Real-life evidence in evaluating effectiveness of treatment in XXXX with a recombinant FVIII concentrate: A non-interventional study in emerging countries.

@entity1 @entity797 @entity548 @entity447
UNASSIGNED: Some progress has been made regarding availability of recombinant factor @entity797 concentrates and prophylaxis for @entity447 in emerging countries, where plasma-derived concentrates were used in the vast majority. Clinical studies to document their introduction and effectiveness are so far not widely available in literature. This non-interventional study evaluates the real-life effectiveness and safety of prophylactic and on-demand treatment with recombinant factor @entity797 formulated with sucrose (rFVIII-FS) for bleed control and preservation of joints in emerging countries from Eastern Europe, North Africa and Middle East area. One hundred and eighty-six @entity1 from 11 countries were enrolled, mean      SD age 12.8      12.7  years. At enrolment, majority (79.6%) had severe @entity447 (<2%  IU  mL(-1) ), 47.8% had a target joint, 15% had an inhibitor history and one @entity1 was on immune tolerance induction. During the 24-month observation period, 58.1% of the @entity1 were prescribed prophylaxis at every visit, 31.7% were on an on-demand regimen. @entity1 with severe @entity447 on prophylaxis (n  =  82) had a mean annual rate of treated @entity548 of 2.8      4.4, whereas it was 19.1      32.0 for the on-demand group (n  =  31), and a mean total Gilbert Score of 9.9      10.3 at baseline and 4.1      6.7 at study end; vs. 15.2      17.3 and 13.7      17.1 for on-demand respectively. The majority of the @entity548 (91.1%) were treated with one or two infusions. Four @entity1 without inhibitor history had a first positive inhibitor test during the study. This study demonstrates the effective use of rFVIII-FS in emerging countries and adds to the established safety profile of rFVIII-FS.
