Turoctocog alfa new treatment option for hemophilia A patients: Study
Researchers from the Department of Clinical Haematology, Haemato‐Oncology, & Bone Marrow (Stem Cell) Transplant Unit, Christian Medical College and Hospital, Ludhiana, India have reported that turoctocog alfa is a safe treatment option for the prophylaxis and treatment of bleeding episodes in previously treated adolescent and adult patients with hemophilia A in the Indian population.
The study is published in the Research and Practice in Thrombosis and Haemostasis.
Turoctocog alfa is a third‐generation FVIII molecule with a truncated B‐domain. Preclinical studies have documented that turoctocog alfa retains full procoagulant activity. Turoctocog alfa is approved for the treatment of hemophilia A and has been demonstrated to have favorable safety and efficacy in previously treated children and adults with severe hemophilia A in two phase III trials—guardian 1 and guardian 3. However, in a recent phase IIIb trial, the guardian 2 extension trial, it was demonstrated that the extended use of turoctocog alfa was safe and effective for the prevention and treatment of bleeding episodes in patients of all age groups.
Also, Hemophilia A is an X chromosome–linked bleeding disorder caused by the deficiency of coagulation factor VIII (FVIII). The majority of the Indian population with hemophilia A use plasma‐derived clotting factors and, in some instances, fresh frozen plasma and cryoprecipitate. Safer and more efficient treatment options are needed for this group of patients.
Keeping this in mind, M. Joseph John DM and his colleagues assessed the safety of turoctocog alfa for the treatment and prophylaxis of bleeding episodes in previously treated Indian patients with moderate or severe hemophilia A. . The secondary objective of this trial was to assess the hemostatic efficacy of turoctocog alfa for prophylaxis and treatment of bleeding episodes in previously treated patients with moderate or severe hemophilia A, they further added.
This multicenter, open‐label, nonrandomized trial included 60 patients who received prophylactic treatment with turoctocog alfa for 8 weeks, which corresponded to a minimum of 20 exposure days. Confirmed development of FVIII inhibitors during the 8‐week treatment period was evaluated.
Other assessments included frequencies of adverse drug reactions (ARs), serious adverse reactions, drug‐related allergic reactions, and infusion reactions during the 12‐week period after the first treatment; hemostatic effect of turoctocog alfa for the treatment of bleeding episodes; and total annualized dose of turoctocog alfa administered during the 8‐week treatment period.
As a result, the authors observed that no incidence of FVIII inhibitors was detected. No safety concerns such as ARs, serious ARs, or drug‐related allergic reactions were noted. The hemostatic success rate for the treatment of bleeding episodes with turoctocog alfa was 81.6%, they noted.
The researchers then finally concluded that "turoctocog alfa is a safe treatment option for the prophylaxis and treatment of bleeding episodes in previously treated adolescent and adult patients with hemophilia A in the Indian population."