To date, however, no randomized comparator studies for prophylaxis with bypassing agents have been carried out and, therefore, it is difficult to substantiate these hypothesized points. To date, effectiveness and safety of prophylaxis with rFVIIa has been described in only a small number of case reports, retrospective studies and a small randomized clinical trial (as discussed). Systematically, obtaining more extensive data on the past
and current use of rFVIIa for prophylaxis in patients with haemophilia and inhibitors would therefore prove useful to clinicians. PROPACT, the Retrospective Prophylaxis Patient Case CollecTion, aims to evaluate the frequency and pattern of bleeding episodes in patients receiving prophylactic treatment with rFVIIa. In addition, the study will assess the selection of patients for prophylaxis, the dose and dosing intervals used, and the safety of rFVIIa in prophylaxis. This international study selleck compound library includes approximately 40 centres in 13 countries and aims to include 50–100 patients. Data are expected to be available in early 2010 (Data on file, Novo Nordisk, 2009). A further retrospective study of the use of rFVIIa as secondary prophylaxis in haemophilic patients
with inhibitors is currently underway in France. This analysis aims to evaluate the various rFVIIa secondary prophylaxis dosing regimens used in 14 French treatment centres and will include 15 patients. The study will also determine the profile of patients selected for prophylaxis and compare the effects of prophylaxis check details with on-demand therapy during the 6 months pre- and post-prophylaxis. Results from this analysis will be incorporated into the PROPACT database. An additional prospective study learn more addressing the efficacy and safety of rFVIIa has been proposed. The EuropeaN initiative to prevent JOInt damage in Haemophilia A children with inhibitors who are candidates for ITI (ENJOIH®) study will investigate prophylactic rFVIIa treatment
compared with on-demand therapy. Analyses include reducing the frequency of joint bleeds and the development of joint damage (including synovitis), as measured by the Haemophilia Joint Health Score (HJHS), in children with haemophilia A and high-responding inhibitors. This study is ready to start in November 2009. This is a crossover study in which patients will be treated with 6 months of on-demand FEIBA® and 6 months of FEIBA® prophylaxis (85 U kg−1± 15% on 3 non-consecutive days weekly) with a 3-month wash-out period between arms. Subjects will be randomly assigned as to sequence of treatment with half receiving the on-demand treatment first, followed by prophylaxis and the other half receiving prophylaxis treatment first, followed by the on-demand treatment period. The primary endpoint is to compare the number of bleeding events in the on-demand and prophylaxis arms. This study is closed and data currently is in examination.