Prophylactic effect of recombinant factor VIIa in factor VII deficient patients

被引:34
作者
Mathijssen, NCJ
Masereeuw, RS
Verbeek, K
Lavergne, JM
Costa, JM
van Heerde, WL
Nováková, IRO
机构
[1] UMC St Radboud Nijmegen, Cent Haematol Lab, NL-6500 MB Nijmegen, Netherlands
[2] UMC St Radboud Nijmegen, Dept Pharmacol & Toxicol, Nijmegen Ctr Mol Life Sci, NL-6500 MB Nijmegen, Netherlands
[3] Amer Hosp Paris, Neuilly, France
[4] UMC St RAdboud Nijmegen, Dept Haematol, Nijmegen, Netherlands
关键词
recombinant factor VIIa; congenital factor Vll deficiency; prophylaxis; factor VII; pharmacokinetics;
D O I
10.1111/j.1365-2141.2004.04942.x
中图分类号
R5 [内科学];
学科分类号
1002 ; 100201 ;
摘要
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 I 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.
引用
收藏
页码:494 / 499
页数:6
相关论文
共 18 条
  • [1] The crystal structure of the complex of blood coagulation factor VIIa with soluble tissue factor
    Banner, DW
    DArcy, A
    Chene, C
    Winkler, FK
    Guha, A
    Konigsberg, WH
    Nemerson, Y
    Kirchhofer, D
    [J]. NATURE, 1996, 380 (6569) : 41 - 46
  • [2] Bauer KA, 1996, HAEMOSTASIS, V26, P155
  • [3] Berrettini M, 2001, HAEMATOLOGICA, V86, P640
  • [4] Perioperative pharmacokinetics of factor VII concentrate during liver surgery in a patient with congenital factor VII deficiency: an individual mathematical model
    Charpiat, B
    Laporte, S
    Mismetti, P
    Debize, G
    Ducerf, C
    [J]. BLOOD COAGULATION & FIBRINOLYSIS, 2002, 13 (05) : 457 - 460
  • [5] Factor VII deficiency in pregnancy treated with recombinant factor VIIa
    Eskandari, N
    Feldman, N
    Greenspoon, JS
    [J]. OBSTETRICS AND GYNECOLOGY, 2002, 99 (05) : 935 - 937
  • [6] Giansily-Blaizot M, 2002, HAEMATOLOGICA, V87, P328
  • [7] Recombinant factor Vlla (NovoSeven®) as a hemostatic agent
    Hedner, U
    [J]. DM DISEASE-A-MONTH, 2003, 49 (01): : 39 - 48
  • [8] Recombinant factor VIIa for the treatment of congenital factor VII deficiency
    Hunault, M
    Bauer, KA
    [J]. SEMINARS IN THROMBOSIS AND HEMOSTASIS, 2000, 26 (04) : 401 - 405
  • [9] Use of recombinant factor VIIa in surgery in factor-VII-deficient patients
    Ingerslev, J
    Knudsen, L
    Hvid, I
    Tange, MR
    Fredberg, U
    Sneppen, O
    [J]. HAEMOPHILIA, 1997, 3 (03) : 215 - 218
  • [10] PHARMACOKINETICS AND PHARMACODYNAMICS OF RECOMBINANT FACTOR VIIA
    LINDLEY, CM
    SAWYER, WT
    MACIK, BG
    LUSHER, J
    HARRISON, JF
    BAIRDCOX, K
    BIRCH, K
    GLAZER, S
    ROBERTS, HR
    [J]. CLINICAL PHARMACOLOGY & THERAPEUTICS, 1994, 55 (06) : 638 - 648