A pooled analysis of studies revealed that preventative treatment with Hemlibra (emicizumab) could be superior to replacement therapy with factor VIII infusions for reducing bleed rates in hemophilia A patients without inhibitors.
Titled “Efficacy of emicizumab prophylaxis versus factor VIII prophylaxis for treatment of hemophilia A without inhibitors: network meta-analysis and sub-group analyses of the intra-patient comparison of the HAVEN 3 trial,” this pool analysis of studies was recently published in the journal Current Medical Research and Opinion and reported on in Hemophilia Today by Ana Pena, PhD.
What is Hemlibra?
Produced by Roche, it is a humanized monoclonal antibody approved in the U.S. as routine preventive treatment for adults and children with hemophilia A, with or without inhibitors. It is approved in Europe as a preventative treatment for all hemophilia A patients with inhibitors and for severe hemophilia A patients with or without inhibitors.
How does it Work?
It acts as a bypassing agent to mimic the activity of factor VIII (FVIII)—the missing clotting factor in hemophilia A patients—that helps blood to clot normally. It also reduced blood loss and prevents damage to joints and tissues from internal bleeding.
With a half-life of almost 30 days, it is administered by subcutaneous injections carrying a lower burden treatment. Hemophilia Today reports that Hemlibra offers potential advantages to conventional replacement therapy (intravenous infusions of the missing clotting factor)—linked to a residual bleed rate and long-term joint problems.
The research team did a pooled analysis (network meta-analyses) of three clinical trials including 1) The A-LONG 2)The LEOPOLD II and 3) Spinart—assessing the efficacy of FVIII therapy given preventatively and on-demand, and of the HAVEN 3 trial, which evaluated Hemlibra prophylaxis and FVIII on demand (called “no prophylaxis group”).
The pooled analysis of the four clinical trials revealed Hemlibra evidenced prophylaxis a superior treatment to FVIII either on demand or as prophylaxis. When comparing total bleed rates Hemlibra prophylaxis was 64% lower as compared to FVIII prophylaxis.
Moreover, the same tendency occurred with HAVEN 3 data as those patients receiving Hemlibra prophylaxis experienced lower rate of targeted bleeds (reduction of 52.8% to 62%) as compared to those patients getting treated with FVIII prophylaxis.
The research team could argue that the results demonstrate superiority of Hemlibra prophylaxis over FVIII prophylaxis in patients with hemophilia A without inhibitors.
This study was funded by Roche—hence an industry-sponsored study.