Advances in Clinical and Experimental Medicine

Title abbreviation: Adv Clin Exp Med
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ISSN 1899–5276 (print)
ISSN 2451-2680 (online)
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Advances in Clinical and Experimental Medicine

2012, vol. 21, nr 4, July-August, p. 519–524

Publication type: review article

Language: English

Single Higher Dose of Recombinant Activated Factor VII in the Treatment of Hemorrhages in Patients with Hemophilia Complicated by Inhibitors

Leczenie pojedynczą dużą dawką rFVIIa pacjentów chorych na hemofilię powikłaną inhibitorem

Paweł Łaguna1,, Andrzej Mital2,

1 Department of Pediatrics Hematology and Oncology, Medical University of Warsaw, Warsaw, Poland

2 Department of Hematology and Transplantology, Medical University of Gdansk, Gdańsk, Poland

Abstract

The development of inhibitors is the most severe complication of treating hemophilia patients with Factor VIII or IX, and providing effective hemostasis for inhibitor patients is challenging. Patients with high responding inhibitors (titer >5 BU/mL) are usually treated with recombinant activated Factor VII (rFVIIa), 90–120 μg/kg every 2–3 hours, or plasma-derived activated prothrombin complex concentrate. The aim of this study was to assess the efficacy and safety of a single dose of 270 μg/kg rFVIIa in inhibitor patients with joint or soft tissue bleeds treated at the Department of Paediatric Hematology and Oncology and the Department of Hematology and Transplantology. 7 inhibitor patients (3 adults aged 23–33 and 4 children aged 3–14) were included in the study. The hemostatic efficacy and tolerability of a single high dose of rFVIIa for home treatment of moderate or severe bleeds was evaluated. Treatment with rFVIIa effectively stopped bleeding in all patients without any adverse events. In most cases a single dose of rFVIIa 270 μg/kg was more effective than 3 or 4 lower doses (90 μg/kg) and less traumatic for pediatric patients. Based on present studies, it can be concluded that a single dose of rFVIIa is effective and well tolerated in the treatment of intra-articular bleeds in patients with hemophilia complicated by the presence of inhibitors.

Streszczenie

Najcięższym powikłaniem leczenia czynnikiem VIII i IX pacjentów chorych na hemofilię jest powstawanie inhibitora. Nowe sposoby leczenia tej grupy pacjentów są dużym wyzwaniem dla leczącego. Pacjenci z wysokim mianem inhibitora (> 5 BU/ml) są zwykle leczeni lekami omijającymi inhibitor, takimi jak: rekombinowany czynnik VIIa (rFVIIa),w dawce 90–120 μg/kg co 2–3 godziny lub aktywowanymi koncentratami zespołu protrombiny. Oceniono skuteczność i bezpieczeństwo pojedynczej dawki rFVIIa – 270 μg/kg u pacjentów z inhibitorem i krwawieniami do stawów lub tkanek miękkich, leczonych w Klinice Pediatrii Hematologii i Onkologii i Klinice Hematologii i Transplantologii. Do badania włączono 7 pacjentów powikłanych inhibitorem (3 osoby dorosłe w wieku 23–33 lat i 4 dzieci w wieku 3–14 lat). Oceniono skuteczność i tolerancję pojedynczej dużej dawki rFVIIa w leczeniu domowym umiarkowanych lub ciężkich krwawień. Leczenie rFVIIa skutecznie hamowało krwawienie u wszystkich pacjentów bez wystąpienia skutków ubocznych. W większości przypadków pojedyncza dawka rFVIIa – 270 μg/kg była bardziej skuteczna niż 3 lub 4 mniejsze dawki (90 μg/kg) i mniej urazowa dla pacjentów pediatrycznych, co wiązało się z mniejszą liczbą iniekcji. Na podstawie obserwacji własnych można powiedzieć, że pojedyncza dawka rFVIIa jest skuteczna i dobrze tolerowana w leczeniu krwawień do stawów u pacjentów chorych na hemofilię powikłaną inhibitorem.

Key words

bypassing agents, hemophilia inhibitors, rFVIIa

Słowa kluczowe

leki omijające, hemofilia, inhibitor, rFVIIa

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