Use of secondary prophylaxis with recombinant activated factor VII in haemophilia A with inhibitor: our experience and the review of the literature
Authors:
P. Kubisz 1; I. Plameňová 1; J. Staško 1; P. Hollý 1; D. Mištuna 2; M. Mýtnik 3
Authors‘ workplace:
Klinika hematológie a transfuziológie, Národné centrum hemostázy a trombózy, Jesseniova lekárska fakulta
1; Univerzity Komenského a Martinská fakultná nemocnica, Martin, SR, 1. chirurgická klinika, Jesseniova
lekárska fakulta Univerzity Komenského a Martinská fakultná nemocnica, Martin, 3Chirurgická klinika
Reimanovej fakultnej nemocnice s poliklinikou, Prešov,
2
Published in:
Transfuze Hematol. dnes,15, 2009, No. 4, p. 210-215.
Category:
Comprehensive Reports, Original Papers, Case Reports
Overview
Coagulation factor VIII (FVIII) inhibitors are the serious complication of replacement therapy with FVIII in hemophilia A. The treatment of hemophiliacs with inhibitor who are not eligible for the immune tolerance induction (ITT) or in whom it fails is problematic. The prophylaxis with bypassing agents (FEIBA, recombinant activated coagulation factor VII (rFVIIa) have been recently studied as a promising alternative. A case of a patient with congenital hemophilia A, in whom the FVIII inhibitor was identified at the age of 3 after more than 50 exposures to human plasma FVIII concentrates is presented. The patient underwent the unsuccessful ITT according to the modified Bonn regimen and his condition was complicated with the developement of the hemophilic arthropathy of the left knee. In order to allow the invasive local treatment (synovectomy, intra-articular glucocorticoids) and intensive physiotherapy the secondary prophylaxis with rFVIIa at the dose of 120 μg/kg 2-3 times a week plus similar dose before the increased physical activity was conducted for 18 months. The prophylactic treatment led to the reduction of the bleeding and the improvement in the quality of life. No progression of the arthropathy as well as no severe bleeding related to the rehabilitation procedures was observed.
Key words:
hemophilia A, FVIII inhibitors, prophylaxis, rFVIIa, hemophilic arthropathy
Sources
1. Wight J, Paisley S. The epidemiology of inhibitors in haemophilia A: a systemic review. Haemophilia 2003; 9: 418-435.
2. Rocino A, Santagostino E, Mancuso ME, Mannucci PM. Immune tolerance induction with recombinant factor VIII in hemophilia A patients with high responding inhibitors. Haematologica, 2006; 91: 558-561.
3. Rodriguez-Merchan EC, Rocino A. Literature review of surgery management in inhibitor patients. Haemophilia 2004; 10 (Suppl. 2): 22-29.
4. Abshire T, Kenet G. Safety update on the use of recombinant factor VIIa and the treatment of congenital and acquired deficiency of factor VIII or IX with inhibitors. Haemophilia 2008; 14: 898-902.
5. 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.
6. Jimenez-Yuste V, Alvarez MT, Marín-Salces M, et al. Prophylaxis in 10 patients with severe haemophilia A and inhibitor: different approaches for different clinical situations. Haemophilia 2008; 14: 1-7.
7. Young G, McDaniel M, Nugent D. Prophylactic recombinant factor VIIa in haemophilia patients with inhibitors. Haemophilia 2005; 11: 203-207.
8. Cooper H, Jones C, Campion E, et al. Rationale for the use of high dose rFVIIa in a high-titre inhibitor patient with haemophilia B during major orthopaedic procedures. Haemophilia 2001; 7: 517-522.
9. Saxon BR, Shanks D, Jory CB, et al. Effective prophylaxis with daily recombinant factor VII (rFVIIa-Novoseven) in a child with high titre inhibitors and a target joint. Thromb Haemost 2001; 86: 1126-1127.
10. Jimenez-Yuste V, Quintana M, Alvarez MT, et al. “Primary prophylaxis“ with rFVIIa in a patient with severe haemophilia A and inhibitor. Blood Coagul Fibrinolysis 2008; 19: 719-720.
11. Blatny J, Kohlerova S, Zapletal O, et al. rFVIIa is effective alternative to aPCC in prophylaxis of bleeding during immune tolerance protocol in haemophiliac with inhibitor. Abstract no. P-M-166, XXIst ISTH Congress, Geneva, July 6–11, 2007.
12. Lopez Fernandez M, Andon Saavedra C, Amor Otero MA, et al. “Primary“ prophylactic treatment with recombinant activated factor VII (rFVIIa) during immune tolerance in a haemophilic child. Abstract no. P-M-161, XXIst ISTH Congress, Geneva, July 6–11, 2007.
13. Marques-Verdier A, Chaleteix C, Soulie B, et al. Secondary prophylaxis with recombinant activated factor VII (rFVIIa) in a severe haemophilia A patient with anti-factor VIII inhibitors. Abstract no. P-T-168, XXIst ISTH Congress, Geneva, July 6–11, 2007.
14. Molina MQ, Bello IF, Jimenez-Yuste V, et al. Prophylaxis with recombinant FVIIa. Abstract no. P-T-170, XXIst ISTH Congress, Geneva, July 6–11, 2007.
15. Tagliaferri A, Rivolta G, De Fanti A, et al. Prophylaxis with rFVIIa during immune tolerance treatment in a haemophiliac child with a target joint. Haemophilia 2006; 12 (Suppl. 2): Abstract no. 23 PO 718.
16. Morfini M, Auerswald G, Kobelt RA, et al. Prophylactic treatment of haemophilia patients with inhibitors: clinical experience with recombinant factor VIIa in European Haemophilia Centres. Haemophilia 2007; 13: 502-507.
17. Hoots WK, Ebbesen LS, Konkle BA, et al. Secondary prophylaxis with recombinant activated factor VII improves health-related quality of life of haemophilia patients with inhibitors. Haemophilia 2008; 14: 466-475.
18. Blatny J, Kohlerova S, Zapletal O, et al. Prophylaxis with recombinant factor VIIa for the management of bleeding episodes during immune tolerance treatment in a boy with severe haemophilia A and high-response inhibitors. Haemophilia 2008; 14: 1140-1142.
19. Brackmann HH, Effenberger E, Hess L, et al. NovoSeven in immune tolerance therapy. Blood Coagul Fibrinolysis 2000; 11 (Suppl. 1): 39-44.
20. Hedner U. Potential role of recombinant factor VIIa in prophylaxis in severe hemophilia patients with inhibitors. J Thromb Haemost 2006; 4: 2498-2500.
21. Miller GJ, Howarth DJ, Attfield JC, et al. Haemostatic factors in human peripheral afferent lymph. Thromb Haemost 2000; 83: 427-432.
22. Almus FE, Rao LV, Fleck RA, et al. Properties of factor VIIa/tissue factor complexes in an umbilical vein model. Blood 1990; 76: 354-360.
23. Leissinger CA, Beeton DL, Ewing NP, et al. Prophylactic treatment with activated prothrombin complex concentrate (FEIBA) reduces the frequency of bleeding episodes in paediatric patients with haemophilia A and inhibitors. Haemophilia 2007; 13: 249-255.
24. Hilgartner MW, Makipernaa A, DiMichele DM. Long-term FEIBA prophylaxis does not prevent progression of existing joint disease. Haemophilia 2003; 9: 261-268.
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Haematology Internal medicine Clinical oncologyArticle was published in
Transfusion and Haematology Today
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