The use of recombinant factor VIIa in children with inherited platelet function disorders

被引:120
作者
Almeida, AM [1 ]
Khair, K [1 ]
Hann, I [1 ]
Liesner, R [1 ]
机构
[1] Great Ormond St Hosp Sick Children, Haemophilia Ctr, Dept Haematol, London WC1N 3JH, England
关键词
platelet dysfunction; factor VIIa; bleeding; efficacy;
D O I
10.1046/j.1365-2141.2003.04286.x
中图分类号
R5 [内科学];
学科分类号
1002 ; 100201 ;
摘要
Inherited deficiencies of platelet surface glycoproteins such as Glanzmann's thrombasthenia (GT) or Bernard-Soulier syndrome (BSS) can lead to a severe bleeding diathesis. In the past, bleeding episodes in these patients have often required platelet transfusion to secure haemostasis but recently a number of patient reports have suggested that recombinant factor VIIa (rVIIa) may also be effective. We have used rVIIa on 33 occasions in seven children with inherited platelet function disorders over a 2-year period: five had GT, one had BSS and one had storage pool disease with a severe phenotype. Bleeding ceased with rVIIa alone in 10 of 28 acute bleeding episodes, but recurred in two of these. The two features that predicted response to rVIIa were the severity of the bleeding and the delay from the onset of bleeding to treatment. Five episodes of planned surgical intervention were treated successfully with rVIIa. Eighteen out of the 28 acute episodes and none of the planned surgical episodes required blood product support. We have found variable efficacy of rVIIa for acute bleeding episodes in this small series of children with inherited platelet function defects but larger studies are warranted, particularly as rVIIa is a relatively low-risk treatment approach for these disorders.
引用
收藏
页码:477 / 481
页数:5
相关论文
共 21 条
[11]   High-dose factor VIIa increases initial thrombin generation and mediates faster platelet activation in thrombocytopenia-like conditions in a cell-based model system [J].
Kjalke, M ;
Ezban, M ;
Monroe, DM ;
Hoffman, M ;
Roberts, HR ;
Hedner, U .
BRITISH JOURNAL OF HAEMATOLOGY, 2001, 114 (01) :114-120
[12]   Platelets and thrombin generation [J].
Monroe, D ;
Hoffman, M ;
Roberts, HR .
ARTERIOSCLEROSIS THROMBOSIS AND VASCULAR BIOLOGY, 2002, 22 (09) :1381-1389
[13]   Platelet activity of high-dose factor VIIa is independent of tissue factor [J].
Monroe, DM ;
Hoffman, M ;
Oliver, JA ;
Roberts, HR .
BRITISH JOURNAL OF HAEMATOLOGY, 1997, 99 (03) :542-547
[14]   Peripartum management of a patient with Glanzmann's thrombasthenia using Thrombelastograph® [J].
Monte, S ;
Lyons, G .
BRITISH JOURNAL OF ANAESTHESIA, 2002, 88 (05) :734-738
[15]  
Peters M, 1998, THROMB HAEMOSTASIS, V80, P352
[16]   Recombinant activated factor VII (NovoSeven®) treatment of platelet-related bleeding disorders [J].
Poon, MC ;
d'Oiron, R .
BLOOD COAGULATION & FIBRINOLYSIS, 2000, 11 :S55-S68
[17]   Recombinant factor VIIa is effective for bleeding and surgery in patients with Glanzmann thrombasthenia [J].
Poon, MC ;
Demers, C ;
Jobin, F ;
Wu, JWY .
BLOOD, 1999, 94 (11) :3951-3953
[18]  
POON MC, 2001, SEMINARS HAEMATOLOGY, V38, pS21
[19]  
Tengborn L, 1996, THROMB HAEMOSTASIS, V75, P981
[20]   Recombinant factor VIIa for bleeding in refractory thrombocytopenia [J].
Vidarsson, B ;
Önundarson, PT .
THROMBOSIS AND HAEMOSTASIS, 2000, 83 (04) :634-635