title: Long-term prophylaxis in severe factor VII deficiency.
abstract: BACKGROUND
  The spectrum of bleeding problems in FVII deficiency is highly variable and FVII levels and causative genetic mutations correlate poorly with the bleeding risk. Long-term prophylaxis is generally initiated in order to prevent subsequent CNS bleeding after a first event or in patients with other major/ life threatening/ frequent bleeding symptoms as gastrointestinal bleeding or hemarthrosis. However few data are available in the literature regarding FVII prophylaxis and clinical decisions cannot be based on evidence.
  
  
  OBJECTIVE
  We report the data available in the literature on FVII prophylaxis and our personal experience regarding three patients affected by severe FVII deficiency.
  
  
  METHODS
  Specific papers on long-term prophylaxis in severe FVII deficiency were identified using the database, PUBMED.
  
  
  RESULTS
  The most frequent indications for long-term prophylaxis were CNS bleeding (58%), hemartrosis (15%) and GI bleeding (9%). Patients were treated with various dosages and frequency. Prophylactic treatment with 10-30U/kg (pdFVII) or 20-30mcg/kg (rFVIIa) twice or three times/weeks was described to be effective.
  
  
  CONCLUSIONS
  In the literature and in our experience, prophylaxis can be considered in patients with severe FVII deficiency and severe bleeding phenotype. A dose of 10-30U/kg (pdFVII) or 20-30 microg/kg (rFVIIa) twice or three times/week is usually administrated, but dose and frequency can be tailored based on the clinical follow-up of the patients. Since hemarthrosis is a frequent manifestation, a suggestion to improve the outcomes of patients with severe FVII deficiency is to monitor joint condition in order to identify early arthropathy that could be another indication to start secondary prophylaxis.
authors.affiliation: Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Dipartimento delle Units Multispecialistiche e dei Trapianti, Unità Operativa Complessa di Ematologia non Tumorale e Coagulopatie, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico and University of Milan, Milan, Italy.
authors.forename: S M
authors.initials: SM
authors.lastname: Siboni
authors.affiliation: Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Dipartimento delle Units Multispecialistiche e dei Trapianti, Unità Operativa Complessa di Ematologia non Tumorale e Coagulopatie, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico and University of Milan, Milan, Italy.
authors.forename: E
authors.initials: E
authors.lastname: Biguzzi
authors.affiliation: Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Dipartimento delle Units Multispecialistiche e dei Trapianti, Unità Operativa Complessa di Ematologia non Tumorale e Coagulopatie, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico and University of Milan, Milan, Italy.
authors.forename: C
authors.initials: C
authors.lastname: Mistretta
authors.affiliation: Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Dipartimento delle Units Multispecialistiche e dei Trapianti, Unità Operativa Complessa di Ematologia non Tumorale e Coagulopatie, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico and University of Milan, Milan, Italy.
authors.forename: I
authors.initials: I
authors.lastname: Garagiola
authors.affiliation: Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Dipartimento delle Units Multispecialistiche e dei Trapianti, Unità Operativa Complessa di Ematologia non Tumorale e Coagulopatie, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico and University of Milan, Milan, Italy.
authors.forename: F
authors.initials: F
authors.lastname: Peyvandi
mesh_terms: D000293:Adolescent; D000328:Adult; D002648:Child; D002675:Child, Preschool; D005168:Factor VII Deficiency / Q000150:complications / Q000235:genetics / Q000517:prevention & control*; D015942:Factor VIIa / Q000494:pharmacology*; D005260:Female; D006801:Humans; D007223:Infant; D007231:Infant, Newborn; D008297:Male; D008875:Middle Aged; D009154:Mutation; D011994:Recombinant Proteins / Q000494:pharmacology; D013997:Time Factors; D055815:Young Adult
chemical_list: D011994:Recombinant Proteins; C103587:recombinant FVIIa; D015942:Factor VIIa
keywords: factor VII deficiency; long-term prophylaxis; rare coagulation disorders