Prophylactic effect of recombinant factor VIIa in factor VII deficient patients.

Abstract:

:Inherited factor VII (FVII) deficiency is a rare autosomal recessive disorder associated with a bleeding tendency. We describe three patients with congenital FVII deficiency who have been treated with activated recombinant factor VII (rVIIa). Two patients had novel mutations and were treated prophylactically with 1.2 mg rVIIa two to three times a week. Patients 1 and 2 had a severe bleeding tendency. The frequency and severity of bleeding decreased by treatment with rVIIa compared with similar treatment with plasma-derived FVII. The third patient with a moderate bleeding phenotype was treated on demand and showed no change in the frequency of bleeding upon treatment with rVIIa or plasma products. The beneficial effect of rVIIa cannot be explained by the rVIIa half-lives. Pharmacokinetical analysis showed rVIIa activity half-lives of 35, 50 and 54 min for patients 1, 2 and 3, respectively. In conclusion, prophylactic treatment of FVII deficient patients with rVIIa appears to be applicable, safe and successful, although the mechanism of action remains to be elucidated.

journal_name

Br J Haematol

authors

Mathijssen NC,Masereeuw R,Verbeek K,Lavergne JM,Costa JM,van Heerde WL,Nováková IR

doi

10.1111/j.1365-2141.2004.04942.x

subject

Has Abstract

pub_date

2004-05-01 00:00:00

pages

494-9

issue

4

eissn

0007-1048

issn

1365-2141

pii

BJH4942

journal_volume

125

pub_type

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