Treatment of Inherited Factor VII Deficiency
Condition: Factor VII Deficiency
MeSH terms: Factor VII Deficiency
Study type: Observational

Summary: 
      FVII deficiency is a rare coagulation disorder. A limited number of patients are found in
      most treatment centres and countries. Treatment demands vary considerably amongst FVII
      deficient patients. Therefore, regular clinical studies will meet with recruitment problems
      in this particular patient population. The present study intends to elucidate the bleeding
      patterns in a well-defined collective of FVII deficiency patients who are carefully
      characterised, to document the actual use of different treatment modalities in different
      subgroups and to evaluate the efficacy and safety of current available treatment modalities
      in bleedings, surgery and prophylaxis. The purpose is to gain some evidence based knowledge
      of treatment of patients with FVII deficiency - an area where treatment decisions are made
      more on personal clinical experience than on consolidated clinical evidence.

      This study intends to register treatment practices as they are actually performed - in a
      structured and documented way.
    

Detailed description: 
      To describe the treatment modalities and outcomes of: - bleeding episodes - surgery -
      prophylaxis in a well defined, international cohort of FVII deficient patients characterised
      following the methodology used by the International Factor VII deficiency Study Group
      (IF7SG).

      To evaluate the presence (in already treated patients) and/or the appearance of inhibiting
      antibodies to FVII and/or therapy-related thrombosis.

      Study Design:

      Prospective observational study on treatment of FVII deficiency patients. This is an outcome
      study conducted through the procedures set up by the IF7SG, in patients already enrolled or
      newly enrolled in the database.

      Study population and products:

      Patients with FVII deficiency (levels of FVII less than 50% of normal or a mutation known to
      be associated to a FVII deficiency) can be enrolled. All pharmaceutical products considered
      useful for treatment of FVII deficiency by the centres can be included in the study.

      Key assessments:

      The database is set up to capture the following assessments, if available:

      Bleeding episodes:

      • Recording of bleeding location, symptom onset and treatment onset & location • Recording of
      substitution therapy, concomitant medications and concomitant illness • Recording of 6 hour
      treatment evaluation for bleeding episodes • Recording of re-bleeding episodes

      Surgery/delivery:

      • Recording of surgery description , date of surgery and indication • Recording of
      substitution therapy, concomitant medications and concomitant illness during surgery •
      Recording of antifibrinolytic drugs used prior, during and/or after surgery •Recording of •
      Recording of overall treatment evaluation

      Prophylaxis:

      • Recording of prophylaxis type (primary/secondary), indication for prophylaxis and
      start/stop date •Recording of prophylaxis therapy, concomitant medications and concomitant
      illness • Recording of bleeding episodes during prophylaxis •Recording of clinical
      prophylaxis evaluation

      For all:

        -  Laboratory values (PT/INR, APTT, FVII:C, platelet count, fibrinogen, inhibitor samples)
           at time points specified in section 7.3.

        -  Adverse events (including thrombotic events, anaphylactic reactions, DIC reactions, Re-
           bleedings and mortality)

        -  Recording of 30 day treatment evaluation (excellent, effective, partly effective,
           ineffective or not evaluable)

        -  Duration of hospital stay (ward, ICU) within 30 days after first product treatment •
           Mortality within 30days after first product administration
    

Intervention name(s): plasma derived Factor VII; recombinant FVIIa; Fresh Frozen Plasma; Activated Prothrombin Complex Concentrates; Virus Inactivated plasma
Intervention type(s): Drug; Drug; Drug; Drug; Drug
Intervention description(s): Treatment of bleeding episodes,treatment during surgery and prophylaxis; Treatment of bleeding episodes,treatment during surgery and prophylaxis; Treatment of bleeding episodes,treatment during surgery and prophylaxis; Treatment of bleeding episodes,treatment during surgery and prophylaxis; Treatment of bleeding episodes,treatment during surgery and prophylaxis
Other intervention name(s): Facteur VII, LFB (plasma-derived);; Factor VII, PFL (plasma-derived); Factor VII, Baxter Immuno (plasma-derived); Novoseven; Blood Transfusion Centre Fresh Frozen Plasma; Octaplas
Intervention MeSH terms: Thrombin
Observational model: Cohort
Time perspective: Prospective
Sampling method: Non-Probability Sample
Study population: 
        Number of patients affected by inherited Factor VII deficiency to be studied Planned number
        of patients to be recruited: >200 Planned number of evaluable bleeding episodes >100
        Planned number of surgical procedures >50 It is planned to include patients to the registry
        from all over the world
      
Biospecimen description: 
      Plasma for Factor VII inhibitor assay is stored and sent to a Central Laboratory for the
      inhibitor determination. Samples are discharged after the inhibitor determination
    
Keywords: Inherited Factor VII deficiency; Inhibitor development in Factor VII deficiency; Bleeding in Factor VII Deficiency; Surgery in Factor VII deficiency; Prophylaxis in Factor VII Deficiency