New era of management of patients with haemophilia - replacement therapy with coagulation factors VIII and IX with an extended half-life (EHL)
Abstract
Replacement therapy with a missing coagulation factor is still the basis of therapy for patients with haemophilia. However, regular prophylactic substitution by standard factors is often burdensome for the patient and his family, especially due to frequent intravenous applications.
This load can result in poor adherence to treatment, which can lead to failure and increased musculoskeletal damage. Among the newest drugs for the treatment of hemophilia A and B are coagulation factors VIII and IX with a prolonged half-life.
Prolongation of the elimination half-life is achieved through various structural modifications, such as pegylation, Fc fusion or single-stranded technology. The prolonged half-life is expected to lead to increased compliance (adherence to treatment) and better prophylactic outcomes of haemophilia treatment, mainly due to prolonged dosing intervals.
Recombinant factors with a prolonged half-life have been evaluated in numerous clinical studies to be well tolerated and effective in the treatment and prevention of bleeding episodes in haemophiliac patients.