Every year, the American Society of Hematology (ASH) holds a conference — a medical congress — to go over the major advancements made in the field.
This year, of course, the conference — the 62nd — was held virtually. And a major highlight was Octapharma USA's three vital studies concerning novel treatments of Hemophilia A and Von Willebrand Disease.
Octapharma is one of the world's largest manufacturers of therapeutic products based on proteins isolated from plasma collected from healthy, voluntary donors. Its therapies are available in more than 100 countries, a sizable share of which are therapies that treat rare bleeding disorders such as Hemophilia A and Von Willebrand Disease.
Both disorders are inherited, which means that they are caused by a genetic mutation that is passed on to the next generation.
Hemophilia A is caused by a genetic mutation that leads to a deficiency of the protein Factor VIII, one of the thirteen blood-clotting proteins called Coagulation Factors. Whereas, Von Willebrand Disease is caused by a genetic mutation that leads to a deficiency of the protein Von Willebrand Factor, another protein critical to the blood-clotting mechanism.
The two are almost identical in that they make a person's immune system unable to clot or plug the blood surrounding a wound, causing uncontrollable bleeding. But the major difference between them is that Hemophilia A, unlike Von Willebrand Disease, also causes bleeding in the joints.
Octapharma's studies focused on the efficacy of NUWIQ and WILATE, FDA-approved protein therapies that treat Hemophilia A and Von Willebrand Disease in children and adults, respectively.
The first study evaluated the long-term efficacy and tolerability of NUWIQ administered to a small set of patients with Hemophilia A. The second and third studies evaluated the efficacy of WILATE: First, in treating patients suffering mild to severe bleeding caused by inherited platelet disorders. Second, in treating bleeding in women suffering from Von Willebrand Disease, during and post-pregnancy.
Both therapies — NUWIQ and WILATE — provide patients with an external supply of Factor VIII and Von Willebrand Factor, respectively, that makes up for their deficiency. The three studies could prove highly instrumental in providing us with a better understanding of rare bleeding disorders and the role of Factor VIII and Von Willebrand Factor in controlling them.
The goal is to create a solution that makes for long-term prevention of bleeding and a significant decrease in the frequency of bleeding episodes. In other words, it is to enhance the lives of people affected by these diseases.
Learn more about plasma at www.iplasma.life