İnhibitörlü Hemofili A Hastalarında Profilaksi

Öz

Aktive protrombin kompleks konsantresi (aPCC) ve rekombinant faktör VIIa (rFVIIa) inhibitör geliştirmiş hemofili hastalarının kanama tedavisinde ve profilaksisinde kullanılmaktadır. Ülkemizde rFVIIa için profilakside endikasyon onayı ve geri ödemesi henüz yoktur. Bu yazıda inhibitörlü hastada aPCC’nin doz ve etkinliği değerlendirilmiştir. Her üç vakamızda da aPCC profilaksisi alırken kanamanın olmaması, iki hastada profilaksi kesildikten sonra tekrarlayan kanamaların görülmesi profilaksi süresinin uzatılabileceğini desteklemektedir.

Anahtar Kelimeler

• Hemofili A, inhibitör, profilaksi

PROPHYLAXIS FOR HEMOPHILIA A PATIENTS WITH INHIBITORS

Öz

Use of activated prothrombin complex concentrate (aPCC) and recombinant factor VIIa (rFVIIa) is indicated for the treatment and prophylaxis of hemophiliac patients who has developed inhibitors. rFVIIa is an off labeled drug for prophylaxis in Turkey and not covered by health insurance. In this manuscript, the efficacy and dosage for aPCC prophylaxis were evaluated. During the aPCC prophylaxis, bleeding did not occur in any of the three cases. However, two patients developed recurrent bleeding attacks after the prophylaxis ceased. Therefore, we suggest that prophylaxis by aPCC should be prolonged to prevent recurrent bleeding attacks in hemophiliacs who developed inhibitors

Anahtar Kelimeler

• Hemophilia A, inhibitory, prophylaxis

Kaynakça

1. Kavaklı K, Şahin F, Demir M, THD Hemofili Çalışma Grubu. He- mofilide İnhibitör Sorunu Tanı ve Tedavi Kılavuzu, Ulusal Tedavi Kılavuzu. 2011; IV. Bölüm: 45-54.
2. Mathev P. Current opinion on inhibitor treatment options. Semin Hematol 2006;43(2 Suppl 4):S8-13.
3. Lessinger C, Gringeri A, Antmen B, Berntrop E, Biasoli C, Car- penter S, et al. Anti- inhibitor Coagulant Complex Prophylaxis in Hemophilia with İnhibitors. N Engl J Med 2011;365(18):1684-92.
4. de Paula EV, Kavakli K, Mahlangu J, Ayob Y, Lentz SR, Morfi- ni M, et al. Recombinate factor VIIa analog (vaterptacog alfa [ac- tivated] for treatment of joint bleeds in hemophilia patients with inhibitors: a randomized controlled trial. J Thromb Haemost. 2012;10(1):81-9.
5. Maclean PS, Richards M, Williams M, Collins P, Liesner R, et al. Treatment related factors and inhibitors development in children with severe hemophilia A. Haemophilia 2011;17(2):282-7.
6. Dimichele D, Negrier C. A retrospective postlicensure survey of FEIBA efficacy and safety. Haemophilia 2006;12(4):352-62.
7. Valentino LA. Assing the benefits of FEIBA prophylaxis in He- mophilia Patients with Inhibitors: A Meta-Analysis. Haemophilia 2008;14(Suppl 2):54(08 PO 73).
8. Jimanez- Yuste V, Radriguez_ Merchan EC, Alvarez MT, Quintana M, Martin-Salces M, Hernandez-Navarro F. Eperiences in the pre- vention of arthrophaty in hemophilia patients with inhibitors. Hae- mophilia 2008;14 Suppl 6:28-35.

9. Ewenstein B, Giangrande P, Morfini M,Tjonnfjord GE, Kraut E, Luu H. Evaluation of FEIBA for prophylaxis in patients with inhi- bitors. Haemophilia 2004; 10(Suppl 3): 114(22 PO 10).
10. Ewing NP, Pullens L, De Guzman C. Anamnesis in patients with hemophilia and inhibitors who receive activated prothrombin complex concentrates for prophylaxis. J Thromb Hemost 2007; 5(Suppl 2): (P_T_158).
11. Negrier C, Goudemand J, Sultan Y, Bertrand M, Rothschild C, La- uroua P. Multicenter retrospective study on the utilization of FE- IBA in France in patients with factor VIII and factor IX inhibi- tors. French FEIBA study Group, Factor Eight Bypassing Activity. Thromb Haemost 1997;77(6):1113-9.