Integrated analysis of safety and efficacy of a plasma- and albumin-free recombinant factor VIII (rAHF-PFM) from six clinical studies in patients with hemophilia A

Expert Opinion on Biological Therapy

Volumn 9, Issue 3, 2009, Pages 273-283

  Shapiro, Amy ^a   Gruppo, Ralph ^b   Pabinger, Ingrid ^c   Collins, Peter W ^d   Hay, Charles R M ^e   Schroth, Phillip ^f   Casey, Kathleen ^f   Patrone, Lisa ^f   Ehrlich, Hartmut ^g   Ewenstein, Bruce M ^f  

^a INDIANA HEMOPHILIA AND THROMBOSIS CENTER   (United States)

^b CINCINNATI CHILDREN'S HOSPITAL MEDICAL CENTER   (United States)

^c MEDICAL UNIVERSITY OF VIENNA   (Austria)

^d UNIVERSITY HOSPITAL OF WALES   (United Kingdom)

^e MANCHESTER UNIVERSITY NHS FOUNDATION TRUST   (United Kingdom)

^f BAXTER HEALTHCARE CORPORATION   (United States)

^g BAXTER INNOVATIONS GMBH   (Austria)

Author keywords

Efficacy; Factor VIII; Hemophilia A; Prophylaxis; rAHF PFM; Safety

Indexed keywords

BLOOD CLOTTING FACTOR 8 INHIBITOR; RECOMBINANT BLOOD CLOTTING FACTOR 8; BLOOD CLOTTING FACTOR 8; F8 PROTEIN, HUMAN; SERUM ALBUMIN;

ADOLESCENT; ADULT; AGED; AREA UNDER THE CURVE; ARTICLE; BLEEDING; BLOOD TRANSFUSION; CHILD; CLINICAL EFFECTIVENESS; CLINICAL STUDY; DIARRHEA; DIZZINESS; DRUG CLEARANCE; DRUG DISTRIBUTION; DRUG EFFICACY; DRUG ERUPTION; DRUG HALF LIFE; DRUG INDUCED HEADACHE; DRUG SAFETY; DYSGEUSIA; FEVER; HEMARTHROSIS; HEMOPHILIA A; HOT FLUSH; HUMAN; INFANT; MAJOR CLINICAL STUDY; MAXIMUM PLASMA CONCENTRATION; OUTCOME ASSESSMENT; PATIENT COMPLIANCE; PRESCHOOL CHILD; PROPHYLAXIS; PROSPECTIVE STUDY; PRURITUS; SCHOOL CHILD; SIDE EFFECT; TREATMENT OUTCOME; TREATMENT RESPONSE; IMMUNOLOGY; MAXIMUM TOLERATED DOSE; META ANALYSIS; MIDDLE AGED; SAFETY;

BOVINAE;

ADOLESCENT; ADULT; AGED; CHILD; CHILD, PRESCHOOL; FACTOR VIII; HEMOPHILIA A; HEMORRHAGE; HUMANS; INFANT; MAXIMUM TOLERATED DOSE; MIDDLE AGED; PATIENT COMPLIANCE; PROSPECTIVE STUDIES; SAFETY; SERUM ALBUMIN; TREATMENT OUTCOME; YOUNG ADULT;

EID: 65549169628     PISSN: 14712598     EISSN: None     Source Type: Journal    
DOI: 10.1517/14712590902729392     Document Type: Article

References (82)

1. 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, DOI:10.1542/peds.108.6.e105
2. Dargaud Y, Négrier C. Haemophilia therapies. Expert Opin Biol Ther 2007;7:651-663 (Pubitemid 46707188)
3. Aronstam A, Arblaster PG, Rainsford SG, et al. Prophylaxis in haemophilia: a double-blind controlled trial. Br J Haematol 1976;33:81-90
4. Aronstam A, Kirk PJ, McHardy J, et al. Twice weekly prophylactic therapy in haemophilia A. J Clin Pathol 1977;30:65-67 (Pubitemid 8066381)
5. Löfqvist T, Nilsson IM, Berntorp E, Pettersson H. Haemophilia prophylaxis in young patients-a long-term follow-up. J Intern Med 1997;241:395-400 (Pubitemid 27244664)
6. Aledort LM, Haschmeyer RH, Pettersson H. A longitudinal study of orthopaedic outcomes for severe factor-VIII-deficient haemophiliacs. The Orthopaedic Outcome Study Group. J Intern Med 1994;236:391-399
7. Fischer K, van der Bom JG, Molho P, et al. Prophylactic versus on-demand treatment strategies for severe haemophilia: a comparison of costs and long-term outcome. Haemophilia 2002;8:745-752 (Pubitemid 35446626)
8. Gringeri A. Prospective controlled studies on prophylaxis: an Italian approach. Haemophilia 2003;9(Suppl 1):38-43 (Pubitemid 36622048)
9. Khoriaty R, Taher A, Inati A, Lee C. A comparison between prophylaxis and on demand treatment for severe haemophilia. Clin Lab Haematol 2005;27:320-323 (Pubitemid 41406701)
10. van den Berg HM, Fischer K, van der Bom JG. Comparing outcomes of different treatment regimens for severe haemophilia. Haemophilia 2003;9(Suppl 1):27-31 (Pubitemid 36622046)
11. Schramm W, Royal S, Kroner B, et al. Clinical outcomes and resource utilization associated with haemophilia care in Europe. Haemophilia 2002;8:33-43 (Pubitemid 34257917)
12. Smith PS, Teutsch SM, Shaffer PA, et al. Episodic versus prophylactic infusions for hemophilia A: a cost-effectiveness analysis. J Pediatr 1996;129:424-431 (Pubitemid 27484150)
13. 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;357:535-544 (Pubitemid 47236278)
14. Roosendaal G, Lafeber F. Prophylactic treatment for prevention of joint disease in hemophilia-cost versus benefit. N Engl J Med 2007;357:603-605 (Pubitemid 47236288)
15. Gouw SC, van der Bom JG, van den BMHM. Treatment-related risk factors of inhibitor development in previously untreated patients with hemophilia A: the CANAL cohort study. Blood 2007;109:4648-4654
16. Morado M, Villar A, Jiménez Yuste V, et al. Prophylactic treatment effects on inhibitor risk: experience in one centre. Haemophilia 2005;11:79-83 (Pubitemid 40468053)
17. Berntorp E, Boulyjenkov V, Brettler D, et al. Modern treatment of haemophilia. Bull World Health Organ 1995;73:691-701 (Pubitemid 26020631)
18. Medical and Scientific Advisory Council (MASAC). Recommendation Concerning Prophylaxis (Regular Administration of Clotting Factor Concentrate to Prevent Bleeding). Document #179 (Replaces #170) ed. National Hemophilia Foundation, 2007. Available from: http://www.hemophilia.org/NHFWeb/MainPgs/ MainNHF. aspx?menuid=57&;contentid=1007
19. Ewenstein BM, Collins P, Tarantino MD, et al. Hemophilia therapy innovation: development of an advanced category recombinant factor VIII by a plasma/albumin-free method. Proceedings of a Special Symposium at the XIXth Congress of the International Society on Thrombosis and Haemostasis, July 12-18, 2003, Birmingham, UK. Semin Hematol 2004;41:1-16
20. Shapiro AD. Anti-hemophilic factor (recombinant), plasma/albumin-free method (octocog-alpha; ADVATE) in the management of hemophilia A. Vasc Health Risk Manag 2007;3:1-11 (Pubitemid 350104112)
21. 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;10:428-437 (Pubitemid 39328049)
22. Blanchette VS, Shapiro AD, Liesner RJ, et al. Plasma and albumin free recombinant factor VIII (rAHF-PFM): pharmacokinetics, efficacy and safety in previously treated pediatric patients. J Thromb Haemost 2008;6:1319-1326 (Pubitemid 352016311)
23. Morfini M, Lee M, Messori A. The design and analysis of half-life and recovery studies for factor VIII and factor IX. Factor VIII/Factor IX Scientific and Standardization Committee of the International Society for Thrombosis and Haemostasis. Thromb Haemost 1991;66:384-386
24. Négrier 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;100:217-223
25. Tarantino MD, Collins PW, Hay PW, 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;10:428-437 (Pubitemid 39328049)
26. Liesner RJ, Khair K, Hann IM. The impact of prophylactic treatment on children with severe haemophilia. Br J Haematol 1996;92:973-978 (Pubitemid 26109507)
27. Manco-Johnson MJ, Nuss R, Geraghty S, et al. Results of secondary prophylaxis in children with severe hemophilia. Am J Hematol 1994;47:113-117 (Pubitemid 24302280)
28. Nilsson IM, Berntorp E, Löfqvist T, Pettersson H. Twenty-five years' experience of prophylactic treatment in severe haemophilia A and B. J Intern Med 1992;232:25-32
29. Petrini P, Lindvall N, Egberg N, Blombäck M. Prophylaxis with factor concentrates in preventing hemophilic arthropathy. Am J Pediatr Hematol Oncol 1991;13:280-287
30. Blanchette VS, Shapiro AD, Liesner RJ, et al. Plasma and albumin free recombinant factor VIII (rAHF-PFM): pharmacokinetics, efficacy and safety in previously treated pediatric patients. J Thromb Haemost 2008;6:1319-1326 (Pubitemid 352016311)
31. Verbruggen B, Novakova I, Wessels H, et al. The Nijmegen modification of the Bethesda assay for Factor VIII: C inhibitors: improved specificity and reliability. Thromb Haemost 1995;73:247-251
32. Björkman S. Prophylactic dosing of factor VIII and factor IX from a clinical pharmacokinetic perspective. Haemophilia 2003;9(Suppl 1):101-108 (Pubitemid 36622057)
33. Carlsson M, Berntorp E, Björkman S, et al. Improved cost-effectiveness by pharmacokinetic dosing of factor VIII in prophylactic treatment of haemophilia A. Haemophilia 1997;3:96-101 (Pubitemid 27187173)
34. Efficacy of antihemophilic factor (recombinant), plasma/albumin-free method (rAHF PFM) in bleed treatment. B Ewenstein, L Patrone, P Schroth, K Casey, S Fritsch, B Pavlova, H Ehrlich; Baxter BioScience, Westlake Village, CA and Vienna, Austria. Poster presented at XXI ISTH Congress. July 6-12, 2006; Geneva, Switzerland
35. Efficacy of antihemophilic factor (recombinant), plasma/albumin-free method (rAHF PFM) in bleed prevention. B Ewenstein, L Patrone, P Schroth, G Spotts, S Fritsch, B Pavlova, H Ehrlich; Baxter BioScience, Westlake Village, CA and Vienna, Austria. Poster presented at XXI ISTH Congress. July 6-12, 2006; Geneva, Switzerland
36. Konkle BA, Ebbesen LS, Erhardtsen E, et al. Randomized, prospective clinical trial of recombinant factor VIIa for secondary prophylaxis in hemophilia patients with inhibitors. J Thromb Haemost 2007;5:1904-1913 (Pubitemid 47288949)
37. Lorenzo JI, Lopez A, Altisent C, Aznar JA. Incidence of factor VIII inhibitors in severe haemophilia: the importance of patient age. Br J Haematol 2001;113:600-603 (Pubitemid 32524377)
38. Chalmers EA, Brown SA, Keeling D, et al. Early factor VIII exposure and subsequent inhibitor development in children with severe haemophilia A. Haemophilia 2007;13:149-155 (Pubitemid 46237946)
39. Oldenburg J, El Maarri O, Schwaab R. Inhibitor development in correlation to factor VIII genotypes. Haemophilia 2002;8 (Suppl 2):23-29 (Pubitemid 34506224)
40. Astermark J, Oldenburg J, Pavlova A, et al. Polymorphisms in the IL10 but not in the IL1beta and IL4 genes are associated with inhibitor development in patients with hemophilia A. Blood 2006;107:3167-3172
41. Astermark J, Oldenburg J, Carlson J, et al. Polymorphisms in the TNFA gene and the risk of inhibitor development in patients with hemophilia A. Blood 2006;108:3739-3745 (Pubitemid 44864553)
42. Santagostino E, Mancuso ME, Rocino A, et al. Environmental risk factors for inhibitor development in children with haemophilia A: a case-control study. Br J Haematol 2005;130:422-427 (Pubitemid 43899592)
43. Ehrlich HJ, Bray GL, Gomperts ED. Comparison of high responder inhibitor frequency in recent studies of previously untreated patients with hemophilia A. Thromb Haemost 1998;79:242-243
44. Kreuz W, Ettingshausen CE, Zyschka A, et al. Inhibitor development in previously untreated patients with hemophilia A: a prospective long-term follow-up comparing plasma-derived and recombinant products. Semin Thromb Hemost 2002;28:285-290 (Pubitemid 34634343)
45. Hay CR. Factor VIII inhibitors in mild and moderate-severity haemophilia A. Haemophilia 1998;4:558-563 (Pubitemid 28351885)
46. Luu H, Kriukov A, Stephens D, et al. The safety and efficacy of ADVATE [Antihemophilic Factor (recombinate), Plasma/Albumin-Free Method (rAHF-PFM)] in routine clinical practice: Results from post-authorization safety surveillance (PASS) in PUPs and PTPs following product switch. Poster presented at 50th Annual Meeting of the American Society of Hematology. December 6-9, 2008; San Francisco, CA
47. 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;83:811-816 (Pubitemid 30411675)
48. Courter SG, Bedrosian CL. Clinical evaluation of B-domain deleted recombinant factor VIII in previously treated patients. Semin Hematol 2001;38:44-51 (Pubitemid 32612098)
49. 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
50. White GC, Courter S, Bray GL, et al. 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;77:660-667 (Pubitemid 27182960)
51. Yoshioka A, Shima M, Fukutake K, et al. Safety and efficacy of a new recombinant FVIII formulated with sucrose (rFVIII-FS) in patients with haemophilia A: a long-term, multicentre clinical study in Japan. Haemophilia 2001;7:242-249 (Pubitemid 32471957)
52. Barnes C, Lillicrap D, Pazmino-Canizares J, et al. Pharmacokinetics of recombinant factor VIII (Kogenate-FS) in children and causes of inter-patient pharmacokinetic variability. Haemophilia 2006;12:40-49 (Pubitemid 44404838)
53. Kelly KM, Butler RB, Farace L, et al. Superior in vivo response of recombinant factor VIII concentrate in children with hemophilia A. J Pediatr 1997;130:537-540 (Pubitemid 27492642)
54. Matucci M, Messori A, Donati-Cori G, et al. Kinetic evaluation of four factor VIII concentrates by model-independent methods. Scand J Haematol 1985;34:22-28 (Pubitemid 15192981)
55. DiMichele DM. Management of factor VIII inhibitors. Int J Hematol 2006;83:119-125 (Pubitemid 44227957)
56. Shapiro AD, Korth-Bradley J, Poon MC. Use of pharmacokinetics in the coagulation factor treatment of patients with haemophilia. Haemophilia 2005;11:571-582 (Pubitemid 41631591)
57. Berntorp E, Björkman S. The pharmacokinetics of clotting factor therapy. Haemophilia 2003;9:353-359 (Pubitemid 36874381)
58. Lee M, Morfini M, Négrier C, Chamouard V. The pharmacokinetics of coagulation factors. Haemophilia 2006;12(Suppl 3):1-7
59. Morfini M, Cinotti S, Bellatreccia A, et al. A multicenter pharmacokinetic study of the B-domain deleted recombinant factor VIII concentrate using different assays and standards. J Thromb Haemost 2003;1:2283-2289
60. 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 (Pubitemid 36511631)
61. Roosendaal G, Lafeber FP. Pathogenesis of haemophilic arthropathy. Haemophilia 2006;12(Suppl 3):117-121
62. Goddard NJ, Mann H. Diagnosis of haemophilic synovitis. Haemophilia 2007;13(Suppl 3):14-19 (Pubitemid 47377654)
63. Luck JV Jr, Silva M, Rodriguez-Merchan EC, et al. Hemophilic arthropathy. J Am Acad Orthop Surg 2004;12:234-245
64. Gilbert MS, Radomisli TE. Therapeutic options in the management of hemophilic synovitis. Clin Orthop Relat Res 1997;343:88-92 (Pubitemid 27450802)
65. Wallny T, Lahaye L, Brackmann HH, et al. Clinical and radiographic scores in haemophilic arthropathies: how well do these correlate to subjective pain status and daily activities? Haemophilia 2002;8:802-808
66. van Genderen FR, Fischer K, Heijnen L, et al. Pain and functional limitations in patients with severe haemophilia. Haemophilia 2006;12:147-153
67. Choiniére M, Melzack R. Acute and chronic pain in hemophilia. Pain 1987;31:317-331 (Pubitemid 18184420)
68. Pollmann H, Externest D, Ganser A, et al. Efficacy, safety and tolerability of recombinant factor VIII (REFACTO) in patients with haemophilia A: interim data from a postmarketing surveillance study in Germany and Austria. Haemophilia 2007;13:131-143 (Pubitemid 46237944)
69. Khoriaty R, Taher A, Inati A, Lee C. A comparison between prophylaxis and on demand treatment for severe haemophilia. Clin Lab Haematol 2005;27:320-323 (Pubitemid 41406701)
70. Janco RL, Maclean WE, Perrin JM, Gortmaker SL. A prospective study of patterns of bleeding in boys with haemophilia. Haemophilia 1996;2:202-206 (Pubitemid 26379914)
71. White GC II, Courter S, Bray GL, et al. A multicenter study of recombinant factor VIII (Recombinate) in previously treated patients with hemophilia A. Thromb Haemost 1997;77:660-667 (Pubitemid 27182960)
72. Pollmann H, Richter H, Ringkamp H, Jürgens H. When are children diagnosed as having severe haemophilia and when do they start to bleed? A 10-year single-centre PUP study. Eur J Pediatr 1999;158(Suppl 3):S166-70 (Pubitemid 30014028)
73. van Dijk K, Fischer K, van der Bom JG, et al. Variability in clinical phenotype of severe haemophilia: the role of the first joint bleed. Haemophilia 2005;11:438-443 (Pubitemid 41283110)
74. Onwuzurike N, Warrier I, Lusher JM. Types of bleeding seen during the first 30 months of life in children with severe haemophilia A and B. Haemophilia 1996;2:137-140 (Pubitemid 26248837)
75. 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 (Pubitemid 34257918)
76. Fischer K, van der Bom JG, Mauser-Bunschoten EP, et al. Changes in treatment strategies for severe haemophilia over the last 3 decades: effects on clotting factor consumption and arthropathy. Haemophilia 2001;7:446-452 (Pubitemid 32923247)
77. Fischer K, van der Bom JG, Molho P, et al. Prophylactic versus on-demand treatment strategies for severe haemophilia: a comparison of costs and long-term outcome. Haemophilia 2002;8:745-752 (Pubitemid 35446626)
78. Hacker MR, Geraghty S, Manco-Johnson M. Barriers to compliance with prophylaxis therapy in haemophilia. Haemophilia 2001;7:392-396 (Pubitemid 32677658)
79. La Greca AM. Issues in adherence with pediatric regimens. J Pediatr Psychol 1990;15:423-436 (Pubitemid 20366292)
80. Kyngas H. Compliance of adolescents with chronic disease. J Clin Nurs 2000;9:549-556
81. World Health Organization. Adherence to long-term therapies project report. In: Sabate E, editor, A Global Initiative Launched in 2001 by the Non-communicable Diseases and Mental Health Cluster of the World Health Organization.Geneva, Switzerland: World Health Organization, 2003. p. 22
82. du Treil S, Rice J, Leissinger CA. Quantifying adherence to treatment and its relationship to quality of life in a well-characterized haemophilia population. Haemophilia 2007;13:493-501