Clinical experience with Optivate ®, high-purity factor VIII (FVIII) product with von Willebrand factor (VWF) in young children with haemophilia A

Haemophilia

Volumn 17, Issue 5, 2011, Pages 737-742

  Matysiak, M ^a   Bobrowska, H ^b   Balwierz, W ^c   Chybicka, A ^d   Kowalczyk, J R ^e   Shaikh Zaidi, R ^f   Gillanders, K ^f   Dash, C H ^f  

^a MEDICAL UNIVERSITY OF WARSAW   (Poland)

^b Specialist Centre for Medical Care of Mother and Children   (Poland)

^c JAGIELLONIAN UNIVERSITY MEDICAL COLLEGE   (Poland)

^d WROCLAW MEDICAL UNIVERSITY   (Poland)

^e MEDICAL UNIVERSITY OF LUBLIN   (Poland)

^f BIO PRODUCTS LABORATORY   (United Kingdom)

Author keywords

Children with haemophilia A; Efficacy; Factor VIII; Plasma derived concentrates; Safety

Indexed keywords

BLOOD CLOTTING FACTOR 8 INHIBITOR; RECOMBINANT BLOOD CLOTTING FACTOR 8; VON WILLEBRAND FACTOR;

ARTICLE; BLEEDING; CHILD; CLINICAL ARTICLE; DRUG EFFICACY; DRUG SAFETY; DRUG TOLERABILITY; HEALTH STATISTICS; HEMARTHROSIS; HEMOPHILIA A; HUMAN; INJECTION SITE PAIN; INJECTION SITE RASH; MALE; MULTICENTER STUDY; MUSCLE BLEEDING; OPEN STUDY; PRESCHOOL CHILD; PRIORITY JOURNAL; PROPHYLAXIS; TREATMENT DURATION;

ANALYSIS OF VARIANCE; CHILD; CHILD, PRESCHOOL; COAGULANTS; DRUG COMBINATIONS; FACTOR VIII; HEMOPHILIA A; HEMORRHAGE; HUMANS; MALE; OUTCOME ASSESSMENT (HEALTH CARE); PROSPECTIVE STUDIES; VON WILLEBRAND FACTOR;

EID: 80052021792     PISSN: 13518216     EISSN: 13652516     Source Type: Journal    
DOI: 10.1111/j.1365-2516.2011.02600.x     Document Type: Article

References (27)

1. Petrini P. Treatment strategies in children with hemophilia. Pediatr Drugs 2002; 4: 427-37.
2. Burnouf T. Safety aspects in the manufacturing of plasma-derived coagulation factor concentrates. Biologicals 1992; 20: 91-100.
3. Teitel JM. Viral safety of haemophilia treatment products. Ann Med 2000; 32: 485-92.
4. Brown SA, Dasani H, Collins PW. Long-term follow up of patients treated with intermediate FVIII concentrate BPL 8Y. Haemophilia 1998; 4: 89-93.
5. Yee TT, Williams MD, Hill FG, Lee CA, Pasi KJ. Absence of inhibitors in previously untreated patients with severe haemophilia A after exposure to a single intermediate purity FVIII product. Thromb Haemost 1997; 78: 1027-9.
6. Colvin BT, Rizza RC, Hill GH et al. Effect of dry-heating of coagulation factor concentrates at 80 degrees C for 72hours on transmission of non-A, non-B hepatitis. Study Group of the UK Haemophilia Centre Directors on Surveillance of Virus Transmission by Concentrates. Lancet 1988; 2: 814-6.
7. Lubetsky A, Martinowitz U, Luboshitz J et al. Efficacy and safety of a factor VIII-von Willebrand factor concentrate 8Y: stability, bacteriological safety, pharmacokinetic analysis and clinical experience. Haemophilia 2002; 8: 622-8.
8. Pasi KJ, Williams MD, Enavat MS, Hill FG. Clinical and laboratory evaluation of the treatment of von Willebrand's disease patients with heat-treated factor VIII concentrate (BPL 8Y). Br J Haematol 1990; 75: 228-33.
9. Rizza CR, Fletcher ML, Kernoff PB. Confirmation of viral safety of dry heated factor VIII concentrate (8Y) prepared by Bio Products Laboratory (BPL): a report on behalf of U.K. Haemophilia Centre Directors. Br J Haematol 1993; 84: 269-72.
10. Thomas D. Clotting factor concentrates - whither purity? Thromb Haemost 1995; 74: 1604-6.
11. Pasi KJ, Hill FG. Safety trial of heated factor VIII concentrate (8Y). Arch Dis Child 1989; 64: 1463-7.
12. ®, a high-purity concentrate of factor VIII with von Willebrand factor, in patients with severe haemophilia A. Haemophilia 2011; 17: 185-90.
13. Dash C, Gillanders K, Akanezi C, Shaikh-Zaidi R, Hay C. Pharmacokinetics of Optivate®, a high purity, FVIII and VWF concentrate in VWD patients. Haemophilia 2010; 16(Suppl. 4): 35. (08P07).
14. Dmoszynska A, Kuliczkowski AKK, Hellmann A et al. Clinical assessment of Optivate, a new high-purity concentrate of factor VIII (FVIII) with von Willebrand factor (VWF), in the management of patients with haemophilia A. Haemophilia 2011; in press.
15. Committee for Proprietary Medicinal Products (CPMP). Note for Guidance on the Clinical Investigation of Human Plasma Derived factor VIII and IX Products (CPMP/BPWG/198/95 rev.1). London: 19 October 2000.
16. Hay CRM, Baglin TP, Collins PW et al The diagnosis and management of factor VIII and IX inhibitors; a guideline from the UK Haemophilia Centre Doctors' Organization (UKHCDO). Br J Haematol 2000; 111: 78-90.
17. Van Dijk K, Fischer K, Van Der Bom JG, Grobbee DE, Van Den HM. Variability in clinical phenotype of severe haemophilia: the role of the first joint bleed. Haemophilia 2005; 11: 438-43.
18. Biss TT, Chan AK, Blanchette VS, Iwenofu Mclimont M, Carcao MD. The use of prophylaxis in 2663 children and adults with haemophilia: results of the 2006 Canadian national haemophilia prophylaxis survey. Haemophilia 2008; 14: 923-30.
19. Van den Berg HM, Fischer K. Prophylaxis for severe hemophilia: experience from Europe and the United States. Semin Thromb Hemost 2003; 29: 49-53.
20. Ljung RR. Prophylactic treatment in Sweden-overtreatment or optimal model. Haemophilia 1998; 4: 409-12.
21. White GC. Parachutes and prophylaxis:they both work. Thromb Haemost 2006; 4: 1226-7.
22. Schramm W. Experience with prophylaxis in Germany. Semin Hematol 1993; 30(3 Suppl. 2): 12-5.
23. Nilsson IM. Experience with prophylaxis in Sweden. Semin Hematol 1993; 30(3 Suppl. 2): 16-9.
24. Liesner RJ, Khair K, Hann IM. The impact of prophylactic treatment on children with severe haemophilia. Br J Haematol 1996; 92: 973-8.
25. Windyga J, Lapaciuk P, Stefanska E et al. Haemophilia in Poland. Haemophilia 2006; 12: 52-7.
26. Rosendaal FR, Nieuwenhuis HK, Van Den Berg HM et al. A sudden increase in Factor VIII inhibitor development in multitransfused hemophilia A patients in the Netherlands. Blood 1993; 81: 2180-6.
27. Peerlinck K, Arnout J, Di Giambatista M et al. Factor VIII inhibitors in previously treated Haemophilia A patients with a double virus-inactivated plasma derived factor VIII. Thromb Haemost 1997; 77: 80-6.