title: Replacement therapy for bleeding episodes in factor VII deficiency. A prospective evaluation.
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 post-rFVIIa, 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.
authors.affiliation: University of Ferrara, Medical School, Via Fossato di Mortara 66, 44121 Ferrara, Italy. gmprivate39@gmail.com
authors.forename: Guglielmo
authors.initials: G
authors.lastname: Mariani
authors.forename: Mariasanta
authors.initials: M
authors.lastname: Napolitano
authors.forename: Alberto
authors.initials: A
authors.lastname: Dolce
authors.forename: Rosario
authors.initials: R
authors.lastname: Pérez Garrido
authors.forename: Angelika
authors.initials: A
authors.lastname: Batorova
authors.forename: Mehran
authors.initials: M
authors.lastname: Karimi
authors.forename: Helen
authors.initials: H
authors.lastname: Platokouki
authors.forename: Günter
authors.initials: G
authors.lastname: Auerswald
authors.forename: Anne-Marie
authors.initials: AM
authors.lastname: Bertrand
authors.forename: Giovanni
authors.initials: G
authors.lastname: Di Minno
authors.forename: Jean F
authors.initials: JF
authors.lastname: Schved
authors.forename: Jens
authors.initials: J
authors.lastname: Bjerre
authors.forename: Jorgen
authors.initials: J
authors.lastname: Ingerslev
authors.forename: Benny
authors.initials: B
authors.lastname: Sørensen
authors.forename: Arlette
authors.initials: A
authors.lastname: Ruiz-Saez
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D001779:Blood Coagulation Factors / Q000008:administration & dosage* / Q000009:adverse effects; D016913:Blood Component Transfusion / Q000009:adverse effects; D002648:Child; D002675:Child, Preschool; D003029:Coagulants / Q000008:administration & dosage* / Q000009:adverse effects; D004334:Drug Administration Schedule; D005168:Factor VII Deficiency / Q000150:complications / Q000175:diagnosis / Q000235:genetics / Q000628:therapy*; D015942:Factor VIIa / Q000008:administration & dosage* / Q000009:adverse effects; D005260:Female; D006470:Hemorrhage / Q000188:drug therapy* / Q000235:genetics; D006801:Humans; D007223:Infant; D007231:Infant, Newborn; D008297:Male; D008875:Middle Aged; D011446:Prospective Studies; D011994:Recombinant Proteins / Q000008:administration & dosage; D012042:Registries; D013997:Time Factors; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: D001779:Blood Coagulation Factors; D003029:Coagulants; D011994:Recombinant Proteins; C025667:prothrombin complex concentrates; D015942:Factor VIIa