Efficacy and safety of BAY 81-8973, a full-length recombinant factor VIII: results from the LEOPOLD I trial

Haemophilia

Volumn 22, Issue 5, 2016, Pages 706-712

  Saxena, K ^a,f   Lalezari, S ^b   Oldenburg, J ^c   Tseneklidou Stoeter, D ^d   Beckmann, H ^d   Yoon, M ^e   Maas Enriquez, M ^d  

^a BOSTON CHILDREN S HOSPITAL   (United States)

^b SHEBA MEDICAL CENTER   (Israel)

^c UNIVERSITY HOSPITAL BONN   (Germany)

^d BAYER PHARMA AG   (Germany)

^e Bayer Inc   (Canada)

^f BAYER CORPORATION   (United States)

Author keywords

clinical studies; efficacy; factor VIII; haemophilia A; prophylaxis; surgery

Indexed keywords

BLOOD CLOTTING FACTOR 8; BLOOD CLOTTING FACTOR 8 ANTIBODY; RECOMBINANT BLOOD CLOTTING FACTOR 8;

ADOLESCENT; ADULT; AGED; ANNUALIZED BLEEDING RATE; ARTICLE; BLEEDING; CARDIOVASCULAR RISK; CHILD; CONTROLLED STUDY; CROSSOVER PROCEDURE; DISEASE SEVERITY; DRUG EFFICACY; DRUG HALF LIFE; DRUG POTENCY; DRUG SAFETY; HEMATOLOGICAL PARAMETERS; HEMOPHILIA A; HEMOSTASIS; HUMAN; INCIDENCE; MAJOR CLINICAL STUDY; MAJOR SURGERY; MALE; MIDDLE AGED; MINOR SURGERY; OPEN STUDY; OPERATIVE BLOOD LOSS; PHASE 1 CLINICAL TRIAL; PHASE 3 CLINICAL TRIAL; PRIORITY JOURNAL; PROPHYLAXIS; RANDOMIZED CONTROLLED TRIAL; SIDE EFFECT; SINGLE DRUG DOSE;

EID: 84990206286     PISSN: 13518216     EISSN: 13652516     Source Type: Journal    
DOI: 10.1111/hae.12952     Document Type: Article

References (14)

1. Valentino LA. Blood-induced joint disease: the pathophysiology of hemophilic arthropathy. J Thromb Haemost 2010; 8: 1895–902.
2. 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.
3. Royal S, Schramm W, Berntorp E et al. Quality-of-life differences between prophylactic and on-demand factor replacement therapy in European haemophilia patients. Haemophilia 2002; 8: 44–50.
4. Vogel JH, Huesslein A, Goudar C et al. BAY 81-8973 – a new full-length recombinant FVIII product using novel manufacturing technologies. Haemophilia 2010; 16: 40.
5. Ishaque A, Thrift J, Murphy JE, Konstantinov K. Over-expression of Hsp70 in BHK-21 cells engineered to produce recombinant factor VIII promotes resistance to apoptosis and enhances secretion. Biotechnol Bioeng 2007; 97: 144–55.
6. Shah A, Delesen H, Garger S, Lalezari S. Pharmacokinetic properties of BAY 81-8973, a full-length recombinant factor VIII. Haemophilia 2015; 21: 766–771.
7. Sommer JM, Moore N, McGuffie-Valentine B et al. Comparative field study evaluating the activity of recombinant factor VIII Fc fusion protein in plasma samples at clinical haemostasis laboratories. Haemophilia 2014; 20: 294–300.
8. Mikaelsson M, Oswaldsson U, Sandberg H. Influence of phospholipids on the assessment of factor VIII activity. Haemophilia 1998; 4: 646–50.
9. Kavakli K, Yang R, Rusen L, Beckmann H, Tseneklidou-Stoeter D, Maas Enriquez M. Prophylaxis versus on-demand treatment with BAY 81-8973, a full-length plasma-protein–free rFVIII product: results from a randomized trial (LEOPOLD II). J Thromb Haemost 2015; 13: 360–9.
10. Lee M, Morfini M, Negrier C, Chamouard V. The pharmacokinetics of coagulation factors. Haemophilia 2006; 12(Suppl. 3): 1–7.
11. European Medicines Agency. Guideline on the clinical investigation of recombinant and human plasma-derived factor VIII products. European Medicines Agency. Available at http://www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2015/06/WC500187409.pdf. Accessed October 7, 2015.
12. ® FS (antihemophilic factor [recombinant] formulated with sucrose). Full Prescribing Information, Bayer, Tarrytown, NY, 2013.
13. Richards M, Williams M, Chalmers E et al. A United Kingdom Haemophilia Centre Doctors' Organization guideline approved by the British Committee for Standards in Haematology: guideline on the use of prophylactic factor VIII concentrate in children and adults with severe haemophilia A. Br J Haematol 2010; 149: 498–507.
14. Feldman BM, Pai M, Rivard GE et al. Tailored prophylaxis in severe hemophilia A: interim results from the first 5 years of the Canadian Hemophilia Primary Prophylaxis Study. J Thromb Haemost 2006; 4: 1228–36.