The biological efficacy profile of BAX 855, a PEGylated recombinant factor VIII molecule

Haemophilia

Volumn 21, Issue 1, 2015, Pages 58-63

  Valentino, L A ^b   Cong, L ^a   Enockson, C ^a   Song, X ^a   Scheiflinger, F ^c   Muchitsch, E M ^c   Turecek, P L ^c   Hakobyan, N ^a  

^a RUSH UNIVERSITY MEDICAL CENTER   (United States)

^b RUSH UNIVERSITY   (United States)

^c BAXTER INNOVATIONS GMBH   (Austria)

Author keywords

ADVATE; BAX 855; Haemarthrosis; Haemophilia; Mouse model; PEGylation

Indexed keywords

RECOMBINANT BLOOD CLOTTING FACTOR 8; BLOOD CLOTTING FACTOR 8; RECOMBINANT PROTEIN;

ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; COMPARATIVE STUDY; CONTROLLED STUDY; DRUG EFFICACY; HEMARTHROSIS; HEMOPHILIA A; HUMAN; JOINT CAVITY; MEDICATION COMPLIANCE; MOUSE; NONHUMAN; PRIORITY JOURNAL; PROPHYLAXIS; SINGLE DRUG DOSE; TREATMENT DURATION; ANIMAL; DISEASE MODEL; HEMORRHAGE;

ANIMALS; DISEASE MODELS, ANIMAL; FACTOR VIII; HEMOPHILIA A; HEMORRHAGE; HUMANS; MICE; RECOMBINANT PROTEINS;

EID: 84921542285     PISSN: 13518216     EISSN: 13652516     Source Type: Journal    
DOI: 10.1111/hae.12532     Document Type: Article

References (18)

1. 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: 535-44.
2. Gringeri A, Lundin B, von Mackensen S, Mantovani L, Mannucci PM. A randomized clinical trial of prophylaxis in children with hemophilia A (the ESPRIT Study). J Thromb Haemost 2011; 9: 700-10.
3. Valentino LA, Mamonov V, Hellmann A et al. A randomized comparison of two prophylaxis regimens and a paired comparison of on-demand and prophylaxis treatments in hemophilia A management. J Thromb Haemost 2012; 10: 359-67.
4. Manco-Johnson MJ, Kempton CL, Reding MT et al. Randomized, controlled, parallel-group trial of routine prophylaxis vs. on-demand treatment with sucrose-formulated recombinant factor VIII in adults with severe hemophilia A (SPINART). J Thromb Haemost 2013; 11: 1119-27.
5. Witmer C, Presley R, Kulkarni R, Soucie JM, Manno CS, Raffini L. Associations between intracranial haemorrhage and prescribed prophylaxis in a large cohort of haemophilia patients in the United States. Br J Haematol 2011; 152: 211-6.
6. Valentino LA, Mamonov V, Hellmann A et al. A randomized comparison of two prophylaxis regimens and a paired comparison of on-demand and prophylaxis treatments in hemophilia A management. J Thromb Haemost 2012; 10: 359-67.
7. Zappa S, McDaniel M, Marandola J, Allen G. Treatment trends for haemophilia A and haemohilia B in the United States: results from the 2010 practice patterns survey. Haemophilia 2012; 18: e140-e153.
8. Pisal DS, Kosloski MP, Balu-Iyer SV. Delivery of therapeutic proteins. J Pharm Sci 2010; 99: 2557-75.
9. Harris JM, Chess RB. Effect of pegylation on pharmaceuticals. Nat Rev Drug Discov 2003; 2: 214-21.
10. Riley T, Riggs-Sauthier J. The Benefits and Challenges of PEGylating Small Molecules. Phar Tech 2008; 32: 1-5.
11. Turcek PL, Scheiflinger F, Siekmann J et al. Biochemical and functional characterization of PEGylated rVWF. Blood 2006; 108: Abstract 1021.
12. Turecek P, Siekmann J, Gritsch H et al. In vitro and in vivo characterization of full-length rFVIII modified with PEG via coupling to primary amino groups. Blood 2007; 110: Abstract 3147.
13. Mei B, Pan C, Jiang H et al. Rational design of a fully active, long-acting PEGylated factor VIII for hemophilia A treatment. Blood 2010; 116: 270-9.
14. Turecek PL, Bossard MJ, Graninger M et al. BAX 855, a PEGylated rFVIII product with prolonged half-life. Development, functional and structural characterisation. Hamostaseologie 2012; 32(Suppl 1): S29-38.
15. Hakobyan N, Enockson C, Cole AA, Sumner DR, Valentino LA. Experimental haemophilic arthropathy in a mouse model of a massive haemarthrosis: gross, radiological and histological changes. Haemophilia 2008; 14: 804-9.
16. Valentino LA, Hakobyan N, Kazarian T, Jabbar KJ, Jabbar AA. Experimental haemophilic synovitis: rationale and development of a murine model of human factor VIII deficiency. Haemophilia 2004; 10: 280-7.
17. Pergantou H, Matsinos G, Papadopoulos A, Platokouki H, Aronis S. Comparative study of validity of clinical, X-ray and magnetic resonance imaging scores in evaluation and management of haemophilic arthropathy in children. Haemophilia 2006; 12: 241-7.
18. Klamroth R, Pollmann H, Hermans C et al. The relative burden of haemophilia A and the impact of target joint development on health-related quality of life: results from the ADVATE Post-Authorization Safety Surveillance (PASS) study. Haemophilia 2011; 17: 412-21.