: Biotechnol Bioeng – volume: 30 start-page: 35 year: 2010 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Prevention of bleeds and FIX consumption publication-title: Hamostaseologie – volume: 16 start-page: 41 year: 1976 end-page: 7 article-title: Evaluation of prophylactic replacement therapy in haemophilia publication-title: Scand J Haematol – volume: 18 start-page: 136 issue: Suppl. 4 year: 2012 end-page: 40 article-title: Models of prophylaxis publication-title: Haemophilia – volume: 18 start-page: 38 issue: Suppl. 1 year: 2012 article-title: What is it like to have a joint bleed? The perspective of adults and adolscents with hemophilia A [abstract] publication-title: Haemophilia – volume: 54 start-page: 419 year: 1991 end-page: 27 article-title: Clinical evaluation of recombinant human factor VIII (BAY w 6240) in the treatment of hemophilia A publication-title: Int J Hematol – volume: 13 start-page: 233 year: 2007 end-page: 43 article-title: Reformulated BeneFix : efficacy and safety in previously treated patients with moderately severe to severe haemophilia B publication-title: Haemophilia – volume: 46 start-page: 1143 year: 2006 end-page: 51 article-title: Removal of small nonenveloped viruses by antibody‐enhanced nanofiltration during the manufacture of plasma derivatives publication-title: Transfusion – volume: 121 start-page: 1039 year: 2013 end-page: 48 article-title: Distinct characteristics of antibody responses against factor VIII in healthy individuals and in different cohorts of hemophilia A patients publication-title: Blood – volume: 14 start-page: 945 year: 2008 end-page: 51 article-title: Effects of secondary prophylaxis started in adolescent and adult haemophiliacs publication-title: Haemophilia – volume: 42 start-page: 190 year: 2002 end-page: 7 article-title: Pharmacokinetic analysis of plasma‐derived and recombinant F IX concentrates in previously treated patients with moderate or severe hemophilia B publication-title: Transfusion – volume: 78 start-page: 261 year: 1997 end-page: 5 article-title: Recombinant factor IX publication-title: Thromb Haemost – volume: 437 start-page: 257 year: 2005 end-page: 61 article-title: The most infectious prion protein particles publication-title: Nature – volume: 35 start-page: 11 issue: Suppl. 2 year: 1998 end-page: 7 article-title: Biochemical characterization of recombinant factor IX publication-title: Semin Hematol – volume: 9 start-page: 357 issue: Suppl. 2 year: 2011 article-title: A multicenter, open‐label study to compare on‐demand treatment with two prophylaxis regimens of recombinant coagulation factor IX in subjects with hemophilia B publication-title: J Thromb Haemost – ident: e_1_2_9_5_1 doi: 10.1182/blood.V98.13.3600 – ident: e_1_2_9_10_1 doi: 10.1016/j.biologicals.2007.04.005 – volume: 35 start-page: 11 issue: 2 year: 1998 ident: e_1_2_9_34_1 article-title: Biochemical characterization of recombinant factor IX publication-title: Semin Hematol – ident: e_1_2_9_39_1 doi: 10.1111/j.1365-2516.2012.02762.x – ident: e_1_2_9_4_1 doi: 10.1182/blood.V79.3.568.568 – ident: e_1_2_9_37_1 doi: 10.1111/j.1365-2516.2008.01791.x – ident: e_1_2_9_15_1 doi: 10.1182/blood-2004-06-2283 – volume: 9 start-page: 371 issue: 2 year: 2011 ident: e_1_2_9_20_1 article-title: Five‐year post‐marketing surveillance of haemophilia B patient receiving a factor IX concentrate: final results [abstract] publication-title: J Thromb Haemost – ident: e_1_2_9_11_1 doi: 10.1038/nature03989 – ident: e_1_2_9_36_1 doi: 10.1111/j.1365-2516.2012.02839.x – ident: e_1_2_9_14_1 doi: 10.1046/j.1365-2516.2003.00757.x – ident: e_1_2_9_25_1 doi: 10.1111/j.1365-2141.2006.06087.x – ident: e_1_2_9_24_1 doi: 10.1055/s-0038-1653759 – ident: e_1_2_9_27_1 doi: 10.1182/blood-2012-07-444877 – volume: 18 start-page: 38 issue: 1 year: 2012 ident: e_1_2_9_38_1 article-title: What is it like to have a joint bleed? 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BAX326 is a recombinant factor IX (rFIX; nonacog gamma) manufactured without the addition of any materials of human or animal origin, and with two... BAX326 is a recombinant factor IX (rFIX; nonacog gamma) manufactured without the addition of any materials of human or animal origin, and with two viral... |
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| SubjectTerms | Adolescent Adult Aged BAX326 Bleeding Blood Coagulation - drug effects Child Factor IX - pharmacokinetics Factor IX - therapeutic use Female haemophilia B Hemophilia B - blood Hemophilia B - drug therapy Humans Male Middle Aged Premedication previously treated patients Quality of Life recombinant factor IX Recombinant Proteins Severity of Illness Index Treatment Outcome Young Adult |
| Title | 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 |
| URI | https://api.istex.fr/ark:/67375/WNG-RPBVPXTX-3/fulltext.pdf https://onlinelibrary.wiley.com/doi/abs/10.1111%2Fhae.12228 https://www.ncbi.nlm.nih.gov/pubmed/23834666 https://www.proquest.com/docview/1490778189 https://www.proquest.com/docview/1492652914 |
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