TY - JOUR
T1 - Replacement therapy for bleeding episodes in factor VII deficiency: A prospective evaluation
AU - Napolitano, Mariasanta
AU - Ingerslev, Jorgen
AU - Sørensen, Benny
AU - Karimi, Mehran
AU - Bertrand, Anne-Marie
AU - Bertrand, Anne-Marie
AU - Mariani, Guglielmo
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 - Minno, Giovanni Di
AU - Schved, Jean F.
AU - Mariani, Guglielmo
AU - Platokouki, Helen
AU - Dolce, Alberto
AU - Mariani, Guglielmo
PY - 2013
Y1 - 2013
N2 - 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.
AB - 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.
KW - Bleeds; Factor VII deficiency; Replacement therapy; Adolescent; Adult; Aged; Blood Coagulation Factors; Child; Child
KW - Newborn; Male; Middle Aged; Prospective Studies; Recombinant Proteins; Registries; Time Factors; Treatment Outcome; Young Adult; Blood Component Transfusion; Hematology
KW - Preschool; Coagulants; Drug Administration Schedule; Factor VII Deficiency; Factor VIIa; Female; Hemorrhage; Humans; Infant; Infant
KW - Bleeds; Factor VII deficiency; Replacement therapy; Adolescent; Adult; Aged; Blood Coagulation Factors; Child; Child
KW - Newborn; Male; Middle Aged; Prospective Studies; Recombinant Proteins; Registries; Time Factors; Treatment Outcome; Young Adult; Blood Component Transfusion; Hematology
KW - Preschool; Coagulants; Drug Administration Schedule; Factor VII Deficiency; Factor VIIa; Female; Hemorrhage; Humans; Infant; Infant
UR - http://hdl.handle.net/10447/75400
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
SP - 238
EP - 247
JO - Thrombosis and Haemostasis
JF - Thrombosis and Haemostasis
SN - 0340-6245
ER -