Treatment of haemophilia A and B and von Willebrand's disease: Summary and conclusions of a systematic review as part of a Swedish health-technology assessment

Haemophilia

Volumn 18, Issue 2, 2012, Pages 158-165

  Berntorp, E ^a   Astermark, J ^a   Baghaei, F ^b   Bergqvist, D ^c   Holmstrom M ^d   Ljungberg, B ^e   Norlund, A ^f   Palmblad, J ^d   Petrini, P ^d   Stigendal, L ^b   Sawe J ^f  

^a LUND UNIVERSITY HOSPITAL   (Sweden)

^b SAHLGRENSKA UNIVERSITY HOSPITAL   (Sweden)

^c UPPSALA UNIVERSITY HOSPITAL   (Sweden)

^d KAROLINSKA UNIVERSITY HOSPITAL   (Sweden)

^e MEDICAL PRODUCTS AGENCY   (Sweden)

^f Swedish Council on Health Technology Assessment   (Sweden)

Author keywords

By pass therapy; Haemophilia; Health technology assessment; Immune tolerance induction; Inhibitor; Von Willebrand's disease

Indexed keywords

ACTIVATED PROTHROMBIN COMPLEX; BLOOD CLOTTING FACTOR 8; BLOOD CLOTTING FACTOR 8 ANTIBODY; BLOOD CLOTTING FACTOR 8 CONCENTRATE; BLOOD CLOTTING FACTOR 9 CONCENTRATE; BLOOD CLOTTING FACTOR CONCENTRATE; RECOMBINANT BLOOD CLOTTING FACTOR 7A; VON WILLEBRAND FACTOR;

COST EFFECTIVENESS ANALYSIS; DOSE CALCULATION; EVIDENCE BASED MEDICINE; FOLLOW UP; HEMARTHROSIS; HEMOPHILIA A; HEMOPHILIA B; HUMAN; JOINT INJURY; PRIORITY JOURNAL; QUALITY OF LIFE; REVIEW; SUBSTITUTION THERAPY; SYSTEMATIC REVIEW; VON WILLEBRAND DISEASE;

BLOOD COAGULATION FACTORS; HEMOPHILIA A; HEMOPHILIA B; HUMANS; JOINT DISEASES; SWEDEN; VON WILLEBRAND DISEASES;

EID: 84857085385     PISSN: 13518216     EISSN: 13652516     Source Type: Journal    
DOI: 10.1111/j.1365-2516.2011.02723.x     Document Type: Review

References (35)

1. Hemophilia A and B and von Willebrand Disease - a systematic review (SBU); 2011 No 208E. ISBN 978-91-85413-44-7. Available at:.
2. Lusher JM, Arkin S, Abildgaard CF, Schwartz RS. Recombinant factor VIII for the treatment of previously untreated patients with hemophilia A. Safety, efficacy, and development of inhibitors. Kogenate Previously Untreated Patient Study Group. N Engl J Med 1993; 328: 453-9.
3. White GC II, Courter S, Bray GL, Lee M, Gomperts ED. A multicenter study of recombinant factor VIII (Recombinate) in previously treated patients with hemophilia A. The Recombinate Previously Treated Patient Study Group. Thromb Haemost 1997; 77: 660-7.
4. Aygoren-Pursun E, Scharrer I. A multicenter pharmacosurveillance study for the evaluation of the efficacy and safety of recombinant factor VIII in the treatment of patients with hemophilia A. German Kogenate Study Group. Thromb Haemost 1997; 78: 1352-6.
5. Courter SG, Bedrosian CL. Clinical evaluation of B-domain deleted recombinant factor VIII in previously untreated patients. Semin Hematol 2001; 38: 52-9.
6. Yoshioka A, Fukutake K, Takamatsu J, Shirahata A. Kogenate post-marketing surveillance Study G. Clinical evaluation of a recombinant factor VIII preparation (Kogenate) in previously untreated patients with hemophilia A. Int J Hematol 2003; 78: 467-74.
7. Lusher JM, Lee CA, Kessler CM, Bedrosian CL. The safety and efficacy of B-domain deleted recombinant factor VIII concentrate in patients with severe haemophilia A. Haemophilia 2003; 9: 38-49.
8. Lusher J, Abildgaard C, Arkin S et al. Human recombinant DNA-derived antihemophilic factor in the treatment of previously untreated patients with hemophilia A: final report on a hallmark clinical investigation. J Thromb Haemost 2004; 2: 574-83.
9. Powell JS, Bush M, Harrison J et al. Safety and efficacy of solvent/detergent-treated antihaemophilic factor with an added 80 degrees C terminal dry heat treatment in patients with haemophilia A. Haemophilia 2000; 6: 140-9.
10. Tarantino MD, Collins PW, Hay CR et al. Clinical evaluation of an advanced category antihaemophilic factor prepared using a plasma/albumin-free method: pharmacokinetics, efficacy, and safety in previously treated patients with haemophilia A. Haemophilia 2004; 10: 428-37.
11. Nemes L, Lissitchkov T, Klukowska A et al. Evaluation of pharmacokinetics, efficacy and safety of Immunate solvent detergent in previously treated patients with severe haemophilia A. Haemophilia 2007; 13: 9-11.
12. Recht M, Nemes L, Matysiak M et al. Clinical evaluation of moroctocog alfa (AF-CC), a new generation of B-domain deleted recombinant factor VIII (BDDrFVIII) for treatment of haemophilia A: demonstration of safety, efficacy, and pharmacokinetic equivalence to full-length recombinant factor VIII. Haemophilia 2009; 15: 869-80.
13. Bray GL, Gomperts ED, Courter S et al. A multicenter study of recombinant factor VIII (recombinate): safety, efficacy, and inhibitor risk in previously untreated patients with hemophilia A. The Recombinate Study Group. Blood 1994; 83: 2428-35.
14. Smith PS, Teutsch SM, Shaffer PA, Rolka H, Evatt B. Episodic versus prophylactic infusions for hemophilia A: a cost-effectiveness analysis. J Pediatr 1996; 129: 424-31.
15. Rodriguez-Merchan EC, Magallon M. The role of a 3-year period of continuous prophylactic concentrate substitution on later development of haemophilic arthropathy. Haemophilia 1997; 3: 108-10.
16. Abshire TC, Brackmann HH, Scharrer I et al. Sucrose formulated recombinant human antihemophilic factor VIII is safe and efficacious for treatment of hemophilia A in home therapy - International Kogenate-FS Study Group. Thromb Haemost 2000; 83: 811-6.
17. Yee TT, Beeton K, Griffioen A et al. Experience of prophylaxis treatment in children with severe haemophilia. Haemophilia 2002; 8: 76-82.
18. Smith MP, Giangrande P, Pollman H, Littlewood R, Kollmer C, Feingold J. A postmarketing surveillance study of the safety and efficacy of ReFacto (St Louis-derived active substance) in patients with haemophilia A. Haemophilia 2005; 11: 444-51.
19. 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.
20. Blanchette VS, Shapiro AD, Liesner RJ et al. Plasma and albumin-free recombinant factor VIII: pharmacokinetics, efficacy and safety in previously treated pediatric patients. J Thromb Haemost 2008; 6: 1319-26.
21. Collins PW, Blanchette VS, Fischer K et al. Break-through bleeding in relation to predicted factor VIII levels in patients receiving prophylactic treatment for severe hemophilia A. J Thromb Haemost 2009; 7: 413-20.
22. Liesner RJ, Khair K, Hann IM. The impact of prophyactic treatment on children with severe haemophilia. Br J Haematol 1996; 92: 973-8.
23. Feldman BM, Pai M, Rivard GE et al. Tailored prophylaxis in severe hemophilia A: interim results from the first 5years of the Canadian Hemophilia Primary Prophylaxis Study. J Thromb Haemost 2006; 4: 1228-36.
24. Shapiro AD, Donfield SM, Lynn HS et al. Defining the impact of hemophilia: the Academic Achievement in Children with Hemophilia Study. Pediatrics 2001; 108: E105.
25. Miners AH, Sabin CA, Tolley KH, Lee CA. Primary prophylaxis for individuals with severe haemophilia: how many hospital visits could treatment prevent? J Intern Med 2000; 247: 493-9.
26. Schwartz RS, Abildgaard CF, Aledort LM et al. Human recombinant DNA-derived antihemophilic factor (factor VIII) in the treatment of hemophilia A. Recombinant Factor VIII Study Group. N Engl J Med 1990; 323: 1800-5.
27. Scharrer I, Brackmann HH, Sultan Y et al. Efficacy of a sucrose-formulated recombinant factor VIII used for 22 surgical procedures in patients with severe haemophilia A. Haemophilia 2000; 6: 614-8.
28. Negrier C, Shapiro A, Berntorp E et al. Surgical evaluation of a recombinant factor VIII prepared using a plasma/albumin-free method: efficacy and safety of Advate in previously treated patients. Thromb Haemost 2008; 100: 217-23.
29. Van den Berg HM, Fischer K. Prophylaxis for severe hemophilia: experience from Europe and the United States. Semin Thromb Hemost 2003; 29: 49-54.
30. Blanchette VS, Manco-Johnson M, Santagostino E, Ljung R. Optimizing factor prophylaxis for the haemophilia population: where do we stand? Haemophilia 2004; 10(Suppl. 4): 97-104.
31. Carcao MD, Aledort L. Prophylactic factor replacement in hemophilia. Blood Rev 2004; 18: 101-13.
32. Hay CR. Prophylaxis in adults with haemophilia. Haemophilia 2007; 13(Suppl. 2): 10-5.
33. Manco-Johnson M. Comparing prophylaxis with episodic treatment in haemophilia A: implications for clinical practice. Haemophilia 2007; 13(Suppl. 2): 4-9.
34. Musso R. Efficacy and safety of recombinant factor VIII products in patients with hemophilia A. Drugs Today (Barc) 2008; 44: 735-50.
35. Lee C. The use of recombinant factor VIII products in previously treated patients with hemophilia A: pharmacokinetics, efficacy, safety, and inhibitor development. Semin Thromb Hemost 2002; 28: 241-6.