Skuteczność kliniczna, farmakokinetyka oraz profil bezpieczeństwa rekombinowanego czynnika krzepnięcia IX u pacjentów z hemofilią B – przegląd systematyczny

*Tomasz Tatara

Summary
Introduction. Haemophilia B is a congenital bleeding disorder caused by a decrease of the factor IX activity in plasma. The diagnosis is based on the measurement of clotting factor activity in plasma. Haemophilia can cause spontaneous bleeding into joints and muscles, excessive bleeding after injuries, accidents, tooth extractions, surgical procedures. The mainstay of treatment is to increase the activity of the blood coagulation factor to inhibit the active bleeding or its prevention.
Aim. Evaluation of clinical efficacy and safety profile rFIX in the prevention of bleeding in patients with haemophilia B. Comparison of pharmacokinetics parameters rFIX vs pdFIX.
Material and methods. Evaluation of clinical efficacy based on the results of primary studies and secondary evidence found in the context of a systematic review. The following literature databases were searched systematically: Medline, Embase, Cochrane Library and others.
Results. Prophylactic administration of rFIX reduces the number of spontaneous bleeding and haemarthroses. Recombinant FIX has a lower in vivo recovery (per 1 IU/kg of concentrate) compared to the same dose of pdFIX. The outcomes one of the comparison of the pharmacokinetics parameters were as follows: mean (± SD) in vivo recovery was 1.3 (± 0.4) IU/dl per 1 IU/kg for pdFIX vs 1.0 (± 0.3) IU/dl per 1 IU/kg for rFIX (P < 0.01) – Lissitchov 2013. Most of the reported mild or moderate adverse reactions rFIX (including rash, allergic reactions, fever and infections) were rare.
Conclusions. Based on the results of the analysis it can be concluded that rFIX shows good efficacy in the treatment of bleedings during prophylaxis/on demand treatment in patients with haemophilia B, with favorable safety profile. Due to differences in the pharmacokinetics parameters of rFIX and pdFIX in relation to the level of recovery, each time an analysis of the target rFIX/pdFIX dose is required.

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