Replacement therapy for bleeding episodes in factor VII deficiency: A prospective evaluation

Mariasanta Napolitano, Jorgen Ingerslev, Benny Sørensen, Mehran Karimi, Anne-Marie Bertrand, Giovanni Di Minno, Jens Bjerre, Arlette Ruiz-Saez, Alberto Dolce, Angelika Batorova, Günter Auerswald, Rosario Perez Garrido, Jean F. Schved, Guglielmo Mariani, Helen Platokouki, Alberto Dolce, Guglielmo Mariani

Risultato della ricerca: Article

35 Citazioni (Scopus)

Abstract

Patients with inherited factor VII (FVII) deficiency display different clinical phenotypes requiring ad hoc management. This study evaluated treatments for spontaneous and traumatic bleeding using data from the Seven Treatment Evaluation Registry (STER). One-hundred one bleeds were analysed in 75 patients (41 females; FVII coagulant activity <1-20%). Bleeds were grouped as haemarthroses (n=30), muscle/subcutaneous haematomas (n=16), epistaxis (n=12), gum bleeding (n=13), menorrhagia (n=16), central nervous system (CNS; n=9), gastrointestinal (GI; n=2) and other (n=3). Of 93 evaluable episodes, 76 were treated with recombinant, activated FVII (rFVIIa), eight with fresh frozen plasma (FFP), seven with plasma-derived FVII (pdFVII) and two with prothrombin-complex concentrates. One-day replacement therapy resulted in very favourable outcomes in haemarthroses, and was successful in muscle/subcutaneous haematomas, epistaxis and gum bleeding. For menorrhagia, single- or multiple-dose schedules led to favourable outcomes. No thrombosis occurred; two inhibitors were detected in two repeatedly treated patients (one postrFVIIa, one post-pdFVII). In FVII deficiency, most bleeds were successfully treated with single 'intermediate' doses (median 60 μg/kg) of rFVIIa. For the most severe bleeds (CNS, GI) short- or long-term prophylaxis may be optimal. © Schattauer 2013.
Lingua originaleEnglish
pagine (da-a)238-247
Numero di pagine9
RivistaThrombosis and Haemostasis
Volume109
Stato di pubblicazionePublished - 2013

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Factor VII Deficiency
Hemarthrosis
Menorrhagia
Epistaxis
Gingiva
Hemorrhage
Hematoma
Factor VIIa
Muscles
Factor VII
Registries
Appointments and Schedules
Thrombosis
Therapeutics
Central Nervous System
Phenotype
recombinant FVIIa

All Science Journal Classification (ASJC) codes

  • Hematology

Cita questo

Napolitano, M., Ingerslev, J., Sørensen, B., Karimi, M., Bertrand, A-M., Minno, G. D., ... Mariani, G. (2013). Replacement therapy for bleeding episodes in factor VII deficiency: A prospective evaluation. Thrombosis and Haemostasis, 109, 238-247.

Replacement therapy for bleeding episodes in factor VII deficiency: A prospective evaluation. / Napolitano, Mariasanta; Ingerslev, Jorgen; Sørensen, Benny; Karimi, Mehran; Bertrand, Anne-Marie; Minno, Giovanni Di; Bjerre, Jens; Ruiz-Saez, Arlette; Dolce, Alberto; Batorova, Angelika; Auerswald, Günter; Garrido, Rosario Perez; Schved, Jean F.; Mariani, Guglielmo; Platokouki, Helen; Dolce, Alberto; Mariani, Guglielmo.

In: Thrombosis and Haemostasis, Vol. 109, 2013, pag. 238-247.

Risultato della ricerca: Article

Napolitano, M, Ingerslev, J, Sørensen, B, Karimi, M, Bertrand, A-M, Minno, GD, Bjerre, J, Ruiz-Saez, A, Dolce, A, Batorova, A, Auerswald, G, Garrido, RP, Schved, JF, Mariani, G, Platokouki, H, Dolce, A & Mariani, G 2013, 'Replacement therapy for bleeding episodes in factor VII deficiency: A prospective evaluation', Thrombosis and Haemostasis, vol. 109, pagg. 238-247.
Napolitano M, Ingerslev J, Sørensen B, Karimi M, Bertrand A-M, Minno GD e altri. Replacement therapy for bleeding episodes in factor VII deficiency: A prospective evaluation. Thrombosis and Haemostasis. 2013;109:238-247.
Napolitano, Mariasanta ; Ingerslev, Jorgen ; Sørensen, Benny ; Karimi, Mehran ; Bertrand, Anne-Marie ; Minno, Giovanni Di ; Bjerre, Jens ; Ruiz-Saez, Arlette ; Dolce, Alberto ; Batorova, Angelika ; Auerswald, Günter ; Garrido, Rosario Perez ; Schved, Jean F. ; Mariani, Guglielmo ; Platokouki, Helen ; Dolce, Alberto ; Mariani, Guglielmo. / Replacement therapy for bleeding episodes in factor VII deficiency: A prospective evaluation. In: Thrombosis and Haemostasis. 2013 ; Vol. 109. pagg. 238-247.
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abstract = "Patients with inherited factor VII (FVII) deficiency display different clinical phenotypes requiring ad hoc management. This study evaluated treatments for spontaneous and traumatic bleeding using data from the Seven Treatment Evaluation Registry (STER). One-hundred one bleeds were analysed in 75 patients (41 females; FVII coagulant activity <1-20{\%}). Bleeds were grouped as haemarthroses (n=30), muscle/subcutaneous haematomas (n=16), epistaxis (n=12), gum bleeding (n=13), menorrhagia (n=16), central nervous system (CNS; n=9), gastrointestinal (GI; n=2) and other (n=3). Of 93 evaluable episodes, 76 were treated with recombinant, activated FVII (rFVIIa), eight with fresh frozen plasma (FFP), seven with plasma-derived FVII (pdFVII) and two with prothrombin-complex concentrates. One-day replacement therapy resulted in very favourable outcomes in haemarthroses, and was successful in muscle/subcutaneous haematomas, epistaxis and gum bleeding. For menorrhagia, single- or multiple-dose schedules led to favourable outcomes. No thrombosis occurred; two inhibitors were detected in two repeatedly treated patients (one postrFVIIa, one post-pdFVII). In FVII deficiency, most bleeds were successfully treated with single 'intermediate' doses (median 60 μg/kg) of rFVIIa. For the most severe bleeds (CNS, GI) short- or long-term prophylaxis may be optimal. {\circledC} Schattauer 2013.",
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AU - Napolitano, Mariasanta

AU - Ingerslev, Jorgen

AU - Sørensen, Benny

AU - Karimi, Mehran

AU - Bertrand, Anne-Marie

AU - Minno, Giovanni Di

AU - Bjerre, Jens

AU - Ruiz-Saez, Arlette

AU - Dolce, Alberto

AU - Batorova, Angelika

AU - Auerswald, Günter

AU - Garrido, Rosario Perez

AU - Schved, Jean F.

AU - Mariani, Guglielmo

AU - Platokouki, Helen

AU - Dolce, Alberto

AU - Mariani, Guglielmo

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KW - Preschool; Coagulants; Drug Administration Schedule; Factor VII Deficiency; Factor VIIa; Female; Hemorrhage; Humans; Infant; Infant

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UR - http://www.schattauer.de/en/magazine/subject-areas/journals-a-z/thrombosis-and-haemostasis/issue/special/manuscript/19113/download.html

M3 - Article

VL - 109

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EP - 247

JO - Thrombosis and Haemostasis

JF - Thrombosis and Haemostasis

SN - 0340-6245

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