Hemocentro Unicamp

1 Introduction The World Federation of Hemophilia (WFH) has been supporting individuals with inherited bleeding disorders for more than 60 years worldwide [1]. Accurate diagnosis is essential for the effective management of these conditions. However, the diagnosis of hereditary bleeding disorders remains challenging, particularly in low-resource settings. Globally, only 31% of patients with hemophilia are currently identified, with the lowest detection rates reported in Africa (8%) and Asia (18%).

Damoctocog alfa pegol (Jivi, Bayer) is an extended half-life recombinant factor VIII product that was approved in 2018 for the treatment and prophylaxis of bleeds in previously treated patients with haemophilia A aged ≥12 years [1]. The efficacy of damoctocog alfa pegol for preventing and treating bleeding in previously treated children aged <12 years with severe haemophilia A was demonstrated in the phase 3, multicentre, open-label, single-arm PROTECT VIII Kids study (NCT01775618) [2].

Abstract A prospective, non-interventional study (270-902) followed 294 adults with severe hemophilia A (SHA) receiving prophylactic factor VIII (FVIII). From these participants, 112 rolled over into a single-arm, multicenter, phase 3 trial (GENEr8-1; NCT03370913) that evaluated efficacy and safety of valoctocogene roxaparvovec, a gene therapy that provides endogenous FVIII in individuals with SHA.